REGENXBIO Presents Positive Twelve-Month Pivotal Data from Phase I/II/III CAMPSIITE® Trial of RGX-121 for Treatment of MPS II
1. RGX-121 shows over 80% CSF HS D2S6 reduction after one year. 2. Positive neurodevelopmental outcomes correlate with biomarker reductions in pivotal trial. 3. RGX-121 could become the first approved treatment for Hunter syndrome. 4. FDA decision on RGX-121 expected by February 8, 2026. 5. RGN-121 has favorable safety profile observed in all trial phases.