Regeneron announces primary endpoint met in Phase 3 OPTIMA trial

Regeneron (REGN) Pharmaceuticals announced the primary endpoint was met in the Phase 3 OPTIMA trial investigating garetosmab in adults with fibrodysplasia ossificans progressiva, FOP. At 56 weeks, both doses of garetosmab, 3 mg/kg and 10 mg/kg, were highly efficacious in reducing the number of new bone lesions as compared to placebo, demonstrating a 94% and 90% reduction, respectively. The Primary endpoint was met, showing a 90% or greater reduction in new HO lesions at 56 weeks, and garetosmab also led to a greater than 99% reduction in the total volume of new HO lesions. Based on these data and the safety profile, the Independent Data Monitoring Committee recommended those receiving placebo be transitioned to garetosmab as soon as possible; U.S. regulatory submission of garetosmab in adults planned for year-end 2025