ACE-031

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ACE-031

ACVR2A-Fc · ActRIIA-hFc · Myostatin inhibitor fusion protein

Clinicalmuscle

A soluble recombinant fusion protein (ActRIIA extracellular domain + IgG1 Fc) that acts as a myostatin trap, blocking myostatin and related TGF-β superfamily ligands to promote muscle growth and reduce muscle wasting.

Half-Life

~11–14 days (Fc fusion extends half-life significantly vs native ActRIIA)

MW

~80,000 Da (dimer with Fc fusion)

Amino Acids

530 AA

Evidence

Clinical

Regulatory Status

Development discontinued (2011). Acceleron paused trials after Phase 2 adverse events. Not approved by FDA. ACE-083 (tissue-specific myostatin inhibitor) is a successor compound in trials. Bimagrumab (anti-ActRIIB antibody) is a related approach by Novartis still in development.

In Plain English

A lab-designed "decoy" protein that soaks up myostatin — the body's main signal that tells muscles to stop growing. By intercepting myostatin before it can bind its real receptor, ACE-031 dramatically increases muscle mass. In a 3-month clinical trial it added over 3 kg of lean muscle. Development was stopped because it also blocked some blood vessel stability signals, causing nosebleeds and capillary problems — a reminder that the myostatin pathway is more complex than just "more is better."

Overview

ACE-031 is a fusion protein developed by Acceleron Pharma consisting of the extracellular domain of activin receptor type IIA (ActRIIA) linked to the Fc portion of human IgG1. By acting as a decoy receptor, ACE-031 sequesters myostatin (GDF-8), GDF-11, activin A/B, and BMP-9/10, preventing them from binding their natural receptors and suppressing muscle (and bone) formation. It was developed for Duchenne muscular dystrophy (DMD) and showed significant lean mass increases in Phase 2 trials, but development was halted after adverse events involving epistaxis, gingival bleeding, and telangiectasias (capillary dilation/bleeding) attributed to its anti-BMP activity.

Common Formats

  • Subcutaneous injection (research protein)
  • Lyophilized powder (research only)

Storage Notes

Protein storage: -80°C long term. Stable at 4°C for 1-2 weeks. Avoid repeated freeze-thaw cycles. Protein concentration per supplier specification.

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Research Sources

(2 vendors)

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Related Compounds

Educational Disclaimer: All information on this page is for educational and research purposes only. This does not constitute medical advice, diagnosis, or treatment recommendation. Consult a qualified healthcare professional.