RARE Daily

Acceleron Scraps FSHD Treatment After Mid-Stage Trial Disappointment

September 17, 2019

Acceleron Pharma said it will discontinue development of its experimental treatment ACE-083 in patients with facioscapulohumeral muscular dystrophy (FSHD) because it did not achieve functional secondary endpoints in the phase 2 trial even though it met the primary endpoint of demonstrating a statistically significant increase in mean total muscle volume.

Photo: Habib Dable, president and CEO of Acceleron

Facioscapulohumeral muscular dystrophy is a rare genetic muscle disorder that mainly affects the muscles of the face, shoulder blades and upper arms. Symptoms usually begin to show before the age of 20.

ACE-083 is a TGF beta protein inhibitor that blocks proteins such as myostatins and activins that are involved in muscle breakdown. Although it built muscle in FSHD patients, it did not improve functional muscle strength. Acceleron expects to present the phase 2 results in FSHD at a future medical meeting

“We are certainly disappointed with these results. As we have stated consistently, for ACE-083 to become an important new therapy for patients with FSHD, it would have to deliver a meaningful functional benefit on top of an ability to grow muscle,” said Habib Dable, president and CEO of Acceleron. “Unfortunately, in this case, the data show no evidence of such a benefit and, therefore, do not support further development of ACE-083 for FSHD. We’re grateful to the patients, families, caregivers, and investigators who participated in this research.”

ACE-083 is still being tested as a treatment for Charcot-Marie-Tooth disease, another rare neuromuscular disorder, for which Acceleron expects topline results from a placebo-controlled phase 2 trial in the first quarter of 2020.

Author: Rare Daily Staff

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