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BioMarin To Advance BMN-701 For Pompe Disease To Next Phase Of Development

BMN-701 Meaningfully Improves Respiratory Endpoints Phase 2/3 Switch Study Expected to Begin by December 2013

Conference Call and Webcast to Be Held Today at 5:00 p.m. ET

SAN RAFAEL, Calif., March 19, 2013 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today results from POM-001, the Phase 1/2 trial for BMN-701, a fusion protein of insulin-like growth factor 2 and acid alpha-glucosidase (IGF2-GAA) for the treatment of late-onset Pompe disease. The results exceeded the company's pre-specified requirements for proceeding to the next phase of development by showing that in the 20 mg/kg every other week dose cohort, three out of 16 patients, or 19 percent, had a greater than 75 meter improvement in 6-minute walk distance, and that there was a 14.1 percent relative improvement in Maximal Expiratory Pressure (MEP) and a 27.0 percent relative improvement in Maximal Inspiratory Pressure (MIP) from pre-treatment baseline to week 24, two important measures of overall respiratory muscle function and strength. Pending a review with regulatory authorities, the company expects to continue development of BMN-701 by initiating a Phase 2/3 switching trial by the end of 2013 in late-onset Pompe patients who have previously been treated with alglucosidase alfa (Myozyme ®/Lumizyme ®).

"More than half of late-onset Pompe patients require ventilatory assistance, and many more patients have impairments directly related to weakness of breathing muscles. This means that a therapy that improves respiratory muscle function substantially would be important and welcome and could help delay premature death in Pompe disease," said Professor Benedikt Schoser of the Friederich-Baur Institute and speaker of the German working group for Pompe disease.

"The findings from this study point the way forward for a potentially meaningful advance in the management of late-onset Pompe patients. Pompe is a disease that destroys muscle tissue throughout the body, including the muscles of respiration. Maximal Expiratory Pressure (MEP) and Maximal Inspiratory Pressure (MIP) are direct measures of respiratory muscle function and likely an important indicator of a drug's effectiveness in this setting. The improvements in MEP and MIP that BMN-701 patients demonstrated is a unique and important finding," said Barry Byrne, M.D., Ph.D., Professor, Pediatrics and Molecular Genetics & Microbiology and Director, University of Florida Powell Center, and lead investigator for POM-001. "If treatment with BMN-701 results in improvements in these important respiratory functional measures in patients who have already experienced a maximal benefit from current therapy, BMN-701 could become an important treatment option," Byrne said.

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