Regenxbio's Hunter Syndrome Gene Therapy Shows Efficacy Signals, Cognitive Development Up To Two Years - Regenxbio (NASDAQ:RGNX)

Regenxbio Inc RGNX has announced a safety update and additional positive interim data from its ongoing Phase 1/2 trial of RGX-121 to treat patients up to 5 years old diagnosed with Mucopolysaccharidosis Type II (MPS II), also known as Hunter Syndrome.
The data will be presented at the American Society of Gene and Cell Therapy 24th Annual Meeting.
Heparan sulfate (HS) levels are a key biomarker of I2S enzyme activity. The patients in Cohorts 1 and 2 demonstrated decreased HS in the cerebrospinal fluid (CSF) for up to 2 years following one-time administration of RGX-121.
Combined median reductions from baseline were 30.3% at Week 8 and 35.0% at the last time point available for each patient.
These patients also demonstrated decreased levels of D2S6, a component of HS, up to 2 years, with median reductions of 44.1% at Week 8 and 40.4% at the last timepoint available for each patient.
Continuous neurocognitive development was observed up to two years after RGX-121 administration.
Five patients have assessments of neurodevelopment function at time points beyond six months, and of those patients, four have continued to demonstrate neurocognitive development.
Two patients in Cohort 1 discontinued enzyme replacement therapy (ERT) over one year after administration of RGX-121.
RGX-121 is an investigational one-time gene therapy designed to deliver the gene that encodes the iduronate-2-sulfatase (I2S) enzyme using the AAV9 vector. It is administered directly to the central nervous system.
Price Action: RGNX shares are up 3.19% at 36.42 during the market session on the last check Friday.

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