Impact of acoramidis on clinical stability in transthyretin amyloid cardiomyopathy: observations from ATTRibute-CM.
Francesco Cappelli, Marianna Fontana, Pablo Garcia-Pavia et al.
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In 30 seconds
This post-hoc analysis of the phase 3, multicentre, randomised ATTRibute-CM trial assessed clinically meaningful changes in NT-proBNP or 6-minute walk distance among participants with transthyretin amyloid cardiomyopathy. Of 611 participants analysed, 409 received acoramidis and 202 placebo. By Month 30, a higher proportion of the acoramidis group met the improvement criteria in either measure: 22.7% versus 8.9% (OR 3.0, 95% CI 1.8-5.1; P < .001). The findings indicate that some participants receiving acoramidis achieved improvement in these measures, rather than only avoiding deterioration.
Key findings
- Clinically meaningful improvement in NT-proBNP or 6-minute walk distance occurred in 22.7% with acoramidis and 8.9% with placebo (OR 3.0, 95% CI 1.8-5.1; P < .001).
- Improvement was defined as an NT-proBNP decrease of >700 ng/L and >30%, or a 6-minute walk distance increase of >35 m from baseline to Month 30.
Why it matters
The analysis highlights that treatment response in ATTR-CM may include measurable improvement in biomarkers or functional capacity for a subset of participants. These outcomes complement the question of whether therapy slows progression, while showing that improvement was not achieved by most participants under the study’s definitions.
What to keep in mind
This was a post-hoc analysis, and the response categories were based on NT-proBNP and 6-minute walk distance rather than a direct measure of overall clinical status.