Denali Therapeutics has announced new interim data from its Phase 1/2 study of DNL310 in MPS II at SSIEM 2023. The data shows rapid and sustained normalization of CSF heparan sulfate to normal healthy levels and improvement in lysosomal function biomarkers. The treatment is generally well tolerated and has shown positive clinical outcome changes in adaptive behavior, cognition, and auditory function. Recruitment for the global Phase 2/3 COMPASS study is ongoing.