Arrowhead Pharmaceuticals Receives $100 Million Milestone Payment from Sarepta Therapeutics for ARO-DM1 Trial Success

Arrowhead Pharmaceuticals has received a $100 million milestone payment from Sarepta Therapeutics for reaching the first enrollment target in a Phase 1/2 clinical study of ARO-DM1. The company expects to achieve the second enrollment target by the end of 2025, potentially earning an additional $200 million. This collaboration demonstrates strong financial backing and the potential of Arrowhead's RNAi therapeutic platform.

Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) has received a $100 million milestone payment from Sarepta Therapeutics (NASDAQ: SRPT) for reaching the first enrollment target in a Phase 1/2 clinical study of ARO-DM1. The milestone was triggered when Arrowhead reached the first of two prespecified enrollment targets and subsequent authorization to dose escalate in the study, which aims to treat type 1 myotonic dystrophy (DM1), the most common adult-onset muscular dystrophy [1].

The milestone payment is part of a broader licensing and collaboration agreement signed between Arrowhead and Sarepta in November 2024, which closed in February 2025. Under the agreement, Sarepta received rights to multiple investigational treatments leveraging Arrowhead's leading Targeted RNAi Molecule (TRiMTM) platform [1].

Arrowhead expects to achieve the second enrollment target by the end of 2025, which would trigger an additional $200 million milestone payment from Sarepta. The agreement also provides for further development milestone payments, sales milestone payments, and tiered royalties on commercial sales [1].

This collaboration underscores strong financial backing and the potential of Arrowhead's RNAi therapeutic platform, which leverages RNA interference (RNAi) to silence specific genes and treat intractable diseases [1].

References:
[1] https://www.businesswire.com/news/home/20250728872484/en/Arrowhead-Pharmaceuticals-Earns-%24100-Million-Milestone-from-Sarepta-Therapeutics