Pharmacy

Shire and Acceleron Pharma collaborate to treat orphan muscle diseases

Category : Pharmacy

Shire,     the     global     specialty biopharmaceutical          company, announced the expansion of its human genetic therapies pipeline through the exclusive licence, in markets outside of North America, for the activin receptor type IIB (ActRIIB) class of molecules being developed by Acceleron Pharma Inc, a private biotechnology company based in Cambridge, Massachusetts. The association will initially investigate ACE-031, Acceleron's lead ActRIIB drug candidate, which is currently in phase 2 of the trial for the treatment of patients with Duchenne Muscular Dystrophy (DMD). DMD is a fatal orphan muscle disease with no current treatment. ACE-031 and other ActRIIB molecules have the potential to be used in other muscular and neuromuscular disorders.

Shire and Acceleron will collaborate on   a   worldwide   development programme to advance ACE-031 into a global phase 2/3 clinical programme designed to exhibit disease modification in DMD patients. They would utilize the facility to produce commercial supplies of the product for both parties Shire will also make a payment of $45 million to Acceleron, who is also eligible to receive additional development, regulatory and sales milestone payments of up to $165 million.


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