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News  ›  GlobeNewswire Inc.

The New England Journal of Medicine Publishes Phase 1/2 Study of Denali Therapeutics’ Tividenofusp Alfa (DNL310) for Hunter Syndrome (MPS II)

GlobeNewswire Inc. Logo GlobeNewswire Inc. • •By Denali Therapeutics Inc.
The New England Journal of Medicine Publishes Phase 1/2 Study of Denali Therapeutics’ Tividenofusp Alfa (DNL310) for Hunter Syndrome (MPS II)

Denali Therapeutics announced publication of Phase 1/2 trial results for tividenofusp alfa (DNL310), an investigational enzyme replacement therapy for Hunter syndrome (MPS II) that crosses the blood-brain barrier. The study showed significant reduction in key disease biomarkers, normalization of heparan sulfate levels in cerebrospinal fluid and urine, and improvements in clinical endpoints including cognition, adaptive behavior, and hearing. The FDA is conducting Priority Review of the Biologics License Application with a decision expected by April 5, 2026.

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Publication of Phase 1/2 trial results in prestigious New England Journal of Medicine showing strong efficacy data with 91% reduction in cerebrospinal fluid heparan sulfate levels, 93% of participants reaching normal levels, and clinical improvements in cognition and hearing. FDA Priority Review with expected decision by April 2026 represents significant regulatory progress. Multiple FDA designations (Breakthrough Therapy, Rare Pediatric Disease, Fast Track, Orphan Drug) support advancement toward potential approval.

Keywords
#Hunter syndrome #MPS II #enzyme replacement therapy #blood-brain barrier #tividenofusp alfa #DNL310 #TransportVehicle platform #FDA approval #lysosomal storage disease #biomarkers

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