Following a tough 2025 for Sarepta, the biotech has posted positive, long-term Phase III data on its Duchenne muscular dystrophy (DMD) gene therapy, Elevidys (delandistrogene moxeparvovec) – though analysts remain sceptical on its impact.
On 26 January, Sarepta shared topline results from part one of the Phase III EMBARK study (NCT05096221), which revealed that Elevidys triggered statistically significant improvements in all key measures of motor function compared with an untreated external control group. The late-stage study enrolled participants who were aged four to seven at the time of the treatment.
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These include an average 4.39-point increase in North Star Ambulatory Assessment (NSAA) scores, as well as a 2.70-second improvement in 10-meter walk/run (10MWR) values and a -6.05 decrease in a patient’s time to rise (TTR).
NSAA values of patients that received Elevidys also remained higher than baseline at the three-year mark. This differs significantly from the external control group, which experienced a commonly observed age-related decline below baseline measures during the study period.
DMD patients given the gene therapy also experienced disease progression at a 70-73% slower rate than their control group counterparts, as determined by analyses of TTR and 10MWR measurements, respectively.
In the three-year follow up, Elevidys’ safety profile remained consistent with previous studies, with no new treatment-emergent adverse events (TEAEs) reported.
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By GlobalDataInvestors seem to be pleased with this three-year outcome, as Sarepta’s stock value climbed nearly 4% from $21.13 at market close on 23 January to $21.96 at market open on 26 January after the EMBARK study results debuted. Sarepta plans to share further results from Elevidys trials in DMD at various upcoming medical meetings.
Positive impact of three-year EMBARK readout dubious
This is a positive step forward for Sarepta after a difficult past year. Elevidys was linked to the deaths of two non-ambulatory DMD patients, resulting in the US Food and Drug Administration (FDA) placing a commercial hold on Elevidys shipments; though this decision was later reversed following a review.
Now, Elevidys is only indicated for patients with ambulatory DMD, and a boxed warning around treatment-associated liver injury risks has been added to its label.
These challenges, alongside infusion reschedules and a rough flu season, caused Elevidys to miss its Q4 2025 sales expectations, with revenue totalling $110.4m. This is notably lower than estimates from investment bank William Blair, which forecasted the gene therapy would make $128.8m in the quarter.
While William Blair analyst, Sami Corwin hails the three-year EMBARK data as positive, she does not believe the readout will impact the prescribing and uptake of Elevidys in the near-term. However, Corwin noted that the readout “reinforces the rationale that Elevidys can alter long-term disease trajectory in ambulatory patients,” which could help to quell investor scepticism on the gene therapy’s efficacy following its prior one-year primary endpoint miss.
Speaking at the 2026 J.P. Morgan Healthcare Conference in San Francisco, Sarepta’s CEO, Douglas Ingram, placed importance on the new cohort in the Phase Ib ENDEAVOR trial (NCT04626674), which will evaluate the potential of Elevidys alongside an immunosuppressant. Through this study, Sarepta hopes to regain the gene therapy’s approval for use in the non-ambulatory DMD patient population. Ingram said the company anticipates data at the back end of 2026.
