Aficamten Improves Exercise and Symptoms in Nonobstructive HCM Trial

A phase III trial involving 517 patients with symptomatic nonobstructive hypertrophic cardiomyopathy found that aficamten significantly boosted peak oxygen uptake and patient-reported health status at 36 weeks compared with placebo. The drug also improved secondary measures including functional class, exercise metrics, and a biomarker of wall stress. These results extend the medication's potential benefit beyond its current approved use for obstructive disease.

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The ACACIA-HCM trial enrolled 517 patients across multiple centers, with results published simultaneously in the New England Journal of Medicine. Aficamten's mechanism involves modulating cardiac contractility, which explains the reversible left ventricular ejection fraction reductions observed in 10.5% of treated patients versus 0.8% on placebo. All heart failure events occurred during the 12-week titration period and were managed with diuretics, suggesting a manageable safety profile with appropriate monitoring.

The drug's FDA approval for obstructive HCM came from the SEQUOIA-HCM trial, and this new data extends its potential utility to a population currently lacking approved treatments. A separate responder analysis published in Circulation examined symptom