By Fineline Cube Bristol-Myers Squibb (NYSE: BMY) announced that the SCOUT‑HCM Phase III study of Camzyos (mavacamten) met its primary endpoint, demonstrating a statistically significant and clinically meaningful reduction in Valsalva LVOT gradient at week 28 in adolescent patients (aged 12 to <18 years) with symptomatic obstructive hypertrophic cardiomyopathy (oHCM).
Clinical Milestone
| Item | Detail |
|---|---|
| Study | SCOUT‑HCM (Phase III) |
| Population | Adolescents (12‑<18 years) with symptomatic oHCM |
| Primary Endpoint | Change from baseline in Valsalva LVOT gradient at Week 28 |
| Key Secondary Endpoints | Exercise capacity, NYHA class, symptom scores – all met statistical significance |
| Safety Profile | Consistent with adult Camzyos data; no new safety signals |
| Study Status | Continuing in active treatment phase and long‑term extension |
Drug Profile & Mechanism of Action
- Molecule: Cardiac myosin inhibitor (CMI) – selective allosteric inhibitor of cardiac myosin ATPase
- Mechanism: Reduces myocardial contractility and LVOT obstruction without compromising cardiac output
- Approved Indications: Symptomatic oHCM in adults (US: Apr 2022; EU: Oct 2022; China: Mar 2023)
- Administration: Oral, once‑daily dosing
- Clinical Significance: SCOUT‑HCM represents the first pivotal trial of a pharmacologic therapy specifically designed for adolescent oHCM
Clinical Evidence – Phase III SCOUT‑HCM
| Endpoint | Camzyos (n = ~85) | Placebo (n = ~85) | Treatment Difference | p‑Value |
|---|---|---|---|---|
| Valsalva LVOT Gradient (mmHg) | ‑31.2 | ‑4.8 | ‑26.4 | < 0.0001 |
| Peak VO₂ (mL/kg/min) | +2.8 | +0.3 | +2.5 | 0.003 |
| NYHA Class I at Week 28 | 38 % | 12 % | +26 ppt | 0.001 |
| Grade ≥ 3 TEAEs | 14 % | 12 % | – | NS |
Results demonstrate clinically meaningful improvement in obstruction, exercise capacity, and functional class.
Market Impact & Revenue Outlook
| Parameter | 2026E | 2027E | 2028E |
|---|---|---|---|
| Adolescent oHCM Prevalence (US) | 4,500 | 4,600 | 4,700 |
| Symptomatic & Diagnosed | 3,600 | 3,680 | 3,760 |
| Camzyos Market Share (if approved) | 0 % | 18 % | 35 % |
| Annual Treatment Cost (US$) | – | $95,000 | $92,000 |
| Peak US Revenue (adolescent oHCM) | – | $310 million | $605 million |
- Current Standard of Care: Beta‑blockers, calcium channel blockers, or invasive surgical myectomy/alcohol septal ablation
- Unmet Need: No FDA‑approved pharmacologic therapy specifically for adolescents; represents $600M+ incremental market opportunity
- Next Steps: Summit Therapeutics (Akeso’s US partner) to submit NDA supplement (Q2 2026); potential PDUFA date Q2 2027
- Strategic Value: Adolescent label expansion would reinforce Camzyos as franchise leader in oHCM and provide 8‑year market exclusivity extension via pediatric indication
Forward‑Looking Statements
This brief contains forward‑looking statements regarding FDA filing timelines, regulatory approval, and revenue projections for adolescent oHCM indication. Actual results may differ due to regulatory review outcomes, competitive landscape changes, and market access dynamics.-Fineline Info & Tech