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Aficamten in symptomatic obstructive hypertrophic cardiomyopathy: the FOREST-HCM long-term study

Albree Tower-Rader1, Ahmad Masri2, Michael E Nassif3

  • 1Cardiology Division, Department of Medicine, 55 Fruit St, Yawkey 5B, Massachusetts General Hospital, Boston, MA 02114, USA.

European Heart Journal
|March 4, 2026
PubMed

Insights

Extended aficamten treatment significantly improved symptoms and hemodynamics in obstructive hypertrophic cardiomyopathy (oHCM) patients. The cardiac myosin inhibitor demonstrated sustained efficacy and a favorable safety profile over 96 weeks.

Area of Science:

  • Cardiology
  • Pharmacology
  • Clinical Trials

Background:

  • Hypertrophic cardiomyopathy (HCM) is characterized by myocardial hypertrophy and often presents with obstructive symptoms.
  • Cardiac myosin inhibitors offer a novel therapeutic approach by targeting the hypercontractility underlying HCM.
  • Aficamten is an oral selective cardiac myosin inhibitor designed to treat symptomatic obstructive HCM (oHCM).

Purpose of the Study:

  • To assess the safety and efficacy of extended treatment with aficamten in patients with symptomatic oHCM.
  • To evaluate long-term hemodynamic and clinical outcomes in patients receiving aficamten.

Main Methods:

  • The FOREST-HCM study (NCT04848506) was an open-label trial for patients who completed a prior aficamten study.
  • 296 patients with oHCM were enrolled, with cumulative exposure reaching 352 patient-years.
  • Safety and efficacy endpoints were assessed up to 96 weeks of follow-up.

Main Results:

  • Aficamten significantly reduced the Valsalva left ventricular outflow tract gradient at Weeks 12 and 96 (P < 0.0001).
  • Clinical improvements included NYHA class and Kansas City Cardiomyopathy Questionnaire scores, with minimal impact on left ventricular ejection fraction (LVEF).
  • Treatment-emergent serious adverse events were low (12.2%), with no deaths or aficamten-related heart failure; LVEF<50% occurred in 3.4% of patients.

Conclusions:

  • Extended aficamten treatment provides early and sustained hemodynamic and clinical benefits in symptomatic oHCM.
  • The cardiac myosin inhibitor demonstrated a favorable safety profile, with low incidences of atrial fibrillation and reduced LVEF.
  • Aficamten represents a promising therapeutic option for long-term management of obstructive hypertrophic cardiomyopathy.
Abstract

Keywords:
AficamtenEfficacyHypertrophic cardiomyopathyOpen-label extensionSafety

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