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BioMarin Acquires Zacharon Pharmaceuticals

Lead Program Focused on the Oral Treatment of MPS III Acquisition Augments Existing Analytical Capabilities and Expertise in Glycobiology

SAN RAFAEL, Calif., Jan. 7, 2013 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today that it has acquired Zacharon Pharmaceuticals, a private biotechnology company based in San Diego focused on developing small molecules targeting pathways of glycan and glycolipid metabolism.

Zacharon drug discovery projects include two ongoing lead optimization programs, inhibition of heparan sulfate synthesis for MPS III and other MPS disorders, and inhibition of ganglioside synthesis for diseases such as Tay Sachs and Sandhoff. Zacharon's proprietary SensiPro ® platform is a powerful technology for analysis of specific carbohydrate structures and therefore the identification of candidate drugs to treat those conditions.

Hank Fuchs, M.D., Executive Vice President and Chief Medical Officer of BioMarin said, "Zacharon's lead program, focused on reducing the accumulation of heparan sulfate, offers the exciting prospect of treating both the CNS and peripheral manifestations of MPS III, and potentially other MPS disorders, with an orally bioavailable small molecule. In general, reducing the synthesis of the target substrate alleviates the burden on the compromised lysosomal system, and this therapeutic approach has been clinically validated with other enzyme inhibitors. Zacharon's deep expertise in glycobiology has generated additional programs for treating lysosomal storage disorders that we expect to progress, and we will leverage that expertise to continue to build BioMarin's existing research and development pipeline into a sustainably leading pipeline."

"The acquisition of Zacharon will further expand our glycobiology expertise and will support our lysosomal storage disease drug development efforts," said Jean-Jacques Bienaimé, Chief Executive Officer of BioMarin. "We are committed to investing in our advancing pipeline, which has evolved through a combination of internal development and targeted acquisitions, such as this."

Under the terms of the stock purchase agreement, BioMarin paid $10 million upfront for 100 percent of Zacharon's share capital and may make potential additional payments for clinical, regulatory and commercial milestones. The costs to be incurred in 2013 of acquiring Zacharon and absorbing its operations is covered by the previously provided 2013 R&D expense guidance.

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