Acceleron has announced the discontinuation of the development of its drug intended for facioscapulohumeral muscular dystrophy (FSHD), ACE-083.
The drug did not achieve statistically significant improvements in functional endpoints relative to placebo in a Phase II trial, which means that Acceleron will not conduct further clinical trials.
Although the drug demonstrated a robust, statistically significant increase in mean total muscle volume, the primary endpoint of the trial, the increase failed to translate to statistically significant improvements in functional tests.
The company says that it is “disappointed” with the results, and that it had “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.
He continued, “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.”
The company also said it is “looking forward to the first quarter of next year”, as it expects topline results from the placebo-controlled Phase II trial of ACE-083 in patients with Charcot-Marie-Tooth disease—a neuromuscular disorder of different pathophysiology.
ACE-083 is an investigational therapy that is not approved for any use in any country.