Already on track to achieve blockbuster status in its third full year on the market, Bristol Myers Squibb’s cardiomyopathy treatment Camzyos has scored in a trial that could pave the way for it to become available to a younger group of patients.
The phase 3 study of the first-in-class cardiac myosin inhibitor on 44 patients ages 12-17 with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) achieved its primary objective and reached statistical significance for multiple secondary endpoints, including those for clinically meaningful aspects of the disease, the company said.
Importantly, given the age group, results in the trial were consistent with the established safety profile of Camzyos in adults, and no new side effects were reported.
The victory comes less than a year after the company revealed the failure of a phase 3 trial of Camzyos in patients with a milder form of the disorder—non-obstructive HCM, showing no statistically significant improvement over placebo in two primary endpoints.
In the most recent study, dubbed Scout-HCM, Camzyos achieved a significant reduction from baseline in Valsalva left ventricular outflow tract (LVOT) gradient at Week 28 versus placebo, BMS said, indicating Camzyos improved LVOT obstruction.
Secondary endpoints included assessments of resting and post-exercise LVOT gradients, peak oxygen consumption, symptoms and health status, plus safety and pharmacokinetic parameters, the company added.
The trial includes treatment over 200 weeks, starting with a 28-week placebo-controlled period, followed by a 28-week active treatment period, when patients randomized to placebo crossed over to treatment with Camzyos and an open-label long-term extension period for up to 144 weeks.
The study is one of the few for pediatric cardiology patients that has “generated positive phase 3 results,” trial investigator Joseph Rossano, M.D., who also is the chief of the Division of Cardiology at Children’s Hospital of Philadelphia, said in a release.
“Treatment options for adolescents with oHCM are currently limited to medical symptom management or invasive surgery,” Rossano added. “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.”
The company said it will break down results from the trial at an upcoming medical conference and will “discuss these data with health authorities.”
“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,” Cristian Massacesi, M.D., the chief medical officer and head of development for BMS, said.
For patients with HCM, muscle fibers in the heart increase in number and thicken, which reduces the space available for blood in the organ, resulting in higher pressure against which the heart has to work. Patients with HCM struggle with shortness of breath, weakness, exhaustion and the inability to do daily physical activities like climbing stairs, lifting weights or playing sports.
Camzyos, which works by blocking the actin-myosin interactions responsible for HCM and, in effect, relaxing the heart, bears a boxed warning for the risk of heart failure and is available under a Risk Evaluation and Mitigation Strategy program.
The FDA approved Camzyos in 2022 and just last month signed off on Cytokinetics’ oHCM treatment Myqorzo. Through a 2012 partnership between Cytokinetics and MyoKardia, the companies helped create Camzyos before BMS bought out MyoKardia for $13.1 billion.
Sales of Camzyos are scaling up quickly. After reaching $602 million in 2024, the company reported the treatment generated $714 million in revenue through the first nine months of 2025, including $296 million in the third quarter.