REGENXBIO Announces Data From Phase I/II/III CAMPSIITE Trial Of Clemidsogene Lanparvovec For Treatment Of Patients With Mucopolysaccharidosis Type II At ICIEM 2025

Author: Benzinga Newsdesk | September 05, 2025 06:08am

• 12-month pivotal data further demonstrate the ability of one-time RGX-121 treatment to improve outcomes for patients with MPS II
  • >80% reduction in CSF levels of HS D2S6, key biomarker of MPS II brain disease, sustained through 1 year
  • Pivotal patients demonstrate continued skill acquisition or stability, stratified by baseline function, through 1 year
• Primary endpoint of CSF HS D2S6 reduction at week 16 met; strong correlation to neurodevelopmental outcomes at 1 year, supporting HS D2S6 as surrogate biomarker reasonably likely to predict clinical benefit
• RGX-121 would be the first and only potential one-time, commercially-available therapy designed to directly address the underlying genetic cause of Hunter syndrome, if approved

ROCKVILLE, Md., Sept. 5, 2025 /PRNewswire/ -- REGENXBIO Inc. (NASDAQ:RGNX) today announced new, positive data from the Phase I/II/III CAMPSIITE® trial of clemidsogene lanparvovec (RGX-121) for the treatment of patients with Mucopolysaccharidosis Type II (MPS II), also known as Hunter syndrome, at the International Congress of Inborn Errors of Metabolism (ICIEM) 2025. REGENXBIO submitted these longer-term pivotal results to the U.S. Food and Drug Administration (FDA) in response to an information request in the ongoing Biologics License Application (BLA) review of RGX-121.

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