Bristol Myers Squibb Reports Positive Phase 3 Results for Camzyos in Adolescents With Obstructive HCM

Bristol Myers Squibb Reports Positive Phase 3 Results for Camzyos in Adolescents With Obstructive HCM

By, Admin 15 January 2026

Bristol Myers Squibb has reported positive topline results from SCOUT-HCM, a Phase 3 trial evaluating Camzyos (mavacamten) in adolescents with obstructive hypertrophic cardiomyopathy (oHCM).

This is the first Phase 3 study of a cardiac myosin inhibitor conducted in adolescent patients.

SCOUT-HCM: Trial at a Glance

SCOUT-HCM evaluated Camzyos in patients aged 12 to under 18 years with symptomatic oHCM.

Key design elements:

  • Phase 3, randomized, double-blind, placebo-controlled
  • 44 adolescent patients enrolled globally
  • Placebo-controlled period of 28 weeks
  • Long-term extension up to 200 weeks

Primary Endpoint Met

The trial met its primary endpoint. Camzyos demonstrated:

  • A statistically significant reduction in Valsalva left ventricular outflow tract (LVOT) gradient
  • Improvement versus placebo at Week 28

This confirms meaningful reduction in LVOT obstruction.

Positive Secondary Outcomes

Multiple secondary endpoints also achieved statistical significance. These included:

  • Resting and post-exercise LVOT gradients
  • Peak oxygen consumption
  • Symptoms and health status measures

The results point to clinically meaningful disease improvement.

Safety Profile Consistent With Adults

Safety outcomes aligned with existing Camzyos data.

Key observations:

  • Safety profile consistent with adult studies
  • No new safety signals identified
  • Well tolerated in the adolescent population

The study continues into active treatment and long-term extension phases.

Why This Matters for Adolescent oHCM?

Adolescent oHCM is rare, serious, and underserved.

Current treatment limitations include:

  • Symptom-focused medical management
  • Side effects from beta-blockers
  • Risks associated with invasive surgery

There are no FDA-approved therapies that directly target disease biology in adolescents.

Expert Perspectives

Bristol Myers Squibb highlighted the broader impact of the results.

  • Camzyos has already transformed adult oHCM care
  • Over 20,000 adult patients have started therapy in the US
  • The company now sees a path to address adolescent disease

Paediatric cardiology leaders emphasized the rarity of positive Phase 3 data in this population.

Camzyos: A First-in-Class Therapy

Camzyos is a selective, reversible cardiac myosin inhibitor.

Its mechanism:

  • Reduces excess myosin–actin cross-bridges
  • Decreases hypercontractility
  • Improves cardiac filling pressures

The drug targets the root cause of oHCM, not just symptoms.

Regulatory and Next Steps

Bristol Myers Squibb plans to:

  • Present full data at an upcoming medical congress
  • Engage with global health authorities on next steps

Camzyos is already approved in 50+ countries for adults with symptomatic oHCM.

Bottom Line

SCOUT-HCM marks a major milestone in paediatric cardiology. For the first time, a cardiac myosin inhibitor has shown Phase 3 efficacy in adolescents with oHCM.

If approved, Camzyos could reshape treatment options for this high-risk, underserved patient group.

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