Pfizer, Zacharon Pharmaceuticals to develop drugs for orphan diseases

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SAN DIEGO—Pharma giant Pfizer Inc. will partner with Zacharon Pharmaceuticals Inc. to develop drugs for orphan diseases, including lysosomal storage disorders (LSDs), in a deal valued at about $210 million, the companies announced last month.

LSDs are a group of more than 40 inherited disorders typically characterized by a deficiency in one or more enzymes that degrade glycans, the carbohydrate chains of glycoproteins, proteoglycans and glycolipids. These deficiencies cause an accumulation of undigested glycan fragments inside the lysosome, leading to progressive deterioration in physical and/or mental state, and eventually, premature death. The family of lysosomal storage diseases includes Gaucher, Fabry, Pompe, mucopolysaccharidoses (MPS I, II, IIIA, IIIB, IIIC), Tay-Sachs, Sandhoff and other related diseases, with a total combined incidence greater than 1 per 8,000 births.

The collaboration includes the potential development of compounds that may be discovered using Zacharon's platform for developing small-molecule drugs targeting specific carbohydrate polymers or glycans.

Under the terms of the agreement, Zacharon, whose sole venture investor is Avalon Ventures, will receive upfront payments and research and development funding to develop drugs against targets that impact lysosomal storage diseases. Zacharon is also eligible under the collaboration for payments for meeting development milestones, plus royalties and sales milestones upon commercialization.

"We are very pleased to be working with Pfizer, which has broad compound development expertise, including in the area of small molecules, which should be quite useful to developing drugs for these orphan diseases," said Dr. Robin Jackman, president and CEO of Zacharon, in a statement. "The collaboration provides validation for the potential that lies in Zacharon's broadly applicable technology platform."

According to Dr. Ed Mascioli, head of Pfizer's Orphan & Genetic Diseases Unit, part of his unit's strategic focus relies on collaborations with companies like Zacharon that have promising technologies to help develop treatments for rare diseases.

"Zacharon is an ideal partner, and we look forward to working with them to develop treatments for lysosomal storage diseases," Mascioli states.

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