Exhibit 99.1
NEWS
CONTACTS:
Joshua Grass (Investors)
BioMarin Pharmaceutical Inc.
(415) 884-6777
Fredda Malkoff (Media)
Feinstein Kean Healthcare
(617) 577-8110
For Immediate Release:
BioMarin Provides Update on Recent Events
Novato, CA, March 31, 2003 - BioMarin Pharmaceutical Inc. (Nasdaq and Swiss SWX
New Market: BMRN) provided updates on the regulatory status of Aldurazyme, its
VibrilaseTM clinical development program and news related to its recent
follow-on offering.
o The U.S. Food and Drug Administration (FDA) formally acknowledged BioMarin
and Genzyme's joint response to remaining items outlined in the complete
response letter received on January 28, 2003 related to the companies'
marketing application for Aldurazyme. The FDA classified the response as a
complete, class 1 response and intends to take action on the resubmission
by April 30, 2003. Aldurazyme (laronidase) is an investigational enzyme
replacement therapy for treatment of mucopolysaccharidosis I (MPS I).
o BioMarin completed its Phase 1a clinical trial of Vibrilase, a topical
enzyme product candidate for serious burns. Results indicate that Vibrilase
did not cause dermal irritation or sensitization in fewer than eight days,
well beyond the expected treatment period of 1-3 days. BioMarin is
currently enrolling patients in a Phase 1b trial, which it expects to
complete in the middle of 2003.
o The underwriters of BioMarin's recent follow-on offering, UBS Warburg LLC,
CIBC World Markets Corp. and SG Cowen Securities Corporation exercised
their option to purchase the entire over-allotment option of 1,125,000
shares of common stock at $10.00 per share. Separately, BioMarin completed
sales of common stock totaling 500,000 shares at a price of $10.00 per
share to Acqua Wellington North American Fund Equities Ltd. under its
existing equity facility. The gross proceeds from the follow-on offering,
the over-allotment, and the sale of stock through the equity facility
totaled $91.25 million. As a result, BioMarin expects to have more than
$140 million in cash and cash equivalents on March 31, 2003.
Aldurazyme Regulatory Update
The FDA has set April 30, 2003 as the formal action date by which it will
respond to BioMarin and Genzyme's application to market Aldurazyme in the United
States. The FDA has notified the companies that they provided a complete, class
I response to the questions issued by the FDA in its January 28, 2003 complete
response letter. The outstanding questions were related to post-marketing
commitments, final product labeling, and completion of the manufacturing
inspections process.
BioMarin and Genzyme formed a joint venture in 1998 to develop and commercialize
Aldurazyme worldwide. Under the terms of the joint venture, if Aldurazyme is
approved for commercial sale, BioMarin will manufacture the product and Genzyme
will market and commercialize it worldwide. The companies have obtained Orphan
Drug designation from the FDA and Orphan Medicinal Product designation from the
European Union.
Vibrilase Program
Overview
BioMarin is developing Vibrilase as a topically applied enzyme for the
debridement of serious burns. In the United States, approximately 45,000 serious
burns require medical attention each year. In order to avoid infection and
promote healing, the burned skin, or eschar, must be removed. This process is
referred to as debridement. Conventional debridement methods employ surgical
techniques, which are frequently associated with significant pain, blood loss
and scarring, especially in burns covering a large fraction of the body.
Surgical debridement of a partial or full-thickness burn can be costly, up to
$10,000 per procedure. Topical burn debridement agents currently available have
a slow onset of action, and are associated with excessive pain due to their
inability to discriminate between burned and healthy tissue. Vibriolysin, the
active enzyme component in Vibrilase, preferentially digests burned skin,
leaving healthy tissue intact. Vibrilase may meet the medical need for a product
that will rapidly and safely debride partial thickness burns, by potentially
reducing the incidence of infection, improving the outcomes of skin grafts on
burns, and ultimately reducing the time to healing.
Clinical Status
A Phase 1a, randomized, placebo-controlled, double-blind study evaluated the
dermal irritation and sensitization potential of 100, 250, and 500 units per
gram of Vibrilase in 118 healthy subjects at one site in the United Kingdom.
Results indicate that Vibrilase produced no evidence of contact sensitization or
irritation for up to eight days, well beyond the expected 1-3 day treatment
period in which Vibrilase would be applied.
A Phase 1b study was initiated in 2002 at one clinical site in the United
Kingdom. Recently, in order to accelerate patient enrollment in the Phase 1b
study, and to prepare for a Phase 2 trial, one additional site was added in
Scotland, and a third site in the Netherlands is expected begin enrolling
patients in the second quarter of 2003. The Phase 1b study is an open-label,
dose escalation and safety study, evaluating a total of 15 patients with partial
thickness burns, with five patients at three doses; 100, 250, and 500 units per
gram. Based on the trial protocol, patients 18 to 75 years of age with partial
thickness burns over 1-25% of their bodies will receive Vibrilase for three
consecutive days, and will be monitored for 28 days, or until healing occurs.
BioMarin expects to complete the Phase 1b Vibrilase trial in the middle of 2003.
BioMarin Pharmaceutical 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., including, without limitation,
statements about: expectations and actions of regulatory authorities, including
the FDA with respect to actions related to Aldurazyme and European authorities
with respect to Vibrilase; the final results of past clinical trials of
Vibrilase; the expected timing, progress, enrollment and conduct of current and
future trials of Vibrilase and the availability of clinical sites for such
trials; and the financial performance of BioMarin. 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 final analysis of results of past
clinical trials; results and timing of current and future clinical trials;
enrollment rates of current and future clinical trials, the content and timing
of decisions by the FDA, the European Commission and other regulatory
authorities concerning Aldurazyme and Vibrilase; BioMarin's financial
performance during the quarter ending March 31, 2003; and those factors detailed
in BioMarin's filings with the Securities and Exchange Commission, including,
without limitation, the factors contained under the caption "Factors That May
Affect Future Results" in BioMarin's 2002 Annual Report on Form 10-K and the
factors contained in BioMarin's reports on Forms 10Q and 8K. 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's press releases and other company information are available online at
http://www.biomarinpharm.com. Information on our website is not incorporated by
reference into this press release.