Molecular Chaperones Target Underlying Defect in Cystic Fibrosis; CFTR Folding and TransportNOVATO, Calif., Aug 22, 2007 /PRNewswire-FirstCall via COMTEX News Network/ -- BioMarin Pharmaceutical
Inc. (Nasdaq and SWX: BMRN) announced today that it has licensed from the
University of California, San Francisco (UCSF) intellectual property covering
compounds demonstrated to improve cystic fibrosis transmembrane conductance
regulator (CFTR) protein functionality. BioMarin expects the lead compounds to
undergo additional animal testing and optimization, with the goal of filing an
IND in 2009.
Emil Kakkis, M.D., Ph.D., Chief Medical Officer of BioMarin commented.
"Dr. Verkman's laboratory has performed pioneering work in the field of CFTR
modulation. The compounds licensed have been developed through a rigorous
high-throughput screening process and have demonstrated a high degree of
potency and specificity for the folding and activation of the abnormal CFTR
protein. We look forward to accelerating the lead optimization activities in
collaboration with Dr. Verkman and his colleagues so that we can take the very
best of these molecules into clinical development."
"Over the last decade, considerable efforts and funding from the Cystic
Fibrosis Foundation and the biotechnology industry have resulted in the
development of therapeutics that have significantly extended and improved the
lives of cystic fibrosis patients," said Dr. Alan Verkman, Professor of
Medicine and Physiology and Director of the Cystic Fibrosis Research
Development Program at the University of California, San Francisco. "Despite
this progress, there is no therapeutic approved that addresses the underlying
molecular defect in cystic fibrosis, and most patients succumb to respiratory
failure as young adults. BioMarin has a strong track record of aggressively
moving disease modifying therapeutics through clinical development, and I am
very pleased that they have made a commitment to further this important work."
"We are pleased to add this program to our growing product development
pipeline. The cystic fibrosis indication aligns well with our existing
product portfolio and core competencies as it is a well-defined and relatively
large orphan disease," said Jean-Jacques Bienaime, Chief Executive Officer of
BioMarin. "It has a clear clinical and regulatory path and will allow us to
leverage our commercial infrastructure targeting specialists."
Cystic fibrosis is an autosomal recessive disease caused by mutations in
the cystic fibrosis transmembrane conductance regulator (CFTR) gene. With an
incidence of approximately 1/3,500 births, cystic fibrosis is the most common,
lethal genetic disease in the U.S. CF affects an estimated 30,000 patients in
the U.S. and approximately 40,000 patients outside the U.S. CFTR is a
transmembrane protein that functions primarily as a chloride channel in the
plasma membrane of epithelial cells. The most common mutation in cystic
fibrosis, the deltaF508 mutation, causes the protein to be misfolded and
through a cascade of events, leads to mucus buildup and ultimately, organ
dysfunction and severe infections in the lungs. The median age of survival of
a CF patient is 36.5 years.
BioMarin develops and commercializes innovative biopharmaceuticals for
serious diseases and medical conditions. The company's product portfolio is
comprised of two 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, and Aldurazyme(R) (laronidase) for
mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a
50/50 joint venture with Genzyme Corporation. Investigational product
candidates include Kuvan(TM) (sapropterin dihydrochloride), a Phase 3 product
candidate for the treatment of phenylketonuria (PKU), and 6R-BH4 for
cardiovascular indications, which is currently in Phase 2 clinical development
for the treatment of peripheral arterial disease and sickle cell disease. For
additional information, please visit http://www.BMRN.com. Information on
BioMarin's website is not incorporated by reference into this press release.
This press release contains forward-looking statements about the business
prospects of BioMarin Pharmaceutical Inc., including, without limitation,
statements about: BioMarin's products and product candidates,
commercialization of BioMarin's products; and actions by regulatory
authorities. 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: our
success in the commercialization of BioMarin's products; the content and
timing of decisions by the U.S. Food and Drug Administration, the European
Commission and other regulatory authorities concerning each of the described
products and product candidates; 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 2006 Annual Report on Form 10-K, as amended, and the factors
contained in BioMarin's reports on Form 10-Q and Form 8-K. Stockholders are
urged not to place undue reliance on forward-looking statements, 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) and Naglazyme(R) are a registered trademarks of BioMarin
Aldurazyme(R) is a registered trademark of BioMarin/Genzyme LLC.
Orapred(R) is a registered trademark of Medicis Pediatrics, Inc. and is
used under license.
Eugenia Shen Susan Berg
BioMarin Pharmaceutical Inc. BioMarin Pharmaceutical Inc.
(415) 506-6570 (415) 506-6594
SOURCE BioMarin Pharmaceutical Inc.
investors, Eugenia Shen, +1-415-506-6570, or media, Susan Berg, +1-415-506-6594, both
of BioMarin Pharmaceutical Inc.