EXHIBIT 99.1
NEWS
Contacts:
Jeremy Price Sharon Karlsberg
Manager, Investor Relations Vice President
BioMarin Pharmaceutical Inc. Feinstein Kean Healthcare
(415) 884-6777 (617) 577-8110
jprice@biomarinpharm.com skarlsberg@fkhealth.com
For Immediate Release:
BioMarin Initiates Phase 2 Trial of AryplaseTM
Enzyme Replacement Therapy for MPS VI
Novato, CA, April 22nd, 2002 - BioMarin Pharmaceutical Inc. (Nasdaq and Swiss
SWX New Market: BMRN) today announced that it began dosing patients last month
in a Phase 2 clinical trial of Aryplase (recombinant human N-acetylgalactosamine
4-sulfatase or arylsulfatase B), an investigational enzyme replacement therapy
for the treatment of mucopolysaccharidosis VI (MPS VI). MPS VI, also known as
Maroteaux-Lamy syndrome, is a life-threatening lysosomal storage disorder for
which no specific drug treatments currently exist. In September 2001, BioMarin
reported positive safety and enzyme activity data from the Company's Phase 1
clinical trial of Aryplase.
The primary objective of this open-label, multi-national Phase 2 clinical trial
will be to evaluate the efficacy, safety and pharmacokinetics of weekly
intravenous infusions of 1.0 mg/kg of Aryplase in 10 MPS VI patients. This dose
represents the higher level of two doses administered in the six-patient Phase 1
trial.
Stuart Swiedler, M.D., Ph.D., BioMarin's Vice President, Clinical Affairs, said,
"Our goal is to assemble a comprehensive set of clinical data that will lead to
the timely advancement of an effective treatment for this debilitating and
currently untreatable genetic disease. We look forward to reporting results from
this trial in the first quarter of 2003. Subsequently, if data from the Phase 2
trial demonstrate positive safety and efficacy outcomes, we anticipate the
initiation of a pivotal Phase 3 trial."
John Jost, Ph.D., BioMarin's Vice President, Manufacturing, added, "Clinical
materials for the Phase 2 trial were produced in our recently upgraded clinical
manufacturing facility, which will also produce the materials for the Phase 3
clinical program. Key modifications to the Aryplase manufacturing process have
resulted in an approximately five-fold increase in plant productivity."
BioMarin has received orphan drug and fast track designations for Aryplase for
the treatment of MPS VI from the U.S. Food and Drug Administration, and the
European Commission has designated Aryplase for the treatment of MPS VI as an
orphan medicinal product.
Aryplase Phase 1 Results
In September 2001, BioMarin reported results from a randomized, double-blind
six-patient Phase 1 clinical trial of Aryplase. The primary objective of this
trial was to evaluate the safety of Aryplase at two dose levels: 0.2 mg/kg and
1.0 mg/kg. The enzyme was well tolerated by all six patients during the initial
24-week treatment stage, and key findings included:
o No treatment-related serious adverse events;
o No significant allergic reactions to the enzyme infusions;
o Urinary glycosaminoglycan (GAG) excretion was reduced by a mean of
70% and 55% in the high and low dose groups, respectively;
o Reduced urinary GAG excretion was evident within three weeks of
initiating treatment.
The reduction in urinary GAGs indicates that Aryplase is breaking down the
complex carbohydrate materials that otherwise accumulate in patients with MPS VI
and lead to the debilitating and life-threatening symptoms of the disease.
About MPS VI
MPS VI is a life-threatening lysosomal storage disorder caused by a deficiency
of the enzyme N-acetylgalactosamine 4-sulfatase (also known as arylsulfatase B).
This deficiency leads to the accumulation of GAGs in the lysosomes, the
digestive organs of the cell. The accumulation of GAGs in the lysosomes leads to
progressive cellular, tissue and organ system dysfunction. Debilitating symptoms
often include impaired cardiac and pulmonary function, delayed physical
development, skeletal and joint deformities, impaired vision and hearing, sleep
disorders, and reduced endurance. Depending on the severity of the disease,
patients die from disease-related complications between childhood and early
adulthood.
BioMarin Pharmaceutical Inc. specializes in the development and
commercialization of therapeutic enzyme products to treat serious,
life-threatening diseases and other conditions.
This press release contains forward-looking statements about the business
prospects of BioMarin Pharmaceutical Inc., including the prospects of Aryplase.
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, the data to be released when the ongoing Aryplase
trial is unblinded, the results of current and proposed clinical trials, actions
of regulatory authorities, 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.
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