On May 6, 2024 Day One Biopharmaceuticals (Nasdaq: DAWN) ("Day One" or the "Company"), a commercial-stage biopharmaceutical company dedicated to developing and commercializing targeted therapies for people of all ages with life-threatening diseases, reported its first quarter 2024 financial results and highlighted recent corporate achievements (Press release, Day One, MAY 6, 2024, View Source [SID1234642691]).

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"We are excited that OJEMDA is now approved and available here in the U.S., and we are grateful to the members of the pediatric brain tumor community whose support of the program has been invaluable," said Jeremy Bender, Ph.D., chief executive officer of Day One. "Our team is focused on executing our U.S. launch, on advancing our other programs, and on exploring opportunities to expand our pipeline."

Program Highlights

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In April 2024, the Company received U.S. Food and Drug Administration (FDA) accelerated approval of OJEMDA (tovorafenib), the first and only FDA approved therapy for the treatment of pediatric patients 6 months of age and older with relapsed or refractory pediatric low-grade glioma (pLGG) harboring a BRAF fusion or rearrangement, or BRAF V600 mutation.

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With OJEMDA now available and the first prescriptions written, patients have begun enrolling in EveryDay Support From Day OneTM, a comprehensive program that offers personalized services for OJEMDA patients and their care teams, including insurance coverage support, financial assistance options and educational resources throughout the treatment journey.

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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 90 sites activated.

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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

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The Company received a rare pediatric disease priority review voucher from the FDA upon OJEMDA’s approval.

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Results from the FIRELIGHT-1 Phase 1b and next steps are expected in the second half of 2024.

First Quarter 2024 Financial Highlights

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Cash Position: Cash, cash equivalents and short-term investments totaled $317.9 million on March 31, 2024. Based on Day One’s current operating plan, management believes it has sufficient capital resources to fund anticipated operations into 2026.

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R&D Expenses: Research and development expenses were $40.2 million for the first quarter of 2024 compared to $27.8 million for the first quarter of 2023. The increase was primarily due to additional employee compensation costs, a payment for the buyback of priority review voucher obligation, and increased clinical trial and manufacturing activities related to Day One’s lead product, OJEMDA.

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G&A Expenses: General and administrative expenses were $26.6 million for the first quarter of 2024 compared to $18.0 million for the first quarter of 2023. The increase was primarily due to additional employee compensation costs, ongoing commercial buildout, and increased professional service expenses to support company growth.

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Net Loss: Net loss totaled $62.4 million for the first quarter of 2024 with non-cash stock compensation expense of $12.6 million, compared to $42.4 million for the first quarter of 2023 with non-cash stock compensation expense of $9.4 million.

Upcoming Events

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2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, May 31-June 4, 2024
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Abstract #10036 titled "Type II RAF inhibitor tovorafenib in relapsed/refractory pediatric low-grade glioma (pLGG): Reversible decreases in growth velocity in the phase 2 FIREFLY-1 trial" will be presented in a poster session on Saturday, June 1 from 1:30-4:30 pm CDT in Hall A

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Goldman Sachs 45th Annual Global Healthcare Conference, June 10-13, 2024

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21st International Symposium on Pediatric Neuro-Oncology (ISPNO), June 28-29, 2024

About OJEMDA

OJEMDA (tovorafenib) is a Type II RAF kinase inhibitor of mutant BRAF V600, wild-type BRAF, and wild-type CRAF kinases.

OJEMDA is indicated for the treatment of patients 6 months of age and older with relapsed or refractory pediatric low-grade glioma (LGG) harboring a BRAF fusion or rearrangement, or BRAF V600 mutation. This indication is approved under accelerated approval based on response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

Tovorafenib is under evaluation as a therapy for patients with pLGG requiring front-line treatment (Phase 3 FIREFLY-2/LOGGIC). It is also being studied in combination with the MEK inhibitor pimasertib for adolescent and adult patient populations with recurrent or progressive solid tumors with MAPK pathway alterations (FIRELIGHT-1).

Tovorafenib was granted Breakthrough Therapy and Rare Pediatric Disease designations by the FDA for the treatment of patients with pLGG harboring an activating RAF alteration, and it was evaluated by the FDA under priority review. 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.

For more information, please visit www.ojemda.com.