January 11, 2011

BioMarin Initiates Phase 1/2 Trial for BMN 673 for the Treatment of Genetically-Defined Cancers

NOVATO, Calif., Jan. 11, 2011 /PRNewswire via COMTEX/ --

BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced the initiation of a Phase 1/2 trial for BMN 673, a poly ADP-ribose polymerase (PARP) inhibitor in development for the treatment of genetically-defined cancers.

"PARP inihibtors have been validated to show survival benefits in cancer patients with tumors that have defects in DNA repair or in combination with DNA damaging agents, and BMN 673 appears to have superior potency, selectivity, and bioavailability as compared to other products in development," said Hank Fuchs, Chief Medical Officer of BioMarin. "There are many possible targets for PARP inhibitors, and we hope to identify which cancers are more susceptible to treatment with BMN 673 in the Phase 1/2 trial. We remain intently focused on developing our R&D pipeline and look forward to many clinical milestones in the coming year."

The Phase 1/2 trial is an open-label study of once daily, orally administered BMN 673 in approximately 70 patients ages 18 and older with advanced or recurrent solid tumors. The primary objective of the study is to establish the maximum tolerated dose of daily oral BMN 673. The secondary objectives are to assess the safety, tolerability, preliminary efficacy and phatrmacodynamic activity of BMN 673, and to determine the pharmacokinetic profile and recommended Phase 2 dose.

About BioMarin

BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio comprises four approved products and multiple clinical and pre-clinical product candidates. Approved products include Naglazyme® (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin; Aldurazyme® (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; Kuvan® (sapropterin dihydrochloride) Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany; and Firdapse (amifampridine phosphate), which has been approved by the European Commission for the treatment of Lambert Eaton Myasthenic Syndrome (LEMS). Other product candidates include GALNS (N-acetylgalactosamine 6-sulfatase), which is currently in clinical development for the treatment of MPS IVA, and PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently in Phase II clinical development for the treatment of PKU. For additional information, please visit www.BMRN.com. Information on BioMarin's website is not incorporated by reference into this press release.

Forward-Looking Statement

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including, without limitation, statements about: the expectations of the development of BMN 673, including the timing of the clinical trials of the candidate, and the possible efficacy of such candidate. 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 content and timing of decisions by the U.S. Food and Drug Administration, the European Commission and other regulatory authorities, results and timing of current and planned clinical and preclinical studies related to such product; our ability to successfully manufacture the product; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's 2009 Annual Report on Form 10-K, and the factors contained in BioMarin's reports on Form 10-Q. 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®, Naglazyme® and Kuvan® are registered trademarks of BioMarin Pharmaceutical Inc.

Firdapse is a trademark of BioMarin Huxley Ltd.

Aldurazyme® is a registered trademark of BioMarin/Genzyme LLC.

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BioMarin Pharmaceutical Inc.

BioMarin Pharmaceutical Inc.

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SOURCE BioMarin Pharmaceutical Inc.


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