October 4, 2016
By Mark Terry, BioSpace.com Breaking News Staff
Cambridge, Massachusetts – Sarepta Therapeutics announced today that it had signed an exclusive license and collaboration deal with UK-based Summit Therapeutics. Sarepta acquired the rights to Summit’s utrophin modulator pipeline, including the lead clinical candidate, ezutromid, to treat Duchenne muscular dystrophy (DMD).
After months of drama, the U.S. Food and Drug Administration (FDA) approved Sarepta’s eteplirsen for DMD on September 19. DMD is a muscle wasting disease caused by mutations in the dystrophin gene. It is a progressive disease that usually causes death in the early twenties, with serious complications that include heart or respiratory-related problems. It mostly affects boys, about 1 in every 3,500 or 5,000 male children.
Utrophin modulation has the potential to treat at least part of DMD regardless of the specific dystrophin gene mutation. Ezutromid is presently being evaluated in a Phase II clinical trial. Both the FDA and the European Medicines Agency (EMA) have granted ezutromid orphan drug status.
The utrophin protein is functionally and structurally similar to dystrophin. Preclinical studies have suggested that when utrophin is still expressed, it has a positive effect on muscle performance.
“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,” said Edward Kaye, Sarepta’s chief executive officer, in a statement. “Summit’s utrophin modulation technology represents a potentially promising approach to treat DMD, which may complement our current approach of exon skipping therapy.”
As part of the deal, Sarepta will hold rights to the portfolio in Europe, as well as Turkey and the Commonwealth of Independent States. Sarepta will also have an option to license Latin American rights to the pipeline. Summit will hold commercialization rights everywhere else.
Sarepta will pay Summit $40 million up front. Summit may receive up to $522 million in various milestone payments, including a $22 million milestone when the last patient in the PhaseOut DMD trial receives the first dose of ezutromid. Escalating royalties that range from low to high-teens are also in the deal, if any of the compounds should make it to market.
The two companies will split research-and-development costs, with Sarepta responsible for 45 percent, and Summit 55 percent, starting in 2018.
“Sarepta Therapeutics has paved the way in the development of disease-modifying therapies for DMD with the first FDA-approved drug in the disease area, making them a strong strategic partner to support our utrophin modulator pipeline,” said Glyn Edwards, Summit’s chief executive officer, in a statement. “This agreement provides us with access to Sarepta’s development, regulatory and commercialization expertise for the continued advancement of our promising utrophin modulator pipeline. We look forward to this partnership and working together to bring great advances to patients and families living with DMD.”
Since September 19, Sarepta has been trading up, currently at $60.95.
Summit Therapeutics stock is currently trading at $8.65, down from $10.02 on September 26.
