Aficamten Improves Exercise Capacity and Health Status in Nonobstructive HCM

In patients with symptomatic nonobstructive hypertrophic cardiomyopathy (HCM), aficamten significantly improved exercise capacity and patient-reported health status compared with placebo at 36 weeks. In this phase 3 multinational, double-blind trial, 517 adults were randomly assigned to receive aficamten or placebo for up to 72 weeks. At week 36, the Kansas City Cardiomyopathy Questionnaire clinical summary score increased by 11.4 points with aficamten versus 8.4 points with placebo, for a significant between-group difference of 3.0 points. Peak oxygen uptake increased by 0.64 ml/kg/min with aficamten but showed essentially no change with placebo, resulting in a significant between-group difference of 0.67 ml/kg/min. Reversible reductions in left ventricular ejection fraction to below 50% occurred more frequently with aficamten than with placebo (10.5% vs. 0.8%), while serious adverse events occurred in 20.2% and 14.7% of patients, respectively. These findings show that aficamten can provide significant improvements in functional capacity and health status in patients with symptomatic nonobstructive HCM. Source: https://www.nejm.org/