Bristol Myers Squibb Reports Positive Phase 3 Results For Camzyos In Adolescent oHCM Patients, Trial Shows Improvment In Cardiac Obstruction In Teens With Hypertrophic Cardiomyopathy

Bristol Myers Squibb has reported positive topline results from the Phase 3 SCOUT-HCM study, which evaluated Camzyos (mavacamten) in adolescents aged 12 to under 18 with symptomatic obstructive hypertrophic cardiomyopathy. The trial met its primary endpoint, showing a statistically significant reduction in left ventricular outflow tract (LVOT) gradient during Valsalva maneuver at Week 28 compared with placebo, demonstrating improved relief of cardiac obstruction.

The study also achieved statistical significance across several secondary endpoints that reflect meaningful clinical benefits for patients. Camzyos was well tolerated in this younger population, with a safety profile consistent with that observed in adults, and no new safety concerns were identified. The trial is ongoing, with treatment and long-term extension phases continuing.

“Adolescent oHCM is a serious, rare disease associated with substantial morbidity and mortality. The SCOUT-HCM topline results highlight the potential for Camzyos to be the first cardiac myosin inhibitor for adolescent patients with oHCM,” said Cristian Massacesi, MD, executive vice president, Chief Medical Officer and Head of Development, Bristol Myers Squibb. “Camzyos has redefined the treatment paradigm for symptomatic oHCM in adults, with over 20,000 patients started in the U.S. alone, and we look forward to the potential opportunity to transform clinical care in the adolescent patient population.”

Bristol Myers Squibb plans to share the full dataset at an upcoming medical congress and engage with regulatory authorities to discuss the implications of the findings. These results represent the first Phase 3 evidence for a cardiac myosin inhibitor in adolescents with obstructive hypertrophic cardiomyopathy.

“The SCOUT-HCM study is important for patients and the field of pediatric cardiology, being one of the very few pediatric cardiology randomized and placebo-controlled clinical trials that has generated positive Phase 3 results,” said Joseph Rossano, MD, Principal Investigator and Chief of the Division of Cardiology at Children’s Hospital of Philadelphia. “Treatment options for adolescents with oHCM are currently limited to medical symptom management or invasive surgery. As a clinician who has cared for patients in this field for decades, I am very excited about the potential opportunity that a therapy like this could hold for the patient population if approved by the FDA."

Camzyos already has the largest global clinical and real-world evidence base in its class for adult oHCM, where it works by reducing excessive heart muscle contractility through targeted inhibition of myosin-actin cross-bridges. The new data support its potential to extend this disease-modifying approach to younger patients.