Regenxbio Inc., recently disclosed topline results from its Phase I/II/III CAMPSIITE® trial, assessing the efficacy of RGX-121 for treating children up to 5 years old diagnosed with Mucopolysaccharidosis Type II (MPS II), also known as Hunter syndrome. The pivotal phase of the trial successfully met its primary endpoint with statistical significance, showcasing the potential of RGX-121 to alter the course of this genetic disorder.

The trial results, presented at the 20th Annual WORLDSymposium™ by Dr. Paul Harmatz from UCSF Benioff Children’s Hospital, indicate that RGX-121 could significantly improve vital brain functions in patients with MPS II. Kenneth T. Mills, President and CEO of REGENXBIO, expressed optimism regarding the trial outcomes and the company’s expedited efforts to file a Biologics License Application (BLA) within the year, following constructive discussions with FDA leadership about the possibility of accelerated approval based on the trial’s comprehensive data.

A key highlight from the trial is the observed reduction in cerebrospinal fluid (CSF) levels of D2S6, a biomarker indicative of brain disease activity in MPS II patients. The pivotal phase reported an 86% median reduction in D2S6 levels, approaching normal ranges. These findings are consistent with earlier phases of the CAMPSIITE trial, where patients demonstrated significant neurodevelopmental improvements, surpassing natural history data expectations.

Furthermore, the trial presented a potential paradigm shift in MPS II treatment strategies, suggesting that patients receiving RGX-121 could either discontinue or avoid initiation of enzyme replacement therapy (ERT), which is currently a standard care practice. This development represents a significant step forward in managing MPS II, offering a one-time gene therapy solution that may reduce the treatment burden on patients and their families.

Regenxbio’s results from the RGX-121 trial contribute meaningful advancements in the treatment landscape for MPS II, showcasing the gene therapy’s potential to alter existing treatment protocols for this genetic disorder. The trial’s success in reducing critical biomarkers and the prospective reduction or elimination of the need for continuous enzyme replacement therapy position RGX-121 as a promising therapeutic option. Regenxbio is moving forward with regulatory processes, preparing to submit a Biologics License Application to the FDA, aiming to introduce this new treatment to patients with Hunter syndrome.

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