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Denali Therapeutics Announces Data Presentations on Enzyme TransportVehicle™ Programs for Hunter Syndrome, Sanfilippo Syndrome Type A and Pompe Disease at Upcoming 2026 WORLDSymposium™
Hunter syndrome (MPS II) presentations will include analysis from continued follow-up of Phase 1/2 data for tividenofusp alfa (DNL310), currently under FDA
About this update from Denali Therapeutics Inc.
[{"type":"text","content":"Hunter syndrome (MPS II) presentations will include analysis from continued follow-up of Phase 1/2 data for tividenofusp alfa (DNL310), currently under FDA Priority ReviewPreliminary data from Phase 1/2 study of DNL126 (ETV:SGSH) for Sanfilippo syndrome type A (MPS IIIA) to be featured in oral presentationPhase 1 study design of DNL952 (ETV:GAA) for Pompe disease and supporting preclinical data will appear in poster session SOUTH SAN FRANCISCO, Calif., Jan. 29, 2026 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (Nasdaq: DNLI) today announced the presentation of clinical and preclinical data from its Enzyme TransportVehicle™ (ETV) programs at the upcoming 22nd Annual WORLDSymposium™ to be held February 2-6, 2026, in San Diego, California. These presentations will demonstrate the broad potential of the ETV to enable the delivery of enzyme replacement therapies to the whole body, including the brain. Two oral presentations will discuss continued follow-up data from the Phase 1/2 clinical study of tividenofusp alfa (DNL310) for Hunter syndrome (mucopolysaccharidosis type II, or MPS II) and preliminary clinical data from the ongoing Phase 1/2 study of DNL126 (ETV:SGSH) for Sanfilippo syndrome type A (MPS IIIA). In addition, three posters will highlight a case study with a non-neuronopathic sibling pair from the Phase 1/2 study of tividenofusp alfa; a community survey conducted in partnership with patient advocacy organization Project Alive examining unmet needs among individuals living with MPS II and their caregivers; and a health outcomes analysis evaluating the clinical and economic burden among individuals treated for MPS II. The U.S. Food and Drug Administration (FDA) is conducting a Priority Review of the Biologics License Application (BLA) for tividenofusp alfa, with a decision expected by April 5, 2026. Denali will also detail the design of the Phase 1 study of DNL952 (ETV:GAA) for Pompe disease and supporting preclinical data in two posters. Details on the WORLDSymposium presentations are below: Platform Presentations Title: Phase I/II Study of Intravenous Tividenofusp Alfa for Mucopolysaccharidosis Type II Presentation #258Date: Thursday, February 5, 2026Session Time: 11:00 AM-noon PST Title: Preliminary Results From Phase I/II, First-in-Human, Open-Label Study of DNL126 in Children With Mucopolysaccharidosis Type IIIA (MPS ...