The trial was conducted with Roche RHHBY.
The Cambridge, Mass., company's shares recently traded at $84.12, up 12%. They have slumped 40% over the past six months.
“In results from the first clinical study using commercially representative material, SRP-9001 demonstrated robust expression of micro-dystrophin and no new safety signals from prior studies,” Sarepta said.
That “supports [the drug’s] potentially differentiated profile for the treatment of Duchenne muscular dystrophy,” the company said.
“SRP-9001 is an investigational gene transfer therapy intended to deliver its micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein.”
In the study, 20 participants ages 4 through 7 were treated with a single infusion of SRP-9001. Symptoms of DMD usually appear in infants and toddlers.
In muscle biopsies from the first 11 patients taken 12 weeks after treatment, “all patients demonstrated robust transduction, with mean micro-dystrophin expression of 55.4% of normal, as measured by western blot,” Sarepta said.
Duchenne muscular dystrophy is a fatal neuromuscular genetic disease that afflicts 1 of every 3,500 to 5,000 males worldwide.
DMD is caused by a change or mutation in the gene that encodes instructions for dystrophin. This is a protein that is required for muscles to work properly and is missing or lacking in people with Duchenne.
In other drug news, TheStreet.com’s Dawn Kawamoto discussed why COVID-19 vaccines could be the start of a vaccine revolution.
And last week, the U.S. Centers for Disease Control and Prevention said people vaccinated against COVID-19 don’t have to wear a mask or socially distance in most places.