Acoramidis Clinical Trials

Acoramidis Clinical Trials

NOTE: The task description references "NCT06563895" as an Alzheimer's disease trial, but this trial is actually for transthyretin (TTR) amyloidosis prevention — NOT Alzheimer's disease. This page documents the actual Acoramidis trials.

Overview

Acoramidis (formerly known as AG10, ALXN2060) is a near-complete (>90%) transthyretin (TTR) stabilizer developed by Eidos Therapeutics, a BridgeBio company. It is designed to mimic the protective properties of the naturally occurring TTR T119M mutation, which prevents TTR from dissociating and forming amyloid deposits.

Acoramidis is being developed for the treatment of TTR amyloidosis, including:

• ATTR-CM: Transthyretin amyloid cardiomyopathy
• ATTR-PN: Transthyretin amyloid polyneuropathy
• Prevention: Asymptomatic TTR variant carriers

Mechanism of Action

Acoramidis binds to the thyroxine binding site on the TTR tetramer, stabilizing the native tetrameric structure and preventing dissociation into monomers — the rate-limiting step in TTR amyloidogenesis.

Key Mechanism Points

• Near-complete stabilization: >90% TTR stabilization across entire dosing interval
• Mimics T119M mutation: Exploits naturally occurring protective mutation
• Oral administration: Twice-daily dosing
• Prevents both cardiac and neurological manifestations

Acoramidis Clinical Trials

NOTE: The task description references "NCT06563895" as an Alzheimer's disease trial, but this trial is actually for transthyretin (TTR) amyloidosis prevention — NOT Alzheimer's disease. This page documents the actual Acoramidis trials.

Overview

Acoramidis (formerly known as AG10, ALXN2060) is a near-complete (>90%) transthyretin (TTR) stabilizer developed by Eidos Therapeutics, a BridgeBio company. It is designed to mimic the protective properties of the naturally occurring TTR T119M mutation, which prevents TTR from dissociating and forming amyloid deposits.

Acoramidis is being developed for the treatment of TTR amyloidosis, including:

• ATTR-CM: Transthyretin amyloid cardiomyopathy
• ATTR-PN: Transthyretin amyloid polyneuropathy
• Prevention: Asymptomatic TTR variant carriers

Mechanism of Action

Acoramidis binds to the thyroxine binding site on the TTR tetramer, stabilizing the native tetrameric structure and preventing dissociation into monomers — the rate-limiting step in TTR amyloidogenesis.

Key Mechanism Points

• Near-complete stabilization: >90% TTR stabilization across entire dosing interval
• Mimics T119M mutation: Exploits naturally occurring protective mutation
• Oral administration: Twice-daily dosing
• Prevents both cardiac and neurological manifestations

Clinical Trials

ACT-EARLY Prevention Trial (NCT06563895)

| Field | Value |
|-------|-------|
| Trial ID | NCT06563895 |
| Phase | Phase 3 |
| Sponsor | Eidos Therapeutics (BridgeBio) |
| Status | RECRUITING |
| Enrollment | 582 (estimated) |
| Start Date | May 12, 2025 |
| Primary Completion | October 2031 |
| Completion Date | December 2032 |

Study Design: Randomized, multicenter, double-blind, placebo-controlled, parallel-group, quadruple-masked Phase 3 trial for prevention in asymptomatic TTR variant carriers.

Primary Outcome: Time to development of ATTR (ATTR-CM or ATTR-PN, whichever occurs first, centrally adjudicated) over approximately 7 years.

Secondary Outcomes:

• Time to development of ATTR-CM (centrally adjudicated)
• Time to development of ATTR-PN (centrally adjudicated)

ATTRibute-CM (NCT04839487)

| Field | Value |
|-------|-------|
| Trial ID | NCT04839487 |
| Phase | Phase 3 |
| Sponsor | Eidos Therapeutics (BridgeBio) |
| Status | COMPLETED |
| Enrollment | 421 |

Results: Acoramidis resulted in a significantly better four-step primary hierarchical outcome containing components of mortality, morbidity, and function than placebo at 30 months in participants with ATTR-CM.

Other Acoramidis Trials

| Trial ID | Phase | Status | Indication |
|----------|-------|--------|------------|
| NCT04769479 | PK | Completed | Pharmacokinetics in healthy subjects |
| NCT04988386 | OLE | Active | Open-label extension in ATTR-CM |
| NCT04958135 | PK | Completed | Food effect study |
| NCT07116473 | OLE | Active | ACT-EARLY OLE for newly diagnosed ATTR-CM |

Clinical Development Timeline

| Year | Milestone |
|------|-----------|
| Phase 1 | Near-complete (>90%) TTR stabilization achieved |
| Phase 2 + OLE | Long-term treatment well-tolerated; sustained TTR stabilization |
| Phase 3 (ATTRibute-CM) | Positive results at 30 months |
| 2025 | ACT-EARLY prevention trial initiated |

No Alzheimer's Disease Trial

Important Note: Despite the task description referencing an "Alzheimer's disease" trial, there is currently no registered ClinicalTrials.gov study investigating Acoramidis for Alzheimer's disease or any other CNS/neurodegenerative disease beyond TTR amyloidosis.

The NCT06563895 trial is specifically for:

• Prevention of ATTR in asymptomatic TTR variant carriers
• Cardiac and peripheral neurological outcomes (ATTR-CM, ATTR-PN)
• NOT for Alzheimer's disease

See Also

• [TTR Protein](/proteins/ttr-protein)
• [TTR Gene](/genes/ttr)
• [Hereditary Transthyretin Amyloidosis](/diseases/hereditary-transthyretin-amyloidosis)
• [TTR Stabilizers](/therapeutics/ttr-stabilizers)
• [Amyloid Cascade Pathway](/mechanisms/amyloid-cascade)
• [Protein Misfolding Disorders](/mechanisms/protein-misfolding)

External Links

• [ClinicalTrials.gov - Acoramidis Studies](https://clinicaltrials.gov/search?query.term=Acoramidis)
• [BridgeBio Pharma - Acoramidis](https://www.bridgebio.com/pipeline)
• [Eidos Therapeutics](https://www.eidosrx.com/)

References