Alexion Pharmaceuticals
Annual Report 2013

Plain-text annual report

Breakthrough Medical Innovation for Patients with Severe and Rare Disorders 2013 Annual Report name: Ar-Alxn-13-Cv-140325-1800 modified: March 25, 2014 6:34 PM “For nine months, Justice was a growing and healthy boy with overflowing amounts of energy. But then one day I went to pick him up and he was limp … the days that followed in the hospital were intense, and we were so afraid for our son. I vividly recall the doctor sitting us down and telling us Justice has atypical hemolytic uremic syndrome. We were overwhelmed and panicked. But then we learned there is a treatment called Soliris, which gave us hope.” — Danielle W., mother of a child with aHUS receiving Soliris name: Ar-Alxn-13-Cv-140325-1800 modified: March 25, 2014 6:34 PM 2013 Accomplishments April Alexion broadens and strengthens its executive leadership team with the addition of Martin Mackay, PhD, as Executive Vice President, Global Head of R&D, and of Saqib Islam, JD, as Senior Vice President, Chief Strategy and Portfolio Officer; Company Co-founder Stephen Squinto, PhD, assumes the newly created position of Executive Vice President, Chief Global Operations Officer May The U.S. Food and Drug Administration (FDA) grants Breakthrough Therapy designation to asfotase alfa for the treatment of perinatal-, infantile- and juvenile-onset hypophosphatasia (HPP) June The New England Journal of Medicine publishes data from two pivotal studies demonstrating that chronic Soliris® (eculizumab) therapy significantly improved clinical outcomes in patients with aHUS Alexion breaks ground on its new global headquarters in New Haven, Connecticut, to support the company’s continued growth The U.S. FDA grants orphan drug designation to eculizumab (Soliris) for the treatment of neuromyelitis optica (NMO) Alexion commences dosing in a study of the company’s cyclic pyranopterin monophosphate (cPMP) replacement therapy in healthy volunteers; cPMP is an investigational therapy for patients with molybdenum cofactor deficiency (MoCD) type A, a severe, ultra-rare and genetic metabolic disorder that causes catastrophic neurologic damage in newborns in the first weeks of life July The Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) issues a positive opinion for orphan medicinal product designation for eculizumab for the treatment of NMO Alexion establishes global supply chain and quality operations in Ireland Alexion commences a multi-dose Phase 1 study of ALXN1007, the company’s novel anti-inflammatory antibody, in healthy volunteers August Alexion initiates a natural history study of its cPMP replacement therapy in patients with MoCD type A September The Ministry of Health, Labour and Welfare (MHLW) in Japan approves Soliris for the treatment of all patients with aHUS Investigators present preliminary data from a single-arm Phase 2 study demonstrating the potential efficacy of eculizumab in preventing early acute antibody-mediated rejection (AMR) in sensitized deceased-donor kidney transplant recipients at the European Society for Organ Transplantation (ESOT) congress Forbes ranks Alexion #2 on its annual list of “The World’s Most Innovative Companies” for the second year in a row Investigators present new data from an ongoing multinational Phase 2 study in which asfotase alfa was associated with early and continued improvement in skeletal mineralization in infants and young children with HPP at the Joint Meeting of Paediatric Endocrinology congress October The U.S. FDA grants Breakthrough Therapy designation to Alexion’s cPMP replacement therapy for the treatment of MoCD type A Alexion expands enrollment in its study evaluating eculizumab in preventing AMR in deceased-donor kidney transplant recipients November Researchers present new data from the largest prospective trial of adult patients with aHUS and the first prospective trial in pediatric patients with aHUS at the American Society of Nephrology (ASN) meeting The ASN meeting also features the presentation of three-year update data from two pivotal Phase 2 extension studies that highlight the long-term benefits of Soliris therapy in patients with aHUS December Researchers present data from multiple studies at the American Society of Hematology (ASH) meeting that enhance understanding of aHUS and paroxysmal nocturnal hemoglobinuria (PNH) to provide optimal care for patients, including new biomarker data that support the need for chronic terminal complement blockade with Soliris in patients with aHUS Early 2014 Alexion announces a significant expansion of its drug discovery capabilities with a broad and long-term strategic agreement with Moderna Therapeutics for Alexion to develop messenger RNA (mRNA) Therapeutics™ for rare diseases The U.S. FDA grants orphan drug designation to eculizumab for prevention of delayed graft function (DGF) in renal transplant patients and, separately, the European Commission grants orphan drug designation to eculizumab for the prevention of DGF after solid organ transplantation Alexion begins screening patients with two different severe neurologic disorders, NMO and myasthenia gravis (MG), for enrollment in separate single multinational registration studies with eculizumab. Additionally, Alexion plans to initiate enrollment in a single registration study with eculizumab in patients with DGF Forward-looking statements: This Annual Report contains forward-looking statements, all of which involve certain assumptions, risks and uncertainties that are beyond Alexion’s control and could cause our actual results to differ materially from the statements described. Forward-looking statements involve significant risks and uncertainties, including those more fully described in our Form 10-K contained within this Annual Report and in the most recent periodic reports on Form 10-Q filed by Alexion with the U.S. Securities and Exchange Commission, and actual results may vary materially. Alexion does not undertake any duty to update any forward-looking statements contained in this Annual Report as a result of new information, future events or otherwise. name: Ar-Alxn-13-Ed-140326-1500 modified: April 1, 2014 1:31 PM Alexion’s consistent strong focus is on developing therapeutic candidates with life-transforming potential for patients with severe and rare disorders. 2 Alexion Pharmaceuticals 2013 Annual Report name: Ar-Alxn-13-Ed-140326-1500 modified: April 1, 2014 1:31 PM To Our Shareholders: 2013 was another year of significant accomplishments for we serve patients with this devastating genetic disease. Alexion in our mission to develop and deliver breakthrough Throughout 2013, our disease awareness and diagnostic medical innovation for patients suffering from severe and programs continued to support optimal patient care — even life-threatening rare disorders. During the year, we: in countries where we have operated the longest — as • Continued to serve new patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) worldwide with Soliris® (eculizumab). physicians identified new patients with PNH and then rapidly initiated Soliris therapy. While we continued our steady performance in our core territories of the U.S., Western Europe and Japan, we increasingly saw important growth in Turkey, Brazil and Russia, and continued with • Continued to optimize our global organization to early initiatives to serve new patients in Korea and various support the further expansion of our commercial and countries in Latin America. In 2014, our plans are driven clinical operations, including the establishment of by the urgency of delivering Soliris to patients with PNH global supply chain operations in Ireland. in both established and newer markets, as most patients • Achieved a series of significant milestones as we progressed our R&D programs toward as many as seven potential approvals, between 2014 and 2018, for new, life-transforming products or indications for patients with severe and devastating diseases — starting with our second product, asfotase alfa, as a treatment for patients with hypophosphatasia (HPP). Serving More Patients with PNH and aHUS with PNH have yet to receive an accurate diagnosis, let alone begin appropriate therapy. aHUS: Strong Performance in the Initial Global Launch In aHUS, we were pleased with our strong progress in 2013 as we move forward with our global launch in the second indication for Soliris. Our performance in 2013 was augmented by initial funding approvals in Western Europe, and our aHUS disease education and Soliris, our breakthrough medical innovation in terminal diagnostic initiatives resulted in a steady increase in complement inhibition, is the first and only treatment the number of new patients in the U.S. and Western approved for patients with PNH and aHUS, two severe Europe commencing Soliris therapy during the year. and life-threatening, ultra-rare disorders for which Near year-end, we were pleased by the aHUS regulatory Soliris is a life-transforming therapy. approval and initial commercial launch in Japan. PNH: Continued Steady Uptake Worldwide In 2013 — more than six years following PNH approval in We remain committed to bringing Soliris therapy to more patients who are living with PNH and aHUS in the initial countries — we observed the continued steady both new and existing markets worldwide. uptake of Soliris across the nearly 50 countries in which Breakthrough Medical Innovation for Patients with Severe and Rare Disorders 3 name: Ar-Alxn-13-Ed-140326-1500 modified: April 1, 2014 1:31 PM “My name is Margarita and I have PNH. When I was diagnosed with PNH 23 years ago, I was experiencing extreme fatigue and debilitating pain. I was terrified that I would not be able to work or even take care of my kids. Fortunately, I was able to enroll in a clinical trial because at the time there were no treatments available. Today I am still taking Soliris, and 2013 was a great year — I got married and proudly watched my son graduate from high school. I am grateful that I am able to build many happy memories with my family.” — Margarita S., patient with PNH receiving Soliris 4 Alexion Pharmaceuticals 2013 Annual Report name: Ar-Alxn-13-Ed-140326-1500 modified: April 1, 2014 1:31 PM Working Toward Future Breakthroughs At the 2013 Annual Congress of the European Society and Product Launches for Organ Transplantation, researchers presented In 2013, we made significant progress across all of preliminary interim data from an open-label, single-arm, our lead development programs. As we advance our multicenter Phase 2 trial of eculizumab in sensitized programs and strengthen our early-stage research, deceased-donor kidney transplant recipients, in which we remain focused exclusively on delivering life- the rates of AMR and related manifestations, such as transforming therapies to patients with devastating graft loss, were substantially lower than expected. In and rare disorders that do not have effective treatment AMR, we also significantly progressed with enrollment options. In these disease settings, we seek to move of at-risk patients in our living-donor study during the forward only if we can provide transformative clinical year. Additionally, we continue to enroll patients in our benefits, as we are doing in PNH and aHUS. expanded deceased-donor AMR study. Eculizumab: Growing Our Portfolio with New Indications As we build on the strong, long-term safety and efficacy profile of Soliris in PNH and aHUS, we are investigating eculizumab in other severe and rare complement- mediated disorders. Two of these programs are in the kidney transplant setting: antibody-mediated rejection (AMR) and delayed graft function (DGF). Acute AMR can lead to severe damage to the transplanted kidney, resulting in rapid loss of function and possible loss of the organ, which makes the risk of AMR a significant clinical barrier to transplantation in many patients. Research suggests that uncontrolled activation of complement, triggered by the binding of donor-specific antibodies DGF is an early and serious complication of organ transplantation that is characterized by the failure of a transplanted organ to function normally immediately following transplantation. In patients undergoing a kidney transplant, DGF leads to the patient requiring dialysis in order to survive. There is currently no approved therapy to prevent DGF in kidney transplant recipients. In addition, a significant number of donor kidneys are reportedly discarded each year due to the risk of DGF and its associated poor clinical outcomes. Following positive discussions with regulatory authorities in 2013, we are preparing to conduct a single registration trial in DGF, and in January 2014, the FDA granted an orphan drug designation to eculizumab for prevention of DGF. (DSAs) to the target proteins (antigens) of the donor Following meetings with regulators in 2013, we now kidney, may be the primary reason for acute AMR in expect to conduct single registration studies in our lead kidney transplant recipients who are sensitized, or have neurology programs with eculizumab in two settings: DSAs, to their donors. Prophylaxis with eculizumab is neuromyelitis optica (NMO) and myasthenia gravis (MG). a potential strategy to prevent acute AMR, for which In early 2014, we began screening patients for each of there is no approved treatment. these double-blind, placebo-controlled studies. Breakthrough Medical Innovation for Patients with Severe and Rare Disorders 5 name: Ar-Alxn-13-Ed-140326-1500 modified: April 1, 2014 1:31 PM “Willem would literally cry for two to three hours a night, screaming that his hands and feet hurt. I felt completely helpless because there was nothing I could do to alleviate the pain, and I wondered what was around the corner for us if it was already this bad. Hearing the diagnosis of hypophosphatasia was devastating because at first we were told that the doctors could only try to manage his symptoms. Willem is now participating in a clinical trial, and we are grateful that progress is being made to treat this devastating condition.” — Linda T., mother of a child with HPP 6 Alexion Pharmaceuticals 2013 Annual Report name: Ar-Alxn-13-Ed-140326-1500 modified: April 1, 2014 1:31 PM NMO is a life-threatening ultra-rare neurological In mid-2013, asfotase alfa received Breakthrough Therapy disorder in which uncontrolled complement activation designation from the U.S. FDA. The Breakthrough Therapy leads to severe damage to the central nervous system, designation is designed to expedite the development including the spinal cord and optic nerve, and our study of a drug to treat a serious or life-threatening disease is focused on patients who continue to experience when preliminary clinical evidence indicates that the relapses despite supportive treatment. The primary drug may demonstrate substantial improvement over endpoint of this study is time to first relapse. existing therapies on one or more clinically significant MG is a debilitating and potentially life-threatening disorder in which uncontrolled complement activation results in destruction and inflammation at the neuromuscular junction, leaving muscles severely weakened. Our study in MG is focused on patients with severe disease who are refractory to other treatment options. The primary endpoint of the study is change in a measure of physical functioning in patients with MG. Asfotase Alfa: Preparing to Launch Our Next Product These guiding principles are clearly illustrated in our asfotase alfa development program for patients with HPP, a chronic, life-threatening, genetic and ultra-rare metabolic disease that leads to progressive damage to multiple vital organs, destruction and deformity of bones and, in too many cases, premature death. Asfotase alfa is designed to address the underlying cause of HPP, which endpoints. In this case, the U.S. FDA recognized asfotase alfa as a Breakthrough Therapy for patients with HPP whose first signs or symptoms occurred prior to 18 years of age, including patients with perinatal-, infantile- and juvenile-onset forms of the disease. Other Highly Innovative Therapeutic Candidates Beyond eculizumab and asfotase alfa, our research and development programs include additional compounds with breakthrough potential. In the metabolic disease area, we continue to accelerate development of our cyclic pyranopterin monophosphate (cPMP) replacement therapy for the treatment of molybdenum cofactor deficiency (MoCD) type A, a severe and life-threatening, ultra-rare, genetic metabolic disorder that causes catastrophic and irreversible neurologic damage within the first weeks of life. We were pleased to report that in 2013 the U.S. FDA granted Breakthrough Therapy designation to cPMP for this indication. affects people of all ages, by normalizing the genetically ALXN1007 is a novel anti-inflammatory antibody. In 2013 defective metabolic process and preventing or reversing we completed dosing in a single-dose study and initiated the severe and potentially life-threatening complications and completed a multi-dose study in healthy volunteers. of lifelong, dysregulated mineral metabolism. We will begin screening in our first proof-of-concept study with ALXN1007 in early 2014. Breakthrough Medical Innovation for Patients with Severe and Rare Disorders 7 name: Ar-Alxn-13-Ed-140326-1500 modified: April 1, 2014 1:31 PM Research Pipeline Preclinical Clinical Studies Registration Studies Market Soliris® (eculizumab) – Franchise Approved Indications Paroxysmal Nocturnal Hemoglobinuria (PNH) Atypical Hemolytic Uremic Syndrome (aHUS) Investigational Indications – Kidney Transplant, Neurology and Nephrology Antibody-Mediated Rejection (AMR) – Living and Deceased Donor Delayed Graft Function (DGF) Relapsing Neuromyelitis Optica (NMO) Severe and Refractory Myasthenia Gravis (MG) Shiga-toxin E. coli-Related (STEC) HUS Soliris Lifecycle Management “Next-Gens” Investigational Candidates – Metabolic Disorders Hypophosphatasia (HPP) Asfotase Alfa Molybdenum Cofactor Deficiency (MoCD) Type A Cyclic Pyranopterin Monophosphate (cPMP) Replacement Therapy (ALXN1101) Investigational Candidates – Inflammatory Disorders ALXN1007 – Anti-inflammatory Antibody ALXN1102/1103 – Anti-Complement 8 Alexion Pharmaceuticals 2013 Annual Report name: Ar-Alxn-13-Ed-140326-1500 modified: April 1, 2014 1:31 PM Additionally, enrollment continues in our Phase 1 the body’s natural processes to produce targeted clinical trial of ALXN1102/1103, the intravenous and proteins. This has the potential to speed the subcutaneous versions, respectively, of Alexion’s novel development and manufacture of treatments for alternative pathway complement inhibitor, which we many rare diseases that are currently untreatable are investigating for the treatment of severe, ultra- with existing technologies. rare inflammatory disorders. Expanding Early-Stage Research Beyond our current clinical development programs, we initiated a collaboration with Ensemble Therapeutics in July 2013 to discover macrocycle drug candidates targeting patients with severe and ultra-rare disorders. The collaboration will utilize Ensemble’s proprietary drug discovery platforms to address several undisclosed drug targets identified by Alexion with the objective of creating highly innovative small molecule therapeutic Continued Strong Financial Discipline and Performance 2013 was another year of sustained growth and profitability for Alexion as we worked to provide Soliris to an increasing number of patients with PNH and aHUS worldwide. For the full year, we recorded sales of $1.55 billion, an increase of 37% compared to 2012. By maintaining fiscal discipline, we achieved a 47% increase in non-GAAP net income to $624.2 million. Our year-on-year revenue growth was robust across candidates. We will have the exclusive worldwide rights all territories we serve. to develop and commercialize candidates arising from the collaboration. In January 2014 we completed a broad, long- During 2013, we further aligned our global structure and made additional investments needed to improve operational efficiency so that we can serve even more term, exclusive strategic agreement with Moderna patients with PNH and aHUS around the world. Therapeutics for the discovery and development of messenger RNA (mRNA) Therapeutics™ to treat Strengthening Our Organization rare diseases. The agreement marks a significant In 2013 we grew to more than 1,800 employees working expansion of our drug discovery capabilities and in company facilities in 25 countries. We significantly an exciting drug discovery platform for Alexion. We broadened and deepened our executive leadership team believe that the technology is optimally positioned with the hiring of Martin Mackay, PhD, as Executive Vice to address the broadest number of severe and rare President, Global Head of R&D, and of Saqib Islam, JD, disorders, as these conditions are typically caused as Senior Vice President, Chief Strategy and Portfolio by single protein deficiencies, and Moderna’s highly Officer. Also during 2013, Alexion Co-founder Stephen innovative technology is designed to directly utilize Squinto, PhD, assumed the newly created position of Breakthrough Medical Innovation for Patients with Severe and Rare Disorders 9 name: Ar-Alxn-13-Ed-140326-1500 modified: April 1, 2014 1:31 PM Back (from left): David L. Hallal, Executive Vice President and Chief Commercial Officer; Saqib Islam, JD, Senior Vice President and Chief Strategy and Portfolio Officer; Stephen P. Squinto, PhD, Executive Vice President and Chief Global Operations Officer; Clare Carmichael, Senior Vice President and Chief Human Resources Officer. Front (from left): John B. Moriarty, Jr., JD, Senior Vice President and General Counsel; Leonard Bell, MD, Chief Executive Officer; Martin Mackay, PhD, Executive Vice President and Global Head of Research & Development; Vikas Sinha, MBA, CA, CPA, Executive Vice President and Chief Financial Officer. “2013 was a year of strong performance for Alexion across a growing number of commercial and clinical objectives. In 2014, we will build on our recent accomplishments as we aggressively push forward the frontiers of medical innovation for the benefit of patients with severe and rare disorders.” — Leonard Bell, MD, Chief Executive Officer 10 Alexion Pharmaceuticals 2013 Annual Report name: Ar-Alxn-13-Ed-140326-1500 modified: April 1, 2014 1:31 PM Soliris Net Product Sales 18 % 13 % 36 % Executive Vice President, Chief occupancy in 2015, is located in Global Operations Officer, further 33 % the Downtown Crossing section of strengthening our capabilities in the manufacturing, quality and supply chain functions. 2013 $1,551,346 New Haven, and we are excited to take part in the redevelopment of the city’s downtown. During 2013 we began to distribute 14 % Soliris from our newly approved contract manufacturing facility in Singapore. We also reached a key milestone in the expansion of our technical operations by establishing global supply chain operations in Ireland. In January 2014, we agreed to purchase what will become Alexion’s first company-owned vialing facility for Soliris and other clinical and 14 % 37 % 35 % Looking Ahead Our track record of accomplish- m e n t s a c r o s s k e y g r o w t h objectives in 2013 has provided a foundation for continued 2012 $1,134,114 innovation and progress in United States Europe Asia-Pacific (primarily Japan) Other 2014 and beyond. As always, we are grateful to everyone who supports our mission and makes our work possible: our employees, our board members commercial products in Ireland. Finally, we are pleased with the continued improvements in our Rhode Island manufacturing and quality operations and look forward to the U.S. FDA reinspection of this facility in 2014. To enable our continued global expansion, in 2013 we broke ground on our new global headquarters at 100 College Street in New Haven, Connecticut. Alexion will be the anchor tenant in the state-of-the-art laboratory and office building. The ground-breaking is a milestone in the growth of our research and business operations, and our return to New Haven will be a homecoming for Alexion, where the company was first established and shareholders, physicians and other health professionals, healthcare authorities around the world and especially the patients, families and caregivers whose needs we strive to meet. Together we will work tirelessly to attain additional breakthroughs in 2014 and beyond. Leonard Bell, MD Chief Executive Officer in 1992. The building, which is expected to open for April 2014 Breakthrough Medical Innovation for Patients with Severe and Rare Disorders 11 name: Ar-Alxn-13-Ed-140326-1500 modified: April 1, 2014 1:31 PM Selected GAAP Financial Results (In thousands, except per share data) Year Ended December 31, 2013 2012 2011 Consolidated Statements of Operations Data: Net product sales Cost of sales Operating expenses: Research and development Selling, general and administrative Acquisition-related costs Impairment of intangible assets Amortization of purchased intangible assets Total operating expenses Operating income Other expense Income before income taxes Income tax provision Net income Earnings per common share — diluted Shares used in computing earnings per common share — diluted $ 1,551,346 $ 1,134,114 $ 783,431 177,556 72,837 93,140 317,093 489,720 5,029 33,521 417 222,732 384,678 22,812 26,300 417 137,421 308,176 13,486 — 382 $ 845,780 $ 656,939 $ 459,465 528,010 1,741 526,269 273,374 252,895 1.27 199,712 $ $ 404,338 6,772 397,566 142,744 254,822 1.28 198,501 $ $ 230,826 1,158 229,668 54,353 175,315 0.91 191,806 $ $ As of December 31, 2013 2012 2011 Consolidated Balance Sheet Data: Cash, cash equivalents and marketable securities $ 1,514,851 $ 989,501 $ 540,865 Trade accounts receivable, net Inventories Property, plant and equipment, net Goodwill and intangible assets, net Deferred tax assets Other assets Total assets 421,752 102,602 201,109 863,792 44,826 168,764 295,598 94,521 165,629 900,323 40,040 127,948 244,288 81,386 165,852 171,243 123,000 68,117 $ 3,317,696 $ 2,613,560 $ 1,394,751 Accounts payable and accrued expenses $ 423,940 $ 271,275 $ 199,653 Deferred revenue Contingent consideration Long-term debt Deferred tax liabilities Other liabilities Total liabilities Total stockholders’ equity 53,801 142,676 113,000 101,265 100,935 935,617 2,382,079 31,266 141,670 149,000 20,994 28,505 642,710 1,970,850 17,905 18,120 — 862 23,719 260,259 1,134,492 Total liabilities and stockholders’ equity $ 3,317,696 $ 2,613,560 $ 1,394,751 12 Alexion Pharmaceuticals 2013 Annual Report name: Ar-Alxn-13-Ed-140326-1500 modified: April 1, 2014 1:31 PM Shareholder Information Directors Executive Management Leonard Bell, MD Chief Executive Officer Stephen P. Squinto, PhD Executive Vice President, Chief Global Operations Officer David L. Hallal Executive Vice President, Chief Commercial Officer Martin Mackay, PhD Executive Vice President, Global Head of Research & Development Vikas Sinha, MBA, CA, CPA Executive Vice President, Chief Financial Officer Clare Carmichael Senior Vice President, Chief Human Resources Officer Saqib Islam, JD Senior Vice President, Chief Strategy & Portfolio Officer John B. Moriarty, Jr., JD Senior Vice President, General Counsel Max Link, PhD1,4 Chairman of the Board Former Chairman of the Board and CEO, Centerpulse AG Former CEO, Corange Former Chairman of the Board and CEO, Sandoz Pharma, Ltd. Leonard Bell, MD Chief Executive Officer William R. Keller2,3 Vice Chairman of Shanghai Association of Foreign Investment Enterprises Senior Consultant of Shanghai Foreign Investment Development Board Former General Manager, Roche China Ltd. Joseph A. Madri, PhD, MD2,4,5 Professor of Pathology, Yale University School of Medicine Larry L. Mathis1,3,5 Former President and CEO, The Methodist Hospital System R. Douglas Norby1,3 Former Senior Vice President, Chief Financial Officer, Tessera Technologies, Inc. Alvin S. Parven2,3 President, ASP Associates Former Vice President, Aetna Health Plans Andreas Rummelt, PhD1,4 CEO, InterPharmaLink AG Former Group Head, Quality Assurance and Technical Operations, Novartis Former Member of Executive Committee, Novartis Former CEO, Sandoz AG Ann M. Veneman2,3 Former Executive Director of UNICEF Former Secretary of US Department of Agriculture Annual Shareholders Meeting To be held on May 5, 2014 5:00 p.m. The Study at Yale 1157 Chapel Street New Haven, CT 06511 tel 203.503.3900 Other Information Global Corporate Headquarters Alexion Pharmaceuticals, Inc. 352 Knotter Drive Cheshire, CT 06410 tel 203.272.2596 fax 203.271.8190 Transfer Agent and Registrar Computershare Trust Company, N.A. 250 Royall Street Canton, MA 02021 Investor Relations Rx Communications 445 Park Avenue, 10th Floor New York, NY 10022 tel 917.322.2569 fax 917.322.2570 Legal Counsel Ropes & Gray LLP Boston, MA Independent Auditors PricewaterhouseCoopers LLP Boston, MA Trading Symbol Listing for Alexion Pharmaceuticals, Inc. is found on the NASDAQ stock market under the symbol ALXN. alexionpharma.com 1 Member of the Audit Committee 2 Member of the Compensation Committee 3 Member of the Nominating and Corporate Governance Committee 4 Member of the Pharmaceutical Compliance and Quality Committee 5 Retiring May 2014 © 2014 Alexion Pharmaceuticals, Inc. Alexion®, Alexion Logo® and Soliris® are trademarks of Alexion Pharmaceuticals, Inc. name: Ar-Alxn-13-Cv-140325-1800 modified: March 25, 2014 6:34 PM Alexion Pharmaceuticals, Inc. 352 Knotter Drive, Cheshire, CT 06410, USA Alexion Pharma International Sàrl Avenue du Tribunal Fédéral 34, 1005, Lausanne, Switzerland Alexion Pharma G.K. Ebisu Prime Square Tower, Tokyo 150-0012, Japan Alexion Pharmaceuticals Australasia Pty Limited 117 Old Pittwater Road, Brookvale NSW Australia, 2100 Alexion Pharma International Trading Block 10A Beckett Way, Park West Business Park, Nangor Road, Dublin 12 alexionpharma.com name: Ar-Alxn-13-Cv-140325-1800 modified: March 25, 2014 6:34 PM 

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