Summit, Sarepta in $584M-plus DMD pipeline license pact in Europe

Buoyed by its recent, and controversial, FDA approval for its Duchenne drug Exondys 51 (eteplirsen), Sarepta ($SRPT) has signed a pact potentially worth more than half a billion dollars with the U.K.’s Summit Therapeutics ($SMMT), giving it rights to its Duchenne candidate in Europe and elsewhere.

Specifically, the two have penned the exclusive license to allow Sarepta rights in Europe, as well as in Turkey and the Commonwealth of Independent States to Summit’s utrophin modulator pipeline, including its lead DMD clinical candidate--the midstage ezutromid.

On top of this, Sarepta also gets the option to license Latin American rights to Summit’s utrophin modulator pipeline. Summit, however, will keep the rights to its pipeline across the rest of the world.

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Ezutromid (aka SMT C1100) works by boosting the protein utrophin in an effort to compensate for the missing dystrophin at the heart of DMD--improving symptoms of the disease without needing to home in on particular mutations.  

This is a different approach from Sarepta’s work with Exondys, which sees its drug target a small subpopulation of DMD patients with a specific mutation--whereas Oxford, U.K.-based Summit wants to treat all DMD patients by modulating levels of utrophin.

Breaking down the deal, Summit gets an upfront fee of $40 million, but could get up to $522 million should testing go well, and is also in line for $22 million milestone upon the first dosing of the last patient in Summit’s ongoing Phase II PhaseOut DMD trial.

It can also look forward to escalating royalties ranging from a low- to high-teens percentage of net sales in the licensed territory, should it gain approval, as well as development and regulatory milestones related to its next-gen utrophin modulators.

In a statement, the pair said: “Sarepta and Summit will share specified utrophin modulator-related research and development costs at a 45%/55% split, respectively, beginning in 2018. If Sarepta elects to exercise its option for Latin American rights, Summit would be entitled to additional fees, milestones and royalties.”

Summit was up by more than 70% premarket on the news this morning.

Edward Kaye, Sarepta’s CEO, said: “This partnership with Summit Therapeutics furthers our commitment to invest in innovative approaches to treating Duchenne and supports our common goal of improving the lives of patients with DMD.

“Summit’s utrophin modulation technology represents a potentially promising approach to treat DMD, which may complement our current approach of exon skipping therapy.”

Last month, the FDA approved Exondys 51 for DMD patients with the dystrophin gene amenable to exon 51 skipping. But the circumstances in which it was approved, on what was regarded by many in the FDA as weak data from just a 12-person study with no placebo-control, proved controversial after CDER Director Dr. Janet Woodcock essentially overruled their concerns and allowed the med on the market.

In documents released by the FDA alongside the approval, the regulator noted that “Dr. Woodcock cautioned that, if Sarepta did not receive accelerated approval for eteplirsen, it would have insufficient funding to continue to study eteplirsen and the other similar drugs in its pipeline.”