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).
