Exhibit 99.1
Contacts:
Joshua A. Grass Susan Ferris
Manager, Investor Relations Manager, Corporate Communications
BioMarin Pharmaceutical Inc. BioMarin Pharmaceutical Inc.
(415) 506-6777 (415) 506-6701
For Immediate Release:
BioMarin Halts Phase 3a Study of Neutralase in CABG and
Terminates the Neutralase Drug Development Program
BioMarin Spending Projected to Decrease in Both 2003 and 2004
Conference Call at 9:00 AM ET (15:00 CET)
Novato, CA, September 22, 2003 - BioMarin Pharmaceutical Inc. (Nasdaq and SWX:
BMRN) announced that it halted its Phase 3a study of NeutralaseTM for the
reversal of anticoagulation by heparin in primary Coronary Artery Bypass Graft
(CABG) surgery and that it has terminated its Neutralase program for all
indications.
The decision to halt the Phase 3 study resulted from a recommendation from an
independent Data Safety Monitoring Board (DSMB) and was based on a review of
data from enrolled patients, which indicated with high probability that
Neutralase would not demonstrate favorable safety and efficacy. Given the
expected risk/benefit profile for Neutralase, BioMarin has decided to stop
development of the drug for all indications.
As a result of the planned reductions in spending related to the discontinuation
of Neutralase development, BioMarin has lowered its projected net loss by $5
million for 2003 and by $13 million for 2004. Revised estimates for both net
loss and cash burn for 2003 and 2004 are included below, as are previous
forecasts for purposes of comparability:
Changes to 2003 and 2004 Forecast ($ in millions)
2003 2004
---- ----
Forecast Current Previous Current Previous
Net Loss $76-$78 $81-$83 $68-$70 $81-$83
Cash Burn $74-$78 $80-$84 $69-$73 $80-$84
As of June 30, 2003, BioMarin had approximately $265 million in cash and cash
equivalents.
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Fredric D. Price, Chairman and Chief Executive Officer of BioMarin stated,
"While we are disappointed that Neutralase will not become a successful product,
it is better to have learned this relatively early in its development at
BioMarin. We have planned BioMarin's product portfolio carefully, and have
adequate resources for our other promising product opportunities, including
AryplaseTM, which is in a pivotal Phase 3 trial."
BioMarin's portfolio includes product candidates for genetic diseases, including
Aryplase for mucopolysaccharidosis VI that is in a Phase 3 clinical trial
expected to be completed at the end of the first quarter of 2004, and a product
candidate for Phenylketonuria (PKU) that the company expects to advance into
clinical-stage development in 2004. VibrilaseTM, an enzyme product candidate for
treatment of serious burns, is nearing completion of a Phase 1b study, and
NeuroTransTM is in preclinical testing as a method to deliver both enzymes and
other therapeutic molecules to the brain via traditional intravenous infusion.
Aldurazyme(R) for mucopolysaccharidosis I is approved in both the United States
and European Union. Aldurazyme is manufactured by BioMarin and is marketed by
BioMarin's joint venture partner, Genzyme Corporation.
BioMarin will host a conference call and webcast to discuss the decision to
terminate Neutralase development today at 9:00 AM ET (15:00 CET). This event can
be accessed on the BioMarin website at: http://investor.biomarinpharm.com.
Date: September 22, 2003
Time: 9:00 AM ET (15:00 CET)
U.S. & Canada Toll-free Dial-in #: 1-888-391-0090 International Dial-in #:
415-537-1988 Replay Toll-free Dial-in #:1-800-633-8625 Replay International
Dial-in #: 402-977-9141
Replay Code #: 21161394
BioMarin develops and commercializes therapeutic enzyme products to treat
serious, life-threatening diseases and conditions.
BioMarin defines cash burn as net cash flow for a fiscal year (determined in
accordance with generally accepted accounting principles) less all proceeds from
capital markets financing activities. BioMarin's proceeds from all capital
markets financing activities in its fiscal year 2003 through the date of this
release are $209.4 million.
Forward-Looking Statement
This press release contains forward-looking statements about the business
prospects of BioMarin Pharmaceutical Inc., including, without limitation,
statements about: the net loss and cash burn of BioMarin for fiscal years 2003
and 2004, the cessation of clinical trials of Neutralase and the cessation of
the development program for Neutralase for all indications, resources for
product candidates, results of clinical trials of Aryplase as well as trials for
a product for PKU and for Vibrilase, and expected timing and progress of current
and future trials. 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
actual results of
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operations of BioMarin for its fiscal years 2003 and 2004, including without
limitation actual revenues and expenses and cash flows; the final analysis of
results of past clinical trials; results and timing of current and future
clinical trials; the content and timing of decisions by regulatory authorities;
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
10-Q and 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's press releases and other company information are available online at
http://www.BMRN.com. Information on BioMarin's website is not incorporated by
reference into this press release.
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