Arrowhead Pharmaceuticals Earns $100M Milestone from Sarepta Therapeutics for Reaching Enrollment Target

Arrowhead Pharmaceuticals has earned a $100 million milestone payment from Sarepta Therapeutics. The milestone was triggered when Arrowhead reached a prespecified enrollment target and received authorization to dose escalate in a clinical trial.

Title: Arrowhead Pharmaceuticals Secures $100 Million Milestone Payment from Sarepta Therapeutics

Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) has earned a significant milestone payment of $100 million from Sarepta Therapeutics (NASDAQ: SRPT). This milestone was achieved when Arrowhead reached the first enrollment target and received authorization to dose escalate in a Phase 1/2 clinical trial of ARO-DM1, an investigational RNA interference (RNAi) therapeutic for the treatment of type 1 myotonic dystrophy (DM1) [1].

The milestone payment is part of a broader licensing and collaboration agreement signed between the two companies in November 2024 and closed in February 2025. Under this agreement, Sarepta received rights to multiple investigational treatments that leverage Arrowhead’s leading Targeted RNAi Molecule (TRiMTM) platform. The agreement covers multiple clinical and preclinical programs in rare, genetic diseases of the muscle, central nervous system, and the lungs, and also allows Sarepta to select up to six new targets for Arrowhead to conduct discovery and preclinical development activities in areas complementary to Sarepta’s leadership in precision genetic medicine for rare diseases [1].

Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes [1].

The milestone payment is expected to be received within 60 days, as per the terms of the agreement. Arrowhead currently expects to achieve the second enrollment target by the end of 2025, which would trigger an additional $200 million milestone payment from Sarepta [1].

Arrowhead's progress in meeting these enrollment targets underscores its commitment to advancing its pipeline of investigational therapies, particularly in the field of rare genetic diseases. The company's ability to secure milestone payments from partners like Sarepta highlights its potential for future growth and success in the biopharmaceutical sector.

References:
[1] Arrowhead Pharmaceuticals, Inc. (2025). Arrowhead Pharmaceuticals, Inc. Earnings $100 Million Milestone Payment from Sarepta Therapeutics. Retrieved from https://www.businesswire.com/news/home/20250728872484/en/