Denali Therapeutics Inc. announced new clinical data and updates on its Enzyme Transport Vehicle $(ETV)$ platform at the 22nd Annual WORLDSymposium™. The company presented continued follow-up results from a Phase 1/2 clinical trial of tividenofusp alfa (DNL310) in Hunter syndrome (MPS II), reinforcing its potential to address the full disease spectrum. Denali has established launch readiness for tividenofusp alfa in anticipation of an April 5, 2026, Prescription Drug User Fee Act (PDUFA) date. Preliminary Phase 1/2 data for DNL126 (ETV:SGSH) in Sanfilippo syndrome type A (MPS IIIA) showed substantial reductions in disease biomarkers, including an 80% mean reduction in cerebrospinal fluid heparan sulfate, with a safety profile generally consistent with established enzyme replacement therapies. Planning for a global Phase 3 confirmatory study is ongoing, with a Biologics License Application (BLA) submission and potential approval expected in 2027. Additionally, the design of the ongoing DNL952 (ETV:GAA) Phase 1 clinical study for Pompe disease was presented, along with preclinical data demonstrating its therapeutic potential to address both muscle and nervous system manifestations.

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