Heart failure & cardiomyopathies

Aficamten Treatment for Symptomatic Obstructive Hypertrophic Cardiomyopathy: 48-Week Results From FOREST-HCM

JACC Heart FailPublished Open-label extension study

Authors' conclusion

Aficamten treatment over 48 weeks was well tolerated and associated with substantial and durable relief of obstruction and symptom burden, lower cardiac biomarker levels, and cardiac phenotypic changes, which may indicate favorable cardiac remodeling.

Study context

Population
Participants with symptomatic obstructive hypertrophic cardiomyopathy from REDWOOD-HCM or SEQUOIA-HCM46 with 48 weeks evaluated
Participants
213 participants
Outcome
LV outflow tract gradient, change from baseline
Follow-up
Week 48
Methodological detailsphase 2/3, ongoing, Enrolment May 2021 to October 2023
  • Phase 2/3
  • Ongoing
  • Enrolment May 2021 to October 2023

Main finding

As reported in the abstract

Mean resting LV outflow tract gradient change

−40 ± 34 mm Hg

Valsalva peak LV outflow tract gradient change

−53 ± 39 mm Hg

Further findings

Symptoms

≥1 NYHA class improvement
82%
20-point improvement in KCCQ-Clinical Summary score
31%

Cardiac structure and biomarkers (mean change)

Maximum LV wall thickness
−1.2 ± 1.6 mm; P < 0.0001
Left atrial volume index
−3.5 ± 6.6 mL/m²; P = 0.0008
Lateral E/e'
−2.2 ± 6.1; P = 0.02
Cardiac biomarkers
P ≤ 0.0031

Safety

Safety and tolerability · 48 weeks

Asymptomatic, transient LVEF <50% (range 47%-49%), neither resulting in drug discontinuation
2 (4.3%)
No new-onset AF

Original publication

Aficamten Treatment for Symptomatic Obstructive Hypertrophic Cardiomyopathy: 48-Week Results From FOREST-HCM. JACC Heart Fail. Published 19 June 2025. doi:10.1016/j.jchf.2025.03.040

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