Cytokinetics announced positive topline results from a pivotal phase 3 clinical trial of aficamten in patients with symptomatic non-obstructive hypertrophic cardiomyopathy (HCM).
Aficamten is an allosteric and reversible inhibitor of cardiac myosin motor activity. The tablets were approved by the FDA under the brand name Myqorzo in December 2025 for symptomatic obstructive hypertrophic cardiomyopathy (oHCM) to reduce cardiac contractility and left ventricular outflow tract obstruction.
Now, results from the ACACIA-HCM trial demonstrate statistically significant improvements from baseline to week 36 compared to placebo in both Kansas City Cardiomyopathy Questionnaire Clinical Summary Score and maximal exercise performance, making it the first clinical trial to demonstrate statistically significant improvements in exercise capacity and symptom burden in patients with non-obstructive HCM.
Cytokinetics says it plans to present the results from ACACIA-HCM at an upcoming medical meeting, as well as discuss them with the FDA and other regulatory agencies.