BioMarin Pharmaceutical acquired Zacharon Pharmaceuticals for $10 million up front. The company may make potential additional payments for clinical, regulatory, and commercial milestones. Zacharon is focused on developing small molecules targeting pathways of glycan and glycolipid metabolism.
Zacharon’s 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 says its SensiPro® platform analyzes specific carbohydrate structures and identifies candidate drugs to treat those conditions.
“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,” says Hank Fuchs, M.D., evp and CMO of BioMarin. “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,” adds Jean-Jacques Bienaime, CEO 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.”
Bienaime is referring to acquisitions such as BioMarin’s 2010 purchase of ZyStor Therapeutics for $22 million up front. ZyStor developed enzyme replacement therapies for lysosomal storage disorders. Earlier that year, BioMarin acquired LEAD Therapeutics, a small private drug discovery and early-stage development company with key compound LT-673, an orally available poly (ADP-ribose) polymerase inhibitor for the treatment of patients with rare, genetically defined cancers. In 2009, BioMarin bought Huxley Pharmaceuticals, which had rights to a form of 3,4-diaminopyridine (3,4-DAP), amifampridine phosphate, for the rare autoimmune disease Lambert Eaton Myasthenic Syndrome.