Sarepta Therapeutics received a $200 million milestone payment from Arrowhead Pharmaceuticals for the second development milestone in its Phase 1/2 study of SRP‑1003, the investigational siRNA therapy for myotonic dystrophy type 1 (DM1). The payment is triggered by the successful completion of Cohort 2 in the multiple‑ascending‑dose (MAD) study and is expected to be paid within 60 days of the announcement.
Cohort 3 of the MAD study is fully enrolled and patients are currently being dosed in Cohort 4, while the final Cohort 5 is slated to begin dosing in early 2026. The milestone payment is part of a series of payments tied to the program’s progress, following an earlier $100 million milestone that was announced in August 2025.
The $200 million inflow is non‑dilutive and strengthens Sarepta’s balance sheet at a time when the company has been aggressively trimming costs. In July 2025 the company announced a restructuring that cut its workforce by 36% and targeted $400 million in annual cost savings. The milestone payment provides the cash needed to accelerate the siRNA platform, which Sarepta has identified as its primary growth engine after the restructuring.
CEO Doug Ingram said the milestone “demonstrates the strong progress of our siRNA programs and reinforces our confidence in the SRP‑1003 platform as a potential best‑in‑class therapy for DM1.” He added that the payment will help fund further development of SRP‑1003 and other siRNA candidates in the pipeline.
Sarepta’s siRNA platform extends beyond DM1 to include investigational therapies for facioscapulohumeral muscular dystrophy, spinocerebellar ataxia type 2, idiopathic pulmonary fibrosis, and Huntington’s disease. The Arrowhead collaboration brings RNAi expertise that complements Sarepta’s delivery technology, positioning the company to capture a broader rare‑disease market and diversify its revenue streams beyond its Duchenne muscular dystrophy franchise.
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