5. Roche/Sarepta

Roche
Roche’s determination to build up its position in gene therapy was apparent in 2019 through a $4.3 billion takeover play for Spark Therapeutics, and its $2.85 billion deal to license rights to Sarepta’s DMD candidate SRP-9001. (Roche)

Licensee: Roche (Genentech)
Licensor: Sarepta Therapeutics
Deal size: $2.85 billion
Upfront: $1.15 billion ($750 million cash/$400 million equity)
Asset: Duchenne muscular dystrophy gene therapy SRP-9001

Roche’s determination to build up its position in gene therapy was apparent in 2019 through a $4.3 billion takeover play for Spark Therapeutics, and its $2.85 billion deal to license rights to Sarepta’s SRP-9001, currently leading the field among gene therapies for inherited muscle-wasting disorder Duchenne muscular dystrophy (DMD).

The upfront fee—made up of $750 million in cash and $400 million in equity—gives Roche the rights to SRP-9001 outside the U.S., with Sarepta also in line to receive up to $1.7 billion in regulatory and sales milestones, plus royalties on sales.

Roche and Sarepta will also share global development expenses, but the scale of the deal for ex-U.S. rights only is a clear indicator of how much commercial potential Roche believes there is in the program.

In early-stage testing involving four patients, SRP-9001 was able to achieve expression levels of up to 96% for microdystrophin—a replacement for the protein that is dysfunctional in DMD—with what seemed to be a benign safety profile.

The levels of microdystrophin expression were well above what Sarepta previously said should be sufficient to affect the course of disease progression in DMD, which prompted the biotech to start a 41-patient placebo-controlled trial (Study 102) that is now fully enrolled with a readout due early in 2021.

At the moment, it looks like SRP-9001 is out in front among DMD gene therapies in clinical development. including candidates from Pfizer and Solid Biosciences which were both placed on clinical hold by the FDA because of safety concerns.

Sarepta’s chief executive, Doug Ingram, said at the J.P. Morgan Healthcare Conference in January that the Roche deal is “the largest ex-U.S. single candidate license of its kind in pharmaceutical history” and would allow SRP-9001 to reach vastly more DMD patients much more quickly.

He went on: “For those who wonder whether that's meaningful, consider this, every single day, every hour of every day, around the world, every hour, a child with [DMD] dies.”

Roche has tried other ways to tackle DMD but suffered a setback last year when it abandoned myostatin inhibitor RG6206, which had reached the phase 3 development stage.

5. Roche/Sarepta