Sarepta Therapeutics has completed enrolment and dosing in the global Phase III EMERGENE trial of its investigational gene therapy, SRP-9003 (bidridistrogene xeboparvovec), aimed at treating limb-girdle muscular dystrophy Type 2E/R4 (LGMD2E/R4).
The study’s primary endpoint is the biomarker expression of the beta-sarcoglycan protein, which is crucial for the progression of LGMD2E/R4, also known as beta-sarcoglycanopathy.
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Data from the trial are expected in the first half of next year.
The multinational, open-label EMERGENE trial involves ambulatory and non-ambulatory subjects aged four and above. Secondary endpoints and outcomes will assess functional measures up to month 60 and evaluate safety parameters.
The gene therapy is designed to deliver a full-length beta-sarcoglycan transgene to skeletal, diaphragm, and cardiac muscle using the AAVrh74 vector.
Subject to a positive pre-Biologics License Application (BLA) meeting and encouraging data from the study, the company plans to submit a BLA for accelerated approval to the US Food and Drug Administration (FDA).
Sarepta Therapeutics research and development head, executive vice president, and chief scientific officer Louise Rodino-Klapac said: “The completion of enrolment in the EMERGENE study marks a significant milestone to bring a potentially disease-modifying treatment to individuals living with LGMD2E, an ultra-rare form of LGMD with no treatments beyond symptom management.
“The design of EMERGENE is an important precedent that informs development plans for Sarepta’s other LGMD pipeline programmes, including our LGMD2D programme, which we just initiated and our LGMD2C programme, which we expect to initiate in the first quarter of 2025, and serves as a pathfinder for heterogeneous neuromuscular gene therapies more broadly.”
In February last year, the company dosed the first subject in the Phase I VOYAGENE trial of SRP-9003 for LGMD2E.
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