Alexion Completes Enrollment in All Four Clinical Trials of Soliris® (eculizumab) in Patients with Atypical Hemolytic Uremic Syndrome (aHUS)
Trials Include Adult and Adolescent Patients with aHUS
CHESHIRE, Conn., Apr 20, 2010 (BUSINESS WIRE) -- Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN) today announced that it has
completed enrolling patients in all four previously announced
prospective, open-label clinical studies investigating Soliris(R)
(eculizumab) as a potential treatment for patients with atypical
Hemolytic Uremic Syndrome (aHUS) being conducted in North America and
multiple European countries. The four studies have achieved the
Company's enrollment targets, with a total of approximately 35 adult and
adolescent patients with aHUS.
About the Studies
All patients in the studies had evidence of thrombotic microangiopathy
prior to enrollment. The studies include patients who had been treated
chronically with plasma therapy and others who were resistant to plasma
therapy. For patients who have been treated chronically with plasma
therapy, patients were screened for an initial 8 week period prior to
initiating treatment with Soliris. Once enrolled, patients in the trials
are treated with Soliris for a period of 26 weeks. All patients have now
commenced treatment.
"Given the potential for rapid and life-threatening deterioration
observed in patients with aHUS, and the inability to predict sudden
worsening in an individual, we are increasingly focused on diligently
advancing our development efforts. We look forward to presenting
preliminary results from these studies later in 2010," said Leonard
Bell, M.D., Chief Executive Officer of Alexion. "In addition, because a
significant number of patients with aHUS are children, we are committed
to expanding our aHUS program to include studies of Soliris in pediatric
patients with aHUS."
Alexion is working with the regulatory authorities in the U.S. and
European Union to finalize protocols for studies of Soliris in patients
younger than 12 years of age. The Company expects to begin a trial in
pediatric aHUS patients approximately mid-year.
About Soliris
Soliris is not approved for the treatment of patients with aHUS and is
being provided to patients in clinical studies on an investigational
basis. Soliris is a first-in-class terminal complement inhibitor
developed from the laboratory through regulatory approval by Alexion.
Soliris has been approved in the U.S., European Union and other
countries as the first treatment for patients with paroxysmal nocturnal
hemoglobinuria (PNH), an ultra-rare, debilitating and life-threatening
blood disorder defined by chronic hemolysis, or the destruction of red
blood cells. Prior to these approvals, there were no therapies
specifically available for the treatment of patients with PNH. Alexion's
innovative approach to complement inhibition has received some of 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. More information on Soliris is
available at www.soliris.net.
About aHUS
Atypical hemolytic-uremic syndrome (aHUS) is a rare and severe genetic
disorder characterized by sudden clinical deterioration -
life-threatening blood clots throughout the body leading to renal
failure, thrombocytopenia, and hemolytic anemia. These clinical
abnormalities are the hallmark of thrombotic microangiopathy.
Patients with aHUS experience poor outcomes, including dialysis, kidney
failure and death, often occurring within the first year following
diagnosis. As in PNH, aHUS is caused by a deficiency in normally
occurring complement inhibitor proteins. Typically, patients with aHUS
have genetic mutations in one of several complement inhibitor proteins
that lead to uncontrolled complement activation. Excessive complement
activation may contribute to severe inflammation of the blood vessels
and blood clotting through the activation of white blood cells,
platelets, and the endothelial cell lining of blood vessels. (1)
The prognosis for patients with aHUS is generally poor. Approximately 70
percent of patients with the most common mutation experience renal
failure, dialysis, or death within one year of the first clinical
episode. (2, 3) Following kidney transplantation, recurrent aHUS causes
kidney failure in up to 60 to 90 percent of patients. (4)
Important Safety Information
Soliris is generally well tolerated in patients with PNH. The most
frequent adverse events observed in clinical studies of patients with
PNH were headache, nasopharyngitis (runny nose), back pain and nausea.
Treatment with Soliris should not alter anticoagulant management because
the effect of withdrawal of anticoagulant therapy during Soliris
treatment has not been established.
The U.S. product label for Soliris also includes a boxed warning:
"Soliris increases the risk of meningococcal infections. Meningococcal
infection may become rapidly life-threatening or fatal if not recognized
and treated early. Vaccinate patients with a meningococcal vaccine at
least two weeks prior to receiving the first dose of Soliris;
revaccinate according to current medical guidelines for vaccine use.
Monitor patients for early signs of meningococcal infections, evaluate
immediately if infection is suspected, and treat with antibiotics if
necessary." During PNH clinical studies, two out of 196 vaccinated PNH
patients treated with Soliris experienced a serious meningococcal
infection. Prior to beginning Soliris therapy, all patients and their
prescribing physicians are encouraged to enroll in the PNH Registry,
which is part of a special risk-management program that involves initial
and continuing education and long-term monitoring for detection of new
safety findings.
About Alexion
Alexion Pharmaceuticals, Inc. is a biopharmaceutical company working to
develop and deliver life-changing drug therapies for patients with
serious and life-threatening medical conditions. Alexion is engaged in
the discovery, development and commercialization of therapeutic products
aimed at treating patients with a wide array of severe disease states,
including hematologic and kidney diseases, transplant, other
inflammatory disorders, and cancer. Soliris is Alexion's first marketed
product. Alexion is evaluating other potential indications for Soliris
as well as other formulations of eculizumab for additional clinical
indications, and is pursuing development of other antibody product
candidates in early stages of development. This press release and
further information about Alexion Pharmaceuticals, Inc. can be found at: www.alexionpharma.com.
[ALXN-G]
Safe Harbor Statement
This news release contains forward-looking statements, including
statements related to anticipated clinical development milestones and
potential health and medical benefits of Soliris (eculizumab) for the
potential treatment of patients with atypical hemolytic uremic syndrome
(aHUS). Forward-looking statements are subject to factors that may cause
Alexion's results and plans to differ from those expected, including for
example, decisions of regulatory authorities regarding marketing
approval or material limitations on the marketing of Soliris for its
current or potential new indications, delays in arranging satisfactory
manufacturing capability and establishing commercial infrastructure, the
possibility that results of published reports or clinical trials are not
predictive of safety and efficacy results of Soliris in broader patient
populations, the risk that clinical trials may not be completed
successfully, the possibility that initial results of commercialization
are not predictive of future rates of adoption of Soliris, the risk that
third parties won't agree to license any necessary intellectual property
to Alexion on reasonable terms or at all, the risk that third party
payors will not reimburse for the use of Soliris at acceptable rates or
at all, and a variety of other risks set forth from time to time in
Alexion's filings with the Securities and Exchange Commission, including
but not limited to the risks discussed in Alexion's Annual Report on
Form 10-K for the period ended December 31, 2009, and in Alexion's other
filings with the Securities and Exchange Commission. Alexion does not
intend to update any of these forward-looking statements to reflect
events or circumstances after the date hereof, except when a duty arises
under law.
References
(1) Ståhl A, Vaziri-Sani F, Heinen S, Kristoffersson A-C, Gydell K-H,
Raafat R, Gutierrez A, Beringer O, Zipfel PF, and Karpman D. Factor H
dysfunction in patients with atypical hemolytic uremic syndrome
contributes to complement deposition on platelets and their activation.
Blood. 2008;111:5307-5315.
(2) Caprioli J, Noris M, Brioschi S, Pianetti G, Castelletti F,
Bettinaglio P, et al. Genetics of HUS: the impact of MCP, CFH, and IF
mutations on clinical presentation, response to treatment, and outcome.
Blood. 2006 Aug 15;108(4):1267-79).
(3) Noris M and Remuzzi G. Atypical Hemolytic-Uremic Syndrome. N Engl J
Med 2009;361:1676-87.
(4) Loirat C, Noris M, Fremeaux-Bacchi V. Complement and the atypical
hemolytic uremic syndrome in children. Pediatr Nephrol. 2008
Nov;23(11):1957-72.
SOURCE: Alexion Pharmaceuticals, Inc.
Alexion Pharmaceuticals, Inc.
Irving Adler, 203-271-8210
Sr. Director Corporate Communications
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