NDA for tovorafenib in relapsed or progressive pLGG accepted for FDA priority review
PDUFA target action date of
“Day One made significant progress this past quarter towards potentially bringing tovorafenib to children with cancer, most notably with the completion of the tovorafenib rolling submission to the FDA,” said
Program Highlights
- In
September 2023 , the Company announced updated FIREFLY-1 data for tovorafenib and completion of the rolling New Drug Application (NDA) submission to theU.S. Food and Drug Administration (FDA) for relapsed or progressive pediatric low-grade glioma (pLGG). - In
October 2023 , Day One announced that the FDA accepted its NDA for Priority Review. The Company anticipates being eligible for a Priority Review Voucher upon potential approval of tovorafenib. - The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of
April 30, 2024 . - Day One presented two case reports at the 2023
Connective Tissue Oncology Society Annual Meeting inNovember 2023 , documenting fusion-driven sarcoma case reports from the FIREFLY-1 and FIRELIGHT-1 studies. - Day One has concluded enrollment in the Phase 2a FIRELIGHT-1 substudy trial of tovorafenib as a monotherapy in patients 12 years and older with relapsed, progressive, or refractory solid tumors harboring MAPK pathway aberrations. Despite observing responses with a generally well-tolerated therapy, a limited duration of response in this relatively rare patient population was observed. Day One will discontinue this monotherapy substudy and re-direct resources to other programs. Results from the substudy will be shared for presentation or publication after the final dataset becomes available.
- 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.
- 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 andAsia , with approximately 70 sites activated.
Corporate Highlights and Upcoming Milestones
- In
August 2023 , Day One entered into a research collaboration and license agreement withSprint Biosciences AB for its Vaccinia Related Kinase 1 (VRK1) program, augmenting the Company’s portfolio of targeted therapies in oncology. - The Company has appointed
Adam Dubow , Day One’s current General Counsel, as one of its executive officers under Section 16 of the Securities Exchange Act of 1934, as amended.Mr. Dubow joined Day One inOctober 2022 and leads the legal and compliance functions.
Third Quarter 2023 Financial Highlights
- Cash Position: Cash, cash equivalents and short-term investments totaled
$405.5 million onSeptember 30, 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
$33.2 million for the third quarter of 2023 compared to$22.0 million for the third quarter of 2022. The increase was primarily due to additional employee compensation costs, an upfront license 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
$18.3 million for the third quarter of 2023 compared to$17.7 million for the third quarter of 2022. The increase was primarily due to additional employee compensation costs, as well as the ongoing build-out of commercial capabilities. - Net Loss: Net loss totaled
$46.2 million for the third quarter of 2023 with non-cash stock compensation expense of$9.6 million , compared to$37.8 million for the third quarter of 2022 with non-cash stock compensation expense of$8.6 million .
Upcoming Events
- Clinical data from the ongoing, open-label, pivotal Phase 2 FIREFLY-1 clinical trial evaluating the investigational agent tovorafenib in relapsed or progressive pLGG will be presented in two plenary presentations at the 2023
Society for Neuro-Oncology Annual Meeting onNovember 17, 2023 . - Piper Sandler 35th Annual Healthcare Conference,
November 28-30, 2023 .
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 has been studied in over 325 patients to date and 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
About
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
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
Consolidated Statements of Operations (unaudited) (in thousands, except shares) | |||||||||||||||
Three Months Ended September 30, | Nine Months Ended September 30, | ||||||||||||||
2023 | 2022 | 2023 | 2022 | ||||||||||||
Operating expenses: | |||||||||||||||
Research and development | $ | 33,163 | $ | 22,035 | $ | 93,173 | $ | 59,598 | |||||||
General and administrative | 18,275 | 17,664 | 53,374 | 44,568 | |||||||||||
Total operating expenses | 51,438 | 39,699 | 146,547 | 104,166 | |||||||||||
Loss from operations | (51,438 | ) | (39,699 | ) | (146,547 | ) | (104,166 | ) | |||||||
Investment income, net | 5,291 | 1,895 | 12,163 | 2,086 | |||||||||||
Other (expense) income, net | (3 | ) | 9 | (22 | ) | 8 | |||||||||
Net loss attributable to common stockholders | (46,150 | ) | (37,795 | ) | (134,406 | ) | (102,072 | ) | |||||||
Net loss per share, basic and diluted | $ | (0.54 | ) | $ | (0.53 | ) | $ | (1.73 | ) | $ | (1.61 | ) | |||
Weighted-average number of common shares used in computing net loss per share, basic and diluted | 85,952,501 | 71,008,993 | 77,682,237 | 63,522,774 |
Selected Consolidated Balance Sheet Data (unaudited) (in thousands) | ||||||||
2023 | 2022 | |||||||
Cash, cash equivalents and short-term investments | $ | 405,538 | $ | 342,269 | ||||
Total assets | 414,179 | 349,062 | ||||||
Total liabilities | 24,552 | 17,023 | ||||||
Accumulated deficit | (404,074 | ) | (269,668 | ) | ||||
Total stockholders’ equity | 389,627 | 332,039 | ||||||
DAY ONE MEDIA
media@dayonebio.com
DAY ONE INVESTORS
pkelleher@lifesciadvisors.com
Source:
2023 GlobeNewswire, Inc., source