REGENERON PHARMACEUTICALS, INC.

Other Programs

•In August 2025, the Company announced that the primary and key secondary endpoints were met in a Phase 3 trial of cemdisiran (siRNA therapy), as both a monotherapy and in combination with pozelimab (C5 antibody), in adults with generalized myasthenia gravis. Cemdisiran monotherapy was numerically better than the combination treatment across these endpoints, showing a statistically significant 2.3-point placebo-adjusted improvement in Myasthenia Gravis Activities of Daily Living (MG-ADL) total score. A U.S. regulatory submission for cemdisiran monotherapy is planned for the first quarter of 2026, pending discussions with the FDA.

•In October 2025, updated data from the pivotal trial of DB-OTO, an AAV-based gene therapy, in children with profound genetic hearing loss due to variants of the otoferlin (OTOF) gene were published in the New England Journal of Medicine and presented at the annual American Academy of Otolaryngology-Head and Neck Surgery meeting. These latest results from the CHORD trial show 11 out of 12 participants have experienced clinically meaningful hearing improvements. A U.S. regulatory submission for DB-OTO is planned for the fourth quarter of 2025.

•In October 2025, the FDA selected DB-OTO to receive one of the Commissioner’s National Priority Vouchers, a pilot program intended to reduce the review time for certain drug and biologic applications to just one to two months.

•In September 2025, the Company announced the primary endpoint was met in a Phase 3 trial of garetosmab in adults with FOP, showing a 90% or greater reduction in new bone lesions (heterotopic ossification, or HO lesions) compared to placebo at 56 weeks, and a greater than 99% reduction in the total volume of new HO lesions. A U.S. regulatory submission for garetosmab is planned for the fourth quarter of 2025.

•In September 2025, the FDA approved Evkeeza® (evinacumab) for the treatment of homozygous familial hypercholesterolemia (HoFH), extending the already approved indication to include children from age 1 to less than 5 years old.

•In September 2025, the Company announced results from the Phase 3 trials evaluating its allergen-blocking antibodies in adults with moderate-to-severe cat allergies (REGN1908 and REGN1909) or birch allergies (REGN5713 and REGN5715). Both trials met their respective primary and key secondary endpoints, with single doses of the allergen-specific antibody blockers significantly reducing allergy symptoms compared to placebo at day 8. Additional Phase 3 development is planned to begin by the end of the year.