NOVATO --  BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today said it acquired privately held biotechnology company ZyStor Therapeutics Inc. in a deal valued at $115 million.

Under the agreement, BioMarin acquired privately held ZyStor of Milwaukee for $22 million up front and up to $93 million more if certain development, regulatory and commercial milestones are achieved. No royalties are owed.

ZyStor develops enzyme-replacement therapies for the treatment of lysosomal storage disorders. Its lead product candidate is ZC-701, developed to treat Pompe disease, a rare progressive degenerative disease of the heart muscle, diaphragm and skeletal muscle affecting one out of 40,000 people born. The total market for Pompe treatment is estimated to be more than $1.0 billion, assuming 3,000 to 6,000 patients have access to high-value therapeutics and an average cost of therapy that is comparable to other enzyme replacement therapies.

"The acquisition of ZyStor gives us the opportunity to introduce a superior product to fulfill an unmet medical need and is a perfect fit in our core business," said Jean-Jacques Bienaime, BioMarin chief executive officer. It not only provides us with a promising product candidate for Pompe disease but also an exciting new platform technology."

Animal and in vitro tests of ZC-701 suggest it could be more effective than commercially available treatments for Pompe disease. Compared to BioMarin's proposed solution, called BMN-103, ZyStor's treatment has a faster clinical development timeline, lower development costs, significantly lower cost of goods and lower capital investment, according to Mr. Bienaime.

The FDA accepted an investigational new drug application for ZC-701. Investigational product has been manufactured, and a clinical study is expected to start in the first quarter of 2011.

Over the next several months BioMarin plans to recruit clinical research hospitals that can conduct clinical studies and finalize the clinical protocol and expects the first patient dosed in the first quarter of 2011.

ZyStor's proprietary Glycosylation independent lysosomal targeting technology can be applicable to other enzyme-replacement therapies and has the potential to deliver more enzyme to lysosomes compared to traditional mannose-6-phosphate targeted approaches, according to Mr. Bienaime.

BMO Capital Markets was ZyStor's financial advisor in the transaction.