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BioMarin Provides BMN 673 Program Update

Source: National Cancer Institute at the National Institutes of Health http://www.cancer.gov/cancertopics/factsheet/Risk/BRCA

About BMN 673 Phase 1 Trial Data

BioMarin is conducting an ongoing Phase 1 study for its poly ADP-ribose polymerase (PARP) inhibitor BMN 673 for the treatment of solid tumors. BMN 673 has exhibited substantial single-agent anti-tumor activity in deleterious germline BRCA breast cancers in the trial. The preliminary data were presented during a poster presentation at the 2013 American Society of Clinical Oncology Annual Meeting on June 3, 2013.

The Phase 1 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 (MTD) of daily oral BMN 673. The secondary objectives are to assess the safety, tolerability, preliminary efficacy and pharmacodynamic activity of BMN 673, and to determine the pharmacokinetic profile. The study design was a standard 3 + 3 dose escalation followed by expansion at MTD in cohorts with selected tumor types to further characterize safety and anti-tumor activity. 

BMN 673 was generally well-tolerated. The dose-limiting toxicity was Grade 4 thrombocytopenia. Myelosuppression, most of which was moderate in severity, occurred in 10-20% of patients with chronic dosing. Fatigue, nausea and alopecia were observed in 20-30% of patients. Signs of activity were seen at doses as low as 100 µg/day, and the maximum tolerated dose was 1000 mg/day (1.0 mg/day), which is the expected dose for further development. BMN 673 also has good bioavailability and a long half-life which supports once-daily dosing. 

To access the poster presented at ASCO

http://www.bmrn.com/pipeline/clinical-trials/asco.php

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), which has been approved by the European Commission for the treatment of Lambert Eaton Myasthenic Syndrome (LEMS). Product candidates include Vimizim (N-acetylgalactosamine 6-sulfatase), formally referred to as GALNS, which successfully completed Phase III clinical development for the treatment of MPS IVA, PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently in Phase III clinical development for the treatment of PKU, BMN-701, a novel fusion protein of insulin-like growth factor 2 and acid alpha glucosidase (IGF2-GAA), which is currently in Phase I/II clinical development for the treatment of Pompe disease, BMN 673, a poly ADP-ribose polymerase (PARP) inhibitor, which is currently in Phase I/II clinical development for the treatment of genetically-defined cancers, and BMN-111, a modified C-natriuretic peptide, which is currently in Phase I clinical development for the treatment of achondroplasia. 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 safety and efficacy of such candidate, and the participation of NBCC in the trial execution. 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; NBCC actual level of involvement with the trials; 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 2012 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.

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