Denali Therapeutics (NASDAQ:DNLI) announced promising Phase 1/2 results for tividenofusp alfa (DNL310) in the treatment of Hunter syndrome, with the data published in The New England Journal of Medicine supporting an FDA Priority Review.
The FDA has set a Prescription Drug User Fee Act (PDUFA) date for April 5, 2026.
In an open-label study of 47 children, with a median age of 5, tividenofusp alfa demonstrated significant reductions in key disease biomarkers.
Notably, cerebrospinal fluid (CSF) heparan sulfate levels were reduced by 91% at Week 24 and maintained at 92% at Week 153 in a subset of 16 patients.
Urine heparan sulfate levels decreased by 88% at Week 24 and 91% at Week 153 in 10 patients.
Additionally, neurofilament light (NfL) levels were reduced by 21% at Week 49 and 76% at Week 153 in 13 patients.
The treatment also showed clinical signals of efficacy, including liver volume normalization at 24 weeks, improved hearing thresholds, and gains in adaptive behavior and cognition.
The most common treatment-related adverse events were infusion-related reactions, though these declined with continued dosing.
Denali highlighted the encouraging results as a step forward in the treatment of Hunter syndrome, a rare lysosomal storage disorder that can lead to severe neurological and physical complications.
The company remains on track for potential FDA approval and is focused on advancing tividenofusp alfa as a promising treatment option for Hunter syndrome.