Denali Therapeutics Announces Positive Interim Data from Phase 1/2 Study with ETV:IDS (DNL310) in Patients with the Lysosomal Storage Disease Hunter Syndrome (MPS II)
?| Source:?Denali Therapeutics Inc.
- Rapid reduction and sustained normalization of heparan sulfate in CSF demonstrated robust and durable CNS activity with intravenous administration, and enhanced peripheral activity with reductions in urine and serum heparan sulfate after switching from standard-of-care idursulfase
- Global Impression of Change scales data suggested clinical improvement in overall MPS II symptoms, cognitive abilities, behavior, and physical abilities
- Exploratory biomarker data demonstrated reductions in CSF lysosomal lipid biomarkers that are consistent with improved lysosomal function; and high variability in exploratory biomarker Nf-L was observed pre- and post-treatment
- Safety profile with up to 43 weeks of dosing was consistent with standard of care enzyme replacement therapy with infusion-related reactions being the most frequently observed adverse events
- Based on these data, Denali is accelerating efforts to initiate a pivotal Phase 2/3 study in 1H 2022
- Management will host a webinar for analysts and investors at 11:30 a.m. Eastern Time today