Sarepta's Stock Rises on Positive Updates on DM1 Therapy

Core Insights - Sarepta Therapeutics' shares increased by 7% following the announcement of progress in its phase I/II study for SRP-1003, a therapeutic for type 1 myotonic dystrophy (DM1) [1][8] Study Progress - The company has completed two cohorts of the study and has enrolled patients in a third cohort, with a positive review from a drug safety committee allowing for the advancement of additional dosing cohorts [2] - Currently, patients are being dosed in a fourth cohort, with plans to start the fifth cohort in Q1 2026, and initial readout from the study is expected early next year [2] Stock Performance - Following the announcement, investor sentiment improved, especially in light of safety concerns surrounding other investigational DM1 therapies from competitors [3] - The stock has faced significant challenges this year, dropping nearly 85% compared to the industry’s 17% growth [6] Milestone Payments - The progress in the study triggered a $200 million milestone payment to Arrowhead Pharmaceuticals, part of a multi-billion-dollar licensing deal for SRP-1003 [7][8] - This is the second milestone payment in 2023, with the first being $100 million in July for reaching enrollment targets [9] Additional Acquisitions - Besides SRP-1003, Sarepta has acquired exclusive rights to three other clinical-stage programs from Arrowhead, which are in separate phase I/II studies [10] - Initial data for the facioscapulohumeral muscular dystrophy (FSHD) program is anticipated in early 2026 [10]