Evidence map›Paper›PMID 42110872›Full record

ArticleCirculation reports2026

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.

Nobuhiro Tahara, Aileen Raizner, Yi Chen, Michele F Mercuri, Masatoshi Minamisawa, Phase 3 Japan Study Investigators

2 registry-linked trialsAbstract read
In one paragraph

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 2 registered trials, which are not on this map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

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.

2 · The registry

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.

NCT03860935 phase3completednot on this map

A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of the Efficacy and Safety of AG10 in Subjects With Symptomatic Transthyretin Amyloid Cardiomyopathy (ATTRibute-CM Trial)

TypeinterventionalSponsorEidos Therapeutics, a BridgeBio companyRan2019 to 2023Enrolled632ConditionsAmyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis, CardiomyopathiesArmsacoramidis, Placebo Oral Tablet
NCT04622046 phase3completednot on this map

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)

TypeinterventionalSponsorAlexion Pharmaceuticals, Inc.Ran2020 to 2025Enrolled25ConditionsSymptomatic Transthyretin Amyloid CardiomyopathyArmsALXN2060
3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

6 authors.

Nobuhiro TaharaDivision of Cardiovascular Medicine, Department of Medicine, Kurume University School of Medicine Fukuoka Japan.
Aileen RaiznerClinical Development, Alexion, AstraZeneca Rare Disease Boston, MA USA.
Yi ChenBiostatistics, Alexion, AstraZeneca Rare Disease Boston, MA USA.
Michele F MercuriClinical Development, Alexion, AstraZeneca Rare Disease Boston, MA USA.
Masatoshi MinamisawaClinical Development, Alexion, AstraZeneca Rare Disease Boston, MA USA.
Phase 3 Japan Study Investigators

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Background: Acoramidis is an oral, highly selective transthyretin (TTR) stabilizer approved for transthyretin amyloid cardiomyopathy (ATTR-CM). This analysis compared efficacy outcomes over a 30-month period between acoramidis-treated patients from the Phase 3 Japan Study (NCT04622046) with propensity score (PS)-matched placebo patients from the global ATTRibute-CM trial (NCT03860935). Methods and Results: In the modified intention-to-treat population, PS matching using an optimal 1 : 2 matching ratio (absolute standardized mean difference ≤0.25 for all covariates) was used to match 25 acoramidis-treated Japanese patients with 50 placebo-treated patients from ATTRibute-CM. Baseline covariates for PS matching were age, sex, body mass index, New York Heart Association class, ATTR-CM genetic status, N-terminal pro-B-type natriuretic peptide (NT-proBNP) level, and 6-min walk distance (6MWD). There were no deaths reported with acoramidis over 30 months, compared with 24% all-cause mortality events among PS-matched placebo patients. There was an 81% reduction in the annualized frequency of cardiovascular-related hospitalizations with acoramidis compared with the PS-matched placebo group. The degree of worsening in 6MWD, Kansas City Cardiomyopathy Questionnaire overall score, and NT-proBNP was less with acoramidis than with placebo. Acoramidis produced a sustained increase in TTR levels and median TTR stabilization assessed using a fluorescent probe exclusion assay. Conclusions: Clinically meaningful benefits were observed favoring acoramidis over placebo (ATTRibute-CM trial) in survival, cardiovascular-related hospitalizations, functional endpoints, and quality of life assessments.

Indexed as

AcoramidisATTRibute-CMEfficacyPropensity scoreTransthyretin amyloid cardiomyopathy (ATTR-CM)

Identifiers

PMID42110872
PMCPMC13153671

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Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.