Myostatin inhibitor ACE-031 treatment of ambulatory boys with Duchenne muscular dystrophy: Results of a randomized, placebo-controlled clinical trial

Muscle Nerve. 2017 Apr;55(4):458-464. doi: 10.1002/mus.25268. Epub 2016 Dec 23.


Introduction: ACE-031 is a fusion protein of activin receptor type IIB and IgG1-Fc, which binds myostatin and related ligands. It aims to disrupt the inhibitory effect on muscle development and provide potential therapy for myopathies like Duchenne muscular dystrophy (DMD).

Methods: ACE-031 was administered subcutaneously every 2-4 weeks to DMD boys in a randomized, double-blind, placebo-controlled, ascending-dose trial. The primary objective was safety evaluation. Secondary objectives included characterization of pharmacokinetics and pharmacodynamics.

Results: ACE-031 was not associated with serious or severe adverse events. The study was stopped after the second dosing regimen due to potential safety concerns of epistaxis and telangiectasias. A trend for maintenance of the 6-minute walk test (6MWT) distance in the ACE-031 groups compared with a decline in the placebo group (not statistically significant) was noted, as was a trend for increased lean body mass and bone mineral density (BMD) and reduced fat mass.

Conclusion: ACE-031 use demonstrated trends for pharmacodynamic effects on lean mass, fat mass, BMD, and 6MWT. Non-muscle-related adverse events contributed to the decision to discontinue the study. Myostatin inhibition is a promising therapeutic approach for DMD. Muscle Nerve 55: 458-464, 2017.

Keywords: 6-minute walk test; Duchenne muscular dystrophy; TGF-β superfamily; lean body mass; myostatin inhibitor; placebo-controlled clinical trial.

Publication types

  • Randomized Controlled Trial

MeSH terms

  • Activin Receptors, Type II / pharmacokinetics
  • Activin Receptors, Type II / pharmacology
  • Activin Receptors, Type II / therapeutic use*
  • Adolescent
  • Body Composition / drug effects
  • Child
  • Child, Preschool
  • Cohort Studies
  • Dose-Response Relationship, Drug
  • Double-Blind Method
  • Drug Administration Schedule
  • Humans
  • Male
  • Muscular Dystrophy, Duchenne / diagnostic imaging
  • Muscular Dystrophy, Duchenne / drug therapy*
  • Myostatin / antagonists & inhibitors*
  • Treatment Outcome*
  • Walking / physiology


  • Myostatin
  • ACVR2B protein, human
  • Activin Receptors, Type II