Regeneron Pharmaceuticals, Inc. Announces Positive 84-Week Results From Phase 3 OPTIMA Trial Evaluating Pasatru In Adults With Fibrodysplasia Ossificans Progressiva

Regeneron (REGN) reported positive long-term results for its rare disease drug Pasatru, showing no new abnormal bone growth in an extended Phase 3 trial. The trial enrolled 63 adults, with zero new lesions in the Pasatru group compared to nine in the placebo group. REGN shares rose 2.8%, their best day in over a month. BofA raised its price target to $908, implying 19% upside. The company plans a new study for children and adolescents later this year.

Regeneron Pharmaceuticals reported 84-week trial data for Pasatru, showing sustained efficacy in treating fibrodysplasia ossificans progressiva. Compared to placebo, Pasatru had fewer new lesions and flare-ups. Common side effects included acne and epistaxis. The FDA approved Pasatru based on 56-week data, and a regulatory review is ongoing in Europe. Regeneron plans a Phase 3 trial in younger patients later this year.

Regeneron (NASDAQ: REGN) reported 84-week data from the Phase 3 OPTIMA trial showing sustained efficacy of Pasatru in adults with FOP, reducing new HO lesions and flare-ups. The trial met its primary endpoint at 56 weeks, with continued benefits observed at 84 weeks. Pasatru was FDA-approved based on the 56-week trial results. The company plans to initiate a Phase 3 trial in adolescents and children with FOP later this year.

Regeneron (REGN) reported 84-week data from its Phase 3 OPTIMA extension trial, showing Pasatru had zero new HO lesions vs. 9 on placebo, with a consistent safety profile. The results extend initial 56-week efficacy data. Next catalysts include OPTIMA 2, EU/Japan approvals, and real-world uptake data.

Regeneron Pharmaceuticals (NASDAQ: REGN) reported 84-week results from the Phase 3 OPTIMA trial of Pasatru, showing sustained efficacy in halting new bone growth and reducing flare-ups in adults with FOP. Pasatru is FDA-approved for this use. The trial involved 63 patients, with 58 completing the extension phase. Pasatru demonstrated a well-tolerated safety profile. Regeneron plans a Phase 3 trial in adolescents and children with FOP.

RBC Capital maintained a 'Sector Perform' rating for Regeneron Pharmaceuticals (REGN) but raised its price target to $810 from $737. REGN is trading at $729.42, which is 20.2% undervalued according to its GF Value™ of $914.40. The company has a GF Score™ of 87/100, indicating strong financial health and growth prospects. 10 gurus hold REGN, with 8 increasing their positions recently.