Alexion
Pharmaceuticals, Inc. (Nasdaq: ALXN) today announced results from three
analyses of Phase III studies examining Soliris(TM) (eculizumab) for the
treatment of paroxysmal nocturnal hemoglobinuria (PNH) at the 12th Congress
of European Hematology, organized by The European Hematology Association
(EHA) in Vienna. These presentations included:
"The Terminal Complement Inhibitor Eculizumab Reduces Thrombosis in
Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)," presented by Dr.
Peter Hillmen of the General Infirmary at the University of Leeds, Leeds,
UK.(1) (Abstract No. 0380)
"Safety and Efficacy of the Terminal Complement Inhibitor Eculizumab in
Patients with Paroxysmal Nocturnal Hemoglobinuria: SHEPHERD Phase III
Clinical Study Results," presented by Dr. Hubert Schrezenmeier of the
University Hospital Ulm in Ulm, Germany.(2) (Abstract No. 0378)
"The Clinical Benefit of Eculizumab Is Demonstrable in all
Subpopulations of Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)
with Hemolysis," presented by Dr. Petra Muus of Radboud University,
Nijmegen, Netherlands.(3) (Abstract No. 0379) Subpopulations were diverse
and included patients with poor bone marrow function, smaller PNH clone
sizes, low levels of hemolysis, less anemia, minimal pre-treatment
transfusion requirements and those receiving steroids or erythropoietin.
Dr. Muus noted, "In published clinical trials, Soliris significantly
reduced hemolysis in all PNH patients, which led to an improvement in
anemia, patient functioning and quality of life, and fewer thrombotic
events. Research presented this week at the Congress in Vienna described
the impact of Soliris on multiple subgroups of PNH patients and showed that
the effect of Soliris was maintained across all subpopulations including in
those patients who had as few as only one or no transfusions in the year
prior to Soliris treatment."
For the full abstracts of these presentations, please visit the
European Hematology Association Web site at
congress.ehaweb/12th.
About PNH
PNH is an acquired genetic blood disorder defined by hemolysis, in
which patients' red blood cells are destroyed by complement, a component of
the body's immune system. Hemolysis can cause one or more of the following
symptoms in patients with PNH: severe anemia, disabling fatigue, recurrent
pain, shortness of breath, pulmonary hypertension, intermittent episodes of
dark colored urine (hemoglobinuria), kidney disease, impaired quality of
life and blood clots (thromboses).(4)(5)
PNH affects an estimated 8,000 to 10,000 people in North America and
Europe.(6) PNH often strikes people in the prime of their lives, with an
average age of onset in the early 30's.(7) Ten percent of all patients
first develop symptoms at 21 years of age or younger.(5) PNH develops
without warning and can occur in men and women of all races, backgrounds
and ages. PNH often goes unrecognized, with delays in diagnosis often
ranging from one to more than 10 years.(8) The estimated median survival
for PNH patients is between 10 and 15 years from the time of
diagnosis.(6)(8)
PNH has been identified more commonly among patients with disorders of
the bone marrow, including aplastic anemia (AA) and myelodysplastic
syndrome (MDS).(9)(10)(11)(12) In patients with thrombosis of unknown
origin, PNH may be an underlying cause.(5)(13)
There are currently no therapies available in Europe specifically for
the treatment of PNH. PNH treatment has been limited to symptom management
through periodic blood transfusions, non-specific immunosuppressive therapy
and, infrequently, bone marrow transplantations - a procedure that carries
considerable mortality risk.(5)(13)
About Soliris
Soliris was approved in March by the U.S. Food and Drug Administration
(FDA) as the first treatment for PNH, a rare, debilitating and life-
threatening blood disorder defined by hemolysis, or the destruction of red
blood cells. In April the Committee for Human Medicinal Products (CHMP) of
the European Medicines Agency (EMEA) adopted a positive opinion
recommending marketing authorization for Soliris for the treatment of all
patients with PNH. Final EU approval is expected in June or July.
Important Safety Information
Soliris is generally well tolerated. The most frequent adverse events
observed in clinical studies were headache, nasopharyngitis (a runny nose),
back pain and nausea.(14) 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. Vaccinate patients
with a meningococcal vaccine at least 2 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." 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 will be enrolled in the Soliris Safety 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.
Please see full prescribing information at soliris.
About Alexion
Alexion Pharmaceuticals is a biotechnology company working to develop
and deliver life-changing drug therapies for patients with serious and
life- threatening medical conditions. Alexion's lead product, Soliris(TM)
(eculizumab), is indicated for the treatment of paroxysmal nocturnal
hemoglobinuria (PNH). Alexion is engaged in the discovery and development
of therapeutic products aimed at treating patients with severe disease
states, including hematologic diseases, cancer and autoimmune disorders,
and in May 2007, received a corporate leadership award from the National
Organization of Rare Disorders (NORD) for the development of Soliris.
Alexion applied for marketing authorization with the European Medicines
Evaluation Agency (EMEA) for Soliris in September 2006, and in April, 2007
the Committee for Human Medicinal Products of the EMEA adopted a positive
opinion recommending marketing authorization for Soliris for the treatment
of PNH. This press release and further information about Alexion
Pharmaceuticals, Inc. can be found at: alexionpharm.
This news release contains forward-looking statements, including
statements related to potential benefits and commercial potential for
Soliris, timing for, and potential regulatory decisions with respect to,
the marketing applications for Soliris in Europe, and interest and
excitement about Soliris in the physician community. Forward-looking
statements are subject to factors that may cause Alexion's results and
plans to differ from those expected, including for example, requests by
regulatory authorities for additional information or data after their
review of our submissions, the need for additional research and testing,
decision of regulatory authorities not to approve (or to materially limit)
marketing of Soliris in Europe or other territories, delays in arranging
satisfactory manufacturing capability and establishing commercial
infrastructure, delays in developing or adverse changes in commercial
relationships, the possibility that results of clinical trials are not
predictive of safety and efficacy results of Soliris in broader patient
populations, the risk that third parties won't agree to license any
necessary intellectual property to us on reasonable terms, the risk that
third party payors will not reimburse for the use of Soliris at acceptable
rates or at all, the risk that Soliris will not generate interest among
physicians, the risk that estimates regarding the number of PNH patients
are inaccurate, the risk that pending litigation may be resolved adversely,
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 Quarterly Report on Form 10-Q
for the period ended March 31, 2007 and in our 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 Hillmen P. The Terminal Complement Inhibitor Eculizumab Reduces
Thrombosis in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH).
Study accepted for presentation at the 12th Congress of European
Hematology, organized by The European Hematology Association (EHA), on
Saturday, June 9 in Vienna, Austria.
2 Schrezenmeier H. Safety and Efficacy of the Terminal Complement
Inhibitor Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria
(PNH): SHEPHERD Phase III Clinical Study Results. Study accepted for
presentation at the 12th Congress of European Hematology, organized by The
European Hematology Association (EHA), on Saturday, June 9 in Vienna,
Austria.
3 Muus P. The Clinical Benefit of Eculizumab is Demonstrable in All
Subpopulations of Patients with Paroxysmal Nocturnal Hemoglobinuira PNH
with Hemolysis. Study accepted for oral presentation at the 12th Congress
of European Hematology, organized by The European Hematology Association
(EHA), on Saturday, June 9 in Vienna, Austria.
4 Rother R, Bell L, Hillmen P, Gladwin M. The clinical sequelae of
intravascular hemolysis and extracellular plasma hemoglobin. JAMA 2005;
293:1653-1662.
5 Parker C, Omine M, Richards S, et al. Diagnosis and management of
paroxysmal nocturnal hemoglobinuria. Blood 2005; 106:3699-3709.
6 Hill A, Platts PJ, Smith A et al. The incidence and prevalence of
paroxysmal nocturnal hemoglobinuria (PNH) and survival of patients in
Yorkshire [abstract]. Blood. 2006;108(11):290a. Abstract 985.
7 Socie G, Mary J Yves, de Gramont A, et al. Paroxysmal nocturnal
haemoglobinuria: long-term follow-up and prognostic factors. Lancet 1996;
348:573-577.
8 Hillmen P. Lewis SM, Bessler M, Luzzatto L, Dacie JV. Natural history
of paroxysmal nocturnal hemoglobinuria. N Engl J Med 1995; 333:1253-1258.
9 Johnson RJ, Hillmen P. Paroxysmal nocturnal hemoglobinemia: Nature's
gene therapy? J Clin Path: Mol Pathol. 2002;55:145-152.
10 Wang, et al. Clinical significance of a minor population of
paroxysmal nocturnal hemoglobinuria-type cells in bone marrow failure
syndrome. Blood 2002;100:3897-3902.
11 Iwanga, et al. Paroxysmal nocturnal haemoglobinuria clones in
patients with myelodysplastic syndromes. Brit J Haem. 1998;102:465-474.
12 Maciejewski, et al. Relationship between bone marrow failure syndromes and the presence of glycophosphatidyl inositol-anchored protein-deficient clones. Brit J Haem. 2001;115:1015-1022
13 Hill A, Richards S, Hillmen P. Recent developments in the
understanding and management of paroxysmal nocturnal haemoglobinuria.
British Journal of Haematology 2007; 137:3, 181-192.
14 Soliris(TM) (eculizumab) prescribing information. Alexion
Pharmaceuticals, Inc., 2006.
Alexion Pharmaceuticals, Inc.
alexionpharm
View drug information on Soliris.
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