DNLI - Denali Therapeutics reports positive DNL310 data in mid-stage hunter syndrome study

Denali Therapeutics (DNLI) announces additional positive interim data from Phase 1/2 study evaluating ETV:IDS (DNL310) for the treatment of both central nervous system ((CNS)) and peripheral manifestations of Hunter syndrome (MPS II).All patients received weekly intravenous doses of DNL310 after switching from idursulfase enzyme replacement therapy on Day 1 of the study. Results across Cohorts A and B showed that, following the switch from idursulfase to DNL310, the levels of heparan sulfate in cerebrospinal fluid ((CSF)) normalized in all patients analyzed (n=15), with rapid response observed in most patients (n=12) by Week 7, which is consistent with crossing of the BBB by DNL310 and activity in tissues of the CNS.Rapid normalization of CSF heparan sulfate at low dose regimens suggest that BBB crossing with Denali’s Transport Vehicle was robust and efficient.Furthermore, the observed decline in urine and serum heparan sulfate was consistent with improved peripheral activity with DNL310.Global Impression of

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Denali Therapeutics reports positive DNL310 data in mid-stage hunter syndrome study