Denali Therapeutics Inc. announced the primary analysis of its Phase 1/2 study in 47 participants with Hunter syndrome (MPS II) for tividenofusp alfa (DNL310), along with additional long-term follow-up data extending over four years. These results were presented at the 21st Annual WORLD Symposium conference.
The long-term data demonstrated substantial and significant reductions in central nervous system and peripheral biomarkers of disease, including cerebrospinal fluid (CSF) and urine heparan sulfate, and neurofilament light (NfL). These biomarkers were reduced to normal and near-normal levels, indicating sustained therapeutic effect.
Clinical outcomes observed included normal liver volume after 24 weeks of treatment, improvements in hearing thresholds across all tested frequencies, and skill gains in most participants on measures of adaptive behavior and cognition. Tividenofusp alfa was generally well tolerated, with most treatment-related adverse events being mild or moderate, and serious adverse events in three participants (6.4%) were manageable.
These compelling clinical data, combined with the recent Breakthrough Therapy designation, further support Denali's plan to submit a Biologics License Application (BLA) in early 2025 for accelerated approval. The company aims to deliver this potential treatment to the Hunter syndrome community in late 2025 or early 2026, and expects this progress to inform and accelerate other lysosomal storage disease programs, such as DNL126.
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