Roche and Sarepta Partner on Duchenne Muscular Dystrophy Therapy


Sarepta Therapeutics and Roche inked a licensing deal that gives Roche exclusive commercial rights to SRP-9001, Sarepta’s investigation gene therapy for Duchenne muscular dystrophy (DMD), outside the U.S.

DMD is a muscle wasting disease caused by mutations in the dystrophin gene. It is a progressive disease that usually causes death in early adulthood, 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.

Sarepta is the only company to have an approved treatment for DMD on the market, Exondys 51, which was approved in 2016 after a contentious and year-long public process. Exondys 51, as the name suggests, is for DMD patients with a confirmed mutation amenable to exon 51 skipping.

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On December 13, the U.S. Food and Drug Administration (FDA) approved Sarepta Therapeutics’ Vyondys 53 (golodirsen) for DMD patients amenable to skipping exon 53.

The agency had originally issued a Complete Response Letter (CRL) in August for Vyondys 53 related to the risk of infections at intravenous infusion ports and renal toxicity observed in preclinical models. After the CRL, Sarepta made a formal dispute resolution request to the agency, which was quickly evaluated and resolved by Peter Stein, director of the Office of New Drugs (OND), which granted the appeal.

The agency is requiring Sarepta to conduct a confirmatory trial, which Sarepta expects to complete by 2024.

SRP-9001 is a micro-dystrophin gene therapy candidate that delivers the microdystrophin-encoding gene directly to muscle tissue. It uses a modified virus, AAVrh74 that has a strong affinity for muscle tissue. It also has a muscle-specific promoter called MHCK7, which is designed to enhance the activity of the gene in heart and skeletal muscles.

Under the terms of the agreement, Roche is paying Sarepta $1.15 billion up front and an equity investment. It is eligible for up to $1.7 billion in regulatory and sales milestones and royalties on net sales, likely to be in the mid-teens. The two companies will equally share global development costs. Sarepta will continue to hold rights to SRP-9001 in the U.S.

“As a mission-driven organization, we are inspired to partner with Roche with the goal of bringing SRP-9001 to patients outside the United States,” said Doug Ingram, president and chief executive officer of Sarepta. “This collaboration will not only increase the speed with which SRP-9001 could benefit DMD patients outside the United States, but will also greatly expand the scope of territories within which we could potentially launch SRP-9001 and improve and safe lives.”

Sarepta will still be responsible for the global development plan and manufacturing plans for SRP-9001. The company has also given Roche an option to buy rights outside the U.S. to certain future DMD-specific programs for separate milestone, royalty and cost-sharing considerations.

“We are excited to enter this licensing agreement with Sarepta,” said James Sabry, head of Roche Pharma Partnering. “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.”

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