Sarepta Hits Record High As Duchenne Muscular Dystrophy Gene Therapy Yields Positive Results In Early-Stage Study

Sarepta Therapeutics Inc SRPT shares, halted shortly before Tuesday's open, opened higher by more than 50 percent.

What Happened

Sarepta released Phase 1/2 data on its microdystrophin gene therapy for Duchenne muscular dystrophy, or DMD, which showed robust levels of microdystrophin, with an average of either 38.2 percent of 53.7 percent of normal levels of the protein. Three boys were evaluated in the trial.

Patients showed decreases in serum creatine kinase, an enzyme associated with muscle damage. The mean reduction of the enzyme was over 87 percent at Day 60.

The company also observed that no serious adverse events were observed in the study.

DMD affects mostly boys and makes its appearance between the ages of 3-5.

"Duchenne is caused by an inherited mutation in a gene that produces dystrophin, a protein required to make muscles function properly," said STAT News senior writer Adam Feuerstein. "Without dystrophin, muscles break down over time, which is why Duchenne patients gradually lose their ability to walk and breathe. The disease is fatal, often by the time boys are in their late teens or early 20s."

Why It's Important

"…Although the data are early and preliminary, these results, if they persist and are confirmed in additional patients, will represent an unprecedented advancement in the treatment of DMD," said Dr. Jerry Mendell, the study's principal investigator.

"The Duchenne gene is one of the largest in the body and cannot fit inside a virus needed to deliver it to cells," Feuerstein wrote. "To solve the delivery conundrum, Mendel and Rodino-Klapac disassembled the dystrophin gene and reconstructed a smaller, 'micro-dystrophin' version containing only the components necessary to make functional protein."

On Monday, Solid Biosciences Inc SLDB rallied about 19 percent after it said the FDA lifted the clinical hold it had imposed on the company's IGNITE Phase I/2 trial of its microdystrophin gene transfter, SGT-001 for treating DMD. Sarepta shares also rose about 3 percent in a sympathy move.

What's Next

Now that the early-stage study has produced positive result with a very small sample population, the company can go ahead with additional patients, studies, measures and time points before it can effectively take an approved product to the affected population.

At time of publication, Sarepta was up 55 percent at $163.02 on roughly four times the average volume.

In sympathy move, Solid Biosciences shares were up 39.45 percent to $40.97.

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