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Denali Therapeutics Announces Key Anticipated 2024 Milestones and Priorities to Further Advance Its Therapeutics Portfolio for Neurodegeneration and Lysosomal Storage Diseases
Complete enrollment in Denali-led, late-stage programs for MPS II and ALS, and establish commercial readinessAdvance broad clinical-stage portfolio of seven
About this update from Denali Therapeutics Inc.
[{"type":"text","content":"Complete enrollment in Denali-led, late-stage programs for MPS II and ALS, and establish commercial readinessAdvance broad clinical-stage portfolio of seven therapeutic product candidates across neurodegeneration (ALS, Parkinson’s disease, MS, FTD-GRN), lysosomal storage disease (MPS II, MPS IIIA), and inflammatory disease (UC)Advance OTV:MAPT targeting tau for Alzheimer’s disease and OTV:SNCA targeting alpha-synuclein for Parkinson’s disease in IND-enabling studiesPrioritize preclinical portfolio with focus on Transport Vehicle-enabled biotherapeutics SOUTH SAN FRANCISCO, Calif., Jan. 08, 2024 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (Nasdaq: DNLI), a biopharmaceutical company developing a broad portfolio of product candidates engineered to cross the blood-brain barrier (BBB) for the treatment of neurodegenerative diseases and lysosomal storage diseases, today announced program progress and expected milestones for 2024, which Chief Executive Officer, Ryan Watts, Ph.D., will highlight during a corporate presentation at the 42nd Annual J.P. Morgan Healthcare Conference on Tuesday, January 9th, at 10:30 a.m. Pacific Time. “In 2024, we expect enrollment to be completed in our late-stage clinical trials for MPS II and ALS, and we are establishing commercial readiness,” said Ryan Watts, Ph.D., Chief Executive Officer of Denali. “In addition, we are excited to announce initiation of clinical development for our second Enzyme Transport Vehicle (TV)-enabled program, DNL126 in MPS IIIA, and that our first two Oligonucleotide TV-enabled programs are in IND-enabling studies targeting tau in Alzheimer’s disease (OTV:MAPT) and alpha-synuclein in Parkinson’s disease (OTV:SNCA). Based on clinical validation and momentum with the TV platform, we have prioritized advancing additional TV programs for common neurodegenerative diseases.” Denali’s 2024 Outlook Expected progress and key milestones in 2024 across Denali’s therapeutic portfolio are summarized below. LATE-STAGE AND MID-STAGE CLINICAL PROGRAMS Tividenofusp alfa (DNL310): MPS II (Hunter syndrome) Tividenofusp alfa (DNL310) is an investigational, intravenously administered, Enzyme Transport Vehicle (ETV)-enabled, iduronate-2-sulfatase (IDS) replacement therapy designed to cross the BBB and address the behavioral, cognitive, and physical manifestations of MPS II (Hunter syndrome). Inter...