Release Details
Denali Therapeutics Reports First Quarter 2025 Financial Results and Business Highlights Including Completion of BLA Rolling Submission for Tividenofusp Alfa for Hunter Syndrome
“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
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
DNL126 (ETV:SGSH) for Sanfilippo syndrome Type A (MPS IIIA)
In
TAK-594/DNL593 (PTV:PGRN) for GRN-related frontotemporal dementia
Denali and Takeda have an ongoing collaboration for the co-development and co-commercialization of DNL593, a therapeutic candidate engineered for the delivery of progranulin (PGRN) across the blood brain barrier (BBB) and into lysosomes for the treatment of frontotemporal dementia (FTD) associated with a mutation in the granulin (GRN) gene. Denali is conducting the ongoing Phase 1/2 study of DNL593 in FTD-GRN.
BIIB122/DNL151 (small molecule LRRK2 inhibitor) for the treatment of Parkinson’s disease (PD)
Denali and Biogen are jointly developing LRRK2 small molecule inhibitors for Parkinson’s disease (PD), with Biogen leading the global Phase 2b LUMA study evaluating the effect of BIIB122 on disease progression in early-stage PD. In
DNL343 (small molecule eIF2B agonist) for the treatment of amyotrophic lateral sclerosis (ALS)
In
IND-ENABLING STAGE PROGRAMS
Denali is advancing additional candidates across its TransportVehicle™ (TV) franchises, i.e., Enzyme TV (ETV), Antibody TV (ATV), and Oligonucleotide TV (OTV), with programs for lysosomal storage diseases, Alzheimer’s disease and Parkinson’s disease. Programs in the Investigational New Drug (IND)-enabling stage include:
ETV
- DNL952 (ETV:GAA) for Pompe disease
- DNL111 (ETV:GCase) for Parkinson’s disease and Gaucher disease
- DNL622 (ETV:IDUA) for Hurler syndrome (MPS I)
ATV
- DNL921 (ATV:Abeta) targeting amyloid beta for Alzheimer’s disease
OTV
- DNL628 (OTV:MAPT) targeting tau for Alzheimer’s disease
- DNL422 (OTV:SNCA) targeting alpha synuclein for Parkinson’s disease
Corporate Updates
In March, Denali officially opened its clinical biomanufacturing facility in
Participation in Upcoming Investor Conferences
Bank of America Healthcare Conference 2025,May 13-15 (Las Vegas )Jefferies Global Healthcare Conference ,June 3-5 (New York City )- Goldman Sachs 46th Annual Global Healthcare Conference,
June 9-11 (Miami ) BTIG Virtual Biotechnology Conference ,July 29
First Quarter 2025 Financial Results
Net loss was
Total research and development expenses were
General and administrative expenses were
Cash, cash equivalents, and marketable securities were approximately
About
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements expressed or implied in this press release include, but are not limited to, statements regarding expectations regarding Denali’s TV platform and its therapeutics and commercial potential; statements made by Denali’s Chief Executive Officer; plans, timelines, and expectations relating to DNL310, including enrollment in the ongoing global Phase 2/3 COMPASS study and the likelihood of global approvals, the timing of planned regulatory filings, and the timing, likelihood, and scope of regulatory approvals and commercial launch; plans, timelines, and expectations related to DNL126, including enrollment in the ongoing Phase 1/2 study, planned engagement with the FDA, and the likelihood and scope of regulatory approvals; plans regarding DNL593 and the ongoing Phase 1/2 study; plans, timelines, and expectations regarding DNL151, including with respect to the ongoing Phase 2b LUMA and Phase 2a BEACON studies; plans and expectations for Denali’s preclinical programs, including the timing of advancement to clinical studies; the impact and scalability of Denali’s in-house manufacturing operations; Denali’s future operating expenses and anticipated cash runway; and Denali’s participation in upcoming investor conferences. All drugs currently being developed by Denali are investigational and have not received regulatory approval for any indication. Actual results are subject to risks and uncertainties and may differ materially from those indicated by these forward-looking statements as a result of these risks and uncertainties, including but not limited to, risks related to: the impact of adverse economic conditions, tariffs, and inflation on Denali’s business and operations; the occurrence of any event, change, or other circumstance that could give rise to the termination of Denali’s agreements with Sanofi, Takeda, Biogen, or other collaborators; Denali’s transition to a late-stage clinical drug development company; Denali’s and its collaborators’ ability to complete the development and, if approved, commercialization of its product candidates; Denali’s and its collaborators’ ability to enroll patients in its ongoing and future clinical trials; Denali’s reliance on third parties for the manufacture and supply of its product candidates for clinical trials; Denali’s dependence on successful development of its blood-brain barrier platform technology and its programs and product candidates; Denali’s and its collaborators’ ability to conduct or complete clinical trials on expected timelines; the risk that preclinical profiles of Denali’s product candidates may not translate in clinical trials; the potential for clinical trials to differ from preclinical, early clinical, preliminary or expected results; the risk of significant adverse events, toxicities, or other undesirable side effects; the uncertainty that product candidates will receive regulatory approval necessary to be commercialized; Denali’s ability to continue to create a pipeline of product candidates or commercialize products; developments relating to Denali’s competitors and its industry, including competing product candidates and therapies; Denali’s ability to obtain, maintain, or protect intellectual property rights related to its product candidates; implementation of Denali’s strategic plans for its business, product candidates, and blood-brain barrier platform technology; Denali’s ability to obtain additional capital to finance its operations, as needed; Denali’s ability to accurately forecast future financial results and hedge against financial risk in the current environment; and other risks and uncertainties, including those described in Denali’s most recent Annual Report on Forms 10-K filed with the
Condensed Consolidated Statements of Operations
(Unaudited)
(In thousands, except share and per share amounts)
Three Months Ended |
|||||||
2025 | 2024 | ||||||
Operating expenses: | |||||||
Research and development | $ | 116,227 | $ | 107,016 | |||
General and administrative | 29,353 | 25,236 | |||||
Total operating expenses | 145,580 | 132,252 | |||||
Gain from divestiture of small molecule programs | — | 14,537 | |||||
Loss from operations | (145,580 | ) | (117,715 | ) | |||
Interest and other income, net | 12,610 | 15,913 | |||||
Net loss | $ | (132,970 | ) | $ | (101,802 | ) | |
Net loss per share, basic and diluted | $ | (0.78 | ) | $ | (0.68 | ) | |
Weighted average number of shares outstanding, basic and diluted | 171,222,030 | 149,404,188 | |||||
Condensed Consolidated Balance Sheets
(Unaudited)
(In thousands)
Assets | |||||
Current assets: | |||||
Cash and cash equivalents | $ | 56,947 | $ | 174,960 | |
Short-term marketable securities | 760,979 | 657,371 | |||
Prepaid expenses and other current assets | 60,712 | 32,105 | |||
Total current assets | 878,638 | 864,436 | |||
Long-term marketable securities | 235,844 | 359,373 | |||
Property and equipment, net | 57,765 | 55,236 | |||
Finance lease right-of-use asset | 50,199 | 47,533 | |||
Operating lease right-of-use asset | 21,963 | 22,861 | |||
Other non-current assets | 26,934 | 24,741 | |||
Total assets | $ | 1,271,343 | $ | 1,374,180 | |
Liabilities and stockholders’ equity | |||||
Current liabilities: | |||||
Accounts payable | $ | 8,458 | $ | 11,137 | |
Accrued compensation | 7,655 | 24,728 | |||
Accrued clinical and other research & development costs | 27,908 | 22,822 | |||
Accrued manufacturing costs | 14,579 | 12,779 | |||
Operating lease liability, current | 8,585 | 8,308 | |||
Deferred research and development funding liability, current | 17,338 | 14,129 | |||
Other accrued costs and current liabilities | 7,354 | 8,305 | |||
Total current liabilities | 91,877 | 102,208 | |||
Operating lease liability, less current portion | 34,449 | 36,673 | |||
Finance lease liability, less current portion | 5,598 | 5,615 | |||
Deferred research funding and development liability, less current portion | 16,733 | — | |||
Total liabilities | 148,657 | 144,496 | |||
Total stockholders’ equity | 1,122,686 | 1,229,684 | |||
Total liabilities and stockholders’ equity | $ | 1,271,343 | $ | 1,374,180 | |
Investor Contact:
Vice President, Investor Relations
[email protected]
(650) 452-2747
Media Contact:
FGS Global
[email protected]
(503) 851-0807

Source: Denali Therapeutics Inc.