Product Description
Acoramidis (AG10) is an investigational, orally-administered small molecule designed to potently stabilize tetrameric transthyretin (TTR). Acoramidis?was designed to mimic a?naturally-occurring?variant of the TTR gene (T119M) that is considered a rescue mutation because it has been shown to prevent or minimize ATTR in individuals carrying pathogenic, or disease-causing, mutations in the TTR gene.
Mechanisms of Action: TTR Stabilizer
Novel Mechanism: No
Modality: Small Molecule
Route of Administration: Oral
FDA Designation: None *
Approval Status: Approved
Approved Countries: Chile | Dominican Republic | Germany | Spain
Approved Indications: None
Known Adverse Events: None
Company: BridgeBio
Company Location: PALO ALTO CA 94301
Company CEO: Neil Kumar
Additional Commercial Interests: None
Clinical Description
Countries in Clinic: Canada, Australia, Belgium, Brazil, Bulgaria, Czech Republic, Denmark, France, Germany, Greece, Ireland, Israel, Italy, Japan, Korea, Netherlands, New Zealand, Poland, Portugal, Spain, United Kingdom, United States
Active Clinical Trial Count: 9
Highest Development Phases
Phase 3: Amyloid Neuropathies, Familial|Amyloidosis, Familial|Cardiomyopathies|Polyneuropathies|Transthyretin Amyloidosis
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
|---|---|---|---|---|---|
| AG10-501 | P3 |
Recruiting |
Transthyretin Amyloidosis |
2031-11-30 |
|
| ACT-EARLY Trial | P3 |
Recruiting |
Amyloidosis, Familial|Transthyretin Amyloidosis|Polyneuropathies|Amyloid Neuropathies, Familial|Cardiomyopathies |
2031-10-01 |
|
| AG10-304 | P3 |
Active, not recruiting |
Transthyretin Amyloidosis |
2028-05-09 |
|
| AG10-301 | P3 |
Active, not recruiting |
Cardiomyopathies|Transthyretin Amyloidosis |
2028-04-15 |