Sarepta jumps on J.P. Morgan update, gene therapy DMD deals

Sarepta and Nationwide Children’s Hospital hope to create a new gene therapy approach to DMD.

Sarepta saw a 21% bump in its share price on Tuesday after it announced a new, early-stage research deal that could help treat all Duchenne muscular dystrophy (DMD) patients, and told investors in San Francisco that its new and controversial med for the disease, Exondys 51 (eteplirsen), had already made $5.4 million after its fall approval.

The research deals are with Nationwide Children’s Hospital and will see the pair work on their microdystrophin gene therapy program, as well as another form of gene therapy.

An initial phase 1/2a trial for the microdystrophin gene therapy is slated to begin at the end of the year and will be done at Nationwide Children’s.

Parent Project Muscular Dystrophy (PPMD) has committed $2.2 million to the trial, with support from additional Duchenne foundations and families.

Sarepta says it has “committed to the trial through a separate research agreement with Nationwide Children’s, and has an exclusive option to license the program,” although exact terms and dollar values were not given.

Sarepta noted that PPMD's grant “provided incentive” for the biotech to help “expand and accelerate this opportunity.”

It also penned an exclusive license agreement with Nationwide for their Galgt2 gene therapy program, originally developed by researcher Dr. Paul Martin.

This early-stage program aims to research a potential surrogate gene therapy approach to DMD, whereby the gene therapy looks to induce genes that make proteins that can perform a similar function as dystrophin.

The goal will be to produce a muscle cell that can function normally even when dystrophin is absent, Sarepta says.

Its current FDA-approved DMD med can only treat certain patients, namely those with the mutation of the dystrophin gene amenable to exon 51 skipping, which affects about 13% of the population with DMD.

It is hoping with these tie-ups it could produce a gene therapy med that could treat many more, if not all, patients with the disease, although this is still some years off.

“We are pleased to have this opportunity to help advance our Galgt2 gene therapy program,” said Dr. Kevin Flanigan, the principal investigator leading the clinical trial. “Our goal is to have this program in the clinic during 2017 and begin to evaluate a therapy that has the potential to treat patients of all ages and disease severity.”

DMD is a rare genetic disorder characterized by progressive muscle deterioration and weakness. The disease primarily affects young boys, and occurs in about one out of every 3,600 male infants worldwide.

“Given the complexities of Duchenne muscular dystrophy, we know that it is going to require multiple treatment approaches,” said Edward Kaye, Sarepta’s CEO.  

“With that goal in mind, we are excited to support clinical development for Nationwide’s gene therapy program with the goal to help all boys with DMD.”

The biotech’s shares jumped 21% on news, first reported by TheStreet and later confirmed by Kaye at JPM, that 250 patients have already started going through the process to get reimbursed for Exondys 51.