Company: DNLI
Filing Date: 2025-12-04
Form Type: 8-K
Source: 0001714899-25-000209
Chunk: 1

Company: Denali Therapeutics Inc.
Filing Date: 2025-12-04
Form: 8-K
Item: Item 8.01
Chunk 1
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Item 8.01 Other Events

On December 4, 2025, Denali provided the following key updates on programs across its development pipeline at its 2025 Investor Day.

Tividenofusp Alfa (ETV:IDS) - MPS II (Hunter Syndrome)

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. A Late Cycle Meeting with the FDA has been completed and labeling discussions are underway. Commercial readiness activities are progressing. Enrollment in Cohort A (neuronopathic) of the Phase 2/3 COMPASS study of tividenofusp alfa is expected to complete in December 2025.

Tividenofusp alfa received Rare Pediatric Disease Designation (“ RPDD”) status for the treatment of Hunter syndrome in January 2019. Denali is in ongoing dialogue with the FDA related to the eligibility of tividenofusp alfa to receive a Rare Pediatric Disease Priority Review Voucher (“ PRV”) upon approval1. Because Denali submitted a filing of its intent to request a PRV after the initial BLA submission, based on discussions with the FDA, the Company may not be eligible to receive the PRV. Therefore, Denali is not including any potential future proceeds from the sale of a PRV in its financial planning. Denali continues to work with the FDA and the FDA will determine whether to award a PRV upon approval of tividenofusp alfa.

DNL126 (ETV:SGSH) - MPS IIIA (Sanfilippo Syndrome Type A)

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. Denali plans to present initial Phase 1/2 data at the 2026 WORLD Symposium. Phase 3 planning is ongoing.

TAK-594/DNL593 (PTV:PGRN) - Frontotemporal Dementia ( GRNMutation)

The Phase 1/2 study of TAK-594/DNL593 is ongoing with screening closed in Cohort B. Initial FTD- GRN patient data are expected in 2026.

1 Congress created Section 529 of the Food, Drug, & Cosmetic Act to incentivize drug development for