(RTTNews) - Denali Therapeutics Inc. (DNLI) says 2026 will be a defining year, as the company readies its first commercial product, Tividenofusp alfa, and advances a broad slate of clinical programs across neurodegenerative diseases and lysosomal storage disorders. Tividenofusp alfa, Denali's TransportVehicle-enabled investigational therapy for Hunter syndrome, is currently under review by the U.S. FDA with an accelerated approval decision expected by April 5, 2026. The company is preparing for a commercial launch pending regulatory clearance, marking what it describes as the first commercial validation of its blood-brain-barrier-crossing platform.
Hunter syndrome, or MPS II, is a rare genetic disorder caused by a deficiency of the IDS enzyme, leading to progressive cognitive and physical decline. Denali's approach is designed to deliver the missing enzyme across the blood-brain barrier, a long-standing challenge in treating neuronopathic forms of the disease.
The company also expects multiple clinical readouts in 2026.
Initial data from a Phase 1/2 study of DNL126 for Sanfilippo syndrome Type A will be presented at the 2026 WORLD Symposium, supporting a potential accelerated approval pathway. Additional data are anticipated from programs targeting frontotemporal dementia, Parkinson's disease, and Alzheimer's disease, Parkinson's disease, and Alzheimer's dementia, including the LRRK2 inhibitor BIIB122/ DNL151, which completed enrolment in a Phase 2b LUMA study in 2025 and is expected to report results this year.
Denali is also planning to initiate first-in-human studies for several TransportVehicle-enabled candidates, including DNL628 for Alzheimer's disease and DNL952 for Pompe disease, following recent regulatory clearances. The FDA has lifted the clinical hold on DNL952, allowing the Phase 1 study to proceed.
The company continues to expand its leadership in transferrin-receptor-enabled therapeutics, advancing enzyme, oligonucleotide, and antibody programs designed to cross the blood-brain barrier.
Partnerships with Biogen and Takeda remain central to its strategy, with shared U.S. commercial rights for key programs and potential royalties from Sanofi-developed assets.
The company ended the third quarter of 2025 with approximately $872.9 million in cash, cash equivalents, and marketable securities, and subsequently raised an additional $200 million through equity financing.
The company entered a royalty funding agreement with Royalty Pharma that could provide up to $275 million tied to future net sales of Tividenofusp alfa.
DNLI has traded between $10.57 and $24.34 over the last year. The stock is currently trading at $16.28, up 1.62%.
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