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October 4, 2016

Sarepta, Summit to Partner on Utrophin Modulator DMD Therapies

  • Sarepta Therapeutics has acquired rights to the pipeline of Summit Therapeutics—led by Phase II Duchenne muscular dystrophy (DMD) candidate ezutromid—for Europe, Turkey, and the Commonwealth of Independent States, the companies said today.

    The companies’ exclusive license and collaboration agreement could generate up to $562 million-plus for Summit.

    Under the agreement, Sarepta obtained an option to license Latin American rights to the pipeline and agreed to collaborate with Summit on developing novel DMD therapies.

    Summit retains commercialization rights to the pipeline in all other countries.

    “Summit's utrophin modulation technology represents a potentially promising approach to treat DMD, which may complement our current approach of exon-skipping therapy,” Sarepta CEO Edward Kaye, M.D., said in a statement.

    Sarepta’s agreement with Summit comes less than a month after Sarepta won the FDA’s first approval of a DMD treatment. Eteplirsen, which is indicated for DMD amenable to exon 51 skipping, will be marketed as Exondys 51 and carry an average cost to patients of $300,000 for a year’s treatment.

    In its latest agreement with Summit, Sarepta agreed to pay $40 million upfront and up to $522 million in payments tied to achieving development, regulatory, and sales milestones for ezutromid—now the subject of a Phase II proof-of-concept trial called PhaseOut DMD.

    Potential milestone payments include $22 million upon the first dosing of the last patient in PhaseOut DMD and escalating royalties ranging from a low to high teens percentage of net sales in territories covered by the license, the companies said.

    Summit will also be eligible to receive development and regulatory milestones related to its next-generation utrophin modulators. Sarepta and Summit said they agreed to share utrophin modulator-related R&D costs at a 45%/55% split, respectively, beginning in 2018.

    Summit would be entitled to additional fees, milestones, and royalties should Sarepta elect to exercise its option for Latin American rights.

    Added Summit CEO Glyn Edwards: “This agreement provides us with access to Sarepta's development, regulatory, and commercialization expertise for the continued advancement of our promising utrophin modulator pipeline.”

    Eteplirsen survived an FDA review process in which the agency went against recommendations by two advisory committees not to approve the treatment. The agency wrestled with concerns about inadequate clinical data—FDA Commissioner Robert Califf, M.D., called for the retraction of a 2013 study—and counterarguments by parents of boys with DMD that neither FDA nor clinical studies had found any safety problems with eteplirsen, and that the drug improved patients’ conditions.

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