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Alexion Pharmaceuticals

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FY2014 Annual Report · Alexion Pharmaceuticals
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Breakthrough Medical Innovation  

for Severe and Life-Threatening Disorders

2014 Annual Report

“I got very sick when I was 19 years old and was told I had six months to a year to live. After 

nearly 16 years of blood transfusions, bone marrow biopsies, several misdiagnoses and life in and 

out of hospitals, I was finally diagnosed with PNH in 2007. Soon after I was told that I had PNH, 

I began to receive regular infusions of Soliris® , which I still get every two weeks. I am grateful for 

Soliris. Today, I am able to be a father, a husband and the man I always aspired to be.”

— Daniel C-F., patient with PNH receiving Soliris

2014 Accomplishments

January
Alexion Pharmaceuticals and Moderna Therapeutics announce exclusive strategic 
agreement to develop Messenger RNA Therapeutics™ for rare diseases

US Food and Drug Administration (FDA) grants orphan drug designation 
(ODD) to eculizumab for the prevention of delayed graft function (DGF) in 
renal transplant patients

February
European Commission (EC) grants ODD to eculizumab for the prevention of DGF 
after solid organ transplantation

March
The National Institute for Health and Clinical Excellence (NICE) Evaluation Committee 
acknowledges that Soliris® (eculizumab) is a very effective treatment for patients 
with atypical hemolytic uremic syndrome (aHUS) and produces substantial quality-
adjusted life year gains of a magnitude rarely seen for a new drug

April
Alexion initiates the rolling submission of a Biologics License Application (BLA) for 
asfotase alfa as a treatment for patients with hypophosphatasia (HPP) with the FDA 

Alexion initiates multinational registration trials of eculizumab as a potential 
treatment for patients with relapsing neuromyelitis optica (NMO) and refractory 
generalized myasthenia gravis (MG)

Alexion announces the appointment of John T. Mollen to its Board of Directors 

Alexion unveils plans for future expansion in Ireland with construction of a new 
global supply chain facility at College Park, Blanchardstown, Dublin

EC grants ODD to eculizumab for the prevention of graft rejection following solid 
organ transplantation

May
FDA approves conversion from accelerated to regular approval for Soliris in aHUS; 
revised label now specifies important longer-term clinical benefits associated with 
chronic and sustained Soliris treatment with inclusion of results from two years 
of ongoing treatment 

Researchers present new data from the first large natural history study in infants 
with HPP, showing 73% mortality reported at five years in infants with severe 
perinatal and infantile HPP, at the joint meeting of the Pediatric Academic Societies 
(PAS) and the Asian Society for Pediatric Research 

The PAS meeting also features data that show early and sustained improvements 
observed in infants, children and juveniles with HPP receiving asfotase alfa in the 
open-label extension phase of two on-going Phase 2 clinical studies 

June
Investigators present new data demonstrating the efficacy of chronic Soliris 
treatment in a broad range of patients with aHUS, including patients with or 
without a history of renal transplant or dialysis, at the European Renal Association–
European Dialysis and Transplant Association Congress

Alexion initiates proof-of-concept clinical study with ALXN1007 in anti-
phospholipid syndrome

FDA grants ODD to eculizumab for the treatment of patients with MG

Alexion and Cincinnati Children’s announce collaboration and establish a fund 
for the advancement of research in rare disease

July
Alexion announces the appointments of David R. Brennan, M. Michele Burns and 
Christopher J. Coughlin to its Board of Directors 

European Medicines Agency (EMA) accepts marketing authorization application 
for asfotase alfa as a treatment for patients with HPP

August
EC grants ODD to eculizumab for the treatment of patients with MG

Alexion initiates multinational registration trial of eculizumab for the prevention 
of DGF after kidney transplantation

September
For  the  third  consecutive  year, Forbes  magazine  names  Alexion  the  #2  
most innovative company in the world on its 2014 list of the World’s 100 Most 
Innovative Companies
Fortune magazine ranks Alexion #20 on its annual list of the 100 Fastest-Growing 
Companies, making it the third fastest-growing company in the healthcare sector

Asfotase alfa granted ODD in Japan by the Ministry of Health, Labour and  
Welfare (MHLW)

Researchers present new data showing improved survival in pediatric patients with 
severe HPP who were treated with investigational asfotase alfa for up to five years at 
the American Society for Bone and Mineral Research annual meeting

Alexion announces the initiation of Phase 1 clinical studies with ALXN1210 and 
ALXN5500, two “next-generation” Soliris molecules

Alexion announces promotion of David Hallal to the newly created position of  
Chief Operating Officer and appoints Mr. Hallal to Alexion’s Board of Directors

October
Alexion announces appointments of Leonard Bell, MD, CEO, to Chairman of the 
Board of Directors and R. Douglas Norby to the newly created Board position of 
Lead Independent Director

Alexion submits New Drug Application to Japan’s MHLW for asfotase alfa as a 
treatment for patients with HPP

Alexion announces the initiation of Phase 1 clinical studies with ALXN1210 and 
ALXN5500, two “next-generation” Soliris molecules

November
Alexion announces plans to establish its new Europe, Middle East & Africa (EMEA) 
regional headquarters in Zurich, Switzerland

Investigators present longer-term outcome data from the largest prospective 
trial of Soliris in aHUS, which underscores the effectiveness of ongoing Soliris 
treatment at the American Society of Nephrology annual meeting

Eculizumab granted ODD by Japan’s MHLW for the treatment of patients with NMO

NICE Highly Specialised Technologies Evaluation Committee issues final 
positive recommendation for national commissioning of Soliris for all patients 
with aHUS in England

Alexion initiates proof-of-concept clinical study with ALXN1007 in gastrointestinal 
graft-versus-host disease

December
Alexion completes rolling BLA submission to FDA for asfotase alfa as a treatment 
for patients with HPP

New data enhancing the clinical knowledge of aHUS and paroxysmal nocturnal 
hemoglobinuria (PNH) and underscoring the effectiveness of Soliris treatment 
presented at the American Society of Hematology annual meeting

Eculizumab granted ODD by Japan’s MHLW for the treatment of patients with MG

Early 2015
Alexion appoints David Hallal as Chief Executive Officer, effective April 1, 2015, 
succeeding Leonard Bell, MD, principal founder of Alexion, who will retire as CEO 
and continue to serve as Chairman of the Board

FDA grants priority review for asfotase alfa as a treatment for patients with 
infantile- and juvenile-onset HPP

New data from first natural history study in juveniles with HPP, showing wide range of 
HPP-related complications and persistent nature of disease, presented as late-breaking 
data at the Endocrine Society’s Annual Meeting and Expo

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 the Annual Report as a result of new information, future events or otherwise. 

Remembering Our Former Chairman

Dr. Max Link was a globally respected leader 
of companies large and small in the pharmaceutical, 

biotechnology and medical device industries over the 

course of nearly four decades. Max was a tireless 

champion of medical innovation and entrepreneurship 

for the benefit of patients, and we were very fortunate 

to have had the benefit of his wisdom, experience and 

global perspective as a member of our Board of Directors 

since our inception in 1992, and as Chairman of the 

Board from 2002 until his untimely passing.

In our earlier years, Max provided invaluable guidance 

during the inevitable challenges we faced as a biotech 

startup. His leadership and fortitude were vital in these 

years. As Chairman, he was a key guide and mentor in 

our transition, starting in 2007, from a development-

stage firm to a global development and commercial firm 

serving patients with devastating and life-threatening 

disorders worldwide. Through his work at Alexion and 

other companies, Max helped to bring a wide array of 

innovative therapies to patients.

Max was a colleague, friend, mentor, confidant and guide 

over the course of 22 years. Each one of us at Alexion – and 

all the patients we serve – has been positively touched by 

the invaluable guidance he provided. We miss Max dearly 

and will carry his memory with us every day. We join with 

his family and close friends in mourning our loss.

Max Link, PhD
(b. 9.26.1940 – d. 10.6.2014) 

To Our Shareholders:

In 2014, Alexion continued to expand its commercial and clinical 

PNH — Reaching More Patients 

operations as we advanced our mission to develop and deliver 

life-transforming therapies for patients with severe and life-

Across our 50-Country Platform 
The steady increase in the number of new patients starting 

threatening rare diseases. During the year, we reached a wide 

on Soliris in 2014 affirms our view that, on a global basis, 

range of significant milestones on behalf of patients and their 

the majority of patients with PNH have yet to receive an 

families while continuing to build a larger and highly efficient 

accurate diagnosis, let alone commence appropriate therapy. 

global enterprise to support a broad portfolio of transformative 

Throughout 2014, as in prior years, we continued to identify 

therapies, starting with our next product, asfotase alfa. Among 

a consistently high number of newly diagnosed patients with 

our achievements in 2014, we: 

•  Continued  to  provide  Soliris®  (eculizumab)  to  an 

increasing number of patients worldwide with paroxysmal 

nocturnal hemoglobinuria (PNH) and atypical hemolytic 

uremic syndrome (aHUS). 

•  Completed the rolling submission of our US Biologics 

License  Application  (BLA)  for  asfotase  alfa  as  a 

treatment for patients with infantile- and juvenile-onset 

hypophosphatasia (HPP), a severe metabolic disorder, 

which the US Food and Drug Administration (FDA) has 

accepted for priority review. In addition, we completed 

our submissions for marketing authorizations for this 

highly innovative therapy in the EU and Japan, and we 

have significantly expanded the body of knowledge about 

HPP and the clinical benefits of asfotase alfa to support 

optimal patient care.

PNH in the US, Western Europe and Japan — the territories 

in which we have operated the longest — and we are also 

observing consistent additions of new patients commencing 

Soliris therapy across Turkey, Brazil and Russia. While we are 

pleased with the continued positive impact we are having 

on the lives of patients with PNH, we know that ongoing 

education is required to further enhance rapid and accurate 

diagnosis and effective treatment. 

aHUS — Strong Performance in the 

Ongoing Global Launch 
We continued to observe a steady addition of new patients 

with aHUS commencing Soliris therapy in the US and 

Europe in 2014, while we made important progress in the 

early stages of serving patients with aHUS in Japan. The 

number of new patients with aHUS being identified by 

physicians — including children who have rapidly progressing, 

life-threatening complications — confirms our view that our 

•  Advanced the first two of our next-generation Soliris 

opportunity to serve patients and families suffering with 

molecules into the clinic, and — based on initial clinical 

aHUS is at least as large as our opportunity to serve the 

data — now intend to advance at least one of these 

PNH community, and perhaps larger. As one measure, in 

molecules into Phase 2 trials in patients with PNH in 2015.

the US — more than three years following their respective 

•  Progressed our other key clinical development programs 

while establishing a strong foundation for future growth 

with an additional 17 preclinical development programs 

spanning diverse modalities and therapeutic areas.

Reaching Patients with PNH and aHUS 
We developed Soliris, the world’s first and only approved 

terminal complement inhibitor, from the laboratory 

through regulatory approvals and commercialization in 

PNH and aHUS, two devastating and life-threatening 

ultra-rare disorders. 

FDA approvals — more patients are currently receiving 

Soliris for aHUS than there had been for PNH. In Europe, 

we are experiencing a similar trend among patients with 

aHUS. These observations are in line with our view that the 

incidence of aHUS is likely higher than PNH. Our educational 

initiatives in aHUS are supported by the strengthening of 

our Soliris labels in the US and Europe, which now specify 

the important longer-term clinical benefits associated with 

chronic and sustained Soliris treatment with inclusion of 

results from two years of ongoing treatment. 

Breakthrough Medical Innovation for Patients with Severe and Life-Threatening Disorders 

3

“I got sick when I was 14 and was in excruciating pain. It took nearly two years for the doctors to 

diagnose me with aHUS. As soon as I was diagnosed, I started receiving Soliris®. While I know that 

I will have aHUS for the rest of my life, I no longer think about it every day, and it is very reassuring 

to finally have a treatment that can help me. Before I got sick I was always interested in medicine, 

but my experience battling aHUS has motivated me to pursue a career in medicine.”

— Julia G., patient with aHUS receiving Soliris

Advancing Our Relentless Mission to 
Provide Transformative Therapies
Beyond PNH and aHUS, we made significant progress across 

Eculizumab: Expanding Our Portfolio 

with New Indications
As we seek to build on the strong, long-term safety and 

all of our lead development programs in 2014, all of which 

efficacy profile of Soliris® in PNH and aHUS, we are 

are focused on first-in-class therapeutic breakthroughs. 

investigating eculizumab as a potential treatment for patients 

Preparing for the Launch of Asfotase Alfa 
As we prepare to provide our next product, asfotase alfa, 

to patients with HPP, we are applying key learnings from 

our experience in serving patients with PNH and aHUS 

for optimizing care with a highly innovative ultra-orphan 

therapy. We expect regulatory decisions in the US, EU 

and Japan this year, and are preparing to serve patients 

in the US and Germany during the first half of 2015 and 

in Japan by year-end. We are working with regulatory 

authorities to obtain these marketing authorizations as 

quickly as possible, given the high rates of mortality, severe 

debilitating effects and current lack of any approved therapy 

for patients with HPP. 

In HPP, as in other ultra-rare disorders, education will be 

critical to helping patients, and our programs will employ 

the growing body of clinical data reflecting the potentially 

transformative impact of asfotase alfa. For example, 

researchers at the Endocrine Society Annual Meeting in 

March 2015 presented new data from a retrospective, 

multinational natural history study of children with HPP. 

Data from this study, which included 32 patients with 

juvenile-onset HPP, demonstrated that children with HPP 

have a substantial disease burden, particularly with regard to 

musculoskeletal abnormalities and growth deficiencies. These 

patients experienced HPP-related disease complications 

and morbidity that persisted despite standard efforts 

to control symptoms.

In parallel to the regulatory filings, we continue to build out 

our initial field-based medical teams and our in-country 

metabolic commercial teams, and they have begun educating 

physicians on the signs and symptoms of HPP and the 

appropriate pathways for a rapid and accurate diagnosis.

with other severe and rare complement-mediated disorders. 

In  neurology,  we  have  development  programs  with 

eculizumab  in  patients  with  two  severe  disorders: 

neuromyelitis optica (NMO) and myasthenia gravis (MG). 

In 2014, we were pleased that eculizumab was granted 

orphan drug designations in Japan for NMO and in the US, 

EU and Japan for MG. NMO is a life-threatening ultra-rare 

neurological disorder in which uncontrolled complement 

activation leads to severe damage to the central nervous 

system in patients, including their spinal cord and optic 

nerve. Our study is focused on patients who continue 

to experience relapses despite supportive treatment. 

Enrollment and dosing are ongoing in the PREVENT study, 

our registration trial in relapsing NMO. 

MG is a debilitating and potentially life-threatening disorder 

in which uncontrolled complement activation results in 

destruction and inflammation at the junction between 

nerves and muscles in patients, leaving their muscles 

severely weakened. The REGAIN study, our registration 

trial in MG, is focused on patients with severe disease who 

are refractory to other treatment options. We expect to 

complete enrollment in REGAIN in 2015. 

In transplant, a multinational registration trial with 

eculizumab in kidney transplant patients at increased risk 

for delayed graft function (DGF) is ongoing, and we expect 

to complete enrollment by the end of the year. 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, 

potentially resulting in the loss of the organ. There is 

currently no approved therapy to prevent DGF in kidney 

transplant recipients. In addition, a significant number of 

Breakthrough Medical Innovation for Patients with Severe and Life-Threatening Disorders 

5

Research Pipeline

Preclinical

Early Clinical  
Development

Advanced Clinical  
Development

Registration Filings

Market

Soliris® (eculizumab)

Approved Indications

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Atypical Hemolytic Uremic Syndrome (aHUS)

Investigational Indications – Transplant and Neurology

Refractory Myasthenia Gravis (MG)

Relapsing Neuromyelitis Optica (NMO)

Delayed Graft Function (DGF)

Antibody-Mediated Rejection (AMR)

Next-Generation Portfolio

ALXN1210

ALXN5500

Investigational Candidates – Metabolic Disorders

Asfotase Alfa

Hypophosphatasia (HPP)

cPMP Replacement Therapy (ALXN1101)

Molybdenum Cofactor Deficiency (MoCD) Type A

Investigational Candidates — Inflammatory Disorders

ALXN1007 Anti-inflammatory Antibody

Antiphospholipid Syndrome (APS)

Gastrointestinal Graft-versus-Host Disease (GI-GVHD)

Preclinical Candidates

Messenger RNA (mRNA) Therapeutics

Other Preclinical Candidates

6 

Alexion Pharmaceuticals, Inc.  2014 Annual Report

donor kidneys are reportedly discarded each year due to 

treatment of patients with molybdenum cofactor deficiency 

the risk of DGF and its associated poor clinical outcomes. 

(MoCD) Type A, a severe and life-threatening, ultra-rare, 

We are also evaluating eculizumab in antibody-mediated 

rejection (AMR), a severe and potentially life-threatening 

condition that can lead to rapid deterioration of function 

and possible loss of the transplanted organ. Later this year 

we expect data from our Phase 2 deceased-donor study 

for the prevention of AMR, as well as the initiation of 

our clinical study with eculizumab for the treatment of 

patients with AMR. We continue to evaluate the results of 

our clinical study with eculizumab for the prevention of 

AMR in patients receiving living-donor transplants, which 

missed its primary endpoint.

Other Highly Innovative Therapeutic Candidates
As the leaders in complement biology, we advanced the 

development of the first two next-generation Soliris® 

molecules in 2014. We initiated Phase I studies of ALXN1210 

genetic metabolic disorder that causes catastrophic and 

irreversible neurologic damage within the first weeks of life. 

The synthetic cPMP bridging study in patients with MoCD 

is ongoing, and we expect to complete enrollment in 2015. 

We are also continuing our retrospective data collection 

and natural history study.

Expanding Early-Stage Research
Within our portfolio of 17 preclinical programs, we have 

initiated preclinical development in our first seven messenger 

RNA (mRNA) programs with our collaborator Moderna 

Therapeutics, focused on the treatment of patients with 

severe and rare disorders. We are targeting our first candidate 

to enter the clinic in 2016. In addition, we entered into 

several other earlier-stage preclinical programs during 2014. 

and ALXN5500, with additional molecules and programs in 

our expanding portfolio at earlier stages of development. 

Strong Financial Performance
2014 was another year of robust growth and profitability 

The data already support advancing both of these molecules 

for Alexion as we provided Soliris to an increasing number 

into further trials — and we intend to progress at least one 

of patients with PNH and aHUS worldwide. Net product 

of them into a Phase 2 PNH trial this year — with additional 

sales for the year increased 44 percent to $2.234 billion, 

indications likely to follow. ALXN1210 is a longer-acting 

compared to $1.551 billion in 2013. Excluding the impact of 

anti-C5 monoclonal antibody suitable for once-monthly 

$88 million for reimbursement of prior year shipments, 2014 

dosing. We are targeting approval of at least one next-

net product sales increased 38 percent to $2.146 billion. 

generation candidate in 2018.

We also have lead development programs with two 

additional highly innovative therapies, ALXN1007 and cyclic 

pyranopterin monophosphate (cPMP).

We have commenced dosing in two Phase 2 proof-of-

concept studies to evaluate the safety and tolerability 

of ALXN1007 in patients with two severe and potentially 

life-threatening auto-immune diseases: graft-versus-host 

disease involving the gastrointestinal tract, or GI-GVHD, 

and antiphospholipid syndrome, or APS. We expect to have 

interim data in the GI-GVHD study later in 2015.

In our metabolic disease area, we continue to advance 

development of our cPMP replacement therapy for the 

By exceeding our revenue target, while maintaining strict 

financial discipline in our growing commercial and clinical 

activities, we reported 2014 non-GAAP EPS of $5.21 per 

diluted share, an increase of 69% year-over-year. Excluding 

the impact of the $88 million in pre-2014 sales, EPS would 

be $4.84, an increase of 57% in 2014. Our year-on-year 

revenue growth was robust across all territories we serve. 

During 2014, we further aligned our global structure and 

invested in improving operational efficiency as we provide 

more therapies to more patients around the world. We 

expanded our Global Supply Chain and Quality operations 

in Ireland, which will include our first company-owned 

fill/finish facility. We are also in the process of moving 

our EMEA headquarters to Zurich, a major center for the 

Breakthrough Medical Innovation for Patients with Severe and Life-Threatening Disorders 

7

Standing (from left): Julie O’Neill, Executive Vice President, Global Operations; Vikas Sinha, MBA, CA, CPA,  
Executive Vice President and Chief Financial Officer; Edward Miller, JD, Senior Vice President,  
Global Chief Compliance Officer; John B. Moriarty, Jr., JD, Executive Vice President, General Counsel;  
Saqib Islam, JD, Executive Vice President, Chief Strategy & Portfolio Officer; Martin Mackay, PhD,  
Executive Vice President and Global Head of Research & Development.

Seated (from left): David Hallal, Chief Operating Officer, CEO-elect; Leonard Bell, MD, Chairman of the Board, 
Chief Executive Officer; Clare Carmichael, Executive Vice President, Chief Human Resources Officer.

“Alexion is at the strongest and most promising point in our history as we move toward 

becoming a multi-product company with our most robust development pipeline ever.  

Our significant accomplishments throughout 2014 have positioned our global organization  

to reach a new set of milestones on behalf of patients in 2015 and beyond as we  

maintain our relentless focus to bring life-transforming treatments to more patients  

with severe and life-threatening diseases worldwide.” 

— Leonard Bell, MD,  Chairman of the Board and Chief Executive Officer

pharmaceutical and biotechnology industries, to maximize 

instrumental in driving our results and building the high-

our ability to attract talent and engage in commercial and 

performing, patient-centered culture for which we are 

academic collaborations. Finally, we have announced the 

known, with accomplishments that include leadership of 

establishment of the Alexion Research and Development 

the highly successful launches of Soliris® for PNH and aHUS 

Center in Paris, the first Alexion research facility outside 

and a key role in the build-out of our 50-country operating 

of North America, which will focus on the discovery of 

platform. As we have worked closely together for nearly 

innovative therapies for patients with severe, rare diseases.

a decade and increasingly shared responsibilities over the 

Recognizing Two Alexion Leaders 
Along with our accomplishments in 2014, we were greatly 

saddened by the unexpected passing of Max Link, PhD, in 

October. Max had been a Director of Alexion since we were 

established in 1992, and served as Chairman of our Board 

of Directors since 2002. Please see page 2 herein to learn 

more about Max — an amazing person, a great friend and 

mentor, and one of our industry’s leading champions of 

innovation for the benefit of patients. 

Also in late 2014, Steve Squinto, PhD, my Alexion co-founder 

and friend, announced his planned retirement from Alexion 

effective January 1, 2015, following 22 years during which he 

past few years, I know that David is the right person to lead 

us into our next chapter of growth, and I look forward to 

continuing to guide the Board and advising the Company 

on strategic matters in my role as Chairman. 

For all that the Alexion team has accomplished for patients 

to date, we are excited that the opportunities ahead of 

us are far greater. In 2015 and beyond, our goals are 

to focus our global skills and resources toward serving 

a continuously increasing number of patients with PNH 

and aHUS … beginning to serve patients with HPP … and 

significantly expanding our portfolio through both internally 

and externally developed therapeutic candidates to provide 

transformative outcomes to patients suffering with severe 

helped to drive Alexion’s evolution into a global leader in the 

and rare disorders. 

development, manufacturing and delivery of biotechnology 

therapeutics. We are very pleased that Steve will continue 

to contribute to our mission as the Chair of our newly 

established Scientific Advisory Board, and likewise pleased 

that Julie O’Neill has now taken on Steve’s most recent 

position as Executive Vice President, Global Operations. 

Looking Ahead
In January 2015, we also announced my own retirement as 

CEO and the appointment of David Hallal, Alexion’s Chief 

Operating Officer, as my successor, effective April 1, 2015. 

As Alexion is at a position of great strength with regard 

to commercial execution, financial discipline, and pipeline 

breadth and growth, the time is right for the Company, for 

David and for me personally to make this transition. In line 

with the thoughtful succession planning process undertaken 

As always, we thank our growing number of employees 

and  the  many  other  people  who  make  our  work 

possible — including researchers, physicians, patients 

and families. We are united in our commitment to serve 

patients with severe and life-threatening disorders through 

breakthrough medical innovation and to transform their 

lives from illness and desperation … into health and hope. 

Leonard Bell, MD 

Chairman and Chief Executive Officer

by our Board, David will become the second CEO in our 

March 2015

company’s history. David has demonstrated outstanding 

leadership over the past decade at Alexion and has been 

Breakthrough Medical Innovation for Patients with Severe and Life-Threatening Disorders 

9

“The medicines we develop need to be transformative because the diseases we strive  

to treat are so devastating. At the same time, the work we do must be global because  

these diseases know no boundaries and affect patients all around the world.”

Martin Mackay, PhD,  Executive Vice President, Global Head of Research & Development

Global Locations

Global and Regional Headquarters

Cheshire, CT, USA
Global Headquarters
North America Regional HQ

Dubai, United Arab Emirates
Middle East Operations
Country Operations 

Dublin, Ireland
Global Supply Chain and Distribution

Lausanne, Switzerland
EMEA Regional Headquarters
International Operations Center
Country Operations

Miami, FL, USA
Latin America Regional Headquarters

Sydney, Australia
Asia-Pacific Regional Headquarters
Country Operations

Tokyo, Japan
Japan Headquarters
Country Operations

Specialized Facilities and Country Operations

Barcelona, Spain
Country Operations

Bogotá, Colombia
Country Operations

Brussels, Belgium 
Government Affairs, EMEA
Country Operations

Buenos Aires, Argentina
Country Operations

Cambridge, MA, USA
Research and Development

Istanbul, Turkey
Country Operations

London, United Kingdom
Country Operations

Mexico City, Mexico
Country Operations

Milan, Italy
Country Operations

Moscow, Russia
Country Operations

Mumbai, India
Global Business Services

Munich, Germany
Country Operations

Osaka, Japan
Country Operations 

Paris, France
European Service Center
Country Operations

São Paulo, Brazil
Country Operations

Shanghai, China
Country Operations

Smithfield, RI, USA
Manufacturing Operations

Stockholm, Sweden
Nordic Country Operations

Toronto, Canada
Country Operations

Washington, DC, USA
Global Government Affairs

Breakthrough Medical Innovation for Patients with Severe and Life-Threatening Disorders 

11

Financial Highlights

Soliris® Net Product Sales 
($ in Millions)

Net Income 
($ in Millions)

$ 2,500

$ 2,000

$ 1,500

$ 1,000

$ 500

$ 0

$ 1,200

$ 1,000

$ 800

$ 600

$ 400

$ 200

$ 0

Other

Asia Pacific

Europe

United States

Non-GAAP

GAAP

2014*

2013

2012

2014

2013

2012

*  Included in Europe revenues for 2014 is a reimbursement of $87.83 million for shipments  

made prior to 2014 as a result of an agreement with the French government.

Earnings Per Share – Diluted 

Cash, Cash Equivalents and Marketable Securities 
($ in Millions)

$ 6.00

$ 5.00

$ 4.00

$ 3.00

$ 2.00

$ 1.00

$ 0.00

$ 2,000

$ 1,500

$ 1,000

$ 500

$ 0

Non-GAAP

GAAP

Marketable 
Securities

Cash and 
Cash Equivalents

2014

2013

2012

2014

2013

2012

Reconciliation of GAAP to Non-GAAP Financial Results
(in thousands expect per share amounts)

Results for the years ended December 31,

GAAP net income

Share-based compensation expense

Acquisition related costs

Upfront and milestone payments related to license and collaboration agreements

Impairment of intangible assets

Restructuring expenses

Amortization of purchased intangible assets

Change in contingent liability from intellectual property settlements

Tax related to acquisition structuring

Non-cash taxes

Non-GAAP net income

GAAP earnings per share - diluted

Non-GAAP earnings per share - diluted

2014

$ 656,912

114,461

20,295

109,925

11,514

15,365

—

—

—

137,449

$ 1,065,921

$ 3.26

$ 5.21

2013

$ 252,895

2012

$ 254,822

76,203

5,029

14,500

33,521

—

417

9,181

—

232,460

$ 624,206

$ 1.27

$ 3.08

54,013

22,812

—

26,300

—

417

(53,377)

21,812

98,364

$ 425,163

$ 1.28

$ 2.13

12 

Alexion Pharmaceuticals, Inc.  2014 Annual Report

Shareholder Information

Directors

Senior Management

Annual Shareholders Meeting

Leonard Bell, MD
Chairman of the Board, Chief Executive Officer

Leonard Bell, MD, 
Chairman of the Board, Chief Executive Officer

David R. Brennan4,5
Former Chief Executive Officer,  
Executive Director, AstraZeneca PLC

M. Michele Burns2,3,5
Center Fellow & Strategic Advisor,  
Stanford University Center on Longevity

Chief Executive Officer, Retirement Policy 
Center, Marsh & McLennan Companies, Inc.

David Hallal
Chief Operating Officer, CEO-elect

Vikas Sinha, MBA, CA, CPA
Executive Vice President, Chief Financial Officer

Martin Mackay, PhD
Executive Vice President,  
Global Head of Research & Development

Clare Carmichael
Executive Vice President,  
Chief Human Resources Officer

Saqib Islam, JD
Executive Vice President,  
Chief Strategy & Portfolio Officer

John B. Moriarty, Jr., JD
Executive Vice President, General Counsel

Julie O’Neill
Executive Vice President, Global Operations

Edward Miller, JD
Senior Vice President,  
Global Chief Compliance Officer

Dominique Monnet
Senior Vice President, Chief Marketing Officer

Carsten Thiel, PhD
Senior Vice President, EMEA & Asia-Pacific

Christopher J. Coughlin1,4
Senior Advisor, McKinsey & Co.

Former Advisor to the Chairman and  
CEO of Tyco International Ltd.

Former Executive Vice President and  
Chief Financial Officer of Tyco

David Hallal
Chief Operating Officer, CEO-elect,  
effective April 1, 2015

William R. Keller2,3,5
Vice Chairman, Shanghai Association  
of Foreign Investment Enterprises

Senior Consultant, Shanghai Foreign 
Investment Development Board

Former General Manager, Roche China Ltd.

John T. Mollen1,2
Former Special Advisor to the Chairman,  
EMC Corporation

Former Executive Vice President, Human 
Resources, EMC Corporation

R. Douglas Norby1,3,4
Lead Independent Director  
Former Senior Vice President, Chief Financial 
Officer, Tessera Technologies, Inc.

Alvin S. Parven1,2,3
President, ASP Associates

Former Vice President, Aetna Health Plans

Andreas Rummelt, PhD4,5
CEO, InterPharmaLink AG

Former Group Head, Quality Assurance  
and Technical Operations, Novartis

Former Member of Executive Committee, Novartis

Former CEO, Sandoz AG

Ann M. Veneman3,4,5
Former Executive Director of UNICEF

Former Secretary of U.S. Department  
of Agriculture

1  Member of the Audit and Finance 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  Member of the Strategy and Risk Committee

©  2015 Alexion Pharmaceuticals, Inc. 

Alexion®, Alexion Logo® and Soliris® are trademarks of Alexion Pharmaceuticals, Inc.

To be held on Wed., May 6, 2015 
5:30 p.m.

The Study at Yale  
1157 Chapel Street  
New Haven, CT 06511

tel 203.503.3900

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 
555 Madison Avenue 
5th Floor 
New York, NY 10022

tel 917.322.2568
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.

alexion.com

About The Cover
“Evie was diagnosed with hypophosphatasia when she was two weeks old, and we were 

so scared that she would not survive because she was having seizures, had difficulty 

breathing and was so fragile since she did not develop bones properly. A few months 

later we were fortunate to enroll Evie in a clinical trial for asfotase alfa. It has been 

a long road, but Evie is now five years old and is a true joy. She loves preschool and 

playing with her sisters — it is a blessing each day to watch who she is becoming.”

— Lindsey E., mother of a child with HPP

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, Ireland

Alexion Services Latin America, Inc. 
600 Brickell Avenue, 16th Floor, Miami, FL 33131, USA

alexion.com