Day One Biopharmaceuticals pany(DAWN) - 2025 Q4 - Annual Results

Financial Performance - As of December 31, 2025, the company reported cash, cash equivalents, and short-term investments of $441.1 million[5]. - OJEMDA net revenue for Q3 2025 was $38.5 million, up from $20.1 million in Q3 2024, representing a 91.5% increase[42]. - Total revenue for the nine months ended September 30, 2025, was $104.5 million, compared to $102.0 million for the same period in 2024, showing a 2.5% increase[42]. - The company reported a net loss of $19.7 million for Q3 2025, compared to a net income of $37.0 million in Q3 2024[42]. - Research and Development expenses for the nine months ended September 30, 2025, were $107.2 million, down from $165.9 million in 2024, a decrease of 35.4%[42]. - Selling, General and Administrative expenses for the nine months ended September 30, 2025, were $86.4 million, slightly up from $85.7 million in 2024[42]. Product Development and Approval - OJEMDA received FDA approval in April 2024 for pediatric patients aged 6 months and older with specific BRAF alterations[4]. - The company plans to advance the Emi-Le program with a path toward registration and has acquired Mersana Therapeutics[5]. - The pivotal Phase 3 trial of tovorafenib in front-line pediatric low-grade glioma (pLGG) is expected to complete enrollment in 1H 2026, with topline data expected mid-2027[31]. - The company plans to complete FIREFLY-2 trial enrollment in the first half of 2026, enabling mid-2027 data readout and potential approval in 2028[43]. - The company anticipates delivering Phase 1 clinical data for Emi-Le by mid-2026 and progressing to later-stage development[43]. - DAY301 is a next-generation antibody-drug conjugate (ADC) targeting PTK7, with Phase 1a data expected in 2H 2026[37]. Market Opportunity and Growth - The company aims to solidify OJEMDA as the second-line standard of care (SOC) in relapsed or refractory pediatric low-grade glioma (pLGG) and expand its market opportunity[5]. - The company anticipates a revenue opportunity exceeding $1 billion across three high unmet need indications[7]. - The number of treatment decisions for relapsed or refractory pediatric low-grade glioma (pLGG) is approximately 1,100 annually, with significant room for market share growth for OJEMDA[14]. - OJEMDA's estimated net product revenues for Q4 2025 are projected to be $52.8 million, reflecting a 37% growth compared to Q3 2025 and a 172% increase from 2024[15]. - The expected net product revenue for OJEMDA in 2026 is estimated to be between $225 million and $250 million, indicating over 50% growth compared to 2025[16]. - OJEMDA has achieved a 60% increase in 2L adoption over the past 12 months, with a growing number of healthcare providers selecting it as their treatment of choice[17]. - The company anticipates a significant increase in prescribing accounts, with 28% sequential growth in accounts treating four or more patients[18]. - OJEMDA's commercial execution is expected to drive strong growth in 2026, supported by favorable payer dynamics and a solidified position as the second-line standard of care[16]. Clinical Data and Efficacy - OJEMDA's median duration of response is reported at 19.4 months, with 77% of patients treatment-free for at least 12 months following 24 months of therapy[12]. - 75% of patients in the Expanded Access Program (EAP) who reached 24 months on OJEMDA continued treatment, demonstrating strong persistency[17]. - The Phase 2 FIREFLY-1 trial reported a 53% overall response rate (ORR) among patients, with 58% completing 26 or more treatment cycles[23]. - Median tumor size change from baseline after the last dose was -51% at 3 months and -55% at 6 months post-treatment[26]. - All 8 patients receiving OJEMDA retreatment were still on therapy at the time of data cutoff, with a median change in tumor size of -38.3%[27]. - In the updated 3-year analysis, 38 patients had RAPNO-defined progressive disease (PD) while on OJEMDA[26]. - No new safety signals were observed in the 3-year update, with 66% of treatment-related adverse events (TRAEs) being grade ≥3[29]. - 77% of 39 patients were treatment-free for at least 12 months, with the median treatment-free interval not yet reached[28]. Intellectual Property - The patent portfolio for OJEMDA includes a composition of matter patent extending to mid-2036, with additional protections potentially extending into the 2040s[20]. Strategic Partnerships - The company is focused on global commercial expansion through partnerships, including a license agreement with Ipsen for OJEMDA outside the U.S.[6]. - The company aims to expand OJEMDA globally, extending its commercial opportunity through collaboration with Ipsen[43].