About ACE-031
Soluble decoy receptor for ActRIIB; sequesters myostatin, activin, and GDF-11 to remove multiple brakes on muscle and bone growth simultaneously
ACE-031 is a soluble decoy receptor fusion protein consisting of the extracellular domain of activin type IIA receptor (ActRIIA) linked to a human IgG1 Fc region, developed by Acceleron Pharma to bind and sequester myostatin, activin, and related TGF-beta superfamily ligands that negatively regulate muscle mass, with the goal of promoting muscle growth in severe wasting conditions including Duchenne muscular dystrophy. By competitively binding circulating myostatin and related ligands, ACE-031 reduces signaling through the Smad2/3 pathway that suppresses muscle satellite cell activation and protein synthesis; in preclinical models of myopathy, blockade of ActRIIA signaling produced significant increases in lean mass, supporting its evaluation in Phase 2 human trials. A Phase 2 randomized placebo-controlled trial in ambulatory boys with Duchenne muscular dystrophy demonstrated significant increases in lean body mass; however, the trial was halted early due to vascular-related adverse events including epistaxis and telangiectasias, attributed to off-target inhibition of angiogenic TGF-beta family ligands, and the Acceleron clinical program was subsequently discontinued. ACE-031 has no FDA approval and is not approved for any indication; commercial development was halted due to the adverse event signal identified in the clinical trial; it is not commercially available, and the vascular safety concern inherent to pan-ActRIIA ligand inhibition represents an unresolved risk that precludes its extrapolation to general performance or muscle enhancement applications.
ACE-031 Benefits & Research Areas
Research Signals
Commonly researched in the context of
Population research notes
These signals reflect research interest areas, not treatment indications.
Regulatory & Evidence
Risk Profile
Higher risk profile in research contexts. Review all contraindications carefully. Not suitable for self-administration without professional oversight.
Regulatory Status
- Availability Status
- Research Only
- FDA Status
- Investigational
Activin receptor IIB-Fc fusion (ACVR2B decoy) by Acceleron Pharma. Phase 2 for Duchenne MD halted 2013 due to AEs (epistaxis, telangiectasia). Development discontinued. Previously held IND.
Regulatory status reflects publicly available information and may change. This is not legal or medical advice.
Research Sources
1 source cited · 1 moderate
1 RCT
Myostatin inhibitor ACE-031 treatment of ambulatory boys with Duchenne muscular dystrophy: Results of a randomized, placebo-controlled clinical trial
Muscle & Nerve · 2017
In a phase 1/2 randomized placebo-controlled trial of ACE-031 in boys with Duchenne muscular dystrophy, subcutaneous administration showed trends toward maintaining 6-minute walk test distance and increasing lean body mass and bone mineral density, though the study was discontinued early due to non-muscle-related safety signals including epistaxis and telangiectasias.
ACE-031 Side Effects & Safety Considerations
Higher risk profile in research contexts. Professional oversight recommended.
Reported contraindications & considerations
Consult a qualified healthcare professional before making any health decisions. This information is educational only and does not constitute medical advice.
Where to Buy ACE-031 — Providers & Availability
2 providersOnline Vendors
2 providersStay updated on verified ACE-031 providers
New verified providers added weekly — delivered to your inbox.
Questions to Ask Your Provider
Frequently Asked Questions — ACE-031
ACE-031 is a soluble decoy receptor fusion protein consisting of the extracellular domain of activin type IIA receptor (ActRIIA) linked to a human IgG1 Fc region, developed by Acceleron Pharma to bind and sequester myostatin, activin, and related TGF-beta superfamily ligands that negatively regulate muscle mass, with the goal of promoting muscle growth in severe wasting conditions including Duchenne muscular dystrophy. By competitively binding circulating myostatin and related ligands, ACE-031 reduces signaling through the Smad2/3 pathway that suppresses muscle satellite cell activation and protein synthesis; in preclinical models of myopathy, blockade of ActRIIA signaling produced significant increases in lean mass, supporting its evaluation in Phase 2 human trials.
myostatin/activin inhibition, muscle mass increase, bone density improvement.
Research on ACE-031 primarily documents effects related to myostatin/activin inhibition and muscle mass increase and bone density improvement. These are areas covered in preclinical and clinical literature — individual response varies and effects depend on context of use.
Reported contraindications and considerations for ACE-031 include cardiovascular disease, telangiectasia risk, bleeding disorders. 1 additional consideration are noted in the safety profile above. This is educational information only — consult a qualified healthcare professional before use.
2 providers in the directory currently offer ACE-031.
In a phase 1/2 randomized placebo-controlled trial of ACE-031 in boys with Duchenne muscular dystrophy, subcutaneous administration showed trends toward maintaining 6-minute walk test distance and increasing lean body mass and bone mineral density, though the study was discontinued early due to non-muscle-related safety signals including epistaxis and telangiectasias.