BioMarin buys ZyStor - and Pompe drug - in staged deal worth a potential $115 million; moves PKU drug into Ph III

In a second acquisition this year, US drugmaker BioMarin Pharmaceutical (Nasdaq: BMRN) says that it has bought ZyStor Therapeutics, 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 metabolic disorder 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 US Food and Drug Administration has already accepted an Investigational New Drug application for ZC-701, for which a clinical study is expected to start in first-quarter 2011.

Earlier this year, BioMarin entered into a stock purchase agreement to acquire LEAD Therapeutics, a small private drug discovery and early stage development company with key compound LT-673, an orally available poly (ADP-ribose) polymerase (PARP) inhibitor for the treatment of patients with rare, genetically defined cancers (The Pharma Letter February 5). Under the terms of that deal, BioMarin paid LEAD' stockholders $18 million upfront and is due to pay an additional $11 million upon acceptance of the IND filing expected by the end of 2010), and up to $68 million for development and launch milestones for LT-673. The yea before, BioMarin took over Huxley Pharma, also in a staged deal, worth up to $58.5 million (TPL October 27, 2009).