PRINCETON, N.J., USA, March 29, 2026
Bristol Myers Squibb has announced positive Phase 3 results from the SCOUT-HCM trial evaluating Camzyos® (mavacamten) in adolescents with symptomatic obstructive hypertrophic cardiomyopathy (oHCM), demonstrating statistically significant improvements in cardiac function and disease-related outcomes. The study met its primary endpoint, showing a clinically meaningful reduction in left ventricular outflow tract (LVOT) gradient at Week 28, positioning Camzyos as a potential first targeted pharmacological therapy for adolescent patients with oHCM.
Phase 3 Trial Meets Primary Endpoint with Strong Efficacy
The SCOUT-HCM trial, a randomized, double-blind, placebo-controlled global study, evaluated 44 adolescent patients aged 12 to <18 years with symptomatic oHCM over a 28-week period. The results demonstrated a significant reduction in Valsalva LVOT gradient, with a least-squares mean difference of −48.0 mm Hg compared to placebo (P < 0.0001), confirming the drug’s strong efficacy.
In addition to the primary endpoint, Camzyos delivered consistent improvements across multiple secondary endpoints, including resting and post-exercise LVOT gradients, diastolic function, and maximal left ventricular wall thickness. The therapy also showed improvements in New York Heart Association (NYHA) class and mitral valve function, highlighting its potential to significantly improve cardiac performance and patient quality of life.
Importantly, a higher proportion of patients achieved clinically meaningful reductions in LVOT gradient to below 30 mm Hg, reinforcing the therapy’s ability to alleviate obstruction and improve cardiac output in this high-risk population.
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Favorable Safety Profile Supports Pediatric Use
Camzyos demonstrated a safety profile consistent with previous adult studies, with no new safety signals identified during the trial. The incidence of treatment-emergent adverse events (TEAEs) was comparable between the Camzyos and placebo groups, and importantly, no patients experienced left ventricular ejection fraction (LVEF) below 50%, a key safety parameter in cardiac therapies.
There were also no treatment discontinuations, deaths, or cases of atrial fibrillation or symptomatic heart failure, indicating strong tolerability in adolescent patients. These findings are particularly significant given the limited treatment options currently available for pediatric oHCM, where existing therapies are often extrapolated from adult data and may carry limitations.
The therapy’s risk evaluation and mitigation strategy (REMS) ensures careful monitoring of cardiac function, further supporting its safe use in clinical practice if approved for this population.
Potential First Targeted Therapy for Adolescent HCM
Camzyos is a cardiac myosin inhibitor (CMI) that directly targets hypercontractility, the underlying cause of oHCM, offering a precision medicine approach to treatment. By reducing excessive cardiac muscle contraction, the drug helps improve blood flow, reduce obstruction, and enhance overall cardiac efficiency.
Currently approved for adults with symptomatic oHCM in multiple regions, Camzyos has been prescribed to over 22,000 patients globally, with a growing body of evidence supporting its long-term benefits. The SCOUT-HCM trial represents a significant step toward expanding its use into adolescent populations, addressing a critical unmet medical need in pediatric cardiology.
The trial findings were presented at the American College of Cardiology (ACC) Annual Scientific Session 2026 and simultaneously published in a leading medical journal, underscoring their scientific credibility and clinical importance.
A Breakthrough in Pediatric Cardiology Treatment
The positive Phase 3 SCOUT-HCM results position Camzyos as a potentially transformative therapy for adolescents with obstructive hypertrophic cardiomyopathy, offering targeted treatment with proven efficacy and safety. As regulatory discussions progress, the therapy could redefine the standard of care in pediatric cardiology, improving outcomes and quality of life for patients with this rare but serious condition.
Source: Bristol Myers Squibb press release
