CAMBRIDGE, Mass. — Sarepta Therapeutics has announced progress in its small interfering RNA (siRNA) collaboration with Arrowhead Pharmaceuticals, reporting that a Phase 1/2 trial of SRP-1003 for type 1 myotonic dystrophy (DM1) has met a key enrollment milestone. The achievement, following a positive safety review and the first predetermined enrollment target, has triggered a $100 million milestone payment to Arrowhead.
To help fund the payment, Sarepta sold 9,265,312 shares of Arrowhead common stock in a privately negotiated block trade, generating at least $174 million in gross proceeds. The company also agreed to transfer 2,660,989 shares of Arrowhead stock back to Arrowhead in lieu of $50 million in cash, effectively prepaying half of the milestone obligation.
“We are very pleased with the progress of our potentially best-in-class siRNA programs, including the advancement of our SRP-1003 program for DM1,” said Doug Ingram, president and CEO of Sarepta. “The sale of our equity investment is a strategic decision to help fund this milestone but does not change our conviction in the utility of the siRNA approach and our confidence in Arrowhead’s work. We look forward to sharing early data from our FSHD and DM1 programs in the second half of this year.”
SRP-1003 is an investigational RNA interference therapy aimed at treating DM1, a rare, progressive neuromuscular disorder. Sarepta expects to release preliminary results from the ongoing Phase 1/2 trial in the second half of 2025. (Source: IANS)
