Day One Biopharmaceuticals (DAWN) 43rd Annual J.P. Morgan Healthcare Conference 2025 summary

Strategic vision and mission

• Focused on developing new medicines for life-threatening diseases in pediatric and adult populations, inspired by urgent pediatric needs.

• Achieved commercial status in 2024 with the U.S. approval and launch of Ojemda (tovorafenib) for relapsed/refractory pediatric low-grade glioma (PLGG) with BRAF alterations.

• Growth strategy centers on in-licensing, pipeline expansion, and establishing first and best-in-class therapies.

• Ex-U.S. commercial partnership with Ipsen established for Ojemda, expanding global reach.

• Maintains a strong financial position, ending 2024 with over $530 million in cash and no debt.

Commercial performance and market execution

• Ojemda generated $57.2 million in net product revenue in 2024, with Q4 revenue of $29 million and 44% quarter-over-quarter growth.

• Achieved approximately 80% reimbursement approval across all payers and high physician awareness in the concentrated PLGG market.

• Commercial model is efficient, with 18 sales reps covering 200 accounts that treat 90% of PLGG patients.

• No significant seasonality or holiday impact observed in Q4 sales; copay card and limited distribution model support patient access.

• Gross-to-net deductions improved after CMS recognized Ojemda's exclusive pediatric indication, reducing rebate rates.

Pipeline and clinical development

• Pivotal Phase 3 Firefly II trial for Ojemda in frontline PLGG is ongoing, with enrollment completion expected in the first half of 2026 and data anticipated 12 months after last patient enrollment.

• DAY301, a PTK7-targeted ADC for solid tumors, is in phase 1A dose escalation, with first dose cohort cleared in January 2025 and plans for histology-specific expansion.

• DAY301 aims to address both pediatric and adult cancers, with a focus on populations with limited therapeutic options.

• DAY301 Phase 1a/b trial uses BOIN design for efficient dose escalation, with potential registrational path pending FDA discussions.

• Pipeline expansion remains a priority, with ongoing business development to add differentiated assets.