Substantial Disease Burden of PNH Demonstrated in Data from International and Asian Patient Registries Presented at European Hematology Association
CHESHIRE, Conn., Jun 14, 2010 (BUSINESS WIRE) -- Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN) today announced new
research evaluating the substantial disease burden of paroxysmal
nocturnal hemoglobinuria (PNH), an ultra-rare blood disorder, in
patients worldwide. The International PNH Registry, involving 580
patients from 99 sites in 14 countries as of May 2010, found that the
debilitating symptoms and life-threatening complications of PNH are
similar across patient populations around the world. Data from a
separate Asian patient registry, which included 286 patients with PNH in
South Korea, examined the clinical manifestations of PNH and concluded
that thrombosis is a strong predictor of mortality in these patients.
The findings of these patient registries support the need for treatment
with Soliris(R) (eculizumab), a first-in-class terminal
complement inhibitor, to improve the prognosis and quality of life for
patients living with PNH. The data were presented at the 15th
Congress of the European Hematology Association held in Barcelona, Spain
on June 10-13, 2010.
"These data quantify the severe and consistent impact of PNH on patients
around the world and make clear the specific risks faced by patients in
Asia," said Leonard Bell, M.D., Chief Executive Officer of Alexion. "The
upcoming launch of Soliris in Japan represents our first major expansion
into the Asia-Pacific region, where we will continue our disease
education and access initiatives to provide the life-transforming
benefits of Soliris to patients in a growing number of countries."
As previously announced, Alexion has accelerated plans for the launch of
Soliris in Japan based on recent approval of pricing and reimbursement
for Soliris by an advisory committee of Japan's Ministry of Health,
Labour and Welfare (MHLW). The Company now expects to serve initial and
increasing numbers of patients with PNH in Japan in the third and fourth
quarters of 2010. In April 2010, Japan's MHLW approved Soliris as a
treatment for patients with PNH, making it the first therapy approved in
Japan for this patient population. Soliris is also approved in the
United States, European Union, Australia, Canada and South Korea as a
treatment for patients with PNH.
Global Disease Burden of PNH
Results from the International PNH Registry, which has enrolled 580
patients from 99 clinical sites in 14 countries as of May 2010, showed
that a history of thrombotic events (TE) was present in patients across
all clone sizes but was significantly higher in patients with larger
clones. Patients with PNH clone greater-than or equal to 50% were more likely to have a history
of TE (20%) compared to patients with smaller PNH clones (p=0.01),
however the risk of a TE is still substantially higher in patients with
PNH clones <10% (4%) than expected in a normal population. (1) The
registry, which continues to evaluate disease burden and determine the
long-term natural history of PNH and treatment outcomes, also showed:
-
Abdominal pain, chest pain, shortness of breath, and fatigue are
prevalent symptoms in PNH patients.
-
These patient-reported symptoms were equally prevalent across all PNH
clone sizes and independent of history of bone marrow disorder.
-
Transfusions are the most common treatment (55%), followed by
anti-coagulants (36%), Soliris (34%) and immunosuppressive therapies
(25%). As Alexion introduces Soliris in additional markets worldwide,
sites participating in the registry are updating the information
collected about specific treatments.
These data were presented by Dr. Alvaro Urbano-Ispizua and colleagues on
Saturday, June 12 from 17:30-18:45 in Hall 6 in a poster presentation
entitled, "Evaluation of Paroxysmal Nocturnal Hemoglobinuria Disease
Burden in Patients Enrolled in the International PNH Registry" (Abstract
#1022).
Fatigue and Impaired Quality of Life in Japanese Patients with PNH
Burden of disease data from the AEGIS study, a 12-week, open-label Phase
II study of Soliris in 29 Japanese patients with PNH, was also presented
at the EHA meeting. The data demonstrate that hemolysis, drives the
risks and burden of disease in PNH independent of anemia. In this study,
the primary endpoint of hemolysis reduction was achieved with high
statistical significance with Soliris treatment. In addition, inhibition
of terminal complement activation with Soliris also improved fatigue,
dyspnea (difficulty breathing) and other significant morbidities of
disease - also independent of anemia - in Japanese patients with PNH.
The data were presented by Dr. Yuzuru Kanakura and colleagues on
Saturday, June 12 from 17:30-18:45 in Hall 6 in a poster presentation
entitled, "Fatigue and Impaired Quality of Life in Patients with
Paroxysmal Nocturnal Hemoglobinuria (PNH) is Associated with Hemolysis,
But Not With Anemia" (Abstract #1042).
Clinical Manifestations and Disease Burden in Asian Patients with PNH
Results from a national data registry in South Korea retrospectively
examined 286 PNH patients and demonstrated that Asian patients with PNH
suffer similar disabling symptoms to those seen in other populations,
including thrombosis, late stage kidney disease, liver dysfunction and
symptoms of pulmonary hypertension. TE are a strong predictor of
mortality in Asian patients (p<0.0001). Other findings include:
-
Abdominal pain is a significant predictor of both early mortality and
TE in Asian patients (p=0.046 and 0.0004, respectively).
-
Prominent symptoms of PNH such as hemoglobinuria (presence of
hemoglobin in the urine), dyspnea and chest pain are also significant
predictors of TE (p=0.015, 0.046 and 0.024, respectively).
-
The risk of TE is high across all granulocyte clone sizes, and
presence of concomitant bone marrow dysfunction does not diminish the
risk of TE.
-
Despite medical intervention with supportive care (78% of patients
used corticosteroids), patients continued to show disabling symptoms,
progressive complications and early mortality.
The data were presented by Dr. Lee Jong Wook and colleagues on Friday,
June 11 from 17:45-19:00 in Hall 6 in two poster presentations entitled,
"Clinical Symptoms of Hemolysis Are Predictive of Disease Burden and
Mortality in Asian Patients with Paroxysmal Nocturnal Hemoglobinuria"
(Abstract # 0506) and "High Prevalence and Mortality Associated with
Thromboembolism in Asian Patients with Paroxysmal Nocturnal
Hemoglobinuria (PNH)" (Abstract #0505).
About PNH
PNH is a rare blood disorder that strikes people of all ages, with an
average age of onset in the early 30s. (2) Approximately 10 percent of
all patients first develop symptoms at 21 years of age or younger. (3)
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 ranging from one to more than 10 years. (4) It is estimated
that approximately one-third of patients with PNH do not survive more
than five years from the time of diagnosis. (4) PNH has been identified
more commonly among patients with disorders of the bone marrow,
including aplastic anemia (AA) and myelodysplastic syndromes (MDS).
(5,6,7) In patients with thrombosis of unknown origin, PNH may be an
underlying cause. (2) More information on PNH is available at www.pnhsource.com.
About Soliris
Soliris (eculizumab) is a first-in-class terminal complement inhibitor
developed from the laboratory through regulatory approval by Alexion.
Soliris has been approved by the healthcare authorities in the U.S.,
European Union, Japan and other countries as the first treatment for
patients with PNH, a rare, debilitating and life-threatening blood
disorder defined by hemolysis, or the destruction of red blood cells.
Prior to these approvals, there was no therapy specifically available
for the treatment of PNH.
Patients with PNH in more than 20 countries now have access to Soliris
therapy through national or private healthcare providers. As the first
terminal complement inhibitor to be approved in countries around the
world, Soliris represents a long-sought breakthrough in medical
innovation. 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.
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 potential health and medical benefits from
Soliris, and the timing of regulatory and commercial milestones for
Soliris in Japan. 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,
delays in arranging satisfactory manufacturing capability and
establishing commercial infrastructure, delays in developing or adverse
changes in commercial relationships, 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-Q for the period
ended March 31, 2010, 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) Fowkes, F.J., Price, J.F. & Fowkes, F.G. Incidence of diagnosed deep
vein thrombosis in the general population: systematic review. European
Journal of Vascular and Endovascular Surgery. 2003: 25, 1-5.
(2) Socié G, Mary J Yves, de Gramont A, et al. Paroxysmal nocturnal
haemoglobinuria: long-term follow-up and prognostic factors. Lancet.
1996: 348:573-577.
(3) Parker C, Omine M, Richards S, et al. Diagnosis and management of
paroxysmal nocturnal hemoglobinuria. Blood. 2005;106 (12):3699-3709.
(4) Hillmen P, Lewis SM, Bessler M, Luzzatto L, Dacie JV. Natural
history of paroxysmal nocturnal hemoglobinuria. N Engl J Med. 1995;
333:1253-1258.
(5) Wang H, Chuhjo T, Yasue S, Omine M, Naka S. Clinical significance of
a minor population of paroxysmal nocturnal hemoglobinuria-type cells in
bone marrow failure syndrome. Blood. 2002;100 (12):3897-3902.
(6) Iwanga M, Furukawa K, Amenomori T, et al. Paroxysmal nocturnal
haemoglobinuria clones in patients with myelodysplastic syndromes. Br J
Haematol. 1998;102 (2):465-474.
(7) Maciejewski JP, Risitano AM, Sloand EM, et al. Relationship between
bone marrow failure syndromes and the presence of glycophosphatidyl
inositol-anchored protein-deficient clones. Br J Haematol.
2001;115:1015-1022.
SOURCE: Alexion Pharmaceuticals, Inc.
Alexion Pharmaceuticals, Inc.
Irving Adler, 203-271-8210
Sr. Director Corporate Communications
or
Makovsky & Company
(Media)
Mark Marmur, 212-508-9670
or
Rx Communications
(Investors)
Rhonda Chiger, 917-322-2569
Copyright Business Wire 2010