Regeneron公布Pasatru(garetosmab)FOP三期OPTIMA研究84周延展期零新增异位骨化病灶数据

Regeneron Pharmaceuticals, Inc. (REGN) reported positive 84-week results from the Phase 3 OPTIMA trial, showing that Pasatru (garetosmab-grts) prevented the formation of new heterotopic ossification (HO) lesions from Week 56 to Week 48 in adults with fibrodysplasia ossificans progressiva, a rare and disabling genetic disorder.
Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare condition in which muscle, ligaments and connective tissue gradually turn into bone. The disease can severely restrict movement and make everyday activities such as walking, eating and breathing increasingly difficult.
Pasatru is the first and only FDA-approved treatment shown to reduce both new abnormal bone growth and clinician-assessed disease flare-ups in adults with FOP. The treatment works by blocking Activin A, a protein involved in the formation of abnormal bone in patients with the disease.
The new data were presented at the American Society for Bone and Mineral Research (ASBMR) Annual Meeting and extend findings from the placebo-controlled portion of the Phase 3 OPTIMA study.
At 56 weeks, the trial met its primary endpoint, demonstrating a reduction of more than 90% in new HO lesion and a substantial decrease in flare-ups compared with placebo.
From Week 56 to Week 84, patients receiving Pasatru had no new HO lesions detected, while nine new lesions were observed in the placebo group. Additionally, one lesion previously detected in the Pasatru 3 mg/kg group at Week 56 was no longer detectable at Week 84.
The treatment also continued to reduce disease flare-ups. During the extension period, one clinician-assessed flare-up was reported in the Pasatru 10 mg/kg group, compared with 14 in the Pasatru 3 mg/kg, and 17 in the placebo group.
Pasatru demonstrated a generally well-tolerated safety profile, through 84 weeks, consistent with earlier findings. The most commonly reported treatment-emergent adverse events included acne and nosebleeds. No deaths were reported during the study.
The Phase 3 OPTIMA trial enrolled 63 adults with active FOP and evaluated intravenous Pasatru at two dose levels compared with placebo. A total of 58 participants continued into the double-blind extension phase through at least 84 weeks.
Regeneron plans to further expand the development of Pasatru, with a Phase 3 study in children and adolescents with FOP, called OPTIMA 2 expected to begin later this year.
REGN has traded between $553.87 and $859.34 over the past year. The stock closed Monday's trading at $762.81, up 2.18%.
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