CAMBRIDGE, Mass. — Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a biotechnology company focused on precision genetic medicines for rare diseases, announced new progress from its Phase 1/2 multiple ascending dose clinical study evaluating SRP-1003, an investigational small interfering RNA therapy for the treatment of myotonic dystrophy type 1 (DM1).
The company reported that the first two dosing cohorts at 1.5 mg/kg and 3 mg/kg have been completed, and cohort 3 at 4.5 mg/kg is fully enrolled and ongoing. Sarepta said a pre-specified review by the study’s drug safety committee was positive, clearing the way for the trial to advance to additional ascending-dose cohorts.
With the study now having reached its predefined enrollment goal, Sarepta said it expects to make a second milestone payment of $200 million to Arrowhead Pharmaceuticals within 60 days, in accordance with its collaboration agreement. Dosing in cohort 4 at 6 mg/kg is now underway, and the company plans to begin dosing the fifth and final cohort at 12 mg/kg in early 2026.
Sarepta noted that SRP-1003 is part of its next-generation siRNA platform, which focuses on chronic treatment approaches for neurodegenerative and pulmonary diseases. In addition to DM1, the platform includes investigational programs for facioscapulohumeral muscular dystrophy, spinocerebellar ataxia type 2, idiopathic pulmonary fibrosis and Huntington’s disease, with preclinical research advancing in spinocerebellar ataxia types 1 and 3. Sarepta also maintains an exclusive collaboration with Arrowhead Pharmaceuticals to develop therapies for skeletal muscle diseases and plans to pursue up to six discovery-stage targets in muscle or central nervous system indications.
The company said it remains committed to expanding its development efforts across rare neuromuscular and neurodegenerative disorders.
More information about Sarepta is available on the company’s website and its social media channels.
