Denali Therapeutics Reports First Quarter 2025 Financial Results and Business Highlights Including Completion of BLA Rolling Submission for Tividenofusp Alfa for Hunter Syndrome

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Denali Therapeutics Inc.
Denali Therapeutics Inc.

SOUTH SAN FRANCISCO, Calif., May 06, 2025 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (Nasdaq: DNLI) today reported financial results for the first quarter ended March 31, 2025, and provided business highlights.

“The completion of our BLA submission for tividenofusp alfa represents a pivotal milestone—not only in our commitment to delivering a potentially transformative therapy to individuals living with Hunter syndrome, but also in Denali’s evolution as a fully integrated, late-stage development and commercial organization,” said Ryan Watts, Ph.D., CEO of Denali Therapeutics. “We are now preparing for commercial launch in late 2025 or early 2026. If approved, tividenofusp alfa would be the first FDA-approved enzyme replacement therapy engineered to cross the blood-brain barrier to treat body and brain manifestations of Hunter syndrome. Our broader TransportVehicle-enabled pipeline continues to advance, including the DNL126 program for Sanfilippo syndrome Type A for which we have an ongoing and productive collaboration with the FDA through the START program. Additionally, the recent launch of our in-house clinical biomanufacturing facility in Salt Lake City further enhances our ability to scale efficiently in the U.S. and supply future programs across lysosomal and neurodegenerative diseases.”

First Quarter 2025 and Recent Program Updates

CLINICAL PROGRAMS

Tividenofusp alfa (DNL310, ETV:IDS) for Hunter syndrome (MPS II)

Today, Denali announced completion of submission of a Biologics License Application (BLA) for tividenofusp alfa under the U.S. Food and Drug Administration’s (FDA’s) accelerated approval pathway based on data from the Phase 1/2 study in participants with Hunter syndrome. The submission of the final BLA modules initiates the FDA’s 60-day filing review process and, upon acceptance of the application, the FDA will communicate the Prescription Drug User Fee Act (PDUFA) target action date. In January 2025, the FDA granted Breakthrough Therapy Designation for tividenofusp alfa for the treatment of individuals with Hunter syndrome (MPS II). Tividenofusp alfa was previously granted Fast Track, Orphan Drug and Rare Pediatric Disease designations. In February 2025, at the WORLD Symposium conference, Denali presented the primary analysis of the Phase 1/2 study in 47 participants with Hunter syndrome in the 24-week treatment period and additional long-term follow-up. Denali continues preparation for the commercialization of tividenofusp alfa with a focus on launch readiness across access, education, and community engagement. Denali is conducting the ongoing global Phase 2/3 COMPASS study to support global regulatory approvals.