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BioMarin Announces Selection Of NAGLU Fusion Protein Drug Development Candidate BMN 250 For The Treatment Of Sanfilippo B (MPS IIIB)

Potential to Add Fourth MPS Treatment to BioMarin Franchise

Two presentations at Lysosomal Disease Network's 10 th Annual WORLDSymposiumâ„¢

SAN RAFAEL, Calif., Feb. 11, 2014 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today that it has selected a new drug development candidate, BMN 250, a novel fusion of alpha-N-acetyglucosaminidase (NAGLU) with a peptide derived from insulin-like growth factor 2 (IGF2), for the treatment of Sanfilippo B syndrome or Mucopolysaccharidosis type IIIB (MPS IIIB). BioMarin has initiated IND-enabling studies and expects to initiate clinical studies with BMN 250 in mid-2015.

Discovered by BioMarin, BMN 250 is an enzyme replacement therapy using recombinant human NAGLU with an IGF2, or Glycosylation Independent Lysosomal Targeting (GILT) tag. BMRN 250 is delivered directly to the brain using BioMarin's patented technology. BioMarin has issued patents which broadly cover delivery of lysosomal enzymes directly into the cerebrospinal fluid to treat lysosomal storage diseases.

"We are pleased to add an exciting new candidate to our pipeline that could be a potentially first-in-class therapy for Sanfilippo B patients who currently have no drug treatment options available," said Jean-Jacques Bienaimé, Chief Executive Officer of BioMarin. "Developing BMN 250 for Sanfilippo B or MPS IIIB brings together the best of BioMarin's scientific and clinical expertise. We are building upon a deep knowledge of MPS diseases, and experience developing fusion proteins and enzyme replacement therapies overall. Adding a fourth treatment for MPS complements our current franchise of two approved therapies for the treatment of MPS I and MPS VI and a third expected for MPS IVA."

Data on the NAGLU fusion protein will be presented at the Lysosomal Disease Network's (LDN) 10 th Annual WORLDSymposiumâ„¢ being held February 11-13 in San Diego, California.

"We are encouraged by the results in the preclinical data where we have seen excellent cellular uptake of the enzyme throughout the brain," said Elizabeth Neufeld, Ph.D., Emerita Member, Brain Research Institute and Professor Emerita, Biological Chemistry, David Geffen School of Medicine at the University of California Los Angeles.  "The animal studies show intracellular storage is cleared with NAGLU-IGF2 treatment which we hope will translate well in the clinic."

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