Global Efficacy of Aficamten in Nonobstructive Hypertrophic Cardiomyopathy: Results From ACACIA-HCM.

In 30 seconds

This randomized, placebo-controlled trial analysis assessed multiple treatment responses to aficamten in symptomatic patients with nonobstructive hypertrophic cardiomyopathy (nHCM), using outcomes measured at week 36. Response criteria covered symptoms, exercise capacity, left atrial volume, septal diastolic velocity, and NT-proBNP. A partial or complete response—improvement in at least three of these five measures—occurred in 53% of patients receiving aficamten versus 14% receiving placebo (P<0.001). Aficamten was associated with improvement across several measures, although the between-group difference in peak oxygen uptake improvement was not statistically significant.

Key findings

• At week 36, at least three favorable outcomes were achieved by 53% with aficamten versus 14% with placebo (P<0.001).
• Symptom burden improved in 71% versus 53%, and septal e' improved in 51% versus 26%, with aficamten versus placebo (P<0.05 for both).
• NT-proBNP decreased by at least 50% in 63% with aficamten versus 5% with placebo (P<0.05).
• Peak oxygen uptake improved in 45% versus 37%; this difference was not statistically significant (P=0.1).

Why it matters

nHCM is associated with substantial morbidity, and the abstract notes that no treatment is approved for the condition. Assessing several domains together suggests aficamten may benefit more than one aspect of disease burden, including symptoms and measures related to diastolic function. The findings also show that response varied by outcome, with no statistically significant difference reported for peak oxygen uptake.

What to keep in mind

The analysis reports outcomes at week 36; longer-term effects are not established by the abstract. The responder categories depend on thresholds across five selected measures.