Key Objectives & Platform - Denali aims to transform lives by delivering biotherapeutics to the whole body, including the brain[11] - The company's TransportVehicle (TV) platform enables systemic delivery of biologics to the brain and other hard-to-target tissues[12, 24] - Denali's TransportVehicle has demonstrated best-in-class properties for brain delivery, modularity, and safety[50] Near-Term Commercial Opportunities - Denali anticipates launching tividenofusp alfa (DNL310) in 2026 and DNL126 in 2027, establishing a commercial foundation for the Enzyme TransportVehicle (ETV) franchise[13] - Tividenofusp alfa has a PDUFA target action date of April 5, 2026, for accelerated approval[61, 81] - The ETV franchise targets lysosomal storage disorders (LSDs), with a combined market opportunity exceeding $1 billion for MPS II and MPS IIIA[13, 107] Pipeline & Milestones - Denali has a broad clinical-stage pipeline, including programs for Alzheimer's disease, with several near-term milestones expected[14] - The company anticipates an approval decision for tividenofusp alfa, ETV:SGSH Phase 1/2 data, and initiation of Phase 1 studies for OTV:MAPT, ATV:Abeta, and ETV:GAA in the first half of 2026[16] - Denali is developing the next generation of enzyme replacement therapies designed to treat brain and body manifestations of serious genetic diseases[61, 64] Financial Position - Denali has a strong financial foundation with approximately $873 million in cash and investments as of Q3 2025, plus $488 million from royalty financing and an equity capital raise in December 2025[146]

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]