Tividenofusp alfa treatment showed reduction and normalization in key disease biomarkers, stabilization or improvement in clinical endpoints including adaptive behavior, cognition and hearing, and normalization of liver volume Most common treatment-related adverse events were infusion-related reactions, which decreased with continued useTividenofusp alfa is an investigational, next-generation enzyme replacement therapy engineered to cross the blood-brain barrierBiologics License Application for tividenofusp ...

Denali's TransportVehicle (TV) Platform - Denali's TransportVehicle (TV) platform enables systemic delivery of biologics to the brain and other hard-to-target tissues[14, 25] - The company's TransportVehicle (TV) is the most clinically validated BBB technology with over 11,000 doses administered[61] - Denali's TransportVehicle (TV) platform has demonstrated best-in-class properties for brain uptake, modularity, safety, and architecture[55] Near-Term Launch Opportunities - Denali plans to launch tividenofusp alfa (DNL310) in 2026 and DNL126 in 2027, which could capture a $1B+ market opportunity[15] - Tividenofusp alfa has a PDUFA target action date of April 5, 2026[168] - Denali is targeting all ~2,000 MPS II patients worldwide in commercially accessible geographies[246] Pipeline and Therapeutic Areas - Denali has a broad clinical-stage pipeline across high-value therapeutic areas, including Alzheimer's disease and Parkinson's disease[16] - The company estimates a total addressable market (TAM) of >$5B for the ETV franchise[63] - Denali estimates a >$5B market potential for BBB-enabled AD therapeutics[68] Financial and Strategic Execution - Denali is well-capitalized and focused on efficient capital allocation and execution timelines for long-term value creation[17] - The company has secured $275M in potential total funding through a royalty financing agreement with Royalty Pharma[443] - Denali projects COGS <20% of revenue due to efficient processes[407]