Regeneron reports sustained 84‑week efficacy of Pasatru in adults with FOP
Regeneron announced that its Phase 3 OPTIMA trial continued to show no new heterotopic ossification lesions in the Pasatru arm through week 84, compared with nine lesions in the placebo group. The extended data also confirmed a marked reduction in flare‑ups, with only one event in the high‑dose Pasatru cohort versus 17 in placebo. Safety remained consistent, with acne and epistaxis reported as the most common adverse events. The company said it will start a Phase 3 OPTIMA 2 study in children and adolescents later this year.
Why it matters
Bank of America raised its price target for Regeneron to $908, implying about 19% upside. The long‑term efficacy data could support broader adoption of Pasatru and sustain revenue growth.
Key facts
- 1The OPTIMA trial enrolled 63 adults with fibrodysplasia ossificans progressiva. stocktitan.net
- 258 participants completed the double‑blind extension phase to at least week 84. stocktitan.net
- 3From weeks 56 to 84, patients receiving Pasatru had zero new HO lesions, while placebo patients had nine new lesions. tradingview.com
- 4At week 84, the Pasatru 10 mg/kg dose recorded one disease flare‑up versus 17 in the placebo group. finance.yahoo.com
- 5The most common treatment‑emergent adverse events through week 84 were acne (Pasatru 10 mg/kg) and epistaxis (Pasatru 3 mg/kg). quiverquant.com
- 6Regeneron plans to initiate the OPTIMA 2 Phase 3 trial in children and adolescents later this year. stocktitan.net
Summary written by AlphAI AI Desk from 5 of 5 sources. Not investment advice. Figures are as stated by the linked sources.