Denali(US:DNLI) Globenewswire·2025-12-30 13:00Core Insights - Denali Therapeutics Inc. announced the publication of results from the Phase 1/2 clinical trial of tividenofusp alfa for Hunter syndrome, with FDA Priority Review for its Biologics License Application expected by April 5, 2026 [1][5] Company Overview - Denali Therapeutics is focused on developing biotherapeutics that can cross the blood-brain barrier using its proprietary TransportVehicle™ platform [13] - The company is advancing a portfolio of therapeutic candidates aimed at treating neurodegenerative and lysosomal storage diseases [13] Clinical Trial Results - The Phase 1/2 trial involved 47 participants, showing that tividenofusp alfa significantly reduced central nervous system and peripheral biomarkers of substrate accumulation and neuronal injury [3][6] - Key findings included a 91% reduction in cerebrospinal fluid levels of heparan sulfate and an 88% reduction in urine levels at Week 24, with normalization of liver volume and improvements in hearing and cognitive skills [11][5] Treatment Mechanism - Tividenofusp alfa is designed to deliver the iduronate 2-sulfatase enzyme to the brain and body, addressing both neurological and physical symptoms of Hunter syndrome [4][7] - The treatment has received multiple designations from the FDA, including Rare Pediatric Disease Designation and Breakthrough Therapy Designation [7] Future Developments - Denali is conducting the Phase 2/3 COMPASS study to support global approval, comparing tividenofusp alfa with standard treatment idursulfase [8] - The company is also exploring the potential of its TransportVehicle platform for delivering other therapeutic agents, including antibodies and oligonucleotides [12]