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Acceleron terminates drug candidate following facioscapulohumeral muscular dystrophy failure

Published on 17/09/19 at 10:27am

Acceleron Pharma has seen its stock fall by almost 4% after it unveiled new Phase 2 data confirming that its investigational therapy ACE-083 failed to meet its functional secondary endpoints in the treatment of facioscapulohumeral muscular dystrophy (FSHD).

The trial results showed that the therapy actually successfully achieved its primary endpoint, demonstrating a robust, statistically significant increase in mean total muscle volume, but this failed to translate into statistically significant improvement in functional tests.

Thus, Acceleron confirmed it would be moving to terminate its clinical trial programme investigating ACE-083 in FSHD.

“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 Chief Executive Officer 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.

“We now look toward the first quarter of next year, when we expect topline results from the placebo-controlled Phase 2 trial of ACE-083 in patients with Charcot-Marie-Tooth disease, a neuromuscular disorder of different pathophysiology.”

Matt Fellows

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