November 24, 2025 — Leads & Copy — Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) has achieved a significant milestone, earning a $200 million payment from Sarepta Therapeutics (NASDAQ: SRPT). This milestone is tied to the progress of ARO-DM1, also known as SRP-1003, an RNA interference (RNAi) therapeutic under investigation for treating type 1 myotonic dystrophy (DM1), the most prevalent adult-onset muscular dystrophy.
The payment is expected to be received by Arrowhead within 60 days, according to the license and collaboration agreement with Sarepta.
The second milestone event was triggered after a drug safety committee review, which led to authorization for dose escalation, and the fulfillment of a pre-specified patient enrollment target.
Patient enrollment in cohort 4 (6 mg/kg) of the multiple ascending dose (MAD) portion of the study is nearing completion. The company anticipates initiating enrollment in cohort 5 (12 mg/kg) during the first quarter of 2026.
Arrowhead Pharmaceuticals focuses on developing medicines for intractable diseases by silencing the genes responsible. Their therapies utilize a range of RNA chemistries and delivery methods to activate the RNA interference mechanism, resulting in rapid and sustained reduction of target gene expression. RNA interference (RNAi) is a process in living cells that inhibits specific gene expression, thereby impacting the production of specific proteins. Arrowhead’s RNAi-based therapeutics harness this natural gene-silencing pathway.
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Source: Arrowhead Pharmaceuticals, Inc.
