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BioMarin Enrolls First Patient in Phase 1/2 Trial of NAGLU Fusion Protein BMN 250 for Treatment of MPS IIIB (Sanfilippo B Syndrome)

SAN RAFAEL, Calif., Apr 21, 2016 (GLOBE NEWSWIRE via COMTEX) --

BioMarin Pharmaceutical Inc. BMRN, -0.39% announced today that it has enrolled the first patient in a Phase 1/2 trial for BMN 250, an investigational enzyme replacement therapy using a novel fusion of recombinant human alpha-N-acetyglucosaminidase (NAGLU) with a peptide derived from insulin-like growth factor 2 (IGF2), for the treatment of Sanfilippo B syndrome or mucopolysaccharidosis IIIB (MPS IIIB). Discovered by BioMarin, BMN 250 is being studied in a multicenter, international clinical trial evaluating safety and tolerability, as well as cognitive function of patients with MPS IIIB receiving BMN 250. Designed to restore functional NAGLU activity in the brain, BMN 250 is administered via intracerebroventricular (ICV) infusion.

"Sanfilippo B has long been a difficult condition to treat due to challenges in effectively delivering a therapy that bypasses the blood brain barrier to address the underlying cause of the disease. BMN 250 is designed to overcome these challenges by leveraging our proprietary technology to deliver enzyme replacement therapies directly to the brain," said Hank Fuchs, M.D., Chief Medical Officer at BioMarin. "BMN 250 is the result of building upon our almost two decades of experience in developing treatments for MPS and our most recent experience with an intracerebroventricular delivery approach, which we have used with another experimental enzyme replacement therapy delivered directly to the brain to treat a form of Batten disease."

The company also announced that BMN 250 has been granted orphan drug designation by the European Commission. In late 2014, BMN 250 also received orphan drug designation from the U.S. Food and Drug Administration. BioMarin has three approved therapies to...


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