Pasatru Shows Zero New HO Lesions in 84-Week FOP Extension, Reinforcing Long-Term Efficacy
REGN sits 41% above its 52-week low of $541 on light trading volume (0.3× avg).
Summary
Regeneron's Pasatru (garetosmab-grts) demonstrated sustained disease-modifying control in the 84-week extension of the Phase 3 OPTIMA trial for fibrodysplasia ossificans progressiva (FOP). From week 56 to 84, patients on Pasatru developed zero new heterotopic ossification (HO) lesions compared to 9 new lesions in the placebo group. One HO lesion previously seen at week 56 in a Pasatru 3 mg/kg patient was no longer detectable at week 84. Flare-ups were also markedly reduced: 1 in the 10 mg/kg arm, 14 in the 3 mg/kg arm, and 17 with placebo. Safety remained consistent with the 56-week profile, though skin and soft tissue infections increased dose-dependently (33% placebo, 53% 3 mg/kg, 78% 10 mg/kg), all resolved without discontinuation. This follows the FDA approval of Pasatru in August 2026 based on the 56-week data. The company also announced plans to start a Phase 3 trial (OPTIMA 2) in adolescents and children later this year. These longer-term results strengthen the commercial outlook for Pasatru in an ultra-rare disease with high unmet need.
At the time of this announcement, REGN was trading at $764.45 on NASDAQ in the Life Sciences sector, with a market capitalization of approximately $78.7B. The 52-week trading range was $541.00 to $859.34. This news item was assessed with positive market sentiment and an importance score of 8 out of 10. Source: GlobeNewswire.