Bristol Myers Squibb Reports Positive Phase 3 SCOUT-HCM Results For Camzyos In Adolescents

Bristol Myers Squibb announced positive results from the Phase 3 SCOUT-HCM trial evaluating Camzyos (mavacamten) in adolescents aged 12 to under 18 with symptomatic obstructive hypertrophic cardiomyopathy (oHCM). The study met its primary endpoint, showing a statistically significant and clinically meaningful reduction in left ventricular outflow tract (LVOT) gradient at Week 28 compared to placebo.

In addition to the primary endpoint, Camzyos demonstrated improvements across several secondary measures, including left ventricular obstruction, diastolic function, wall thickness, NYHA class, and mitral valve function. The trial, which enrolled 44 patients with NYHA class II–III symptoms, also showed notable reductions in both resting and post-exercise LVOT gradients, reinforcing its therapeutic potential in this younger patient population.

“Pediatric HCM is a rare cardiac disorder that is associated with severe, sometimes life-threatening, symptoms,” said Joseph Rossano, MD, Principal Investigator of SCOUT-HCM and Chief of the Division of Cardiology at Children’s Hospital of Philadelphia. “With no approved therapies for pediatric patients with oHCM and current recommendations for pharmacological therapy primarily extrapolated from evidence obtained from adult studies, the positive results of this trial represent a significant advance in the field of pediatric cardiology and the potential for a meaningful new therapy for adolescent patients if approved by the FDA.”

Structural benefits were also observed, including reductions in maximal left ventricular wall thickness and improvements in cardiac function markers such as the E/e’ ratio. These findings suggest that Camzyos may positively impact both functional and structural aspects of the disease.

“The SCOUT-HCM results underscore the potential for Camzyos to become the first CMI for adolescents, reinforcing our leadership in the CMI space and our role in reshaping the scientific understanding of oHCM and how the disease is diagnosed, evaluated and potentially treated,” said Cristian Massacesi, MD, executive vice president, Chief Medical Officer and Head of Development, Bristol Myers Squibb. “With these meaningful safety and efficacy data, we are excited about the potential to provide a paradigm-changing treatment for adolescents and their families.”

The safety profile of Camzyos in adolescents was consistent with that seen in adults, with similar rates of adverse events between treatment and placebo groups. No new safety concerns were identified, and there were no treatment discontinuations, deaths, or serious cardiac complications reported during the study period.