Denali Therapeutics (DNLI) investor relations material

Strategic focus and technology platform

• Advancing transport vehicle technology to cross the blood-brain barrier, enabling delivery of therapeutics to the brain and other tissues using transferrin receptor targeting.

• Achieved significant milestones in Hunter syndrome with robust biomarker and clinical improvements, and submitted the first BLA using this technology in the U.S.

• Expanding the ETV franchise to additional indications, including Sanfilippo and Pompe, with ongoing regulatory and IND filings.

• Preparing for the first commercial launch and multiple new INDs in the coming year, with a focus on Alzheimer's and other neurodegenerative diseases.

R&D day and pipeline highlights

• R&D Day on December 4th will focus on engineering brain and whole-body delivery, ETV franchise foundation, and Alzheimer's programs targeting Tau and Aβ.

• Emphasis on the unique engineering principles of the transport vehicle platform compared to conventional approaches.

• Guest speakers will discuss opportunities in lysosomal storage diseases and Pompe.

Hunter syndrome (Tivi) commercialization and market outlook

• Tivi (Tividenofusp alfa) targets Hunter syndrome, with a PDUFA date in April 2026 and a robust clinical package supporting accelerated approval.

• Market opportunity benchmarked against EloPrase, with global annual sales of $650–700 million, and pricing around $500,000 per patient per year.

• Launch expected to follow an S-curve adoption, with modest revenues in the first year and focus on patient uptake as a key metric.

• Small, focused sales team planned, leveraging known patient and physician base for efficient commercialization.