Sarepta’s Duchenne gene therapy slows disease progression, three-year data shows
Sarepta Therapeutics’ gene therapy for a muscle‑wasting disorder slowed disease progression and showed sustained benefit in patients’ ability to control and coordinate movement three years after treatment, sending its shares up about 4% premarket.
The therapy, Elevidys, helped patients perform physical activities such as jumping, standing on one leg, and hopping on one foot, better than a pre-planned group of untreated patients from outside the trial, the company said.
The late-stage study enrolled participants who were aged four to seven at the time of the treatment and were still able to walk.
The company said those treated with Elevidys showed a 70% or higher slowing of disease progression as measured by time to rise from the floor and time to complete a 10-meter walk or run, compared to the external control group.
Sarepta has faced heightened scrutiny over the safety and effectiveness of the therapy after two non-ambulatory teenage boys died last year due to acute liver failure associated with the treatment.
Oppenheimer analysts said while investors are generally skeptical of studies using external control groups, they expect the data to improve physician and patient perceptions of Elevidys’ benefit-risk profile and help accelerate commercial uptake.
Sarepta, which has been grappling with weakening sales, said no new treatment-related safety signals were observed.
Elevidys is approved to treat patients aged four and older who are still able to walk, although it had failed to show a statistically significant improvement in motor function in boys aged four to seven after 52 weeks in a late-stage study.
The prescribing information for Elevidys carries the U.S. health regulator’s most serious safety warning for acute serious liver injury and acute liver failure, as well as stringent monitoring requirements after treatment.
