Regeneron Reports Positive 84-Week Trial Data for FOP Drug Pasatru
Regeneron Pharmaceuticals, Inc. (REGN) reported encouraging 84-week Phase 3 trial results for Pasatru (garetosmab-grts), demonstrating sustained long-term effectiveness in treating adults with fibrodysplasia ossificans progressiva (FOP). Presented at the American Society for Bone and Mineral Research Annual Meeting, the updated OPTIMA trial data showed that the therapy continued to halt abnormal bone formation and control disease flare-ups following its recent approval by the Food and Drug Administration.
FOP is an ultra-rare genetic disorder that causes soft tissues such as muscles, tendons, and ligaments to permanently transform into bone, progressively restricting physical movement. Between weeks 56 and 84 of the trial, patients in the Pasatru group experienced zero new abnormal bone lesions, compared to nine in the placebo group. Additionally, patients taking the 10 mg/kg dose of Pasatru had only one clinician-assessed flare-up during this timeframe, compared to 17 in the placebo group. The safety profile remained generally consistent with earlier 56-week results, and soft tissue infections were successfully managed without treatment disruptions.
Looking ahead, Regeneron plans to launch OPTIMA 2, a Phase 3 study evaluating Pasatru in adolescent and pediatric patients with FOP later this year. Following the data release, Regeneron shares traded more than 2% higher.
Key points
- Regeneron Pharmaceuticals presented positive 84-week Phase 3 OPTIMA trial data for its FOP drug Pasatru at the ASBMR Annual Meeting.
- Patients taking Pasatru had zero new abnormal bone lesions between weeks 56 and 84, compared to nine lesions in the placebo group.
- The 10 mg/kg dose saw just one clinician-assessed flare-up during the period, compared with 17 flare-ups in the placebo group.
- Regeneron plans to start the OPTIMA 2 Phase 3 trial for children and adolescents with FOP later this year.
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How we writeDisclaimerQuestions and answers
What is Pasatru and what condition does it treat?
Pasatru is a fully human monoclonal antibody developed by Regeneron Pharmaceuticals to treat fibrodysplasia ossificans progressiva (FOP), an ultra-rare genetic connective tissue disorder that turns soft tissues into bone.
What were the key findings from Regeneron's 84-week FOP trial?
Between weeks 56 and 84 of the Phase 3 OPTIMA trial, adult patients treated with Pasatru experienced zero new abnormal bone lesions and significantly fewer flare-ups compared to those given a placebo.
What are Regeneron's next steps for testing Pasatru in FOP?
Regeneron plans to initiate the OPTIMA 2 Phase 3 clinical trial later this year to evaluate Pasatru in pediatric and adolescent patients with FOP.