ATTRibute-CM was a randomised double-blind trial which aimed to evaluate the safety and efficacy of acoramidis in a cohort of patients diagnosed with transtherytin amyloid cardiomyopathy (ATTR-CM). 632 patients were enrolled in the trial, and were randomised 2:1 to receive either oral acoramidis 800mg twice daily, or placebo for 30 months. Patients had the option tafmidis after 12 months.
The analysis of primary endpoints, including Finkelstein-Schoenfeld hierarchy of ACM, CVH, NT-proBNP, and 6MWD, showed highly significant results. The win ratio was 1.8 with a p-value of less than 0.0001. In 58% of cases where there was a tie in the win ratio, ACM and CVH factors combined were used to break the tie.
1. What are the unmet needs of patients and currently available treatments for ATTR?
2. Can you summarise the mechanism of action of the study drug?
3. What was the study design, patient population and outcome measures?
4. What are the key results?
5. Were there any safety events?
6. What are the take-home messages?
7. What are the next steps?
Recorded on-site at ESC Congress 2023, Amsterdam.
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Editors: Mirjam Boros and Jordan Rance
Video Specialists: Dan Brent, Tom Green, Mike Knight, Oliver Miles