Performance/Muscle

ACE-031

Research Reagent · Laboratory Use Only

Quick Answer

What are the research findings on ACE-031 as a myostatin inhibitor in clinical studies?

ACE-031 (bimagrumab precursor/ACVR2B-Fc fusion protein) is an investigational myostatin pathway inhibitor studied for muscle-wasting conditions. Phase II trials in Duchenne muscular dystrophy showed increased lean muscle mass but were halted due to adverse effects including telangiectasia and epistaxis. Research continues into selective ACVR2B pathway modulation for neuromuscular disease.

PMID41686840↗DOI10.1111/j.1365-2249.2012.04654.x↗⟳ Reconstitution Calculator
Scientific AbstractPMID 41686840 · 2026

, myostatin and activin A is associated with improvements in murine skeletal muscle mass and function. The efficacy of a similar treatment approach in a non-human primate (NHP) model would suggest a greater likelihood of success in the treatment of humans suffering from chronic myopathies. In the present study, we elucidate the potential therapeutic benefit of ACE-031, a therapeutic protein consisting of the ActRIIB extracellular region fused to human IgG1, in the common marmoset (Callithrix jacchus).

Marmosets were randomized to receive ACE-031 or vehicle control (10 mM Tris buffered saline; TBS) for 14 weeks. Body composition was measured weekly throughout the experimental period and morphometric analysis and contractile properties of skeletal muscle were assessed terminally. There was a significant main effect of time and time x treatment interaction for lean body mass, such that marmosets administered ACE-031 were greater at euthanasia compared to baseline; this was not observed in the vehicle-treated controls.

Biceps brachii exhibited a significant increase in the cross-sectional area of both type I and type II fibers and ex vivo contractile properties of the EDL showed an increase in absolute and specific force production. The efficacy of ACE-031 in non-human primates provides optimism that a therapeutic strategy that targets multiple negative regulators of skeletal muscle may be beneficial in treating myopathies in humans.

Source:10.1111/j.1365-2249.2012.04654.x
Mechanistic Research SummaryCurated from PubMed

This data is for laboratory research purposes only. Not for human or animal consumption.

What is ACE-031?

ACE-031 is a recombinant fusion protein consisting of the activin receptor type IIB (ActRIIB) extracellular domain fused to human IgG1 Fc region, designed to antagonize myostatin and activin A signaling in skeletal muscle. This therapeutic approach demonstrated efficacy in promoting muscle growth and contractile function in non-human primate models.

Mechanism of Action

ACE-031 functions as a ligand trap by binding and sequestering myostatin and activin A—endogenous negative regulators of skeletal muscle growth. By blocking these TGF-β superfamily ligands from engaging their cognate ActRIIB receptors, ACE-031 de-represses the Smad2/3 signaling pathway, allowing unopposed myogenic differentiation and hypertrophy through alternative ActRI signaling.

Observed Laboratory Results

  • Lean body mass: Common marmosets receiving ACE-031 demonstrated significant increases in lean body mass from baseline to week 14 (time × treatment interaction, p<0.05), whereas vehicle-treated controls showed no change.
  • Muscle fiber cross-sectional area: Biceps brachii samples exhibited significant hypertrophy in both Type I (slow-twitch oxidative) and Type II (fast-twitch glycolytic) fiber populations.
  • Contractile force production: Extensor digitorum longus (EDL) muscle ex vivo analysis revealed increased absolute force and specific force (force normalized to muscle cross-sectional area), indicating enhanced contractile protein organization and function.
Clinical Research ParametersHuman Study Registry

No registered clinical trials or indexed human study data currently available for ACE-031 via ClinicalTrials.gov or PubMed. This compound may be at preclinical or early research stages.

Search ClinicalTrials.gov ↗Search PubMed ↗
Research Compliance Notice

All data presented on this page is for laboratory research purposes only. ACE-031 is referenced here as a research reagent. This page does not constitute medical advice, clinical guidance, or endorsement of any compound for human or animal use. All referenced studies are available via PubMed (PMID: 41686840) and the DOI-linked journal publication. Researchers must consult applicable institutional and regulatory frameworks before conducting any protocols.

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