Cytokinetics, Incorporated (NASDAQ:CYTK) today announced positive topline results from ACACIA-HCM (Assessment Comparing Aficamten to Placebo on Cardiac Endpoints In Adults with Non-Obstructive HCM), the pivotal Phase 3 clinical trial of aficamten in patients with symptomatic non-obstructive hypertrophic cardiomyopathy (HCM).

ACACIA-HCM met both dual primary endpoints, demonstrating statistically significant improvements from baseline to Week 36 compared to placebo in both Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS) and maximal exercise performance.

The improvement in KCCQ was robust and consistent throughout the treatment period in participants on aficamten. Following washout, KCCQ decreased for participants on aficamten to match the placebo group. At Week 36, pVO2 increased for participants on aficamten, while it remained unchanged for participants on placebo, consistent with prior trials of aficamten in obstructive HCM. 

Statistically significant (p<0.001) improvements compared to placebo were observed in key secondary endpoints including the proportion of participants with improvements in New York Heart Association (NYHA) Functional Class, the composite z-score of ventilatory efficiency and pVO2, and NT-proBNP.