Roche has signed a licensing deal worth up to $2.85 billion with Sarepta Therapeutics for the exclusive ex-US commercial rights to the latter’s SRP-9001 (AAVrh74.MHCK7.micro-dystrophin), an investigational gene therapy for Duchenne muscular dystrophy (DMD).

As per the agreement terms, Sarepta Therapeutics will get an upfront payment made up of $750 million in cash and $400 million in equity by the Swiss pharma giant. Additionally, the US gene therapy company will be entitled to be paid up to $1.7 billion in the form of regulatory and sales milestones payments and royalties on net sales of the investigational DND gene therapy.

Furthermore, Roche and Therapeutics will equally share the global development expenses to be incurred on SRP-9001.

The Swiss pharma giant said that the collaboration will help speed up access to the potential DND gene therapy for patients outside the US.

Commenting on the Roche, Sarepta deal, James Sabry – head of Roche Pharma Partnering said: “We are excited to enter this licensing agreement with Sarepta. 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.”

Currently, in clinical development for DMD, SRP-9001 is designed to supply the microdystrophin-encoding gene directly to the muscle tissue to enable the targeted production of the microdystrophin protein.

Doug Ingram – president and CEO of Sarepta Therapeutics, commenting on the Roche, Sarepta deal, said: “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.

“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.”

As part of the deal, the Swiss pharma giant also has an option to acquire the ex-US rights to certain future DMD-specific programs from the US gene therapy company, in exchange for separate milestone and royalty considerations, and also cost sharing.

The transaction is subject to the expiration or termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 and other customary conditions. The parties anticipate that the agreement will close in the first quarter of 2020.

The Roche, Sarepta deal, which is subject to the expiration or termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 and other customary conditions, is likely to close in Q1 2020.

Recently, Roche signed a deal worth up to $1.39 billion to acquire Promedior, a Massachusetts-based biotech company. (Read about Roche acquisition of Promedior here)

  Duchenne muscular dystrophy, Gene therapy company, James Sabry, Massachusetts, Roche, Sarepta Therapeutics, SRP-9001, USA