REGENXBIO Completes Cohort 2 Enrollment and Reports Positive Interim AFFINITY DUCHENNE® Trial Data

REGENXBIO has successfully completed patient recruitment for the second group in the Phase I/II AFFINITY DUCHENNE® clinical trial, which is examining the safety and effectiveness of RGX-202. RGX-202 is a promising one-time AAV gene therapy designed to treat Duchenne muscular dystrophy by introducing an optimized microdystrophin gene. The latest three-month evaluation of a patient from the first dose group has shown a significant increase in microdystrophin levels, with a 6.6-year-old patient achieving 83.4% of the control expression level. The company is on schedule to start a pivotal trial in the latter half of 2024 and will discuss these interim findings during a conference call on February 7th.
The drug RGX-202 has shown a good safety profile, with no serious drug-related adverse events reported in five patients aged between 4.4 and 12.1 years across the first two dose levels. These patients have been monitored for a period ranging from three weeks to over nine months post-treatment. Positive biomarker data from the third patient at the first dose level indicates a substantial reduction in serum creatinine kinase levels, a marker for muscle injury, which is typically elevated in Duchenne patients. The measured expression levels of RGX-202 microdystrophin at three months post-treatment were on average 44.4%, with a standard deviation of 36.5%.
REGENXBIO anticipates determining the pivotal dose in mid-2024 and sharing preliminary strength and functionality data for both dose levels. The company intends to utilize the RGX-202 microdystrophin expression as a proxy for a Biologics License Application (BLA) submission under an accelerated approval pathway. The AFFINITY DUCHENNE trial is a multicenter, open-label study aimed at assessing the safety and clinical efficacy of a single intravenous dose of RGX-202 in pediatric patients with Duchenne, aged 4 to 11 years. The study design incorporates a short-term immunosuppression regimen and specific inclusion criteria based on dystrophin gene mutation status.
RGX-202 incorporates a novel microdystrophin gene with a C-Terminal domain that aids in recruiting essential proteins to the muscle cell membrane, potentially enhancing muscle resistance to damage. The therapy also features enhancements to improve gene expression and reduce the risk of immune responses. Duchenne muscular dystrophy is a severe and progressive disease that affects approximately 1 in 3,500 to 5,000 male births annually, caused by the absence of the dystrophin protein, which is crucial for muscle cell integrity and function.
REGENXBIO is a clinical-stage biotech firm focused on gene therapy with a proprietary AAV gene delivery platform, NAV Technology, which includes over 100 novel AAV vectors. The company is working towards advancing five AAV therapeutics to pivotal or commercial stages by 2025 as part of its "5x'25" strategy. 

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