CAMBRIDGE, Mass.–(BUSINESS WIRE)–Acceleron Pharma Inc. (NASDAQ:XLRN), a leading biopharmaceutical company in the discovery and development of TGF-beta superfamily therapeutics to treat serious and rare diseases, today announced that treatment with ACE-083 in patients with facioscapulohumeral muscular dystrophy (FSHD) did not achieve functional secondary endpoints in the Phase 2 trial.
Although ACE-083 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. As a result, Acceleron will not conduct further clinical trials of 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.”
Dable added: “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.”
In this Phase 2 trial in patients with FSHD, ACE-083 was generally well tolerated. Adverse events were mostly mild to moderate (Grade 1 or 2) and injection-site related. Acceleron expects to present results at a future medical meeting.
ACE-083 is an investigational therapy that is not approved for any use in any country, Acceleron noted in its press release.