Aficamten trial brings positive news for patients living with non-obstructive hypertrophic cardiomyopathy
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.
A recent clinical trial has shown promising results for patients with non-obstructive hypertrophic cardiomyopathy (HCM). Hypertrophic cardiomyopathy is an inherited disorder where the heart muscle becomes abnormally thick, impacting blood flow and heart function. While non-obstructive HCM is a common condition with limited treatment options, the ACACIA-HCM trial explored the effects of the cardiac myosin inhibitor aficamten.
The double-blind, phase 3 trial, conducted at 182 international sites, involved 517 adults with symptomatic non-obstructive HCM. Participants were randomly assigned to receive either aficamten or a placebo for up to 72 weeks. The primary endpoints measured were changes in symptom burden and exercise capacity from baseline to week 36.
Results demonstrated significant improvements in symptom burden and exercise capacity with aficamten treatment. At 36 weeks, the Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS) improved by 11.4 in the aficamten group compared to 8.4 in the placebo group (p = 0.02). Additionally, maximal exercise performance (pVO2) increased by 0.64 mL/kg/min in the aficamten group, while remaining almost unchanged in the placebo group (p = 0.003).
These improvements were observed as early as 12 weeks, with a pronounced return of symptoms after stopping aficamten treatment. Secondary endpoints also showed significant benefits, such as reduced New York Heart Association functional class, improved submaximal exercise performance, and decreased markers of heart strain (NT-proBNP).
Safety was managed effectively, with a higher incidence of low ejection fraction (less than 50%) in the aficamten group (10%) compared to placebo (1%). However, this was mitigated through dose adjustments.
Principal investigator Dr. Ahmad Masri of Oregon Health & Science University expressed optimism about the trial results, emphasizing that they provide "really positive news for patients with symptomatic non-obstructive HCM." For the first time, aficamten has shown clinically meaningful improvements in symptoms and exercise capacity by targeting the underlying cause of the disease.
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