Denali's Brain-Penetrant Enzyme Therapy Shows Durable Effect With CNS Impact In Hunter Syndrome - Denali

Denali Therapeutics Inc (NASDAQ:DNLI) announced additional interim data from a Phase 1/2 study evaluating ETV:IDS (DNL310) for the central nervous system (CNS) and peripheral manifestations of Hunter syndrome (MPS II).
DNL30 is an investigational brain-penetrant enzyme replacement therapy.
The results will be presented today at MPS 2021, the 16th International Symposium on MPS and Related Diseases.
This interim analysis included data on five patients enrolled in Cohort A and 12 patients enrolled in Cohort B. Results across Cohorts A, and B showed that, following the switch from idursulfase to DNL310, heparan sulfate levels in cerebrospinal fluid (CSF) normalized in all patients analyzed (n=15).
Rapid response was observed in most patients (n=12) by Week 7, which is consistent with the crossing of the blood-brain barrier (BBB) and activity in tissues of the CNS.
Furthermore, the observed decline in urine and serum heparan sulfate was consistent with improved peripheral activity with DNL310.
Exploratory clinical data suggest improved clinical symptoms and function for all five patients enrolled in Cohort A.
Data showed clinical improvement in overall MPS II symptoms, cognitive abilities, behavior, and physical abilities.
The safety profile of DNL310 remained consistent with the standard of care enzyme replacement therapy.
DNL310 was generally well tolerated, with the most common treatment-emergent adverse events being infusion-related reactions.
Infusion reactions occurred in 12 of 17 (71%) patients, and the majority had mild or moderate reactions.
Denali is accelerating efforts to initiate a pivotal Phase 2/3 study in 1H 2022.
Price Action: DNLI shares are trading lower by 4.55% at $60.99 premarket on the last check Monday.

DNLIDenali Therapeutics Inc

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