ACE-031 1mg

ACE-031 (Activin Type IIB Receptor-Fc Fusion Protein)

ACE-031 is a recombinant fusion protein composed of the extracellular domain of the human activin receptor type IIB (ActRIIB) linked to the Fc domain of human IgG1. It acts as a soluble decoy receptor that binds to activins and myostatin (GDF-8), thereby inhibiting their signaling through endogenous ActRIIB receptors. This mechanism increases muascle mass and strength in preclinical models by reducing negative regulation of skeletal muscle growth.

Overview

ACE-031 was designed to block signaling from members of the transforming growth factor-β (TGF-β) superfamily, primarily myostatin and activins, which suppress muscle growth and differentiation. By sequestering these ligands, ACE-031 prevents their interaction with cell-surface receptors, leading to enhanced myogenesis and hypertrophy in muscle tissue.
Research applications focus on muscle wasting, sarcopenia, cachexia, and neuromuscular disorders such as Duchenne muscular dystrophy (DMD). The molecule serves as a critical research tool in studying the ActRIIB–myostatin pathway and anabolic regulation in skeletal muscle.

Chemical Makeup

• Type: Recombinant human fusion protein
• Structure: Soluble extracellular domain of ActRIIB linked to human IgG1 Fc
• Molecular Weight: ~95–100 kDa (monomeric)
• Expression System: Recombinant CHO cells
• Purity: ≥98% (per COA)
• Form: Lyophilized protein
• Size: 1 mg, 2 mg, and 5 mg vials available

Research and Clinical Studies

Myostatin and Activin Inhibition
ACE-031 binds circulating myostatin and activins, neutralizing their inhibitory effects on skeletal muscle growth. Preclinical models demonstrate increased muscle fiber diameter, lean mass, and grip strength following administration.

Muscle and Bone Metabolism Research
In addition to muscle effects, ACE-031 has been shown to increase bone mineral density and improve markers of bone formation, suggesting cross-talk between muscle and bone anabolic pathways.

Neuromuscular Disease Models
ACE-031 has been evaluated in mouse and primate models of Duchenne muscular dystrophy (DMD), where it improved muscle mass and functional outcomes, supporting its use in studies of muscle degenerative disease mechanisms.

Metabolic and Energy Balance
By increasing lean body mass and reducing adiposity, ACE-031 serves as a model compound in research investigating muscle metabolism, insulin sensitivity, and energy expenditure.

Pharmacokinetics
ACE-031 exhibits an extended circulating half-life due to the Fc fusion, allowing for intermittent dosing in experimental protocols (typically 3–4 days in rodents; longer in primates).

ACE-031 is available for research and laboratory purposes only. Not for human consumption.

References

1. Lee SJ, et al. Regulation of muscle mass by myostatin and activin signaling. Nat Rev Mol Cell Biol. 2020;21(5):269–280. https://pubmed.ncbi.nlm.nih.gov/32152592/
   
2. Lach-Trifilieff E, et al. Inhibition of ActRIIB signaling increases muscle mass and strength. Proc Natl Acad Sci U S A. 2014;111(17):E1774–E1782. https://pubmed.ncbi.nlm.nih.gov/24706799/
   
3. Cadena SM, et al. Administration of ACE-031 increases muscle mass and reduces fat in preclinical models. Am J Physiol Endocrinol Metab. 2010;299(6):E965–E974. https://pubmed.ncbi.nlm.nih.gov/20858706/
   
4. Campbell C, et al. Pharmacologic blockade of myostatin in Duchenne muscular dystrophy: results of a clinical trial with ACE-031. Muscle Nerve. 2017;55(4):458–464. https://pubmed.ncbi.nlm.nih.gov/27560668/
   
5. Lawlor MW, et al. Inhibition of myostatin signaling improves muscle function in DMD models. Skelet Muscle. 2011;1(1):34. https://pubmed.ncbi.nlm.nih.gov/22087763/
   
6. Rodino-Klapac LR, et al. Gene therapy and myostatin blockade in muscle disorders. Mol Ther. 2013;21(1):132–140. https://pubmed.ncbi.nlm.nih.gov/23151419/
   
7. Ploquin C, et al. ActRIIB blockade alters muscle and bone composition in primates. J Endocrinol. 2012;213(2):183–195. https://pubmed.ncbi.nlm.nih.gov/22495698/
   
8. Morine KJ, et al. Systemic inhibition of activin type II receptors promotes muscle growth and fat loss. Am J Physiol Endocrinol Metab. 2010;299(5):E776–E788. https://pubmed.ncbi.nlm.nih.gov/20858707/
   
9. Attie KM, et al. Clinical pharmacology of ACE-031 in healthy volunteers. Muscle Nerve. 2013;47(3):416–423. https://pubmed.ncbi.nlm.nih.gov/23335387/
   
10. Campbell C, et al. Safety and pharmacodynamic effects of ACE-031 in DMD boys: phase 1 study. Neurology. 2012;78(8):627–635. https://pubmed.ncbi.nlm.nih.gov/22357796/