BioMarin Initiates Phase 1 Trial for BMN 673 in Patients With Advanced Hematological Malignancies BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced the initiation of a Phase 1 trial for BMN 673, a poly ADP-ribose polymerase (PARP)...

NOVATO, Calif. , July 14, 2011 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN ) today announced the initiation of a Phase 1 trial for BMN 673, a poly ADP-ribose polymerase (PARP) inhibitor, for the treatment of patients with advanced hematological malignancies. A Phase 1/2 trial for BMN 673 for the treatment of patients with solid tumors was initiated in January 2011 and is ongoing.

"We are excited to be at the forefront of studying the potential benefit of PARP inhibitors in hematological malignancies," said Hank Fuchs , M.D., Chief Medical Officer of BioMarin. "Our approach to the development of our PARP inhibitor is to identify tumor mutations that are more susceptible to treatment with BMN 673 and to target subgroups based on the genetic basis of the disease. We are committed to the advancement of our pipeline and look forward to many clinical milestones over the next 18 months."

This Phase 1 trial is a two-arm, open-label dose escalation study to determine the maximum tolerated dose and to assess the safety, pharmacokinetics, pharmacodynamics and preliminary efficacy of once daily, oral BMN 673 in patients with acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), chronic lymphocytic leukemia (CLL) or mantle cell lymphoma (MCL). The study will enroll approximately 80 patients across multiple sites in the U.S. and UK.

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 GALNS (N-acetylgalactosamine 6-sulfatase), which is currently in Phase III clinical development for the treatment of MPS IVA, amifampridine phosphate (3,4-diaminopyridine phosphate), which is currently in Phase III clinical development for the treatment of LEMS in the U.S., PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently in Phase II 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, and 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. 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

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.

SOURCE BioMarin Pharmaceutical Inc.