Benzinga | Jul 26, 2021 06:59AM EDT

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

* 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.