DNLI - Denali Therapeutics posts positive data from early-stage DNL310 Hunter syndrome trial

Denali Therapeutics (DNLI) announces additional positive interim results from its ongoing Phase 1/2 study evaluating DNL310 as a potential brain-penetrant enzyme replacement therapy for treating both central nervous system ((CNS)) and peripheral manifestations of Hunter syndrome ((MPS II)), a rare neurodegenerative lysosomal storage disorder.Interim analysis included data on five patients enrolled in Cohort A of the study, who all received 3 months of weekly intravenous doses of DNL310 after switching from idursulfase enzyme replacement therapy.Normal levels of heparan sulfate, a glycosaminoglycan (GAG), in cerebrospinal fluid (CSF) that were seen after four weeks of dosing in four of five patients were sustained after three months of dosing (mean 85% reduction across Cohort A; p<0.001).Reductions in downstream exploratory CSF biomarkers, GM3 and BMP (lysosomal lipids), of 39% and 15%, respectively, were observed after eight weeks of dosing, consistent with improvement in lysosomal function.DNL310 was generally well tolerated with no dose reductions and all five patients continue in the study.The

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Denali Therapeutics posts positive data from early-stage DNL310 Hunter syndrome trial