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Acoramidis

Alternative Names: acoramidis, ag-10, ag10, ag 10, ALXN2060, ALXN-2060
Latest Update: 2024-12-16
Latest Update Note: Clinical Trial Update

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: Not 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
Additonal Commercial Interests: None

Clinical Description

Map of Global Clinical Trials for Acoramidis

Countries in Clinic: Australia, Belgium, Brazil, Canada, Czech Republic, Denmark, Greece, Ireland, Israel, Italy, Japan, Korea, Netherlands, New Zealand, Poland, Portugal, Spain, United Kingdom, United States

Active Clinical Trial Count: 8

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

ACT-EARLY Trial

P3

Recruiting

Amyloid Neuropathies, Familial|Polyneuropathies|Transthyretin Amyloidosis|Amyloidosis, Familial|Cardiomyopathies

2031-10-01

AG10-304

P3

Unknown Status

Transthyretin Amyloidosis

2028-05-09

AG10-301

P3

Active, not recruiting

Cardiomyopathies|Transthyretin Amyloidosis

2028-04-15

AG10-202

P2

Active, not recruiting

Cardiomyopathies

2027-07-01

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