Denali Therapeutics reported that the results of its open‑label Phase 1/2 study of tividenofusp alfa (DNL310) for Hunter syndrome were published in the January 1 2026 issue of the New England Journal of Medicine. The publication confirms that the investigational enzyme replacement therapy is safe, well‑tolerated, and capable of delivering an enzyme across the blood‑brain barrier.
The study enrolled 47 pediatric patients and demonstrated dramatic biomarker reductions: cerebrospinal fluid heparan sulfate fell 91 % at week 24, with 93 % of participants reaching normal levels, while urinary heparan sulfate dropped 88 % at the same time point. These results provide the first peer‑reviewed evidence that the Transport Vehicle platform can address both central nervous system and peripheral manifestations of Hunter syndrome.
Denali’s Biologics License Application is under FDA priority review, and the agency has set an anticipated PDUFA action date of April 5 2026. The date is an extension from the original January 5 2026 target, granted after Denali submitted a major amendment with updated clinical pharmacology data. The extension reflects the FDA’s need for additional information but also confirms continued regulatory momentum.
Chief Executive Officer Ryan Watts said the company is “committed to working with regulators, physicians, and advocates to bring this important therapy to individuals and families living with Hunter syndrome.” Acting Chief Medical Officer Dr. Peter Chin added that the data “show that after treatment with tividenofusp alfa, the majority of participants had normalization of heparan sulfate levels in both cerebrospinal fluid and urine to levels seen in unaffected children.”
Analysts at Stifel, Jefferies, and William Blair have maintained buy ratings and price targets in the $37–$40 range, citing the robust biomarker data, the first‑in‑class mechanism, and the company’s priority review status as key drivers of approval confidence. The publication strengthens Denali’s competitive position against existing enzyme replacement therapies that do not cross the blood‑brain barrier and positions DNL310 for a potential commercial launch in 2026.
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