REGENXBIO's RGX-121 MPS II Trial Meets Primary Endpoint

On February 7, 2024, REGENXBIO Inc. (Nasdaq: RGNX) revealed that the Phase I/II/III CAMPSIITE® clinical trial for RGX-121, a treatment for Mucopolysaccharidosis Type II (MPS II), also known as Hunter syndrome, has successfully met its primary endpoint with high statistical significance (p value of 0.00016). RGX-121 is a one-time gene therapy that targets the underlying genetic cause of the disease, potentially improving brain function and allowing patients to forego or discontinue enzyme replacement therapy.
The pivotal data indicate that RGX-121-treated patients showed a significant reduction in cerebrospinal fluid (CSF) levels of D2S6, a biomarker for brain disease activity, with an 86% median decrease observed. This reduction is nearly to normal levels and is consistent with the dose-finding phase of the trial, where patients demonstrated neurodevelopmental progress over four years. Notably, 80% of patients at the pivotal dose level were free from enzyme replacement therapy at the last reported time point.
The company is preparing to submit a Biologics License Application (BLA) in 2024 under an accelerated approval pathway, following a positive meeting with the FDA. REGENXBIO's manufacturing process, NAVXpress™, is used to produce commercial-scale cGMP material for the trial. The trial will continue to gather neurodevelopmental data and outcomes reported by caregivers.
RGX-121 has received multiple designations from regulatory bodies, including Orphan Drug Product, Rare Pediatric Disease, Fast Track, and Regenerative Medicine Advanced Therapy from the U.S. FDA, and ATMP classification from the European Medicines Agency. MPS II is a rare genetic disorder characterized by the accumulation of glycosaminoglycans (GAGs) due to a deficiency in the I2S enzyme, leading to progressive damage to cells, tissues, and organs, particularly in the CNS.
REGENXBIO is a clinical-stage biotechnology company focused on gene therapy with a proprietary AAV gene delivery platform. The company aims to advance five AAV therapeutics into pivotal-stage or commercial products by 2025 as part of its "5x'25" strategy.

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