Sarepta Provides Progress Update for SRP-1003, its Investigational siRNA treatment for Myotonic Dystrophy Type 1
Sarepta Therapeutics, Inc. (SRPT)
Last sarepta therapeutics, inc. earnings: 2/26 04:05 pm
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Source: Business Wire
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today shared progress in the Phase 1/2 multiple ascending dose (MAD) clinical study of SRP-1003 (formerly ARO-DM1), an investigational small interfering RNA (siRNA) therapeutic for the treatment of type 1 myotonic dystrophy (DM1).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.About Sarepta’s siRNA PlatformSarepta’s next-generation siRNA platform is
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SRPT
Earnings
- 11/3/25 - Beat
SRPT
Sec Filings
- 12/2/25 - Form 4
- 11/19/25 - Form 4
- 11/14/25 - Form SCHEDULE
- SRPT's page on the SEC website