Rare Daily Staff

Sarepta Therapeutics and Roche have entered into a blockbuster licensing agreement granting Roche exclusive rights to commercialize Sarepta’s experimental micro-dystrophin gene therapy SRP-9001 to treat Duchenne muscular dystrophy outside the United States.

Under the terms of the agreement, Sarepta will receive an upfront payment of $1.15 billion comprised of $750 million in cash and $400 million in equity. In addition, Sarepta is eligible to receive up to $1.7 billion regulatory and sales milestones, and royalties on net sales. Sarepta will continue to be responsible for the clinical development and manufacturing of SRP-9001 with global clinical development costs shared equally with Roche. Roche also has an option to acquire ex-U.S. rights to certain future DMD-specific programs, in exchange for separate milestone and royalty considerations, and cost sharing.

Duchenne muscular dystrophy (DMD) is a rare genetic disorder characterized by progressive muscle deterioration and weakness that primarily affects boys with symptoms beginning as early as three years of age. It is the most common type of muscular dystrophy. DMD is caused by an absence of dystrophin, a protein that helps keep muscle cells intact. Progressive muscle weakness in the lower limbs spreads to the arms, neck and other areas of the body. The condition is universally fatal, and death usually occurs before the age of 30 generally due to respiratory or cardiac failure. DMD occurs in about one out of every 3,600 male infants worldwide.

Currently Sarepta’s two approved exon-skipping DMD treatments address about a fifth of the total DMD population. Its experimental gene therapy SRP-9001, currently in clinical development for DMD, is designed to deliver the micro-dystrophin-encoding gene directly to the muscle tissue for the targeted production of the micro-dystrophin protein. It could potentially address the entire DMD population and provide a cure for the debilitating and fatal disorder.

“This collaboration will not only increase the speed with which SRP-9001 could benefit patients outside the United States, but will also greatly expand the scope of territories within which we could potentially launch SRP-9001 to improve and save lives,” said Doug Ingram, president and CEO of Sarepta.

The deal affirms Roche’s moves to be a leader in gene therapy, coming a week after receiving Federal Trade Commission approval to acquire gene therapy pioneer Spark Therapeutics for $4.3 billion.

The size of the deal also reflects the potential for Sarepta’s gene therapy, which

uses the AAVrh74 vector for delivery, which may avoid many of the safety issues that have plagued AAV9 vector delivery approaches and speaks to Sarepta’s ability to the scale up that will be needed for global commercial manufacturing.

“By working together to provide SRP-9001 to patients, we hope to fundamentally transform the lives of patients and families living with this devastating disorder for which there are currently only limited treatment options,” said James Sabry, head of partnering at Roche.

Photo: Doug Ingram, president and CEO of Sarepta

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