Overview

• The pivotal OPTIMA study in adults met its primary endpoint, with 56-week results showing 94% and 90% fewer new lesions at 3 mg/kg and 10 mg/kg versus placebo.
• Regeneron plans a U.S. regulatory submission by year-end 2025 and expects to pursue global filings in 2026.
• A pediatric and adolescent Phase 3 study, OPTIMA 2, is expected to begin next year.
• Safety findings included more skin and soft-tissue infections at the higher dose, reduced musculoskeletal pain in treated groups, no deaths, and no discontinuations on garetosmab at 56 weeks.
• Garetosmab neutralizes Activin A, addressing a disease driven by ALK-2 mutations that affects roughly 1,000 people worldwide.