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REGENXBIO Presents Additional Positive Interim Data from Phase I/II Trial of RGX-121 for the Treatment of MPS II (Hunter Syndrome) at 18th Annual WORLDSymposium™ 2022
ROCKVILLE, Md., Feb. 9, 2022 /PRNewswire/ -- RGX-121, a one-time gene therapy for MPS II, continues to be well-tolerated with no drug-related SAEs across
About this update from Regenxbio Inc.
[{"type":"text","content":"ROCKVILLE, Md., Feb. 9, 2022 /PRNewswire/ -- \nRGX-121, a one-time gene therapy for MPS II, continues to be well-tolerated with no drug-related SAEs across three dose levels Dose-dependent reductions in CSF biomarkers observed; Cohort 3 patients approach normal levels of D2S6 biomarker Measures of neurodevelopmental function from patients in Cohorts 1 and 2 demonstrate continued developmental skill acquisition up to two years after RGX-121 administration Evidence of systemic enzyme expression and biomarker activity continues to be observed Cohort 3 expansion using commercial-scale cGMP material is planned to start in 1H 2022 REGENXBIO Inc. (Nasdaq: RGNX) today announced additional positive interim data from Cohorts 1-3 of the ongoing Phase I/II 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, are being presented at the 18th Annual WORLDSymposium™. \n\"We are pleased to share additional data from patients in Cohorts 1-3 of our RGX-121 Phase I/II trial,\" said Steve Pakola, M.D., Chief Medical Officer of REGENXBIO. \"The interim data demonstrate dose-dependent reductions in CSF biomarkers across cohorts, and we are encouraged by the neurodevelopmental trajectories in patients from Cohorts 1 and 2, now up to two years after administration of RGX-121. We look forward to providing additional updates in this trial.\"\n\"MPS II patients are in need of new treatment options that could address the neurological manifestations of the disease and help address the neurodevelopmental decline observed with currently available therapies,\" said Roberto Giugliani, M.D., Ph.D., Professor, Department of Genetics, UFRGS, Medical Genetics Service, HCPA, Porto Alegre, Brazil. \"The data presented today continues to support the early positive signals seen in the emerging clinical profile of RGX-121 for the treatment of MPS II. I am encouraged by the biomarker activity and impact on neurodevelopmental improvements observed in this trial, and I look forward to additional updates.\" \nRGX-121 is an investigational one-time gene therapy designed to deliver the gene that encodes the iduronate-2-sulfatase (I2S) enzyme using the AAV9 vector. The primary endpoint of the trial is to evaluate the safety of RGX-121. Secondary and exploratory endpoints include bi...