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REGENXBIO Announces Pivotal Trial of RGX-121 for the Treatment of MPS II Achieves Primary Endpoint
Results support BLA submission in 2024 using the accelerated approval pathwayPrimary endpoint of patients achieving reduction in CSF biomarker of MPS II
About this update from Regenxbio Inc.
[{"type":"text","content":"Results support BLA submission in 2024 using the accelerated approval pathwayPrimary endpoint of patients achieving reduction in CSF biomarker of MPS II disease was met with statistical significance (p value of 0.00016) Patients treated with RGX-121 have showed continued improvement in neurodevelopmental skill acquisition up to four years and discontinued intravenous enzyme therapyCompany plans to discuss these results as part of a full rare disease program update on its conference call today, Wednesday, February 7, 4:30 p.m. ETROCKVILLE, Md., Feb. 7, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced topline results from the Phase I/II/III CAMPSIITE® trial of RGX-121 for the treatment of patients up to 5 years old diagnosed with Mucopolysaccharidosis Type II (MPS II), also known as Hunter syndrome, demonstrating that the pivotal phase of the trial met its primary endpoint with statistical significance.\nThe results were presented at the 20th Annual WORLDSymposium™ by Paul Harmatz, M.D., UCSF Benioff Children's Hospital and trial investigator.\n\"The data from this pivotal trial supports that RGX-121 changes the course of disease by restoring the gene missing in boys with Hunter syndrome and has the potential to significantly improve vital brain function for patients living with this debilitating disease,\" said Kenneth T. Mills, President and CEO of REGENXBIO. \"We are excited about these results and working quickly to complete activities to file the BLA this year. We have shared CAMPSIITE results with FDA leadership, and they have confirmed that, based on the totality of the evidence, they are open to accelerated approval if supported by review of the full data.\"\n\"There is currently no treatment to address fatal neuronopathic CNS disease in MPS II, and I am encouraged by the topline data from the pivotal trial of RGX-121,\" said Dr. Harmatz. \"A one-time gene therapy that can help these boys develop beyond the natural history of the disease and may allow them to discontinue enzyme replacement therapy or remain ERT-naïve represents a meaningful breakthrough.\"\nData SummaryIn the pivotal phase, MPS II patients treated with RGX-121 achieved decreased cerebrospinal fluid (CSF) levels of D2S6, a key biomarker of brain disease activity, below maximum attenuated disease levels at 16 weeks (p value of 0.00016). Pat...