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BioMarin to Advance BMN-701 for Pompe Disease to Next Phase of Development

Posted on: March 22, 2013

San Rafael, Calif, March 19, 2013 – 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®).

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