Locally acting ACE-083 increases muscle volume in healthy volunteers

Muscle Nerve. 2018 Jun;57(6):921-926. doi: 10.1002/mus.26113. Epub 2018 Mar 15.


Introduction: ACE-083 is a locally acting follistatin-based therapeutic that binds myostatin and other muscle regulators and has been shown to increase muscle mass and force in neuromuscular disease mouse models. This first-in-human study examined these effects.

Methods: In this phase 1, randomized, double-blind, placebo-controlled, dose-ranging study in healthy postmenopausal women, ACE-083 (50-200 mg) or placebo was administered unilaterally into rectus femoris (RF) or tibialis anterior (TA) muscles as 1 or 2 doses 3 weeks apart.

Results: Fifty-eight postmenopausal women were enrolled, 42 ACE-083 and 16 placebo. No serious adverse events (AE), dose-limiting toxicities, or discontinuations resulting from AEs occurred. Maximum (mean ± SD) increases in RF and TA muscle volume were 14.5% ± 4.5% and 8.9% ± 4.7%, respectively. No significant changes in mean muscle strength were observed.

Discussion: ACE-083 was well tolerated and resulted in significant targeted muscle growth. ACE-083 may have the potential to increase muscle mass in a wide range of neuromuscular disorders. Muscle Nerve 57: 921-926, 2018.

Trial registration: ClinicalTrials.gov NCT02257489.

Keywords: ACE-083; muscle volume; muscular dystrophy; myostatin; neuromuscular disease.

Publication types

  • Clinical Trial, Phase I
  • Randomized Controlled Trial

MeSH terms

  • Aged
  • Double-Blind Method
  • Female
  • Follistatin / pharmacology*
  • Healthy Volunteers
  • Humans
  • Middle Aged
  • Muscle Strength / drug effects*
  • Muscle, Skeletal / drug effects*
  • Organ Size / drug effects
  • Recombinant Proteins / pharmacology*


  • Follistatin
  • Recombinant Proteins

Associated data

  • ClinicalTrials.gov/NCT02257489