Presented in Hot Line 1 at ESC Congress 2026 in Munich and published simultaneously in the NEJM: in 517 patients with symptomatic non-obstructive hypertrophic cardiomyopathy, aficamten improved both KCCQ-CSS (difference 3.0 points, p=0.021) and peak VO2 (difference 0.67 mL/kg/min, p=0.003) at 36 weeks versus placebo. NYHA class improved in 42 percent versus 28 percent, NT-proBNP fell by more than half, and 53 percent versus 13 percent responded in three or more domains. The price: LVEF below 50 percent in 10.5 percent versus 0.8 percent, and more serious adverse events. A first drug for a third of the HCM population, with a modest effect size and a safety signal that demands echo-guided titration.
