Shire has licensed the activin receptor type IIB (ActRIIB) class of molecules being developed for the potential treatment of Duchenne Muscular Dystrophy (DMD), from Acceleron Pharma, Inc. The collaboration will initially investigate ACE-031, Acceleron’s lead ActRIIB drug candidate currently in Phase II development. DMD is a fatal muscle disease with no current treatment. ACE-031 and other ActRIIB molecules have the potential to be used in other muscular and neuromuscular disorders with high unmet medical need.

The two companies will collaborate on a worldwide development program to advance ACE-031 into Phase II/III development in DMD patients. Shire will use its Lexington, MA manufacturing facility to produce commercial supplies of the product. Upon marketing authorization, Acceleron will commercialize ACE-031 in the U.S. and Canada and Shire has exclusive rights in the rest of the world.

Acceleron will receive an upfront payment of $45 million and is eligible to receive development, regulatory and sales milestone payments of as much as $165 million for the commercialization of ACE-031 in DMD, as much as $288 million for other indications and molecules, and royalties on product sales. Shire will also contribute to global development costs.

“This collaboration is an excellent strategic fit to the work that Shire is already doing on behalf of patients with rare diseases,” said Sylvie Gregoire, president of Shire Human Genetic Therapies. “Working with Acceleron on the development of ACE-031 for DMD allows us to use our expertise to help patients suffering from this devastating disease, as well as expand our pipeline into a new therapeutic area.”

“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 John Knopf, Ph.D., chief executive officer of Acceleron. “Shire’s international presence and its proven leadership and dedicated focus on orphan diseases forms the basis for successful collaboration to bring innovative therapies to patients with enormous unmet medical need.”