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BioMarin Initiates Phase 1/2 Clinical Trial for GALNS for Morquio A Syndrome NOVATO, Calif., April 21, 2009 /PRNewswire-FirstCall
via COMTEX/ -- BioMarin
Pharmaceutical Inc. (Nasdaq: BMRN)
announced today the initiation of a
Phase
1/2 clinical trial for BMN-110 or N-acetylgalactosamine
6-sulfatase (GALNS),
intended for the treatment of the
lysosomal storage disorder
ucopolysaccharidosis Type IVA (MPS
IVA), or Morquio A Syndrome. The
company expects to report initial
results in the first half of 2010.
"We plan to leverage our clinical,
manufacturing and regulatory
expertise to
bring a new therapeutic option to
the significant number of untreated
Morquio patients around the world,"
said Henry Fuchs, M.D., Chief
Medical Officer of BioMarin. "GALNS
has been shown in mice to reach
important tissues including
cartilage and different zones of the
bone such as bone marrow, calcified
bone and importantly, the growth
plate. Our experiments have also
shown that GALNS is taken up into
human Morquio chondrocytes in vitro
and reaches the lysosome to clear
keratan sulfate."
Chris Hendriksz, M.D., Consultant in
Metabolic Disorders, Birmingham
Children's Hospital, added, "This is
a very exciting announcement for our
patients who have been waiting so
long for a potential therapy. Hope
of a therapeutic is now within reach
for these patients, and Birmingham
Children's Hospital is very honoured
to be part of this exciting
development."
The Phase 1/2 study is designed as
an open-label, within-patient dose
escalation trial in approximately 20
patients followed by a treatment
continuation phase. All patients to
be enrolled in the study have
already been identified. During the
dose escalation phase of the study,
subjects will receive weekly
intravenous infusions of BMN-110 in
three consecutive 12-week dosing
intervals. The objectives of the
Phase 1/2 study will be to evaluate
safety, pharmacokinetics,
pharmacodynamics and to identify the
optimal dose of GALNS for future
studies.
BioMarin has developed and
manufactures two FDA-approved enzyme
replacement therapies, one for the
treatment of MPS I and one for the
treatment of MPS VI. Naglazyme(R) (galsulfase)
for MPS VI is wholly developed and
commercialized by BioMarin.
Aldurazyme(R) (laronidase) for MPS I
is
manufactured by BioMarin and
marketed by Genzyme Corporation.
About MPS IVA
Mucopolysaccharidosis IVA (MPS IVA,
also known as Morquio A Syndrome) is
a
disorder characterized by deficient
activity of N-acetylgalactosamine
6-sulfatase (GALNS) causing
excessive lysosomal storage of
keratan sulfate (KS). This excessive
storage causes a systemic skeletal
dysplasia, short stature, and joint
abnormalities, which limit mobility
and endurance. Malformation of the
thorax as well as macrophage
dysfunction in the lung likely
impairs respiratory function and
contributes to sinopulmonary
infections. Odontoid dysplasia and
ligamentous laxity can commonly
cause cervical spinal instability
and potentially spinal cord
compression. Other symptoms may
include recurrent infections,
hearing loss, corneal clouding,and
heart valvular disease. Initial
symptoms often become vident in the
first five years of life. Depending
on severity of the disorder, age of
diagnosis will vary. Many patients
become wheelchair dependent in their
second decade of life and undergo
numerous surgeries to alleviate
life-threatening conditions caused
by the underlying enzyme deficiency.
The incidence estimates for MPS IVA
vary widely, between one in 200,000
livee
births to one in 300,000 live
births. Approximately 400 patients
worldwide have been identified and
tracked through the International
Morquio Organization (IMO) survey.
There are already more MPS IVA
patients identified through this
registry than there are MPS VI
patients being treated with
Naglazyme worldwide. Based on the
number of identified patients to
date, the prevalence of patients
with MPS IVA appears similar to that
with MPS I.
About BioMarin
BioMarin develops and commercializes
innovative biopharmaceuticals for
serious diseases and medical
conditions. The company's product
portfolio
comprises three approved products
and multiple clinical and
preclinical
product candidates. Approved
products include Naglazyme(R) (galsulfase)
for
mucopolysaccharidosis VI (MPS VI), a
product wholly developed and
commercialized by BioMarin;
Aldurazyme(R) (laronidase) for
ucopolysaccharidosis I (MPS I), a
product which was developed through
a 50/50 joint venture with Genzyme
Corporation; and Kuvan(R) (sapropterin
dihydrochloride) Tablets, a product
for the treatment of phenylketonuria
(PKU), developed in partnership with
Merck Serono, a division of Merck
KGaA of Darmstadt, Germany. Other
product candidates include PEG-PAL (PEGylated
recombinant phenylalanine ammonia
lyase), which is currently in Phase
1 clinical development for the
treatment of PKU. For additional
information, please visit
www.BMRN.com. Information on
BioMarin's website is not
incorporated by reference into this
press release.
Forward-Looking Statement
This press release contains
forward-looking statements about the
business
prospects of BioMarin Pharmaceutical
Inc., including, without limitation,
statements about: the development of
its program for MPS IVA, and
particularly the timing and conduct
of clinical trials related thereto,
and expectations regarding filings
with regulatory agencies. These
forward-looking statements are
predictions and involve risks and
uncertainties such that actual
results may differ materially from
these statements. These risks and
uncertainties include, among others:
the results of current and planned
pre-clinical trials related to the
enzyme replacement therapy for MPS
IVA; the content and timing of
decisions by the U.S. Food and Drug
Administration, EMEA and other
regulatory agencies, particularly
with respect to the enzyme
replacement therapy for MPS IVA, and
those factors detailed in BioMarin's
filings with the Securities and
Exchange Commission, including,
without limitation, the factors
contained under the caption "Risk
Factors" in BioMarin's 2008 Annual
Report on Form 10-K. Stockholders
are urged not to place undue
reliance on forward-looking
tatements, which speak only as of
the date hereof. BioMarin is under
no obligation, and expressly
disclaims any obligation to update
or alter any forward-looking
statement, whether as a result of
new information, future events or
otherwise.
BioMarin(R), Naglazyme(R) and
Kuvan(R) are registered trademarks
of BioMarin
Pharmaceutical Inc.
Aldurazyme(R) is a registered
trademark of BioMarin/Genzyme LLC.
Contacts:
Investors Media
Eugenia Shen Susan Berg
BioMarin Pharmaceutical Inc.
BioMarin Pharmaceutical Inc.
(415) 506-6570 (415) 506-6594
SOURCE BioMarin Pharmaceutical Inc. |
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