Sarepta Therapeutics Inc. (NASDAQ:SRPT) released three-year follow-up results from its EMBARK study, showing that its gene therapy ELEVIDYS slowed functional decline in ambulatory patients with Duchenne muscular dystrophy.
The analysis covered 52 patients who received ELEVIDYS at an average age of nine. After three years, treated patients maintained average scores on the North Star Ambulatory Assessment (NSAA) above their starting levels. By contrast, an external control group of 73 untreated patients showed the typical age-related deterioration, with scores falling below baseline.
Compared with the control group, patients who received ELEVIDYS experienced a 73% slower rate of disease progression based on Time to Rise testing and a 70% reduction when measured by the 10-meter walk/run test. Sarepta said the treatment benefit became more pronounced over time, with the functional gap between treated and untreated patients widening between the second and third years.
All three primary motor function endpoints showed statistically significant advantages for ELEVIDYS. On average, treated patients scored 4.39 points higher on the NSAA (p=0.0002), completed the Time to Rise test 6.05 seconds faster (p<0.0001), and finished the 10-meter walk/run 2.70 seconds quicker (p=0.0039) than the external control group.
According to the company, no new safety concerns related to the therapy emerged during the three-year observation period.
ELEVIDYS is approved by the U.S. Food and Drug Administration for ambulatory patients aged four and older with confirmed Duchenne gene mutations. More than 1,200 patients worldwide have received the therapy in both clinical trials and real-world use. Sarepta oversees U.S. regulatory approval and commercialization, while Roche manages markets outside the United States under a collaboration established in 2019.