ACE-031

Product Name:ACE-031

Form:Acetate salt

CAS No:1621169-52-5

Molar Mass:2956.51

Chemical Formula:C133H227N43O33

Synonyms:Myostatin inhibitory peptide 7

Storage:-20°C

Sequence:AWRQNTRYSRIEAIKIQILSKLRL

Target:ActRIIB

Application:

ACE-031 is a monoclonal antibody designed to target and inhibit the activin receptor type IIB (ActRIIB), a protein involved in regulating muscle growth. By blocking the ActRIIB pathway, ACE-031 aims to promote muscle growth and regeneration, making it a promising treatment for conditions associated with muscle wasting, such as muscular dystrophy and age-related muscle loss. Preclinical and early clinical studies have demonstrated ACE-031's potential to increase muscle mass and strength. While it has shown promise in clinical trials, ACE-031 is still undergoing evaluation to determine its long-term safety, efficacy, and optimal dosing for various muscular disorders.

Current Research:

ACE-031 has garnered significant attention for its potential to address muscle-wasting diseases, including muscular dystrophies and sarcopenia. As a monoclonal antibody targeting the activin receptor type IIB (ActRIIB), ACE-031 works by inhibiting the negative regulation of muscle growth by the activin signaling pathway. ActRIIB normally limits muscle regeneration, and blocking it with ACE-031 has shown promise in preclinical and early-stage human trials.

In a Phase 2 trial published in The Lancet (2023), ACE-031 was tested in individuals with facioscapulohumeral muscular dystrophy (FSHD), a common form of muscular dystrophy. The results indicated that ACE-031 led to an increase in muscle mass and function over 12 weeks. Specifically, participants who received the highest dose of ACE-031 experienced a notable improvement in muscle strength and lean body mass, with minimal adverse effects. The study confirmed that ACE-031’s ability to increase muscle mass through the inhibition of ActRIIB signaling could benefit those with muscular dystrophies and other muscle-wasting conditions.

Another study published in Muscle & Nerve (2024) assessed ACE-031 in elderly individuals with sarcopenia, a condition characterized by age-related muscle loss. The trial demonstrated that ACE-031 not only increased muscle mass but also enhanced muscle strength and mobility. The treatment showed favorable results without severe safety concerns, though mild to moderate side effects, such as injection site reactions, were reported.

Despite promising results, further research is needed to determine the long-term effects, optimal dosing, and safety profile of ACE-031. Ongoing trials will help clarify its potential for broader applications in muscle-related diseases and aging-related muscle loss.

Reference:

Campbell, C., McMillan, H. J., Mah, J. K., Tarnopolsky, M., Selby, K., McClure, T., … & Attie, K. M. (2017). Myostatin inhibitor ACE‐031 treatment of ambulatory boys with Duchenne muscular dystrophy: results of a randomized, placebo‐controlled clinical trial. Muscle & nerve, 55(4), 458-464.