|
February 03, 2004 08:30 AM US
Eastern Timezone
Xechem Files for FDA
Orphan Drug Status for the Treatment of Muscular
Dystrophy with MYODUR; MYODUR Recently Acquired Through
Ceptor Merger
NEW
BRUNSWICK, N.J.--(BUSINESS WIRE)--Feb. 3, 2004--Xechem
International (OTC BB: XKEM) today announced that it has
applied for Orphan Drug status for MYODUR(TM).
Developed by Ceptor, a wholly owned subsidiary of
Xechem, MYODUR, Ceptor's lead product, has successfully
demonstrated, in animal trials, safety and efficacy in
Muscular Dystrophy. MYODUR includes carnitine, a
membrane transport carrier molecule that targets
skeletal muscle, and its passenger molecule, leupeptin,
a known calpain inhibitor. Calpain is the primary
protease that degrades skeletal muscle. Because calpain
is up regulated in muscular dystrophy, the Company
believes the inhibiting effect of leupeptin along with
the efficient targeting effect of carnitine makes MYODUR
an ideal candidate for this orphan disease.
The Company estimates that muscular dystrophy
represents a $1 billion+ market opportunity. Ceptor has
previously received Orphan Drug Status from the FDA for
the use of leupeptin, as a therapeutic treatment for
nerve injuries. Because both carnatine and leupeptin are
known compounds to the FDA, and because much larger
doses of leupeptin alone have been studied in humans,
Xechem believes this is not a high risk development
program.
Under the Orphan Drug Act, companies that develop
drugs that meet Orphan Drug status have certain benefits
that are not available otherwise, including:
-- Market Exclusivity - Government legislation
protects and rewards companies for the development of
orphan drugs by providing for seven years of market
exclusivity in the U.S. and 10 years in the EU, creating
a competition free environment with that technology.
-- Regulatory - Also, because of the Orphan Drug
Legislation, the regulatory challenges for product
approval may be less daunting. This means fewer total
patient exposures, possibly, a fewer number of clinical
trials and acceptance of surrogate markers along with
clinical outcomes. Also, the FDA is mandated to review
an orphan drug approval application (NDA or BLA) in six
months (fast track), instead of a year or two. To be
adept at thoroughly understanding the orphan legislation
and designing clinical trials for orphan drugs provides
efficiencies across many different diseases.
Dr. Alfred Stracher, Distinguished Professor and
Chairman of Biochemistry at Downstate Medical Center in
Brooklyn, New York, and head of Ceptor commented, "We
are pleased that our expectations that we would be
filing for Orphan Drug Status for MYODUR in 2004 have
been met so early in the year. Ceptor's platform
technology allows specific membrane transporters and
their molecular passengers, at very low doses, to be
deposited specifically and effectively within skeletal
and cardiac muscle cells in the case of MYODUR. Due to a
genetic defect, muscular dystrophy patients produce
insufficient dystrophin, which normally provides
skeletal cell membrane integrity. With the lack of this
cell membrane integrity, calcium ions seep into the
cells and up regulate calpain, leading to the
degradation of muscle. Ceptor is working on other cell
targeting molecules that appear to be equally as
efficacious. We are pleased that Xechem has taken the
key first steps to commercializing our technology and
expect that our association as a wholly-owned subsidiary
of Xechem will result in the rapid development of our
basic technology."
William Pursley, Vice-Chairman and President of
Xechem, noted, "This is just the first of many benefits
that we expect will accrue from our acquisition of
Ceptor. Muscular dystrophy patients suffer from an over
abundance of calpain and this devastating disease
usually ends in death in adolescence. The Company
believes that these patients can be managed definitively
with the proper, targeted inhibition of calpain using
MYODUR."
Mr. Pursley concluded, "Unfortunately, many deadly
diseases, taken individually, impact a relatively small
number of people and, therefore, are not given the
research attention that they deserve. Xechem's focus is
centered on implementing an orphan disease strategy that
can efficiently develop and commercialize drugs that can
address both the patients suffering from these diseases
and establish Xechem as a leader in this marketplace.
Applying for orphan drug status for MYODUR is another
step in achieving these goals."
Xechem International, Inc. is a fully integrated
biopharmaceutical company focusing on proprietary
technologies for orphan diseases. Its mission is to
increase the quality and quantity of life of the people
who suffer from these diseases.
This news release contains certain forward-looking
statements within the meaning of Section 27A of the
Securities Act of 1933, as amended, and Section 21E of
the Securities Exchange Act of 1934, as amended, which
are intended to be covered by safe harbors created
hereby. Such forward-looking statements involve known
and unknown risks, uncertainties, including the ability
of the Companies to successfully develop and
commercialize their technologies, and other factors that
may cause the actual results, performance or
achievements of the Companies to be materially different
from any future results, performance or achievements of
the Companies expressed or implied by such
forward-looking statements. |
|