Last reviewed · How we verify
ACE-031
Activates the activin receptor type II-A (ActRIIA), leading to increased myostatin inhibition.
ACE-031 is an investigational drug developed by Acceleron Pharma, now part of Merck & Co., for the treatment of Duchenne Muscular Dystrophy. Despite promising early results, Phase 2 trials were terminated due to safety concerns. The drug is a fusion protein that targets the ActRIIB receptor, aiming to increase muscle mass and strength.
At a glance
| Generic name | ACE-031 |
|---|---|
| Also known as | ActRIIB-IgG1 |
| Sponsor | Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA |
| Drug class | Myostatin inhibitor |
| Target | Activin receptor type II-A (ActRIIA) |
| Modality | Biologic |
| Therapeutic area | Oncology |
| Phase | Phase 1 |
Mechanism of action
ACE-031 is a soluble form of the activin receptor type II-A (ActRIIA) that acts as a decoy receptor to inhibit myostatin, a protein that limits muscle growth and repair.
Approved indications
- Muscular dystrophy
- Muscle wasting
Common side effects
- Injection site reaction
- Fatigue
- Headache
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape: