Roche gets back to gene therapy

DMD Deal

As soon as the Federal Trade Commission (FTC) told Roche it could acquire Spark Therapeutics and its numerous treatments for genetically driven diseases, the Swiss company embarked on a $1.2 billion deal with Sarepta Therapeutics for the biotech's experimental Duchenne muscular dystrophy gene therapy.

Because of the scale of the collaboration, financial analysts think that Sarepta has a clear lead in DMD, according to an article in Biopharma Dive by Jonathan Gardner. The article pointed out that “its rivals have stumbled: $750 million in cash and a $400 million share purchase gives Roche rights to the therapy, called SRP-9001, in markets outside the United States.” In addition, the collaboration will entail cost sharing for global development and as much as $1.7 billion in regulatory and sales milestones, “with royalties on sales estimated in the mid-teens percentage.”

Sarepta CEO Douglas Ingram told analysts that the total value of the deal might be as much as $10 billion. As he explained, "It's a number one gets to fairly easily with even modest penetration ex-U.S."

Because of the deal, Swiss pharma companies have become the biggest global players in gene therapy, Gardner said. Novartis purchased AveXis for $8.7 billion for the spinal muscular atrophy treatment Zolgensma, By acquiring Spark for $4.8 billion, Roche obtained Luxturna, a gene therapy for a rare form of blindness, along with experimental drugs to treat hemophilia.

Analysts see the deals as a competition in rare forms of degenerative diseases. Roche owns rights to risdiplam, a non-gene-therapy treatment for SMA. With AveXis, Novartis gained assets for treating Rett syndrome and amyotrophic lateral sclerosis (ALS).

Ingram said that the Sarepta deal came after a "robust, competitive process" said, but he did not say which other bidders were involved. Big pharma's attention to SRP-9001 was probably increased by the struggles of other experimental gene therapies. Solid Biosciences' SGT-001 has been put on a clinical hold from the Food and Drug Administration (FDA), and Pfizer's drug has had safety issues. Ingram added that his company's plans to market the treatment independently worldwide helped to raise the value of the winning offer. A Roche spokesperson claimed that the timing of the Sarepta announcement had nothing to do with the FTC's decision to clear the Spark deal.

Sarepta believes that Roche will be able to provide access to markets such as China that it would not be able to reach on its own. Ingram said, "There are 60,000 DMD patients living in China, but Roche has a lot of work to do before it figures out how to get to China."

Currently, SRP-9001 is in a placebo-controlled trial. The company intends to begin a clinical trial using the commercial gene therapy supply in mid-2020.

Being able to share clinical development costs will be critical in establishing the clinical benefit of SRP-9001. As Ingram said, "We're doing a very significant set of other studies that gets at ambulatory patients, non-ambulatory, younger kids, older kids. It's multi-national, multi-center and much larger than [the current clinical trial]. There is a significant amount of research [funding] requirements."