Shire, Acceleron to Collaborate on Treatments for Rare Muscle Diseases

wheelchair-handicap-rampShire Pharmaceuticals has signed a $498 million collaboration agreement with Acceleron Pharma to develop and commercialize treatments for muscular and neuromuscular disorders. Under the terms of the agreement, Cambridge, Mass.-based Acceleron will receive a $45 million up-front payment and could receive up to an additional $165 million in milestone payments for the successful commercialization of ACE-031. ACE-031 is a drug candidate for the treatment of Duchenne muscular dystrophy (DMD), an inherited muscle disease for which there is no known cure. The drug is currently undergoing a Phase 2a clinical trial. Shire also licensed a group of molecules from Acceleron that have the potential to treat other muscular and neuromuscular disorders.

DMD is a severe form of muscular dystrophy characterized by rapid muscle wasting. The disorder is caused by a mutation on the DMD gene that impairs the body’s ability to produce the protein dystrophin. Without enough dystrophin, the body’s muscles become damaged over time. Complications of DMD include decreased mobility, deformities such as scoliosis, cardiomyopathy (weakened or abnormal heart muscle), respiratory infections or failure, and mental impairment. Patients rarely survive past their early 30s; death is usually caused by lung disorders. The disorder usually affects males, occurring in about 1 out of every 3,600 male newborns.

Another company developing a DMD therapy is Switzerland-based Santhera Pharmaceuticals. Santhera ‘s lead compound, Catena, is being investigated for five separate conditions. Catena has been granted orphan drug status in the U.S. and Europe for DMD and is currently in Phase III trials for the disorder. In an early-stage trial, Catena was found to improve or slow the decline in cardiac function and improve certain respiratory parameters in DMD patients.

Portland, Oregon-based AVI BioPharma is currently investigating several drug candidates for DMD. The company’s lead product candidate is AVI-4658, formulated to slow the progression of DMD and improve quality of life for selected patients who suffer from the disorder. AVI-4658 is undergoing a Phase 1b/2 trial in the United Kingdom.

If you know of any other companies working to slow the progression of DMD, or even cure the disorder, we’d like to hear about them. Please post your reply in comments.

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