Date: 2016-10-04
Type of information: Licensing agreement
Compound: utrophin modulator pipeline including ezutromid
Company: Sarepta Therapeutics (USA - MA) Summit Therapeutics (UK)
Therapeutic area: Rare diseases - Genetic diseases - Neuromuscular diseases
Type agreement: licensing R&D
Action mechanism: utrophin modulator. SMT C1100 is a proprietary, orally available small molecule with a novel mechanism of action for DMD. SMT C1100 acts to modify the progression of DMD by replacing dystrophin with an endogenous, functionally similar protein called utrophin. SMT C1100 is designed to upregulate and maintain the production of utrophin. Utrophin is a protein that is highly expressed in regenerating muscle, but decreases as the muscle fibre matures and is eventually replaced by dystrophin, a protein that maintains the integrity and healthy function of muscles. Patients with DMD are unable to make dystrophin, resulting in muscle fibre degeneration. However, if utrophin is continually expressed in the mature muscle fibre, it can replace the function of dystrophin and thereby overcome the deficit in patients with DMD.
This approach is expected to be a universal treatment for all DMD patients regardless of whether the disease was caused by an inherited or spontaneous genetic mutation. Summit has demonstrated in nonclinical efficacy studies that SMT C1100 is capable of increasing utrophin to restore and maintain the healthy function of muscles.
Disease: Duchenne muscular dystrophy
Details: * On October 4, 2016, Sarepta Therapeutics and Summit Therapeutics announced that they have entered into an exclusive license and collaboration agreement granting Sarepta rights in Europe, as well as in Turkey and the Commonwealth of Independent States (‘the licensed territory’), to Summit’s utrophin modulator pipeline, including its lead clinical candidate, ezutromid, for the treatment of Duchenne muscular dystrophy. As part of the agreement, Sarepta also obtains an option to license Latin American rights to Summit’s utrophin modulator pipeline. Summit retains commercialization rights in all other countries. Ezutromid is currently in a Phase 2 proof of concept trial called PhaseOut DMD.
Financial terms: Under the terms of the agreement, Summit will receive an upfront fee of $40 million. In addition, Summit will be eligible for future ezutromid related development, regulatory and sales milestone payments totalling up to $522 million, including a $22 million milestone upon the first dosing of the last patient in Summit’s PhaseOut DMD trial, and escalating royalties ranging from a low to high teens percentage of net sales in the licensed territory. Summit will also be eligible to receive development and regulatory milestones related to its next-generation utrophin modulators. 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.
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