Overview
- Full ACACIA-HCM data released on Aug. 28, 2026 confirm that aficamten met both co‑primary endpoints for symptomatic nonobstructive hypertrophic cardiomyopathy.
- Patients on aficamten had greater improvement in patient‑reported health status and exercise capacity than placebo, with a 3.0‑point least‑squares mean advantage on KCCQ‑CSS and a 0.67 mL/kg/minute gain in peak VO2.
- The trial enrolled 517 adults and showed that 53% of treated patients had meaningful gains across at least three disease domains versus 13% on placebo.
- Safety signals included more serious adverse events, a higher rate of left ventricular ejection fraction falls below 50% (10.5% versus 0.8%), and a cluster of early heart‑failure events that were largely managed by dose down‑titration or diuretics.
- Cytokinetics plans a supplemental U.S. filing in Q4 2026 and analysts project large sales, but the drug did not improve heart structure or lower cardiovascular event rates in the trial so long‑term benefits remain unproven and competitors are preparing late‑stage nHCM studies.