Rare Daily Staff

BioMarin said its selected a compound that it plans to advance to clinical development for the treatment of Friedreich’s Ataxia, a rare genetic disorder that causes progressive neurologic and cardiac decline.

The company made the announcement during its annual R&D day where it provided investors an update on its pipeline. The experimental candidate, BMN 290, is a selective chromatin modulation therapy. The function of chromatin, a mix of proteins knowns as histones and genetic material, is to compress DNA within the cells’ nucleus. In preclinical models of Friedreich’s Ataxia, BMN 290 increased frataxin expression in affected tissue more than two-fold, the company said.

BMN 290 is a second-generation compound derived from a compound the company acquired in 2014 from Repligen when it acquired Repligen’s histone deacetylase inhibitor library and related intellectual property for an upfront payment of $2 million and potential future milestone payments and royalties.

Repligen had human clinical data showing increases in the protein frataxin in FA patients. Production of frataxin is reduced in patients with FA and the gene that produces it is mutated in these patients.

There is a growing pipeline of potential therapies in development for FA. It can be found on the FARA website.

October 19, 2017