Denali Therapeutics Inc. announced the completion of its Biologics License Application (BLA) submission for tividenofusp alfa under the U.S. Food and Drug Administration’s (FDA’s) accelerated approval pathway for Hunter syndrome. This milestone initiates the FDA’s 60-day filing review process, with a Prescription Drug User Fee Act (PDUFA) target action date to be communicated upon acceptance. The company is preparing for a potential commercial launch in late 2025 or early 2026, positioning tividenofusp alfa as a potential first FDA-approved enzyme replacement therapy engineered to cross the blood-brain barrier for Hunter syndrome.
For the first quarter ended March 31, 2025, Denali reported a net loss of $133.0 million, an increase from a net loss of $101.8 million in the first quarter of 2024. Total research and development expenses rose by approximately $9.2 million to $116.2 million, driven by increased investment in TV programs like DNL126 and preclinical OTV programs, as well as higher lab consumables, consultants, and general facilities costs due to the commencement of operations at Denali's new large molecule manufacturing facility in Salt Lake City, Utah. General and administrative expenses also increased by $4.1 million to $29.4 million, primarily due to BLA submission activities and commercial launch preparations for tividenofusp alfa.
As of March 31, 2025, Denali held approximately $1.05 billion in cash, cash equivalents, and marketable securities. The company also provided an update on DNL343 for ALS, stating that additional analyses in March 2025 did not demonstrate a treatment effect on neurofilament light (NfL), a biomarker of neuronal damage. Consequently, the active treatment extension in Regimen G of the Phase 2/3 HEALEY ALS Platform Trial was discontinued, though DNL343 was generally well tolerated.
In a corporate update, Denali officially opened its clinical biomanufacturing facility in Salt Lake City, Utah, in March 2025. This facility expands the company's U.S. manufacturing capabilities, strengthening supply chain control and operational efficiency for its complex biologic therapeutics. The facility has begun manufacturing drug supply for clinical trials, supporting the expansion of Denali's TV-enabled therapeutic portfolio.
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