CAMBRIDGE, Mass. — 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 Platform
Sarepta’s next-generation siRNA platform is focused on chronically administered therapies for neurodegenerative and pulmonary diseases and includes investigational treatments for:
- Facioscapulohumeral muscular dystrophy (FSHD)
- Myotonic dystrophy type 1 (DM1)
- Spinocerebellar ataxia type 2 (SCA2)
- Idiopathic Pulmonary Fibrosis (IPF)
- Huntington’s disease (HD)
Sarepta is also pursuing preclinical programs for Spinocerebellar ataxia type 1 (SCA1) and Spinocerebellar ataxia type 3 (SCA3) and has an exclusive collaboration with Arrowhead Pharmaceuticals to develop therapies for skeletal muscle diseases, with plans to pursue up to six discovery targets in muscle or central nervous system disorders.
About Sarepta Therapeutics
Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (Duchenne) and are building a robust portfolio of programs across muscle, central nervous system, and cardiac diseases. For more information, please visit www.sarepta.com or follow us on LinkedIn, X, Instagram and Facebook.
Internet Posting of Information
We routinely post information that may be important to investors in the ‘For Investors’ section of our website at www.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.
Investor Contacts
Ian Estepan, 617-274-4052
[email protected]
Ryan Wong, 617-800-4112
[email protected]
Media Contact:
Tracy Sorrentino, 617-301-8566
[email protected]