Sarepta's Elevidys shows durable benefit in ambulatory Duchenne patients, analysts see turnaround potential

Jefferies analysts said Sarepta Therapeutics could become a turnaround story after the company presented new data on its Duchenne muscular dystrophy gene therapy Elevidys.

The late-breaking poster displayed two-year results from 25 patients aged 8-12 who received Elevidys in the Phase 3 EMBARK and Phase 1b ENDEAVOR studies. The data were shown at the 31st World Muscle Society Congress in Hiroshima, Japan.

Compared with well-matched external controls, the patients demonstrated clinically meaningful improvements in ambulatory motor function, time to rise from the floor and gait speed. Sarepta reported a sustained treatment effect and a slowing of disease progression, supporting Elevidys for ambulatory DMD patients.

Analysts view the results as a positive catalyst, adding to a series of milestones that could extend through 2027.