After assuring investors last week that beleaguered Sarepta Therapeutics would likely fulfill its financial duties in a deal with Arrowhead Pharmaceuticals, the latter has publicly stated that it has surpassed the threshold to earn its $100 million milestone payment.
The payment is tied to Arrowhead hitting the first enrollment target in a phase 1/2 trial for an investigational RNA interference (RNAi) therapeutic designed to treat type 1 myotonic dystrophy (DM1). The $100 million check is also dependent upon regulatory approval to launch dose escalation in the same study.
Arrowhead said it has met both of these conditions in a July 28 release and now expects to receive the $100 million within 60 days.
The company also said it plans on reaching the second enrollment target by the end of this year, which would trigger a $200 million payment from Sarepta.
Back in November of last year, Sarepta struck a major deal with Arrowhead, making a $500 million upfront payment and a $325 million equity investment to secure rights to seven programs. The pact requires Sarepta to pay $50 million a year for five years plus the $300 million in milestone payments associated with the DM1 study’s enrollment.
The announcement comes a few days after Arrowhead said it believes Sarepta will remain good on the deal and comes amid escalating problems for Sarepta stemming from several deaths occurring in patients who received its gene therapies.
Investors have called for more transparency surrounding the genetic medicine company’s clinical trials, and the company has halted all shipments of its Duchenne muscular dystrophy treatment Elevidys after a public back-and-forth with the FDA.
Earlier this month, Sarepta unveiled a massive restructure that included laying off 500 staffers and the discontinuation of several limb-girdle muscular dystrophy (LGMD) programs in efforts to save $400 million annually. The company will also pivot away from gene therapies to focus on siRNA, which underpins the Arrowhead collaboration.
Shortly thereafter, news publication BioCentury reported a patient death had occurred in one of the LGMD trials in June, an event that was not initially publicly shared by Sarepta.
The FDA has since placed a clinical hold across all of the company’s investigational LGMD trials, which are evaluating gene therapies engineered by Sarepta using the same viral vector as Elevidys.