Researchers to Present New Data on Soliris® (eculizumab) as a Treatment for Patients with aHUS at ASN Annual Meeting

  Researchers to Present New Data on Soliris® (eculizumab) as a Treatment for
  Patients with aHUS at ASN Annual Meeting

Business Wire

CHESHIRE, Conn. -- October 16, 2013

Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN) today announced that researchers
are scheduled to present data from clinical studies of Soliris^® (eculizumab)
in patients with atypical hemolytic uremic syndrome (aHUS), a genetic,
life-long, ultra-rare disease associated with vital organ failure and
premature death. Data will be presented at the annual meeting of the American
Society of Nephrology (ASN), being held from November 5 –10, 2013 in Atlanta,
Georgia. Presentations will include:

  *Results from a prospective clinical trial of Soliris in pediatric patients
    with aHUS, following the published results of a retrospective study in
    pediatric patients with aHUS
  *Results from an expanded adult population, representing the largest
    prospective trial of Soliris in adult patients with aHUS
  *Three-year follow-up data from two pivotal Phase 2 extension studies that
    highlight the long-term benefits of Soliris therapy in patients with aHUS

Abstracts summarizing these presentations were published today on the ASN
website and can be accessed using the links below.

Soliris is approved in the United States, European Union, Japan and other
countries as a treatment for patients with aHUS. Soliris is also approved in
nearly 50 countries as a 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). Both aHUS and PNH are life-threatening, ultra-rare diseases that
are caused by chronic uncontrolled complement activation.

Soliris and aHUS

The following abstract will be presented in an oral session on Friday,
November 8, 2013 from 4:30 – 6:30 p.m. Eastern Standard Time (EST):

Abstract [FR-OR057]: “Eculizumab Inhibits Thrombotic Microangiopathy (TMA) and
Improves Renal Function in Adult Atypical Hemolytic Uremic Syndrome (aHUS)
Patients,” Fakhouri et al.

Accessible at:
http://www.abstracts2view.com/asn_2013/view.php?nu=5593&terms=&type=abstract

The following abstracts will be presented in a poster session on Saturday,
November 9, 2013 from 10:00 a.m. – 12:00 p.m., Eastern Standard Time (EST):

Abstract [SA-PO852]: “Eculizumab Maintains Efficacy in Atypical Hemolytic
Uremic Syndrome (aHUS) Patients with Progressing Thrombotic Microangiopathy
(TMA): 3-Year Update,” Gaber, et al.

Accessible at:
http://www.abstracts2view.com/asn_2013/view.php?nu=5311&terms=&type=abstract

Abstract [SA-PO850]: “Eculizumab in Atypical Hemolytic Uremic Syndrome (aHUS)
Patients with Long Disease Duration and Chronic Kidney Disease (CKD):
Sustained Efficacy at 3 Years,” Delmas, et al.

Accessible at:
http://www.abstracts2view.com/asn_2013/view.php?nu=5506&terms=&type=abstract

Abstract [SA-PO849]: “Eculizumab Inhibits Thrombotic Microangiopathy (TMA) and
Improves Renal Function in Pediatric Atypical Hemolytic Uremic Syndrome (aHUS)
Patients,” Greenbaum, et al.

Accessible at:
http://www.abstracts2view.com/asn_2013/view.php?nu=5579&terms=&type=abstract

Abstract [SA-PO853]: “An Observational, Non-Interventional, Multicenter,
Multinational Registry of Patients with Atypical Hemolytic Uremic Syndrome
(aHUS): Initial Patient Characteristics,” Licht, et al.

Accessible at:
http://www.abstracts2view.com/asn_2013/view.php?nu=5184&terms=&type=abstract

About Soliris

Soliris is a first-in-class terminal complement inhibitor developed from the
laboratory through regulatory approval and commercialization by Alexion.
Soliris is also approved in the US, European Union, Japan 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, the European Union, Japan 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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