Company: DNLI
Filing Date: 2025-12-09
Form Type: 424B5
Source: 0001628280-25-056085
Chunk: 6

Company: Denali Therapeutics Inc.
Filing Date: 2025-12-09
Form: 424B5
Chunk 6
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 and is essential for our survival, it also prevents the delivery of medicines to the brain in sufficient quantities to have therapeutic effect. We have invented, developed, and validated a proprietary technology, called the TransportVehicle TM ("TV"), to address the BBB challenge. The TV has a modular design enabling delivery of large molecules, i.e., enzymes, oligonucleotides, and antibodies, to all tissues of the body, including the brain, by crossing the BBB after systemic administration. Over the last few decades, large molecule biotherapeutics have enabled medical breakthroughs in treating a wide array of serious diseases, but with very limited success in CNS diseases. Now, with the invention and validation of our TV technology, we are leading the field in delivering on the potential of biotherapeutics to transform the lives of individuals with neurodegenerative, lysosomal, and other serious diseases.

We are building a broad portfolio of therapeutic candidates by investing in our TV franchises, i.e., Enzyme TV ("ETV"), Oligonucleotide TV ("OTV"), and Antibody TV ("ATV"), to advance programs for rare diseases, such as lysosomal storage diseases, and common diseases, such as Alzheimer's disease and Parkinson's disease. Our most advanced TV-enabled program is tividenofusp alfa (DNL310, ETV:IDS) for the potential treatment of mucopolysaccharidosis II ("MPS II", or Hunter syndrome). We submitted a biologics license application for tividenofusp alfa under the FDA's accelerated approval pathway in May 2025. The Biologics License Application (“BLA”) for tividenofusp alfa remains under review with the U.S. Food and Drug Administration (“FDA”), with a Prescription Drug User Fee Act (“PDUFA”) target action date of April 5, 2026. We are preparing for a potential commercial launch thereafter. Our second most advanced TV-enabled program is DNL126 (ETV:SGSH) for MPS IIIA (Sanfilippo syndrome). The Phase 1/2 study of DNL126 remains on track for completion in 2026, supporting a potential accelerated approval pathway and commercial launch by the end of 2027. Our TV-enabled portfolio also includes DNL593 (PTV:PGRN) for frontotemporal dementia-granulin (“FTD-GRN”), DNL628 (OTV:MAPT) for Alzheimer’s disease, DNL952 (ETV:G