Bristol Myers Squibb has reported sustained efficacy and a consistent safety profile for its cardiac drug Camzyos (mavacamten) in adolescents with symptomatic obstructive hypertrophic cardiomyopathy (oHCM).

 

This, with new 56-week data showing no new safety signals and no cases of left ventricular ejection fraction (LVEF) falling below 50%.

 

The findings from the Phase 3 SCOUT-HCM trial strengthen the clinical evidence supporting Camzyos as the first approved cardiac myosin inhibitor for paediatric patients with symptomatic oHCM and reinforce its position as a treatment option for patients aged 12 to under 18 years.

 

The 56-week findings were consistent with results reported at 28 weeks, demonstrating sustained reductions in left ventricular outflow tract (LVOT) obstruction and improvements across several secondary measures.

 

“These results are significant as they build upon the understanding of the meaningful impact of Camzyos in pediatric patients with symptomatic oHCM, evaluated in a 56-week study that included a 28-week placebo-controlled period. This is the first time a cardiac myosin inhibitor has been evaluated in this patient population for this length of time and in a double-blind study,” said Cordula Wolf, Associate Professor, Technical University of Munich (TUM) University Hospital German Heart Centre. 

 

“These results serve as an important resource for healthcare providers and patients when discussing their care plan and the potential of Camzyos, the first approved treatment option for pediatric patients in the US with symptomatic oHCM.”

 

SCOUT-HCM enrolled 44 adolescents aged 12 to under 18 years with symptomatic oHCM. Of these, 43 completed the initial 28-week double-blind, placebo-controlled phase and moved into the active-treatment period.

 

At Week 28, all 43 patients received Camzyos through Week 56, including those who had initially received placebo.

 

Patients in both treatment groups recorded significant reductions in Valsalva LVOT gradients following treatment. Those who received Camzyos throughout the study recorded a mean reduction of 48.1 mmHg from baseline after 56 weeks.

 

Patients who switched from placebo to Camzyos also achieved a comparable degree of reduction.

 

Improvements were also observed across several secondary endpoints, while the overall safety profile remained consistent with earlier findings.

 

Importantly, no patient experienced an LVEF below 50% during the 56-week evaluation period.

 

Treatment-related treatment-emergent adverse events and serious adverse events remained low. In the group receiving Camzyos throughout the study, five treatment-related adverse events and two serious events were reported over 56 weeks. In the group that switched from placebo to Camzyos, one treatment-related adverse event and no serious events were reported during 28 weeks of active treatment.

 

No new safety signals emerged compared with previous SCOUT-HCM findings or Phase 3 studies involving adult patients.

 

“Symptomatic oHCM is a serious condition for which having effective and safe treatments is crucial. These additional SCOUT-HCM results provide added confidence in Camzyos to healthcare providers as they consider treatment options for their pediatric patients with oHCM,” said Cristian Massacesi, executive vice president, chief medical officer and head of development, Bristol Myers Squibb. 

 

“BMS is committed to researching and developing new treatments to address cardiovascular disease. The data presented at these congresses helps us better understand symptomatic oHCM and the role of Camzyos in delivering more meaningful outcomes for patients and their families.”