DNLI - Denali Therapeutics Announces Presentations on Its Investigational Blood-Brain Barrier (BBB)-Crossing Enzyme Replacement Therapies at the Upcoming 2024 WORLDSymposium™ | Benzinga
SOUTH SAN FRANCISCO, Calif., Feb. 01, 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 upcoming presentations from its Enzyme Transport Vehicle (ETV) development programs, tividenofusp alfa (DNL310) and DNL126 (ETV:SGSH), to be given at the 20th Annual WORLDSymposium™, which will be held February 4-9, 2024, in San Diego, California.
WORLDSymposium™ Presentation Details on DNL310 and DNL126
Presentations on DNL310 will include new two-year data on clinical outcomes and previously presented data on biomarkers from the ongoing Phase 1/2 study in MPS II. In addition, an oral presentation will highlight data from preclinical studies of DNL126 in an MPS IIIA mouse model. The presentation schedule at WORLDSymposium™ is as follows:
Title: Interim Analysis of a Phase 1/2 Study of Weekly Intravenous DNL310 (Brain-Penetrant Enzyme Replacement Therapy) in Mucopolysaccharidosis Type II
Session: Clinical Applications Platform Presentations
Date: Wednesday, February 7, 2024
Time: 9:00 AM Pacific Time
Title: Somatic Outcomes in a Phase 1/2 Study of Weekly Intravenous DNL310 (Brain-Penetrant Enzyme Replacement Therapy) in Mucopolysaccharidosis Type II (Poster #43)
Session: Clinical Applications – Poster Session III
Date: Wednesday, February 7, 2024
Time: 3:00 PM - 5:00 PM Pacific Time
Title: DNL310 Phase 1/2 Case Study Demonstrates Properties of Raw, Standard and Growth Scale Scores for Adaptive Behavior Scales (Poster #80)
Session: Contemporary Forum – Poster Session IV
Date: Thursday, February 8, 2024
Time: 3:00 – 5:00 PM Pacific Time
Title: DNL310 Normalizes Primary Storage Substrates, Corrects Biomarkers of Lysosomal Dysfunction and Reduces Biomarkers of Neuronal Injury (Neurofilament Light Chain) in MPS II: 2-Year Interim Analysis of a Phase 1/2 Study (Poster #34)
Session: Contemporary Forum – Poster Session IV
Date: Thursday, February 8, 2024
Time: 3:00 – 5:00 PM Pacific Time
Title: ETV:SGSH, a Brain-Penetrant Enzyme Transport Vehicle for SGSH, Improves Lysosomal and Microglial Morphology, Degeneration and Cognitive Behavior in MPS IIIA Mice
Session: Contemporary Forum Platform Presentations
Date: Thursday, February 8, 2024
Time: 1:00 PM Pacific Time
Denali is also participating in or sponsoring the following events being held at the WORLDSymposium™:
Title: 3rd Annual Robert J. Gorlin Symposium – Beyond the Blood Brain barrier: Strategies for Treating the CNS
Course Director: Jeanine R. Jarnes, PharmD, MSc, BCOP, BCPS
Date: Tuesday, February 6, 2024
Time: 5:15 – 7:30 PM Pacific Time
Title: From Prevailing to Pioneering – Current and Emerging Biomarkers in Neurodegenerative Lysosomal Diseases
Date: Wednesday, February 7, 2024
Time: 5:15 – 6:15 PM Pacific Time
About MPS II (Hunter syndrome)
MPS II, also called Hunter syndrome, is a rare genetic disease that affects over 2,000 individuals, primarily males, world-wide, and leads to behavioral, cognitive, and physical symptoms ultimately resulting in shortened lifespan. MPS II is caused by mutations in the iduronate-2-sulfatase (IDS) gene, which leads to a deficiency of the IDS enzyme. Symptoms often begin emerging around age two and include physical complications, including organ dysfunction, joint stiffness, hearing loss and impaired growth, and ...