Regenxbio announces positive data from trial for MPS II treatment
Betsy Goodfellow | February 8, 2024 | News story | Research and Development | Hunter syndrome, MPS II, Neurology, Regenxbio, clinical trial
Regenxbio has announced topline results from the phase 1/2/3 CAMPSIITE trial of RGX-121 for the treatment of patients up to five years of age who have been diagnosed with Mucopolysaccharidosis Type II (MPS II), also known as Hunter syndrome.
The trial met its primary endpoint of achieving reduction in CSF biomarker of MPS II disease with statistical significance, with patients treated with RGX-121 showing continued improvement in neurodevelopmental skill acquisition up to four years and discontinued intravenous enzyme therapy.
Kenneth T Mills, president and CEO of Regenxbio, commented: “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. We are excited about these results and working quickly to complete activities to file the BLA this year. We have shared CAMPSIITE results with US Food and Drug Administration (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.”
Paul Harmatz MD, UCSF Benioff Children’s Hospital, US, and trial investigator, added: “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. 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.”
Betsy Goodfellow
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