Acoramidis Evidence: Trial Results and Peer-Reviewed Publications

Hipa.ai Research · Source: PubMed & ClinicalTrials.gov / AACT · Last updated:

The clinical evidence base for Acoramidis comprises 11 peer-reviewed publications across 8 journals, 1 pivotal-trial primary-outcome rows reported to ClinicalTrials.gov, spanning indications including Amyloid Neuropathies, Familial. Most recent publication: Efficacy of Acoramidis in Adults With Transthyretin Amyloid Cardiomyopathy: Propensity Score Comparison Between a Single-Arm Open-Label Phase 3 Trial in Japan and a Global Double-Blind Phase 3 Trial., Circ Rep, 2026.

Top peer-reviewed publications

Curated set of pivotal-trial result papers and recent publications in high-tier journals.

  1. Efficacy of Acoramidis in Adults With Transthyretin Amyloid Cardiomyopathy: Propensity Score Comparison Between a Single-Arm Open-Label Phase 3 Trial in Japan and a Global Double-Blind Phase 3 Trial.
    Tahara N, Raizner A, Chen Y, et al. · Circ Rep · 2026
    PubMed: PMID 42110872 · NCT03860935 (ATTRibute-CM) · Amyloid Neuropathies, Familial
  2. Comparison of in-clinic assessment of 6MWT by conventional method and using wearable sensors for patients with ATTR-CM.
    Soman P, Khouri MG, Lenihan D, et al. · Future Cardiol · 2025
    PubMed: PMID 39878480 · NCT03860935 (ATTRibute-CM) · Amyloid Neuropathies, Familial
  3. Long-Term Durability of Acoramidis Efficacy in Transthyretin Amyloid Cardiomyopathy: Open-Label Extension of the ATTRibute-CM Randomized Clinical Trial.
    Soman P, Cuddy SAM, Gillmore JD, et al. · JAMA Cardiol · 2026
    PubMed: PMID 41911489 · NCT04988386 (AG10) · Amyloid Neuropathies, Familial
  4. Early Increase in Serum Transthyretin by Acoramidis Independently Predicts Improved Survival in TTR Amyloid Cardiomyopathy.
    Maurer MS, Judge DP, Gillmore JD, et al. · J Am Coll Cardiol · 2025
    PubMed: PMID 40398971 · NCT03860935 (ATTRibute-CM) · Amyloid Neuropathies, Familial
  5. Long-Term Efficacy and Safety of Acoramidis in ATTR-CM: Initial Report From the Open-Label Extension of the ATTRibute-CM Trial.
    Judge DP, Gillmore JD, Alexander KM, et al. · Circulation · 2025
    PubMed: PMID 39556242 · NCT04988386 (AG10) · Amyloid Neuropathies, Familial
  6. Efficacy and Safety of Acoramidis in Transthyretin Amyloid Cardiomyopathy.
    Gillmore JD, Judge DP, Cappelli F, et al. · N Engl J Med · 2024
    PubMed: PMID 38197816 · NCT03860935 (ATTRibute-CM) · Amyloid Neuropathies, Familial
  7. A plain language review of the ATTRibute-CM study: efficacy and safety of acoramidis in transthyretin amyloid cardiomyopathy.
    Gillmore JD, Judge DP, Cappelli F, et al. · Future Cardiol · 2026
    PubMed: PMID 41384357 · NCT03860935 (ATTRibute-CM) · Amyloid Neuropathies, Familial
  8. Efficacy of Acoramidis in Wild-Type and Variant Transthyretin Amyloid Cardiomyopathy: Results From ATTRibute-CM and Its Open-Label Extension.
    Alexander KM, Davis MK, Akinboboye O, et al. · JAMA Cardiol · 2026
    PubMed: PMID 41205147 · NCT04988386 (AG10) · Amyloid Neuropathies, Familial

Primary-outcome results across pivotal trials

Per-arm reported values from Phase 2/3 and Phase 3 trials with results posted to ClinicalTrials.gov.

TrialIndicationPrimary endpointArmValue
NCT03860935
ATTRibute-CM
Amyloid Neuropathies, FamilialA Hierarchical Combination of All-Cause Mortality, Cumulative Frequency of CV-related Hospitalization, Change From Baseline in NT-proBNP and Change From Baseline in 6MWT at the Last Available Visit Where Both Subjects Had Non-missing Assessments.
Baseline up to Month 30
Acoramidis HCl 800 mg63.7 Percent of Wins from Win Ratio
Placebo35.9 Percent of Wins from Win Ratio

Publications by year

2021–2026: 11 publications.

2021
1
2024
1
2025
5
2026
4

Publications by indication

Amyloid Neuropathies, Familial (11)

  • Efficacy of Acoramidis in Adults With Transthyretin Amyloid Cardiomyopathy: Propensity Score Comparison Between a Single-Arm Open-Label Phase 3 Trial in Japan and a Global Double-Blind Phase 3 Trial.
    Circ Rep · 2026 · PMID 42110872 · NCT03860935
  • Long-Term Durability of Acoramidis Efficacy in Transthyretin Amyloid Cardiomyopathy: Open-Label Extension of the ATTRibute-CM Randomized Clinical Trial.
    JAMA Cardiol · 2026 · PMID 41911489 · NCT04988386
  • A plain language review of the ATTRibute-CM study: efficacy and safety of acoramidis in transthyretin amyloid cardiomyopathy.
    Future Cardiol · 2026 · PMID 41384357 · NCT03860935
  • Efficacy of Acoramidis in Wild-Type and Variant Transthyretin Amyloid Cardiomyopathy: Results From ATTRibute-CM and Its Open-Label Extension.
    JAMA Cardiol · 2026 · PMID 41205147 · NCT04988386
  • Comparison of in-clinic assessment of 6MWT by conventional method and using wearable sensors for patients with ATTR-CM.
    Future Cardiol · 2025 · PMID 39878480 · NCT03860935

Publications by journal

Trial-results highlights

Acoramidis is a medication that has been investigated in clinical trials for the treatment of familial amyloid neuropathies, a specific type of amyloidosis. A pivotal study, known as ATTRibute-CM and identified as NCT03860935, evaluated the effectiveness of Acoramidis HCl 800 mg when compared to a placebo. This trial observed participants and assessed various outcomes over a period extending from baseline measurements up to Month 30.

The primary outcome in NCT03860935 was designed as a comprehensive hierarchical endpoint. This endpoint was determined at the last available visit where both subjects had non-missing data, and it combined several key clinical measures:

  • All-cause mortality
  • The cumulative frequency of cardiovascular-related hospitalization
  • The change from baseline in N-terminal pro-B-type natriuretic peptide (NT-proBNP)
  • The change from baseline in the 6-minute walk test (6MWT)
For this specific primary outcome, the arm receiving Acoramidis HCl 800 mg achieved 63.7 Percent of Wins from the Win Ratio. In contrast, the placebo arm in NCT03860935 reported 35.9 Percent of Wins from the Win Ratio for the same endpoint.

All values are sourced from primary registry reporting, and individual published papers should be consulted for clinical decisions.

All Acoramidis publications (11)

2026 (4 papers)

  1. Efficacy of Acoramidis in Adults With Transthyretin Amyloid Cardiomyopathy: Propensity Score Comparison Between a Single-Arm Open-Label Phase 3 Trial in Japan and a Global Double-Blind Phase 3 Trial.
    Tahara N, Raizner A, Chen Y, et al. · Circ Rep · 2026 · Derived
    PubMed: PMID 42110872 · NCT03860935 (ATTRibute-CM) · Amyloid Neuropathies, Familial
  2. Long-Term Durability of Acoramidis Efficacy in Transthyretin Amyloid Cardiomyopathy: Open-Label Extension of the ATTRibute-CM Randomized Clinical Trial.
    Soman P, Cuddy SAM, Gillmore JD, et al. · JAMA Cardiol · 2026 · Derived
    PubMed: PMID 41911489 · NCT04988386 (AG10) · Amyloid Neuropathies, Familial
  3. A plain language review of the ATTRibute-CM study: efficacy and safety of acoramidis in transthyretin amyloid cardiomyopathy.
    Gillmore JD, Judge DP, Cappelli F, et al. · Future Cardiol · 2026 · Derived
    PubMed: PMID 41384357 · NCT03860935 (ATTRibute-CM) · Amyloid Neuropathies, Familial
  4. Efficacy of Acoramidis in Wild-Type and Variant Transthyretin Amyloid Cardiomyopathy: Results From ATTRibute-CM and Its Open-Label Extension.
    Alexander KM, Davis MK, Akinboboye O, et al. · JAMA Cardiol · 2026 · Derived
    PubMed: PMID 41205147 · NCT04988386 (AG10) · Amyloid Neuropathies, Familial

2025 (5 papers)

  1. Comparison of in-clinic assessment of 6MWT by conventional method and using wearable sensors for patients with ATTR-CM.
    Soman P, Khouri MG, Lenihan D, et al. · Future Cardiol · 2025 · Derived
    PubMed: PMID 39878480 · NCT03860935 (ATTRibute-CM) · Amyloid Neuropathies, Familial
  2. Early Increase in Serum Transthyretin by Acoramidis Independently Predicts Improved Survival in TTR Amyloid Cardiomyopathy.
    Maurer MS, Judge DP, Gillmore JD, et al. · J Am Coll Cardiol · 2025 · Derived
    PubMed: PMID 40398971 · NCT03860935 (ATTRibute-CM) · Amyloid Neuropathies, Familial
  3. Long-Term Efficacy and Safety of Acoramidis in ATTR-CM: Initial Report From the Open-Label Extension of the ATTRibute-CM Trial.
    Judge DP, Gillmore JD, Alexander KM, et al. · Circulation · 2025 · Derived
    PubMed: PMID 39556242 · NCT04988386 (AG10) · Amyloid Neuropathies, Familial
  4. Efficacy of Acoramidis on All-Cause Mortality and Cardiovascular Hospitalization in Transthyretin Amyloid Cardiomyopathy.
    Judge DP, Alexander KM, Cappelli F, et al. · J Am Coll Cardiol · 2025 · Derived
    PubMed: PMID 40074465 · NCT03860935 (ATTRibute-CM) · Amyloid Neuropathies, Familial
  5. Contemporary Oral Medication Use and Frequency in Patients with Transthyretin Amyloid Cardiomyopathy.
    Dasgupta N, Poulsen SH, Emdin M, et al. · Am J Cardiovasc Drugs · 2025 · Derived
    PubMed: PMID 40750720 · NCT03860935 (ATTRibute-CM) · Amyloid Neuropathies, Familial

2024 (1 paper)

  1. Efficacy and Safety of Acoramidis in Transthyretin Amyloid Cardiomyopathy.
    Gillmore JD, Judge DP, Cappelli F, et al. · N Engl J Med · 2024 · Derived
    PubMed: PMID 38197816 · NCT03860935 (ATTRibute-CM) · Amyloid Neuropathies, Familial

2021 (1 paper)

  1. Updates in Cardiac Amyloidosis Diagnosis and Treatment.
    Stern LK, Kittleson MM · Curr Oncol Rep · 2021 · Derived
    PubMed: PMID 33725199 · NCT03860935 (ATTRibute-CM) · Amyloid Neuropathies, Familial

Sources and methodology

This page summarizes published evidence for general reference and does not constitute medical advice. For clinical decisions, consult the linked primary publications and your healthcare provider. Data sourced from PubMed and the ClinicalTrials.gov / AACT database maintained by the Clinical Trials Transformation Initiative (CTTI).