EXPLORER-HCM and VALOR-HCM trials:
Significant benefit observed in both primary endpoints1
EXPLORER-HCM
Trial design
A 30-week, Phase 3 clinical trial for patients with NYHA Class II-III obstructive HCM (N=251).1
At baseline, 92% of patients were on background therapy
(BB or CCB).1,2
Observed outcomes
2x as many patients taking CAMZYOS achieved the primary composite functional endpoint (symptoms and exercise capacity) vs. placebo: 37% with CAMZYOS (n=45/123) vs. 17% with placebo (n=22/128).
Treatment difference: 19% (95% CI: 8.67-30.13; p=0.0005)
VALOR-HCM
Trial design
A 16-week, Phase 3 clinical trial for patients with severely symptomatic obstructive HCM (N=112) who were eligible for SRT.1
At baseline,95% of patients were on background therapy (BB, CCB, disopyramide or combination therapy).1,5
Oberved outcomes
>3x as many patients were able to avoid SRT at 16 weeks (no longer guideline-eligible for SRT at Week 16 and did not choose to undergo SRT by Week 16*) with CAMZYOS vs. placebo: 18% (n=10/56) of patients taking CAMZYOS met the primary composite endpoints vs. 77% (n=43/56) taking placebo. Two patients in each group decided to proceed with SRT.
Treatment difference: 59% (95% CI: 44.0-73.9; p=0.0001)
Clinical studies – long-term results
MAVA-LTE: A cohort of EXPLORER-HCM4†
5-year extension study designed to evaluate long-term efficacy, safety and clinically guided dosing for CAMZYOS.
VALOR-HCM LTE: Long-term study of VALOR-HCM patients5†
Extension study designed to evaluate long-term effects of CAMZYOS through Week 128 in patients who were eligible and referred for SRT.
BB=beta blocker; CCB=calcium channel blocker; HCM=hypertrophic cardiomyopathy; NYHA=New York Heart Association; SRT=septal reduction therapy
* Patient decision to proceed with SRT: CAMZYOS treatment group n=2 (4%), placebo group n=2 (4%). SRT status not evaluable (imputed as meeting guideline criteria): CAMZYOS treatment group n=0, placebo group n=2 (4%).1
† The duration of this study is longer than the data in the Product Monograph.
References: 1. CAMZYOS (mavacamten capsules) Product Monograph. Bristol Myers Squibb Canada, February 14, 2024. 2. Olivotto I, et al. Mavacamten for treatment of symptomatic obstructive hypertrophic cardiomyopathy (EXPLORER-HCM): a randomized, double-blind, placebo-controlled, phase 3 trial. Lancet 2020;396:759-69. 3. Desai MY, et al. Myosin inhibition in patients with obstructive hypertrophic cardiomyopathy referred for septal reduction therapy. J Am Coll Cardiol 2022;80(2):95-108. 4. Garcia-Pavia P, et al. Long-term effect of mavacamten in obstructive hypertrophic cardiomyopathy. Eur Heart J 2024;45(47):5071-83. 5. Desai MY, et al. Mavacamten in Patients With Hypertrophic Cardiomyopathy Referred for Septal Reduction: Week 128 Results From VALOR- HCM. Circulation 2025;151:1378-90.