Alexion Pharmaceuticals Annual Report 2010

Plain-text annual report

Transforming Patients’ Lives Through Innovation A l e x i o n P h a r m a c e u t i c a l s , I n c . 2 0 1 0 A n n u a l R e p o r t 2010 Annual Report 3/30/11 9:24 PM 3/30/11 9:24 PM “Before Soliris I couldn’t walk down the block without getting short of breath. Now I am at the baseball ﬁ elds with my boys and chasing my little girl around the playground. I can’t imagine not having the energy to keep up with my kids.” Ruthie M. Patient with PNH receiving Soliris® since 2007 76856 Cover.indd 2 76856 Cover.indd 2 4/1/11 4:27 PM 4/1/11 4:27 PM 2010 Accomplishments February November Alexion appoints Andreas Rummelt to its Board of Directors Two Phase II studies of eculizumab in adult and adolescent April Soliris® (eculizumab) receives marketing approval in Japan for patients with paroxysmal nocturnal hemoglobinuria (PNH) Alexion completes enrollment in Phase II clinical trials of eculizumab in adult and adolescent patients with atypical hemolytic uremic syndrome (aHUS) May patients with aHUS – one in patients resistant or intolerant to plasma therapy and the second trial in patients receiving chronic plasma therapy – meet primary and secondary endpoints with high statistical and clinical signiﬁ cance; data presented for the ﬁ rst time at the annual American Society of Nephrology (ASN) meeting Investigators present positive data on 24 patients from an investigator-initiated trial with eculizumab in patients at elevated risk for AHR at the ASN meeting Alexion appoints Ann M. Veneman to its Board of Directors December Investigators present positive interim data on 16 patients Australian government makes a landmark decision concluding from an investigator-initiated trial with eculizumab in patients that Soliris treatment substantially extends the lives of at elevated risk for acute humoral rejection (AHR) at the patients with PNH American Transplant Congress June Japan’s Ministry of Health, Labour and Welfare (MHLW) lists Soliris for reimbursement for patients with PNH in Japan Soliris data from International and Asian patient registries, presented at the European Hematology Association (EHA) meeting, show substantial disease burden of PNH Researchers at Innsbruck, Austria conference report early clinical experience that supports further investigation of eculizumab for the treatment of patients with diseases characterized by uncontrolled complement activation that share a common pathology of thrombotic microangiopathy (TMA), including aHUS, MPGN and CAPS July Alexion aligns its commercial operations into distinct Therapeutic Areas in Hematology and Nephrology August Alexion receives FDA approval of its Rhode Island manufacturing facility for Soliris supply, providing a second source for commercial and clinical needs October Alexion commences a Phase II study of eculizumab in pediatric patients with aHUS Alexion earns the Connecticut Green Building Council Green Advocate Award and two LEED Gold Certiﬁ cations for environmental sustainability Multiple presentations by physicians at the annual meeting of the American Society of Hematology (ASH) show a growing body of evidence regarding the long-term use of Soliris as a treatment for patients with PNH: (cid:129) Independent European investigators show that studied Soliris-treated patients had survival rates similar to an age- and gender-matched population of normal individuals (cid:129) A retrospective assessment of 195 PNH clinical trial patients demonstrates that three-year Soliris treatment was associated with a rapid and sustained reduction in hemolysis in all patients (cid:129) Data from the Korean PNH registry underscore that hemolysis is associated with increased mortality and thrombosis Early 2011 Alexion submits marketing applications to the FDA and European Medicines Agency for eculizumab as a treatment for patients with aHUS Alexion acquires Taligen Therapeutics and creates the Alexion Translational Medicine Group to accelerate development of an expanded portfolio including innovative Taligen complement inhibitors for patients with severe and ultra-rare disorders and ophthalmic disorders Alexion acquires investigational cPMP replacement therapy from Orphatec Pharmaceuticals for infants suffering from molybdenum cofactor deﬁ ciency (MoCD) Type A, a catastrophic, ultra-rare genetic neurologic disorder Alexion Pharmaceuticals 1 2010 Annual Report 76856 Color Text.indd ed1 76856 Color Text.indd ed1 4/4/11 3:14 PM 4/4/11 3:14 PM “These ground-breaking results show that eculizumab signiﬁ cantly increased platelets and reduced the life-threatening blood clot process that caused severe damage to the kidney and other organs in these patients with aHUS.” Christophe Legendre, M.D. Professor of Nephrology at Université René Descartes-Hôpital Necker, Paris aHUS Study Investigator and Presenter of Phase II Data at ASN Meeting, November 2010 76856 Color Text.indd ed2 76856 Color Text.indd ed2 3/30/11 10:33 PM 3/30/11 10:33 PM To Our Shareholders: 2010 was a year of remarkable strides in Alexion’s mission have world-leading expertise, such as complement inhibition to develop and deliver innovative therapies to transform the and the development of therapies for patients suffering with lives of patients suffering from severe, ultra-rare and life- ultra-rare disorders. threatening diseases. During the year, we: (cid:129) Expanding our capabilities to move early-stage product (cid:129) Served more patients with paroxysmal nocturnal candidates more rapidly through clinical development with hemoglobinuria (PNH) in existing countries – helping to the establishment of our new Translational Medicine Group increase the awareness and diagnosis of PNH and raising the based in Cambridge, Massachusetts. understanding of the role that Soliris® (eculizumab) can play in transforming the lives of patients with PNH. (cid:129) Expanded our ability to serve patients with PNH, atypical hemolytic uremic syndrome (aHUS) and other ultra-rare and severe diseases by aligning our operations into distinct Therapeutic Areas in Hematology and Nephrology. (cid:129) Pursuing these initiatives with the same ﬁ nancial discipline that has guided us since before the launch of Soliris. Soliris in PNH Since 2007, Soliris, Alexion’s ﬁ rst approved product, has truly transformed the lives of patients and families struggling with (cid:129) Completed two Phase II clinical studies evaluating PNH, a debilitating and life-threatening ultra-rare disease eculizumab in patients with aHUS, demonstrating characterized by hemolysis, the destruction of red blood that eculizumab substantially reduced thrombotic cells. In 2010 – largely through improved education about, microangiopathy (TMA), stabilized or improved kidney and awareness of, PNH – we again made strong progress in function, and enhanced quality of life in study patients. bringing Soliris to more patients in our core territories of the (cid:129) Broadened our pipeline programs to include more than a dozen clinical trials with eculizumab for patients with additional severe and rare disorders. (cid:129) Ensured a reliable supply of product for the treatment of patients through the U.S. and E.U. commissioning of our Rhode Island manufacturing facility as an additional source of supply. (cid:129) Achieved another year of proﬁ table growth to help ensure the Company’s long-term ability to reach its objectives to beneﬁ t more patients with ultra-rare and life-threatening diseases. Today, in early 2011, we are building on these accomplishments by: (cid:129) Accelerating our aHUS development program. Based on the landmark data from our clinical studies of eculizumab, we have now submitted, earlier than expected, marketing applications for aHUS to the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). (cid:129) Broadening our diverse pipeline programs with eculizumab and other promising compounds, including those acquired early in 2011 from Taligen Therapeutics and Orphatec Pharmaceuticals – working at all times in areas in which we U.S., Western Europe and Japan. Our robust initial launch in Japan in the second half of the year was especially important as we strive to meet the needs of more patients. Today, we are further extending our reach by scaling up our efforts in new major territories, focusing on Turkey, Brazil and Russia in 2011 and 2012. These countries are home to a greater aggregate population than the United States, offering a powerful opportunity to serve even more patients with PNH. At the same time, we are continuing to reach new patients in more than a dozen other smaller nations. Deepening the Understanding of PNH Our operations in all countries continue to be supported by a growing body of evidence, which the medical community has been developing for several years, identifying the debilitating and life-threatening nature of PNH and the utility of Soliris in treating patients with the disease. In December 2010, landmark data on the long-term use of Soliris in patients with PNH were presented at the annual meeting of the American Society of Hematology (ASH). While historically, up to 35% of studied patients with PNH have died within ﬁ ve years of diagnosis, independent investigators presented a study in which PNH Alexion Pharmaceuticals 3 2010 Annual Report 76856 Color Text.indd ed3 76856 Color Text.indd ed3 3/30/11 10:33 PM 3/30/11 10:33 PM 8 4 11 2 10 7 9 17 6 1 14 3 12 5 16 13 15 Map Key: Soliris in PNH Approved for Marketing Marketing Application Under Review Available Under Alternative Access Programs Ofﬁ ce Locations 1 Cheshire, CT, USA 7 Lausanne, Switzerland North America Regional and Global Headquarters 2 Barcelona, Spain Country Operations 3 Bogotá, Colombia Country Operations 4 Brussels, Belgium Country Operations 5 Buenos Aires, Argentina Country Operations 6 Cambridge, MA, USA Translational Medicine Group EMEA Regional Headquarters International Operations Center Country Operations 8 London, United Kingdom Country Operations 9 Milan, Italy Country Operations 10 Munich, Germany Country Operations 11 Paris, France European Service Center Country Operations 12 São Paulo, Brazil Country Operations 13 Shanghai, China Commercial Operations 14 Smithﬁ eld, RI, USA Global Manufacturing 15 Sydney, Australia Asia/Paciﬁ c Regional Headquarters Country Operations 16 Tokyo, Japan Country Operations 17 Toronto, Canada Country Operations Alexion Pharmaceuticals 4 2010 Annual Report 76856 Color Text.indd ed4 76856 Color Text.indd ed4 3/31/11 6:47 PM 3/31/11 6:47 PM patients treated with Soliris for up to eight years achieved Unique Expertise in Ultra-Rare Disorders survival comparable to an age- and gender-matched normal population. Researchers further reported ﬁ ndings from a multinational study of patients treated with Soliris for up to 36 months in which all patients demonstrated rapid and sustained In the ﬁ rst half of 2011, we mark the fourth anniversaries of the initial launches of Soliris in the U.S. and the larger European countries. Our learnings during these years, combined with the global infrastructure we have established, reduction in LDH (an indicator of hemolysis), and more than half give us greater conﬁ dence than ever in fulﬁ lling our showed a marked improvement in long-term kidney function. Separately, the Australian government concluded in December that Soliris treatment substantially extends the lives of patients with PNH – a decision that bolsters the view in other countries that patient access to Soliris is vitally important. The growing recognition of the long-term beneﬁ ts of Soliris treatment for patients suffering from PNH among international experts provides a strong underpinning for governments and other payors to facilitate access to Soliris for patients around the world. Optimizing Care Through Heightened Awareness and Diagnosis As hematologists and oncologists gain a better understanding of the severe and progressive nature of PNH and the clinical beneﬁ ts that Soliris can provide to patients, they are increasingly requesting PNH laboratory testing services for patients at higher risk for having PNH. In response to this growing demand, in 2010 the International Clinical Cytometry Society published PNH testing guidelines, a pivotal milestone for physicians and patients, who have historically endured years of missed opportunities for correct diagnoses. The Society’s new guidelines have already inﬂ uenced numerous labs to improve their testing methods to the new global standard. Despite the progress on so many fronts, we know that the majority of patients with PNH worldwide – and even in our core territories – have yet to receive an accurate diagnosis or appropriate treatment. To address this critical need for patients with this progressive disease, we are accelerating the international rollout of our PNH education efforts to facilitate a greater awareness of the signs of PNH so that patients no longer suffer needlessly. We are conﬁ dent that by working closely with the medical community to deepen the understanding of commitment to the objective that every patient with PNH who can beneﬁ t from Soliris will have access to Soliris. At the same time, we have developed a unique set of skills and resources – clinical, regulatory and commercial – that can be applied to the needs of patients suffering with other ultra-rare and severe disorders. These include capabilities in developing innovative novel therapies, designing clinical trials in disease areas where trials have not previously been conducted, recruiting trial patients from very small populations of patients scattered around the world, and working with authorities to provide access to a highly innovative approved therapy once it becomes available. As 2011 proceeds, we are accelerating and expanding our efforts on all these fronts, with a special emphasis on our lead development programs in nephrology. Beyond PNH: Innovation with Eculizumab As the world’s ﬁ rst approved terminal complement inhibitor, eculizumab was a long-sought breakthrough innovation for patients with PNH – and the ﬁ rst signiﬁ cant hope for patients with a number of other ultra-rare, debilitating and often life- threatening complement-mediated diseases. In such disorders, the patient’s healthy tissue is destroyed by complement, a component of the normal immune system – either as a result of excessive complement activation or from a deﬁ ciency of naturally occurring inhibitors of complement. For several years, we have noted the increasing level of interest of independent researchers who have sought to investigate eculizumab as a potential therapy for patients with other severe and ultra- rare conditions in hematology, nephrology, transplant and neurology, as well as for patients with debilitating disorders PNH, we’re positively inﬂ uencing the entire cycle of care – from within ophthalmology. accurate diagnosis to successful treatment. Alexion Pharmaceuticals 5 2010 Annual Report 76856 Color Text.indd ed5 76856 Color Text.indd ed5 3/30/11 10:33 PM 3/30/11 10:33 PM “Our Cambridge-based Translational Medicine Group will accelerate the development of our expanded portfolio of innovative products that aim to transform the lives of patients with severe and untreated disorders.” Stephen P. Squinto, Ph.D. Executive Vice President, Head of Research and Development 76856 Color Text.indd ed6 76856 Color Text.indd ed6 3/31/11 6:47 PM 3/31/11 6:47 PM Accelerated Development Program in aHUS with aHUS, as well as a further adult study, are ongoing in the Our lead nephrology program – eculizumab as a treatment for United States, Europe and Canada. patients with aHUS – is one example of how the dedication of independent investigators can potentially lead to positive outcomes for patients who otherwise lack any effective treatment option. aHUS is an ultra-rare, life-threatening disease in which chronic uncontrolled complement activation causes blood clots in small blood vessels throughout the body (thrombotic microangiopathy or TMA), leading to kidney failure, stroke, heart attack and death. There are no current treatment options that have been shown to be safe or effective for the treatment of patients with aHUS. Indeed, within one year of diagnosis, 60% of patients with aHUS, many of whom are children, will require dialysis, undergo a kidney transplant, or die from the disease. In early 2009, the medical literature began to highlight the ﬁ rst case reports of the successful use of eculizumab to treat individual patients with aHUS. Soon after, Alexion commenced company-sponsored studies in two cohorts of adult and adolescent patients with aHUS: one group who were resistant or intolerant to plasma therapy (PT), and the other group who were receiving PT chronically. In November 2010, investigators reported highly encouraging results from these Phase II studies at the American Society of Nephrology (ASN) conference. These studies met their primary and secondary endpoints with strong statistical and clinical signiﬁ cance: (cid:129) Patients resistant or intolerant to PT demonstrated increased platelet counts and became TMA-free; their kidney function improved, and they were able to stop dialysis and had improved quality of life. (cid:129) Patients receiving chronic PT became free of TMA events and intervention, and experienced stabilized or improved kidney function, and had improved quality of life. These clinically and statistically meaningful results suggested that terminal complement inhibition with Soliris has the potential to change the course of aHUS. Given the strength of the Phase II data, we have accelerated the timeline for development, operational build-out and launch of Soliris in this new indication. A separate study in pediatric patients Kidney Transplant: Acute Humoral Rejection In the second lead program within our emerging nephrology franchise, eculizumab is being investigated as a treatment for patients undergoing kidney transplant who are at elevated risk for acute humoral rejection (AHR). Encouraging data presented by independent investigators in November 2010 showed that eculizumab signiﬁ cantly reduced AHR, post-transplant plasma therapy requirements and splenectomy in a study of 24 transplant patients compared to a historical control population. Enrollment in this study is expected to be completed in 2011. Separately, we expect to initiate two global studies in kidney transplant patients at elevated risk for AHR – one for patients undergoing living-donor transplant and the other for patients undergoing deceased-donor transplant. An additional investigator-initiated study in transplant patients at risk from blood-type (ABO) incompatibilities is ongoing. Eculizumab Programs in Additional Renal, Hematologic and Neurologic Conditions Beyond our lead development programs in aHUS and AHR, eculizumab continues to be evaluated across a spectrum of ultra-rare and severe complement-mediated diseases. Research includes investigator-initiated and Alexion-sponsored studies in Nephrology (dense deposit disease, C3 nephropathy and transplant in catastrophic anti-phospholipid syndrome), Hematology (cold agglutinin disease) and Neurology (neuromyelitis optica and myasthenia gravis). Preliminary data from several of these studies are expected in 2011. Beyond Eculizumab: Expanding Innovation, Accelerating Development As we seek to transform the lives of more patients with severe disorders, we are focused on broadening our portfolio of early- stage product candidates, whether through internal research or by applying our development, clinical and regulatory skills to other highly innovative compounds. Alexion Pharmaceuticals 7 2010 Annual Report 76856 Color Text.indd ed7 76856 Color Text.indd ed7 4/1/11 4:21 PM 4/1/11 4:21 PM Preclinical Phase I Phase II Phase III Regulatory Market Research Pipeline Hematology Paroxysmal Nocturnal Hemoglobinuria (PNH) Cold Agglutinin Disease* ANCA Vasculitis* Inﬂ ammatory Disorders Nephrology atypical Hemolytic Uremic Syndrome (aHUS) – Adults & Adolescents atypical Hemolytic Uremic Syndrome (aHUS) – Pediatrics Presensitized Kidney Transplant (AHR)* MPGN II* / C3 Nephropathy* ABO Incompatible Renal Transplant* Kidney Transplant for CAPS* Neurology Myasthenia Gravis Neuromyelitis Optica* Molybdenum Cofactor Deﬁ ciency Type A Ophthalmology Age-Related Macular Degeneration (AMD) Age-Related Macular Degeneration (AMD) Age-Related Macular Degeneration (AMD) Oncology Chronic Lymphocytic Leukemia Multiple Myeloma Rare Solid Tumor Pipeline Key Soliris® (eculizumab) cPMP Replacement Therapy Samalizumab TT30 TA106 * Investigator-Initiated Trial Alexion Pharmaceuticals 8 2010 Annual Report 76856 Color Text.indd ed8 76856 Color Text.indd ed8 4/1/11 4:24 PM 4/1/11 4:24 PM Novel Approaches to Ultra-Rare and Severe Disorders opportunities arise, as demonstrated by our ophthalmology In early 2011, we announced two strategic acquisitions in line program. An investigator-sponsored Phase II proof-of-concept with our mission to serve patients with ultra-rare and severe study continues to investigate intravenous eculizumab disorders and based on our proven competencies: as a treatment for patients with dry age-related macular (cid:129) In January, we completed the acquisition of Taligen Therapeutics, through which we have gained several highly innovative complement inhibitors with mechanisms of action distinct from Soliris and with potential uses for patients with ultra-rare, systemic disorders, as well as for other patients with severe ophthalmic conditions. Through Taligen, we have also gained new colleagues: a team of talented researchers who form the nucleus of our new Translational Medicine Group based in Cambridge, Massachusetts, enabling us to move drug candidates more rapidly through preclinical stages and into trials. degeneration, or dry AMD, a disease that causes blurred central vision and can lead to vision loss. Importantly, with the acquisition of Taligen, our ophthalmology program has grown to include several additional early-stage drug candidates with the potential for intravitreal use in patients with AMD. By accelerating the investigation of Taligen’s distinct and targeted alternative pathway complement inhibitors while completing the current eculizumab study, we are in a stronger position to optimize an approach to complement inhibition for local ophthalmic use. Oncology (cid:129) In February 2011, we acquired Orphatec Pharmaceuticals’ In December 2010, investigators presented interim results investigational therapy for infants suffering with molybdenum from the ﬁ rst Phase I/II trial of samalizumab, a ﬁ rst-in-class cofactor deﬁ ciency (MoCD) Type A, an ultra-rare metabolic anti-CD200 antibody and our lead oncology compound. The disease affecting newborns in which a genetic deﬁ ciency of study showed that in patients with B-cell lymphocytic leukemia the normally occurring metabolite cPMP causes a deﬁ ciency (B-CLL) or multiple myeloma (MM), samalizumab was well of molybdenum cofactor, leading to uncontrollable seizures, tolerated, exhibited a dose-dependent response, and showed catastrophic brain damage and death. There are currently no initial evidence of anti-tumor activity. We look forward to treatment options for patients with MoCD Type A, and survival completing this study and further investigating samalizumab in in newborns with the disease is generally measured in weeks patients suffering with a rare solid tumor later in 2011. or months. As with most conditions this rare, the suffering of families is heightened by a paucity of research and drug development. This new investigational therapy is designed to replace the deﬁ cient cPMP, providing the ﬁ rst ray of hope for an effective therapy for these infants and their families. Continued Strong Financial Performance We have served an increasing number of patients with PNH each year since launch, and our ﬁ nancial performance in 2010 reﬂ ects this trend. Net sales of Soliris totaled $541.0 million Given the urgent medical need, our Translational Medicine in 2010, representing a 40% increase from 2009. Non-GAAP Group is focused on rapidly completing work necessary for our net income increased 54% to $167.3 million, or $1.78 per MoCD program to move forward. Our development teams are share. As we grew our business and our pipeline initiatives, we also preparing to introduce a novel anti-inﬂ ammatory antibody, remained committed to maintaining ﬁ nancial discipline and developed internally by Alexion, into clinical studies during 2011. limited non-GAAP operating expense growth to 30%. Ophthalmology While our primary focus is on the development and delivery of breakthrough therapies for patients suffering with ultra-rare disorders, we are committed to employing our pioneering expertise in complement inhibition to develop highly innovative treatments for other patients when such Tight ﬁ nancial controls also enabled us to remain strongly cash positive in 2010, ﬁ nishing the year with $361.6 million in cash, cash equivalents and marketable securities, compared to $176.2 million in 2009. Subsequent to the end of 2010, we used a portion of this cash reserve to fund the acquisitions of Taligen Therapeutics and assets from Orphatec Pharmaceuticals. Alexion Pharmaceuticals 9 2010 Annual Report 76856 Color Text.indd ed9 76856 Color Text.indd ed9 4/1/11 4:25 PM 4/1/11 4:25 PM “Alexion is driven by a mission to transform patients’ lives. I am conﬁ dent that we will bring more therapies to more people, and provide even greater value to the physicians and healthcare delivery systems who are as dedicated as we are to combating the suffering caused by devastating rare diseases.” Leonard Bell, M.D. Chief Executive Ofﬁ cer 76856 Color Text.indd ed10 76856 Color Text.indd ed10 3/30/11 10:33 PM 3/30/11 10:33 PM Strengthening Our Human Capital In 2010, we added exceptional talent to the strong management team at Alexion. Thomas Bock, M.D., joined Alexion as Senior Vice President of Global Medical Affairs, leading our medical research collaborations, scientiﬁ c communications and medical country operations worldwide. Sarah Boyce joined us as Vice President of the Global Nephrology Franchise, with worldwide responsibility for establishing this therapeutic area, and Henric Bjarke joined Alexion as Vice President, North America Commercial Operations. In 2011, with the acquisition of Taligen, we welcomed Abbie Celniker, Ph.D., as head of Alexion’s Translational Foundation. Our employees are involved in a number of programs that support our local communities, including HomeFront, a project to renovate and restore homes for Medicine Group. Dr. Celniker leads a cross-functional team people in need. of world-class scientists in accelerating the investigation and development of novel molecules. She brings more than 20 years’ experience in building biologics-focused businesses. Today, Alexion has approximately 800 employees in 20 locations around the world. Our growing global team comprises many of the most talented people in our industry. Commitment to Global Citizenship As we accelerate our efforts on behalf of patients, Alexion remains committed to being a good citizen of the communities and countries in which we operate. In 2010, we earned two Leadership in Energy and Environmental Design (LEED) certiﬁ cation awards for our global headquarters and research facilities in Cheshire, CT. These gold-standard LEED certiﬁ cations, awarded by the U.S. Green Building Council, recognize Alexion for constructing, operating and maintaining high-performance commercial interiors. We also were honored with the 2010 Connecticut Green Building Council (CTGBC) Green Advocate Award for our efforts to promote environmental stewardship and sustainable business practices. Alexion also supports nonproﬁ t organizations that play an important role in disease education and access to medicines, especially for rare diseases. Through a variety of activities and donations, we support the National Organization for Rare Disorders (NORD), the European Organization for Rare Moving More Rapidly in 2011 By all measures, 2010 was an outstanding year for Alexion and for the patients we serve today and expect to serve tomorrow. Our fundamental focus in 2011 is to serve more patients with PNH in our core territories while expanding to serve patients in additional important geographies – all while fulﬁ lling the vast potential of our broader pipeline portfolio. Alexion is driven by a mission to transform patients’ lives. I am conﬁ dent that we will bring more therapies to more people, and provide even greater value to the physicians and healthcare delivery systems who are as dedicated as we are to combating the suffering caused by devastating and ultra-rare diseases. I thank our shareholders, board of directors and dedicated employees for your belief in our mission. Alexion has never been better positioned to grow through innovation and thus bring health and hope to more patients worldwide. Leonard Bell, M.D. Chief Executive Ofﬁ cer Diseases (EURORDIS) and the PNH Research & Support April 2011 Alexion Pharmaceuticals 11 2010 Annual Report 76856 Color Text.indd ed11 76856 Color Text.indd ed11 3/31/11 6:48 PM 3/31/11 6:48 PM Selected Financial Highlights (In thousands, except per share data) Year Ended December 31, Revenues: Net product sales Contract research revenue Total revenues Cost of sales Operating expenses: Research and development Selling, general and administrative Total operating expenses Operating income Other income (expense) Income before income taxes Income tax provision (beneﬁ t) Net income Earnings per common share Basic Diluted Shares used in computing earnings per share Basic Diluted 2010 2009 2008 $ 540,957 $ 386,800 $ 259,004 — 540,957 64,437 98,394 227,488 — 386,800 45,059 81,915 172,767 95 259,099 28,366 62,581 133,543 325,882 254,682 196,124 150,638 (1,627) 149,011 51,981 87,059 (3,745) 83,314 (211,852) 1 34,609 121 34,730 1,581 $ 97,030 $ 295,166 $ 33,149 $ $ 1.09 1.04 $ $ 3.46 3.26 $ $ 0.43 0.39 89,271 93,037 85,326 90,582 77,680 89,967 As of December 31, 2010 2009 2008 Consolidated Balance Sheet Data: Cash, cash equivalents, and marketable securities $ 361,605 $ 176,220 $ 138,012 Trade accounts receivable, net Inventories Total current assets Property, plant and equipment, net Intangible assets, net Deferred tax assets Total assets Accounts payable and accrued expenses License payable Mortgage loan Convertible notes Total liabilities 168,732 62,165 113,731 40,885 74,476 49,821 646,556 373,456 277,101 162,240 24,146 154,569 164,691 28,589 194,308 139,885 32,325 3,397 1,012,037 786,401 477,551 123,056 — — 3,718 152,301 78,445 — — 9,918 98,045 54,855 25,000 44,000 97,222 230,550 Total stockholders’ equity 859,736 688,356 247,001 Total liabilities and stockholders’ equity 1,012,037 786,401 477,551 1 In 2009 we determined that it was more likely than not that a signiﬁ cant portion of our deferred tax assets in the United States, primarily net operating losses and research and development credits, would be realized. Accordingly, we recorded a tax beneﬁ t of $215,516 as a result of reversing the valuation allowance on these deferred tax asseets. 76856 Color Text.indd ed12 76856 Color Text.indd ed12 3/30/11 10:33 PM 3/30/11 10:33 PM Shareholder Information Directors Max Link, Ph.D.1,4 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, M.D. Chief Executive Ofﬁ cer 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, Ph.D., M.D.2,4 Professor of Pathology, Yale University School of Medicine Larry L. Mathis1,3 Former President and CEO, The Methodist Hospital System R. Douglas Norby1,3 Former Senior Vice President, Chief Financial Ofﬁ cer, Tessera Technologies, Inc. Alvin S. Parven2,3 President, ASP Associates Former Vice President, Aetna Health Plans Andreas Rummelt, Ph.D.1,4 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 U.S. Department of Agriculture Senior Management Leonard Bell, M.D. Chief Executive Ofﬁ cer Stephen P. Squinto, Ph.D. Executive Vice President, Head of Research and Development Patrice Coissac Senior Vice President, President, Alexion Pharma International Sàrl Thomas I.H. Dubin, J.D. Senior Vice President and Chief Legal Ofﬁ cer David L. Hallal Senior Vice President, Global Commercial Operations Vikas Sinha, M.B.A., C.A., C.P.A. Senior Vice President and Chief Financial Ofﬁ cer Camille L. Bedrosian, M.D. Senior Vice President and Chief Medical Ofﬁ cer Thomas Bock, M.D. Senior Vice President, Global Medical Affairs Abbie Celniker, Ph.D. Senior Vice President, Translational Medicine Group M. Stacy Hooks, Ph.D. Senior Vice President, Technical Operations Claude Nicaise, M.D. Senior Vice President, Strategic Development and Global Regulatory James P. Bilotta, M.B.A. Vice President and Chief Information Ofﬁ cer Sarah Boyce Vice President, Global Nephrology Franchise Daniel N. Caron, M.S. Vice President, Site Operations and Engineering Margaret M. Olinger, M.B.A. Vice President, Global Hematology Franchise Jeremy P. Springhorn, Ph.D. Vice President, Corporate Strategy and Business Development Heidi L. Wagner, J.D. Vice President, Global Government Affairs Annual Shareholders Meeting To be held on May 11, 2011 10:00 a.m. The Study at Yale 1157 Chapel Street New Haven, CT 06511 tel 203.503.3900 fax 203.503.3901 Other Information 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 Hartford, CT 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 © 2011 Alexion Pharmaceuticals, Inc. Alexion®, Alexion Logo®, Soliris® and OneSource™ are trademarks of Alexion Pharmaceuticals, Inc. 76856 Cover.indd 2 76856 Cover.indd 2 4/1/11 4:27 PM 4/1/11 4:27 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 Brooksvale NSW Australia, 2100 Alexion Pharma Argentina SRL Ing. Butty 240 5to Piso (C1001AFB) Buenos Aires, Argentina www.alexionpharma.com 76856 Cover.indd 1 76856 Cover.indd 1 3/30/11 9:24 PM 3/30/11 9:24 PM

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