ACE-031
Also known as ActRIIB-Fc, Ramatercept
A fusion protein of the activin type IIB receptor and an antibody Fc fragment that traps myostatin and related ligands. It was tested in Duchenne muscular dystrophy before its trial was stopped.
Clinical-stage research
Size
1 option- Catalog no.
- AE1
- Specification
- 1ml*10vials
- Per kit
- 10 vials
For laboratory research use only. Not for human or animal consumption. Buyers must be 21 or older.
Overview
ACE-031 is a soluble decoy receptor made by fusing the extracellular domain of activin receptor type IIB to the Fc portion of human IgG1. It binds myostatin and other TGF-beta family ligands that act through this receptor, preventing them from limiting muscle growth. In healthy volunteers, single doses increased lean mass and thigh muscle volume. A subsequent trial in boys with Duchenne muscular dystrophy was halted early because of nosebleeds and dilated small blood vessels. The compound was not developed further.
Class: Soluble activin receptor type IIB fusion protein
Mechanism of action
How the compound acts, as described in the literature.
Acts as a ligand trap: the soluble ActRIIB domain binds myostatin, activins and GDF-11 before they reach cell-surface receptors.
Blocking ActRIIB signaling reduces SMAD2/3-mediated inhibition of muscle growth, increasing muscle mass in animals and humans.
The IgG1-Fc fusion extends circulating half-life to roughly 10–15 days in human studies.
Because the trap also binds ligands such as BMP9 involved in vascular biology, effects beyond muscle can occur.
Published research
Selected studies, each linked to its PubMed record. Summaries describe what the studies reported.
2005
Rodent study
PMID 16330774Regulation of muscle growth by multiple ligands signaling through activin type II receptors.
Lee SJ, et al. · Proc Natl Acad Sci U S A
Showed that a soluble ActRIIB receptor increased muscle mass in mice beyond what myostatin loss alone produced, implicating multiple ligands.
2013
Phase 1 trial
PMID 23169607A single ascending-dose study of muscle regulator ACE-031 in healthy volunteers.
Attie KM, et al. · Muscle Nerve
In healthy postmenopausal women, single doses were generally well tolerated and increased lean body mass (3.3%) and thigh muscle volume (5.1%) at day 29 in the highest-dose group; injection-site erythema occurred.
2017
Phase 2 RCT
PMID 27462804Myostatin inhibitor ACE-031 treatment of ambulatory boys with Duchenne muscular dystrophy: Results of a randomized, placebo-controlled clinical trial.
Campbell C, et al. · Muscle Nerve
Showed non-significant trends toward maintained walking distance, lean mass and bone density, but the trial was stopped early because of epistaxis and telangiectasias.
Evidence & safety
What the research does not show, and the adverse events and regulatory status it reports.
The phase 2 trial in boys with Duchenne muscular dystrophy was stopped early for safety concerns, nosebleeds and telangiectasias (dilated small blood vessels), and the compound was not developed further. Efficacy findings were trends that did not reach statistical significance. It is not approved for any use.
Specifications
All sizes, exactly as catalogued.
| Catalog no. | Product | Specification | Vials per kit |
|---|---|---|---|
| AE1 | ACE-031 | 1ml*10vials | 10 |
For laboratory research use only. Not for human or animal consumption. Not intended to diagnose, treat, cure or prevent any disease. Research summaries are provided for educational purposes and do not describe this product’s use.