Denali Therapeutics Announces Primary Analysis and Long-Term Follow-Up of Phase 1/2 Study in Hunter Syndrome (MPS II) with Tividenofusp Alfa

• Long-term data demonstrate that robust reductions and normalization in key biomarkers from baseline were maintained over time with continued improvement in hearing, cognition and adaptive behavior
• Long-term safety data with median follow-up of two years, and out to more than four years, demonstrate that tividenofusp alfa was generally well tolerated
• Regulatory submission for accelerated approval is planned for early 2025; U.S. launch preparation is ongoing to deliver tividenofusp alfa to families with MPS II in late 2025 or early 2026

Excerpt from the Press Release:

SOUTH SAN FRANCISCO, Calif., Feb. 06, 2025 (GLOBE NEWSWIRE) — Denali Therapeutics Inc. (NASDAQ: DNLI), today announced the primary analysis of the Phase 1/2 study in 47 participants with Hunter syndrome (MPS II) in the 24-week treatment period and additional long-term follow-up of its investigational therapeutic tividenofusp alfa (DNL310). These data, along with recent Breakthrough Therapy designation, further support the company’s plan to submit a biologics license application (BLA) in early 2025 for accelerated approval and deliver this potential treatment to the Hunter syndrome community in late 2025 or early 2026. The Phase 1/2 results are being presented this week at the 21st Annual WORLDSymposium™ conference in San Diego, California.

“Longer-term clinical data add to confidence that normalization of key biomarkers endures over time and that treatment with tividenofusp alfa is associated with continued improvement in hearing, cognition and behavior, which is meaningful to affected individuals and their families,” said Joseph Muenzer, M.D., Ph.D., Director of the Muenzer MPS Center and Professor in Pediatric Genetics at the University of North Carolina at Chapel Hill School of Medicine as well as an investigator in the Phase 1/2 study. “I look forward to new treatment options urgently needed by the Hunter syndrome community that effectively address the full spectrum of the disease.”

“Our primary analysis in 47 participants with MPS II and the additional long-term data in up to more than four years, support the potential of tividenofusp alfa to address neurocognitive, behavioral, and physical effects for all individuals living with MPS II.

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