(RTTNews) - Regenxbio Inc. (RGNX) Wednesday reported additional positive interim data from the Phase I/II/III CAMPSIITE study of its drug candidate RGX-121 for the treatment of patients up to 5 years old diagnosed with Mucopolysaccharidosis Type II (MPS II), also known as Hunter Syndrome.
Hunter syndrome is a genetic disorder, caused by a missing or malfunctioning enzyme, leading to many physical and mental developmental issues.
Data from the Phase I/II portion of the CAMPSIITE trial, where the primary endpoint is to evaluate the safety of RGX-121, were presented at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium.
RGX-121, a one-time gene therapy for MPS II, continued to be well-tolerated with no drug-related SAEs across three dose levels, Regenxbio said.
Secondary and exploratory endpoints include cerebral spinal fluid (CSF) glycosaminoglycans (GAGs), neurodevelopmental assessments, caregiver reported outcomes and systemic biomarkers.
New data showed largest median reduction in CSF GAGs in Cohort 3 patients approaching normal levels at 48 weeks.
Positive interim data supports recently announced plan to file Biologics License Application in 2024 using the accelerated approval pathway, the company added.
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