Preliminary results from a Phase 1/2 clinical trial evaluating REGENXBIO’s (NASDAQ:RGNX) gene therapy RGX-121 in patients with mucopolysaccharidosis type II (MPS II), also known as Hunter’s syndrome, showed a treatment effect.
Three patients in Cohort 1 received an intercisternal dose (administered directly into a part of the brain) of MGX-121. Levels of heparan sulfate, high levels of which are associated with neurocognitive decline, dropped an average of 33% from baseline to week 8.
Two patients who progressed beyond week 24 showed stable neurocognitive development, one with normal development at week 48 and the other, who was diagnosed with neurocognitive decline prior to dosing, was stable (based on preliminary assessments).
The estimated completion date is December 2022.
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