Aficamten trial brings positive news for patients living with non-obstructive hypertrophic cardiomyopathy
· Medical Xpressby European Society of Cardiology
edited by Swati Mestri, reviewed by Andrew Zinin
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Aficamten treatment was associated with significant improvements in symptom burden and exercise capacity in patients with symptomatic non-obstructive hypertrophic cardiomyopathy, according to results presented today in a Hot Line session at ESC Congress 2026 and published simultaneously in the New England Journal of Medicine.
Hypertrophic cardiomyopathy (HCM) is an inherited disease in which the heart muscle becomes abnormally thick. HCM can be obstructive, when the thickened muscle blocks blood flow, or non-obstructive, when blood flow is not blocked but heart function is still affected.
"Patients with non-obstructive HCM experience limiting symptoms that affect their daily lives. But despite this being a relatively common disorder, there are no effective therapies," explained principal investigator Dr. Ahmad Masri of Oregon Health & Science University in Portland, Oregon.
"Building on our experience with the cardiac myosin inhibitor aficamten in obstructive HCM, we conducted the ACACIA-HCM trial to investigate its effects on symptom burden and exercise capacity in patients with non-obstructive HCM."
This was a double-blind, phase 3 trial conducted at 182 international sites. A total of 517 adults with symptomatic non-obstructive HCM were randomized 1:1 to aficamten or placebo for up to 72 weeks. The dose of aficamten was adjusted based on left ventricular ejection fraction (LVEF).
The dual primary endpoints were changes in Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS) and maximal exercise performance (pVO2) from baseline to week 36. The mean age of participants was 55 years, 54% were women and baseline LVEF was 68%.
Aficamten significantly improved the primary endpoint related to symptom burden. At 36 weeks, KCCQ-CSS improved by 11.4 in the aficamten group and 8.4 in the placebo group (p = 0.02). Masri noted that improvements were seen "as early as 12 weeks and there was a pronounced return of symptoms after aficamten treatment was stopped."
Aficamten also significantly improved the primary endpoint related to maximal exercise performance at 36 weeks. pVO2 improved by 0.64 mL/kg/min in the aficamten group and was almost unchanged (−0.03 mL/kg/min) in the placebo group (p = 0.003).
Significant improvements with aficamten were also seen in secondary endpoints, including New York Heart Association functional class, submaximal exercise performance and NT-proBNP, a marker of heart strain.
Regarding safety, more patients had LVEF of less than 50% with aficamten than with placebo (10% vs. 1%), but this was managed with dose adjustment.
In conclusion, Masri said, "Results from the ACACIA-HCM trial provide really positive news for patients with symptomatic non-obstructive HCM. For the first time, we have shown clinically meaningful improvements in symptoms and exercise capacity with a treatment that targets the cause of the disease."
Journal information: New England Journal of Medicine
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Swati Mestri
Swati Mestri holds a bachelor's degree in Electronics Engineering and has worked as a content editor since 2019. She has experience editing research documents across technology, health care, and materials science, and has a particular interest in technology and space. Full profile →
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Citation: Aficamten trial brings positive news for patients living with non-obstructive hypertrophic cardiomyopathy (2026, August 28) retrieved 28 August 2026 from https://medicalxpress.com/news/2026-08-aficamten-trial-positive-news-patients.html This document is subject to copyright. Apart from any fair dealing for the purpose of private study or research, no part may be reproduced without the written permission. The content is provided for information purposes only.