Denali Therapeutics Reports Third Quarter 2026 Financial Results and Business Highlights
- AVLAYAH® (tividenofusp alfa-eknm) net product revenue accelerated to
$17.8 million in its second full commercial quarter - Continued growth expectations drive Q4 2026 net product revenue guidance of
$30.0 million to$32.0 million - AVLAYAH recognized as the “Best Biotechnology Product” of 2026 by
The Galien Foundation - Zafinofusp alfa advancing as Denali’s second potential product, with BLA submission on track for 2027 seeking accelerated approval for Sanfilippo syndrome type A (MPS IIIA)
- Five TransportVehicle™ (TV) programs expected to generate data in 2027: zafinofusp alfa (ETV:SGSH), DNL593 (PTV:PGRN), DNL628 (OTV:MAPT), DNL921 (ATV:Abeta) and DNL952 (ETV:GAA)
- Q3 2026 net income of
$70.1 million included a one-time$194.0 million net gain on the sale of a Priority Review Voucher, bringing cash, cash equivalents and marketable securities to$1.0 billion - Conference call and webcast today at
4:30 p.m. ET
“Families and physicians are telling us that AVLAYAH is making a difference for children with Hunter syndrome, and the results this quarter reflect that,” said
Third Quarter 2026 and Recent Program Updates
COMMERCIAL PRODUCT
AVLAYAH for Hunter syndrome (MPS II)
AVLAYAH (tividenofusp alfa-eknm) is the first and only
AVLAYAH's
$17.8 million in AVLAYAH net product revenue in the second full quarter of commercial availability.- Coverage now exceeds 70% of
U.S . commercial lives, up from more than 50% in the prior quarter, and 20 state Medicaid programs have published coverage of AVLAYAH. - A permanent J-code became effective
October 1, 2026 , streamlining billing and reimbursement for treatment centers. - Fourth-quarter 2026 net product revenue is expected to be
$30.0 million to$32.0 million .
Denali also completed enrollment of Cohort B, for a total enrollment of 65 participants across Cohorts A and B, in the global Phase 2/3 COMPASS study. This is an important step toward generating confirmatory evidence, expanding the
CLINICAL PROGRAMS
Lysosomal Storage Diseases
Zafinofusp alfa (DNL126; ETV:SGSH) for Sanfilippo syndrome type A (MPS IIIA)
Zafinofusp alfa is an investigational ETV-enabled enzyme replacement therapy designed to systemically deliver N-sulfoglucosamine sulfohydrolase (SGSH) throughout the body, including the brain, for the treatment of Sanfilippo syndrome type A. In the third quarter, the Phase 1/2 study completed the prespecified treatment duration for the primary endpoint. The study enrolled a total of 20 participants across the pediatric age range. A Biologics License Application (BLA) submission in 2027 seeking accelerated approval is on track.
Preliminary data from dose-finding cohorts in the ongoing Phase 1/2 study of zafinofusp alfa, presented at WORLDSymposium 2026, showed mean reductions across age groups of 80% and 83% at week 49 in the key disease biomarkers cerebrospinal fluid heparan sulfate (CSF HS) and urine HS, respectively, including normalization of CSF HS in some participants and normalization of liver volume in all assessed participants.
Denali plans to present additional data from the Phase 1/2 study at a medical congress in the first quarter of 2027. Start-up activities are underway for a global Phase 3 confirmatory study.
DNL593 (PTV:PGRN) for FTD-GRN
DNL593 is an investigational Protein TransportVehicle™ (PTV)-enabled protein replacement therapy designed to systemically deliver progranulin (PGRN) across the blood-brain barrier for the treatment of granulin (GRN)-related frontotemporal dementia (FTD-GRN). In
DNL952 (ETV:GAA) for Pompe disease
DNL952 is an investigational ETV-enabled enzyme replacement therapy designed to systemically deliver acid alpha-glucosidase (GAA) to muscle tissue and the brain by crossing the blood-brain barrier for the treatment of Pompe disease. The current standard of care is enzyme replacement therapy. Progressive motor weakness, respiratory failure and neurologic symptoms remain unmet needs. The Phase 1 study of DNL952 is enrolling participants with late-onset Pompe disease (LOPD), and initial clinical data are expected in 2027.
Alzheimer's Disease
DNL628 (OTV:MAPT) for Alzheimer's disease
DNL628 is an investigational Oligonucleotide TransportVehicle™ (OTV)-enabled antisense oligonucleotide designed for systemic delivery across the blood-brain barrier to reduce tau by targeting the MAPT gene for the treatment of Alzheimer’s disease. Initial clinical biomarker data from the ongoing Phase 1b study of DNL628 in participants with Alzheimer’s disease are expected in the first half of 2027.
DNL921 (ATV:Abeta) for Alzheimer's disease
DNL921 is an investigational Antibody TransportVehicle™ (ATV)-enabled antibody designed for systemic delivery across the blood-brain barrier to target amyloid plaques in Alzheimer's disease. Safety and clinical proof-of-concept data from the ongoing Phase 1/1b study of DNL921 in healthy volunteers and participants with Alzheimer's disease are expected in 2027.
Parkinson’s Disease
DNL151 (LRRK2 inhibitor) for Parkinson's disease
DNL151 is an investigational small molecule inhibitor of leucine-rich repeat kinase 2 (LRRK2) for the treatment of Parkinson’s disease. Denali is conducting the Phase 2a BEACON study in participants with LRRK2-positive Parkinson’s disease, with data expected in the first half of 2027.
IND-ENABLING STAGE PROGRAMS
Denali has multiple additional programs in the investigational new drug (IND)-enabling stage, including 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 October, Denali announced that AVLAYAH was recognized as the “Best Biotechnology Product” of 2026 by
Participation in Upcoming Investor Conferences
- Stifel 2026 Healthcare Conference,
November 10-12 (New York ) UBS Global Biopharma Conference ,November 16-17 (London )Jefferies Global Healthcare Conference ,November 16-19 (London )- 9th Annual
Evercore Healthcare Conference ,November 30-December 2 (Coral Gables) - J.P. Morgan 2027 Healthcare Conference,
January 11-14 (San Francisco )
Third Quarter 2026 Financial Results
Net product revenue was
Cost of goods sold was
Total research and development expenses were
Selling, general and administrative expenses were
Intangible asset amortization was
Net income was
Cash, cash equivalents and marketable securities were approximately
Conference Call and Webcast Information
Denali will host a conference call today,
About the Denali TransportVehicle™ Platform
The blood-brain barrier (BBB) is essential in maintaining the brain’s microenvironment and protecting it from harmful substances and pathogens circulating in the bloodstream. Historically, the BBB has posed significant challenges to drug development for central nervous system diseases by preventing most drugs from reaching the brain in therapeutically relevant concentrations. Denali’s TransportVehicle™ (TV) platform is a proprietary technology designed to effectively deliver large therapeutic molecules such as antibodies, enzymes and oligonucleotides throughout the whole body, including the brain, by crossing the BBB after intravenous administration. The TV platform is based on engineered Fc domains that bind to specific natural transport receptors, such as transferrin receptor and CD98 heavy chain amino acid transporter, which are expressed at the BBB and deliver the TV and its therapeutic cargo to the brain through receptor-mediated transcytosis. In animal models, antibodies and enzymes engineered with the TV platform demonstrate more than 10- to 30-fold greater brain exposure than similar antibodies and enzymes without this technology. Oligonucleotides engineered with the TV platform demonstrate more than a 1,000-fold greater brain exposure in primates than systemically delivered oligonucleotides without this technology. Improved exposure and broad distribution in the brain may increase therapeutic efficacy by enabling widespread achievement of therapeutically relevant concentrations of product candidates. The TV platform has been clinically validated, with AVLAYAH® (tividenofusp alfa-eknm) as the first FDA-approved medicine leveraging transferrin receptor to cross the BBB.
About Denali Therapeutics
Denali Therapeutics Inc. is a biotechnology company pioneering a new class of biotherapeutics designed to cross the blood-brain barrier (BBB) using its proprietary TransportVehicle™ platform. With the first FDA-approved biologic specifically designed to cross the BBB, 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 life for people with neurodegenerative diseases, lysosomal storage disorders and other serious diseases. For more information, please visit www.denalitherapeutics.com.
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 for Denali’s TransportVehicle™ (TV) platform, including the Enzyme TransportVehicle™ (ETV) franchise and its therapeutic and commercial potential; plans, timelines and expectations relating to AVLAYAH® (tividenofusp alfa-eknm) and expected revenues, including expectations regarding demand, payer coverage, and reimbursement; expectations related to the ongoing Phase 2/3 COMPASS study of tividenofusp alfa, including the timing and availability of data and the likelihood that it will generate confirmatory evidence to support continued approval, U.S. label expansion, and global regulatory submissions; plans, timelines and expectations related to zafinofusp alfa, including with respect to the ongoing Phase 1/2 study and the planned Phase 3 confirmatory study, the timing and availability of data, the timing and occurrence of a planned BLA submission, and the likelihood and timing of accelerated approval; plans, timelines and expectations related to DNL593, including the ongoing Phase 1/2 study and the timing and availability of data; plans, timelines and expectations related to DNL952 and the timing and availability of data from the ongoing Phase 1 study; plans, timelines and expectations related to DNL628, including the ongoing Phase 1b study and the timing and availability of clinical biomarker data; plans, timelines and expectations related to DNL921, including the Phase 1/1b study and the timing and availability of data; plans, timelines and expectations related to DNL151, including the timing and availability of data from the ongoing Phase 2a BEACON study; plans, timelines and expectations for IND-enabling stage programs; plans regarding participation in upcoming investor conferences; forecasts of future revenues and operating expenses; expectations regarding costs of goods sold; and statements by Denali's Chief Executive Officer. Actual results may differ materially from those expressed or implied by these forward-looking statements due to a variety of risks and uncertainties. These include, but are not limited to, risks arising from adverse economic conditions and their impact on Denali’s business and operations; Denali’s ability to complete the development and, if approved, the commercialization of product candidates; reliance on third-party manufacturers and suppliers for clinical trial and commercial materials; difficulties enrolling patients for ongoing and future clinical trials; potential delays or failures in meeting expected clinical trial timelines; discrepancies between preclinical, early-stage or preliminary clinical results and later-stage trial outcomes; the risk that interim or topline clinical results may not be predictive of final study results or longer-term outcomes; the risk that the FDA may not accept the data or surrogate endpoints Denali intends to use to support accelerated approval, may require additional or different studies, or may withdraw an accelerated approval if confirmatory studies do not verify clinical benefit; significant adverse events or other undesirable side effects; the uncertainty surrounding regulatory approvals required for commercialization in the U.S., Europe or other international jurisdictions, including uncertainties related to the FDA’s policies; developments relating to Denali's competitors and competing product candidates; Denali’s ability to obtain, maintain or protect intellectual property rights related to its product candidates; the implementation and success 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 revenues and operating expenses in the current environment; quarterly variability in net product revenue and the risk that revenue growth may not continue at recent rates; and other risks and uncertainties, including those described in Denali's Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC) on February 26, 2026, its Quarterly Report on Form 10-Q filed with the SEC on August 6, 2026, and Denali’s future SEC filings. Except for AVLAYAH, Denali's product candidates are investigational, and their safety and efficacy profiles have not yet been established. Denali undertakes no obligation to update or revise any forward-looking statements, to conform these statements to actual results or reflect changes in Denali’s expectations, except as required by law.
Condensed Consolidated Statements of Operations (Unaudited) (In thousands, except share and per share amounts) |
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| Three Months Ended |
Nine Months Ended |
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| 2026 | 2025 | 2026 | 2025 | ||||||||||||
| Revenue: | |||||||||||||||
| Product revenue, net | $ | 17,778 | $ | — | $ | 21,382 | $ | — | |||||||
| Total revenue | 17,778 | — | 21,382 | — | |||||||||||
| Operating expenses: | |||||||||||||||
| Cost of goods sold | 192 | — | 285 | — | |||||||||||
| Research and development | 106,102 | 101,950 | 306,967 | 320,873 | |||||||||||
| Selling, general and administrative | 37,485 | 35,484 | 107,279 | 97,104 | |||||||||||
| Intangible asset amortization | 750 | — | 1,500 | — | |||||||||||
| Total operating expenses | 144,529 | 137,434 | 416,031 | 417,977 | |||||||||||
| Loss from operations | (126,751 | ) | (137,434 | ) | (394,649 | ) | (417,977 | ) | |||||||
| Other income: | |||||||||||||||
| Gain on sale of priority review voucher, net | 194,000 | — | 194,000 | — | |||||||||||
| Interest and other income, net | 2,877 | 10,532 | 14,775 | 33,986 | |||||||||||
| Total other income | 196,877 | 10,532 | 208,775 | 33,986 | |||||||||||
| Net income (loss) | $ | 70,126 | $ | (126,902 | ) | $ | (185,874 | ) | $ | (383,991 | ) | ||||
| Net income (loss) per share: | |||||||||||||||
| Basic | $ | 0.37 | $ | (0.74 | ) | $ | (0.99 | ) | $ | (2.24 | ) | ||||
| Diluted | $ | 0.37 | $ | (0.74 | ) | $ | (0.99 | ) | $ | (2.24 | ) | ||||
| Weighted-average shares used in calculating net income (loss) per share: | |||||||||||||||
| Basic | 188,387,660 | 172,421,492 | 187,452,793 | 171,702,183 | |||||||||||
| Diluted | 192,077,855 | 172,421,492 | 187,452,793 | 171,702,183 | |||||||||||
Condensed Consolidated Balance Sheets (Unaudited) (In thousands) |
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2026 |
2025 |
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| Assets | |||||
| Current assets: | |||||
| Cash and cash equivalents | $ | 102,870 | $ | 205,326 | |
| Short-term marketable securities | 413,326 | 662,553 | |||
| Accounts receivable, net | 18,882 | — | |||
| Inventory | 26,275 | — | |||
| Prepaid expenses and other current assets | 30,355 | 32,779 | |||
| Total current assets | 591,708 | 900,658 | |||
| Long-term marketable securities | 491,376 | 98,322 | |||
| Property and equipment, net | 47,907 | 52,402 | |||
| Finance lease right-of-use asset | 45,784 | 48,531 | |||
| Operating lease right-of-use asset | 15,612 | 19,002 | |||
| Intangible asset, net | 34,500 | — | |||
| Other non-current assets | 29,148 | 25,939 | |||
| Total assets | $ | 1,256,035 | $ | 1,144,854 | |
| Liabilities and stockholders' equity | |||||
| Current liabilities: | |||||
| Accounts payable | $ | 13,700 | $ | 505 | |
| Accrued expenses and other current liabilities | 81,558 | 97,846 | |||
| Total current liabilities | 95,258 | 98,351 | |||
| Operating lease liability, less current portion | 19,280 | 27,210 | |||
| Finance lease liability, less current portion | 5,449 | 5,532 | |||
| Liability related to the revenue participation right agreement | 209,661 | — | |||
| Total liabilities | 329,648 | 131,093 | |||
| Total stockholders' equity | 926,387 | 1,013,761 | |||
| Total liabilities and stockholders’ equity | $ | 1,256,035 | $ | 1,144,854 | |
Investor Contact:
hansen@dnli.com
Media Contact:
epatton@dnli.com
Source: Denali Therapeutics Inc.