Arrowhead Pharmaceuticals receives $200 million from Sarepta milestone

Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) announced it earned a $200 million milestone payment from Sarepta Therapeutics Inc. (NASDAQ: SRPT) after achieving the second development milestone in a Phase 1/2 clinical study of ARO-DM1, an investigational RNA interference therapeutic for type 1 myotonic dystrophy.

The milestone was reached following a drug safety committee review, authorization to dose escalate, and achievement of a patient enrollment target, according to the company's statement. Arrowhead expects to receive the payment within 60 days under its license and collaboration agreement with Sarepta.

ARO-DM1, also designated SRP-1003, targets type 1 myotonic dystrophy, described as the most common adult-onset muscular dystrophy. Patient enrollment in cohort 4, which receives a 6 mg/kg dose in the multiple ascending dose portion of the study, is nearly complete.

The company plans to initiate enrollment in cohort 5, which will receive a 12 mg/kg dose, in the first quarter of 2026.

Arrowhead develops RNA interference-based therapeutics that silence genes associated with diseases. The technology uses the body's natural gene silencing pathway to reduce production of specific proteins linked to various conditions.

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