Denali Shares Phase 1/2 Results for Rare Disease Treatment as FDA Reviews Accelerated Approval

Denali Therapeutics Inc. (NASDAQ:DNLI) today announced the publication of results from the open-label Phase 1/2 clinical trial of its investigational, next-generation enzyme replacement therapy (ERT), tividenofusp alfa (DNL310), for the treatment of Hunter syndrome (mucopolysaccharidosis type II, or MPS II) in the January 1, 2026 issue of The New England Journal of Medicine. The U.S. Food and Drug Administration (FDA) is conducting a Priority Review of the Biologics License Application (BLA) for tividenofusp alfa, which is supported by these data and for which Denali is seeking accelerated approval. A decision by the FDA on the tividenofusp alfa BLA is expected by April 5, 2026.

MPS II is a life-limiting lysosomal storage disease caused by a deficiency in the iduronate 2-sulfatase (IDS) enzyme, which is needed to break down complex sugars called glycosaminoglycans (GAGs). In individuals with MPS II, GAGs build up in cells throughout the body, including the brain, resulting in progressive damage to organs and tissues starting at a young age. MPS II occurs along a spectrum of disease severity, with approximately two-thirds of individuals developing progressive neurocognitive decline (neuronopathic MPS II). Current therapies do not cross the blood-brain barrier and therefore lack the potential to address the impact of the disease on cognitive abilities and behavior.