Global pharma powerhouse Bristol Myers Squibb has announced a potential breakthrough for a rare and serious heart condition.
The company has said that its Phase 3 SCOUT-HCM trial evaluating Camzyos (mavacamten) in adolescents with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) met its primary endpoint,
The study, the first to test a cardiac myosin inhibitor (CMI) in patients aged 12 to 17, showed a statistically significant reduction in Valsalva left ventricular outflow tract (LVOT) gradient at Week 28 compared with placebo, demonstrating Camzyos’ effectiveness in improving LVOT obstruction.
Multiple secondary endpoints also reached statistical significance, highlighting improvements in clinically meaningful aspects of the disease. Safety results were consistent with Camzyos’ adult profile, with no new safety signals reported. The trial continues with active treatment and long-term extension periods.
“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.”
“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, 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 is backed by the largest global clinical trial and real-world evidence base in the CMI class, further cementing its role in transforming care for adults with symptomatic oHCM by reducing hypercontractility through inhibition of excess myosin-actin cross-bridges in the heart’s sarcomere.
