Release Details
Denali Therapeutics Reports Full Year 2019 Financial Results and Business Highlights
“2019 was an extraordinary year for Denali with significant progress across our portfolio in programs targeting Alzheimer’s, ALS and Hunter syndrome, as well as completing our first patient trial for a LRRK2 inhibitor in Parkinson’s disease,” said
Recent Business Highlights
- Biomarker proof of concept for LRRK2 inhibitor program (DNL201 and DNL151) - In
January 2020 , Denali presented positive results from a Phase 1b study with DNL201 in patients with Parkinson’s disease and from a Phase 1 study with DNL151 in healthy volunteers. Both clinical trials showed dose-dependent target engagement, improvement in biomarkers of lysosomal function and demonstrated safety profiles supporting progression to further development in upcoming Phase 2/3 studies.
- First-in-human dosing of EIF2B activator program (DNL343) - In
February 2020 , Denali commenced dosing of its small molecule activator of EIF2B, DNL343, in healthy volunteers. DNL343 is a brain penetrant small molecule EIF2B activator designed to resolve cellular integrated stress response and restore protein translation and RNA metabolism in ALS, Frontotemporal Dementia and other neurodegenerative diseases.
- IND accepted for DNL310 for Hunter syndrome - In
January 2020 , the IND for DNL310 was accepted and Denali plans to initiate a Phase 1/2 study for DNL310 in Hunter syndrome patients in the first half of 2020. DNL310 is an intravenously administered recombinant form of the iduronate 2-sulfatase (“IDS”) enzyme engineered to cross the BBB using Denali’s proprietary Transport Vehicle (“TV”) technology. It is intended to treat overall clinical manifestations of Hunter syndrome, including cognitive and behavioral function, which are not addressed by the current standard of care.
- Pipeline update - In
January 2020 , Denali disclosed two additional biotherapeutic programs in its portfolio, which are engineered to cross the BBB using Denali’s proprietary TV technology.- DNL593 (PTV:Progranulin ("PGRN")) is an intravenously administered recombinant form of PGRN intended to restore normal levels of PGRN in the brain without interfering with normal PGRN processing. PGRN is genetically linked to Frontotemporal Dementia and other neurodegenerative diseases.
- ETV:N-sulphoglucosamine sulphohydrolase (“SGSH”), is an intravenously administered recombinant form of the enzyme SGSH, which is defective in patients with San Filippo syndrome. ETV:SGSH is intended to treat overall clinical manifestations of San Filippo syndrome, including cognitive and behavioral function.
- Balance sheet strengthened - In
January 2020 , Denali raised net proceeds of approximately$194.1 million through a public offering of its common stock.
Fourth Quarter and Full Year 2019 Financial Results
For the quarter ended
Collaboration revenue was
Total research and development expenses were
General and administrative expenses were
Cash, cash equivalents, and marketable securities were
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 Denali's plan to initiate a Phase 1/2 study for DNL310 in Hunter syndrome patients in the first half of 2020; Denali’s expectations regarding key milestones in 2020 including advancing its LRRK2 program into late stage clinical testing and establishing clinical biomarker proof-of-concept with its first large molecule program, ETV:IDS (DNL310) in Hunter syndrome, enabled by its blood-brain barrier delivery Transport Vehicle platform; and statements made by Denali’s Chief Executive Officer.
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: Denali’s early stages of clinical drug development; Denali’s ability to complete the development and, if approved, commercialization of its product candidates; the risk of the occurrence of any event, change or other circumstance that could give rise to the termination of Denali’s collaboration agreements; Denali’s dependence on successful development of its BBB platform technology; Denali’s ability to conduct or complete clinical trials on expected timelines; 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 develop commercially successful products; 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; and other risks, including those described in Denali’s most recent Annual Report on Form 10-K, most recent Quarterly Report on From 10-Q and Denali’s future reports to be filed with the
Condensed Consolidated Statements of Operations | |||||||||||||||
(Unaudited) | |||||||||||||||
(In thousands, except share and per share amounts) | |||||||||||||||
Three Months Ended |
Twelve Months Ended |
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2019 | 2018 | 2019 | 2018 | ||||||||||||
Collaboration revenue | $ | 4,672 | $ | 125,676 | $ | 26,678 | $ | 129,160 | |||||||
Operating expenses: | |||||||||||||||
Research and development | 51,551 | 39,909 | 193,382 | 143,183 | |||||||||||
General and administrative | 10,879 | 11,045 | 46,480 | 32,349 | |||||||||||
Total operating expenses | 62,430 | 50,954 | 239,862 | 175,532 | |||||||||||
Income (loss) from operations | (57,758 | ) | 74,722 | (213,184 | ) | (46,372 | ) | ||||||||
Interest and other income, net | 3,808 | 2,811 | 15,219 | 10,132 | |||||||||||
Income (loss) before income taxes | (53,950 | ) | 77,533 | (197,965 | ) | (36,240 | ) | ||||||||
Income tax benefit (provision) | (75 | ) | — | 351 | — | ||||||||||
Net income (loss) | $ | (54,025 | ) | $ | 77,533 | $ | (197,614 | ) | $ | (36,240 | ) | ||||
Net income (loss) per share, basic | $ | (0.56 | ) | $ | 0.82 | $ | (2.07 | ) | $ | (0.39 | ) | ||||
Net income (loss) per share, diluted | $ | (0.56 | ) | $ | 0.79 | $ | (2.07 | ) | $ | (0.39 | ) | ||||
Weighted average number of shares outstanding, basic | 96,078,950 | 94,299,096 | 95,608,208 | 92,621,991 | |||||||||||
Weighted average number of shares outstanding, diluted | 96,078,950 | 97,746,224 | 95,608,208 | 92,621,991 |
Condensed Consolidated Balance Sheets | |||||||
(Unaudited) | |||||||
(In thousands) | |||||||
Assets | |||||||
Current assets: | |||||||
Cash and cash equivalents | $ | 79,449 | $ | 77,123 | |||
Short-term marketable securities | 335,907 | 387,174 | |||||
Prepaid expenses and other current assets | 14,675 | 16,539 | |||||
Total current assets | 430,031 | 480,836 | |||||
Long-term marketable securities | 39,886 | 147,881 | |||||
Property and equipment, net | 46,732 | 25,162 | |||||
Operating lease right-of-use asset | 33,923 | — | |||||
Other non-current assets | 2,659 | 8,105 | |||||
Total assets | $ | 553,231 | $ | 661,984 | |||
Liabilities and stockholders' equity | |||||||
Current liabilities: | |||||||
Accounts payable | $ | 2,590 | $ | 1,891 | |||
Accrued expenses and other current liabilities | 24,015 | 19,468 | |||||
Contract liabilities | 18,739 | 11,427 | |||||
Total current liabilities | 45,344 | 32,786 | |||||
Contract liabilities, less current portion | 43,753 | 57,350 | |||||
Operating lease liability, less current portion | 68,865 | — | |||||
Deferred rent, less current portion | — | 24,532 | |||||
Other non-current liabilities | 379 | 471 | |||||
Total liabilities | 158,341 | 115,139 | |||||
Total stockholders' equity | 394,890 | 546,845 | |||||
Total liabilities and stockholders’ equity | $ | 553,231 | $ | 661,984 | |||
Contacts:
(646) 495-2706
lhyland@gpg.com
or
Morgan Warners
(202) 295-0124
mwarners@gpg.com
Source: Denali Therapeutics Inc.