Acceleron Announces Topline Results from the Phase 2 Trial of ACE-083 in Patients with Facioscapulohumeral Muscular Dystrophy
– ACE-083 did not achieve statistically significant improvements in functional endpoints relative to placebo –
– Acceleron to discontinue development of ACE-083 in facioscapulohumeral muscular dystrophy –
– Topline results from the Phase 2 trial of ACE-083 in Charcot-Marie-Tooth disease expected in Q1 2020 -
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
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.
FSHD Phase 2 Trial Design
The two-part Phase 2 clinical trial was designed to evaluate ACE-083 in FSHD patients with muscle weakness in the biceps brachii (BB) and the tibialis anterior (TA), a muscle in the lower leg involved in foot dorsiflexion (raising the foot at the ankle). Part 1 was an open-label, dose-escalation study, with ACE-083 administered by injection into the BB or TA muscle to evaluate safety and increases in muscle volume over a 3-month treatment period. Part 2 was a randomized, double-blind, placebo-controlled study using the optimal dose level selected in Part 1. A total of 56 patients were randomized in Part 2 to receive either placebo or ACE-083 and were evaluated for changes in muscle volume, fat fraction, strength, function, quality of life, and safety over a 6-month primary treatment period, followed by a 6-month open-label treatment period.
For additional information about this clinical trial, please visit www.clinicaltrials.gov.
Acceleron is a clinical-stage biopharmaceutical company dedicated to the discovery, development, and commercialization of therapeutics to treat serious and rare diseases. The Company's leadership in the understanding of TGF-beta biology and protein engineering generates innovative compounds that engage the body's ability to regulate cellular growth and repair.
Acceleron focuses its research and development efforts in hematologic, neuromuscular, and pulmonary diseases. In hematology, the Company and its global collaboration partner,
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Acceleron Pharma Inc.
Todd James, IRC, 617-649-9393
Vice President, Investor Relations and Corporate Communications
Ed Joyce, 617-649-9242
Director, Investor Relations
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Director, Corporate Communications