ACE-083 Reference

Reference summary

Thomas 2022 Neurology (CMT, n=44, intramuscular ACE-083 to tibialis anterior) and Statland 2022 Muscle Nerve (FSHD) are the two published Phase 2 datasets. Both reported MRI evidence of muscle hypertrophy at the injected site but no significant gains on functional endpoints (6-minute walk, foot-drop measures, FSHD functional composite). The program was discontinued before Phase 3.

Regulatory and posture

Categories

Growth Factor

Aliases

Locally-injected follistatin-Fc fusion protein, Acceleron Pharma development code

Evidence posture

human - Phase 2 trials showed muscle growth without functional benefit; development discontinued.

Regulatory status

Not FDA-approved. Development discontinued. ACE-083 was Acceleron Pharma's locally-injected follistatin-Fc fusion designed to produce focal muscle hypertrophy without the systemic vascular signals seen with ACE-031. Two Phase 2 trials were completed in Charcot-Marie-Tooth disease (Thomas 2022 Neurology) and facioscapulohumeral muscular dystrophy (Statland 2022 Muscle Nerve). Both reported MRI-measured muscle volume increases without translating into clinically meaningful functional improvement, and Acceleron discontinued the program in 2019. WADA-prohibited at all times as a myostatin-pathway agent (S4).

Content review status

research reference