ACE-031

Third-Party Tested

Independent lab verified

Batch-Specific CoA

Publicly accessible

YPB.249

Reference number

1mg

Lyophilized vial

Origin

ACE-031 was developed by Acceleron Pharma (now part of Merck) as a soluble activin receptor type IIB (ActRIIB) Fc fusion protein. It was designed to act as a ligand trap, binding and sequestering myostatin, activin A, GDF-11, and other TGF-beta superfamily ligands that normally signal through ActRIIB to limit muscle growth.

Research Lineage

Attie et al. published Phase I clinical results in 2013, demonstrating increases in lean body mass and thigh muscle volume. A Phase II trial in Duchenne muscular dystrophy patients was initiated but discontinued due to epistaxis and telangiectasia side effects, attributed to off-target inhibition of BMP9 signaling. The compound continues to be used as a research tool for understanding ActRIIB signaling.

Mechanism of Action

ACE-031 consists of the extracellular domain of human ActRIIB fused to an IgG1 Fc domain. It acts as a soluble decoy receptor, binding circulating myostatin, activin A, and GDF-11 with high affinity and preventing them from activating cell-surface ActRIIB. This removes the negative regulatory signal on muscle growth, resulting in hypertrophy through de-repression of the Akt/mTOR anabolic pathway.

Structural Notes

Recombinant fusion protein. ActRIIB extracellular domain (residues 19-134) fused to human IgG1 Fc. Homodimer. Molecular weight: ~90 kDa.