Shares of Sarepta Therapeutics (SRPT) skyrocketed more than 169% after the company posted positive results from a walking test of its investigational muscular dystrophy drug.
The positive trial results overshadowed an announcement that the U.S. Department of Defense was dropping the Ebola portion of its contract for hemorrhagic fever virus therapeutics with Sarepta due to defense budget cuts.
The developer of RNA-based therapeutics said its compound, eteplirsen, met the primary endpoint of the study by improving the outcome in a 6-minute walk test over the placebo with patients with Duchenne muscular dystrophy.
Administered once weekly at either a 30 milligram dosage or 50 mg for 48 weeks, eteplirsen showed a “statistically significant increase” in dystrophin-positive fibers, Sarepta said.
"These data represent a significant milestone and a defining moment of progress and hope for patients with DMD and their families, as well as for those of us in the scientific community who have been pursuing potential treatments for this devastating and deadly disease for decades," said Dr. Jerry Mendell, director of the Centers for Gene Therapy and Muscular Dystrophy at Nationwide Children's Hospital and this study’s principal investigator.
Duchenne muscular dystrophy is a genetic neuromuscular disorder that leads to severe progressive muscle loss and premature death.
By addressing the underlying cause of DMD, the investigational drug helps enable dystrophin production and slows the progression of the disease.
While eteplirsen is targeted at patients with a specific genetic mutation, Mendell said he thinks there are benefits for all DMD patients.