BioMarin Pharmaceutical has acquired ZyStor Therapeutics for $22 million upfront and as much as an additional $93 million if certain development, regulatory and commercial milestones are achieved. ZyStor is a privately-held biotechnology company developing enzyme replacement therapies (ERT) for the treatment of lysosomal storage disorders. ZyStor’s lead product candidate ZC-701 is a fusion of insulin-like growth factor 2 and alpha glucosidase (IGF2-GAA) in development for Pompe disease.

The FDA has accepted the IND for ZC-701 and a clinical study is expected to begin in 1Q11. According to the company, in vitro studies demonstrate that ZC-701 has more than 10 times higher affinity for the mannose-6-phosphate receptor compared to Myozyme, enabling delivery of higher levels of enzyme to the lysosomes of muscle cells of Pompe patients.

“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.It not only provides us with a promising product candidate for Pompe disease but also an exciting new platform technology,” said Jean-Jacques Bienaime, chief executive officer of BioMarin.

Loren Peterson, president and chief executive officer of ZyStor said, “We are very pleased to conclude this transaction with BioMarin for the development of ZC-701.BioMarin has a proven track record of successfully and expeditiously developing value-added therapies for orphan diseases, with particular strength in the field of enzyme replacement therapies for lysosomal storage disorders.”