Acceleron Announces Publication of ACE-083 Phase 1 Trial Results in Muscle & Nerve
“These unprecedented increases in total muscle volume produced in the
Phase 1 trial of ACE-083 were critical in leading us to rapidly advance
into two Phase 2 trials in two distinct diseases of focal muscle
weakness, and we are thrilled that the Muscle & Nerve journal
has selected this study for publication,” said
The article, entitled “Locally Acting ACE-083 Increases Muscle Volume in Healthy Volunteers” is now available online and will be published in a future print issue of Muscle & Nerve. The Phase 1 trial was a randomized, double-blind, placebo-controlled, dose-ranging study in healthy volunteers. The key objectives of the trial were safety and tolerability, as well as assessing changes in total muscle volume of the treated muscles via magnetic resonance imaging (MRI) of the RF and TA muscles.
ACE-083 is currently being evaluated in two Phase 2 trials in FSHD and Charcot-Marie-Tooth (CMT) disease. The Company recently announced preliminary results from Part 1 of the FSHD trial with mean total volume increases of over 12% in the tibialis anterior and biceps brachii muscle cohorts. The final Part 1 results from both Phase 2 trials are expected in the second half of 2018. Part 2 of the FSHD trial is expected to be initiated in the second quarter of 2018 with results expected in the second half of 2019. Part 2 of the CMT trial is expected to be initiated before the end of 2018.
Presentations outlining the Phase 1 results included in the publication are available online under the science page on the Company’s website at www.acceleronpharma.com.
ACE-083 is a therapeutic candidate, based on the naturally-occurring protein follistatin, which utilizes the "Myostatin+" approach to inhibit multiple TGF-beta ligands. It is designed to have a concentrated effect along targeted muscles to maximize growth and strength selectively in the muscles into which the drug is administered. Acceleron is developing ACE-083 for diseases such as facioscapulohumeral dystrophy (FSHD) and Charcot-Marie-Tooth (CMT) disease, in which improved muscle strength in target muscles may provide a clinical benefit and enhance quality of life.
Acceleron is a Cambridge-based, 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
Candice Ellis, 617-649-9226
Manager, Investor Relations and Corporate Communications
Brad Miles, 646-513-3125