Shots:

• Denali reports data from the P-I/II study evaluating DNL310 (qw- IV) for both CNS and peripheral MPS II. All patients received doses of DNL310 after switching from idursulfase enzyme replacement therapy on Day 1
• Results of Cohorts A & B showed the levels of heparan sulfate in CSF normalized in all patients analyzed with rapid response observed in patients @7wks.- and is consistent with crossing of the BBB by DNL310 and activity in tissues of the CNS
• Exploratory biomarker data demonstrated reductions in CSF lysosomal lipid biomarkers & high variability in exploratory biomarker Nf-L was observed pre- & post-treatment. Safety profile with up to 43wks. of dosing was consistent with SOC

­ Ref: Denali | Image: Trading View