Researchers to Present Clinical Trial Data With Soliris® (eculizumab) Treatment in Both Approved Indications – PNH and aHUS

  Researchers to Present Clinical Trial Data With Soliris® (eculizumab)
  Treatment in Both Approved Indications – PNH and aHUS – at ASH Annual
  Meeting

55th Annual Meeting of the American Society of Hematology

Business Wire

CHESHIRE, Conn. -- November 7, 2013

Alexion Pharmaceuticals, Inc. (Nasdaq:ALXN) today announced that researchers
will present data from clinical studies of Soliris^® (eculizumab) as a
treatment for patients with paroxysmal nocturnal hemoglobinuria (PNH) and
atypical hemolytic uremic syndrome (aHUS), two life-threatening and ultra-rare
diseases caused by chronic uncontrolled complement activation, at the 55th
Annual Meeting of the American Society of Hematology (ASH). Soliris is the
first and only approved treatment for PNH and aHUS. Abstracts summarizing
these data are published on the ASH website and can be accessed using the
links below. The ASH annual meeting will be held December 7-10, 2013, at the
Ernest N. Morial Convention Center in New Orleans.

Soliris was first approved in 2007 and is now approved in nearly 50 countries
as a treatment for patients with PNH, a debilitating, ultra-rare and
life-threatening blood disorder characterized by complement-mediated hemolysis
(destruction of red blood cells). Soliris is also approved in the United
States (2011), European Union (2011), Japan (2013) and other countries as a
treatment for patients with aHUS, a genetic, chronic and ultra-rare disease
associated with vital organ failure and premature death.

Soliris and PNH

The following abstract will be presented in a poster session on Saturday,
December 7, 2013 from 5:30 – 7:30 p.m., Central Standard Time (CST):

Abstract 1241: “Baseline Assessment of GPI-anchored Protein Deficient Blood
Cells in Patients with Bone Marrow Failure (The OPTIMA study),” Obara, et al.

Accessible at: https://ash.confex.com/ash/2013/webprogram/Paper63472.html

The following abstracts will be presented in a poster session on Monday,
December 9, 2013 from 6:00 – 8:00 p.m., Central Standard Time (CST):

Abstract 3715: “Periodic Evaluation of the Clone Size is Mandatory in PNH: A
Study of the Spanish Cohort of the International PNH Registry,” Villegas, et
al.

Accessible at: https://ash.confex.com/ash/2013/webprogram/Paper60116.html

Abstract 3720: “Clinical Signs and Symptoms in Non-transfused Patients with
Paroxysmal Nocturnal Hemoglobinuria from a Korean Prospective PNH Registry,”
Lee, et al.

Accessible at: https://ash.confex.com/ash/2013/webprogram/Paper61567.html

Soliris and aHUS

The following abstracts will be presented in a poster session on Sunday,
December 8, 2013 from 6:30 - 8:30 p.m., Central Standard Time (CST):

Abstract 2184: “Biomarkers of Complement and Endothelial Activation,
Inflammation, Thrombosis, and Renal Injury in Patients with Atypical Hemolytic
Uremic Syndrome (aHUS) Treated with Eculizumab,” Cofiell, et al.

Accessible at:https://ash.confex.com/ash/2013/webprogram/Paper63810.html

Abstract 2191: “Eculizumab Inhibits Thrombotic Microangiopathy (TMA) and
Improves Renal Function in Pediatric Patients with Atypical Hemolytic Uremic
Syndrome (aHUS),” Greenbaum, et al.

Accessible at:https://ash.confex.com/ash/2013/webprogram/Paper61643.html

Abstract 2179: “Eculizumab Inhibits Thrombotic Microangiopathy (TMA) and
Improves Renal Function in Adult Patients with Atypical Hemolytic Uremic
Syndrome (aHUS),” Fakhouri, et al.

Accessible at:https://ash.confex.com/ash/2013/webprogram/Paper61803.html

Abstract 2186: “Time to Hematologic and Renal Improvements in Atypical
Hemolytic Uremic Syndrome Patients with Long Disease Duration and Chronic
Kidney Disease (CKD) Treated with Eculizumab,” Licht, et al.

Accessible at:https://ash.confex.com/ash/2013/webprogram/Paper62059.html

The following abstract will be presented in a poster session on Sunday,
December 9, 2013 from 6:00 - 8:00 p.m., Central Standard Time (CST):

Abstract 3426: “Time to Hematologic and Renal Improvements in aHUS Patients
with Progressing Thrombotic Microangiopathy Treated with Eculizumab Over Two
Years,” Muus, et al.

Accessible at:https://ash.confex.com/ash/2013/webprogram/Paper60646.html

About Soliris

Soliris is a first-in-class terminal complement inhibitor developed from the
laboratory through regulatory approval and commercialization by Alexion.
Soliris is approved in the US (2007), European Union (2007), Japan (2010) and
other countries as the first and only treatment for patients with paroxysmal
nocturnal hemoglobinuria (PNH), a debilitating, ultra-rare and
life-threatening blood disorder, characterized by complement-mediated
hemolysis (destruction of red blood cells).

Soliris is also approved in the US (2011), the European Union (2011), Japan
(2013) and other countries as the first and only treatment for patients with
atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated
thrombotic microangiopathy, a debilitating, ultra-rare and life-threatening
genetic disorder characterized by complement-mediated thrombotic
microangiopathy (blood clots in small vessels). The effectiveness of Soliris
in aHUS is based on the effects on thrombotic microangiopathy (TMA) and renal
function. Prospective clinical trials in additional patients are ongoing to
confirm the benefit of Soliris in patients with aHUS. Soliris is not indicated
for the treatment of patients with Shiga-toxin E. coli related hemolytic
uremic syndrome (STEC-HUS).

Alexion's breakthrough approach in complement inhibition has received the
pharmaceutical industry's highest honors: the 2008 Prix Galien USA Award for
Best Biotechnology Product with broad implications for future biomedical
research and the 2009 Prix Galien France Award in the category of Drugs for
Rare Diseases.

Important Safety Information

The US product label for Soliris includes a boxed warning: "Life-threatening
and fatal meningococcal infections have occurred in patients treated with
Soliris. Meningococcal infection may become rapidly life-threatening or fatal
if not recognized and treated early. Comply with the most current Advisory
Committee on Immunization Practices (ACIP) recommendations for meningococcal
vaccination in patients with complement deficiencies. Immunize patients with a
meningococcal vaccine at least 2 weeks prior to administering the first dose
of Soliris, unless the risks of delaying Soliris therapy outweigh the risk of
developing a meningococcal infection. (See Serious Meningococcal Infections
(5.1) for additional guidance on the management of meningococcal infection.)
Monitor patients for early signs of meningococcal infections and evaluate
immediately if infection is suspected. Soliris is available only through a
restricted program under a Risk Evaluation and Mitigation Strategy (REMS).
Under the Soliris REMS, prescribers must enroll in the program. Enrollment in
the Soliris REMS program and additional information are available by
telephone: 1-888-soliris (1-888-765-4747)."

In patients with PNH, the most frequently reported adverse events observed
with Soliris treatment in clinical studies were headache, nasopharyngitis
(runny nose), back pain and nausea. Soliris treatment of patients with PNH
should not alter anticoagulant management because the effect of withdrawal of
anticoagulant therapy during Soliris treatment has not been established. In
patients with aHUS, the most frequently reported adverse events observed with
Soliris treatment in clinical studies were hypertension, upper respiratory
tract infection, diarrhea, headache, anemia, vomiting, nausea, urinary tract
infection, and leukopenia. Please see full prescribing information for
Soliris, including boxed WARNING regarding risk of serious meningococcal
infection.

About Alexion

Alexion Pharmaceuticals, Inc. is a biopharmaceutical company focused on
serving patients with severe and ultra-rare disorders through the innovation,
development and commercialization of life-transforming therapeutic products.
Alexion is the global leader in complement inhibition, and has developed and
markets Soliris^® (eculizumab) as a treatment for patients with PNH and aHUS,
two debilitating, ultra-rare and life-threatening disorders caused by chronic
uncontrolled complement activation. Soliris is currently approved in nearly 50
countries for the treatment of PNH, and in the United States, Europe, Japan
and other countries for the treatment of aHUS. Alexion is evaluating other
potential indications for Soliris and is pursuing development of four other
innovative biotechnology product candidates which are being investigated
across additional severe and ultra-rare disorders beyond PNH and aHUS. This
press release and further information about Alexion Pharmaceuticals, Inc. can
be found at www.alexionpharma.com.

[ALXN-G]

Contact:

Alexion Pharmaceuticals, Inc.
Irving Adler, 203-271-8210
Executive Director, Corporate Communications
or
Kim Diamond, 203-439-9600
Senior Director, Corporate Communications
or
Investors:
Rx Communications
Rhonda Chiger, 917-322-2569
 
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