ArticleCirculation reports2026
Efficacy and Safety of Acoramidis in Japanese Adults With Symptomatic Transthyretin Amyloid Cardiomyopathy.
Article in Circulation reports, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT04622046 (A Phase 3, Prospective, Multicenter, Open Label, 2-Part Study of the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of ALXN2060 in Japanese Participants With Symptomatic Transthyretin Amyloid Cardiomyopathy), which is not on this map. Cited by 3 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Phase 3, Prospective, Multicenter, Open Label, 2-Part Study of the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of ALXN2060 in Japanese Participants With Symptomatic Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
Who cites it
3 citing papers in PubMed.
- Clinical Factors Related to Cardiac Conduction Disturbance in Patients With Wild-Type Transthyretin Amyloid Cardiomyopathy Undergoing Tafamidis Treatment.Circulation reports · 2026Article
- Disease-Modifying Therapies for Transthyretin Amyloid Cardiomyopathy: Current Evidence and Emerging Strategies.Journal of clinical medicine research · 2026Review
- 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.Circulation reports · 2026Article
Corrections and comments
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Authors and funding
18 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Background: This multicenter open-label single-arm 2-part prospective Phase 3 trial (NCT04622046) evaluated the efficacy, safety, pharmacokinetics, and pharmacodynamics of acoramidis, an oral, highly selective transthyretin (TTR) stabilizer, in Japanese patients with wild-type or hereditary symptomatic transthyretin amyloid cardiomyopathy (ATTR-CM). Methods and Results: Twenty-five patients (mean [±SD] age 76.5±6.3 years; 88% men) received acoramidis hydrochloride (800 mg twice daily) for up to 30 months. Primary endpoints were change from baseline to Month 12 in the 6-min walk distance (6MWD) and all-cause mortality and cardiovascular-related hospitalizations over 30 months. No deaths were reported over 30 months. The annualized frequency of cardiovascular-related hospitalizations was 0.133. Clinically meaningful treatment benefits were observed in least-squares mean change from baseline to Month 12 for 6MWD (-3.9 m; 95% confidence interval [CI] -22.9, 15.1 m) and to Month 30 for 6MWD (-36.2 m; 95% CI -58.5 to -13.9 m), Kansas City Cardiomyopathy Questionnaire overall score (-7.0; 95% CI -14.7, 0.8), TTR levels (9.4 mg/dL; 95% CI 6.7, 12.0 mg/dL), and median N-terminal pro B-type natriuretic peptide levels (169.5 pg/mL; interquartile range -557.0, 700.0 pg/mL). The median TTR stabilization (fluorescent probe exclusion assay) was 95.6%. Acoramidis was well tolerated. Conclusions: Acoramidis prolongs survival, provides functional and quality-of-life benefits, and is well tolerated in Japanese patients with ATTR-CM, consistent with results from the global ATTRibute-CM Phase 3 trial.
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