Physicians' Academy for Cardiovascular Education

Phase 2 study with cardiac myosin inhibitor in non-obstructive HCM

News - May 22, 2023

Evaluation of Aficamten in Patients with Symptomatic Non-obstructive Hypertrophic Cardiomyopathy: REDWOOD-HCM Cohort 4

Presented at the ESC Heart Failure 2023 by: Ahmad Masri, MD- Portland, OR, USA

Introduction and methods

Aficamten is a cardiac myosin inhibitor targeting myocardial hypercontractility and impaired relaxation. An phase 2, open label, dose finding study evaluated the safety and efficacy of aficamten in patients with non-obstructive HCM.

A total of 41 patients (mean age 56 years, 59% female) with symptomatic non-obstructive HCM (NYHA class II/III, LVEF ≥ 60%, NT-proBNP >300 pg/mL) entered the study. Patients received 10 weeks of treatment, followed by 4 weeks of wash-out. Available doses were 5, 10, 15 mg. Doses were up-titrated if LVEF ≥55%, maintained if LVEF 50-54%, down-titrated if LVEF<50% and discontinued if LVEF <40%.

Main results


This phase 2, open-label, dose finding study showed that aficamten was generally well tolerated in patients with non-obstructive HCM. A modest reduction of -5.5% LVEF was observed by week 10, which was rapidly reversed during the wash-out period. The majority of patients reported improvement of symptoms. Aficamten will be further investigated in patients with non-obstructive HCM in a phase 3 study.

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