EPIDYS Trial Supports Givinostat's Efficay, SRP-9001 in EMBARK Study, Eteplirsen Shows Long-Term Safety

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Welcome to this special edition of Neurology News Network. I’m Marco Meglio. This week’s episode is centered around coverage of the 2024 MDA Clinical and Scientific Conference.

In a new supportive post hoc analysis from the phase 3 EPIDYS trial, findings showed a positive significant difference with givinostat (Italfarmaco SpA), a HDAC inhibitor, in motor function among patients with Duchenne muscular dystrophy in comparison with controls. These results are consistent with efficacy in the prespecified analyses and emphasize the potential efficacy of givinostat in a broader context. In the overall intention-to-treat (ITT) population (n = 179), investigators reported that givinostat displayed a statistically significant difference compared with the control in change from baseline at 18 months in the timed 4 stair climb. Overall, investigators noted that the treatment effect estimates relating to the key secondary end points consistently favored givinostat over the control, which supports the primary end point result.

New findings from part 1 of the phase 3 EMBARK study (NCT05096221) assessing Sarepta Therapeutics’ gene therapy delandistrogene moxeparvovec (SRP-9001) showed that the agent failed to meet its primary end point in change among patients with Duchenne muscular dystrophy (DMD); however, did demonstrate several benefits on other secondary analyses. Presented at the 2024 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, held March 3-6, in Orlando, Florida, treatment with the agent resulted in a nominal difference in change on North Star Ambulatory Assessment (NSAA) total score vs placebo over 52 weeks (SRP-9001 change: 2.6; n = 63; placebo: 1.9; n = 61). Using a cohort of ambulatory patients aged between 4 and 8 years with a confirmed DMD mutation within exons 18-79, there were no identified new safety signals, reinforcing the favorable safety profile.

At the 2024 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, held March 3-6, in Orlando, Florida, investigators presented real-world, interim data from the phase 4 EVOLVE trial, a confirmatory study assessing the treatment effects of eterplirsen (Exondys 51; Sarepta Therapeutics). All told, in a subgroup of patients, treatment with the exon-skipping agent demonstrated persisted effects and a safety profile that was consistent with previous clinical trials. The interim analysis included patients who were nonambulatory at eteplirsen treatment initiation of became nonambulatory after eteplirsen initiation. Of the 123 eteplirsen-treated patients enrolled in the study as of December 2021, 41 (33%) were nonambulatory at treatment initiation.

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