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

Aficamten significantly improves multiple clinical measures in 53% of patients with nonobstructive hypertrophic cardiomyopathy compared to 14% on placebo.

• Why it matters: Nonobstructive hypertrophic cardiomyopathy has no approved treatment and is linked to significant morbidity, highlighting the need for effective therapies.
• What they did: The ACACIA-HCM trial randomized 517 symptomatic nHCM patients to aficamten or placebo, assessing symptom burden, exercise capacity, left atrial volume, diastolic function, and NT-proBNP at 36 weeks.
• The result: Aficamten led to greater improvements in symptom burden (71% vs. 53%), exercise capacity (45% vs. 37%), and other measures, with a number needed to treat as low as 1.7 for NT-proBNP reduction, suggesting broad clinical benefits.