What is ACE-031?
ACE-031 was a soluble form of ActRIIB (Activin Receptor Type IIB) developed by Acceleron Pharma as a myostatin inhibitor for muscular dystrophy. It acts as a "trap" for myostatin and related ligands, preventing them from inhibiting muscle growth.
Development History
- Developed for Duchenne muscular dystrophy
- Phase 2 clinical trials conducted
- Discontinued due to safety signals
- Primarily nosebleeds and gum bleeding
- Development halted in 2011
Mechanism
Ligand Trap
ACE-031 works by:
- Circulating as soluble receptor
- Binding myostatin and GDFs
- Preventing muscle inhibition
- Allowing enhanced muscle growth
Clinical Results (Before Discontinuation)
Phase 2 showed:
- Increased lean mass
- Improved muscle volume
- But safety concerns emerged
- Bleeding-related adverse events
Current Status
Discontinued
- Development stopped
- Safety concerns unresolved
- Related compounds in development
- Acceleron pursued other programs
Summary
Key Points:
- Classification: ActRIIB-Fc fusion
- Mechanism: Myostatin trap
- Status: DISCONTINUED
- Reason: Safety concerns (bleeding)