Results of First Interim Efficacy Analysis for Riquent Phase 3 ASPEN Trial: Continuation of the Trial is Futile

Friday, February 13, 2009 General News
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NOVATO, Calif., Feb. 12 BioMarin PharmaceuticalInc. (Nasdaq: BMRN) announced today that in the first interim efficacyanalysis (IEA) for the Riquent(R) Phase 3 ASPEN trial, the Independent DataMonitoring Board (DMB) determined that the continuation of the trial isfutile. BioMarin and partner La Jolla Pharmaceutical have decided to stop thestudy, unblind the data and evaluate all of the clinical results includingsecondary endpoints such as SLE (Systemic Lupus Erythematosus) diseaseactivity indices and proteinuria.

"While the results of this first interim efficacy analysis are obviouslydisappointing, we decided to take a calculated risk on this program and withthe carefully staged agreement, we have spent a total of $15 million, arelatively modest amount for a late Phase 3 asset addressing a large marketopportunity," said Jean-Jacques Bienaime, Chief Executive Officer of BioMarin."We remain dedicated to advancing our development pipeline by makinginvestments in future growth and continue to look for attractive late-stagein-licensing or acquisition opportunities."

About BioMarin

BioMarin develops and commercializes innovative biopharmaceuticals forserious diseases and medical conditions. The company's product portfoliocomprises three approved products and multiple clinical and preclinicalproduct candidates. Approved products include Naglazyme(R) (galsulfase) formucopolysaccharidosis VI (MPS VI), a product wholly developed andcommercialized by BioMarin; Aldurazyme(R) (laronidase) formucopolysaccharidosis I (MPS I), a product which was developed through a 50/50joint venture with Genzyme Corporation; and Kuvan(R) (sapropterindihydrochloride) Tablets, a product for the treatment of phenylketonuria(PKU), developed in partnership with Merck Serono, a division of Merck KGaA ofDarmstadt, Germany. Other product candidates include 6R-BH4 for cardiovascularindications, which is currently in Phase 2 clinical development for thetreatment of sickle cell disease, and PEG-PAL (PEGylated recombinantphenylalanine ammonia lyase), which is currently in Phase 1 clinicaldevelopment for the treatment of PKU. For additional information, please visit Information on BioMarin's website is not incorporated byreference into this press release.

Forward-Looking Statement

This press release contains forward-looking statements about the businessprospects of BioMarin Pharmaceutical Inc., including, without limitation,statements about: expectations related to the clinical trials and continueddevelopment of abetimus sodium, also known as Riquent. These forward-lookingstatements are predictions and involve risks and uncertainties such thatactual results may differ materially from these statements. These risks anduncertainties include, among others: results and timing of current and plannedpreclinical studies and clinical trials, including the ASPEN study; theenrollment rate of such study and the actual rate of renal flares in suchstudy; the content and timing of decisions by the U.S. Food and DrugAdministration, the European Commission regarding abetimus sodium; La JollaPharmaceutical's ability to and success in executing the ASPEN study and itsability to produce sufficient quantities of material for such study; and thosefactors detailed in BioMarin's filings with the Securities and ExchangeCommission, including, without limitation, the factors contained under thecaption "Risk Factors" in BioMarin's 2007 Annual Report on Form 10-K, and thefactors contained in BioMarin's reports on Form 10-Q. Stockholders are urgednot to place undue reliance on forward-looking statements, which speak only asof the date hereof. BioMarin is under no obligation, and expressly disclaimsany obligation to update or alter any forward-looking statement, whether as aresult of new information, future events or otherwise.

BioMarin(R), Naglazyme(R) and Kuvan(R) are registered trademarks ofBioMarin Pharmaceutical Inc.Aldurazyme(R) is a registered trademark of BioMarin/Genzyme LLC. Riquent(R) is a registered trademark of La Jolla Pharmaceutical. Contact: Investors Media Eugenia Shen Susan Berg BioMarin Pharmaceutical Inc. BioMarin Pharmaceutical Inc. (415) 506-6570 (415) 506-6594

SOURCE BioMarin Pharmaceutical Inc.

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