ACE-031
ACE-031 (Activin Receptor Type IIB-Fc Fusion Protein)
Myostatin trap designed for muscle wasting diseases
ACE-031 is a soluble activin receptor type IIB fusion protein that acts as a myostatin and activin trap. It was developed by Acceleron Pharma for Duchenne muscular dystrophy.
Admin routes
Subcutaneous
Popularity
Niche
Side effects
Generally mild
AU vendors
0 rated
✓Key benefits
📈What to expect
Measurable increases in lean body mass detected in clinical trials
3-4% lean mass increase observed in Phase 1 trial at highest doses
Based on community reports and published research. Individual results vary significantly.
💊Dosing protocols
DMD trial dose (reference only)
1-3 mg/kg
Every 2 weeks (subcutaneous)
Trial was designed for 12+ months
Dosing information is sourced from published research and community protocols. This is not a recommendation. Consult a healthcare professional.
Research status|Phase 2 trial halted due to safety concerns
Overview
ACE-031 works by acting as a decoy receptor that binds myostatin and other TGF-beta superfamily ligands before they can signal through their normal receptors. This effectively removes the brakes on muscle growth. Acceleron Pharma developed it and tested it in a Phase 2 clinical trial for Duchenne muscular dystrophy in boys aged 4-16. While the trial showed significant increases in lean body mass and bone mineral density, it was halted due to safety concerns including nosebleeds, gum bleeding, and dilated blood vessels in the skin. Acceleron pivoted to ACE-083 (a localised version) and later luspatercept for blood disorders.
⚙️How it works
ACE-031 is a fusion protein combining the extracellular domain of the activin type IIB receptor with the Fc portion of human IgG1. It acts as a ligand trap, binding myostatin, GDF-11, activins, and other TGF-beta superfamily members with high affinity. By sequestering these growth-inhibitory ligands, it removes the negative regulation of muscle growth, allowing increased muscle protein synthesis and reduced muscle protein breakdown.
⚡Side effects
📅Research history
Acceleron Pharma begins ACE-031 development
Phase 1 healthy volunteer study shows +3.6% lean mass in 4 weeks
Phase 2 trial in DMD halted due to vascular safety signals
Final clinical data published
Why the trial was stopped
The Phase 2 trial in DMD boys was halted after several patients developed nosebleeds, gum bleeding, and telangiectasias (small dilated blood vessels visible on the skin). These effects were attributed to the broad ligand-trapping activity of ACE-031. Because it doesn't only trap myostatin but also activins and other TGF-beta family members, it disrupts vascular biology. This broad activity was the compound's strength (more muscle growth) and its weakness (off-target vascular effects).
References
- [1]Campbell C, et al. 'Myostatin inhibitor ACE-031 treatment of ambulatory boys with Duchenne muscular dystrophy.' Muscle & Nerve, 2017.
- [2]Attie KM, et al. 'A single ascending-dose study of muscle regulator ACE-031 in healthy volunteers.' Muscle & Nerve, 2013.
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Disclaimer: This guide is for educational and informational purposes only. It is not medical advice. The dosing protocols listed are sourced from published research and community reports and do not constitute a recommendation. Always consult a qualified healthcare professional before using any peptide. Australian regulations classify many peptides as Schedule 4 (prescription-only) substances. Check current TGA guidelines before purchasing.