Arrowhead, Sarepta Advance on Pact

Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that it has earned a $100-million milestone payment from Sarepta Therapeutics (NASDAQ: SRPT).

This morning’s news release reported that the milestone was triggered when Arrowhead reached the first of two prespecified enrollment targets and subsequent authorization to dose escalate in a Phase 1/2 clinical study of ARO-DM1, an investigational RNA interference (RNAi) therapeutic for the treatment of type 1 myotonic dystrophy (DM1), the most common adult-onset muscular dystrophy.
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.

In accordance with the license and collaboration agreement, Arrowhead expects to receive this payment within 60 days.

Arrowhead and Sarepta signed a global licensing and collaboration agreement in November 2024, which closed in February 2025, whereby Sarepta received rights to multiple investigational treatments that leverage Arrowhead’s leading Targeted RNAi Molecule platform.

The release goes on to say 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.

ARWR shares vaulted 44 cents, or 2.8%, to $16.30, first thing Monday while those for SRPT acquired 13 cents, or 1.1%, to $12.06.