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Acoramidis reduces risk of ACM and CVM regardless of baseline NT-proBNP and sTTR levelsNew trial shows acoramidis works for heart muscle disease

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Key Takeaway
Note that acoramidis reduces risk of ACM and CVM regardless of baseline NT-proBNP or sTTR levels.

This Phase 3 randomized controlled trial enrolled 632 patients with transthyretin amyloid cardiomyopathy. The study evaluated the efficacy of acoramidis compared to a placebo over a follow-up period of 30 months. Secondary outcomes included time to ACM/first CVH, time to CVM/first CVH, Kansas City Cardiomyopathy Questionnaire scores, and 6-minute walk distance.

Results indicated that higher baseline NT-proBNP and lower sTTR were associated with greater disease severity. Specifically, baseline NT-proBNP and sTTR were each independently associated with ACM/first CVH and CVM/first CVH. Furthermore, the combination of higher baseline NT-proBNP and lower sTTR conferred a higher risk and shorter time to event.

Despite these baseline variations, acoramidis reduced the risk of ACM/first CVH and CVM/first CVH regardless of the patient's baseline NT-proBNP, sTTR, National Amyloidosis Center stage, or Geriatric Nutritional Risk Index. Safety and tolerability data were not reported. Because this is a post hoc analysis of baseline biomarkers, results should be interpreted with caution regarding the specific magnitude of effect.

How this fits prior evidence

How this fits prior evidence: This finding extends the existing evidence for acoramidis in transthyretin amyloid cardiomyopathy. It confirms that acoramidis provides consistent clinical efficacy regardless of baseline biomarkers, complementing the previously reported finding that acoramidis was associated with lower diuretic intensification in transthyretin amyloid cardiomyopathy.

Living with transthyretin amyloid cardiomyopathy can be frightening because the disease often progresses in ways that are hard to predict. Doctors use specific markers, like NT-proBNP and sTTR, to try and understand how severe the condition is and how quickly it might progress. These markers help them see the current state of the heart muscle.

A large study of 632 patients looked at how these markers relate to the drug acoramidis. The researchers found that while high levels of one marker and low levels of another were linked to more severe disease, the drug worked consistently for everyone. This means acoramidis reduced the risk of major heart events regardless of what those initial test numbers were.

This is important because it suggests the medication provides reliable protection for patients across different stages of the disease. While the study was a look back at data already collected, it offers a clearer picture of how the drug performs for people with different baseline health profiles.

What this means for you:
Acoramidis reduces heart risks for patients regardless of their initial biomarker levels.

Common questions

How does acoramidis work for patients with this heart condition?

The study of 632 patients showed that acoramidis reduced the risk of major heart events. Importantly, the drug worked consistently for patients regardless of their initial test levels, their stage of disease, or their nutritional risk scores.

What role do NT-proBNP and sTTR play in diagnosis?

These are markers used to measure disease severity. The study found that higher levels of NT-proBNP and lower levels of sTTR were linked to more severe disease. Combining these two measurements can help doctors better stage the disease and predict risks.

Does the treatment work differently based on a patient's initial health status?

No, the study found that acoramidis provided consistent clinical results. It reduced risks for patients regardless of their starting NT-proBNP or sTTR levels, meaning the drug's effectiveness did not change based on those specific initial markers.

Study Details

Study typeRct
Sample sizen = 632
EvidenceLevel 2
PublishedOct 2026
View Original Abstract ↓
BACKGROUND: Transthyretin amyloid cardiomyopathy (ATTR-CM) carries high risk for all-cause mortality (ACM), cardiovascular mortality (CVM), and cardiovascular hospitalization (CVH). NT-proBNP (N-terminal pro-B-type natriuretic peptide) and serum TTR (sTTR) are prognostic biomarkers in transthyretin amyloid cardiomyopathy, but their combined value for integrated risk stratification remains unknown. We evaluated the relationship between baseline NT-proBNP and sTTR and their prognostic value in ATTRIBUTE-CM (Efficacy and Safety of Acoramidis in Transthyretin Amyloid Cardiomyopathy). METHODS: ATTRIBUTE-CM was a phase 3, randomized, double-blind, placebo-controlled trial. Among 632 participants randomized to acoramidis or placebo, baseline NT-proBNP and sTTR concentrations were evaluated post hoc for associations with baseline characteristics, Kansas City Cardiomyopathy Questionnaire overall scores, 6-minute walk distance, National Amyloidosis Center stage, Geriatric Nutritional Risk Index score, and subsequent time to ACM/first CVH and CVM/first CVH through Month 30. RESULTS: Higher baseline NT-proBNP and lower sTTR were associated with greater disease severity and were inversely correlated. Using multivariable linear regression, sTTR remained inversely correlated with NT-proBNP. Using multivariable Cox analyses, baseline NT-proBNP and sTTR were each independently associated with ACM/first CVH and CVM/first CVH. Higher baseline NT-proBNP combined with lower sTTR conferred higher risk and shorter time to event. Acoramidis reduced risk of ACM/first CVH and CVM/first CVH regardless of baseline NT-proBNP/sTTR, National Amyloidosis Center stage, or Geriatric Nutritional Risk Index. CONCLUSIONS: Baseline NT-proBNP and sTTR were additively prognostic for ACM/first CVH and CVM/first CVH in patients with transthyretin amyloid cardiomyopathy. Their joint measurement may be useful for enhanced staging, risk stratification, and prognostication in transthyretin amyloid cardiomyopathy. Acoramidis demonstrated consistent clinical efficacy regardless of baseline NT-proBNP/sTTR status. REGISTRATION: URL: https://clinicaltrials.gov/study/NCT03860935; Unique Identifier: NCT03860935. Registered: February 2019.
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