Denali Therapeutics Reveals Enzyme TransportVehicle Progress Across Three Clinical Programs For Treatment of Lysosomal Storage Disorders; Analysis From Continued Follow-Up Of Phase 1/2 Clinical Trial Data In Hunter Syndrome Reinforces Potential For Tividenofusp Alfa To Address Full Disease Spectrum; Preliminary Phase 1/2 Study Data Show Treatment With DNL126 Reduced Disease Biomarkers In Cerebrospinal Fluid And Peripheral Tissue

• Analysis from continued follow-up of Phase 1/2 clinical trial data in Hunter syndrome (MPS II) reinforces potential for tividenofusp alfa (DNL310) to address full disease spectrum
• Launch readiness established in anticipation of April 5, 2026, Prescription Drug User Fee Act (PDUFA) date for tividenofusp alfa
• Preliminary Phase 1/2 study data show treatment with DNL126 (ETV:SGSH) substantially reduced disease biomarkers in cerebrospinal fluid (CSF) and peripheral tissues, including an 80% mean reduction in CSF heparan sulfate, in Sanfilippo syndrome type A (MPS IIIA); safety profile generally consistent with established enzyme replacement therapies
• Design of ongoing DNL952 (ETV:GAA) Phase 1 clinical study presented in addition to preclinical data that show therapeutic potential to treat both muscle and nervous system manifestations of Pompe disease
  
  
  
   

SOUTH SAN FRANCISCO, Calif., Feb. 05, 2026 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (NASDAQ:DNLI) today announced data from programs in Hunter syndrome (mucopolysaccharidosis type II, MPS II), Sanfilippo syndrome type A (MPS IIIA) and Pompe disease that highlight the potential of its Enzyme TransportVehicle™ (ETV) to enable the delivery of enzyme replacement therapies (ERT) to the whole body, including the brain. Data were presented this week at the 22nd Annual WORLDSymposium™ in San Diego, California.

"The data presented at this year's WORLDSymposium reflect the strong momentum of our Enzyme TransportVehicle franchise as we continue to prepare for the potential commercial launch of tividenofusp alfa for Hunter syndrome and make meaningful progress across lysosomal storage disorders," said Peter Chin, M.D., Acting Chief Medical Officer and Head of Development of Denali Therapeutics. "From advancing our program in Sanfilippo to expanding into muscle disease with Pompe, we are continuing to broaden the reach of our ETV platform with the goal of delivering new medicines in areas of high unmet need. This progress is made possible through constructive collaboration with regulatory authorities and close partnership with the community, listening to and learning from them to better understand treatment journeys of individuals and their families living with these debilitating diseases."