Sarepta Takes Another Big Step Forward With DMD Treatment

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Sarepta Therapeutics Inc. (NASDAQ: SRPT) saw its shares hit a new multiyear high on Friday after the firm announced that it received a key approval from the U.S. Food and Drug Administration (FDA). Specifically, the firm announced that its Investigational New Drug (IND) application for the GALGT2 gene therapy program was cleared by the FDA. Also Nationwide Children’s Hospital is on track to initiate the Phase 1/2a clinical trial in individuals with Duchene muscular dystrophy (DMD) by year-end 2017.

The program is exploring a potential surrogate gene therapy approach to treat DMD, targeting the dystroglycan complex to preserve muscle function. In animal models, overexpression of GALGT2 results in muscle that function normally even in the absence of dystrophin.

Overall, this approach represents a potential new pathway to treat the root cause of DMD.

Back in January 2017, Sarepta announced a license agreement with Nationwide Children’s for the GALGT2 gene therapy program.

Douglas Ingram, Sarepta’s president and CEO, commented:

The field of gene therapy represents a potentially transformative approach to the treatment of Duchenne muscular dystrophy. We are very proud to be partnered with gene therapy luminaries Drs. Flanigan and Martin as they rapidly advance our partnered GALGT2 program and begin to treat DMD patients this year.

Ingram continued:

The GALGT2 program offers the potential to treat the majority of dystrophin gene mutations responsible for this devastating disease, DMD, as well as having potential utility in other muscular dystrophies, and perfectly exemplifies our strategy of working with urgency to find and progress potentially life changing new treatments for DMD through agnostically investing in precision genetic medicine.

While the broad markets have had a fantastic year in 2017, Sarepta has done even better, with the stock up about 86% year to date, excluding Friday’s move. However, over the past 52 weeks the stock is only up about 36%.

Shares of Sarepta were last seen up about 5% at $53.71, with a consensus analyst price target of $69.24 and a 52-week range of $26.26 to $53.79.