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Sarepta Therapeutics Shared Progress In Phase 1/2 Multiple Ascending Dose Study Of SRP-1003 (Formerly ARO-DM1), An Investigational Small Interfering RNA Therapeutic For Type 1 Myotonic Dystrophy
Sarepta Therapeutics, Inc. SRPT | 21.53 | -1.01% |
Cohorts 1 (1.5 mg/kg) and 2 (3 mg/kg) of the study are complete, and cohort 3 (4.5 mg/kg) is fully enrolled and ongoing. Following a positive, pre-specified drug safety committee review, the study advances with additional drug escalating cohorts. Having achieved the pre-specified patient enrollment target, a second milestone payment of USD$200 million will be made to Arrowhead Pharmaceuticals within 60 days.
Patients are currently being dosed in cohort 4 (6 mg/kg) with plans to initiate dosing in the final cohort, cohort 5 (12 mg/kg), in early 2026.


