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Additional Positive Interim Data from Phase I/II/III CAMPSIITE™ Trial of REGENXBIO's RGX-121 for the Treatment of MPS II (Hunter Syndrome) Presented at 19th Annual WORLDSymposiumTM
RGX-121, a potential one-time gene therapy for the treatment of MPS II, continues to be well-tolerated with no drug-related SAEs across three dose
About this update from Regenxbio Inc.
[{"type":"text","content":"RGX-121, a potential one-time gene therapy for the treatment of MPS II, continues to be well-tolerated with no drug-related SAEs across three dose levelsAdditional data from patients in Cohort 3 using pivotal program dose level continue to demonstrate largest reductions in CSF GAGs, continuing to approach normal levels at 48 weeksNew, longer-term clinical measures demonstrated continued improvement in neurodevelopmental and daily activity skill acquisition up to three years after RGX-121 administrationPositive interim data continues to support plan to file Biologics License Application in 2024 using the accelerated approval pathway ROCKVILLE, Md., Feb. 22, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced additional positive interim data 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. The results were presented at the 19th Annual WORLDSymposium™.\n\"These new results demonstrate sustained reductions in CSF GAGs and an encouraging, long-term clinical profile of RGX-121 up to three years,\" said Steve Pakola, M.D., Chief Medical Officer of REGENXBIO. \"GAGs measured in CSF, specifically heparan sulfate, reflect disease manifestations in the CNS and are a direct cause of disease pathophysiology. Our data provide additional evidence to support the finding that meaningful changes in CSF heparan sulfate is an appropriate and reliable surrogate endpoint reasonably likely to predict the clinical benefit of CNS-targeted therapies for MPS II. We are excited to have taken RGX-121 into a pivotal program and plan to file a Biologics License Application in 2024 using the accelerated approval pathway.\"\n\"Treatment options to address the neurological manifestations of MPS II remain a significant unmet medical need for patients,\" said Can Ficicioglu, M.D., Ph.D., Professor of Pediatrics at the Perelman School of Medicine, University of Pennsylvania. \"The data presented today are encouraging and continue to show the potential of a one- time gene therapy to provide meaningful, durable clinical benefits to the MPS II community. I look forward to continuing to follow RGX-121 as it progresses through the pivotal program.\"\nRGX-121 is an investigational, one-time gene therapy designed to delive...