Evidence map›Paper›PMID 42307157›Full record

ArticleJACC. CardioOncology2026

Differential Transthyretin Stabilization in Patients With Wild Type and Variant Transthyretin Amyloidosis.

Daniel P Judge, Alan X Ji, Isabella A Graef, Martha Grogan, Prem Soman, Francesco Cappelli, Marianna Fontana, Ahmad Masri, Julian D Gillmore, Pablo Garcia-Pavia and 3 more

Registry-linked trialAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in JACC. CardioOncology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03860935 (A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of the Efficacy and Safety of AG10 in Subjects With Symptomatic Transthyretin Amyloid Cardiomyopathy), which is 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
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

13 authors.

Daniel P JudgeMedical University of South Carolina, Charleston, South Carolina, USA. Electronic address: judged@musc.edu.
Alan X JiBridgeBio Pharma, Inc. San Francisco, California, USA.
Isabella A GraefStanford University, Stanford, California, USA.
Martha GroganMayo Clinic, Rochester, Minnesota, USA.
Prem SomanUniversity of Pittsburgh Medical Center, Pittsburgh, Pennsylvania, USA.
Francesco CappelliTuscan Regional Amyloidosis Centre, Careggi University Hospital, Florence, Italy.
Marianna FontanaNational Amyloidosis Centre, University College London, Royal Free Hospital, London, United Kingdom.
Ahmad MasriOregon Health & Science University, Portland, Oregon, USA.
Julian D GillmoreNational Amyloidosis Centre, University College London, Royal Free Hospital, London, United Kingdom.
Pablo Garcia-PaviaHospital Universitario Puerta de Hierro Majadahonda, IDIPHISA, CIBERCV and Centro Nacional de Investigaciones Cardiovasculares, Madrid, Spain.
Xiaofan CaoBridgeBio Pharma, Inc. San Francisco, California, USA.
Jonathan C FoxBridgeBio Pharma, Inc. San Francisco, California, USA.
Uma SinhaBridgeBio Pharma, Inc. San Francisco, California, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundTransthyretin amyloid cardiomyopathy (ATTR-CM) is a progressive, often fatal disease caused by transthyretin (TTR) tetramer destabilization, leading to amyloid deposition from either age-related wild type TTR or pathogenic TTR variants. TTR variants are less stable than wild type TTR, leading to lower serum TTR (sTTR) and worse clinical outcomes. TTR stabilizers, tafamidis and acoramidis, are approved for treatment of patients with ATTR-CM.

objectivesThe aim of this study was to evaluate the differences in stabilizing effect and magnitude of sTTR increases for acoramidis and tafamidis using data from the ATTRibute-CM (Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Cardiomyopathy) trial.

methodsStabilizing potency was analyzed using sTTR as an in vivo readout and by applying 2 orthogonal pharmacodynamic assays: Western blot and fluorescent probe exclusion. In vitro analyses used blood samples from patients with variant ATTR-CM at clinically relevant concentrations of acoramidis (10 μM) and tafamidis (16-26 μM).

resultsIn ATTRibute-CM, treatment with acoramidis (n = 234) resulted in a greater rise in sTTR from baseline to month 30 vs placebo plus tafamidis (n = 34). Acoramidis achieved greater TTR stabilization than placebo plus tafamidis at month 30 by Western blot (90.2% [n = 83] vs 60.6% [n = 6]) and fluorescent probe exclusion (99.7% [n = 71] vs 68.0% [n = 4]), although this was limited by a small sample size. Subsequent in vitro analysis corroborated acoramidis was a more effective TTR stabilizer than tafamidis across all 51 individual participant samples tested, representing 17 unique variants.

conclusionsAcoramidis is a near-complete stabilizer of wild type and variant TTR. Although in vitro comparisons between acoramidis and tafamidis suggest greater stabilization by acoramidis, randomized prospective trial data comparing these TTR stabilizers are lacking, and further investigation is warranted. (Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Cardiomyopathy [ATTRibute-CM]; NCT03860935).

Indexed as

acoramidisATTR-CMtafamidistransthyretinTTR stabilizationvariant

Identifiers

PMID42307157
PMCPMC13282820

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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.