Acceleron inks $498M pact with Shire for lead muscle drug

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Cambridge, MA-based Acceleron has inked a $498 million development deal with Shire covering the ex-U.S. rights to its closely watched lead therapy, ACE-031. Shire is paying $45 million upfront and committing itself to up to $453 million more in a slate of milestones to snag the international marketing rights to the therapy--as well as other molecules targeting the activin receptor type IIB pathway--which is designed to regulate the growth of skeletal muscle. And Acceleron will hold on to the North American rights to the drug as it lays the foundation for its first commercial launch.

ACE-031 is in a Phase IIa trial for Duchenne Muscular Dystrophy and only weeks ago earned orphan and accelerated-review status at the FDA. The drug has gained the spotlight for its promise to build muscle and increase strength by inhibiting signaling through a cell surface receptor called activin receptor type IIB. DMD is a fatal orphan muscle disease and there's no therapy approved to treat it, offering the biotech a high unmet medical need to target for its first indication.

The deal is structured in a way that it gives Acceleron and Shire an opening to mount a pivotal Phase II/III study designed to demonstrate disease modification after the IIa wraps. That late-stage trial would likely get underway sometime around the end of next year, Steven Ertel, Acceleron's vice president for corporate development, tells FierceBiotech. Ertel wasn't ready to speculate about the development schedule following that trial launch, but he also acknowledges that a therapy like 031 has the potential to ultimately treat a number of conditions.

"Muscle loss is such an exciting area for drug development," adds Ertel. "It's a really underserved area. There's no safe and effective therapy that targets skeletal muscles. Muscle is a really exciting area for researchers."

"The structure of this collaboration allows Acceleron to retain commercial rights in North America with the opportunity to build a highly valuable business while collaborating with an ideal partner for ACE-031," said Acceleron CEO John Knopf, Ph.D., in a statement. It also makes good sense for Shire, which specializes in orphan drugs.

- here's the Acceleron release

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