Sarepta Therapeutics (SRPT) said Friday that new data from two separate studies of its gene therapy Elevidys showed high levels of protein expression in toddlers and statistically significant functional improvements in older boys with Duchenne muscular dystrophy.
In the Endeavor study, children who were 2 years old at the time of treatment showed an average protein expression level of nearly 94% twelve weeks after dosing, the company said.
The safety profile in this group aligned with earlier findings, with nausea and vomiting as the most common side effects, Sarepta said, adding that elevated liver enzymes occurred in two participants but resolved with steroids.
Sarepta said that in the Embark trial, boys aged 8 to 9 years who were treated after receiving a placebo in the earlier phase showed statistically significant gains one year after treatment.
Compared to an external control group, they improved by 4.75 points on the North Star Ambulatory Assessment, reduced time to rise by nearly 7 seconds, and completed a 10-meter walk/run about 4.76 seconds faster, the company said.
No new safety concerns emerged over two years, according to Sarepta.
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