Roche, Sarepta Therapeutics sign $1.15B deal for ex-US rights to gene therapy for Duchenne’s

An analyst wrote that the deal would create value for Sarepta, which has a gene therapy in Phase II development for Duchenne muscular dystrophy, SRP-9001, but lacks the resources to commercialize it abroad.

A biotech company developing therapies for Duchenne muscular dystrophy has signed a deal with one of the world’s largest drugmakers that an analyst noted would give it much-needed muscle to market its investigational gene therapy abroad.

Basel, Switzerland-based Roche said Monday it had signed a $1.15 billion deal with Sarepta Therapeutics to obtain exclusive launch and commercialization rights to SRP-9001, Sarepta’s investigational gene therapy for DMD, outside the U.S. The deal also includes up to $1.7 billion in regulatory and sales milestones, plus royalties on sales.

Shares of Cambridge, Massachusetts-based Sarepta were up 9.4% pre-market on the Nasdaq and were up 9% when when markets opened. Roche’s shares, which trade on the SIX Swiss Exchange, were mostly flat.

SRP-9001 is currently undergoing a 40-patient, randomized and placebo-controlled Phase II study that opened in December of last year, with the protocol page listing sites at the University of California Los Angeles and Nationwide Children’s Hospital in Columbus, Ohio. The company currently markets two therapies for DMD, both antisense oligonucleotide drugs: Exondys 51 (eteplirsen), for DMD involving a gene amenable to exon 51 skipping; and Vyondys 53 (golodirsen), which won approval earlier this month for patients in whom the gene is amenable to exon 53 skipping.

Other gene therapies in development for DMD include Pfizer’s PF-06939926 and another by Audentes Therapeutics. Astellas Pharma announced earlier this month it would spend $3 billion to acquire Audentes, while Vertex Pharmaceuticals said in June it would acquire another developer of gene therapies for DMD, Exonics Therapeutics, for $245 million.

Early data on four patients from a Phase I/IIa trial of SRP-9001 were presented at a conference in October 2018, showing an increase in the micro-dystrophin gene, a surrogate for improvement in muscular strength in DMD, a disease that causes progressive muscle degeneration and weakness. Updated data on the same patients were presented at another conference in March of this year.

“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,” Sarepta CEO Doug Ingram said in a statement. “In addition to the validation that comes from joining forces with Roche, this licensing agreement – one of the most significant ex-U.S. licensing transactions in biopharma – will provide Sarepta with 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.”

In a note to investors, Cowen analyst Ritu Baral called the deal “incredibly value creating” for Sarepta given Roche’s ability to advance SRP-9001’s approval and commercialization faster than Sarepta would be able to do on its own, given the biotech company’s lack of operations or expertise outside the U.S. Indeed, she wrote, Sarepta’s many activities – including optimizing the manufacturing of the therapy and finishing pivotal clinical trials, as well as other clinical trial programs ranging from early-stage to Phase III, it is unlikely Sarepta would be able to launch SRP-9001 abroad in a timely manner or obtain optimal reimbursement for it.

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