ROCKVILLE, Md. - REGENXBIO Inc. (NASDAQ: RGNX) today announced positive topline results from its pivotal CAMPSIITE® trial of RGX-121 for Mucopolysaccharidosis Type II (MPS II), also known as Hunter syndrome. The trial met its primary endpoint with statistical significance, indicating a reduction in a crucial cerebrospinal fluid (CSF) biomarker for the disease.
The Phase I/II/III trial demonstrated that patients up to 5 years old showed a median 86% reduction in CSF levels of D2S6, a biomarker of brain disease activity, after treatment with RGX-121. These results suggest potential improvement in vital brain function for those living with this debilitating condition. Furthermore, patients treated with RGX-121 continued to exhibit neurodevelopmental skill acquisition for up to four years and were able to discontinue standard intravenous enzyme replacement therapy (ERT).
REGENXBIO plans to use these findings to file a Biologics License Application (BLA) in the second half of 2024, aiming for accelerated approval. The company has already engaged with the FDA, which has shown openness to this pathway based on the evidence presented.
As of January 3, 2024, the gene therapy has been well tolerated across 25 patients in all trial phases. The pivotal program utilized REGENXBIO's proprietary NAVXpress™ manufacturing process. The trial also collected neurodevelopmental data and caregiver-reported outcomes.
RGX-121, an investigational one-time gene therapy, is designed to deliver the gene for the iduronate-2-sulfatase enzyme directly to the central nervous system. The treatment has received several designations from the U.S. Food and Drug Administration, including Orphan Drug Product and Fast Track, indicating its potential to address an unmet medical need.
MPS II is a rare genetic disorder that leads to the buildup of glycosaminoglycans (GAGs), causing severe cell and organ dysfunction, particularly affecting the central nervous system. There is no current treatment for the fatal neuronopathic CNS manifestation of the disease.
This article is based on a press release statement from REGENXBIO Inc.
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