Regenxbio 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, or MPS II, also known as Hunter Syndrome. The results were presented at the 19th Annual WORLDSymposium. "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."
Published first on TheFly
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