Table 1.

Summary of Hypertrophic Cardiomyopathy Trials.

Study	Group	Primary Endpoint Achieved	Secondary Endpoint Achieved	Adverse Event
PIONEER-HCM	N=11 Cohort A- Mavacamten 10–15 mg with no background therapy for 12 weeks N=10 Cohort B- Mavacamten 2–5 mg with beta blockers for 12 weeks	Cohort A- ↓ peak exercise LVOT gradient- 90 mm Hg (95% CI -138 to -41; p=0.008) Cohort B-↓ peak exercise LVOT gradient -25 mm Hg (95% CI -47 to -3.0 mm Hg; p=0.02)	↑ pVO2 ↓ Resting and Valsalva LVOT gradients ↓ NT-proBNP Improved NYHA, KCCQ-CCS & NRS score	Cohort A- recurrence of atrial fibrillation requiring hospitalization in 1 patient; transient ↓EF in 3 patients
EXPLORER-HCM	N=251 Mavacamten 2.5–15 mg vs. placebo for 30 weeks	↑ in pVO2 > 1.5 mL/kg/min and ↓ NYHA>1 class or ↑ in pVO2 > 3.0mL/kg/min and no worsening of NYHA class 37% vs. 17% (difference +19.4%, 95% CI 8.7 to 30.1; p=0.0005)	↑ pVO2 ↓ Post-exercise LVOT gradient ↓ NYHA class > 1 Improved KCCQ-CCS	7 mavacamten patients had a decrease in EF <50% that recovered 1 death in placebo
VALOR-HCM	N=112 Mavacamten 2.5–15 mg vs. placebo for 16 weeks of patients referred for SRT within the past 12 months	Decided to proceed with SRT or was guideline eligible for SRT 18% vs. 77% (p<0.0001)	↓ Post-exercise LVOT gradient ↓ NYHA class >1 Improved KCCQ-CCS ↓ NT-proBNP and troponin I	2 mavacamten patients had a decrease in EF <50% that recovered

Abbreviations: EF, ejection fraction; KCCQ-CCS, Kansas City Cardiomyopathy Questionnaire Overall Summary Score; LVOT, left ventricular outflow tract; NT-proBNP, N-terminal pro hormone B-type natriuretic peptide; NYHA, New York Heart Association; pVO2, peak oxygen consumption; SRT, septal reduction therapy.