BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that it has acquired ZyStor Therapeutics, Inc. (ZyStor), a privately-held biotechnology company developing enzyme replacement therapies (ERT) for the treatment of lysosomal storage disorders. ZyStor's lead product candidate is ZC-701, a novel fusion of insulin-like growth factor 2 and alpha glucosidase (IGF2-GAA) in development for Pompe disease.
Under the terms of the agreement, BioMarin acquired ZyStor for $22 million upfront and up to an additional $93 million if certain development, regulatory and commercial milestones are achieved. There are no royalties owed. The FDA has accepted an investigational new drug (IND) application for ZC-701, investigational product has been manufactured and a clinical study is expected to start in Q1 2011.
In vitro studies demonstrate that ZC-701 has more than ten times higher affinity for the mannose-6-phosphate receptor compared to Myozyme, which enables delivery of higher levels of enzyme to the lysosomes of muscle cells of Pompe patients. Studies in the Pompe mouse model indicate that ZC-701 clears glycogen to lower levels in skeletal, heart and diaphragm muscle compared to Myozyme and at similar levels compared to second generation compounds that have been tested. Many experts believe that an enzyme with more efficient uptake into muscle cells would lead to more effective treatment of the disease. 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.