ACE-083
Also known as
Locally acting follistatin-based ligand trap investigated for lean mass in muscular dystrophy. Moderate evidence grade — two Phase 2 RCTs showed targeted muscle volume gains without significant functional improvement.
Evidence
Multiple human trials, mixed outcomes
Risk band
Known AEs, monitoring advised
Research goals
Summary
ACE-083 is a locally acting, follistatin-based ligand trap designed to induce hypertrophy in the specific muscle into which it is injected [1,4]. In a Phase I randomized trial in postmenopausal women, intramuscular ACE-083 was well tolerated and produced significant targeted muscle growth (up to ~14.5% increase in rectus femoris volume), though without significant changes in muscle strength [1]. Two subsequent Phase 2 randomized trials, in facioscapulohumeral muscular dystrophy and Charcot-Marie-Tooth disease, confirmed significant increases in total muscle volume but found that this did not translate into greater functional improvement compared with placebo, with mild-to-moderate injection-site reactions being the most common adverse events [2,3]. Preclinical models support localized hypertrophy with some functional gains, but the human trials indicate that increased muscle size alone did not yield clinical benefit [4,3]. Overall, the human evidence is reasonably developed but the therapeutic has not demonstrated meaningful functional efficacy.
Sources
- 1. Locally acting ACE-083 increases muscle volume in healthy volunteers Muscle & nerve, 2018.
- 2. Randomized phase 2 study of ACE-083, a muscle-promoting agent, in facioscapulohumeral muscular dystrophy Muscle & nerve, 2022.
- 3. Randomized Phase 2 Study of ACE-083 in Patients With Charcot-Marie-Tooth Disease Neurology, 2022.
- 4. Follistatin-based ligand trap ACE-083 induces localized hypertrophy of skeletal muscle with functional improvement in models of neuromuscular disease Scientific reports, 2019.
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