Phase 3 study with aficamten in non-obstructive HCM meets dual primary endpoints
In ACACIA-HCM, the cardiac myosin inhibitor aficamten improved KCCQ-CSS and maximal exercise performance (pVO2) in patients with symptomatic non-obstructive hypertrophic cardiomyopathy (HCM) compared with placebo.
Positive results of the phase 3 ACACIA-HCM trial, in which the effect of the cardiac myosin inhibitor aficamten was evaluated in non-obstructive HCM, have been announced. The trial met both dual primary endpoints, showing statistically significant improvements in patient-reported symptoms (KCCQ-Clinical Summary Score (CSS)) and maximal exercise performance (pVO2) compared with placebo at 36 weeks.
Aficamten significantly improved KCCQ-CSS (least square mean difference: 3.0; 95%CI: 0.5-5.5; P=0.021) and pVO2 (least square mean difference: 0.67 mL/kg/min; 95%CI: 0.22-1.1; P=0.003) from baseline to 36 weeks compared with placebo.
Treatment with aficamten also improved key secondary endpoints, including NYHA functional class, the composite z-score of ventilatory efficiency and pVO2, and NT-proBNP levels.
ACACIA-HCM was a randomized, double-blind, placebo-controlled phase 3 trial in which 516 patients with symptomatic non-obstructive HCM were randomized 1:1 to aficamten or placebo. The dual primary endpoints were the changes in KCCQ-CSS and pVO2 from baseline to 36 weeks.
There were no new safety signals observed. LVEF <50% occurred in 10% of patients treated with aficamten and 1% of patients receiving placebo. Two patients receiving aficamten experienced a serious adverse event of HF associated with LVEF <50%. Treatment interruptions due to LVEF <40% occurred in 3% of aficamten-treated participants.
Full results from the ACACIA-HCM trial will be presented at an upcoming medical meeting.