Regeneron's Phase 3 OPTIMA Trial In Fibrodysplasia Ossificans Progressiva Meets Primary Endpoint

(RTTNews) - Regeneron Pharmaceuticals, Inc. (REGN) Wednesday said that the Phase 3 OPTIMA trial evaluating garetosmab in adults with fibrodysplasia ossificans progressiva met its primary goal.

Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare genetic disorder in which muscles, tendons, and ligaments are progressively replaced by bone, ultimately leading to severe disability.

In the OPTIMA study, FOP patients aged 18 and older were randomized to receive either garetosmab or placebo every four weeks. The study met its primary endpoint, showing a 90% or greater reduction in new heterotopic ossification (HO) lesions at 56 weeks. Treatment with garetosmab also resulted in a more than 99% reduction in the total volume of new HO lesions.

Based on these results and the safety profile, the Independent Data Monitoring Committee (IDMC) has recommended that patients receiving placebo be transitioned to garetosmab as soon as possible.

The company plans to submit garetosmab for U.S. regulatory approval in adults by the end of 2025.