Shares of Sarepta Therapeutics soared more than 50 percent Tuesday after the company presented preliminary data for its experimental treatment for patients with Duchenne muscular dystrophy.
The rare genetic disease causes muscle wasting and can be fatal before patients turn 30. Symptoms usually start in early childhood, usually between 3- and 5-years old. It primarily affects boys. But in rare cases can affect girls.
Sarepta shared results from three children to receive the gene therapy in a phase 1/2a clinical trial.
Creatine kinase is an enzyme found within muscle cells and can leak into the bloodstream when damage has occurred in the muscle. Elevated levels of it can indicate someone has Duchenne muscular dystrophy.
Patients that received Sarepta’s drug showed an average reduction of more than 87 percent in their creatine kinase levels on day 60.
Sarepta closed Monday with a market value of $6.9 billion, according to FactSet. That rocketed higher to about $10.6 billion during Tuesday’s surge.
The company’s shares have gained nearly 200 percent this year.