-----BEGIN PRIVACY-ENHANCED MESSAGE----- Proc-Type: 2001,MIC-CLEAR Originator-Name: webmaster@www.sec.gov Originator-Key-Asymmetric: MFgwCgYEVQgBAQICAf8DSgAwRwJAW2sNKK9AVtBzYZmr6aGjlWyK3XmZv3dTINen TWSM7vrzLADbmYQaionwg5sDW3P6oaM5D3tdezXMm7z1T+B+twIDAQAB MIC-Info: RSA-MD5,RSA, H02wHKIXEQEsSMruJr2s+WkXNM/gTSbHhDCOr2KszcdqJbKKBRxbpMNP+2RhVEzf DWUVgiZNKheaFJELflorWA== 0000950123-09-054268.txt : 20091029 0000950123-09-054268.hdr.sgml : 20091029 20091029080602 ACCESSION NUMBER: 0000950123-09-054268 CONFORMED SUBMISSION TYPE: 10-K PUBLIC DOCUMENT COUNT: 11 CONFORMED PERIOD OF REPORT: 20090831 FILED AS OF DATE: 20091028 DATE AS OF CHANGE: 20091029 FILER: COMPANY DATA: COMPANY CONFORMED NAME: RAPTOR PHARMACEUTICALS CORP. CENTRAL INDEX KEY: 0001203944 STANDARD INDUSTRIAL CLASSIFICATION: PHARMACEUTICAL PREPARATIONS [2834] IRS NUMBER: 980379351 STATE OF INCORPORATION: DE FISCAL YEAR END: 0831 FILING VALUES: FORM TYPE: 10-K SEC ACT: 1934 Act SEC FILE NUMBER: 000-50720 FILM NUMBER: 091143208 BUSINESS ADDRESS: STREET 1: 9 COMMERCIAL BLVD, SUITE 200 CITY: NOVATO STATE: CA ZIP: 94949 BUSINESS PHONE: 415-382-1390 MAIL ADDRESS: STREET 1: 9 COMMERCIAL BLVD, SUITE 200 CITY: NOVATO STATE: CA ZIP: 94949 FORMER COMPANY: FORMER CONFORMED NAME: HIGHLAND CLAN CREATIONS CORP. DATE OF NAME CHANGE: 20060531 FORMER COMPANY: FORMER CONFORMED NAME: HIGHLAND CLAN CREATIONS CORP DATE OF NAME CHANGE: 20021106 10-K 1 c91590e10vk.htm FORM 10-K Form 10-K
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UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM 10-K
     
þ   ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934
For the fiscal year ended August 31, 2009
     
o   TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934
For the transition period from                      to                     
Commission file number 000-50720
Raptor Pharmaceuticals Corp.
 
(Exact name of registrant issuer as specified in its charter)
     
Delaware   98-0379351
     
(State or other jurisdiction of incorporation or
organization)
  (I.R.S. Employer Identification No.)
9 Commercial Blvd., Suite 200, Novato, CA 94949
 
(Address of principal executive offices)
(415) 382-8111
 
(Registrant’s telephone number, including area code)
N/A
 
(Former name, former address and former fiscal year, if changed since last report)
Securities registered pursuant to Section 12(b) of the Act:
     
Title of Each Class*
Common Stock, $.001 par value
Preferred Share Purchase Rights
  Name of Each Exchange on Which Registered*
The NASDAQ Capital Market

Securities registered under Section 12(g) of the Act:*

None
Indicate by check mark if the registrant is a well-known seasoned issuer, as defined in Rule 405 of the Securities Act.* Yes o No þ
Indicate by check mark if the registrant is not required to file reports pursuant to Section 13 or Section 15(d) of the Act. * Yes o No þ
Indicate by check mark whether the registrant (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days.* Yes þ No o
Indicate by check mark whether the registrant has submitted electronically and posted on its corporate Website, if any, every Interactive Data File required to be submitted and posted pursuant to Rule 405 of Regulation S-T (§ 232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit and post such files).* Yes o No þ
Indicate by check mark if disclosure of delinquent filers in response to Item 405 of Regulation S-K is not contained in this form, and will not be contained, to the best of registrant’s knowledge, in definitive proxy or information statements incorporated by reference in Part III of this Form 10-K or any amendment to this Form 10-K.* o
Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, or a smaller reporting company.* See definition of “large accelerated filer,” “accelerated filer” and “smaller reporting company” in Rule 12b-2 of the Exchange Act:
Large accelerated filer o Accelerated filer o Non-accelerated filer o (Do not check if a smaller reporting company) Smaller reporting company þ
Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act.)* Yes o No þ
Indicate the number of shares outstanding of each of the issuer’s classes of common stock, as of the latest practicable date: 18,829,842 shares common stock, par value $0.001, outstanding as of October 23, 2009.* The aggregate market value of the voting and non-voting stock held by non-affiliates of the registrant as of October 23, 2009 was $66.7 million.*
The documents incorporated by reference are as follows:
None.
 
 

 

 


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* This information relates to the registrant’s sole stockholder, Raptor Pharmaceutical Corp., a Delaware corporation. As of September 28, 2009, the registrant’s common stock, par value $0.001, was registered under Section 12(g) of the Act. On September 29, 2009, the registrant completed a merger whereby it became a wholly-owned subsidiary of Raptor Pharmaceutical Corp. as further described in the immediately subsequent Explanatory Note. As of September 28, 2009, the registrant would have checked, or left blank, as applicable, each check box set forth on this cover page in the same manner as checked, or left blank, as applicable, as set forth on this cover page. As of October 23, 2009, the registrant has one share of common stock outstanding that is held by Raptor Pharmaceutical Corp. As of February 28, 2009, the last business day of the registrant’s most recently completed second fiscal quarter, the aggregate market value of the voting and non-voting stock held by non-affiliates of the registrant was $15.9 million.

 

 


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EXPLANATORY NOTE
On July 27, 2009, we entered into an Agreement and Plan of Merger and Reorganization, herein referred to as the Merger Agreement, with TorreyPines Therapeutics, Inc., a Delaware corporation, herein referred to as TorreyPines, and ECP Acquisition, Inc., a Delaware corporation and wholly-owned subsidiary of TorreyPines, herein referred to as Merger Sub. On September 29, 2009, on the terms and subject to the conditions set forth in the Merger Agreement, pursuant to a stock-for-stock reverse triangular merger, or the Merger, Merger Sub was merged with and into us and we survived such Merger as a wholly-owned subsidiary of TorreyPines. Immediately prior to such Merger and in connection therewith, TorreyPines effected a 1-for-17 reverse stock split of its common stock and changed its corporate name to “Raptor Pharmaceutical Corp.”
The stockholder proposals of TorreyPines regarding the reverse stock split, corporate name change, and issuance of shares of TorreyPines’ common stock in such merger and resulting change of control of TorreyPines, among others, were approved by the stockholders of TorreyPines at the Annual Stockholders Meeting of Torrey Pines held on September 28, 2009. Our stockholder proposal regarding the adoption of the Merger Agreement, among others, was approved by our stockholders at our Annual Stockholders Meeting held on September 28, 2009.
As a result of the Merger and in accordance with the Merger Agreement, each share of our common stock outstanding immediately prior to the effective time of the Merger was converted into the right to receive 0.2331234 shares of Raptor Pharmaceutical Corp.’s common stock, on a post 1-for-17 reverse-split basis. Each option and warrant to purchase our common stock outstanding immediately prior to the effective time of the Merger was assumed by Raptor Pharmaceutical Corp. at the effective time of the Merger, with each share of our common stock underlying such options and warrants being converted into the right to receive 0.2331234 shares of Raptor Pharmaceutical Corp.’s common stock, on a post 1-for-17 reverse split basis, rounded down to the nearest whole share of Raptor Pharmaceutical Corp.’s common stock. Following the Merger, each such option or warrant has an exercise price per share of Raptor Pharmaceutical Corp.’s common stock equal to the quotient obtained by dividing the per share exercise price of our common stock subject to such option or warrant by 0.2331234, rounded up to the nearest whole cent. For example, if we originally issued 1,000,000 shares in a financing at $0.32 per share before the Merger, this Annual Report would reflect this transaction in its post-Merger shares and stock price of 233,123 shares (1,000,000 x .2331234) at $1.373 ($0.32 divided by .2331234) per share; however, each of the pre- and post-Merger proceeds in this example totals approximately $320,000.
Immediately following the effective time of the Merger, our former stockholders owned approximately 95% of the outstanding common stock of Raptor Pharmaceutical Corp. and former TorreyPines stockholders owned approximately 5% of the outstanding common stock of Raptor Pharmaceutical Corp., in each case without taking into account any of the shares of common stock of TorreyPines or ours, respectively, that were issuable pursuant to outstanding options or warrants of TorreyPines or ours, respectively, outstanding as of the effective time of the Merger. Although we became the wholly-owned subsidiary of Raptor Pharmaceutical Corp., we were the “accounting acquirer” in the Merger and our board of directors and officers manage and operate the combined company.
The common stock of Raptor Pharmaceutical Corp. began trading on The NASDAQ Capital Market under the ticker symbol, “RPTPD” on September 30, 2009. Effective October 27, 2009, such ticker symbol changed to “RPTP”.
Following the effective time of the Merger, the business conducted by us immediately prior to the Merger became primarily the business conducted by Raptor Pharmaceutical Corp. Unless otherwise mentioned or unless the context requires otherwise (e.g., our consolidated financial statements as of August 31, 2009, and the notes to such consolidated financial statements included elsewhere in this Annual Report on Form 10-K or a reference to an event or circumstance that occurred prior to the effective time of the Merger on September 29, 2009), all references in this Annual Report on Form 10-K to “we,” “us,” “our,” the “Company,” “Raptor” and similar references refer to the public company formerly known as TorreyPines Therapeutics, Inc. and now known as Raptor Pharmaceutical Corp., including its wholly-owned direct and indirect subsidiaries (which includes Raptor Pharmaceuticals Corp., TPTX, Inc., Raptor Discoveries Inc. (f/k/a Raptor Pharmaceutical Inc.) and Raptor Therapeutics Inc. (f/k/a Bennu Pharmaceutical Inc.)), following the name change and completion of the Merger. All references to “TorreyPines” refer to TorreyPines Therapeutics, Inc., prior to the name change and the completion of the Merger. Unless otherwise mentioned or unless the context requires otherwise, all discussions in this Annual Report on Form 10-K regarding our business includes the programs of the combined business of Raptor Pharmaceutical Corp., including its wholly-owned direct and indirect subsidiaries, and our plan of operations includes costs related to the Merger with TorreyPines. Unless otherwise mentioned or unless the context requires otherwise, all discussions in this Annual Report on Form 10-K regarding our common stock, our stock price, and our stock options and warrants to purchase our common stock have been converted to their equivalent post-Merger number of shares and equivalent post-Merger stock prices and exercises prices.
This Annual Report on Form 10-K reflects our financial statements as of August 31, 2009, which date precedes the Merger, because the Merger was consummated after our fiscal year-ended August 31, 2009.

 

 


 

RAPTOR PHARMACEUTICALS CORP.
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 Exhibit 4.11
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 Exhibit 21.1
 Exhibit 23.1
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 Exhibit 32.1
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PART I
FORWARD-LOOKING STATEMENTS
This Annual Report contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. In some cases, these statements can be identified by the use of terminology such as “believes,” “expects,” “anticipates,” “plans,” “may,” “might,” “will,” “could,” “should,” “would,” “projects,” “anticipates,” “predicts,” “intends,” “continues,” “estimates,” “potential,” “opportunity” or the negative of these terms or other comparable terminology. All statements, other than statements of historical facts, included in this Annual Report, including our financial condition, future results of operation, projected revenues and expenses, business strategies, operating efficiencies or synergies, competitive positions, growth opportunities for existing intellectual properties, technologies, products, plans, and objectives of management, markets for our securities, and other matters, are about us and our industry that involve substantial risks and uncertainties and constitute forward-looking statements for the purpose of the safe harbor provided by Section 27A of the Securities Act of 1933, as amended, or the Securities Act, and Section 21E of the Securities Exchange Act of 1934, as amended, or the Exchange Act. Such forward-looking statements, wherever they occur, are necessarily estimates reflecting the best judgment of our senior management on the date on which they were made, or if no date is stated, as of the date of this Annual Report. You should not place undue reliance on these statements, which only reflect information available as of the date that they were made. Our business’ actual operations, performance, development and results might differ materially from any forward-looking statement due to various known and unknown risks, uncertainties, assumptions and contingencies, including those described in the section titled “Risk Factors,” and including, but not limited to, the following:
   
our need for, and our ability to obtain, additional funds;
 
   
uncertainties relating to clinical trials and regulatory reviews;
 
   
our dependence on a limited number of therapeutic compounds;
 
   
the early stage of the products we are developing;
 
   
the acceptance of any of our future products by physicians and patients;
 
   
competition and dependence on collaborative partners;
 
   
loss of key management or scientific personnel;
 
   
our ability to obtain adequate intellectual property protection and to enforce these rights;
 
   
our ability to avoid infringement of the intellectual property rights of others; and
 
   
the other factors and risks described under the section captioned “Risk Factors” as well as other factors not identified therein.
Although we believe that the expectations reflected in the forward-looking statements are reasonable, the factors discussed in this Annual Report could cause actual results or outcomes to differ materially and/or adversely from those expressed in any forward-looking statements made by us or on our behalf, and therefore we cannot guarantee future results, levels of activity, performance or achievements and you should not place undue reliance on any such forward-looking statements. We cannot give you any assurance that the forward-looking statements included in this Annual Report will prove to be accurate and the forward-looking events discussed in this Annual Report may not occur. In light of the significant uncertainties inherent in the forward-looking statements included in this Annual Report, you should not regard the inclusion of this information as a representation by us or any other person that the results or conditions described in those statements or our objectives and plans will be achieved.

 

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ITEM 1: BUSINESS
Overview
We believe that we are building a balanced pipeline of drug candidates that may expand the reach and benefit of existing therapeutics. Our product portfolio includes both candidates from our proprietary drug targeting platforms and in-licensed and acquired product candidates.
Our current pipeline includes three clinical development programs which we are actively developing. We also have three other clinical-stage product candidates, for which we are seeking business development partners but are not actively developing, and we have four preclinical product candidates we are developing, three of which are based upon our proprietary drug-targeting platforms.
Clinical Development Programs
Our three active clinical development programs are based on an existing therapeutic that we are reformulating for potential improvement in safety and/or efficacy and for application in new disease indications. These clinical development programs include the following:
   
DR Cysteamine for the potential treatment of nephropathic cystinosis, or cystinosis, a rare genetic disorder;
 
   
DR Cysteamine for the potential treatment of non-alcoholic steatohepatitis, or NASH, a metabolic disorder of the liver; and
 
   
DR Cysteamine for the potential treatment of Huntington’s Disease, or HD.
Other Clinical-Stage Product Candidates
We have three clinical-stage product candidates for which we are seeking partners:
   
Convivia™ for the potential management of acetaldehyde toxicity due to alcohol consumption by individuals with aldehyde dehydrogenase, or ALDH2 deficiency, an inherited metabolic disorder; and
 
   
Tezampanel and NGX426, non-opioids for the potential treatment of: migraine, acute pain, and chronic pain.
Preclinical Product Candidates
Our preclinical platforms consist of targeted therapeutics, which we are developing for the potential treatment of multiple indications, including liver diseases, neurodegenerative diseases and breast cancer:
Our receptor-associated protein, or RAP, platform consists of: HepTide™ for the potential treatment of primary liver cancer and hepatitis C; and NeuroTrans™ to potentially deliver therapeutics across the blood-brain barrier for treatment of a variety of neurological diseases.
Our mesoderm development protein, or Mesd, platform consists of WntTide™ for the potential treatment of breast cancer.
We are also examining our glutamate receptor antagonists, tezampanel and NGX426, for the potential treatment of thrombosis and spasticity disorders.

 

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DRUG PRODUCT CANDIDATE   DISEASE INDICATION   STAGE OF DEVELOPMENT
Delayed release,
enterically coated
cysteamine bitartrate,
or DR Cysteamine
  cystinosis   Phase IIb
(ongoing, open IND)
Orphan Product Designation
 
       
DR Cysteamine
  NASH   Phase IIa
(ongoing, open IND)
 
       
DR Cysteamine
  HD   Phase II
(planned for 2010)
Orphan Product Designation
 
       
ConviviaTM
  ALDH2 Deficiency, or Ethanol
Intolerance
  Business Development Opportunity
(Phase IIa completed)
 
       
Tezampanel and NGX 426
  Migraine and Pain   Business Development Opportunity
(Phase I/II completed)
 
       
HepTideTM
  Hepatocellular Carcinoma, or HCC and Hepatitis   Preclinical
(ongoing)
 
       
WntTideTM
  Breast Cancer   Preclinical
(ongoing)
 
       
NeuroTransTM
  Neurodegenerative Diseases   Preclinical
Roche collaboration
(ongoing)
 
       
Tezampanel and NGX 426
  Thrombosis and Spasticity Disorder   Preclinical
Future Activities
Over the next 12 months, we plan to conduct research and development activities based upon our DR Cysteamine clinical programs and continued development of our preclinical product candidates. We also plan to actively seek business development partners for our ConviviaTM product candidate and Tezampanel and NGX426. We may also develop future in-licensed technologies and acquired technologies. A brief summary of our primary objectives in the next 12 months for our research and development activities is provided below. Our plans for research and development activities over the next 12 months can only be implemented if we are successful in raising significant funds during this period. In addition, there can be no assurances that our research and development activities will be successful. If we do not make important progress towards achieving at least one of our major clinical objectives, this could adversely impact our ability to raise significant additional funds, which could adversely impact our ability to continue as a going concern.
Clinical Development Programs
We develop clinical-stage drug product candidates which are: internally discovered therapeutic candidates based on our novel drug delivery platforms and in-licensed or purchased clinical-stage products which may be new chemical entities in mid-to-late stage clinical development, currently approved drugs with potential efficacy in additional indications, and treatments that we could repurpose or reformulate as potentially more effective or convenient treatments for a drug’s currently approved indications.

 

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Development of DR Cysteamine for the Potential Treatment of Nephropathic Cystinosis or Cystinosis
Our DR Cysteamine product candidate is a proprietary delayed-release, enteric-coated microbead formulation of cysteamine bitartrate contained in a gelatin capsule. We are investigating DR Cysteamine for the potential treatment of cystinosis.
We believe that immediate-release cysteamine bitartrate, a cystine-depleting agent, is currently the only U.S. Food and Drug Administration, or FDA, and the European Medicines Agency, or EMEA, approved drug to treat cystinosis, a rare genetic disease. Immediate-release cysteamine is effective at preventing or delaying kidney failure and other serious health problems in cystinosis patients. However, patient compliance is challenging due to the requirement for frequent dosing and gastrointestinal side effects. Our DR Cysteamine for the potential treatment of cystinosis is designed to mitigate some of these difficulties. It is expected to be dosed twice daily, compared to the current every-six-hour dosing schedule. In addition, DR Cysteamine is designed to pass through the stomach and deliver the drug directly to the small intestine, where it is more easily absorbed into the bloodstream and may result in fewer gastrointestinal side effects.
The FDA granted orphan drug designation for DR Cysteamine for the treatment of cystinosis in 2006.
In June 2009, we commenced our Phase IIb clinical trial of DR Cysteamine in cystinosis, in which we have enrolled nine cystinosis patients with histories of compliance using the currently available immediate-release form of cysteamine bitartrate. The clinical trial, which is being conducted at the University of California at San Diego, or UCSD, is evaluating safety, tolerability, pharmacokinetics and pharmacodynamics of a single dose of DR Cysteamine in patients. Release of data from the study is expected in the fourth calendar quarter of 2009. We plan to follow the Phase IIb clinical study with a pivotal, Phase III clinical study in cystinosis patients anticipated to commence in early 2010. While we plan to commercialize DR Cysteamine in the U.S. by ourselves, we are actively reviewing potential development partners for DR Cysteamine for markets outside of the U.S. with companies that have experience in clinical development and commercialization of orphan drugs in various ex-U.S. countries.
Development of DR Cysteamine for the Potential Treatment of Non-Alcoholic Steatohepatitis or NASH
In October 2008, we commenced a clinical trial in collaboration with UCSD to investigate a prototype formulation of DR Cysteamine for the treatment of NASH in juvenile patients. In October 2009, we announced positive findings from the completed treatment phase of this open-label Phase IIa clinical trial. At the completion of the initial six-month treatment phase, the study achieved the primary endpoint: mean blood levels of alanine aminotransferase, or ALT, a common biomarker for NASH, were reduced by over 50%. Additionally, over half of the study participants had achieved normalized ALT levels by the end of the treatment phase.
There are no currently approved drug therapies for NASH, and patients are limited to lifestyle changes such as diet, exercise and weight reduction to manage the disease. DR Cysteamine represents an important potential treatment option for patients with NASH. Although NASH is most common in insulin-resistant obese adults with diabetes and abnormal serum lipid profiles, its prevalence is increasing among juveniles as obesity rates rise within this patient population. Although most patients are asymptomatic and feel healthy, NASH causes decreased liver function and can lead to cirrhosis, liver failure and end-stage liver disease.
The NASH trial entails six months of treatment followed by a six-month post-treatment monitoring period. Eligible patients with baseline ALT and aspartate aminotransferase or AST measurements at least twice that of normal levels were enrolled to receive twice-daily, escalating oral doses of up to 1,000 mg of DR Cysteamine. The trial currently has enrolled eleven NASH patients between 11-18 years old. No major adverse events were reported during the six-month treatment phase. Trial subjects continue to be monitored during the six-month post-treatment period currently underway. Full results are being submitted for peer review by UCSD and us, and are expected to be presented in 2010.

 

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Development of DR Cysteamine for the Potential Treatment of Huntington’s Disease or HD
Huntington’s Disease, or HD, is a fatal, inherited degenerative neurological disease affecting about 30,000 people in the U.S. and a comparable number of people in Europe. We are not aware of any treatment for HD other than therapeutics that minimize symptoms such as the uncontrollable movements and mood swings resulting from HD. We are collaborating with a French institution, CHU d’ Angers, on a Phase II clinical trial investigating DR Cysteamine in HD patients, anticipated to begin in early 2010. We are providing the clinical trial materials for the study, which is sponsored by CHU d’ Angers and funded in part by a grant from the French government. We were granted Orphan Drug Designation in the U.S. by the FDA for cysteamine as a potential treatment for HD in 2008.
Other Clinical-Stage Product Candidates
We have three clinical-stage product candidates for which we are seeking partners.
ConviviaTM for Liver Aldehyde Dehydrogenase Deficiency
Convivia™ is our proprietary oral formulation of 4-methylpyrazole, or 4-MP, intended for the potential treatment of acetaldehyde toxicity resulting from alcohol consumption in individuals with ALDH2 deficiency, which is an inherited disorder of the body’s ability to breakdown ethanol, commonly referred to as alcohol intolerance. 4-MP is presently marketed in the U.S. and E.U. in an intravenous form as an anti-toxin. ConviviaTM is designed to lower systemic levels of acetaldehyde (a carcinogen) and reduce symptoms, such as tachycardia and flushing, associated with alcohol consumption by ALDH2-deficient individuals. ConviviaTM is a capsule designed to be taken approximately 30 minutes prior to consuming an alcoholic beverage.
In 2008, we completed a Phase IIa dose escalation clinical trial of oral 4-MP with ethanol in ALDH2 deficient patients. The study results demonstrated that the active ingredient in ConviviaTM significantly reduced heart palpitations (tachycardia), which are commonly experienced by ALDH2 deficient people who drink, at all dose levels tested. The study also found that the 4-MP significantly reduced peak acetaldehyde levels and total acetaldehyde exposure in a subset of the study participants who possess specific genetic variants of the liver ADH and ALDH2 enzymes. We believe that this subset represents approximately one-third of East Asian populations. We are actively seeking corporate partnerships with pharmaceutical companies in selected Asian countries to continue clinical development of ConviviaTM in those countries.
Tezampanel and NGX426 for the Potential Treatment of Migraine and Pain
Tezampanel and NGX426, the oral prodrug of tezampanel, are what we believe to be first-in-class compounds that may represent novel treatments for both pain and non-pain indications. Tezampanel and NGX426 are receptor antagonists that target and inhibit a specific group of receptors—the AMPA and kainate glutamate receptors—found in the brain and other tissues. While normal glutamate production is essential, excess glutamate production, either through injury or disease, has been implicated in a number of diseases and disorders. Published data show that during a migraine, increased levels of glutamate activate AMPA and kainate receptors, result in the transmission of pain and, in many patients, the development of increased pain sensitivity.
By acting at both the AMPA and kainate receptor sites to competitively block the binding of glutamate, tezampanel and NGX426 have the potential to treat a number of diseases and disorders. These include chronic pain, such as migraine and neuropathic pain, muscle spasticity and a condition known as central sensitization, a persistent and acute sensitivity to pain.
Results of a Phase IIb clinical trial of tezampanel were released in October 2007. In the trial, a single dose of tezampanel given by injection was statistically significant compared to placebo in treating acute migraine headache. This was the sixth Phase II trial in which tezampanel has been shown to have analgesic activity. Based on a review of the Phase II data, the FDA previously agreed that tezampanel may move forward into a Phase III program for acute migraine.
In December 2008, results of NGX426 in a human experimental model of cutaneous pain, hyperalgesia and allodynia demonstrated a statistically significant reduction in spontaneous pain, hyperalgesia and allodynia compared to placebo following injections of capsaicin (i.e., chili oil) under the skin. In February 2009, results from a Phase 1 multiple dose trial of NGX426 showed that the compound is safe and well-tolerated in healthy male and female subjects when dosed once daily for five consecutive days.

 

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We are currently seeking program funding, development collaborations or out-licensing partners for the migraine and pain programs.
Preclinical Product Candidates
We are also developing a drug-targeting platform based on the proprietary use of RAP and Mesd. We believe that these proteins may have therapeutic applications in cancer, infectious diseases and neurodegenerative diseases, among others.
These applications are based on the assumption that our targeting molecules can be engineered to bind to a selective subset of receptors with restricted tissue distribution under particular conditions of administration. We believe these selective tissue distributions can be used to deliver drugs to the liver or to other tissues, such as the brain.
In addition to selectively transporting drugs to specific tissues, selective receptor binding constitutes a means by which receptor function might be specifically controlled, either through modulating its binding capacity or its prevalence on the cell surface. Mesd is being engineered for this latter application.
HepTideTM for Hepatocellular Carcinoma and Hepatitis C
Drugs currently used to treat primary liver cancer are often toxic to other organs and tissues. We believe that the pharmacokinetic behavior of RAP (i.e., the determination of the fate or disposition of RAP once administered to a living organism) may diminish the non-target toxicity and increase the on-target efficacy of attached therapeutics.
In preclinical studies of our radio-labeled HepTideTM (a variant of RAP), HepTideTM , our proprietary drug-targeting peptide was shown to distribute predominately to the liver. Radio-labeled HepTideTM which was tested in a preclinical research model of HCC, at the National Research Council in Winnipeg, Manitoba, Canada, showed 4.5 times more delivery to the liver than the radio-labeled control. Another study of radio-labeled HepTideTM in a non-HCC preclinical model, showed 7 times more delivery to the liver than the radio-labeled control, with significantly smaller amounts of radio-labeled HepTideTM delivery to other tissues and organs
HCC is caused by the malignant transformation of hepatocytes, epithelial cells lining the vascular sinusoids of the liver, or their progenitors. HepTideTM has shown to bind to lipoprotein receptor-related protein, or LRP1, receptors on hepatocytes. We believe that the pharmacokinetics and systemic toxicity of a number of potent anti-tumor agents may be controlled in this way.
There are additional factors that favor the suitability of RAP as an HCC targeting agent:
   
RAP is captured by hepatocytes with efficiency, primarily on first-pass.
 
   
Late-stage HCC is perfused exclusively by the hepatic artery, while the majority of the liver is primarily perfused through the portal vein.
Studies have shown that the RAP receptor, LRP1, is well expressed on human HCC and under-expressed on non-cancerous, but otherwise diseased, hepatocytes. Also, LRP1 expression is maintained on metastasized HCC. These factors will favor delivery of RAP peptide-conjugated anti-tumor agents to tumor cells, whether in the liver or at metastasized sites.
We are evaluating conjugates between HepTideTM and a chemotherapeutic for testing in vitro and in appropriate preclinical models for the potential treatment of HCC.

 

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We are also evaluating conjugates between HepTideTM and an antiviral agent for testing in vitro and in appropriate preclinical models for the potential treatment of hepatitis C.
NeuroTransTM for the Potential Treatment of Diseases Affecting the Brain
Hundreds of known genetic and neurodegenerative diseases affect the brain. Drugs often have difficulty reaching these disease-affected areas because the brain has evolved a protective barrier, commonly referred to as the blood-brain barrier.
Part of the solution to the medical problem of neurodegenerative diseases is the creation of effective brain targeting and delivery technologies. One of the most obvious ways of delivering therapeutics to the brain is via the brain’s extensive vascular network. Treating these diseases by delivering therapeutics into the brain in a minimally invasive way, including through a natural receptor mediated transport mechanism called transcytosis, is a vision shared by many researchers and clinicians in the neuroscience and neuromedical fields.
NeuroTrans™ is our proprietary RAP-based technology program to research the delivery of therapeutics across the blood-brain barrier. We believe our NeuroTrans™ platform may provide therapies that will be safer, less intrusive and more effective than current approaches in treating a wide variety of brain disorders.
In preclinical studies, NeuroTrans™ has been conjugated to a variety of protein drugs, including enzymes and growth factors, without interfering with the function of either fusion partner. Studies indicate that radio-labeled NeuroTrans™ may be transcytosed across the blood-brain barrier and that fusions between NeuroTrans™ and therapeutic proteins may be manufactured economically. Experiments conducted in collaboration with Stanford University in 2008 support the NeuroTrans™ peptide’s ability to enhance the transport of cargo molecules into the cells that line the blood-brain barrier.
In June 2009, we entered into a collaboration and licensing agreement with F. Hoffman — La Roche Ltd. and Hoffman—La Roche Inc., or Roche, to evaluate therapeutic delivery across the blood-brain barrier utilizing NeuroTrans™. Under terms of the agreement, Roche has funded studies of select molecules attached to NeuroTrans™. The agreement provides Roche with an exclusive worldwide license to NeuroTrans™ for use in the delivery of diagnostic and therapeutic molecules across the blood-brain barrier. Roche’s and our scientists will actively collaborate on the project. We have received an initial upfront payment for the collaboration to cover our portion of the initial studies, and may earn development milestone payments and royalties in exchange for the licensing of NeuroTrans™ to Roche.
WntTideTM for the Potential Treatment of Cancer
Human Mesd is a natural inhibitor of the receptor LRP6. LRP6 has recently been shown to play a role in the progression of some breast tumors. Studies in the laboratory of Professor Guojun Bu, one of our scientific advisors, at the Washington University in St. Louis Medical School have demonstrated the potential of Mesd and related peptides to target these tumors. These molecules and applications are licensed to us from Washington University.
WntTide™ is our proprietary, Mesd-based peptide that we are developing as a potential therapeutic to inhibit the growth and metastasis of tumors over-expressing LRP5 or LRP6. We have licensed the use of Mesd from Washington University in St. Louis for the potential treatment of cancer and bone density disorders.
In April 2009, Washington University conducted a preclinical study of WntTide™ in a breast cancer model which showed tumor inhibition. The results of this study were presented at the 2nd Annual Wnt Conference in Washington, D.C., in June 2009 and will be published in the fourth quarter of 2009. We are currently planning another breast tumor preclinical model study with researchers at Washington University in the continued development of WntTide™.

 

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Tezampanel and NGX426 for the Potential Treatment of Thrombotic Disorder
Research conducted at Johns Hopkins University or JHU by Craig Morrell, D.V.M., Ph.D., and Charles Lowenstein, M.D. demonstrated the importance of glutamate release in promoting platelet activation and thrombosis. Research shows that platelets treated with an AMPA/kainate receptor antagonist such as tezampanel or NGX426 are more resistant to glutamate-induced aggregation than untreated platelets. This identifies the AMPA/kainate receptors on platelets targeted by tezampanel or NGX426 as a new antithrombotic target with a different mechanism of action than Plavix®, aspirin or tPA. We have licensed the intellectual property of Tezampanel and NGX 426 for the treatment of thrombotic disorder from JHU and are in discussions with potential collaborators regarding the development of this product candidate. Research conducted in Martin Marsala’s lab at UCSD has demonstrated the utility of tezampanel in reducing spasticity elicited by activation of AMPA receptors on spinal astrocytes following ischemic events. We intend to further assess application of tezampanel in the treatment of spasticity.
Other Development Areas
Securing Additional and Complementary Technology Licenses from Others
We plan to establish additional research collaborations with prominent universities and research labs currently working on the development of potential targeting molecules, and to secure licenses from these universities and labs for technology resulting from the collaboration. No assurances can be made regarding our ability to establish such collaborations over the next 12 months, or at all. We intend to focus our in-licensing and product candidate acquisition activities on identifying complementary therapeutics, therapeutic platforms that offer a number of therapeutic targets, and clinical-stage therapeutics based on existing approved drugs in order to create proprietary reformulations to improve safety and efficacy or to expand such drugs’ clinical indications through additional clinical trials. We may obtain these products through collaborations, joint ventures or through merger and/or acquisitions with other biotechnology companies.
Strategic Acquisitions
Reverse Merger with TorreyPines
In July 2009, we entered into an Agreement and Plan of Merger and Reorganization, or the Merger Agreement, with TorreyPines Therapeutics, Inc., a Delaware corporation, or TorreyPines, and ECP Acquisition, Inc., a Delaware corporation and wholly-owned subsidiary of TorreyPines, or Merger Sub. On September 29, 2009, on the terms and subject to the conditions set forth in the Merger Agreement, Merger Sub was merged with and into us and we survived such Merger as a wholly-owned subsidiary of TorreyPines. Immediately prior to such Merger and in connection therewith, TorreyPines effected a 1-for-17 reverse stock split of its common stock and changed its corporate name to “Raptor Pharmaceutical Corp.”
As of immediately following the effective time of the Merger, our former stockholders owned approximately 95% of the outstanding common stock of Raptor Pharmaceutical Corp. and former TorreyPines stockholders owned approximately 5% of the outstanding common stock of Raptor Pharmaceutical Corp., in each case without taking into account any of the shares of common stock of TorreyPines or ours, respectively, that were issuable pursuant to outstanding options or warrants of TorreyPines or ours, respectively, outstanding as of the effective time of the Merger. Although we became the wholly-owned subsidiary of Raptor Pharmaceutical Corp., we were the “accounting acquirer” in the Merger and our board of directors and officers manage and operate the combined company. The common stock of Raptor Pharmaceutical Corp. began trading on The NASDAQ Capital Market under the ticker symbol, “RPTPD” in September 2009. Effective October 27, 2009, such ticker symbol changed to “RPTP”. Future filings with the Securities and Exchange Commission, or SEC, with respect to our business will be made by Raptor Pharmaceutical Corp.

 

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Purchase of ConviviaTM
In October 2007, we purchased certain assets of Convivia, Inc., or Convivia, including intellectual property, know-how and research reports related to a product candidate targeting liver ALDH2 deficiency, a genetic metabolic disorder. We hired Convivia’s chief executive officer and founder, Thomas E. (Ted) Daley, as President of our clinical development division. In exchange for the assets related to the ALDH2 deficiency program, what we now calls ConviviaTM , we issued to Convivia 46,625 shares of our common stock, an additional 46,625 shares of our common stock to a third party in settlement of a convertible loan between the third party and Convivia, and another 8,742 shares of our common stock in settlement of other obligations of Convivia. Mr. Daley, as the former sole stockholder of Convivia, may earn additional shares of our common stock based on certain triggering events or milestones related to the development of the Convivia assets. In addition, Mr. Daley may earn cash bonuses based on the same triggering events pursuant to his employment agreement. In January 2008, Mr. Daley earned a $30,000 cash bonus pursuant to his employment agreement as a result of the milestone of our execution of a formulation agreement for manufacturing ConviviaTM with Patheon. In March 2008, we issued to Mr. Daley 23,312 shares of our common stock pursuant to our Convivia purchase agreement as a result of the milestone of our execution of an agreement to supply us with the active pharmaceutical ingredient for ConviviaTM and two $10,000 cash bonuses pursuant to his employment agreement for reaching his six-month and one-year employment anniversaries. In October 2008, we issued to Mr. Daley 23,312 shares of our common stock valued at $27,000 and a $30,000 cash bonus as a result of fulfilling a clinical milestone.
Purchase of DR Cysteamine
In December 2007, through a merger between Encode Pharmaceuticals, Inc., or Encode, and our wholly-owned subsidiary, Raptor Therapeutics, we purchased certain assets, including the clinical development rights to DR Cysteamine. Under the terms of and subject to the conditions set forth in the merger agreement, we issued 802,946 shares of our common stock to the stockholders of Encode, or Encode Stockholders, options, or Encode Options, to purchase up to, in the aggregate, 83,325 shares of our common stock to the optionholders of Encode, or Encode Optionholders, and warrants, or Encode Warrants, to purchase up to, in the aggregate, 256,034 shares of our common stock to the warrantholders of Encode, or Encode Warrantholders, and together with the Encode Stockholders and Encode Optionholders, referred to herein collectively as the Encode Securityholders, as of the date of such agreement. Such common stock, Encode Options to purchase our common stock, and Encode Warrants to purchase our common stock combine for an aggregate amount of 1,142,304 shares of our common stock issuable to the Encode Securityholders as of the closing of the merger. The Encode Securityholders are eligible to receive up to an additional 559,496 shares of our common stock, Encode Options and Encode Warrants to purchase our common stock in the aggregate based on certain triggering events related to regulatory approval of DR Cysteamine, an Encode product program, if completed within the five year anniversary date of the merger agreement.
As a result of the Encode merger, we received the exclusive worldwide license to DR Cysteamine, referred to herein as the License Agreement, developed by clinical scientists at the UCSD, School of Medicine. In consideration of the grant of the license, we are obligated to pay an annual maintenance fee of $15,000 until we begin commercial sales of any products developed pursuant to the License Agreement. In addition to the maintenance fee, we are obligated to pay during the life of the License Agreement: milestone payments ranging from $20,000 to $750,000 for orphan indications and from $80,000 to $1,500,000 for non-orphan indications upon the occurrence of certain events, if ever; royalties on commercial net sales from products developed pursuant to the License Agreement ranging from 1.75% to 5.5%; a percentage of sublicense fees ranging from 25% to 50%; a percentage of sublicense royalties; and a minimum annual royalty commencing the year we begin commercially selling any products pursuant to the License Agreement, if ever. Under the License Agreement, we are obligated to fulfill predetermined milestones within a specified number of years ranging from 0.75 to 6 years from the effective date of the License Agreement, depending on the indication. In addition, we are obligated, among other things, to spend annually at least $200,000 for the development of products (which we satisfied, as of our fiscal year ended August 31, 2009 by spending approximately $4.1 million on such programs) pursuant to the License Agreement. To-date, we have paid $250,000 in milestone payments to UCSD based upon the initiation of clinical trials in cystinosis and in NASH. To the extent that we fail to perform any of our obligations under the License Agreement, UCSD may terminate the license or otherwise cause the license to become non-exclusive.

 

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Company History
Corporate Structure
We were incorporated in the State of Nevada on April 1, 2002 under the name of Highland Clan Creations Corp., or HCCC. On June 9, 2006, HCCC merged with our wholly-owned subsidiary, Raptor Pharmaceuticals Corp. incorporated on May 5, 2006 in Delaware. As a result, HCCC was reincorporated from the State of Nevada to the State of Delaware and changed our name to Raptor Pharmaceuticals Corp. HCC was a publicly traded company quoted on the OTC Bulletin Board and upon the merger traded under the ticker RPTP.
On May 25, 2006, we acquired 100% of the outstanding capital stock of Raptor Discoveries (f/k/a Raptor Pharmaceutical Inc.) (incorporated in Delaware on September 8, 2005), a development-stage research and development company and on June 9, 2006, we disposed of our former wholly-owned subsidiary, Bodysentials Health & Beauty Inc., which sold nutritional milkshakes and drinks on the Internet. On August 1, 2007, we formed Raptor Therapeutics Inc. (f/k/a Bennu Pharmaceuticals Inc.) as our wholly-owned subsidiary for the purpose of developing clinical-stage drug product candidates through to commercialization.
On September 29, 2009, we merged with a subsidiary of TorreyPines in a reverse triangular merger pursuant to which we became a wholly-owned subsidiary of TorreyPines and our stockholders exchanged their shares in us for shares in TorreyPines. TorreyPines changed its name to Raptor Pharmaceutical Corp. and began trading on the NASDAQ Capital Market under the ticker symbol “RPTPD”. Effective October 27, 2009, the ticker symbol changed to “RPTP”. Our Board and officers manage the combined company.
Financing History
Initial Investors
On May 25, 2006, in exchange for all of the outstanding common stock of Raptor Pharmaceutical Inc., we issued 1,864,987 shares of common stock to the Raptor Pharmaceutical Inc. stockholders including 699,370 shares of our common stock to each of Christopher M. Starr, Ph.D., and Todd C. Zankel, Ph.D., our Chief Executive Officer and Chief Scientific Officer, respectively, 233,123 shares of our common stock to Erich Sager, a member of our board of directors and 233,123 shares of common stock to an unrelated third party. These initial stockholders of Raptor Pharmaceutical Inc. purchased common stock of Raptor Pharmaceutical Inc. when we were a privately held company for the following amounts of proceeds: Dr. Starr $5,000; Dr. Zankel $5,000; Mr. Sager $100,000 and the unrelated third party $200,000.
$5 Million Financing and the 2006 Reverse Merger
Pursuant to an agreement dated March 8, 2006, with HCCC, on May 25, 2006, we closed a $5 million financing concurrent with a reverse merger. As part of that agreement, HCCC loaned us $0.2 million to be repaid with accrued interest upon the earlier of six months or the closing of the financing. Also, the agreement stated that pending the closing of at least a $3.5 million financing, HCCC would be obligated to issue 186,499 units as fees to a placement agent and $30,000 in commissions to an investment broker. In the financing HCCC sold 1,942,695 units at $2.57 per unit. Each unit consisted of one share of our common stock and one common stock purchase warrant exercisable for one share of our common stock at $2.57 per share. The warrants were exercisable for 18 months and expired on November 25, 2007. Gross proceeds from the financing were $5 million and net proceeds after the repayment of the $0.2 million loan plus interest and the deduction of commissions and legal fees totaled approximately $4.6 million. Prior to the warrants expiring, we received $3,895,000 in gross proceeds from the exercise of warrants in exchange for 1,513,359 shares of our common stock.
Issuance of Common Stock Pursuant to Stock Option Exercises
Since inception, we received $8,700 from the exercise of stock options resulting in the issuance of 3,380 shares of our common stock.

 

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2008 and 2009 Private Placements
In May and June 2008, referred to herein as the 2008 Closing, we entered into a securities purchase agreement, referred to herein as the 2008 Purchase Agreement, with eight investors, referred to herein as the 2008 Investors, for the private placement of our units, each unit comprised of one share of our common stock and one warrant to purchase one half of one share of our common stock, at a purchase price of $2.15 per unit. At the 2008 Closing, we sold an aggregate of 4,662,468 shares of common stock and two-year warrants with an exercise price of $3.22 or $3.86 per share, depending on when such warrants were exercised, if at all, to purchase up to an aggregate of 2,331,234 shares of common stock to the 2008 Investors for aggregate gross proceeds to us of $10 million. Pursuant to the 2008 Purchase Agreement, we agreed to prepare and file a registration statement within 60 days after the 2008 Closing with the SEC covering the common stock (including common stock underlying warrants, including warrants issued to placement agents) sold pursuant to the 2008 Purchase Agreement, subject to volume limitations, and to use our commercially reasonable efforts to cause such registration statement to become effective with the SEC thereafter and to remain effective until the earlier to occur of the date (i) that is the second anniversary of the 2008 Closing, (ii) the date such securities may be sold under Rule 144 during any 90-day period and (iii) such time as all of such securities have been publicly sold. We filed the registration statement in July 2008, which the SEC declared effective on August 7, 2008
In connection with the 2008 Closing, we issued warrants and a cash fee to placement agents to compensate them for placing investors into the financing. Placement agents were issued warrants exercisable for 7% of our common stock issued and issuable under the warrants issued to investors as part of the financing units and a cash fee based upon a percentage of the proceeds of the sale of the units of the 2008 Closing. In connection with the 2008 Closing, we issued placement agent warrants to purchase 489,559 shares of our common stock at an exercise price of $2.36 per share for a five year term and cash fees to placement agents totaling $700,000. One of the placement agents compensated, Limetree Capital, was issued warrants to purchase 438,890 shares of our common stock and paid a cash commission of $627,550. One of our board members serves on the board of Limetree Capital.
On April 29, 2009, in order to reflect then-current market prices, we notified the 2008 Investors that we were offering, in exchange for their 2008 warrants, new warrants to purchase our common stock at an exercise price of $1.29 per share, but only to the extent such exchange of the original warrants and exercise of the new warrants, including the delivery of the exercise price, occurred on or prior to July 17, 2009, such offer is herein referred to as the 2009 Warrant Exchange. The warrants that were not exchanged prior to or on July 17, 2009 retained their original exercise prices of $3.86 per share and original expiration date of May 21, 2010. We received approximately $2.6 million of proceeds from warrant exercises that resulted in the issuance of 2,031,670 shares of our common stock pursuant to the exchange described above.
In August 2009, referred to herein as the 2009 Closing, we entered into a securities purchase agreement, referred to herein as the 2009 Purchase Agreement, with four investors, referred to herein as the 2009 Investors, for the private placement of our units, each unit comprised of one share of our common stock and one warrant to purchase one half of one share of our common stock, at a purchase price of $1.37 per unit. At the 2009 Closing, we sold an aggregate of 1,738,226 shares of common stock and two-year warrants with an exercise price of $2.57 or $3.22 per share, depending on when such warrants were exercised, if at all, to purchase up to an aggregate of 869,113 shares of common stock to the 2009 Investors for aggregate gross proceeds to us of $2,386,000. Pursuant to the 2009 Purchase Agreement, we agreed to prepare and file a registration statement within 75 days after the 2009 Closing with the SEC covering the common stock (including common stock underlying warrants, including warrants issued to placement agents) sold pursuant to the 2009 Purchase Agreement, subject to volume limitations.
In connection with the 2009 Closing, we issued warrants and a cash fee to Limetree Capital, the sole placement agent, to compensate them for placing investors into the financing. The placement agent was issued warrants exercisable for 7% of our common stock issued and issuable under the warrants issued to investors as part of the financing units and a cash fee based upon a percentage of the proceeds of the sale of the units of the 2009 Closing introduced by Limetree. In connection with the 2009 Closing, we issued to the placement agent warrants to purchase 129,733 shares of our common stock at an exercise price of $1.50 per share for a five year term and paid a cash fee of $59,360.

 

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Proprietary Rights
We purchased from BioMarin the intellectual property owned by BioMarin for the research and development of the RAP technologies, including two patents, two pending patent applications and two provisional patent applications in review in the U.S., and countries in Europe and Asia and two trademarks for NeuroTrans™. Subsequent to the purchase from BioMarin, we have filed four additional patent applications for our RAP technologies. As of October 23, 2009, we have eight patent applications under prosecution in the U.S. and internationally. Two of these applications relate to cysteamine and the remaining six cover the RAP platform. Of the six RAP platform patents, two have been allowed in the U.S., as of July 14, and August 4, 2009, and another was allowed in Japan, Australia and Europe during the first half of 2009. All other applications are awaiting examination in a variety of countries. We also entered into an exclusive worldwide license agreement with Washington University for our Mesd program for the treatment of cancer and bone diseases. We fund the prosecution of a patent application covering this technology, entering national phase in the U.S. and internationally in November, 2009. In October 2007, we acquired intellectual property assets from Convivia, Inc., a privately held pharmaceutical company, including four filed patents for 4-MP as a potential treatment for ALDH2 deficiency. Since the acquisition of Convivia, Inc. assets, we filed a provisional patent for trans-dermal formulation of 4-MP, a provisional patent for genotype specific methods for treating human subjects using 4-methylpyrazole and a patent based on botanically derived compound for treatment of ALDH2 deficiency. In December 2007, we acquired an exclusive worldwide license agreement to pending patent applications from UCSD relating to our DR Cysteamine program. In March 2008, we amended our license with UCSD to add exclusive worldwide rights to develop DR Cysteamine for the potential treatment of NASH. Through our reverse merger with TorreyPines, we have a license from Eli Lilly & Co. for the intellectual property related to tezampanel and NGX426 for pain indications and a license of tezampanel and NGX 426 for the treatment of thrombotic disorder from JHU. We fund the prosecution of a patent covering this technology, which entered national phase in the U.S. in August, 2009.
Regulatory Exclusivities
Orphan Drug Designation
We have been granted access to an Orphan Drug Designation from the FDA for use of DR Cysteamine to potentially treat cystinosis and the use of Cysteamine to potentially treat HD and Batten Disease. The Orphan Drug Act of 1983 generally provides incentives, including marketing exclusivity and tax benefits, to companies that undertake development and marketing of products to treat relatively rare diseases, which are defined as meaning diseases for which fewer than 200,000 persons in the U.S. would be likely to receive the treatment. A drug that receives orphan drug status is entitled to up to seven years of exclusive marketing in the U.S. for that indication. Equivalent European regulations would give us ten years of marketing exclusivity for that indication in Europe. DR Cysteamine has already been granted Orphan Drug Designation by the FDA and we plan to submit an orphan drug application in Europe. We cannot be sure that we will be granted orphan drug status or that it would prove advantageous. In addition, the testing and approval process will likely require a significant commitment of time, effort, and expense on our part. If we fail to obtain or maintain orphan drug exclusivity for some of our drug product candidates, our competitors may sell products to treat the same conditions and our results of operations and revenues will be affected.
Facilities
Our primary offices are located at 9 Commercial Blvd., Suite 200, Novato, CA 94949. Our phone number is (415) 382-8111 and our facsimile number is (415) 382-1368. Our website is located at www.raptorpharma.com.
Competition
Cystinosis
The only pharmaceutical product currently approved by FDA and EMEA to treat cystinosis that we are aware of is Cystagon ® (rapid release cysteamine bitartrate capsules), marketed in the U.S. by Mylan Pharmaceuticals, and by Recordati and Swedish Orphan International in markets outside of the U.S. Cystagon ®

 

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was approved by FDA in 1994 and is the standard of care for cystinosis treatment. While we believe that our DR Cysteamine formulation will be well received in the market due to what we believe will be reduced dose frequency and improved tolerability, if we receive marketing approval, we anticipate that Cystagon ® will remain a well-established competitive product which may retain many patients, especially those for whom the dose schedule and tolerability do not pose significant problems.
We are not aware of any pharmaceutical company with an active program to develop an alternative therapy for cystinosis. There are companies developing and/or marketing products to treat symptoms and conditions related to, or resulting from cystinosis, but none developing products to treat the underlying metabolic disorder. Academic researchers in the U.S. and Europe are pursuing potential cures for cystinosis through gene therapy and stem cell therapy, as well as pro-drug approaches as alternatives to cysteamine bitartrate for cystinosis treatment. The development timeline for these approaches is many years.
Huntington’s Disease
We are not aware of any currently available treatment alternatives for HD, although there are products available such as Haldol, Klonopin and Xenazine to treat uncontrollable movements and mood swings that result from the disease. There are several pharmaceutical companies pursuing potential cures and treatments for HD, as well as numerous academic- and foundation-sponsored research efforts.
Companies with HD product candidates in development include Medivation, Inc., Amarin, Eli Lilly & Co., and Pfizer. Several other companies have drug candidates in preclinical development. Additionally, nutritional supplements including creatinine and coenzyme Q10 have been investigated as potential treatments for HD. The Huntington Study Group sponsors numerous studies of potential therapies for HD, including coenzyme Q10 and the antibiotic minocycline.
NASH
We are not aware of any currently available treatment options for NASH. Weight loss, healthy diet, abstinence from alcohol and increased physical activity are typically suggested to slow the onset of NASH. There are numerous therapies being studied for NASH, including anti-oxidants (Vitamin E, betaine, Moexipril from Univasc), insulin sensitizing agents (Actos ® from Takeda Pharmaceuticals for type 2 diabetes, in an ongoing phase III study for NASH sponsored by University of Texas) and drugs to improve blood flow (Trental ® from Aventis for treatment of intermittent claudication, which is reported to have failed to meet endpoints in a phase II study for NASH). Gilead Sciences is developing a pan-caspase inhibitor for NASH. Other products being studied for NASH include Byetta from Amylin, in an ongoing phase II/III study for NASH; and siliphos, or milk thistle, in a UCSD phase II study for NASH.
ALDH2 Deficiency
ALDH2 deficiency affects hundreds of millions of people worldwide and is especially prevalent in East Asian populations. The association of this metabolic disorder with serious health risks, including liver diseases and digestive tract cancers, has been documented in numerous peer-reviewed studies over the last 10 years. We are not aware of any pharmaceutical products currently approved for this indication, either in the U.S. or internationally. However, given the size of the potential patient population and the emerging awareness of this disorder as a serious health risk, we expect there are or will be other pharmaceutical companies, especially those with commercial operations in Asian countries, developing products to treat the symptoms of this condition. Many of these competitors may have greater resources, and existing commercial operations in the Asian countries which we expect will be the primary markets for this product.
Additionally, there are non-pharmaceutical products available such as supplements and traditional remedies, especially in some Asian countries, which are claimed to be effective in reducing the symptoms associated with ALDH2 deficiency and other physical reactions to ethanol consumption. Although we are not aware of any study which has demonstrated the efficacy of such non-pharmaceutical alternatives, these products may compete with our ALDH2 deficiency product candidate if it is approved for marketing.

 

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Migraine
Triptans are the most commonly prescribed drugs for the treatment of moderate to severe migraine. There are seven triptans approved for use and Imitrex ® , marketed by GlaxoSmithKline, dominates the market. Other triptans are: Zomig ® , Maxalt ® , Amerge ® , Frova™, Axert ® , and Relpax ®. According to PhRMA’s 2008 report, Medicines in Development for Neurologic Disorders, there are more than 30 companies seeking to develop compounds to treat migraine and pain disorders or to obtain additional indications to broaden the use of currently approved pain relieving prescription medications. This list includes most of the large pharmaceutical companies such as Abbott Laboratories, AstraZeneca, Eisai, Elan, Eli Lilly, GlaxoSmithKline, Merck, Pfizer, and Wyeth Pharmaceuticals as well as small and mid-sized biotechnology companies.
Pain
In the neuropathic pain market, we would compete with companies such as Pfizer, marketing Neurontin and Lyrica® , and Eli Lilly, marketing Cymbalta® in addition to opioids approved for treating neuropathic pain, off-label uses of products to treat neuropathic pain and generic products. Given the size of the neuropathic pain market, approximately $3.5 billion in 2006 and expected to double by 2016, it is likely that most of the large pharmaceutical companies as well as many biotechnology companies will look to develop compounds to treat neuropathic pain. Since the licensing of tezampanel, Lilly has continued development of more potent and specific molecules (e.g., iGluR5 antagonists) targeting the same receptors as tezampanel and NGX424 and based on the same chemistry (i.e., tetrahydroisoquinoline moiety) as tezampanel and NGX424. Their third generation candidate is now in phase II studies for osteoarthritis and peripheral neuropathy.
Primary Liver Cancer
Surgical resection of the primary tumor or liver transplantation remains the only curative options for HCC patients. The acute and tragic nature of this aggressive cancer and the widely preserved unmet medical need continues to attract a significant level of interest in finding ways of treating this disease. For example, there are currently over 140 ongoing clinical trials actively recruiting patients with HCC listed in the ClinicalTrials.gov website. Many of these trials are designed to evaluate ways of locally administering chemotherapeutics or various ways of performing surgical resections of the tumors. One drug that was approved in 2007 for treatment of inoperable HCC is currently the standard-of-care for this disease due to its claims of enhancing overall survival time. This enhancement has been determined to be even smaller within the Asian population of inoperable HCC patients. We believe that a number of biotechnology and pharmaceutical companies may have internal programs targeting the development of new therapeutics that may be useful in treating HCC in the future.
Hepatitis
It has been estimated that approximately 3% of the world’s population is chronically infected with hepatitis C, which translates to nearly 200 million people infected worldwide. Due to the latency of hepatitis C virus, or HCV, infection and slow disease progression, along with a lag in awareness of the disease, the number of patients with HCV is increasing and expecting to peak in the next 20-30 years. Over 50,000 people die of HCV infection every year. Up to 75% of chronically infected individuals carry the genotype I strain of HCV. The most effective current treatment for chronic HCV infection is Interferon, but nearly 60% of patients infected with genotype 1 do not show a sustained viral reduction with Interferon treatment, and the remaining 40% of such genotype 1 HCV cases are without any therapy.
The significant number of interferon non-responders has created a need for second generation therapies and a large number of pharmaceutical companies have active therapeutic programs to meet the requirements of this large and growing market. There are currently 28 compounds in clinical development for the treatment of chronic HCV infection. A large number of these clinical compounds are small molecule antivirals being developed by pharmaceutical companies including Novartis, Kemin, Vertex and Migenix. In addition, over a dozen non-interferon immunomodulators are currently under clinical development by companies including SciClone, Schering-Plough, Chiron and Innogenetics. These compounds are designed to attack different parts of the Hepatitis C virus and its ability to replicate or enhance the body’s immune system to better recognize and destroy the virus. Most clinicians now believe that eventually these and future drugs will be used in combination to treat chronic HCV.

 

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Brain Delivery
We believe we will be competing with other pharmaceutical and biotechnology companies that provide, or are attempting to develop product candidates to provide, remedies and treatments for brain and neurodegenerative diseases.
Three approaches are primarily used to solve the problem of reaching the brain with therapeutic compounds:
   
Neurosurgery or invasive techniques.
 
   
Pharmacological techniques, which include less than 2% of currently available drugs.
 
   
Physiologically based techniques, such as transcytosis.
Invasive techniques include bone marrow transplants or implants of polymers with drugs imbedded in the material for slow release. These implants extend from the skull surface to deep into brain tissue sites and use a permeation enhancer. Mannitol induced osmotic shock that creates leaks in the blood-brain barrier allowing intravenous administered chemotherapeutics into the brain is used in the treatment of brain tumors, but is not appropriate for administration of drugs for chronic therapies. Companies active in developing treatments based on these invasive technologies include Alza Corporation, Ethypharm, Guilford Pharmaceuticals, Medtronic Inc., Neurotech, and Sumitomo Pharmaceutical.
Other invasive procedures utilize catheter-based delivery of the drug directly into the brain. This technique has proven useful in the treatment of brain tumors, but has not been successful in distributing drugs throughout the entire brain. Amgen Inc. recently conducted clinical trials for the treatment of Parkinson’s disease using intrathecal delivery through the use of various catheter/pump techniques.
The physiological route is a popular approach to cross the blood-brain barrier via lipid mediated free diffusion or by facilitated transport. This is the most common strategy used for the development of new neuropharmaceuticals, but has experienced limited success as it requires that the drug have sufficient lipophilic or fat-soluble properties so that it can pass through lipid membranes. The current method of delivery by this route, however, is nonspecific to the brain and side effects are common since most organs are exposed to the drug. Furthermore, many of the potential lipophilic therapeutic molecules are substrates for the blood-brain barrier’s multi-drug resistant proteins, which actively transport the therapeutic agent back into the blood. Consequently, large doses need to be used so that sufficient amounts of the drug reach the brain. These high doses can result in significant side effects as the drug is delivered to essentially all tissues of the body, which is extremely inefficient. Companies and organizations that are developing treatments based on various physiological approaches include Angiochem, AramGen Technology, to-BBB, Xenoport Inc., Bioasis, Oregon Health and Science University Neuro-oncology, Xenova Group Ltd., d-Pharm, Neurochem Inc., and Vasogen Inc.
Thrombotic Disorder
A number of anti-platelet drugs are already available on the market. These include the ADP receptor antagonist Plavix, the cyclooxygenase (and hence thromboxane) inhibitor, aspirin, and injectable integrin (IIb/IIIA) blockers such as Integrelin. Each drug has strengths and weaknesses (which predominantly involve excess bleeding). Since anti-thrombotic drugs are a multi-billion dollar market, it is likely that a large number of companies have additional therapies in development.
Because, many of our competitors have greater capital resources and larger overall research and development staffs and facilities, than us, there can be no assurances that we will be successful in competing in the areas discussed above. See the section under “Risk Factors” titled, “If our competitors succeed in developing products and technologies that are more effective than ours, or if scientific developments change our understanding of the potential scope and utility of our drug product candidates, then our technologies and future drug product candidates may be rendered less competitive.”

 

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Government Regulations of the Biotechnology Industry
Regulation by governmental authorities in the U.S. and foreign countries is a significant factor in the development, manufacture, and expected marketing of our drug product candidates and in our ongoing research and development activities. The nature and extent to which such regulation will apply to us will vary depending on the nature of any drug product candidates developed. We anticipate that all of our drug product candidates will require regulatory approval by governmental agencies prior to commercialization.
In particular, human therapeutic products are subject to rigorous preclinical and clinical testing and other approval procedures of the FDA and similar regulatory authorities in other countries. Various federal statutes and regulations also govern or influence testing, manufacturing, safety, labeling, storage, and record-keeping related to such products and their marketing. The process of obtaining these approvals and the subsequent compliance with the appropriate federal statutes and regulations requires substantial time and financial resources. Any failure by us or our collaborators to obtain, or any delay in obtaining, regulatory approval could adversely affect the marketing of any of our drug product candidates, our ability to receive product revenues, and our liquidity and capital resources.
The FDA’s Modernization Act codified the FDA’s policy of granting “fast track” review of certain therapies targeting “orphan” indications and other therapies intended to treat severe or life threatening diseases and having potential to address unmet medical needs. Orphan indications are defined by the FDA as having a prevalence of less than 200,000 patients in the U.S. We anticipate that certain genetic diseases and primary liver cancer which could potentially be treated using our technology could qualify for fast track review under these revised guidelines. There can be no assurances, however, that we will be able to obtain fast track designation and, even with fast track designation, it is not guaranteed that the total review process will be faster or that approval will be obtained, if at all, earlier than would be the case if the drug product candidate had not received fast-track designation.
Before obtaining regulatory approvals for the commercial sale of any of our products under development, we must demonstrate through preclinical studies and clinical trials that the product is safe and efficacious for use in each target indication. The results from preclinical studies and early clinical trials might not be predictive of results that will be obtained in large-scale testing. Our clinical trials might not successfully demonstrate the safety and efficacy of any product candidates or result in marketable products.
In order to clinically test, manufacture, and market products for therapeutic use, we will have to satisfy mandatory procedures and safety and effectiveness standards established by various regulatory bodies. In the U.S., the Public Health Service Act and the Federal Food, Drug, and Cosmetic Act, as amended, and the regulations promulgated thereunder, and other federal and state statutes and regulations govern, among other things, the testing, manufacture, labeling, storage, record keeping, approval, advertising, and promotion of our current and proposed product candidates. Product development and approval within this regulatory framework takes a number of years and involves the expenditure of substantial resources.
The steps required by the FDA before new drug products may be marketed in the U.S. include:
   
completion of preclinical studies;
 
   
the submission to the FDA of a request for authorization to conduct clinical trials on an investigational new drug application, or IND, which must become effective before clinical trials may commence;
 
   
adequate and well-controlled Phase I, Phase II and Phase III clinical trials to establish and confirm the safety and efficacy of a drug candidate;
 
   
submission to the FDA of a new drug application, or NDA, for the drug candidate for marketing approval; and
 
   
review and approval of the NDA by the FDA before the product may be shipped or sold commercially.

 

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In addition to obtaining FDA approval for each product, each product manufacturing establishment must be registered with the FDA and undergo an inspection prior to the approval of an NDA. Each manufacturing facility and its quality control and manufacturing procedures must also conform and adhere at all times to the FDA’s cGMP regulations. In addition to preapproval inspections, the FDA and other government agencies regularly inspect manufacturing facilities for compliance with these requirements. If, as a result of these inspections, the FDA determines that any equipment, facilities, laboratories or processes do not comply with applicable FDA regulations and conditions of product approval, the FDA may seek civil, criminal, or administrative sanctions and/or remedies against us, including the suspension of the manufacturing operations. Manufacturers must expend substantial time, money and effort in the area of production and quality control to ensure full technical compliance with these standards.
Preclinical testing includes laboratory evaluation and characterization of the safety and efficacy of a drug and its formulation. Preclinical testing results are submitted to the FDA as a part of an IND which must become effective prior to commencement of clinical trials. Clinical trials are typically conducted in three sequential phases following submission of an IND. Phase I represents the initial administration of the drug to a small group of humans, either patients or healthy volunteers, typically to test for safety (adverse effects), dosage tolerance, absorption, distribution, metabolism, excretion and clinical pharmacology, and, if possible, to gain early evidence of effectiveness. Phase II involves studies in a small sample of the actual intended patient population to assess the efficacy of the drug for a specific indication, to determine dose tolerance and the optimal dose range and to gather additional information relating to safety and potential adverse effects. Once an investigational drug is found to have some efficacy and an acceptable safety profile in the targeted patient population, Phase III studies are initiated to further establish clinical safety and efficacy of the therapy in a broader sample of the general patient population, in order to determine the overall risk-benefit ratio of the drug and to provide an adequate basis for any physician labeling. During all clinical studies, we must adhere to Good Clinical Practice, or GCP, standards. The results of the research and product development, manufacturing, preclinical studies, clinical studies and related information are submitted in an NDA to the FDA.
The process of completing clinical testing and obtaining FDA approval for a new drug is likely to take a number of years and require the expenditure of substantial resources. If an application is submitted, there can be no assurance that the FDA will review and approve the NDA. Even after initial FDA approval has been obtained, further studies, including post-market studies, might be required to provide additional data on safety and will be required to gain approval for the use of a product as a treatment for clinical indications other than those for which the product was initially tested and approved. Also, the FDA will require post-market reporting and might require surveillance programs to monitor the side effects of the drug. Results of post-marketing programs might limit or expand the further marketing of the products. Further, if there are any modifications to the drug, including changes in indication, manufacturing process, labeling or a change in manufacturing facility, an NDA supplement might be required to be submitted to the FDA.
The rate of completion of any clinical trials will be dependent upon, among other factors, the rate of patient enrollment. Patient enrollment is a function of many factors, including the size of the patient population, the nature of the trial, the availability of alternative therapies and drugs, the proximity of patients to clinical sites and the eligibility criteria for the study. Delays in planned patient enrollment might result in increased costs and delays, which could have a material adverse effect on us.
We do not know whether our IND for future products or the protocols for any future clinical trials will be accepted by the FDA. We do not know if our clinical trials will begin or be completed on schedule or at all. Even if completed, we do not know if these trials will produce clinically meaningful results sufficient to support an application for marketing approval. The commencement of our planned clinical trials could be substantially delayed or prevented by several factors, including:
   
a limited number of, and competition for, suitable patients with particular types of disease for enrollment in clinical trials;
 
   
delays or failures in obtaining regulatory clearance to commence a clinical trial;

 

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delays or failures in obtaining sufficient clinical materials;
 
   
delays or failures in reaching agreement on acceptable clinical trial agreement terms or clinical trial protocols with prospective sites; and
 
   
delays or failures in obtaining Institutional Review Board, or IRB, approval to conduct a clinical trial at a prospective site.
The completion of our clinical trials could also be substantially delayed or prevented by several factors, including:
   
slower than expected rates of patient recruitment and enrollment;
 
   
failure of patients to complete the clinical trial;
 
   
unforeseen safety issues;
 
   
lack of efficacy during clinical trials;
 
   
inability or unwillingness of patients or medical investigators to follow our clinical trial protocols;
 
   
inability to monitor patients adequately during or after treatment; and
 
   
regulatory action by the FDA for failure to comply with regulatory requirements.
Failure to comply with applicable FDA requirements may result in a number of consequences that could materially and adversely affect us. Failure to adhere to approved trial standards and GCPs in conducting clinical trials could cause the FDA to place a clinical hold on one or more studies which would delay research and data collection necessary for product approval. Noncompliance with GCPs could also have a negative impact on the FDA’s evaluation of an NDA. Failure to adhere to GMPs and other applicable requirements could result in FDA enforcement action and in civil and criminal sanctions, including but not limited to fines, seizure of product, refusal of the FDA to approve product approval applications, withdrawal of approved applications, and prosecution.
Whether or not FDA approval has been obtained, approval of a product by regulatory authorities in foreign countries must be obtained prior to the commencement of marketing of the product in those countries. The requirements governing the conduct of clinical trials and product approvals vary widely from country to country, and the time required for approval might be longer or shorter than that required for FDA approval. Although there are some procedures for unified filings for some European countries, in general, each country at this time has its own procedures and requirements. There can be no assurance that any foreign approvals would be obtained.
In most cases, if the FDA has not approved a drug product candidate for sale in the U.S., the drug product candidate may be exported for sale outside of the U.S. only if it has been approved in any one of the following: the European Union, Canada, Australia, New Zealand, Japan, Israel, Switzerland and South Africa. Specific FDA regulations govern this process.
In addition to the regulatory framework for product approvals, we and our collaborative partners must comply with federal, state, and local laws and regulations regarding occupational safety, laboratory practices, the use, handling and disposition of radioactive materials, environmental protection and hazardous substance control, and other local, state, federal and foreign regulation. All facilities and manufacturing processes used by third parties to produce our drug candidates for clinical use in the United States must conform with cGMPs. These facilities and practices are subject to periodic regulatory inspections to ensure compliance with cGMP requirements. Their failure to comply with applicable regulations could extend, delay, or cause the termination of clinical trials conducted for our drug candidates. The impact of government regulation upon us cannot be predicted and could be material and adverse. We cannot accurately predict the extent of government regulation that might result from future legislation or administrative action.

 

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Scientific Advisory Board
The following describes the background of our Scientific Advisory Board.
Stephen C. Blacklow, M.D., Ph.D. Over the last ten years, Dr. Blacklow’s research team has achieved international recognition both for their mechanistic and structural studies of proteins of the LDL receptor family, and for their work on the structure and function of human Notch proteins. Recently, Dr. Blacklow’s team determined the structure of RAP d3 complex with receptor. Dr. Blacklow graduated from Harvard College summa cum laude in 1983, and received his M.D. and Ph.D. in bioorganic chemistry from Harvard University in 1991. Dr. Blacklow is a board-certified pathologist and an Associate Professor of Pathology at Harvard Medical School where he is the Director of the Harvard M.D.-Ph.D. program, basic sciences track. He has directed a research laboratory at the Brigham and Women’s Hospital, a teaching affiliate of the Harvard Medical School, since 1998.
Guojun Bu, Ph.D. Guojun Bu, Ph.D., is a molecular and cell biologist and a leader in the field of the LDL receptor family. Dr. Bu obtained his undergraduate degree from the Beijing Normal University in China. He then studied biochemistry and molecular biology in the Department of Biochemistry at Virginia Tech where he received his Ph.D. Dr. Bu moved to the Washington University School of Medicine for a postdoctoral training in cell biology where he later became a member of the faculty. He is currently Professor of Pediatrics, and of Cell Biology and Physiology. Among the numerous awards that he has received, Dr. Bu has been a Faculty Scholar of the Alzheimer’s Association and an Established Investigator of the American Heart Association. He currently serves as the Editor-in-Chief of Molecular Neurodegeneration.
Ranjan Dohil, M.D. Ranjan Dohil, M.D., is Professor of Pediatrics at the University of California, San Diego, within the Division of Gastroenterology, Hepatology and Nutrition. He has also served as the conference director of the first Cystinosis Research Foundation International Conference in Newport Beach (April 3-5 2008). An interest in childhood acid-peptic disorders led Dr. Dohil to study patients with cystinosis taking cysteamine. He subsequently went on to study the pharmacokinetics of cysteamine with the intention of helping to develop a new formulation of cysteamine that would result in an improved quality of life for patients with cystinosis. Dr. Dohil also has a research interest in eosinophilic esophagitis, a condition that over the past few years has increased in incidence. Within this field, his work has led to the development of a treatment that is becoming more widely used. Dr. Dohil undertook his medical training at the University of Wales College of Medicine in Cardiff, U.K. He has served as a physician in many hospitals over his career including the University Hospital of Wales in Cardiff, U.K., the British Military Hospital in Iserlohn, Germany, and British Columbia’s Children’s Hospital in Vancouver, Canada. He is a reviewer for several respected academic publications, including the Journal of Pediatrics, Pediatrics and the American Journal of Gastroenterology.
William C. Mobley, M.D., Ph.D. After completing undergraduate training in Chemistry and Zoology at the University of Nebraska at Lincoln, William C. Mobley, M.D., Ph.D., received his M.D. and Ph.D. in Neuroscience from Stanford University. Dr. Mobley trained in Pathology and Pediatrics at the Stanford University Hospital and completed a residency and fellowship in Neurology at Johns Hopkins University Hospital, where he also was Chief Resident in Pediatric Neurology. In 1985, he joined the faculty of the University of California, San Francisco School of Medicine where he rose to the rank of Professor of Neurology, Pediatrics and the Neuroscience Program and served as the Director of Child Neurology. In 1991, he was named Derek Denny Brown Scholar of the American Neurological Association. From 1997 to 2005, he served as the Chair of the Department of Neurology and Neurological Sciences at Stanford University, and he held the John E. Cahill Family Endowed Chair. He was appointed Director of the Neuroscience Institute at Stanford. While at Stanford his laboratory studied the signaling biology of neurotrophic factors in the normal nervous system and in animal models of neurological disorders, including Alzheimer’s disease, Down’s syndrome and peripheral neuropathy. He is the recipient of both the Zenith Award and the Temple Award from the Alzheimer’s Association and is a Fellow of the Royal College of Physicians. He was chosen to receive the Cotzias Award of the American Academy of Neurology for 2004. Dr. Mobley is Past President of the Association of University Professors of Neurology and is President of The Professors of Child Neurology. He was recently elected to the Institute of Medicine of the National Academy of Sciences. Dr Mobley now serves as the chair of the department of neurosciences at the University of California, San Diego School of Medicine since April 2009.

 

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Jerry Schneider, M.D. Jerry Schneider, M.D. is Research Professor of Pediatrics and Dean for Academic Affairs Emeritus at the University of California, San Diego (UCSD) School of Medicine. He also serves as a member of the Board of Directors and Chair of the Scientific Review Board for the Cystinosis Research Foundation. Over the course of his distinguished career, Dr. Schneider has been actively involved in the study of metabolic diseases. An expert on the diagnosis and treatment of cystinosis, Dr. Schneider has published over 150 papers on cystinosis and related subjects over the past 40 years. Since 1969 he has been associated with the UCSD School of Medicine in both academic and research capacities. Dr. Schneider earned his M.D. from Northwestern University. He received postgraduate training at Johns Hopkins University, the National Institutes of Health (NIH), and the Centre de Genetique Moleculaire, Gif-sur-Yvette, France. He was also a Guggenheim Fellow and a Fogarty Senior Fellow at the Imperial Cancer Research Fund Laboratories in London, England.
Sam Teichman, M.D., FACC, FACP Sam Teichman, M.D., is an independent consultant in the area of strategic drug discovery and development. He has worked on over 40 medical products in various stages of development from the earliest identification of leads in research to supporting commercial-stage products. Most recently, Dr. Teichman served as Vice President and Chief Development Officer at ARYx Therapeutics, where he was involved in identifying and advancing three products from the research stage into clinical development. During the past 20 years, Dr. Teichman has held senior level executive positions at Genentech, Medco Research (now part of King Pharmaceuticals), Glycomed (now part of Ligand Pharmaceuticals), and Mimetix. He has provided scientific advisory services and has acted in an interim executive role for numerous early-stage and established biotechnology companies. Dr. Teichman holds an M.D. from Columbia University and a B.S. in Chemistry from Columbia College, Columbia University. He is board certified in Internal Medicine and Cardiology. Dr. Teichman is a Fellow of the American College of Cardiology (FACC) and the American College of Physicians (FACP). Dr. Teichman served as Associate Clinical Professor of Medicine at University of California in San Francisco from 1990 to 2001. He has more than 40 original publications, reviews and abstracts published in peer-reviewed and invited medical journals.
Legal Proceedings
Several lawsuits were filed against TorreyPines in February 2005 in the U.S. District Court for the Southern District of New York asserting claims under Sections 10(b) and 20(a) of the Exchange Act and Rule 10b-5 thereunder on behalf of a class of purchasers of TorreyPines’ common stock during the period from June 26, 2003, through and including February 4, 2005, referred to as the class period. Dr. Marvin S. Hausman, M.D., a former director and TorreyPines’ former Chief Executive Officer, and Dr. Gosse B. Bruinsma, M.D., also a former director and TorreyPines’ former Chief Executive Officer, were also named as defendants in the lawsuits. These actions were consolidated into a single class action lawsuit in January 2006. On April 10, 2006, the class action plaintiffs filed an amended consolidated complaint. TorreyPines filed its answer to that complaint on May 26, 2006. TorreyPines’ motion to dismiss the consolidated amended complaint was filed on May 26, 2006 and was submitted to the court for a decision in September 2006. On March 31, 2009 the U.S. District Court for the Southern District of New York dismissed the proceedings. On April 24, 2009, an appeal was filed with the United States Court of Appeals for the Second Circuit by the class action plaintiffs. Our response to such appeal was filed on October 23, 2009. We do not anticipate that this claim, if successful, would burden the Company with any additional liability above and beyond the insurance coverage provided under the insurance policy that we currently maintain.
Other than as described above, we know of no material, active or pending legal proceedings against us, nor are we involved as a plaintiff in any material proceedings or pending litigation. There are no proceedings in which any of our directors, officers or affiliates, or any registered or beneficial stockholders are an adverse party or have a material interest adverse to us.
Research and Development
We are a research and development company and our plan is to focus our efforts in the discovery, research, preclinical and clinical development of our RAP based platforms, complementary technologies and clinical drug candidates to provide therapies that we believe will be safer, less intrusive, and more effective than current approaches in treating a wide variety of brain disorders and neurodegenerative diseases, genetic disorders and cancer. During the period from September 8, 2005 (inception) to August 31, 2009, we incurred approximately $14.9

 

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million ($6.6 million and $5.6 million for the years ended August 31, 2009 and 2008, respectively) in research and development costs. Please see the section titled, “Management’s Discussion and Analysis of Financial Condition and Results of Operations” in this Annual Report on Form 10-K for our planned research and development activities for the next twelve months.
Compliance with Environmental Laws
We estimate the annual cost of compliance with environmental laws, comprised primarily of hazardous waste removal, will be approximately $5,000.
Employees
We presently have twelve full time employees, including eight executives, one scientist, one program director and one clinical development assistant in our research and development department and one senior manager in our finance department. Three of the executives were retained as part of the merger with TorreyPines in September 2009. Based on our current plan, over the next 12 month period, we anticipate hiring a regulatory director. We also plan to supplement our human resources needs through consultants and contractors as needed.
ITEM 1A: RISK FACTORS
An investment in our common stock involves a high degree of risk. Before investing in our common stock, you should consider carefully the specific risks detailed in this “Risk Factors” section before making a decision to invest in our common stock, together with all of the other information contained in this Annual Report. If any of these risks occur, our business, results of operations and financial condition could be harmed, the price of our common stock could decline, and you may lose all or part of your investment.
Risks Related to Our Business
If we fail to obtain the capital necessary to fund our operations, our financial results, financial condition and our ability to continue as a going concern will be adversely affected and we will have to delay or terminate some or all of our product development programs.
Our consolidated financial statements as of August 31, 2009 have been prepared assuming that we will continue as a going concern. As of August 31, 2009, Raptor had an accumulated deficit of approximately $21.9 million. We expect to continue to incur losses for the foreseeable future and will have to raise substantial cash to fund our planned operations.
We believe that our cash and cash equivalents balances as of October 23, 2009, will be sufficient to meet our obligations into the first calendar quarter of 2010. We are currently in the process of reviewing strategic partnerships and collaborations in order to fully fund our preclinical and clinical programs through the end of 2010. We also anticipate raising additional capital in the fourth calendar quarter of 2009. These estimates are based on assumptions that may prove to be wrong. In addition to the activities described herein above, we anticipate that we will need to raise funds in the future for the continued development of our drug development programs. We will need to sell equity or debt securities to raise significant additional funds. The sale of additional securities is likely to result in additional dilution to our stockholders. Additional financing may not be available in amounts or on terms satisfactory to us or at all. We may be unable to raise additional financing due to a variety of factors, including our financial condition, the status of our research and development programs, and the general condition of the financial markets. If we fail to raise significant additional financing, we will have to delay or terminate some or all of our research and development programs, our financial condition and operating results will be adversely affected and we may have to cease our operations.
If we obtain significant additional financing, we expect to continue to spend substantial amounts of capital on our operations for the foreseeable future. The amount of additional capital we will need depends on many factors, including:

 

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the progress, timing and scope of our preclinical studies and clinical trials;
 
   
the time and cost necessary to obtain regulatory approvals;
 
   
the time and cost necessary to develop commercial manufacturing processes, including quality systems, and to build or acquire manufacturing capabilities;
 
   
the time and cost necessary to respond to technological and market developments; and
 
   
any changes made or new developments in our existing collaborative, licensing and other corporate relationships or any new collaborative, licensing and other commercial relationships that we may establish.
Moreover, our fixed expenses such as rent, collaboration and license payments and other contractual commitments are substantial and will likely increase in the future. These fixed expenses are likely to increase because we expect to enter into:
   
additional licenses and collaborative agreements;
 
   
contracts for manufacturing, clinical and preclinical research, consulting, maintenance and administrative services; and
 
   
financing facilities.
We are an early development stage company and have not generated any revenues to date and have a limited operating history. Many of our drug product candidates are in the concept stage and have not undergone significant testing in preclinical studies or any testing in clinical trials. Moreover, we cannot be certain that our research and development efforts will be successful or, if successful, that our drug product candidates will ever be approved for sale or generate commercial revenues. We have a limited relevant operating history upon which an evaluation of our performance and prospects can be made. We are subject to all of the business risks associated with a new enterprise, including, but not limited to, risks of unforeseen capital requirements, failure of drug product candidates either in preclinical testing or in clinical trials, failure to establish business relationships, and competitive disadvantages against larger and more established companies.
If we do not make important progress towards achieving at least one of our major clinical objectives, as outlined in the section titled, “Management’s Discussion and Analysis of Financial Conditions and Results of Operations” in this Annual Report on Form 10-K, this could adversely impact our ability to raise significant additional funds, which could adversely impact our ability to continue as a going concern.
The current disruptions in the financial markets could affect our ability to obtain financing on favorable terms (or at all).
The U.S. credit markets have recently experienced historic dislocations and liquidity disruptions which have caused financing to be unavailable in many cases and, even if available, have caused the cost of prospective financings to increase. These circumstances have materially impacted liquidity in the debt markets, making financing terms for borrowers able to find financing less attractive, and in many cases have resulted in the unavailability of certain types of debt financing. Continued uncertainty in the debt and equity markets may negatively impact our ability to access financing on favorable terms or at all. In addition, Federal legislation to deal with the current disruptions in the financial markets could have an adverse affect on our ability to raise other types of financing.

 

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Even if we are able to develop our drug product candidates, we may not be able to receive regulatory approval, or if approved, we may not be able to generate significant revenues or successfully commercialize our products, which would adversely affect our financial results and financial condition and we would have to delay or terminate some or all of our research product development programs.
All of our drug product candidates are at an early stage of development and will require extensive additional research and development, including preclinical testing and clinical trials, as well as regulatory approvals, before we can market them.
Since TorreyPines’ inception in 1997 and since we began operations in 2005, both companies have dedicated substantially all of their resources to the research and development of their technologies and related compounds. All of our compounds currently are in preclinical or clinical development, and none have been submitted for marketing approval. Our preclinical compounds may not enter human clinical trials on a timely basis, if at all, and we may not develop any product candidates suitable for commercialization.
We cannot predict if or when any of the drug product candidates we intend to develop will be approved for marketing. There are many reasons that we may fail in our efforts to develop our drug product candidates. These include:
   
the possibility that preclinical testing or clinical trials may show that our drug product candidates are ineffective and/or cause harmful side effects;
 
   
our drug product candidates may prove to be too expensive to manufacture or administer to patients;
 
   
our drug product candidates may fail to receive necessary regulatory approvals from the FDA or foreign regulatory authorities in a timely manner, or at all;
 
   
our drug product candidates, if approved, may not be produced in commercial quantities or at reasonable costs;
 
   
our drug product candidates, if approved, may not achieve commercial acceptance;
 
   
regulatory or governmental authorities may apply restrictions to our drug product candidates, which could adversely affect their commercial success; and
 
   
the proprietary rights of other parties may prevent us or our potential collaborative partners from marketing our drug product candidates.
If we fail to develop our drug product candidates, our financial results and financial condition will be adversely affected, we will have to delay or terminate some or all of our research product development programs and may be forced to cease operations.
If we are limited in our ability to utilize acquired or licensed technologies, we may be unable to develop, out-license, market and sell our product candidates, which could cause delayed new product introductions, and/or adversely affect our reputation, any of which could have a material adverse effect on our business, prospects, financial condition, and operating results.
We have acquired and licensed certain proprietary technologies, discussed in the following risk factors, and plan to further license and acquire various patents and proprietary technologies owned by third parties. These agreements are critical to our product development programs. These agreements may be terminated, and all rights to the technologies and product candidates will be lost, if we fail to perform our obligations under these agreements and licenses in accordance with their terms including, but not limited to, our ability to make all payments due under such agreements. Our inability to continue to maintain these technologies could materially adversely affect our business, prospects, financial condition, and operating results. In addition, our business strategy depends on the successful development of these licensed and acquired technologies into commercial products, and, therefore, any limitations on our ability to utilize these technologies may impair our ability to develop, out-license, market and sell our product candidates, delay new product introductions, and/or adversely affect our reputation, any of which could have a material adverse effect on our business, prospects, financial condition, and operating results.

 

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If the purchase or licensing agreements we entered into are terminated, we will lose the right to use or exploit our owned and licensed technologies, in which case we will have to delay or terminate some or all of our research and development programs, our financial condition and operating results will be adversely affected and we may have to cease our operations.
We entered into an asset purchase agreement with BioMarin Pharmaceutical Inc., or BioMarin, for the purchase of intellectual property related to the receptor-associated protein, or RAP, technology, a licensing agreement with Washington University for mesoderm development protein, or Mesd, and a licensing agreement with UCSD for DR Cysteamine. BioMarin, Washington University and UCSD may terminate their respective agreements with us upon the occurrence of certain events, including if we enter into certain bankruptcy proceedings or if we materially breach our payment obligations and fail to remedy the breach within the permitted cure periods. Although we are not currently involved in any bankruptcy proceedings or in breach of these agreements, there is a risk that we may be in the future, giving BioMarin, Washington University and UCSD the right to terminate their respective agreements with us. We have the right to terminate these agreements at any time by giving prior written notice. If the BioMarin, Washington University or UCSD agreements are terminated by either party, we would be forced to assign back to BioMarin, in the case of the BioMarin agreement, all of our rights, title and interest in and to the intellectual property related to the RAP technology, would lose our rights to the Mesd technology, in the case of the Washington University agreement and would lose our rights to DR Cysteamine, in the case of UCSD. Under such circumstances, we would have no further right to use or exploit the patents, copyrights or trademarks in those respective technologies. If this happens, we will have to delay or terminate some or all of our research and development programs, our financial condition and operating results will be adversely affected, and we may have to cease our operations. If we lose our rights to the intellectual property related to the RAP technology purchased by us from BioMarin, our agreement with Roche regarding the evaluation of therapeutic delivery across the blood-brain barrier utilizing NeuroTrans™ would likely be terminated and any milestone or royalty payments from Roche to us would thereafter cease to accrue.
If we fail to compete successfully with respect to acquisitions, joint venture and other collaboration opportunities, we may be limited in our ability to develop our drug product candidates.
Our competitors compete with us to attract established biotechnology and pharmaceutical companies or organizations for acquisitions, joint ventures, licensing arrangements or other collaborations. Collaborations include licensing proprietary technology from, and other relationships with, academic research institutions. If our competitors successfully enter into partnering arrangements or license agreements with academic research institutions, we will then be precluded from pursuing those specific opportunities. Since each of these opportunities is unique, we may not be able to find a substitute. Other companies have already begun many drug development programs, which may target diseases that we are also targeting, and have already entered into partnering and licensing arrangements with academic research institutions, reducing the pool of available opportunities.
Universities and public and private research institutions also compete with us. While these organizations primarily have educational or basic research objectives, they may develop proprietary technology and acquire patents that we may need for the development of our drug product candidates. We will attempt to license this proprietary technology, if available. These licenses may not be available to us on acceptable terms, if at all. If we are unable to compete successfully with respect to acquisitions, joint venture and other collaboration opportunities, we may be limited in our ability to develop new products.
If we do not achieve our projected development goals in the time frames we announce and expect, the credibility of our management and our technology may be adversely affected and, as a result, the price of our common stock may decline.
For planning purposes, we estimate the timing of the accomplishment of various scientific, clinical, regulatory and other product development goals, which we sometimes refer to as milestones. These milestones may include the commencement or completion of scientific studies and clinical trials and the submission of regulatory

 

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filings. From time to time, we may publicly announce the expected timing of some of these milestones. All of these milestones will be based on a variety of assumptions. The actual timing of these milestones can vary dramatically compared to our estimates, in many cases for reasons beyond our control. If we do not meet these milestones as publicly announced, our stockholders may lose confidence in our ability to meet these milestones and, as a result, the price of our common stock may decline.
Our product development programs will require substantial additional future funding which could impact our operational and financial condition.
It will take several years before we are able to develop marketable drug product candidates, if at all. Our product development programs will require substantial additional capital to successfully complete them, arising from costs to:
   
conduct research, preclinical testing and human studies;
 
   
establish pilot scale and commercial scale manufacturing processes and facilities; and
 
   
establish and develop quality control, regulatory, marketing, sales, finance and administrative capabilities to support these programs.
Our future operating and capital needs will depend on many factors, including:
   
the pace of scientific progress in our research and development programs and the magnitude of these programs;
 
   
the scope and results of preclinical testing and human clinical trials;
 
   
our ability to obtain, and the time and costs involved in obtaining regulatory approvals;
 
   
our ability to prosecute, maintain, and enforce, and the time and costs involved in preparing, filing, prosecuting, maintaining and enforcing patent claims;
 
   
competing technological and market developments;
 
   
our ability to establish additional collaborations;
 
   
changes in our existing collaborations;
 
   
the cost of manufacturing scale-up; and
 
   
the effectiveness of our commercialization activities.
We base our outlook regarding the need for funds on many uncertain variables. Such uncertainties include the success of our research initiatives, regulatory approvals, the timing of events outside our direct control such as negotiations with potential strategic partners and other factors. Any of these uncertain events can significantly change our cash requirements as they determine such one-time events as the receipt or payment of major milestones and other payments.
Significant additional funds will be required to support our operations and if we are unable to obtain them on favorable terms, we may be required to cease or reduce further development or commercialization of our drug product programs, to sell some or all of our technology or assets, to merge with another entity or cease operations.

 

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If we fail to demonstrate efficacy in our preclinical studies and clinical trials our future business prospects, financial condition and operating results will be materially adversely affected.
The success of our development and commercialization efforts will be greatly dependent upon our ability to demonstrate drug product candidate efficacy in preclinical studies, as well as in clinical trials. Preclinical studies involve testing drug product candidates in appropriate non-human disease models to demonstrate efficacy and safety. Regulatory agencies evaluate these data carefully before they will approve clinical testing in humans. If certain preclinical data reveals potential safety issues or the results are inconsistent with an expectation of the drug product candidate’s efficacy in humans, the regulatory agencies may require additional more rigorous testing, before allowing human clinical trials. This additional testing will increase program expenses and extend timelines. We may decide to suspend further testing on our drug product candidates or technologies if, in the judgment of our management and advisors, the preclinical test results do not support further development.
Moreover, success in preclinical testing and early clinical trials does not ensure that later clinical trials will be successful, and we cannot be sure that the results of later clinical trials will replicate the results of prior clinical trials and preclinical testing. The clinical trial process may fail to demonstrate that our drug product candidates are safe for humans and effective for indicated uses. This failure would cause us to abandon a drug product candidate and may delay development of other drug product candidates. Any delay in, or termination of, our preclinical testing or clinical trials will delay the filing of our investigational new drug application, or IND, and new drug application, or NDA, as applicable, with the FDA and, ultimately, our ability to commercialize our drug product candidates and generate product revenues. In addition, some of our clinical trials will involve small patient populations. Because of the small sample size, the results of these early clinical trials may not be indicative of future results. Following successful preclinical testing, drug product candidates will need to be tested in a clinical development program to provide data on safety and efficacy prior to becoming eligible for product approval and licensure by regulatory agencies. From first clinical trial through product approval can take at least eight years, on average in the U.S.
If any of our future clinical development drug product candidates become the subject of problems, including those related to, among others:
   
efficacy or safety concerns with the drug product candidates, even if not justified;
 
   
unexpected side-effects;
 
   
regulatory proceedings subjecting the drug product candidates to potential recall;
 
   
publicity affecting doctor prescription or patient use of the drug product candidates;
 
   
pressure from competitive products; or
 
   
introduction of more effective treatments.
our ability to sustain our development programs will become critically compromised. For example, efficacy or safety concerns may arise, whether or not justified, that could lead to the suspension or termination of our clinical programs.
Each clinical phase is designed to test attributes of drug product candidates and problems that might result in the termination of the entire clinical plan can be revealed at any time throughout the overall clinical program. The failure to demonstrate efficacy in our clinical trials would have a material adverse effect on our future business prospects, financial condition and operating results.

 

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If we do not obtain the support of new, and maintain the support of existing, key scientific collaborators, it may be difficult to establish products using our technologies as a standard of care for various indications, which may limit our revenue growth and profitability and could have a material adverse effect on our business, prospects, financial condition and operating results.
We will need to establish relationships with additional leading scientists and research institutions. We believe that such relationships are pivotal to establishing products using our technologies as a standard of care for various indications. Although we have established a Medical and Scientific Advisory Board and research collaborations, there is no assurance that our Advisory Board members and our research collaborators will continue to work with us or that we will be able to attract additional research partners. If we are not able to maintain existing or establish new scientific relationships to assist in our research and development, we may not be able to successfully develop our drug product candidates.
If the manufacturers upon whom we rely fail to produce in the volumes and quality that we require on a timely basis, or to comply with stringent regulations applicable to pharmaceutical manufacturers, we may face delays in the development and commercialization of, or be unable to meet demand for, our products, if any, and may lose potential revenues.
We do not currently manufacture our drug product candidates, and does not currently plan to develop the capacity to do so. The manufacture of pharmaceutical products requires significant expertise and capital investment, including the development of advanced manufacturing techniques and process controls. Manufacturers of pharmaceutical products often encounter difficulties in production, particularly in scaling up initial production. These problems include difficulties with production costs and yields, quality control, including stability of the product candidate and quality assurance testing, shortages of qualified personnel, as well as compliance with strictly enforced federal, state and foreign regulations. Our third-party manufacturers and key suppliers may experience manufacturing difficulties due to resource constraints or as a result of labor disputes, unstable political environments at foreign facilities or financial difficulties. If these manufacturers or key suppliers were to encounter any of these difficulties, or otherwise fail to comply with their contractual obligations, our ability to timely launch any potential product candidate, if approved, would be jeopardized.
In addition, all manufacturers and suppliers of pharmaceutical products must comply with cGMP requirements enforced by the FDA, through its facilities inspection program. The FDA is likely to conduct inspections of our third party manufacturer and key supplier facilities as part of their review of any of our NDAs. If our third party manufacturers and key suppliers are not in compliance with cGMP requirements, it may result in a delay of approval, particularly if these sites are supplying single source ingredients required for the manufacture of any potential product. These cGMP requirements include quality control, quality assurance and the maintenance of records and documentation. Furthermore, regulatory qualifications of manufacturing facilities are applied on the basis of the specific facility being used to produce supplies. As a result, if a manufacturer for us shifts production from one facility to another, the new facility must go through a complete regulatory qualification and be approved by regulatory authorities prior to being used for commercial supply. Our manufacturers may be unable to comply with these cGMP requirements and with other FDA, state and foreign regulatory requirements. A failure to comply with these requirements may result in fines and civil penalties, suspension of production, suspension or delay in product approval, product seizure or recall, or withdrawal of product approval. If the safety of any quantities supplied is compromised due to a our third party manufacturer’s or key supplier’s failure to adhere to applicable laws or for other reasons, we may not be able to obtain regulatory approval for or successfully commercialize our products.
If we fail to obtain or maintain orphan drug exclusivity for some of our drug product candidates, our competitors may sell products to treat the same conditions and our revenues will be reduced.
As part of our business strategy, we intend to develop some drugs that may be eligible for FDA and European Union, or EU, orphan drug designation. Under the Orphan Drug Act, the FDA may designate a product as an orphan drug if it is a drug intended to treat a rare disease or condition, defined as a patient population of less than 200,000 in the U.S. The company that first obtains FDA approval for a designated orphan drug for a given rare disease receives marketing exclusivity for use of that drug for the stated condition for a period of seven years. Orphan drug exclusive marketing rights may be lost if the FDA later determines that the request for designation was materially defective or if the manufacturer is unable to assure sufficient quantity of the drug. Similar regulations are available in the EU with a 10-year period of market exclusivity.

 

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Because the extent and scope of patent protection for some of our drug products is particularly limited, orphan drug designation is especially important for our products that are eligible for orphan drug designation. For eligible drugs, we plan to rely on the exclusivity period under Orphan Drug Act designation to maintain a competitive position. If we do not obtain orphan drug exclusivity for our drug products that do not have patent protection, our competitors may then sell the same drug to treat the same condition and our revenues will be reduced.
Even though we have obtained orphan drug designation for DR Cysteamine for the potential treatment of nephropathic cystinosis, the potential treatment of HD and the potential treatment of Batten Disease and even if we obtain orphan drug designation for our future drug product candidates, due to the uncertainties associated with developing pharmaceutical products, we may not be the first to obtain marketing approval for any orphan indication. Further, even if we obtain orphan drug exclusivity for a product, that exclusivity may not effectively protect the product from competition because different drugs can be approved for the same condition. Even after an orphan drug is approved, the FDA can subsequently approve the same drug for the same condition if the FDA concludes that the later drug is safer, more effective or makes a major contribution to patient care. Orphan drug designation neither shortens the development time or regulatory review time of a drug, nor gives the drug any advantage in the regulatory review or approval process.
The fast-track designation for our drug product candidates, if obtained, may not actually lead to a faster review process and a delay in the review process or in the approval of our products will delay revenue from the sale of the products and will increase the capital necessary to fund these product development programs.
Although we have received Orphan Drug Designations from the FDA as described above, our drug product candidates may not receive an FDA fast-track designation or priority review. Without fast-track designation, submitting an NDA and getting through the regulatory process to gain marketing approval is a lengthy process. Under fast-track designation, the FDA may initiate review of sections of a fast-track drug’s NDA before the application is complete. However, the FDA’s time period goal for reviewing an application does not begin until the last section of the NDA is submitted. Additionally, the fast-track designation may be withdrawn by the FDA if the FDA believes that the designation is no longer supported by data emerging in the clinical trial process. Under the FDA policies, a drug candidate is eligible for priority review, or review within a six-month time frame from the time a complete NDA is accepted for filing, if the drug candidate provides a significant improvement compared to marketed drugs in the treatment, diagnosis or prevention of a disease. A fast-track designated drug candidate would ordinarily meet the FDA’s criteria for priority review. The fast-track designation for our drug product candidates, if obtained, may not actually lead to a faster review process and a delay in the review process or in the approval of our products will delay revenue from the sale of the products and will increase the capital necessary to fund these product development programs.
Because the target patient populations for some of our products are small, we must achieve significant market share and obtain high per-patient prices for our products to achieve profitability.
Our clinical development of DR Cysteamine targets diseases with small patient populations, including nephropathic cystinosis and HD. If we are successful in developing DR Cysteamine and receive regulatory approval to market DR Cysteamine for a disease with a small patient population, the per-patient prices at which we could sell DR Cysteamine for these indications are likely to be relatively high in order for us to recover our development costs and achieve profitability. We believe that we will need to market DR Cysteamine for these indications worldwide to achieve significant market penetration of this product.
We may not be able to market or generate sales of our products to the extent anticipated.
Assuming that we are successful in developing our drug product candidates and receive regulatory clearances to market our products, our ability to successfully penetrate the market and generate sales of those products may be limited by a number of factors, including the following:

 

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Certain of our competitors in the field have already received regulatory approvals for and have begun marketing similar products in the U.S., the EU, Japan and other territories, which may result in greater physician awareness of their products as compared to ours.
 
   
Information from our competitors or the academic community indicating that current products or new products are more effective than our future products could, if and when it is generated, impede our market penetration or decrease our future market share.
 
   
Physicians may be reluctant to switch from existing treatment methods, including traditional therapy agents, to our future products.
 
   
The price for our future products, as well as pricing decisions by our competitors, may have an effect on our revenues.
 
   
Our future revenues may diminish if third-party payers, including private healthcare coverage insurers and healthcare maintenance organizations, do not provide adequate coverage or reimbursement for our future products.
There are many difficult challenges associated with developing proteins that can be used to transport therapeutics across the blood-brain barrier.
Our RAP technology has a potential clinical use as a drug transporter through the blood-brain barrier. However, we do not know that our technology will work or work safely. Many groups and companies have attempted to solve the critical medical challenge of developing an efficient method of transporting therapeutic proteins from the blood stream into the brain. Unfortunately, these efforts to date have met with little success due in part to a lack of adequate understanding of the biology of the blood-brain barrier and to the enormous scientific complexity of the transport process itself.
In the research and development of our RAP technology, we will certainly face many of the same issues that have caused these earlier attempts to fail. It is possible that:
   
We or our collaborator/licensee will not be able to produce enough RAP drug product candidates for testing;
 
   
the pharmacokinetics, or where the drug distributes in the body, of our RAP drug product candidates will preclude sufficient binding to the targeted receptors on the blood-brain barrier;
 
   
the targeted receptors are not transported across the blood-brain barrier;
 
   
other features of the blood-brain barrier, apart from the cells, block access molecules to brain tissue after transport across the cells;
 
   
the targeted receptors are expressed on the blood-brain barrier at densities insufficient to allow adequate transport of our RAP drug product candidates into the brain;
 
   
targeting of the selected receptors induces harmful side-effects which prevent their use as drugs; or
 
   
that we or our collaborator/licensee’s RAP drug product candidates cause unacceptable side-effects.
Any of these conditions may preclude the use of RAP or RAP fusion compounds from potentially treating diseases affecting the brain.

 

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If our competitors succeed in developing products and technologies that are more effective than our own, or if scientific developments change our understanding of the potential scope and utility of our drug product candidates, then our technologies and future drug product candidates may be rendered less competitive.
We face significant competition from industry participants that are pursuing similar technologies that we are pursuing and are developing pharmaceutical products that are competitive with our drug product candidates. Nearly all of our industry competitors have greater capital resources, larger overall research and development staffs and facilities, and a longer history in drug discovery and development, obtaining regulatory approval and pharmaceutical product manufacturing and marketing than we do. With these additional resources, our competitors may be able to respond to the rapid and significant technological changes in the biotechnology and pharmaceutical industries faster than we can. Our future success will depend in large part on our ability to maintain a competitive position with respect to these technologies. Rapid technological development, as well as new scientific developments, may result in our compounds, drug product candidates or processes becoming obsolete before we can recover any of the expenses incurred to develop them. For example, changes in our understanding of the appropriate population of patients who should be treated with a targeted therapy like we are developing may limit the drug’s market potential if it is subsequently demonstrated that only certain subsets of patients should be treated with the targeted therapy.
Our reliance on third parties, such as collaborators, university laboratories, contract manufacturing organizations and contract or clinical research organizations, may result in delays in completing, or a failure to complete, preclinical testing or clinical trials if they fail to perform under our agreements with them.
In the course of product development, we may engage university laboratories, other biotechnology or companies or contract or clinical manufacturing organizations to manufacture drug material for us to be used in preclinical and clinical testing and collaborators and contract or clinical research organizations to conduct and manage preclinical studies and clinical trials. If we engage these organizations to help us with our preclinical and clinical programs, many important aspects of this process have been and will be out of our direct control. If any of these organizations we may engage in the future fail to perform their obligations under our agreements with them or fail to perform preclinical testing and/or clinical trials in a satisfactory manner, we may face delays in completing our clinical trials, as well as commercialization of any of our drug product candidates. Furthermore, any loss or delay in obtaining contracts with such entities may also delay the completion of our clinical trials, regulatory filings and the potential market approval of our drug product candidates.
Companies and universities that have licensed product candidates to us for research, clinical development and marketing are sophisticated competitors that could develop similar products to compete with our products which could reduce our future revenues.
Licensing our product candidates from other companies, universities or individuals does not always prevent them from developing non-identical but competitive products for their own commercial purposes, nor from pursuing patent protection in areas that are competitive with us. While we seek patent protection for all of our owned and licensed product candidates, our licensors or assignors who created these product candidates are experienced scientists and business people who may continue to do research and development and seek patent protection in the same areas that led to the discovery of the product candidates that they licensed or assigned to us. By virtue of the previous research that led to the discovery of the drugs or product candidates that they licensed or assigned to us, these companies, universities, or individuals may be able to develop and market competitive products in less time than might be required to develop a product with which they have no prior experience and may reduce our future revenues from such product candidates.
Uncertainties regarding healthcare reform and third-party reimbursement may impair our ability to raise capital, form collaborations and if any of our product candidates become marketable, sell such products.
The continuing efforts of governmental and third-party payers to contain or reduce the costs of healthcare through various means may harm our business. For example, in some foreign markets, the pricing or profitability of healthcare products is subject to government control. In the United States, there have been, and we expect there will continue to be, a number of federal and state proposals to implement similar government control. The

 

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implementation or even the announcement of any of these legislative or regulatory proposals or reforms could harm our business if any of our product candidates become marketable by reducing the prices we or our partners are able to charge for our products (if marketable), impeding our ability to achieve profitability, raise capital or form collaborations. In addition, the availability of reimbursement from third-party payers determines, in large part, the demand for healthcare products in the United States and elsewhere. Examples of such third-party payers are government and private insurance plans. Significant uncertainty exists as to the reimbursement status of newly approved healthcare products and third-party payers are increasingly challenging the prices charged for medical products and services. If we succeed in bringing one or more products to the market, reimbursement from third-party payers may not be available or may not be sufficient to allow us to sell such products on a competitive or profitable basis.
Any future product revenues could be reduced by imports from countries where our product candidates are available at lower prices.
Even if we obtain FDA approval to market our potential products in the United States, our sales in the United States may be reduced if our products are imported into the United States from lower priced markets, whether legally or illegally. In the United States, prices for pharmaceuticals are generally higher than in the bordering nations of Canada and Mexico. There have been proposals to legalize the import of pharmaceuticals from outside the United States. If such legislation were enacted, our potential future revenues could be reduced.
The use of any of our drug product candidates in clinical trials may expose us to liability claims.
The nature of our business exposes us to potential liability risks inherent in the testing, manufacturing and marketing of our drug product candidates. While we are in clinical stage testing, our drug product candidates could potentially harm people or allegedly harm people and we may be subject to costly and damaging product liability claims. Some of the patients who participate in clinical trials are already critically ill when they enter a trial. The waivers we obtain may not be enforceable and may not protect us from liability or the costs of product liability litigation. Although we currently carry a $3 million clinical product liability insurance policy, it may not be sufficient to cover future claims. We currently do not have any clinical or product liability claims or threats of claims filed against us.
Our future success depends, in part, on the continued service of our management team.
Our success is dependent in part upon the availability of our senior executive officers, including our Chief Executive Officer, Dr. Christopher M. Starr, our Chief Scientific Officer, Dr. Todd C. Zankel, our Chief Financial Officer, Kim R. Tsuchimoto, Ted Daley, the President of our clinical development subsidiary and Dr. Patrice P. Rioux, Chief Medical Officer of our clinical development subsidiary. The loss or unavailability to us of any of these individuals or key research and development personnel, and particularly if lost to competitors, could have a material adverse effect on our business, prospects, financial condition, and operating results. We have no key-man insurance on any of our employees.
There is intense competition for qualified scientists and managerial personnel from numerous pharmaceutical and biotechnology companies, as well as from academic and government organizations, research institutions and other entities. In addition, we will rely on consultants and advisors, including scientific and clinical advisors, to assist us in formulating our research and development strategy. All of our consultants and advisors will be employed by other employers or be self-employed, and will have commitments to or consulting or advisory contracts with other entities that may limit their availability to us. There is no assurance that we will be able to retain key employees and/or consultants. If key employees terminate their employment, or if insufficient numbers of employees are retained to maintain effective operations, our development activities might be adversely affected, management’s attention might be diverted from managing our operations to hiring suitable replacements, and our business might suffer. In addition, we might not be able to locate suitable replacements for any key employees that terminate, or that are terminated from, their employment with us and we may not be able to offer employment to potential replacements on reasonable terms, which could negatively impact our product candidate development timelines and may adversely affect our future revenues and financial condition.

 

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Our success depends on our ability to manage our growth.
If we are able to raise significant additional financing, we expect to continue to grow, which could strain our managerial, operational, financial and other resources. With the addition of our clinical-stage programs and with our plan to in-license and acquire additional clinical-stage product candidates, we will be required to retain experienced personnel in the regulatory, clinical and medical areas over the next several years. Also, as our preclinical pipeline diversifies through the acquisition or in-licensing of new molecules, we will need to hire additional scientists to supplement our existing scientific expertise over the next several years.
Our staff, financial resources, systems, procedures or controls may be inadequate to support our operations and our management may be unable to take advantage of future market opportunities or manage successfully our relationships with third parties if we are unable to adequately manage our anticipated growth and the integration of new personnel.
Our executive offices and laboratory facility are located near known earthquake fault zones, and the occurrence of an earthquake or other catastrophic disaster could cause damage to our facility and equipment, or that of our third-party manufacturers or single-source suppliers, which could materially impair our ability to continue our product development programs.
Our executive offices and laboratory facility are located in the San Francisco Bay Area near known earthquake fault zones and are vulnerable to significant damage from earthquakes. We and the third-party manufacturers with whom we contract and our single-source suppliers of raw materials are also vulnerable to damage from other types of disasters, including fires, floods, power loss and similar events. If any disaster were to occur, our ability to continue our product development programs, could be seriously, or potentially completely impaired. The insurance we maintain may not be adequate to cover our losses resulting from disasters or other business interruptions.
We will incur increased costs as a result of recently enacted and proposed changes in laws and regulations and our management will be required to devote substantial time to comply with such laws and regulations.
We face burdens relating to the recent trend toward stricter corporate governance and financial reporting standards. Legislation or regulations such as Section 404 of the Sarbanes-Oxley Act of 2002, or the Sarbanes-Oxley Act, as well as other rules implemented by the SEC and NASDAQ, follow the trend of imposing stricter corporate governance and financial reporting standards have led to an increase in the costs of compliance for companies similar to us, including increases in consulting, auditing and legal fees. New rules could make it more difficult or more costly for us to obtain certain types of insurance, including directors’ and officers’ liability insurance, and we may be forced to accept reduced policy limits and coverage or incur substantially higher costs to obtain the same or similar coverage. The impact of these events could also make it more difficult for us to attract and retain qualified persons to serve on our board of directors, our board committees or as executive officers. Failure to comply with these new laws and regulations may impact market perception of our financial condition and could materially harm our business. Additionally, it is unclear what additional laws or regulations may develop, and we cannot predict the ultimate impact of any future changes in law. Our management and other personnel will need to devote a substantial amount of time to these requirements.
In addition, the Sarbanes-Oxley Act requires, among other things, that we maintain effective internal controls for financial reporting and disclosure controls and procedures. In particular, we must perform system and process evaluation and testing of our internal controls over financial reporting to allow management to report on the effectiveness of our internal controls over financial reporting, as required by Section 404 of the Sarbanes-Oxley Act. Our compliance with Section 404 will require that we incur substantial accounting and related expense and expend significant management efforts. In the future, we may need to hire additional accounting and financial staff to satisfy the ongoing requirements of Section 404. Moreover, if we are not able to comply with the requirements of Section 404, or we or our independent registered public accounting firm identifies deficiencies in our internal controls over financial reporting that are deemed to be material weaknesses, the market price of our stock could decline and we could be subject to sanctions or investigations by NASDAQ, the SEC or other regulatory authorities.

 

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We may be required to suspend, repeat or terminate our clinical trials if they do not meet regulatory requirements, the results are negative or inconclusive, or if the trials are not well designed, which may result in significant negative repercussions on our business and financial condition.
Before regulatory approval for any potential product can be obtained, we must undertake extensive clinical testing on humans to demonstrate the tolerability and efficacy of the product, both on our own terms, and as compared to the other principal drugs on the market that have the same therapeutic indication. We cannot provide assurance that we will obtain authorization to permit product candidates that are already in the preclinical development phase to enter the human clinical testing phase. In addition, we cannot provide assurance that any authorized preclinical or clinical testing will be completed successfully within any specified time period by us, or without significant additional resources or expertise to those originally expected to be necessary. We cannot provide assurance that such testing will show potential products to be safe and efficacious or that any such product will be approved for a specific indication. Further, the results from preclinical studies and early clinical trials may not be indicative of the results that will be obtained in later-stage clinical trials. In addition, we or regulatory authorities may suspend clinical trials at any time on the basis that the participants are being exposed to unacceptable health risks.
Completion of clinical tests depends on, among other things, the number of patients available for testing, which is a function of many factors, including the number of patients with the relevant conditions, the nature of the clinical testing, the proximity of patients to clinical testing centers, the eligibility criteria for tests as well as competition with other clinical testing programs involving the same patient profile but different treatments. We will rely on third parties, such as contract research organizations and/or co-operative groups, to assist us in overseeing and monitoring clinical trials as well as to process the clinical results and manage test requests, which may result in delays or failure to complete trials, if the third parties fail to perform or to meet the applicable standards. A failure by us or such third parties to keep to the terms of a product program development for any particular product candidate or to complete the clinical trials for a product candidate in the envisaged time frame could have significant negative repercussions on our business and financial condition.
If we fail to establish and maintain collaborations or if our partners do not perform, we may be unable to develop and commercialize our product candidates, which may adversely affect our future revenues and financial condition.
We have entered into collaborative arrangements with third parties to develop and/or commercialize product candidates. Additional collaborations might be necessary in order for us to fund our research and development activities and third-party manufacturing arrangements, seek and obtain regulatory approvals and successfully commercialize existing and future product candidates. If we fail to maintain the existing collaborative arrangements held by us or fail to enter into additional collaborative arrangements, the number of product candidates from which we could receive future revenues would decline.
Our dependence on collaborative arrangements with third parties will subject us to a number of risks that could harm our ability to develop and commercialize products:
   
collaborative arrangements might not be on terms favorable to us;
 
   
disagreements with partners may result in delays in the development and marketing of products, termination of collaboration agreements or time consuming and expensive legal action;
 
   
we cannot control the amount and timing of resources partners devote to product candidates or their prioritization of product candidates, and partners may not allocate sufficient funds or resources to the development, promotion or marketing of our product candidates, or may not perform their obligations as expected;
 
   
partners may choose to develop, independently or with other companies, alternative products or treatments, including products or treatments which compete with ours;

 

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agreements with partners may expire or be terminated without renewal, or partners may breach collaboration agreements with us;
 
   
business combinations or significant changes in a partner’s business strategy might adversely affect that partner’s willingness or ability to complete their obligations to us; and
 
   
the terms and conditions of the relevant agreements may no longer be suitable.
We cannot assure you that we will be able to negotiate future collaboration agreements or that those currently in existence will make it possible for us to fulfill our objectives.
We may not complete our clinical trials in the time expected, which could delay or prevent the commercialization of our products, which may adversely affect our future revenues and financial condition.
Although for planning purposes we forecast the commencement and completion of clinical trials, the actual timing of these events can vary dramatically due to factors such as delays, scheduling conflicts with participating clinicians and clinical institutions and the rate of patient enrollment. Clinical trials involving our product candidates may not commence nor be completed as forecasted. In certain circumstances we will rely on academic institutions or clinical research organizations to conduct, supervise or monitor some or all aspects of clinical trials involving our product candidates. We will have less control over the timing and other aspects of these clinical trials than if we conducted them entirely on our own. These trials may not commence or be completed as we expect. They may not be conducted successfully. Failure to commence or complete, or delays in, any of our planned clinical trials could delay or prevent the commercialization of our product candidates and harm our business and may adversely affect our future revenues and financial condition.
If we fail to keep pace with rapid technological change in the biotechnology and pharmaceutical industries, our product candidates could become obsolete, which may adversely affect our future revenues and financial condition.
Biotechnology and related pharmaceutical technology have undergone and are subject to rapid and significant change. We expect that the technologies associated with biotechnology research and development will continue to develop rapidly. Our future will depend in large part on our ability to maintain a competitive position with respect to these technologies. Any compounds, products or processes that we develop may become obsolete before we recover any expenses incurred in connection with developing such products, which may adversely affect our future revenues and financial condition.
Risks Related to Our Intellectual Property
If we are unable to protect our proprietary technology, we may not be able to compete as effectively and our business and financial prospects may be harmed.
Where appropriate, we seek patent protection for certain aspects of our technology. Patent protection may not be available for some of the drug product candidates we are developing. If we must spend significant time and money protecting our patents, designing around patents held by others or licensing, potentially for large fees, patents or other proprietary rights held by others, our business and financial prospects may be harmed.
The patent positions of biopharmaceutical products are complex and uncertain.
We own or license patent applications related to certain of our drug product candidates. However, these patent applications do not ensure the protection of our intellectual property for a number of reasons, including the following:
   
We do not know whether our patent applications will result in issued patents. For example, we may not have developed a method for treating a disease before others developed similar methods.

 

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Competitors may interfere with our patent process in a variety of ways. Competitors may claim that they invented the claimed invention prior to us. Competitors may also claim that we are infringing on their patents and therefore cannot practice our technology as claimed under our patents, if issued. Competitors may also contest our patents, if issued, by showing the patent examiner that the invention was not original, was not novel or was obvious. In litigation, a competitor could claim that our patents, if issued, are not valid for a number of reasons. If a court agrees, we would lose that patent. As a company, we have no meaningful experience with competitors interfering with our patents or patent applications.
 
   
Enforcing patents is expensive and may absorb significant time of our management. Management would spend less time and resources on developing drug product candidates, which could increase our operating expenses and delay product programs.
 
   
Receipt of a patent may not provide much practical protection. If we receive a patent with a narrow scope, then it will be easier for competitors to design products that do not infringe on our patent.
 
   
In addition, competitors also seek patent protection for their technology. Due to the number of patents in our field of technology, we cannot be certain that we do not infringe on those patents or that we will not infringe on patents granted in the future. If a patent holder believes our drug product candidate infringes on its patent, the patent holder may sue us even if we have received patent protection for our technology. If someone else claims we infringe on their technology, we would face a number of issues, including the following:
   
Defending a lawsuit takes significant time and can be very expensive.
 
   
If a court decides that our drug product candidate infringes on the competitor’s patent, we may have to pay substantial damages for past infringement.
   
A court may prohibit us from selling or licensing the drug product candidate unless the patent holder licenses the patent to us. The patent holder is not required to grant us a license. If a license is available, we may have to pay substantial royalties or grant cross licenses to our patents.
 
   
Redesigning our drug product candidates so we do not infringe may not be possible or could require substantial funds and time.
It is also unclear whether our trade secrets are adequately protected. While we use reasonable efforts to protect our trade secrets, our employees or consultants may unintentionally or willfully disclose our information to competitors. Enforcing a claim that someone else illegally obtained and is using our trade secrets, like patent litigation, is expensive and time consuming, and the outcome is unpredictable. In addition, courts outside the U.S. are sometimes less willing to protect trade secrets. Our competitors may independently develop equivalent knowledge, methods and know-how.
We may also support and collaborate in research conducted by government organizations, hospitals, universities or other educational institutions. These research partners may be unwilling to grant us any exclusive rights to technology or products derived from these collaborations prior to entering into the relationship.
If we do not obtain required licenses or rights, we could encounter delays in our product development efforts while we attempt to design around other patents or even be prohibited from developing, manufacturing or selling drug product candidates requiring these licenses. There is also a risk that disputes may arise as to the rights to technology or drug product candidates developed in collaboration with other parties.

 

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If our agreements with employees, consultants, advisors and corporate partners fail to protect our intellectual property, proprietary information or trade secrets, it could have a significant adverse effect on us.
We have taken steps to protect our intellectual property and proprietary technology, by entering into confidentiality agreements and intellectual property assignment agreements with our employees, consultants, advisors and corporate partners. Such agreements may not be enforceable or may not provide meaningful protection for our trade secrets or other proprietary information in the event of unauthorized use or disclosure or other breaches of the agreements, and we may not be able to prevent such unauthorized disclosure. Monitoring unauthorized disclosure is difficult, and we do not know whether the steps we have taken to prevent such disclosure are, or will be, adequate. Furthermore, the laws of some foreign countries may not protect our intellectual property rights to the same extent as do the laws of the United States.
Risks Related to Our Common Stock
We are obligated to issue additional common stock based on our contractual obligations, if we meet certain triggering events, if at all. When we issue such additional common stock, this will result in dilution to common stockholders at the time such additional common stock is issued.
Future milestone payments, as more fully set forth under “Contractual Obligations with Thomas E. Daley (as assignee of the dissolved Convivia, Inc.)” and “Contractual Obligations with Former Encode Securityholders” discussed elsewhere in this Annual Report on Form 10-K, relating to our acquisition of the Convivia assets and merger with Encode will result in dilution. We may be required to make additional contingent payments of up to 664,400 shares of our common stock, in the aggregate, under the terms of our acquisition of Convivia assets and merger with Encode, based on milestones related to certain future marketing and development approvals obtained with respect to Convivia and Encode product candidates. The issuance of any of these shares will result in further dilution to our existing stockholders.
In May and June 2008, pursuant to a securities purchase agreement for a private placement of units, we issued 4,662,468 shares of our common stock and two-year warrants to purchase up to, in the aggregate, 2,331,234 shares of our common stock as well as five-year warrants to purchase up to, in the aggregate, 489,559 shares of our common stock to placement agents in such private placement. On April 29, 2009, in order to reflect current market prices, we notified the holders of warrants purchased in the May/June 2008 private placement that we were offering, in exchange for such warrants, new warrants to purchase our common stock at an exercise price of $1.29 per share, but only to the extent such exchange of the original warrants and exercise of the new warrants, including the delivery of the exercise price, occurred on or prior to July 17, 2009. The warrants that were not exchanged prior to or on July 17, 2009 retained their original exercise prices of $3.86 per share and original expiration date of May 21, 2010. We received approximately $2.6 million of proceeds from warrant exercises that resulted in the issuance of 2,031,670 shares of our common stock pursuant to the exchange described above. In August 2009, pursuant to a securities purchase agreement for a private placement of units, we issued 1,738,226 shares of our common stock and two-year warrants to purchase up to, in the aggregate, 869,113 shares of our common stock as well as a five-year warrant to purchase up to, in the aggregate, 129,733 shares of our common stock to a placement agent in such private placement. These stock issuances and other future issuances of common stock underlying unexpired and unexercised warrants have and will result in, significant dilution to our stockholders.
In connection with other collaborations, joint ventures, license agreements or future financings that we may enter into in the future, we may issue additional shares of common stock or other equity securities, and the value of the securities issued may be substantial and create additional dilution to our existing and future common stockholders.
Because we do not intend to pay any cash dividends on our common stock, investors seeking dividend income or liquidity should not purchase shares of our common stock.
We have not declared or paid any cash dividends on our common stock since our inception, and we do not anticipate paying any such cash dividends for the foreseeable future. Investors seeking dividend income or liquidity should not invest in our common stock.

 

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Our stock price is volatile and the trading in our common stock may be limited.
Our stock price has been and we anticipate that it will continue to be subject to significant fluctuations. Market prices for securities of early-stage pharmaceutical, biotechnology and other life sciences companies have historically been particularly volatile and trading in such securities has often been limited. Some of the factors that may cause the market price of our common stock to fluctuate include:
   
the results of our current and any future clinical trials of our drug candidates;
 
   
the results of ongoing preclinical studies and planned clinical trials of our preclinical drug candidates;
 
   
the entry into, or termination of, key agreements, including key strategic alliance agreements;
 
   
the results and timing of regulatory reviews relating to the approval of our drug candidates;
 
   
the initiation of, material developments in, or conclusion of litigation to enforce or defend any of our intellectual property rights;
 
   
failure of any of our drug candidates, if approved, to achieve commercial success;
 
   
general and industry-specific economic conditions that may affect our research and development expenditures;
 
   
the results of clinical trials conducted by others on drugs that would compete with our drug candidates;
 
   
issues in manufacturing our drug candidates or any approved products;
 
   
the loss of key employees;
 
   
the introduction of technological innovations or new commercial products by our competitors;
 
   
changes in estimates or recommendations by securities analysts, if any, who cover our common stock;
 
   
future sales of our common stock;
 
   
changes in the structure of health care payment systems; and
 
   
period-to-period fluctuations in our financial results.
Moreover, the stock markets in general have experienced substantial volatility that has often been unrelated to the operating performance of individual companies. These broad market fluctuations may also adversely affect the trading price of our common stock.
In the past, following periods of volatility in the market price of a company’s securities, stockholders have often instituted class action securities litigation against those companies. Such litigation can result in substantial costs and diversion of management attention and resources, which could significantly harm our profitability and reputation.
Our stock is a penny stock. Trading of our stock may be restricted by the SEC’s penny stock regulations and the FINRA’s sales practice requirements, which may limit a stockholder’s ability to buy and sell our stock.

 

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Our common stock is a penny stock. The SEC has adopted Rule 15g-9 which generally defines “penny stock” to be any equity security that has a market price less than $5.00 per share or an exercise price of less than $5.00 per share, subject to certain exceptions. Our securities are covered by the penny stock rules, which impose additional sales practice requirements on broker-dealers who sell to persons other than established customers and institutional accredited investors. The penny stock rules require a broker-dealer, prior to a transaction in a penny stock not otherwise exempt from the rules, to deliver a standardized risk disclosure document in a form prepared by the SEC which provides information about penny stocks and the nature and level of risks in the penny stock market. The broker-dealer also must provide the customer with current bid and offer quotations for the penny stock, the compensation of the broker-dealer and its salesperson in the transaction and monthly account statements showing the market value of each penny stock held in the customer’s account. The bid and offer quotations, and the broker-dealer and salesperson compensation information, must be given to the customer orally or in writing prior to effecting the transaction and must be given to the customer in writing before or with the customer’s confirmation. In addition, the penny stock rules require that prior to a transaction in a penny stock not otherwise exempt from these rules, the broker-dealer must make a special written determination that the penny stock is a suitable investment for the purchaser and receive the purchaser’s written agreement to the transaction. These disclosure requirements may have the effect of reducing the level of trading activity in the secondary market for the stock that is subject to these penny stock rules. Consequently, these penny stock rules may affect the ability of broker-dealers to trade our securities. We believe that the penny stock rules discourage investor interest in and limit the marketability of our common stock.
In addition to the “penny stock” rules promulgated by the SEC, the Financial Industry Regulatory Authority, or FINRA, has adopted rules that require that in recommending an investment to a customer, a broker-dealer must have reasonable grounds for believing that the investment is suitable for that customer. Prior to recommending speculative low priced securities to their non-institutional customers, broker-dealers must make reasonable efforts to obtain information about the customer’s financial status, tax status, investment objectives and other information. Under interpretations of these rules, the FINRA believes that there is a high probability that speculative low priced securities will not be suitable for at least some customers. The FINRA requirements make it more difficult for broker-dealers to recommend that their customers buy our common stock, which may limit your ability to buy and sell our stock.
Anti-takeover provisions in our stockholder rights plan and in our certificate of incorporation and bylaws may prevent or frustrate attempts by stockholders to change the board of directors or current management and could make a third-party acquisition of us difficult.
We are a party to a stockholder rights plan, also referred to as a poison pill, which is intended to deter a hostile takeover of us by making such proposed acquisition more expensive and less desirable to the potential acquirer. The stockholder rights plan and our certificate of incorporation and bylaws, as amended, contain provisions that may discourage, delay or prevent a merger, acquisition or other change in control that stockholders may consider favorable, including transactions in which stockholders might otherwise receive a premium for their shares. These provisions could limit the price that investors might be willing to pay in the future for shares of our common stock.
An adverse determination, if any, in the class action suit in which we are a defendant, or our inability to obtain or maintain directors’ and officers’ liability insurance, could have a material adverse affect on us.
A class action securities lawsuit was filed against TorreyPines, as described in the section titled, “Legal Proceedings” in this Annual Report on Form 10-K. We are defending against this action vigorously; however, we do not know what the outcome of the proceedings will be and, if we do not prevail, we may be required to pay substantial damages or settlement amounts. Furthermore, regardless of the outcome, we may incur significant defense costs, and the time and attention of our key management may be diverted from normal business operations. If we are ultimately required to pay significant defense costs, damages or settlement amounts, such payments could materially and adversely affect our operations and results. We have purchased liability insurance, however, if any costs or expenses associated with the litigation exceed the insurance coverage, we may be forced to bear some or all of these costs and expenses directly, which could be substantial and may have an adverse effect on our business, financial condition, results of operations and cash flows. In any event, publicity surrounding the lawsuits and/or any outcome unfavorable to us could adversely affect our reputation and stock price. The uncertainty associated with substantial unresolved lawsuits could harm our business, financial condition and reputation.

 

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We have certain obligations to indemnify our officers and directors and to advance expenses to such officers and directors. Although we have purchased liability insurance for our directors and officers, if our insurance carriers should deny coverage, or if the indemnification costs exceed the insurance coverage, we may be forced to bear some or all of these indemnification costs directly, which could be substantial and may have an adverse effect on our business, financial condition, results of operations and cash flows. If the cost of the liability insurance increases significantly, or if this insurance becomes unavailable, we may not be able to maintain or increase our levels of insurance coverage for our directors and officers, which could make it difficult to attract or retain qualified directors and officers.
ITEM 1B: UNRESOLVED STAFF COMMENTS
None.
ITEM 2: PROPERTIES
Effective April 1, 2006, we entered into a three year lease for 2,892 square feet of combined laboratory and office space with an additional three year option. Our original monthly base rent was $5,206. Effective April 1, 2007, we leased an additional 3,210 square feet in order to expand our office space and our base rent increased to $9,764 per month. In June 2008, our rent increased to $10,215 reflecting a Consumer Price Index increase of 3% plus an increase in operating costs for the period from April 1, 2008 to March 31, 2009. In September 2008, we executed a lease addendum replacing the one three-year extension with two two-year extensions commencing on April 1, 2009 and renegotiated the first two-year extension base rent to $10,068 with an adjustment after the first year for CPI between 3% (minimum) and 5% (maximum). The facility is located in an industrial park at 9 Commercial Blvd, Suite 200, Novato, California 94949. We also store cell line back ups at an off site cell bank, a commercial facility specifically licensed for such purpose. Our current facility is expected to be adequate for the foreseeable future.
ITEM 3: LEGAL PROCEEDINGS
Several lawsuits were filed against TorreyPines in February 2005 in the U.S. District Court for the Southern District of New York asserting claims under Sections 10(b) and 20(a) of the Exchange Act and Rule 10b-5 thereunder on behalf of a class of purchasers of TorreyPines’ common stock during the period from June 26, 2003, through and including February 4, 2005, referred to as the class period. Dr. Marvin S. Hausman, M.D., a former director and TorreyPines’ former Chief Executive Officer, and Dr. Gosse B. Bruinsma, M.D., also a former director and TorreyPines’ former Chief Executive Officer, were also named as defendants in the lawsuits. These actions were consolidated into a single class action lawsuit in January 2006. On April 10, 2006, the class action plaintiffs filed an amended consolidated complaint. TorreyPines filed its answer to that complaint on May 26, 2006. TorreyPines’ motion to dismiss the consolidated amended complaint was filed on May 26, 2006 and was submitted to the court for a decision in September 2006. On March 31, 2009 the U.S. District Court for the Southern District of New York dismissed the proceedings. On April 24, 2009, an appeal was filed with the United States Court of Appeals for the Second Circuit by the class action plaintiffs. Our response to such appeal was filed on October 23, 2009. We do not anticipate that this claim, if successful, would burden the Company with any additional liability above and beyond the insurance coverage provided under the insurance policy that we currently maintain.
Other than as described above, we know of no material, active or pending legal proceedings against us, nor are we involved as a plaintiff in any material proceedings or pending litigation. There are no proceedings in which any of our directors, officers or affiliates, or any registered or beneficial stockholders are an adverse party or have a material interest adverse to us.
ITEM 4: SUBMISSION OF MATTERS TO A VOTE OF SECURITY HOLDERS
The following items were submitted for stockholder vote at our annual meeting, held on September 28, 2009:

 

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The total number of shares voted in person or by proxy was 11,403,587 out of 17,857,555 (64%) shares issued and outstanding on the record date of August 27, 2009.
The following directors were nominated to our Board of Directors:
                 
    Number of shares     Number of shares  
Name of nominee   voted FOR     WITHHELD  
Christopher M. Starr, Ph.D.
    11,904,766       2,741  
Raymond W. Anderson
    11,401,363       2,223  
Erich Sager
    11,401,177       2,410  
Richard L. Franklin, M.D., Ph.D.
    11,401,491       2,096  
A proposal to adopt that certain Agreement and Plan of Merger and Reorganization dates as of July 27, 2009, by and among TorreyPines Therapeutics, Inc., ECP Acquisition, Inc. and Raptor Pharmaceuticals Corp.
         
Number of shares voted FOR:
    11,008,116  
Number of shares voted AGAINST:
    9,884  
Number of shares ABSTAINING:
    303  
A proposal to ratify the appointment of Burr, Pilger & Mayer, LLP as our independent registered public accounting firm for the fiscal year ended August 31, 2009.
         
Number of shares voted FOR:
    11,390,756  
Number of shares voted AGAINST:
    8,810  
Number of shares ABSTAINING:
    4,021  
A proposal to approve any motion to adjourn Raptor Pharmaceuticals Corp. annual meeting, if necessary, to solicit additional proxies if there are not sufficient votes in favor of adoption of the Agreement of Plan of Merger and Reorganization.
         
Number of shares voted FOR:
    11,387,437  
Number of shares voted AGAINST:
    16,149  
Number of shares ABSTAINING:
    0  
PART II
ITEM 5: MARKET FOR REGISTRANT’S COMMON EQUITY AND RELATED STOCKHOLDER MATTERS AND ISSUER PURCHASES OF EQUITY SECURITIES
Market Information
In connection with the closing of the merger with TorreyPines, the common stock of the combined company commenced trading on the NASDAQ Capital Market on September 30, 2009, under the ticker symbol “RPTPD” with 18,822,162 shares outstanding. Effective October 27, 2009, our ticker symbol changed to “RPTP”.
On September 29, 2009, the last reported sale price on the FINRA OTC Bulletin Board for our common stock was $2.79 per share. We have never paid any cash dividends on our common stock and we do not anticipate paying cash dividends in the foreseeable future. There is no public trading market for our warrants.

 

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Up to September 29, 2009, our common stock was quoted on the FINRA OTC Bulletin Board under the symbol “RPTP”. Prior to June 8, 2006, our common stock had not traded in the public market. The following table sets forth the quarterly high and low bid prices for our common stock for the period from June 8, 2006 through our quarter ended August 31, 2009, as reported by the FINRA OTC Bulletin Board, which reflects inter-dealer quotations, without retail mark-up, mark-down or commission and may not represent actual transactions.
                 
Period   High     Low  
 
               
2008 Fiscal Quarters:
               
September 1, 2007 — November 30, 2007
  $ 2.83     $ 2.02  
December 1, 2007 — February 29, 2008
  $ 2.66     $ 1.63  
March 1, 2008 — May 31, 2008
  $ 2,87     $ 2.02  
June 1, 2008 — August 31, 2008
  $ 2,49     $ 1,63  
 
               
2009 Fiscal Quarters:
               
September 1, 2008— November 30, 2008
  $ 2.06     $ 0.51  
December 1, 2008— February 29, 2009
  $ 1.67     $ 0.43  
March 1, 2009— May 31, 2009
  $ 1.63     $ 0.64  
June 1, 2009— August 31, 2009
  $ 2.23     $ 1.24  
Holders of Record
Immediately prior to the Merger, as of September 29, 2009, there were 49 holders of record of 17,857,555 outstanding shares of our common stock, excluding shares held in book-entry form through The Depository Trust Company, and we estimate that the number of beneficial owners of shares of our common stock was approximately 1,000 as of such date. Additionally, on such date, options, held by twenty-five persons to acquire up to, in the aggregate, 989,213 shares, and warrants held by fifteen persons to acquire up to, in the aggregate, 2,011,522 shares, of our common stock, were outstanding.
As of October 21, 2009, there were 340 holders of record of 18,829,842 outstanding shares of our common stock, excluding shares held in book-entry form through The Depository Trust Company, and we estimate that the number of beneficial owners of shares of our common stock was approximately 4,800 as of such date. Additionally, on such date, options, held by twenty-five persons to acquire up to, in the aggregate, 989,213 shares, and warrants held by fifteen persons to acquire up to, in the aggregate, 2,011,522 shares, of our common stock, were outstanding.
Dividends
We have never declared or paid cash dividends on our common stock and do not anticipate paying any cash dividends on our shares of common stock in the foreseeable future. We expect to retain future earnings, if any, for use in our development activities and the operation of our business. The payment of any future cash dividends will be subject to the discretion of our Board of Directors and will depend, among other things, upon our results of operations, financial condition, cash requirements, prospects and other factors that our Board of Directors may deem relevant. Additionally, our ability to pay future cash dividends may be restricted by the terms of any future financing.
Purchase of Equity Securities and Affiliated Purchasers
We have not repurchased any shares of our common stock since inception. For a discussion regarding our unregistered equity issuances during our fiscal year ended August 31, 2009, please refer to the 2009 Warrant Exchange and the 2009 Closing described under the heading, “2008 and 2009 Private Placements” under Item 1 to this Annual Report on Form 10-K which such discussion is incorporated herein.

 

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ITEM 6: SELECTED FINANCIAL DATA
The following table shows selected historical consolidated financial and operating information for, and as of the end of, each of the periods indicated and should be read in conjunction with sections titled “Management’s Discussion and Analysis of Financial Condition and Results of Operation” and “Business” and our consolidated financial statements and the corresponding notes to those consolidated financial statements included elsewhere in this Annual Report. The following tables set forth our consolidated balance sheet data as of August 31, 2009 and 2008, and our consolidated statements of operations data for the years ended August 31, 2009 and 2008 and for the cumulative period from September 8, 2005 (inception) to August 31, 2009 which were audited by Burr, Pilger & Mayer, LLP, an independent registered public accounting firm.
                         
                    For the period  
                    from  
                    September 8,  
                    2005  
                    (inception) to  
    For the year ended     For the year ended     August 31,  
    August 31, 2009     August 31, 2008     2009  
Revenues:
  $       $     $  
 
                       
Operating expenses:
                       
 
                       
General and administrative
    2,687,993       2,229,140       6,956,240  
 
                       
Research and development
    6,570,119       5,558,871       14,874,284  
In-process research and development
          240,625       240,625  
 
                 
 
                       
Total operating expenses
    9,258,112       8,028,636       22,071,149  
 
                 
 
                       
Loss from operations
    (9,258,112 )     (8,028,636 )     (22,071,149 )
 
                       
Interest income
    36,744       77,871       301,903  
Interest expense
    (2,526 )     (103,198 )     (109,937 )
 
                 
Net loss
  $ (9,223,894 )   $ (8,053,963 )   $ (21,879,183 )
 
                 
 
                       
Net loss per share:
                       
Basic and diluted
  $ (0.64 )   $ (0.81 )        
 
                   
 
                       
Weighted average shares outstanding used to compute:
                       
Basic and diluted
    14,440,254       9,893.612          
 
                   
                 
    August 31,  
Balance Sheet Data:   2009     2008  
Cash and cash equivalents
  $ 3,701,787     $ 7,546,912  
Working capital
    2,739,904       6,659,225  
Total assets
    6,578,574       10,620,770  
Long-term portion of capital lease obligations
    6,676        
Total liabilities
    1,075,613       1,003,280  
Total stockholders’ equity
    5,502,961       9,617,490  

 

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ITEM 7: MANAGEMENT’S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS
PLAN OF OPERATION
Overview
You should read the following discussion in conjunction with our consolidated financial statements as of August 31, 2009, and the notes to such consolidated financial statements included elsewhere in this Annual Report on Form 10-K. This “Management’s Discussion and Analysis of Financial Condition and Results of Operations” section contains forward-looking statements. Please see “Forward-Looking Statements” for a discussion of the uncertainties, risks and assumptions associated with these statements. Our actual results and the timing of certain events could differ materially from those anticipated in these forward-looking statements as a result of certain factors, including those discussed below and elsewhere in this Annual Report on Form 10-K, particularly under the heading “Risk Factors.”
Unless otherwise mentioned or unless the context requires otherwise (e.g., our consolidated financial statements as of August 31, 2009, and the notes to such consolidated financial statements included elsewhere in this Annual Report on Form 10-K, or a reference to an event or circumstance that occurred prior to the effective time of the Merger on September 29, 2009), all references in this Annual Report on Form 10-K to “we,” “us,” “our,” the “Company,” “Raptor” and similar references refer to the public company formerly known as TorreyPines Therapeutics, Inc. and now known as Raptor Pharmaceutical Corp., including its wholly-owned direct and indirect subsidiaries (which includes Raptor Pharmaceuticals Corp., TPTX, Inc., Raptor Discoveries Inc. (f/k/a Raptor Pharmaceutical Inc.) and Raptor Therapeutics Inc. (f/k/a Bennu Pharmaceuticals Inc.)), following the name change and completion of the Merger.
Plan of Operation and Overview
We believe that we are building a balanced pipeline of drug candidates that may expand the reach and benefit of existing therapeutics. Our product portfolio includes both candidates from our proprietary drug targeting platforms and in-licensed and acquired product candidates.
Our current pipeline includes three clinical development programs which we are actively developing. We also have three other clinical-stage product candidates, which we are seeking business development partners but are not actively developing, and we have four preclinical product candidates we are developing, three of which are based upon our proprietary drug-targeting platforms.
Clinical Development Programs
Our three active clinical development programs are based on an existing therapeutic that we are reformulating for potential improvement in safety and/or efficacy and for application in new disease indications. These clinical development programs include the following:
   
DR Cysteamine for the potential treatment of nephropathic cystinosis, or cystinosis, a rare genetic disorder;
 
   
DR Cysteamine for the potential treatment of non-alcoholic steatohepatitis, or NASH, a metabolic disorder of the liver; and
 
   
DR Cysteamine for the potential treatment of Huntington’s Disease, or HD.

 

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Other Clinical-Stage Product Candidates
We have three clinical-stage product candidates for which we are seeking partners:
   
Convivia™ for the potential management of acetaldehyde toxicity due to alcohol consumption by individuals with aldehyde dehydrogenase, or ALDH2 deficiency, an inherited metabolic disorder; and
 
   
Tezampanel and NGX426, non-opioids for the potential treatment of: migraine, acute pain, and chronic pain.
Preclinical Product Candidates
Our preclinical platforms consist of targeted therapeutics, which we are developing for the potential treatment of multiple indications, including liver diseases, neurodegenerative diseases and breast cancer:
Our receptor-associated protein, or RAP, platform consists of: HepTide™ for the potential treatment of primary liver cancer and hepatitis C; and NeuroTrans™ to potentially deliver therapeutics across the blood-brain barrier for treatment of a variety of neurological diseases.
Our mesoderm development protein, or Mesd, platform consists of WntTide™ for the potential treatment of breast cancer.
We are also examining our glutamate receptor antagonists, tezampanel and NGX426, for the potential treatment of thrombotic disorder.
Future Activities
Over the next 12 months, we plan to conduct research and development activities based upon our DR Cysteamine clinical programs and continued development of our preclinical product candidates. We also plan to actively seek business development partners for our ConviviaTM product candidate and Tezampanel and NGX426. We may also develop future in-licensed technologies and acquired technologies. A brief summary of our primary objectives in the next 12 months for our research and development activities is provided below. Our plans for research and development activities over the next 12 months can only be implemented if we are successful in raising significant funds during this period. In addition, there can be no assurances that our research and development activities will be successful. If we do not make important progress towards achieving at least one of our major clinical objectives, this could adversely impact our ability to raise significant additional funds, which could adversely impact our ability to continue as a going concern.
Clinical Development Programs
We develop clinical-stage drug product candidates which are: internally discovered therapeutic candidates based on our novel drug delivery platforms and in-licensed or purchased clinical-stage products which may be new chemical entities in mid-to-late stage clinical development, currently approved drugs with potential efficacy in additional indications, and treatments that we could repurpose or reformulate as potentially more effective or convenient treatments for a drug’s currently approved indications.
Development of DR Cysteamine for the Potential Treatment of Nephropathic Cystinosis or Cystinosis
Our DR Cysteamine product candidate is a proprietary delayed-release, enteric-coated microbead formulation of cysteamine bitartrate contained in a gelatin capsule. We are investigating DR Cysteamine for the potential treatment of cystinosis.
Immediate-release cysteamine bitartrate, a cystine-depleting agent, is currently the only U.S. Food and Drug Administration, or FDA, and the European Medicines Agency, or EMEA, approved drug to treat cystinosis, a rare genetic disease. Immediate-release cysteamine is effective at preventing or delaying kidney failure and other serious health problems in cystinosis patients. However, patient compliance is challenging due to the requirement

 

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for frequent dosing and gastrointestinal side effects. Our DR Cysteamine for the potential treatment of cystinosis is designed to mitigate some of these difficulties. It is expected to be dosed twice daily, compared to the current every-six-hour dosing schedule. In addition, DR Cysteamine is designed to pass through the stomach and deliver the drug directly to the small intestine, where it is more easily absorbed into the bloodstream and may result in fewer gastrointestinal side effects.
The FDA granted orphan drug designation for: DR Cysteamine for the treatment of cystinosis in 2006.
In June 2009, we commenced our Phase IIb clinical trial of DR Cysteamine in cystinosis, in which we have enrolled nine cystinosis patients with histories of compliance using the currently available immediate-release form of cysteamine bitartrate. The clinical trial, which is being conducted at the University of California at San Diego, or UCSD, is evaluating safety, tolerability, pharmacokinetics and pharmacodynamics of a single dose of DR Cysteamine in patients. Release of data from the study is expected in the fourth calendar quarter of 2009. We plan to follow the Phase IIb clinical study with a pivotal, Phase III clinical study in cystinosis patients anticipated to commence in early 2010. While we plan to commercialize DR Cysteamine in the U.S. by ourselves, we are actively reviewing potential development partners for DR Cysteamine for markets outside of the U.S. with companies that have experience in clinical development and commercialization of orphan drugs in various ex-U.S. countries.
Development of DR Cysteamine for the Potential Treatment of Non-Alcoholic Steatohepatitis or NASH
In October 2008, we commenced a clinical trial in collaboration with UCSD to investigate a prototype formulation of DR Cysteamine for the treatment of NASH in juvenile patients. In October 2009, we announced positive findings from the completed treatment phase of this open-label Phase IIa clinical trial. At the completion of the initial six-month treatment phase, the study achieved the primary endpoint: mean blood levels of alanine aminotransferase, or ALT, a common biomarker for NASH, were reduced by over 50%. Additionally, over half of the study participants had achieved normalized ALT levels by the end of the treatment phase.
There are no currently approved drug therapies for NASH, and patients are limited to lifestyle changes such as diet, exercise and weight reduction to manage the disease. DR Cysteamine represents an important potential treatment option for patients with NASH. Although NASH is most common in insulin-resistant obese adults with diabetes and abnormal serum lipid profiles, its prevalence is increasing among juveniles as obesity rates rise within this patient population. Although most patients are asymptomatic and feel healthy, NASH causes decreased liver function and can lead to cirrhosis, liver failure and end-stage liver disease.
The NASH trial entails six months of treatment followed by a six-month post-treatment monitoring period. Eligible patients with baseline ALT and aspartate aminotransferase or AST measurements at least twice that of normal levels were enrolled to receive twice-daily, escalating oral doses of up to 1,000 mg of DR Cysteamine. The trial currently has enrolled eleven NASH patients between 11-18 years old. No major adverse events were reported during the six-month treatment phase. Trial subjects continue to be monitored during the six-month post-treatment period currently underway. Full results are being submitted for peer review by UCSD and us, and are expected to be presented in 2010.
Development of DR Cysteamine for the Potential Treatment of Huntington’s Disease or HD
Huntington’s Disease, or HD, is a fatal, inherited degenerative neurological disease affecting about 30,000 people in the U.S. and a comparable number of people in Europe. We are not aware of any treatment for HD other than therapeutics that minimize symptoms such as the uncontrollable movements and mood swings resulting from HD. We are collaborating with a French institution, CHU d’ Angers, on a Phase II clinical trial investigating DR Cysteamine in HD patients, anticipated to begin in early 2010. We are providing the clinical trial materials for the study, which is sponsored by CHU d’ Angers and funded in part by a grant from the French government. We were granted Orphan Drug Designation in the U.S. by the FDA for cysteamine as a potential treatment for HD in 2008.

 

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Other Clinical-Stage Product Candidates
We have three clinical-stage product candidates for which we are seeking partners.
ConviviaTM for Liver Aldehyde Dehydrogenase Deficiency
Convivia™ is our proprietary oral formulation of 4-methylpyrazole, or 4-MP, intended for the potential treatment of acetaldehyde toxicity resulting from alcohol consumption in individuals with ALDH2 deficiency, which is an inherited disorder of the body’s ability to breakdown ethanol, commonly referred to as alcohol intolerance. 4-MP is presently marketed in the U.S. and E.U. in an intravenous form as an anti-toxin. ConviviaTM is designed to lower systemic levels of acetaldehyde (a carcinogen) and reduce symptoms, such as tachycardia and flushing, associated with alcohol consumption by ALDH2-deficient individuals. ConviviaTM is a capsule designed to be taken approximately 30 minutes prior to consuming an alcoholic beverage.
In 2008, we completed a Phase IIa dose escalation clinical trial of oral 4-MP with ethanol in ALDH2 deficient patients. The study results demonstrated that the active ingredient in ConviviaTM significantly reduced heart palpitations (tachycardia), which are commonly experienced by ALDH2 deficient people who drink, at all dose levels tested. The study also found that the 4-MP significantly reduced peak acetaldehyde levels and total acetaldehyde exposure in a subset of the study participants who possess specific genetic variants of the liver ADH and ALDH2 enzymes. We believe that this subset represents approximately one-third of East Asian populations. We are actively seeking corporate partnerships with pharmaceutical companies in selected Asian countries to continue clinical development of ConviviaTM in those countries.
Tezampanel and NGX426 for the Potential Treatment of Migraine and Pain
Tezampanel and NGX426, the oral prodrug of tezampanel, are first-in-class compounds that may represent novel treatments for both pain and non-pain indications. Tezampanel and NGX426 are receptor antagonists that target and inhibit a specific group of receptors—the AMPA and kainate glutamate receptors—found in the brain and other tissues. While normal glutamate production is essential, excess glutamate production, either through injury or disease, has been implicated in a number of diseases and disorders. Published data show that during a migraine, increased levels of glutamate activate AMPA and kainate receptors, result in the transmission of pain and, in many patients, the development of increased pain sensitivity.
By acting at both the AMPA and kainate receptor sites to competitively block the binding of glutamate, tezampanel and NGX426 have the potential to treat a number of diseases and disorders. These include chronic pain, such as migraine and neuropathic pain, muscle spasticity and a condition known as central sensitization, a persistent and acute sensitivity to pain.
Results of a Phase IIb clinical trial of tezampanel were released in October 2007. In the trial, a single dose of tezampanel given by injection was statistically significant compared to placebo in treating acute migraine headache. This was the sixth Phase II trial in which tezampanel has been shown to have analgesic activity. Based on a review of the Phase II data, the FDA previously agreed that tezampanel may move forward into a Phase III program for in acute migraine.
In December 2008, results of NGX426 in a human experimental model of cutaneous pain, hyperalgesia and allodynia demonstrated a statistically significant reduction in spontaneous pain, hyperalgesia and allodynia compared to placebo following injections of capsaicin (i.e., chili oil) under the skin. In February 2009, results from a Phase 1 multiple dose trial of NGX426 showed that the compound is safe and well-tolerated in healthy male and female subjects when dosed once daily for five consecutive days.
We are currently seeking program funding, development collaborations or out-licensing partners for the migraine and pain programs.

 

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Preclinical Product Candidates
We are also developing a drug-targeting platform based on the proprietary use of RAP and Mesd. We believe that these proteins may have therapeutic applications in cancer, infectious diseases and neurodegenerative diseases, among others.
These applications are based on the assumption that our targeting molecules can be engineered to bind to a selective subset of receptors with restricted tissue distribution under particular conditions of administration. We believe these selective tissue distributions can be used to deliver drugs to the liver or to other tissues, such as the brain.
In addition to selectively transporting drugs to specific tissues, selective receptor binding constitutes a means by which receptor function might be specifically controlled, either through modulating its binding capacity or its prevalence on the cell surface. Mesd is being engineered for this latter application.
HepTideTM for Hepatocellular Carcinoma and Hepatitis C
Drugs currently used to treat primary liver cancer are often toxic to other organs and tissues. We believe that the pharmacokinetic behavior of RAP (i.e., the determination of the fate or disposition of RAP once administered to a living organism) may diminish the non-target toxicity and increase the on-target efficacy of attached therapeutics.
In preclinical studies of our radio-labeled HepTideTM (a variant of RAP), HepTideTM , our proprietary drug-targeting peptide was shown to distribute predominately to the liver. Radio-labeled HepTideTM which was tested in a preclinical research model of HCC, at the National Research Council in Winnipeg, Manitoba, Canada, showed 4.5 times more delivery to the liver than the radio-labeled control. Another study of radio-labeled HepTideTM in a non-HCC preclinical model, showed 7 times more delivery to the liver than the radio-labeled control, with significantly smaller amounts of radio-labeled HepTideTM delivery to other tissues and organs
HCC is caused by the malignant transformation of hepatocytes, epithelial cells lining the vascular sinusoids of the liver, or their progenitors. HepTideTM has shown to bind to lipoprotein receptor-related protein, or LRP1, receptors on hepatocytes. We believe that the pharmacokinetics and systemic toxicity of a number of potent anti-tumor agents may be controlled in this way.
There are additional factors that favor the suitability of RAP as an HCC targeting agent:
   
RAP is captured by hepatocytes with efficiency, primarily on first-pass.
 
   
Late-stage HCC is perfused exclusively by the hepatic artery, while the majority of the liver is primarily perfused through the portal vein.
Studies have shown that the RAP receptor, LRP1, is well expressed on human HCC and under-expressed on non-cancerous, but otherwise diseased, hepatocytes. Also, LRP1 expression is maintained on metastasized HCC. These factors will favor delivery of RAP peptide-conjugated anti-tumor agents to tumor cells, whether in the liver or at metastasized sites.
We are evaluating conjugates between HepTideTM and a chemotherapeutic for testing in vitro and in appropriate preclinical models for the potential treatment of HCC.
We are also evaluating conjugates between HepTideTM and an antiviral agent for testing in vitro and in appropriate preclinical models for the potential treatment of hepatitis C.

 

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NeuroTransTM for the Potential Treatment of Diseases Affecting the Brain
Hundreds of known genetic and neurodegenerative diseases affect the brain. Drugs often have difficulty reaching these disease-affected areas because the brain has evolved a protective barrier, commonly referred to as the blood-brain barrier.
Part of the solution to the medical problem of neurodegenerative diseases is the creation of effective brain targeting and delivery technologies. One of the most obvious ways of delivering therapeutics to the brain is via the brain’s extensive vascular network. Treating these diseases by delivering therapeutics into the brain in a minimally invasive way, including through a natural receptor mediated transport mechanism called transcytosis, is a vision shared by many researchers and clinicians in the neuroscience and neuromedical fields.
NeuroTrans™ is our proprietary RAP-based technology program to research the delivery of therapeutics across the blood-brain barrier. We believe our NeuroTrans™ platform may provide therapies that will be safer, less intrusive and more effective than current approaches in treating a wide variety of brain disorders.
In preclinical studies, NeuroTrans™ has been conjugated to a variety of protein drugs, including enzymes and growth factors, without interfering with the function of either fusion partner. Studies indicate that radio-labeled NeuroTrans™ may be transcytosed across the blood-brain barrier and, that fusions between NeuroTrans™ and therapeutic proteins may be manufactured economically. Experiments conducted in collaboration with Stanford University in 2008 support the NeuroTrans™ peptide’s ability to enhance the transport of cargo molecules into the cells that line the blood-brain barrier.
In June 2009, we entered into a collaboration and licensing agreement with F. Hoffman — La Roche Ltd. and Hoffman—La Roche Inc., or Roche, to evaluate therapeutic delivery across the blood-brain barrier utilizing NeuroTrans™. Under terms of the agreement, Roche has funded studies of select molecules attached to NeuroTrans™. The agreement provides Roche with an exclusive worldwide license to NeuroTrans™ for use in the delivery of diagnostic and therapeutic molecules across the blood-brain barrier. Roche’s and our scientists will actively collaborate on the project. We have received an initial upfront payment for the collaboration to cover our portion of the initial studies, and may earn development milestone payments and royalties in exchange for the licensing of NeuroTrans™ to Roche.
WntTideTM for the Potential Treatment of Cancer
Human Mesd is a natural inhibitor of the receptor LRP6. LRP6 has recently been shown to play a role in the progression of some breast tumors. Studies in the laboratory of Professor Guojun Bu, one of our scientific advisors, at the Washington University in St. Louis Medical School have demonstrated the potential of Mesd and related peptides to target these tumors. These molecules and applications are licensed to us from Washington University.
WntTide™ is our proprietary, Mesd-based peptide that we are developing as a potential therapeutic to inhibit the growth and metastasis of tumors over-expressing LRP5 or LRP6. We have licensed the use of Mesd from Washington University in St. Louis for the potential treatment of cancer and bone density disorders.
In April 2009, Washington University conducted a preclinical study of WntTide™ in a breast cancer model which showed tumor inhibition. The results of this study were presented at the 2nd Annual Wnt Conference in Washington, D.C., in June 2009 and will be published in the second calendar half of 2009. We are currently planning another breast tumor preclinical model study with researchers at Washington University in the continued development of WntTide™.
Tezampanel and NGX426 for the Potential Treatment of Thrombotic Disorder
Research conducted at Johns Hopkins University or JHU by Craig Morrell, D.V.M., Ph.D., and Charles Lowenstein, M.D. demonstrated the importance of glutamate release in promoting platelet activation and thrombosis. Research shows that platelets treated with an AMPA/kainate receptor antagonist such as tezampanel or NGX426 are more resistant to glutamate-induced aggregation than untreated platelets. This identifies the AMPA/kainate receptors on platelets as a new antithrombotic target with a different mechanism of action than Plavix®, aspirin or tPA. We have licensed the intellectual property for the treatment of thrombotic disorder from JHU and are in discussions with potential collaborators regarding this product candidate.

 

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Other Development Areas
Securing Additional and Complementary Technology Licenses from Others
We plan to establish additional research collaborations with prominent universities and research labs currently working on the development of potential targeting molecules, and to secure licenses from these universities and labs for technology resulting from the collaboration. No assurances can be made regarding our ability to establish such collaborations over the next 12 months, or at all. We intend to focus our in-licensing and product candidate acquisition activities on identifying complementary therapeutics, therapeutic platforms that offer a number of therapeutic targets, and clinical-stage therapeutics based on existing approved drugs in order to create proprietary reformulations to improve safety and efficacy or to expand such drugs’ clinical indications through additional clinical trials. We may obtain these products through collaborations, joint ventures or through merger and/or acquisitions with other biotechnology companies.
Strategic Acquisitions
Reverse Merger with TorreyPines
In calendar 2009, we began reviewing potential reverse merger candidates that would be a strategic fit and allow us the opportunity to list our stock on a national stock exchange such as NASDAQ. As a result of that search, we decided to engage in a reverse triangular merger in which our management, Board and our drug product programs would continue to be the focus of the post-merger company while gaining a NASDAQ listing and a couple of potential drug product candidates. In September 2009, our stockholders approved the merger agreement and we closed the reverse merger with TorreyPines and began trading on the NASDAQ Capital Market under the ticker symbol “RPTPD” as “Raptor Pharmaceutical Corp.” Effective October 27, 2009, our ticker changed to “RPTP” and all of our future SEC filings will be as Raptor Pharmaceutical Corp. At the closing of the reverse merger, our stockholders owned 95% of the post-merger company and the original TorreyPines stockholders owned 5% of the post-merger company.
Purchase of ConviviaTM
In October 2007, we purchased certain assets of Convivia, Inc., or Convivia, including intellectual property, know-how and research reports related to a product candidate targeting liver ALDH2 deficiency, a genetic metabolic disorder. We hired Convivia’s chief executive officer and founder, Thomas E. (Ted) Daley, as President of our clinical development division. In exchange for the assets related to the ALDH2 deficiency program, what we now calls ConviviaTM , we issued to Convivia 46,625 shares of our common stock, an additional 46,625 shares of our common stock to a third party in settlement of a convertible loan between the third party and Convivia, and another 8,742 shares of our common stock in settlement of other obligations of Convivia. Mr. Daley, as the former sole stockholder of Convivia, may earn additional shares of our common stock based on certain triggering events or milestones related to the development of the Convivia assets. In addition, Mr. Daley may earn cash bonuses based on the same triggering events pursuant to his employment agreement. In January 2008, Mr. Daley earned a $30,000 cash bonus pursuant to his employment agreement as a result of the milestone of our execution of a formulation agreement for manufacturing ConviviaTM with Patheon. In March 2008, we issued to Mr. Daley 23,312 shares of our common stock pursuant to our Convivia purchase agreement as a result of the milestone of our execution of an agreement to supply us with the active pharmaceutical ingredient for ConviviaTM and two $10,000 cash bonuses pursuant to his employment agreement for reaching his six-month and one-year employment anniversaries. In October 2008, we issued to Mr. Daley 23,312 shares of our common stock valued at $27,000 and a $30,000 cash bonus as a result of fulfilling a clinical milestone.

 

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Purchase of DR Cysteamine
In December 2007, through a merger between Encode Pharmaceuticals, Inc., or Encode, and our wholly-owned subsidiary, Raptor Therapeutics, we purchased certain assets, including the clinical development rights to DR Cysteamine. Under the terms of and subject to the conditions set forth in the merger agreement, we issued 802,946 shares of our common stock to the stockholders of Encode, or Encode Stockholders, options, or Encode Options, to purchase up to, in the aggregate, 83,325 shares of our common stock to the optionholders of Encode, or Encode Optionholders, and warrants, or Encode Warrants, to purchase up to, in the aggregate, 256,034 shares of our common stock to the warrantholders of Encode, or Encode Warrantholders, and together with the Encode Stockholders and Encode Optionholders, referred to herein collectively as the Encode Securityholders, as of the date of such agreement. Such common stock, Encode Options to purchase our common stock, and Encode Warrants to purchase our common stock combine for an aggregate amount of 1,142,304 shares of our common stock issuable to the Encode Securityholders as of the closing of the merger. The Encode Securityholders are eligible to receive up to an additional 559,496 shares of our common stock, Encode Options and Encode Warrants to purchase our common stock in the aggregate based on certain triggering events related to regulatory approval of DR Cysteamine, an Encode product program, if completed within the five year anniversary date of the merger agreement.
As a result of the Encode merger, we received the exclusive worldwide license to DR Cysteamine, referred to herein as the License Agreement, developed by clinical scientists at the UCSD, School of Medicine. In consideration of the grant of the license, we are obligated to pay an annual maintenance fee of $15,000 until we begin commercial sales of any products developed pursuant to the License Agreement. In addition to the maintenance fee, we are obligated to pay during the life of the License Agreement: milestone payments ranging from $20,000 to $750,000 for orphan indications and from $80,000 to $1,500,000 for non-orphan indications upon the occurrence of certain events, if ever; royalties on commercial net sales from products developed pursuant to the License Agreement ranging from 1.75% to 5.5%; a percentage of sublicense fees ranging from 25% to 50%; a percentage of sublicense royalties; and a minimum annual royalty commencing the year we begin commercially selling any products pursuant to the License Agreement, if ever. Under the License Agreement, we are obligated to fulfill predetermined milestones within a specified number of years ranging from 0.75 to 6 years from the effective date of the License Agreement, depending on the indication. In addition, we are obligated, among other things, to spend annually at least $200,000 for the development of products (which we satisfied, as of our fiscal year ended August 31, 2009 by spending approximately $4.1 million on such programs) pursuant to the License Agreement. To-date, we have paid $250,000 in milestone payments to UCSD based upon the initiation of clinical trials in cystinosis and in NASH. To the extent that we fail to perform any of our obligations under the License Agreement, UCSD may terminate the license or otherwise cause the license to become non-exclusive.
Application of Critical Accounting Policies
Our consolidated financial statements and accompanying notes are prepared in accordance with generally accepted accounting principles used in the U.S. Preparing financial statements requires management to make estimates and assumptions that affect the reported amounts of assets, liabilities, revenue, and expenses. These estimates and assumptions are affected by management’s application of accounting policies. We believe that understanding the basis and nature of the estimates and assumptions involved with the following aspects of our consolidated financial statements is critical to an understanding of our consolidated financial position.
We believe the following critical accounting policies require us to make significant judgments and estimates in the preparation of our consolidated financial statements.
Fair Value of Financial Instruments
The carrying amounts of certain of our financial instruments including cash and cash equivalents, prepaid expenses, accounts payable, accrued liabilities and capital lease liability approximate fair value due to their short maturities.

 

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Cash and Cash Equivalents
We consider all highly liquid investments with original maturities of three months or less to be cash equivalents.
Intangible Assets
Intangible assets include the intellectual property and other rights relating to DR Cysteamine and our RAP technology. Our intangible assets are amortized using the straight-line method over the estimated useful life of 20 years, which is the life of our intellectual property patents. The 20 year estimated useful life is also based upon the typical development, approval, marketing and life cycle management timelines of pharmaceutical drug products.
Fixed Assets
Fixed assets, which mainly consist of leasehold improvements, lab equipment, computer hardware and software and capital lease equipment, are stated at cost. Depreciation is computed using the straight-line method over the related estimated useful lives, except for leasehold improvements and capital lease equipment, which are depreciated over the shorter of the useful life of the asset or the lease term. Significant additions and improvements that have useful lives estimated at greater than one year are capitalized, while repairs and maintenance are charged to expense as incurred.
Impairment of Long-Lived Assets
We evaluate our long-lived assets for indicators of possible impairment by comparison of the carrying amounts to future net undiscounted cash flows expected to be generated by such assets when events or changes in circumstances indicate the carrying amount of an asset may not be recoverable. Should an impairment exist, the impairment loss would be measured based on the excess carrying value of the asset over the asset’s fair value or discounted estimates of future cash flows. We have not identified any such impairment losses to date.
Income Taxes
Income taxes are recorded under the liability method, under which deferred tax assets and liabilities are determined based on the difference between the financial statement and tax bases of assets and liabilities using enacted tax rates in effect for the year in which the differences are expected to affect taxable income. Valuation allowances are established when necessary to reduce deferred tax assets to the amount expected to be realized.
Research and Development
We are an early development stage company. Research and development costs are charged to expense as incurred. Research and development expenses include scientists’ salaries, lab collaborations, preclinical studies, clinical trials, clinical trial materials, regulatory and clinical consultants, lab supplies, lab services, lab equipment maintenance and small equipment purchased to support the research laboratory, amortization of intangible assets and allocated executive, human resources and facilities expenses.
In-Process Research and Development
We record in-process research and development expense for a product candidate acquisition where there is not more than one potential product or usage for the assets being acquired. We review each product candidate acquisition to determine if the purchase price should be expensed as in-process research and development or capitalized and amortized over the life of the asset.
Stock-Based Compensation
In May 2006, our stockholders approved the 2006 Equity Compensation Plan, referred to herein as the Plan. The Plan’s term is ten years and allows for the granting of options to our employees, directors and consultants.

 

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Typical option grants are for ten years with exercise prices at or above market price based on the last closing price as of the date prior to the grant date as quoted on the OTC Bulletin Board or relevant stock market or exchange and vest over four years as follows: 6/48ths on the six month anniversary of the date of grant; and 1/48th per month thereafter.
Effective September 1, 2006, our stock-based compensation is accounted for in accordance with Statement of Financial Accounting Standards, or SFAS, No. 123(R), or SFAS 123(R), Share-Based Payment and related interpretations. Under the fair value recognition provisions of this statement, share-based compensation cost is measured at the grant date based on the value of the award and is recognized as expense over the vesting period. Determining the fair value of share-based awards at the grant date requires judgment, including estimating future stock price volatility and employee stock option exercise behavior. If actual results differ significantly from these estimates, stock-based compensation expense and results of operations could be materially impacted.
In March 2005, the SEC issued Staff Accounting Bulletin, or SAB, No. 107, or SAB 107, which offers guidance for SFAS 123(R). SAB 107 was issued to assist preparers by simplifying some of the implementation challenges of SFAS 123(R) while enhancing the information that investors receive. SAB 107 creates a framework that is premised on two overarching themes: (a) considerable judgment will be required by preparers to successfully implement SFAS 123(R), specifically when valuing employee stock options; and (b) reasonable individuals, acting in good faith, may conclude differently on the fair value of employee stock options. Key topics covered by SAB 107 include valuation models, expected volatility and expected term. We are applying the principles of SAB 107 in conjunction with our adoption of SFAS 123(R).
For the three month period ended August 31, 2009, stock-based compensation expense was based on the Black-Scholes option-pricing model assuming the following: risk-free interest rate of 3.2%; 7 year expected life; 240% volatility; 10% turnover rate; and 0% dividend rate.
We based our Black-Scholes inputs on the following factors: the risk-free interest rate was based upon our review of current constant maturity treasury bill rates for seven years; the expected life was based upon our assessment of the ten-year term of the stock options issued along with the fact that we are a development-stage company and our anticipation that option holders will exercise stock options when the company is at a more mature stage of development; the volatility was based on the actual volatility of our common stock price as quoted on the over the counter bulletin board; the turnover rate was based on our assessment of our size and the minimum potential for employee turnover at our current development-stage; and the dividend rate was based on our current decision to not pay dividends on our stock at our current development stage.
If factors change and different assumptions are employed in the application of SFAS 123(R), the compensation expense recorded in future periods may differ significantly from what was recorded in the current period. See Note 8 of our consolidated financial statements for further discussion of our accounting for stock based compensation.
We recognize as consulting expense the fair value of options granted to persons who are neither employees nor directors. Stock options issued to consultants are accounted for in accordance with the provisions of Emerging Task Force, or EITF, Issue No. 96-18, Accounting for Equity Instruments that are Issued to Other Than Employees for Acquiring, or in Conjunction with Selling, Goods or Services. The fair value of expensed options is based on the Black-Scholes option-pricing model assuming the same factors as stock-based compensation expense discussed above.
On November 10, 2005, the Financial Accounting Standards Board, or FASB, issued FASB Staff Position, or FSP, No. FAS 123(R)-3 Transition Election Related to Accounting for Tax Effects of Share-Based Payment Awards . This FSP provides a practical transition election related to the accounting for the tax effects of share-based payment awards to employees, as an alternative to the transition guidance for the additional paid-in capital pool (APIC pool) in paragraph 81 of SFAS 123(R). The alternative transition method includes simplified methods to establish the beginning balance of the APIC pool related to the tax effects of employee stock-based compensation, and to determine the subsequent impact on the APIC pool and statements of cash flows of the tax effects of employee stock-based compensation awards that are outstanding upon adoption of SFAS 123(R). The guidance in this FSP is effective after November 10, 2005. We may take up to one year from the later of adoption of SFAS 123(R) or the effective date of this FSP to evaluate our available transition alternatives and make our one-time election. We have elected the “short-form” method to calculating excess tax benefits available for use in offsetting future tax shortfalls in accordance with FSP FAS 123(R)-3.

 

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Results of Operations
Years ended August 31, 2009 and 2008
General and Administrative
General and administrative expenses (including officer and employee compensation allocated to general and administrative expenses) for the year ended August 31, 2009 increased by $0.45 million compared to the prior year. The increase was primarily due to (A) an increase of $0.29 million in legal and other costs accrued related to the TorreyPines merger, $0.17 million in employee salaries, bonuses, benefits and other employment-related costs due to employee raises that occurred in July 2008, milestone related bonuses paid in October 2008, recruiting fees related to the hiring of our Director of Program Management in October 2008 and our Chief Medical Officer in April 2009, offset by prior year’s performance bonuses not repeated in the current year, plus (B) an increase of $0.20 million in administrative consulting due to the retention of a strategic business advisor in May 2008, investor relations consultants in February 2009 and for the redesign of our website in November 2008, and (C) an increase of $0.08 million of board fees and expenses due to the addition of a new board member in July 2008, all of which were partially offset by (i) the increase of support services allocation to research and development expenses of $0.19 million (ii) a decrease of $0.05 million in amortization and depreciation related to fully depreciated fixed assets, and (iii) a decrease of $0.05 million in travel due to a reduction in attendance at tradeshows and conferences.
Research and Development
Research and development expenses (including officer and employee compensation allocated to research and development) for the year ended August 31, 2009 increased by $1.00 million over the prior year primarily due to (A) the costs associated with the formulation manufacturing expenses of our proprietary formulation of DR Cysteamine of $1.21 million in preparation for our clinical trials in cystinosis, (B) an increase of $0.34 million in research and development consulting related to the preparation for our pre-IND meeting with the FDA and in preparation for our IND submission, (C) an increase of $0.25 million in salaries and benefits due to the hiring of our director of program management in October 2008 and our Chief Medical Officer in April 2009, (D) an increase of $0.25 million in milestone payments for the commencement of the NASH trial in October 2008 and cystinosis trial in June 2009, (E) an increase of $0.23 million in clinical trial costs for our NASH indication, and (F) an increase of $0.19 million in allocated support services, all of which were partially offset by (i) a decrease of $0.54 million due to the ConviviaTM clinical trial in the prior year that did not repeat in the current year, (ii) a $0.30 reduction in lab personnel expenses due to a collaboration reimbursement, (iii) a decrease of $0.27 million of HepTideTM conjugates that were manufactured in the prior year but did not repeat in the current year, (iv) a decrease in lab collaboration fees of $0.24 million due to the lapse of the Stanford collaboration on NeuroTrans™, (v) a decrease of $0.10 million in preclinical studies due to the reduction of resources allocated to preclinical programs and (vi) a decrease of $0.02 million in tradeshow costs which we incurred in the prior year but not in the current year.
Research and development expenses include the following: (in $ millions)
                                 
    Estimated            
    FYE   Cumulative Through   FYE   FYE
Major Program (stage of development)   August 31, 2010   August 31, 2009   August 31, 2009   August 31, 2008
DR Cysteamine — All Indications (clinical)
    6.0       5.0       4.0       1.0  
ConviviaTM (clinical)
    0.1       2.1       0.4       1.7  
HepTideTM (preclinical)
    0.1       1.6       0.4       0.7  
NeuroTransTM (preclinical)
          0.3       (0.3 )     0.3  
WntTideTM (preclinical)
          0.3       0.1       0.2  
Minor or Inactive Programs
          0.7       0.1       0.2  
R & D Personnel and Other Costs Not Allocated to Programs
    1.7       4.9       1.9       1.5  
Total Research & Development Expenses
    7.9       14.9       6.6       5.6  

 

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Major Program expenses recorded as general and administrative expenses: (in $ millions)
                                 
    Estimated            
    FYE   Cumulative Through   FYE   FYE
Major Program (stage of development)   August 31, 2010   August 31, 2009   August 31, 2009   August 31, 2008
DR Cysteamine — All Indications (clinical)
    0.10       0.20       0.12       0.08  
ConviviaTM (clinical)
    0.01       0.09       0.05       0.04  
HepTideTM (preclinical)
    0.05       0.17       0.07       0.05  
NeuroTransTM (preclinical)
    0.03       0.15       0.05       0.05  
WntTideTM (preclinical)
    0.01       0.06       0.01       0.02  
Additional major program expenses include patent fees and patent expenses which were recorded as general and administrative expenses as these fees are to support patent applications (not issued patents).
Any of our major programs could be partnered for further development and/or could be accelerated, slowed or ceased due to scientific results or challenges in obtaining funding. We will need significant additional funding in order to pursue our plans for the next 12 months. In addition, the timing and costs of development of our programs beyond the next 12 months is highly uncertain and difficult to estimate. See Item 1A titled Risk Factors for further discussion about the risks and uncertainties pertaining to drug development.
Current Status of Major Programs
Please refer to the section titled, “Management’s Discussion and Analysis of Financial Condition and Results of Operations” in this Annual Report on Form 10-K for a detailed discussion of each of our major programs. In summary, DR Cysteamine is being developed in cystinosis, NASH and HD. In June 2009, we began our Phase IIb clinical trial to study DR Cysteamine in cystinosis patients. In February 2009, we completed enrollment of our NASH clinical trial. Both our NASH and cystinosis clinical trials are currently ongoing. We anticipate studying DR Cysteamine in a Phase III clinical trial in cystinosis patients and a Phase IIa clinical trial in HD patients in early 2010.
Our ConviviaTM product candidate completed our initial clinical study in 2008 and we are actively seeking to partner any further development of our Convivia TM product candidate with an Asian company where our potential market exists. We are seeking program funding for our Tezampanel and NGX426 product candidates, however, no development costs will be incurred until such funding is obtained, if at all. NeuroTransTM is currently being studied under a collaboration agreement with Roche. HepTideTM and WntTideTM are undergoing preclinical proof of concept studies, which will require further study prior to potentially moving into a clinical phase of development.
In-Process Research and Development Expenses
In-process research and development expenses decreased by $0.24 million over the year ended August 31, 2008 due to the recording of the purchase of our ConviviaTM program during the year ended August 31, 2008. No such expense was incurred in the year ended August 31, 2009. In-process research and development expenses were calculated based on the value of our stock issued in connection with the purchase of certain intellectual property rights to develop ConviviaTM (4-MP) for the treatment of acetaldehyde toxicity.
Interest Income
Interest income decreased by $0.041 million during the year ended August 31, 2009 over the year ended August 31, 2008 due to the significant decrease in annual money market interest rates from an average of 2% in 2008 to an average of less than 1% in 2009.
Interest expense
Interest expense decreased by $0.10 million in the year ended August 31, 2009 over the year ended August 31, 2008 due to the capitalized finder’s fee of 46,625 shares of our common stock valued at $102,000 (which was paid in connection with a convertible loan), which was amortized as interest expense from August 2007 to April 2008, the term of the convertible loan. No draws were made on the convertible loan prior to its expiration.

 

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Years ended August 31, 2008 and 2007
General and Administrative
General and administrative expenses (including officer and employee compensation allocated to general and administrative expenses) for the fiscal year ended August 31, 2008 increased by $0.7 million over the prior fiscal year primarily due to the costs incurred during our fiscal year ended August 31, 2008 for the patent expenses for our clinical programs of $0.1 million, the salary and benefits of our clinical subsidiary’s President of $0.3 million, and legal and accounting expenses attributable to our clinical subsidiary of $0.3 million.
Research and Development
Research and development expenses (including officer and employee compensation allocated to research and development) for the fiscal year ended August 31, 2008 increased by $3.4 million over the prior fiscal year primarily due to the costs incurred during our fiscal year ended August 31, 2008 associated with our Phase IIa clinical trial for the ConviviaTM program of $0.6 million, clinical and regulatory consulting for ConviviaTM of $0.6 million and DR Cysteamine of $0.6 million, executive, finance and facilities costs allocated to the research and development department of our clinical division of $0.5 million, formulation manufacturing expenses of the proprietary formulation of ConviviaTM of $0.3 million and DR Cysteamine of $0.1 million, preclinical studies of ConviviaTM of $0.2 million and of DR Cysteamine of $0.1 million, and amortization of intangible assets related to the purchase of DR Cysteamine of $0.1 million and incremental executive, finance and facilities costs allocated to the research and development department of our clinical division of $0.5 million.
In-Process Research and Development Expenses
In-process research and development expenses increased by $0.24 million over the prior fiscal year due to the recording of the purchase of our ConviviaTM program during our fiscal year ended August 31, 2008. No such expense was incurred in the prior year. In-process research and development expenses were calculated based on the value of our stock issued in connection with the purchase of certain intellectual property rights to develop ConviviaTM (4-MP) for the treatment of acetaldehyde toxicity. For further details about the calculation of in-process research and development expenses, please refer to Note 7 of our audited financial statements located elsewhere in this Annual Report on Form 10-K.
Interest Income
Interest income decreased by $0.07 million over the prior fiscal year due to the significant decrease in money market interest rates from 4.5% during the fiscal year ended August 31, 2007 to an average of approximately 2% during the fiscal year ended August 31, 2008, which was partially offset by the increase in money market balances during the fiscal year ended August 31, 2008 due to the $10 million raised in May and June 2008.
Interest expense
Interest expense increased by $0.1 million over the prior fiscal year due to the capitalized finder’s fee of 46,625 shares of our common stock paid in connection with a convertible loan. These shares were valued at $102,000, which was amortized as interest expense from August 2007 to April 2008, the term of the convertible loan. No draws were made on the loan prior to its expiration.
Liquidity and Capital Resources
Capital Resource Requirements
As of August 31, 2009, we had approximately $3.7 million in cash, approximately $1.1 million in current liabilities and approximately $2.7 million of net working capital. Our forecasted average monthly cash expenditures for the next twelve months are approximately $875,000.

 

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We believe that our cash and cash equivalents balances as of August 31, 2009 will be sufficient to meet our obligations into the first calendar quarter of 2010. We are currently in the process of reviewing strategic partnerships and collaborations in order to fully fund our preclinical and clinical programs through the end of 2010. We also anticipate raising additional capital in the fourth calendar quarter of 2009.
On April 29, 2009, in order to reflect current market prices, we notified the holders of warrants purchased in the May/June 2008 private placement that we were offering, in exchange for such warrants, new warrants to purchase our common stock at an exercise price of $1.29 per share, but only to the extent such exchange of the original warrants and exercise of the new warrants, including the delivery of the exercise price, occurred on or prior to July 17, 2009. The warrants that were not exchanged prior to or on July 17, 2009 retained their original exercise prices of $3.86 per share and original expiration date of May 21, 2010. We received approximately $2.6 million of proceeds from warrant exercises that resulted in the issuance of 2,031,670 shares of our common stock pursuant to the exchange described above.
On August 21, 2009, we entered into a Securities Purchase Agreement, dated as of August 21, 2009, with four investors for the private placement of units at a purchase price of $1.37 per unit, each unit was comprised of one share of our common stock, par value $0.001 per share and one warrant to purchase one half of one share of our common stock. At the closing, we sold an aggregate of 1,738,226 units to the investors, comprised of an aggregate of 1,738,226 shares of our common stock and warrants, for aggregate gross proceeds of $2,386,000. The investor warrants, exercisable for two years from the closing, entitle the investors to purchase, in the aggregate, up to 869,113 shares of our common stock and have an exercise price of $2.57 per share during the first year and $3.22 per share during the second year, depending on when such investor warrants are exercised, if at all.
There can be no assurance that we will be able to obtain funds required for our continued operation. There can be no assurance that additional financing will be available to us or, if available, that it can be obtained on commercially reasonable terms. If we are not able to obtain financing on a timely basis, we will not be able to meet our obligations as they become due and we will be forced to scale down or perhaps even cease the operation of our business. This also may be the case if we become insolvent or if we breach our asset purchase agreement with BioMarin or our licensing agreements with Washington University and UCSD due to non-payment (and we do not cure our non-payment within the stated cure period). If this happens, we would lose all rights to the RAP technology assigned to us by BioMarin and/or the rights to Mesd licensed to us by Washington University and/or the rights to DR Cysteamine licensed to us by UCSD, depending on which agreement is breached. If we lose our rights to the intellectual property related to the RAP technology purchased by us from BioMarin, our agreement with Roche would likely be terminated and any milestone or royalty payments from Roche to us would thereafter cease to accrue.
We will need to raise significant long-term financing in order to implement our 12 month operating plan. If we are able to raise significant additional financing, for the next 12 months we intend to expend a total of approximately $10.5 million to implement our operating plan of researching and developing our DR Cysteamine clinical programs, our RAP based platform, our licensed technologies, as well as continuing business development efforts for our other clinical-stage product candidates.
Specifically, we estimate our operating expenses and working capital requirements for the next 12 months to be as follows:
         
Estimated spending for the next 12 months:        
Research and development activities
  $ 6,600,000  
Research and development compensation and benefits
    1,300,000  
General and administrative activities
    1,400,000  
General and administrative compensation and benefits
    1,180,000  
Capital expenditures
    20,000  
 
     
 
       
Total estimated spending for the next 12 months
  $ 10,500,000  
 
     

 

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We anticipate that we will not be able to generate revenues from the sale of products until we further develop our drug product candidates and obtain the necessary regulatory approvals to market our future drug product candidates, which could take several years or more, if we are able to do so at all. Accordingly, our cash flow projections are subject to numerous contingencies and risk factors beyond our control, including successfully developing our drug product candidates, market acceptance of our drug product candidates, competition from well-funded competitors, and our ability to manage our expected growth. It is likely that for many years, we will not be able to generate internal positive cash flow from the sales of our drug product candidates sufficient to meet our operating and capital expenditure requirements.
There is substantial doubt about our ability to continue as a going concern as the continuation of our business is dependent upon obtaining further long-term financing, the successful development of our drug product candidates and related technologies, the successful and sufficient market acceptance of any product offerings that we may introduce and, finally, the achievement of a profitable level of operations. The issuance of additional equity securities by us is likely to result in a significant dilution in the equity interests of our current stockholders. Obtaining commercial loans, assuming those loans would be available, including on acceptable terms, will increase our liabilities and future cash commitments.
Research and Development Activities
We plan to conduct further research and development, seeking to support several clinical trials for DR Cysteamine, improve upon our RAP-based and in-licensed technology and continue business development efforts for our other clinical-stage product candidates in the next 12 months. We plan to conduct research and development activities by our own laboratory staff and also by engaging contract research organizations, clinical research organizations and contract manufacturing organizations. We also plan to incur costs for the production of our clinical study drug candidate, DR Cysteamine, clinical trials, clinical and medical advisors and consulting and collaboration fees. Assuming we obtain additional long-term financing, we anticipate our research and development costs for the next 12 months, excluding in-house research and development compensation, will be approximately $6.6 million. We will need to scale down our research and development plans and expenses detailed herein in the next fiscal year if we are not able to raise significant additional financing over the next three months as detailed in the section titled, “Capital Resource Requirements.”
Officer and Employee Compensation
We currently employ eight executive officers. We also have one permanent scientific staff member, two permanent clinical development staff members and one permanent finance staff member. Assuming we obtain significant additional long-term financing, we anticipate spending up to approximately $2.48 million in officer and employee compensation during the next 12 months, of which $1.30 million is allocated to research and development expenses, $0.68 million is allocated to general and administrative expenses and $0.50 million is related to the TorreyPines personnel and is allocated to general and administrative expenses. Cash received as a result of the merger with TorreyPines is sufficient to cover the TorreyPines personnel expense obligations of $0.50 million. We will need to scale down our officer and employee compensation expenses detailed herein in the next fiscal year if we are not able to raise significant additional financing over the next 12 months as detailed in the section titled, “Capital Resource Requirements.”
General and Administrative
Assuming we obtain additional long-term financing, we anticipate spending approximately $1.40 million on general and administrative costs in the next 12 months. These costs will consist primarily of legal and accounting fees, patent legal fees, investor relations expenses, board fees and expenses, insurance, rent and facility support expenses, excluding finance and administrative compensation. We will need to scale down our general and administrative plans and expenses detailed herein in the next fiscal year if we are not able to raise significant additional financing over the next 12 months as detailed in the section titled, “Capital Resource Requirements.”

 

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Capital Expenditures
We anticipate spending approximately $20,000 in the next 12 months on capital expenditures for lab equipment and office furniture. We will need to scale down our capital expenditures detailed herein in the next fiscal year if we are not able to raise significant additional financing over the next 12 months as detailed in the section titled, “Capital Resource Requirements.”
Contractual Obligations with BioMarin
Pursuant to the terms of the asset purchase agreement we entered into with BioMarin for the purchase of intellectual property related to our RAP based technology (including NeuroTransTM ), we are obligated to make the following milestone payments to BioMarin upon the achievement of the following events:
   
$50,000 (paid by us in June 2006) within 30 days after we receive total aggregate debt or equity financing of at least $2,500,000;
 
   
$100,000 (paid by us in June 2006) within 30 days after we receive total aggregate debt or equity financing of at least $5,000,000;
 
   
$500,000 upon our filing and acceptance of an investigational new drug application for a drug product candidate based on our NeuroTransTM product candidate;
 
   
$2,500,000 upon our successful completion of a Phase II human clinical trial for a drug product candidate based on our NeuroTransTM product candidate;
 
   
$5,000,000 upon our successful completion of a Phase III human clinical trial for a drug product candidate based on our NeuroTransTM product candidate;
 
   
$12,000,000 within 90 days of our obtaining marketing approval from the FDA or other similar regulatory agencies for a drug product candidate based on our NeuroTransTM product candidate;
 
   
$5,000,000 within 90 days of our obtaining marketing approval from the FDA or other similar regulatory agencies for a second drug product candidate based on our NeuroTransTM product candidate;
 
   
$5,000,000 within 60 days after the end of the first calendar year in which our aggregated revenues derived from drug product candidates based on our NeuroTransTM product candidate exceed $100,000,000; and
 
   
$20,000,000 within 60 days after the end of the first calendar year in which our aggregated revenues derived from drug product candidates based on our NeuroTransTM product candidate exceed $500,000,000.
In addition to these milestone payments, we are also obligated to pay BioMarin a royalty at a percentage of our aggregated revenues derived from drug product candidates based on our NeuroTransTM product candidate. On June 9, 2006, we made a milestone payment in the amount of $150,000 to BioMarin because we raised $5,000,000 in our May 25, 2006 private placement financing. If we become insolvent or if we breach our asset purchase agreement with BioMarin due to non-payment and we do not cure our non-payment within the stated cure period, all of our rights to RAP technology (including NeuroTransTM) will revert back to BioMarin.
Contractual Obligations with Thomas E. Daley (assignee of the dissolved Convivia, Inc.)
Pursuant to the terms of the asset purchase agreement, or the Asset Purchase Agreement that we entered into with Convivia, Inc. and Thomas E. Daley, pursuant to which we purchased intellectual property related to our 4-MP product candidate program, Mr. Daley will be entitled to receive the following, if at all, in such amounts and only to the extent certain future milestones are accomplished by us, as set forth below:

 

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23,312 shares of our restricted, unregistered common stock within fifteen (15) days after we enter into a manufacturing license or other agreement to produce any product that is predominantly based upon or derived from any assets purchased from Convivia, or Purchased Assets, in quantity, referred to as Product, if such license agreement is executed within one (1) year of execution of the Asset Purchase Agreement or, if thereafter, 11,656 shares of our restricted, unregistered common stock. Should we obtain a second such license or agreement for a Product, Mr. Daley will be entitled to receive 11,656 shares of our restricted, unregistered common stock within 30 days of execution of such second license or other agreement. On March 31, 2008, we issued 23,312 shares of our common stock valued at $56,000 to Mr. Daley pursuant to this milestone reflecting the execution of an agreement to supply the active pharmaceutical ingredient for ConviviaTM , combined with the execution of a formulation agreement to produce the oral formulation of ConviviaTM
 
   
23,312 shares of our restricted, unregistered common stock within fifteen (15) days after we receive our first patent allowance on any patents which constitute part of the Purchased Assets in any one of certain predetermined countries, or Major Market.
 
   
11,656 shares of our restricted, unregistered common stock within fifteen (15) days after we receive our second patent allowance on any patents which constitute part of the Purchased Assets different from the patent referenced in the immediately preceding bullet point above in a Major Market.
 
   
23,312 shares of our restricted, unregistered common stock within fifteen (15) days of completion of predetermined benchmarks in a Major Market by us or our licensee of the first phase II human clinical trial for a Product, or Successful Completion if such Successful Completion occurs within one (1) year of execution of the Asset Purchase Agreement or, if thereafter, 11,656 shares of our restricted, unregistered common stock within thirty (30) days of such Successful Completion. In October 2008, we issued 23,312 shares of our common stock valued at $27,000 and a $30,000 cash bonus (pursuant to Mr. Daley’s employment agreement) to Mr. Daley pursuant to the fulfillment of this milestone.
 
   
11,656 shares of our restricted, unregistered common stock within fifteen (15) days of a Successful Completion in a Major Market by us or our licensee of the second phase II human clinical trial for a Product (other than the Product for which a distribution is made under the immediately preceding bullet point above).
 
   
23,312 shares of our restricted, unregistered common stock within fifteen (15) days after we or our licensee applies for approval to market and sell a Product in a Major Market for the indications for which approval is sought, or Marketing Approval.
 
   
11,656 shares of our restricted, unregistered common stock within fifteen (15) days after we or our licensee applies for Marketing Approval in a Major Market (other than the Major Market for which a distribution is made under the immediately preceding bullet point above).
 
   
46,625 shares of our restricted, unregistered common stock within fifteen (15) days after we or our licensee obtains the first Marketing Approval for a Product from the applicable regulatory agency in a Major Market.
 
   
23,312 shares of our restricted, unregistered common stock within fifteen (15) days after we or our licensee obtains Marketing Approval for a Product from the applicable regulatory agency in a Major Market (other than the Major Market for which a distribution is made under the immediately preceding bullet point above).

 

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As discussed above, in aggregate, we issued to Mr. Daley, 46,625 shares of our common stock valued at $83,000 and paid $30,000 in cash bonuses related to ConviviaTM milestones along with another $20,000 in cash bonuses related to employment milestones pursuant to Mr. Daley’s employment agreement.
Contractual Obligations with Former Encode Securityholders
Pursuant to the terms of the merger agreement, or the Merger Agreement, we entered into with Encode Pharmaceuticals, Inc. and Nicholas Stergis, former Encode securityholders will be entitled to receive the following, if at all, in such amounts and only to the extent certain future milestones are accomplished by us, as set forth below:
   
Restricted, unregistered common stock, stock options to purchase our common stock, and warrants to purchase our common stock in an amount equal to, in the aggregate, 116,562 shares of our common stock upon the receipt by it at any time prior to the fifth-year anniversary of the Merger Agreement of approval to market and sell a product for the treatment of cystinosis predominantly based upon and derived from the assets acquired from Encode, or Cystinosis Product, from the applicable regulatory agency (e.g., FDA and European Agency for the Evaluation of European Medical Products or EMEA) in a given major market in the world.
 
   
Restricted, unregistered common stock, stock options to purchase our common stock, and warrants to purchase our common stock in an amount equal to 442,934 shares of our common stock upon the receipt by us at any time prior to the fifth anniversary of the Merger Agreement of approval to market and sell a product, other than a Cystinosis Product, predominantly based upon and derived from the assets acquired from Encode, from the applicable regulatory agency (e.g., FDA and EMEA) in a given major market in the world.
If within five years from the date of the Merger Agreement, there occurs a transaction or series of related transactions that results in the sale of all or substantially all of the assets acquired from Encode other than to our affiliate in such case where such assets are valued at no less than $2.5 million, the former Encode stockholders will be entitled to receive, in the aggregate, restricted, unregistered common stock, stock options to purchase our common stock, and warrants to purchase our common stock in an amount equal to 559,496 shares of common stock, less the aggregate of all milestone payments previously made or owing, if any.
Pursuant to the terms of the Merger Agreement, we will at any time following 140 days from the closing date of the merger and prior to the expiration of the fourth anniversary of the Merger Agreement, grant to an Encode stockholder the right to demand the registration of its portion of the initial restricted, unregistered common stock issued to it in connection with the execution of the Merger Agreement and future restricted, unregistered common stock issued to it in the future relating to the milestone payments outlined above, if any.
Contractual Obligations with UCSD
As a result of the merger of our clinical subsidiary and Encode, we received the exclusive worldwide license to DR Cysteamine, or License Agreement for use in the field of human therapeutics for metabolic and neurologic disorders, developed by clinical scientists at the UCSD, School of Medicine. DR Cysteamine is a proprietary, delayed-release, enteric-coated microbead formulation of cysteamine bitartrate, a cystine depleting agent currently approved by the FDA. Cysteamine bitartrate is prescribed for the management of the genetic disorder known as cystinosis, a lysosomal storage disease. The active ingredient in DR Cysteamine has also demonstrated potential in studies as a treatment for other metabolic and neurodegenerative diseases, such as HD and NASH.
In consideration of the grant of the license, prior to the merger, Encode paid an initial license fee and we will be obligated to pay an annual maintenance fee of $15,000 until we begin commercial sales of any products developed pursuant to the License Agreement. In addition to the maintenance fee, we will be obligated to pay during the life of the License Agreement: milestone payments ranging from $20,000 to $750,000 for orphan indications and from $80,000 to $1,500,000 for non-orphan indications upon the occurrence of certain events, if ever; royalties on commercial net sales from products developed pursuant to the License Agreement ranging from

 

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1.75% to 5.5%; a percentage of sublicense fees ranging from 25% to 50%; a percentage of sublicense royalties; and a minimum annual royalty commencing the year we begin commercially selling any products pursuant to the License Agreement, if ever. Under the License Agreement, we are obligated to fulfill predetermined milestones within a specified number of years ranging from 0.75 to 6 years from the effective date of the License Agreement, depending on the indication. In addition, we are obligated to, among other things, annually spend at least $200,000 for the development of products (which, as of our fiscal year ended August 31, 2009 by spending approximately $4.1 million on such programs) pursuant to the License Agreement. To date, we have paid $250,000 in milestone payments to UCSD based upon the initiation of clinical trials in cystinosis and in NASH. To the extent that we fail to perform any of our obligations under the License Agreement, UCSD may terminate the license or otherwise cause the license to become non-exclusive.
Off-Balance Sheet Arrangements
We do not have any outstanding derivative financial instruments, off-balance sheet guarantees, interest rate swap transactions or foreign currency contracts. We do not engage in trading activities involving non-exchange traded contracts.
Reverse Acquisition
We are treating the TorreyPines merger as a reverse acquisition and the reverse acquisition will be accounted for as a recapitalization.
For accounting purposes, Raptor Pharmaceuticals Corp. is considered the accounting acquirer in the reverse acquisition. The historical financial statements that will be reported in future periods will be those of Raptor Pharmaceuticals Corp. consolidated with its subsidiaries and with its parent, Raptor Pharmaceutical Corp. (formerly TorreyPines). Earnings per share for periods prior to the reverse merger have been restated to reflect the number of equivalent shares received by our former stockholders.
Going Concern
Due to the uncertainty of our ability to meet our current operating and capital expenses, in their reports on our audited financial statements for the years ended August 31, 2009, 2008, 2007 and for the period September 8, 2005 (inception) to August 31, 2006, our independent registered public accounting firm, Burr, Pilger & Mayer, LLP included an explanatory paragraph regarding substantial doubt about our ability to continue as a going concern. Our financial statements contain additional note disclosures describing the circumstances that led to this disclosure by our independent registered public accounting firm.
New Accounting Pronouncements.
In September 2006, the FASB issued SFAS No. 157, Fair Value Measurements, or SFAS 157. SFAS 157 defines fair value, establishes a framework for measuring fair value in accordance with generally accepted accounting principles, and expands disclosures about fair value measurements. SFAS 157 does not require any new fair value measurements; rather, it applies under other accounting pronouncements that require or permit fair value measurements. The provisions of SFAS 157 are to be applied prospectively as of the beginning of the fiscal year in which it is initially applied, with any transition adjustment recognized as a cumulative-effect adjustment to the opening balance of retained earnings. The provisions of SFAS 157 are effective for fiscal years beginning after November 15, 2007; therefore, we adopted SFAS 157 as of September 1, 2008 for financial assets and liabilities. In accordance with FASB Staff Position 157-2, Effective Date of FASB Statement No. 157 , we elected to defer the adoption of the provisions of SFAS 157 for our non-financial assets and non-financial liabilities. See Note 5, Fair Value Measurements, in our consolidated financial statements as of August 31, 2009, regarding the disclosure of the value of our cash equivalents.
In February 2007, the FASB issued SFAS 159, The Fair Value Option for Financial Assets and Financial Liabilities, Including an Amendment of FASB Statement No. 115, which permits the measurement of many financial instruments and certain other asset and liabilities at fair value on an instrument-by-instrument basis (the fair value option). The guidance is applicable for fiscal years beginning after November 15, 2007; therefore, we adopted SFAS 159 as of September 1, 2008. We have determined that SFAS 159 had no material impact on our financial results for the year ended August 31, 2009.

 

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In June 2007, the EITF reached a consensus on EITF No. 07-3, Accounting for Nonrefundable Advance Payments for Goods or Services to Be Used in Future Research and Development Activities, or EITF 07-3. EITF 07-3 specifies the timing of expense recognition for non-refundable advance payments for goods or services that will be used or rendered for research and development activities. EITF 07-3 was effective for fiscal years beginning after December 15, 2007, and early adoption is not permitted; therefore, we adopted EITF 07-3 as of September 1, 2008. We have determined that EITF 07-3 had no material impact on our financial results for the year ended August 31, 2009.
In December 2007, the EITF reached a consensus on EITF No. 07-1, Accounting for Collaborative Arrangements Related to the Development and Commercialization of Intellectual Property, or EITF 07-1. EITF 07-1 discusses the appropriate income statement presentation and classification for the activities and payments between the participants in arrangements related to the development and commercialization of intellectual property. The sufficiency of disclosure related to these arrangements is also specified. EITF 07-1 is effective for fiscal years beginning after December 15, 2008. As a result, EITF 07-1 is effective for us as of September 1, 2009. We are currently evaluating the impact of EITF 07-1 on our financial position and results of operations. Based upon the nature of our business, EITF 07-1 could have a material impact on our financial position and consolidated results of operations in future years.
In December 2007, FASB issued Statement No. 141 (Revised 2007), Business Combinations, or SFAS 141(R), and SFAS No. 160, Accounting and Reporting of Non-controlling Interests in Consolidated Financial Statements, an amendment of ARB No. 51, or SFAS 160. These statements will significantly change the financial accounting and reporting of business combination transactions and non-controlling (or minority) interests in consolidated financial statements. SFAS 141(R) requires companies to: (i) recognize, with certain exceptions, 100% of the fair values of assets acquired, liabilities assumed, and non-controlling interests in acquisitions of less than a 100% controlling interest when the acquisition constitutes a change in control of the acquired entity; (ii) measure acquirer shares issued in consideration for a business combination at fair value on the acquisition date; (iii) recognize contingent consideration arrangements at their acquisition-date fair values, with subsequent changes in fair value generally reflected in earnings; (iv) with certain exceptions, recognize pre-acquisition loss and gain contingencies at their acquisition-date fair values; (v) capitalize in-process research and development assets acquired; (vi) expense, as incurred, acquisition-related transaction costs; (vii) capitalize acquisition-related restructuring costs only if the criteria in SFAS No. 146, Accounting for Costs Associated with Exit or Disposal Activities, are met as of the acquisition date; and (viii) recognize changes that result from a business combination transaction in an acquirer’s existing income tax valuation allowances and tax uncertainty accruals as adjustments to income tax expense. SFAS 141(R) is required to be adopted concurrently with SFAS 160 and is effective for business combination transactions for which the acquisition date is on or after the beginning of the first annual reporting period beginning on or after December 15, 2008 (our fiscal 2010). Early adoption of these statements is prohibited. We believe the adoption of these statements will have a material impact on significant acquisitions, if any, completed after September 1, 2009.
In March 2008, the FASB issued SFAS No. 161, Disclosures about Derivative Instruments and Hedging Activities, or SFAS 161. This statement will require enhanced disclosures about derivative instruments and hedging activities to enable investors to better understand their effects on an entity’s financial position, financial performance, and cash flows. It is effective for financial statements issued for fiscal years and interim periods beginning after November 15, 2008, with early application encouraged. We adopted SFAS 161 on December 1, 2008 and have determined that SFAS 161 had no material impact on our financial results for the year ended August 31, 2009.
In May 2008, FASB issued SFAS No. 162, The Hierarchy of Generally Accepted Accounting Principles, or SFAS 162. This standard is intended to improve financial reporting by identifying a consistent framework, or hierarchy, for selecting accounting principles to be used in preparing financial statements that are presented in conformity with U.S. GAAP for non-governmental entities. SFAS 162 is effective 60 days following the U.S. Securities and Exchange Commission’s approval of the Public Company Accounting Oversight Board amendments to AU Section 411, the meaning of “Present Fairly in Conformity with GAAP”. We have determined it did not have a material impact on our consolidated financial statements.

 

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In May 2008, the FASB released FSP APB 14-1 Accounting For Convertible Debt Instruments That May Be Settled in Cash Upon Conversion (Including Partial Cash Settlement), or FSP APB 14-1, that alters the accounting treatment for convertible debt instruments that allow for either mandatory or optional cash settlements. FSP APB 14-1 specifies that issuers of such instruments should separately account for the liability and equity components in a manner that will reflect the entity’s nonconvertible debt borrowing rate when interest cost is recognized in subsequent periods. Furthermore, it would require recognizing interest expense in prior periods pursuant to retrospective accounting treatment. FSP APB 14-1 is effective for financial statements issued for fiscal years beginning after December 15, 2008; therefore, we anticipate adopting FSP APB 14-1 as of September 1, 2009. We are in the process of evaluating the impact, if any, of FSP APB 14-1 on our consolidated financial statements.
In June 2008, the FASB issued EITF No. 07-5, or EITF 07-5, Determining Whether an Instrument (or Embedded Feature) is Indexed to an Entity’s Own Stock. EITF 07-5 requires entities to evaluate whether an equity-linked financial instrument (or embedded feature) is indexed to our own stock by assessing the instrument’s contingent exercise provisions and settlement provisions. Instruments not indexed to their own stock fail to meet the scope exception of SFAS No. 133, Accounting for Derivative Instruments and Hedging Activities , paragraph 11(a), and should be classified as a liability and marked-to-market. The statement is effective for fiscal years beginning after December 15, 2008 and interim periods within those fiscal years and is to be applied to outstanding instruments upon adoption with the cumulative effect of the change in accounting principle recognized as an adjustment to the opening balance of retained earnings. We are adopting EITF 07-5 as of September 1, 2009 and is in the process of evaluating the impact, if any, of EITF 07-5 on our consolidated financial statements.
In April 2008, the FASB issued FSP SFAS No. 142-3, Determination of the Useful Life of Intangible Assets, or FSP SFAS 142-3. FSP SFAS 142-3 provides guidance with respect to estimating the useful lives of recognized intangible assets acquired on or after the effective date and requires additional disclosure related to the renewal or extension of the terms of recognized intangible assets. FSP SFAS 142-3 is effective for fiscal years and interim periods beginning after December 15, 2008. We are adopting FSP SFAS 142-3 as of September 1, 2009 and are currently evaluating the impacts and disclosures of this standard, but do not expect FSP SFAS 142-3 to have a material impact on our consolidated financial statements.
In May 2009, the FASB issued SFAS No. 165, Subsequent Events, or SFAS 165. SFAS 165 establishes general standards of accounting for and disclosure of events that occur after the balance sheet date but before financial statements are issued or are available to be issued. SFAS 165 defines the period after the balance sheet date during which management of a reporting entity should evaluate events or transactions that may occur for potential recognition or disclosure in the financial statements, and the circumstances under which an entity should recognize events or transactions occurring after the balance sheet date in our financial statements. SFAS 165 is effective for fiscal years and interim periods ending after June 15, 2009. We adopted SFAS 165 as of August 31, 2009 and anticipate that the adoption will impact the accounting and disclosure of future transactions. Our management has evaluated and disclosed subsequent events from the balance sheet date of August 31, 2009 through October 27, 2009, the day before the date our consolidated financial statements included in this Annual Report on Form 10-K, were filed with the SEC.
FSP FAS 107-1 and APB 28-1 amends FASB Statement No. 107, Disclosures about Fair Value of Financial Instruments, to require disclosures about the fair value of financial instruments for interim reporting periods of publicly traded companies as well as in annual financial statements. This FSP also amends APB Opinion No. 28, Interim Financial Reporting, to require those disclosures in summarized financial information at interim reporting periods. The adoption of FSP FAS 107-1 did not have a material impact on our consolidated financial statements.
In June 2009, the FASB issued SFAS No. 167, Amendments to FASB Interpretation, or FIN, No. 46(R), or FIN 46(R), or SFAS 167. The amendments include: (1) the elimination of the exemption for qualifying special purpose entities, (2) a new approach for determining who should consolidate a variable-interest entity, and (3) changes to when it is necessary to reassess who should consolidate a variable-interest entity. This statement is effective for fiscal years beginning after November 15, 2009, and for interim periods within that first annual reporting period. We are currently evaluating the impact of this standard, however, we do not expect SFAS 167 to have a material impact on our consolidated financial statements.

 

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In June 2009, FASB issued SFAS No. 168, The FASB Accounting Standards CodificationTM and the Hierarchy of Generally Accepted Accounting Principles, a replacement of FASB Statement No. 162, or the Codification. The Codification, which was launched on July 1, 2009, became the single source of authoritative nongovernmental U.S. GAAP, superseding existing FASB, American Institute of Certified Public Accountants (AICPA), Emerging Issues Task Force (EITF) and related literature. The Codification eliminates the GAAP hierarchy contained in SFAS No. 162 and establishes one level of authoritative GAAP. All other literature is considered non-authoritative. This Statement is effective for financial statements issued for interim and annual periods ending after September 15, 2009. We will adopt this Statement for our first fiscal 2010 quarter ending November 30, 2009. There will be no change to our consolidated financial statements due to the implementation of this Statement.
ITEM 7A: QUANTITATIVE AND QUALITATIVE DISCLOSURES ABOUT MARKET RISK
Interest Rate Market Risk
Our exposure to market risk for changes in interest rates relates primarily to our investment portfolio. By policy, we place our investments with highly rated credit issuers and limit the amount of credit exposure to any one issuer. As stated in our policy, we seek to improve the safety and likelihood of preservation of our invested funds by limiting default risk and market risk.
We mitigate default risk by investing in high credit quality securities and by positioning our portfolio to respond appropriately to a significant reduction in a credit rating of any investment issuer or guarantor. The portfolio includes only marketable securities with active secondary or resale markets to ensure portfolio liquidity.
As of August 31, 2009, our investment portfolio does not include any investments with significant exposure to the subprime mortgage market issues. Based on our investment portfolio, which consists 100% of money market accounts, and interest rates at August 31, 2009, we believe that a 100 basis point decrease in interest rates could result in a potential loss of future interest income of approximately $37,000 annually, however it would have no effect on the fair value of the money market principal balances.
Of our total consolidated cash and cash equivalent balance of approximately $3.7 million as of August 31, 2009, our money market balances represent $3.5 million or 95%.
Our debt obligations consist of our capital lease to finance our photocopier, which carries a fixed imputed interest rate and, as a result, we are not exposed to interest rate market risk on our capital lease obligations. The carrying value of our capital lease obligation approximates its fair value at August 31, 2009.

 

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ITEM 8: FINANCIAL STATEMENTS AND SUPPLEMENTARY DATA
Documents filed as part of this annual report on Form 10-K:
Financial Statements
         
    Page  
    66  
 
       
    67  
 
       
    68  
 
       
    69  
 
       
    73  
 
       
    74  
 
       

 

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REPORT OF INDEPENDENT REGISTERED PUBLIC ACCOUNTING FIRM
To the Board of Directors and Stockholders of
Raptor Pharmaceuticals Corp.
We have audited the accompanying consolidated balance sheets of Raptor Pharmaceuticals Corp. and its subsidiaries (the “Company”) (a development stage enterprise) as of August 31, 2009 and 2008 and the related consolidated statements of operations, stockholders’ equity and cash flows for the years ended August 31, 2009 and 2008 and the cumulative amounts from September 8, 2005 (inception) to August 31, 2009. These consolidated financial statements are the responsibility of the Company’s management. Our responsibility is to express an opinion on these consolidated financial statements based on our audits.
We conducted our audits in accordance with the standards of the Public Company Accounting Oversight Board (United States). Those standards require that we plan and perform the audit to obtain reasonable assurance about whether the financial statements are free of material misstatement. The Company is not required to have, nor were we engaged to perform an audit of the Company’s internal control over financial reporting. Our audit included consideration of internal control over financial reporting as a basis for designing audit procedures that are appropriate in the circumstances, but not for the purpose of expressing an opinion on the effectiveness of the Company’s internal control over financial reporting. Accordingly, we express no such opinion. An audit includes examining, on a test basis, evidence supporting the amounts and disclosures in the consolidated financial statements, assessing the accounting principles used and significant estimates made by management, as well as evaluating the overall financial statement presentation. We believe that our audits provide a reasonable basis for our opinion.
In our opinion, the consolidated financial statements referred to above present fairly, in all material respects, the consolidated financial position of Raptor Pharmaceuticals Corp. and its subsidiaries as of August 31, 2009 and 2008 and the consolidated results of their operations and cash flows for the years ended August 31, 2009 and 2008 and the cumulative amounts from September 8, 2005 (inception) to August 31, 2009 in conformity with accounting principles generally accepted in the United States of America.
The accompanying consolidated financial statements have been prepared assuming that the Company will continue as a going concern. As discussed in Note 2 to the consolidated financial statements, the Company is in the development stage and has not generated any revenue to date. These factors raise substantial doubt about the Company’s ability to continue as a going concern. Management’s plans in regard to these matters are also described in Note 2. The financial statements do not include any adjustments relating to the recoverability and classification of asset carrying amounts or the amount and classification of liabilities that might result should the Company be unable to continue as a going concern.
As discussed in Note 14 to the consolidated financial statements, effective September 29, 2009, the Company completed a reverse merger with TorreyPines Therapeutics, Inc. The combined company is called Raptor Pharmaceutical Corp.
     
/s/ Burr, Pilger & Mayer LLP
   
San Francisco, California
   
October 27, 2009

 

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PART I — FINANCIAL INFORMATION
Item 1. Financial Statements.
Raptor Pharmaceuticals Corp.
(A Development Stage Company)
Consolidated Balance Sheets
                 
    August 31, 2009     August 31, 2008  
ASSETS
               
Current assets:
               
 
               
Cash and cash equivalents
  $ 3,701,787     $ 7,546,912  
Prepaid expenses and other
    107,054       115,594  
 
           
Total current assets
    3,808,841       7,662,506  
 
               
Intangible assets, net
    2,524,792       2,663,291  
Fixed assets, net
    144,735       194,766  
Deposits
    100,206       100,207  
 
           
Total assets
  $ 6,578,574     $ 10,620,770  
 
           
 
               
 
               
LIABILITIES AND STOCKHOLDERS’ EQUITY
               
 
               
Liabilities
               
Current liabilities:
               
 
               
Accounts payable
  $ 613,577     $ 565,593  
Accrued liabilities
    451,243       432,434  
Deferred rent
          2,951  
Capital lease liability — current
    4,117       2,302  
 
           
Total current liabilities
    1,068,937       1,003,280  
 
               
Capital lease liability — long-term
    6,676        
 
           
Total liabilities
    1,075,613       1,003,280  
 
           
Commitments and contingencies
               
 
               
Stockholders’ equity:
               
Preferred stock, $0.001 par value, 15,000,000 shares authorized, zero shares issued and outstanding
           
Common stock, $0.001 par value, 150,000,000 shares authorized 17,857,555 and 14,064,345 shares issued and outstanding as at August 31, 2009 and 2008, respectively
    17,858       14,064  
Additional paid-in capital
    27,364,286       22,258,715  
Deficit accumulated during development stage
    (21,879,183 )     (12,655,289 )
 
           
Total stockholders’ equity
    5,502,961       9,617,490  
 
           
 
               
Total liabilities and stockholders’ equity
  $ 6,578,574     $ 10,620,770  
 
           
The accompanying notes are an integral part of these financial statements.

 

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Raptor Pharmaceuticals Corp.
(A Development Stage Company)
Consolidated Statements of Operations
                         
                    For the cumulative  
                    period  
                    from September 8, 2005  
    For the year ended August 31,     (inception) to  
    2009     2008     August 31, 2009  
 
                       
Revenues:
  $     $     $  
 
                 
 
                       
Operating expenses:
                       
General and administrative
    2,687,993       2,229,140       6,956,240  
Research and development
    6,570,119       5,558,871       14,874,284  
In-process research and development
          240,625       240,625  
 
                 
Total operating expenses
    9,258,112       8,028,636       22,071,149  
 
                 
 
                       
Loss from operations
    (9,258,112 )     (8,028,636 )     (22,071,149 )
 
                       
Interest income
    36,744       77,871       301,903  
Interest expense
    (2,526 )     (103,198 )     (109,937 )
 
                 
Net loss
  $ (9,223,894 )   $ (8,053,963 )   $ (21,879,183 )
 
                 
 
                       
Net loss per share:
                       
Basic and diluted
  $ (0.64 )   $ (0.81 )        
 
                   
 
                       
Weighted average shares outstanding used to compute:
                       
Basic and diluted
    14,440,254       9,893,612          
 
                   
The accompanying notes are an integral part of these financial statements.

 

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Raptor Pharmaceuticals Corp.
(A Development Stage Company)
Consolidated Statements of Stockholders’ Equity
For the period from September 8, 2005 (inception) to August 31, 2006
                                                 
                                    Deficit        
                                    accumulated        
                    Additional     Receivable     during the        
    Common stock     paid-in     from     development        
    Shares     Amount     Capital     stockholders     stage     Total  
Balance at September 8, 2005, issuance of common stock to founders at $0.004 per share, net of retirement of common stock upon reverse merger
    1,398,740     $ 1,399     $ 8,601     $ (10,000 )   $     $  
 
                                               
Common stock issued in May 2006 at $0.43 per share pursuant to a stock purchase agreement dated February 2006
    233,123       233       99,767       (100,000 )            
 
                                               
Common stock issued in May 2006 at $0.86 per share pursuant to a stock purchase agreement dated February 2006
    233,123       233       199,767                   200,000  
 
                                               
Common stock issued on May 25, 2006 at $2.57 per share, net of fundraising costs of $217,534
    1,942,695       1,943       4,780,523                   4,782,466  
 
                                               
Common stock and warrants issued for a placement fee in connection with May 25, 2006 financing
    186,499       186       (186 )                  
 
                                               
Common stock issued in connection with reverse merger in May 2006
    2,914,042       2,914       (2,914 )                  
 
                                               
Warrant subscribed pursuant to a consulting agreement dated September 2005
                60                   60  
 
                                               
Consultant stock-based compensation expense
                23,500                   23,500  
 
                                               
Repayment of receivable from stockholders
                      110,000             110,000  
 
                                               
Net loss
                            (969,250 )     (969,250 )
 
                                   
 
                                               
Balance at August 31, 2006
    6,908,222     $ 6,908     $ 5,109,118     $     $ (969,250 )   $ 4,146,776  
 
                                   
The accompanying notes are an integral part of these financial statements.

 

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Raptor Pharmaceuticals Corp.
(A Development Stage Company)
Consolidated Statements of Stockholders’ Equity
For the year ended August 31, 2007
                                         
                            Deficit accumulated        
                    Additional     during the        
    Common stock     paid-in     development        
    Shares     Amount     Capital     stage     Total  
 
                                       
Balance at September 1, 2006
    6,908,222     $ 6,908     $ 5,109,118     $ (969,250 )   $ 4,146,776  
 
                                       
Exercise of common stock warrants
    765,422       766       1,969,234             1,970,000  
 
                                       
Exercise of common stock options
    3,380       3       8,697               8,700  
 
                                       
Consultant stock-based compensation expense
                95,731             95,731  
 
                                       
Employee stock-based compensation expense
                368,978             368,978  
 
                                       
Net loss
                      (3,632,076 )     (3,632,076 )
 
                             
 
                                       
Balance at August 31, 2007
    7,677,024     $ 7,677     $ 7,551,758     $ (4,601,326 )   $ 2,958,109  
 
                             
The accompanying notes are an integral part of these financial statements.

 

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Raptor Pharmaceuticals Corp.
(A Development Stage Company)
Consolidated Statements of Stockholders’ Equity
For the year ended August 31, 2008
                                         
                            Deficit accumulated        
                    Additional     during the        
    Common stock             paid-in     development        
    Shares     Amount     capital     stage     Total  
 
                                       
Balance at September 1, 2007
    7,677,024     $ 7,677     $ 7,551,758     $ (4,601,326 )   $ 2,958,109  
 
                                       
Exercise of common stock warrants
    747,938       748       1,924,252             1,925,000  
 
                                       
Consultant stock-based compensation expense
    2,040       2       240,227             240,229  
 
                                       
Employee stock-based compensation expense
    23,312       23       491,532             491,555  
 
                                       
Issuance of common stock for loan placement fee
    46,625       47       101,953             102,000  
 
                                       
Issuance of common stock for the purchase of Convivia, Inc. assets
    101,992       102       240,523             240,625  
 
                                       
Issuance of common stock for the merger with Encode Pharmaceuticals, Inc.
    802,946       803       2,657,197             2,658,000  
 
                                       
Issuance of common stock and warrants for the sale of units in a private placement at $2.14 per unit, including placement agent warrants, net of fundraising costs of $944,065
    4,662,468       4,662       9,051,273             9,055,935  
 
                                       
Net loss
                      (8,053,963 )     (8,053,963 )
 
                             
 
                                       
Balance at August 31, 2008
    14,064,345     $ 14,064     $ 22,258,715     $ (12,655,289 )   $ 9,617,490  
 
                             
The accompanying notes are an integral part of these financial statements.

 

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Raptor Pharmaceuticals Corp.
(A Development Stage Company)
Consolidated Statements of Stockholders’ Equity
For the year ended August 31, 2009
                                         
                            Deficit accumulated        
                    Additional     during the        
    Common stock     paid-in     development        
    Shares     Amount     Capital     stage     Total  
 
                                       
Balance at August 31, 2008
    14,064,345     $ 14,064     $ 22,258,715     $ (12,655,289 )   $ 9,617,490  
 
                                       
Exercise of common stock warrants
    2,031,671       2,032       2,612,468             2,614,500  
 
                                       
Consultant stock-based compensation expense
                  48,094             48,094  
 
                                       
Employee stock-based compensation expense
    23,312       23       354,471             354,494  
 
                                       
Issuance of common stock and warrants for the sale of units in a private placement at $1.37 per unit, including placement agent warrants, net of fundraising costs of $293,724
    1,738,227       1,739       2,090,538             2,092,277  
 
                                       
Net loss
                      (9,223,894 )     (9,223,894 )
 
                             
 
                                       
Balance at August 31, 2009
    17,857,555     $ 17,858     $ 27,364,286     $ (21,879,183 )   $ 5,502,961  
 
                             
The accompanying notes are an integral part of these financial statements.

 

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Raptor Pharmaceuticals Corp.
(A Development Stage Company)
Consolidated Statements of Cash Flows
                         
                    For the cumulative period  
                    from September 8,  
    For the year ended August 31,     2005 (inception) to  
    2009     2008     August 31, 2009  
Cash flows from operating activities:
                       
Net loss
  $ (9,223,894 )   $ (8,053,963 )   $ (21,879,183 )
Adjustments to reconcile net loss to net cash used in operating activities:
                       
Employee stock-based compensation exp.
    354,494       491,555       1,215,027  
Consultant stock-based compensation exp.
    48,094       240,229       407,614  
Amortization of intangible assets
    138,499       94,834       245,208  
Depreciation of fixed assets
    84,693       126,888       350,940  
In-process research and development
          240,625       240,625  
Amortization of capitalized finder’s fee
          102,000       102,000  
Capitalized acquisition costs previously expensed
          38,000       38,000  
Changes in assets and liabilities:
                       
Prepaid expenses
    8,540       81,500       (107,053 )
Intangible assets
                (150,000 )
Deposits
          (80,000 )     (100,206 )
Accounts payable
    47,984       449,914       613,576  
Accrued liabilities
    18,809       231,114       451,348  
Deferred rent
    (2,951 )     (8,064 )     (105 )
 
                 
Net cash used in operating activities
    (8,525,732 )     (6,045,368 )     (18,572,209 )
 
                 
Cash flows from investing activities:
                       
Purchase of fixed assets
    (22,734 )     (13,227 )     (476,350 )
 
                 
Cash flows from financing activities:
                       
Proceeds from the sale of common stock
    2,386,000       10,000,000       17,386,000  
Proceeds from the exercise of common stock warrants
    2,614,500       1,925,000       6,509,500  
Proceeds from the exercise of common stock options
                8,700  
Fundraising costs
    (293,724 )     (944,065 )     (1,455,323 )
Proceeds from the sale of common stock to initial investors
                310,000  
Proceeds from bridge loan
                200,000  
Repayment of bridge loan
                (200,000 )
Principal payments on capital lease
    (3,435 )     (2,500 )     (8,531 )
 
                 
Net cash provided by financing activities
    4,703,341       10,978,435       22,750,346  
 
                 
Net increase (decrease) in cash and cash equivalents
    (3,845,125 )     4,919,840       3,701,787  
Cash and cash equivalents, beginning of period
    7,546,912       2,627,072        
 
                 
Cash and cash equivalents, end of period
  $ 3,701,787       7,546,912     $ 3,701,787  
 
                 
Supplemental disclosure of non-cash financing activities:
                       
Acquisition of equipment in exchange for capital lease
  $ 14,006     $     $ 21,403  
 
                 
Interest paid
  $ 2,526     $ 1,198     $ 7.937  
 
                 
Notes receivable issued in exchange for common stock
  $     $     $ 110,000  
 
                 
Common stock issued for a finder’s fee
  $     $ 102,000     $ 102,000  
 
                 
Common stock issued in asset purchase
  $     $ 2,898,624     $ 2,898,624  
 
                 
The accompanying notes are an integral part of these financial statements.

 

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RAPTOR PHARMACEUTICALS CORP.
(A Development Stage Company)
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS
(1) NATURE OF OPERATIONS AND BUSINESS RISKS
The accompanying consolidated financial statements reflect the results of operations of Raptor Pharmaceuticals Corp. (the “Company” or “Raptor”) and have been prepared in accordance with the accounting principles generally accepted in the United States of America.
Raptor is a publicly-traded biotechnology company dedicated to speeding the delivery of new treatment options to patients by enhancing existing therapeutics through the application of highly specialized drug targeting platforms and formulation expertise. The Company focuses on underserved patient populations where it can have the greatest potential impact. Raptor’s preclinical division bioengineers novel drug candidates and drug-targeting platforms derived from the human receptor-associated protein (“RAP”) and related proteins, while Raptor’s clinical division advances clinical-stage product candidates towards marketing approval and commercialization. Raptor’s clinical programs include DR Cysteamine for the potential treatment of nephropathic cystinosis, non-alcoholic steatohepatitis (“NASH”), and Huntington’s Disease. Raptor also has two clinical stage product candidates in which the Company is seeking to out-license or a development partnership: ConviviaTM for the potential treatment of aldehyde dehydrogenase (“ALDH2”) deficiency; and Tezampanel and NGX426, a non-opioid solution designed to treat chronic pain. Raptor’s preclinical programs target cancer, neurodegenerative disorders and infectious diseases. HepTide™ is designed to utilize engineered RAP-based peptides conjugated to drugs to target delivery to the liver to potentially treat primary liver cancer and hepatitis. NeuroTrans™ represents engineered RAP peptides created to target receptors in the brain and are currently, in collaboration with Roche, undergoing preclinical evaluation for their ability to enhance the transport of therapeutics across the blood-brain barrier. WntTide™ is based upon Mesd and Mesd peptides that the Company is studying in a preclinical breast cancer model for WntTide™’s potential inhibition of Wnt signaling through LRP5, which may block cancers dependent on signaling through LRP5 or LRP6. Raptor is also examining Tezampanel and NGX426, for the treatment of thrombotic disorder. The Company’s fiscal year end is August 31.
The Company is subject to a number of risks, including: the need to raise capital through equity and/or debt financings; the uncertainty whether the Company’s research and development efforts will result in successful commercial products; competition from larger organizations; reliance on licensing proprietary technology of others; dependence on key personnel; uncertain patent protection; and dependence on corporate partners and collaborators.
See the section titled “Risk Factors” in Part I Item 1A of this Annual Report on Form 10-K.
(2) SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES
(a) Basis of Presentation
The Company’s consolidated financial statements include the accounts of the Company’s wholly owned subsidiaries, Raptor Discoveries Inc. (f/k/a Raptor Pharmaceutical Inc.) and Raptor Therapeutics Inc. (f/k/a Bennu Pharmaceuticals Inc.) incorporated in Delaware on September 8, 2005 (date of inception) and August 1, 2007, respectively. All inter-company accounts have been eliminated. The Company’s consolidated financial statements have been prepared on a going concern basis, which contemplates the realization of assets and the settlement of liabilities and commitments in the normal course of business. Through August 31, 2009, the Company had accumulated losses of approximately $21.9 million. Management expects to incur further losses for the foreseeable future. Management believes that the Company’s cash and cash equivalents at August 31, 2009 will be sufficient to meet the Company’s obligations into the first calendar quarter of 2010. The Company is currently in the process of reviewing strategic partnerships and collaborations in order to fully fund its preclinical and clinical programs through the end of 2010. If the Company is not able to close a strategic transaction, the Company anticipates raising additional capital in the fourth calendar quarter of 2009. Until the Company can generate sufficient levels of cash from its operations, the Company expects to continue to finance future cash needs primarily through proceeds from equity or debt financings, loans and collaborative agreements with corporate partners or through a business combination with a company that has such financing in order to be able to sustain its operations until the Company can achieve profitability and positive cash flows, if ever.

 

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RAPTOR PHARMACEUTICALS CORP.
(A Development Stage Company)
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS
On September 29, 2009, upon the closing of the merger with TorreyPines (as discussed further in the Note 14, Subsequent Events), Raptor’s stockholders exchanged each share of Raptor’s common stock into .2331234 shares of the post-merger company and the exercise prices and stock prices were divided by .2331234 to reflect the post-merger equivalent stock prices and exercise prices. Therefore, all shares of common stock and exercise prices of common stock options and warrants are reported in these consolidated financial statements on a post-merger basis.
The Company’s independent registered public accounting firm has audited our consolidated financial statements for the years ended August 31, 2009 and 2008. The October 27, 2009 audit opinion included a paragraph indicating substantial doubt as to the Company’s ability to continue as a going concern due to the fact that the Company is in the development stage and has not generated any revenue to date.
Management plans to seek additional debt and/or equity financing for the Company through private or public offerings or through a business combination or strategic partnership, but it cannot assure that such financing or transaction will be available on acceptable terms, or at all. The uncertainty of this situation raises substantial doubt about the Company’s ability to continue as a going concern. The accompanying consolidated financial statements do not include any adjustments that might result from the failure to continue as a going concern.
(b) Use of Estimates
The preparation of financial statements in conformity with United States generally accepted accounting principles requires management to make certain estimates and assumptions that affect the reported amounts of assets and liabilities, disclosure of contingent assets and liabilities as of the dates of the consolidated financial statements and the reported amounts of revenues and expenses during the reporting period. Actual results could differ from those estimates.
(c) Fair Value of Financial Instruments
The carrying amounts of certain of the Company’s financial instruments including cash and cash equivalents, prepaid expenses, accounts payable, accrued liabilities and capital lease liability approximate fair value due to their short maturities.
(d) Segment Reporting
The Company has determined that it operates in two operating segments, preclinical development and clinical development. Operating segments are components of an enterprise for which separate financial information is available and are evaluated regularly by the Company in deciding how to allocate resources and in assessing performance. The Company’s chief executive officer assesses the Company’s performance and allocates its resources. Below is a break-down of the Company’s net loss and total assets by operating segment:
                                                 
    For the year ended August 31,  
    2009     2008  
    Preclinical     Clinical     Total     Preclinical     Clinical     Total  
Net loss
  $ (2,920,598 )   $ (6,303,295 )   $ (9,223,894 )   $ (3,834,895 )   $ (4,219,068 )   $ (8,053,963 )
Total assets
    683,828       5,894,746       6,578,574       2,646,598       7,974,172       10,620,770  
(e) Cash and Cash Equivalents
The Company considers all highly liquid investments with original maturities of three months or less to be cash equivalents.

 

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RAPTOR PHARMACEUTICALS CORP.
(A Development Stage Company)
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS
(f) Intangible Assets
Intangible assets include the intellectual property and other rights relating to DR Cysteamine and to the RAP technology. The intangible assets are amortized using the straight-line method over the estimated useful life of 20 years, which is the life of the intellectual property patents. The 20 year estimated useful life is also based upon the typical development, approval, marketing and life cycle management timelines of pharmaceutical drug products.
(g) Fixed Assets
Fixed assets, which mainly consist of leasehold improvements, lab equipment, computer hardware and software and capital lease equipment, are stated at cost. Depreciation is computed using the straight-line method over the related estimated useful lives, except for leasehold improvements and capital lease equipment, which are depreciated over the shorter of the useful life of the asset or the lease term. Significant additions and improvements that have useful lives estimated at greater than one year are capitalized, while repairs and maintenance are charged to expense as incurred.
(h) Impairment of Long-Lived Assets
The Company evaluates its long-lived assets for indicators of possible impairment by comparison of the carrying amounts to future net undiscounted cash flows expected to be generated by such assets when events or changes in circumstances indicate the carrying amount of an asset may not be recoverable. Should an impairment exist, the impairment loss would be measured based on the excess carrying value of the asset over the asset’s fair value or discounted estimates of future cash flows. The Company has not identified any such impairment losses to date.
(i) Income Taxes
Income taxes are recorded under the liability method, under which deferred tax assets and liabilities are determined based on the difference between the financial statement and tax bases of assets and liabilities using enacted tax rates in effect for the year in which the differences are expected to affect taxable income. Valuation allowances are established when necessary to reduce deferred tax assets to the amount expected to be realized.
(j) Research and Development
The Company is an early development stage company. Research and development costs are charged to expense as incurred. Research and development expenses include scientists’ salaries, lab collaborations, preclinical studies, clinical trials, clinical trial materials, regulatory and clinical consultants, lab supplies, lab services, lab equipment maintenance and small equipment purchased to support the research laboratory, amortization of intangible assets and allocated executive, human resources and facilities expenses.
(k) In-Process Research and Development
The Company records in-process research and development expense for a product candidate acquisition where there is not more than one potential product or usage for the assets being acquired. The Company reviews each product candidate acquisition to determine if the purchase price should be expensed as in-process research and development or capitalized and amortized over the life of the asset.

 

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RAPTOR PHARMACEUTICALS CORP.
(A Development Stage Company)
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS
(l) Net Loss per Share
Net loss per share is calculated by dividing net loss by the weighted average shares of common stock outstanding during the period. Diluted net income per share is calculated by dividing net income by the weighted average shares of common stock outstanding and potential shares of common stock during the period. For all periods presented, potentially dilutive securities are excluded from the computation of fully diluted net loss per share as their effect is anti-dilutive. Potentially dilutive securities include:
                 
    August 31,  
    2009     2008  
Warrants to purchase common stock
    2,057,990       3,090,814  
Options to purchase common stock
    989,213       907,602  
 
           
Total potentially dilutive securities
    3,047,203       3,998,416  
 
           
     
(m)  
Stock Option Plan
Effective September 1, 2006, the Company adopted the provisions of SFAS No. 123 (revised 2004), Share-Based Payment (“SFAS 123R”) in accounting for its 2006 Equity Incentive Plan, as amended. Under SFAS 123R, compensation cost is measured at the grant date based on the fair value of the equity instruments awarded and is recognized over the period during which an employee is required to provide service in exchange for the award, or the requisite service period, which is usually the vesting period. The fair value of the equity award granted is estimated on the date of the grant. The Company previously applied Accounting Principles Board (“APB”) Opinion No. 25, Accounting for Stock Issued to Employees, and related interpretations and provided the required pro forma disclosures required by SFAS No. 123, Accounting for Stock-Based Compensation. The Company accounts for stock options issued to third parties, including consultants, in accordance with the provisions of the EITF Issue No. 96-18, Accounting for Equity Instruments That Are Issued to Other Than Employees for Acquiring, or in Conjunction with Selling Goods or Services. See Note 8, “Stock Option Plan” for further discussion of employee stock-based compensation.
(n) Recent Accounting Pronouncements
In September 2006, the Financial Accounting Standards Board (“FASB”) issued SFAS No. 157, Fair Value Measurements (“SFAS 157”). SFAS 157 defines fair value, establishes a framework for measuring fair value in accordance with generally accepted accounting principles, and expands disclosures about fair value measurements. SFAS 157 does not require any new fair value measurements; rather, it applies under other accounting pronouncements that require or permit fair value measurements. The provisions of SFAS 157 are to be applied prospectively as of the beginning of the fiscal year in which it is initially applied, with any transition adjustment recognized as a cumulative-effect adjustment to the opening balance of retained earnings. The provisions of SFAS 157 are effective for fiscal years beginning after November 15, 2007; therefore, the Company adopted SFAS 157 as of September 1, 2008 for financial assets and liabilities. In accordance with FASB Staff Position 157-2, Effective Date of FASB Statement No. 157, the Company elected to defer the adoption of the provisions of SFAS 157 for its non-financial assets and non-financial liabilities. See Note 5, Fair Value Measurements, regarding the disclosure of the Company’s value of its cash equivalents.
In February 2007, the FASB issued SFAS 159, The Fair Value Option for Financial Assets and Financial Liabilities, Including an Amendment of FASB Statement No. 115, which permits the measurement of many financial instruments and certain other asset and liabilities at fair value on an instrument-by-instrument basis (the fair value option). The guidance is applicable for fiscal years beginning after November 15, 2007; therefore, the Company adopted SFAS 159 as of September 1, 2008. The Company has determined that SFAS 159 had no material impact for the year ended August 31, 2009.
In June 2007, the Emerging Issues Task Force (“EITF”) reached a consensus on EITF No. 07-3, Accounting for Nonrefundable Advance Payments for Goods or Services to Be Used in Future Research and Development Activities (“EITF 07-3”). EITF 07-3 specifies the timing of expense recognition for non-refundable advance payments for goods or services that will be used or rendered for research and development activities. EITF 07-3 was effective for fiscal years beginning after December 15, 2007, and early adoption is not permitted; therefore, the Company adopted EITF 07-3 as of September 1, 2008. The Company has determined that EITF 07-3 had no material impact on its financial results for the year ended August 31, 2009.

 

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RAPTOR PHARMACEUTICALS CORP.
(A Development Stage Company)
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS
In December 2007, the EITF reached a consensus on EITF No. 07-1, Accounting for Collaborative Arrangements Related to the Development and Commercialization of Intellectual Property (“EITF 07-1”). EITF 07-1 discusses the appropriate income statement presentation and classification for the activities and payments between the participants in arrangements related to the development and commercialization of intellectual property. The sufficiency of disclosure related to these arrangements is also specified. EITF 07-1 is effective for fiscal years beginning after December 15, 2008. As a result, EITF 07-1 is effective for the Company as of September 1, 2009. The Company is currently evaluating the impact of EITF 07-1 on its financial position and results of operations. Based upon the nature of the Company’s business, EITF 07-1 could have a material impact on its financial position and consolidated results of operations in future years.
In December 2007, FASB issued SFAS 141(R) and SFAS No. 160, Accounting and Reporting of Non-controlling Interests in Consolidated Financial Statements, an amendment of ARB No. 51 (“SFAS 160”). These statements will significantly change the financial accounting and reporting of business combination transactions and non-controlling (or minority) interests in consolidated financial statements. SFAS 141(R) requires companies to: (i) recognize, with certain exceptions, 100% of the fair values of assets acquired, liabilities assumed, and non-controlling interests in acquisitions of less than a 100% controlling interest when the acquisition constitutes a change in control of the acquired entity; (ii) measure acquirer shares issued in consideration for a business combination at fair value on the acquisition date; (iii) recognize contingent consideration arrangements at their acquisition-date fair values, with subsequent changes in fair value generally reflected in earnings; (iv) with certain exceptions, recognize pre-acquisition loss and gain contingencies at their acquisition-date fair values; (v) capitalize in-process research and development (“IPR&D”) assets acquired; (vi) expense, as incurred, acquisition-related transaction costs; (vii) capitalize acquisition-related restructuring costs only if the criteria in SFAS No. 146, Accounting for Costs Associated with Exit or Disposal Activities, are met as of the acquisition date; and (viii) recognize changes that result from a business combination transaction in an acquirer’s existing income tax valuation allowances and tax uncertainty accruals as adjustments to income tax expense. SFAS 141(R) is required to be adopted concurrently with SFAS 160 and is effective for business combination transactions for which the acquisition date is on or after the beginning of the first annual reporting period beginning on or after December 15, 2008 (our fiscal 2010). Early adoption of these statements is prohibited. The Company believes the adoption of these statements will have a material impact on significant acquisitions completed after September 1, 2009.
In March 2008, the FASB issued SFAS No. 161, Disclosures about Derivative Instruments and Hedging Activities (“SFAS 161”). This statement will require enhanced disclosures about derivative instruments and hedging activities to enable investors to better understand their effects on an entity’s financial position, financial performance, and cash flows. It is effective for financial statements issued for fiscal years and interim periods beginning after November 15, 2008, with early application encouraged. The Company adopted SFAS 161 on December 1, 2008 and has determined that SFAS 161 had no material impact on its financial results for the year ended August 31, 2009.
In May 2008, FASB issued SFAS No. 162, The Hierarchy of Generally Accepted Accounting Principles (“SFAS 162”). This standard is intended to improve financial reporting by identifying a consistent framework, or hierarchy, for selecting accounting principles to be used in preparing financial statements that are presented in conformity with U.S. GAAP for non-governmental entities. SFAS No. 162 is effective 60 days following the U.S. Securities and Exchange Commission’s approval of the Public Company Accounting Oversight Board amendments to AU Section 411, the meaning of “Present Fairly in Conformity with GAAP”. The Company adopted SFAS 162 as of September 1, 2009 and has determined the adoption did not have a material impact on its consolidated financial statements.
In May 2008, the FASB released FASB Staff Position (“FSP”) APB 14-1 Accounting For Convertible Debt Instruments That May Be Settled in Cash Upon Conversion (Including Partial Cash Settlement) (“FSP APB 14-1”) that alters the accounting treatment for convertible debt instruments that allow for either mandatory or optional cash settlements. FSP APB 14-1 specifies that issuers of such instruments should separately account for the liability and equity components in a manner that will reflect the entity’s nonconvertible debt borrowing rate when interest cost is recognized in subsequent periods. Furthermore, it would require recognizing interest expense in prior periods pursuant to retrospective accounting treatment. FSP APB 14-1 is effective for financial statements issued for fiscal years beginning after December 15, 2008; therefore, the Company anticipates adopting FSP APB 14-1 as of September 1, 2009. The Company is in the process of evaluating the impact, if any, of FSP APB 14-1 on its consolidated financial statements.

 

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RAPTOR PHARMACEUTICALS CORP.
(A Development Stage Company)
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS
In June 2008, the FASB issued EITF No. 07-5 (“EITF 07-5”), Determining Whether an Instrument (or Embedded Feature) is Indexed to an Entity’s Own Stock. EITF 07-5 requires entities to evaluate whether an equity-linked financial instrument (or embedded feature) is indexed to its own stock by assessing the instrument’s contingent exercise provisions and settlement provisions. Instruments not indexed to their own stock fail to meet the scope exception of SFAS No. 133, Accounting for Derivative Instruments and Hedging Activities, paragraph 11(a), and should be classified as a liability and marked-to-market. The statement is effective for fiscal years beginning after December 15, 2008 and interim periods within those fiscal years and is to be applied to outstanding instruments upon adoption with the cumulative effect of the change in accounting principle recognized as an adjustment to the opening balance of retained earnings. The Company is adopting EITF 07-5 as of September 1, 2009 and is in the process of evaluating the impact, if any, of EITF 07-5 on its consolidated financial statements.
In April 2008, the FASB issued FSP SFAS No. 142-3, Determination of the Useful Life of Intangible Assets (“FSP SFAS 142-3”). FSP SFAS 142-3 provides guidance with respect to estimating the useful lives of recognized intangible assets acquired on or after the effective date and requires additional disclosure related to the renewal or extension of the terms of recognized intangible assets. FSP SFAS 142-3 is effective for fiscal years and interim periods beginning after December 15, 2008. The Company is adopting FSP SFAS 142-3 as of September 1, 2009 and is currently evaluating the impacts and disclosures of this standard, but does not expect FSP SFAS 142-3 to have a material impact on the Company’s consolidated financial statements.
In May 2009, the FASB issued SFAS No. 165, Subsequent Events (“SFAS 165”). SFAS 165 establishes general standards of accounting for and disclosure of events that occur after the balance sheet date but before financial statements are issued or are available to be issued. SFAS 165 defines the period after the balance sheet date during which management of a reporting entity should evaluate events or transactions that may occur for potential recognition or disclosure in the financial statements, and the circumstances under which an entity should recognize events or transactions occurring after the balance sheet date in its financial statements. SFAS 165 is effective for fiscal years and interim periods ending after June 15, 2009. The Company has adopted SFAS 165 as of August 31, 2009 and anticipates that the adoption will impact the accounting and disclosure of future transactions. The Company’s management has evaluated and disclosed subsequent events from the balance sheet date of August 31, 2009 through October 27, 2009, the day before the date that these consolidated financial statements were filed with the Securities and Exchange Commission in this Annual Report on Form 10-K.
FSP FAS 107-1 and APB 28-1 amends FASB Statement No. 107, Disclosures about Fair Value of Financial Instruments, to require disclosures about the fair value of financial instruments for interim reporting periods of publicly traded companies as well as in annual financial statements. This FSP also amends APB Opinion No. 28, Interim Financial Reporting, to require those disclosures in summarized financial information at interim reporting periods. The adoption of FSP FAS 107-1 did not have a material impact on the Company’s consolidated financial statements.
In June 2009, the FASB issued SFAS No. 167, Amendments to FASB Interpretation (“FIN”) No. 46(R) (“FIN 46(R)”), (“SFAS 167”). The amendments include: (1) the elimination of the exemption for qualifying special purpose entities, (2) a new approach for determining who should consolidate a variable-interest entity, and (3) changes to when it is necessary to reassess who should consolidate a variable-interest entity. This statement is effective for fiscal years beginning after November 15, 2009, and for interim periods within that first annual reporting period. The Company is currently evaluating the impact of this standard, however, it does not expect SFAS 167 will have a material impact on its consolidated financial statements.

 

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RAPTOR PHARMACEUTICALS CORP.
(A Development Stage Company)
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS
In June 2009, the Financial Accounting Standards Board (FASB) issued Statement of Financial Accounting Standards (SFAS) No. 168, The FASB Accounting Standards CodificationTM and the Hierarchy of Generally Accepted Accounting Principles, a replacement of FASB Statement No. 162, (the Codification). The Codification, which was launched on July 1, 2009, became the single source of authoritative nongovernmental U.S. GAAP, superseding existing FASB, American Institute of Certified Public Accountants (AICPA), Emerging Issues Task Force (EITF) and related literature. The Codification eliminates the GAAP hierarchy contained in SFAS No. 162 and establishes one level of authoritative GAAP. All other literature is considered non-authoritative. This Statement is effective for financial statements issued for interim and annual periods ending after September 15, 2009. The Company will adopt this Statement for its first fiscal 2010 quarter ending November 30, 2009. There will be no change to the Company’s consolidated financial statements due to the implementation of this Statement.
(3) INTANGIBLE ASSETS
On January 27, 2006, BioMarin Pharmaceutical Inc. (“BioMarin”) assigned the intellectual property and other rights relating to the RAP technology to the Company. As consideration for the assignment of the RAP technology, BioMarin will receive milestone payments based on certain financing and regulatory triggering events. No other consideration was paid for this assignment. The Company has recorded $150,000 of intangible assets on the consolidated balance sheets as of August 31, 2009 and 2008 based on the estimated fair value of its agreement with BioMarin.
On December 14, 2007, the Company acquired the intellectual property and other rights to develop DR Cysteamine to treat various indications from the University of California at San Diego (“UCSD”) by way of a merger with Encode Pharmaceuticals, Inc. (“Encode”), a privately held research and development company, which held the intellectual property license with UCSD. The intangible assets, recorded at approximately $2.6 million acquired in the merger with Encode, were primarily based on the value of the Company’s common stock and warrants issued to the Encode stockholders as reflected in the table below:
         
Raptor common stock issued (number of shares)
    802,946  
Raptor common stock issuable upon marketing approval by a regulatory agency of DR Cysteamine for Cystinosis (number of shares)
    81,910  
 
     
Total shares of common stock used to value the transaction
    884,856  
Average closing price of Raptor’s common stock 2 days before and after the close of the merger
  $ 2.514  
 
     
Value of Raptor common stock portion of transaction
  $ 2,224,254  
Value (based on Carpenter model) of warrants issued in connection with transaction, net of legal fees
    395,746  
 
     
Intangible asset (IP license) related to the Encode merger, gross
  $ 2,620,000  
Intangible asset related to NeuroTransTM purchase from BioMarin, gross
    150,000  
 
     
Total gross intangible assets
    2,770,000  
Less accumulated amortization
    (245,208 )
 
     
Intangible assets, net
  $ 2,524,792  
 
     
The intangible assets are being amortized monthly over 20 years, which are the life of the intellectual property patents and the estimated useful life. The 20 year estimated useful life is also based upon the typical development, approval, marketing and life cycle management timelines of pharmaceutical drug products. During the years ended August 31, 2009 and 2008 and the cumulative period from September 8, 2005 (inception) to August 31, 2009, the Company amortized $138,499, $94,834, and $245,208, respectively, of intangible assets to research and development expense.
The following table summarizes the actual and estimated amortization expense for our intangible assets for the periods indicated:
         
Amortization period   Amortization expense  
September 8, 2005 (inception) to August 31, 2006 – actual
  $ 4,375  
Fiscal year ending August 31, 2007 – actual
    7,500  
Fiscal year ending August 31, 2008 – actual
    94,833  
Fiscal year ending August 31, 2009 – actual
    138,500  
Fiscal year ending August 31, 2010 – estimate
    138,500  
Fiscal year ending August 31, 2011 – estimate
    138,500  
Fiscal year ending August 31, 2012 – estimate
    138,500  
Fiscal year ending August 31, 2013 – estimate
    138,500  
Fiscal year ending August 31, 2014 – estimate
    138,500  

 

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RAPTOR PHARMACEUTICALS CORP.
(A Development Stage Company)
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS
(4) FIXED ASSETS
Fixed assets consisted of:
                         
Category   August 31, 2009     August 31, 2008     Estimated useful lives
Leasehold improvements
  $ 113,422     $ 113,422     Shorter of life of asset or lease term
Office furniture
    3,188       3,188     7 years
Laboratory equipment
    277,303       277,303     5 years
Computer hardware and software
    80,437       59,703          
3 years
                       
Capital lease equipment
    14,006       7,397     Shorter of life of asset or lease term
 
                   
Total at cost
    488,356       461,013          
Less: accumulated depreciation
    (343,621 )     (266,247 )        
 
                   
Total fixed assets, net
  $ 144,735     $ 194,766          
 
                   
Depreciation expense for the years ended August 31, 2009 and 2008 and the cumulative period from September 8, 2005 (inception) to August 31, 2009 was $84,693, $126,888 and $350,940, respectively. Accumulated depreciation on capital lease equipment was $3,951 and $5,446 as of August 31, 2009 and 2008, respectively.
(5) FAIR VALUE MEASUREMENT
The Company uses a fair-value approach to value certain assets and liabilities. Fair value is the price that would be received to sell an asset or paid to transfer a liability in an orderly transaction between market participants at the measurement date. As such, fair value is a market-based measurement that should be determined based on assumptions that market participants would use in pricing an asset or liability. The Company uses a fair value hierarchy, which distinguishes between assumptions based on market data (observable inputs) and an entity’s own assumptions (unobservable inputs). The hierarchy consists of three levels:
 
Level one — Quoted market prices in active markets for identical assets or liabilities;
 
 
Level two — Inputs other than level one inputs that are either directly or indirectly observable; and
 
 
Level three — Unobservable inputs developed using estimates and assumptions, which are developed by the reporting entity and reflect those assumptions that a market participant would use.
Determining which category an asset or liability falls within the hierarchy requires significant judgment. The Company evaluates its hierarchy disclosures each quarter. Assets and liabilities measured at fair value on a recurring basis at August 31, 2009 are summarized as follows:
                                         
Assets   Level 1     Level 2     Level 3     August 31, 2009  
Fair value of cash equivalents
  $ 3,515,353     $             $     $ 3,515,353  
 
                             
Total
  $ 3,515,353     $ $           $     $ 3,513,353  
 
                             

 

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RAPTOR PHARMACEUTICALS CORP.
(A Development Stage Company)
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS
Cash equivalents represent the fair value of our investment in two money market accounts as of August 31, 2009
(6) ACCRUED LIABILITIES
Accrued liabilities consisted of:
                 
    August 31, 2009     August 31, 2008  
Legal fees primarily due to TorreyPines merger (for August 31, 2009)
  $ 195,552     $ 51,503  
TorreyPines joint proxy/prospectus
    109,011        
Salaries and wages
    57,351       44,165  
Accrued vacation
    38,109       17,728  
Consulting — research and development
    21,000       7,578  
Auditing and tax preparation fees
    19,720       66,307  
Patent costs
    10,500       10,000  
Clinical trial costs
          114,514  
Preclinical studies
          48,165  
Consulting — administrative
          30,000  
Lab reagents
          27,024  
Prepaid conference expense
          5,490  
Other
          9,960  
 
           
Total accrued liabilities
  $ 451,243     $ 432,434  
 
           
(7) IN-PROCESS RESEARCH AND DEVELOPMENT
On October 17, 2007, the Company purchased certain assets of Convivia, Inc. (“Convivia”) including intellectual property, know-how and research reports related to a product candidate targeting liver aldehyde dehydrogenase (“ALDH2”) deficiency, a genetic metabolic disorder. The Company issued an aggregate of 101,991 shares of its restricted, unregistered common stock to the seller and other third parties in settlement of the asset purchase. Pursuant to Financial Accounting Standard (“FAS”) 2 Paragraph 11(c), Intangibles Purchased From Others, the Company has expensed the value of the common stock issued in connection with this asset purchase as in-process research and development expense. The amount expensed was based upon the closing price of Raptor’s common stock on the date of the closing of the asset purchase transaction of $2.359 per share multiplied by the aggregate number of shares of Raptor common stock issued or 101,991 for a total expense of $240,625 recorded on Raptor’s consolidated statement of operations during the year ended August 31, 2008.
(8) STOCK OPTION PLAN
Effective September 1, 2006, the Company began recording compensation expense associated with stock options and other forms of equity compensation in accordance with SFAS 123R, as interpreted by Staff Accounting Bulletin No. 107 (“SAB 107”). Prior to September 1, 2006, the Company accounted for stock options according to the provisions of Accounting Principles Board (“APB”) Opinion No. 25, Accounting for Stock Issued to Employees, and related interpretations, and therefore no related compensation expense was recorded for awards granted with no intrinsic value. The Company adopted the modified prospective transition method provided for under SFAS 123R, and consequently has not retroactively adjusted results from prior periods. Under this transition method, compensation cost associated with stock options now includes: (1) quarterly amortization related to the remaining unvested portion of all stock option awards granted prior to September 1, 2006, based on the grant date value estimated in accordance with the original provisions of SFAS 123; and (2) quarterly amortization related to all stock option awards granted subsequent to September 1, 2006, based on the grant date fair value estimated in accordance with the provisions of SFAS 123R. In addition, the Company records consulting expense over the vesting period of stock options granted to consultants. The compensation expense for stock-based compensation awards includes an estimate for forfeitures and is recognized over the requisite service period of the options, which is typically the period over which the options vest, using the straight-line method. Employee stock-based compensation expense for the years ended August 31, 2009 and 2008 and for the cumulative period from September 8, 2005 (inception) to August 31, 2009 was $354,494, $491,555, and $1,215,027 of which cumulatively $1,029,990 was included in general and administrative expense and $185,037 was included in research and development expense. No employee stock compensation costs were recognized for the period from September 8, 2005 (inception) to August 31, 2006, which was prior to the Company’s adoption of SFAS 123R.

 

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RAPTOR PHARMACEUTICALS CORP.
(A Development Stage Company)
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS
Stock-based compensation expense was based on the Black-Scholes option-pricing model assuming the following:
                                 
            Expected              
    Risk-free     life of stock     Annual     Annual  
Period*   interest rate     option     volatility     turnover rate  
September 8, 2005 (inception) to August 31, 2006**
    5 %   10 years     100 %     0 %
 
                               
Quarter ended November 30, 2006
    5 %   8 years     100 %     10 %
 
                               
Quarter ended February 28, 2007
    5 %   8 years     100 %     10 %
 
                               
Quarter ended May 31, 2007
    5 %   8 years     100 %     10 %
 
                               
Quarter ended August 31, 2007
    4 %   8 years     100 %     10 %
 
                               
Quarter ended November 30, 2007
    3.75 %   8 years     109 %     10 %
 
                               
Quarter ended February 29, 2008
    2 %   8 years     119 %     10 %
 
                               
Quarter ended May 31, 2008
    2 %   8 years     121 %     10 %
 
                               
Quarter ended August 31, 2008
    2.5 %   8 years     128 %     10 %
 
                               
Quarter ended November 30, 2008
    1.5 %   7 years     170 %     10 %
 
                               
Quarter ended February 28, 2009
    2.0 %   7 years     220 %     10 %
 
                               
Quarter ended May 31, 2009
    2.6 %   7 years     233 %     10 %
 
                               
Quarter ended August 31, 2009
    3.2 %   7 years     240 %     10 %
     
*  
Dividend rate is 0% for all period presented.
 
**  
Stock-based compensation expense was recorded on the consolidated statements of operations commencing on the effective date of SFAS 123R, September 1, 2006. Prior to September 1, 2006, stock based compensation was reflected only in the footnotes to the consolidated statements of operations, with no effect on the consolidated statements of operations, per the guidelines of APB No. 25. Consultant stock-based compensation expense has been recorded on the consolidated statements of operations since inception.
If factors change and different assumptions are employed in the application of SFAS 123R, the compensation expense recorded in future periods may differ significantly from what was recorded in the current period.
The Company recognizes as an expense the fair value of options granted to persons who are neither employees nor directors. The fair value of expensed options was based on the Black-Scholes option-pricing model assuming the same factors shown in the stock-based compensation expense table above. Stock-based compensation expense for consultants for the years ended August 31, 2009 and 2008 and for the cumulative period from September 8, 2005 (inception) to August 31, 2009, were $48,094, $240,229 and $407,614, respectively, of which cumulatively $101,836 was included in general and administrative expense and $305,778 was included in research and development expense.

 

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RAPTOR PHARMACEUTICALS CORP.
(A Development Stage Company)
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS
A summary of the activity in the 2006 Equity Compensation Plan, as amended, is as follows:
                                 
                            Weighted average  
            Weighted average             fair value of options  
    Option shares     exercise price     Exercisable     granted  
Outstanding at September 8, 2005
                       
Granted
    580,108     $ 2.64           $ 2.47  
Exercised
                       
Canceled
                       
 
                             
Outstanding at August 31, 2006
    580,108     $ 2.64       4,010     $ 2.47  
Granted
    107,452     $ 2.56           $ 2.31  
Exercised
    (3,381 )   $ 2.57           $ 2.40  
Canceled
                         
 
                             
Outstanding at August 31, 2007
    684,179     $ 2.63       273,236     $ 2.45  
Granted
    223,439     $ 2.27           $ 2.21  
Exercised
                       
Canceled
                       
 
                             
Outstanding at August 31, 2008
    907,618     $ 2.54       600,837     $ 2.39  
Granted
    81,595     $ 1.13           $ 1.04  
Exercised
                       
Canceled
                       
 
                             
Outstanding at August 31, 2009
    989,213     $ 2.42       826,303     $ 2.28  
 
                             
The weighted average intrinsic values of stock options outstanding and expected to vest and stock options exercisable as of August 31, 2009 and 2008 were $57,039 and $5,500 respectively.
There were 406,147 options available for grant under the 2006 Equity Compensation Plan, as amended, as of August 31, 2009. As of August 31, 2009, the options outstanding consisted of the following:
                                         
    Options outstanding     Options exercisable  
            Weighted                
            average                
    Number of     remaining     Weight     Number of     Weighted  
Range of   options     contractual life     average exercise     options     average exercise  
exercise prices   outstanding (#)     (yrs.)     price ($)     exercisable (#)     price ($)  
$0 to $1.50
    81,595       9.46       1.13       21,855       1.43  
$1.51 to $2.00
    32,058       8.95       1.88       8,012       1.88  
$2.01 to $2.50
    199,424       8.31       2.34       155,711       2.37  
$2.51 to $3.00
    676,136       6.87       2.63       640,725       2.63  
 
                                   
 
    989,213       7.44       2.42       826,303       2.54  
 
                                   
At August 31, 2009, the total unrecognized compensation cost was approximately $205,000. The weighted average period over which it is expected to be recognized is 2.75 years.

 

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RAPTOR PHARMACEUTICALS CORP.
(A Development Stage Company)
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS
(9) INCOME TAXES
The provision for income taxes differs from the amount estimated by applying the statutory federal income tax rate to income (loss) before taxes as follows:
                                 
    August 31,  
    2009     2008  
 
                               
Federal tax (Benefit) at statutory rate
  $ (3,132,608 )     -34.00 %   $ (2,738,000 )     -34.00 %
State tax (benefit) at statutory rate, net of federal tax benefit
    (629,304 )     -6.83 %     (470,000 )     -5.83 %
Change in valuation allowance
    5,069,715       55.02 %     3,208,000       39.83 %
 
                               
Research and development credits
    (1,325,036 )     -14.38 %           0.00 %
Other
    17,233       0.19 %           0.00 %
 
                       
 
                               
Provision for Income Taxes
  $ (0 )     (0 )   $ 0       0  
 
                       
Deferred tax assets (liabilities) consist of the following (in thousands):
                 
    August 31,  
    2009     2008  
Deferred Tax Assets
               
Net Operating Loss Carryforwards
  $ 4,722,078     $ 3,248,000  
Capitalized Start-Up Costs
    1,615,625       612,000  
Stock Option Expense
    207,169       114,000  
Research Credit
    2,223,767       84,000  
Capital Loss Carryforwards
    47,600       0  
Basis Difference for Fixed Assets and Intangibles
    277,941        
Accruals
    24,823        
Valuation Allowance
    (9,119,003 )     (4,058,000 )
 
           
 
               
Gross Deferred Tax Asset
  $ 0     $ 0  
 
           
As of August 31, 2009, the Company had net operating loss carryforwards for federal and state income tax purposes of approximately $11.6 million and $13.3 million respectively, which expire beginning after the year 2022 and 2016, respectively. As of August 31, 2009, the Company had federal and state research and development credits of $2.2 million and $.1 million respectively. The federal credits expire beginning after the year 2026 and the state credits have no expiration.
The valuation allowance increased approximately $5.1 million during the period ending August 31, 2009, primarily as a result of current year losses.
Utilization of the Company’s net operating loss may be subject to substantial annual limitation due to the ownership change limitations provided by the Internal Revenue Code and similar state provisions. Such an annual limitation could result in the expiration of the net operating loss before utilization.

 

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RAPTOR PHARMACEUTICALS CORP.
(A Development Stage Company)
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS
In July 2006, the FASB released Final Interpretation No. 48, Accounting for Uncertainty in Income Taxes (“FIN 48”). FIN 48 prescribes the minimum recognition threshold a tax position is required to meet before being recognized in the financial statements. This interpretation also provides guidance on the recognition of income tax assets and liabilities, classification of current and deferred income tax assets and liabilities, accounting for interest and penalties associated with tax positions, accounting for income taxes in interim periods, and income tax disclosures. FIN 48 also requires additional disclosure of the beginning and ending unrecognized tax benefits and details regarding the uncertainties that may cause the unrecognized benefits to increase or decrease within a twelvemonth period. FIN 48 is effective for fiscal years beginning after December 15, 2006, with the cumulative effect of the change in accounting principle, if any, recorded as an adjustment to opening retained earnings.
The Company adopted FIN 48 as of September 1, 2007. As a result of the implementation, the Company recognized no adjustment in the liability for unrecognized income tax benefits. The Company’s policy will be to recognize interest and penalties related to income taxes in income tax expense. The Company is not aware of any pending income tax audits. Significant components of the Company’s deferred tax assets for income tax purposes are net operating loss carryforwards, capitalized start-up costs, stock-based compensation and research credits. Due to the Company’s lack of earning history, any deferred assets recorded have been fully offset by a valuation allowance.
(10) ISSUANCE OF COMMON STOCK
ISSUANCE OF COMMON STOCK PURSUANT TO COMMON STOCK WARRANT EXERCISES AND STOCK OPTION EXERCISES
During the cumulative period from September 8, 2005 (inception) through August 31, 2009, the Company received $3.895 million from the exercises of common stock warrants issued in the Company’s May 2006 financing. The Company issued an aggregate of 1,513,359 shares of common stock for the warrants, which had an exercise price of $2.57 per share and expired on November 25, 2007.
During the cumulative period from September 8, 2005 (inception) through August 31, 2009, the Company received $8,700 from the exercise of stock options resulting in the issuance of 3,380 shares of common stock. Total common stock outstanding as of August 31, 2009 was 17,857,555 shares.
ISSUANCE OF COMMON STOCK PURSUANT TO AN ASSET PURCHASE AGREEMENT WITH CONVIVIA, INC.
On October 18, 2007, the Company purchased certain assets of Convivia, Inc. (“Convivia”) including intellectual property, know-how and research reports related to a product candidate targeting liver aldehyde dehydrogenase (“ALDH2”) deficiency, a genetic metabolic disorder. The Company hired Convivia’s chief executive officer and founder, Thomas E. (Ted) Daley, as President of its clinical division. In exchange for the assets related to the ALDH2 deficiency program, the Company issued to Convivia 46,625 shares of its restricted, unregistered common stock, an additional 46,625 shares of its restricted, unregistered common stock to a third party in settlement of a convertible loan between the third party and Convivia, and another 8,742 shares of restricted, unregistered common stock in settlement of other obligations of Convivia. Mr. Daley, as the former sole stockholder of Convivia (now dissolved), may earn additional shares of the Company based on certain triggering events or milestones related to the development of Convivia assets. In addition, Mr. Daley may earn cash bonuses based on the same triggering events pursuant to his employment agreement. In January 2008, Mr. Daley earned a $30,000 cash bonus pursuant to his employment agreement for executing the Patheon formulation agreement for manufacturing ConviviaTM. In March 2008, Mr. Daley earned a $10,000 cash bonus pursuant to his employment agreement and was issued 23,312 shares of valued at $56,000 based on the execution of an agreement to supply the Company with the active pharmaceutical ingredient for ConviviaTM pursuant to the asset purchase agreement. In October 2008, Mr. Daley was issued 23,312 shares of restricted Raptor common stock valued at $27,000 and earned a $30,000 cash bonus (pursuant to Mr. Daley’s employment agreement) pursuant to the fulfillment of a clinical milestone. Pursuant to FAS 2, Paragraph 11(c), the accounting guidelines for expensing research and development costs, the Company has expensed the value of the stock issued in connection with this asset purchase (except for milestone bonuses, which are expensed as compensation expense) as in-process research and development expense in the amount of $240,625 on its consolidated statement of operations for the year ended August 31, 2008.

 

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RAPTOR PHARMACEUTICALS CORP.
(A Development Stage Company)
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS
MERGER OF RAPTOR’S CLINICAL DEVELOPMENT SUBSIDIARY AND ENCODE PHARMACEUTICALS, INC.
On December 14, 2007, the Company entered into a Merger Agreement (the “Merger Agreement”), dated as of the same date, by and between the Company, its clinical development subsidiary and Encode Pharmaceuticals, Inc. (“Encode”), a privately held development stage company. Pursuant to the Merger Agreement, a certificate of merger was filed with the Secretary of State of the State of Delaware and Encode was merged with and into the Company’s clinical development subsidiary. The existence of Encode ceased as of the date of the Merger Agreement. Pursuant to the Merger Agreement and the certificate of merger, the Company’s clinical development subsidiary, as the surviving corporation, continued as a wholly-owned subsidiary of the Company. Under the terms of and subject to the conditions set forth in the Merger Agreement, the Company issued 802,946 shares of restricted, unregistered shares of the Company’s common stock, par value $.001 per share (the “Common Stock”) to the stockholders of Encode (the “Encode Stockholders”), options (“Company Options”) to purchase 83,325 shares of Common Stock to the optionholders of Encode (the “Encode Optionholders”), and warrants (“Company Warrants”) to purchase 256,034 restricted, unregistered shares of Common Stock to the warrantholders of Encode (the “Encode Warrantholders”, and together with the Encode Stockholders and Encode Optionholders, the “Encode Securityholders”), as of the date of such Agreement. Such Common Stock, Company Options to purchase Common Stock, and Company Warrants to purchase Common Stock combine for an aggregate amount of 1,142,305 shares of Common Stock issuable to the Encode Securityholders as of the closing of the Merger. The purchase price was valued at $2.6 million, which is reflected as intangible assets on the Company’s consolidated balance sheet as of August 31, 2008, primarily based on the value the Company’s common stock and warrants issued to Encode stockholders. The Encode Securityholders are eligible to receive up to an additional 559,496 shares of Common Stock, Company Options and Company Warrants to purchase Common Stock in the aggregate based on certain triggering events related to regulatory approval of DR Cysteamine, an Encode product program described below, if completed within the five year anniversary date of the Merger Agreement. The Company recorded this transaction as an asset purchase rather than a business combination, as Encode had not commenced planned principle operations, such as generating revenues from its drug product candidate.
As a result of the Merger, the Company received the exclusive worldwide license to DR Cysteamine (“License Agreement”), developed by clinical scientists at the UCSD, School of Medicine. DR Cysteamine is a proprietary enterically coated formulation of cysteamine bitartrate, a cystine depleting agent currently approved by the U.S. Food and Drug Administration (“FDA”). Cysteamine bitartrate is prescribed for the management of the genetic disorder known as nephropathic cystinosis (“cystinosis”), a lysosomal storage disease. The active ingredient in DR Cysteamine has also demonstrated potential in studies as a treatment for other metabolic and neurodegenerative diseases, such as Huntington’s Disease and Non-alcoholic steatohepatitis (“NASH”).
In consideration of the grant of the license, the Company will be obligated to pay an annual maintenance fee until it begins commercial sales of any products developed pursuant to the License Agreement. In addition to the maintenance fee, the Company will be obligated to pay during the life of the License Agreement: milestone payments ranging from $20,000 to $750,000 for orphan indications and from $80,000 to $1,500,000 for non-orphan indications upon the occurrence of certain events, if ever; royalties on commercial net sales from products developed pursuant to the License Agreement ranging from 1.75% to 5.5%; a percentage of sublicense fees ranging from 25% to 50%; a percentage of sublicense royalties; and a minimum annual royalty commencing the year the Company begins commercially selling any products pursuant to the License Agreement, if ever. Under the License Agreement, the Company is obligated to fulfill predetermined milestones within a specified number of years ranging from 0.75 to 6 years from the effective date of the License Agreement, depending on the indication. To the extent that the Company fails to perform any of the obligations, UCSD may terminate the license or otherwise cause the license to become non-exclusive. To-date, Raptor has paid $250,000 in milestone payments to UCSD based upon the initiation of clinical trials in cystinosis and in NASH.

 

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RAPTOR PHARMACEUTICALS CORP.
(A Development Stage Company)
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS
ISSUANCES OF COMMON STOCK AND WARRANTS IN CONNECTION WITH THE SALE OF UNITS IN A PRIVATE PLACEMENT
On May 21, 2008 (the “Initial Closing”) Raptor entered into a Securities Purchase Agreement (the “Purchase Agreement”), with eight investors (the “Initial Investors”) for the private placement of units of the Company, each unit comprised of one share of Raptor’s Common Stock and one warrant to purchase one half of one share of Raptor’s Common Stock, at a purchase price of $2.14 per unit. Immediately subsequent to the Initial Closing, the Company and each Initial Investor entered into an Amendment to the Securities Purchase Agreement, principally in order to increase the amount able to be raised by the Company in the private placement and to extend the outside closing date of such private placement (the “Amendment”, and, together with the Purchase Agreement, the “Amended Purchase Agreement”), dated as of May 21, 2008.
At the Initial Closing, the Company sold an aggregate of 1,030,405 shares of Common Stock (the “Initial Shares”) to the Initial Investors for aggregate gross proceeds of $2,210,000 and issued to the Initial Investors warrants (the “Initial Warrants”). The Initial Warrants, exercisable for two years from the Initial Closing, entitle the Initial Investors to purchase up to an aggregate of 515,203 shares of Common Stock of the Company (the “Initial Warrant Stock”) and have an exercise price of either $3.22 or $3.86 per share, depending on when such Initial Warrants are exercised, if at all, and were valued at approximately $675,000 (using the following Black -Scholes pricing model assumptions: risk-free interest rate 2%; expected term 2 years and annual volatility 121.45%).
On May 30, 2008 Raptor sold an additional $150,000 of units at the same terms as outlined in the May 21, 2008 closing discussed above. As a result, the Company issued 69,937 shares of the Company’s Common Stock and warrants to purchase 34,969 shares of the Company’s Common Stock valued at approximately $40,000 (using the following Black -Scholes pricing model assumptions: risk-free interest rate 2%; expected term 2 years and annual volatility 121.45%).
On June 27, 2008 Raptor sold an additional $7,640,000 of units at the same terms as outlined in the May 21, 2008 closing discussed above. As a result, the Company issued 3,562,126 shares of the Company’s Common Stock and warrants to purchase 1,781,063 shares of the Company’s Common Stock valued at approximately $2.3 million (using the following Black -Scholes pricing model assumptions: risk-free interest rate 2%; expected term 2 years and annual volatility 121.45%).
In connection with the May / June 2008 private placement, the Company issued warrants and a cash fee to placement agents to compensate them for placing investors into the financing. Placement agents were issued warrants exercisable for 7% of Common Stock issued and issuable under the warrants issued to investors as part of the financing units and a cash fee based upon the proceeds of the sale of the units of the private placement. In connection with the sale of units, the Company issued placement agent warrants to purchase 489,559 shares of Raptor’s Common Stock at an exercise price of $2.36 per share for a five year term (valued at approximately $960,000 using the following Black -Scholes pricing model assumptions: risk-free interest rate 2%; expected term 5 years and annual volatility 121.45%) and cash fees to placement agents totaling $700,000. Of the placement agents compensated, Limetree Capital was issued warrants to purchase 438,890 shares of Raptor’s Common Stock and cash commission of $627,550. One of our Board members serves on the board of Limetree Capital.
On April 29, 2009, in order to reflect current market prices, Raptor notified the holders of warrants purchased in the May/June 2008 private placement that the Company was offering, in exchange for such warrants, new warrants to purchase its common stock at an exercise price of $1.29 per share, but only to the extent such exchange of the original warrants and exercise of the new warrants, including the delivery of the exercise price, occurred on or prior to July 17, 2009. The new warrants were valued at approximately $2.3 million based on the following Black -Scholes pricing model assumptions: risk-free interest rate 0.55%; expected term 1 year and annual volatility 231.97%. The warrants that were not exchanged prior to or on July 17, 2009 retained their original exercise prices of $3.86 per share and original expiration date of May 21, 2010. The Company received $2,614,500 of proceeds from warrant exercises that resulted in the issuance of 2,031,670 shares of Raptor’s common stock pursuant to the exchange described above.

 

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RAPTOR PHARMACEUTICALS CORP.
(A Development Stage Company)
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS
On August 21, 2009 Raptor entered into a securities purchase agreement, dated as of August 21, 2009, with four investors for the private placement of units of the Company at a purchase price of $1.37 per unit, each unit comprised of one share of Raptor’s common stock, par value $0.001 per share and one warrant to purchase one half of one share of Raptor’s common stock. Pursuant to the securities purchase agreement, the Company sold an aggregate of 1,738,226 units to the investors for aggregate gross proceeds of $2,386,000. The 1,738,226 units comprised of an aggregate of 1,738,226 shares of common stock and warrants to purchase up to 869,113 shares of Raptor’s common stock valued at $1.0 million (using the following Black -Scholes pricing model assumptions: risk-free interest rate 1.11%; expected term 2 years and annual volatility 240.29%). The warrants, exercisable for two years from the closing, entitle the investors to purchase, in the aggregate, up to 869,113 shares of Raptor’s common stock and have an exercise price of either $2.57 until the first anniversary of issuance or $3.22 per share after the first anniversary of issuance.
In connection with the August 2009 private placement, the Company issued warrants and a cash fee to Limetree Capital as its sole placement agent to compensate them for placing investors into the financing. Limetree Capital was issued warrants exercisable for 7% of common stock issued and issuable under the warrants issued to investors as part of the financing units and a 3.5% cash fee based upon the proceeds of the sale of the units of the August 2009 private placement. Limetree Capital was issued a five-year warrant to purchase 129,733 shares of Raptor’s Common Stock at an exercise price of $1.50 per share (valued at approximately $171,000 using the following Black - -Scholes pricing model assumptions: risk-free interest rate 2.58%; expected term 5 years and annual volatility 240.29%) and cash commission of $59,360. One of our Board members serves on the board of Limetree Capital.
The following is a summary of common stock outstanding as of August 31, 2009:
             
        Common Stock  
Transaction   Date   Issued  
 
           
Founders’ shares
  Sept. 2005     1,398,742  
Seed round
  Feb. 2006     466,247  
PIPE concurrent with reverse merger
  May 2006     1,942,695  
Shares issued in connection with reverse merger
  May 2006     3,100,541  
Warrant exercises
  Jan. – Nov. 2007     1,513,359  
Stock option exercises
  Mar. 2007     3,380  
Loan finder’s fee
  Sept. 2007     46,625  
Convivia asset purchase
  Oct. 2007 – Nov. 2008     148,616  
Encode merger DR Cysteamine asset purchase
  Dec. 2007     802,946  
Shares issued pursuant to consulting agreement
  May 2008     2,040  
PIPE — initial tranche
  May 2008     1,030,405  
PIPE — second tranche
  May 2008     69,937  
PIPE — third tranche
  June 2008     3,562,126  
Warrant exercises from warrant exchange
  June/July 2009     2,031,670  
PIPE
  August 2009     1,738,226  
 
         
Total shares of common stock outstanding
        17,857,555  
 
         

 

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RAPTOR PHARMACEUTICALS CORP.
(A Development Stage Company)
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS
(11) WARRANTS
The table reflects the number common stock warrants outstanding as of August 31, 2009:
                         
    Number of              
    shares              
    exercisable     Exercise price     Expiration date  
Summary of outstanding warrants:
                       
Issued in lieu of deferred legal fees
    13,987     $ 2.57       2/13/2011  
Issued in connection with Encode merger
    22,725     $ 2.40       12/13/2015  
Issued in connection with Encode merger
    233,309     $ 2.87       12/13/2015  
Issued to PIPE investors in May / June 2008
    299,564     $ 3.86       5/21/2010  
Issued to placement agents in May / June 2008
    489,559     $ 2.36       5/21/2013  
Issued to PIPE investors in August 2009
    869,113     $ 2.57/$3.22 *     8/21/2011  
Issued to placement agents in August 2009
    129,733     $ 1.50       8/21/2014  
 
                     
Total warrants outstanding
    2,057,990     $ 2.67 **        
 
                     
     
*  
First year exercisable at $2.57; second year exercisable at $3.22
 
**  
Average exercise price
(12) COMMITMENTS AND CONTINGENCIES
CONTRACTUAL OBLIGATIONS WITH BIOMARIN
Pursuant to the terms of the asset purchase agreement the Company entered into with BioMarin Pharmaceutical Inc. (“BioMarin”) for the purchase of intellectual property related to our receptor-associated protein (“RAP”) based technology (including NeuroTrans™), we are obligated to make the following milestone payments to BioMarin upon the achievement of the following events:
$50,000 (paid by the Company in June 2006) within 30 days after Raptor receives total aggregate debt or equity financing of at least $2,500,000;
$100,000 (paid by the Company in June 2006) within 30 days after Raptor receives total aggregate debt or equity financing of at least $5,000,000;
$500,000 upon the Company’s filing and acceptance of an investigational new drug application for a drug product candidate based on the NeuroTrans™ product candidate;
$2,500,000 upon the Company’s successful completion of a Phase II human clinical trial for a drug product candidate based on the NeuroTrans™ product candidate;
$5,000,000 upon on the Company’s successful completion of a Phase III human clinical trial for a drug product candidate based on the NeuroTrans™ product candidate;
$12,000,000 within 90 days of the Company’s obtaining marketing approval from the FDA or other similar regulatory agencies for a drug product candidate based on the NeuroTrans™ product candidate;
$5,000,000 within 90 days of the Company’s obtaining marketing approval from the FDA or other similar regulatory agencies for a second drug product candidate based on the NeuroTrans™ product candidate;
$5,000,000 within 60 days after the end of the first calendar year in which the Company’s aggregated revenues derived from drug product candidates based on the NeuroTrans™ product candidate exceed $100,000,000; and
$20,000,000 within 60 days after the end of the first calendar year in which the Company’s aggregated revenues derived from drug product candidates based on the NeuroTrans™ product candidate exceed $500,000,000.

 

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RAPTOR PHARMACEUTICALS CORP.
(A Development Stage Company)
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS
In addition to these milestone payments, the Company is also obligated to pay BioMarin a royalty at a percentage of the Company’s aggregated revenues derived from drug product candidates based on the NeuroTransTM product candidate. On June 9, 2006, the Company made a milestone payment in the amount of $150,000 to BioMarin because the Company raised $5,000,000 in its May 25, 2006 private placement financing. If the Company becomes insolvent or if the Company breaches its asset purchase agreement with BioMarin due to non-payment and the Company does not cure its non-payment within the stated cure period, all of the Company’s rights to the RAP technology (including NeuroTransTM) will revert back to BioMarin.
CONTRACTUAL OBLIGATIONS WITH THOMAS E. DALEY (ASSIGNEE OF THE DISSOLVED CONVIVIA, INC.)
Pursuant to the terms of the asset purchase agreement (“Asset Purchase Agreement”), the Company entered into with Convivia, Inc. and Thomas E. Daley for the purchase of intellectual property related to its 4-MP product candidate program, Mr. Daley will be entitled to receive the following, if at all, in such amounts and only to the extent certain future milestones are accomplished by the Company (or any of its subsidiaries thereof), as set forth below:
23,312 shares of Raptor’s restricted, unregistered Common Stock within fifteen (15) days after the Company enters into a manufacturing license or other agreement to produce any product that is predominantly based upon or derived from any assets purchased from Convivia (“Purchased Assets”) in quantity (“Product”) if such license agreement is executed within one (1) year of execution of the Asset Purchase Agreement or, if thereafter, 11,656 shares of Raptor’s restricted, unregistered Common Stock. Should the Company obtain a second such license or agreement for a Product, Mr. Daley will be entitled to receive 11,656 shares of the Company’s restricted, unregistered Common Stock within 30 days of execution of such second license or other agreement. On March 31, 2008, the Company issued 23,312 shares of Raptor’s Common Stock valued at $56,000 to Mr. Daley pursuant to this milestone reflecting the execution of an agreement to supply the active pharmaceutical ingredient for ConviviaTM, combined with the execution of a formulation agreement to produce the oral formulation of ConviviaTM.
23,312 shares of the Company’s restricted, unregistered Common Stock within fifteen (15) days after it receives its first patent allowance on any patents which constitute part of the Purchased Assets in any one of certain predetermined countries (“Major Market”).
11,656 shares of the Company’s restricted, unregistered Common Stock within fifteen (15) days after the Company receives its second patent allowance on any patents which constitute part of the Purchased Assets different from the patent referenced in the immediately preceding bullet point above in a Major Market.
23,312 shares of the Company’s restricted, unregistered Common Stock within fifteen (15) days of completing predetermined benchmarks in a Major Market by the Company or its licensee of the first phase II human clinical trial for a Product (“Successful Completion”) if such Successful Completion occurs within one (1) year of execution of the Asset Purchase Agreement or, if thereafter, 11,656 shares of the Company’s restricted, unregistered Common Stock within thirty (30) days of such Successful Completion. In October 2008, the Company issued 23,312 shares of Raptor’s Common Stock valued at $27,000 and a $30,000 cash bonus (pursuant to Mr. Daley’s employment agreement) to Mr. Daley pursuant to the fulfillment of this milestone.
11,656 shares of the Company’s restricted, unregistered Common Stock within fifteen (15) days of a Successful Completion in a Major Market by the Company’s or its licensee of the second phase II human clinical trial for a Product (other than the Product for which a distribution is made under the immediately preceding bullet point above).
23,312 shares of the Company’s restricted, unregistered Common Stock within fifteen (15) days after the Company or its licensee applies for approval to market and sell a Product in a Major Market for the indications for which approval is sought (“Marketing Approval”).

 

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RAPTOR PHARMACEUTICALS CORP.
(A Development Stage Company)
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS
11,656 shares of the Company’s restricted, unregistered Common Stock within fifteen (15) days after the Company or its licensee applies for Marketing Approval in a Major Market (other than the Major Market for which a distribution is made under the immediately preceding bullet point above).
46,625 shares of the Company’s restricted, unregistered Common Stock within fifteen (15) days after the Company or its licensee obtains the first Marketing Approval for a Product from the applicable regulatory agency in a Major Market.
23,312 shares of the Company’s restricted, unregistered Common Stock within fifteen (15) days after the Company or its licensee obtains Marketing Approval for a Product from the applicable regulatory agency in a Major Market (other than the Major Market for which a distribution is made under the immediately preceding bullet point above).
As discussed above, in aggregate, the Company has issued to Mr. Daley, 46,625 shares of Raptor’s common stock valued at $83,000 and paid $30,000 in cash bonuses related to ConviviaTM milestones along with another $20,000 in cash bonuses related to employment milestones pursuant to Mr. Daley’s employment agreement.
CONTRACTUAL OBLIGATIONS WITH FORMER ENCODE STOCKHOLDERS AND UCSD RELATING TO THE ACQUISITION OF THE DR CYSTEAMINE LICENSE
As a result of the merger between our clinical subsidiary and Encode, as discussed in Note 10 above, the Encode Securityholders are eligible to receive up to an additional 559,496 shares of Raptor’s common stock, Company Options and Company Warrants to purchase Raptor’s common stock in the aggregate based on certain triggering events related to regulatory approval of DR Cysteamine, an Encode product program, if completed within the five year anniversary date of the merger agreement.
Also as a result of the merger, the Company will be obligated to pay an annual maintenance fee to UCSD for the exclusive license to develop DR Cysteamine for certain indications of $15,000 until it begins commercial sales of any products developed pursuant to the License Agreement. In addition to the maintenance fee, the Company will be obligated to pay during the life of the License Agreement: milestone payments ranging from $20,000 to $750,000 for orphan indications and from $80,000 to $1,500,000 for non-orphan indications upon the occurrence of certain events, if ever; royalties on commercial net sales from products developed pursuant to the License Agreement ranging from 1.75% to 5.5%; a percentage of sublicense fees ranging from 25% to 50%; a percentage of sublicense royalties; and a minimum annual royalty commencing the year we begin commercially selling any products pursuant to the License Agreement, if ever. Under the License Agreement, the Company is obligated to fulfill predetermined milestones within a specified number of years ranging from 0.75 to 6 years from the effective date of the License Agreement, depending on the indication. In addition, the Company is obligated to, among other things, secure $1 million in funding prior to December 18, 2008 (which the Company has fulfilled by raising $10 million in its May/June 2008 private placement) and annually spend at least $200,000 for the development of products (which, as of its fiscal year ended August 31, 2009, the Company has fulfilled by spending approximately $4.1 million on such programs) pursuant to the License Agreement. To-date, we have paid $250,000 in milestone payments to UCSD based upon the initiation of clinical trials in cystinosis and in NASH. To the extent that the Company fails to perform any of its obligations under the License Agreement, then UCSD may terminate the license or otherwise cause the license to become non-exclusive.
OFFICE LEASES
In March 2006, the Company entered into a lease for the Company’s executive offices and research laboratory in Novato, California. Base monthly payments were $5,206 per month subject to annual rent increase of between 3% to 5%, based on the Consumer Price Index (“CPI”). In March 2006, the Company paid $20,207 as a security deposit on this lease, which expired in March 2009. Effective April 1, 2007, the Company leased additional office space adjoining the existing leased space, increasing our base rent to $9,764 per month without extending the term of the original lease. The original lease allows for one three-year extension at the market rate and up to $18,643 in reimbursement for tenant improvements. In June 2008, the Company’s rent increased to $10,215 reflecting a CPI increase of 3% plus an increase in operating costs for the period from April 1, 2008 to March 31, 2009. In September 2008, the Company executed a lease addendum replacing the one three-year extension with two two-year extensions commencing on April 1, 2009 and renegotiated the first two-year extension base rent to $10,068 with an adjustment after the first year for CPI between 3% (minimum) and 5% (maximum). During the year ended August 31, 2009 and 2008 and the cumulative period from September 8, 2005 (inception) to August 31, 2009, the Company paid $128,830, $128,268 and $368,395, respectively, in rent.

 

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RAPTOR PHARMACEUTICALS CORP.
(A Development Stage Company)
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS
The minimum future lease payments under this operating lease assuming a 3% CPI increase per year are as follows:
         
Period   Amount  
Fiscal year ending August 31, 2010
  $ 124,226  
September 1, 2010 to March 31, 2011
    73,698  
CAPITAL LEASE
In June 2006, the Company leased a photocopier machine for 36 months at $242 per month. There was no purchase option at the end of the lease. Based on the fair value and estimated useful life of the photocopier and the life of the lease and the photocopier, the Company has accounted for the lease as a capital lease. In September 2008, the Company replaced the originally leased photocopier with a new photocopier which is subject to a 39-month lease at $469 per month. There were no penalties imposed for cancelling the original lease.
The future lease payments under the capital lease are as follows:
         
Period   Amount  
Fiscal year ending August 31, 2010
  $ 5,625  
Fiscal year ending August 31, 2011
    5,625  
September 1, 2011 to December 31, 2011
    1,875  
 
     
Total future capital lease payments
    13,125  
Less interest
    (2,333 )
 
     
Total current and long-term capital lease liability
  $ 10,792  
 
     
Interest rate on the capital lease is 17% based on the lessor’s implicit rate of return.
RESEARCH COLLABORATION AGREEMENTS
In September 2008, the Company signed a research collaboration agreement with a research hospital. The research collaboration agreement requires the Company to pay an aggregate of $150,000 over one year, which includes the salary, benefits and overhead of one research scientist along with laboratory equipment and supplies necessary to carry out the research at the university. During the year ended August 31, 2009, the Company entered into two preclinical research contracts to tests its proprietary molecules.
The future commitments pursuant to the research agreement are as follows:
         
Period   Amount  
September 1, 2009 through August 31, 2010
  $ 77,484  

 

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RAPTOR PHARMACEUTICALS CORP.
(A Development Stage Company)
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS
CLINICAL STUDY AGREEMENTS
In November 2008, the Company sponsored a clinical study with the University of California, San Diego to study a prototype formulation of DR Cysteamine in NASH patients. In May 2009, the Company entered into a clinical study collaboration agreement with the University of California, San Diego, to study DR Cysteamine in patients with cystinosis. Also in May 2009, the Company entered into an agreement with a clinical research organization to monitor the cystinosis trial. The future commitments pursuant to these clinical study agreements are as follows:
         
Period   Amount  
September 1, 2009 through August 31, 2010
  $ 487,436  
FORMULATION / MANUFACTURING AGREEMENTS
In April 2008, the Company executed an agreement with a contract manufacturing organization to formulate and manufacture DR Cysteamine for its cystinosis program. The costs are invoiced to the Company in installments throughout the formulation and manufacturing process. Also in July 2008, the Company executed a supply agreement with a contract manufacturer for the active pharmaceutical agreement of DR Cysteamine. The future commitments pursuant to these contracts are as follows:
         
Period   Amount  
September 1, 2009 through August 31, 2010
  $ 1,244,783  
(13) RELATED PARTY TRANSACTIONS
Pursuant to the terms of the Share Purchase Agreement, the Company issued to each of Drs. Starr and Zankel (its Chief Executive Officer and its Chief Scientific Officer, respectively) 699,370 shares of the Company’s common stock and to Erich Sager (one of the Company’s directors) 233,123 shares of its common stock. Mr. Sager purchased his shares pursuant to a promissory note when the Company was privately held in February 2006 in the amount of $100,000 plus accrued interest at 8% per annum. Mr. Sager repaid $50,000 of the note on February 8, 2006, another $50,000 on March 9, 2006 and $373 of accrued interest on April 11, 2006. Drs. Starr and Zankel and Mr. Sager did not own any shares of the Company’s common stock at the time when the Share Purchase Agreement was first approved and executed.
In connection with the May / June 2008 private placement, the Company issued to Limetree Capital warrants to purchase 438,890 shares of Raptor’s Common Stock and $627,550 in cash commissions. In connection with the August 2009 private placement, the Company issued to Limetree Capital warrants to purchase 129,733 shares of Raptor’s Common Stock and $59,360 in cash commissions. Also, commencing on April 1, 2009, we engaged Limetree to support our investor relations efforts in Europe for a retainer of $2,500 per month. Through August 31, 2009, we have paid $12,500 in such fees to Limetree. One of our Board members serves on the Board of Limetree Capital.
In the ordinary course of business, Raptor’s officers occasionally utilize their personal credit cards or cash to pay for expenses on behalf of the Company and the Company reimburses the officers within 30 days.
(14) SUBSEQUENT EVENTS
The Company’s management has evaluated and disclosed subsequent events from the balance sheet date of August 31, 2009 through October 27, 2009, the day before the date that these financial statements were filed with the Securities and Exchange Commission in the Annual Report on Form 10-K.
On September 29, 2009, Raptor and TorreyPines Therapeutics, Inc. completed a reverse merger. The combined company is named “Raptor Pharmaceutical Corp.” and commenced trading on September 30, 2009 on the NASDAQ Capital Market under the ticker symbol “RPTP.” Pursuant to NASDAQ’s regulations, for the first 20 trading days the ticker symbol will be “RPTPD”. Effective October 27, 2009, Raptor’s ticker symbol changed to “RPTP”.

 

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RAPTOR PHARMACEUTICALS CORP.
(A Development Stage Company)
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS
In connection with the exchange of shares in the merger, Raptor and TorreyPines stockholders own 95% and 5% of the outstanding shares of the combined company, respectively. Raptor stockholders received 17,881,300 shares of the combined company’s common stock in exchange for the 76,703,147 shares of Raptor common stock outstanding immediately prior to the closing of the merger. On September 29, 2009, TorreyPines’ board of directors, with the consent of Raptor’s board of directors, acted to effect a reverse stock split of the issued and outstanding shares of TorreyPines’ common stock such that every 17 shares of TorreyPines’ common stock outstanding immediately prior to the effective time of the Merger would represent one share of TorreyPines’ common stock. Due to the reverse stock split implemented by TorreyPines, the 15,999,058 shares of TorreyPines common stock outstanding immediately prior to the closing of the merger became 941,121 shares of the combined company’s common stock.
In connection with the merger and subject to the same conversion factor as the Raptor common stock (.2331234), the combined company assumed all of Raptor’s stock options and warrants outstanding at the time of the merger. The combined company also retained the TorreyPines stock options and warrants outstanding at the merger, subject to the same adjustment factor as the TorreyPines common stock to give effect to the 1 for 17 reverse split.
The combined company is headquartered in Novato, California and is managed by Raptor’s existing management team including Christopher M. Starr, Ph.D., as Chief Executive Officer and director, Todd C. Zankel, Ph.D., as Chief Scientific Officer, Kim R. Tsuchimoto, C.P.A., as Chief Financial Officer, Ted Daley, as President of the clinical division and Patrice P. Rioux., M.D., Ph.D., as Chief Medical Officer of the clinical division.
There were a number of factors on which Raptor’s board of directors relied in approving the merger, including, having access to an expanded pipeline of product candidates and having development capabilities across a wider spectrum of diseases and markets. Another primary reason for Raptor’s board of directors’ decision to merge with TorreyPines was the benefit anticipated from the additional liquidity expected from Raptor’s assumption of TorreyPines’ NASDAQ listing. This liquidity benefit is the primary factor behind the goodwill recognized in the transaction (see below). The goodwill has been assigned to our clinical segment and is expected to be fully deductible for tax purposes. Below is a preliminary purchase consideration and breakdown of the assets acquired in the merger with TorreyPines (in millions, except for %):
         
Purchase Consideration (post-merger shares/share price) Closing price of TorreyPines on September 29, 2009 (date of closing of merger)
  $ 4.23  
TorreyPines shares outstanding on September 29, 2009
    941,121  
 
     
Subtotal
  $ 4.00 million  
Value of options and warrants assumed
  0.44 million  
Liabilities assumed
  0.59 million  
 
     
Total preliminary purchase consideration
  $ 5.03 million  
 
     
                 
Asset Allocation   Value (millions)     %  
Cash and equivalents
  $ 0.58       12  
Other current assets
    0.07       1  
Accrued liabilities
    (0.06 )     -1  
 
           
Working capital
    0.59       12  
Intangible assets:
               
In-process research & development
    0.90       18  
Licenses
    0.24       5  
 
           
Total identifiable assets
    1.73       35  
Plus Goodwill
    3.30       65  
 
           
Total assets acquired
  $ 5.03       100  
 
           

 

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RAPTOR PHARMACEUTICALS CORP.
(A Development Stage Company)
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS
Acquisition costs incurred by the Company related to the merger are expected to be approximately $0.5 million.
If the reverse merger had occurred on September 1, 2007, the Company’s revenues would have increase by approximately $2.3 million from license and option fees earned by TorreyPines in 2008 for total pro forma revenues of $2.3 million for the year ended August 31, 2008. Net loss would have decreased by approximately $1.5 million due to an increase of revenues of $2.3 million described above partially offset by $0.8 million in transaction costs and costs associated with obligations owed to the TorreyPines employees for a pro forma net loss of $6.6 million for the year ended August 31, 2008. For the year ended August 31, 2009, the Company’s revenues would have increased by approximately $1.8 million from other revenues earned by TorreyPines for total pro forma revenues of $1.8 million. Net loss would have decreased by approximately $2.1 million due to the increase of revenues of $1.8 million as described above, plus the reduction of $0.3 million of transaction costs which would have occurred during our year ended August 31, 2008, for a pro forma net loss of $7.1 million.
On October 1, 2009, Raptor Pharmaceutical Corp. announced the appointment of Llew Keltner, M.D., Ph.D., to the Company’s board of directors. Dr. Keltner is currently CEO and President of Light Sciences Oncology, a privately-held biotechnology company developing a late-stage, light-activated therapy for hepatocellular cancer and other solid tumors. He is also CEO of EPISTAT, an international healthcare technology transfer, corporate risk management and healthcare strategy company that he founded in 1972.
 

 

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ITEM 9: CHANGES IN AND DISAGREEMENTS WITH ACCOUNTANTS ON ACCOUNTING AND FINANCIAL DISCLOSURE
None.
ITEM 9A(T): CONTROLS AND PROCEDURES
As of August 31, 2009, we performed an evaluation of the effectiveness of our disclosure controls and procedures that are designed to ensure that the information required to be disclosed in the reports that we file or submit under the Securities Exchange Act of 1934, as amended, is recorded, processed, summarized and reported within the time periods specified in the SEC’s rules and forms. Disclosure controls and procedures include, without limitation, controls and procedures designed to ensure that information required to be disclosed by us in the reports that we file or submit under the Securities Exchange Act of 1934, as amended, is accumulated and communicated to the our management, including our principal executive and principal financial officers, or persons performing similar functions, as appropriate to allow timely decisions regarding required disclosure. Based on our evaluation, our management, including our Chief Executive Officer and Chief Financial Officer, has concluded that our disclosure controls and procedures (as defined in Rules 13a-15(e) and 15d-15(e) of the Securities Exchange Act of 1934, as amended) as of August 31, 2009, are effective at such reasonable assurance level.
Our management, under the supervision of our Chief Executive Officer and Chief Financial Officer, is responsible for establishing and maintaining adequate internal control over our financial reporting, as defined in Rules 13a-15(f) and 15d-15(f) of the Securities Exchange Act of 1934, as amended. The Company’s internal control over financial reporting is defined as a process designed to provide reasonable assurance regarding the reliability of financial reporting and the preparation of financial statements for external purposes in accordance with generally accepted accounting principles. Internal control over financial reporting includes policies and procedures that: (i) pertain to the maintenance of records that in reasonable detail accurately and fairly reflect our transactions and asset dispositions; (ii) provide reasonable assurance that transactions are recorded as necessary to permit the preparation of our financial statements in accordance with generally accepted accounting principles, and that our receipts and expenditures are being made only in accordance with authorizations of our management and directors; and (iii) provide reasonable assurance regarding the prevention or timely detection of unauthorized acquisition, use or disposition of assets that could have a material effect on our financial statements.
Due to its inherent limitations, internal control over financial reporting may not prevent or detect misstatements. Also, projections of any evaluation of effectiveness to future periods are subject to the risk that controls may become inadequate because of changes in conditions, or that the degree of compliance with the policies or procedures may deteriorate.
Under the supervision and with the participation of our management, including our Chief Executive Officer and Chief Financial Officer, we conducted an evaluation of the effectiveness of our internal control over financial reporting based on the framework in Internal Control—Integrated Framework issued by the Committee of Sponsoring Organizations of the Treadway Commission. Based on our evaluation under the framework in Internal Control—Integrated Framework, our management concluded that our internal control over financial reporting was effective as of August 31, 2009.
This Annual Report does not include an attestation report of our registered public accounting firm regarding internal control over financial reporting. Management’s report was not subject to attestation by our registered public accounting firm pursuant to temporary rules of the SEC that permit us to provide only management’s report in this Annual Report.
Changes in Internal Control Over Financial Reporting
During the most recent fiscal quarter, there have not been any significant changes in our internal control over financial reporting or in other factors that have materially affected, or are reasonably likely to materially affect, our internal control over financial reporting.

 

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ITEM 9B. OTHER INFORMATION
None.
PART III
ITEM 10: DIRECTORS, EXECUTIVE OFFICERS AND CORPORATE GOVERNANCE
In accordance with General Instructions G(3) of Form 10-K, we incorporate information regarding our directors, executive officers and corporate governance into this section by reference from section captioned “Directors, Executive Officers and Corporate Governance” in the amendment to this Form 10-K to be filed with the SEC not later than 120 days after the end of our fiscal year, August 31, 2009.
ITEM 11: EXECUTIVE COMPENSATION
In accordance with General Instructions G(3) of Form 10-K, we incorporate information regarding executive compensation into this section by reference from the section captioned “Executive Compensation” in the amendment to this Form 10-K to be filed with the SEC not later than 120 days after the end of our fiscal year, August 31, 2009.
ITEM 12: SECURITY OWNERSHIP OF CERTAIN BENEFICIAL OWNERS AND MANAGEMENT AND RELATED STOCKHOLDER MATTERS
In accordance with General Instructions G(3) of Form 10-K, we incorporate information regarding security ownership of certain beneficial owners and management and related stockholder matters into this section by reference from the section captioned “Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters” in the amendment to this Form 10-K to be filed with the SEC not later than 120 days after the end of our fiscal year, August 31, 2009.
ITEM 13: CERTAIN RELATIONSHIPS AND RELATED TRANSACTIONS, AND DIRECTOR INDEPENDENCE
In accordance with General Instructions G(3) of Form 10-K, we incorporate information regarding certain relationships and related transactions, and director independence into this section by reference from the section captioned “Certain Relationships and Related Transactions, and Director Independence” in the amendment to this Form 10-K to be filed with the SEC not later than 120 days after the end of our fiscal year, August 31, 2009.
ITEM 14: PRINCIPAL ACCOUNTING FEES AND SERVICES
In accordance with General Instructions G(3) of Form 10-K, we incorporate information regarding our principal accounting fees and services into this section by reference from the section captioned “Principal Accounting Fees and Services” in the amendment to this Form 10-K to be filed with the SEC not later than 120 days after the end of our fiscal year, August 31, 2009.

 

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PART IV
ITEM 15: EXHIBITS, FINANCIAL STATEMENT SCHEDULES
The following documents are filed as a part of this Report:
Financial Statements
The following consolidated financial statements of Raptor Pharmaceuticals Corp. and the Independent Registered Public Accounting Firm’s Report issued thereon, are incorporated by reference in Part II, Item 8:
         
    Page  
    66  
 
       
    67  
 
       
    68  
 
       
    69  
 
       
    73  
 
       
    74  
 
       

 

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Exhibits
                                     
            Filed    
Exhibit       Here   Incorporated by Reference
No.   Exhibit Description   with   Form   File No.   Exhibit   Filing Date   Filed By
  2.1    
Share Purchase Agreement dated May 2, 2006 by and between the Highland Clan Creations Corp. and Universal Financial Consulting Inc.
      8-K   000-50720     10.1     5/4/06   Registrant
  2.2    
Share Purchase Agreement dated as of March 9, 2006 by and among Highland Clan Creations Corp., Christopher Starr, Erich Sager, Todd Zankel and Falcon Corporate Investments Ltd.
      8-K   000-50720     10.1     3/14/06   Registrant
  2.3    
Agreement and Plan of Merger between Highland Clan Creations Corp. and Raptor Pharmaceuticals Corp. Effective May 15, 2006
      Schedule 14C   000-50720   Schedule A   5/17/06   Registrant
  2.4    
Agreement and Plan of Merger and Reorganization, dated July 27, 2009, by and among the Registrant, TorreyPines Therapeutics, Inc. and ECP Acquisition, Inc., a Delaware corporation
      8-K   000-50720     2.1     7/28/09   Registrant
  3.1    
Certificate of Incorporation of the Registrant
      Schedule 14C   000-50720   Schedule B   5/17/06   Registrant
  3.2    
Bylaws of the Registrant
      Schedule 14C   000-50720   Schedule C   5/17/06   Registrant
  3.3    
Certificate of Designation of Series A Junior Participating Preferred Stock of the Registrant
      8-A   000-50720     3.1     12/9/08   Registrant
  4.2    
Rights Agreement, between the Registrant and Nevada Agency and Trust Company, dated May 13, 2005
      8-A   000-50720     4.1     12/9/08   Registrant
  4.3    
Form of Rights Certificate of the Registrant (included in Exhibit 4.2)
                          Registrant
  4.4    
Amendment to Rights Agreement of the Registrant
      8-K   000-50720     4.1     7/28/09   Registrant
  4.5    
Warrant to purchase common stock dated December 14, 2007 issued to Flower Ventures, LLC
      10QSB/A   000-50720     4.1     4/15/08   Registrant
  4.6    
Warrant to purchase common stock dated December 14, 2007 issued to ICON Partners, LP
      10QSB/A   000-50720     4.2     4/15/08   Registrant
  4.7    
Securities Purchase Agreement, dated as of May 21, 2008, by and among the Registrant and the investors listed on the signature pages thereto
      10-QSB   000-50720     10.1     7/9/08   Registrant
  4.8    
Amendment to Securities Purchase Agreement, dated May 21, 2008, by and among the Registrant and the investors listed on the signature pages thereto
      10-QSB   000-50720     10.2     7/9/08   Registrant

 

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Table of Contents

                                     
            Filed    
Exhibit       Here   Incorporated by Reference
No.   Exhibit Description   with   Form   File No.   Exhibit   Filing Date   Filed By
  4.9    
Form of 2008 Warrant to Purchase Shares of Common Stock of the Registrant
      8-K   000-50720     4.1     5/22/08   Registrant
  4.10    
Form of 2008 Placement Agent Warrant to Purchase Shares of Common Stock of the Registrant
      8-K/A   000-50720     4.2     5/28/08   Registrant
  4.11    
Form of Warrant Exchange Agreement, dated July 17, 2009
  X                        
  4.12    
Securities Purchase Agreement, dated August 21, 2009, by and among the Registrant and the investors listed on the signature pages thereto
  X                        
  4.13    
Form of 2009 Warrant to Purchase Shares of Common Stock of the Registrant
      8-K   000-50720     4.1     8/25/09   Registrant
  4.14    
Form of 2009 Placement Agent Warrant to Purchase Shares of Common Stock of the Registrant
      8-K   000-50720     4.2     8/25/09   Registrant
  10.1    
Employment Agreement between the Registrant and Dr. Christopher Starr dated May 1, 2006
      8-K   000-50720     10.5     5/26/06   Registrant
  10.2    
First Amendment to the Employment Agreement between the Registrant and Dr. Christopher Starr effective January 1, 2009
      8-K   000-50720     10.1     1/5/09   Registrant
  10.3    
Employment Agreement between the Registrant and Dr. Todd Zankel dated May 15, 2006
      8-K   000-50720     10.6     5/26/06   Registrant
  10.4    
First Amendment to the Employment Agreement between the Registrant and Dr. Todd Zankel effective January 1, 2009
      8-K   000-50720     10.3     1/5/09   Registrant
  10.5    
Employment Agreement between the Registrant and Ms. Kim Tsuchimoto dated May 1, 2006
      8-K   000-50720     10.7     5/26/06   Registrant
  10.6    
First Amendment to the Employment Agreement between the Registrant and Ms. Kim Tsuchimoto effective January 1, 2009
      8-K   000-50720     10.7     1/5/09   Registrant

 

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            Filed    
Exhibit       Here   Incorporated by Reference
No.   Exhibit Description   with   Form   File No.   Exhibit   Filing Date   Filed By
  10.7    
Employment Agreement between Raptor Therapeutics and Thomas E. Daley dated September 7, 2007
      10QSB   000-50720     10.1     1/14/08   Registrant
  10.8    
First Amendment to the Employment Agreement between Raptor Therapeutics and Thomas E. Daley effective January 1, 2009
      8-K   000-50720     10.4     1/5/09   Registrant
  10.9    
Offer Letter from Raptor Therapeutics dated as of April 8, 2009 for Patrice Rioux, M.D., Ph.D
      8-K   000-50720     10.1     4/14/09   Registrant
  10.10    
2006 Equity Incentive Plan, as amended
      S-8   333-140944     4.3     2/28/07   Registrant
  10.11    
2008 Plan Amendment to 2006 Equity Incentive Plan
      10-K/A   000-50720     10.5     12/23/08   Registrant
  10.12    
Asset Purchase Agreement between Raptor Therapeutics, Registrant and Convivia, Inc. dated October 17, 2007
      10QSB   000-50720     10.3     1/14/08   Registrant
  10.13    
Merger agreement between Raptor Therapeutics and Encode Pharmaceuticals, Inc. dated December 14, 2007*
      10QSB/A   000-50720     10.1     4/15/08   Registrant
  10.14    
Pharmaceutical development services agreement between Raptor Therapeutics and Patheon Pharmaceuticals Inc. dated January 7, 2008*
      10QSB/A   000-50720     10.2     4/15/08   Registrant
  10.15    
License agreement between Encode Pharmaceuticals, Inc. (acquired by Raptor Therapeutics) and Regents of the University of California dated October 31, 2007*
      10QSB/A   000-50720     10.3     4/15/08   Registrant
  10.16    
Amendment number one to license agreement between Encode Pharmaceuticals, Inc. (acquired by Raptor Therapeutics) and the Regents of the University of California dated February 29, 2008*
      10QSB/A   000-50720     10.4     4/15/08   Registrant
  10.17    
Securities Purchase Agreement, dated as of May 21, 2008, by and among the Registrant and the investors listed on the signature pages thereto (included in Exhibit 4.7)
                          Registrant

 

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            Filed    
Exhibit       Here   Incorporated by Reference
No.   Exhibit Description   with   Form   File No.   Exhibit   Filing Date   Filed By
  10.18    
Amendment to Securities Purchase Agreement, dated as of May 21, 2008, by and among the Registrant and the investors listed on the signature pages thereto (included in Exhibit 4.8)
                           
  10.19    
Collaboration and License Agreement, dated as of June 3, 2009, by and between F. Hoffmann-La Roche Ltd., Hoffmann-La Roche Inc. and Raptor Discoveries Inc.*
  X                        
  10.20    
Supply Agreement, dated July 20, 2009, by and between Raptor Therapeutics and Mylan Pharmaceuticals Inc.*
  X                        
  10.21    
Securities Purchase Agreement, dated August 21, 2009, by and among the Registrant and the investors listed on the signature pages thereto (included in Exhibit 4.12)
                           
  21.1    
Subsidiaries of Registrant
  X                        
  23.1    
Consent of Independent Registered Public Accounting Firm Burr, Pilger, Mayer, LLP
  X                        
  24.1    
Power of Attorney (included in the signature page hereto)
  X                        
  31.1    
Certification of Christopher M. Starr, Ph.D., Chief Executive Officer and Director
  X                        
  31.2    
Certification of Kim R. Tsuchimoto, Chief Financial Officer, Secretary and Treasurer
  X                        
  32.1    
Certification of Christopher M. Starr, Ph.D., Chief Executive Officer and Director, and of Kim R. Tsuchimoto, Chief Financial Officer, Secretary and Treasurer
  X                        
     
X  
Filed herewith
 
*  
Certain portions of this agreement have been redacted and submitted to the Securities and Exchange Commission under a confidential treatment request pursuant to Rule 24b-2.

 

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SIGNATURES
Pursuant to the requirements of Section 13 or 15(d) of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned, thereunto duly authorized.
         
  RAPTOR PHARMACEUTICALS CORP.

Dated: October 28, 2009
 
 
  By:   /s/ Kim R. Tsuchimoto    
    Kim R. Tsuchimoto   
    Chief Financial Officer, Secretary and Treasurer
(Principal Financial Officer and Principal Accounting Officer) 
 
KNOW ALL PERSONS BY THESE PRESENTS, that each person whose signature appears below constitutes and appoints Christopher M. Starr, Ph.D. and Kim R. Tsuchimoto, his attorney-in-fact, with the power of substitution, for him in any and all capacities, to sign any amendments to the Report on Form 10-K and to file the same, with exhibits thereto and other documents in connection therewith, with the Securities and Exchange Commission, hereby ratifying and confirming all that each of said attorneys-in-fact, or his substitute or substitutes, may do or cause to be done by virtue hereof.
Pursuant to the requirements of the Securities Exchange Act of 1934, this report has been signed below by the following persons on behalf of the registrant and in the capacities and on the dates indicated:
         
Signatures   Title   Date
 
       
/s/ Christopher M. Starr
 
Christopher M. Starr, Ph.D.
  Chief Executive Officer and Director
(Principal Executive Officer)
  October 28, 2009
 
       
/s/ Kim R. Tsuchimoto
  Chief Financial Officer, Secretary and Treasurer   October 28, 2009
 
Kim R. Tsuchimoto
  (Principal Financial Officer and Principal
Accounting Officer)
   
 
       
/s/ Erich Sager
  Director   October 28, 2009
 
Erich Sager
       
 
       
/s/ Raymond William Anderson
  Director   October 28, 2009
 
Raymond William Anderson
       
 
       
/s/ Richard L. Franklin
  Director   October 28, 2009
 
Richard L. Franklin, M.D., Ph.D.
       
 
       
/s/ Llew Keltner
  Director   October 28, 2009
 
Llew Keltner, M.D., Ph.D.
       

 

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The following exhibits are filed as part of, or incorporated by reference into this Report:
Exhibits
                                     
            Filed    
Exhibit       Here   Incorporated by Reference
No.   Exhibit Description   with   Form   File No.   Exhibit   Filing Date   Filed By
  2.1    
Share Purchase Agreement dated May 2, 2006 by and between the Highland Clan Creations Corp. and Universal Financial Consulting Inc.
      8-K   000-50720     10.1     5/4/06   Registrant
  2.2    
Share Purchase Agreement dated as of March 9, 2006 by and among Highland Clan Creations Corp., Christopher Starr, Erich Sager, Todd Zankel and Falcon Corporate Investments Ltd.
      8-K   000-50720     10.1     3/14/06   Registrant
  2.3    
Agreement and Plan of Merger between Highland Clan Creations Corp. and Raptor Pharmaceuticals Corp. Effective May 15, 2006
      Schedule 14C   000-50720   Schedule A   5/17/06   Registrant
  2.4    
Agreement and Plan of Merger and Reorganization, dated July 27, 2009, by and among the Registrant, TorreyPines Therapeutics, Inc. and ECP Acquisition, Inc., a Delaware corporation
      8-K   000-50720     2.1     7/28/09   Registrant
  3.1    
Certificate of Incorporation of the Registrant
      Schedule 14C   000-50720   Schedule B   5/17/06   Registrant
  3.2    
Bylaws of the Registrant
      Schedule 14C   000-50720   Schedule C   5/17/06   Registrant
  3.3    
Certificate of Designation of Series A Junior Participating Preferred Stock of the Registrant
      8-A   000-50720     3.1     12/9/08   Registrant
  4.2    
Rights Agreement, between the Registrant and Nevada Agency and Trust Company, dated May 13, 2005
      8-A   000-50720     4.1     12/9/08   Registrant
  4.3    
Form of Rights Certificate of the Registrant (included in Exhibit 4.2)
                          Registrant
  4.4    
Amendment to Rights Agreement of the Registrant
      8-K   000-50720     4.1     7/28/09   Registrant
  4.5    
Warrant to purchase common stock dated December 14, 2007 issued to Flower Ventures, LLC
      10QSB/A   000-50720     4.1     4/15/08   Registrant
  4.6    
Warrant to purchase common stock dated December 14, 2007 issued to ICON Partners, LP
      10QSB/A   000-50720     4.2     4/15/08   Registrant
  4.7    
Securities Purchase Agreement, dated as of May 21, 2008, by and among the Registrant and the investors listed on the signature pages thereto
      10-QSB   000-50720     10.1     7/9/08   Registrant
  4.8    
Amendment to Securities Purchase Agreement, dated May 21, 2008, by and among the Registrant and the investors listed on the signature pages thereto
      10-QSB   000-50720     10.2     7/9/08   Registrant

 

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            Filed    
Exhibit       Here   Incorporated by Reference
No.   Exhibit Description   with   Form   File No.   Exhibit   Filing Date   Filed By
  4.9    
Form of 2008 Warrant to Purchase Shares of Common Stock of the Registrant
      8-K   000-50720     4.1     5/22/08   Registrant
  4.10    
Form of 2008 Placement Agent Warrant to Purchase Shares of Common Stock of the Registrant
      8-K/A   000-50720     4.2     5/28/08   Registrant
  4.11    
Form of Warrant Exchange Agreement, dated July 17, 2009
  X                        
  4.12    
Securities Purchase Agreement, dated August 21, 2009, by and among the Registrant and the investors listed on the signature pages thereto
  X                        
  4.13    
Form of 2009 Warrant to Purchase Shares of Common Stock of the Registrant
      8-K   000-50720     4.1     8/25/09   Registrant
  4.14    
Form of 2009 Placement Agent Warrant to Purchase Shares of Common Stock of the Registrant
      8-K   000-50720     4.2     8/25/09   Registrant
  10.1    
Employment Agreement between the Registrant and Dr. Christopher Starr dated May 1, 2006
      8-K   000-50720     10.5     5/26/06   Registrant
  10.2    
First Amendment to the Employment Agreement between the Registrant and Dr. Christopher Starr effective January 1, 2009
      8-K   000-50720     10.1     1/5/09   Registrant
  10.3    
Employment Agreement between the Registrant and Dr. Todd Zankel dated May 15, 2006
      8-K   000-50720     10.6     5/26/06   Registrant
  10.4    
First Amendment to the Employment Agreement between the Registrant and Dr. Todd Zankel effective January 1, 2009
      8-K   000-50720     10.3     1/5/09   Registrant
  10.5    
Employment Agreement between the Registrant and Ms. Kim Tsuchimoto dated May 1, 2006
      8-K   000-50720     10.7     5/26/06   Registrant
  10.6    
First Amendment to the Employment Agreement between the Registrant and Ms. Kim Tsuchimoto effective January 1, 2009
      8-K   000-50720     10.7     1/5/09   Registrant

 

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            Filed    
Exhibit       Here   Incorporated by Reference
No.   Exhibit Description   with   Form   File No.   Exhibit   Filing Date   Filed By
  10.7    
Employment Agreement between Raptor Therapeutics and Thomas E. Daley dated September 7, 2007
      10QSB   000-50720     10.1     1/14/08   Registrant
  10.8    
First Amendment to the Employment Agreement between Raptor Therapeutics and Thomas E. Daley effective January 1, 2009
      8-K   000-50720     10.4     1/5/09   Registrant
  10.9    
Offer Letter from Raptor Therapeutics dated as of April 8, 2009 for Patrice Rioux, M.D., Ph.D
      8-K   000-50720     10.1     4/14/09   Registrant
  10.10    
2006 Equity Incentive Plan, as amended
      S-8   333-140944     4.3     2/28/07   Registrant
  10.11    
2008 Plan Amendment to 2006 Equity Incentive Plan
      10-K/A   000-50720     10.5     12/23/08   Registrant
  10.12    
Asset Purchase Agreement between Raptor Therapeutics, Registrant and Convivia, Inc. dated October 17, 2007
      10QSB   000-50720     10.3     1/14/08   Registrant
  10.13    
Merger agreement between Raptor Therapeutics and Encode Pharmaceuticals, Inc. dated December 14, 2007*
      10QSB/A   000-50720     10.1     4/15/08   Registrant
  10.14    
Pharmaceutical development services agreement between Raptor Therapeutics and Patheon Pharmaceuticals Inc. dated January 7, 2008*
      10QSB/A   000-50720     10.2     4/15/08   Registrant
  10.15    
License agreement between Encode Pharmaceuticals, Inc. (acquired by Raptor Therapeutics) and Regents of the University of California dated October 31, 2007*
      10QSB/A   000-50720     10.3     4/15/08   Registrant
  10.16    
Amendment number one to license agreement between Encode Pharmaceuticals, Inc. (acquired by Raptor Therapeutics) and the Regents of the University of California dated February 29, 2008*
      10QSB/A   000-50720     10.4     4/15/08   Registrant
  10.17    
Securities Purchase Agreement, dated as of May 21, 2008, by and among the Registrant and the investors listed on the signature pages thereto (included in Exhibit 4.7)
                          Registrant
  10.18    
Amendment to Securities Purchase Agreement, dated as of May 21, 2008, by and among the Registrant and the investors listed on the signature pages thereto (included in Exhibit 4.8)
                           

 

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            Filed    
Exhibit       Here   Incorporated by Reference
No.   Exhibit Description   with   Form   File No.   Exhibit   Filing Date   Filed By
  10.19    
Collaboration and License Agreement, dated as of June 3, 2009, by and between F. Hoffmann-La Roche Ltd., Hoffmann-La Roche Inc. and Raptor Discoveries Inc.*
  X                        
  10.20    
Supply Agreement, dated July 20, 2009, by and between Raptor Therapeutics and Mylan Pharmaceuticals Inc.*
  X                        
  10.21    
Securities Purchase Agreement, dated August 21, 2009, by and among the Registrant and the investors listed on the signature pages thereto (included in Exhibit 4.12)
                           
  21.1    
Subsidiaries of Registrant
  X                        
  23.1    
Consent of Independent Registered Public Accounting Firm Burr, Pilger, Mayer, LLP
  X                        
  24.1    
Power of Attorney (included in the signature page hereto)
  X                        
  31.1    
Certification of Christopher M. Starr, Ph.D., Chief Executive Officer and Director
  X                        
  31.2    
Certification of Kim R. Tsuchimoto, Chief Financial Officer, Secretary and Treasurer
  X                        
  32.1    
Certification of Christopher M. Starr, Ph.D., Chief Executive Officer and Director, and of Kim R. Tsuchimoto, Chief Financial Officer, Secretary and Treasurer
  X                        
     
X  
Filed herewith
 
*  
Certain portions of this agreement have been redacted and submitted to the Securities and Exchange Commission under a confidential treatment request pursuant to Rule 24b-2.

 

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EX-4.11 2 c91590exv4w11.htm EXHIBIT 4.11 Exhibit 4.11

EXHIBIT 4.11

WARRANT AMENDMENT AND SUBSCRIPTION AGREEMENT

This Warrant Amendment and Subscription Agreement (this “Agreement”) is entered into as of July 17, 2009 between Raptor Pharmaceuticals Corp., a Delaware corporation (the “Company”), and the undersigned warrantholder (“Investor”), with respect to the exercise of the those warrants (collectively, the ”Warrants”) to purchase shares of the Company’s common stock, par value $.001 per share (“Common Stock”) issued to Investor in connection with the Company’s private placement of units (consisting of the Company’s common stock and warrants) pursuant to the terms of that certain Securities Purchase Agreement, dated May 21, 2008, by and among the Company, the Investor, and the other parties named therein, as amended by that certain Amendment to Securities Purchase Agreement, dated May 21, 2008 (collectively, the “Purchase Agreement”). Pursuant to this Agreement, Investor will purchase the number of shares of Common Stock set forth on the subscription forms of such Warrants (the “Subscription Forms”) and delivered to the Company.

  a.  
Amendment of Warrant and Warrant Exercise.

The Company and Investor hereby amend the Warrants to provide for the reduction in the exercise price thereof to $0.30 per share of Common Stock, but only with respect to those shares of Common Stock for which the Warrants are exercised in accordance herewith on or before July 17, 2009 (the “Offer Expiration Date”), and in consideration of this Agreement, Investor hereby exercises the Warrants to the extent provided herein to be effective on or before the Offer Expiration Date. Other than as expressly set forth herein, nothing in this Agreement shall be deemed to alter, vary or otherwise affect the terms, conditions, and provisions of the Warrants.

Upon (i) the Company’s receipt and acceptance of this signed Agreement, (ii) receipt by the Company of the Warrants with Subscription Forms properly completed and duly executed by the Investor with respect to the Common Stock for which these Warrants are being exercised, and (iii) receipt by the Company of an amount equal to $0.30 per share of Common Stock for which these Warrants are being exercised as set forth on the applicable Subscription Forms, payable in cash or by check for same day funds, all of such items in clauses (i), (ii) and (iii) to occur on or before the Offer Expiration Date, then the Company, will sell to Investor, and Investor will purchase and receive from the Company, the number of shares of Common Stock (the “Exercised Warrant Stock”) subject to the Warrants and set forth in the applicable Subscription Forms. If any completed Subscription Form is for less than all of the common stock subject to the applicable Warrant, a new warrant certificate incorporating the terms of the original Warrant, including, without limitation, the original exercise prices, for the remaining number of shares of common stock not so exercised hereby will be issued to the Investor. For the avoidance of doubt, such new warrant certificate would include the exercise price of: (i) $0.75 per share to the extent that such Warrant is exercised prior to the first anniversary date of the original issue date, and (ii) $0.90 per share to the extent that such Warrant is exercised after the first anniversary date, and prior to the second anniversary date, of the original issue date.

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  b.  
Investor Representations.

Investor hereby represents and warrants to the Company that:

(1) INVESTOR ACKNOWLEDGES THIS IS A HIGHLY SPECULATIVE INVESTMENT. INVESTOR ACKNOWLEDGES THAT THE COMPANY’S CURRENT CAPITAL RESOURCES, WITHOUT REGARD TO POTENTIAL PROCEEDS FROM THIS OFFER OR ANY FUTURE FINANCING OR OTHER POTENTIAL SOURCES OF FUTURE FUNDING, WILL ONLY BE SUFFICIENT TO FUND THE COMPANY UNTIL MID-2009. THERE CAN BE NO ASSURANCE THAT FUNDS WILL BE AVAILABLE TO OPERATE BEYOND THAT DATE. IF NOT, THE COMPANY WOULD BE REQUIRED TO DELAY PROGRAMS OR POSSIBLY CEASE OPERATIONS.

(2) Investor has received and read, prior to its acquisition of the Exercised Warrant Stock, the Company’s most recent Annual Report on Form 10-K for the Company’s fiscal year ended August 31, 2008 filed with the Securities and Exchange Commission (the “Commission”) on September 30, 2008 (as amended by the Company’s Forms 10-K/A filed with the Commission on December 23, 2008 and April 20, 2009), and its Quarterly Report on Form 10-Q for the second quarter of fiscal year 2009 filed with the Commission on April 13, 2009, including, without limitation, the statements of risk factors set forth therein.

(3) Investor acknowledges that even with funds raised through exercise of the Warrants, substantial additional capital resources will be required to fund the Company’s operations. The Investor acknowledges that there can be no assurance the Company will be able to raise sufficient additional capital funds, or that such financing will be available on acceptable terms.

(4) Investor has had, prior to its acquisition of the Exercised Warrant Stock, the opportunity to ask questions of and receive answers from the Company in order to obtain additional information or to verify the accuracy of any information furnished to it or to which it has had access. Investor has received all information that it has requested regarding the Company and believes that such information is sufficient to make an informed decision with respect to the acquisition of the Exercised Warrant Stock.

(5) Investor is an “accredited investor” as that term is defined in Rule 501 of Regulation D promulgated under the Securities Act.

(6) Investor’s jurisdiction of formation or incorporation (if applicable) and principal place of business or its residency as set forth on the signature page hereof by the Investor are accurate.

(7) The acquisition by Investor of the Exercised Warrant Stock hereunder does not violate or conflict with any law or regulation applicable to Investor.

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(8) No person engaged by Investor has, or will have, any right or claim against the Company for any commission, fee or other compensation as a finder or broker, or in any similar capacity.

(9) Investor hereby incorporates by reference and reaffirms each acknowledgement, representation and warranty made by it in each of the Purchase Agreement and the Warrants as if made on the date hereof.

  c.  
Investor Covenants.

Investor hereby further agrees with the Company as follows:

(1) All certificates evidencing the Exercised Warrant Stock shall bear a legend in substantially the following form:

“THE SECURITIES REPRESENTED BY THIS CERTIFICATE HAVE NOT BEEN REGISTERED UNDER THE SECURITIES ACT OF 1933, AS AMENDED, OR THE SECURITIES LAWS OF ANY STATE OF THE UNITED STATES. THE SECURITIES MAY NOT BE SOLD, OFFERED FOR SALE, PLEDGED, HYPOTHECATED, TRANSFERRED OR ASSIGNED IN THE ABSENCE OF AN EFFECTIVE REGISTRATION STATEMENT FOR THE SECURITIES UNDER APPLICABLE SECURITIES LAWS, OR UNLESS OFFERED, SOLD, PLEDGED, HYPOTHECATED OR TRANSFERRED PURSUANT TO AN AVAILABLE EXEMPTION FROM THE REGISTRATION REQUIREMENTS OF THOSE LAWS. THE COMPANY SHALL BE ENTITLED TO REQUIRE AN OPINION OF COUNSEL SATISFACTORY TO THE COMPANY THAT SUCH REGISTRATION IS NOT REQUIRED TO THE EXTENT THAT SUCH OPINION IS REQUIRED PURSUANT TO THAT CERTAIN SECURITIES PURCHASE AGREEMENT UNDER WHICH THE SECURITIES WERE ISSUED.”

“Until the Distribution Date (as defined in the Rights Agreement referred to below), this certificate also evidences and entitles the holder hereof to certain Rights as set forth in a Stockholder Rights Agreement, dated as of December 5, 2008 (as such may be amended from time to time, the “Rights Agreement”), between Raptor Pharmaceuticals Corp. (the “Company”) and Nevada Agency & Trust Company, as Rights Agent, the terms of which are hereby incorporated herein by reference and a copy of which is on file at the principal executive offices of the Company. Under certain circumstances, as set forth in the Rights Agreement, such Rights may be evidenced by separate certificates and may no longer be evidenced by this certificate. The Company will mail or arrange for the mailing of a copy of the Rights Agreement to the holder of this certificate without charge after the receipt of a written request therefor. As described in the Rights Agreement, Rights issued to any Person who becomes an “Acquiring Person” or its “Affiliates” or “Associates” (as defined in the Rights Agreement) and certain related Persons, whether currently held by or on behalf of such Person or by any subsequent holder, shall become null and void.”

(2) The instruments or certificates representing the Exercised Warrant Stock, and each instrument or certificate issued in transfer thereof, will also bear any legend required under any applicable state securities law.

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(3) Any proposed sale, assignment, transfer or pledge of any Exercised Warrant Stock by Investor shall be made in accordance with Section 3.7 of the Purchase Agreement.

(4) Investor consents to the Company’s making a notation on its records or giving instructions to any transfer agent of the Common Stock in order to implement the restrictions on transfer of the Exercised Warrant Stock.

  d.  
Miscellaneous.

(1) Waivers and Amendments. The rights of the parties hereto may be waived, amended or modified (either generally or in a particular instance, either retroactively or prospectively and either for a specified period of time or indefinitely) only in a writing signed by both of the parties hereto.

(2) Severability. Should any one or more of the provisions of this Agreement be determined to be illegal or unenforceable, all other provisions of this Agreement shall be given effect separately from the provision or provisions determined to be illegal or unenforceable and shall not be affected thereby.

(3) Parties in Interest. All the terms and provisions of this Agreement shall be binding upon and inure to the benefit of the respective successors of the parties hereto.

(4) Headings. The headings of the paragraphs of this Agreement have been inserted for convenience of reference only and do not constitute a part of this Agreement.

(5) Governing Law; Jurisdiction; Waiver of Jury Trial. This Agreement will be governed by and interpreted in accordance with the laws of the State of Delaware without regard to the principles of conflict of laws. Each of the parties hereto irrevocably submits and consents to the exclusive jurisdiction of the courts of the State of California located in Marin County and the United States District Court for the Northern District of California for the purpose of any suit, action, proceeding or judgment relating to or arising out of this Agreement and the transactions contemplated hereby. Each party hereto irrevocably waives any objection to the laying of venue of any such suit, action or proceeding brought in such courts and irrevocably waives any claim that any such suit, action or proceeding brought in any such court has been brought in an inconvenient forum. EACH OF THE PARTIES HERETO WAIVES ANY RIGHT TO REQUEST A TRIAL BY JURY IN ANY LITIGATION WITH RESPECT TO THIS AGREEMENT AND REPRESENTS THAT COUNSEL HAS BEEN CONSULTED SPECIFICALLY AS TO THIS WAIVER.

(6) Counterparts. This Agreement may be executed in any number of counterparts (including by facsimile) and by different parties hereto in separate counterparts, with the same effect as if all parties had signed the same document. All such counterparts shall be deemed an original, shall be construed together and shall constitute one and the same instrument.

(7) NOTHING HEREIN SHALL CONSTITUTE OR BE CONSTRUED AS TAX OR INVESTMENT ADVICE. [Signature page follows]

 

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IN WITNESS WHEREOF, the undersigned has caused this Agreement to be duly executed as of the date first above written.

RAPTOR PHARMACEUTICALS CORP.,
a Delaware corporation

By:                                                                        
Kim R. Tsuchimoto
Chief Financial Officer, Secretary and Treasurer

ACCEPTED AND AGREED TO:

INVESTOR

Signature:                                                        

Name:                                                              

Principal place of business or residency:

                                                                       

                                                                       

                                                                       

Jurisdiction of formation or incorporation (if applicable):

                                                                       

[Signature Page to Warrant Amendment and Subscription Agreement]

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EX-4.12 3 c91590exv4w12.htm EXHIBIT 4.12 Exhibit 4.12

EXHIBIT 4.12

RAPTOR PHARMACEUTICALS CORP.
SECURITIES PURCHASE AGREEMENT

This Securities Purchase Agreement (this “Agreement”) is made as of August 21, 2009 (the “Effective Date”), by and among Raptor Pharmaceuticals Corp., a Delaware corporation (the Company”), and the individuals and entities identified on the signature pages hereto (each, a “Purchaser” and, collectively, the “Purchasers”).

Introduction

The Company and the Purchasers are executing and delivering this Agreement in reliance upon the exemption from securities registration afforded by Section 4(2) of the Securities Act and Rule 506 promulgated thereunder.

The Purchasers desire to purchase from the Company, and the Company desires to sell to the Purchasers, upon the terms and conditions stated in this Agreement, up to a maximum of $2.4 million of the Company’s common stock, par value $0.001 per share (the “Common Stock”), and warrants to purchase Common Stock of the Company.

The capitalized terms used herein and not otherwise defined herein have the meanings given them in Article 7.

In consideration of the premises and the mutual covenants contained herein and other good and valuable consideration, the receipt and sufficiency of which are hereby acknowledged, the Company and the Purchasers (severally and not jointly) hereby agree as follows:

ARTICLE 1

Purchase and Sale of Securities

Section 1.1 Purchase and Sale of Securities. At the Closing, upon the terms and subject to the conditions herein, the Company will issue and sell to each Purchaser, and each Purchaser will, severally and not jointly, purchase from the Company the number of shares of Common Stock and the number of warrants to purchase shares of Common Stock, pursuant to a Warrant substantially in the form of Exhibit B attached hereto, as set forth opposite such Purchaser’s name under the heading “Shares” and “Warrants,” respectively, on Exhibit A attached hereto, as the same may be amended from time to time after each Subsequent Closing or otherwise, as appropriate. The purchase price for each Share and related Warrant (together, a unit) will be $0.32, subject to change at the discretion of those officers so authorized by the Company’s Board of Directors (the “Purchase Price”). The Shares (as defined below) and Warrants (as defined below) are being sold hereunder as units, each unit consisting of one Share and a Warrant to purchase one-half of a share of Common Stock.

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Section 1.2 Payment. Upon the Closing Date, each Purchaser shall deliver such Purchaser’s aggregate Purchase Price set forth opposite its name on Exhibit A attached hereto, by wire transfer of immediately available funds in accordance with wire instructions provided to it by the Company at least two Business Days prior to the Closing. At or promptly following the Closing, and after receiving the aggregate Purchase Price set forth opposite each Purchaser’s name on Exhibit A attached hereto, by wire transfer of immediately available funds, the Company will instruct its transfer agent to credit and deliver to each Purchaser certificate(s) representing the number of Shares set forth on Exhibit A attached hereto and will deliver Warrants to purchase the Warrant Shares (as defined below).

Section 1.3 Closing Date. The closing of the transactions contemplated by this Agreement (the “Closing”) will take place at 5 p.m. (Pacific Time) on the first Business Day after the satisfaction or waiver of the conditions set forth in Article 5 (excluding conditions that, by their nature, cannot be satisfied until the Closing), the actual time and date of the Closing being referred to herein as the “Closing Date”. The anticipated Closing Date is August 2009. The Closing will be held at the offices of Paul, Hastings, Janofsky & Walker, LLP, 515 South Flower Street, Floor 25, Los Angeles, California 90071, or at such other time and place as shall be agreed upon by the Company and the Majority Purchasers.

Section 1.4 Subsequent Closings. After the Closing, additional Shares (which, together with the Shares issued at the Closing, shall not exceed 7.5 million shares in the aggregate), and additional Warrants (which, together with the Warrants issued at the Closing, shall not represent the right to acquire more than 3.75 million shares of Warrant Shares in the aggregate) may be issued at the discretion of the Company at one or more subsequent closings (each a “Subsequent Closing”) which are held on or before August 21, 2009. Each Subsequent Closing shall be effective upon the date (a “Subsequent Closing Date”) of the Company’s receipt from a Purchaser of a wire transfer of funds in the amount of the Purchase Price for the Securities being purchased by such Purchaser at such Subsequent Closing. Effective upon each such Subsequent Closing, the applicable Purchaser shall also enter into and become a party to this Agreement as if such Purchaser had executed such agreement at the Closing. At the Closing, the Company shall prepare Exhibit A with respect to the Purchasers purchasing the Shares and Warrants at the Closing. Promptly after each Subsequent Closing, the Company shall amend Exhibit A as appropriate. The shares of Common Stock, the warrants to purchase shares of Common Stock, and the Common Stock underlying such warrants, that are sold pursuant to and in accordance with Section 1.1 and this Section 1.4 are referred to herein as the “Shares,the “Warrants,and the “Warrant Shares,” respectively, and are referred to herein collectively as the “Securities.

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ARTICLE 2

Representations and Warranties of the Company

The Company hereby represents and warrants to the Purchasers that, as of the date hereof, except as set forth in the Public Filings or the corresponding section of the disclosure schedules delivered to the Purchasers herewith:

Section 2.1 Organization and Qualification. The Company is duly incorporated, validly existing and in good standing under the laws of the State of Delaware, with corporate power and authority to conduct its business as currently conducted as disclosed in the Public Filings. The Company is duly qualified to do business and is in good standing in every jurisdiction in which the nature of the business conducted by it or property owned by it makes such qualification necessary, except where the failure to be so qualified or in good standing, as the case may be, would not reasonably be expected to have a Material Adverse Effect.

Section 2.2 Authorization; Enforcement. The Company has all requisite corporate power and authority to enter into and to perform its obligations under the Transaction Agreements, to consummate the transactions contemplated hereby and thereby and to issue the Securities in accordance with the terms hereof. The execution, delivery and performance of the Transaction Agreements by the Company and the consummation by it of the transactions contemplated hereby and thereby (including the issuance of the Securities) have been duly authorized by the Company’s Board of Directors and no further consent or authorization of the Company, its Board of Directors, or its stockholders is required. Each Transaction Agreement has been duly executed by the Company and, assuming the due authorization, execution and delivery of the Transaction Agreements by each Purchaser a party thereto, constitutes a legal, valid and binding obligation of the Company enforceable against the Company in accordance with its terms, except as enforceability may be limited by applicable bankruptcy, insolvency, reorganization, or moratorium or similar laws affecting creditors’ and contracting parties’ rights generally and except as enforceability may be subject to general principles of equity and except as rights to indemnity and contribution may be limited by state, federal, or other securities laws or public policy underlying such laws.

Section 2.3 Capitalization. As of June 18, 2009, the authorized capital stock of the Company consisted of 100,000,000 shares of Common Stock and 10,000,000 shares of preferred stock, par value $0.001 per share, of which 60,430,047 shares of Common Stock were issued and outstanding and no shares of preferred stock were issued and outstanding. All of the issued and outstanding shares of Common Stock have been duly authorized, validly issued and are fully paid and nonassessable. Options and warrants to purchase an aggregate of 17,501,503 shares of Common Stock were outstanding as of June 18, 2009. Except as disclosed in or contemplated by the Public Filings, the Company does not have outstanding any options to purchase, or any preemptive rights or other rights to subscribe for or to purchase, any securities or obligations convertible into, or any contracts or commitments to issue or sell, shares of its capital stock or any such options, rights, convertible securities or obligations other than options granted under the Company’s stock option plans.

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Section 2.4 Issuance of Securities. The Shares and the Warrant Shares have been duly authorized and, upon issuance in accordance with the terms of this Agreement (and in case of the Warrant Shares, the Warrants), will be validly issued, fully paid and non-assessable and will not be subject to preemptive rights or other similar rights of stockholders of the Company.

Section 2.5 No Conflicts; Government Consents and Permits.

(a) The execution, delivery and performance of each Transaction Agreement by the Company and the consummation by the Company of the transactions contemplated hereby and thereby (including the issuance of the Securities) will not: (i) conflict with or result in a violation of any provision of its Certificate of Incorporation or Bylaws; (ii) violate or conflict with, or result in a breach of any provision of, or constitute a default under, any material agreement to which the Company is a party; or (iii) result in a violation of any law, rule, regulation, order, judgment or decree of any governmental authority applicable to the Company, except for such conflicts, breaches, defaults, and violations as would not reasonably be expected to have a Material Adverse Effect.

(b) The Company is not required to obtain any consent, authorization or order of, or make any filing or registration with, any court or governmental agency or any regulatory or self regulatory agency in order for it to execute, deliver or perform any of its obligations under this Agreement in accordance with the terms hereof, or to issue and sell the Securities in accordance with the terms hereof other than such as have been made or obtained or any filings required to be made under federal, state or other securities laws.

(c) The Company has all franchises, permits, licenses, and any similar authority necessary for the conduct of its business as now being conducted by it, except for such franchise, permit, license or similar authority, the lack of which would not reasonably be expected to have a Material Adverse Effect. The Company has not received any written notice of any proceeding relating to revocation or modification of any such franchise, permit, license, or similar authority except where such revocation or modification would not reasonably be expected to have a Material Adverse Effect.

Section 2.6 Public Filings, Financial Statements.

(a) The Company has timely filed all reports, schedules, forms, statements and other documents required to be filed by it with the U.S. Securities and Exchange Commission (the “SEC”) since January 1, 2009, pursuant to the reporting requirements of the Exchange Act, except for those filings not timely filed as would not reasonably be expected to have a Material Adverse Effect.

(b) As of their respective dates, the Public Filings complied as to form in all material respects with the requirements of the Exchange Act or the Securities Act, as the case may be, and the rules and regulations of the SEC promulgated thereunder applicable to the Public Filings, and none of the Public Filings, at the time they were filed with the SEC, contained any untrue statement of a material fact or omitted to state a material fact required to be stated therein or necessary in order to make the statements therein, in light of the circumstances

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under which they were made, not misleading. The Financial Statements have been prepared in accordance with accounting principles generally accepted in the United States, consistently applied, during the periods involved (except (i) as may be otherwise indicated in the Financial Statements or the notes thereto, or (ii) in the case of unaudited interim statements, to the extent they may not include footnotes, may be condensed or summary statements or may conform to the SEC’s rules and instructions for Reports on Form 10-Q or 10-QSB) and fairly present in all material respects the consolidated financial position of the Company as of the dates thereof and the consolidated results of its operations and cash flows for the periods then ended (subject, in the case of unaudited statements, to normal and recurring year-end audit adjustments).

Section 2.7 Absence of Litigation. As of the date hereof, there is no action, suit, proceeding or investigation before or by any court, public board, government agency, self-regulatory organization or body pending or, to the Company’s knowledge, threatened against the Company that if determined adversely to the Company would reasonably be expected to have a Material Adverse Effect. To the knowledge of the Company, there is not pending, any investigation by the SEC involving the Company or any current or former director or officer of the Company. The Company has not received any stop order or other order suspending the effectiveness of any registration statement filed by the Company under the Exchange Act or Securities Act and, to the Company’s knowledge, the SEC has not issued any such order.

Section 2.8 Intellectual Property Rights. To the Company’s knowledge, the Company owns or possesses, or believes it can obtain on reasonable terms, licenses or sufficient rights to use all patents, patent applications, patent rights, inventions, know-how, trade secrets, trademarks, trademark applications, service marks, service names, trade names and copyrights necessary to enable it to conduct its business as conducted as of the date hereof (the “Intellectual Property”). To the Company’s knowledge, the Company has not infringed the intellectual property rights of third parties and no third party, to the Company’s knowledge, is infringing the Intellectual Property, in each case, which could reasonably be expected to result in a Material Adverse Effect. Except as disclosed in the Public Filings, there are no material options, licenses or agreements relating to the Intellectual Property, nor is the Company bound by or a party to any material options, licenses or agreements relating to the patents, patent applications, patent rights, inventions, know-how, trade secrets, trademarks, trademark applications, service marks, service names, trade names or copyrights of any other Person. As of the date hereof, there is no material claim or action or proceeding pending or, to the Company’s knowledge, threatened, that challenges the right of the Company with respect to any Intellectual Property.

Section 2.9 Placement Agents. The Company has taken no action that would give rise to any claim by any Person for brokerage commissions, placement agent’s fees or similar payments relating to this Agreement or the transactions contemplated hereby, except for dealings with the Placement Agents, whose commissions and fees will be paid by the Company.

Section 2.10 No Material Adverse Change. Since January 1, 2008, except as described in the Public Filings and except for cash expenditures in the ordinary course of business, there has not been a material adverse change in the business, financial condition, assets or results of operations of the Company other than its continuing losses from operations. Since January 1, 2008: (i) there has not been any dividend or distribution of any kind declared, set aside for payment, paid or made by the Company on any class of capital stock; and (ii) the Company has

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not sustained any material loss or interference with the Company’s business from fire, explosion, flood or other calamity, whether or not covered by insurance, or from any labor disturbance or dispute or any action, order or decree of any court or arbitrator or governmental or regulatory authority.

Section 2.11 Taxes. The Company has filed (or has obtained an extension of time within which to file) all necessary federal, state and foreign income and franchise tax returns and has paid all taxes shown as due on such tax returns, except where the failure to so file or the failure to so pay would not reasonably be expected to have a Material Adverse Effect and except those taxes being contested in good faith and for which adequate reserves have been provided.

Section 2.12 No Manipulation of Stock. The Company has not taken, nor will it take, directly or indirectly any action designed to stabilize or manipulate the price of the Common Stock or any security of the Company to facilitate the sale or resale of any of the Shares.

ARTICLE 3

Purchaser’s Representations and Warranties

Each Purchaser represents and warrants to the Company, severally and not jointly, with respect to itself and its purchase hereunder, that:

Section 3.1 Investment Purpose. The Purchaser is purchasing the Securities for its own account and not with a present view toward the public sale or distribution thereof and has no intention of selling or distributing any of such Securities or any arrangement or understanding with any other Persons regarding the sale or distribution of such Securities except in accordance with the provisions of Article 6 and except as would not result in a violation of the Securities Act. The Purchaser will not, directly or indirectly, offer, sell, pledge, transfer or otherwise dispose of (or solicit any offers to buy, purchase or otherwise acquire or take a pledge of) any of the Securities except in accordance with the provisions of Article 6 or pursuant to and in accordance with the Securities Act.

Section 3.2 Questionnaires. The Purchaser has completed the Accredited Purchaser Qualification Questionnaire, in the form of Exhibit C attached hereto, and has submitted such Accredited Purchaser Qualification Questionnaire to the Company in connection with its purchase of the Securities. The Accredited Purchaser Qualification Questionnaire was accurate and correct when delivered and is accurate and correct as of the date hereof and the representations, warranties and covenants contained herein and in the applicable schedules attached hereto will be true and correct both as of the date of execution of this Subscription Agreement and on the Closing Date. In addition to completing and delivering the aforementioned, the Purchaser agrees to provide any additional information that is requested by the Company in order to confirm that such Purchaser has complied with any applicable securities laws to which it is subject.

Section 3.3 Reliance on Exemptions. The Purchaser understands that the Securities are being offered and sold to it in reliance upon specific exemptions from the

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prospectus and registration requirements of federal, state, and other securities laws, as applicable, and that the Company is relying upon the truth and accuracy of, and the Purchaser’s compliance with, the representations, warranties, agreements, acknowledgments and understandings of the Purchaser set forth herein in order to determine the availability of such exemptions and the eligibility of the Purchaser to acquire the Securities.

Section 3.4 Information. The Purchaser has been furnished with all relevant materials relating to the business, finances and operations of the Company necessary to make an investment decision, and materials relating to the offer and sale of the Securities, that have been requested by the Purchaser, including, without limitation, the Company’s Public Filings, and the Purchaser has had the opportunity to review the Public Filings. The Purchaser has been afforded the opportunity to ask questions of the Company. Neither such inquiries nor any other investigation conducted by or on behalf of such Purchaser or its representatives or counsel shall modify, amend or affect such Purchaser’s right to rely on the truth, accuracy and completeness of the Public Filings and the Company’s representations and warranties contained herein.

Section 3.5 Acknowledgement of Risk.

(a) The Purchaser acknowledges and understands that its investment in the Securities involves a significant degree of risk, including, without limitation: (i) the Company remains a development stage business with limited operating history and requires substantial funds in addition to the proceeds from the sale of the Securities; (ii) an investment in the Company is speculative, and only Purchasers who can afford the loss of their entire investment should consider investing in the Company and the Securities; (iii) the Purchaser may not be able to liquidate its investment; (iv) transferability of the Securities is extremely limited; (v) in the event of a disposition of the Securities, the Purchaser could sustain the loss of its entire investment; and (vi) the Company has not paid any dividends on its Common Stock since inception and does not anticipate the payment of dividends in the foreseeable future. Such risks are more fully set forth in the Public Filings;

(b) The Purchaser is able to bear the economic risk of holding the Securities for an indefinite period, and has knowledge and experience in financial and business matters such that it is capable of evaluating the risks of the investment in the Securities; and

(c) The Purchaser has, in connection with the Purchaser’s decision to purchase Securities, not relied upon any representations or other information (whether oral or written) other than as set forth in the representations and warranties of the Company contained herein, and the Purchaser has, with respect to all matters relating to the Transaction Agreements and the offer and sale of the Securities, relied solely upon the advice of such Purchaser’s own counsel and has not relied upon or consulted any counsel to any placement agent or counsel to the Company.

Section 3.6 Governmental Review. The Purchaser understands that no federal or state agency or any other government or governmental agency has passed upon or made any recommendation or endorsement of the Securities or an investment therein.

Section 3.7 Transfer or Resale. The Purchaser understands that:

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(a) the Securities have not been and are not being registered under the Securities Act (other than as contemplated in Article 6), or any applicable federal, state, or other securities laws and, consequently, the Purchaser may have to bear the risk of owning the Securities for an indefinite period of time because the Securities may not be transferred unless: (i) the resale of the Securities is registered pursuant to an effective registration statement under the Securities Act, as contemplated in Article 6; (ii) the Purchaser has delivered to the Company an opinion of counsel (in form, substance and scope reasonably satisfactory to the Company) to the effect that the Securities to be sold or transferred may be sold or transferred pursuant to an exemption from such registration; or (iii) the Securities are sold or transferred pursuant to Rule 144;

(b) any sale of the Securities made in reliance on Rule 144 may be made only in accordance with the terms of Rule 144 and, if Rule 144 is not applicable, any resale of the Securities under circumstances in which the seller (or the person through whom the sale is made) may be deemed to be an underwriter (as that term is defined in the Securities Act) may require compliance with some other exemption under the Securities Act or the rules and regulations of the SEC thereunder; and

(c) except as set forth in Article 6, neither the Company nor any other Person is under any obligation to register the resale of the Shares or the Warrant Shares under the Securities Act, or any applicable federal, state or other securities laws or to comply with the terms and conditions of any exemption thereunder.

Section 3.8 Legends. The Purchaser understands the certificates representing the Securities will bear restrictive legends in the following forms (and a stop-transfer order may be placed against transfer of the certificates for such Securities):

“THE SECURITIES REPRESENTED BY THIS CERTIFICATE HAVE NOT BEEN REGISTERED UNDER THE SECURITIES ACT OF 1933, AS AMENDED, OR THE SECURITIES LAWS OF ANY STATE OF THE UNITED STATES OR ANY OTHER JURISDICTION. THE SECURITIES MAY NOT BE SOLD, OFFERED FOR SALE, PLEDGED, HYPOTHECATED, TRANSFERRED OR ASSIGNED IN THE ABSENCE OF AN EFFECTIVE REGISTRATION STATEMENT FOR THE SECURITIES UNDER APPLICABLE SECURITIES LAWS, OR UNLESS OFFERED, SOLD, PLEDGED, HYPOTHECATED OR TRANSFERRED PURSUANT TO AN AVAILABLE EXEMPTION FROM THE REGISTRATION REQUIREMENTS OF THOSE LAWS. THE COMPANY SHALL BE ENTITLED TO REQUIRE AN OPINION OF COUNSEL SATISFACTORY TO THE COMPANY THAT SUCH REGISTRATION IS NOT REQUIRED TO THE EXTENT THAT SUCH OPINION IS REQUIRED PURSUANT TO THAT CERTAIN SECURITIES PURCHASE AGREEMENT UNDER WHICH THE SECURITIES WERE ISSUED.

“UNTIL THE DISTRIBUTION DATE (AS DEFINED IN THE RIGHTS AGREEMENT REFERRED TO BELOW), THIS CERTIFICATE ALSO EVIDENCES AND ENTITLES THE HOLDER HEREOF TO CERTAIN

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RIGHTS AS SET FORTH IN A STOCKHOLDER RIGHTS AGREEMENT, DATED AS OF DECEMBER 5, 2008 (AS SUCH MAY BE AMENDED FROM TIME TO TIME, THE “RIGHTS AGREEMENT”), BETWEEN RAPTOR PHARMACEUTICALS CORP. (THE “COMPANY”) AND NEVADA AGENCY & TRUST COMPANY, AS RIGHTS AGENT, THE TERMS OF WHICH ARE HEREBY INCORPORATED HEREIN BY REFERENCE AND A COPY OF WHICH IS ON FILE AT THE PRINCIPAL EXECUTIVE OFFICES OF THE COMPANY. UNDER CERTAIN CIRCUMSTANCES, AS SET FORTH IN THE RIGHTS AGREEMENT, SUCH RIGHTS MAY BE EVIDENCED BY SEPARATE CERTIFICATES AND MAY NO LONGER BE EVIDENCED BY THIS CERTIFICATE. THE COMPANY WILL MAIL OR ARRANGE FOR THE MAILING OF A COPY OF THE RIGHTS AGREEMENT TO THE HOLDER OF THIS CERTIFICATE WITHOUT CHARGE AFTER THE RECEIPT OF A WRITTEN REQUEST THEREFOR. AS DESCRIBED IN THE RIGHTS AGREEMENT, RIGHTS ISSUED TO ANY PERSON WHO BECOMES AN “ACQUIRING PERSON” OR ITS “AFFILIATES” OR “ASSOCIATES” (AS DEFINED IN THE RIGHTS AGREEMENT) AND CERTAIN RELATED PERSONS, WHETHER CURRENTLY HELD BY OR ON BEHALF OF SUCH PERSON OR BY ANY SUBSEQUENT HOLDER, SHALL BECOME NULL AND VOID.”

Notwithstanding anything herein to the contrary, the Purchaser understands the certificates representing the Securities will bear any other restrictive legends (and a stop-transfer order may be placed against transfer of the certificates for such Securities with respect thereof) that the Company deems necessary or that are otherwise required pursuant to applicable securities laws.

Section 3.9 Authorization; Enforcement. The Purchaser has the requisite power and authority to enter into the Transaction Agreements and to consummate the transactions contemplated hereby and thereby. The Purchaser has taken all necessary action to authorize the execution, delivery and performance of the Transaction Agreements. Each Transaction Agreement has been duly executed by the Purchaser and, assuming the due authorization, execution and delivery of the Transaction Agreements by the Company, constitutes a legal, valid and binding obligation of the Purchaser enforceable against the Purchaser in accordance with its terms, except as enforceability may be limited by applicable bankruptcy, insolvency, reorganization, moratorium or similar laws affecting creditors’ and contracting parties’ rights generally and except as enforceability may be subject to general principles of equity and except as rights to indemnity and contribution may be limited by federal, state or other securities laws or public policy underlying such laws.

Section 3.10 Residency. The Purchaser certifies that it is a resident of the jurisdiction set forth immediately below such Purchaser’s name on the signature pages hereto.

Section 3.11 No Net Short Position. Between the time the Purchaser learned about the Offering (or was first contacted regarding an investment in the Company) and the first public announcement of the Offering by the Company, neither the Purchaser nor any Person acting on its behalf or pursuant to any understanding with it, has engaged, nor has, nor will, the

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Purchaser, directly or indirectly, cause any Person to (but shall cause such Persons not to) engage in any short sales or similar transactions with respect to the Common Stock. Additionally, the Purchaser understands and acknowledges, severally and not jointly with any other Purchaser, that the SEC currently takes the position that coverage of short sales of the Common Stock “against the box” prior to the date such shares are registered is a violation of Section 5 of the Securities Act, as set forth in Item 65, Section 5 under Section A, of the Manual of Publicly Available Telephone Interpretations, dated July 1997, compiled by the Office of Chief Counsel, Division of Corporation Finance.

Section 3.12 Acknowledgements Regarding Placement Agents.

(a) The Purchaser acknowledges that the Placement Agents are acting as the non-exclusive placement agents on a “best efforts” basis for the Securities being offered hereby and will be compensated by the Company for acting in such capacity. The Purchaser represents that (i) (x) the Purchaser was contacted regarding the sale of the Securities by one of the Placement Agents (or an authorized agent or representative thereof) or (y) the Purchaser contacted the Company directly or the Company contacted the Purchaser directly regarding the sale of the Securities and (ii) no Securities were offered or sold to it by means of any form of general solicitation or general advertising.

(b) The Purchaser acknowledges that (i) the Company and each Placement Agent have entered into a Placement Agency Agreement under which each Placement Agent will introduce to the Company accredited investors, (ii) in consideration for such services, the Company will pay each Placement Agent a placement fee equal to 7% of the gross proceeds of Securities sold to investors introduced by such Placement Agent in the Offering and the Company will issue to each Placement Agent a warrant to purchase a number of shares of Common Stock equal to 7% of the number of units issued to investors (inclusive of the Shares and Warrant Shares) introduced by such Placement Agent in the Offering, (iii) Limetree Capital, one of the Placement Agents, is an affiliate of the Company, (iv) the Company will pay to each Placement Agent expenses incurred by it in connection with the Offering that are pre-approved by the Company, and (v) Erich Sager, a partner of Limetree Capital, is a member of our Board of Directors and a stockholder of the Company.

Section 3.13 Anti-Money Laundering Laws. The Purchaser represents and warrants to, and covenants with, the Company that: (i) the Purchaser is in compliance with Executive Order 13224 and the regulations administered by the U.S. Department of the Treasury (“Treasury”) Office of Foreign Assets Control; (ii) the Purchaser, its parents, subsidiaries, affiliated companies, officers, directors and partners, and to the Purchaser’s knowledge, its shareholders, owners, employees, and agents, are not on the List of Specially Designated Nationals and Blocked Persons (“SDN List”) maintained by Treasury and have not been designated by Treasury as a financial institution of primary money laundering concern; (iii) to the Purchaser’s knowledge after reasonable investigation, all of the funds to be used to acquire the Securities are derived from legitimate sources and are not the product of illegal activities; (iv) the Purchaser is in compliance with all other applicable anti-money laundering laws and regulations and has implemented, if applicable, an anti-money laundering compliance program in accordance

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with the requirements of the Bank Secrecy Act, as amended by the USA PATRIOT Act, Pub. L. 107-56; (v) none of the funds being used to purchase the Securities are to the Purchaser’s knowledge proceeds obtained or derived directly or indirectly as a result of illegal activities; and (vi) to the best of its knowledge (A) none of the funds to be provided by the Purchaser are being tendered on behalf of a person or entity who has not been identified to the Purchaser, and (B) it shall promptly notify the Company if the Purchaser discovers that any of such representations cease to be true, and to provide the Company with appropriate information in connection therewith.

ARTICLE 4

Covenants

Section 4.1 Expenses. The Company and each Purchaser shall be severally and not jointly liable for, and each shall pay, its own expenses incurred in connection with the negotiation, preparation, execution and delivery of this Agreement, including, without limitation, attorneys’ and consultants’ fees and expenses.

Section 4.2 Publicity. No Purchaser shall originate any press release or other public announcement, written or oral, relating to this Agreement, or to performance hereunder or the existence of any arrangement among the parties hereto without the prior approval of the Company, except to the extent that such press release or announcement is required by applicable law. The Purchasers acknowledge that the Company will be required to file a copy of this Agreement, and the other agreements and instruments contemplated hereby, with the SEC and to describe these transactions in its public filings and public announcements.

Section 4.3 Sales by Purchasers. Each Purchaser will sell any Securities held by it in compliance with the requirements for an exemption from the prospectus requirement and registration under the Securities Act, any applicable federal, state or other securities laws and the rules and regulations promulgated thereunder. No Purchaser will make any sale, transfer or other disposition of the Securities in violation of federal, state or other securities laws.

Section 4.4 Reservation of Common Stock. The Company shall reserve and keep available at all times during which the Warrants are exercisable, free of preemptive rights, a sufficient number of shares of Common Stock for the purpose of enabling the Company to issue Warrant Shares pursuant to this Agreement.

Section 4.5 Reports. If required by applicable securities legislation, regulations, rules, instruments, policies or orders or by any securities commission or other regulatory authority, the Purchaser will execute, deliver and file such reports, undertakings and other documents with respect to the issue of the Securities as may be required.

ARTICLE 5

Conditions to Closing

Section 5.1 Conditions to Obligations of the Company. The Company’s obligation to complete the sale and issuance of the Securities and deliver such stock certificate(s)

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representing the Shares and Warrants to each Purchaser is subject to the fulfillment, or waiver by the Company, as of the Closing Date (or, if applicable, the Subsequent Closing Date) of the following conditions:

(a) Receipt of Funds. The Company shall have received immediately available funds in the full amount of the purchase price for the Shares and Warrants being purchased hereunder as set forth opposite such Purchaser’s name on Exhibit A attached hereto.

(b) Minimum Aggregate Investment. The Company shall have received at the Closing at least $2.24 million of aggregate proceeds from the sale of the Shares and Warrants hereunder.

(c) Representations and Warranties. The representations and warranties made by each Purchaser in Article 3 shall be true and correct in all material respects as of the Closing Date or, if applicable, the Subsequent Closing Date (except for representations and warranties that speak as of a specific date, which shall be true and correct in all material respects as of such date).

(d) Covenants. All covenants, agreements and conditions contained in this Agreement to be performed by the Purchasers on or prior to the Closing Date (or, if applicable, the Subsequent Closing Date) shall have been performed or complied with in all material respects.

(e) Qualifications. The Company shall have obtained all necessary permits and qualifications, including all blue sky permits and qualifications, or secured exemptions therefrom, required by any state for the offer and sale of the Securities.

(f) Absence of Litigation. No proceeding challenging this Agreement or the transactions contemplated hereby, or seeking to prohibit, alter, prevent or materially delay the Closing, shall have been instituted or be pending before any court, arbitrator, governmental body, agency or official.

(g) No Governmental Prohibition. The sale of the Securities by the Company shall not be prohibited by any law or governmental order or regulation.

(h) No Stop Order. No stop order or suspension of trading shall have been imposed by the SEC or any other governmental or regulatory body with respect to public trading in the Common Stock after the date of the Agreement.

Section 5.2 Conditions to Purchasers’ Obligations at the Closing. Each Purchaser’s obligation to complete the purchase of the Shares and Warrants is subject to the fulfillment, or waiver by the Majority Purchasers, as of the Closing Date of the following conditions:

(a) Representations and Warranties. The representations and warranties made by the Company in Article 2 shall be true and correct in all material respects as of the Closing Date or, if applicable, the Subsequent Closing Date (except for representations and warranties

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that speak as of a specific date, which shall be true and correct in all material respects as of such date).

(b) Covenants. All covenants, agreements and conditions contained in this Agreement to be performed by the Company on or prior to the Closing Date (or, if applicable, the Subsequent Closing Date) shall have been performed or complied with in all material respects.

(c) Qualifications. The Company shall have obtained all necessary permits and qualifications, including all blue sky permits and qualifications, or secured exemptions therefrom, required by any state for the offer and sale of the Securities.

(d) Transfer Agent Instructions. The Company shall have delivered to its transfer agent irrevocable instructions to issue to such Purchaser or in such nominee name(s) as designated by such Purchaser in writing certificates representing such number of Shares set forth opposite such Purchaser’s name on Exhibit A attached hereto.

(e) Absence of Litigation. No proceeding challenging this Agreement or the transactions contemplated hereby, or seeking to prohibit, alter, prevent or materially delay the Closing, shall have been instituted or be pending before any court, arbitrator, governmental body, agency or official.

(f) No Governmental Prohibition. The sale of the Securities by the Company shall not be prohibited by any law or governmental order or regulation.

(g) No Stop Order. No stop order or suspension of trading shall have been imposed by the SEC or any other governmental or regulatory body with respect to public trading in the Common Stock after the date of the Agreement.

ARTICLE 6

Registration Rights

Section 6.1 Required Registration.

(a) Subject to Section 6.1(b), the Company shall no later than 75 days after the Closing Date, subject to receipt of necessary information from the Purchasers, prepare and file with the SEC a registration statement covering the resale of all of the Registrable Securities (the “Registration Statement”).

(b) Notwithstanding anything herein to the contrary, in the event that the Company, in its sole discretion, decides to limit the amount of shares of Common Stock that may be included in the Registration Statement (such number of shares of Common Stock which the Company decides to include in the Registration Statement, the “Allowable Maximum”), the number of Registrable Securities sought to be included in the Registration Statement shall be cutback and removed from the Registration Statement until the aggregate number of such Registrable Securities to be included in the Registration Statement equals the Allowable Maximum. Such cutbacks will be in the following order:

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(i) first there shall be excluded any securities of the Company included or to be included in the Registration Statement, whether pursuant to piggyback or demand registration rights or otherwise requested to be included, other than the Registrable Securities, the shares of Common Stock to be issued to the Placement Agents (the “Placement Agents Shares”), and the shares of Common Stock underlying the warrant(s) to be issued to the Placement Agents (the “Placement Agents Warrant Shares”); next

(ii) the Placement Agents Warrant Shares shall be excluded; next

(iii) the Placement Agents Shares shall be excluded; next

(iv) the Warrant Shares shall be excluded; and next

(v) the Shares shall be excluded, until the Allowable Maximum is not exceeded.

Except as specified in the preceding sentence, any required cutbacks within each of Sections 6.1(b)(iv) and 6.1(b)(v) shall be applied to such Holders pro rata in accordance with the number of Warrant Shares or Shares, respectively, then-held by such Holders and sought to be included in such Registration Statement.

Section 6.2 Registration Expenses and Selling Expenses. All Registration Expenses incurred in connection with any registration, qualification, exemption or compliance pursuant to Section 6.1 shall be borne by the Company. All Selling Expenses incurred in connection with any registrations under this Agreement shall be borne by the Holders of the securities so registered pro rata on the basis of the number of shares so registered and all Selling Expenses relating to the sale of securities registered or by or on behalf of any Holder shall be borne by such Holders.

Section 6.3 Registration Period Covenants. At its expense, during the period that the Company keeps the Registration Statement effective, in its discretion (the “Registration Period”), the Company shall:

(a) advise the Holders:

(i) within five Business Days of when the Registration Statement or any amendment thereto has been filed with the SEC and within five Business Days of receiving notice or obtaining knowledge of the effectiveness of the Registration Statement or any post-effective amendment thereto;

(ii) within five Business Days of any request by the SEC for amendments or supplements to the Registration Statement or the prospectus included therein or for additional information;

(iii) within five Business Days of receiving notice or obtaining knowledge of the issuance by the SEC of any stop order suspending the effectiveness of the Registration Statement or the initiation of any proceedings for such purpose; and

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(iv) within five Business Days of the occurrence of any event that requires the making of any changes in the Registration Statement or the prospectus so that, as of such date, the statements therein are not misleading and do not omit to state a material fact required to be stated therein or necessary to make the statements therein (in the case of the prospectus, in light of the circumstances under which they were made) not misleading;

(b) use its commercially reasonable efforts to obtain the withdrawal of any order suspending the effectiveness of the Registration Statement as soon as reasonably practicable;

(c) promptly deliver to each such Holder, without charge, as many copies of the prospectus included in such Registration Statement and any amendment or supplement thereto as such Holder may reasonably request in writing; and the Company consents to the use, consistent with the provisions hereof, of the prospectus or any amendment or supplement thereto by each of the selling Holders of Registrable Securities in connection with the offering and sale of the Registrable Securities covered by the prospectus or any amendment or supplement thereto;

(d) if a Holder so requests in writing, deliver to each Holder, without charge, (i) one copy of the following documents, other than those documents available via the Electronic Data Gathering, Analysis, and Retrieval (EDGAR) system: (A) its annual report to its stockholders, if any (which annual report shall contain financial statements audited in accordance with generally accepted accounting principles in the United States of America by a firm of certified public accountants of recognized standing); (B) if not included in substance in its annual report to stockholders, its annual report on Form 10-K (or similar form); (C) its definitive proxy statement (or similar form) with respect to its most recent annual meeting of stockholders; (D) each of its quarterly reports to its stockholders, and, if not included in substance in its quarterly reports to stockholders, its quarterly report on Form 10-Q (or similar form); and (E) a copy of the full Registration Statement (the foregoing, in each case, excluding exhibits); and (ii) if explicitly requested, all exhibits excluded by the parenthetical to the immediately preceding clause (E);

(e) prior to any public offering of Registrable Securities pursuant to the Registration Statement, promptly take such actions as may be necessary to register or qualify or obtain an exemption for offer and sale under the securities or blue sky laws of such United States jurisdictions as any such Holders reasonably request in writing, provided that the Company shall not for any such purpose be required to qualify generally to transact business as a foreign corporation in any jurisdiction where it is not so qualified, consent to general service of process in any such jurisdiction, or subject itself to any material tax in any such jurisdiction where it is not so then subject; and

(f) upon the occurrence of any event contemplated by Section 6.3(a)(iv) above, except for such times as the Company is permitted hereunder to suspend the use of the prospectus forming part of the Registration Statement, the Company shall use its commercially reasonable efforts to as soon as reasonably practicable prepare a post-effective amendment to the Registration Statement or a supplement to the related prospectus, or file any other required document so that, as thereafter delivered to purchasers of the Registrable Securities included therein, the prospectus will not include any untrue statement of a material fact or omit to state

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any material fact necessary to make the statements therein, in light of the circumstances under which they were made, not misleading.

Section 6.4 Certain Limitations. The Holders shall have no right to take any action to restrain, enjoin or otherwise delay any registration pursuant to Section 6.1 as a result of any controversy that may arise with respect to the interpretation or implementation of this Agreement.

Section 6.5 Indemnity.

(a) To the extent permitted by law, the Company shall indemnify each Holder and each person controlling such Holder within the meaning of Section 15 of the Securities Act, with respect to which the Registration Statement that has been effected pursuant to this Agreement, against all claims, losses, damages and liabilities (or action in respect thereof), including any of the foregoing incurred in settlement of any litigation, commenced or threatened (subject to Section 6.5(c)), arising out of or based on any untrue statement (or alleged untrue statement) of a material fact contained in the Registration Statement, prospectus, any amendment or supplement thereof, or based on any omission (or alleged omission) to state therein a material fact required to be stated therein or necessary to make the statements therein not misleading, in light of the circumstances in which they were made, or any violation by the Company of any rule or regulation promulgated by the Securities Act applicable to the Company and relating to any action or inaction required of the Company in connection with any such registration, qualification or compliance, and will reimburse each Holder and each person controlling such Holder, for reasonable legal and other out-of-pocket expenses reasonably incurred in connection with investigating or defending any such claim, loss, damage, liability or action as incurred; provided that the Company will not be liable in any such case to the extent that any untrue statement or omission or allegation thereof is made in reliance upon and in conformity with written information furnished to the Company by or on behalf of such Holder for use in preparation of the Registration Statement, prospectus, amendment or supplement; provided further that the Company will not be liable in any such case where the claim, loss, damage or liability arises out of or is related to the failure of such Holder to comply with the covenants and agreements contained in this Agreement respecting sales of Registrable Securities, and except that the foregoing indemnity agreement is subject to the condition that, insofar as it relates to any such untrue statement or alleged untrue statement or omission or alleged omission made in the preliminary prospectus but eliminated or remedied in the amended prospectus on file with the SEC at the time the Registration Statement becomes effective or in the amended prospectus filed with the SEC pursuant to Rule 424(b) or in the prospectus subject to completion under Rule 434 of the Securities Act, which together meet the requirements of Section 10(a) of the Securities Act (the “Final Prospectus”), such indemnity shall not inure to the benefit of any such Holder or any such controlling person, if a copy of the Final Prospectus furnished by the Company to the Holder for delivery was not furnished to the person or entity asserting the loss, liability, claim or damage at or prior to the time such furnishing is required by the Securities Act and the Final Prospectus would have cured the defect giving rise to such loss, liability, claim or damage.

(b) Each Holder will severally, and not jointly, indemnify the Company, each of its directors and officers, and each person who controls the Company within the meaning of Section 15 of the Securities Act, against all claims, losses, damages and liabilities (or actions in

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respect thereof), including any of the foregoing incurred in settlement of any litigation, commenced or threatened (subject to Section 6.5(c) below), arising out of or based on any untrue statement (or alleged untrue statement) of a material fact contained in the Registration Statement, prospectus, or any amendment or supplement thereof, incident to any such registration, or based on any omission (or alleged omission) to state therein a material fact required to be stated therein or necessary to make the statements therein not misleading, in light of the circumstances in which they were made, and will reimburse the Company, such directors and officers, and each person controlling the Company for reasonable legal and other out-of-pocket expenses reasonably incurred in connection with investigating or defending any such claim, loss, damage, liability or action as incurred, in each case to the extent, but only to the extent, that such untrue statement or omission or allegation thereof is made in reliance upon and in conformity with written information furnished to the Company by or on behalf of the Holder for use in preparation of the Registration Statement, prospectus, amendment or supplement. Notwithstanding the foregoing, a Holder’s aggregate liability pursuant to this subsection (b) shall be limited to the net amount received by the Holder from the sale of the Registrable Securities giving rise to such claims, losses, damages and liabilities (and actions in respect thereof).

(c) Each party entitled to indemnification under this Section 6.5 (the “Indemnified Party”) shall give notice to the party required to provide indemnification (the “Indemnifying Party”) promptly after such Indemnified Party has actual knowledge of any claim as to which indemnity may be sought, and shall permit the Indemnifying Party (at its expense) to assume the defense of any such claim or any litigation resulting therefrom; provided that the Indemnified Party may participate in such defense at such Indemnified Party’s expense; provided further that the failure of any Indemnified Party to give notice as provided herein shall not relieve the Indemnifying Party of its obligations under this Agreement, unless such failure is materially prejudicial to the Indemnifying Party in defending such claim or litigation. An Indemnifying Party shall not be liable for any settlement of an action or claim effected without its written consent (which consent will not be unreasonably withheld, conditioned or delayed). No Indemnifying Party, in its defense of any such claim or litigation, shall, except with the consent (such consent not to be unreasonably withheld, conditioned or delayed) of the Indemnified Party consent to entry of any judgment or enter into any settlement which does not include as an unconditional term thereof the giving by the claimant or plaintiff to such Indemnified Party of a release from all liability in respect to such claim or litigation.

(d) If the indemnification provided for in this Section 6.5 is held by a court of competent jurisdiction to be unavailable to an Indemnified Party or is insufficient to hold such Indemnified Party harmless with respect to any loss, liability, claim, damage or expense referred to therein, then the Indemnifying Party shall contribute to the amount paid or payable by such Indemnified Party as a result of such loss, liability, claim, damage or expense in such proportion as is appropriate to reflect the relative fault of the Indemnifying Party on the one hand and of the Indemnified Party on the other in connection with the statements or omissions which resulted in such loss, liability, claim, damage or expense as well as any other relevant equitable considerations. The relative fault of the Indemnifying Party and of the Indemnified Party shall be determined by reference to, among other things, whether the untrue or alleged untrue statement of a material fact or the omission to state a material fact relates to information supplied by the Indemnifying Party or by the Indemnified Party and the parties’ relative intent, knowledge, access to information and opportunity to correct or prevent such statement or omission.

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Notwithstanding the foregoing, a Holder’s aggregate liability pursuant to this subsection (d) shall be limited to the net amount received by the Holder from the sale of Registrable Securities giving rise to such loss, liability, claim, damage or expense (or actions in respect thereof).

Section 6.6 Additional Covenants and Agreements of the Holders.

(a) Each Holder agrees that, upon receipt of any notice from the Company of the happening of any event requiring the preparation of a supplement or amendment to a prospectus relating to Registrable Securities so that, as thereafter delivered to the Holders, such prospectus shall not contain an untrue statement of a material fact or omit to state any material fact required to be stated therein or necessary to make the statements therein not misleading, each Holder will immediately discontinue disposition of Registrable Securities pursuant to the Registration Statement and prospectus contemplated by this Agreement until its receipt of copies of the supplemented or amended prospectus from the Company and, if so directed by the Company, each Holder shall deliver to the Company all copies, other than permanent file copies then in such Holder’s possession, of the prospectus covering such Registrable Securities current at the time of receipt of such notice.

(b) Each Holder shall suspend, upon request of the Company, any disposition of Registrable Securities pursuant to the Registration Statement and prospectus contemplated by Section 6.1 during no more than two periods of no more than 45 calendar days each during any 12-month period to the extent that the Board of Directors of the Company determines in good faith that the Company may be required to disclose any material corporate development which disclosure may have a Material Adverse Effect on the Company.

(c) As a condition to the inclusion of its Registrable Securities, each Holder shall furnish to the Company such information regarding such Holder and the distribution proposed by such Holder as the Company may request in writing, including completing one or more registration statement questionnaires in the form(s) provided by the Company or as shall be required in connection with any registration referred to in this Article 6, duly executed by the Holder, in each case within three Business Days after such information is requested by the Company. The Holder covenants that upon the Company’s request, such Holder will promptly notify the Company of any changes in such information or other information, under signature of the Holder, as the Company may request in connection with any registration referred to in this Article 6.

(d) Each Holder hereby covenants with the Company: (i) not to make any sale of the Registrable Securities without effectively causing the prospectus delivery requirements under the Securities Act to be satisfied; and (ii) if such Registrable Securities are to be sold by any method or in any transaction other than on a national securities exchange or in the over-the-counter market, in privately negotiated transactions, or in a combination of such methods, to notify the Company at least five Business Days prior to the date on which the Holder first offers to sell any such Registrable Securities.

(e) Each Holder acknowledges and agrees that the Registrable Securities sold pursuant to the Registration Statement are not transferable on the books of the Company unless the stock certificate submitted to the transfer agent evidencing such Registrable Securities is

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accompanied by a certificate reasonably satisfactory to the Company to the effect that: (i) the Registrable Securities have been sold in accordance with such Registration Statement; and (ii) the requirement of delivering a current prospectus has been satisfied, together with any other documentation required by the transfer agent.

(f) Each Holder agrees not to take any action with respect to any distribution deemed to be made pursuant to such Registration Statement which would constitute a violation of Regulation M under the Exchange Act or any other applicable rule, regulation or law.

(g) At the end of the Registration Period, the Holders shall discontinue sales of shares pursuant to the Registration Statement upon receipt of notice from the Company of its intention to remove from registration the shares covered by the Registration Statement which remain unsold, and such Holders shall notify the Company of the number of shares registered which remain unsold immediately, but in any event no later than three Business Days, following receipt of such notice from the Company.

Section 6.7 Additional Covenants and Agreements of the Company. With a view to making available to the Holders the benefits of certain rules and regulations of the SEC which at any time permit the sale of the Registrable Securities to the public without registration, so long as the Holders own Registrable Securities, the Company shall use its commercially reasonable efforts to:

(a) make and keep public information available, as those terms are understood and defined in Rule 144 under the Securities Act, at all times;

(b) file with the SEC in a timely manner all reports and other documents required of the Company under the Exchange Act; and

(c) so long as a Holder owns any Registrable Securities, furnish to such Holder, upon any reasonable request, a written statement by the Company as to its compliance with Rule 144 under the Securities Act, and of the Exchange Act, a copy of the most recent annual or quarterly report of the Company, and such other reports and documents of the Company as such Holder may reasonably request in writing in availing itself of any rule or regulation of the SEC allowing a Holder to sell any such securities without registration.

Section 6.8 Assignment of Registration Rights. The rights to cause the Company to register Registrable Securities granted to the Holders by the Company under Section 6.1 may not be assigned or transferred to any other Person under any circumstances.

ARTICLE 7

Definitions

Section 7.1 Definitions. The following capitalized terms have the following meanings in this Agreement:

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“Affiliate” means, with respect to any Person, any other Person controlling, controlled by or under direct or indirect common control with such Person (for the purposes of this definition “control,” when used with respect to any specified Person, shall mean the power to direct the management and policies of such person, directly or indirectly, whether through ownership of voting securities, by contract or otherwise; and the terms “controlling” and “controlled” shall have meanings correlative to the foregoing).

“Business Day” means a day Monday through Friday on which banks are generally open for business in Novato, California.

“Closing” has the meaning set forth in Section 1.3.

“Closing Date” has the meaning set forth in Section 1.3.

“Exchange Act” means the Securities Exchange Act of 1934, as amended.

“Filing Date” has the meaning set forth in Section 6.1.

“Final Prospectus” has the meaning set forth in Section 6.6(a).

“Financial Statements” means the financial statements of the Company included in the Public Filings.

“Holder” means any person holding Registrable Securities.

“Indemnified Party” has the meaning set forth in Section 6.5(c).

“Indemnifying Party” has the meaning set forth in Section 6.5(c).

“Intellectual Property” has the meaning set forth in Section 2.8.

“Majority Purchasers” means, as of the Closing Date or a Subsequent Closing Date, the Purchasers who have purchased prior to such date or are then purchasing as of such date a majority of the Shares, as set forth on Exhibit A attached hereto.

“Material Adverse Effect” means a material adverse effect on the business, operations, assets, financial condition, market capitalization or results of operations of the Company and its subsidiaries, taken as a whole.

“Offering” means the private placement of the Securities contemplated by this Agreement.

“Person” means any person, individual, corporation, limited liability company, partnership, trust or other nongovernmental entity or any governmental agency, court, authority or other body (whether foreign, federal, state, local or otherwise).

“Placement Agents” means, collectively, Limetree Capital and any other firm serving as a placement agent in connection with this Offering.

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Placement Agents Shares has the meaning set forth in Section 6.1(b)(i).

Placement Agents Warrant Shares” has the meaning set forth in Section 6.1(b)(i).

“Public Filings” means the Company’s Annual Report filed with the SEC on Form 10-K/A on December 23, 2008 for the fiscal year ended August 31, 2008, and Quarterly Report filed with the SEC on Form 10-Q on April 13, 2009 for the quarterly period ended February 28, 2009.

“Purchasers” mean the Purchasers whose names are set forth on the signature pages of this Agreement, and their permitted transferees.

“Purchase Price” has the meaning set forth in Section 1.1.

“register,” “registered” and “registration” refer to the registration effected by preparing and filing a registration statement in compliance with the Securities Act, and the declaration or ordering of the effectiveness of such registration statement.

“Registrable Securities” means (i) the Shares and (ii) the Warrant Shares; provided, however, that securities shall only be treated as Registrable Securities if and only for so long as they (A) have not been disposed of pursuant to a registration statement declared effective by the SEC; or (B) have not been sold in a transaction exempt from the registration and prospectus delivery requirements of the Securities Act so that all transfer restrictions and restrictive legends with respect thereto are removed upon the consummation of such sale.

“Registration Expenses” means all expenses incurred by the Company in complying with Section 6.1, including, without limitation, all registration, qualification and filing fees, printing expenses, escrow fees, fees and expenses of counsel for the Company, blue sky fees and expenses and the expense of any special audits incident to or required by any such registration (but excluding any Selling Expenses and the costs and fees of legal counsel for any Holder).

“Registration Period” has the meaning set forth in Section 6.3(a).

“Registration Statement” has the meaning set forth in Section 6.1.

“Rule 144” means Rule 144 promulgated under the Securities Act, or any successor rule.

“SEC” means the United States Securities and Exchange Commission.

“Securities” has the meaning set forth in Section 1.4.

“Securities Act” means the Securities Act of 1933, as amended, and the rules and regulations thereunder, or any similar successor statute.

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“Selling Expenses” means all selling commissions and underwriting discounts applicable to the sale of Registrable Securities and all fees and expenses of legal counsel for any Holder.

“Shares” has the meaning set forth in Section 1.1.

“Subsequent Closing” has the meaning set forth in Section 1.4.

“Subsequent Closing Date” has the meaning set forth in Section 1.4.

“Warrant Shares” has the meaning set forth in Section 1.1.

“Warrants” has the meaning set forth in Section 1.1.

“Transaction Agreements” means, collectively, this Agreement and the Warrants.

Section 7.2 Certain Interpretations. Except where expressly stated otherwise in this Agreement, the following rules of interpretation apply to this Agreement: (i) “or” is not exclusive and “include”, “includes” and “including” are not limiting; (ii) definitions contained in this Agreement are applicable to the singular as well as the plural forms of such terms; (iii) references to an agreement or instrument mean such agreement or instrument as from time to time amended, modified or supplemented; (iv) references to a Person are also to its successors and permitted assigns; (v) references to an “Article”, “Section”, “Exhibit”, or “Schedules” refer to an Article of, a Section of, an Exhibit to, or a Schedule to, this Agreement, respectively; and (vi) words importing the masculine gender include the feminine or neuter and, in each case, vice versa.

ARTICLE 8

Governing Law; Miscellaneous

Section 8.1 Governing Law; Jurisdiction; Waiver of Jury Trial. This Agreement will be governed by and interpreted in accordance with the laws of the State of Delaware without regard to the principles of conflict of laws. Each of the parties hereto irrevocably submits and consents to the exclusive jurisdiction of the courts of the State of California located in Marin County and the United States District Court for the Northern District of California for the purpose of any suit, action, proceeding or judgment relating to or arising out of this Agreement and the transactions contemplated hereby. Each party hereto irrevocably waives any objection to the laying of venue of any such suit, action or proceeding brought in such courts and irrevocably waives any claim that any such suit, action or proceeding brought in any such court has been brought in an inconvenient forum. EACH OF THE PARTIES HERETO WAIVES ANY RIGHT TO REQUEST A TRIAL BY JURY IN ANY LITIGATION WITH RESPECT TO THIS AGREEMENT AND REPRESENTS THAT COUNSEL HAS BEEN CONSULTED SPECIFICALLY AS TO THIS WAIVER.

Section 8.2 Rights of Purchasers Inter Se. Each Purchaser shall have the absolute right to exercise or refrain from exercising any right or rights which such Purchaser may

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have by reason of this Agreement or any of the Securities, including, without limitation, the right to consent to the waiver of any obligation of the Company under this Agreement and to enter into an agreement with the Company for the purpose of modifying this Agreement or any agreement effecting any such modification, and such Purchaser shall not incur any liability to any other Purchaser or Purchasers with respect to exercising or refraining from exercising any such right or rights.

Section 8.3 Exculpation Among Purchasers. Each Purchaser acknowledges that it is not relying upon any other Purchaser, or any officer, director, employee, agent, partner, member or affiliate of any such other Purchaser, in making its investment or decision to invest in the Company or in monitoring such investment. Each Purchaser agrees that no Purchaser nor any Affiliate or controlling person, officer, director, stockholder, partner, member, agent or employee of any Purchaser shall be liable for any action heretofore or hereafter taken or omitted to be taken by any of them relating to or in connection with the Company or the Securities, or both. Without limiting the generality of the foregoing, no Purchaser (nor any of its Affiliates, officers, directors, stockholders, partners, members, agents or employees) shall have any obligation, liability or responsibility whatsoever for the accuracy, completeness or fairness of any or all information about the Company or its properties, business or financial and other affairs, acquired by such Purchaser from the Company or its officers, directors, employees, agents, representatives, counsel or auditors, and in turn provided to another Purchaser, nor shall any such Purchaser (or such other Person) have any obligation or responsibility whatsoever to provide any such information to any other Purchaser (or such other Person) or to continue to provide any such information if any information is provided.

Section 8.4 Counterparts; Signatures by Facsimile. This Agreement may be executed in two or more counterparts, all of which are considered one and the same agreement and will become effective when counterparts have been signed by each party and delivered to the other parties. This Agreement, once executed by a party, may be delivered to the other parties hereto by facsimile transmission of a copy of this Agreement bearing the signature of the party so delivering this Agreement.

Section 8.5 Headings. The headings of this Agreement are for convenience of reference only, are not part of this Agreement and do not affect its interpretation.

Section 8.6 Severability. If any immaterial provision of this Agreement is invalid or unenforceable under any applicable statute or rule of law, then such provision will be deemed modified in order to conform with such statute or rule of law. Any provision hereof that may prove invalid or unenforceable under any law will not affect the validity or enforceability of any other provision hereof.

Section 8.7 Entire Agreement; Amendments.

(a) This Agreement (including all schedules and exhibits hereto), each other Transaction Agreement, and any confidentiality agreement entered into between the Company and a Purchaser (which confidentiality agreement shall continue to be in full force and effect) constitute the entire agreement among the parties hereto with respect to the subject matter hereof and thereof. There are no restrictions, promises, warranties or undertakings, other than those set

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forth or referred to herein or therein. This Agreement supersedes all prior agreements and understandings among the parties hereto with respect to the subject matter hereof. No provision of this Agreement may be waived or amended other than by an instrument in writing signed by the Company and the Majority Purchasers. Any amendment or waiver effected in accordance with this Section 8.7 shall be binding upon each holder of any Securities purchased under this Agreement at the time outstanding (including securities into which such Securities are convertible and for which such Securities are exercisable), each future holder of all such Securities, and the Company.

(b) Upon the effectuation of each such waiver, or amendment, the Company shall promptly give written notice thereof to the Purchasers who have not previously consented thereto in writing.

(c) Each Purchaser acknowledges that by operation of this Section 8.7 the Majority Purchasers have the right and power to diminish or eliminate certain rights of such Purchaser under this Agreement.

Section 8.8 Notices. All notices required or permitted hereunder shall be in writing and shall be deemed effectively given: (a) upon personal delivery to the party to be notified; (b) when sent by confirmed email or facsimile if sent during normal business hours of the recipient; if not, then on the next business day; (c) five days after having been sent by registered or certified mail, return receipt requested, postage prepaid; or (d) one business day after deposit with a nationally recognized overnight courier, specifying next day delivery, with written verification of receipt. The addresses for such communications are:

If to the Company:

Raptor Pharmaceuticals Corp.
9 Commercial Boulevard, Suite 200
Novato, CA 94949
Attn: Christopher Starr, Ph.D., CEO
Fax: 415-382-1368
Email: cstarr@raptorpharma.com

With copies to (which copies shall not constitute notice to the Company):

Paul, Hastings, Janofsky & Walker, LLP
515 South Flower Street, 25th Floor
Los Angeles, CA 90071-2228
Attn: Siobhan McBreen Burke
Fax: 213-627-0705
Email: siobhanburke@paulhastings.com

If to a Purchaser: To the address set forth immediately below such Purchaser’s name on the signature pages hereto. Each party will provide ten days’ advance written notice to the other parties of any change in its address.

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Section 8.9 Successors and Assigns. This Agreement is binding upon and inures to the benefit of the parties and their successors and permitted assigns. No Purchaser may assign this Agreement or any rights or obligations hereunder without the prior written consent of the Company.

Section 8.10 Third Party Beneficiaries. This Agreement is intended for the benefit of the parties hereto, their respective successors and permitted assigns, and is not for the benefit of, nor may any provision hereof be enforced by, any other Person.

Section 8.11 Further Assurances. Each party will do and perform, or cause to be done and performed, all such further acts and things, and will execute and deliver all other agreements, certificates, instruments and documents, as another party may reasonably request in order to carry out the intent and accomplish the purposes of this Agreement and the consummation of the transactions contemplated hereby.

Section 8.12 No Strict Construction. The language used in this Agreement is deemed to be the language chosen by the parties to express their mutual intent, and no rules of strict construction will be applied against any party.

Section 8.13 Independent Nature of Purchasers’ Obligations and Rights. The obligations of each Purchaser under this Agreement are several and not joint with the obligations of any other Purchaser, and no Purchaser shall be responsible in any way for the performance of the obligations of any other Purchaser under this Agreement. Each Purchaser shall be entitled to independently protect and enforce its rights, including without limitation the rights arising out of this Agreement, and it shall not be necessary for any other Purchaser to be joined as an additional party in any proceeding for such purpose.

[Signature Pages Follow]

 

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COMPANY:

Raptor Pharmaceuticals Corp.

By: /s/ Kim R. Tsuchimoto                                           
Name: Kim R. Tsuchimoto
Its: Chief Financial Officer

PURCHASERS:

Rune Kjeken

By: /s/ Rune Kjeken                                                      
Name: Rune Kjeken

VP Bank (Switzerland) Ltd.

By: /s/ Daniel Lacher /s/ Andre Roth                       
Name: Daniel Lacher            Andre Roth
Its: Vice President            Vice President

Charles B. Scoville, Jr. Trust,
Daniel B. Scholefield & Thomas J. Scoville,
Trustees

By: /s/ Daniel B. Scholefield                                        
Name: Daniel B. Scholefield
Its: Trustee

Aran Asset Management SA.

By: /s/ Michael C. Thalmann                                        
Name: Michael C. Thalmann
Its: Chairman / CEO

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EX-10.19 4 c91590exv10w19.htm EXHIBIT 10.19 Exhibit 10.19
EXECUTION VERSION
EXHIBIT 10.19
Collaboration and License Agreement
This Agreement is entered into with effect as of the Effective Date (as defined below)
by and between
F. Hoffmann-La Roche Ltd
with an office and place of business at Grenzacherstrasse 124, 4070 Basel, Switzerland (“Roche Basel”);
and
Hoffmann-La Roche Inc.
with an office and place of business at 340 Kingsland Street, Nutley, New Jersey 07110, U.S.A. (“Roche Nutley”; Roche Basel and Roche Nutley together referred to as “Roche”)
on the one hand;
and
Raptor Pharmaceutical Inc.
with their office and place of business at 9 Commercial Blvd., Suite 200, Novato, California 94949. (“Raptor”)
on the other hand.
WHEREAS, Raptor has expertise in the RAP proteins used to deliver therapeutics across the blood brain barrier and possesses its Nuerotrans, proprietary technology and intellectual property rights relating thereto; and
WHEREAS, Roche has expertise in the research, development, manufacture and commercialization of pharmaceutical products; and

 

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WHEREAS, Roche wishes to develop and commercialize products containing pharmaceutically active compounds and RAP proteins as a delivery vehicle, and explore potential applications of such products in various therapeutic areas; and
WHEREAS, Raptor wishes to work with Roche in generating, selecting and testing RAP proteins for use with pharmaceutically active compounds in products and is willing to grant to Roche the right to make, use, offer for sale, sell, import and export such products under certain of its intellectual property rights, as contemplated herein.
NOW, THEREFORE, in consideration of the mutual covenants and promises contained in this Agreement and other good and valuable consideration, the receipt and sufficiency of which are hereby acknowledged, the Parties hereto, intending to be legally bound, do hereby agree as follows:
1. Definitions
As used in this Agreement, the following terms, whether used in the singular or plural, shall have the following meanings:
1.1 Affiliate
The term “Affiliate” shall mean, as to a Party:
  a)  
An entity that owns directly or indirectly, a controlling interest in such Party, by stock ownership or otherwise;
  b)  
any entity in which such Party owns a controlling interest, by stock ownership or otherwise, or;
 
  c)  
any entity under common control with such Party, directly or indirectly.
For purposes of this paragraph, “controlling interest” and “control” mean ownership of more than fifty percent (50%) of the voting stock permitted to vote for the election of the board of directors or any other arrangement resulting in control of or the right to control the management and the affairs of the entity or Party in question. Anything to the contrary in this paragraph notwithstanding, Chugai Pharmaceutical Co., Ltd, a Japanese corporation, (“Chugai”) shall not be deemed an Affiliate of Roche unless Roche provides written notice to Raptor of its desire to include Chugai as an Affiliate of Roche.
1.2 Agreement
The term “Agreement” shall mean this document including any and all appendices and amendments to it as may be added and/or amended from time to time in accordance with the provisions of this Agreement.
1.3 Agreement Term
The term “Agreement Term” shall mean the term of this Agreement as set forth in Section 14.1.

 

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1.4 Business Day
The term “Business Day” shall mean 9.00 a.m. to 5.00 p.m. local time on a day other than a Saturday, Sunday or bank or other public or federal holiday in Switzerland or the State of New Jersey in the US.
1.5 Calendar Quarter
The term “Calendar Quarter” shall mean each period of three (3) consecutive calendar months, ending March 31, June 30, September 30, and December 31.
1.6 Calendar Year
The term “Calendar Year” shall mean each twelve (12) month period ending on December 31.
1.7 Change of Control
The term “Change of Control” shall mean, with respect to Raptor: (a) the acquisition by any Third Party of beneficial ownership of more than [*****] of the then-outstanding common shares or voting power of Raptor, other than acquisitions by employee benefit plans sponsored or maintained by Raptor or by existing shareholders of Raptor; or (b) the consummation of a business combination with a Third Party, unless, following such business combination, the stockholders of Raptor immediately prior to such business combination beneficially own directly or indirectly more than [*****] of the then-outstanding common shares or voting power of the entity resulting from such business combination or of the ultimate parent of such resulting entity.
1.8 Clinical Study
The term “Clinical Study” shall mean a Phase I Study, a Phase II Study or a Phase III Study, as applicable.
1.9 Commercially Reasonable Efforts
The term “Commercially Reasonable Efforts” shall mean [*****].
1.10 Confidential Information
The term “Confidential Information” shall mean any and all information, data or know-how (including Know-How), whether technical or non-technical, oral or written, that is disclosed by one Party or its Affiliates (“Disclosing Party”) to the other Party or its Affiliates (“Receiving Party”). Confidential Information shall not include any information, data or know-how which:
(i) was generally available to the public at the time of disclosure, or information which becomes available to the public after disclosure by the Disclosing Party other than through fault (whether by action or inaction) of the Receiving Party,
(ii) can be shown by clear and convincing written records to have been already known to the Receiving Party prior to its receipt from the Disclosing Party,
(iii) is obtained at any time lawfully from a Third Party rightfully in possession of such information, data or know-how and under circumstances permitting its use or disclosure,
(iv) is developed independently by the Receiving Party as evidenced by clear and convincing written records other than through knowledge of Confidential Information, or
(v) is approved in writing by the Disclosing Party for release by the Receiving Party.
The terms of this Agreement shall be considered Confidential Information of both Parties.

 

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1.11 Control
The term “Control” shall mean (as an adjective or as a verb including conjugations and variations such as “Controls” “Controlled” or “Controlling”) (a) with respect to Patent Rights and/or Know-How, the possession by a Party of the ability to grant a license or sublicense of such Patent Rights and/or Know-How without violating the terms of any agreement or arrangement between such Party and any other party and (b) with respect to proprietary materials, the possession by a Party of the ability to supply such proprietary materials to the other Party as provided herein without violating the terms of any agreement or arrangement between such Party and any other party [*****].
1.12 Cover
The term “Cover” shall mean (as an adjective or as a verb including conjugations and variations such as “Covered,” “Coverage” or “Covering”) that the developing, making, using, offering for sale, promoting, selling, exporting or importing of a given compound, formulation or product would infringe a Valid Claim in the absence of a license under the Patent Rights to which such Valid Claim pertains. The determination of whether a compound, formulation, process or product is Covered by a particular Valid Claim shall be made on a country-by-country basis.
1.13 Dollars
The term “Dollars” shall mean US dollars.
1.14 Effective Date
The term “Effective Date” shall mean June 3, 2009.
1.15 EU
The term “EU” shall mean the European Union, as it may be constituted from time to time.
Exclusivity Period
The term “Exclusivity Period” shall mean [*****]. The Exclusivity Period can be extended pursuant to Section 6.1.1.
1.17 FDA
The term “FDA” shall mean the Food and Drug Administration of the United States of America.
1.18 Field
The term “Field” shall mean all human uses for delivery of diagnostic or therapeutic agents across the blood brain barrier for the diagnosis treatment and/or prevention of any Indication.
1.19 First Commercial Sale
The term “First Commercial Sale” shall mean the first invoiced sale of a Product to a Third Party by the Roche Group.

 

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1.20 Handle
The term “Handle” shall mean preparing, filing, prosecuting (including interference and opposition proceedings) and maintaining (including interferences, reissue, re-examination, revocation and opposition proceedings).
1.21 Indication
The term “Indication” shall mean any human disease or condition, or sign or symptom of a human disease or condition, in each case determined by reference to the World Health Organization International Classification of Diseases, version 10, as it may be revised and updated from time to time.
1.22 Initiation
The term “Initiation” shall mean the date that a human is first dosed with the Product in a Clinical Study conducted by or on behalf of Roche or any of its Affiliates or Sublicensees and approved by the respective Regulatory Authority.
1.23 Insolvency Event
The term “Insolvency Event” shall mean circumstances under which a Party (i) has a receiver or similar officer appointed over all or substantially all of its assets or undertaking; (ii) passes a resolution for winding-up (other than a winding-up for the purpose of, or in connection with, any solvent amalgamation, reorganization or restructuring) or a court makes an order to that effect or a court makes an order for administration (or any equivalent order in any jurisdiction); (iii) enters into any voluntary composition or arrangement with its creditors (other than relating to a solvent amalgamation, reorganization or restructuring) relating to all or substantially all of its assets; or (iv) permanently ceases to carry on business; in the case of each of the foregoing clauses (i) and (ii), where the event shall have continued for [*****] undismissed, unbonded and undischarged.
1.24 Inventions
The term “Invention” shall mean an invention that is conceived and/or reduced to practice in the course of performing an activity pursuant to the Research Plan under this Agreement. Under this definition, an Invention may be made by employees of Raptor or of its Affiliates solely or jointly with a Third Party (a “Raptor Invention”), by employees of the Roche Group solely or jointly with a Third Party (a “Roche Invention”), or jointly by employees of Raptor or its Affiliates and by employees of a member of the Roche Group with or without a Third Party (a “Joint Invention”). Notwithstanding the above, all intellectual property related solely to Transport Candidates shall be deemed to be a Raptor Invention, and all intellectual property related solely to Products (excluding Transport Candidates included therein) shall be deemed to be a Roche Invention.
1.25 Joint Know-How
The term “Joint Know-How” shall mean Know-How that is made jointly by the Parties or their Affiliates or their Sublicensees in the course of performing an activity pursuant to this Agreement.
1.26 Joint Patent Rights
The term “Joint Patent Rights” shall mean Patent Rights claiming a Joint Invention.

 

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1.27 Joint Steering Committee
The term “Joint Steering Committee” or “JSC” shall mean the steering committee, otherwise referred to as the JSC, further described in Article 5.
1.28 Know-How
The term “Know-How” shall mean data, knowledge and information, including materials, samples, chemical manufacturing data, toxicological data, pharmacological data, preclinical data, assays, platforms, formulations, specifications, quality control testing data, whether or not patentable.
1.29 Law
The term “Law” shall mean any law, statute, rule, regulation, ordinance or other pronouncement having the effect of law of any federal, national, multinational, state, provincial, county, city or other political subdivision, domestic or foreign.
1.30 Major Market Countries
The term “Major Market Countries” shall mean the U.S., Germany, France, United Kingdom, Italy, Spain and Japan.
1.31 NDA
The term “NDA” shall mean a new drug application or a Biologics License Application, including all necessary documents, data, and other information concerning a Product, required for Regulatory Approval of the Product as a pharmaceutical product by the FDA or an equivalent application to the equivalent agency in any other country or group of countries (e.g. the marketing authorization application (MAA) in the EU).
1.32 Net Sales
The term “Net Sales” shall mean the amount calculated by subtracting from the amount of Adjusted Gross Sales (as defined below) a lump sum deduction of [*****] of Adjusted Gross Sales [*****].
For purposes of this definition of “Net Sales”, “Adjusted Gross Sales” shall mean [*****] less deductions (which deductions shall in all cases be as consistently applied by Roche to its products) for [*****].
In case of doubt, the “Adjusted Gross Sales” [*****].
1.33 Non-cancellable Obligation
The term “Non-cancellable Obligation” shall mean a Third Party Cost which, in either case, cannot be canceled without penalty upon termination of this Agreement.
1.34 Party
The term “Party” shall mean Raptor or Roche, as the case may be, and “Parties” shall mean Raptor and Roche collectively.
1.35 Patent Rights
The term “Patent Rights” shall mean all rights under any patent or patent application, in any country of the Territory, including any patents issuing on such patent application, and further including any substitution, extension or supplementary protection certificate, reissue, reexamination, renewal, division, continuation or continuation-in-part of any of the foregoing.

 

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1.36 Person
The term “Person” shall mean any corporation, limited or general partnership, limited liability company, joint venture, trust, unincorporated association, governmental body, authority, bureau or agency, any other entity or body, or an individual.
1.37 Phase I Study
The term “Phase I Study” shall mean a human clinical trial in any country that would satisfy the requirements of 21 C.F.R. § 312.21(a), as amended from time to time.
1.38 Phase II Study
The term “Phase II Study” shall mean a human clinical trial, for which the primary endpoints include a determination of dose ranges and/or a preliminary determination of efficacy in patients being studied as described in 21 C.F.R. § 312.21(b).
1.39 Phase III Study
The term “Phase III Study” shall mean a human clinical trial that is prospectively designed to demonstrate statistically whether a product is safe and effective for use in humans in a manner sufficient to obtain regulatory approval to market such product in patients having the disease or condition being studied as described in 21 C.F.R. § 312.21(c).
1.40 Product
The term “Product” shall mean any product comprising (1) a Transport Candidate, and (2) a pharmaceutically active ingredient or an ingredient for the diagnosis or prognosis of an Indication.
1.41 Regulatory Approval
The term “Regulatory Approval” shall mean any approvals (including, if applicable, pricing and reimbursement approvals), licenses, registrations or authorizations by Regulatory Authority, necessary for the marketing and sale of a Product in the Field in a regulatory jurisdiction in the Territory.
1.42 Raptor Base Patent Rights
The term “Raptor Base Patent Rights” shall mean any and all Raptor Patent Rights which are Controlled by Raptor as of the Effective Date, said Patent Rights being listed in Appendix 1.42 of this Agreement.
1.43 Raptor Know-How
The term “Raptor Know-How” shall mean all Know-How that Raptor Controls as of the Effective Date and during the Agreement Term that is necessary or useful for the discovery, manufacture, development or commercialization of Products in the Field.
1.44 Raptor Patent Rights
The term “Raptor Patent Rights” shall mean the Patent Rights that Raptor Controls as of the Effective Date and during the Agreement Term, necessary or useful for the discovery, manufacture, development or commercialization of or Covering a Product in the Field. The term Raptor Patent Rights shall include Raptor Base Patent Rights.

 

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1.45 Research Plan
The term “Research Plan” shall mean the plan of research outlining the work expected to be performed by each Party, a copy of which initial plan is attached as Appendix 1.45, as such plan is approved by the JSC and as it may be updated from time to time as provided in this Agreement.
1.46 Research Program
The term “Research Program” shall mean the activities undertaken by the Parties pursuant to the Research Plan to identify Products, and such other activities with regard to Transport Candidates and Products as the Parties may agree in writing.
1.47 Research Term
The term “Research Term” shall mean the period of time in which the Research Program shall be conducted, commencing on the Effective Date and continuing until the completion of Development Phase 1 or Development Phase 2, unless earlier terminated pursuant to Section 14.2.
1.48 Roche Group
The term “Roche Group” shall mean collectively Roche, its Affiliates and its Sublicensees.
1.49 Roche Patent Rights
The term “Roche Patent Rights” shall mean all Patent Rights Controlled by Roche Covering a Product or that otherwise claim Roche Inventions.
1.50 Royalty Term
The term “Royalty Term” shall mean, with respect to a Product and for a given country, the period of time commencing on the date of First Commercial Sale of the Product in such country and ending on the later of the date that is [*****].
1.51 Sublicensee
The term “Sublicensee” means an entity to which Roche or its Affiliates have licensed rights pursuant to this Agreement.
1.52 Territory
The term “Territory” shall mean all countries of the world.
1.53 Third Party
The term “Third Party” shall mean a Person other than (i) Raptor or any of its Affiliates or (ii) a member of the Roche Group.
1.54 Transport Candidate
The term “Transport Candidate” shall mean any receptor associated protein or any derivative or fragment thereof Covered by Raptor Base Patent Rights or invented or discovered in the conduct of the research collaboration, and any protein that has seventy-five percent (75%) or greater homology thereto.
1.55 US
The term “US” shall mean the United States of America and its territories and possessions.

 

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1.56 Valid Claim
The term “Valid Claim” shall mean, as applicable, a claim in any (i) unexpired and issued Raptor Patent Rights or Joint Patent Rights that have not been disclaimed, revoked or held invalid by a final nonappealable decision of a court of competent jurisdiction or government agency or (ii) pending patent application of any Raptor Patent Rights or Joint Patent Rights in any country of the Territory that has been on file with the applicable patent office for [*****] from the earliest date to which the patent application claims its earliest priority.
1.57 Additional Definitions
Each of the following definitions is set forth in the Section of this Agreement indicated below:
         
Definition   Section
“Accounting Period”
    7.1  
“Adjusted Gross Sales”
    1.32  
“Alliance Director”
    5.6  
“Bankruptcy Code”
    15  
[*****]
       
“Breaching Party”
    14.2.1  
“Chairperson”
    5.2  
“Chugai”
    1.1  
“Development Phase 1”
    3.1.1  
“Development Phase 2”
    3.1.1  
“Disclosing Party”
    1.10  
“Genentech”
    1.1  
[*****]
       
“Indemnified Party”
    12.3  
“Indemnifying Party”
    12.3  
“Joint Invention”
    1.24  
“Material(s)”
    13.7  
“Member(s)”
    5.2  
“Non-Breaching Party”
    14.2.1  
“Notice of Comments”
    13.4 (b)
“Payment Breach”
    14.2.1  
“Peremptory Notice Period”
    14.2.1  
“Publishing Party”
    13.4 (b)
“Raptor”
  Preamble
“Raptor Invention”
    1.24  
“Receiving Party”
    1.10  
“Roche Basel”
  Preamble
“Roche Entity”
    1.1  
“Roche Invention”
    1.24  
“Roche Nutley”
  Preamble
“SEC”
    11.4  
“Subject Disclosure”
    13.3.1  

 

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2. Grant of License
2.1 Licenses
2.1.1 Commercial Licenses
Subject to the terms and conditions of this Agreement, Raptor grants to Roche and its Affiliates an exclusive (even as to Raptor except as provided below), non-transferable (except as permitted under Section 16.4) right and license under Raptor’s interest in the Raptor Patent Rights and Raptor Know-How and Raptor’s interest in the Joint Patent Rights and Joint Know-How to use, make, have made, import, offer to sell, sell and have sold Transport Candidates and Products in the Field in the Territory.
2.1.2 Research Cross Licenses
Each Party grants to the other Party and its Affiliates during the Research Term a non-exclusive right and license under Know-How and Patent Rights Controlled by such Party and that are necessary or useful for the discovery, manufacture or development of Products solely to enable the other Party and its Affiliates to perform the activities contemplated under the Research Plan under this Agreement.
Notwithstanding the exclusive license grant to Roche and its Affiliates in Section 2.1.1, Raptor reserves the rights under the Raptor Patent Rights, Raptor Know-How, Joint Patent Rights and Joint Know-How to use, make, have made, and transfer Transport Candidates and Products in the Field in the Territory solely for performing the Research Project.
2.2 Sublicense
The rights and licenses granted to Roche and its Affiliates shall include the right of Roche to grant written sublicenses to Affiliates and Third Parties under such rights and licenses, in whole or in part, solely to the extent necessary to make, use, offer for sale, sell or import Transport Candidates or Products in the Territory for use in the Field, provided that Roche will be responsible for the sublicensees’ compliance to the applicable terms and conditions of this Agreement.
2.3 Exclusivity
During the Exclusivity Period, Raptor shall work exclusively with Roche on the use of Transport Candidates to cross the blood brain barrier. The Exclusivity Period can be extended if Roche makes the payments set forth in Section 6.1.1.
2.4 No Other Rights
Except for the rights expressly granted under this Article 2, no license, right, title or interest of any nature whatsoever is granted hereunder by implication, estoppel, reliance or otherwise, by Raptor to Roche or any of Roche’s Affiliates, and any of Raptor’s rights to Raptor Patent Rights, Raptor Know-How, Joint Patent Rights and Joint Know-How not specifically licensed to Roche under this Article 2 shall be retained by Raptor.
3. Research Collaboration

 

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3.1 Conduct of the Research Program
3.1.1 Scope
Initially, Roche and Raptor shall conduct a mutually agreed-upon Research Program pursuant to the Research Plan attached as Appendix 1.45 with the objective of performing the initial feasibility study with full RAP protein and existing fragments (“Development Phase 1”). Upon successful completion of the Development Phase 1, Roche will have the option to retain Raptor to perform an additional Research Plan to expand the NeuroTransTM platform to develop neurobiologicals for delivery to the brain (“Development Phase 2”) subject to mutual agreement on the scope of Development Phase 2 and payment of prospective fees based on FTEs. The activities conducted in connection with the Research Program will be overseen by the JSC.
3.1.2 Commercially Reasonable Efforts
Roche and Raptor shall each use Commercially Reasonable Efforts to perform their respective tasks and obligations in conducting the activities assigned to it in the then-current Research Plan.
3.1.3 Research Plan
The Parties will conduct the Research Program in accordance with the Research Plan. The Research Plan will set forth (i) the scope of the Research Program and the resources that are expected to be dedicated to the activities contemplated within the scope of the Research Program, including the responsibilities of each Party and (ii) specific objectives for each year, which objectives will be updated or amended, as appropriate, by the JSC as research progresses. The initial Research Plan for activities is attached hereto as Appendix 1.45. The JSC shall review the Research Plan on an ongoing basis and may amend the Research Plan, subject to Section 5.7. Any such changes shall be reflected in written amendments to the Research Plan.
3.1.4 Duration
The Research Program shall commence on the Effective Date and shall continue until the end of the Research Term.
4. Diligence and Third Party Obligations
4.1 Diligence
Roche and Raptor shall each use Commercially Reasonable Efforts to perform their other respective activities contemplated by this Agreement or as may be agreed upon in any subsequent written agreements with respect to the subject matter hereof. [*****]
[*****]
5. Governance
5.1 Joint Steering Committee
Within sixty (60) days after the Effective Date of this Agreement, the Parties shall establish a Joint Steering Committee to oversee the Research Program activities under this Agreement.

 

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5.2 Members
The JSC shall be composed of four (4) persons (each a “Member”). Roche and Raptor each shall be entitled to appoint two (2) Members with appropriate seniority and expertise. Each Party may replace any of its Members and appoint a person to fill the vacancy arising from each such replacement. A Party that replaces a Member shall notify the other Party at least ten (10) days prior to the next scheduled meeting of the JSC. Both Parties shall use Commercially Reasonable Efforts to keep an appropriate level of continuity in representation. Both Parties may invite a reasonable number of additional experts and/or advisors who are subject to confidentiality obligations at least as restrictive as those set forth in Article 13 to attend part or the whole JSC meeting with prior notification to the JSC. Members may be represented at any meeting by another person designated by the absent Member. The JSC shall be chaired by a Roche Member (“Chairperson”).
5.3 Meetings
The Chairperson or his/her delegate is responsible for sending invitations and agendas for all JSC meetings to all Members at least ten (10) days before the next scheduled meeting of the JSC. The venue for the meetings shall be agreed by the JSC. The JSC shall hold meetings at least twice per Calendar Year, either in person or by tele-/video-conference, and in any case as frequently as the Members of the JSC may agree shall be necessary, but not more than four (4) times a year. The Alliance Director of each Party shall attend the JSC meetings as permanent participant.
5.4 Minutes
The Chairperson is responsible for designating a Member to record in reasonable detail and circulate draft minutes of JSC meetings to all members of the JSC for comment and review within twenty (20) days after the relevant meeting. The Members of the JSC shall have thirty (30) days to provide comments. The Party preparing the minutes shall incorporate timely received comments and distribute finalized minutes to all Members of the JSC within sixty (60) days of the relevant meeting. Both Parties shall approve the final version of the minutes before its distribution.
5.5 Decisions
5.5.1 Decision Making Authority
Subject to Section 5.7, the JSC shall decide matters related to the Research Program, as well as approve and amend the Research Plan.
5.5.2 Consensus; Good Faith
The Members of the JSC shall act in good faith to cooperate with one another and seek agreement with respect to issues to be decided by the JSC. The Parties shall endeavor to make decisions by consensus.
5.5.3 Failure to Reach Consensus
If the JSC is unable to decide a matter by consensus, then Roche shall have the final decision authority on any matter.
5.6 Alliance Director
Each Party shall appoint an Alliance Director (“Alliance Director”). The Alliance Directors shall be the point of contact within each Party with responsibility for facilitating communication and collaboration between the Parties. They are permanent participants of the JSC meetings (but not members of the JSC) and may attend JSC meetings as appropriate. The Alliance Directors shall seek to facilitate resolution of potential and pending issues and potential disputes to enable the JSC to reach consensus and avert escalation of such issues or potential disputes.

 

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5.7 Limitations of Authority
Neither the JSC, nor Roche unilaterally, shall have the authority to amend or waive any terms of this Agreement or impose additional obligations on Raptor without Raptor’s prior written consent.
5.8 Expenses
Each Party shall be responsible for its own expenses including travel and accommodation costs incurred in connection with the JSC.
5.9 Lifetime
The JSC shall exist during the Research Term and for six (6) months thereafter.
6. Payment
6.1 Signing Fee
Within thirty (30) days after the Effective Date and receipt of an invoice from Raptor, Roche shall pay to Raptor a fee of [*****].
6.1.1 Extension of Exclusivity Period
Roche can extend the Exclusivity Period for an additional twelve (12) months by notifying Raptor in writing on or before the end of the Exclusivity Period. Roche shall also pay Raptor [*****] within thirty (30) days after such notice has been provided, and Roche has received an invoice for such amount from Raptor.
6.2 Events
Roche shall pay to Raptor up to a total of [*****] in relation to the achievement of events with respect to each Product. The event payments under this Section 6.2 shall be paid by Roche according to the following schedule of development events:
     
[*****]
  [*****]
Each event payment for a given Product shall be paid only once for a given Product, the first time a Product reaches such event, regardless of the number of times such events are reached for the given Product.
Upon achievement by Roche, its Affiliates and/or Sublicensees of any of the foregoing events, Roche shall promptly (and in no event more than thirty (30) days following achievement thereof) notify Raptor and event payments shall be paid by Roche to Raptor within thirty (30) days after occurrence of the applicable event and receipt of an invoice from Raptor.

 

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6.3 Royalty Payments
6.3.1 Royalty Term
Royalties shall be payable by Roche on Net Sales of Products on a Product-by Product and country-by-country basis until the expiry of the Royalty Term for a Product in a given country. Thereafter, the licenses shall be fully paid up and royalty-free with respect to such Product in such country, on a Product-by-Product and country-by-country basis.
Subject to the remainder of this Section 6.3, the following royalty rates shall apply to the respective tiers of worldwide, aggregate Calendar Year Net Sales of a Product in the Territory, on an incremental basis, as follows:
[*****]
For the purpose of calculating royalties payable with respect to a Product, royalties on Calendar Year Net Sales shall be subject to [*****]
7. Accounting and reporting
7.1 Timing of Payments
Roche shall calculate royalties on Net Sales quarterly as of March 31, June 30, September 30 and December 31 (each being the last day of an “Accounting Period”) and shall pay royalties on Net Sales within sixty (60) days after the end of each Accounting Period in which such Net Sales occur.
7.2 Late Payment
Any payment under this Agreement that is not paid on or before the date such payment is due shall bear interest, to the extent permitted by applicable law [*****] calculated on the number of days such payment is overdue. [*****]
7.3 Method of Payment
Royalties on Net Sales and all other amounts payable by Roche hereunder shall be paid by or on behalf of Roche in U.S. Dollars. All payments due to Raptor hereunder shall be made directly from an account located in either (at Roche’s option) the United States or Switzerland to account(s) designated by Raptor.
7.4 Currency Conversion
Whenever calculating royalties requires conversion from any currency, Roche shall make such conversion as follows: When calculating the Net Sales for countries other than the United States of America, Roche shall convert the amount of such sales in currencies other than Swiss Francs into Swiss Francs using for internal foreign currency translation Roche’s then current standard practices actually used on a consistent basis in preparing its audited financial statements.
Upon converting the amount of Adjusted Gross Sales into Swiss Francs, Roche shall convert into US Dollars (or other currency) [*****].

 

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7.5 Reporting
With each payment Roche shall provide Raptor in writing for the relevant Calendar Quarter on a Product-by-Product basis the following information with respect to each of the following territories: (1) the United States, (2) the Major Market Countries (other than the United States) and Canada, and (3) all territories other than those set forth in the foregoing clause (1) and (2):
a) Net Sales and Adjusted Gross Sales;

b) taxes withheld, if applicable;
; and
d) Total royalty payable to Raptor.
7.6 United States Dollars
All dollar ($) amounts specified in this Agreement are United States dollar amounts.
7.7 Nonrefundable
All payments made by Roche to Raptor under this Agreement shall be non-refundable and non-creditable, except for those set forth in Section 6.3 or as set forth in 6.2 or 9.3.
8. Taxes
Raptor shall pay all sales, turnover, income, revenue, value added, and other taxes levied on account of any payments accruing or made to Raptor under this Agreement. If provision is made in law or regulation of any country for withholding of taxes of any type, levies or other charges with respect to any royalty payable under this Agreement to Raptor, then Roche shall promptly pay such tax, levy or charge for and on behalf of Raptor to the proper governmental authority, and shall promptly furnish Raptor with receipt of payment. Roche shall be entitled to deduct any such tax, levy or charge actually paid from royalty due Raptor or be promptly reimbursed by Raptor if no further payments are due Raptor. Each Party agrees to reasonably assist the other Party in claiming exemption from such deductions or withholdings under double taxation or similar agreement or treaty from time to time in force and in minimizing the amount required to be so withheld or deducted.
9. Auditing
9.1 Raptor Right to Audit
9.1.1 Roche shall keep, and shall require its and its Affiliates and Sub-licensees to keep, for three (3) years, full, true and accurate books of account containing all particulars that may be necessary for the purpose of calculating all royalties and all other amounts payable under this Agreement. Such books of accounts shall be kept at Roche’s or the relevant Affiliate’s or Sublicensee’s principal place of business. At the expense of Raptor, Raptor has the right to engage an independent public accountant to perform, on behalf of Raptor an audit of such books and records of Roche and its Affiliates and Sub-licensees, that are deemed necessary by Raptor’s independent public accountant to report on Net Sales of Products for the period or periods requested by Raptor and the correctness of any report or payments made under this Agreement.

 

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9.1.2 Upon timely request and at least thirty (30) Business Days prior written notice from Raptor, such audit shall be conducted in the countries specifically requested by Raptor, during regular business hours in such a manner as to not unnecessarily interfere with Roche’s normal business activities, and shall be limited to results in the three (3) Calendar Years prior to audit notification.
9.1.3 Such audit shall not be performed more frequently than once per Calendar Year nor more frequently than once with respect to records covering any specific period of time. The auditors shall not interpret the agreement.
9.1.4 All information, data documents and abstracts herein referred to shall be used only for the purpose of verifying royalty statements, shall be treated as Roche Confidential Information subject to the obligations of this Agreement and need neither be retained more than the longer of one (1) year after completion of an audit hereof, if an audit has been requested; nor more than three (3) years from the end of the Calendar Year to which each shall pertain; nor more than one (1) year after the date of termination of this Agreement.
9.2 Sharing of reports
The final audit report shall be shared by Roche and Raptor.
9.3 Over- or Underpayment
If the audit reveals an underpayment, Roche shall reimburse Raptor for the amount of the underpayment within thirty (30) days with interest as set forth in Section 7.2. If the audit reveals an overpayment, Roche shall have the right to credit the amount of such overpayment against the next royalty payment payable to Raptor hereunder. [*****] Section 16.2 shall apply to this Section 9.3.
9.4 Duration of Audit Rights
The failure of Raptor to request verification of any royalty calculation within the period during which corresponding records must be maintained under this Section 10 will be deemed to be acceptance of the royalty payments and reports.
10. Intellectual Property
10.1 Ownership of Inventions
Raptor shall own all Raptor Inventions, Roche shall own all Roche Inventions, and Raptor and Roche shall jointly own all Joint Inventions. Each Party shall require all of its employees, and shall cause its Affiliates performing under this Agreement to require all of such Affiliates’ employees, to assign all Inventions made by them to such Party or such Party’s Affiliates, as the case may be.
The determination of inventorship for Inventions shall be in accordance with US inventorship laws.
Except as specifically set forth herein, this Agreement shall not be construed as (i) granting any license or right under any intellectual property rights; or (ii) representing any commitment by either party to enter into any additional agreement, by implication or otherwise.

 

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10.2 Prosecution of Patent Rights Claiming Raptor Inventions
Raptor shall, at its expense discretion, (i) Handle all Raptor Patent Rights, (ii) consult with Roche as to the Handling of such Raptor Patent Rights, (iii) apprise Roche of significant events (such as issuance, reissuance, reexamination, interference, revocation or opposition proceedings) encountered in such Handling, and (iv) provide to Roche an annual report identifying all Raptor Patent Rights and their status. At Raptor’s reasonable request, Roche shall cooperate with the Handling of all Raptor Patent Rights. Raptor will not abandon any Raptor Patent Rights without the prior consent of Roche. Roche acknowledges that if Raptor (based on consultation with Roche) elects not to Handle, or (with prior consent of Roche) elects to abandon, any Raptor Patent Rights that Cover a Product to which Roche retains a license under Section 2.1, BioMarin has the right to take over the prosecution of such Raptor Patent Rights pursuant to the Asset Purchase Agreement.
10.3 Prosecution of Patent Rights Claiming Roche Inventions
Roche shall, at its own expense and discretion, (i) Handle all Roche Patent Rights that claim Roche Inventions, (ii) consult with Raptor as to the Handling of such Roche Patent Rights, (iii) apprise Raptor of significant events (such as issuance, reissuance, reexamination, interference, revocation or opposition proceedings) encountered in such Handling, and (iv) provide to Raptor an annual report identifying all Roche Patent Rights and their status. At Roche’s expense and reasonable request, Raptor shall cooperate with the Handling of all such Roche Patent Rights.
10.4 Prosecution of Patent Rights Claiming Joint Inventions
10.4.1 Roche shall (i) Handle all Patent Rights that claim Joint Inventions, (ii) consult with Raptor as to the Handling of such Patent Rights, and (iii) furnish to Raptor copies of all documents relevant to any such Handling. Roche shall furnish such documents and consult with Raptor in sufficient time before any action by Roche is due to allow Raptor to provide comments thereon, which comments Roche must consider. Raptor shall reasonably cooperate, with the Handling of all such Patent Rights. If Roche wishes to abandon any of such Patent Rights, then sufficiently in advance to permit Raptor to undertake Handling of such Patent Rights without a loss of rights, Roche shall offer to assign its interest in such Patent Rights to Raptor and, upon Raptor’s request, shall promptly assign its interest in such Patent Rights to Raptor.
10.4.2 The Parties shall equally share all reasonable and documented external costs incurred in Handling Patent Rights claiming Joint Inventions. Each Party shall have the right, exercisable by written notice, to terminate its obligation to share such costs. If Roche exercises such right, then from and after the date thereof Roche shall assign its interest in such Patent Right to Raptor, which Patent Right shall be deemed to be a Raptor Patent Right for purposes of this Agreement. If Raptor exercises such right, then from and after the date thereof Raptor shall assign its interest in such Patent Right to Roche, which Patent Right shall be deemed to be a Roche Patent Right for purposes of this Agreement. If neither Party Handles any such Patent Right, then the underlying Invention shall be deemed Joint Know-How.
11. Representations and Warranties
Except as expressly set forth in a letter dated as of even date herewith from Raptor to Roche, Raptor represents and warrants to Roche as of the Effective Date as follows:

 

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11.1 Ownership of Patent Rights
Raptor is the exclusive owner of all right, title and interest in, or is the exclusive licensee of, the Raptor Base Patent Rights. Appendix 1.42 contains a complete and accurate list of all patents and patent applications included in the Raptor Base Patent Rights as of the Effective Date. The Raptor Base Patent Rights are free and clear of all liens, claims, security interests and other encumbrances of any kind or nature [*****]. Raptor has not granted any licenses to the Raptor Patent Rights to any Third Party, nor has Raptor effectuated any prior transfer, sale or assignment of any part of the Raptor Patent Rights to a Third Party, that conflicts in any material respect with any of the terms of this Agreement or the licenses granted to Roche hereunder.
11.2 Inventors
All of Raptor’s employees, officers and consultants performing work on behalf of Raptor under this Agreement have executed agreements with Raptor requiring assignment to Raptor of all Inventions made by such individuals during the course of their employment or consulting relationship with Raptor.
11.3 Grants
Raptor has the right to grant Roche and its Affiliates the rights and licenses described in this Agreement.
11.4 Authorization
The execution, delivery and performance of this Agreement by Raptor: (i) are within the corporate power of Raptor; (ii) have been duly authorized by all necessary corporate action; (iii) are not in contravention of any provision of the certificate of incorporation of Raptor; (iv) to the knowledge of Raptor, will not violate any Law or regulation or any order or decree of any court of governmental instrumentality; (v) will not violate the terms of any indenture, mortgage, deed of trust, lease, agreement, or other instrument to which Raptor is a party or by which Raptor or any of its property is bound, which violation would have a material adverse effect on the financial condition of Raptor or on the ability of Raptor to perform its obligations hereunder; and (vi) do not require any filing or registration with, or the consent or approval of, any governmental body, agency, authority or any other Person, which has not been made or obtained previously, except for any filing required to comply with the laws and regulations of the Securities and Exchange Commission (“SEC”) as permitted under Section 13.3.3.
11.5 Validity of Patent Rights
To the best of Raptor’s knowledge, the claims in any issued patent included in the Raptor Base Patent Rights licensed to Roche pursuant to this Agreement are valid and enforceable. To Raptor’s knowledge, there is no dispute concerning inventorship of any Raptor Base Patent Rights.
11.6 Ownership and Validity of Know-How
To the best of Raptor’s knowledge, the Raptor Know-How has not been misappropriated from any Third Party. Raptor has taken reasonable measures to protect the confidentiality of the Raptor Know-How.
11.7 No Claims
There are no claims or investigations pending or, to the best of Raptor’s knowledge, threatened against Raptor or any of its Affiliates, at Law or in equity, or before or by any governmental authority relating to the subject matter of this Agreement or that would materially adversely affect Raptor’s ability to perform its obligations hereunder.

 

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11.8 No Conflict
Raptor is not under any obligation to any Person, contractual or otherwise, that conflicts in any material respect with the terms of this Agreement.
11.9 Roche Representations and Warranties
Roche represents and warrants to Raptor as of the Effective Date as follows:
(a) The execution, delivery and performance of this Agreement by Roche: (i) are within the corporate power of Roche; (ii) have been duly authorized by all necessary corporate action; (iii) are not in contravention of any provision of the certificate of incorporation of Roche; (iv) to the knowledge of Roche, will not violate any Law or regulation or any order or decree of any court of governmental instrumentality; (v) will not violate the terms of any indenture, mortgage, deed of trust, lease, agreement, or other instrument to which Roche is a party or by which Roche or any of its property is bound, which violation would have a material adverse effect on the financial condition of Roche or on the ability of Roche to perform its obligations hereunder; and (vi) do not require any filing or registration with, or the consent or approval of, any governmental body, agency, authority or any other Person, which has not been made or obtained previously.
(b) There are no claims or investigations pending or, to Roche’s knowledge, threatened against Roche or any of its Affiliates, at Law or in equity, or before or by any governmental authority relating to the subject matter of this Agreement or that would materially adversely affect Roche’s ability to perform its obligations hereunder.
(c) Roche is not under any obligation to any Person, contractual or otherwise, that conflicts in any material respect with the terms of this Agreement.
11.10 No Other Representations; Disclaimer
EXCEPT AS OTHERWISE EXPRESSLY SET FORTH IN THIS AGREEMENT, NEITHER PARTY MAKES ANY REPRESENTATIONS OR EXTENDS ANY WARRANTIES OF ANY KIND, EXPRESS OR IMPLIED, INCLUDING, WITHOUT LIMITATION, ANY WARRANTIES OF MERCHANTABILITY, FITNESS FOR A PARTICULAR PURPOSE OR NON-INFRINGEMENT OF RIGHTS OF THIRD PARTIES, WITH RESPECT TO ANY PRODUCT, TRANSPORT CANDIDATE, KNOW-HOW, MATERIALS OR PATENT RIGHTS.
12. Indemnification
12.1 Indemnification by Roche
Roche shall indemnify, hold harmless and defend Raptor, its Affiliates and their respective directors, officers, employees and agents from and against any and all losses, expenses, cost of defense (including without limitation reasonable attorneys’ fees, witness fees, damages, judgments, fines and amounts paid in settlement) and any amounts Raptor becomes legally obligated to pay because of any claim or claims against it to the extent that such claim or claims arise out of (a) activities related to Products (including, without limitation, product liability claims) conducted by or on behalf of Roche, its Affiliates or Sublicensees, (b) the breach of any of Roche’s representations or warranties hereunder, or (c) any infringement of any Third Party Patent Rights or misappropriation of any Third Party Know-How in connection with the manufacture, use, offer for sale, sale, import or export of any Products, in all cases except to the extent such losses, expenses, costs and amounts are due to the gross negligence or willful misconduct of Raptor or breach of any of Raptor’s representations and warranties hereunder.

 

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12.2 Indemnification by Raptor
Raptor shall indemnify, hold harmless and defend Roche, its Affiliates and their respective directors, officers, employees and agents from and against any and all losses, expenses, cost of defense (including without limitation reasonable attorneys’ fees, witness fees, damages, judgments, fines and amounts paid in settlement) and any amounts Roche becomes legally obligated to pay because of any claim or claims against it to the extent that such claim or claims arise out of the breach of any of Raptor’s representations or warranties hereunder, in all cases except to the extent such losses, expenses, costs and amounts are due to the gross negligence or willful misconduct of Roche or breach of any of Roche’s representations and warranties hereunder.
12.3 Procedure
In the event of a claim by a Third Party against any indemnitee entitled to indemnification under this Agreement (in such capacity, the “Indemnified Party”), the Indemnified Party shall promptly notify the other Party (in such capacity, the “Indemnifying Party”) in writing of the claim (it being understood that the failure by the Indemnified Party to give prompt notice of a Third Party claim as provided in this Section 12.3 shall not relieve the Indemnifying Party of its indemnification obligation under this Agreement except and only to the extent that such Indemnifying Party is actually prejudiced as a result of such failure to give prompt notice). Within thirty (30) days after delivery of such notification, the Indemnifying Party may, upon written notice thereof to the Indemnified Party, undertake and solely manage and control, at its sole expense and with counsel reasonably satisfactory to the Indemnified Party, the defense of the claim. If the Indemnifying Party does not undertake such defense, the Indemnified Party shall control such defense. The Party not controlling such defense shall cooperate with the other Party and may, at its option and expense, participate in such defense; provided that, if the Indemnifying Party assumes control of such defense and the Indemnified Party in good faith concludes, based on advice from counsel, that the Indemnifying Party and the Indemnified Party have conflicting interests with respect to such action, suit, proceeding or claim, the Indemnifying Party shall be responsible for the reasonable fees and expenses of counsel to the Indemnified Party solely in connection therewith. The Party controlling such defense shall keep the other Party advised of the status of such action, suit, proceeding or claim and the defense thereof and shall consider recommendations made by the other Party with respect thereto. The Indemnifying Party shall not be liable for any litigation costs or expenses incurred by the Indemnified Party without the Indemnifying Party’s written consent. The Indemnified Party shall not settle any such action, suit, proceeding or claim without the prior written consent of the Indemnifying Party, which shall not be unreasonably withheld, delayed or conditioned. The Indemnifying Party shall not settle any such action, suit, proceeding or claim, or consent to any judgment in respect thereof, that does not include a complete and unconditional release of the Indemnified Party from all liability with respect thereto, that imposes any liability or obligation on the Indemnified Party or that acknowledges fault by the Indemnified Party without the prior written consent of the Indemnified Party.
12.4 No Consequential Damages
IN NO EVENT SHALL EITHER RAPTOR OR ROCHE BE LIABLE FOR SPECIAL, INDIRECT, INCIDENTAL, EXEMPLARY, MULTIPLE OR CONSEQUENTIAL DAMAGES ARISING OUT OF THIS AGREEMENT BASED ON CONTRACT, TORT OR ANY OTHER LEGAL THEORY. NOTHING IN THIS SECTION 12.4 IS INTENDED TO LIMIT OR RESTRICT (A) THE INDEMNIFICATION RIGHTS OR OBLIGATIONS OF EITHER PARTY UNDER ARTICLE 12 OR (B) REMEDIES AVAILABLE TO EITHER PARTY WITH RESPECT TO A BREACH OF ARTICLE 13.

 

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13. Obligation Not to Disclose Confidential Information
13.1 Non-Use and Non-Disclosure
During the Agreement Term and for five (5) years thereafter, the Receiving Party shall (i) treat Confidential Information provided by the Disclosing Party as it would treat its own information of a similar nature (but with no less than reasonable care), (ii) take reasonable precautions not to disclose such Confidential Information to Third Parties, without the Disclosing Party’s prior written consent, (iii) not use such Confidential Information other than for fulfilling its obligations under this Agreement and (iv) shall disclose such Confidentiality Information only to the Receiving Party’s employees, consultants, advisors and contractors who have a need-to-know such Confidential Information to perform their obligations in connection herewith, solely under conditions of confidentiality and non-use that are at least as restrictive as those set forth in this Article 13.
13.2 Permitted Disclosure
Notwithstanding the obligation of non-use and non-disclosure set forth in Section 13.1, the Parties recognize the need for certain exceptions to this obligation, specifically set forth below, with respect to press releases, patent rights, publications, and certain commercial considerations.
13.3 Press Releases; Disclosure of Terms
13.3.1 During the Agreement Term, the content of any press release or public disclosure relating to this Agreement or the Parties’ activities hereunder (each such press release or public disclosure, a “Subject Disclosure”) shall be mutually agreed by the Parties, which agreement shall not be unreasonably withheld, delayed or conditioned, except that a Party may, without the other Party’s agreement, issue or make such Subject Disclosure (a) if the contents of such Subject Disclosure have previously been made public other than through a breach of this Agreement by the issuing Party, or (b) if it is advised by counsel that such Subject Disclosure is required by applicable Law, including without limitation the rules or regulations of the SEC or similar regulatory agency in a country other than the United States or of any stock exchange or other securities trading institution.
13.3.2 In light of the foregoing, each Party shall provide the other Party with a draft Subject Disclosure that requires such other Party’s approval hereunder at least five (5) Business Days prior to its intended issuance or disclosure for such other Party’s review. During such period, the reviewing Party shall either (i) approve the draft Subject Disclosure and permit the submitting Party to issue such Subject Disclosure, (ii) contact the submitting Party to discuss modification to the draft Subject Disclosure, or (iii) contact the submitting Party and disapprove the Subject Disclosure, in each case subject to the reviewing Party’s obligation not to unreasonably withhold, condition or delay any approval which is sought by the submitting Party with respect to such draft Subject Disclosure. If the reviewing Party asks for modification, then the submitting Party shall either make such modification or work with the reviewing Party to arrive at a Subject Disclosure that is agreeable to both Parties.

 

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13.3.3 Notwithstanding anything in this Agreement to the contrary, each Party shall have the right to disclose the terms of this Agreement [*****] and (b) as required by applicable Law, including without limitation the rules or regulations of the SEC or similar regulatory agency in a country other than the United States or of any stock exchange or other securities trading institution. In the event that this Agreement shall be included in any report, statement or other document filed by either Party or an Affiliate of either Party with the SEC or similar regulatory agency in a country other than the United States or any stock exchange or other securities trading institution, such Party shall use, or shall cause such Party’s Affiliate, as the case may be, to use, reasonable efforts to obtain confidential treatment from the SEC, similar regulatory agency, stock exchange or other securities trading institution of any financial information or other information of a competitive or confidential nature, and shall include in such confidentiality request such provisions of this Agreement as may be reasonably requested by the other Party.
13.4 Publications
During the Agreement Term, the following restrictions shall apply with respect to disclosure by any Party of Confidential Information relating to the Product (or any component thereof) in any publication or presentation:
  a)  
Both Parties acknowledge that it is their policy for the studies and results thereof to be registered and published in accordance with their internal guidelines. Roche, in accordance with its internal policies and procedures, shall have the right to publish all studies and clinical trials conducted under this Agreement, and results thereof, on the clinical trial registries which are maintained by or on behalf of Roche.
  b)  
A Party (“Publishing Party”) shall provide the other Party with a copy of any proposed publication or presentation at least sixty (60) days (or at least thirty (30) days in the case of oral presentations) prior to submission for publication or presentation so as to provide such other party with an opportunity to recommend any changes it reasonably believes are necessary to continue to maintain the Confidential Information of the non-Publishing Party in accordance with the requirements of this Agreement. If such non-Publishing Party notifies (“Notice of Comments”) the Publishing Party in writing, within sixty (60) days after receipt of the copy of the proposed publication or presentation (or at least thirty (30) days in the case of oral presentations) that such publication or presentation in its reasonable judgment (i) contains an Invention, conceived and reduced to practice either solely or jointly by the non-Publishing Party, for which such Party desires to obtain patent protection or (ii) contains Confidential Information of the Non-Publishing Party or could otherwise be reasonably expected to have a material adverse effect on the commercial value of any Confidential Information disclosed by the non-Publishing Party to the Publishing Party, the Publishing Party shall delete such Confidential Information or other relevant disclosure and prevent such publication or delay such publication for a mutually agreeable period of time. In the case of Inventions, the proposed publication or presentation shall be delayed for a period reasonably sufficient to permit the timely preparation and filing of a patent application(s) on such Invention, and in no event less than an additional ninety (90) days from the date of the Notice of Comments.

 

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13.5 Commercial Considerations
Nothing in this Agreement shall prevent Roche or its Affiliates from disclosing Confidential Information of Raptor to (i) governmental agencies to the extent required or desirable to secure government approval for the development, manufacture or sale of Product in the Territory, (ii) Third Parties acting on behalf of Roche and bound by obligations of confidentiality and non-use which are at least as restrictive as those set forth in this Article 13, solely to the extent reasonably necessary for the development, manufacture or sale of Product in the Territory, or (iii) Third Parties bound by obligations of confidentiality and non-use which are at least as restrictive as those set forth in this Article 13, solely to the extent reasonably necessary to market the Product in the Territory. The Receiving Party may disclose Confidential Information of the Disclosing Party to the extent that such Confidential Information is required to be disclosed by the Receiving Party (x) to comply with applicable Laws, or (y) to the extent necessary to exercise the rights granted to or retained by the Receiving Party under this Agreement in connection with the Handling of Patent Rights or to defend or prosecute litigation, provided that the Receiving Party provides prior written notice of such disclosure to the Disclosing Party and, to the extent practicable, takes reasonable and lawful actions to, and reasonably cooperates with the Disclosing Party in the Disclosing Party’s efforts to, minimize the degree of such disclosure.
13.6 Clinical Trial Registry.
Roche, in accordance with its internal policies and procedures, shall have the right to publish all studies, clinical trials and results thereof regarding Products on the clinical trial registries which are maintained by or on behalf of Roche.
13.7 Material Transfer.
Either Party may provide the other Party with sample molecules and other materials (“Material(s)”) in connection with the Research Plan. The Party supplying the Materials shall provide the other Party with all safety information and handling instructions pertaining to the Material that it is aware of. The party receiving any Materials will use the Materials solely for carrying out the Research Plan and for no other purposes and will not transfer the Materials to any third party. The Party providing the Materials will retain all rights in and title to the Materials. Under no circumstances shall the Party receiving the Material use the Material in humans or chemically or biologically modify the Material, except in accordance with the Research Plan. The Party receiving the Material shall take no action to determine the chemical structure of the Material or to reverse engineer the Material.
14. Term and Termination
14.1 Commencement and Term
The term of this Agreement shall commence upon the Effective Date and, unless this Agreement is terminated sooner as provided in this Article 14, expire on the date when no royalty or other payment obligations under this Agreement are or will become due (“Agreement Term”). Upon expiration of this Agreement, all licenses from Raptor to Roche shall be fully paid-up, perpetual and irrevocable.
14.2 Termination
14.2.1 Termination for Breach
A Party (in such capacity, the “Non-Breaching Party”) shall have the right to terminate this Agreement in its entirety or on a country-by-country or Product-by Product basis in the event the other Party (in such capacity, the “Breaching Party”) is in material breach under this Agreement.
The Non-Breaching Party shall provide written notice to the Breaching Party, which notice shall identify the material breach and the countries in which the Non-Breaching Party intends to have this Agreement terminate. The Breaching Party shall have a period of sixty (60) days (or thirty (30) days with respect to any payment breach (“Payment Breach”)) after such written notice is provided to cure such material breach (“Peremptory Notice Period”).

 

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If any breach set forth above in this Section 14.2.1 is not cured within the Peremptory Notice Period, this Agreement shall effectively terminate in its entirety or with respect to specific country(ies), or Product(s), as applicable, at the end of such Peremptory Notice Period, unless the Breaching Party provides written notice to Non-Breaching Party during such Peremptory Notice Period that the Breaching Party disputes in good faith the existence of such breach, in which event this Agreement shall terminate only upon the issuance by an arbitrator pursuant to Section 16.3 of an award upholding the basis for termination (or once the Breaching Party is no longer disputing such basis in good faith, if earlier). Notwithstanding the foregoing, (a) in the case of a Payment Breach that is so arbitrated but that is upheld in an arbitration award as a basis for termination, this Agreement shall not terminate if both (x) Roche shall have paid all disputed amounts into a Third Party escrow within thirty (30) days after the commencement of the arbitration, and (y) within fifteen (15) days after such arbitration award (or such time as Roche is no longer disputing such amounts, if earlier), all amounts held to be owed to Raptor (or that Roche is no longer disputing) are paid to Raptor, and (b) in the case of a material breach by Raptor, this Agreement shall not terminate unless and until (x) an arbitral tribunal issues a final award pursuant to Section 16.3 upholding such basis for termination (or unless and until Raptor is no longer disputing such basis, if earlier) and (y) within twenty (20) days thereafter Raptor fails to cure the breach(es) on which the arbitral tribunal based such award.
14.2.2 Termination for Insolvency Event
A Party shall have the right to terminate this Agreement upon notice to the other Party, if such other Party incurs an Insolvency Event.
14.2.3 Termination by Roche without a Cause
Roche shall have the right to terminate this Agreement at any time on a Product-by-Product, patent-by-patent, and country-by-country basis upon sixty (60) days prior written notice. The effective date of termination under this Section 14.2.3 shall be the date sixty (60) days after Roche provides such written notice to Raptor. Upon termination of this Agreement in any country, Roche will cease manufacturing, marketing, and/or selling the Products in such country, if and only if the Royalty Term has not yet expired.
14.3 Consequences of Termination
14.3.1 Termination by Raptor for Breach by Roche, or Roche Insolvency Event; Termination by Roche without Cause
Upon any termination of this Agreement with respect to any patent(s) and/or country(ies) by Roche without cause pursuant to Section 14.2.3, all rights and licenses granted by Raptor to Roche under this Agreement with respect to such terminated patent(s) and/or terminated country(ies) shall also terminate on the effective date of termination.

 

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Upon any termination of this Agreement in its entirety or with respect to any country(ies), or Product(s) by Raptor for an uncured material breach by Roche pursuant to Section 14.2.1, for a Roche Insolvency Event pursuant to Section 14.2.2, or any termination of this Agreement in its entirety or with respect to any Product(s) by Roche without cause pursuant to Section 14.2.3 all rights and licenses granted by Raptor to Roche under this Agreement, in the terminated country(ies) and/or with respect to the terminated Product(s), shall also terminate on the effective date of termination.
14.3.2 Termination by Roche for Breach by Raptor or Raptor Insolvency Event
Upon any termination of this Agreement with respect to any country(ies) or in its entirety by Roche for an uncured material breach by Raptor pursuant to Section 14.2.1, all rights and obligations of each Party under this Agreement, with respect to the terminated country(ies) or this Agreement in its entirety, shall terminate on the effective date of termination (except as set forth in Section 14.4); provided, however, that Roche shall have the right to elect, by written notice to Raptor, to (i) continue the licenses granted to Roche by Raptor under Section 2.1.1, (and, (ii) solely if and to the extent that the discovery, manufacture and development activities contemplated under the Research Plan have not been completed with respect to any Product as of the effective date of termination, continue the licenses granted to Roche by Raptor under Section 2.1.2 of this Agreement, in each case subject to Roche’s continued compliance with (a) Roche’s payment obligations under Article 6 (Payments) with respect to Products developed and/or commercialized by Roche under such licenses, and (b) the terms of Section 2.2 (Sublicense), Article 7 (Accounting and Reporting), and Article 9 (Auditing) .
Upon any termination of this Agreement in its entirety by Roche for a Raptor Insolvency Event pursuant to Section 14.2.2, all rights and licenses granted to Roche by Raptor under this Agreement in the terminated country(ies) shall also terminate on the effective date of termination.
14.3.3 Royalty and Payment Obligations
Raptor shall use Commercially Reasonable Efforts to mitigate the cost of Non-cancellable Obligations, and Roche shall cooperate with Raptor in such efforts, and Raptor shall promptly notify Roche as to the amount of such mitigation. In the event of termination of this Agreement by Raptor under Section 14.2.1 or 14.2.2, or by Roche under Section 14.2.3, Roche will reimburse Raptor for Non-cancellable Obligations, except to the extent any Non-cancellable Obligations have been or will be mitigated by Raptor.
Termination of this Agreement by a Party, for any reason, shall not release Roche from any obligation to pay royalties or make any payments to Raptor which are due and payable prior to the effective date of termination.
Other than as set forth in the first paragraph of this Section 14.3.3, termination of this Agreement by a Party, for any reason, will release Roche from any obligation to pay royalties or make any payments to Raptor which would otherwise become due or payable on or after the effective date of termination.
14.4 Survival
Article 6 (Payment) (solely to the extent that any amounts are due hereunder but not paid as of the effective date of termination), Article 7 (Accounting and Reporting), Article 8 (Taxes), Article 9 (Auditing), Article 10 (Intellectual Property), , Article 12 (Indemnification), Section 13.1 (Non-Use and Non-Disclosure), Section 13.2 (Permitted Disclosure), Section 14.1 (Commencement and Term), Section 14.2 (Termination), Section 14.3 (Consequences of Termination), Section 14.4 (Survival), Section 16.1 (Governing Law), Section 16.2 (Disputes), Section 16.3 (Arbitration), Section 16.4 (Assignment), and Section 16.14 (Actions of Affiliates) shall survive any expiration or termination of this Agreement for any reason in accordance with their terms.

 

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15. Bankruptcy
All licenses (and to the extent applicable rights) granted under or pursuant to this Agreement by Raptor to Roche are, and shall otherwise be deemed to be, for purposes of Section 365(n) of Title 11, US Code (the “Bankruptcy Code”) licenses of rights to “intellectual property” as defined under the Bankruptcy Code. Unless Roche elects to terminate this Agreement, the Parties agree that Roche, as a licensee or sub-licensee of such rights under this Agreement, shall retain and may fully exercise all of its rights and elections under the Bankruptcy Code, subject to the continued performance of its obligations under this Agreement.
16. Miscellaneous
16.1 Governing Law
This Agreement shall be governed by and construed in accordance with the laws of the State of New Jersey, U.S.A., without reference to its conflict of laws principles, and shall not be governed by the United Nations Convention of International Contracts on the Sale of Goods (the Vienna Convention).
16.2 Disputes
Unless otherwise set forth in this Agreement, in the event of any dispute in connection with this Agreement, such dispute shall be referred to the respective executive officers of the Parties designated below or their designees, for good faith negotiations attempting to resolve the dispute. The designated executive officers are as follows:
     
For Raptor:
  CEO
For Roche:
  Head of Pharma Partnering
16.3 Arbitration
Should the Parties fail to agree within one (1) month after a dispute is referred to the executive officers for resolution under Section 16.2, it shall be finally settled by arbitration in accordance with the commercial arbitration rules of the American Arbitration Association (AAA) as in force at the time when initiating the arbitration. The tribunal shall consist of a single arbitrator who shall be appointed in accordance with such rules. The place of arbitration shall be New York City, New York. The language to be used shall be English. The award through arbitration shall be final and binding. Either Party may enter any such award in a court having jurisdiction or may make application to such court for judicial acceptance of the award and an order of enforcement, as the case may be. Nothing in Section 16.2 or 16.3 shall prevent or limit either Party from seeking or obtaining injunctive relief from any court of competent jurisdiction.
16.4 Assignment
Neither Party may assign this Agreement or any of its rights or obligations under this Agreement to a Third Party absent the prior written consent of the other Party, except that either Party may, without the other Party’s prior written consent, make an assignment to any of its Affiliates or in connection with the merger or sale of all or substantially all of the assets or business of such Party to which this Agreement relates. Any permitted assignment shall be binding on the successors of the assigning Party. Any assignment notwithstanding, the assigning Party in all cases shall remain primarily liable hereunder for the prompt and punctual payment and performance of all obligations of the assignee (i.e., to that extent, no such assignment shall constitute a novation or otherwise release the assigning Party from liability hereunder).

 

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16.5 Debarment
  (a)  
Raptor hereby certifies that it has not been debarred under the provisions of the Generic Drug Enforcement Act of 1992, 21 U.S.C. Sec. 335a(a) and (b). In the event that during the term of this Agreement Raptor or any of its employees (i) becomes debarred; or (ii) receives notice of an action or threat of an action with respect to its debarment, Raptor agrees to immediately notify Roche. Raptor also agrees that in the event that it becomes debarred it shall immediately cease all activities relating to this Agreement.
  (b)  
In the event that Raptor becomes debarred, this Agreement shall automatically terminate, without any further action or notice by either party. In the event that Roche receives notice from Raptor or otherwise becomes aware that (i) a debarment action has been brought against Raptor or any of its employees; or (ii) Raptor has been threatened with a debarment action, then Roche shall have the right to terminate this Agreement immediately.
  (c)  
Raptor hereby certifies that it has not and will not use in any capacity the services of any individual, corporation, partnership or association which has been debarred under 21 U.S.C. Sec. 335(a) or (b). In the event that Raptor becomes aware of the debarment or threatened debarment of any individual, corporation, partnership or association providing services to Raptor which directly or indirectly relate to the activities under this Agreement, Raptor shall notify Roche immediately. Upon the receipt of such notice by Roche or if Roche otherwise becomes aware of such debarment or threatened debarment, Roche shall have the right to terminate this Agreement immediately.
16.6 Independent Contractor
No employee or representative of either Party shall have any authority to bind or obligate the other Party to this Agreement for any sum or in any manner whatsoever or to create or impose any contractual or other liability on the other Party without said Party’s prior written approval. For all purposes, and notwithstanding any other provision of this Agreement to the contrary, Raptor’s legal relationship to Roche under this Agreement shall be that of independent contractor.
16.7 Unenforceable Provisions and Severability
If any of the provisions of this Agreement are held to be void or unenforceable, then such void or unenforceable provisions shall be replaced by valid and enforceable provisions which will achieve as far as possible the economic business intentions of the Parties. However, the remainder of this Agreement will remain in full force and effect, provided that the material interests of the Parties are not affected, i.e. the Parties would presumably have concluded this Agreement without the unenforceable provisions.

 

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16.8 Waiver
The failure by either Party to require strict performance and/or observance of any obligation, term, provision or condition under this Agreement will neither constitute a waiver thereof nor affect in any way the right of the respective Party to require such performance and/or observance. The waiver by either Party of a breach of any obligation, term, provision or condition hereunder shall not constitute a waiver of any subsequent breach thereof or of any other obligation, term, provision or condition.
16.9 Appendices
All Appendices to this Agreement shall form an integral part to this Agreement.
16.10 Entire Understanding
This Agreement contains the entire understanding between the Parties with respect to the within subject matter and supersedes any and all prior agreements, understandings and arrangements, whether written or oral.
16.11 Amendments
No amendments of the terms and conditions of this Agreement shall be binding upon either Party unless in writing and signed by both Parties.
16.12 Notice
All notices which are required or permitted hereunder shall be in writing and sufficient if delivered personally, sent by facsimile (and promptly confirmed by personal delivery, registered or certified mail or overnight courier), sent by nationally recognized overnight courier or sent by registered or certified mail, postage prepaid, return receipt requested, addressed as follows:
     
if to Raptor, to:
  Raptor Pharmaceutical Inc.
 
  9 Commercial Blvd., Suite 200
 
  Novato, CA 94949
 
  Attn: Christopher M. Starr, Ph.D., CEO
 
  Facsimile No.: 415-382-1458
 
   
And:
   
 
   
if to Roche, to:
  F. Hoffmann-La Roche Ltd
 
  Grenzacherstrasse 124
 
  4070 Basel
 
  Switzerland
 
  Attn: Legal Department
 
  Facsimile No.: +41 61 688 13 96
 
   
And:
  Hoffmann-La Roche Inc.
 
  340 Kingsland Street
 
  Nutley, New Jersey 07110
 
  U.S.A.
 
  Attn. Corporate Secretary
 
  Facsimile No.: +1 973 235-3500

 

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or to such other address as the Party to who notice is to be given may have furnished to the other Party in writing in accordance herewith. The effective date of notice shall be the actual date of receipt by the Party receiving the same.
16.13 Change of Control
In the event of a Change of Control of Raptor pursuant to which Raptor is acquired by, or consummates a business combination with, a direct competitor of Roche or such a direct competitor acquires more than [*****] of the common shares or voting power of Raptor, Roche shall not be required under to provide any competitively-sensitive information to such Roche competitor. A direct competitor of Roche is any pharmaceutical or biotechnology company which markets or is developing one or more products directed to an Indication or target for which Roche or its Affiliates are developing and/or commercializing products.
16.14 Actions of Affiliates
For purposes of clarity, each Party may perform its obligations hereunder personally or through one or more Affiliates, provided, that such Party shall nonetheless be primarily liable for the performance of its Affiliates and for any failure by its Affiliates to comply with the restrictions, limitations and obligations set forth in this Agreement. To the extent that the rights granted to a Party hereunder may be and are exercised by an Affiliate of such Party, such Affiliate shall be bound by the corresponding obligations of such Party. Notwithstanding any of the foregoing, Roche Nutley and Roche Basel shall be jointly and severally liable under this Agreement.
16.15 Execution in Counterparts
This Agreement may be executed in counterparts, each of which counterparts, when so executed and delivered, shall be deemed an original and all of which counterparts, taken together, shall constitute one and the same instrument.
* * * * *
[Signature Page to Follow]

 

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EXECUTION VERSION
IN WITNESS WHEREOF, the Parties have entered into this Agreement as of the Effective Date.
Raptor Pharmaceutical Inc.
By: /s/ Kim R. Tsuchimoto
Name: Kim R. Tsuchimoto
Title: Chief Financial Officer
F. Hoffmann-La Roche Ltd
             
By:
  [Illegible]   By:   /s/ Stefan Arnold
 
           
Name:
      Name:   Stefan Arnold
 
           
Title:
      Title:   Head Corporate Law Pharma
 
           
Hoffmann-La Roche Inc.        
 
           
By:
  /s/ A. Waseem Malick        
 
           
Name:
  A. Waseem Malick        
 
           
Title:
  VP, PARD        
[*****] Raptor Pharmaceutical Corp. has requested confidential treatment of certain portions of this agreement which have been omitted and filed separately with the U.S. Securities and Exchange Commission pursuant to Rule 24b-2 under the Securities Exchange Act of 1934.
[Signature Page to Collaboration and License Agreement]

 

 


 

Appendix 1.42
Raptor Base Patent Rights as of the Effective Date
[*****]
[Appendix 1.42-1]

 

 


 

Appendix 1.45
Initial Research Plan
[*****]
[Appendix 1.45]

 

 

EX-10.20 5 c91590exv10w20.htm EXHIBIT 10.20 Exhibit 10.20

EXHIBIT 10.20

SUPPLY AGREEMENT

This Supply Agreement (“Agreement”) is effective as of this 20th day of July, 2009 (“Effective Date”), by and between Mylan Pharmaceuticals Inc., a corporation duly organized under the laws of the state of West Virginia (MYLAN”), and Raptor Therapeutics Inc., a corporation duly organized under the laws of Delaware (“RAPTOR”). RAPTOR and MYLAN may be referred to herein as the “Party” or “Parties.”

WHEREAS, RAPTOR desires to enter into this Agreement for the supply of the Active Pharmaceutical Ingredient (“API”) from MYLAN; and

WHEREAS, MYLAN desires to enter into this Agreement and grant such supply of the API to RAPTOR.

NOW, THEREFORE, WITNESSETH that for and in consideration of the mutual promises and covenants set forth herein and for other good and valuable consideration, the receipt and sufficiency of all of which are hereby acknowledged, the Parties, intending to be legally bound, agree as follows:

I.  
SCOPE OF AGREEMENT

  1.  
RAPTOR shall purchase from MYLAN Active Pharmaceutical Ingredient (“API”) that meets the specifications, as further defined in ATTACHMENT I, as may be ordered by RAPTOR in accordance with the terms and conditions herein.

  2.  
MYLAN shall supply API to RAPTOR that meets the specifications, as further defined in ATTACHMENT I. MYLAN shall procure all supplies of API from [*****]. MYLAN shall not change or modify the specifications set forth on ATTACHMENT I in any manner that would impact the manufacturing or processing activities related to the supply of API herein without first giving RAPTOR prior written notice of no less than [*****]. MYLAN shall notify RAPTOR of any notice of change or modification issued by CAMBREX within [*****] of the receipt of said notice.

[*****]

  3.  
MYLAN shall support the filing of RAPTOR’s New Drug Application(s) (“NDA(s)”) for products containing API and commercial production thereof by providing API, technical documentation, and regulatory information as requested by RAPTOR to RAPTOR. MYLAN shall authorize RAPTOR (or its designees) the right to reference all such technical documentation and regulatory information to support any RAPTOR NDA, including all Drug Master Files provided to a regulatory authority by or on behalf of MYLAN or its permitted contractors [*****].

II.  
FORECAST AND SUPPLY

  1.  
MYLAN shall supply the API to RAPTOR pursuant to the product, price, volume, and time period listed in ATTACHMENT II.

  2.  
RAPTOR shall provide a non-binding, [*****] rolling forecast [*****] for the supply of API (“PO”) to MYLAN, [*****] in advance of delivery to RAPTOR.

Page 1 of 9

1


 

  3.  
MYLAN shall schedule the purchase of the API based on [*****] shall accept such binding POs received from RAPTOR at least [*****] in advance of delivery.

  4.  
In the event of a supply shortage of the API where MYLAN is unable to fulfill RAPTOR’S requirements set forth in a particular PO, MYLAN shall immediately notify RAPTOR. RAPTOR may modify or cancel such Purchase Orders accordingly, without penalty, upon written notice of no less than [*****]before the delivery date specified in such PO to MYLAN.

  5.  
[*****] A “DELAY” shall mean any shipment of API delivered pursuant to a PO of which more than [*****] of the quantities ordered in the applicable PO is delivered later than [*****] after the delivery date specified in the applicable PO through no fault of MYLAN.

III.  
PAYMENT

  1.  
RAPTOR shall remit payment for the API [*****] after RAPTOR’s receipt and approval of MYLAN’s invoice therefor. Notwithstanding the foregoing, RAPTOR may withhold payment for any shipment or portion thereof that has been rejected by RAPTOR for nonconformance under Section IV.3 below.

IV.  
SHIPPING AND RETURNS

  1.  
All shipments of API from MYLAN to RAPTOR will be shipped [*****].

  2.  
RAPTOR will receive and test API within [*****] of delivery. RAPTOR will notify MYLAN of non-conforming results within [*****].

  3.  
MYLAN shall accept and replace returns of API that are not in conformance with the applicable specifications or standards set forth under this agreement when identified by RAPTOR, or refund the purchase price of the API, within [*****] of receipt of the API by RAPTOR.

V.  
QUALITY CONTROL

  1.  
MYLAN shall provide API that meets the United States Pharmacopeia (“USP”) and International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (“ICH”) requirements.

  2.  
MYLAN shall provide API in accordance with ATTACHMENT I, the Drug Master Files (“DMF”) for the manufacture of API provided by or on behalf of MYLAN or its permitted contractors and current Good Manufacturing Practices (“cGMPs”) set forth by the FDA or other regulatory authorities. The Parties shall use commercially reasonable efforts to negotiate and execute a mutually agreeable quality agreement prior RAPTOR issuing its first purchase order for API to MYLAN pursuant to the Agreement.

  3.  
MYLAN shall promptly provide the necessary resources to respond to the FDA or other regulatory authority determined deficiencies and information requests regarding the API and the process used by the manufacturer to manufacture the API (including providing access to the applicable manufacturing facilities used to manufacture the API). MYLAN

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2


 

shall provide to RAPTOR copies of all such determined deficiencies, information requests, inspection reports and other correspondence of the FDA or other regulatory authority related to the manufacture of API within [*****] after obtaining knowledge thereof.

  4.  
MYLAN will allow RAPTOR to audit MYLAN’s storage and shipping facilities annually during normal business hours and after receiving at least [*****] prior written notice. In addition, MYLAN shall use commercially reasonable efforts to facilitate an annual request by RAPTOR to audit other facilities not owned by MYLAN that are directly related to the manufacture of API, upon receiving at least [*****] prior written notice.

  5.  
MYLAN shall generate and maintain complete and accurate records (including files, certificates and authorizations) and samples as necessary to evidence compliance with this Agreement, and applicable laws, regulatory requirements, and other requirements of applicable governmental authorities relating to the manufacture of API. All such records and samples shall be securely maintained for a period of not less than [*****] from the date received or generated by MYLAN or such period as may be required by regulatory requirements. Upon RAPTOR’S request, MYLAN shall provide RAPTOR with reasonable access to, and copies and portions of, such records and samples and any supporting data relating thereto, and MYLAN shall not dispose of such records or samples without first offering to transfer such records or samples to RAPTOR at RAPTOR’S expense.

VI.  
WARRANTIES AND REPRESENTATIONS

  1.  
MYLAN warrants and represents the following:

  (i)  
MYLAN is a corporation duly organized, validly existing and in good standing under the laws of West Virginia;

  (ii)  
MYLAN has all requisite power and authority to enter into this Agreement and to carry out its obligations hereunder;

  (iii)  
The person signing this Agreement has the necessary corporate authority to legally bind MYLAN to the terms set forth herein;

  (iv)  
The execution by MYLAN of this Agreement and the performance by MYLAN of the terms set forth herein will not cause MYLAN to be in material conflict with or constitute a material breach of any agreement or understanding with any third party;

  (v)  
To MYLAN’s knowledge and belief, there are no suits, adverse third party allegations or actions, claims, proceedings, or investigations pending or threatened by or before any court, by or before any government body or agency, or any individual related, to the matters set forth herein;

  (vi)  
The execution of this Agreement and MYLAN’s performance hereunder do not and will not be in material conflict with any law, ordinance, statute or regulation or any current Good Laboratory Practice (“cGLP”), cGMP, or FDA guidelines or policies;

Page 3 of 9

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  (vii)  
MYLAN is not debarred and MYLAN has not and will not use in any capacity the services of any person debarred under subsection 306(a) or (b) of the Generic Drug Enforcement Act of 1992. If at any time this representation and warranty is no longer accurate, MYLAN shall immediately notify RAPTOR of such fact;

  (viii)  
MYLAN has and will maintain throughout the term of this Agreement all permits, licenses, registrations and other forms of governmental authorization and approval as required by law in order for MYLAN to execute and deliver this Agreement and to perform its obligations hereunder in accordance with all applicable laws;

  (ix)  
MYLAN represents and warrants that the API supplied to MYLAN shall meet the agreed specifications, as further defined in ATTACHMENT I; the United States Pharmacopeia (“USP”) requirements; and the International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (“ICH”) requirements

  2.  
RAPTOR warrants and represents the following:

  (i)  
RAPTOR is a corporation duly organized, validly existing and in good standing under the laws of the state of Delaware;

  (ii)  
RAPTOR has all requisite power and authority to enter into this Agreement and to carry out its obligations hereunder;

  (iii)  
The person signing this Agreement has the necessary corporate authority to legally bind RAPTOR to the terms set forth herein;

  (iv)  
The execution by RAPTOR of this Agreement and the performance by RAPTOR of the terms set forth herein will not cause RAPTOR to be in material conflict with or constitute a material breach of any agreement or understanding with any third party;

  (v)  
To RAPTOR’s knowledge and belief, there are no suits, adverse third party allegations, actions, claims, proceedings, or investigations pending or threatened by or before any court, by or before any government body or agency, or any individual, related to the matters set forth herein;

  (vi)  
The execution of this Agreement and RAPTOR’s performance hereunder do not and will not be in material conflict with any law, ordinance, statute or regulation;

  (vii)  
RAPTOR is not debarred and RAPTOR has not and will not use in any capacity the services of any person debarred under subsections 306(a) or (b) of the Generic Drug Enforcement Act of 1992. If at any time this representation and warranty is no longer accurate, RAPTOR shall immediately notify MYLAN of such fact; and

  (viii)  
RAPTOR has and will maintain throughout the term of this Agreement all federal, state and local permits, licenses, registrations and other forms of governmental authorization and approval as required by law in order for

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RAPTOR to execute and deliver this Agreement and to perform its obligations hereunder in accordance with all applicable laws.

VII.  
TERM AND TERMINATION

  1.  
This Agreement shall begin on the Effective Date written above and shall continue in full force and effect for a period of [*****] (“Initial Term”). Thereafter, the term of this Agreement shall continue in full force and effect until terminated in accordance with this Section VII.

  2.  
After the Initial Term, either Party may terminate this Agreement with [*****] prior written notice to the non-terminating Party.

  3.  
This Agreement shall be subject to immediate termination in the event the manufacture, distribution, or sale of the API would materially contravene any applicable law [*****].

  4.  
Upon the termination of this Agreement; pursuant to Section VII(3), due to a material breach of the terms of this Agreement by MYLAN, or a unilateral termination by MYLAN pursuant to Section VII, RAPTOR shall have no obligation to purchase any API ordered in any PO. Termination of this Agreement shall not affect the rights and obligations of either Party that may have accrued prior to the date of termination, or any right or obligation contained in Sections III, IV, V.1, V.2 (with respect to outstanding POs to be fulfilled) and Sections V.3, V.4, V.5, VI, VII.4, VIII, IX, X, XI, XII, XIII, XIV, XV.

VIII.  
INDEMNIFICATION

  1.  
RAPTOR agrees to indemnify, defend and hold MYLAN harmless from and against any losses resulting from or arising out of [*****].

  2.  
MYLAN agrees to indemnify, defend and hold RAPTOR harmless from and against any losses resulting from or arising out of [*****].

IX.  
CONFIDENTIALITY

Information exchanged pursuant to this Agreement, shall be subject to the terms and conditions of that certain Confidentiality Agreement executed and entered into on the 7th day of April, 2009, by and between Raptor Pharmaceuticals Corp., the parent company of Raptor Therapeutics Inc., and Mylan Pharmaceuticals Inc. (the “CDA”). The terms and conditions of the CDA notwithstanding, a Party may disclose the Confidential Information of the disclosing Party to extent required to exercise its rights or perform its obligations hereunder.

X.  
ASSIGNMENT

RAPTOR may not assign this Agreement or any of its rights or obligations hereunder, to a third party, without the express, prior, written consent of MYLAN; except that RAPTOR may assign this Agreement without such consent to any successor to all or substantially all of RAPTOR’S assets or business to which this Agreement relates. Any assignment in violation of this Section X shall be null and void.

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XI.  
GOVERNING LAW

Any and all actions between the Parties regarding the interpretation or application of any term or provision contained herein shall be governed by and interpreted in accordance with the laws of the State of New York, excluding its conflict of laws principles. MYLAN and RAPTOR each do hereby respectively consent and agree that the courts of the State of New York shall have jurisdiction with respect to any and all actions brought hereunder.

XII.  
LIMITATION OF LIABILITY

In no event shall either Party be liable to the other Party or its Affiliates for special, punitive, indirect, incidental, exemplary or consequential loss or damage, or for lost profits, based on a contract, tort, or any other legal theory, arising out any breach of this Agreement or otherwise relating to the subject matter of this Agreement, except as may be specifically and expressly stated in this Agreement.

[*****]

XIII.  
NOTICES

Any notice of communication to be given hereunder shall be in writing and be delivered personally, mailed, or sent via facsimile transmission, as follows:

     
If to MYLAN:   Mylan Pharmaceuticals Inc.
 
  781 Chestnut Ridge Road
 
  Morgantown, West Virginia 26505
 
  Attention:
 
  Facsimile No.:
     
 
  With a copy to:
     
 
  Deputy General Counsel
 
  Mylan Inc.
 
  1500 Corporate Drive
 
  Canonsburg, PA 15317
 
  Facsimile No.: (724) 514-1870
     
If to RAPTOR:
   
     
 
  Raptor Therapeutics Inc.
 
  9 Commercial Boulevard
 
  Suite 200
 
  Novato, CA 94949

XIV.  
SEVERABILITY

If any provision of this Agreement is found invalid or unenforceable by a court of law, the remainder of this Agreement shall continue in full force and effect.

Page 6 of 9

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XV.  
WAIVER

A waiver by either Party of any terms, provisions, or condition of this Agreement shall not constitute a precedent or bind either Party to a waiver of any succeeding default or other breach of the same of any other term, provision, or conditions of this Agreement.

XVI.  
COUNTERPARTS

This Agreement may be executed in two or more counterparts, each of which shall be considered an original but all of which shall constitute one agreement.

XVII.  
ENTIRE AGREEMENT

The terms and conditions of this Agreement, ATTACHMENT I, ATTACHMENT II, and the CDA, express the entire agreement between MYLAN and RAPTOR and supersede all prior drafts and/or understandings between the Parties.

IN WITNESS of their agreement to the terms and conditions contained herein, MYLAN and RAPTOR, intending to be legally bound, have caused the following signatures to be affixed hereto:

     
MYLAN PHARMACEUTICALS INC.   RAPTOR THERAPEUTICS INC.
     
BY: /s/ Harry A. Korman
  BY: /s/ Thomas E. Daley
     
PRINT NAME: Harry A. Korman
  PRINT NAME: Thomas E. Daley
     
TITLE: President, North America
  TITLE: President

[*****]   Raptor Pharmaceutical Corp. has requested confidential treatment of certain portions of this agreement which have been omitted and filed separately with the U.S. Securities and Exchange Commission pursuant to Rule 24b-2 under the Securities Exchange Act of 1934.

Page 7 of 9

7


 

ATTACHMENT I

[*****]

Page 8 of 9

8


 

ATTACHMENT II

[*****]

Page 9 of 10

9

EX-21.1 6 c91590exv21w1.htm EXHIBIT 21.1 Exhibit 21.1

EXHIBIT 21.1

Raptor Therapeutics Inc. (f/k/a Bennu Pharmaceuticals Inc.)

Raptor Discoveries Inc. (f/k/a Raptor Pharmaceutical Inc.)

 

EX-23.1 7 c91590exv23w1.htm EXHIBIT 23.1 Exhibit 23.1

BPM_LLP_logo_4c

EXHIBIT 23.1  

CONSENT OF INDEPENDENT REGISTERED PUBLIC ACCOUNTING FIRM  

We hereby consent to the incorporation by reference in the Registration Statements on Form S-3 No. 333-140944 and No. 333-162430 of Raptor Pharmaceutical Corp. of our report dated October 27, 2009, relating to the consolidated financial statements of Raptor Pharmaceuticals Corp. (which report expresses an unqualified opinion and includes an explanatory paragraph regarding uncertainty about Raptor Pharmaceuticals Corp.’s ability to continue as a going concern), which appear in Raptor Pharmaceuticals Corp’s Annual Report on Form 10-K for the year ended August 31, 2009. We also consent to the reference to our firm under the heading “Experts” in such Registration Statements.

/s/ Burr Pilger & Mayer, LLP
San Francisco, California
October 27, 2009

 

EX-31.1 8 c91590exv31w1.htm EXHIBIT 31.1 Exhibit 31.1

EXHIBIT 31.1

CERTIFICATION OF PRINCIPAL EXECUTIVE OFFICER PURSUANT TO SECTION 302 OF
THE SARBANES-OXLEY ACT OF 2002

I, Christopher M. Starr, certify that:

1. I have reviewed this Annual Report on Form 10-K of Raptor Pharmaceuticals Corp.;

2. Based on my knowledge, this report does not contain any untrue statement of a material fact or omit to state a material fact necessary to make the statements made, in light of the circumstances under which such statements were made, not misleading with respect to the period covered by this report;

3. Based on my knowledge, the financial statements, and other financial information included in this report, fairly present in all material respects the financial condition, results of operations and cash flows of the registrant as of, and for, the periods presented in this report;

4. The registrant’s other certifying officer and I are responsible for establishing and maintaining disclosure controls and procedures (as defined in Exchange Act Rules 13a-15(e) and 15d-15(e)) and internal control over financial reporting (as defined in Exchange Act Rules 13a-15(f) and 15d-15(f)) for the registrant and have:

a. Designed such disclosure controls and procedures, or caused such disclosure controls and procedures to be designed under our supervision, to ensure that material information relating to the registrant, including its condensed consolidated subsidiaries, is made known to us by others within those entities, particularly during the period in which this report is being prepared:

b. Designed such internal control over financial reporting, or caused such internal control over financial reporting to be designed under our supervision, to provide reasonable assurance regarding the reliability of financial reporting and the preparation of financial statements for external purposes in accordance with generally accepted accounting principles;

c. Evaluated the effectiveness of the registrant’s disclosure controls and procedures and presented in this report our conclusions about the effectiveness of the disclosure controls and procedures, as of the end of the period covered by this report based on such evaluation; and

d. Disclosed in this report any change in the registrant’s internal control over financial reporting that occurred during the registrant’s most recent fiscal quarter (the registrant’s fourth fiscal quarter in the case of an annual report) that has materially affected, or is reasonably likely to materially affect, the registrant’s internal control over financial reporting; and

5. The registrant’s other certifying officer and I have disclosed, based on our most recent evaluation of internal control over financial reporting, to the registrant’s auditors and the audit committee of the registrant’s board of directors (or persons performing the equivalent functions):

a. All significant deficiencies and material weaknesses in the design or operation of internal control over financial reporting which are reasonably likely to adversely affect the registrant’s ability to record, process, summarize and report financial information; and

b. Any fraud, whether or not material, that involves management or other employees who have a significant role in the registrant’s internal control over financial reporting.

Date: October 28, 2009

By: /s/ Christopher M. Starr
Christopher M. Starr, Ph.D.
Chief Executive Officer and Director
(Principal Executive Officer)

 

EX-31.2 9 c91590exv31w2.htm EXHIBIT 31.2 Exhibit 31.2

EXHIBIT 31.2

CERTIFICATION OF PRINCIPAL FINANCIAL OFFICER PURSUANT TO SECTION 302 OF
THE SARBANES-OXLEY ACT OF 2002

I, Kim R. Tsuchimoto, certify that:

1. I have reviewed this Annual Report on Form 10-K of Raptor Pharmaceuticals Corp.;

2. Based on my knowledge, this report does not contain any untrue statement of a material fact or omit to state a material fact necessary to make the statements made, in light of the circumstances under which such statements were made, not misleading with respect to the period covered by this report;

3. Based on my knowledge, the financial statements, and other financial information included in this report, fairly present in all material respects the financial condition, results of operations and cash flows of the registrant as of, and for, the periods presented in this report;

4. The registrant’s other certifying officer and I are responsible for establishing and maintaining disclosure controls and procedures (as defined in Exchange Act Rules 13a-15(e) and 15d-15(e)) and internal control over financial reporting (as defined in Exchange Act Rules 13a-15(f) and 15d-15(f)) for the registrant and have:

a. Designed such disclosure controls and procedures, or caused such disclosure controls and procedures to be designed under our supervision, to ensure that material information relating to the registrant, including its condensed consolidated subsidiaries, is made known to us by others within those entities, particularly during the period in which this report is being prepared:

b. Designed such internal control over financial reporting, or caused such internal control over financial reporting to be designed under our supervision, to provide reasonable assurance regarding the reliability of financial reporting and the preparation of financial statements for external purposes in accordance with generally accepted accounting principles;

c. Evaluated the effectiveness of the registrant’s disclosure controls and procedures and presented in this report our conclusions about the effectiveness of the disclosure controls and procedures, as of the end of the period covered by this report based on such evaluation; and

d. Disclosed in this report any change in the registrant’s internal control over financial reporting that occurred during the registrant’s most recent fiscal quarter (the registrant’s fourth fiscal quarter in the case of an annual report) that has materially affected, or is reasonably likely to materially affect, the registrant’s internal control over financial reporting; and

5. The registrant’s other certifying officer and I have disclosed, based on our most recent evaluation of internal control over financial reporting, to the registrant’s auditors and the audit committee of the registrant’s board of directors (or persons performing the equivalent functions):

a. All significant deficiencies and material weaknesses in the design or operation of internal control over financial reporting which are reasonably likely to adversely affect the registrant’s ability to record, process, summarize and report financial information; and

b. Any fraud, whether or not material, that involves management or other employees who have a significant role in the registrant’s internal control over financial reporting.

Date: October 28, 2009

By: /s/ Kim R. Tsuchimoto
Kim R. Tsuchimoto
Chief Financial Officer, Secretary and Treasurer
(Principal Financial Officer and Principal Accounting Officer)

 

EX-32.1 10 c91590exv32w1.htm EXHIBIT 32.1 Exhibit 32.1

Exhibit 32.1

Certification Pursuant to Section 906 of the Sarbanes-Oxley Act of 2002

Securities and Exchange Commission
450 Fifth Street, N.W
Washington, D.C. 20549

Ladies and Gentlemen:

The certification set forth below is being furnished to the Securities and Exchange Commission solely for the purpose of complying with Rule 13a-14(b) or Rule 15d-14(b) of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), and with Section 1350 of Chapter 63 of Title 18 of the United States Code. A signed original of this written statement required by Section 906 has been provided to Raptor Pharmaceuticals Corp. (the “Company”) and will be retained by the Company and furnished to the Securities and Exchange Commission or its staff upon request.

Christopher M. Starr, Ph.D., the Chief Executive Officer and Director of the Company. and Kim R. Tsuchimoto, the Chief Financial Officer, Secretary and Treasurer of the Company each certify that:

1.  
this Annual Report on Form 10-K (the “Report”) fully complies with the requirements of Section 13(a) or 15(d) of the Exchange Act; and

2.  
the information contained in the Report fairly presents, in all material respects, the financial condition and results of operations of Raptor Pharmaceuticals Corp.

IN WITNESS WHEREOF, the undersigned have set their hands hereto as of the 28th day of October, 2009.

By: /s/ Christopher M. Starr
Christopher M. Starr, Ph.D.
Chief Executive Officer and Director

By: /s/ Kim R. Tsuchimoto
Kim R. Tsuchimoto
Chief Financial Officer, Secretary and Treasurer
(Principal Financial Officer and Principal Accounting Officer)
 

 

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