Day One Reports Fourth Quarter and Full Year 2023 Financial Results and Corporate Progress
- Strong cash position of $366.3 million at the end of 2023 providing runway into 2026.
- Phase 2 FIREFLY-1 registrational data for tovorafenib published in Nature Medicine.
- Upcoming PDUFA target action date for tovorafenib NDA in April 2024.
- Continued advancement in Phase 3 front-line trial with tovorafenib and exploration of pipeline additions.
- FDA accepted NDA for Priority Review of tovorafenib in October 2023.
- Hiring of 18 sales representatives in the U.S. for commercial preparedness of tovorafenib launch.
- Enrollment ongoing in Phase 3 FIREFLY-2/LOGGIC clinical trial for tovorafenib.
- Patient enrollment continues in Phase 1b/2 substudy of the FIRELIGHT-1 trial.
- Promotion of Elly Barry, MD to Chief Medical Officer.
- Addition of Habib Dable and Dr. William Grossman to the Board of Directors.
- Expected Phase 2 dose and schedule in FIRELIGHT-1 trial in 2H 2024.
- Participation in the 44th Annual TD Cowen Health Care Conference on March 5, 2024.
- Increase in R&D and G&A expenses for the fourth quarter and full year ended December 31, 2023 compared to 2022.
- Net loss of $54.5 million for the fourth quarter of 2023 with non-cash stock compensation expense.
- Net loss of $188.9 million for the year ended December 31, 2023.
Insights
The financial results of Day One Biopharmaceuticals, with a cash position of $366.3 million at the end of 2023, suggest a stable financial footing that can sustain operations into 2026. This runway is critical for investors assessing the company's risk profile, especially given the capital-intensive nature of drug development. The increase in R&D expenses to $130.5 million for the full year, up from $85.6 million in the prior year, reflects the company's investment in the clinical advancement of tovorafenib. While this increase indicates progress in their lead product candidate's development, it also contributes to the reported net loss of $188.9 million for the year. The non-cash stock compensation expenses, which are a common tool for biotech companies to attract and retain talent, have also risen, suggesting an expanding team and increased operational scale. For stakeholders, the financial health of Day One is a balance between the costs of advancing their pipeline and the potential market opportunity upon successful product launch.
The acceptance of the New Drug Application (NDA) for tovorafenib by the FDA for Priority Review and the setting of a PDUFA date are significant milestones. The Priority Review designation indicates that the FDA considers the drug to have the potential to provide significant improvements in the treatment of a serious condition. The focus on pediatric low-grade glioma (pLGG), a rare and often difficult-to-treat cancer, could position Day One to address an unmet medical need, potentially leading to orphan drug status and associated market exclusivities. The publication of Phase 2 FIREFLY-1 trial results in a prestigious journal like Nature Medicine adds scientific credibility and visibility to the company's research efforts. The ongoing Phase 3 trial and the combination study with pimasertib are also important to track as they will provide further data on efficacy and safety, which are crucial for eventual market penetration and competitive positioning.
The hiring of 18 sales representatives in anticipation of tovorafenib's approval indicates a proactive commercial strategy by Day One. This early buildout of a sales force is a strategic move to ensure immediate market penetration upon drug approval. The potential approval of tovorafenib and the eligibility for a Priority Review Voucher, which can be sold or used for future drug applications, represent significant commercial opportunities. The presence of seasoned biotechnology veterans joining the Board of Directors can be seen as a move to strengthen the company's governance and strategic decision-making in the oncology space. The market will be closely watching the recommended Phase 2 dose and schedule expected in the second half of 2024 as it will provide insights into the drug's commercial viability and potential treatment protocols.
PDUFA target action date for tovorafenib NDA in relapsed or progressive pLGG remains set for April 30, 2024
Phase 2 FIREFLY-1 tovorafenib registrational data published in Nature Medicine
Ended 2023 with
BRISBANE, Calif., Feb. 26, 2024 (GLOBE NEWSWIRE) -- Day One Biopharmaceuticals (Nasdaq: DAWN) (“Day One” or the “Company”), a clinical-stage biopharmaceutical company dedicated to developing and commercializing targeted therapies for people of all ages with life-threatening diseases, today announced its fourth quarter and full year 2023 financial results and highlighted recent corporate achievements.
“We have a monumental year ahead of us at Day One with the upcoming PDUFA date for tovorafenib,” said Jeremy Bender, Ph.D., chief executive officer of Day One. “Our team is trained and ready to deliver our first expected commercial medicine to children in need of new treatment options. We also continue to advance our Phase 3 front-line trial with tovorafenib and are actively exploring other potential additions to our pipeline for children and adults living with cancer.”
Program Highlights
- In October 2023, Day One announced that the U.S. Food and Drug Administration (FDA) accepted its New Drug Application (NDA) for Priority Review of tovorafenib. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of April 30, 2024. The Company anticipates being eligible for a Priority Review Voucher upon potential approval of tovorafenib.
- In November 2023, Nature Medicine published the registrational Phase 2 FIREFLY-1 trial results investigating tovorafenib in patients with BRAF-altered, relapsed or progressive pediatric low-grade glioma (pLGG).
- In the fourth quarter of 2023, Day One continued its commercial preparedness for the approval and launch of tovorafenib with the hiring of 18 sales representatives in the U.S.
- The pivotal Phase 3 FIREFLY-2/LOGGIC clinical trial evaluating tovorafenib as a front-line therapy in patients aged 6 months to 25 years with pLGG continues to enroll in the United States, Canada, Europe, Australia and Asia, with more than 80 sites activated.
- Patient enrollment continues in the Phase 1b/2 substudy (102b) of the FIRELIGHT-1 trial evaluating the combination of tovorafenib with the Company’s investigational MEK inhibitor, pimasertib.
Corporate Highlights and Upcoming Milestones
- Elly Barry, MD, has been promoted to Chief Medical Officer where she will lead the execution and expansion of Day One’s clinical development programs. Most recently, Dr. Barry was Head of Clinical Development at Day One where she played an integral role in the execution of the Company’s clinical programs. Prior to joining the Company in 2021, Dr. Barry was Global Clinical Lead for Pediatric Oncology at Pfizer, as well as Head of Pfizer’s Pediatric Oncology Leadership Team where she oversaw multiple oncology clinical programs. She has replaced Raphaël Rousseau, MD, PhD, who has transitioned into a strategic advisory consulting role into the second quarter of 2024.
- Day One welcomed seasoned biotechnology veterans Habib Dable and Dr. William Grossman to its Board of Directors. Both individuals bring deep expertise and leadership in oncology to the Company’s Board.
- The recommended Phase 2 dose and schedule in the FIRELIGHT-1 clinical trial is expected in 2H 2024.
Fourth Quarter and Full Year 2023 Financial Highlights
- Cash Position: Cash, cash equivalents and short-term investments totaled
$366.3 million on December 31, 2023. Based on Day One’s current operating plan, management believes it has sufficient capital resources to fund anticipated operations into 2026. - R&D Expenses: Research and development expenses were
$37.3 million and$130.5 million for the fourth quarter and full year ended December 31, 2023, respectively, as compared to$26.0 million and$85.6 million for the same periods in 2022. The increase was primarily due to additional employee compensation costs, a milestone payment, as well as clinical trial and manufacturing activities related to Day One’s lead product candidate, tovorafenib. - G&A Expenses: General and administrative expenses were
$22.2 million and$75.5 million for the fourth quarter and full year ended December 31, 2023, respectively, as compared to$16.7 million and$61.3 million for the same periods in 2022. The increase was primarily due to additional employee compensation costs, an ongoing commercial buildout, and professional service expenses to support company growth. - Net Loss: Net loss totaled
$54.5 million for the fourth quarter of 2023 with non-cash stock compensation expense of$10.8 million , compared to$40.1 million for the fourth quarter of 2022 with non-cash stock compensation expense of$6.8 million . Net loss was$188.9 million for the year ended December 31, 2023, with non-cash stock compensation expense of$39.3 million , compared to$142.2 million for the year ended December 31, 2022, with non-cash stock compensation expense of$27.2 million .
Upcoming Events
- 44th Annual TD Cowen Health Care Conference
- Management will participate in a fireside chat on Tuesday, March 5 at 9:50 a.m. ET. A live and archived audio webcast of the discussion will be available by visiting the Events & Presentations section of the Company’s website.
About Tovorafenib
Tovorafenib is an investigational, oral, brain-penetrant, highly selective type II RAF kinase inhibitor designed to target a key enzyme in the MAPK signaling pathway, which is being investigated in primary brain tumors or brain metastases of solid tumors. Tovorafenib is currently under evaluation in two pivotal clinical trials for pLGG. Tovorafenib is also being evaluated as a combination therapy for adolescent and adult patient populations with recurrent or progressive solid tumors with MAPK pathway aberrations (FIRELIGHT-1).
Tovorafenib has been granted Breakthrough Therapy and Rare Pediatric Disease designations by the FDA for the treatment of patients with pLGG harboring an activating RAF alteration. Tovorafenib has also received Orphan Drug designation from the FDA for the treatment of malignant glioma, and from the European Commission for the treatment of glioma.
About Day One Biopharmaceuticals
Day One Biopharmaceuticals is a clinical-stage biopharmaceutical company that believes when it comes to pediatric cancer, we can do better. We put kids first and are developing targeted therapies that deliver to their needs. Day One was founded to address a critical unmet need: the dire lack of therapeutic development in pediatric cancer. The Company’s name was inspired by “The Day One Talk” that physicians have with patients and their families about an initial cancer diagnosis and treatment plan. Day One aims to re-envision cancer drug development and redefine what’s possible for all people living with cancer—regardless of age—starting from Day One.
Day One partners with leading clinical oncologists, families, and scientists to identify, acquire, and develop important emerging cancer treatments. The Company’s lead product candidate, tovorafenib, is an investigational, oral, brain-penetrant, highly selective type II RAF kinase inhibitor. The Company’s pipeline also includes pimasertib, an investigational, oral, highly selective small molecule inhibitor of mitogen‐activated protein kinases 1 and 2 (MEK-1/-2). Day One is based in Brisbane, California. For more information, please visit www.dayonebio.com or find the Company on LinkedIn or X.
Cautionary Note Regarding Forward-Looking Statements
This press release contains “forward-looking” statements within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to: Day One’s plans to develop cancer therapies, expectations from current clinical trials, the execution of the Phase 2 and Phase 3 clinical trials for tovorafenib as designed, any expectations about safety, efficacy, timing and ability to complete clinical trials, release data results, the ability of Day One to obtain regulatory approvals for and to commercialize tovorafenib and other candidates in development, and the ability of tovorafenib to treat pLGG or related indications.
Statements including words such as “believe,” “plan,” “continue,” “expect,” “will,” “develop,” “signal,” “potential,” or “ongoing” and statements in the future tense are forward-looking statements. These forward-looking statements involve risks and uncertainties, as well as assumptions, which, if they do not fully materialize or prove incorrect, could cause our results to differ materially from those expressed or implied by such forward-looking statements.
Forward-looking statements are subject to risks and uncertainties that may cause Day One’s actual activities or results to differ significantly from those expressed in any forward-looking statement, including risks and uncertainties in this press release and other risks set forth in our filings with the Securities and Exchange Commission, including Day One’s ability to develop, obtain regulatory approval for or commercialize any product candidate, Day One’s ability to protect intellectual property, the potential impact of global business or macroeconomic conditions, including as a result of inflation, changing interest rates, potential instability in the global banking system, uncertainty with respect to the federal debt ceiling and budget and potential government shutdowns related thereto and global regional conflicts and the sufficiency of Day One’s cash, cash equivalents and investments to fund its operations. These forward-looking statements speak only as of the date hereof and Day One specifically disclaims any obligation to update these forward-looking statements or reasons why actual results might differ, whether as a result of new information, future events or otherwise, except as required by law.
| Day One Biopharmaceuticals, Inc. Consolidated Statements of Operations (unaudited) (in thousands, except shares) | ||||||||||||
| Year Ended December 31, | ||||||||||||
| 2023 | 2022 | 2021 | ||||||||||
| Operating expenses: | ||||||||||||
| Research and development | $ | 130,521 | $ | 85,618 | $ | 43,584 | ||||||
| General and administrative | 75,543 | 61,291 | 29,159 | |||||||||
| Total operating expenses | 206,064 | 146,909 | 72,743 | |||||||||
| Loss from operations | (206,064 | ) | (146,909 | ) | (72,743 | ) | ||||||
| Investment income, net | 17,187 | 4,746 | 4 | |||||||||
| Other expense, net | (40 | ) | (18 | ) | (15 | ) | ||||||
| Net loss | (188,917 | ) | (142,181 | ) | (72,754 | ) | ||||||
| Net loss attributable to redeemable convertible noncontrolling interest | — | — | (2,109 | ) | ||||||||
| Exchange of redeemable noncontrolling interest shares – deemed dividend | — | — | (99,994 | ) | ||||||||
| Net loss attributable to common stockholders/members | $ | (188,917 | ) | $ | (142,181 | ) | $ | (170,639 | ) | |||
| Net loss per share, basic and diluted | $ | (2.37 | ) | $ | (2.17 | ) | $ | (4.62 | ) | |||
| Weighted-average number of common shares used in computing net loss per share, basic and diluted | 79,773,004 | 65,466,773 | 36,960,569 | |||||||||
| Day One Biopharmaceuticals, Inc. Selected Consolidated Balance Sheet Data (unaudited) (in thousands) | ||||||||
| December 31, 2023 | December 31, 2022 | |||||||
| Cash, cash equivalents and short-term investments | $ | 366,347 | $ | 342,269 | ||||
| Total assets | 376,048 | 349,062 | ||||||
| Total liabilities | 29,508 | 17,023 | ||||||
| Accumulated deficit | (458,585 | ) | (269,668 | ) | ||||
| Total stockholders’ equity | 346,540 | 332,039 | ||||||
DAY ONE MEDIA
Laura Cooper, Head of Communications
media@dayonebio.com
DAY ONE INVESTORS
LifeSci Advisors, PJ Kelleher
pkelleher@lifesciadvisors.com
FAQ
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