BioMarin Reports Promising Results of Pompe Phase 1/2 Trial

Category: Archive
March 20, 2013

BioMarin Pharmaceutical Inc. announced 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 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.

Read more in the press release: http://investors.bmrn.com/releasedetail.cfm?ReleaseID=749572 (external link to the web pages of BioMarin) or http://files.shareholder.com/downloads/ABEA-3W276N/2377789652x0x646934/ace1187d-41f6-463d-979d-34611854c1bf/BMRN_News_2013_3_19_General_Releases.pdf (PDF download).


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