Summary of Day One Biopharmaceuticals FY Conference Call Company Overview - **Company**: Day One Biopharmaceuticals (NasdaqGS:DAWN) - **Industry**: Biopharmaceuticals, focusing on treatments for pediatric patients with life-threatening diseases Key Points and Arguments Product Development and Pipeline - **Ojenda**: Over 1,000 children treated; positioned to become the standard of care for relapsed or refractory pediatric lobar glioma (PLGG) [3][4] - **Firefly-2 Trial**: Enrollment completion anticipated in the first half of 2026, aiming to expand Ojenda's use into frontline PLGG [3][19] - **Acquisition of Mersana**: Completed in Q4 2025, adding the Emmy Lee program to the portfolio, which is expected to contribute significantly to revenue [4][20] - **Day 301 Program**: Early clinical results are promising, with potential applications in multiple adult and pediatric indications [6][23] Financial Performance - **Cash Position**: Ended 2025 with over $440 million in cash and no debt, allowing for continued investment in high-potential programs [5] - **Revenue Growth**: - Q4 2025 net product revenue reached $52.8 million, a 37% increase from Q3 [12] - Full-year 2025 net product revenues totaled $155.4 million, up 172% year-over-year [12] - Guidance for 2026 net product revenue for Ojenda is projected at $225-$250 million [16] Market Dynamics - **Patient Population**: Approximately 1,100 treatment-eligible relapsed refractory PLGG patients annually [17] - **Market Expansion**: Anticipated EME approval in 2026 for global expansion, led by partner Ipsen [17] - **Physician Adoption**: Increasing confidence in multi-year treatments, contributing to growth in patient persistence and treatment duration [17][31] Clinical Data and Efficacy - **Ojenda's Efficacy**: - 19.4 months duration of response and 42.6 months median time to next treatment observed [10] - 75% of patients who took a drug holiday after 24 months remained treatment-free for 12 months [11] - **Emmy Lee Program**: Early evidence of monotherapy efficacy in adenoid cystic carcinoma (ACC), with a potential market opportunity of $300 million [20][21] Strategic Focus - **Dual Growth Model**: Combination of durable revenue from Ojenda and clinical catalysts from new programs like Emmy Lee and Day 301 [24] - **Commitment to Patients**: Focus on developing new medicines that significantly improve patient outcomes [24] Additional Important Content - **Payer Dynamics**: High reimbursement rates (over 90%) and favorable gross-to-net dynamics due to a significant percentage of Medicaid patients [37] - **Physician Education**: Increased efforts in educating physicians and caregivers to improve treatment management and patient outcomes [33][34] - **Future Updates**: Anticipated updates on Emmy Lee and Day 301 programs in mid to late 2026, with a focus on safety and efficacy data [43][44]

Company Overview - Geron Corp. is a single-product biotech company that launched an infusion drug, Imetelstat, in June 2024 for the treatment of myelodysplastic syndrome (MDS) [1] - The company is also developing Imetelstat for a second cancer indication, myelofibrosis [1] Leadership and Expertise - The executive has over 10 years of experience in the biotechnology and healthcare industry, along with 15 years of biomedical research experience [1] - The executive has served as Investment Director at a major VC&PE fund in Russia, focusing on life sciences, and has been on the Board of Directors for several biotech startups in the US and Russia [1] - The executive's background includes academic appointments at the University of Pittsburgh and Russian State Medical University, with over 50 peer-reviewed publications [1] Product Development and Strategy - The company emphasizes a systematic analysis approach to estimate risks, clinical values, and commercial prospects of innovative projects [1] - The goal is to improve patients' lives by designing new medicines and translating ideas into commercially viable products [1]