S-4 1 t1501262-s4.htm FORM S-4 t1501262-s4 - none - 26.8206818s
As filed with the Securities and Exchange Commission on May 28, 2015
Registration No. 333-        ​
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM S-4
REGISTRATION STATEMENT
UNDER
THE SECURITIES ACT OF 1933
Alexion Pharmaceuticals, Inc.
(Exact name of registrant as specified in its charter)
Delaware
13-3648318
(State or other jurisdiction of incorporation or organization)
(I.R.S. Employer Identification Number)
352 Knotter Drive,
Cheshire, Connecticut 06410
(203) 272-2596
(Address, Including Zip Code, and Telephone Number, Including Area Code, of Registrant’s Principal Executive Offices)
David Hallal
Chief Executive Officer
Alexion Pharmaceuticals, Inc.
352 Knotter Drive
Cheshire, Connecticut 06410
(203) 272-2596
(Address, Including Zip Code, and Telephone Number, Including Area Code, of Agent for Service)
Copies to:
John B. Moriarty, Jr.
Executive Vice President,
General Counsel
Alexion Pharmaceuticals, Inc.
352 Knotter Drive
Cheshire, Connecticut 06410
(203) 272-2596
Daniel A. Neff
Mark Gordon
Wachtell, Lipton,
Rosen & Katz
51 West 52nd Street
New York, New York 10019
(212) 403-1000
Thomas Beetham
Senior Vice President,
General Counsel &
Chief Legal Officer
Synageva BioPharma Corp.
33 Hayden Avenue
Lexington, Massachusetts 02421
(781) 357-9900
Matthew G. Hurd
Krishna Veeraraghavan
Sullivan & Cromwell LLP
125 Broad Street
New York, New York 10004
(212) 558-4000
Paul M. Kinsella
Ropes & Gray LLP
800 Boylston Street
Boston, Massachusetts 02199
(617) 951-7000
Approximate date of commencement of proposed sale of the securities to the public: As soon as practicable after the effectiveness of this registration statement and the satisfaction or waiver of the conditions to the transactions described herein.
If the securities being registered on this Form are being offered in connection with the formation of a holding company and there is compliance with General Instruction G, check the following box:
If this Form is filed to register additional securities for an offering pursuant to Rule 462(b) under the Securities Act, check the following box and list the Securities Act registration statement number of the earlier effective registration statement for the same offering.
If this Form is a post-effective amendment filed pursuant to Rule 462(d) under the Securities Act, check the following box and list the Securities Act registration statement number of the earlier effective registration statement for the same offering.
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 the definitions of  “large accelerated filer,” “accelerated filer” and “smaller reporting company” in Rule 12b-2 of the Exchange Act. (Check one):
Large accelerated filer Accelerated filer
Non-accelerated filer
   (Do not check if a smaller reporting company)
Smaller reporting company
If applicable, place an ☒ in the box to designate the appropriate rule provision relied upon in conducting this transaction:
Exchange Act Rule 13e-4(i) (Cross-Border Issuer Tender Offer)    Exchange Act Rule 14d-1(d) (Cross-Border Third-Party Tender Offer)   
CALCULATION OF REGISTRATION FEE
Title of each class of
securities to be registered
Amount to be
registered
Proposed maximum offering
price per share
Proposed maximum
aggregate offering price
Amount of
registration fee
Common stock, par value $0.0001 per share
27,425,229 shares(1)
N/A
$ 4,140,246,925.10(2) $ 481,096.69(3)
(1)
Represents the maximum number of shares of Alexion Pharmaceuticals, Inc. (“Alexion”) common stock estimated to be issuable upon consummation of the transactions, calculated by multiplying the exchange ratio of 0.6581 by 41,673,346 shares of common stock of Synageva BioPharma Corp. (“Synageva”), which is the sum of 37,225,329, the number of shares of Synageva common stock outstanding as of May 15, 2015, plus 4,250,191, the number of shares of Synageva common stock reserved for issuance under existing Synageva equity plans, plus 197,826, the number of shares of Synageva common stock that would be entitled to receive the transaction consideration if Synageva made a grant of restricted stock units with the maximum number of shares of Synageva common stock underlying such restricted stock units as permitted under the transaction agreement, and then rounding up. In accordance with Rule 416, this registration statement also covers an indeterminate number of additional shares of Alexion securities as may be issuable as a result of stock splits, stock dividends or similar transactions.
(2)
Estimated solely for the purpose of calculating the registration fee pursuant to Rule 457 under the Securities Act on the basis of the market value of the shares of Synageva common stock to be exchanged in the transactions, computed in accordance with Rule 457(f)(1) and Rule 457(f)(3) based on (a) the product of  (i) $214.35, the average of the high and low sales prices per share of Synageva common stock on May 21, 2015, as reported by Nasdaq, and (ii) 41,673,346, the estimated number of shares of Synageva common stock to be exchanged in the transactions for the transaction consideration, less (b) the product of  (x) $115.00, the per-share cash consideration that will be paid by Alexion to Synageva stockholders in the transactions, and (y) 41,673,346, the estimated number of shares of Synageva common stock to be exchanged in the transactions for the transaction consideration.
(3)
The amount of the filing fee, calculated in accordance with Rule 457(c) and Rule 457(f) under the Securities Act, equals 0.00011620 multiplied by the proposed maximum offering price. Pursuant to Rule 457(p), $473,736.18 of this registration fee is offset by the registration fee of  $473,736.18 paid in connection with the Registration Statement on Form S-4 initially filed by Alexion on May 22, 2015.
The registrant hereby amends this registration statement on such date or dates as may be necessary to delay its effective date until the registrant shall file a further amendment which specifically states that this registration statement shall thereafter become effective in accordance with Section 8(a) of the Securities Act of 1933 or until the registration statement shall become effective on such date as the Securities and Exchange Commission, acting pursuant to said Section 8(a), may determine.

The information in this document is not complete and may change. The registrant may not complete the transactions and issue these securities until the registration statement filed with the U.S. Securities and Exchange Commission is effective. This document is not an offer to sell these securities and the registrant is not soliciting an offer to buy these securities in any state or jurisdiction in which such offer is not permitted.
PRELIMINARY AND SUBJECT TO CHANGE, DATED [•] [•], 2015
Explanatory Note: The purpose of the transactions agreed to between Alexion Pharmaceuticals, Inc. and Synageva BioPharma Corp. is for Alexion to acquire all of the outstanding equity of Synageva. A wholly owned subsidiary of Alexion previously commenced an exchange offer to achieve such purpose, including by filing a Registration Statement on Form S-4 in connection with the exchange offer (the “Exchange Offer S-4”). However, the transaction agreement between the parties also gives Alexion the right under certain circumstances to terminate the exchange offer and effect the acquisition through a “long-form” merger. This document relates to that alternative long-form merger approach and will be used only in the event that the exchange offer is terminated. The filing of this document does not impact the exchange offer nor does this document replace or affect the Exchange Offer S-4.
[MISSING IMAGE: lg_synageva-lowres.jpg]
[•][•], 2015​
Dear Stockholder:
As previously announced, on May 5, 2015, Synageva BioPharma Corp. (“Synageva”) entered into an agreement and plan of reorganization (the “transaction agreement”) with Alexion Pharmaceuticals, Inc. (“Alexion”), Pulsar Merger Sub Inc., a direct wholly owned subsidiary of Alexion (the “Offeror”), and Galaxy Merger Sub LLC, a direct wholly owned subsidiary of Alexion (“Merger Sub”). Under the transaction agreement, the Offeror will merge with and into Synageva, with Synageva continuing as the interim surviving corporation (the “first merger”). Immediately thereafter, the corporation surviving the first merger will merge with and into Merger Sub, with Merger Sub continuing as the final surviving company and a direct wholly owned subsidiary of Alexion (the “second merger” and, together with the first merger, the “mergers”). We believe that the proposed transactions bring together two companies with complementary product offerings and product candidates for patients with devastating and rare diseases.
If the transaction agreement is adopted by Synageva stockholders and the first merger is completed, for each share of Synageva common stock that you hold (other than those shares for which appraisal rights are validly exercised or those shares owned by Synageva, Alexion or their respective subsidiaries), you will be entitled to receive:

$115.00 in cash; and

0.6581 shares of Alexion common stock with cash in lieu of any fractional shares of Alexion common stock;
in each case, without interest, but subject to any required withholding taxes (such consideration, the “transaction consideration”).
Synageva common stock is listed on the NASDAQ Global Select Market under the symbol “GEVA.” Alexion common stock is listed on the NASDAQ Global Select Market under the symbol “ALXN.” On [•] [•], 2015, the last practicable trading day prior to the date of this proxy statement/prospectus, the last reported sale price per share of Alexion common stock on the NASDAQ Global Select Market was $[•].
The first merger cannot be completed unless Synageva stockholders holding a majority of the outstanding shares of Synageva common stock as of the close of business on [•] [•], 2015 vote in favor of the adoption of the transaction agreement at the special meeting of Synageva stockholders to be held on [•] [•], 2015, referred to as the special meeting. Your vote is very important, regardless of the number of shares of Synageva common stock you own. Whether or not you expect to attend the special meeting in person, please vote or otherwise submit a proxy to vote your shares as promptly as possible so that your shares may be represented and voted at the special meeting.

In addition, at the special meeting you also will be asked to approve the adjournment of the special meeting under certain circumstances and to approve, on an advisory (non-binding) basis, the “golden parachute” compensation payments that will or may be paid by Synageva to its named executive officers in connection with the mergers.
THE SYNAGEVA BOARD OF DIRECTORS HAS UNANIMOUSLY DETERMINED THAT THE TERMS OF THE TRANSACTION AGREEMENT AND THE TRANSACTIONS CONTEMPLATED BY THE TRANSACTION AGREEMENT, INCLUDING THE FIRST MERGER, ARE FAIR TO, AND IN THE BEST INTERESTS OF, SYNAGEVA AND ITS STOCKHOLDERS, DETERMINED THAT IT IS THE BEST INTERESTS OF SYNAGEVA AND ITS STOCKHOLDERS TO ENTER INTO, AND DECLARED ADVISABLE, THE TRANSACTION AGREEMENT, AND APPROVED THE EXECUTION AND DELIVERY BY SYNAGEVA OF THE TRANSACTION AGREEMENT. THE SYNAGEVA BOARD OF DIRECTORS UNANIMOUSLY RECOMMENDS THAT YOU VOTE “FOR” THE ADOPTION OF THE TRANSACTION AGREEMENT, “FOR” THE ADVISORY “GOLDEN PARACHUTE” COMPENSATION PROPOSAL AND “FOR” THE ADJOURNMENT OF THE SPECIAL MEETING, IF NECESSARY, TO SOLICIT ADDITIONAL PROXIES.
The obligations of Synageva and Alexion to complete the mergers are subject to the satisfaction or waiver of the conditions set forth in the transaction agreement. See “Transaction Agreement — Conditions to the Transactions.”
The first merger will entitle Synageva stockholders to appraisal rights under the General Corporation Law of the State of Delaware (the “DGCL”). To exercise appraisal rights, a Synageva stockholder must strictly comply with all of the procedures under the DGCL. These procedures are described more fully in the section entitled “The Transactions — Dissenters’ Rights.”
Additional information about Synageva, Alexion and the mergers is contained in the accompanying proxy statement/prospectus. For a discussion of risk factors that you should consider in evaluating the transactions, see “Risk Factors” beginning on page 20 of the accompanying proxy statement/prospectus. The market price of Alexion common stock will continue to fluctuate following the date of the special meeting. Consequently, at the time of the special meeting, the market value of the stock portion of the transaction consideration will not yet be determined. We urge you to read the accompanying proxy statement/prospectus carefully and in its entirety.
Thank you for your consideration and continued support.
Sincerely,
Sanj K. Patel
President and Chief Executive Officer
Neither the Securities and Exchange Commission nor any state securities commission has approved or disapproved of the securities to be issued under the proxy statement/prospectus or determined that the proxy statement/prospectus is accurate or complete. Any representation to the contrary is a criminal offense.
This document is dated [•] [•], 2015, and is first being mailed to stockholders of Synageva on or about [•] [•], 2015.

[MISSING IMAGE: lg_synageva-lowres.jpg]
Lexington, Massachusetts
NOTICE OF SPECIAL MEETING OF STOCKHOLDERS
To Be Held On [•] [•], 2015
MERGER PROPOSAL — YOUR VOTE IS VERY IMPORTANT
Dear Stockholder,
You are cordially invited to attend a special meeting of stockholders of Synageva BioPharma Corp., a Delaware corporation, to be held on [•] [•], 2015, at [•], local time, at [•]. The purpose of the meeting will be to consider and vote upon the following matters:
(1)
to adopt the agreement and plan of reorganization, dated as of May 5, 2015, among Synageva, Alexion Pharmaceuticals, Inc. (“Alexion”), Pulsar Merger Sub Inc. and Galaxy Merger Sub LLC, a copy of which is attached as Annex A to the proxy statement/​prospectus accompanying this notice (the “transaction agreement”), and approve the transactions contemplated by the transaction agreement, including the merger of Pulsar Merger Sub Inc. with and into Synageva, with Synageva as the surviving corporation and a direct, wholly owned subsidiary of Alexion;
(2)
to approve, on an advisory (non-binding) basis, specified compensatory arrangements between Synageva and its named executive officers relating to the proposed transactions with Alexion, as described in the accompanying proxy statement/prospectus; and
(3)
to approve any motion to adjourn the special meeting, or any adjournments or postponements thereof, to another time or place if necessary or appropriate to solicit additional proxies if there are insufficient votes at the time of the special meeting to adopt the transaction agreement and approve the transactions contemplated by the transaction agreement.
THE SYNAGEVA BOARD OF DIRECTORS UNANIMOUSLY RECOMMENDS THAT SYNAGEVA STOCKHOLDERS VOTE “FOR” EACH OF THE PROPOSALS.
The above matters, the transaction agreement and the proposed merger are described in more detail in the accompanying proxy statement/prospectus. Please read the proxy statement/prospectus carefully and in its entirety in deciding how to vote.
The record date for the Synageva special meeting is [•] [•], 2015. Only holders of record of shares of Synageva common stock at the close of business on the record date are entitled to notice of, and to vote at, the Synageva special meeting, or any adjournment or postponement thereof. A list of stockholders entitled to vote at the special meeting will be available at [•], during regular business hours for a period not less than 10 days before the special meeting, as well as during the special meeting.
Adoption of the transaction agreement (including the transactions contemplated by the transaction agreement) by Synageva stockholders is a condition to completion of the merger and requires the affirmative vote, in person or by proxy, of holders of a majority of the outstanding shares of Synageva common stock entitled to vote thereon. Therefore, your vote is very important. Your failure to vote your shares will have the same effect as a vote “against” the adoption of the transaction agreement (including the transactions contemplated by the transaction agreement). Whether or not you plan to attend the special meeting, please promptly vote your shares of Synageva common stock by calling the toll-free number found on your proxy card, by accessing the internet site found on your proxy card or by marking, dating, signing and returning all proxy cards you receive. By providing your proxy, you do not restrict your right to vote in person at the Synageva special meeting. If your Synageva shares are held in the name of a broker, dealer, commercial bank, trust company or other nominee, please follow the instructions on the voting instruction form(s) furnished by such nominee that is the record holder.

Do not send any Synageva stock certificates at this time. If the merger is completed, you will be notified of the procedures for exchanging your stock certificates for the transaction consideration.
By Order of the Board of Directors,
Sanj K. Patel
President and Chief Executive Officer
Lexington, Massachusetts
[•] [•], 2015

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EXPLANATORY NOTE
Pursuant to the terms and subject to the conditions set forth in the Agreement and Plan of Reorganization (the “transaction agreement”), entered into by Alexion Pharmaceuticals, Inc. (“Alexion”), Pulsar Merger Sub Inc. (the “Offeror”), Galaxy Merger Sub LLC (“Merger Sub”) and Synageva BioPharma Corp. (“Synageva”) on May 5, 2015, as agreed between Alexion and Synageva, Alexion proposes to acquire control of, and ultimately all of the outstanding equity in, Synageva.
The transaction agreement contemplates that Alexion will commence an exchange offer as the first step in such plan to acquire all of the outstanding shares of Synageva and that, if the exchange offer is completed (such that Alexion will own at least a majority of the outstanding shares of Synageva common stock), Alexion will acquire the remaining shares of outstanding Synageva common stock through the merger of the Offeror with and into Synageva (the “first merger”), with Synageva surviving the merger as a direct wholly owned subsidiary of Alexion. If the offer is completed (such that Alexion will own at least a majority of the outstanding shares of Synageva common stock), the first merger will be governed by Section 251(h) of the General Corporation Law of the State of Delaware (the “DGCL”), and accordingly no stockholder vote will be required to consummate the first merger.
Alternatively, Alexion has the right under certain circumstances as set forth in the transaction agreement, to terminate the exchange offer and seek to instead effect the first merger through a “long-form” merger governed by Section 251(c) of the DGCL, and accordingly a vote of Synageva’s stockholders will be required to consummate the first merger. If the offer is terminated and the parties instead propose to effect the transactions through a long-form merger, Synageva will convene a meeting of Synageva stockholders to seek their approval of the transaction agreement.
The proxy statement/prospectus forming a part of this registration statement relates to the completion of the transactions pursuant to a long-form merger subject to the vote of Synageva stockholders and will not be used unless and until the exchange offer is terminated under the circumstances described in the transaction agreement and the parties seek to complete the transactions through a long-form merger. In such circumstances, the proxy statement/prospectus forming a part of this registration statement will be mailed to Synageva stockholders and will be used to solicit votes of Synageva stockholders in favor of adoption of the transaction agreement. The proxy statement/prospectus forming a part of this registration statement is not being and will not be used with respect to the exchange offer.
v

ADDITIONAL INFORMATION
As permitted by the SEC, this document incorporates by reference important business and financial information about Alexion, Synageva and their respective subsidiaries from documents filed with the SEC that have not been included in or delivered with this document.
This information is available without charge at the SEC’s website at www.sec.gov, as well as from other sources.
You can obtain the documents incorporated by reference in this document, without charge, by requesting them in writing or by telephone at the following address and telephone number.
Investor Relations
Alexion Pharmaceuticals, Inc.
352 Knotter Drive
Cheshire, Connecticut 06410
(203) 272-2596
http://www.alxn.com
If you would like to request documents, in order to receive timely delivery prior to the date of the Synageva special meeting, please make your request at least five business days prior to the date of the Synageva special meeting. Unless the special meeting is adjourned or postponed, this means that the latest you should request documents is [•] [•], 2015.
See also “Where To Obtain Additional Information.”
Synageva has supplied all information contained or incorporated by reference in this document relating to Synageva, and Alexion has supplied all information contained or incorporated by reference in this document relating to Alexion. Both Synageva and Alexion have both contributed information relating to the transactions.
vi

QUESTIONS AND ANSWERS ABOUT THE SYNAGEVA SPECIAL MEETING
Below are some of the questions that you as a holder of shares of Synageva common stock may have regarding the Synageva special meeting of stockholders and answers to those questions. You are urged to carefully read the remainder of this document, the annexes to this document and the other information referred to or incorporated by reference in this document because the information contained in this section and in the “Summary” section is not complete. See “Where To Obtain Additional Information.”
As used in this document, unless otherwise indicated or the context requires: “Alexion” (or “we,” “us” and “our”) refers to Alexion Pharmaceuticals, Inc., a Delaware corporation, and its consolidated subsidiaries; the “Offeror” refers to Pulsar Merger Sub Inc., a Delaware corporation and direct wholly owned subsidiary of Alexion; “Merger Sub” refers to Galaxy Merger Sub LLC, a Delaware limited liability company and direct wholly owned subsidiary of Alexion; and “Synageva” refers to Synageva BioPharma Corp., a Delaware corporation, and its consolidated subsidiaries.
Why have I received these materials?
You are receiving this document as a stockholder of Synageva BioPharma Corp. (“Synageva”). Synageva has agreed to be acquired by Alexion Pharmaceuticals, Inc. (“Alexion”) pursuant to the terms and subject to the conditions set forth in the Agreement and Plan of Reorganization (the “transaction agreement”), dated as of May 5, 2015, among Synageva, Alexion, Pulsar Merger Sub Inc., a direct wholly owned subsidiary of Alexion (the “Offeror”) and Galaxy Merger Sub LLC, a direct wholly owned subsidiary of Alexion (“Merger Sub”).
Pursuant to the transaction agreement and subject to the satisfaction or waiver of the conditions to the transactions, the Offeror will be merged with and into Synageva (the “first merger”), with Synageva surviving the merger as a direct wholly owned subsidiary of Alexion. At the effective time of the first merger, each outstanding share of Synageva common stock (other than certain cancelled, converted and dissenting shares, as described elsewhere in this document) will be automatically converted into the right to receive $115.00 in cash and 0.6581 shares of Alexion common stock, with cash in lieu of any fractional shares of Alexion common stock and in each case, without interest and subject to any applicable withholding taxes. Immediately following the first merger, the corporation surviving the first merger will merge with and into Merger Sub (the “second merger” and, together with the first merger, the “mergers”), with Merger Sub surviving the second merger as a direct wholly owned subsidiary of Alexion.
This document serves as the proxy statement through which Synageva will solicit proxies to obtain the approval of Synageva stockholders to adopt the transaction agreement and approve the transactions contemplated by the transaction agreement, including the first merger. This document also serves as the prospectus by which Alexion will issue shares of its common stock as part of the transaction consideration in the first merger. This proxy statement/prospectus contains important information, and you should read it carefully and in its entirety.
Who is Alexion?
As a result of the first merger, the Synageva business will be held in a direct wholly owned subsidiary of Alexion. Alexion is a biopharmaceutical company focused on serving patients with severe and rare disorders through the innovation, development and commercialization of life-transforming therapeutic products. Alexion is the global leader in complement inhibition and has developed and markets Soliris® (eculizumab) (“Soliris”) as a treatment for patients with paroxysmal nocturnal hemoglobinuria (“PNH”) and atypical hemolytic uremic syndrome (“aHUS”), two debilitating, rare and life-threatening disorders caused by chronic uncontrolled activation of the complement component of the immune system. Soliris is currently approved in nearly 50 countries for the treatment of PNH, and in nearly 40 countries for the treatment of aHUS. Alexion is evaluating other potential indications for Soliris in additional severe and devastating diseases beyond PNH and aHUS in which uncontrolled complement activation is the underlying mechanism, and is progressing in various stages of development with additional product candidates as potential treatments for patients with severe and life-threatening ultra-rare disorders. In 2014, Alexion filed for regulatory approval with the U.S. Food and Drug Administration (“FDA”), European Medicines Agency (“EMA”) and the Japanese Ministry of Health, Labor and Welfare (“MHLW”) for
1

StrensiqTM (asfotase alfa) (“Strensiq”), a targeted enzyme replacement therapy in Phase II clinical trials for patients with hypophosphatasia (“HPP”) an ultra-rare, genetic, and life-threatening metabolic disease characterized by impaired phosphate and calcium regulation, leading to progressive damage to multiple vital organs including destruction and deformity of bones, profound muscle weakness, seizures, impaired renal function, and respiratory failure. In July 2014, the EMA validated Alexion’s Marketing Authorization Application (“MAA”) for Strensiq for the treatment of HPP. In March 2015, the FDA accepted for Priority Review the Biologics License Application (“BLA”) for Strensiq for treatment of patients with infantile- and juvenile-onset HPP. Alexion has approximately 2,400 employees and serves patients in 50 countries.
Why is Alexion proposing the mergers?
The board of directors of Alexion unanimously determined that the terms of the transaction agreement and the transactions contemplated by the transaction agreement, including the first merger, are fair to, and in the best interests of, Alexion and its stockholders. See “The Transactions — Alexion’s Reasons for the Transactions” for more information.
What matters are being voted on at the Synageva special meeting?
At the Synageva special meeting, Synageva stockholders will be asked to vote upon the following proposals:

Proposal No. 1 — Adoption of the Transaction Agreement and Approval of the Transactions Contemplated by the Transaction Agreement. Adopt the transaction agreement and approve the transactions contemplated by the transaction agreement, including the first merger;

Proposal No. 2 — Advisory Vote on Specified Compensatory Arrangements. Approve, on a non-binding advisory basis, specified compensatory arrangements between Synageva and its named executive officers that are based on or otherwise relate to the mergers as described in the section of this document entitled “The Transactions — Interests of Certain Persons in the Transactions — Golden Parachute Compensation”, approval of which is not a condition to completion of the mergers; and

Proposal No. 3 — Approval of Possible Adjournment of the Synageva Special Meeting. Approve any motion to adjourn the special meeting, or any adjournments or postponements thereof, to another time or place if necessary or appropriate to solicit additional proxies if there are insufficient votes at the time of the special meeting to adopt the transaction agreement and approve the transactions contemplated by the transaction agreement.
Does the Synageva board of directors support the transactions and recommend a vote in favor of the proposal to adopt the transaction agreement and the other proposals?
Yes. The Synageva board of directors unanimously determined that the terms of the transaction agreement and the transactions contemplated by the transaction agreement, including the first merger, are fair to, and in the best interests of, Synageva and its stockholders, determined that it is in the best interests of Synageva and its stockholders to enter into, and declared advisable, the transaction agreement and approved the execution and delivery by Synageva of the transaction agreement, the performance by Synageva of its covenants and agreements contained in the transaction agreement and the consummation of the mergers and the other transactions contemplated by the transaction agreement on the terms and subject to the conditions contained in the transaction agreement. The Synageva board of directors unanimously recommends that Synageva stockholders vote “FOR” the proposals to adopt the transaction agreement, to approve on a non-binding advisory basis, specified compensatory arrangements between Synageva and its named executive officers that are based on or otherwise relate to the mergers and to adjourn the special meeting, if necessary or appropriate, to solicit additional proxies if there are not sufficient votes in favor of the first proposal. See “The Transactions — Synageva’s Reasons for the Transactions; Recommendation of Synageva’s Board of Directors.”
Does Synageva’s largest shareholder support the transactions and plan to vote in favor of the proposals?
Yes. Concurrently with the execution of the transaction agreement, certain affiliates of Baker Bros. Advisors L.P., an entity controlled by Felix J. Baker, a director of Synageva (collectively, “Baker Brothers”),
2

and Thomas J. Tisch, a director of Synageva, entered into voting and support agreements with Alexion and the Offeror, pursuant to which, among other things and subject to the terms and conditions of such voting and support agreements, such stockholders agreed to vote all shares of Synageva common stock beneficially owned by them in favor of the adoption of the transaction agreement and the approval of the transactions contemplated by the transaction agreement, and any other matter necessary to consummate such transactions, and not to vote in favor of, or tender their shares into, any competing offer or takeover proposal. The Baker Brothers and Mr. Tisch together own approximately 33.36% of Synageva’s outstanding common stock. See “Voting and Support Agreements.” It is also expected that Synageva’s other directors and executive officers who are stockholders of Synageva will vote “FOR” each of the proposals, although none of them has entered into any agreement requiring them to do so, except as described above.
What will I receive for my shares of Synageva common stock if the first merger is completed?
If the first merger is completed, at the effective time of the first merger, each share of Synageva common stock, par value $0.001 per share, that is outstanding immediately prior to the effective time of the first merger, unless appraisal rights under Delaware law for such shares are properly exercised and other than shares held in treasury by Synageva or shares held by Alexion, any subsidiary of Alexion or any subsidiary of Synageva, will be converted in the first merger into the right to receive:

$115.00 in cash, without interest and less any applicable withholding taxes (the “cash consideration”); and

0.6581 shares of Alexion common stock, par value $0.0001 per share, together with cash in lieu of any fractional shares of Alexion common stock, without interest and less any applicable withholding taxes (the “stock consideration”).
We refer to the cash consideration and stock consideration above collectively as the “transaction consideration.”
When and how will I receive the transaction consideration in exchange for my shares of Synageva common stock?
Synageva stockholders of record will be sent a letter of transmittal requesting their stock certificates and containing instructions for exchanging shares of Synageva common stock from the exchange agent for the first merger shortly after the effective time of the first merger. See “Transaction Agreement —  Transaction Consideration — Exchange of Synageva Stock Certificates for the Transaction Consideration.” Synageva stockholders should not send in their common stock certificates with their proxy cards.
What vote is required to approve the proposals?
The following votes are required to approve the proposals at the Synageva special meeting:

Proposal No. 1 — Adoption of the Transaction Agreement and Approval of the Transactions Contemplated by the Transaction Agreement. Provided a quorum of stockholders is present in person or by proxy at the special meeting, in order to adopt the transaction agreement, holders of a majority of the outstanding shares of Synageva common stock must cast a vote in favor of the proposal.
Approval of this proposal is a condition to the completion of the transactions. See also “Transaction Agreement — Conditions to the Transactions.” A failure to vote, an abstention or a failure to instruct your broker, dealer, commercial bank, trust company or other nominee on how to vote your shares with respect to this proposal will have the same effect as a vote “AGAINST” this proposal.

Proposal No. 2 — Advisory Vote on Specified Compensatory Arrangements. Provided a quorum of stockholders is present in person or by proxy at the special meeting, in order to approve the specified compensatory arrangements between Synageva and its named executive officers that are
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based on or otherwise relate to the mergers, holders of a majority of the shares of Synageva common stock present in person or represented by proxy at the special meeting and entitled to vote must cast a vote in favor of the proposal. Such approval is not a condition to completion of the mergers.

Proposal No. 3 — Approval of Possible Adjournment of the Synageva Special Meeting. If there are not sufficient votes to adopt the transaction agreement at the time of the special meeting or any adjournments or postponements thereof, a majority of the votes present in person or by proxy (whether or not a quorum is present) may adjourn the special meeting to another time and place in order to solicit additional proxies.
What are the conditions to completion of the transactions?
The transactions are conditioned upon, among other things, the following:

Synageva Stockholder Approval — the transaction agreement having been adopted (and the transactions contemplated by the transaction agreement having been approved) by holders of at least a majority of the outstanding shares of Synageva common stock entitled to vote thereon;

Regulatory Approval — any waiting period (and extensions thereof) applicable to the offer and the mergers under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, as amended (the “HSR Act”), having expired or been terminated;

Effectiveness of Form S-4 — the registration statement on Form S-4 of which this document is a part having been declared effective by the U.S. Securities and Exchange Commission (the “SEC”) under the U.S. Securities Act of 1933, as amended (the “Securities Act”), and no stop order having been issued or proceeding seeking a stop order having been initiated or threatened by the SEC;

Listing of Alexion Common Stock — the shares of Alexion common stock to be issued in the first merger having been approved for listing on the NASDAQ Global Select Market (“Nasdaq”), subject to official notice of issuance;

Accuracy of Representations — the representations and warranties of each party contained in the transaction agreement being true and correct as of May 5, 2015 and the closing date, subject to specified materiality standards;

Compliance with Covenants — each party having complied in all material respects with its covenants under the transaction agreement;

No Legal Prohibition — there being no injunction by any court or other tribunal of competent jurisdiction or law that has been adopted and is effective that, in each case, prohibits or makes illegal the consummation of the mergers; and

Tax Opinions — the receipt of written opinions by Alexion and Synageva from their respective legal counsel, dated as of the closing date, to the effect that the mergers, taken together, will qualify as a “reorganization” within the meaning of Section 368(a) of the Internal Revenue Code of 1986, as amended (the “Code”).
The transactions are subject to certain other conditions set forth in the section entitled “The Transaction Agreement — Conditions to the Transactions.”
Alexion’s obligation to consummate the mergers are not conditioned upon any financing arrangements or contingencies (although the availability of the debt financing contemplated by the commitment letter (described elsewhere in this document) is subject to the satisfaction of the conditions set forth in the commitment letter). See “The Transaction — Source and Amount of Funds.”
How long will it take to complete the proposed transactions?
The transactions are currently expected to be completed in mid-2015, subject to the satisfaction or waiver of the conditions described in “Transaction Agreement — Conditions to the Transactions.”
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If the first merger is completed, will Synageva continue as a public company?
No. If the first merger is completed, Synageva will no longer be publicly traded, and the Synageva business will be held in a wholly owned subsidiary of Alexion.
When and where is the Synageva special meeting being held?
The Synageva special meeting is being held on [•] [•], 2015, at [•] local time, at [•].
Who can vote at the Synageva special meeting?
Only stockholders listed on Synageva’s records at the close of business on [•] [•], 2015, the record date for the special meeting, are entitled to receive notice of and to vote at the special meeting, or any adjournments or postponements of the special meeting.
If your shares are registered directly in your name with Synageva’s transfer agent, you are considered, with respect to those shares, the “stockholder of record.” You are entitled to vote any such shares you held of record as of the close of business on the record date.
If you own your shares of Synageva common stock through a broker, dealer, commercial bank, trust company or other nominee, you are considered the beneficial owner of shares held in “street name,” and your broker, dealer, commercial bank, trust company or other nominee who is considered, with respect to those shares, the stockholder of record. As the beneficial owner of shares held in street name, you have the right to direct your broker, dealer, commercial bank, trust company or other nominee how to vote any shares that it held of record as of the close of business on the record date on your behalf.
How many shares of Synageva common stock were outstanding as of the close of business on the record date?
As of the close of business on the record date, there were [•] shares of Synageva common stock outstanding and entitled to vote at the special meeting.
What constitutes a quorum for the Synageva special meeting?
In order for business to be conducted at the Synageva special meeting, a quorum must be present. The stockholders present, in person or by proxy, holding a majority of the outstanding shares of Synageva common stock entitled to vote as of the close of business on the record date will constitute a quorum for the transaction of business at the Synageva special meeting.
How do I vote my shares of Synageva common stock if I am a stockholder of record?
If you are a Synageva stockholder of record, you may submit a proxy to tell the persons named as proxy holders how to vote your shares. If you properly complete, sign, date and return the proxy card enclosed with this document, your shares will be voted in accordance with your instructions. Synageva stockholders may also submit a proxy over the internet at [•] or by telephone toll free at [•] by close of business on the day immediately preceding the Synageva special meeting. More detailed voting instructions are printed on the proxy card you received. Any such method of submitting a proxy will enable your shares to be represented and voted at the special meeting.
If you submit a proxy, the named proxy holders will vote all shares at the meeting for which your proxy has been properly submitted and not revoked. If you sign and return your proxy card but do not mark your card to tell the proxy holders how to vote with respect to any particular proposal, your shares will be voted “FOR” such proposal.
Synageva stockholders may also vote in person at the Synageva special meeting, although attending the special meeting will not in and of itself constitute a vote or serve to revoke a properly submitted proxy. Synageva recommends that you submit your proxy even if you plan to attend the Synageva special meeting. If you vote by proxy, you may change your vote, among other ways, if you attend and vote at the Synageva special meeting.
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How do I vote my shares of Synageva common stock if I hold my shares in street name?
If your shares of Synageva common stock are held in an account through a broker, dealer, commercial bank, trust company or other nominee, you must instruct the broker, dealer, commercial bank, trust company or other nominee how to vote your shares by following the instructions that broker, dealer, commercial bank, trust company or other nominee provides you along with this document. Your broker, dealer, commercial bank, trust company or other nominee may have an earlier deadline by which you must provide instructions to it as to how to vote your shares, so you should read carefully the materials provided to you by your broker, dealer, commercial bank, trust company or other nominee. If you do not receive materials from your broker, dealer, commercial bank, trust company or other nominee, you should contact your broker, dealer, commercial bank, trust company or other nominee to seek such materials to ensure that your Synageva shares are voted at the special meeting.
If you do not provide voting instructions to your broker, dealer, commercial bank, trust company or other nominee, it will nevertheless be entitled to vote your shares on “discretionary” items but will not be permitted to do so on “non-discretionary” items. None of the proposals at the Synageva special meeting are discretionary matters. As such, without your instructions, nominees do not have discretionary authority to vote on any of the proposals to be voted on at the Synageva special meeting.
Synageva stockholders who hold their shares in street name may also vote in person at the Synageva special meeting, provided that they bring a copy of a brokerage or bank statement to the Synageva special meeting reflecting their stock ownership as of the close of business on the record date, although attending the special meeting will not in and of itself constitute a vote or serve to revoke a properly submitted proxy provided on a Synageva stockholder’s behalf. Synageva recommends that you instruct your broker, dealer, commercial bank, trust company or other nominee how to vote your shares even if you plan to attend the Synageva special meeting.
If I plan on attending the special meeting in person, should I still submit a proxy?
Yes. Whether or not you plan to attend the special meeting, you should submit a proxy. Even if you submit a proxy, you may change your vote by voting in person by ballot at the special meeting. Attendance at the special meeting will not, in and of itself, serve to revoke your proxy.
What if I receive more than one set of proxy cards or more than one email asking me to vote?
If you receive more than one set of proxy cards or more than one e-mail instructing you to vote, it means your shares are registered in more than one name or are registered in different accounts. Please complete, date, sign and return each proxy card (or otherwise submit a proxy by internet or telephone) or respond to each e-mail, to ensure that all your shares are voted.
Can I change my vote after I have submitted a proxy?
Yes. You may revoke your proxy and change your vote at any time before it is voted at the special meeting.
If you are a stockholder of record, you may revoke your proxy by:

voting again by telephone or on the internet, because only your latest telephone or internet vote will be counted;

properly completing, signing, dating and returning another proxy card with a later date;

voting in person at the special meeting; or

giving written notice of such revocation to Synageva’s Secretary prior to or at the special meeting.
If you hold your shares beneficially in “street name,” you may revoke your proxy by:

following the instructions of your broker, dealer, commercial bank, trust company or other nominee regarding the revocation of proxies (note that your broker, dealer, commercial bank, trust company or other nominee may have an earlier deadline with respect to instructing it with respect to the revocation of proxies); or
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voting in person at the special meeting, provided that you bring a copy of a brokerage or bank statement to the Synageva special meeting reflecting your stock ownership as of the close of business on the record date.
Will I have the right to have my shares of Synageva common stock appraised?
If the first merger is completed, holders of shares of Synageva common stock will be entitled to exercise appraisal rights in connection with the first merger if they did not vote in favor of the transaction agreement and first merger at the Synageva special meeting and satisfy the other requirements prescribed by Delaware law.
Synageva stockholders who comply with the applicable statutory procedures under the General Corporation Law of the State of Delaware (the “DGCL”) will be entitled to receive a judicial determination of the fair value of their shares of Synageva common stock (exclusive of any element of value arising from the accomplishment or expectation of the first merger) and to receive payment of such fair value in cash. Any such judicial determination of the fair value of shares of Synageva common stock could be based upon considerations other than, or in addition to, the price paid in the first merger and the market value of shares of Synageva common stock. The value so determined could be higher or lower than the price per Synageva share paid by Alexion pursuant to the first merger. You should be aware that opinions of investment banking firms as to the fairness from a financial point of view of the consideration payable in a sale transaction, such as the first merger, are not opinions as to fair value under applicable Delaware law.
Under Section 262 of the DGCL, where a merger is approved under Section 251(c) of the DGCL, either a constituent corporation before the effective date of the merger, or the surviving corporation within 10 days thereafter, shall notify each of the holders of any class or series of stock of such constituent corporation who are entitled to appraisal rights of the approval of the merger or consolidation and that appraisal rights are available for any or all shares of such class or series of stock of such constituent corporation, and shall include in such notice a copy of Section 262 of the DGCL. This proxy statement/prospectus will constitute the formal notice of appraisal rights under Section 262 of the DGCL.
The foregoing summary of the rights of dissenting stockholders under the DGCL does not purport to be a complete statement of the procedures to be followed by Synageva stockholders desiring to exercise any available appraisal rights under Section 262 of the DGCL, and is qualified in its entirety by the full text of Section 262 of the DGCL, which is attached to this document as Annex E. See also “The Transactions — Dissenters’ Rights.”
Who can help answer my questions about the Synageva special meeting?
You may contact Synageva’s proxy solicitor, [•], with any questions about the Synageva special meeting or the proposals, including questions about how to vote, or to request additional copies of the proxy materials, at:
[•]
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SUMMARY
This section summarizes material information presented in greater detail elsewhere in this document. However, this summary does not contain all of the information that may be important to Synageva stockholders. You are urged to carefully read the remainder of this document, the annexes to this document and the other information referred to or incorporated by reference in this document because the information contained in this section and in the “Questions and Answers About the Offer” section is not complete. See “Where To Obtain Additional Information.”
Purpose of the Transactions (Page 74)
The purpose of the transactions that have been agreed to between Alexion and Synageva is for Alexion to acquire control of, and ultimately the entire equity interest in, Synageva.
Transaction Consideration (Page 123)
The transaction consideration consists of:

$115.00 in cash, without interest and less any applicable withholding taxes (the “cash consideration”); and

0.6581 shares of Alexion common stock, together with cash in lieu of any fractional shares of Alexion common stock, without interest and less any applicable withholding taxes (the “stock consideration” and, together with the cash consideration, the “transaction consideration”).
Synageva stockholders will not receive any fractional shares of Alexion common stock in the first merger, and each Synageva stockholder who otherwise would be entitled to receive a fraction of a share of Alexion common stock pursuant to the first merger will be paid an amount in cash (without interest) in lieu thereof, based on the volume weighted average closing sale price of one share of Alexion common stock as reported on Nasdaq for the 10 consecutive trading days ending on the third business day prior to the closing date (the “Alexion Trading Price”).
The Mergers (Page 121)
The first merger and the second merger (which we refer to collectively as the “mergers”) will be completed as soon as practicable following receipt of Synageva stockholder approval of the transaction agreement, assuming the satisfaction or waiver of the other conditions at such time. The first merger will be subject to Section 251(c) of the DGCL, which means that a vote of Synageva stockholders will be required to complete the first merger. Accordingly Alexion anticipates that the merger will be completed on the same day as the meeting of Synageva stockholders if Synageva stockholders approve the transaction agreement at such meeting and assuming the satisfaction or waiver of the other closing conditions set forth in the transaction agreement as of such date.
In the first merger, the Offeror will merge with and into Synageva, with Synageva surviving the merger. At the effective time of the first merger, each outstanding share of Synageva common stock (other than shares held by stockholders validly exercising appraisal rights under Delaware law, shares held in treasury by Synageva or shares held by Alexion, any subsidiary of Alexion or any subsidiary of Synageva) will be converted into the right to receive the transaction consideration. After the first merger, the Synageva business will be held in a direct wholly owned subsidiary of Alexion, and the former stockholders of Synageva will no longer have any direct ownership interest in the surviving corporation.
Immediately following the first merger, the surviving corporation will merge with and into Merger Sub, with Merger Sub surviving the second merger. From and after the effective time of the second merger, the surviving company holding the Synageva business will be a limited liability company rather than a corporation.
The Companies (Page 72)
Alexion
Alexion Pharmaceuticals, Inc.
352 Knotter Drive
Cheshire, Connecticut 06410
(203) 272-2596
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Alexion is a biopharmaceutical company focused on serving patients with severe and rare disorders through the innovation, development and commercialization of life-transforming therapeutic products. Alexion is the global leader in complement inhibition and has developed and markets Soliris as a treatment for patients with PNH and aHUS, two debilitating, rare and life-threatening disorders caused by chronic uncontrolled activation of the complement component of the immune system. Soliris is currently approved in nearly 50 countries for the treatment of PNH, and in nearly 40 countries for the treatment of aHUS. Alexion is evaluating other potential indications for Soliris in additional severe and devastating diseases beyond PNH and aHUS in which uncontrolled complement activation is the underlying mechanism, and is progressing in various stages of development with additional product candidates as potential treatments for patients with severe and life-threatening ultra-rare disorders. In 2014, Alexion filed for regulatory approval with the FDA, EMA and the MHLW for Strensiq, a targeted enzyme replacement therapy in Phase II clinical trials for patients with HPP, an ultra-rare, genetic, and life-threatening metabolic disease characterized by impaired phosphate and calcium regulation, leading to progressive damage to multiple vital organs including destruction and deformity of bones, profound muscle weakness, seizures, impaired renal function, and respiratory failure. In July 2014, the EMA validated Alexion’s MAA for Strensiq for the treatment of HPP. In March 2015, the FDA accepted for Priority Review the BLA for Strensiq for treatment of patients with infantile- and juvenile-onset HPP. Alexion has approximately 2,400 employees and serves patients in 50 countries
Alexion is a Delaware corporation that was established in 1992 and became a public company in 1996. Its shares are traded on Nasdaq under the ticker symbol “ALXN.”
Offeror
Pulsar Merger Sub Inc.
c/o Alexion Pharmaceuticals, Inc.
352 Knotter Drive
Cheshire, Connecticut 06410
(203) 272-2596
The Offeror is a Delaware corporation and a direct wholly owned subsidiary of Alexion. The Offeror was incorporated on April 28, 2015 for the purpose of making an exchange offer and consummating the first merger. The Offeror has engaged in no business activities to date and it has no material assets or liabilities of any kind, other than those incident to its formation and those incurred in connection with the exchange offer and the mergers.
Merger Sub
Galaxy Merger Sub LLC
c/o Alexion Pharmaceuticals, Inc.
352 Knotter Drive
Cheshire, Connecticut 06410
(203) 272-2596
Merger Sub is a Delaware limited liability company and direct wholly owned subsidiary of Alexion. Merger Sub was formed on April 28, 2015 for the purpose of consummating the second merger. Merger Sub has engaged in no business activities to date and it has no material assets or liabilities of any kind, other than those incident to its formation and those incurred in connection with the mergers.
Synageva
Synageva BioPharma Corp.
33 Hayden Avenue
Lexington, Massachusetts 02421
(781) 357-9900
Synageva is a biopharmaceutical company focused on the discovery, development and commercialization of therapeutic products for patients with rare diseases. Synageva has a pipeline of protein therapeutic programs for rare diseases with unmet medical needs that are at various stages of
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development. It is planning for a global launch of its lead product, sebelipase alfa for lysosomal acid lipase deficiency (LAL Deficiency) under the proposed brand name of KanumaTM. Synageva also has an active investigational new drug application with the FDA to evaluate a second program, SBC-103, a first mover-enzyme replacement therapy program for mucopolysaccharidosis IIIB (also known as Sanfilippo B syndrome), and a third pipeline program, SBC-105, a first-mover enzyme therapy in preclinical development for rare disorders of calcification, including the first planned target indication for generalized calcification in infants.
Synageva is a Delaware corporation that was established in 2008 and became a publicly traded company in 2011. Its shares trade on Nasdaq under the ticker symbol “GEVA.”
Voting and Support Agreements (Page 143)
Concurrently with the execution of the transaction agreement, certain affiliates of Baker Bros. Advisors L.P., an entity controlled by Felix J. Baker, a director of Synageva, and Thomas J. Tisch, a director of Synageva, entered into voting and support agreements with Alexion and the Offeror, pursuant to which, among other things and subject to the terms and conditions of such voting and support agreements, such stockholders agreed to vote all shares of Synageva common stock beneficially owned by them in favor of the adoption of the transaction agreement and the approval of the transactions contemplated by the transaction agreement, and any other matter necessary to consummate such transactions, and not to vote in favor of, or tender their shares into, any competing offer or takeover proposal. The Baker Brothers and Mr. Tisch together own approximately 33.36% of Synageva’s outstanding common stock. The voting and support agreement entered into among Alexion, the Offeror and the Baker Brothers is attached to this document as Annex B, and the voting and support agreement entered into among Alexion, the Offeror and Mr. Tisch is attached to this document as Annex C.
Conditions to the Transactions (Page 125)
Completion of the transactions is subject to certain conditions, including, among others:

receipt of Synageva stockholder approval;

receipt of required regulatory clearance under the HSR Act;

lack of legal prohibitions;

the listing of the shares of Alexion common stock to be issued in the first merger on Nasdaq;

the receipt of an opinion by each party from its legal counsel regarding the tax treatment of the mergers;

the effectiveness of the registration statement on Form S-4 of which this document is a part;

the truth and accuracy of the other party’s representations and warranties made in the transaction agreement, subject to specified materiality standards; and

the other party’s material compliance with its covenants under the transaction agreement.
Treatment of Synageva Equity Awards; Employee Stock Purchase Plan (Page 124)
Consideration for Options
As agreed by Alexion under the terms of the transaction agreement, if the mergers are consummated, the vesting of all options to acquire shares of Synageva common stock (each, a “Stock Option”) outstanding immediately prior to effective time of the first merger will accelerate, such that the Stock Options will become fully vested and cancelled, and the holders thereof will be entitled to receive (without interest) an amount in cash and shares of Alexion common stock equal to (i) the cash consideration and the stock consideration each multiplied by a number of shares of Synageva common stock based on the intrinsic spread value of such Stock Option, based on a $230.00 stock price divided by (ii) $230.00, with the cash portion of such amount rounded down to the nearest cent and with the portion of such amount payable in shares of Alexion common stock rounded down to the nearest one thousandth of a share. Each
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holder of a Stock Option who would otherwise be entitled to receive a fraction of a share of Alexion common stock under the transaction agreement in respect of a Stock Option (after aggregating all of the consideration due with respect to all shares of Synageva common stock underlying such Stock Option) will be paid an amount in cash (without interest) equal to (x) such fractional part of a share of Alexion common stock multiplied by (y) the Alexion Trading Price, rounded down to the nearest cent. Any such consideration will be paid less applicable taxes, which will be deducted first from the cash portion of the consideration payable in respect of the Stock Options. Any Stock Option with a per-share exercise price that equals or exceeds $230.00 will be cancelled without any consideration therefor.
Consideration for Restricted Stock Units
As agreed by Alexion under the terms of the transaction agreement, if the mergers are consummated, the vesting of all restricted stock units (“RSUs”) outstanding immediately prior to the effective time of the first merger other than the “Rolled 2015 RSUs Award” (as defined below) will be accelerated, such that all such RSUs will become fully vested and be cancelled, and the holders thereof will be entitled to receive (without interest) an amount in cash and a number of shares of Alexion common stock equal to the transaction consideration in respect of each share of Synageva common stock subject to such RSUs outstanding immediately prior to the effective time of the first merger. Any such consideration will be paid less applicable taxes, which will be deducted first from the cash portion of the consideration payable in respect of the RSUs.
With respect to one half of each RSU award that is granted after the signing of the transaction agreement (such portion of the award, the “Rolled 2015 RSUs Award”) that is outstanding immediately prior to the effective time of the first merger, such Rolled 2015 RSUs Award will be converted into an RSU award in respect of Alexion common stock, with the number of shares of Alexion common stock underlying such converted award determined by multiplying (x) the number of shares of Synageva common stock subject to such Rolled 2015 RSUs Award by (y) the sum of  (1) the stock consideration and (2) the quotient of the cash consideration, divided by the Alexion Trading Price, with each converted award to continue to be subject to the same terms and conditions as were applicable to the related Rolled 2015 RSUs Award immediately prior to the effective time of the first merger (including accelerated vesting upon a termination without “cause” or resignation for “good reason” within two years following the effective time of the first merger). The other half of each RSU award that is granted after the signing of the transaction agreement will be treated as provided in the immediately preceding paragraph.
Synageva 2014 Employee Stock Purchase Plan
Each outstanding offering period under Synageva’s 2014 Employee Stock Purchase Plan (the “ESPP”) that is in progress as of the date of the execution of the transaction agreement will terminate, and all accumulated contributions to purchase shares of Synageva common stock under the ESPP will be used to purchase shares of Synageva common stock, on the earlier of  (x) the scheduled purchase date for such offering period, and (y) the date that is seven business days prior to the effective time of the first merger. Only current participants in the ESPP may continue to participate in the ESPP and no participant may increase payroll deductions from those in effect at the time the transaction agreement was executed. Synageva will suspend the commencement of any future offering periods under the ESPP unless and until the transaction agreement is terminated, and the ESPP will terminate prior to the effective time of the first merger (with any participant payroll deductions not applied to the purchase of shares of Synageva common stock under the ESPP returned to the applicable participant). Synageva currently expects that the final offering period under the ESPP will be the currently outstanding offering period, which is expected to end on June 30, 2015.
Regulatory Approvals (Page 101)
Completion of the transactions is subject to the expiration or termination of the waiting period applicable to the transactions under the HSR Act. The parties to the transaction agreement are required to use their respective reasonable best efforts to consummate the transactions, including by taking all reasonable actions necessary to obtain any antitrust or other regulatory approvals.
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Source and Amount of Funds (Page 109)
Alexion estimates the aggregate amount of cash consideration required to consummate the first merger will be approximately $4.6 billion, plus related fees and expenses. Alexion anticipates that the funds needed to complete the transactions will be derived from a combination of  (i) available cash on hand and (ii) third-party debt financing. In connection with entering into the transaction agreement, Alexion executed a commitment letter (the “commitment letter”), dated May 5, 2015, with Bank of America, N.A., Merrill Lynch, Pierce, Fenner & Smith Incorporated, JPMorgan Chase Bank, N.A. and J.P. Morgan Securities LLC (collectively, the “commitment parties”), that provides a commitment, subject to the satisfaction of certain conditions, for a $3.0 billion five-year senior secured term loan facility and a $500 million five-year senior secured revolving credit facility.
Alexion’s obligation to consummate the transactions is not conditioned upon any financing arrangements or contingencies (although the availability of the debt financing contemplated by the commitment letter is subject to the satisfaction of the conditions set forth in the commitment letter).
Listing of Alexion Common Stock (Page 139)
Alexion will submit the necessary applications to seek to cause the shares of its common stock to be issued as transaction consideration in the first merger to be approved for listing on Nasdaq. Approval of this listing is a condition to completion of the transactions.
Comparative Market Price and Dividend Matters (Page 145)
Alexion common stock is listed on Nasdaq under the symbol “ALXN,” and Synageva common stock is listed on Nasdaq under the symbol “GEVA.” The following table sets forth the closing prices of Alexion common stock and Synageva common stock on Nasdaq as reported on May 5, 2015, the trading day prior to public announcement of execution of the transaction agreement, and on May 27, 2015, the most recent practicable trading date prior to the filing of this document. The table also shows the implied value of one share of Synageva common stock on such dates, which was calculated by adding (1) the per-share cash consideration of  $115.00 and (2) the product of the exchange ratio of 0.6581 multiplied by the closing price of Alexion common stock on such date.
Per-Share
Synageva
Closing Price
Per-Share
Alexion
Closing Price
Implied
Transaction
Value of
Synageva Share
May 5, 2015
$ 95.87 $ 168.55 $ 225.92
May 27, 2015
$ 215.77 $ 168.12 $ 225.64
The market value of the stock portion of the transaction consideration will change as the market value of Alexion common stock fluctuates until the date of the Synageva special meeting and thereafter. Synageva stockholders should obtain current market quotations for shares of Synageva common stock and Alexion common stock before deciding how to vote their Synageva shares.
Ownership of Alexion After the Transactions (Page 96)
Alexion estimates that former stockholders of Synageva will own, in the aggregate, approximately 11.6% of the shares of Alexion common stock outstanding immediately following completion of the transactions.
Comparison of Stockholders’ Rights (Page 162)
The rights of Alexion stockholders are different in some respects from the rights of Synageva stockholders. Therefore, Synageva stockholders will have different rights as stockholders once they become Alexion stockholders.
Material U.S. Federal Income Tax Consequences (Page 155)
It is intended that the mergers, taken together, qualify as a “reorganization” within the meaning of Section 368(a) of the Code. It is a condition to each of Alexion’s and Synageva’s obligation to complete the
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mergers that it receive a written opinion from its legal counsel, Wachtell, Lipton, Rosen & Katz and Sullivan & Cromwell LLP, respectively, to the effect that the mergers, taken together, will qualify as a “reorganization” within the meaning of Section 368(a) of the Code. Accordingly, assuming the receipt and accuracy of such opinions, U.S. holders (as defined under “Material U.S. Federal Income Tax Consequences”) of shares of Synageva common stock that receive a combination of shares of Alexion common stock and cash (other than cash received in lieu of fractional shares of Alexion common stock) as transaction consideration for shares of Synageva common stock pursuant to the first merger generally will recognize gain (but not loss) in an amount equal to the lesser of  (i) the amount by which the sum of the fair market value of Alexion common stock and cash received by the U.S. holder exceeds such U.S. holder’s adjusted tax basis in its shares of Synageva common stock surrendered and (ii) the amount of cash received by such U.S. holder. Non-U.S. holders (as defined under “Material U.S. Federal Income Tax Consequences”) of shares of Synageva common stock that receive the transaction consideration pursuant to the first merger may be subject to U.S. withholding tax with respect to cash received.
Holders of Synageva common stock should read the section entitled “Material U.S. Federal Income Tax Consequences” for a more complete discussion of the U.S. federal income tax consequences of the transactions. Tax matters can be complicated, and the tax consequences of the transactions to a particular holder will depend on such holder’s particular facts and circumstances. Synageva stockholders should consult their own tax advisors to determine the specific consequences to them of receiving the transaction consideration pursuant to the first merger.
Accounting Treatment (Page 111)
In accordance with United States generally accepted accounting principles (“GAAP”), Alexion will account for the acquisition of shares in the transactions under the acquisition method of accounting for business combinations.
13

SELECTED HISTORICAL CONSOLIDATED FINANCIAL DATA OF ALEXION
The following table sets forth certain selected financial information for Alexion as of the end of and for the periods indicated. The selected consolidated statements of operations data for the years ended December 31, 2014, December 31, 2013 and December 31, 2012 and the selected consolidated balance sheet data as of December 31, 2014 and December 31, 2013 are derived from, and qualified by reference to, the audited consolidated financial statements included in Alexion’s Annual Report on Form 10-K for the fiscal year ended December 31, 2014, which is incorporated by reference into this document. The selected consolidated statements of operations data for the three months ended March 31, 2015 and March 31, 2014 and the selected consolidated balance sheet data as of March 31, 2015 are derived from, and qualified by reference to, Alexion’s unaudited condensed consolidated financial statements included in Alexion’s Quarterly Report on Form 10-Q for the quarter ended March 31, 2015, which is incorporated by reference into this document. The selected consolidated statements of operations data for the years ended December 31, 2011 and December 31, 2010 and the selected consolidated balance sheet data as of December 31, 2012, December 31, 2011 and December 31, 2010 are derived from Alexion’s audited consolidated financial statements, which are not incorporated by reference into this document, and the selected consolidated balance sheet data as of March 31, 2014 are derived from Alexion’s unaudited condensed consolidated financial statements, which are not incorporated by reference into this document. You should read this summary selected financial data together with Alexion’s “Management’s Discussion and Analysis of Financial Condition and Results of Operations” and Alexion’s historical consolidated financial statements and the notes thereto. The historical results are not necessarily indicative of results to be expected in the future. See “Where To Obtain Additional Information.”
Selected Consolidated Statements of Operations Data
Three Months
Ended
March 31,
2015
Three Months
Ended
March 31,
2014
Year Ended
December 31,
2014
Year Ended
December 31,
2013
Year Ended
December 31,
2012
Year Ended
December 31,
2011
Year Ended
December 31,
2010
(Amounts in thousands, except per share amounts)
Net product sales
$ 600,333 $ 566,616 $ 2,233,733 $ 1,551,346 $ 1,134,114 $ 783,431 $ 540,957
Cost of sales:
Cost of sales
69,399 32,939 173,862 168,375 126,214 93,140 64,437
Change in contingent liability from intellectual property settlements
9,181 (53,377)
Total cost of sales
69,399 32,939 173,862 177,556 72,837 93,140 64,437
Operating expenses:
Research and development
221,080 191,457 513,782 317,093 222,732 137,421 98,394
Selling, general and administrative
187,116 129,291 630,209 489,720 384,678 308,176 226,766
Acquisition-related costs
11,979 (38) 20,295 5,029 22,812 13,486 722
Impairment of intangible assets
3,464 11,514 33,521 26,300
Restructuring expenses
7,052 15,365
Amortization of purchased intangible
assets
417 417 382
Total operating expenses
427,227 324,174 1,191,165 845,780 656,939 459,465 325,882
Operating income
103,707 209,503 868,706 528,010 404,338 230,826 150,638
Other income (expense)
3,238 2,408 3,401 (1,741) (6,772) (1,158) (1,627)
Income before income taxes
106,945 211,911 872,107 526,269 397,566 229,668 149,011
Income tax provision
15,622 52,557 215,195 273,374 142,744 54,353 51,981
Net income
$ 91,323 $ 159,354 $ 656,912 $ 252,895 $ 254,822 $ 175,315 $ 97,030
Earnings per common share
Basic
$ 0.46 $ 0.81 $ 3.32 $ 1.29 $ 1.34 $ 0.96 $ 0.54
Diluted
$ 0.45 $ 0.79 $ 3.26 $ 1.27 $ 1.28 $ 0.91 $ 0.52
Shares used in computing earnings per common share
Basic
199,361 197,797 198,103 195,532 190,461 183,220 178,542
Diluted
202,034 201,804 201,623 199,712 198,501 191,806 186,074
14

Selected Consolidated Balance Sheet Data
March 31,
2015
March 31,
2014
December 31,
2014
December 31,
2013
December 31,
2012
December 31,
2011
December 31,
2010
(Amounts in thousands)
Cash, cash equivalents and marketable securities
$ 1,925,092 $ 1,557,478 $ 1,961,566 $ 1,514,851 $ 989,501 $ 540,865 $ 361,605
Total assets
4,414,925 3,379,666 4,201,962 3,317,696 2,613,560 1,394,751 1,012,037
Long-term debt and convertible notes (current
and noncurrent)
45,500 93,500 57,500 113,000 149,000 3,718
Contingent consideration (current and noncurrent)
174,950 142,638 162,971 142,676 141,670 18,120
Facility lease obligation
114,912 38,417 107,099 32,230
Total stockholders’ equity
3,516,902 2,700,978 3,302,018 2,382,079 1,970,850 1,134,492 859,736
15

SELECTED HISTORICAL CONSOLIDATED FINANCIAL DATA OF SYNAGEVA
The following table sets forth certain selected financial information for Synageva as of the end of and for the periods indicated. The selected consolidated statements of operations data for the years ended December 31, 2014, December 31, 2013 and December 31, 2012 and the selected consolidated balance sheet data as of December 31, 2014 and December 31, 2013 are derived from, and qualified by reference to, the audited consolidated financial statements included in Synageva’s Annual Report on Form 10-K for the fiscal year ended December 31, 2014, which is incorporated by reference into this document. The selected consolidated statements of operations data for the three months ended March 31, 2015 and March 31, 2014 and the selected consolidated balance sheet data as of March 31, 2015 are derived from, and qualified by reference to, Synageva’s unaudited consolidated financial statements included in Synageva’s Quarterly Report on Form 10-Q for the quarter ended March 31, 2015, which is incorporated by reference into this document. The selected consolidated statements of operations data for the years ended December 31, 2011 and December 31, 2010 and the selected consolidated balance sheet data as of December 31, 2012, December 31, 2011 and December 31, 2010 are derived from Synageva’s audited consolidated financial statements, which are not incorporated by reference into this document, and the selected consolidated balance sheet data as of March 31, 2014 are derived from Synageva’s unaudited consolidated financial statements, which are not incorporated by reference into this document. You should read this summary selected financial data together with Synageva’s “Management’s Discussion and Analysis of Financial Condition and Results of Operations” and Synageva’s historical consolidated financial statements and the notes thereto. The historical results are not necessarily indicative of results to be expected in the future. See “Where To Obtain Additional Information.”
Selected Consolidated Statements of Operations Data
Three Months
Ended
March 31,
2015
Three Months
Ended
March 31,
2014
Year Ended
December 31,
2014
Year Ended
December 31,
2013
Year Ended
December 31,
2012
Year Ended
December 31,
2011
Year Ended
December 31,
2010
(in thousands except per share data)
Revenues:
Royalty revenue
$ 927 $ 1,447 $ 6,000 $ 7,042 $ 7,023 $ 1,083 $
Collaboration and license revenue
139 492 6,332 7,931 1,016 595
Total revenue
927 1,586 6,492 13,374 14,954 2,099 595
Costs and expenses:
Research and development
38,207 27,868 142,638 79,644 37,347 17,346 9,866
Selling, general and administrative
21,671 9,804 54,498 27,560 17,396 9,268 3,852
Amortization of developed technology
222 390 1,489 2,073 3,232 504
Total costs and expenses
60,100 38,062 198,625 109,277 57,975 27,118 13,718
Loss from operations
(59,173) (36,476) (192,133) (95,903) (43,021) (25,019) (13,123)
Other (expense) income, net
(252) (5) (238) 159 (259) 2,295
Interest income (expense), net
83 75 263 342 72 (28) 4
Loss before provision for income taxes
(59,342) (36,406) (192,108) (95,402) (42,949) (25,306) (10,824)
Provision for income taxes
259 18 540 48
Net loss
(59,601) (36,424) (192,648) (95,450) (42,949) (25,306) (10,824)
Basic and diluted loss per common share
$ (1.63) $ (1.16) $ (5.89) $ (3.40) $ (1.90) $ (8.58) $ (338.25)
Weighted average shares used in basic
and diluted per common share
computations
36,495 31,338 32,719 28,087 22,579 2,950 32
16

Selected Consolidated Balance Sheet Data
At
March 31,
2015
At
March 31,
2014
At
December 31,
2014
At
December 31,
2013
At
December 31,
2012
At
December 31,
2011
At
December 31,
2010
Consolidated Balance Sheet Data:
(in thousands)
Cash, cash equivalents, and short-term investments
$ 710,561 $ 575,218 $ 446,908 $ 408,733 $ 218,953 $ 60,232 $ 14,715
Working capital
694,426 568,402 432,589 402,803 212,028 56,393 14,285
Total assets
768,546 623,104 504,203 447,949 243,256 83,298 16,982
Accumulated deficit
(506,488) (290,663) (446,887) (254,239) (158,789) (115,840) (90,534)
Total stockholders’ equity
735,126 601,092 473,239 430,201 230,177 74,048 15,403
17

SELECTED UNAUDITED PRO FORMA COMBINED FINANCIAL DATA
The following selected unaudited pro forma combined financial information has been prepared to give effect to the mergers and the debt financing.
The unaudited pro forma combined statements of operations give effect to the mergers and the debt financing as if they had occurred on January 1, 2014. The unaudited pro forma combined balance sheet gives effect to the mergers and the debt financing as if they had occurred on March 31, 2015. The unaudited pro forma combined financial information was prepared using the acquisition method of accounting. See “The Transactions — Accounting Treatment.”
The summary selected unaudited pro forma combined financial information has been prepared for illustrative purposes only and does not purport to represent what the actual consolidated results of operations or the consolidated financial position of Alexion would have been had the mergers and the debt financing occurred on the dates assumed, nor is this information necessarily indicative of future consolidated results of operations or financial position. The unaudited pro forma combined financial information includes adjustments and assumptions that are factually supportable and that Alexion believes are reasonable. These assumptions, however, are only preliminary and may vary significantly from the fair values that will be recorded upon completion of the mergers and the debt financing. The unaudited pro forma combined statements of operations are based upon the historical financial statements of Alexion and Synageva and include all adjustments that give effect to the events directly attributable to the mergers and the debt financing, and are expected to have a continuing impact and are factually supportable. See “Risk Factors — Alexion’s and Synageva’s actual financial positions and results of operations may differ materially from the unaudited pro forma combined financial data included in this document.” The following information has been derived from, and should be read in conjunction with, the unaudited pro forma combined financial statements and the related notes included in this document. See “Unaudited Pro Forma Combined Financial Statements.”
Selected Unaudited Pro Forma Combined Statements of Operations
Three Months Ended March 31, 2015
Year Ended December 31, 2014
Alexion
Historical
Synageva
Historical
Pro Forma
Adjustments
Pro Forma
Combined
Alexion
Historical
Synageva
Historical
Pro Forma
Adjustments
Pro Forma
Combined
(in thousands, except per share data)
Net income (loss)
$ 91,323 $ (59,601) $ (11,685) $ 20,037 $ 656,912 $ (192,648) $ (47,818) $ 416,446
Earnings (loss) per common share:
Basic
$ 0.46 $ (1.63) $ 0.09 $ 3.32 $ (5.89) $ 1.86
Diluted
$ 0.45 $ (1.63) $ 0.09 $ 3.26 $ (5.89) $ 1.83
Selected Unaudited Pro Forma Combined Balance Sheet
As of March 31, 2015
Alexion
Historical
Synageva
Historical
Pro Forma
Adjustments
Pro Forma
Combined
(in thousands)
Total assets
$ 4,414,925 $ 768,546 $ 7,164,311 $ 12,347,782
Total liabilities
$ 898,023 $ 33,420 $ 3,795,400 $ 4,726,843
Total stockholders’ equity
$ 3,516,902 $ 735,126 $ 3,368,911 $ 7,620,939
18

UNAUDITED COMPARATIVE PER SHARE DATA
The following table reflects historical information about basic and diluted earnings per share, cash dividends per share and book value per share for the three months ended March 31, 2015 and for the year ended December 31, 2014, in each case, on a historical basis, and for Alexion and Synageva on an unaudited pro forma combined basis after giving effect to the mergers and the debt financing. The pro forma data of the combined company assume the acquisition of 100% of the shares of Synageva common stock by Alexion and were derived by combining the historical consolidated financial information of Alexion and Synageva as described elsewhere in this document. For a discussion of the assumptions and adjustments made in preparing the unaudited pro forma combined financial information presented in this document, see “Unaudited Pro Forma Combined Financial Statements.”
Synageva stockholders should read the information presented in the following table together with the historical financial statements of Alexion and Synageva and the related notes, which are incorporated herein by reference, and the “Unaudited Pro Forma Combined Financial Statements” appearing elsewhere in this document. The pro forma data are unaudited and for illustrative purposes only. Synageva stockholders should not rely on this information as being indicative of the historical results that would have been achieved during the periods presented had the companies always been combined or the future results that the combined company will achieve after the consummation of the offer and the mergers. This pro forma information is subject to risks and uncertainties, including those discussed in “Risk Factors.”
Alexion
Historical
Synageva
Historical
Pro Forma
Combined
Pro Forma
Equivalent
Synageva
Share
Net income (loss) per share attributable to common stockholders for the three months ended March 31, 2015:
Basic earnings (loss) per share
$ 0.46 $ (1.63) $ 0.09 $ 0.06
Diluted earnings (loss) per share
$ 0.45 $ (1.63) $ 0.09 $ 0.06
Cash dividends declared per share for the three months ended March 31, 2015
$ $ $ $
Book value per share as of March 31, 2015
$ 17.61 $ 19.87 $ 33.75 $ 22.21
Net income (loss) per share attributable to common stockholders for the year ended December 31, 2014:
Basic earnings (loss) per share
$ 3.32 $ (5.89) $ 1.86 $ 1.22
Diluted earnings (loss) per share
$ 3.26 $ (5.89) $ 1.83 $ 1.20
Cash dividends declared per share for the year ended December 31, 2014
$ $ $ $
19

RISK FACTORS
Synageva stockholders should carefully read this document and the other documents referred to or incorporated by reference into this document, including in particular the following risk factors, in deciding how to vote their shares of Synageva common stock at the Synageva special meeting.
Risks Relating to the Transactions and to the Combined Company
The stock portion of the transaction consideration is fixed and will not be adjusted. Because the market price of Alexion common stock may fluctuate, Synageva stockholders cannot be sure of the market value of the transaction consideration they will receive for their Synageva shares in connection with the transactions.
In connection with the first merger, Synageva stockholders will receive cash and a fixed number of Alexion shares of common stock for each of their shares of Synageva common stock (i.e., 0.6581 Alexion shares for each Synageva share). Because the number of shares of Alexion common stock comprising the stock portion of the transaction consideration will not vary based on the market value of Alexion common stock, the portion of the market value of the transaction consideration that Synageva stockholders will receive in the first merger that is based on the value of Alexion common stock will vary based on the price of such stock at the time the transaction consideration is received. The market price of Alexion common stock may decline after the date of this document, after you vote your shares and/or after the first merger is completed.
A decline in the market price of Alexion common stock could result from a variety of factors beyond Alexion’s control, including, among other things, the possibility that Alexion may not achieve the expected benefits of the acquisition of Synageva as rapidly or to the extent anticipated, including to the extent Alexion is unable to effectively identify patients with LAL Deficiency or as a result of adverse legal or regulatory developments; Synageva’s business may not perform as anticipated following the transactions, including if preclinical and clinical trials of KanumaTM (“Kanuma”) and Synageva’s other product candidates do not produce positive results or are delayed, if serious side effects are identified during drug development, if a narrow label is received or if regulatory and marketing approval and commercialization of Kanumaand Synageva’s other product candidates is not achieved on the expected time frame or at all (see “Risk Factors — Risks Related to Synageva’s Business”); the effect of Alexion’s acquisition of Synageva on Alexion’s financial results may not meet the expectations of Alexion, financial analysts or investors; the addition and integration of Synageva’s business may be unsuccessful, take longer or be more disruptive than anticipated; or Alexion’s creditworthiness may be adversely affected as a result of Alexion’s increased indebtedness incurred to finance the mergers.
Because the transactions will not be completed until certain conditions have been satisfied or waived, a significant period of time may pass between the time you vote your shares and the time that the first merger is completed. Therefore, at the time you vote your shares of Synageva common stock, you will not know the exact market value of the stock portion of the transaction consideration that will be issued if the first merger is completed.
See “Comparative Market Price and Dividend Matters” of this document. You are urged to obtain current market quotations for shares of Synageva common stock and for shares of Alexion common stock.
The transactions remain subject to conditions that Alexion cannot control.
The transactions are subject to a number of conditions, including receipt of Synageva stockholder approval, receipt of required regulatory clearance under the HSR Act, lack of legal prohibitions, the listing on Nasdaq of the shares of Alexion common stock to be issued in the transactions, the receipt of opinions of Synageva’s and Alexion’s respective legal counsel regarding the tax treatment of the transactions, the effectiveness of the registration statement on Form S-4 of which this document is a part, the truth and accuracy of each party’s representations and warranties made in the transaction agreement, subject to specified materiality standards, and each party’s material compliance with its covenants under the transaction agreement. There are no assurances that all of the conditions to the transactions will be satisfied or that the conditions will be satisfied in the time frame expected. If the conditions to the transactions are not met, then the parties, subject to the terms and conditions of the transaction agreement, will not be required to complete the transactions. See “The Transaction Agreement — Conditions to the Transactions.”
20

If the transactions are completed, Synageva stockholders will receive Alexion common stock as part of the transaction consideration and will accordingly become Alexion stockholders. Alexion common stock may be affected by different factors than Synageva common stock, and Alexion stockholders will have different rights than Synageva stockholders.
Upon consummation of the transactions, Synageva stockholders will receive shares of Alexion common stock as part of the transaction consideration and will accordingly become Alexion stockholders. Alexion’s business differs from that of Synageva, and Alexion’s results of operations and the trading price of Alexion common stock may be adversely affected by factors different from those that would affect Synageva’s results of operations and stock price.
In addition, holders of shares of Alexion common stock will have rights as Alexion stockholders that differ from the rights they had as Synageva stockholders before the transactions. For a comparison of the rights of Alexion stockholders to the rights of Synageva stockholders, see “Comparison of Stockholders’ Rights.”
Synageva stockholders will have a reduced ownership and voting interest in the combined company.
Immediately following consummation of the first merger, Synageva stockholders will collectively own approximately 11.6% of the outstanding shares of Alexion common stock. Consequently, Synageva stockholders will not be able to exercise as much influence over the management and policies of the combined company as they currently exercise over Synageva.
Alexion may fail to realize all of the anticipated benefits of the transactions or those benefits may take longer to realize than expected.
The full benefits of the transactions may not be realized as expected or may not be achieved within the anticipated time frame, or at all. Failure to achieve the anticipated benefits of the transactions could adversely affect Alexion’s results of operations or cash flows, cause dilution to the earnings per share of Alexion, decrease or delay the expected benefits of the transactions and negatively affect the price of Alexion common stock.
In addition, Alexion and Synageva will be required to devote significant attention and resources prior to closing to prepare for the post-closing operation of the combined company, and Alexion will be required post-closing to devote significant attention and resources to successfully align the business practices and integrate the operations of Alexion and Synageva. This process may disrupt the businesses and, if ineffective, would limit the anticipated benefits of the transactions.
Alexion and Synageva will incur direct and indirect costs as a result of the transactions.
Alexion and Synageva will incur substantial expenses in connection with and as a result of completing the transactions and, following the completion of the mergers, Alexion expects to incur additional expenses in connection with combining the businesses, operations, policies and procedures of Alexion and Synageva. Factors beyond Alexion’s control could affect the total amount or timing of those expenses, many of which, by their nature, are difficult to estimate accurately. Moreover, diversion of management focus and resources from the day-to-day operation of the business to matters relating to the transactions could adversely affect each company’s business, regardless of whether the mergers are completed.
The receipt of shares of Alexion common stock in the first merger may be taxable to Synageva stockholders.
The transactions are contingent upon the receipt of an opinion by each of Alexion and Synageva from their respective legal counsel to the effect that the mergers, taken together, will qualify as a “reorganization” within the meaning of Section 368(a) of the Code. However, if the mergers are not treated as component parts of an integrated transaction for U.S. federal income tax purposes, if the mergers are not completed or if the transactions otherwise fail to qualify as a “reorganization” within the meaning of Section 368(a) of the Code, the receipt of cash and shares of Alexion common stock in the first merger will be taxable to such Synageva stockholders for U.S. federal income tax purposes.
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Alexion’s and Synageva’s actual financial positions and results of operations may differ materially from the unaudited pro forma combined financial data included in this document.
The unaudited pro forma combined financial information contained in this document is presented for illustrative purposes only and may differ materially from what Alexion’s actual financial position or results of operations would have been had the transactions been completed on the dates indicated. The unaudited pro forma combined financial information has been derived from the audited and unaudited historical financial statements of Alexion, and certain adjustments and assumptions have been made regarding the combined company after giving effect to the transactions. The assets and liabilities of Synageva have been measured at fair value based on various preliminary estimates using assumptions that Alexion management believes are reasonable utilizing information currently available. The process for estimating the fair value of acquired assets and assumed liabilities requires the use of judgment in determining the appropriate assumptions and estimates. These estimates may vary significantly as additional information becomes available and as additional analyses are performed. Differences between preliminary estimates in the unaudited pro forma combined financial information and the final acquisition accounting may occur and are not necessarily indicative of financial position or results of operations in future periods or that would have been realized in historical periods presented.
In addition, the assumptions used in preparing the unaudited pro forma combined financial information may not prove to be accurate, and other factors may affect Alexion’s financial condition or results of operations following the closing. Any potential decline in Alexion’s financial condition or results of operations may cause significant variations in the share price of Alexion. See “Unaudited Pro Forma Combined Financial Statements.”
Alexion will incur significant additional indebtedness in connection with the transactions, which will decrease Alexion’s business flexibility and increase its interest expense.
The consolidated indebtedness of Alexion as of March 31, 2015 was approximately $160 million. Alexion’s pro forma indebtedness as of March 31, 2015, after giving effect to the transactions and the anticipated incurrence and extinguishment of indebtedness in connection therewith, will be approximately $3.6 billion. Alexion’s substantially increased indebtedness following completion of the transactions could have the effect, among other things, of reducing Alexion’s flexibility to respond to changing business and economic conditions and will increase Alexion’s interest expense. Alexion will also incur various costs and expenses associated with the debt financing. The amount of cash required to pay interest on Alexion’s increased indebtedness levels following completion of the transactions, and thus the demands on Alexion’s cash resources, will be greater than the amount of cash required to service the indebtedness of Alexion prior to the transactions. Alexion’s increased indebtedness following completion of the transactions could also reduce funds available for working capital, capital expenditures, acquisitions and other general corporate purposes and may create competitive disadvantages for Alexion relative to other companies with lower indebtedness levels. If Alexion does not achieve the expected benefits and cost savings from the transactions, or if the financial performance of the combined company does not meet current expectations, then Alexion’s ability to service its indebtedness may be adversely impacted.
It is expected that the debt financing will bear interest at variable interest rates. If interest rates increase, variable rate debt will create higher debt service requirements, which could further adversely affect Alexion’s cash flows.
Moreover, Alexion may be required to raise substantial additional financing to fund working capital, capital expenditures, acquisitions or other general corporate requirements. Alexion’s ability to arrange additional financing will depend on, among other factors, Alexion’s financial position and performance, as well as prevailing market conditions and other factors beyond Alexion’s control. Alexion cannot assure you that it will be able to obtain additional financing on terms acceptable to Alexion or at all.
The definitive documentation governing the debt financing has not been finalized. However, it is expected that the definitive documentation governing the debt financing will contain various affirmative and negative covenants that impose restrictions on Alexion and certain of its subsidiaries and that may affect their ability to operate their businesses. In addition, such documentation is expected to contain financial covenants that will require Alexion to maintain certain financial ratios. See “Source and Amount
22

of Funds.” The ability of Alexion and its subsidiaries to comply with these provisions may be affected by events beyond their control. Failure to comply with these covenants could result in an event of default, which, if not cured or waived, could accelerate Alexion’s repayment obligations.
The transaction agreement limits Synageva’s ability to pursue alternative transactions, and in certain instances requires payment of a termination fee, which could deter a third party from proposing an alternative transaction.
The transaction agreement contains provisions that, subject to certain exceptions, limit Synageva’s ability to solicit, initiate or knowingly encourage or knowingly facilitate any inquiries regarding or the making of any proposal or offer that constitutes or could reasonably be expected to lead to an alternative takeover proposal. See “Transaction Agreement — No Solicitation of Other Offers by Synageva.” In addition, under specified circumstances, Synageva is required to pay a termination fee of  $325 million if the transaction agreement is terminated. See “Transaction Agreement — Termination Fee.” It is possible that these or other provisions might discourage a potential competing acquiror that might have an interest in acquiring all or a significant part of Synageva from considering or proposing an acquisition or might result in a potential competing acquiror proposing to pay a lower per share price to acquire Synageva than it might otherwise have proposed to pay.
If the value of Alexion’s business, together with any synergies to be achieved from Alexion’s acquisition of Synageva, is less than the value of the transaction consideration, the trading price of shares of Alexion common stock could decrease.
If investors believe that the value of the cash consideration and stock consideration to be received for Synageva shares in connection with the first merger, together with transaction costs, is greater than the value of Synageva’s business, together with any synergies expected to be achieved from Alexion’s acquisition of Synageva, the trading price of Alexion common stock could decrease and the transactions could have a dilutive effect on the value of common shares held by Alexion stockholders (including former Synageva stockholders).
Uncertainty during pendency of the transactions may cause suppliers, customers or other business partners to delay or defer decisions concerning Alexion and/or Synageva or re-negotiate agreements with Alexion and/or Synageva, and completion of the transactions could cause suppliers, customers and other business partners to terminate or re-negotiate their relationships with the combined company.
The transactions will be completed only if specified conditions are met, many of which are outside the control of Alexion and Synageva. In addition, both parties have rights to terminate the transaction agreement under specified circumstances. Accordingly, there may be uncertainty regarding the consummation of the transactions, both as to whether they will be consummated and when. This uncertainty may cause suppliers, customers or other business partners of Alexion and/or Synageva to delay or defer decisions concerning such company’s products or businesses, or may seek to change existing agreements with Alexion and/or Synageva, which could negatively affect their respective businesses, results of operations and financial conditions.
Additionally, if the transactions are completed, certain suppliers, customers or other business partners may attempt to terminate or change their relationships with the combined company, for example if such counterparties had prior experiences with either Alexion or Synageva that caused them to be dissatisfied with Alexion or Synageva. These decisions could have an adverse effect on the business of the combined company.
Alexion’s acquisition of Synageva could trigger certain change-of-control or similar provisions contained in Synageva’s agreements with third parties that could permit such parties to terminate or re-negotiate those agreements.
Synageva may be a party to agreements that permit a counterparty to terminate an agreement or receive payments because the transactions would cause a default or violate an anti-assignment, change-of-control or similar clause in such agreement. If this happens, Alexion may have to seek to replace that agreement with a new agreement or make additional payments under such agreement. However, Alexion may be unable to replace a terminated agreement on comparable terms or at all. Depending on the
23

importance of such agreement to Synageva’s business, the failure to replace a terminated agreement on similar terms or at all, and requirements to pay additional amounts, may increase the costs to Alexion of operating Synageva’s business or decrease the expected benefits of the transactions to the combined company.
The stock prices of Alexion and Synageva common stock may be adversely affected if the transactions are not completed.
If the mergers are not completed, the prices of Alexion common stock and Synageva common stock may decline to the extent that the current market prices of such common stock reflect a market assumption that the mergers will be completed and have value.
Failure to effectively retain, attract and motivate key employees could diminish the anticipated benefits of the transactions.
The success of the acquisition of Synageva will depend in part on the attraction, retention and motivation of personnel critical to the business and operations of the combined company due to, for example, their technical skills or industry and management expertise. Employees and consultants may experience uncertainty about their future roles with Alexion and Synageva during the pendency of the transactions or after their completion. Alexion and Synageva, while similar and sharing a number of core values, do not have identical corporate cultures, and some employees or consultants may not want to work for the combined company. In addition, competitors may recruit employees during Alexion’s integration of Synageva. If the companies are unable to attract, retain and motivate personnel that are critical to the successful integration and future operation of the companies, the combined company could face disruptions in its operations, loss of existing customers, key information, expertise or know-how and unanticipated additional recruiting and training costs. In addition, the loss of key personnel could diminish the anticipated benefits of the acquisition of Synageva.
The opinion of Synageva’s financial advisor will not reflect changes in circumstances between the signing of the transaction agreement and the completion of the first merger.
Synageva has not obtained an updated opinion from its financial advisor as of the date of this document and does not expect to receive an updated opinion prior to completion of the first merger. Changes in the operations and prospects of Synageva or Alexion, general market and economic conditions and other factors that may be beyond the control of Synageva or Alexion, and on which Synageva’s financial advisor’s opinion was based, may significantly alter the value of Synageva or Alexion or the prices of Synageva or Alexion common stock by the time the offer and the mergers are completed. The opinion does not speak as of the time the offer and the mergers will be completed or as of any date other than the date of such opinion. Because Synageva’s financial advisor will not be updating its opinion, the opinion will not address the fairness of the transaction consideration from a financial point of view at the time the mergers are completed.
Risks Related to Alexion’s Business
Alexion depends heavily on the success of its lead product, Soliris. If Alexion is unable to increase sales of Soliris, or obtain approval or commercialize Soliris in new territories for the treatment of PNH, aHUS or for additional indications, or if Alexion is significantly delayed or limited in doing so, Alexion’s business may be materially harmed.
Alexion’s ability to generate revenues will continue to depend on commercial success of Soliris and whether physicians, patients and health care payers view Soliris as therapeutically effective and safe relative to cost. Since Alexion launched Soliris in the United States in April 2007, essentially all of its revenue has been attributed to sales of Soliris, and Alexion expects that Soliris product sales will continue to contribute to a significant percentage or almost all of its total revenue over the next several years.
In September and November 2011, Alexion obtained marketing approval in the United States and the European Union, respectively, for Soliris for the treatment of a second indication, aHUS. In September 2013, the MHLW approved Soliris for the treatment of patients with aHUS in Japan.
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Alexion dedicates significant resources to the worldwide commercialization of Soliris. Alexion has established sales and marketing capabilities in the United States and in many countries throughout the world. Alexion cannot guarantee that any marketing application for Soliris for the treatment of PNH, aHUS or any other indication, will be approved or maintained in any country where Alexion seeks marketing authorization to sell Soliris. In certain countries, Alexion continues discussions with authorities to finalize operational, reimbursement, price approval and funding processes so that Alexion may, upon conclusion of such discussions, commence commercial sales of Soliris for the treatment of PNH in those countries. Alexion has had and will continue to have similar discussions with authorities to facilitate the commercialization of Soliris for the treatment of aHUS in certain countries in the European Union. Alexion’s ability to complete such processes successfully is subject to the risks and uncertainties described in this prospectus/offer to exchange. Alexion cannot guarantee that it will be able to obtain reimbursement for Soliris or successfully commercialize Soliris in any additional countries, or that Alexion will be able to maintain coverage or reimbursement at anticipated levels in any country in which it has already received marketing approval, including the U.S., certain European countries, or Japan. As a result, sales in certain countries may be delayed or never occur, or may be subsequently reduced.
The commercial success of Soliris and Alexion’s ability to generate and increase revenues will depend on several factors, including the following:

receipt of marketing approvals for Soliris for the treatment of PNH and aHUS in new territories, and the maintenance of marketing approvals in the United States, the European Union, Japan and other territories;

Alexion’s ability to obtain sufficient coverage or reimbursement by government or third-party payers and its ability to maintain coverage or reimbursement at anticipated levels;

establishment and maintenance of Alexion’s commercial manufacturing capabilities either by Alexion or through third-party manufacturers;

the number of patients with PNH and aHUS, and the number of those patients who are diagnosed with PNH and aHUS and identified to us;

the number of patients with PNH and aHUS that may be treated with Soliris;

successful continuation of commercial sales in the United States, Japan and in European countries where Alexion is already selling Soliris for the treatment of PNH and aHUS, and successful launch in countries where Alexion has not yet obtained, or only recently obtained, marketing approval or commenced sales;

acceptance of Soliris and maintenance of safety and efficacy in the medical community; and

Alexion’s ability to develop, register and commercialize Soliris for indications other than PNH and aHUS.
If Alexion is not successful in increasing sales of Soliris in the United States, Europe and Japan and commercializing in the rest of the world, or are significantly delayed or limited in doing so, Alexion may experience surplus inventory, Alexion’s business may be materially harmed and it may need to significantly curtail operations.
If Alexion is unable to obtain, or maintain at anticipated levels, reimbursement for Soliris from government health administration authorities, private health insurers and other organizations, Alexion’s pricing may be affected or its product sales, results of operations or financial condition could be harmed.
Alexion may not be able to sell Soliris on a profitable basis or Alexion’s profitability may be reduced if it is required to sell its product at lower than anticipated prices or reimbursement is unavailable or limited in scope or amount. Soliris is significantly more expensive than traditional drug treatments and almost all patients require some form of third party coverage to afford its cost. Alexion’s future revenues and profitability will be adversely affected if Alexion cannot depend on governmental payers, such as Medicare and Medicaid in the United States or country specific governmental organizations in foreign countries, and private third-party payers to defray the cost of Soliris to patients. These entities may refuse to provide coverage and reimbursement with respect to Soliris, determine to provide a lower level of coverage and
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reimbursement than anticipated, or reduce previously approved levels of coverage and reimbursement, including in the form of higher mandatory rebates or modified pricing terms. In any such case, Alexion’s pricing or reimbursement for Soliris may be affected and Alexion’s product sales, results of operations or financial condition could be harmed.
In certain countries where Alexion sells or is seeking or may seek to commercialize Soliris, including certain countries where Alexion both sells Soliris for the treatment of PNH and sells or seeks to commercialize Soliris for the treatment of aHUS, if approved by the appropriate regulatory authority, pricing, coverage and level of reimbursement of prescription drugs are subject to governmental control. Alexion may be unable to timely or successfully negotiate coverage, pricing, and reimbursement on terms that are favorable to it, or such coverage, pricing, and reimbursement may differ in separate regions in the same country. In some foreign countries, the proposed pricing for a drug must be approved before it may be lawfully marketed. The requirements governing drug pricing vary widely from country to country, and Alexion cannot guarantee that Alexion will have the capabilities or resources to successfully conclude the necessary processes and commercialize Soliris in every, or even most countries in which Alexion seek to sell Soliris.
Reimbursement sources are different in each country and in each country may include a combination of distinct potential payers, including private insurance and governmental payers. For example, the European Union member states’ authorities may restrict the range of medicinal products for which their national health insurance systems provide reimbursement and adopt additional measures to control the prices of medicinal products for human use. This includes the use of reference pricing and Health Technology Assessment (“HTA”). HTA is the procedure according to which the assessment of the public health impact, therapeutic impact and the economic and societal impact of the use of a given medicinal product in the national healthcare systems of the individual country is conducted. HTA generally focuses on the clinical efficacy and effectiveness, safety, cost, and cost-effectiveness of individual medicinal products as well as their potential implications for the healthcare system. These elements of medicinal products are compared with other treatment options available on the market. The national authorities of some European Union member states may from time to time approve a specific price for the medicinal product. Others may adopt a system of direct or indirect controls on the profitability of the company placing the medicinal product on the national market. Some countries have and others may seek to impose limits on the aggregate reimbursement for Soliris or for the use of Soliris for certain indications. In such cases, Alexion’s commercial operations in such countries and Alexion’s results of operations and business are and may be adversely affected. Alexion’s results of operations may suffer if it is unable to successfully and timely conclude reimbursement, price approval or funding processes and market Soliris in such foreign countries or if coverage and reimbursement for Soliris is limited or reduced. If Alexion is not able to obtain coverage, pricing or reimbursement on terms acceptable to Alexion or at all, or if such terms should change in any foreign countries, Alexion may not be able to or it may determine not to sell Soliris for one or more indications in such countries, or Alexion could decide to sell Soliris at a lower than anticipated price in such countries, and Alexion’s revenues may be adversely affected as a result.
The potential increase in the number of patients receiving Soliris may cause third-party payers to modify or limit coverage or reimbursement for Soliris for the treatment of PNH, aHUS, or both indications.
Changes in pricing or the amount of reimbursement in countries where Alexion currently commercializes Soliris may also reduce its profitability and worsen its financial condition. In the United States, the European Union member states, and elsewhere, there have been, and Alexion expects there will continue to be, efforts to control and reduce health care costs. Third party payers decide which drugs they will pay for and establish reimbursement and co-payment levels. Government and other third-party payers in the United States and the European Union member states are increasingly challenging the prices charged for health care products, examining the cost effectiveness of drugs in addition to their safety and efficacy, and limiting or attempting to limit both coverage and the level of reimbursement for prescription drugs.
A significant reduction in the amount of reimbursement or pricing for Soliris in one or more countries may have a material adverse effect on Alexion’s business. See additional discussion below under the headings “Changes in healthcare law and implementing regulations, including those based on recently
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enacted legislation, as well as changes in healthcare policy and Government initiatives that affect coverage and reimbursement of drug products may impact Alexion’s business in ways that Alexion cannot currently predict and these changes could adversely affect Alexion’s business and financial condition” and “The credit and financial market conditions may aggravate certain risks affecting Alexion’s business.” In addition, certain countries establish pricing and reimbursement amounts by reference to the price of the same or similar products in other countries. If coverage or the level of reimbursement is limited in one or more countries, Alexion may be unable to obtain or maintain anticipated pricing or reimbursement in current or new territories.
Many third-party payers cover only selected drugs, making drugs that are not preferred by such payer more expensive for patients, and require prior authorization or failure on another type of treatment before covering a particular drug. Third-party payers may be especially likely to impose these obstacles to coverage for higher-priced drugs such as Soliris.
Payers in the U.S. also are increasingly considering new metrics as the basis for reimbursement rates, such as average sales price (“ASP”), average manufacturer price, and actual acquisition cost. The existing data for reimbursement based on these metrics is relatively limited, although certain states have begun to survey acquisition cost data for the purpose of setting Medicaid reimbursement rates. The Centers for Medicare and Medicaid Services (“CMS”), the federal agency that administers Medicare and the Medicaid Drug Rebate Program, has begun posting drafts of this retail survey price information on at least a monthly basis in the form of draft National Average Drug Acquisition Cost (“NADAC”) files, which reflect retail community pharmacy invoice costs, and National Average Retail Price (“NARP”) files, which reflect retail community pharmacy prices to consumers. In July 2013, CMS suspended the publication of draft NARP data, pending funding decisions. In November 2013, CMS moved to publishing final rather than draft NADAC data and has since made updated NADAC data publicly available on a weekly basis. Therefore, it may be difficult to project the impact of these evolving reimbursement mechanics on the willingness of payers to cover Soliris.
Even in countries where patients have access to insurance, their insurance co-payment amounts or annual or lifetime caps on reimbursements may represent a barrier to obtaining or continuing Soliris. Alexion has financially supported non-profit organizations which assist patients in accessing treatment for PNH and aHUS, including Soliris. Such organizations assist patients whose insurance coverage leaves them with prohibitive co-payment amounts or other expensive financial obligations. Such organizations’ ability to provide assistance to patients is dependent on funding from external sources, and Alexion cannot guarantee that such funding will be provided at adequate levels, if at all. Alexion has also provided Soliris without charge to patients who have no insurance coverage for drugs through related charitable purposes. Alexion is not able to predict the financial impact of the support it may provide for these and other charitable purposes; however, substantial support could have a material adverse effect on Alexion’s profitability in the future.
Alexion is also focusing development efforts on the use of eculizumab for the treatment of additional diseases. The success of these programs depends on many factors, including those described in this prospectus/offer to exchange. As Soliris is approved by regulatory agencies for indications other than PNH and aHUS, the potential increase in the number of patients receiving Soliris may cause third-party payers to refuse coverage or reimbursement for Soliris for the treatment of PNH, aHUS or for any other approved indication, or provide a lower level of coverage or reimbursement than anticipated or currently in effect.
Alexion may not be able to maintain market acceptance of Soliris among the medical community or patients, or gain market acceptance of Alexion’s products in the future, which could prevent it from maintaining profitability or growth.
Alexion cannot be certain that Soliris will maintain market acceptance in a particular country among physicians, patients, health care payers, and others. Although Alexion has received regulatory approval for Soliris in certain territories, including the United States, Japan and the European Union, such approvals do not guarantee future revenue. Alexion cannot predict whether physicians, other health care providers, government agencies or private insurers will determine or continue to accept that Soliris is safe and therapeutically effective relative to its cost. Physicians’ willingness to prescribe, and patients’ willingness to accept, Alexion’s products, such as Soliris, depends on many factors, including prevalence and severity of
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adverse side effects in both clinical trials and commercial use, the timing of the market introduction of competitive drugs, lower demonstrated clinical safety and efficacy compared to other drugs, perceived lack of cost-effectiveness, pricing and lack of availability of reimbursement from third-party payers, convenience and ease of administration, effectiveness of Alexion’s marketing strategy, publicity concerning the product, Alexion’s other product candidates or competing products, and availability of alternative treatments, including bone marrow transplant as an alternative treatment for PNH. The likelihood of physicians to prescribe Soliris for patients with aHUS may also depend on how quickly Soliris can be delivered to the hospital or clinic and Alexion’s distribution methods may not be sufficient to satisfy this need. In addition, Alexion is aware that medical doctors have determined not to continue Soliris treatment for some patients with aHUS.
Health insurance programs may restrict coverage of some products by using payer formularies under which only selected drugs are covered, variable co-payments that make drugs that are not preferred by the payer more expensive for patients, and by using utilization management controls, such as requirements for prior authorization or failure on another type of treatment. Payers may especially impose these obstacles to coverage for higher-priced drugs, and consequently Alexion’s drug products may be subject to payer-driven restrictions. In addition, in some foreign countries, the proposed pricing for a drug must be approved before it may be lawfully marketed. The requirements governing drug pricing vary widely from country to country. For example, European Union member states may restrict the range of medicinal products for which their national health insurance systems provide reimbursement and to control the prices and/or reimbursement of medicinal products for human use. A European Union member state may approve a specific price or level of reimbursement for the medicinal product, or it may instead adopt a system of direct or indirect controls on the profitability of the company placing the medicinal product on the market. The reimbursement or budget identified by a government or non-government payer for Alexion’s products, including Soliris in a new indication, if obtained, may be adversely affected by the reimbursement or budget for Soliris in previously approved indications and/or adversely affect the reimbursement or budget for Soliris in such previously approved indication by that payer.
If Soliris fails to achieve or maintain market acceptance among the medical community or patients in a particular country, Alexion may not be able to market and sell it successfully in such country, which would limit Alexion’s ability to generate revenue and could harm Alexion’s overall business.
If Alexion or any third party manufacturer or provider fails to provide sufficient quantities of Soliris or Alexion’s product candidates, including Soliris for new indications, Alexion could experience product shortages, its commercialization of Soliris may be stopped or delayed, its clinical trials could be disrupted or regulatory approvals could be delayed.
Soliris is manufactured by Alexion at Alexion’s Rhode Island manufacturing facility (“ARIMF”) and by Lonza Group Ltd. (“Lonza”). Alexion depends on a very limited number of third party providers for the manufacture and supply of Soliris and Alexion’s product candidates. The manufacture of Soliris and Alexion’s product candidates is difficult, requiring a multi-step controlled process and even minor problems or deviations could result in defects or failures. Manufacture of Alexion’s products, including Soliris, is highly technical, and only a small number of companies have the ability and capacity to manufacture Alexion’s products for its development and commercialization needs. Due to the highly technical requirements of manufacturing Alexion’s products and the strict quality and control specifications, Alexion and its third party providers may be unable to manufacture or supply Alexion’s products despite Alexion’s and their efforts. In addition, Alexion cannot be certain that any third party will be able or willing to honor the terms of its agreement, including any obligations to manufacture Alexion’s products in accordance with regulatory requirements and to Alexion’s quality specifications and volume requirements.
Alexion cannot be certain that it, Lonza or Alexion’s other third party providers will be able to perform uninterrupted supply chain services. The failure to manufacture appropriate supplies of Soliris, on a timely basis, or at all, may prevent or interrupt the commercialization of Soliris. If Alexion, Lonza or Alexion’s other third party providers were unable to manufacture Soliris for any period for any reason, including due to the loss of approvals, or if Alexion, Lonza or Alexion’s other third party providers do not obtain approval for the manufacturing of Soliris in the respective facility by the applicable regulatory agencies, Alexion may incur substantial loss of sales. See also Alexion’s Risk Factor “If Alexion or its
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contract manufacturers fail to comply with continuing United States and foreign regulations, Alexion could lose its approvals to market Soliris or Alexion’s manufacturers could lose their approvals to manufacture Soliris or Alexion’s product candidates, and Alexion’s business would be seriously harmed.” Alexion may also lose any redundancy in its manufacturing capabilities if it is no longer able to perform operations at ARIMF or any other facility. The failure to manufacture appropriate supplies of Alexion’s product candidates, on a timely basis, or at all, may prevent or interrupt clinical development of Alexion’s products, including Soliris for new indications. If Alexion is forced to find an alternative supplier or other third party providers, in addition to loss of sales and disruption to patients, Alexion may also incur significant costs and experience significant delay in establishing a new arrangement.
Alexion is authorized to sell Soliris that is manufactured by Lonza and at ARIMF in the United States, the European Union, Japan and certain other territories. However, manufacturing Soliris for commercial sale in certain other territories may only be performed at a single facility until such time as Alexion has received the required regulatory approval for an additional facility, if ever. Alexion will continue to depend entirely on one facility to manufacture Soliris for commercial sale in such other territories until that time.
Alexion has obtained marketing approval for Soliris for the treatment of patients with aHUS in the United States, the European Union, Japan and other territories. Alexion expects that the demand for Soliris will increase. Alexion may underestimate demand, or experience product interruptions at ARIMF, Lonza or a facility of a third party provider, including as a result of risks and uncertainties described in this report. If Alexion, Lonza or Alexion’s other third party providers do not manufacture sufficient quantities of Soliris to satisfy demand, Alexion’s business will be materially harmed.
Alexion depends on a very limited number of third party providers for other services with respect to its clinical and commercial requirements, including product filling, finishing, packaging, and labeling. Alexion has changed or added third party fill/finish providers in the past in order to support uninterrupted supply, and may do so in the future. Alexion currently relies on three third party fill/finish providers to support its commercial requirements in the United States and the European Union, and two to support requirements in Japan. No guarantee can be made that regulators will approve additional third party fill/finish providers in a timely manner or at all, or that any third party fill/finish providers will be able to perform such services for sufficient product volumes for any country or territory. Alexion does not have control over any third party provider’s compliance with Alexion’s internal or external specifications or the rules and regulations of the FDA, EMA, competent authorities of the European Union member states, MHLW or any other applicable regulations or standards. In the past, Alexion has had to write off and incur other charges and expenses for production that failed to meet requirements, including with respect to recalls initiated in 2013 and 2014.
Any difficulties or delays in Alexion’s third party manufacturing of Soliris, or any failure of Alexion’s third party providers to comply with Alexion’s internal and external specifications or any applicable rules, regulations and standards could increase Alexion’s costs, constrain its ability to satisfy demand for Soliris from customers, cause Alexion to lose revenue or incur penalties for failure to deliver product, make Alexion postpone or cancel clinical trials, or cause Alexion’s products to be recalled or withdrawn, such as the voluntary recalls that Alexion initiated in 2013 and 2014 due to the presence of visible particles in a limited number of vials in specific lots. Even if Alexion is able to find alternatives they may ultimately be insufficient for Alexion’s needs.
Due to the nature of the current market for third-party commercial manufacturing, many arrangements require substantial penalty payments by the customer for failure to use the manufacturing capacity for which it contracted. Penalty payments under these agreements typically decrease over the life of the agreement, and may be substantial initially and de minimis or non-existent in the final period. The payment of a substantial penalty could harm Alexion’s financial condition.
In April 2014, Alexion acquired a fill/finish facility in Ireland to support global distribution of Soliris and Alexion’s other clinical and commercial products. To date, Alexion has relied entirely on third party fill/finish providers and have never operated Alexion’s own fill/finish facility. Alexion cannot guarantee that it will be able to successfully complete the appropriate validation processes or obtain the necessary regulatory approvals, or that it will be able to perform fill/finish services at this facility to support its product requirements.
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Many additional factors could cause production interruptions at ARIMF or at the facilities of Lonza or Alexion’s third party providers, including natural disasters, labor disputes, acts of terrorism or war, human error, equipment malfunctions, contamination, or raw material shortages. The occurrence of any such event could adversely affect Alexion’s ability to satisfy demand for Soliris, which could materially and adversely affect Alexion’s operating results.
If Alexion or its contract manufacturers fail to comply with United States and foreign regulations, Alexion or its manufacturers could lose Alexion’s approvals to market Soliris or Alexion’s product candidates, and Alexion’s business would be seriously harmed.
Alexion cannot guarantee that it will be able to maintain its regulatory approvals for Soliris. If Alexion does not maintain its regulatory approvals for Soliris, the value of Alexion’s company and its results of operations will be materially harmed. Alexion and its current and future partners, contract manufacturers and suppliers are subject to rigorous and extensive regulation by governmental authorities around the world, including the FDA, EMA, the competent authorities of the European Union member states, and MHLW. If Alexion or a regulatory agency discovers previously unknown problems with a product, such as adverse events of unanticipated severity or frequency, or problems with the facility where the product is manufactured, a regulatory agency may impose restrictions on that product, the manufacturing facility or us. For example, in March 2013, Alexion received the Warning Letter from the FDA relating to compliance with cGMP at ARIMF. In August 2014 Alexion announced that it received a Form 483 with three observations following an FDA inspection at ARIMF. If Alexion does not resolve outstanding concerns expressed by the FDA in the Warning Letter and the August 2014 Form 483 to the satisfaction of the FDA, EMA or any other regulatory agency, or Alexion or its third-party providers, including Alexion’s product fill/finish providers, packagers and labelers, fail to comply fully with applicable regulations then Alexion may be required to initiate a recall or withdrawal of its products.
The safety profile of any product continues to be closely monitored by the FDA and comparable foreign regulatory authorities after approval. Regulations continue to apply after product approval, and cover, among other things, testing, manufacturing, quality control, finishing, filling, labeling, advertising, promotion, risk mitigation, adverse event reporting requirements, and export of biologics. For example, the risk management program established in 2007 upon the FDA’s approval of Soliris for the treatment of PNH was replaced with a Risk Evaluation and Mitigation Strategy (“REMS”) program, approved by the FDA in 2010. The REMS program requires mandatory physician certification in the United States. Each physician must certify that the physician is aware of the potential risks associated with the administration of Soliris and that the physician will inform each patient of these risks using educational material approved by the FDA. In November 2014, Alexion met with the FDA Drug Safety and Risk Management Advisory Committee to discuss adjustments to the REMS with elements to assure safe use. A majority of the Committee favored revising the REMS and made suggestions for streamlining prescriber assessments and broadening the program’s educational outreach. Changes to the Soliris REMS could be costly and burdensome to implement.
As a condition of approval for marketing Soliris, governmental authorities may require Alexion to conduct additional studies. For example, in connection with the approval of Soliris in the United States, European Union and Japan, for the treatment of PNH, Alexion agreed to establish a PNH Registry, monitor immunogenicity, monitor compliance with vaccination requirements, and determine the effects of anticoagulant withdrawal among PNH patients receiving eculizumab, and, specifically in Japan, Alexion agreed to conduct a trial in a limited number of Japanese PNH patients to evaluate the safety of a meningococcal vaccine. Further, in connection with the approval of Soliris in the United States for the treatment of aHUS, Alexion agreed to establish an aHUS Registry and complete additional human clinical studies in adult and pediatric patients. In the United States, for example, the FDA can propose to withdraw approval for a product if it determines that such additional studies are inadequate or if new clinical data or information shows that a product is not safe for use in an approved indication. Alexion is required to report any serious and unexpected adverse experiences and certain quality problems with Soliris to the FDA, the EMA, the competent authorities of the European Union member states, MHLW, and certain other health agencies. Alexion or any health agency may have to notify health care providers of any such developments.
The discovery of any previously unknown problems with Soliris, a manufacturer or a facility may result in restrictions on Soliris, a manufacturer or a facility, including withdrawal of Soliris from the
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market, batch failures, or interruption of production or a product recall such as the recalls Alexion announced and voluntarily initiated in 2013 and 2014. Certain changes to an approved product, including the way it is manufactured or promoted, often require prior regulatory approval before the product as modified may be marketed. Alexion’s manufacturing and other facilities and those of any third parties manufacturing Soliris will be subject to inspection prior to grant of marketing approval by each regulatory authority where Alexion seeks marketing approval and subject to continued review and periodic inspections by the regulatory authorities, such as the inspections that resulted in issuance of the Warning Letter. Alexion and any third party Alexion would use to manufacture Soliris for sale, including Lonza, must also be licensed by applicable regulatory authorities.
The FDA requires reporting of certain information on side effects and adverse events reported during clinical studies and after marketing approval. Non-compliance with safety reporting requirements could result in regulatory action that may include civil action or criminal penalties.
Failure to comply with the laws and requirements, including statutes and regulations, administered by the FDA, the EMA, the competent authorities of the European Union member states, the MHLW or other agencies, including without limitation, failures or delays in resolving the concerns raised by the FDA in the Warning Letter, could result in:

a product recall;

a product withdrawal;

significant administrative and judicial sanctions, including, warning letters or untitled letters;

significant fines and other civil penalties;

suspension, variation or withdrawal of a previously granted approval for Soliris;

interruption of production;

operating restrictions, such as a shutdown of production facilities or production lines, or new manufacturing requirements;

suspension of ongoing clinical trials;

delays in approving or refusal to approve Alexion’s products including pending BLAs or BLA supplements for Soliris or asfotase alfa, or a facility that manufactures Alexion’s products;

seizing or detaining product;

requiring Alexion or Alexion’s partners to enter into a consent decree, which can include imposition of various fines, reimbursements for inspection costs, required due dates for specific actions and penalties for noncompliance;

injunctions; and/or

criminal prosecution.
If the use of Soliris harms people, or is perceived to harm patients even when such harm is unrelated to Soliris, Alexion’s regulatory approvals could be revoked or otherwise negatively impacted and Alexion could be subject to costly and damaging product liability claims.
The testing, manufacturing, marketing and sale of drugs for use in humans exposes Alexion to product liability risks. Side effects and other problems from using Soliris could (1) lessen the frequency with which physicians decide to prescribe Soliris, (2) encourage physicians to stop prescribing Soliris to their patients who previously had been prescribed Soliris, (3) cause serious adverse events and give rise to product liability claims against Alexion, and (4) result in Alexion’s need to withdraw or recall Soliris from the marketplace. Some of these risks are unknown at this time.
Alexion tested Soliris in only a small number of patients. The FDA marketing approval for the treatment of patients with aHUS was based on two prospective studies in a total of 37 adult and adolescent patients, together with a retrospective study that included 19 pediatric patients. PNH and aHUS are ultra-rare diseases. As more patients use Soliris, including more children and adolescents, new risks and side
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effects may be discovered, the rate of known risks or side effects may increase, and risks previously viewed as less significant could be determined to be significant. Previously unknown risks and adverse effects of Soliris may also be discovered in connection with unapproved uses of Soliris, which may include administration of Soliris under acute emergency conditions, such as the Enterohemorrhagic E. coli health crisis in Europe, primarily Germany, that began in May 2011. Alexion does not promote, or in any way support or encourage the promotion of Soliris for unapproved uses in violation of applicable law, but physicians are permitted to use products for unapproved purposes and Alexion is aware of such uses of Soliris. In addition, Alexion is studying and expects to continue to study Soliris in diseases other than PNH and aHUS in controlled clinical settings, and independent investigators are doing so as well. In the event of any new risks or adverse effects discovered as new patients are treated for approved indications and as Soliris is studied in or used by patients for other indications, regulatory authorities may delay or revoke their approvals, Alexion may be required to conduct additional clinical trials and safety studies, make changes in labeling of Soliris, reformulate Soliris or make changes and obtain new approvals for Alexion’s and its suppliers’ manufacturing facilities. Alexion may also experience a significant drop in the potential sales of Soliris, experience harm to its reputation and the reputation of Soliris in the marketplace or become subject to lawsuits, including class actions. Any of these results could decrease or prevent any sales of Soliris or substantially increase the costs and expenses of commercializing and marketing Soliris.
Alexion may be sued by people who use Soliris, whether as a prescribed therapy, during a clinical trial, during an investigator initiated study, or otherwise. Many patients who use Soliris are already very ill. Any informed consents or waivers obtained from people who enroll in Alexion’s trials or use Soliris may not protect Alexion from liability or litigation. Alexion’s product liability insurance may not cover all potential types of liabilities or may not cover certain liabilities completely. Moreover, Alexion may not be able to maintain its insurance on acceptable terms. In addition, negative publicity relating to the use of Soliris or a product candidate, or to a product liability claim, may make it more difficult, or impossible, for Alexion to market and sell Soliris. As a result of these factors, a product liability claim, even if successfully defended, could have a material adverse effect on Alexion’s business, financial condition or results of operations.
Patients who use Soliris already often have severe and advanced stages of disease and known as well as unknown significant pre-existing and potentially life-threatening health risks, including, for example, bone marrow failure, kidney failure and thrombosis. During the course of treatment, patients may suffer adverse events, including death, for reasons that may or may not be related to Soliris. Such events could subject Alexion to costly litigation, require it to pay substantial amounts of money to injured patients, delay, negatively impact or end Alexion’s opportunity to receive or maintain regulatory approval to market Soliris, or require Alexion to suspend or abandon its commercialization efforts. Even in a circumstance in which Alexion does not believe that an adverse event is related to Soliris, the investigation into the circumstance may be time consuming or inconclusive. These investigations may interrupt Alexion’s sales efforts, delay its regulatory approval process in other countries, or impact and limit the type of regulatory approvals Soliris receives or maintains.
Some patients treated with Soliris for PNH and other diseases, including patients who have participated in Alexion’s clinical trials, have died or suffered potentially life-threatening diseases either during or after ending their Soliris treatments. In particular, use of C5 Inhibitors, such as Soliris, is associated with an increased risk for certain types of infection, including meningococcal infection. Serious cases of meningococcal infection can result in severe illness, including but not limited to brain damage, loss of limbs or parts of limbs, kidney failure, or death. Under controlled settings, patients in Alexion’s eculizumab trials all receive vaccination against meningococcal infection prior to first administration of Soliris and patients who are prescribed Soliris in most countries are required by prescribing guidelines to be vaccinated prior to receiving their first dose. A physician may not have the opportunity to timely vaccinate a patient in the event of an acute emergency episode, such as in a patient presenting with aHUS or during the health crisis that began in May 2011 in Europe, principally in Germany, due to the epidemic of infections from Enterohemorrhagic E. coli. Vaccination does not, however, eliminate all risk of meningococcal infection. Additionally, in some countries there may not be any vaccine approved for general use or approved for use in infants and children. Some patients treated with Soliris who had been vaccinated have nonetheless experienced meningococcal infection, including patients who have suffered serious illness or death. Each such incident is required to be reported to appropriate regulatory agencies in accordance with relevant regulations.
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Alexion is also aware of a potential risk for PNH patients who delay a dose of Soliris or discontinue their treatment of Soliris. Treatment with Soliris blocks complement and allows complement-sensitive PNH red blood cells to increase in number. If treatment with Soliris is thereafter delayed or discontinued, a greater number of red blood cells therefore would become susceptible to destruction when the patient’s complement system is no longer blocked. The rapid destruction of a larger number of a patient’s red blood cells may lead to numerous complications, including death. Several PNH patients in Alexion’s studies of Soliris have received delayed doses or discontinued their treatment. In none of those circumstances were significant complications shown to be due to rapid destruction of a larger number of PNH red blood cells; however, Alexion has not studied the delay or termination of treatment in enough patients to determine that such complications in the future are unlikely to occur. Additionally, such delays or discontinuations may be associated with significant complications without evidence of such rapid cell destruction.
Alexion is aware of a risk for aHUS patients who delay or miss a dose of Soliris or discontinue their treatment of Soliris. Treatment with Soliris blocks complement and inhibits complement-mediated Thrombotic microangiopathy (“TMA”). After missing a dose or discontinuing Soliris, blood clots may form in small blood vessels throughout the body, causing a reduction in platelet count. The reduction in platelet count may lead to numerous complications, including changes in mental status, seizures, angina, thrombosis, renal failure or even death. In Alexion’s aHUS clinical studies, such TMA complications were observed in some patients who missed a dose.
Clinical evaluations of outcomes in the post-marketing setting are required to be reported to appropriate regulatory agencies in accordance with relevant regulations. Determination of significant complications associated with the delay or discontinuation of Soliris could have a material adverse effect on Alexion’s ability to sell Soliris.
If Alexion is unable to establish and maintain effective sales, marketing and distribution capabilities, or to enter into agreements with third parties to do so, Alexion will be unable to successfully commercialize Soliris.
Alexion is marketing and selling Soliris ourselves in the United States, Europe, Japan and several other territories. If Alexion is unable to establish and/or expand its capabilities to sell, market and distribute Soliris for the treatment of PNH, aHUS or, if approved by the necessary regulatory agencies, other future indications, either through Alexion’s own capabilities or by entering into agreements with others, or to maintain such capabilities in countries where Alexion has already commenced commercial sales, Alexion will not be able to successfully sell Soliris. In that event, Alexion will not be able to generate significant revenues. Alexion cannot guarantee that it will be able to establish and maintain its own capabilities or enter into and maintain any marketing or distribution agreements with third-party providers on acceptable terms, if at all. Even if Alexion hires the qualified sales and marketing personnel it needs to support its objectives, or enter into marketing and distribution agreements with third parties on acceptable terms, Alexion may not do so in an efficient manner or on a timely basis. Alexion may not be able to correctly judge the size and experience of the sales and marketing force and the scale of distribution capabilities necessary to successfully market and sell Soliris. Establishing and maintaining sales, marketing and distribution capabilities are expensive and time-consuming. Alexion’s expenses associated with building up and maintaining the sales force and distribution capabilities around the world may be disproportionate compared to the revenues Alexion may be able to generate on sales of Soliris. Alexion cannot guarantee that it will be successful in commercializing Soliris.
If Alexion markets Soliris in a manner that violates health care fraud and abuse laws and other laws regulating marketing and promotion, Alexion may be subject to investigations and civil or criminal penalties.
In addition to FDA and related regulatory requirements, Alexion is subject to health care “fraud and abuse” laws, such as the federal False Claims Act (“FCA”), the anti-kickback provisions of the federal Social Security Act, and other state and federal laws and regulations. The federal Anti-Kickback Statute prohibits, among other things, knowingly and willfully offering, paying, soliciting or receiving any remuneration, directly or indirectly, in cash or in kind to induce, or reward the purchasing, leasing, ordering or arranging for or recommending the purchase, lease or order of any health care item or service reimbursable under Medicare, Medicaid, or other federal health care programs. This statute has been interpreted to apply broadly to arrangements between pharmaceutical manufacturers on the one hand and
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prescribers, patients, purchasers and formulary managers on the other. Liability may be established without a person or entity having actual knowledge of the federal Anti-Kickback Statute or specific intent to violate it. In addition, the Patient Protection and Affordable Care Act, as amended by the Health Care and Education Reconciliation Act of 2010 (collectively, the “PPACA”), amended the Social Security Act to provide that the government may assert that a claim including items or services resulting from a violation of the federal anti-kickback statute constitutes a false or fraudulent claim for purposes of the FCA. A conviction for violation of the Anti-Kickback Statute requires mandatory exclusion from participation in federal health care programs. Although there are a number of statutory exemptions and regulatory safe harbors protecting certain common activities from prosecution, the exemptions and safe harbors are drawn narrowly, and practices that involve remuneration to those who prescribe, purchase, or recommend pharmaceutical products, including certain discounts, education and research grants, purchase of speaking or consulting services, and patient assistance programs, may be subject to scrutiny or penalty if they do not qualify for an exemption or safe harbor. Alexion seeks to comply with the anti-kickback laws and with the available statutory exemptions and safe harbors. However, Alexion’s practices may not in all cases fit within the safe harbors, and Alexion’s practices may therefore be subject to case-by-case scrutiny.
The FCA prohibits any person from knowingly presenting, or causing to be presented, a false or fraudulent claim for payment of government funds, or knowingly making, using or causing to be made or used, a false record or statement material to a false or fraudulent claim. Pharmaceutical companies have been investigated and have reached substantial financial settlements with the Federal government under the FCA for a variety of alleged promotional and marketing activities, such as allegedly providing free product to customers with the expectation that the customers would bill federal programs for the product; providing consulting fees and other benefits to physicians to induce them to prescribe products; reporting inflated prices to private publications that were then used by federal programs to set reimbursement rates; engaging in promotion for uses that the FDA has not approved, or “off-label” uses that caused claims to be submitted to Federal programs for non-covered off-label uses; and submitting inflated best price information to the Medicaid Rebate Program.
The majority of states also have statutes similar to the federal anti-kickback law and false claims laws that apply to items and services reimbursed under Medicaid and other state programs, or, in several states, apply regardless of the payer. In addition, several U.S. states and localities have enacted legislation requiring pharmaceutical companies to establish marketing compliance programs, file periodic reports with the state or make periodic public disclosures on sales, marketing, pricing, clinical trials, and other activities. Some state laws prohibit certain marketing-related activities including the provision of gifts, meals or other items to certain health care providers. Similar legislation is being considered in other states. Additionally, PPACA enacted the Physician Payment Sunshine Act, being implemented as the Open Payments program, that requires manufacturers to track and report to the federal government, for public dissemination, payments and other transfers of value made to physicians and teaching hospitals. Many of these requirements are new and there is limited guidance on many aspects of how they will be interpreted, implemented and enforced. Nonetheless, if Alexion is found not to be in full compliance with these laws, Alexion could face enforcement action and fines and other penalties, and could receive adverse publicity.
Sanctions under these federal and state fraud and abuse laws may include civil monetary penalties, exclusion of a manufacturer’s products from reimbursement under government programs, monetary damages, criminal fines, and imprisonment. Efforts to ensure that Alexion’s business arrangements continue to comply with applicable healthcare laws and regulations could be costly. Because of the breadth of these laws and the narrowness of the safe harbors and because government scrutiny in this area is high, it is possible that some of Alexion’s business activities could come under that scrutiny. Even if Alexion is not determined to have violated these laws, government investigations into these issues typically require the expenditure of significant resources and generate negative publicity, which could also harm Alexion’s financial condition. Responding to government investigations or whistleblower lawsuits, defending any claims raised, and any resulting fines, damages, penalties, settlement payments or administrative actions, as well as any related actions brought by stockholders or other third parties, could have a material impact on Alexion’s reputation, business and financial condition and divert the attention of Alexion’s management from operating Alexion’s business.
Although physicians in the United States are permitted to, based on their medical judgment, prescribe products for indications other than those cleared or approved by the FDA, manufacturers are prohibited
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from promoting their products for such off-label uses. In the United States, Alexion markets Soliris for PNH and aHUS and provide promotional materials and training programs to physicians regarding the use of Soliris for PNH and aHUS. Although Alexion believe its marketing materials and training programs for physicians do not constitute off-label promotion of Soliris, the FDA, the U.S. Department of Justice (the “DOJ”), or other federal or state government agencies may disagree. If the FDA or other government agencies determine that Alexion’s promotional materials, training or other activities constitute off-label promotion of Soliris, it could request that Alexion modify its training or promotional materials or other activities or subject Alexion to regulatory enforcement actions, including the issuance of a warning letter, injunction, seizure, civil fine and criminal penalties. It is also possible that other federal or state enforcement authorities might take action if they believe that the alleged improper promotion led to the submission and payment of claims for an unapproved use, which could result in significant fines or penalties under other statutory authorities, such as laws prohibiting false or fraudulent claims for payment of government funds. Even if it is later determined Alexion is not in violation of these laws, Alexion may be faced with negative publicity, incur significant expenses defending its position and have to divert significant management resources from other matters.
Similar strict restrictions are imposed on the promotion and marketing of drug products in the European Union, where a large portion of Alexion’s non-U.S. business is conducted, and other territories. Laws in the European Union, including in the individual European Union member states, require promotional materials and advertising for drug products to comply with the product’s Summary of Product Characteristics (“SmPC”), which is approved by the competent authorities. Promotion of a medicinal product which does not comply with the SmPC is considered to constitute off-label promotion. The off-label promotion of medicinal products is prohibited in the European Union and in other territories. The promotion of medicinal products that are not subject to a marketing authorization is also prohibited in the European Union. Laws in the European Union, including in the individual European Union member states, also prohibit the direct-to-consumer advertising of prescription-only medicinal products. Violations of the rules governing the promotion of medicinal products in the European Union and in other territories could be penalized by administrative measures, fines and imprisonment.
Interactions between pharmaceutical companies and physicians are also governed by strict laws, regulations, industry self-regulation codes of conduct and physicians’ codes of professional conduct in the individual European Union member states. The provision of any inducements to physicians to prescribe, recommend, endorse, order, purchase, supply, use or administer a medicinal product is prohibited. A number of European Union member states have introduced additional rules requiring pharmaceutical companies to publicly disclose their interactions with physicians and to obtain approval from employers, professional organizations and/or competent authorities before entering into agreements with physicians. These rules have been supplemented by provisions of related industry codes. Additional countries may consider or implement similar laws and regulations. Violations of these rules could lead to reputational risk, public reprimands, and/or the imposition of fines or imprisonment.
If Alexion fails to comply with the Foreign Corrupt Practices Act or other similar legal requirements, Alexion may be subject to criminal and civil penalties and other remedial measures, which could have a material adverse effect on Alexion’s reputation, business, results of operations or financial condition.
Alexion is subject to the United States Foreign Corrupt Practices Act (“FCPA”), the U.K. Bribery Act, and other anti-corruption laws and regulations that generally prohibit companies and their intermediaries from offering or making improper payments to government officials and/or other persons for the purpose of obtaining or retaining business. Worldwide regulators are increasing their regulatory and enforcement efforts in this area. For example, the Bribery Act in the United Kingdom, effective as of July 2011 applies to any company incorporated in or “carrying on business” in the United Kingdom, regardless of the country in which the alleged bribery activity occurs and even if the inappropriate activity is undertaken by Alexion’s international distribution partners. Alexion’s policies mandate compliance with these anti-bribery laws. Alexion may operate in many parts of the world that are recognized as having a greater potential for governmental and commercial corruption. Alexion cannot assure that its policies and procedures will always protect it from reckless or criminal acts committed by its employees or third-party intermediaries.
Recent years have seen a substantial increase in anti-bribery law enforcement activity by U.S. regulators, with more frequent and aggressive investigations, enforcement proceedings and sanctions by
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both the DOJ and the SEC, including with respect to the pharmaceutical industry, increased enforcement activity by non-U.S. regulators and increases in criminal and civil proceedings brought against companies and individuals. Increasing regulatory scrutiny of the promotional activities of pharmaceutical companies also has been observed in a number of European Union member states.
Laws, including those governing promotion, marketing and anti-kickback/anti-bribery provisions, and industry regulations are often strictly enforced. In the United States, additional governmental resources are being added to enforce these laws and to prosecute companies and individuals believed to be violating them. For example, PPACA included a number of provisions aimed at strengthening the government’s ability to pursue anti-kickback and false claims cases against pharmaceutical manufacturers and other healthcare entities, including substantially increased funding for healthcare fraud enforcement activities, enhanced investigative powers for government authorities, and amendments to the FCA that make it easier for the government and whistleblowers to pursue cases for alleged kickback and false claim violations. Alexion anticipates that government scrutiny of pharmaceutical sales and marketing practices will continue for the foreseeable future and continue to subject Alexion to the risk of government investigations and whistleblower lawsuits. If Alexion fails to comply with laws governing promotion, marketing and anti-kickback/anti-bribery provisions, Alexion may be subject to criminal and civil penalties and other remedial measures, which could have a material adverse effect on its reputation, business, results of operations or financial condition.
On May 8, 2015, Alexion received a subpoena in connection with an investigation by the Enforcement Division of the SEC requesting information related to Alexion’s grant-making activities and compliance with the FCPA. While the subpoena seeks information related to Alexion’s activities and policies and procedures worldwide, it notes in particular Japan, Brazil, Turkey and Russia. The subpoena also seeks information related to Alexion’s recalls of specific lots of Soliris and related securities disclosures. Alexion is committed to compliance with applicable laws and regulations and strives to operate at the highest ethical standards in all of its markets. Alexion is cooperating with the SEC’s investigation, which is in its early stages. At this time, Alexion is unable to predict the duration, scope or outcome of the SEC investigation.
Any determination that Alexion’s operations or activities are not, or were not, in compliance with existing United States or foreign laws or regulations, including by the SEC pursuant to its investigation of Alexion’s compliance with the FCPA and other matters, could result in the imposition of a broad range of civil and criminal sanctions against Alexion and certain of its directors, officers and/or employees, including injunctive relief, disgorgement, substantial fines or penalties, imprisonment, interruptions of business, debarment from government contracts, loss of supplier, vendor or other third-party relationships, termination of necessary licenses and permits, and other legal or equitable sanctions. Other internal or government investigations or legal or regulatory proceedings, including lawsuits brought by private litigants, may also follow as a consequence. Violations of these laws may result in criminal or civil sanctions, which could disrupt Alexion’s business and result in a material adverse effect on its reputation, business, results of operations or financial condition. Cooperating with and responding to the SEC in connection with its investigation of Alexion’s FCPA practices and other matters, as well as responding to any future U.S. or foreign governmental investigation or whistleblower lawsuit, could result in substantial expenses, and could divert management’s attention from other business concerns and could have a material adverse effect on Alexion’s business and financial condition and growth prospects.
None of Alexion’s product candidates except for Soliris has received regulatory approvals. Soliris has not been approved for any indication other than for the treatment of patients with PNH and aHUS. If Alexion is unable to obtain regulatory approvals to market one or more of its product candidates, including asfotase alfa and Soliris for other indications, Alexion’s business may be adversely affected.
All of Alexion’s product candidates except Soliris and asfotase alfa are in early stages of development, and Alexion does not expect its early stage product candidates to be commercially available for several years, if at all. Although Alexion is preparing for a commercial launch of Strensiq for the treatment of hypophosphatasia, Alexion does not know when or if Strensiq will be approved by the FDA, EMA or any other regulatory agency. Alexion completed a rolling submission of its BLA for Strensiq in the U.S., which allowed completed portions of the application to be submitted and reviewed by the FDA on an ongoing basis. While the FDA accepted the application in March 2015, Alexion cannot predict how long the
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approval process will take or when Alexion will receive approval, if at all. Alexion does not know when or if Alexion’s other product candidates will be approved. Unfavorable clinical trial results, failure to comply with regulatory requirements, resolve pending concerns described in the Warning Letter, and inadequate manufacturing processes are examples of problems that could prevent approval. In addition, Alexion may encounter delays or rejections due to additional government regulation from future legislation, administrative action or changes in the FDA policy. Even if the FDA approves a product, the approval will be limited to those indications covered in the approval.
Outside the United States, Alexion’s ability to market any of its potential products is dependent upon receiving marketing approvals from the appropriate regulatory authorities. These foreign regulatory approval processes include all of the risks associated with the FDA approval process described above. If Alexion is unable to receive regulatory approvals, it will be unable to commercialize its product candidates, and Alexion’s business may be adversely affected.
Completion of preclinical studies or clinical trials does not guarantee advancement to the next phase of development.
Completion of preclinical studies or clinical trials does not guarantee that Alexion will initiate additional studies or trials for Alexion’s product candidates, that if further studies or trials are initiated what the scope and phase of the trial will be or that they will be completed, or that if these further studies or trials are completed, that the design or results will provide a sufficient basis to apply for or receive regulatory approvals or to commercialize products. Results of clinical trials could be inconclusive, requiring additional or repeat trials. Data obtained from preclinical studies and clinical trials are subject to varying interpretations that could delay, limit or prevent regulatory approval. Data that Alexion believes is highly clinically significant, including the results of Alexion’s HPP trials, could be interpreted differently by the FDA or other regulatory agencies. The results generated in clinical studies of Strensiq which Alexion believes to be positive, do not ensure that the product will be approved and the FDA or other regulatory agency could require additional preclinical or clinical data. If the design or results achieved in Alexion’s clinical trials are insufficient to proceed to further trials or to regulatory approval of Alexion’s product candidates, the company could be materially adversely affected. Failure of a clinical trial to achieve its pre-specified primary endpoint, such as the Phase II Soliris trial for AMR that Alexion announced in January 2015, generally increases the likelihood that additional studies or trials will be required if Alexion determine to continue development of the product candidate, reduces the likelihood of timely development of and regulatory approval to market the product candidate, and may decrease the chances for successfully achieving the primary endpoint in scientifically similar indications.
There are many reasons why drug testing could be delayed or terminated.
For human trials, patients must be recruited and each product candidate must be tested at various doses and formulations for each clinical indication. In addition, to ensure safety and effectiveness, the effect of drugs often must be studied over a long period of time, especially for the chronic diseases that Alexion is studying. Many of Alexion’s programs focus on diseases with small patient populations and insufficient patient enrollment in Alexion’s clinical trials could delay or cause Alexion to abandon a product development program. Alexion may decide to abandon development of a product candidate at any time due to unfavorable results or other reasons, or Alexion may have to spend considerable resources repeating clinical trials or conducting additional trials, either of which would increase costs and delay any revenue from those product candidates, if any.
Additional factors that can cause delay, impairment or termination of Alexion’s clinical trials or product development efforts include:

delay or failure in obtaining institutional review board (“IRB”), approval or the approval of other reviewing entities to conduct a clinical trial at each site;

delay or failure in reaching agreement on acceptable terms with prospective contract research organizations (“CROs”), and clinical trial sites, the terms of which can be subject to extensive negotiation and may vary significantly among different CROs and trial sites;
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withdrawal of clinical trial sites from Alexion’s clinical trials as a result of changing standards of care or the ineligibility of a site to participate in Alexion’s clinical trials;

clinical sites and investigators deviating from trial protocol, failing to conduct the trial in accordance with regulatory requirements, or dropping out of a trial;

slow patient enrollment, including, for example, due to the rarity of the disease being studied;

delay or failure in having patients complete a trial or return for post-treatment follow-up;

long treatment time required to demonstrate effectiveness;

lack of sufficient supplies of the product candidate;

disruption of operations at the clinical trial sites;

adverse medical events or side effects in treated patients, and the threat of legal claims and litigation alleging injuries;

failure of patients taking the placebo to continue to participate in Alexion’s clinical trials;

insufficient clinical trial data to support effectiveness of the product candidates;

lack of effectiveness or safety of the product candidate being tested;

lack of sufficient funds;

inability to meet required specifications or to manufacture sufficient quantities of the product candidate for development or commercialization activities in a timely and cost-efficient manner;

decisions by regulatory authorities, the IRB, ethics committee, or Alexion, or recommendation by a data safety monitoring board, to suspend or terminate clinical trials at any time for safety issues or for any other reason;

failure to obtain the necessary regulatory approvals for the product candidate or the approvals for the facilities in which such product candidate is manufactured; and

decisions by competent authorities, IRBs or ethics committees to demand variations in protocols or conduct of clinical trials.
The regulatory approval process is costly and lengthy and Alexion may not be able to successfully obtain all required regulatory approvals.
In March 2015, the FDA accepted Alexion’s application for Strensiq as a treatment for patients with HPP. In July 2014, the MAA for Strensiq was validated by the EMA. In October 2014, Alexion submitted a New Drug Application for Strensiq to Japan’s MHLW.
The preclinical development, clinical trials, manufacturing, marketing and labeling of pharmaceuticals are all subject to extensive regulation by numerous governmental authorities and agencies in the United States, the European Union and other territories. Alexion must obtain regulatory approval for each of its product candidates, such as Strensiq, before marketing or selling any of them. It is not possible to predict how long the approval processes of the FDA or any other applicable federal or foreign regulatory authority or agency for any of Alexion’s product candidates will take or whether any such approvals ultimately will be granted. For example, the EMA transitioned the MAA for Strensiq from an accelerated assessment to a regular assessment. The FDA and foreign regulatory agencies have substantial discretion in the drug approval process, and positive results in preclinical testing or early phases of clinical studies offer no assurance of success in later phases of the approval process. The approval process varies from country to country and the requirements governing the conduct of clinical trials, product manufacturing, product licensing, pricing and reimbursement vary greatly from country to country. Generally, preclinical and clinical testing of product candidates can take many years and require the expenditure of substantial resources, and the data obtained from these tests and trials can be susceptible to varying interpretations that could delay, limit or prevent regulatory approval. If Alexion encounters significant delays in the regulatory
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process, this may prevent Alexion from continuing to develop its product candidates due to excessive costs or otherwise. Any delay in obtaining, or failure to obtain, approvals could adversely affect the marketing of Alexion’s products and its ability to generate product revenue. The risks associated with the approval process include:

failure of Alexion’s product candidates to meet a regulatory agency’s requirements for safety, efficacy and quality;

disagreement over interpretation of data from preclinical studies or clinical trials;

restricted distribution or limitation on the indicated uses for which a product may be marketed;

unforeseen safety issues or side effects and potential requirements to establish REMS or post-marketing obligations;

disapproval of the manufacturing processes or facilities of third-party manufacturers with which Alexion contract for clinical and commercial supplies; and

governmental or regulatory delays and changes in regulatory requirements and guidelines.
The FDA or a comparable foreign regulatory authority may require more information, including additional preclinical or clinical data, to support approval, which may delay or prevent approval and Alexion’s commercialization plans, or Alexion may decide to abandon the development program. If Alexion were to obtain approval, regulatory authorities may approve any of Alexion’s product candidates for fewer or more limited indications than Alexion request, may grant approval contingent on the performance of costly post-marketing clinical trials, or may approve a product candidate with a label that is not desirable for the successful commercialization of that product candidate. In addition, if Alexion’s product candidate produces undesirable side effects or safety issues, the FDA may require the establishment of REMS or a comparable foreign regulatory authority may require the establishment of a similar strategy, that may, for instance, restrict distribution of Alexion’s products and impose burdensome implementation requirements on it. Any of the foregoing scenarios could materially harm the commercial prospects of Alexion’s product candidates.
If Alexion cannot obtain new patents, maintain its existing patents and protect the confidentiality and proprietary nature of its trade secrets and other intellectual property, Alexion’s business and competitive position will be harmed.
In order to protect Alexion’s drugs and technology more effectively, Alexion needs to obtain and maintain patents covering the drugs and technologies it develops. Alexion has and may in the future obtain patents or the right to practice patents through ownership or license. Alexion’s patent applications may not result in the issue of patents in the United States or other countries. Alexion’s patents may not afford adequate protection for its products. Third parties may challenge Alexion’s patents, and have challenged Alexion’s patents in the past. If any of Alexion’s patents are narrowed, invalidated or become unenforceable, competitors may develop and market products similar to ours that do not conflict with or infringe Alexion’s patents rights, which could have a material adverse effect on Alexion’s financial condition. Alexion may also finance and collaborate in research conducted by government organizations, hospitals, universities or other educational or research institutions. Such research partners may be unwilling to grant Alexion exclusive rights to technology or products developed through such collaborations. There is also a risk that disputes may arise as to the rights to technology or products developed in collaboration with other parties. Soliris and Alexion’s drug candidates are expensive and time-consuming to test and develop. Even if Alexion obtains and maintain patents, Alexion’s business may be significantly harmed if the patents are not broad enough to protect Alexion’s drugs from copycat products.
In addition, Alexion’s business requires using sensitive technology, techniques and proprietary molecules that Alexion protects as trade secrets. However, Alexion may also rely heavily on collaboration with, or discuss the potential for collaboration with, suppliers, outside scientists and other drug companies. Collaboration and discussion of potential collaboration present a strong risk of exposing Alexion’s trade secrets. If Alexion’s trade secrets were exposed, it would help Alexion’s competitors and adversely affect Alexion’s business prospects.
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If Alexion is found to be infringing on patents owned by others, Alexion may be forced to pay damages to the patent owner and/or obtain a license to continue the manufacture, sale or development of its drugs. If Alexion cannot obtain a license, it may be prevented from the manufacture, sale or development of its drugs, including Soliris, which would adversely affect Alexion’s business.
Parts of Alexion’s technology, techniques and proprietary molecules and potential drug candidates, including those which are or may be in-licensed, may be found to infringe patents owned by or granted to others. Alexion previously reported that Novartis and other third parties have filed civil lawsuits against Alexion claiming infringement of their intellectual property rights. Each of these matters has been resolved, however, additional third parties may claim that the manufacture, use or sale of Soliris or other drugs under development infringes patents owned or granted to such third parties. In addition to the civil actions referenced above, Alexion has in the past received, and may in the future receive, notices from third parties claiming that their patents may be infringed by the development, manufacture or sale of Soliris or some of Alexion’s drug candidates. Alexion is aware of patents owned by third parties that might be claimed by such third parties to be infringed by the development and commercialization of Soliris and some of Alexion’s drug candidates. In respect to some of these patents, Alexion has obtained licenses, or expect to obtain licenses. However, with regard to such other patents, Alexion has determined in its judgment that:

Soliris and Alexion’s product candidates do not infringe the patents;

the patents are not valid; or

Alexion has identified and tested or are testing various modifications that Alexion believes should not infringe the patents and which should permit commercialization of Alexion’s product candidates.
Any holder of these patents or other patents covering similar technology could sue Alexion for damages and seek to prevent Alexion from manufacturing, selling or developing its drugs. Legal disputes can be costly and time consuming to defend. If Alexion cannot successfully defend against any future actions or conflicts, if they arise, Alexion may incur substantial legal costs and may be liable for damages, be required to obtain costly licenses or need to stop manufacturing, using or selling Soliris, which would adversely affect Alexion’s business. Alexion may seek to obtain a license prior to or during legal actions in order to reduce further costs and the risk of a court determination that Alexion’s product infringes the third party’s patents. A required license may be costly or may not be available on acceptable terms, if at all. A costly license, or inability to obtain a necessary license, could have a material adverse effect on Alexion’s business.
There can be no assurance that Alexion would prevail in a patent infringement action or that Alexion would be able to obtain a license to any third-party patent on commercially reasonable terms or any terms at all; successfully develop non-infringing alternatives on a timely basis; or license alternative non-infringing technology, if any exists, on commercially reasonable terms. Any impediment to Alexion’s ability to manufacture, use or sell approved forms of Soliris or Alexion’s product candidates could have a material adverse effect on Alexion’s business and prospects.
It is possible that Alexion could lose market exclusivity for a product earlier than expected, which would harm Alexion’s competitive position.
In Alexion’s industry, much of an innovative product’s commercial value is realized while it has market exclusivity. When market exclusivity expires and biosimilar or generic versions of the product are approved and marketed, there can be substantial decline in the innovative product’s sales.
Market exclusivity for Soliris is based upon patent rights and certain regulatory forms of exclusivity. The scope of Soliris patent rights vary from country to country and are dependent on the availability of meaningful legal remedies in each country. The failure to obtain patent and other intellectual property rights, or limitations on the use, or loss of such rights, could be material to Alexion’s business. In some countries, patent protections for Soliris may not exist because certain countries did not historically offer the right to obtain specific types of patents or Alexion did not file patents in those markets. Also, the patent environment is unpredictable and the validity and enforceability of patents cannot be predicted with
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certainty. Absent relevant patent protection for a product, once regulatory exclusivity periods expire, biosimilar or generic versions of the product can be approved and marketed. Even prior to the expiration of regulatory exclusivity, a competitor could seek to obtain marketing approval by submitting its own clinical trial data.
Alexion cannot guarantee that it will achieve its financial goals, including Alexion’s ability to maintain profitability on a quarterly or annual basis in the future.
Until the quarter ended June 30, 2008, Alexion had never been profitable since it was incorporated in January 1992. Alexion has maintained profitability on a quarterly basis since the quarter ended June 30, 2008 and on an annual basis beginning with the year ended December 31, 2008. Alexion believes that it formulates its annual operating budgets with reasonable assumptions and targets, however Alexion cannot guarantee that it will be able to generate sufficient revenues or control expenses to achieve its financial goals, including continued profitability. Even if Alexion does achieve profitability in any subsequent quarters, Alexion may not be able to sustain or increase profitability on a quarterly or annual basis. You should not consider Alexion’s revenue growth in recent periods as indicative of its future performance. Alexion’s revenue in future periods could decline. Alexion may make errors in predicting and reacting to relevant business trends or Alexion’s business may be subject to factors beyond its control, which could harm Alexion’s operations. Since Alexion began its business, it has focused on research and development of product candidates. Alexion cannot guarantee that it will be successful in marketing and selling Soliris on a continued basis in countries or regions where it has obtained marketing approval, including the United States, Europe and Japan, and Alexion does not know when it will have Soliris available for sale in territories where it has applied or will apply for marketing approval, if ever. Alexion will have substantial expenses as it continues its research and development efforts, continue to conduct clinical trials and continue to develop manufacturing, sales, marketing and distribution capabilities in the United States and abroad. The achievement of Alexion’s financial goals, including the extent of Alexion’s future profitability, depends on many factors, including Alexion’s ability to successfully market Soliris in the United States, the European Union and Japan and other territories, Alexion’s ability to obtain regulatory, pricing, coverage, and reimbursement approvals of Alexion’s drug candidates, such as asfotase alfa, and for Soliris in additional territories and other indications, Alexion’s ability to successfully market Soliris in additional territories, Alexion’s ability to successfully manufacture and commercialize its drug candidates and Alexion’s ability to successfully bring its other product candidates to the major commercial markets throughout the world.
If Alexion’s competitors get to the marketplace before it does, or with better or less expensive drugs, it may not be profitable to continue to produce Soliris and Alexion’s product candidates.
The FDA, EC and the MHLW granted orphan drug designation for Soliris in the treatment of PNH and the FDA and EC granted orphan drug designation for aHUS. Orphan drug status entitles Soliris to market exclusivity for a total of seven years in the United States and for ten years in the European Union and Japan. However, if a competitive product that is the same as or similar to Soliris, as defined under the applicable regulations, is shown to be clinically superior to Soliris in the treatment of PNH or aHUS, or if a competitive product is different from Soliris, as defined under the applicable regulations, the orphan drug exclusivity Alexion has obtained may not block the approval of such competitive product. Several biotechnology and pharmaceutical companies throughout the world have programs to develop complement inhibitor therapies or have publicly announced their intentions to develop drugs which target the inflammatory effects of complement in the immune system. Pharmaceutical companies have publicly announced intentions to establish or develop rare disease programs and these companies may introduce products that are competitive with ours. These and other companies, many of which have significantly greater resources than us, may develop, manufacture, and market better or cheaper drugs than Soliris or Alexion’s product candidates. They may establish themselves in the marketplace before Alexion for Soliris for other indications or for any of Alexion’s other product candidates. Other pharmaceutical companies also compete with Alexion to attract academic research institutions as drug development partners, including for licensing these institutions’ proprietary technology. If Alexion’s competitors successfully enter into such arrangements with academic institutions, Alexion will be precluded from pursuing those unique opportunities and may not be able to find equivalent opportunities elsewhere.
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If Alexion fails to recruit and retain personnel, Alexion may not be able to implement Alexion’s business strategy.
Alexion is highly dependent upon the efforts of its executive officers, and other key personnel in its commercial and technical organizations. There is intense competition in the biopharmaceutical industry for qualified commercial and technical personnel. Alexion’s business is specialized and global and Alexion must attract and retain highly qualified individuals across many geographies. Alexion may not be able to continue to attract and retain the qualified personnel necessary for developing, manufacturing and commercializing Alexion’s products and product candidates.
Alexion is subject to environmental laws and potential exposure to environmental liabilities.
Alexion is subject to various federal, state and local environmental laws and regulations that govern its operations, including Alexion’s manufacturing operations at ARIMF and in Ireland, the handling and disposal of non-hazardous and hazardous wastes, such as medical and biological wastes, and emissions and discharges into the environment, such as air, soils and water sources. Failure to comply with such laws and regulations could result in costs for corrective action, penalties or the imposition of other liabilities. Alexion is also subject to laws and regulations that impose liability and clean-up responsibility for releases of hazardous substances into the environment. Under certain of these laws and regulations, a current or previous owner or operator of property may be liable for the costs of remediating its property or locations to which wastes were sent from its facilities, without regard to whether the owner or operator knew of, or necessarily caused, the contamination. Such obligations and liabilities, which to date have not been material, could have a material impact on Alexion’s business and financial condition.
Alexion is seeking to expand its business through acquisitions and Alexion may not realize the benefits of such acquisitions.
Alexion’s business strategy includes expanding its products and capabilities. Alexion may seek additional acquisitions or in-licensing of businesses or products to expand Alexion’s products and capabilities. Acquisitions of new businesses or products and in-licensing of new products may involve numerous risks, including:

substantial cash expenditures;

potentially dilutive issuance of equity securities;

incurrence of debt and contingent liabilities, some of which may be difficult or impossible to identify at the time of acquisition;

difficulties in assimilating the operations of the acquired companies;

diverting Alexion’s management’s attention away from other business concerns;

risks of entering markets in which Alexion has limited or no direct experience;

the potential loss of Alexion’s key employees or key employees of the acquired companies; and

failure of any acquired businesses or products or in-licensed products to achieve the scientific, medical, commercial or other results anticipated.
A substantial portion of Alexion’s strategic efforts are focused on opportunities for rare disorders and life-saving therapies. The availability of such development opportunities is limited. Alexion may not be able to identify opportunities that are acceptable to it or its shareholders. Several companies have publicly announced intentions to establish or develop rare disease programs. For these and other reasons, Alexion may not be able to acquire the rights to additional product candidates and approved products on terms that it or its shareholders find acceptable, or at all. The development or expansion of Alexion’s business, any acquired business or any acquired or in-licensed products may require a substantial capital investment by Alexion. Alexion may not have these necessary funds or they might not be available to Alexion on
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acceptable terms or at all. Alexion may also seek to raise funds by selling shares of its capital stock, which could dilute current stockholders’ ownership interest in Alexion’s company, or securities convertible into Alexion’s capital stock, which could dilute current stockholders’ ownership interest in Alexion’s company upon conversion.
Even if Alexion is able to successfully identify and complete acquisitions and other strategic transactions, it may not be able to integrate them or take full advantage of them. An acquisition or other strategic transaction may not result in short-term or long-term benefits to Alexion. Alexion may also incorrectly judge the value or worth of an acquired company or business or an acquired or in-licensed product.
To effectively manage Alexion’s current and future potential growth, Alexion must continue to effectively grow and manage its global employee base, and enhance its operational and financial processes. Supporting Alexion’s growth strategy will require significant capital expenditures and management resources, including investments in research and development, sales and marketing, manufacturing and other areas of Alexion’s operations. If Alexion does not successfully manage its current growth and do not successfully execute Alexion’s strategy, then its business and financial results may be adversely affected and it may incur asset impairment or restructuring charges.
Alexion’s business could be affected by litigation, government investigations and enforcement actions.
Alexion operates in many jurisdictions in a highly regulated industry and Alexion could be subject to litigation, government investigation and enforcement actions on a variety of matters in the United States or foreign jurisdictions, including, without limitation, intellectual property, regulatory, product liability, environmental, whistleblower, Qui Tam, false claims, privacy, anti-kickback, anti-bribery, securities, commercial, employment, and other claims and legal proceedings which may arise from conducting Alexion’s business. Legal proceedings, government investigations and enforcement actions can be expensive and time consuming. An adverse outcome could result in significant damages awards, fines, penalties, exclusion from the federal healthcare programs, healthcare debarment, injunctive relief, product recalls, reputational damage and modifications of Alexion’s business practices, which could have a material adverse effect on Alexion’s business and results of operations.
The intended efficiency of Alexion’s corporate structure depends on the application of the tax laws and regulations in the countries where it operate and Alexion may have exposure to additional tax liabilities or its effective tax rate could change, which could have a material impact on Alexion’s results of operations and financial position.
As a company with international operations, Alexion is subject to income taxes, as well as non-income based taxes, in both the United States and various foreign jurisdictions. Significant judgment is required in determining Alexion’s worldwide tax liabilities. Although Alexion believes its estimates are reasonable, the ultimate outcome with respect to the taxes it owes may differ from the amounts recorded in Alexion’s financial statements. If the Internal Revenue Service, or other taxing authority, disagrees with the positions Alexion takes, Alexion could have additional tax liability, and this could have a material impact on Alexion’s results of operations and financial position. Alexion’s effective tax rate could be adversely affected by changes in the mix of earnings in countries with different statutory tax rates, changes in the valuation of deferred tax assets and liabilities, changes in tax laws and regulations, changes in interpretations of tax laws, including pending tax law changes, changes in Alexion’s manufacturing activities and changes in Alexion’s future levels of research and development spending.
Alexion has designed its corporate structure, the manner in which it develops and uses its intellectual property, and its intercompany transactions between its affiliates in a way that is intended to enhance its operational and financial efficiency and increase Alexion’s overall profitability. The application of the tax laws and regulations of various countries in which Alexion operates and to Alexion’s global operations is subject to interpretation. Alexion also must operate its business in a manner consistent with its corporate structure to realize such efficiencies. The tax authorities of the countries in which Alexion operates may challenge its methodologies for valuing developed technology or for transfer pricing. If tax authorities determine that the manner in which Alexion operates results in its business not achieving the intended tax consequences, Alexion’s effective tax rate could increase and harm Alexion’s financial position and results of operations.
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In addition, the United States government and other governments are considering and may adopt tax reform measures that significantly increase Alexion’s worldwide tax liabilities. The U.S. Congress, the Organization for Economic Co-operation and Development and other government agencies in countries where Alexion and its affiliates operate have focused on issues related to the taxation of multinational corporation, including, for example, in the area of  “base erosion and profit shifting,” where payments are made between affiliates from a jurisdiction with high tax rates to a jurisdiction with lower tax rates. Alexion established operations in Ireland in 2013 and recently, Ireland tax authorities announced changes to the treatment of non-resident Irish entities. The changes are not expected to impact existing non-resident Irish entities, such as ours, until after December 31, 2020. These changes and other prospective changes in the United States and other countries in which Alexion and its affiliates operate could increase Alexion’s effective tax rate, and harm its financial position and results of operations.
Alexion’s sales and operations are subject to the economic, political, legal and business conditions in the countries in which Alexion does business, and Alexion’s failure to operate successfully or adapt to changes in these conditions could cause its sales and operations to be limited or disrupted.
Since 2007, Alexion has significantly expanded its operations and expect to continue to do so in the future. Alexion’s operations in foreign countries subject Alexion to the following additional risks:

fluctuations in currency exchange rates;

political or economic determinations that adversely impact pricing or reimbursement policies;

economic problems or political instability that disrupt health care payment systems;

difficulties or inability to obtain financing in markets;

unexpected changes in tariffs, trade barriers and regulatory requirements;

difficulties enforcing contractual and intellectual property rights;

changes in laws, regulations or enforcement practices with respect to Alexion’s business, including without limitation laws relating to reimbursement, competition, pricing and sales and marketing of Alexion’s products;

trade restrictions and restrictions on direct investments by foreign entities;

compliance with tax, employment and labor laws;

costs and difficulties in recruiting and retaining qualified managers and employees to manage and operate the business in local jurisdictions;

costs and difficulties in managing and monitoring international operations; and

longer payment cycles.
Alexion’s business and marketing methods are also subject to regulation by the governments of the countries in which Alexion operates. The FCPA and similar anti-bribery laws in other countries prohibit companies and their representatives from offering, promising, authorizing or making payments to foreign officials for the purpose of obtaining or retaining business. Alexion has policies and procedures designed to help ensure that Alexion and its representatives, including Alexion’s employees, comply with such laws, however Alexion cannot guarantee that these policies and procedures will protect it against liability under the FCPA or other anti-bribery laws for actions taken by Alexion’s representatives. Failure to comply with the laws and regulations of the countries in which Alexion operate could materially harm Alexion’s business.
Alexion conducts, or anticipates that it will conduct, a substantial portion of its business in currencies other than the U.S. dollar and Alexion is exposed to fluctuations in foreign currency exchange rates in the normal course of its business. See also Risk Factor “Currency fluctuations and changes in exchange rates could adversely affect Alexion’s revenue growth, increase its costs and negatively affect Alexion’s profitability.”
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The credit and financial market conditions may aggravate certain risks affecting Alexion’s business.
Sales of Soliris and other products are or will be dependent, in large part, on reimbursement from government health administration organizations and private and governmental third-party payers, and also co-payments from individual patients in certain situations. As a result of adverse credit and financial market conditions, and the overall financial climate, these governmental organizations and payers, and/or individuals, may reduce or delay initiation of treatment, may be unable to satisfy their reimbursement obligations, may delay payment or may seek to reduce reimbursement for Alexion’s products, including Soliris, in the future, which could have a material adverse effect on Alexion’s business and results of operations. Soliris is approved for the treatment of patients with PNH and aHUS in the United States, the European Union and Japan and for the treatment of PNH in several other territories. If Soliris is approved in additional territories for PNH, aHUS, or for additional indications that are under clinical development, the reimbursement risks and uncertainties associated with adverse credit and financial market conditions may be exacerbated due to increases in the number of patients receiving Soliris that require reimbursement. Payment defaults by a government payer could require Alexion to expense previously recorded revenue as uncollectible, and might cause Alexion to end or restrict sales to patients in that country. Further, the risk of payment default by a government payer could require Alexion to revise its revenue recognition policies in regard to that payer, causing revenue to be recorded only on a cash basis, and Alexion may be required to end or restrict sales to patients in that country.
Alexion continues to monitor economic conditions, including volatility associated with U.S. and international economies, associated impacts on the financial markets and Alexion’s business, and the sovereign debt issues in Europe.
Alexion may not be able to successfully mitigate or prevent its exposures to volatile economic and financial conditions and Alexion’s failure to operate successfully or adapt to changes in these conditions could cause its sales and operations to be limited or disrupted or otherwise harm Alexion’s business.
Additionally, Alexion relies upon third-parties for certain parts of its business, including Lonza, licensees, wholesale distributors of Soliris, contract clinical trial providers, contract manufacturers and other third-party suppliers and financial institutions. Because of the volatility in the financial markets, there may be a disruption or delay in the performance or satisfaction of commitments to Alexion by these third parties which could have a material adverse effect on its business and results of operations.
Currency fluctuations and changes in exchange rates could adversely affect Alexion’s revenue growth, increase its costs and negatively affect Alexion’s profitability.
Alexion conducts, or anticipates that it will conduct, a substantial portion of its business in currencies other than the U.S. dollar. Alexion is exposed to fluctuations in foreign currency exchange rates in the normal course of its business and Alexion expects these exposures to increase during 2015 if the strengthening of the U.S. dollar continues. The exposures result from portions of Alexion’s revenues, as well as the related receivables, and expenses that are denominated in currencies other than the U.S. dollar, including the Euro, Japanese Yen, British Pound, Swiss Franc, and Russian Ruble. Alexion manages its foreign currency transaction risk within specified guidelines through the use of derivatives. All of Alexion’s derivative instruments are utilized for risk management purposes, and Alexion does not use derivatives for speculative trading purposes. Alexion enters into foreign exchange forward contracts, with durations of up to 60 months, to hedge exposures resulting from portions of Alexion’s forecasted revenues, including intercompany revenues, that are denominated in currencies other than the U.S. dollar. The purpose of the hedges of revenue is to reduce the volatility of exchange rate fluctuations on Alexion’s operating results and to increase the visibility of the foreign exchange impact on forecasted revenues. Further, Alexion enters into foreign exchange forward contracts, with durations of approximately 30 days, designed to limit the balance sheet exposure of monetary assets and liabilities. Alexion enters into these hedges to reduce the impact of fluctuating exchange rates on Alexion’s operating results. Gains and losses on these hedge transactions are designed to offset gains and losses on underlying balance sheet exposures. While Alexion attempts to hedge certain currency risks, currency fluctuations between the U.S. dollar and the currencies in which Alexion does business have, in the past, caused foreign currency transaction gains and losses and have also impacted the amounts of revenues and expenses calculated in U.S. dollars and will likely do so in the future. Likewise, past currency fluctuations have at times resulted in foreign currency transaction gains, and there can be no assurance that these gains can be reproduced.
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Changes in healthcare law and implementing regulations, including those based on recently enacted legislation, as well as changes in healthcare policy and coverage and reimbursement of drug products may impact Alexion’s business in ways that Alexion cannot currently predict and these changes could adversely affect Alexion’s business and financial condition.
Governments in countries where Alexion operates have adopted or have shown significant interest in pursuing legislative initiatives to reduce costs of health care. Any such government-adopted health care measures could adversely impact the pricing of Soliris or the amount of coverage and reimbursement available for Soliris from governmental agencies or other third-party payers.
For example, the PPACA was adopted in the United States in March 2010. This law substantially changes the way healthcare is financed by both governmental and private insurers in the U.S., and significantly impacts the pharmaceutical industry. PPACA contains a number of provisions that are expected to impact Alexion’s business and operations, in some cases in ways Alexion cannot currently predict. Changes that may affect Alexion’s business include those governing enrollment in federal healthcare programs, reimbursement changes, rules regarding prescription drug benefits under health insurance exchanges, expansion of the 340B program, expansion of state Medicaid programs, and fraud and abuse enforcement. These changes will impact existing government healthcare programs and will result in the development of new programs, including Medicare payment for performance initiatives and improvements to the physician quality reporting system and feedback program.
PPACA contains several provisions that have or could potentially impact Alexion’s business. PPACA made significant changes to the Medicaid Drug Rebate Program. Effective March 23, 2010, rebate liability expanded from fee-for-service Medicaid utilization to include the utilization of Medicaid managed care organizations as well. With regard to the amount of the rebates owed, PPACA increased the minimum Medicaid rebate from 15.1% to 23.1% of the average manufacturer price for most innovator products; changed the calculation of the rebate for certain innovator products that qualify as line extensions of existing drugs; and capped the total rebate amount for innovator drugs at 100% of the average manufacturer price. In addition, PPACA and subsequent legislation changed the definition of average manufacturer price. Finally, PPACA requires pharmaceutical manufacturers of branded prescription drugs, such as Soliris, to pay a branded prescription drug fee to the federal government beginning in 2011. Each individual pharmaceutical manufacturer pays a prorated share of the branded prescription drug fee of $3.0 billion in 2014 (and set to increase in ensuing years), based on the dollar value of its branded prescription drug sales to certain federal programs identified in the law. Sales of  “orphan drugs” — those designated under section 526 of the FDCA, like Soliris — are excluded from this fee as long as no non-orphan indications have been approved for the orphan drug.
In 2012, CMS issued proposed regulations to implement the changes to the Medicaid Drug Rebate Program under PPACA but has not yet issued final regulations. CMS is currently expected to release the final regulations in 2015. Moreover, in the future, Congress could enact legislation that further increases Medicaid drug rebates or other costs and charges associated with participating in the Medicaid Drug Rebate Program. The issuance of regulations and coverage expansion by various governmental agencies relating to the Medicaid Drug Rebate Program has and will continue to increase Alexion’s costs and the complexity of compliance, has been and will be time-consuming, and could have a material adverse effect on Alexion’s results of operations.
Additional provisions of PPACA, some of which became effective in 2011, may negatively affect Alexion’s revenues in the future. For example, as part of PPACA’s provisions closing a coverage gap that currently exists in the Medicare Part D prescription drug program (commonly known as the “donut hole”), Alexion is required to provide a 50% discount on branded prescription drugs dispensed to beneficiaries within this donut hole.
PPACA also expanded the Public Health Service’s 340B drug pricing discount program. The 340B pricing program requires participating manufacturers to agree to charge statutorily-defined covered entities no more than the 340B “ceiling price” for the manufacturer’s covered outpatient drugs. PPACA expanded the 340B program to include additional types of covered entities: certain free-standing cancer hospitals, critical access hospitals, rural referral centers and sole community hospitals, each as defined by PPACA. PPACA exempts “orphan drugs” — those designated under section 526 of the FDCA, such as Soliris —
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from the ceiling price requirements for these newly-eligible entities. On July 21, 2014, the Health Resources and Services Administration (“HRSA”) which administers the 340B program, issued an interpretive rule to implement the orphan drug exception which interprets the orphan drug exception narrowly. It exempts orphan drugs from the ceiling price requirements for the newly-eligible entities only when the orphan drug is used for its orphan indication. The newly-eligible entities are entitled to purchase orphan drugs at the ceiling price when the orphan drug is not used for its orphan indication. A manufacturer trade group has filed a lawsuit challenging the interpretive rule as inconsistent with the statutory language. That challenge remains ongoing. The uncertainty regarding how the statutory orphan drug exception will be applied will increase the complexity of compliance, will make compliance more time-consuming, and could negatively impact Alexion’s results of operations. If HRSA’s narrow interpretation of the scope of the orphan drug exemption prevails, it could potentially negatively impact the price Alexion is paid for Soliris by certain entities for some uses and increase the complexity of compliance with the 340B program.
In addition, Alexion’s industry may be affected by broader legislation addressing federal spending, including, for example, a sequester required by the Budget Control Act of 2011, Pub. L. No. 112-25, as amended by the American Taxpayer Relief Act of 2012, Pub. L. 112-240, that took effect in April 2013 and was expended by the Bipartisan Budget Act of 2013, Pub. L. No. 113-67. Under the sequestration, Medicare payments for all items and services, including drugs and biologicals, have been reduced by 2%. This 2% reduction in Medicare payments affects all Parts of the Medicare program and could impact sales of Soliris. As another example, the governments of Germany and Spain each approved increases to mandatory rebates on the sales of pharmaceutical products.
Alexion expects that the implementation of current laws and policies, the amendment of those laws and policies in the future, as well as the adoption of new laws and policies, could have a material adverse effect on Alexion’s industry generally and on Alexion’s ability to maintain or increase its product sales or successfully commercialize Alexion’s product candidates, or could limit or eliminate Alexion’s future spending on development projects. In many cases, these government initiatives, even if enacted into law, are subject to future rulemaking by regulatory agencies. Although Alexion has evaluated these government initiatives and the impact on its business, Alexion cannot know with certainty whether any such law, rule or regulation will adversely affect coverage and reimbursement of Soliris, or to what extent, until such laws, rules and regulations are promulgated, implemented and enforced. The announcement or adoption of regulatory or legislative proposals could delay or prevent Alexion’s entry into new markets, affect its reimbursement or sales in the markets where Alexion is already selling Soliris and materially harm Alexion’s business, financial condition and results of operations.
If Alexion fails to comply with its reporting and payment obligations under the Medicaid Drug Rebate Program, Medicare, or other governmental pricing programs, Alexion could be subject to additional reimbursement requirements, penalties, sanctions and fines which could have a material adverse effect on Alexion’s business, financial condition, results of operations and growth prospects.
Medicare is a U.S. federal government insurance program that covers individuals aged 65 years or older, certain younger individuals with certain disabilities, and individuals with End-Stage Renal Disease. The primary Medicare programs that may affect reimbursement for Soliris are Medicare Part B, which covers physician services and outpatient care, and Medicare Part D, which provides a voluntary outpatient prescription drug benefit. Medicare Part B provides limited coverage of certain outpatient drugs and biologicals that are reasonable and necessary for diagnosis or treatment of an illness or injury. Under Part B, reimbursement is based on a fixed percentage of the applicable product’s ASP. Manufacturers calculate ASP based on a statutory formula and must report ASP information to the CMS, the federal agency that administers Medicare and the Medicaid Drug Rebate Program, on a quarterly basis.
Medicaid is a government health insurance program for low-income children, families, pregnant women, and people with disabilities. It is jointly funded by the federal and state governments, and it is administered by individual states within parameters established by the federal government. Coverage and reimbursement for drugs and biologicals thus varies by state. Drugs and biologicals may be covered under the medical or pharmacy benefit. State Medicaid programs may impose utilization management controls, such as prior authorization, step therapy, or quantity limits on drugs and biologicals. Medicaid also includes the Medicaid Drug Rebate Program, under which Alexion is required to pay a rebate to each state
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Medicaid program for quantities of Soliris that are dispensed to Medicaid beneficiaries and paid for by a state Medicaid program as a condition of having federal funds being made available to the states for Soliris under Medicaid and Medicare Part B. Those rebates are based on pricing data reported by Alexion on a monthly and quarterly basis to CMS. These data include the average manufacturer price and the best price for Soliris.
Federal law requires that any company that participates in the Medicaid Drug Rebate Program also participate in the Public Health Service’s 340B drug pricing program in order for federal funds to be available for the manufacturer’s drugs under Medicaid and Medicare Part B. The 340B pricing program requires participating manufacturers to agree to charge statutorily-defined covered entities no more than the 340B “ceiling price” for the manufacturer’s covered outpatient drugs. These 340B covered entities include a variety of community health clinics and other entities that receive health services grants from the Public Health Service, as well as hospitals that serve a disproportionate share of low-income patients. The 340B ceiling price is calculated using a statutory formula, which is based on the average manufacturer price and rebate amount for the covered outpatient drug as calculated under the Medicaid Drug Rebate Program.
Pricing and rebate calculations vary among products and programs. The calculations are complex and are often subject to interpretation by Alexion, governmental or regulatory agencies and the courts. The Medicaid rebate amount is computed each quarter based on Alexion’s submission to CMS of Alexion’s current average manufacturer price and best price for the quarter. If Alexion becomes aware that its reporting for a prior quarter was incorrect, or has changed as a result of recalculation of the pricing data, Alexion is obligated to resubmit the corrected data for a period not to exceed twelve quarters from the quarter in which the data originally were due. Such restatements and recalculations increase Alexion’s costs for complying with the laws and regulations governing the Medicaid Drug Rebate Program. Any corrections to Alexion’s rebate calculations could result in an overage or underage in Alexion’s rebate liability for past quarters, depending on the nature of the correction. Price recalculations also may affect the ceiling price at which Alexion is required to offer its products to certain covered entities, such as safety-net providers, under the 340B drug discount program.
Alexion is liable for errors associated with its submission of pricing data. In addition to retroactive rebates and the potential for 340B program refunds, if Alexion is found to have knowingly submitted false average manufacturer price, ASP, or best price information to the government, Alexion may be liable for civil monetary penalties in the amount of  $100,000 per item of false information. If Alexion is found to have made a misrepresentation in the reporting of its ASP, the Medicare statute provides for civil monetary penalties of up to $10,000 for each misrepresentation for each day in which the misrepresentation was applied. Alexion’s failure to submit monthly/quarterly average manufacturer price, ASP, and best price data on a timely basis could result in a civil monetary penalty of  $10,000 per day for each day the information is late beyond the due date. Such failure also could be grounds for CMS to terminate Alexion’s Medicaid drug rebate agreement, pursuant to which Alexion participates in the Medicaid program. In the event that CMS terminates Alexion’s rebate agreement, federal payments may not be available under Medicaid or Medicare Part B for Alexion’s covered outpatient drugs.
In September 2010, CMS and the Office of Inspector General indicated that they intend to pursue more aggressively those companies who fail to report these data to the government in a timely manner. Governmental agencies may also make changes in program interpretations, requirements or conditions of participation, some of which may have implications for amounts previously estimated or paid. Alexion cannot assure you that its submissions will not be found by CMS to be incomplete or incorrect.
Federal law requires that for a company to be eligible to have its products paid for with federal funds under the Medicaid program as well as to be purchased by certain federal agencies and grantees, it also must participate in the Department of Veterans Affairs (“VA”) Federal Supply Schedule (“FSS”) pricing program. To participate, Alexion is required to enter into an FSS contract with the VA, under which Alexion must make its innovator “covered drugs” available to the “Big Four” federal agencies — the VA, the Department of Defense (“DoD”) the Public Health Service, and the Coast Guard — at pricing that is capped pursuant to a statutory federal ceiling price, or FCP, formula set forth in Section 603 of the Veterans Health Care Act of 1992 (“VHCA”). The FCP is based on a weighted average non-federal average manufacturer price (“Non-FAMP”) which manufacturers are required to report on a quarterly and annual
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basis to the VA. If a company misstates Non-FAMPs or FCPs it must restate these figures. Pursuant to the VHCA, knowing provision of false information in connection with a Non-FAMP filing can subject a manufacturer to penalties of  $100,000 for each item of false information.
FSS contracts are federal procurement contracts that include standard government terms and conditions, separate pricing for each product, and extensive disclosure and certification requirements. All items on FSS contracts are subject to a standard FSS contract clause that requires FSS contract price reductions under certain circumstances where pricing is reduced to an agreed “tracking customer.” Further, in addition to the “Big Four” agencies, all other federal agencies and some non-federal entities are authorized to access FSS contracts. FSS contractors are permitted to charge FSS purchasers other than the Big Four agencies “negotiated pricing” for covered drugs that is not capped by the FCP; instead, such pricing is negotiated based on a mandatory disclosure of the contractor’s commercial “most favored customer” pricing. Alexion offer dual pricing on Alexion’s FSS contract.
In addition, pursuant to regulations issued by the DoD TRICARE Management Activity, now the Defense Health Agency, to implement Section 703 of the National Defense Authorization Act for Fiscal Year 2008, each of Alexion’s covered drugs is listed on a Section 703 Agreement under which Alexion has agreed to pay rebates on covered drug prescriptions dispensed to TRICARE beneficiaries by TRICARE network retail pharmacies. Companies are required to list their innovator products on Section 703 Agreements in order for those products to be eligible for DoD formulary inclusion. The formula for determining the rebate is established in the regulations and Alexion’s Section 703 Agreement and is based on the difference between the annual Non-FAMP and the FCP (as described above, these price points are required to be calculated by Alexion under the VHCA).
If Alexion overcharges the government in connection with its FSS contract or Section 703 Agreement, whether due to a misstated FCP or otherwise, Alexion is required to refund the difference to the government. Failure to make necessary disclosures and/or to identify contract overcharges can result in allegations against Alexion under the FCA and other laws and regulations. Unexpected refunds to the government, and responding to a government investigation or enforcement action, would be expensive and time-consuming, and could have a material adverse effect on Alexion’s business, financial condition, results of operations and growth prospects.
Alexion may be subject to numerous and varying privacy and security laws, and Alexion’s failure to comply could result in penalties and reputational damage.
Alexion is subject to laws and regulations covering data privacy and the protection of personal information including health information. The legislative and regulatory landscape for privacy and data protection continues to evolve, and there has been an increasing focus on privacy and data protection issues which may affect Alexion’s business. In the U.S., some of the laws that may apply include state security breach notification laws, state health information privacy laws and federal and state consumer protections laws which impose requirements for the collection, use, disclosure and transmission of personal information. Each of these laws may be subject to varying interpretations by courts and government agencies, creating complex compliance issues for Alexion. If Alexion fails to comply with applicable laws and regulations Alexion could be subject to penalties or sanctions. Accordingly, Alexion could be subject to criminal penalties if it knowingly obtains individually identifiable health information from a covered entity in a manner that is not authorized or permitted by the federal Health Insurance Portability and Accountability Act of 1996, as amended (“HIPAA”) or for aiding and abetting the violation of HIPAA.
In addition, the receipt of personal health information in connection with Alexion’s clinical trial initiatives is subject to state and federal human subject protection laws. These laws could create liability for Alexion if one of its research collaborators were to use or disclose research subject information without consent and in violation of applicable laws.
Numerous other countries have, or are developing, laws governing the collection, use and transmission of personal information as well. European Union member states and other jurisdictions have adopted data protection laws and regulations, which impose significant compliance obligations. For example, the European Union Data Protection Directive, as implemented into national laws by the European Union member states, imposes strict obligations and restrictions on the ability to collect, analyze, and transfer
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personal data, including health data from clinical trials and adverse event reporting. Data protection authorities from different European Union member states may interpret the European Union Data Protection Directive and national laws differently, which adds to the complexity of processing personal data in the European Union, and guidance on implementation and compliance practices are often updated or otherwise revised. The European Union Data Protection Directive prohibits the transfer of personal data to countries outside of the European Union member states that are not considered by the European Commission to provide an adequate level of data protection. These countries include the United States. Any failure to comply with the rules arising from the European Union Data Protection Directive and related national laws of European Union member states could lead to government enforcement actions and significant penalties against Alexion, and adversely impact Alexion’s operating results.
A proposal for an European Union Data Protection Regulation, intended to replace the current European Union Data Protection Directive, is currently under consideration. The European Union Data Protection Regulation is expected to introduce new data protection requirements in the European Union and substantial fines for breaches of the data protection rules. If the draft European Union Data Protection Regulation is adopted in its current form, it may increase Alexion’s responsibility and liability in relation to personal data that Alexion processes and it may be required to put in place additional mechanisms ensuring compliance with the new European Union data protection rules.
Security breaches, cyber-attacks, or other disruptions could expose Alexion to liability and affect its business and reputation.
Alexion collects, stores, and transmits sensitive information including intellectual property, proprietary business information and personal information in connection with business operations. Alexion has implemented information security measures to protect patients’ personal information against the risk of inappropriate and unauthorized external use and disclosure. However, despite these measures, and due to the ever changing information cyber-threat landscape, Alexion may be subject to data breaches through cyber-attacks perpetrated by individuals that attempt to compromise Alexion’s security controls. If Alexion’s systems were to fail or be disrupted for an extended period of time Alexion could lose product sales and Alexion’s revenue and reputation would suffer. In the event Alexion’s systems were to be breached by an unauthorized third-party, they could potentially access confidential personal information, which could cause Alexion to suffer reputational damage and loss of customer confidence. Such incidents would result in notification obligations to affected individuals and government agencies, legal claims or proceedings, and liability under federal and state laws that protect the privacy and security of personal information. Any one of these events could cause Alexion’s business to be materially harmed and Alexion’s results of operations would be adversely impacted.
If the trading price of Alexion’s common stock continues to fluctuate in a wide range, Alexion’s stockholders will have uncertainty with respect to an investment in its common stock.
The trading price of Alexion’s common stock has been volatile and may continue to be volatile in the future. Factors such as announcements of fluctuations in Alexion’s or its competitors’ operating results or clinical or scientific results, fluctuations in the trading prices or business prospects of Alexion’s competitors and collaborators, changes in Alexion’s prospects, particularly with respect to sales of Soliris, failure to resolve, delays in resolving or other developments with respect to the issues raised in the Warning Letter, and market conditions for biopharmaceutical stocks in general could have a significant impact on the future trading prices of Alexion’s common stock. In particular, the trading price of the common stock of many biopharmaceutical companies, including ours, has experienced extreme price and volume fluctuations, which have at times been unrelated to the operating performance of the companies whose stocks were affected. This is due to several factors, including general market conditions, sales of Soliris, the announcement of the results of Alexion’s clinical trials or product development and the results of Alexion’s efforts to obtain regulatory approval for Alexion’s products. While Alexion cannot predict its future performance, if Alexion’s stock price continues to fluctuate in a wide range, an investment in Alexion’s common stock may result in considerable uncertainty for an investor.
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Anti-takeover provisions of Delaware law, provisions in Alexion’s charter and bylaws could make a third-party acquisition of Alexion difficult and may frustrate any attempt to remove or replace Alexion’s current management.
Because Alexion is a Delaware corporation, the anti-takeover provisions of Delaware law could make it more difficult for a third party to acquire control of Alexion, even if the change in control would be beneficial to stockholders. Alexion is subject to the provisions of Section 203 of the DGCL, which prohibits a person who owns in excess of 15% of its outstanding voting stock from merging or combining with Alexion for a period of three years after the date of the transaction in which the person acquired in excess of 15% of Alexion’s outstanding voting stock, unless the merger or combination is approved in a prescribed manner.
Alexion’s corporate charter and by-law provisions may discourage certain types of transactions involving an actual or potential change of control that might be beneficial to Alexion or its stockholders. Alexion’s bylaws provide that special meetings of Alexion’s stockholders may be called only by the Chairman of the Board, the President, the Secretary or a majority of the Board of Directors, or upon the written request of stockholders who together own of record 50% of the outstanding stock of all classes entitled to vote at such meeting. Alexion’s bylaws also specify that the authorized number of directors may be changed only by resolution of the board of directors. Alexion’s charter does not include a provision for cumulative voting for directors, which may have enabled a minority stockholder holding a sufficient percentage of a class of shares to elect one or more directors. Under Alexion’s charter, Alexion’s board of directors has the authority, without further action by stockholders, to designate up to 5,000 shares of preferred stock in one or more series. The rights of the holders of common stock will be subject to, and may be adversely affected by, the rights of the holders of any class or series of preferred stock that may be issued in the future.
Alexion’s board of directors decided to accelerate the expiration of Alexion’s shareholder rights plan after reviewing Alexion’s governance profile and current practices, considering the vote results on a related non-binding shareholder proposal presented at Alexion’s 2014 annual meeting of shareholders, and determining that it was in the best interests of Alexion and Alexion’s shareholders. The shareholder rights plan expired in March 2015.
Alexion’s corporate charter and bylaw provisions and stockholder rights plan may discourage certain types of transactions involving an actual or potential change of control that might be beneficial to Alexion or Alexion’s stockholders. Alexion’s bylaws provide that special meetings of its stockholders may be called only by the Chairman of the Board, the President, the Secretary or a majority of the board of directors, or upon the written request of stockholders who together own of record 50% of the outstanding stock of all classes entitled to vote at such meeting. Alexion’s bylaws also specify that the authorized number of directors may be changed only by resolution of the board of directors. Alexion’s charter does not include a provision for cumulative voting for directors, which may have enabled a minority stockholder holding a sufficient percentage of a class of shares to elect one or more directors. Under Alexion’s charter, its board of directors has the authority, without further action by stockholders, to designate up to 5,000 shares of preferred stock in one or more series. The rights of the holders of common stock will be subject to, and may be adversely affected by, the rights of the holders of any class or series of preferred stock that may be issued in the future.
Pursuant to Alexion’s stockholder rights plan, each share of common stock has an associated preferred stock purchase right. The rights will not trade separately from the common stock until, and are exercisable only upon, the acquisition or the potential acquisition through tender offer by a person or group of 20% or more of the outstanding common stock. The rights are designed to make it more likely that all of Alexion’s stockholders receive fair and equal treatment in the event of any proposed takeover of Alexion and to guard against the use of partial tender offers or other coercive tactics to gain control of Alexion. These provisions could delay or discourage transactions involving an actual or potential change in control of Alexion or its management, including transactions in which stockholders might otherwise receive a premium for their shares over then current prices. These provisions could also limit the ability of stockholders to remove current management or approve transactions that stockholders may deem to be in their best interests and could adversely affect the price of Alexion’s common stock.
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Risks Related to Synageva’s Business
Synageva is largely dependent on the success of Kanuma. All of Synageva’s product candidates, including Kanuma, are still in development. Preclinical and clinical trials of Synageva’s product candidates may not be successful.
Synageva’s business prospects are largely dependent upon the successful development and commercialization of Kanuma. Before Synageva can commercialize any of its product candidates, including Kanuma, it needs to:

conduct substantial research and development;

undertake preclinical and clinical testing and other costly and time consuming measures;

scale-up and transfer manufacturing processes while maintaining consistent product quality; and

pursue and obtain marketing and manufacturing approvals and, in some jurisdictions, pricing and reimbursement approvals.
This process involves a high degree of risk and takes many years. Synageva’s product development efforts with respect to a product candidate may fail for many reasons, including:

failure of the product candidate in preclinical studies;

failure to obtain, or delays in obtaining, the required regulatory approvals to initiate or continue clinical studies for the product candidate;

failure of later trials to confirm positive results from earlier preclinical studies or clinical trials;

delays or difficulty enrolling patients in clinical trials, particularly for disease indications with small patient populations;

failure to identify a sufficient number of patients who meet the clinical trial enrollment criteria and/or who would support commercial launch and subsequent commercialization efforts;

patients exhibiting adverse reactions to the product candidate or indications of other safety concerns;

insufficient clinical trial data to support the safety, effectiveness or superiority of the product candidate;

inability to produce proteins with favorable or superior characteristics using Synageva’s proprietary EW manufacturing platform;

failure to obtain, or delays in obtaining, the required regulatory approvals for the product candidate, or the facilities or processes used to manufacture the product candidate; or

changes in the regulatory or pricing and reimbursement environments could make development of a new product or further development of an existing product for a new indication no longer desirable.
Few research and development projects result in commercial products, and success in preclinical studies or clinical trials often is not replicated in later studies.
Synageva may decide to abandon development of a product candidate or service at any time, or it may be required to expend considerable resources repeating clinical trials or conducting additional trials, either of which would increase costs of development and delay any revenue from those programs. In addition, a regulatory authority may delay or deny an approval because it is not satisfied with the design, conduct, or results of clinical trials or due to its assessment of the data Synageva supplies.
Synageva has neither obtained marketing approval, nor commercialized any of its current product candidates.
Synageva has submitted a BLA to the FDA and an MAA to the EMA for Kanuma as a treatment for patients with LAL Deficiency, but has neither obtained marketing approval nor commercialized any of its current product candidates and does not know if or when it will receive marketing approval or generate
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revenue from the direct sale of an approved product, including Kanuma. Synageva has only limited clinical experience, which might prevent it from successfully designing or implementing a clinical trial for any of the indications Synageva currently, or in the future, target. Synageva may not be able to demonstrate that its product candidates meet the appropriate standards for regulatory approval, including because it may be unsuccessful in convincing regulatory authorities of the adequacy of the design of its trials or the sufficiency of the data generated from its clinical and other studies. If Synageva is not successful in conducting and managing its preclinical development activities or clinical trials or obtaining regulatory approvals, Synageva might not be able to commercialize its lead programs, or might be significantly delayed in doing so, which will materially harm its business.
If Synageva’s preclinical or clinical studies do not produce positive results or are delayed or if serious side effects are identified during drug development, it may experience delays, incur additional costs, receive a narrow label, or ultimately be unable to obtain regulatory approval and commercialize its product candidates.
Before obtaining regulatory approval for the sale of its product candidates, Synageva must conduct, at its own expense, extensive preclinical tests to demonstrate the safety of its product candidates in animals, and clinical trials to demonstrate the safety and efficacy of its product candidates in humans. Preclinical and clinical testing is expensive, difficult to design and implement and can take many years to complete. A failure of one or more preclinical studies or clinical trials can occur at any stage of testing. Synageva may experience numerous events during, or as a result of, preclinical testing and the clinical trial process, including for potential new indications, which could delay or prevent the receipt of regulatory approval for, or the commercialization of, Synageva’s product candidates, including:

Synageva’s preclinical tests or clinical trials may produce negative or inconclusive results, and Synageva may decide or regulators may require us, to conduct additional preclinical testing prior to initiating clinical trials, or to suspend ongoing studies;

Synageva may decide, or regulators may require it, to change the design of its preclinical studies or clinical trials in ways that may slow their progress, delay the initiation of clinical trials, or Synageva may abandon projects that it expects to be promising;

a regulatory authority or institutional review board may not authorize Synageva to commence a clinical trial or conduct a clinical trial at a prospective trial site;

conditions imposed on Synageva by the FDA or any non-U.S. regulatory authority regarding the scope or design of Synageva’s clinical trials may require Synageva to resubmit its clinical trial protocols to these authorities or to institutional review boards or ethics committees for re-review due to changes in the regulatory environment;

the number of patients required for clinical trials may be larger than Synageva anticipates or is able to enroll, or participants may drop out of, or not qualify for, clinical trials at a higher rate than Synageva anticipates;

Synageva’s third-party contractors or clinical investigators may fail to comply with regulatory requirements or fail to meet their contractual obligations to Synageva in a timely manner or at all;

Synageva might have to suspend or terminate one or more of its clinical trials if it, a regulatory authority or an institutional review board or ethics committee determines that the participants are being exposed to unacceptable health risks;

a regulatory authority or institutional review board or ethics committee may require that Synageva hold, suspend or terminate clinical research for various reasons, including noncompliance with regulatory requirements;

a regulatory authority may require that Synageva conduct additional clinical research to provide additional information regarding the efficacy or safety of Kanuma;

the cost of Synageva’s clinical trials may be greater than Synageva anticipates;
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the supply or quality of Synageva’s product candidates or other materials necessary to conduct its clinical trials may be insufficient or inadequate or Synageva may not be able to reach agreements on acceptable terms with prospective contract manufacturing organizations;

Synageva may not be able to produce, or sufficiently test, comparable or consistent drug materials derived from different manufacturing facilities operated by it or from processes run by third party manufacturers which could impact Synageva’s ability or timing with respect to receiving regulatory approval for its product candidates;

Synageva may not be able to reach agreements on acceptable terms with prospective clinical research organizations;

if approved, Synageva may not be able to reach agreements on acceptable terms with commercial distributors and other logistics providers; or

the effects of Synageva’s product candidates may not be the desired effects, may include undesirable side effects, or the product candidates may have other unexpected characteristics.
Synageva may obtain approval for indications that are not as broad as intended or entirely different than those indications for which it sought approval. For example, even though Synageva met the pre-specified primary and six secondary endpoints in the Phase 3 clinical trial for children and adults, potential regulatory approval could be limited to the treatment of LAL Deficiency only in infants, and that would represent a small portion of the total LAL Deficiency patient population. If Synageva is required to conduct additional clinical trials or other testing of its product candidates beyond those that it currently contemplates or is unable to successfully complete its clinical trials or other testing or if the results of these trials or tests are not positive or are only modestly positive or if there are safety concerns, Synageva may:

be delayed in obtaining, or may not be able to obtain, marketing approval for one or more of Synageva’s product candidates;

incur substantial additional costs in conducting such additional clinical trials or other testing;

obtain regulatory approval for only a narrower indication or an indication that might be otherwise qualified or constrained; or

have the product removed from the market after obtaining marketing approval.
Synageva’s product development costs will also increase if it experiences delays in testing or approvals. Synageva does not know whether any preclinical tests or clinical trials will be initiated as planned, will need to be restructured or will be completed on schedule, if at all. Due to the limited term of a patent, significant preclinical or clinical trial delays could also shorten the period of time from marketing approval to patent expiration, during which Synageva may benefit from patent protection of its product candidates. Such delays could allow Synageva’s competitors to bring products to market before it does, impairing Synageva’s ability to commercialize its products or product candidates.
Synageva may find it difficult to enroll patients in its clinical trials.
Potential patients for Synageva’s product candidates may not be adequately diagnosed or identified with the diseases being targeted by its product candidates. Kanuma is being developed to treat LAL Deficiency, which is very rare. Based on prevalence estimates published in the medical literature, Synageva estimates there are at least 3,000 children and adults with LAL Deficiency in the major reimbursable markets. In addition, Synageva is recruiting patients to enroll in its Phase 1/2 trial for MPS IIIB. Synageva may not be able to initiate or continue clinical trials if it is unable to locate a sufficient number of eligible patients to participate in the clinical trials required by the FDA or other non-U.S. regulatory agencies. In addition, the process of finding and diagnosing patients may prove costly. Synageva’s inability to enroll a sufficient number of patients for any of its current or future clinical trials would result in significant delays or may require it to abandon one or more clinical trials altogether.
The results of Synageva’s clinical trials may not prove sufficient to obtain regulatory approval of its product candidates, and subsequent trials may fail to replicate promising data seen in earlier preclinical studies and clinical trials.
Promising results in Synageva’s preclinical studies or clinical trials may not be replicated in ongoing and future studies or trials, and final data analysis may differ from interim data analysis. Even though
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Synageva has reported positive top-line results from its Phase 3 clinical trial in children and adults with LAL Deficiency and Synageva’s Phase 2/3 trial in infants with LAL Deficiency, and even if its other clinical trials for Kanuma are conducted and completed as planned, the results may not prove sufficient to obtain regulatory approval or result in a restricted product label that could negatively impact commercialization. Success in preclinical testing does not ensure success in clinical trials, and success in early stage clinical trials does not ensure success in later clinical trials. This can be due to a variety of reasons, including variations in patient populations, or the inability of certain patients to complete all assessments required by the clinical trial protocol, adjustments to clinical trial protocols or designs as compared to earlier testing or trials, variations in the data that could produce inconclusive or uninterpretable results, or the use of additional trial sites or investigators. Clinical trial data are subject to differing interpretations, and regulatory agencies may not concur with Synageva’s analysis of clinical trial data or its implications, which may result in delays in the regulatory approval process. Ongoing and future studies, including for new indications, may, for example, indicate safety concerns that regulatory authorities view as unacceptable. Final data analysis of Synageva’s completed, ongoing and future clinical trials may fail to demonstrate that its product candidates are sufficiently safe and effective for pursued or new indications. Any such failure could cause Synageva to abandon a product candidate, substantially delay development of other product candidates, or require substantial expenditures to conduct additional trials. Both preclinical and clinical data are often susceptible to varying interpretations that may delay, limit or prevent initiation of clinical trials, regulatory approvals or commercialization. Any delay in, or termination of, Synageva’s clinical trials would delay its obtaining regulatory approval of the affected product candidate and, consequently, its ability to commercialize that product candidate and potentially its other product candidates. Development and commercialization of therapies for rare diseases requires expenditure of significant funds with no assurance of success.
A regulatory authority may deny or delay approval of Synageva’s product candidates, including Kanuma, because it is not satisfied with the structure or conduct of Synageva’s clinical trials or due to its assessment of the data Synageva supplies, or may determine to approve Synageva’s product candidates for use in narrow patient populations.
A regulatory authority may not agree with the design or endpoints of Synageva’s clinical trials. Synageva sought advice from EMA for its Kanuma development plan, including the Phase 3 clinical trial design for Kanuma in children and adults with LAL Deficiency and EMA was supportive of Synageva’s plan in principle. Synageva did not seek a special protocol assessment with the FDA and does not have agreement with the FDA regarding the design or primary endpoint utilized in the Phase 3 clinical trial. Accordingly, Synageva believes that it will need to demonstrate efficacy based on a totality of evidence including success on multiple endpoints in Synageva’s clinical studies to support a favorable risk-benefit profile and provide substantial evidence of efficacy of Kanuma for the treatment of LAL Deficiency, including data from the Phase 3 clinical trial in children and adults, the Phase 2/3 open-label trial in infants with LAL Deficiency, as well as from the natural history studies for LAL Deficiency. Based on FDA feedback, it will be essential to link the primary endpoint for the Phase 3 clinical trial, alanine aminotransferase (“ALT”) normalization, to other evidence of clinical benefit. Synageva also will need to provide evidence to the FDA demonstrating that ALT normalization, alone or in combination with other endpoints, is reasonably likely to predict clinical benefit. If Synageva is unable to do so, even though it met the pre-specified primary endpoint and six secondary endpoints in the Phase 3 clinical trial and the primary endpoint in the Phase 2/3 study in infants, Synageva may not receive regulatory approval or it may receive narrower labeling with respect to the total LAL Deficiency patient population, unless and until it successfully completes additional clinical trials, if ever. In addition, regulatory authorities may not believe that Synageva has provided sufficient safety data or adequately demonstrated clinical benefit in the patient population studied in the clinical trial. Clinical data is subject to varied interpretations, and regulatory authorities may disagree with Synageva’s assessments of data. In any such case, a regulatory authority could insist that Synageva provide additional data or conduct additional clinical studies, which could substantially delay or even prevent commercialization efforts, particularly if Synageva is required to conduct additional pre-approval clinical studies. A positive opinion by the Committee for Medicinal Products for Human Use (“CHMP”) is required for EMA approval and requires agreement among a majority of CHMP members, each of whom may have differing opinions on the strength of the evidence Synageva may provide.
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Priority review for Synageva’s drug product candidates may not actually lead to a faster review process, if at all.
The FDA has accepted for review the BLA for Kanuma for the treatment of LAL Deficiency. The FDA granted Synageva’s request for Priority Review, which shortens the regulatory review period. The FDA established a target action date of September 8, 2015 under the PDUFA. The FDA review timeline for first cycle review could be longer and the FDA could issue a complete response letter requiring additional data to be submitted.
Because FDA also requires parallel approval of a New Animal Drug Application (“NADA”) for transgenic products such as Kanuma, this could create delays in the review and approval process for the BLA. A lengthier review process will delay revenue from the sale of products and will increase the capital necessary to fund Synageva’s product development programs. In addition, Kanuma received Fast Track Designation by the FDA, and Breakthrough Therapy designation by the FDA for LAL Deficiency presenting in infants. The practical implications of Fast Track and Breakthrough Therapy designation on the regulatory review and approval process cannot be determined at this time and may not lead to a faster review or approval. In addition, the approval of the NADA and BLA will require successful completion of inspections and resolution of any significant issues raised during these inspections.
Synageva also submitted an MAA to the EMA for Kanuma as a treatment for patients with LAL Deficiency. The EMA validated the MAA and granted Synageva’s request for accelerated assessment, which has the potential to shorten the EMA’s regulatory review time. However, the review timelines to reach a CHMP opinion and EMA action will depend in part on how efficiently Synageva responds to questions which stop the clock during the review. In addition, the approval of the MAA will require successful completion of inspections and resolution of any significant issues raised during these inspections.
Synageva’s product candidates, including Kanuma, if approved by any regulatory authorities could be subject to labeling and other restrictions, and Synageva will be subject to ongoing regulatory obligations, oversight and continued regulatory review, which may result in significant additional expense.
Any regulatory approvals that Synageva obtains for its product candidates will be subject to limitations on the approved indicated uses or patient population for which the product may be recommended for use or marketed, or to the conditions of approval, including a possible risk evaluation and mitigation strategy or post-marketing commitments, requirements, or follow-up measures. In addition, if the FDA, EMA or other regulatory authorities approve a product candidate, the manufacturing processes, labeling, packaging, distribution, storage, adverse event reporting, dispensation, distribution, advertising, promotion and recordkeeping for the product will be subject to extensive and ongoing regulatory requirements. These requirements will include submissions of safety and other post-marketing information and reports, ongoing maintenance of product registration, as well as continued compliance with current good manufacturing practices (“cGMPs”), good clinical practices and good laboratory practices. If Synageva does not comply with the applicable regulations and requirements, the range of possible sanctions includes issuance of adverse publicity, product recalls or seizures, fines, total or partial suspensions of production and/or distribution, suspension of marketing applications, and enforcement actions, including injunctions and civil or criminal prosecution. The FDA and comparable international regulatory agencies can withdraw a product’s approval under some circumstances, such as the failure to comply with regulatory requirements or unexpected safety issues.
Regulatory approvals could also contain requirements for potentially costly post-marketing testing, including Phase 4 clinical trials and extraordinary requirements for surveillance to monitor the safety and efficacy of the drug product. Post-marketing studies and/or post-market surveillance may suggest that a product causes undesirable side effects which present an increased risk to the patient. If data Synageva collects from post-marketing studies suggest that one of its approved products may present a risk to safety, the regulatory authorities could withdraw Synageva’s product approval, suspend production or place other labeling or marketing restrictions on Synageva’s products. If regulatory sanctions are applied or if regulatory approval is delayed or withdrawn, the value of Synageva and its business, financial condition and operating results will be adversely affected.
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If the market opportunities for Synageva’s product candidates are smaller than it believes they are, Synageva’s revenues may be adversely affected and its business may suffer.
Synageva focuses its research and product development on treatments for rare diseases. Synageva’s projections of both the number of people who have these diseases, as well as the subset of people with these diseases who have the potential to benefit from treatment with its product candidates, are based on estimates. In addition, the awareness of LAL Deficiency among treating health care providers is low. Currently, most reported estimates of the prevalence of these diseases are based on studies of small subsets of the population of specific geographic areas, which are then extrapolated to estimate the prevalence of the diseases in the broader world population. Based on prevalence estimates published in the medical literature, Synageva estimates there are at least 3,000 children and adults with LAL Deficiency in the major reimbursable markets. In addition, there is no prevalent population for infants with LAL Deficiency, since these infants almost never survive beyond the first year of life. These estimates may prove to be incorrect and new studies may change the estimated prevalence of these diseases. If the estimates are incorrect, and the prevalence rate is lower than Synageva anticipates, or if an approved indication is limited in any way, Synageva’s commercial business may suffer.
The commercial success of any product candidate that Synageva may develop, including Kanuma, will depend upon the degree of market acceptance by physicians, patients, third party payors and others in the medical community.
Any future product that Synageva may bring to the market, including Kanuma, may not gain market acceptance by physicians, patients, third party payors and others in the medical community. If Synageva’s products do not achieve an adequate level of acceptance, it may not generate significant product revenue and may not become profitable. The degree of market acceptance of these product candidates, if approved for commercial sale, will depend on a number of factors, including:

the prevalence and severity of any side effects of the product, including any limitations or warnings contained in a product’s approved labeling;

the perception of clinical benefit, safety, and potential advantages over alternative treatments;

relative convenience and ease of administration;

the willingness of the target patient population to try new therapies and of physicians to prescribe these therapies;

the strength of marketing and distribution support and timing of market introduction of competitive products;

publicity concerning Synageva’s products or competing products and treatments; and

sufficient third party insurance coverage or reimbursement in the countries or geographies where patients live.
Even if a potential product displays a favorable efficacy and safety profile in preclinical and clinical trials, market acceptance of the product will not be known until after it is launched. Synageva’s efforts to educate the medical community and third party payors on the benefits of the product candidates may require significant resources and may never be successful. Such efforts to educate the marketplace may require more resources than are required by the conventional technologies marketed by Synageva’s competitors.
Uncertainties relating to third-party reimbursement and health care reform measures could limit payments or reimbursements for future products that Synageva may develop could materially adversely affect its business.
In the U.S. and elsewhere, sales of prescription drugs depend in part on the consumers’ ability to obtain reimbursement for the cost of the drugs from third-party payors, such as private and government insurance programs. Third-party payors are increasingly challenging the prices charged for medical products and services, including those related to rare diseases, in an effort to promote cost containment measures and alternative health care delivery systems. Synageva’s prospects for achieving profitability will depend heavily
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upon the availability of adequate reimbursement for the use of its approved product candidates from governmental and other third party payors, both in the U.S. and in other markets. Reimbursement by a third party payor may depend upon a number of factors, including the third party payor’s determination that use of a product is:

a covered benefit under its health plan;

safe, effective and medically necessary;

appropriate for the specific patient;

cost-effective; and

neither experimental nor investigational.
Obtaining reimbursement approval for a product from each governmental or other third party payor is a time consuming and costly process that could require Synageva to provide supporting scientific, clinical and cost effectiveness data for the use of Synageva’s products to each payor. Synageva may not be able to provide data sufficient to gain acceptance with respect to reimbursement or might need to conduct post-marketing studies in order to demonstrate the cost-effectiveness of any future products to such payors’ satisfaction. Such studies might require Synageva to commit a significant amount of management time and financial and other resources. Even when a payor determines that a product is eligible for reimbursement, the payor may impose coverage limitations that preclude payment for some uses that are approved by the FDA or non-U.S. regulatory authorities. In addition, there is a risk that full reimbursement may not be available for high priced products. Moreover, eligibility for coverage does not imply that any product will be reimbursed in all cases or at a rate that allows Synageva to make a profit or even cover its costs. Interim payments for new products, if applicable, may also not be sufficient to cover costs and may not be made permanent. Third party payors may attempt to contain health care costs by demanding price discounts or rebates and limiting both the types and variety of drugs that they will cover and the amounts that they will pay for drugs. As a result, they may not cover or provide adequate payment for Synageva’s products. Synageva’s products might not ultimately be considered cost-effective. Adequate third-party reimbursement might not be available to enable Synageva to maintain price levels sufficient to realize an appropriate return on Synageva’s investment in product development.
Reimbursement rates vary according to the use of the drug and the clinical setting in which it is used, may be based on payments allowed for lower-cost products that already are reimbursed, may be incorporated into existing payments for other products or services and may reflect budgetary constraints and/or imperfections in the data used to calculate these rates. Net prices for products are reduced by mandatory discounts or rebates required by government health care programs and privately-negotiated discounts. The U.S. federal government, state governments and private payors frequently pursue actions against pharmaceutical and biotechnology companies alleging that the companies have overstated prices in order to inflate reimbursement rates. Any such action could adversely affect the pricing of and revenues from Synageva’s products.
Specialty pharmaceuticals are drugs that are prescribed by specialist physicians to treat rare or life-threatening conditions and typically address smaller patient populations. Each of Synageva’s product candidates is a specialty pharmaceutical product. The increasing availability and use of innovative specialty pharmaceuticals, combined with their relative higher cost as compared to other types of pharmaceutical products, is beginning to generate significant third party payor interest in developing cost-containment strategies targeted to this sector. The increasing use of health technology assessments in markets around the world and the financial challenges faced by many governments may lead to significant adverse effects on Synageva’s business.
Any legislation or regulatory changes or relaxation of laws that restrict imports of drugs from other countries also could reduce the net price Synageva receives for its products.
Synageva is subject to regulations regarding the manufacturing of therapeutic proteins and, if it is unable to comply, or if it or any third party provider fails to provide sufficient quantities of material, Synageva may experience delays and incur additional costs.
Synageva and its third party suppliers are subject to ongoing periodic unannounced inspections by the FDA, corresponding state agencies or non-U.S. regulatory authorities to ensure strict compliance with
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cGMPs and other government regulations and corresponding foreign standards. The cGMP requirements govern manufacturing, quality control and documentation policies and procedures. Complying with cGMP and non-U.S. regulatory requirements will require that Synageva expend time, money, and effort in production, recordkeeping, and quality control to assure that the product candidate or any approved product meets applicable specifications and other requirements. Synageva and its contract manufacturers and testing laboratories must also pass pre-approval inspections prior to regulatory approvals. Failure to pass a pre-approval inspection might significantly delay regulatory approval of Synageva’s product candidates. If Synageva or its contract manufacturers or testing laboratories fail to comply with these requirements, Synageva would be subject to possible regulatory action and might be limited in the jurisdictions in which it is permitted to sell its products. As a result, Synageva’s business, financial condition, and results of operations might be materially harmed.
Synageva currently manufactures the therapeutic protein product candidates that it is developing using both internal resources and external contract manufacturers; however, Synageva has limited experience in manufacturing or procuring products in commercial quantities and its manufacturing system has never been utilized to produce a product approved by regulatory authorities for commercial use. Synageva may not be able to manufacture enough product to conduct clinical trials or for later commercialization at an acceptable cost or at all. Synageva may also experience shortages in supply of its products which would have a material adverse impact on its business, financial condition and financial operations. Synageva may not be able to produce, or sufficiently test comparable drug materials derived from different manufacturing facilities operated by it or from processes run by its third party manufacturing partners, which could impact Synageva’s ability or timing with respect to receiving regulatory approval for its product candidates. In addition, a number of other factors could cause production interruptions at Synageva’s facilities or the facilities of its third-party providers, including equipment malfunctions, facility or product contamination, labor problems, raw material shortages or contamination, natural disasters, disruption in utility services, terrorist activities, human error or disruptions in the operations of its suppliers. Synageva’s product candidates are biologics and are very difficult to manufacture. Synageva employs multiple steps to attempt to control the manufacturing processes. Problems with these manufacturing processes, even minor deviations from the normal process, could result in product defects, contamination or manufacturing failures that result in lot failures, product recalls, product liability claims and insufficient material for clinical trials or commercial use. Certain of the raw materials required in the manufacturing and the formulation of Synageva’s product candidates are derived from biological sources, including egg white and human serum albumin. Such raw materials are difficult to procure and may be subject to contamination or recall. Also, some countries in which Synageva may operate could restrict the use of certain biologically derived substances in the manufacture of drugs. A material shortage, contamination, recall, or restriction on the use of certain biologically derived substances in the manufacture of Synageva’s products could adversely impact or disrupt manufacturing or could result in a withdrawal of Synageva’s product candidates or any approved products. As a result, Synageva’s business, financial condition, and results of operations might be materially harmed.
Synageva’s current and anticipated future reliance on a limited number of third parties to complete the manufacturing process for its products exposes Synageva to certain risks.
Synageva currently rely on third parties to complete the manufacturing process, including purifying, finishing, filling, labeling and testing Synageva’s products. Synageva have recently entered into agreements with third parties related to these steps for the commercial supply of Kanuma, and as Synageva do not have experience in producing Kanuma for commercial use, Synageva may experience unanticipated delays or issues in this process. Synageva’s reliance on a limited number of third-party manufacturers exposes Synageva to the following risks:

Synageva might be unable to identify or enter into agreements with manufacturers for clinical or commercial supply on acceptable terms or at all because the number of potential manufacturers is limited and the FDA and other regulatory authorities must approve any replacement contractor. This approval would generally require new testing and compliance inspections. In addition, a new manufacturer would have to be educated in, or develop substantially equivalent processes for, production of Synageva’s products prior to receipt of FDA approval, if any.
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Synageva’s third-party manufacturers might be unable to formulate and manufacture the relevant drugs in the volume and of the quality required to meet Synageva’s clinical and commercial needs, if any.

Synageva’s third-party contract manufacturers might not perform as agreed or might not remain in the contract manufacturing business for the time required to supply possible clinical trials or to successfully produce, store and distribute Synageva’s products.

Drug manufacturers are subject to ongoing periodic unannounced inspections by regulatory authorities, corresponding state agencies and non-U.S. regulatory authorities to ensure strict compliance with cGMP, and other government regulations and corresponding foreign standards. Synageva does not have complete or direct control over third-party manufacturers’ compliance with these regulations and standards.

If any third-party manufacturer makes improvements in the manufacturing process for the relevant products, Synageva might not own, or might have to share, the intellectual property rights to the innovation with Synageva’s licensors.

Synageva might compete with other companies for access to these manufacturers’ facilities and might be subject to manufacturing delays if the manufacturers give other clients higher priority than Synageva.
Each of these risks could delay Synageva’s clinical trials or the approval, if any, of Synageva’s product candidates by the FDA or the commercialization of Synageva’s product candidates and could result in higher costs or deprive Synageva of potential product revenues. As a result, Synageva’s business, financial condition, and results of operations might be materially harmed.
Even if Synageva obtains regulatory approval for its product candidates, if Synageva is unable to successfully develop internal and external commercialization capabilities, it will be unable to successfully commercialize them.
Synageva currently has limited internal capabilities for the commercialization of any product candidates that may be approved. In order to commercialize a product if approved, Synageva must develop its sales, marketing, contracting, distribution and reimbursement capabilities. Synageva will need to commit significant time and financial and managerial resources to develop a medical affairs team, a marketing and sales force with technical expertise, and sufficient distribution capabilities.
Factors that may inhibit Synageva’s efforts to develop its commercialization capabilities include:

Synageva’s inability to recruit and retain adequate numbers of effective medical and commercial personnel or manage a potential substantial increase in its number of full-time employees in a short period;

Synageva’s inability to train sales personnel, who may have limited experience with Synageva or its future products, to deliver a consistent message regarding the underlying disease that complies with regulatory requirements and be effective (after regulatory approval) in convincing physicians to prescribe Synageva’s future products;

Synageva’s inability to equip medical and sales personnel with effective materials, including medical and sales literature to help them educate physicians and other healthcare providers regarding applicable rare diseases and Synageva’s future products;

Synageva’s inability to develop or obtain sufficient operational functions to support its commercial activities; and

unforeseen costs and expenses associated with creating and sustaining an independent sales and marketing organization.
If Synageva is not successful in building a medical affairs and sales, marketing and other commercial infrastructure, it will have difficulty commercializing any future products, which would adversely affect Synageva’s business and financial condition.
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In addition, Synageva plans to rely on third parties to help distribute its future products to patients. Synageva expects that it will need to contract with third-party logistics companies to warehouse and distribute any approved products, and coordinate prescription intake and distribution, reimbursement adjudication, patient financial support, and ongoing compliance support. This distribution network will require significant coordination with Synageva’s sales and marketing and finance organizations. If Synageva is unable to effectively establish and manage the distribution process, the commercial launch and sales of any future products Synageva may commercialize, will be delayed or severely compromised and Synageva’s results of operations may be harmed.
Synageva may be subject, directly or indirectly, to healthcare fraud and abuse laws, false claims laws, and health information privacy and security laws. If Synageva is unable to comply, or has not fully complied, with such laws, Synageva could face substantial penalties and it could affect Synageva’s ability to develop, market and sell its potential products.
Synageva’s operations may be directly, or indirectly through its customers, subject to various federal and state fraud and abuse laws, including, without limitation, the federal Anti-Kickback Statute, the federal False Claims Act, and physician sunshine laws and regulations. The laws and regulations concerning promotion and marketing of drug products and dialogistic tests may impact, among other things, Synageva’s disease awareness and education programs, as well as its proposed sales and marketing activities for any of its products that might receive regulatory approval, including Kanuma. In addition, Synageva may be subject to patient privacy regulation by both the federal government and the states in which Synageva conducts its business. The laws that may affect Synageva’s ability to operate include, but are not necessarily limited to:

federal healthcare program anti-kickback law, which prohibits, among other things, persons from soliciting, receiving or providing remuneration, directly or indirectly, to induce either the referral of an individual for a healthcare item or service, or the purchasing or ordering of an item or service, for which payment may be made under a federal healthcare program such as Medicare or Medicaid;

federal false claims law, which prohibits, among other things, individuals or entities from knowingly presenting or causing to be presented, claims for payment by government funded programs such as Medicare or Medicaid that are false or fraudulent, and which may apply to Synageva by virtue of statements and representations made to customers or third parties;

HIPAA and the Health Information Technology and Clinical Health Act, which prohibit executing a scheme to defraud healthcare programs; impose requirements relating to the privacy, security, and transmission of individually identifiable health information; and require notification to affected individuals and regulatory authorities of certain breaches of security of individually identifiable health information;

the federal physician sunshine requirements under the health care reform laws requires manufacturers of drugs, devices, biologics, and medical supplies to report annually to the U.S. Department of Health and Human Services information related to payments and other transfers of value to physicians, other healthcare providers, and teaching hospitals, and ownership and investment interests held by physicians and other healthcare providers and their immediate family members and applicable group purchasing organizations; and

state laws comparable to each of the above federal laws, such as, for example, anti-kickback and false claims laws applicable to commercial insurers and other non-federal payors, requirements for mandatory corporate regulatory compliance programs, and laws relating to patient data privacy and security. Similar strict restrictions are imposed on the promotion and marketing of drug products in the European Union and other countries. Violations of the rules governing the promotion of drug products in the European Union could be penalized by administrative measures, fines and imprisonment. In addition, many countries have strict data privacy laws and violators could be subject to administrative measures and fines.
Interactions between pharmaceutical companies and physicians are also governed by strict laws, regulations, industry self-regulation codes of conduct and physicians’ codes of professional conduct in the individual European Union member states. The provision of any inducements to physicians to prescribe,
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recommend, endorse, order, purchase, supply, use or administer a drug product is prohibited. A number of European Union member states have introduced additional rules requiring pharmaceutical companies to publicly disclose their interactions with physicians and to obtain approval from employers, professional organizations and/or competent authorities before entering into agreements with physicians. Violations of these rules could lead to the imposition of fines or imprisonment.
Laws, including those governing promotion, marketing and anti-kickback provisions, industry regulations and professional codes of conduct are often strictly enforced. Increasing regulatory scrutiny of the promotional activities of pharmaceutical companies has been observed in the U.S. and a number of European Union member states. Synageva is also subject to the FCPA, the U.K. Bribery Act, and other anti-corruption laws and regulations pertaining to its efforts in this area. For example, the Bribery Act in the United Kingdom entered into force in July 2011 applies to any company incorporated in or “carrying on business” in the United Kingdom, regardless of the country in which the alleged bribery activity occurs and even if the inappropriate activity is undertaken by Synageva’s international distribution partners.
If Synageva infringes the rights of third parties, it might have to forgo selling its future products, pay damages, or defend litigation.
If Synageva’s product candidates or FUZEON, methods, processes, or other technologies infringe the proprietary rights of other parties, Synageva could incur substantial costs and might have to:

obtain rights or licenses from such third parties, which might not be available on commercially reasonable terms, if at all;

abandon an infringing product candidate;

redesign products or processes to avoid infringement;

stop using the subject matter claimed in the patents held by others;

pay damages; and/or

engage in litigation or administrative proceedings which might be costly whether Synageva wins or lose, and which could result in a substantial diversion of financial and management resources.
Any of these events could substantially harm Synageva’s earnings, financial condition, and operations.
Synageva’s business depends on protecting its intellectual property.
Synageva and its licensors are pursuing intellectual property protection for Kanuma and other product candidates in the form of patent applications that have been and will continue to be filed in the U.S. and in other countries; however, there can be no assurance that patents will issue with the scope for which they are originally filed, if at all.
If Synageva and its licensors do not obtain protection for Synageva’s respective intellectual property rights and its products are not, or are no longer, protected by regulatory exclusivity protection, such as orphan drug protection, Synageva’s competitors might be able to develop and commercialize competing drugs.
Synageva’s success, competitive position, and future revenues, if any, depend in part on its ability and the abilities of its licensors to obtain and maintain patent protection for Synageva’s products, methods, processes, and other technologies, to preserve Synageva’s trade secrets, to prevent third parties from infringing on Synageva’s proprietary rights, and to operate without infringing on the proprietary rights of third parties. Synageva currently holds various issued patents and exclusive licenses to issued patents and owns and has exclusive licenses to various patent applications, in each case in the U.S. as well as rights under foreign patents and patent applications. Synageva anticipates filing additional patent applications both in the U.S. and in other countries, as appropriate. However, the patent process is subject to numerous risks and uncertainties, and there can be no assurance that Synageva will be successful in protecting its products by obtaining and defending patents. These risks and uncertainties include the following:

Synageva’s patent rights might be challenged, invalidated, or circumvented, or otherwise might not provide any competitive advantage;
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Synageva’s competitors, many of which have substantially greater resources than Synageva does and many of which might make significant investments in competing technologies, might seek, or might already have obtained, patents that will limit, interfere with, make obsolete, or eliminate Synageva’s ability to make, use, and sell its potential products either in the U.S. or in international markets;

governments may adopt regulations requiring compulsory licensing of IP rights, and governments or courts may render decisions enforcing those regulations;

as a matter of public policy regarding worldwide health concerns, there might be significant pressure on the U.S. government and other international governmental bodies to limit the scope of patent protection both inside and outside the U.S. for disease treatments that prove successful; and

countries other than the U.S. might have less restrictive patent laws than the U.S., giving foreign competitors the ability to exploit these laws to create, develop, and market competing products.
In addition, the USPTO and patent offices in other jurisdictions have often required that patent applications concerning pharmaceutical and/or biotechnology-related inventions be limited or narrowed substantially to cover only the specific innovations exemplified in the patent application, thereby limiting the scope of protection against competitive challenges. Thus, even if Synageva or its licensors are able to obtain patents, the patents might be substantially narrower than anticipated.
Patent and other intellectual property protection is crucial to the success of Synageva’s business and prospects, and there is a risk that such protections will prove inadequate. Synageva’s business and prospects might be materially harmed if these protections prove insufficient.
On September 16, 2011, the Leahy-Smith America Invents Act (the “Leahy-Smith Act”), was signed into law. The Leahy-Smith Act includes a number of significant changes to United States patent law, including provisions that affect the way patent applications will be prosecuted and may also affect patent litigation. The USPTO has issued regulations and procedures to govern administration of the Leahy-Smith Act, but many of the substantive changes to patent law associated with the Leahy-Smith Act have only recently become effective. Accordingly, it is not clear what, if any, impact the Leahy-Smith Act will have on the operation of Synageva’s business. However, the Leahy-Smith Act and its implementation could increase the uncertainties and costs surrounding the prosecution of Synageva’s patent applications and the enforcement or defense of its issued patents, all of which could have a material adverse effect on Synageva’s business and financial condition.
Synageva relies on trade secret protections through confidentiality agreements with its employees and third parties, and the breach of these agreements could adversely affect Synageva’s business and prospects.
Synageva relies on trade secrets, which Synageva seeks to protect, in part, through confidentiality and non-disclosure agreements with its employees, collaborators, suppliers, and other parties. There can be no assurance that these agreements will not be breached, that Synageva would have adequate remedies for any such breach, or that Synageva’s trade secrets will not otherwise become known to or independently developed by its competitors. Synageva might be involved from time to time in litigation to determine the enforceability, scope, and validity of its proprietary rights. Any such litigation could result in substantial cost and divert management’s attention from operations. If any of these events occurs, or Synageva otherwise loses protection for its trade secrets or proprietary know-how, the value of this information may be greatly reduced.
Synageva is dependent on certain license relationships.
Synageva has licensed technology that is related to its proprietary expression technology from the University of Georgia, University of Minnesota and Pangenix. In addition, Synageva obtained exclusive worldwide rights to multiple patents and patent applications relating to the use of LAL for the treatment of LAL Deficiency and atherosclerosis from Shire Human Genetics Therapies, Inc. and its affiliates and Cincinnati Children’s Hospital Research Foundation. Synageva might enter into additional licenses in the future. Licenses to which Synageva is a party contain, and Synageva expect that any future licenses will contain, provisions requiring up-front, milestone, and royalty payments to licensors and other conditions to
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maintaining the license rights. If Synageva fails to comply with its obligations under any such license, the applicable licensor may have the right to terminate the license on relatively short notice and as a result, Synageva may not be able to commercialize product candidates or technologies that were covered by the applicable license. Also, the milestone and other payments associated with these licenses will make it less profitable for Synageva to develop its product candidates.
Synageva expects to rely on orphan drug exclusivity for Kanuma and SBC-103. A competitor may receive orphan drug marketing authorization prior to Synageva for the same indication for which Synageva is seeking approval.
Approval of Kanuma and SBC-103 as orphan drugs would grant Synageva seven years of marketing exclusivity under the Federal Food, Drug, and Cosmetic Act, and up to 10 years of marketing exclusivity in Europe. While the orphan drug designation for Kanuma and SBC-103 will provide market exclusivity in the U.S., Europe and Japan, Synageva will not be able to rely on market exclusivity to prevent other companies from obtaining regulatory approval in these territories for the same active ingredient for the same indication beyond that timeframe. Furthermore, the marketing exclusivity in Europe can be reduced from 10 years to six years if the initial designation criteria have significantly changed since the market authorization of the orphan drug or orphan exclusivity may be revoked if Synageva cannot reliably supply the market. Even if Synageva has orphan drug designation for a particular drug indication, Synageva cannot guarantee that another company also with orphan drug designation will not receive marketing authorization for the same indication before Synageva does. If that were to happen, Synageva’s applications for that indication may not be approved until the competing company’s period of exclusivity has expired. Also, Synageva cannot guarantee that another company with orphan drug designation will not receive marketing authorization for the same indication at the same time Synageva does. In this case, both companies would receive market exclusivity, which could have a material adverse effect on sales in that market. Even if Synageva is the first to obtain marketing authorization for an orphan drug indication, there are circumstances under which a competing product may be approved for the same indication during the seven-year period of marketing exclusivity in the U.S., such as if the later product is shown to be clinically superior to Synageva’s product, or if the later product is a different drug than Kanuma or SBC-103. Further, the seven-year marketing exclusivity in the U.S. would not prevent competitors from obtaining approval of the same compound for other indications or the use of other types of drugs for the same use as the orphan drug.
Synageva faces significant competition from other pharmaceutical and biotechnology companies. Synageva’s operating results will suffer if it fails to compete effectively.
The pharmaceutical and biotechnology industries are intensely competitive and subject to rapid and significant technological change. Synageva’s competitors include organizations such as major multinational pharmaceutical companies, established biotechnology companies and specialty pharmaceutical and generic drug companies. Many competitors have greater financial and other resources than Synageva has, such as larger research and development staff, more extensive marketing, distribution, sales and manufacturing organizations and experience, more extensive clinical trial and regulatory experience, expertise in prosecution of intellectual property rights and access to development resources like personnel and technology. As a result, these companies may develop or improve existing technologies that make Synageva’s manufacturing technology or product candidates obsolete or they may obtain regulatory approval more rapidly than Synageva is able to and may be more effective in selling and marketing their products.
If Synageva is unable to retain and recruit qualified scientists and advisors, or if any of Synageva’s key executives, key employees or key consultants discontinues his or her employment or consulting relationship with it, it may delay Synageva’s development efforts or otherwise harm Synageva’s business.
The loss of any of Synageva’s key executives, employees or key consultants could impede the achievement of its research, development and commercial objectives. Furthermore, recruiting and retaining qualified scientific personnel to perform research and development work in the future is critical to Synageva’s success. Synageva must also recruit and retain personnel experienced in manufacturing, marketing, selling, distributing, supporting and otherwise commercializing approved therapeutic products. Synageva may be unable to attract and retain personnel on acceptable terms given the competition among
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biotechnology, biopharmaceutical, and health care companies, universities, and non-profit research institutions for experienced scientists and other disciplines. Competition for employees may impact Synageva’s ability to recruit and retain qualified personnel in the future. Certain of Synageva’s officers, directors, scientific advisors, and/or consultants may from time to time serve as officers, directors, scientific advisors, and/or consultants of other biopharmaceutical or biotechnology companies. Synageva does not maintain “key man” insurance policies on any of its officers or employees. Synageva currently has employment contracts with its Chief Executive Officer, Sanj K. Patel, and other executive officers which provide for certain severance benefits. Consistent with Synageva’s current employment policies, all of its employees are employed “at will” and, therefore, each employee may leave Synageva’s employment at any time. If Synageva is unable to retain its existing employees, including qualified scientific personnel, and attract additional qualified candidates, Synageva’s business and results of operations could be adversely affected. Synageva is not aware of any key personnel who intend to retire or otherwise leave Synageva in the near future.
Synageva may pursue rapid expansion of Synageva’s workforce or diversify Synageva’s business strategy through mergers, acquisitions, licensing arrangements or other contractual arrangements with third parties which may require substantial resources and substantial amounts of time from members of Synageva’s senior management and involve numerous risks.
Synageva may spend substantial resources to hire additional employees or pursue acquisitions of new technologies or businesses that Synageva would expect to be complementary to Synageva’s current technologies or business focus through mergers, acquisitions, licensing arrangements or other contractual arrangements with third parties. Acquisitions of technologies, companies or product rights involve numerous risks, including potential difficulties in the integration of acquired operations such as retaining key employees of an acquired business, integrating research and development programs, not meeting financial objectives, increased costs, undisclosed liabilities not covered by insurance or terms of acquisition, and diversion of management’s attention and resources in connection with an acquisition. Synageva cannot ensure that Synageva will be successful in identifying, executing, and integrating acquisitions in the future.
If Synageva fail to manage projected growth, its results and operations may be adversely affected.
With Synageva’s growth and preparation for a potential commercial launch of Kanuma for the treatment of LAL Deficiency and the continued progress of its preclinical-stage programs, Synageva will be required to retain existing and add required new qualified and experienced personnel in the commercial, regulatory, manufacturing, quality, program management, clinical and medical areas over the next several years. Also, as Synageva’s preclinical pipeline diversifies through internal discoveries, or the acquisition or in-licensing of new molecules, Synageva will need to hire additional scientists to supplement its existing scientific expertise over the next several years.
Synageva’s staff, financial resources, systems, procedures or controls may be inadequate to support its expanding operations and Synageva’s management may be unable to take advantage of future market opportunities or manage successfully Synageva’s relationships with third parties if Synageva is unable to adequately manage its anticipated growth and the integration of new personnel.
Synageva’s operations are subject to the economic, political, legal and business conditions in the countries in which Synageva does business, and Synageva’s failure to operate successfully or adapt to changes in these conditions could cause Synageva’s operations to be limited or disrupted.
Synageva has expanded its operations outside of the United States and expects to continue to do so in the future. Synageva’s current operations in foreign countries subject it to certain risks that could cause Synageva’s operations to be limited or disrupted, including volatility in international economies, inflation, political instability, difficulties enforcing contractual and intellectual property rights, changes in laws, regulations or enforcement practices with respect to Synageva’s business, compliance with tax, privacy, employment and labor laws, costs and difficulties in recruiting and retaining qualified managers and employees to manage and operate the business in local jurisdictions and costs and difficulties in managing and monitoring international operations.
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Synageva is exposed to product liability and preclinical and clinical liability risks which could place a substantial financial burden upon it, should it be sued, if it does not have adequate liability insurance or general insurance coverage for such a claim.
Synageva’s business exposes it to potential product liability and other liability risks that are inherent in the testing, manufacturing and marketing of products like ours. Foreign regulations or clinical sites may require it, as sponsor, to be liable for medical outcomes even if an adverse event is not directly related to Synageva’s product candidate. In addition, the use in Synageva’s clinical trials of pharmaceutical formulations and products that Synageva’s potential collaborators may develop and the subsequent sale of these formulations or products by Synageva or its potential collaborators may cause Synageva to bear a portion or all of the product liability risks. As is common for companies sponsoring such clinical testing, Synageva carries product liability insurance. This insurance may in some instances may be insufficient to offset a negative judgment or settlement payment. As a result, a successful liability claim or series of claims brought against Synageva could have a material adverse effect on Synageva’s business, financial condition and results of operations.
Security breaches and other disruptions could compromise Synageva’s information and expose it to liability, which would cause Synageva’s business and reputation to suffer.
In the ordinary course of Synageva’s business, it collects and stores sensitive data, including intellectual property, Synageva’s proprietary business information and data about Synageva’s patients, suppliers, and business partners, and personally identifiable information. The secure maintenance of this information is critical to Synageva’s operations and business strategy. Some of this information could be an attractive target of criminal attack by malicious third parties with a wide range of motives and expertise, including organized criminal groups, “hactivists,” patient groups, disgruntled current or former employees, and others. Hacker attacks are of ever-increasing levels of sophistication, and despite Synageva’s security measures, its information technology and infrastructure may be vulnerable to such attacks or may be breached due to employee error or malfeasance. Any such breach could compromise Synageva’s networks and the information stored there could be accessed, publicly disclosed, lost or stolen. Furthermore, if Synageva’s systems become compromised, it may not promptly discover the intrusion. Like other companies in Synageva’s industry, Synageva has experienced attacks to its data and systems, including malware and computer viruses. Attacks could have a material impact on Synageva’s business, operations or financial results. Any access, disclosure or other loss of information could result in legal claims or proceedings, liability under laws that protect the privacy of personal information, disrupt Synageva’s operations, and damage Synageva’s reputation which could adversely affect Synageva’s business.
If Synageva does not achieve its projected development and commercialization goals in the time frames it expects and announces, the credibility of Synageva’s management and its organizational competence may be adversely affected.
For planning purposes, Synageva estimates the timing of the accomplishment of various scientific, preclinical, clinical, regulatory, market launch and commercialization goals, which Synageva sometimes refers to as milestones. These milestones may include the commencement or completion of scientific, preclinical and clinical studies, the submission of regulatory filings and eventual product launch.
From time to time, Synageva may publicly announce the estimated 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 Synageva’s estimates, in many cases for reasons beyond its control. For example, clinical trials may be delayed due to factors such as regulatory agency approval, institutional review board approvals, qualification of clinical sites, scheduling conflicts with participating clinicians and clinical institutions and the rate of patient enrollment. In most circumstances, Synageva relies on academic institutions, major medical institutions, governmental research organizations (U.S. or internationally based), clinical research organizations or contract manufacturing organizations to conduct, supervise or monitor some or all aspects of clinical trials involving Synageva’s product candidates. Synageva will have limited control over the timing and other aspects of these clinical trials.
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If Synageva does not meet the milestones as publicly announced (or as projected by various analysts who follow us), Synageva’s stockholders or potential stockholders may lose confidence in Synageva’s ability to meet overall product development and commercialization goals and, as a result, the price of Synageva’s common stock may decline.
Synageva’s success will depend in part on relationships with third parties. Any adverse changes in these relationships could adversely affect Synageva’s business, financial condition, or results of operations.
Synageva’s success will be dependent on its ability to maintain and renew business relationships with third parties and to establish new business relationships. There can be no assurance that Synageva’s management will be able to maintain such business relationships, or enter into or maintain new business contracts and other business relationships, on acceptable terms, if at all. The failure to maintain important business relationships could have a material adverse effect on Synageva’s business, financial condition, or results of operations.
Synageva’s charter documents and indemnification agreements require Synageva to indemnify its directors and officers to the fullest extent permitted by law, which may obligate Synageva to make substantial payments and to incur significant insurance-related expenses.
Synageva’s charter documents require Synageva to indemnify its directors and officers to the fullest extent permitted by law. This could require Synageva, with some legally prescribed exceptions, to indemnify its directors and officers against any and all expenses, judgments, penalties, fines, and amounts reasonably paid in defense or settlement of an action, suit, or proceeding brought against any of them by reason of the fact that he or she is or was Synageva’s director or officer. In addition, expenses incurred by a director or officer in defending any such action, suit, or proceeding must be paid by Synageva in advance of the final disposition of that action, suit or proceeding if Synageva receives an undertaking by the director or officer to repay the advance if it is ultimately determined that he or she is not entitled to be indemnified. Synageva has also entered into indemnification agreements with each of its directors and officers. In furtherance of these indemnification obligations, Synageva maintains directors’ and officers’ insurance in the amount of $30,000,000. For future renewals, if Synageva is able to retain coverage, Synageva may be required to pay a higher premium for its directors’ and officers’ insurance than in the past and/or the amount of its insurance coverage may be decreased.
Synageva may be unable to raise the substantial additional capital that it will need to further develop and commercialize its products.
As is typical of biotechnology companies at Synageva’s stage of development, Synageva’s operations consume substantial amounts of cash and it will need substantial additional funds to further develop and commercialize its products.
While Synageva will need to seek additional funding, Synageva may not be able to obtain financing on acceptable terms, or at all. In addition, the terms of Synageva’s financings may be dilutive to, or otherwise adversely affect, holders of Synageva’s common stock. Synageva may also seek additional funds through arrangements with collaborators or other third parties. These arrangements would generally require Synageva to relinquish rights to some of its technologies, product candidates or products, and Synageva may not be able to enter into such agreements, on acceptable terms, if at all. If Synageva is unable to obtain additional funding on a timely basis, it may be required to curtail or terminate some or all of its development programs, including some or all of its product candidates.
Synageva has incurred significant losses since its inception and anticipates that it will continue to incur losses for the foreseeable future. Synageva is a company with limited historical revenues, which makes it difficult to assess its future viability.
Synageva is a clinical-stage biopharmaceutical company. Biopharmaceutical product development is a highly speculative undertaking and involves a substantial degree of risk. Synageva has incurred significant losses since its inception, and at March 31, 2015, Synageva had an accumulated deficit of approximately $506.5 million. Synageva expects its expenses to increase in connection with its efforts to seek approval for and commercialize Kanuma and Synageva’s research and development of its other product candidates,
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including but not limited to, SBC-103 and SBC-105. As a result, Synageva expects to continue to incur significant research and development and other expenses related to its ongoing operations for the foreseeable future. If any of Synageva’s product candidates fail in clinical trials or do not gain regulatory approval, or if any of Synageva’s product candidates, if approved, fail to achieve market acceptance, Synageva may never become profitable. Even if Synageva achieves profitability in the future, it may not be able to sustain profitability in subsequent periods. Synageva’s prior losses, combined with expected future losses, have had and will continue to have an adverse effect on Synageva’s stockholders’ equity and working capital.
Synageva’s ability to use net operating loss carry forwards to reduce future tax payments may be limited if there is or has been a change in ownership of Synageva, or if taxable income does not reach sufficient levels.
Utilization of Synageva’s net operating loss (“NOL”) and research and development (“R&D”) credit carry forwards to reduce future tax payments depends in part on whether taxable income reaches sufficient levels prior to the expiration of these deferred tax assets. Synageva’s federal NOL carryforwards begin to expire in 2018 and Synageva’s state NOL carryforwards began to expire in 2014. Synageva has federal orphan drug credits and federal and state research tax credit carryforwards, which begin expiring in 2018 and 2023, respectively. Utilization of these deferred tax assets also may be subject to a substantial annual limitation under Section 382 of the Code due to ownership change limitations that have occurred previously or that could occur in the future. These ownership changes may limit the amount of NOL and R&D credit carry forwards that can be utilized annually to offset future taxable income and tax, respectively. As part of the merger of Trimeris and Synageva BioPharma Corp., a privately held Delaware corporation in 2011, Synageva acquired federal tax attributes that are significantly limited under Section 382 of the Code.
A valuation allowance of  $185.2 million and $128.8 million has been established at December 31, 2014 and 2013, respectively, to offset Synageva’s potential deferred tax assets.
Synageva may have exposure to additional tax liabilities which could have a material impact on its results of operations and financial position.
As a result of Synageva’s international operations, Synageva is subject to income taxes, as well as non-income based taxes, in both the United States and various foreign jurisdictions. Significant judgment is required in determining Synageva’s worldwide tax liabilities. Although Synageva believes its estimates are reasonable, the ultimate outcome with respect to the taxes Synageva owes may differ from the amounts recorded in its financial statements. If the Internal Revenue Service, or other taxing authority, disagrees with the positions Synageva takes, Synageva could have additional tax liability, and this could have a material impact on its results of operations and financial position. In addition, the United States government and other governments are considering and may adopt tax reform measures that significantly increase Synageva’s worldwide tax liabilities which could materially harm Synageva’s business, financial condition and results of operations.
Synageva’s management is required to devote substantial time to comply with public company regulations.
As a public company, Synageva incurs significant legal, accounting and other expenses. Sarbanes-Oxley and rules implemented by the SEC and Nasdaq impose various requirements on public companies, including those related to corporate governance practices. Synageva’s management and other personnel will need to devote substantial time to these requirements.
Sarbanes-Oxley requires, among other things, that Synageva maintain effective internal controls for financial reporting and disclosure controls and procedures. In particular, Synageva is required to perform system and process evaluation and testing of Synageva’s internal controls over financial reporting to allow management and Synageva’s independent registered public accounting firm to report on the effectiveness of Synageva’s internal controls over financial reporting, as required by Section 404 of Sarbanes-Oxley (“Section 404”). If Synageva is not able to comply with the requirements of Section 404, or if Synageva or its independent registered public accounting firm identifies deficiencies in Synageva’s internal controls over financial reporting that are deemed to be material weaknesses, the market price of Synageva’s stock could decline and Synageva could be subject to sanctions or investigations by Nasdaq, the SEC or other regulatory authorities.
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The market price and trading volume of Synageva’s common stock may be volatile.
The market price of Synageva’s common stock could fluctuate significantly for many reasons, including the following factors:

announcements of preclinical, clinical or regulatory developments or technological innovations by Synageva or its competitors;

changes in Synageva’s relationship with its licensors and other strategic partners;

Synageva’s quarterly operating results;

declines in sales of FUZEON;

developments in patent or other technology ownership rights;

public concern regarding the safety of Synageva’s products;

additional funds may not be available on terms that are favorable to Synageva and, in the case of equity financings, may result in dilution to Synageva’s stockholders;

government regulation of drug pricing; and

general changes in the economy, the financial markets or the pharmaceutical or biotechnology industries.
Additional factors beyond Synageva’s control may also have an impact on the price of Synageva’s stock. For example, to the extent that other large companies within Synageva’s industry experience declines in their stock price, Synageva’s stock price may decline as well. In addition, when the market price of a company’s common stock drops significantly, stockholders often institute securities class action lawsuits against the company. A lawsuit against Synageva could cause it to incur substantial costs and could divert the time and attention of Synageva’s management and other resources.
Future sales of substantial amounts of Synageva’s common stock, or the perception that such sales could occur, could adversely affect the market price of Synageva’s common stock.
Future sales into the public market of substantial amounts of Synageva’s common stock, or securities convertible or exchangeable into shares of Synageva’s common stock, including shares of Synageva’s common stock issued upon exercise of options and warrants, or perceptions that such sales could occur, could adversely affect the market price of Synageva’s common stock and Synageva’s ability to raise capital in the future.
Ownership of Synageva’s common stock is highly concentrated, and it may prevent you and other stockholders from influencing significant corporate decisions and may result in conflicts of interest that could cause Synageva’s stock price to decline.
Synageva’s executive officers and directors, together with their respective affiliates, beneficially own and control a significant portion of Synageva’s common stock. Accordingly, these executive officers, directors and their affiliates, acting individually or as a group, have substantial influence over the outcome of a corporate action requiring stockholder approval, including the election of directors, any merger, consolidation or sale of all or substantially all of Synageva’s assets or any other significant corporate transaction. These stockholders may also exert influence in delaying or preventing a change in control, even if such change in control would benefit Synageva’s other stockholders. In addition, the significant concentration of stock ownership may adversely affect the market value of Synageva’s common stock due to investors’ perception that conflicts of interest may exist or arise.
Anti-takeover provisions in Synageva’s charter 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 Synageva difficult.
Synageva’s certificate of incorporation and bylaws 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 Synageva’s common stock.
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Synageva has never declared or paid dividends on its common stock and does not anticipate paying dividends in the foreseeable future.
Synageva’s business requires significant funding, and Synageva does not anticipate paying any cash dividends on its common stock in the foreseeable future.
Synageva has broad discretion in how it uses its resources, and Synageva may not use its cash and investments effectively or in ways with which you agree.
Synageva’s management has broad discretion as to the application of company resources. Synageva’s stockholders may not agree with the manner in which Synageva’s management chooses to allocate and spend Synageva’s cash, cash equivalents and investments. Moreover, Synageva’s management may use Synageva’s resources for corporate purposes that may not increase the market price of its common stock.
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FORWARD-LOOKING STATEMENTS
Information both included and incorporated by reference in this document may contain forward-looking statements, which may be identified by their use of terms such as “intend,” “plan,” “may,” “should,” “will,” “anticipate,” “believe,” “could,” “estimate,” “expect,” “forecast,” “continue,” “potential,” “opportunity,” “project” and similar terms. These statements are based on certain assumptions and analyses that Alexion’s management or Synageva’s management believe are appropriate under the circumstances. However, these statements are subject to risks, uncertainties and assumptions. Should one or more of these risks or uncertainties materialize, or should the assumptions prove incorrect, actual results may differ materially from those expected, estimated or projected. Forward-looking statements speak only as of the date they are made, and neither Alexion nor Synageva undertakes any obligation to publicly update or revise any of them in light of new information, future events or otherwise.
All subsequent written and oral forward-looking statements attributable to Alexion, Synageva or any person acting on Alexion’s or Synageva’s behalf are qualified by the cautionary statements in this section.
Factors that could have a material adverse effect on Alexion’s or Synageva’s operations and future prospects or the consummation of the offer and the mergers, many of which are difficult to predict and beyond the control of Alexion or Synageva, include, but are not limited to:

failure to satisfy the conditions to consummate the transactions;

the occurrence of any event, change or other circumstances that could give rise to the termination of the transaction agreement;

the failure of the transactions to close in a timely manner or at all for any other reason;

the availability of financing for the transactions on the terms anticipated or at all;

the ability to successfully integrate Alexion and Synageva following completion of the transactions;

realization of the expected benefits of the transactions in a timely manner or at all;

the amount of the costs, fees, expenses and charges related to the mergers;

effects of the pendency of the transactions on relationships with employees, suppliers, customers and other business partners;

general political, economic and business conditions and industry conditions;

changes in laws or regulations;

challenges to intellectual property;

competition from other products, including other drugs treating the same diseases as the companies’ products and product candidates;

difficulties inherent in the research and development process;

adverse litigation or government action;

the inherent uncertainty associated with financial or other projections; and

the ability to implement and achieve business strategies successfully.
These risks and uncertainties, along with the risk factors discussed under “Risk Factors” in this document, should be considered in evaluating any forward-looking statements contained in this document.
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THE COMPANIES
Alexion
Alexion is a biopharmaceutical company focused on serving patients with severe and rare disorders through the innovation, development and commercialization of life-transforming therapeutic products. Alexion is the global leader in complement inhibition and has developed and markets Soliris as a treatment for patients with PNH and aHUS, two debilitating, rare and life-threatening disorders caused by chronic uncontrolled activation of the complement component of the immune system. Soliris is currently approved in nearly 50 countries for the treatment of PNH, and in nearly 40 countries for the treatment of aHUS. Alexion is evaluating other potential indications for Soliris in additional severe and devastating diseases beyond PNH and aHUS in which uncontrolled complement activation is the underlying mechanism, and is progressing in various stages of development with additional product candidates as potential treatments for patients with severe and life-threatening ultra-rare disorders. In 2014, Alexion filed for regulatory approval with the FDA, EMA and the MHLW for Strensiq, a targeted enzyme replacement therapy in Phase II clinical trials for patients with HPP an ultra-rare, genetic, and life-threatening metabolic disease characterized by impaired phosphate and calcium regulation, leading to progressive damage to multiple vital organs including destruction and deformity of bones, profound muscle weakness, seizures, impaired renal function, and respiratory failure. In July 2014, the EMA validated Alexion’s MAA for Strensiq for the treatment of HPP. In March 2015, the FDA accepted for Priority Review the BLA for Strensiq for treatment of patients with infantile- and juvenile-onset HPP. Alexion has approximately 2,400 employees and serves patients in 50 countries.
Alexion is a Delaware corporation that was established in 1992 and became a public company in 1996. Its shares are traded on Nasdaq under the ticker symbol “ALXN.”
The address and telephone number of Alexion’s principal executive offices is 352 Knotter Drive, Cheshire, Connecticut 06410, (203) 272-2596.
Alexion also maintains an Internet site at www.alxn.com. Alexion’s website and the information contained therein or connected thereto shall not be deemed to be incorporated herein, and you should not rely on any such information in making an investment decision.
Recent Developments
On May 8, 2015, Alexion received a subpoena in connection with an investigation by the Enforcement Division of the SEC requesting information related to Alexion’s grant-making activities and compliance with the FCPA. While the subpoena seeks information related to Alexion’s activities and policies and procedures worldwide, it notes in particular Japan, Brazil, Turkey and Russia. The subpoena also seeks information related to Alexion’s recalls of specific lots of Soliris and related securities disclosures. Alexion is committed to compliance with applicable laws and regulations and strives to operate at the highest ethical standards in all of its markets. Alexion is cooperating with the SEC’s investigation, which is in its early stages. At this time, Alexion is unable to predict the duration, scope or outcome of the SEC investigation.
Offeror
The Offeror is a Delaware corporation and a direct wholly owned subsidiary of Alexion. The Offeror was incorporated on April 28, 2015 for the purpose of making an exchange offer and consummating the first merger. The Offeror has engaged in no business activities to date and it has no material assets or liabilities of any kind, other than those incident to its formation and those incurred in connection with the exchange offer and the mergers.
The address and telephone number of the Offeror’s principal executive offices is c/o Alexion Pharmaceuticals, Inc., 352 Knotter Drive, Cheshire, Connecticut 06410, (203) 272-2596.
Merger Sub
Merger Sub is a Delaware limited liability company and direct wholly owned subsidiary of Alexion. Merger Sub was formed on April 28, 2015 for the purpose of consummating the second merger. Merger Sub has engaged in no business activities to date and it has no material assets or liabilities of any kind, other than those incident to its formation and those incurred in connection with the mergers.
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The address and telephone number of Merger Sub’s principal executive offices is c/o Alexion Pharmaceuticals, Inc., 352 Knotter Drive, Cheshire, Connecticut 06410, (203) 272-2596.
Synageva
Synageva is a biopharmaceutical company focused on the discovery, development and commercialization of therapeutic products for patients with rare diseases. Synageva has a pipeline of protein therapeutic programs for rare diseases with unmet medical needs that are at various stages of development. It is planning for a global launch of its lead product, sebelipase alfa for lysosomal acid lipase deficiency (LAL Deficiency) under the proposed brand name of KanumaTM. Synageva also has an active investigational new drug application with the FDA to evaluate a second program, SBC-103, a first mover-enzyme replacement therapy program for mucopolysaccharidosis IIIB (also known as Sanfilippo B syndrome), and a third pipeline program, SBC-105, a first-mover enzyme therapy in preclinical development for rare disorders of calcification, including the first planned target indication for generalized calcification in infants.
Synageva is a Delaware corporation that was established in 2008 and became a publicly traded company in 2011. Its shares trade on Nasdaq under the ticker symbol “GEVA.”
The address and telephone number of Synageva’s principal executive offices is 33 Hayden Avenue, Lexington, Massachusetts 02421, (781) 357-9900.
Synageva also maintains an Internet site at www.synageva.com. Synageva’s website and the information contained therein or connected thereto shall not be deemed to be incorporated herein, and you should not rely on any such information in making an investment decision.
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THE TRANSACTIONS
General
In the first merger, the Offeror will merge with and into Synageva with Synageva surviving the merger, and each share of Synageva common stock outstanding as of immediately prior to the effective time of the first merger (other than certain converted, cancelled and dissenting shares, as described elsewhere in this document) will be converted into the right to receive the transaction consideration.
The transaction consideration consists of:

$115.00 in cash, without interest and less any applicable withholding taxes; and

0.6581 shares of Alexion common stock, together with cash in lieu of any fractional shares of Alexion common stock, without interest and less any applicable withholding taxes.
Synageva stockholders will not receive any fractional shares of Alexion common stock in the first merger, and each Synageva stockholder who otherwise would be entitled to receive a fraction of a share of Alexion common stock pursuant to the first merger will be paid an amount in cash (without interest) in lieu thereof. See “Transaction Agreement — Transaction Consideration.”
Immediately following the first merger, the surviving corporation will merge with and into Merger Sub in the second merger.
As described elsewhere in this document, the purpose of the transactions as agreed between Alexion and Synageva is for Alexion to acquire control of, and ultimately all of the outstanding equity in, Synageva. The transaction agreement provides for two alternative means to achieve that purpose.
The transaction agreement contemplated in the first instance that Alexion would cause the Offeror to commence an exchange offer offering to exchange the transaction consideration for each outstanding share of Synageva common stock, and that, if the exchange offer were completed, Alexion would acquire the remaining shares of outstanding Synageva common stock through the first merger. If the offer were completed (such that Alexion would own at least a majority of the outstanding shares of Synageva common stock), the first merger would be governed by Section 251(h) of the DGCL, and accordingly no stockholder vote would be required to consummate the first merger.
However, the transaction agreement also entitled Alexion, under specified circumstances, to cause the termination of the exchange offer and to seek to instead effect the first merger through a “long-form” merger governed by Section 251(c) of the DGCL, and accordingly a vote of Synageva’s stockholders would be required to consummate the first merger. The transaction agreement provides that if the offer is terminated and the parties instead propose to effect the transactions through a long-form merger, Synageva will convene a meeting of Synageva stockholders to seek their approval of the transaction agreement.
If you have received this document in the mail, it means that the Offeror has terminated the exchange offer and the parties are instead seeking your approval of the transaction agreement in order to complete the transactions through a long-form merger subject to a stockholder vote.
Background of the Transactions
The Synageva board of directors, with the assistance of Synageva’s senior management, has regularly reviewed Synageva’s research and development activities relating to its product candidates, the potential for commercializing its product candidates, and the strategic alternatives available to Synageva to maximize stockholder value. As part of this review, the Synageva board of directors has periodically considered whether the continued execution of Synageva’s business strategy as a standalone company, or a possible license or sale of assets to, or a business combination with, a third party would provide the best avenue to enhance stockholder value. In January 2015, Synageva raised approximately $308.7 million in a public offering of shares of Synageva common stock to help fund Synageva’s expected cash needs as an independent entity. Investment funds advised by an adviser affiliated with Dr. Baker purchased 1,000,000 shares of Synageva common stock in the January 2015 public offering.
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The Alexion board of directors, with the assistance of Alexion’s senior management, has regularly reviewed opportunities for expanding Alexion’s metabolic rare disease franchise and pipeline, including by acquisition of development stage companies. For the reasons described under “— Alexion’s Reasons for the Transactions,” during these reviews Alexion had identified Synageva as a potential acquisition opportunity. On February 16, 2015, the Alexion board of directors met telephonically, together with members of Alexion’s senior management and representatives of Lazard Frères & Co. LLC, Alexion’s financial advisor, and Wachtell, Lipton, Rosen & Katz, Alexion’s external mergers and acquisitions counsel (“Wachtell Lipton”), to discuss the possibility of making an acquisition proposal for Synageva. As a result of this meeting, the Alexion board of directors authorized senior management to communicate to Synageva Alexion’s interest in acquiring Synageva for $175 per share (“Alexion’s $175 Proposal”). In addition to this meeting, the Alexion board of directors met, telephonically or in person, together with members of Alexion’s senior management and representatives of its financial and legal advisors, in advance of each subsequent proposal made to Synageva and in advance of final approval of the transaction agreement on May 5, 2015, to discuss the status of the transaction, receive presentations from management and the external advisors and to provide guidance to Alexion’s senior management, including Dr. Leonard Bell and Mr. David Hallal, and during the negotiation of the transaction agreement, to Alexion’s legal and financial advisors. At the time, Dr. Bell was Alexion’s Chief Executive Officer and was and continues to serve as Chairman of the Alexion board of directors. Mr. Hallal, who at the time was Alexion’s Chief Operating Officer and Chief Executive Officer-elect, became Alexion’s Chief Executive Officer on April 1, 2015.
On February 18, 2015, Dr. Bell telephoned Dr. Felix Baker, Chairman of the Synageva board of directors, to communicate Alexion’s $175 Proposal, which represented a premium of approximately 77.0% to Synageva’s closing price on February 17, 2015, a premium of approximately 61.7% to Synageva’s volume weighted average closing price for the 30 days ended February 17, 2015 and a premium of approximately 44.7% to Synageva’s all-time high closing price. Later on February 18, 2015, Dr. Bell telephoned Mr. Sanj K. Patel, Synageva’s President and Chief Executive Officer, to discuss Alexion’s $175 Proposal. Dr. Baker and Mr. Patel thereafter contacted Synageva’s external legal and financial advisors regarding Alexion’s $175 Proposal. Following the February 18, 2015 calls with Dr. Baker and Mr. Patel, Dr. Bell sent Dr. Baker a letter dated that same day confirming Alexion’s $175 Proposal, which Dr. Baker distributed to the members of the Synageva board of directors. In the letter, Alexion invited a member of the Synageva board of directors to serve on the Alexion board of directors following completion of the transaction between the parties.
On February 21, 2015, the Synageva board of directors held a meeting to discuss Alexion’s $175 Proposal. Representatives of Sullivan & Cromwell LLP, Synageva’s external mergers and acquisitions counsel (“Sullivan & Cromwell”), representatives of Ropes & Gray LLP, Synageva’s external corporate counsel (“Ropes & Gray”), and representatives of Goldman, Sachs & Co., Synageva’s financial advisor (“Goldman Sachs”), attended the meeting. In engaging Ropes & Gray as one of its external legal advisors, senior management of Synageva and Synageva’s board of directors were aware of Ropes & Gray’s representation of Alexion in various unrelated matters. In addition, senior management of Synageva and Synageva’s board of directors were aware that Goldman Sachs had previously provided financial services or underwriting services to Alexion, and Goldman Sachs advised the Synageva board of directors that during the two year period ended February 18, 2015, the Investment Banking Division of Goldman Sachs had not received any compensation for financial services or underwriting services provided to Alexion. Representatives of Sullivan & Cromwell and Ropes & Gray reviewed with the Synageva board of directors their fiduciary duties. The members of the Synageva board of directors discussed Alexion’s $175 Proposal among themselves and with representatives of Goldman Sachs, Sullivan & Cromwell and Ropes & Gray, and outlined the information and advice that they wished to receive from Synageva’s senior management and Synageva’s external advisors.
On March 2, 2015, the Synageva board of directors held another meeting to discuss Alexion’s $175 Proposal. Representatives of Goldman Sachs, Sullivan & Cromwell and Ropes & Gray attended the meeting. Synageva’s senior management reviewed certain financial projections concerning Synageva’s product candidates and described various aspects of Synageva’s research, development and commercialization plans. Representatives of Goldman Sachs reviewed certain indicative financial analyses for Synageva and provided an analysis of Alexion’s $175 Proposal in relation to these analyses. A representative of Sullivan & Cromwell described the directors’ fiduciary duties under various scenarios. In
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addition, the members of the Synageva board of directors, while not making a decision on whether to do so, also discussed whether Synageva should solicit the interest of other potentially interested counterparties for a potential transaction with Synageva. The directors discussed the risks attendant to such potential solicitations and to engaging in a transaction with Alexion or another party at this time, including the potential disruption at a critical juncture to Synageva’s business and the regulatory and launch process for its lead product candidate and the diversion of senior management time and attention. Following discussions, it was the consensus of the Synageva board of directors that Dr. Baker should communicate to Dr. Bell that the proposed price contained in Alexion’s $175 Proposal did not appear compelling in light of what they believed Synageva’s standalone value to be, and that, accordingly, Synageva was not interested in discussing a potential transaction with Alexion on the terms outlined in Alexion’s $175 Proposal.
On March 5, 2015, Dr. Baker telephoned Dr. Bell to inform him that Synageva was not interested in discussing a potential transaction with Alexion on the terms outlined in Alexion’s $175 Proposal. Dr. Bell informed Dr. Baker that Alexion could potentially improve the terms of Alexion’s $175 Proposal if it were provided with certain nonpublic information about Synageva and its product candidates that would assist Alexion in assessing its valuation of Synageva.
On March 5, 2015, representatives of Sullivan & Cromwell provided to representatives of Wachtell Lipton a draft confidentiality agreement incorporating a two-year standstill provision pursuant to which Alexion would be prohibited from taking certain actions with respect to Synageva for such period. From that date through March 9, 2015, representatives of Sullivan & Cromwell and Wachtell Lipton negotiated the terms of the confidentiality agreement, which was entered into by Synageva and Alexion on March 9, 2015.
On March 10, 2015, Mr. Patel and other members of Synageva’s senior management met with Dr. Bell and Mr. Hallal. At this meeting Synageva provided to Alexion information concerning Synageva’s product candidates.
On March 17, 2015, Dr. Bell telephoned Dr. Baker to communicate Alexion’s interest in acquiring Synageva for $195 per share in an unspecified mix of cash and Alexion common stock (“Alexion’s $195 Proposal”), which represented a premium of approximately 97.2% to Synageva’s closing price on February 17, 2015 which was the day prior to the date Alexion provided Alexion’s $175 Proposal, a premium of approximately 80.2% to Synageva’s volume weighted average closing price for the 30 days prior to the date Alexion provided Alexion’s $175 Proposal and a premium of approximately 61.3% to Synageva’s all-time high closing price. Dr. Bell subsequently sent Dr. Baker a letter confirming Alexion’s $195 Proposal, which Dr. Baker distributed to the members of the Synageva board of directors.
On March 18, 2015, the Synageva board of directors held a meeting to discuss Alexion’s $195 Proposal. Representatives of Goldman Sachs, Sullivan & Cromwell and Ropes & Gray attended the meeting. The members of the Synageva board of directors discussed Alexion’s $195 Proposal among themselves and with representatives of Goldman Sachs, Sullivan & Cromwell and Ropes & Gray. The Synageva board of directors also discussed the risks of engaging in a potential transaction of this type at this time, including the fact that Synageva was in a critical juncture in the regulatory and launch process with respect to its lead product candidate and that senior management would be required to divert their attention from that process in order to engage in pursuing a transaction with Alexion. The Synageva board of directors also discussed Synageva’s standalone prospects and discussed that they were confident in the ability of senior management to continue to operate Synageva as an independent company. Following the discussion, it was the consensus of the Synageva board of directors that Dr. Baker should communicate to Dr. Bell that the proposed price contained in Alexion’s $195 Proposal did not appear compelling in light of what they believed Synageva’s standalone value to be and that, accordingly, Synageva was not interested in discussing a potential transaction with Alexion on the terms outlined in Alexion’s $195 Proposal. After the conclusion of the meeting Dr. Baker so informed Dr. Bell. Dr. Bell undertook to discuss the matter again with the Alexion board of directors.
On March 24, 2015, Dr. Bell telephoned Dr. Baker to communicate Alexion’s interest in acquiring Synageva for $212 per share in an unspecified mix of cash and Alexion common stock (“Alexion’s $212 Proposal”), which represented a premium of approximately 114.4% to Synageva’s closing price on February 17, 2015, which was the day prior to the date Alexion provided Alexion’s $175 Proposal, a
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premium of approximately 95.9% to Synageva’s volume weighted average closing price for the 30 days prior to the date Alexion provided Alexion’s $175 Proposal and a premium of approximately 75.3% to Synageva’s all-time high closing price. During the conversation, Dr. Bell stated that in order for Alexion to improve its proposal further, Alexion would need to receive and be satisfied with additional information concerning Synageva. Dr. Bell subsequently sent Dr. Baker a letter confirming Alexion’s $212 Proposal, which Dr. Baker distributed to the members of the Synageva board of directors.
On March 25, 2015, the Synageva board of directors held a meeting to discuss Alexion’s $212 Proposal. Representatives of Goldman Sachs, Sullivan & Cromwell and Ropes & Gray attended the meeting. The members of the Synageva board of directors discussed Alexion’s $212 Proposal among themselves and with representatives of Goldman Sachs, Sullivan & Cromwell and Ropes & Gray. Following the discussion, it was the consensus of the Synageva board of directors that Dr. Baker should communicate to Dr. Bell that the proposed price contained in Alexion’s $212 Proposal did not appear compelling in light of what they believed Synageva’s standalone value to be and that, accordingly, Synageva was not interested in engaging in a potential transaction with Alexion on the terms outlined in Alexion’s $212 Proposal. While the Synageva board of directors considered the risks of permitting Alexion to have access to additional information concerning Synageva and the diversion of senior management’s time and attention in providing such information, it determined to authorize Synageva’s senior management to provide certain limited information concerning Synageva to Alexion so that Alexion could determine whether it could improve its offer. On March 26, 2015, Dr. Baker so informed Dr. Bell.
On March 30, 2015, Synageva made available to Alexion an online datasite containing certain information concerning Synageva and its product candidates. From March 30 through May 1, 2015, Alexion and its representatives engaged in due diligence review of Synageva.
Also on March 30, 2015, Mr. Patel and other members of Synageva’s senior management met with Mr. Hallal and other members of Alexion’s senior management. At this meeting, Synageva provided to Alexion certain information concerning Synageva and its product candidates. In telephone conversations on March 31, 2015 and April 1, 2015, Dr. Bell provided feedback from the diligence review and Dr. Baker requested that Alexion provide any revisions to Alexion’s $212 Proposal early in the week of April 6, 2015.
On April 2, 2015, the Synageva board of directors held a meeting to discuss the status of discussions between Synageva and Alexion. Representatives of Ropes & Gray attended the meeting. The members of the Synageva board of directors discussed the status of discussions among themselves, and discussed the advisability of causing Synageva’s senior management to continue its engagement with Alexion during a critical juncture in Synageva’s regulatory and launch process for Synageva’s lead product candidate.
In a telephone conversation on April 8, 2015, Dr. Bell requested additional time to formulate Alexion’s communication to the Synageva board of directors, and Dr. Baker requested that Alexion provide its communication by the end of that week.
On April 9, 2015, the Synageva board of directors held a meeting to discuss the status of communications between Synageva and Alexion. Representatives of Goldman Sachs, Sullivan & Cromwell and Ropes & Gray attended the meeting. Dr. Baker and Mr. Patel updated the Synageva board of directors on the status of the discussions and thereafter members of the Synageva board of directors discussed the potential transaction with Alexion among themselves and with representatives of Goldman Sachs, Sullivan & Cromwell and Ropes & Gray.
On April 12, 2015, Dr. Bell telephoned Dr. Baker to reiterate Alexion’s interest in acquiring Synageva for $212 per share, to be paid 60% in cash and 40% in the form of Alexion common stock (“Alexion’s Reconfirmed $212 Proposal”). Dr. Bell subsequently sent Dr. Baker a letter containing Alexion’s Reconfirmed $212 Proposal, which Dr. Baker distributed to the members of the Synageva board of directors. In the letter, Alexion invited Dr. Baker to serve on the Alexion board of directors following completion of the transaction between the parties.
On April 13, 2015, the Synageva board of directors held a meeting to discuss Alexion’s Reconfirmed $212 Proposal. Representatives of Goldman Sachs, Sullivan & Cromwell and Ropes & Gray attended the meeting. The members of the Synageva board of directors discussed Alexion’s Reconfirmed $212 Proposal among themselves and with representatives of Goldman Sachs, Sullivan & Cromwell and Ropes & Gray.
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Following this discussion, it was the consensus of the Synageva board of directors that Dr. Baker should communicate to Dr. Bell that the proposed price contained in Alexion’s Reconfirmed $212 Proposal did not appear compelling in light of what they believed Synageva’s standalone value to be and that, accordingly, Synageva was not interested in engaging in a potential transaction with Alexion on the terms outlined in Alexion’s Reconfirmed $212 Proposal. Later that day, Dr. Baker so informed Dr. Bell.
On April 16, 2015, Dr. Bell telephoned Dr. Baker to communicate Alexion’s interest in acquiring Synageva for $230 per share, with the transaction consideration to be comprised of 50% cash and 50% Alexion common stock (“Alexion’s $230 Proposal”), which represented a premium of approximately 132.6% to Synageva’s closing price on February 17, 2015, which was the day prior to the date Alexion provided Alexion’s $175 Proposal, a premium of approximately 112.5% to Synageva’s volume weighted average closing price for the 30 days prior to the date Alexion provided Alexion’s $175 Proposal and a premium of approximately 90.2% to Synageva’s all-time high closing price. Dr. Bell stated that this would be Alexion’s final offer and that Alexion would not bid higher. Dr. Bell stated that in order to proceed with Alexion’s $230 Proposal, Alexion would need to receive and be satisfied with additional information concerning Synageva. Dr. Bell subsequently sent Dr. Baker a letter confirming that Alexion’s $230 Proposal was Alexion’s best and final offer, which Dr. Baker distributed to the members of the Synageva board of directors.
On April 17, 2015, the Synageva board of directors held a meeting to discuss Alexion’s $230 Proposal. The meeting was also attended by representatives of Goldman Sachs, Sullivan & Cromwell and Ropes & Gray. The members of the Synageva board of directors discussed Alexion’s $230 Proposal among themselves and with representatives of Goldman Sachs, Sullivan & Cromwell and Ropes & Gray. While not coming to a conclusion on whether to do so, the members of the Synageva board of directors discussed, in light of the discussions with Alexion, whether Synageva should solicit the interest of other potentially interested counterparties in a potential transaction with Synageva and the risks attendant to doing so, including the increased potential for leaks of information and the associated risks, the lack of senior management bandwidth during a critical juncture in Synageva’s regulatory and launch process with respect to its lead product candidate, and the incremental diversion of employee time and attention associated with interacting with multiple potential counterparties. Representatives of Goldman Sachs discussed with the Synageva board of directors the paucity of potentially interested and capable counterparties based on the criteria of whether such parties had both the capacity to compete with the terms proposed by Alexion and the demonstrated interest in Synageva’s area of interest. Following this discussion, it was the consensus of the Synageva board of directors that Dr. Baker should communicate to Dr. Bell that Synageva was willing to explore a transaction with Alexion on the terms contained in Alexion’s $230 Proposal, that Synageva believed that its large stockholders would support such a transaction, that Synageva was willing to provide to Alexion certain additional information concerning Synageva but also that Synageva and its advisors would require access to certain information regarding Alexion in order to determine whether the significant stock component contained in Alexion’s $230 Proposal was acceptable. On April 18, 2015, Dr. Baker so informed Dr. Bell.
On April 19, 2015 representatives of Wachtell Lipton provided to representatives of Sullivan & Cromwell a draft transaction agreement providing for a two-step process in which Alexion would conduct an exchange offer to be followed by a short-form merger, together with a draft support agreement to be entered into by certain stockholders specified by Alexion.
From April 20 through May 1, the parties, primarily through their external legal counsel (Sullivan & Cromwell for Synageva and Wachtell Lipton for Alexion) negotiated the transaction documentation and the significant transaction terms. The issues resolved between the parties during this period included issues relating to certainty of consummation of the transaction and the definition of  “Material Adverse Effect” to be used in the transaction agreement; the Synageva board’s flexibility to change its recommendation of the transaction; and the ability of the Synageva board of directors to terminate the transaction agreement in order to accept a superior proposal from a third party. The parties also agreed on a structure for the transaction whereby the exchange offer to be followed by a short-form merger (if the offer conditions were satisfied) was to be pursued, with an option for Alexion to elect to terminate the exchange offer if any of the offer conditions had not been met after July 12, 2015 and proceed alternatively by having Synageva call a meeting of its stockholders to vote to approve the transaction agreement.
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During the week of April 20, 2015, representatives of Wachtell Lipton furnished to representatives of Sullivan & Cromwell a draft confidentiality agreement incorporating a two-year standstill provision pursuant to which Synageva would be prohibited from taking certain actions with respect to Alexion for such period. Representatives of Sullivan & Cromwell and Wachtell Lipton negotiated this confidentiality agreement, which was entered into by Synageva and Alexion as of April 22, 2015. On that date, Alexion made available to Synageva certain information concerning Alexion.
On April 24, 2015, Mr. Hallal and other representatives of Alexion and met in person with senior management and other representatives of Synageva during which Alexion provided to Synageva certain information concerning Alexion’s business, financial position, products and product candidates.
On April 26, 2015, the Synageva board of directors held a meeting to discuss the status of the negotiations and of the parties’ respective due diligence investigations. The meeting was also attended by representatives of Goldman Sachs, Sullivan & Cromwell and Ropes & Gray. The members of the Synageva board of directors discussed the potential transaction with Alexion among themselves and with representatives of Goldman Sachs, Sullivan & Cromwell and Ropes & Gray. In connection with this discussion, the Synageva board of directors considered whether Synageva should solicit interest from other potential counterparties regarding a potential transaction with Synageva. Members of the Synageva board of directors and representatives of Goldman Sachs discussed the paucity of potentially interested counterparties based on the criteria of whether that such parties had both the capacity to compete with the terms proposed by Alexion and the demonstrated interest in Synageva’s area of interest. Following this discussion, and based on the economic terms of Alexion’s $230 Proposal, the risks associated with contacting other potentially interested parties, including the increased potential for leaks of information and the associated risks, the appropriate allocation of senior management bandwidth during a critical juncture in Synageva’s regulatory and launch process for Synageva’s lead product candidate, the incremental diversion of employee time and attention associated with interacting with multiple potential counterparties, the Synageva board of directors’ and Goldman Sachs’ views concerning the likelihood that contacting additional parties would generate proposals with values exceeding Alexion’s $230 Proposal and the Synageva board of directors’ intention to ensure that any transaction agreement with Alexion would permit the Synageva board of directors to terminate the transaction agreement in order to accept a superior proposal from a third party, the Synageva board of directors determined not to direct Synageva management to contact other potentially interested parties. Members of the Synageva board of directors also discussed the importance of determining expeditiously whether a mutually agreeable agreement with respect to a transaction could be reached with Alexion, and instructed Dr. Baker to inform Dr. Bell that Synageva wanted to reach this determination by May 4, 2015. On April 26, 2015, Dr. Baker so informed Dr. Bell.
On April 29, 2015, the Synageva board of directors held a meeting to discuss the status of the negotiations and the provisions of the draft transaction agreement. The meeting was also attended by representatives of Goldman Sachs, Sullivan & Cromwell and Ropes & Gray. Among other things, the Synageva board of directors emphasized to its negotiating representatives the importance of maintaining flexibility for the Synageva board of directors to change its recommendation of the transaction under appropriate circumstances.
On May 2, 2015, Dr. Bell and Dr. Baker spoke by telephone, and Dr. Bell stated, among other things, that Alexion expected its due diligence investigation to require several more days, and potentially up to one additional week. Dr. Baker stated that he believed the Synageva board of directors would not be willing to delay signing and announcement of the transaction for any additional period, but that if Alexion were willing to move forward without delay, Synageva would be willing to consider including in the transaction agreement certain post-signing provisions concerning certain regulatory and other matters relating to Synageva’s product candidates. Later on May 2, 2015, the Synageva board of directors held a meeting to discuss the status of the negotiations. The meeting was also attended by representatives of Goldman Sachs, Sullivan & Cromwell and Ropes & Gray. The Synageva board of directors discussed the status of the negotiations, and directed Dr. Baker to terminate discussions with Alexion unless Alexion committed to moving forward on a more expeditious timeline. Following this meeting, Dr. Bell and Dr. Baker spoke by telephone, and agreed to terminate discussions between the parties.
On May 4, 2015, Dr. Bell telephoned Dr. Baker and stated that he had discussed matters with the Alexion board of directors, including Synageva’s willingness to consider certain post-signing provisions
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with regard to certain regulatory and lead product matters, and that Alexion would be willing to move forward to negotiate and finalize documentation for the potential transaction in time for a public announcement on May 6, 2015. At Dr. Baker’s request, Dr. Bell confirmed to Dr. Baker that it was a requirement for Alexion that Dr. Baker agree to be appointed to the Alexion board of directors following consummation of the transaction between the parties.
On May 4, 2015, the Synageva board of directors held a meeting to discuss the communication from Dr. Bell. Following discussion among the members of the Synageva board of directors, the Synageva board of directors directed Dr. Baker and Synageva’s management and advisors to resume discussions with Alexion.
Late on May 4, 2015, representatives of Wachtell Lipton provided representatives of Sullivan & Cromwell with a revised draft of the transaction agreement. Representatives of Sullivan & Cromwell and Wachtell Lipton negotiated the transaction agreement over the night of May 4 and the morning of May 5, 2015. On the morning of May 5, 2015, Dr. Bell and Dr. Baker spoke by telephone to discuss the few remaining open negotiation points.
In the late afternoon on May 5, 2015, the Synageva board of directors held a meeting to discuss the status of the negotiations. The meeting was also attended by representatives of Goldman Sachs, Sullivan & Cromwell and Ropes & Gray. In this meeting, Synageva management updated the Synageva board of directors concerning Synageva’s due diligence investigation concerning Alexion, and representatives of Goldman Sachs provided the Synageva board of directors with a financial review of Alexion.
In the evening of May 5, 2015, representatives of Sullivan & Cromwell and Wachtell Lipton continued to negotiate the transaction agreement.
Later in the evening on May 5, 2015, the Synageva board of directors again held a meeting. The meeting was also attended by representatives of Goldman Sachs, Sullivan & Cromwell and Ropes & Gray. Representatives of Sullivan & Cromwell reviewed the terms of the proposed transaction agreement with the members of the Synageva board of directors and a representative of Sullivan & Cromwell described the directors’ fiduciary duties. Representatives of Goldman Sachs presented Goldman Sachs’s financial analysis of the proposed transaction consideration and rendered to the Synageva board of directors its oral opinion, subsequently confirmed in writing, that as of May 5, 2015, and based upon and subject to the assumptions made, procedures followed, matters considered and limitations on the scope of the review undertaken by Goldman Sachs as set forth in such written opinion, the consideration to be paid to the holders of shares of Synageva common stock pursuant to the transaction agreement was fair from a financial point of view to such holders, as in “— Opinion of Synageva’s Financial Advisor.” Such opinion is attached to this document as Annex D.
Following consideration of the transaction agreement and the transactions contemplated by the transaction agreement, the Synageva board of directors unanimously (i) approved and declared advisable the transaction agreement, the offer, the mergers and the other transactions contemplated by the transaction agreement; (ii) determined that the terms of the transaction agreement, the offer, the mergers and the other transactions contemplated by the transaction agreement are fair to and in the best interests of Synageva and to holders of Synageva common stock; (iii) authorized and approved the transaction agreement, the offer, the mergers and the other transactions contemplated by the transaction agreement; and (iv) recommended that the holders of Synageva commons stock accept the offer, tender their shares of Synageva common stock into the offer and, if a vote of Synageva stockholders is required by applicable law to consummate the first merger, adopt the transaction agreement at a meeting of holders of Synageva common stock duly called and held for such purpose. During the meeting of the Synageva board of directors, the compensation committee of the Synageva board of directors reviewed the terms of, and approved, certain Synageva employment compensation, severance and other employee benefit arrangements with respect to the employees of Synageva.
After the closing of trading on Nasdaq on May 5, 2015, Synageva, Alexion, the Offeror and Merger Sub executed the transaction agreement, Alexion and certain stockholders of Synageva executed the voting and support agreements, and on May 6, 2015, Synageva and Alexion issued a joint press release announcing the execution of the transaction agreement, the voting and support agreements and the forthcoming commencement of the exchange offer.
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On May 22, 2015, the Offeror commenced the offer.
On [•] [•], 2015, the Offeror terminated the exchange offer in order to seek to instead effect the transactions through a long-form merger subject to a stockholder vote.
Alexion’s Reasons for the Transactions
Alexion’s board of directors unanimously approved the transaction agreement and determined that the transaction agreement and the transactions contemplated by the transaction agreement, including the mergers and the issuance of Alexion common stock as part of the transaction consideration, are fair to, and in the best interests of, Alexion and its stockholders.
In reaching its determination, Alexion’s board of directors consulted with Alexion’s management, as well as with Alexion’s legal and financial advisors, and considered a variety of factors weighing favorably towards the transactions, including the factors described below.

Expected Benefits of the Transaction.   The Alexion board of directors believes that the transactions will allow Alexion to realize a number of significant benefits, including the following:

Expanded Metabolic Franchise.   The acquisition of Synageva is expected to expand Alexion’s premier metabolic rare disease franchise with the addition of Kanuma, an investigational therapy expected to obtain marketing approval and be launched in 2015 for the treatment of LAL Deficiency, a rare and potentially life-threatening metabolic condition with no available treatment to-date. Alexion believes that Kanuma will complement its existing portfolio, which includes Strensiq, an investigational therapy also awaiting marketing approval and expected to be launched in 2015, for the treatment of HPP, another rare and potentially life-threatening metabolic disease. If both Strensiq and Kanuma obtain marketing approval in 2015 as expected, Alexion could market both products using a single metabolic sales force. If both products are approved in 2015, together with Soliris, a therapy developed by Alexion and approved in the U.S., the European Union and Japan as the first and only treatment for patients with PNH and aHUS, also potentially life-threatening and rare disorders, Alexion would be marketing and selling three highly innovative therapies serving patients with four devastating and rare diseases. In addition, the acquisition is expected to create a metabolic rare disease franchise pipeline consisting of two candidates in clinical trials and a further 14 in pre-clinical development. Alexion believes that the transactions will increase the ability to serve more patients, more quickly, with Kanuma, by utilizing Alexion’s 50-country operating platform.

Strategic and Operational Fit.   Because both companies share an exclusive focus on developing and commercializing life-transforming therapies for patients suffering from devastating and rare diseases, Alexion believes that the acquisition will strengthen its position as a global industry leader by taking advantage of what Alexion already knows well and does well. Alexion anticipates that it will be able to apply its medical, regulatory, clinical and commercial know-how, and utilize its OneSource treatment support program and 50-country operating platform, to maximize the opportunities to serve patients using Synageva’s drug portfolio. In particular, Alexion believes that it can leverage its experience and proven expertise in developing and commercializing Soliris to facilitate the regulatory application process of Kanuma and, subject to receipt of marketing approvals, effectively identify and serve patients with LAL Deficiency.

Robust Rare Disease Pipeline.   The Alexion board of directors expects that the combination will create the most robust rare disease pipeline in the biotech or pharmaceutical industry, notably adding Synageva’s SBC-103 for MPS IIIB to Alexion’s clinical development programs and growing portfolio of highly innovative product candidates. As a result, the combined company would have eight product candidates in clinical trials for 11 indications, including two products under review for registration, and more than 30 pre-clinical programs across a range of therapeutic modalities, including 12 from Synageva’s novel drug discovery platform. Alexion anticipates that at least four pre-clinical candidates from the combined pipelines would enter the clinic by year-end 2016.
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Greater Manufacturing Capabilities.   It is also expected that the transactions would result in expanded manufacturing capabilities, as Synageva would bring to Alexion three upstream facilities and a proprietary manufacturing technology, that can be used to produce proteins with human-like glycosylation patterns.

Shared Core Values and Culture.   The Alexion board also viewed favorably the similarities of Synageva’s and Alexion’s core values, with both companies nurturing a patient-centric culture focused on improving the lives of individuals with devastating diseases that do not have effective treatment options.

Financial Benefits.   Alexion expects to achieve annual cost synergies beginning in 2015 in connection with the transactions, and expects to achieve aggregate synergies of approximately $150 million by 2017. In addition, the transactions are anticipated to accelerate and diversify Alexion’s revenues beginning in 2015 and to be accretive to non-GAAP earnings per share in 2018.

Market Conditions and Diligence.   Alexion’s board also took into account current financial market conditions and the current and historical market prices and volatility of, and trading information with respect to, shares of Synageva and Alexion common stock. The board of directors further considered its familiarity with the business operations, strategy, earnings and prospects of each of Alexion and Synageva and the scope and results of the due diligence investigation conducted by Alexion’s management and advisors with respect to Synageva.

Financial Terms of the Transaction.   Alexion’s board of directors reviewed the amount and form of consideration to be paid in the transaction, the fact that the exchange ratio is fixed, the expected pro forma ownership of the combined company and other financial terms of the transactions.