Sarepta Therapeutics has announced new outcomes from its open-label Phase Ib Study 9001-103, also referred to as the ENDEAVOR trial, of adeno-associated virus (AAV)-based gene transfer therapy Elevidys (delandistrogene moxeparvovec-rokl) to treat Duchenne muscular dystrophy (DMD).
The multi-cohort ENDEAVOR study is aimed at evaluating the therapy’s safety and protein expression in male subjects with DMD.
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Across seven cohorts, the trial enrolled 55 subjects and dosed participants who were aged between four and seven years old at the time of treatment, with varying degrees of mobility, and those younger than four years old.
The primary endpoint of the trial is the change from baseline in Elevidys micro-dystrophin protein expression quantity at 12 weeks, as per western blot measurement, with secondary outcomes measuring dystrophin-positive fibres.
In addition, the trial’s exploratory endpoints evaluate the change in vector genome copies per nucleus, the North Star Ambulatory Assessment (NSAA), and timed functional tests.
Inclusive of the first 12-week duration, subjects will undergo a follow-up period of a total of five years.
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By GlobalDataIn cohort 6, subjects aged two years old at the time of treatment showed a mean expression of 93.87% of normal dystrophin levels when treated with the therapy, with 79.9% dystrophin-positive fibres. These outcomes were observed in biopsies 12 weeks post-treatment.
The safety seen in this cohort was found to be in line with the clinical and real-world applications of the therapy.
Sarepta noted that the company has shared safety and expression from the trial’s cohort 4, in which subjects were aged three at the time of treatment and showed a mean protein level of 99.64% in biopsies taken at the same post-treatment interval.
More than 25 subjects below four years of age were treated in Sarepta’s clinical trials.
Sarepta Therapeutics research and development head and chief scientific officer Louise Rodino-Klapac said: “The strength of the biomarker results that we are seeing in younger patients is extremely encouraging, and we have a meeting with the US Food and Drug Administration next month to discuss expanding the Elevidys label to include younger patients.”
Last December, Sarepta completed enrolment and dosing for the Phase III EMERGENE trial of SRP-9003 (bidridistrogene xeboparvovec), targeting limb-girdle muscular dystrophy Type 2E/R4.
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