EX-99.1 2 dex991.htm PRESS RELEASE Press Release

Exhibit 99.1

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Investors     Media
Eugenia Shen     Bob Purcell
BioMarin Pharmaceutical Inc.     BioMarin Pharmaceutical Inc.
(415) 506-6570     (415) 506-3267

For Immediate Release:

BioMarin Announces First Quarter 2011 Financial Results

Net Product Revenue Growth of 30% in the First Quarter of 2011; Improved Top and Bottom-Line Guidance

Conference Call and Webcast to Be Held Today at 5:00 p.m. ET

Financial Highlights ($ in millions, except per share data, unaudited)

 

Item    Q1 2011    Q1 2010 Comparison
Total BioMarin Revenue    $109.5    28.8% increase
Total Net Product Revenue    $109.1    29.7% increase
Naglazyme Net Product Revenue    $60.6    24.7% increase
Aldurazyme BioMarin Net Product Revenue*    $18.7    $14.2
Kuvan Net Product Revenue    $26.7    25.9% increase
Firdapse Net Product Revenue    $3.1    $0.1
GAAP Net Income (Loss)    $(4.4)    $1.2
GAAP Net Income (Loss) per share    $(0.04) (basic and diluted)    $0.01 (basic and diluted)
Non-GAAP Adjusted EBITDA    $17.3    $14.8
Non-GAAP Adjusted EBITDA per share    $0.16 (basic), $0.14 (diluted)    $0.15 (basic), $0.14 (diluted)

 

* Net product transfer revenue had a positive $1.8 million impact on net Aldurazyme revenue to BioMarin in the first quarter of 2011 and a negative $1.7 million impact on net Aldurazyme revenue to BioMarin in the first quarter of 2010.

Novato, Calif., April 28, 2011 – BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced financial results for the first quarter of 2011. GAAP net loss was $4.4 million ($0.04 per diluted share) for the first quarter of 2011, compared to GAAP net income of $1.2 million ($0.01 per diluted share) for the first quarter of 2010. Non-GAAP adjusted EBITDA was $17.3 million ($0.14 per diluted share) for the first quarter of 2011, compared to non-GAAP adjusted EBITDA of $14.8 million ($0.14 per diluted share) for the first quarter of 2010. Non-GAAP adjusted EBITDA excludes depreciation and amortization, contingent consideration expense, interest income and expense, income taxes, stock compensation expense and material non-recurring items. The reconciliation of the non-GAAP measures to the GAAP net income in the first quarter of 2011 is detailed in the table provided near the end of the press release.

As of March 31, 2011, BioMarin had cash, cash equivalents and short and long-term investments totaling $394.0 million, as compared to $402.3 million as of December 31, 2010. Cash and investment balances declined $8.3 million in the first quarter of 2011 due primarily to the timing of cash receipts that were collected in early April. For 2011, the company expects to remain cash flow neutral to slightly positive.

“Our clinical pipeline is advancing with more programs in the clinic than ever in the history of the company,” said Jean-Jacques Bienaimé, Chief Executive Officer of BioMarin. “We saw a strong quarter from our commercial business, driven in part by the timing of a Naglazyme order from Brazil early in the quarter, along with consistent new patient adds around the world. We remain focused on growing the commercial business and the successful advancement of our pipeline projects.”

 

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Net Product Revenue (in millions)

 

     Three Months Ended March 31,  
     2011      2010      $ Change      % Change  

Naglazyme (1)

   $ 60.6       $ 48.6       $ 12.0         24.7

Kuvan (2)

   $ 26.7       $ 21.2       $ 5.5         25.9

Firdapse (3)

   $ 3.1       $ 0.1       $ 3.0         100 %+ 

 

(1) Changes in foreign currency rates, net of hedges, had a positive $0.1 million impact on Naglazyme sales in the three months ended March 31, 2011. Naglazyme revenues experience quarterly fluctuations due to the timing of government ordering patterns in certain countries.
(2) The quantity of commercial tablets dispensed to patients in the U.S. increased 21.1 percent in the first quarter of 2011 compared to the first quarter of 2010.
(3) A product for the treatment of Lambert Eaton Myasthenic Syndrome (LEMS) which was launched in the EU in April 2010.

 

     Three Months Ended March 31,  
     2011      2010     $ Change      % Change  

Aldurazyme revenue reported by Genzyme (4)

   $ 42.8       $ 39.9      $ 2.9         7.3

Royalties due from Genzyme

     16.9         15.9        1.0      

Incremental (previously recognized)

     1.8         (1.7     3.5      
                            

Total Aldurazyme net product revenues (5)

   $ 18.7       $ 14.2      $ 4.5      
                            

 

(4) Changes in foreign currency rates caused an increase to Aldurazyme sales by Genzyme of $0.4 million in the three months ended March 31, 2011. In the first quarter of 2011, the number of Aldurazyme vials shipped increased 6.4 percent over the first quarter of 2010.
(5) To the extent units shipped to third party customers by Genzyme exceed BioMarin inventory transfers to Genzyme, BioMarin records a decrease in net product revenue from the royalty payable to BioMarin for the amount of previously recognized product transfer revenue. If BioMarin inventory transfers exceed units shipped to third party customers by Genzyme, BioMarin will record incremental net product transfer revenue for the period.

2011 Guidance

Revenue Guidance ($ in millions)

 

Item

  

    2011 Guidance    

  

Previous 2011 Guidance

Total BioMarin Revenues

   $422 to $452    $417 to $452

Total Net Product Revenues

   $416 to $446    $411 to $446

Naglazyme Net Product Revenue

   $211 to $225    $206 to $225

Kuvan Net Product Revenue

   Unchanged    $112 to $120

Aldurazyme Net Product Revenue to BioMarin

   Unchanged    $79 to $83

Firdapse Net Product Revenue

   Unchanged    $14 to $18

 

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Selected Income Statement Guidance ($ in millions)

 

Item

   2011 Guidance   Previous 2011 Guidance
Cost of Sales (% of Total Revenue)    Unchanged   18% to 20%
Selling, General and Admin. Expense    Unchanged   $164 to $174
Research and Development Expense    Unchanged   $195 to $205
Amortization and Contingent Consideration    Unchanged   $15
Interest Income    Unchanged   $3
Income Tax Expense (Benefit)    Unchanged   $15
GAAP Net Income (Loss)    $(52) to $(42)   $(60) to $(48)
Stock Compensation Expense    Unchanged   $43
Non-GAAP Adjusted EBITDA    $51 to $61   $43 to $55

Anticipated Upcoming Milestones

2Q 2011: Initiation of pivotal Phase III trial for Firdapse for LEMS in the U.S.

3Q 2011: Top-line results from Phase II trial for PEG-PAL, including daily dosing and formulation studies

1Q 2012: Initiation of Phase III trial for PEG-PAL

1Q 2012: Initiation of Phase I trial for BMN-111 for Achondroplasia

1H 2012: 510k approval and commercial availability of the handheld blood Phe monitor for PKU

2H 2012: Top-line results for Phase III trial for GALNS for MPS IVA

2H 2012: Top-line results for PKU-016 Kuvan neurocognitive study

2H 2012: Top-line results for Phase I/II trial for BMN-701 for Pompe disease

4Q 2012/1Q 2013: Market authorization application filings for GALNS for MPS IVA

Research and Development Programs

BioMarin continues to make significant investments in research and development to ensure persistent growth of the company. The current pipeline includes programs in various stages of development that are focused on treating a range of serious unmet medical needs.

Advanced Clinical Programs

 

 

GALNS for MPS IVA: BioMarin is enrolling patients in the randomized, double-blind, placebo-controlled Phase III study with primary endpoint of six-minute walk distance, 24 weeks in treatment duration and treatment doses of two mg/kg/week and two mg/kg/every other week. The company expects to enroll approximately 160 patients across 40 centers worldwide and to report top-line results in the second half of 2012.

 

 

Kuvan Outcomes Study: PKU-016, a randomized, placebo-controlled, 13-week Kuvan outcomes study is ongoing. Endpoints include clinically validated measures of neuropsychiatric symptoms and if successful, may enable a label amendment. The company expects to report top-line results in the second half of 2012.

 

 

Hand-Held Blood Phe Monitor: Several programs are underway to expand and protect the market and to improve the ability of healthcare providers and patients to better manage PKU. Regulatory approval and commercial availability of the handheld blood Phe monitor are expected in the first half of 2012.

 

 

Firdapse: BioMarin expects to initiate a Phase III trial for LEMS in the U.S. in the second quarter of 2011.

Mid-Stage Clinical Programs

 

 

PEG-PAL for PKU: Top-line results for the Phase II study, including a study comparing daily and weekly dosing and a formulation study are expected in the third quarter of 2011. The company expects to initiate a Phase III trial in the first quarter of 2012.

Early-Stage Clinical Programs

 

 

BMN-673 (PARP inhibitor): The company initiated a Phase I/II trial in the first quarter of 2011. The trial is an open-label study of once daily, orally administered BMN-673 in patients ages 18 and older with advanced or recurrent solid tumors. The primary objective of the study is to establish the maximum tolerated dose of daily oral BMN-673 and to obtain preliminary efficacy data in an expanded cohort of patients with genetically-defined tumors.

 

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BMN-701 for Pompe Disease: BioMarin initiated a Phase I/II trial in Pompe patients in the first quarter of 2011. The trial is an open-label study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamic and clinical activity of BMN-701 administered as an intravenous infusion every two weeks at doses of 5 mg/kg, 10 mg/kg and 20 mg/kg. The company expects to enroll up to 30 patients between the ages of 13 and 65 years old with late-onset Pompe disease for a treatment period of 24 weeks. The primary objective of the study is to establish the maximum tolerated dose of every other week administration of BMN-701 and to treat an expanded cohort of patients at the maximum tolerated dose. Top-line results are expected in the second half of 2012.

Preclinical Programs

 

 

BMN-111 for Achondroplasia: BioMarin expects to initiate a Phase I trial by the first quarter of 2012. BMN-111 is an analog of C-type Natriuretic Peptide (CNP), a small cyclic peptide that is a positive regulator of bone growth, for achondroplasia, the most common form of dwarfism. There are approximately between 18,000 and 24,000 patients in the U.S. and Europe, 25 percent of which is the estimated addressable market.

 

 

Other early stage programs: BioMarin is working on multiple early development opportunities.

Non-GAAP Financial Information and Reconciliation

The above results for the three months ended March 31, 2011 and March 31, 2010 and financial guidance for the year ending December 31, 2011 are presented both as determined in accordance with GAAP and on a non-GAAP basis. As used in this release, non-GAAP income is based on GAAP Earnings Before Interest, Taxes, Depreciation and Amortization (EBITDA) adjusted to exclude non-cash stock compensation expense, contingent consideration expense and certain nonrecurring material items (adjusted EBITDA).

The following tables detail the reconciliation of non-GAAP to GAAP financial metrics:

Reconciliation of GAAP Net Income (Loss) to Non-GAAP Adjusted EBITDA

(in millions)

(unaudited)

 

           Three Months Ended
March 31,
    Year Ending
December 31, 2011
Guidance
      NOTES     2011     2010    

GAAP Net Income (Loss)

     $ (4.4   $ 1.2      $(52) - $(42)

Interest expense, net

       1.4        1.2      6

Income tax expense

       4.8        0.6      15

Depreciation

       4.7        3.4      24

Amortization

       0.9        0.1      4
                      

EBITDA (Loss)

       7.4        6.5      (3) - 7

Stock-based compensation

       10.4        8.5      43

Gain on sale of equity investments

       —          (0.9   —  

Contingent consideration

     (1)        (0.5     0.7      11
                      

Adjusted EBITDA

     $ 17.3      $ 14.8      $51 - $61
                      

 

(1) Represents the changes in the fair value of contingent acquisition consideration payable for the period.

BioMarin believes that this non-GAAP information is useful to investors, taken in conjunction with BioMarin’s GAAP information because it provides additional information regarding the performance of BioMarin’s core ongoing business, Naglazyme, Kuvan, Aldurazyme and Firdapse and development of its pipeline. By providing information about both the overall GAAP financial performance and the non-GAAP measures that focus on continuing operations, the company

 

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believes that the additional information enhances investors’ overall understanding of the company’s business and prospects for the future. Further, the company uses both the GAAP and the non-GAAP results and expectations internally for its operating, budgeting and financial planning purposes and uses the adjusted EBITDA methodology in establishing corporate goals for internal compensation programs.

Diluted Earnings Per Share Calculation

As of March 31, 2011 and March 31, 2010, the shares related to our outstanding convertible debt are 19.1 million and 26.3 million, respectively. The calculation of non-GAAP adjusted EBITDA diluted earnings per share for the quarter ended March 31, 2011 and March 31, 2010 includes the 19.1 million shares and the 10.3 million shares, respectively, related to the company’s convertible debt, as their impact is considered dilutive.

Conference Call Details

BioMarin will host a conference call and webcast to discuss first quarter 2011 financial results today, Thursday, April 28, at 5:00 p.m. ET. This event can be accessed on the investor section of the BioMarin website at www.BMRN.com.

Date: April 28, 2011

Time: 5:00 p.m. ET

U.S. / Canada Dial-in Number: 888.396.2386

International Dial-in Number: 617.847.8712

Participant Code: 42725170

Replay Dial-in Number: 888.286.8010

Replay International Dial-in Number: 617.801.6888

Replay Code: 56138656

About BioMarin

BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company’s product portfolio comprises four approved products and multiple clinical and pre-clinical product candidates. Approved products include Naglazyme® (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin; Aldurazyme® (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; Kuvan® (sapropterin dihydrochloride) Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany; and Firdapse™ (amifampridine), which has been approved by the European Commission for the treatment of Lambert Eaton Myasthenic Syndrome (LEMS). Product candidates include GALNS (N-acetylgalactosamine 6-sulfatase), which is currently in Phase III clinical development for the treatment of MPS IVA, PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently in Phase II clinical development for the treatment of PKU, BMN-701, a novel fusion protein of insulin-like growth factor 2 and acid alpha glucosidase (IGF2-GAA), which is currently in Phase I/II clinical development for the treatment of Pompe disease, and BMN-673, a poly ADP-ribose polymerase (PARP) inhibitor, which is currently in Phase I/II clinical development for the treatment of genetically-defined cancers. For additional information, please visit www.BMRN.com. Information on BioMarin’s website is not incorporated by reference into this press release.

Forward-Looking Statement

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including, without limitation, statements about: the expectations of revenue and sales related to Naglazyme, Kuvan, Firdapse, and Aldurazyme; the financial performance of the BioMarin as a whole; the timing of BioMarin’s clinical trials of GALNS, Firdapse, PEG-PAL, BMN-673 , BMN-701 and other product candidates; the continued clinical development and commercialization of Aldurazyme, Naglazyme, Kuvan, Firdapse, and its product candidates; and actions by regulatory authorities. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: our success in the

 

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continued commercialization of Naglazyme, Kuvan, and Firdapse; Genzyme Corporation’s success in continuing the commercialization of Aldurazyme; results and timing of current and planned preclinical studies and clinical trials, particularly with respect to GALNS, Firdapse, PEG-PAL, BMN 673 and BMN 701; our ability to successfully manufacture our products and product candidates; the content and timing of decisions by the U.S. Food and Drug Administration, the European Commission and other regulatory authorities concerning each of the described products and product candidates; the market for each of these products and particularly Aldurazyme, Naglazyme, Kuvan and Firdapse; actual sales of Aldurazyme, Naglazyme Kuvan and Firdapse; Merck Serono’s activities related to Kuvan; and those factors detailed in BioMarin’s filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption “Risk Factors” in BioMarin’s 2009 Annual Report on Form 10-K, and the factors contained in BioMarin’s reports on Form 10-Q. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.

BioMarin®, Naglazyme®, Kuvan® and Firdapse™ are registered trademarks of BioMarin Pharmaceutical Inc.

Aldurazyme® is a registered trademark of BioMarin/Genzyme LLC.

 

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BIOMARIN PHARMACEUTICAL INC.

CONSOLIDATED BALANCE SHEETS

March 31, 2011 and 2010

(In thousands of U.S. dollars, except share and per share amounts)

 

     March 31,
2011
    December 31,
2010 (1)
 
     (unaudited)        
ASSETS     

Current assets:

    

Cash and cash equivalents

   $ 90,782      $ 88,079   

Short-term investments

     161,799        186,033   

Accounts receivable, net (allowance for doubtful accounts $969 and $63, respectively)

     106,198        86,576   

Inventory

     110,067        109,698   

Other current assets

     34,984        33,874   
                

Total current assets

     503,830        504,260   

Investment in BioMarin/Genzyme LLC

     1,133        1,082   

Long-term investments

     141,378        128,171   

Property, plant and equipment, net

     217,884        221,866   

Intangible assets, net

     102,692        103,648   

Goodwill

     53,055        53,364   

Long-term deferred tax assets

     231,947        236,017   

Other assets

     12,227        14,215   
                

Total assets

   $ 1,264,146      $ 1,262,623   
                

LIABILITIES AND STOCKHOLDERS’ EQUITY

    

Current liabilities:

    

Accounts payable and accrued liabilities

   $ 75,986      $ 83,844   
                

Total current liabilities

     75,986        83,844   

Convertible debt

     377,521        377,521   

Other long-term liabilities

     89,799        84,001   
                

Total liabilities

     543,306        545,366   
                

Stockholders’ equity:

    

Common stock, $0.001 par value: 250,000,000 shares authorized at March 31, 2011 and December 31, 2010 : 110,873,519 and 110,634,465 shares issued and outstanding at March 31, 2011 and December 31, 2010, respectively

     111        111   

Additional paid-in capital

     1,103,885        1,090,188   

Company common stock held by Nonqualified Deferred Compensation Plan

     (1,733     (1,965

Accumulated other comprehensive income (loss)

     (5,787     188   

Accumulated deficit

     (375,636     (371,265
                

Total stockholders’ equity

     720,840        717,257   
                

Total liabilities and stockholders’ equity

   $ 1,264,146      $ 1,262,623   
                

 

(1) December 31, 2010 balances were derived from the audited consolidated financial statements.

 

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BIOMARIN PHARMACEUTICAL INC.

CONSOLIDATED STATEMENTS OF OPERATIONS

For the Three Months Ended March 31, 2011 and 2010

(In thousands, except for per share data)

 

     2011     2010  
     (unaudited)     (unaudited)  

REVENUES:

    

Net product revenues

   $ 109,076      $ 84,073   

Collaborative agreement revenues

     125        201   

Royalty and license revenues

     255        679   
                

Total revenues

     109,456        84,953   

OPERATING EXPENSES:

    

Cost of sales (excludes amortization of developed product technology)

     20,796        17,412   

Research and development

     45,017        30,097   

Selling, general and administrative

     41,037        34,000   

Intangible asset amortization and contingent consideration

     312        654   
                

Total operating expenses

     107,162        82,163   
                
INCOME FROM OPERATIONS      2,294        2,790   

Equity in the loss of BioMarin/Genzyme LLC

     (542     (691

Interest income

     782        1,190   

Interest expense

     (2,141     (2,429

Net gain from sale of investments

     —          927   
                
INCOME BEFORE INCOME TAXES      393        1,787   

Provision for income taxes

     4,764        636   
                
NET INCOME (LOSS)    $ (4,371   $ 1,151   
                
NET INCOME (LOSS) PER SHARE, BASIC & DILUTED    $ (0.04   $ 0.01   
                

Weighted average common shares outstanding, basic

     110,652        101,144   

Weighted average common shares outstanding, diluted

     110,743        103,720   

Total stock-based compensation expense included in the Consolidated Statements of Operations is as follows:

 

     Three Months Ended
March  31,
 
     2011      2010  
     (unaudited)      (unaudited)  

Cost of sales

   $ 1,402       $ 1,028   

Research and development

     3,674         3,182   

Selling, general and administrative

     5,304         4,336   
                 
   $ 10,380       $ 8,546   
                 

###

 

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