Repligen Corporation announced today that it has entered into an asset purchase agreement with BioMarin Pharmaceutical Inc. to advance Repligen's histone deacetylase inhibitor (HDACi) portfolio. The HDACi portfolio includes multiple orally bioavailable small molecule compounds as well as enabling technologies. Under the terms of the agreement, Repligen will receive an upfront payment of $2 million from BioMarin and it has the potential to receive up to $160 million in future milestone payments for the development, regulatory approval and commercial sale of portfolio compounds included in the agreement. In addition, Repligen is eligible to receive royalties on sales of therapeutic products originating from the HDACi portfolio. Potential applications of the HDACi portfolio include Friedreich's ataxia and other neurological disorders.
"The outlicensing of our HDAC portfolio, which includes the Friedreich's ataxia program, is consistent with our objective to realize financial value from discontinued therapeutic development programs as we fully focus on the expansion of Repligen's bioprocessing business," said Walter C. Herlihy, Ph.D., President and Chief Executive Officer of Repligen. "BioMarin is an ideal partner for the HDACi program based on the company's history of successfully developing and commercializing first-to-market and best-in-class therapies for people with serious rare disorders."
About Friedreich's ataxia
Friedreich's ataxia (FA) is a progressive, neurological disorder that affects approximately 20,000 people in the United States and Europe, typically resulting in wheelchair dependence in young adulthood and early death due to cardiac failure. It is caused by mutations in the FXN gene, and is inherited in an autosomal recessive manner. FXN mutations result in reduced expression of frataxin protein, manifesting in progressive neurological and cardiac damage. Major neurological symptoms include muscle weakness and ataxia, a loss of balance and coordination. These symptoms typically appear between 10 and 15 years of age, but FA has been diagnosed in people from ages 2 to 50 with earlier onset associated with a more severe course.