Treatment with aficamten (Myqorzo) improved exercise capacity as well as health status and symptoms in patients with symptomatic nonobstructive hypertrophic cardiomyopathy (HCM) in a large, double-blind, placebo-controlled trial.
The 517-patient phase III trial, ACACIA-HCM, met both dual primary endpoints, including change from baseline to week 36 in peak oxygen uptake and patient-reported health status on the Kansas City Cardiomyopathy Questionnaire clinical summary score (KCCQ-CSS), according to Ahmad Masri, MD, of Oregon Health & Science University Medical Group in Portland, Oregon, speaking at the European Society of Cardiology's annual meeting. Results were published simultaneously in the New England Journal of Medicine (NEJM).
At 36 weeks, the mean change in peak oxygen uptake was 0.64 mL/kg/min in the aficamten group, versus -0.03 mL/kg/min in the placebo group, for a least-squares mean difference of 0.67 mL/kg/min (P=0.003).
KCCQ-CSS values rose an average of 11.4 points in the aficamten group versus 8.4 points with placebo, for a least-squares mean difference of 3.0 points (P=0.02).
Aficamten, a cardiac myosin inhibitor, was approved by the FDA last December to improve functional capacity and symptoms in patients with symptomatic obstructive HCM, on the strength of data from the SEQUOIA-HCM trial. This new trial extends those findings to patients with symptomatic nonobstructive HCM.
