Exhibit 99.1
BioMarin Provides Update on NeuroTransTM Brain Delivery Program
Novato, California, May 15th, 2002 - BioMarin Pharmaceutical Inc. (Nasdaq and
Swiss SWX New Market: BMRN) today announced that it has initiated preclinical
studies of NeuroTrans and taken key steps to continue building the technology's
intellectual property position. NeuroTrans is the proprietary brain delivery
technology that BioMarin is developing to treat neurological problems associated
with many lysosomal storage disorders. BioMarin obtained the NeuroTrans platform
through its recently completed acquisition of Synapse Technologies Inc.
Intravenously administered protein therapeutics - including lysosomal enzymes -
have not been shown to penetrate the blood-brain barrier. As a result, treatment
of the central nervous system impairment that is typical of certain lysosomal
storage disorders remains a critical unmet medical need. The NeuroTrans
technology is designed to use a natural human protein (p97) to transport enzymes
and other drugs across the blood-brain barrier and into brain cells to treat
neurological disorders.
Reinhard Gabathuler, Ph.D., BioMarin's Vice President, Brain Research, said,
"Three key steps are required in order for NeuroTrans to serve as a potential
treatment for the neurological effects of lysosomal storage disorders: (1) the
attachment of lysosomal enzymes to p97 molecules; (2) the active transport of
these enzymes across the blood-brain barrier; and (3) the delivery of these
enzymes to the lysosomes of brain cells. Preliminary results from both recent
and ongoing preclinical studies indicate that NeuroTrans may meet each of these
requirements."
Preclinical Studies
Since announcing on January 14, 2002 that it had entered into a definitive
agreement to acquire Synapse, BioMarin has initiated multiple preclinical
studies of NeuroTrans both in-house as well as through strategic brain delivery
collaborations with researchers at Stanford University, the University of
California at Los Angeles, and the University of Quebec at Montreal. The
Company's initial preclinical efforts have focused on three areas:
o Enzyme Attachment: BioMarin is evaluating methods for attaching
lysosomal enzymes to p97, whereby p97 will act as a `carrier' molecule
that pulls the enzyme `payload' across the blood-brain barrier.
o Transport Efficiency: BioMarin is using an in vitro blood-brain barrier
model to measure the rate at which p97-linked lysosomal enzymes are
transported across the blood-brain barrier.
o Biological Uptake: BioMarin is working with normal and lysosomal
storage disorder animal models to assess the biodistribution and
bioactivity of intravenously administered p97-linked lysosomal enzymes
in the brain and in other tissues.
Christopher Starr, Ph.D., BioMarin's Co-Founder and Senior Vice President,
Research and Development, said, "Throughout 2002, BioMarin will continue to
conduct preclinical studies to validate the NeuroTrans technology as a potential
treatment for lysosomal storage disorders and to identify an initial indication
for future clinical studies. The Company's goal for this year is to gather a
substantial set of preclinical efficacy data to prepare for an IND filing."
Intellectual Property
As a part of the Synapse transaction, BioMarin acquired the rights to U.S.
Patent No. 5,981,194, entitled, "Use of p97 and iron binding proteins as
diagnostic and therapeutic agents." In addition, the U.S. Patent and Trademark
Office recently issued BioMarin a notice of allowance for a continuation-in-part
(CIP) application. BioMarin believes this patent creates a strong intellectual
property foundation for NeuroTrans by granting claims for both composition of
matter and methods of delivering therapeutic compounds linked to p97 across the
blood-brain barrier. Furthermore, BioMarin has filed three other patent
applications related to NeuroTrans based on internal research efforts.
Terms of the Acquisition
Under the terms of the agreement, on March 21, 2002, BioMarin acquired 100% of
the outstanding shares of Synapse for approximately US$10.2 million by
exchanging 885,240 shares of BioMarin common stock at a deemed value of US$11.50
per share. BioMarin may also be required to make contingent payments to former
Synapse shareholders in either cash or BioMarin stock of up to approximately
US$6.0 million upon reaching certain development milestones.
Twelve Synapse employees joined BioMarin at the close of the acquisition and now
operate out of BioMarin's Vancouver, British Columbia office, which serves as
the hub of the NeuroTrans program.
BioMarin specializes in the development and commercialization of therapeutic
enzyme products to treat serious, life-threatening diseases and conditions.
This press release contains forward-looking statements about the business
prospects of BioMarin Pharmaceutical Inc., and the acquisition of Synapse
Technologies Inc. These forward-looking statements are predictions and involve
risks and uncertainties such that actual results may differ materially from
these statements. Results may differ materially depending on the progress of
BioMarin's product programs, including the ability to integrate the research
programs being acquired from Synapse, the actual results of the current and
proposed preclinical efforts and current and proposed clinical trials, actions
of regulatory authorities, future availability of capital, future actions in the
pharmaceutical market and developments by competitors, and those factors
detailed in BioMarin's filings with the Securities and Exchange Commission such
as 10Q, 10K and 8K reports. 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.
Contacts:
Jeremy Price
Manager, Investor Relations
BioMarin Pharmaceutical Inc.
(415) 884-6777
jprice@biomarinpharm.com
Sharon Karlsberg
Vice President
Feinstein Kean Healthcare
(617) 577-8110
skarlsberg@fkhealth.com
"Safe Harbor" Statement under the Private Securities Litigation Reform Act of
1995: Statements in this press release regarding BioMarin Pharmaceutical Inc.'s
business which are not historical facts are "forward-looking statements" that
involve risks and uncertainties. For a discussion of such risks and
uncertainties, which could cause actual results to differ from those contained
in the forward-looking statements, see "Risk Factors" in the Company's Annual
Report or Form 10-K for the most recently ended fiscal year.