REGENXBIO Presents Interim Results of RGX-121 in P-I/II/III (CAMPSIITE) Trial for MPS II (Hunter Syndrome) at SSIEM 2022
Shots:
- The P-I/II/III (CAMPSIITE) trial evaluates RGX-121 (2.9x1011GC/g) in ~10 patients aged 4mos. to ~5yrs. with MPS II across the US, Brazil & Canada. The company plan to file BLA in 2024
- As of Aug 1, 2022, RGX-121 was well-tolerated across all cohorts with no drug-related SAEs, biomarker data from patients in all three cohorts showed dose-dependent reductions of CSF GAGs, reductions of HS with dose-dependent reductions @8, 24 & 48wk. post-RGX-121 administration, the median decrease in CSF HS from baseline was 33.5%/52.9%/62.5% in cohort 1/2/3 @48wk.
- Reduction in HS D2S6 with dose-dependent reductions & median reduction from baseline of 31.9%/69.4%/83.1% in cohort 1/2/3, positive impact on neurodevelopmental function, improved I2S protein concentration levels in plasma
Ref: PR Newswire | Image: REGENXBIO
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