Sarepta Joins Roche to Speed DMD Gene Therapy Development, Access Outside US

Sarepta Therapeutics is partnering with Roche to accelerate the development and expand the availability of SRP-9001 — its leading gene therapy candidate for Duchenne muscular dystrophy (DMD) — outside the Unites States.

DMD is caused by deficient production of dystrophin — an essential protein for muscle integrity — due to mutations in the DMD gene.

SRP-9001 uses a modified and harmless adeno-associated virus (AAV) — AAVrh74 — to deliver the micro-dystrophin gene, a shorter but functional copy of the DMD gene, to the muscle tissue.

In addition to using the viral vector’s ability to specifically target muscle cells, SRP-9001 is intended to promote the activity of the micro-dystrophin gene in skeletal and heart muscle cells — the main targets of DMD.

By providing a source of functional micro-dystrophin protein to the most affected tissues, one-time treatment with SRP-9001 aims to slow or halt muscle degeneration in people with DMD.

Positive data from preclinical studies and a Phase 1/2 trial (NCT03375164) led to a placebo-controlled Phase 2 clinical trial, Study-102 (NCT03769116), evaluating the safety and effectiveness of a single dose of SRP-9001 in as many as 41 boys with DMD. The study is scheduled to end in 2022.

Results from the Phase 1/2 trial found that after nine months of treatment, four boys showed micro-dystrophin production in the muscles, as well as improved muscle function and no signs of adverse effects.

Sarepta said the collaboration combines its potential gene therapy with Roche’s global reach, commercial presence, and regulatory expertise to speed patients’ access to SRP-9001 outside 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,” Doug Ingram, Sarepta’s president and CEO, said in a press release.

Sarepta will give Roche exclusive commercial rights to SRP-9001 in countries other than the U.S. in exchange for an upfront payment of $1.15 billion. The company may also receive up to $1.7 billion in regulatory and sales milestone payments, in addition to royalties on net sales.

While Sarepta will continue to be responsible for SRP-9001’s clinical development and manufacturing, the companies will split global development costs. In the future, Roche will have the option to acquire rights to other DMD-specific programs from Sarepta outside the U.S., under additional milestone and royalty payments, as well as cost sharing.

“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 save lives,” Ingram said.

The agreement not only validates SRP-9001’s therapeutic potential, but also provides “the resources and focus to accelerate our gene therapy engine and, if successful, bring SRP-9001 to patients as quickly as possible, potentially transforming the lives of countless DMD patients across the globe,” he said.

James Sabry, head of pharma partnering at Roche, said his company is “excited” about the partnership, and hopes it will “fundamentally transform the lives of patients and families” affected by DMD.

A replay of a conference call Sarepta hosted to discuss the partnership is available here.