Denali Therapeutics Reports Fourth Quarter and Full Year 2025 Financial Results and Business Highlights
- Tividenofusp alfa (DNL310; ETV:IDS) launch readiness established ahead of April 5, 2026 Prescription Drug User Fee Act (PDUFA) target action date for Hunter syndrome
- DNL126 (ETV:SGSH) Phase 1/2 preliminary data presented at 2026 WORLDSymposium™, supporting plans to pursue an accelerated approval path in Sanfilippo syndrome type A
- Start-up activities underway for DNL628 (OTV:MAPT) Phase 1b study for Alzheimer's disease and DNL952 (ETV:GAA) Phase 1 study for late-onset Pompe disease
- TransportVehicle™ platform and clinical pipeline progressing across lysosomal storage disorders and neurodegenerative diseases
SOUTH SAN FRANCISCO, Calif., Feb. 26, 2026 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (Nasdaq: DNLI) today reported financial results for the fourth quarter and full year ended December 31, 2025, and provided business highlights.
“In 2025, we made meaningful progress toward delivering urgently needed treatment options for people living with neurodegenerative diseases and lysosomal storage disorders, building on the strong scientific foundation that defines Denali,” said Ryan Watts, Ph.D., Chief Executive Officer of Denali Therapeutics. “We established commercial readiness for the anticipated launch of tividenofusp alfa for individuals and families affected by Hunter syndrome and continued advancing our TransportVehicle platform across serious neurologic and systemic diseases that impact millions worldwide.
"In 2026, we are focused on launching tividenofusp alfa and transforming life for individuals living with other serious diseases. Data presented at WORLDSymposium support our plans to pursue an accelerated approval path for DNL126 in Sanfilippo syndrome type A. We are also initiating clinical studies of DNL628 (OTV:MAPT) in Alzheimer’s disease and DNL952 (ETV:GAA) in late-onset Pompe disease. Over the next three years, we expect to advance four to six additional programs into the clinic, guided by our commitment to the patients we serve.”
Fourth Quarter 2025 and Recent Program Updates
CLINICAL PROGRAMS
Tividenofusp alfa (DNL310; ETV:IDS) for Hunter syndrome (mucopolysaccharidosis type II [MPS II])
Denali has established commercial launch readiness in anticipation of a regulatory decision on the Biologics License Application (BLA) for tividenofusp alfa under the U.S. Food and Drug Administration (FDA) accelerated approval pathway with a Prescription Drug User Fee Act (PDUFA) target action date of April 5, 2026. Results from the open-label Phase 1/2 clinical trial of tividenofusp alfa were published in the January 1, 2026 issue of The New England Journal of Medicine. The ongoing global Phase 2/3 COMPASS study is expected to generate confirmatory evidence and support global regulatory submissions; enrollment in Cohort A (neuronopathic participants) was completed in December 2025.
DNL126 (ETV:SGSH) for Sanfilippo syndrome type A (MPS IIIA)
In February 2026, Denali presented preliminary Phase 1/2 data at WORLDSymposium demonstrating treatment with DNL126 resulted in substantial reductions in disease biomarkers in cerebrospinal fluid (CSF heparan sulfate and GM3) and the periphery (urinary heparan sulfate) with a safety profile generally consistent with established enzyme replacement therapies. These preliminary data support an accelerated approval path in Sanfilippo syndrome type A. Planning for a global Phase 3 confirmatory study is ongoing.
TAK-594/DNL593 (PTV:PGRN) for GRN-related frontotemporal dementia (FTD-GRN)
TAK-594/DNL593 is an intravenously administered progranulin replacement therapy utilizing Denali’s Protein TransportVehicle™ (PTV) technology to deliver progranulin across the blood-brain barrier (BBB) and into the brain for individuals with FTD-GRN. Enrollment in the ongoing Phase 1/2 study is complete with a total of 40 participants with FTD-GRN enrolled. Initial FTD-GRN patient data are expected in 2026. The program is being developed in collaboration with Takeda.
DNL952 (ETV:GAA) for Pompe disease
DNL952 is enabled by Denali’s Enzyme TransportVehicle™ (ETV) and designed to enhance delivery of the missing enzyme, GAA, into muscle tissues and across the BBB into the brain. In January 2026, Denali announced that the FDA had lifted the clinical hold on the Investigational New Drug (IND) application for DNL952. Phase 1 study start-up activities are underway.
DNL628 (OTV:MAPT) for Alzheimer's disease
DNL628 is enabled by Denali’s Oligonucleotide TransportVehicle™ (OTV) and is designed to cross the BBB and reduce the tau protein by targeting the MAPT gene that encodes for tau. In January 2026, Denali announced that the Clinical Trial Application (CTA) for the Phase 1b study of DNL628 had been approved and study start-up activities are underway.
BIIB122/DNL151 (small molecule LRRK2 inhibitor) for Parkinson’s disease
A clinical data readout of the global Phase 2b LUMA study of BIIB122 for early-stage Parkinson’s disease is expected in mid-2026. Denali’s Phase 2a BEACON study in LRRK2-associated Parkinson’s disease remains ongoing. The LRRK2 program is being developed in collaboration with Biogen.
SAR443122/DNL758 (eclitasertib; small molecule RIPK1 inhibitor) for ulcerative colitis
The Phase 2 study of eclitasertib in participants with moderate to severe ulcerative colitis is expected to have results in the first half of 2026. The program is being developed by Sanofi.
IND-ENABLING STAGE PROGRAMS
Denali has multiple additional programs in the IND-enabling stage including DNL921 (ATV:Abeta) for Alzheimer’s disease; DNL111 (ETV:GCase) for Parkinson’s disease and Gaucher disease; DNL622 (ETV:IDUA) for MPS I; and DNL422 (OTV:SNCA) for Parkinson’s disease.
Corporate Updates
In December, Denali announced two funding events. The first was a $275.0 million synthetic royalty funding agreement with Royalty Pharma plc based on future net sales of tividenofusp alfa. The second was a successful public offering of common stock and pre-funded warrants totaling approximately $200.0 million in net proceeds.
Participation in Upcoming Investor Conferences
- TD Cowen 46th Annual Healthcare Conference, March 2-4, 2026, Boston
- UBS Biotech Summit Miami - Catalyst for Change, March 8-10, 2026, Miami
- Leerink Global Healthcare Conference, March 8-11, 2026, Miami
- Jefferies 2026 Biotech on the Beach Summit, March 10-11, 2026, Miami
Fourth Quarter and Full Year 2025 Financial Results
Net losses were $128.5 million and $512.5 million for the quarter and year ended December 31, 2025, respectively, compared to net losses of $114.8 million and $422.8 million for the quarter and year ended December 31, 2024, respectively.
Total research and development expenses were $97.9 million and $418.8 million for the quarter and year ended December 31, 2025, respectively, compared to $99.8 million and $396.4 million for the quarter and year ended December 31, 2024, respectively. The increase of approximately $22.4 million for the year ended December 31, 2025, compared to the comparative period in the prior year was primarily attributable to higher external research and development costs related to multiple preclinical and clinical TransportVehicle programs, as well as increased personnel and other operating expenses associated with our large molecule manufacturing facility in Salt Lake City, Utah. These increases were partially offset by lower external expenses related to small molecule programs, which also contributed to the $1.9 million decrease in research and development expenses for the quarter ended December 31, 2025, compared to the same period in the prior year.
General and administrative expenses were $39.5 million and $136.6 million for the quarter and year ended December 31, 2025, respectively, compared to $30.1 million and $105.4 million for the quarter and year ended December 31, 2024, respectively. The increases of $9.4 million and $31.1 million for the quarter and year ended December 31, 2025, compared to the comparative period in the prior year were primarily driven by headcount increases and other activities associated with preparing for the potential commercial launch for tividenofusp alfa.
Cash, cash equivalents and marketable securities were approximately $966.2 million as of December 31, 2025.
About Denali Therapeutics
Denali Therapeutics Inc. is a biotechnology company pioneering a new class of biotherapeutics designed to cross the blood-brain barrier using its proprietary TransportVehicle™ platform. With a clinically validated delivery platform and a growing portfolio of therapeutic candidates across all stages of development, Denali is advancing toward its goal of delivering effective medicines to transform the lives of people living with neurodegenerative diseases, lysosomal storage disorders and other serious diseases. For more information, please visit www.denalitherapeutics.com.
Denali Therapeutics Inc.
Condensed Consolidated Statements of Operations
(Unaudited)
(In thousands, except share and per share amounts)
| Three Months Ended December 31, | Twelve Months Ended December 31, | ||||||||||||||
| 2025 | 2024 | 2025 | 2024 | ||||||||||||
| Operating expenses: | |||||||||||||||
| Research and development | $ | 97,905 | $ | 99,787 | $ | 418,778 | $ | 396,440 | |||||||
| General and administration | 39,460 | 30,059 | 136,564 | 105,438 | |||||||||||
| Total operating expenses | 137,365 | 129,846 | 555,342 | 501,878 | |||||||||||
| Gain from divestiture of small molecule programs | — | — | — | 14,537 | |||||||||||
| Loss from operations | (137,365 | ) | (129,846 | ) | (555,342 | ) | (487,341 | ) | |||||||
| Interest and other income, net | 8,918 | 15,161 | 42,904 | 64,636 | |||||||||||
| Loss before income taxes | (128,447 | ) | (114,685 | ) | (512,438 | ) | (422,705 | ) | |||||||
| Income tax expense | (102 | ) | (68 | ) | (102 | ) | (68 | ) | |||||||
| Net loss | $ | (128,549 | ) | $ | (114,753 | ) | $ | (512,540 | ) | $ | (422,773 | ) | |||
| Net loss per share, basic and diluted | $ | (0.73 | ) | $ | (0.67 | ) | $ | (2.97 | ) | $ | (2.57 | ) | |||
| Weighted average number of shares outstanding, basic and diluted | 175,458,962 | 170,086,146 | 172,649,097 | 164,473,772 | |||||||||||
Denali Therapeutics Inc.
Condensed Consolidated Balance Sheets
(Unaudited)
(In thousands)
| December 31, 2025 | December 31, 2024 | ||||||
| Assets | |||||||
| Current assets: | |||||||
| Cash and cash equivalents | $ | 205,326 | $ | 174,960 | |||
| Short-term marketable securities | 662,553 | 657,371 | |||||
| Prepaid expenses and other current assets | 32,779 | 32,105 | |||||
| Total current assets | 900,658 | 864,436 | |||||
| Long-term marketable securities | 98,322 | 359,373 | |||||
| Property and equipment, net | 52,402 | 55,236 | |||||
| Finance lease right-of-use asset | 48,531 | 47,533 | |||||
| Operating lease right-of-use asset | 19,002 | 22,861 | |||||
| Other non-current assets | 25,939 | 24,741 | |||||
| Total assets | $ | 1,144,854 | $ | 1,374,180 | |||
| Liabilities and stockholders' equity | |||||||
| Current liabilities: | |||||||
| Accounts payable | $ | 3,330 | $ | 11,137 | |||
| Accrued expenses and other current liabilities | 95,021 | 91,071 | |||||
| Total current liabilities | 98,351 | 102,208 | |||||
| Operating lease liability, less current portion | 27,210 | 36,673 | |||||
| Finance lease liability, less current portion | 5,532 | 5,615 | |||||
| Total liabilities | 131,093 | 144,496 | |||||
| Total stockholders' equity | 1,013,761 | 1,229,684 | |||||
| Total liabilities and stockholders’ equity | $ | 1,144,854 | $ | 1,374,180 | |||
Disclaimer: The content of this article solely reflects the author's opinion and does not represent the platform in any capacity. This article is not intended to serve as a reference for making investment decisions.
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