Alexion Pharmaceuticals

2011 Annual Report

Transforming lives through innovation in ultra-rare diseases

“My daughter was three years old when I was diagnosed with aHUS.  
I was so scared that I would not see her start kindergarten, much less 
graduate from college, get married and have her own children. After 
years of hospitalizations and constant dialysis, I was put on Soliris in 
2011. I feel extremely blessed to be living a normal life now.”

Jill Z.,  Patient with aHUS receiving Soliris®

2011 Accomplishments

Soliris® (eculizumab) becomes available to patients with 

Alexion receives FDA approval for Soliris as the first and only 

paroxysmal nocturnal hemoglobinuria (PNH) in Australia 

treatment for adult and pediatric patients with aHUS in the 

following a pivotal government reimbursement decision  

United States; the same day, the European Committee for 

that Soliris is life-saving

Alexion acquires Taligen Therapeutics and creates the Alexion 

Translational Medicine Group

Alexion acquires an investigational cPMP replacement 

therapy from Orphatec Pharmaceuticals for infants suffering 

from molybdenum cofactor deficiency (MoCD) Type A, a 

Medicinal Products for Human Use (CHMP) recommends that 

Soliris be approved for the treatment of pediatric and adult 

patients with aHUS in Europe

An exploratory, 14-patient Phase 2 study of eculizumab in 

patients with severe and refractory myasthenia gravis shows  

a strong disease-improvement signal 

catastrophic, ultra-rare genetic metabolic disorder

The European Commission grants marketing authorization for 

Alexion is added to the NASDAQ-100 Index, a list of the 

largest domestic and international non-financial companies on 

the NASDAQ Stock Market

Alexion responds to the earthquake and tsunami in Japan by 

supporting relief efforts and working to maintain continuity of 

Soliris therapy for PNH patients in Japan 

Soliris as the first and only treatment for adult and pediatric 

patients with aHUS in Europe

At the American Society of Nephrology (ASN) annual meeting, 

clinical investigators present longer-term data showing 

significant and sustained benefits of Soliris in patients with 

aHUS enrolled in Phase 2 extension studies

In a separate presentation at the ASN annual meeting, 

investigators report strong interim data from an open-label 

clinical trial to investigate eculizumab as a treatment for 

Alexion submits marketing applications to the US Food and 

patients with STEC-HUS

Drug Administration (FDA) and the European Medicines 

Agency (EMA) for Soliris as a treatment for patients with 

atypical hemolytic uremic syndrome (aHUS)

Researchers present nine studies related to Soliris in patients with 

PNH and aHUS at the 16th Congress of the European Hematology 

Association (EHA); data include the consequences of PNH and the 

positive impact of Soliris on long-term outcomes, and final results 

from the pivotal Phase 2 studies of Soliris in patients with aHUS

Alexion responds to the Enterohemorrhagic Escherichia coli 

(EHEC) crisis in Germany by providing free compassionate 

access to Soliris for several hundred patients and initiating 

an open-label clinical trial to investigate eculizumab as a 

treatment for Shiga toxin-producing E. coli hemolytic uremic 

syndrome (STEC-HUS) resulting from the EHEC outbreak

Data presented at the American Society of Hematology (ASH) 

annual meeting underscore the severity of PNH and confirm the 

importance of consistently testing high-risk patients for PNH 

Alexion agrees to acquire Enobia Pharma Corp. and 

asfotase alfa, the first potential treatment for patients 

with hypophosphatasia (HPP), an ultra-rare, inherited, 

life-threatening, metabolic disease for which there are no 

approved or effective treatments

The New England Journal of Medicine publishes data 

from a Phase 2 study of asfotase alfa in life-threatening 

hypophosphatasia; the study met its primary endpoint with 

90% of patients showing substantial skeletal healing at 

24 weeks, and achieved key secondary endpoints including 

improvement in cognitive development and motor and 

Provincial reimbursement decisions in Canada significantly 

pulmonary function 

broaden access to Soliris for Canadian patients with PNH

Alexion Pharmaceuticals  Transforming lives through innovation in ultra-rare diseases 

1

J
a
n
u
a
r
y
F
e
b
r
u
a
r
y
M
a
r
c
h
A
p
r
i
l
J
u
n
e
J
u
l
y
S
e
p
t
e
m
b
e
r
N
o
v
e
m
b
e
r
D
e
c
e
m
b
e
r
E
a
r
l
y
 
2
0
1
2
“My life changed dramatically in 2002 when I went from an active and 
energetic event security manager who was busy planning a future 
to suddenly having debilitating pain, extreme fatigue, and long 
hospitalizations. It took two years until I was diagnosed with PNH, and 
then I was terrified that I would not be able to be part of a future with 
my wife. Today I am taking Soliris, sharing happy moments with my 
growing family, and living like other 35-year-old husbands and fathers.”

Victor E., Patient with PNH receiving Soliris

To Our Shareholders:

In 2011, Alexion reached new levels in our mission to transform 

•	 Advance our eculizumab clinical programs in Shiga toxin-

the lives of patients with severe and ultra-rare disorders. By 

producing E. coli hemolytic uremic syndrome (STEC-HUS), 

acting with urgency and strategic focus on behalf of the 

acute humoral transplant rejection (AHR), severe and 

patients and families we serve, we exceeded the ambitious 

refractory neuromyelitis optica (NMO), and severe and 

objectives we had set for the year. Our remarkable progress in 

refractory myasthenia gravis 

2011 includes:

•	 Drive development of asfotase alfa, a highly innovative 

•	 Bringing Soliris® (eculizumab) to more patients with 

enzyme replacement therapy that has the potential 

paroxysmal nocturnal hemoglobinuria (PNH) in our core 

to become the first treatment for patients with 

territories of the United States, Western Europe, and Japan, 

hypophosphatasia (HPP), an ultra-rare, inherited, and life-

as well as to patients in new countries 

threatening metabolic disorder

•	 Achieving regulatory approvals in the US and European 

•	 Accelerate the development of additional novel therapies, 

Union for Soliris as the first and only treatment for patients 

including cPMP replacement therapy for newborns with a 

with atypical hemolytic uremic syndrome (aHUS)

fatal metabolic disorder; ALXN1007, our anti-inflammatory 

•	 Deepening our pipeline through three strategic acquisitions 

of highly innovative product candidates focused on what we 

know well and do well: developing and delivering innovative 

and transformative treatments for patients with severe and 

ultra-rare disorders

•	 Advancing our lead development programs, which now 

include five highly innovative biotechnology drug candidates 

including Soliris, in eight severe and ultra-rare disorders 

beyond PNH and aHUS

antibody; and TT30, a novel inhibitor of the alternative 

complement pathway

Serving More Patients with PNH

PNH is an ultra-rare blood disorder in which chronic, 

uncontrolled activation of the complement system causes 

destruction of red blood cells (hemolysis), leading to severe 

clinical manifestations, including recurring blood clots, 

progressive kidney disease, and significantly shortened 

lifespans. Historically, up to 35% of patients with PNH died 

•	 Sustaining high growth while maintaining strong  

within five years of diagnosis. 

financial discipline

Today, we are building on this clinical and commercial expertise 

as we continue to expand our commitment to patients with 

severe and ultra-rare disorders. We entered 2012 with the widest 

global commercial operations and the deepest development 

pipeline in our Company’s history. As we look ahead, we are 

reaching even further with a growth strategy designed to deliver 

first-in-class, highly innovative therapies to more patients with 

ultra-rare and life-threatening disorders. In 2012, we will:

•	 Expand our global presence in PNH to bring the transformative 

benefits of Soliris to more patients in more countries

•	 Build on our strong medical, regulatory, and commercial 

capabilities worldwide to bring Soliris to a growing number 

of patients with aHUS in the US and EU

Since receiving FDA approval for PNH in 2007, Soliris has been 

changing outcomes for patients and families suffering from 

the disease. In addition to dramatic clinical improvements in 

PNH manifestations, long-term retrospective data published by 

independent researchers in the journal Blood reported that the 

survival of studied patients with PNH who were treated with 

Soliris was no different than the survival of healthy, normal 

individuals. These data suggest that patients who once faced 

early mortality can now hope to live a normal life. 

In 2011, we continued the trajectory of growth we have 

achieved since launch, bringing Soliris to more patients with 

PNH, primarily in our core territories of the US, Japan, and 

Western Europe. We also widened our approach by assembling 

operational leadership that will enable us to serve patients 

in Turkey, Brazil, and Russia, as well as other countries in the 

Alexion Pharmaceuticals  Transforming lives through innovation in ultra-rare diseases 

3

Alexion is serving a growing number of patients around the world. 
Soliris is currently approved in more than 40 countries for the 
treatment of PNH, and in the United States and the European 
Union for the treatment of aHUS.

Middle East, Latin America, Asia-Pacific, and Eastern Europe. 

after, on November 24, 2011, the European Commission granted 

In all territories, our work on behalf of patients is focused 

approval for Soliris as the first treatment for aHUS in the EU. 

on disease education and awareness, with an emphasis on 

In both the US and EU, the aHUS labels for Soliris are broad 

diagnostic testing of patients with a higher likelihood of 

and strong, including patients regardless of age, clinical profile, 

having PNH. Importantly, in our core territories, we continue 

identifiable genetic mutations, or history of supportive care. 

to observe that a majority of new patients who have started 

on Soliris are also newly diagnosed with PNH, reflecting the 

positive impact of these initiatives.

The importance of Soliris to the aHUS community is clear. 

Historically, more than one-half of patients with aHUS have died, 

required kidney dialysis, or had permanent renal damage within 

Our disease education efforts are supported by the growing 

one year of diagnosis. For many patients, life with aHUS meant 

body of clinical data underscoring the severity of PNH and the 

frequent hospitalizations, reliance on dialysis, progressive damage 

significant impact of Soliris on survival. For example, data from 

to vital organs, and the threat of stroke, seizure, heart attack, and 

the South Korean National Registry, presented at the American 

other devastating events. Today, with ongoing Soliris treatment, 

Society of Hematology’s annual conference in December 

patients on dialysis have the chance to become and remain dialysis-

2011, showed that any PNH patient with elevated LDH, a 

free, and patients have the hope of improving and restoring kidney 

measure of hemolysis, is at risk for serious complications due 

function as well as returning to work, school, and their normal lives 

to uncontrolled complement activation, reinforcing the need 

for the first time since their devastating diagnosis. 

for early intervention. These and other independent studies are 

helping physicians make better-informed treatment decisions 

on behalf of their patients with PNH. 

However, despite our substantial progress over the five years 

since Soliris was approved for the treatment of PNH, we 

know that the majority of PNH patients still do not receive 

appropriate care. This is why our focus remains on expanding 

our presence in core territories, serving more patients in 

additional countries, and continuing to build a common, 

global understanding of PNH and its diagnosis and treatment. 

Bringing Life-Transforming Hope to  
Families Battling aHUS

Leveraging Our Expertise for a Successful aHUS Launch

The US and EU approvals of Soliris for the treatment of patients 

with aHUS exemplify our mission of transforming the lives of 

patients with severe and ultra-rare disorders. In both regions, 

we are leveraging the unique expertise we gained from the 

global rollout of Soliris for PNH to bring Soliris therapy to 

an increasing number of patients with aHUS. The US aHUS 

launch has been under way since late 2011, and we have 

begun to serve initial patients across the country. In the EU, 

reimbursement discussions are under way in Western Europe, 

and we plan to launch in major European countries throughout 

2012 and 2013. As with PNH, our key objectives in aHUS are 

to build a common global understanding of the disease among 

In 2007, Soliris began transforming the lives of people living 

physicians and patients, facilitate broad and unrestricted access 

with PNH. In the fall of 2011, patients and families suffering 

to Soliris, and ensure appropriate utilization of Soliris. 

from aHUS gained hope for a similar transformation. 

Our educational efforts are bolstered by the growing body of 

aHUS is a chronic, ultra-rare, and life-threatening disease in 

compelling clinical evidence supporting the use of Soliris in both 

which a genetic deficiency in one or more complement regulatory 

adult and pediatric patients with aHUS. In November, data were 

genes causes lifelong uncontrolled complement activation, 

presented at the American Society of Nephrology (ASN) annual 

resulting in systemic thrombotic microangiopathy (TMA), the 

meeting from the extensions of two pivotal Phase 2 studies: 

formation of blood clots in small blood vessels throughout the 

one in patients with a long duration of disease and substantial 

body. On September 23, 2011, the FDA approved Soliris for the 

organ damage despite previously receiving long-term plasma 

treatment of patients with aHUS – the first-ever approval for an 

exchange/infusion (PE/PI), and one in patients with a shorter 

aHUS treatment and the second indication for Soliris. Shortly 

duration of disease with progressing clinical TMA complications. 

Alexion Pharmaceuticals  Transforming lives through innovation in ultra-rare diseases 

5

Research Pipeline: Lead Programs

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Atypical Hemolytic Uremic Syndrome (aHUS)

Shiga toxin E. coli-related Hemolytic Uremic Syndrome (STEC-HUS)

Presensitized Kidney Transplant (Acute Humoral Rejection)

Severe and Refractory Myasthenia Gravis

Severe and Refractory Neuromyelitis Optica*

Hypophosphatasia

Molybdenum Cofactor Deficiency Type A

Inflammatory Disorders

Inflammatory Disorders

Pipeline Key

  Hematology

Inflammatory Disorders

  Metabolic

  Nephrology

  Neurology

 * 

Investigator-Initiated Trial

P
r
e
c
l
i
n
i
c
a
l
P
h
a
s
e
 
I
P
h
a
s
e
 
I
I
P
h
a
s
e
 
I
I
I
M
a
r
k
e
t
S
o
l
i
r
i
s
fi
 
(
e
c
u
l
i
z
u
m
a
b
)
A
s
f
o
t
a
s
e
 
A
l
f
a
c
P
M
P
 
R
e
p
l
a
c
e
m
e
n
t
 
T
h
e
r
a
p
y
T
T
3
0
A
L
X
N
1
0
0
7
 
Both studies demonstrated that ongoing treatment with Soliris 

without treatment options. Our ongoing development program 

suppressed complement-mediated TMA, maintained or further 

for eculizumab is focused on diseases for which current 

improved longer-term renal function, and enhanced quality of 

supportive approaches are ineffective or nonexistent, where 

life. Additional long-term data from these studies, presented in 

the mechanism of action of the disease is well understood, 

December at the American Society of Hematology (ASH) annual 

and for which eculizumab has the potential to have a life-

meeting, further illustrate the compelling clinical benefits of 

transforming impact. Our lead programs in nephrology and 

Soliris and support early and ongoing treatment.

neurology all share these characteristics. 

Since the 2007 approval of Soliris for the treatment of PNH, 

In nephrology, scientists presented very encouraging interim 

we have had the objective that every patient with PNH who 

data at the ASN meeting in November from our STEC-

can benefit from Soliris will have access to Soliris. Now, we 

HUS clinical trial, which was initiated in response to the 

have expanded that objective – and our long-standing access 

Enterohemorrhagic Escherichia coli (EHEC) crisis in Germany. 

initiatives – to include patients with aHUS. Our OneSource™ 

The interim findings showed that eight weeks of treatment 

Treatment Support program helps patients with both disorders 

with eculizumab substantially improved serious morbidities 

navigate the reimbursement processes in the US, the 

among studied patients with STEC-HUS. Soliris-treated patients 

Complement Foundation provides Soliris at no cost to patients 

experienced rapid, significant, and sustained reduction in TMA, 

who cannot obtain insurance, and patient assistance programs 

as well as reversal of organ damage, underscoring the critical 

are in place through third parties for patients whose insurance 

role of uncontrolled complement activation in the TMA process. 

leaves significant gaps with regard to treatment-related 

Final data are expected this year. 

expenses. Beyond the US, we are working with governments 

in major markets worldwide to ensure that patients with 

aHUS can have access to Soliris therapy. Alexion also supports 

the work of patient organizations in many countries that are 

involved in education and advocacy for rare diseases. 

A Robust Pipeline Focused on Severe  
and Ultra-Rare Disorders

We finished 2011 with the most robust and promising pipeline 

in our Company’s history. Today, our R&D team is investigating 

five highly innovative compounds, including eculizumab, in 

eight severe and ultra-rare diseases beyond PNH and aHUS. 

Our aim is not just to provide incremental benefits but rather 

to dramatically alter the course of severe and ultra-rare 

diseases that have a devastating impact on patients’ lives.

Exploring the Potential of Eculizumab and  

Other Innovative Complement Inhibitors

Also in nephrology, we have recently begun enrolling patients 

in our Alexion-sponsored multinational living-donor trial for 

the prophylaxis of acute humoral rejection (AHR) for patients 

undergoing kidney transplant who are at elevated risk of rejection, 

as measured by the presence of high levels of donor-specific 

antibodies. Patients in the study will be treated with eculizumab 

for nine weeks post-transplant and then observed for a year.

In neurology, we have two clinical development programs under 

way – one in severe and refractory myasthenia gravis, and 

another in patients with severe neuromyelitis optica (NMO). Data 

from our company-sponsored Phase 2 study in myasthenia gravis, 

presented in the fall of 2011 at the Myasthenia Gravis Foundation 

of America meeting, showed a strong disease-improvement 

signal in a group of 14 patients. In NMO, enrollment has been 

completed in an investigator-initiated Phase 2 clinical trial, with 

data expected in the second half of this year. 

We are also expanding our pipeline in complement inhibitors 

As the world’s first and only approved terminal complement 

beyond eculizumab with a Phase 1 development program 

inhibitor, Soliris represents a long-awaited medical 

for TT30, a unique inhibitor of the alternative complement 

breakthrough – not only for patients with PNH and aHUS, but 

pathway. Once we have data from the current Phase 1 study, 

also for patients with numerous other complement-mediated 

we can better evaluate the therapeutic potential of TT30 for 

disorders that are also severe, ultra-rare, life-threatening, and 

various disease targets. 

Alexion Pharmaceuticals  Transforming lives through innovation in ultra-rare diseases 

7

“Alexion enters 2012 with the most robust and innovative pipeline in 
our history, with five highly innovative compounds, including Soliris, 
currently being investigated at various stages of development across 
eight severe and ultra-rare indications beyond PNH and aHUS.”

Stephen P. Squinto, PhD  Executive Vice President and Head of R&D

 
Innovation in Severe and Ultra-Rare Disorders Beyond 

our objectives for this innovative therapy are to advance the 

Eculizumab and Complement

pediatric development program, expand the adult development 

As we expand our development activities, we have sharpened 

program, and optimize commercial-scale manufacturing. We 

our focus on what we know well and do well – using our 

look forward to moving with urgency to bring an approved, life-

proven skills in severe and ultra-rare disorders to develop first-

transforming treatment to patients with HPP and their families. 

in-class, highly innovative therapies. We put this strategy into 

action most recently with the acquisition of Enobia Pharma, a 

company well aligned with our values and areas of focus. The 

acquisition, which closed in February 2012, brings us asfotase 

alfa, a highly innovative, late-stage compound with the 

potential to transform the lives of patients with HPP, a severe, 

ultra-rare, and life-threatening metabolic disorder for which 

there are no approved or effective therapies.

Additionally, we are accelerating the development of a 

highly innovative cPMP replacement therapy as a potential 

treatment for molybdenum cofactor deficiency (MoCD) Type 

A, a devastating, ultra-rare disorder in newborns. MoCD Type A 

is among the rarest and most deadly disorders that can affect 

a newborn, with survival typically measured only in weeks or 

months. There are currently no treatment options for this disease, 

which means children will either die or survive with devastating 

Due to a genetic defect, patients with HPP are deficient in an 

brain damage. Early experience with the cPMP replacement 

enzyme known as tissue non-specific alkaline phosphatase. 

therapy has shown encouraging results, and in 2011 we made 

Without this enzyme, patients can face severe outcomes 

substantial progress in our manufacturing process for the 

including progressive damage to multiple vital organs, destruction 

treatment, allowing us to begin conducting IND-enabling studies.

and deformity of bones, profound muscle weakness, impaired 

renal function, and respiratory failure. Tragically, about one-half 

of newborns with severe HPP do not survive past their first 

birthday, due to a profound bone mineralization defect and 

compromised respiratory function. Older children with HPP may 

not be able to climb a single stair or take a single step. 

By targeting replacement of the missing enzyme directly to 

the necessary tissue, asfotase alfa is designed to normalize the 

defective metabolic process and prevent or reverse the severe and 

life-threatening complications of life-long uncontrolled mineral 

metabolism in patients with HPP. In Phase 2 studies, asfotase alfa 

demonstrated the potential to bring the first real hope to patients 

and families facing this devastating disorder. In a study recently 

published in the New England Journal of Medicine, treatment 

with asfotase alfa led to a striking improvement in skeletal 

abnormalities, pulmonary and physical function, and cognitive 

development in infants and young children with HPP. These and 

other findings in patients of all ages provide strong support for 

the potential of asfotase alfa to transform the lives of patients 

with HPP by correcting the enzyme deficiency that underlies the 

mortality and morbidities of the disease. 

Asfotase alfa was awarded orphan drug designation in the US 

and EU in 2008 and Fast Track status in the US in 2009. In 2012, 

Another key drug candidate in our pipeline is ALXN1007, 

a novel antibody designed to target rare and severe 

inflammatory disorders. ALXN1007 is a product of our 

proprietary antibody discovery technologies. We have 

commenced Phase 1 clinical trials in healthy volunteers.

Continued Strong Financial Performance

In 2011, our world-class research, clinical, regulatory, and 

commercial capabilities delivered sustained high growth and 

positioned us for continued success in 2012. Soliris sales for 

2011 totaled $783 million, representing a 45% increase from 

the previous year and the 19th consecutive quarter of growth 

for our Company. By serving an increasing number of patients 

while maintaining rigorous financial discipline, we achieved 

non-GAAP net income of $266 million, or $1.38 per diluted 

share, a 59% increase from 2010.

We also finished 2011 with cash, cash equivalents, and 

marketable securities totaling $541 million, up from $362 

million in 2010. Importantly, the Enobia acquisition was 

executed in early 2012 with strong financial discipline, using 

cash on hand and bank debt, thus adding a significant late-

stage asset to our pipeline with an investment well within our 

strict financial parameters. 

Alexion Pharmaceuticals  Transforming lives through innovation in ultra-rare diseases 

9

“2011 was a year of strong performance in all of our major global 
initiatives. In 2012 we will reach further, driving forward with the 
same passion and commitment we have demonstrated over the 
years to transform the lives of more patients and families suffering 
with more disorders that are severe, devastating, and ultra-rare.”

Leonard Bell, MD  Chief Executive Officer 

Strengthening Our Human Capital

Throughout 2011, we continued to strengthen and expand 

our world-class capabilities in translational medicine, drug 

development, regulatory affairs, and commercial operations. 

To help guide this growth and success, we added exceptional 

talent to our management team. Clare Carmichael joined as 

Senior Vice President and Chief Human Resources Officer, with 

extensive experience in building cohesive organizations with 

integrated, high-performance teams. Claus Weisemann, PhD 

also joined as Senior Vice President, Corporate Quality and 

Compliance, with responsibility for global quality initiatives 

and regulatory compliance worldwide.

Alexion has more than 1,100 employees in Company facilities 

in more than 20 countries. Our employees are among the best 

and brightest in our industry. We are grateful for their sense 

of urgency and steadfast commitment to serving patients with 

severe, ultra-rare disorders.

Global Citizenship

During 2011, we demonstrated our deep commitment to 

patients following an unusually widespread EHEC outbreak in 

Germany. Physicians faced the daunting challenge of caring 

for a subset of patients who developed STEC-HUS, a severe 

complication caused by uncontrolled complement activation. 

Alexion responded immediately, providing eculizumab at no 

cost to hundreds of patients who had no other treatment 

option. Our medical team worked closely and urgently with 

German health authorities to initiate one of the largest 

In addition, thanks to the dedication of our employees, we 

are involved in a broad range of charitable programs in our 

local communities, including the renovation and restoration of 

homes for people in need, scholarship grants, and fundraising 

for organizations involved in public health initiatives. And in 

2011, we again expanded our commitment to environmental 

sustainability and energy conservation with the installation 

of a micro-turbine cogeneration system at our global 

headquarters in Connecticut – a state-of-the-art technology 

that reduces greenhouse gas emissions by 50% while 

generating both electricity and heat for our labs and offices.

Looking Ahead in 2012

Our progress and performance during 2011 position us for 

even greater achievements on behalf of more patients in 2012. 

We are strengthening our capabilities, expanding our global 

reach, and advancing our deep pipeline of highly innovative, 

life-transforming therapies for patients with severe and 

ultra-rare disorders. We are grateful for those who support 

us in fulfilling this vital mission: our employees, our Board, 

our shareholders, and the patients, families, physicians and 

healthcare systems we serve around the world. Together we 

are reaching further. Together we are transforming lives. 

compassionate access programs for an already-approved drug 

Leonard Bell, MD 

in the developed world. 

Chief Executive Officer

April 2012

From left: Stephen Squinto, PhD, Executive Vice President and Head of Research and Development; Leonard Bell, MD, Chief Executive 

Officer; Patrice Coissac, Senior Vice President and President, Alexion Pharma International Sàrl; Clare Carmichael, Senior Vice President 

and Chief Human Resources Officer; Thomas Dubin, JD, Senior Vice President and Chief Legal Officer; Vikas Sinha, MBA, CA, CPA,  

Senior Vice President and Chief Financial Officer; David Hallal, Senior Vice President, Global Commercial Operations

Alexion Pharmaceuticals  Transforming lives through innovation in ultra-rare diseases 

11

Selected Financial Highlights (In thousands, except per share data)

Net product sales

Cost of sales

$  783,431

93,140

$  540,957

64,437

$  386,800

45,059

Research and development

Selling, general and administrative

Acquisition-related costs

Amortization of purchased intangible assets

Operating income

Other expense

Income before income taxes

Income tax provision (benefit)

137,421

308,176

13,486

382

230,826

1,158

229,668

54,353

98,394

226,766

722

—

150,638

1,627

149,011

51,981

81,915

172,767

—

—

87,059

3,745

83,314

(211,852) 2

Earnings per common share — diluted1

Shares used in computing earnings per share — diluted1

$ 

0.91

191,806

$ 

0.52

186,074

$ 

1.63

181,164

Cash, cash equivalents, and marketable securities

$  540,865

$  361,605

$  176,220

Trade accounts receivable, net

Inventories

Property, plant and equipment, net

Goodwill and intangible assets, net

Deferred tax assets

Other assets

Accounts payable and accrued expenses

Deferred revenue

Contingent consideration

Other liabilities

244,288

81,386

165,852

171,243

123,000

68,117

202,093

17,905

18,120

22,141

168,732

62,165

162,240

44,100

174,212

38,983

123,056

2,896

—

26,349

113,731

40,885

164,691

48,543

211,034

31,297

78,445

1,652

—

17,948

1  On May 20, 2011, we effected a two-for-one stock split, paid in the form of a 100% stock dividend. Stockholders of record at the close of trading on May 2, 2011 were 
issued one additional share of common stock for each share owned by such shareholder. All share and per share data presented in the accompanying table has been 
retroactively restated to reflect the stock split.

2  In 2009, we determined that it was more likely than not that a significant 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 benefit of $215,516 as a result of reversing the valuation allowance on these deferred tax assets.

12 

Alexion Pharmaceuticals  Transforming lives through innovation in ultra-rare diseases

Y
e
a
r
 
E
n
d
e
d
 
D
e
c
e
m
b
e
r
 
3
1
,
 
 
2
0
1
1
 
 
2
0
1
0
 
 
2
0
0
9
 
 
 
 
 
 
 
 
 
O
p
e
r
a
t
i
n
g
 
e
x
p
e
n
s
e
s
:
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
T
o
t
a
l
 
o
p
e
r
a
t
i
n
g
 
e
x
p
e
n
s
e
s
 
 
4
5
9
,
4
6
5
 
 
3
2
5
,
8
8
2
 
 
2
5
4
,
6
8
2
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
N
e
t
 
i
n
c
o
m
e
 
$
 
1
7
5
,
3
1
5
 
$
 
9
7
,
0
3
0
 
$
 
2
9
5
,
1
6
6
 
 
 
 
 
 
 
 
 
A
s
 
o
f
 
D
e
c
e
m
b
e
r
 
3
1
,
 
 
2
0
1
1
 
 
2
0
1
0
 
 
2
0
0
9
C
o
n
s
o
l
i
d
a
t
e
d
 
B
a
l
a
n
c
e
 
S
h
e
e
t
 
D
a
t
a
:
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
T
o
t
a
l
 
a
s
s
e
t
s
 
 
1
,
3
9
4
,
7
5
1
 
 
1
,
0
1
2
,
0
3
7
 
 
7
8
6
,
4
0
1
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
T
o
t
a
l
 
l
i
a
b
i
l
i
t
i
e
s
 
 
2
6
0
,
2
5
9
 
 
1
5
2
,
3
0
1
 
 
9
8
,
0
4
5
T
o
t
a
l
 
s
t
o
c
k
h
o
l
d
e
r
s
(cid:146)
 
e
q
u
i
t
y
 
 
1
,
1
3
4
,
4
9
2
 
 
8
5
9
,
7
3
6
 
 
6
8
8
,
3
5
6
T
o
t
a
l
 
l
i
a
b
i
l
i
t
i
e
s
 
a
n
d
 
s
t
o
c
k
h
o
l
d
e
r
s
(cid:146)
 
e
q
u
i
t
y
 
 
1
,
3
9
4
,
7
5
1
 
 
1
,
0
1
2
,
0
3
7
 
 
7
8
6
,
4
0
1
Our Global Locations

Cheshire, CT, USA
North America Regional and  
Global Headquarters

Barcelona, Spain
Country Operations

Bogotá, Colombia
Country Operations

Brussels, Belgium
Country Operations

Buenos Aires, Argentina
Country Operations

Cambridge, MA, USA
Translational Medicine Group
HPP Program Group

Istanbul, Turkey
Country Operations

Lausanne, Switzerland
EMEA Regional Headquarters
International Operations Center
Country Operations

London, United Kingdom
Country Operations

Mexico City, Mexico
LatAm Regional Headquarters
Country Operations

Milan, Italy
Country Operations

Montréal, Canada
Translational Medicine Group

Moscow, Russia
Country Operations

Mumbai, India
Global Business Services

Munich, Germany
Country Operations

Paris, France
European Service Center
Country Operations

São Paulo, Brazil
Country Operations

Shanghai, China
Commercial Operations

Smithfield, RI, USA
Global Manufacturing

Stockholm, Sweden
Nordic Country Operations

Sydney, Australia
Asia-Pacific Regional Headquarters
Country Operations

Tokyo, Japan
Country Operations

Toronto, Canada
Country Operations

Shareholder Information

Directors

Senior Management 

Annual Shareholders Meeting

To be held on May 7, 2012
5:00 p.m.
Westin Providence Hotel
One West Exchange Street
Providence, RI  02903
tel  401.598.8000
fax 401 598.8200

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

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

Leonard Bell, MD
Chief Executive Officer 

Stephen P. Squinto, PhD
Executive Vice President,  
Head of Research and Development

Clare Carmichael
Senior Vice President, 
Chief Human Resources 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.

Patrice Coissac
Senior Vice President,  
President, Alexion Pharma International Sàrl

Thomas I.H. Dubin, JD
Senior Vice President and Chief Legal Officer

Joseph A. Madri, PhD, MD2,4
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 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

David L. Hallal 
Senior Vice President,  
Global Commercial Operations

Vikas Sinha, MBA, CA, CPA
Senior Vice President and Chief Financial Officer 

Camille L. Bedrosian, MD
Senior Vice President and Chief Medical Officer

Thomas Bock, MD, MBA
Senior Vice President, Global Medical Affairs

M. Stacy Hooks, PhD
Senior Vice President, Technical Operations

Claude Nicaise, MD
Senior Vice President, Strategic  
Development and Global Regulatory 

Claus Weisemann, PhD
Senior Vice President,  
Corporate Quality and Compliance

James P. Bilotta, MBA
Vice President and Chief Information Officer

Daniel N. Caron, MS
Vice President, Site Operations and Engineering 

Sven Ante (Bill) Lundberg, MD
Vice President, Head of Translational Medicine

Margaret M. Olinger, MBA 
Vice President, Global Hematology Franchise 

Jeremy P. Springhorn, PhD
Vice President, Corporate Strategy  
and Business Development 

Jeroen van Beek, PhD
Vice President, Global Nephrology Franchise

Heidi L. Wagner, JD
Vice President, Global Government Affairs

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

© 2012 Alexion Pharmaceuticals, Inc. 
Alexion®, Alexion Logo®, Soliris® and OneSource™  
are trademarks of Alexion Pharmaceuticals, Inc.

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 Mexico S de RL de CV 
Paseo de los Tamarindos 90 Torre 1 Piso 14, Col. Bosques de la Lomas, CP 05120 D.F. Mexico

www.alexionpharma.com