ACE-031
ACE-031 (ramatercept) is a soluble activin receptor type IIB fused to an antibody Fc fragment, designed to act as a decoy trap for myostatin and related ligands. It was tested clinically for Duchenne muscular dystrophy, where it increased muscle volume but trials were halted over safety signals including nosebleeds and gum bleeding (telangiectasias). It is a large biologic, not a peptide suited to casual subcutaneous dosing.
01 Overview
ACE-031 works by mopping up myostatin and other activin-receptor ligands before they can signal, releasing the brake on muscle growth without directly targeting the receptor on muscle. Acceleron and partners advanced it into human trials for Duchenne muscular dystrophy in boys.
Early-phase results showed increases in lean and muscle mass, but the program was stopped because of vascular safety signals — notably epistaxis (nosebleeds), gum bleeding, and dilated blood vessels (telangiectasias) — consistent with off-target effects on the ActRIIB/BMP pathway in vasculature. Its bodybuilding use is entirely off-label and based on the muscle-mass signal while ignoring the reason its trials ended.
02 Mechanism
A soluble ActRIIB-Fc fusion protein that binds and sequesters myostatin, activins and related TGF-beta ligands, blocking their inhibitory signalling and permitting muscle growth.
03 Dosing
| Tier | Dose | Route | Notes |
|---|---|---|---|
| Trial-derived | 1–3 mg/wk | SubQ | Trials dosed roughly 1-3 mg/kg every 2-4 weeks; per-kg dosing means figures here are indicative only. |
04 Effects
| Effect | Magnitude | Evidence | |
|---|---|---|---|
| Increased muscle volume / lean massIncreased thigh muscle volume and lean mass observed in DMD trial subjects. | Measurable increases in early trials | Clinical | |
| Raised bone/muscle biomarkersChanges consistent with anabolic and bone effects were seen. | Biomarker-level | Clinical |
05 Side effects
| Effect | Severity | Frequency | Evidence | Countermeasures |
|---|---|---|---|---|
| Dilated blood vessels / skin changesTelangiectasias (visible dilated vessels) observed. | Moderate | Reported in trials | Clinical | |
| Vascular bleeding (epistaxis, gum bleeding, telangiectasia)Nosebleeds, gum bleeding and dilated small vessels led to trial suspension. | Severe | Trial-limiting | Clinical |