Acceleron Pharma Inc. and Shire plc have signed a development and commercialization deal associated with Acceleron’s novel molecules linked to skeletal muscle growth. The agreement will bring in $45 million in upfront payments to Acceleron, along with up to $165 million in milestone payments and up to $288 million additionally for other molecules, as well as royalty payments on product sales.
The collaboration deal focuses on Acceleron’s ACE-031, an investigational protein therapeutic intended to build muscle. The drug candidate is in Phase 2a clinical trials for the treatment of Duchenne Muscular Dystrophy (DMD), a fatal muscular disease that currently has no approved treatments.
Developing ACE-031 jointly and moving it to Phase 2/3 clinical trials is the goal of the two companies, according to a news release today. Shire will be responsible for manufacturing commercial supplies of ACE-031 at its Lexington facility and, if approved by the U.S. Food and Drug Administration, Acceleron will hold U.S. and Canadian commercialization rights, while Shire will hold commercialization rights to the rest of the world.
Last month, Acceleron won orphan drug status for ACE-031, in addition to fast track status, from the FDA, to treat DMD, which primarily affects boys and occurs in approximately 1 in every 3,500 live male births. Few patients survive beyond their late-20s when their heart and respiratory muscles weaken and eventually fail.
U.K.-based Shire has its Human Genetics Therapies division headquartered in Cambridge and Lexington.