Acceleron Pharma and Shire announce joint development of ACE-031 for DMD

Acceleron Pharma, Inc., a biopharmaceutical company developing novel therapeutics that modulate the growth of cells and tissues including muscle, bone, fat, red blood cells and the vasculature, and Shire plc (LSE: SHP, NASDAQ: SHPGY), the global specialty biopharmaceutical company, today announced a joint development and commercialization agreement for ACE-031 and other novel molecules targeting the activin receptor type IIB (ActRIIB) pathway. This pathway plays critical roles in regulating the growth of skeletal muscle.

“This collaboration is an excellent strategic fit to the work that Shire is already doing on behalf of patients with rare diseases”

Shire will receive an exclusive license to Acceleron's ActRIIB molecules, including ACE-031, in markets outside of North America. Acceleron will retain all commercial rights in North America. The collaboration will initially investigate ACE-031, Acceleron's lead ActRIIB drug candidate, currently in a Phase 2a trial for the treatment of patients with Duchenne Muscular Dystrophy (DMD). DMD is a fatal orphan muscle disease with no currently approved treatment. ACE-031 and other ActRIIB molecules also have the potential to be used in other muscular and neuromuscular disorders with high unmet medical need.

Shire and Acceleron will jointly collaborate on a worldwide development program to advance ACE-031 into a global Phase 2/3 clinical program designed to demonstrate disease modification in DMD patients. Shire will utilize its Lexington manufacturing facility to produce commercial supplies of the product for both parties. If marketing authorization is received, Acceleron will commercialize ACE-031 in the U.S. and Canada, and Shire will commercialize the therapy in the rest of the world.

Under the terms of the agreement, Shire will make an upfront cash payment to Acceleron of $45 million. Acceleron is eligible to receive additional development, regulatory and sales milestone payments of up to $165 million for ACE-031 in DMD, up to an additional $288 million for other indications and molecules, and royalty payments on product sales. Shire and Acceleron will share development costs in North America and Europe, and Shire will be responsible for development costs in other markets.

"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 their proven leadership and dedicated focus on orphan diseases forms the basis for a successful collaboration that will bring innovative therapies to patients with enormous unmet medical need."

"This collaboration is an excellent strategic fit to the work that Shire is already doing on behalf of patients with rare diseases," said Sylvie Grégoire, 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, and expands our pipeline into a new therapeutic area."