Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that it has earned a $200-million milestone payment from Sarepta Therapeutics (NASDAQ: SRPT). The milestone was earned when Arrowhead achieved the second development milestone event in a Phase 1/2 clinical study of ARO-DM1, also called SRP-1003, an investigational RNA interference (RNAi) therapeutic for the treatment of type 1 myotonic dystrophy (DM1), the most common adult-onset muscular dystrophy. In accordance with the license and collaboration agreement with Sarepta, Arrowhead expects to receive this payment within 60 days.
The second milestone event was reached following a drug safety committee review, subsequent authorization to dose escalate, and achievement of a pre-specified patient enrollment target. Accrual of patients in cohort 4 (6 mg/kg) of the multiple ascending dose (MAD) portion of the study is nearly complete and the company intends to initiate enrollment in cohort 5 (12 mg/kg) in the first quarter of 2026.
Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them.
Shares in ARWR rocketed $3.76, or 9.3%, to $44.20, while those for SRPT gained $1.24, or 7%, to $18.93.
