Shares of Sarepta Therapeutics Inc. jumped Wednesday, after the drug developer said its lead candidate, a potential treatment for Duchenne muscular dystrophy, continued to help patients in an extended mid-stage trial.
THE SPARK: The Cambridge, Mass., company said patients taking the treatment, eteplirsen, saw less than a 6 percent decline in walking ability through 84 weeks. Some patients have been taking the drug for the study's entire duration, while others took a placebo for the first six months.
The patients were evaluated with a six-minute walking test, and the drug appeared to be well-tolerated with no serious side effects.
CEO Chris Garabedian said in a statement the company has now demonstrated walking stability for over a year and a half in boys who are 11 years old on average. He noted that many boys with Duchenne muscular dystrophy have lost their ability to walk by then.
THE BIG PICTURE: Duchenne muscular dystrophy is a rare and fatal genetic disease that causes increasing muscle weakness and affects one of every 3,500 boys worldwide. Sarepta said death usually occurs before patients reach age 30.
Sarepta has no drugs on the market. Eteplirsen is its most advanced product.
SHARE ACTION: Up 4 percent, or $1.53, to $39.68 in trading Wednesday morning, while broader trading indexes fell slightly. Shares had climbed about 48 percent so far this year as of Tuesday's close.