Bristol Myers Squibb (BMS) has reported longer-term data from its Phase III clinical trial assessing Camzyos (mavacamten) in paediatric patients with symptomatic obstructive hypertrophic cardiomyopathy (oHCM).
The late-breaker data were presented at the Hypertrophic Cardiomyopathy Medical Society Scientific Sessions in Chicago, Illinois.
Go deeper with GlobalData
Discover B2B Marketing That Performs
Combine business intelligence and editorial excellence to reach engaged professionals across 36 leading media platforms.
The SCOUT-HCM trial (NCT06253221) enrolled 44 paediatric patients aged 12 to less than 18 years, with 43 completing the initial 28-week placebo-controlled phase before receiving Camzyos through week 56.
After 56 weeks, patients on continuous mavacamten achieved a mean baseline reduction of 48.1mmHg in Valsalva left ventricular outflow tract (LVOT) gradient, with a similar reduction observed in placebo patients switched to active drug after the initial 28-week period.
No participant experienced a left ventricular ejection fraction below 50% across 56 weeks.
Five treatment-related adverse events and two serious events occurred in the continuous arm over 56 weeks, whilst the placebo-mavacamten arm recorded one event and no serious events over 28 weeks.
After the initial 28-week period, patients taking Camzyos experienced an average reduction of 48.5 mmHg in their Valsalva LVOT gradient, compared to just 0.5 mmHg in the placebo group. The topline data for the initial 28-week period was released on 14 January 2026, with full data following in March.
BMS executive vice-president, chief medical officer and development head Cristian Massacesi said: “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 paediatric patients with oHCM.
“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.”
The US Food and Drug Administration (FDA) approved Camzyos in September 2026 for paediatric patients weighing at least 30kg based on 28-week SCOUT-HCM results, following an initial approval for adults in 2022.
Camzyos is an allosteric cardiac myosin inhibitor designed to reduce LVOT obstruction by addressing hypercontractility.
Regulatory agencies have approved the drug in over 60 countries and regions across five continents, supported by Phase III studies in China (EXPLORER-CN) and Japan (HORIZON-HCM).
In the European Union (EU), Camzyos is only indicated for adults, whilst US indications include both adults and eligible paediatric patients.
In September this year, BMS reported positive top-line results from the registrational Phase II QUINTESSENTIAL trial (NCT06297226) of arlocabtagene autoleucel (arlo-cel; BMS-986393) in adults with quadruple-class exposed relapsed and refractory multiple myeloma (r/r MM).
