Metabolic Replacement Therapy Designed to Prevent Severe Brain
Damage, Early Mortality in Infants with Molybdenum Cofactor Deficiency
(MoCD) Type A
CHESHIRE, Conn.--(BUSINESS WIRE)--
Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN) announced that it has
purchased patents and assets from Germany-based Orphatec Pharmaceuticals
GmbH related to an investigational therapy for patients with molybdenum
cofactor deficiency (MoCD) Type A, a devastating ultra-rare genetic
disorder characterized by severe brain damage and rapid death in
newborns. Orphatec is a privately held development-stage biotechnology
company with headquarters in Cologne, Germany. In addition, Alexion has
established a research collaboration with key MoCD researchers from
Orphatec to accelerate development of the investigational therapy.
About MoCD Type A
MoCD Type A is an ultra-rare metabolic disease affecting newborns in
which a genetic deficiency of cPMP causes a deficiency of molybdenum
cofactor which in turn leads to catastrophic brain damage, with survival
generally measured in weeks or months. Deficiency of the cofactor leads
to accumulation of neurotoxic sulfite, resulting in uncontrollable
seizures, severe and rapid neurological damage, and death. There are
currently no treatment options for patients with MoCD Type A.
About the cPMP Replacement Therapy
The investigational therapy is designed to replace the deficient cPMP,
which enables MoCD production so that the infant's body can eliminate
the toxic sulfite. Scientific discoveries underlying this highly
innovative therapy were pioneered in Germany, and have led to
encouraging early clinical experience with cPMP replacement therapy in
several newborns. Investigators in Germany and Australia have reported
clinically meaningful results in the first patient treated. (1)
"An essential part of Alexion's mission is to employ our skills in drug
development and delivery on behalf of patients with ultra-rare and
severe disorders who have no other hope. Our goal with this acquisition
is to provide a first-in-class, life-transforming treatment for newborns
and their families devastated by MoCD Type A," said Leonard Bell, M.D.,
Chief Executive Officer of Alexion. "While development of a therapy for
this type of very rare neonatal disorder involves significant commitment
and risk, we are dedicated to driving forward expeditiously to
investigate the potential of this innovative metabolic therapy."
"We appreciate Alexion's commitment to infants with MoCD Type A who
currently have no chance of survival," said Guenter Schwarz, Ph.D.,
Professor and Chair in Biochemistry, Department of Chemistry & Center
for Molecular Medicine Cologne, Germany, and a leader of the original
Orphatec MoCD team. "Alexion's proven expertise in developing drugs for
patients suffering from ultra-rare disorders now brings meaningful hope
that the work begun by the Orphatec team can result in the first-ever
approved treatment for these most helpless of patients and their
families."
The assets were purchased with an upfront cash payment of approximately
$3 million plus contingent payments which would be earned upon reaching
various development, regulatory, and commercial milestones.
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, neurologic disorders,
ophthalmic, transplant, other inflammatory disorders, and cancer.
Soliris® (eculizumab), Alexion's first marketed product, is approved in
more than 35 countries as a therapy for patients with PNH, a
debilitating and ultra-rare life-threatening blood disorder. Alexion is
evaluating other potential indications for Soliris and is pursuing
development of other innovative biotechnology 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 benefits from the acquired cPMP
replacement therapy assets. Forward-looking statements are
subject to factors that may cause Alexion's results and plans to differ
from those expected, including for example those development,
manufacturing, regulatory, commercialization and 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 September
30, 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. Veldman A, et al. Successful treatment of molybdenum cofactor
deficiency type A with cPMP Pediatrics. 2010 May;125(5):e1249-54.
Alexion Pharmaceuticals, Inc.
Irving Adler, 203-271-8210
Senior
Director, Corporate Communications and Public Policy
or
Media:
Makovsky
& Company
Mark Marmur, 609-354-8135
or
Investors:
Rx
Communications
Rhonda Chiger, 917-322-2569
Source: Alexion Pharmaceuticals, Inc.
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