Sarepta (SRPT) shared progress in the Phase 1/2 multiple ascending dose clinical study of SRP-1003, an investigational small interfering RNA therapeutic for the treatment of type 1 myotonic dystrophy. Cohorts 1 and 2 of the study are complete, and cohort 3 is fully enrolled and ongoing. Following a 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 $200M will be made to Arrowhead Pharmaceuticals (ARWR) within 60 days. Patients are currently being dosed in cohort 4 with plans to initiate dosing in the final cohort, cohort 5, in early 2026.
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