Company Overview - Day One Biopharmaceuticals is focused on developing and commercializing targeted therapies for life-threatening diseases, particularly in pediatric cancer [3][4] - The company aims to address the critical unmet need in pediatric cancer therapeutic development, inspired by the initial conversations between physicians and families regarding cancer diagnosis and treatment [3] Upcoming Events - Day One will host a live conference call and webcast on August 5, 2025, at 4:30 p.m. ET to report its financial results and discuss corporate progress for the second quarter of 2025 [1] - The live audio of the webcast will be accessible on the company's Media & Investors page, with an archived version available for replay for 30 days following the event [2] Pipeline and Partnerships - The company's pipeline includes targeted cancer treatments such as tovorafenib (OJEMDA™) and DAY301 [4] - Day One collaborates with leading clinical oncologists, families, and scientists to identify, acquire, and develop important cancer treatments [4] Communication Channels - Day One utilizes its Investor Relations website, social media platforms, and other channels to disseminate news, announcements, and financial performance updates [5]

Company Overview and Financial Highlights - Day One is a commercial-stage biopharmaceutical company focused on developing targeted medicines for childhood and adult diseases[11] - As of March 31, 2025, Day One had approximately $473 million in cash, cash equivalents, and short-term investments[16,82] - OJEMDA net product revenue since launch reached $87.7 million[43] - Q1 2025 OJEMDA net product revenue was $30.5 million, representing an 11% increase compared to Q4 2024[43,47] - Cumulative prescriptions for OJEMDA since launch totaled 2,571 as of March 31, 2025[43] OJEMDA Clinical Data and Market Opportunity - OJEMDA demonstrated an overall response rate (ORR) of 51% in 76 evaluable patients with relapsed or refractory pLGG harboring a BRAF fusion or rearrangement, or BRAF V600 mutation[29,30] - The addressable U.S opportunity for OJEMDA is estimated to be approximately 2,000-3,000 patients[38,39] Pipeline Development - Enrollment completion is expected in the first half of 2026 for the FIREFLY-2 pivotal Phase 3 trial in front-line pLGG[18] - The first dose cohort for DAY301, a PTK7-targeted ADC, was cleared in January 2025[18,65]

Summary of Day One Biopharmaceuticals (DAWN) FY Conference Call - June 10, 2025 Company Overview - **Company**: Day One Biopharmaceuticals - **Product**: Ogemda (for treating pediatric low-grade gliomas, PLGG) Key Industry Insights - **Market Dynamics**: Ogemda has been on the market for over a year, with a strong initial launch due to an expanded access program that established a patient base early on [3][5] - **Patient Population**: The estimated U.S. relapsed PLGG patient population is around 26,000, with 2,000 to 3,000 patients on treatment at any given time [7][8] - **Adoption Rate**: The launch is expected to be slow and steady, akin to a rare disease launch, with gradual physician adoption and experience being critical for establishing Ogemda as a standard of care [11][12] Financial Performance - **Growth**: The company has seen steady and incremental gains, with double-digit growth anticipated moving forward [5][31] Clinical Insights - **Treatment Duration**: The median duration of treatment observed in clinical trials is approximately 24 months, consistent with commercial settings [34][45] - **Adverse Events (AEs)**: There is a noted increase in early dropouts due to skin toxicities, which are common with MAP kinase inhibitors [38][41] Physician Engagement - **Prescribing Behavior**: Efforts are focused on encouraging physicians to transition from off-label treatments to Ogemda, with educational initiatives and data publication being key strategies [13][14] - **Physician Confidence**: The adoption of Ogemda is heavily influenced by physician experiences regarding efficacy and safety, with a need for ongoing education about managing AEs [27][28][30] Regulatory and Market Expansion - **European Filing**: The European Medicines Agency (EMA) has received the filing for Ogemda, with expectations for additional filings in other countries [51] - **Frontline Study**: The FIREFLY two trial is progressing well, with enrollment expected to complete in the first half of next year [52] Strategic Development - **Business Development**: The company is actively pursuing additional high-quality assets for clinical development, focusing on both adult and pediatric oncology indications [60][64] - **Leadership Changes**: A new head of R&D has been appointed, expected to enhance the company's strategic direction in oncology [68] Additional Considerations - **Off-Label Use**: Approximately 10% of prescriptions are for off-label use, primarily in adult solid tumors, but this proportion is expected to decrease as the PLGG market grows [35][42] - **Long-Term Data**: There is a strong emphasis on generating long-term data to reassure physicians and support the adoption of Ogemda [15][16] This summary encapsulates the key points discussed during the conference call, highlighting the company's strategic focus, market dynamics, and clinical insights related to Ogemda and its patient population.

Core Insights - Day One Biopharmaceuticals has appointed Dr. Michael Vasconcelles as Head of Research & Development, bringing over 25 years of oncology expertise to the company [2][3][4] - Dr. Vasconcelles will play a crucial role in advancing the company's pipeline and supporting the growth of its lead product, OJEMDA [3][5] Company Overview - Day One Biopharmaceuticals is focused on developing targeted therapies for life-threatening diseases, particularly in pediatric cancer, addressing a significant unmet need in therapeutic development [6][7] - The company aims to redefine cancer drug development and improve outcomes for patients of all ages from the moment of diagnosis [6] Leadership and Experience - Dr. Vasconcelles has a robust background in oncology research and development, having held leadership roles at ImmunoGen, Flatiron Health, Unum Therapeutics, and Takeda [4][5] - His previous experience includes transforming ImmunoGen into a global biotech enterprise and overseeing clinical development at Genzyme [4] Strategic Goals - The company is committed to expanding its pipeline of first- or best-in-class medicines, with a focus on delivering transformative therapies for children with cancer [3][5] - Dr. Vasconcelles' leadership is expected to enhance the company's medical and scientific strategies, driving growth and innovation [3][5]

Core Insights - Day One Biopharmaceuticals (NASDAQ: DAWN) has completed one year since the launch of Ojemda (tovorafenib) for treating pediatric low-grade glioma, but the recent earnings updates did not lead to a stock price increase [1] Company Overview - The company focuses on developing treatments for pediatric low-grade glioma, a type of brain tumor [1] - Ojemda is the primary product launched by the company, aimed at addressing a significant medical need in the pediatric population [1] Market Performance - Despite the launch anniversary, the stock has not experienced a rally following the earnings updates, indicating potential investor concerns or market conditions affecting performance [1]

Summary of Conference Call Company and Industry Overview - The conference call discusses the performance and commercialization of **Ogemda**, a drug recently launched by the company, which has generated approximately **$90 million** in revenue within the first **11 months** of its launch [1][2] - The company is optimistic about achieving **double-digit growth** moving forward [2] Key Performance Metrics - The company reported **$54 million** in revenue for Ogemda in **2024**, significantly exceeding the initial consensus estimate of **$12 million** [3] - In **Q1**, net product sales reached over **$30 million**, indicating a potential annual run rate of **$120 million** if growth remains stable [4] Market Dynamics and Growth Potential - The company is focusing on expanding the use of Ogemda among prescribers, particularly in earlier lines of therapy, as many have initially used it in later line patients [5][6] - There is a significant opportunity to increase the number of patients treated with Ogemda, especially among healthier patients who are likely to stay on the drug longer [6] - The company has identified **200 centers** in the U.S. where **90%** of PLGG patients are treated, allowing for an efficient commercial model [11] Prescriber Engagement - The company has categorized prescriber accounts into three priority levels based on volume, with **100%** of priority one accounts having tried Ogemda, **75%** of priority two, and **35%** of priority three [11][12] - The focus is on deepening the engagement with prescribers to increase the number of patients treated and to educate them on the appropriate use of Ogemda [14] Compliance and Treatment Duration - Compliance with Ogemda has been reported as excellent, with high rates of on-time refills and minimal drug holidays observed [24][25] - The anticipated treatment duration in the commercial setting is expected to differ from clinical trials, with ongoing monitoring of patient experiences [22][23] Regulatory and Market Expansion - The company has filed for regulatory approval in the EU, with an anticipated decision by mid-2026, and expects a similar patient population in Europe as in the U.S. [40][41] - Pricing in Europe is expected to be lower than in the U.S., but the company sees this as a significant opportunity for market expansion [42] Business Development Strategy - The company is actively pursuing business development opportunities, particularly in oncology, and is interested in acquiring early clinical assets that align with its strategic focus [66][67] - The recent acquisition of **Day Three Zero One**, a PTK7 targeted ADC, exemplifies the company's strategy to invest in promising assets with clinical validation [56][63] Conclusion - The company is in a strong financial position and is focused on expanding the market for Ogemda while also exploring new business development opportunities in oncology [1][66] - The ongoing clinical trials and regulatory filings are expected to enhance the company's growth prospects and market presence in the coming years [48][49]

Company Performance - Day One Biopharmaceuticals reported a quarterly loss of $0.35 per share, better than the Zacks Consensus Estimate of a loss of $0.46, and an improvement from a loss of $0.72 per share a year ago, representing an earnings surprise of 23.91% [1] - The company posted revenues of $30.76 million for the quarter ended March 2025, surpassing the Zacks Consensus Estimate by 4.90%, compared to zero revenues a year ago [2] - The stock has lost about 41% since the beginning of the year, while the S&P 500 has declined by 3.9% [3] Earnings Outlook - The current consensus EPS estimate for the coming quarter is -$0.44 on revenues of $34.88 million, and for the current fiscal year, it is -$1.67 on revenues of $149.97 million [7] - The estimate revisions trend for Day One Biopharmaceuticals is currently unfavorable, resulting in a Zacks Rank 4 (Sell) for the stock, indicating expected underperformance in the near future [6] Industry Context - The Medical - Biomedical and Genetics industry, to which Day One Biopharmaceuticals belongs, is currently in the top 31% of over 250 Zacks industries, suggesting a favorable industry outlook [8] - Empirical research indicates a strong correlation between near-term stock movements and trends in earnings estimate revisions, which can be tracked by investors [5]

Financial Data and Key Metrics Changes - In Q1 2025, net product revenue for Ogemda reached $30.5 million, reflecting an 11% increase compared to the previous quarter, driven by a 16% growth in quarterly prescriptions [8][22][23] - Operating expenses, excluding cost of sales, were $68.9 million, which included $12.9 million in non-cash stock-based compensation [22][23] - The company maintained a cash balance of $473 million with no debt at the end of Q1 2025 [23][24] Business Line Data and Key Metrics Changes - Ogemda prescriptions grew to over 2,500 since launch, marking a 16% increase over the prior quarter [12][15] - The company reported a rebound in new patient starts in April, indicating strong demand despite initial seasonality effects in January [13][14] Market Data and Key Metrics Changes - The U.S. market for relapsed/refractory PLGG patients is estimated to have approximately 26,000 individuals, with about half likely in long-term remission [16][18] - The company aims to establish Ogemda as the second-line standard of care in this market, with a focus on increasing prescriber adoption and usage depth [19][20] Company Strategy and Development Direction - The company is focused on three priorities for 2025: establishing Ogemda as the standard of care, advancing the clinical development pipeline for FIREFLY two and DAY three zero one, and expanding the product portfolio [7][10] - The company is actively evaluating business development opportunities to enhance its clinical stage portfolio and drive long-term growth [10][24] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the continued growth of Ogemda, citing high prescriber adoption rates and patient demand [9][11] - The company remains well-positioned financially, with a disciplined investment approach to support growth while managing resources responsibly [24][25] Other Important Information - The company anticipates that costs and operating expenses will remain relatively consistent throughout the year, allowing for strategic investments in growth opportunities [23][24] - Management highlighted the importance of educating physicians on the benefits and appropriate use of Ogemda to drive deeper adoption [20][21] Q&A Session Summary Question: What are you seeing in terms of duration of therapy in the marketplace and how docs plan to use the drug long term? - Management noted that it is early to comment on duration, but a high percentage of patients continue on therapy month after month, consistent with expectations from the FIREFLY one study [28][29] Question: Can you provide a rough percentage of revenue from new patients versus ongoing patients? - Management indicated that the dynamics of new patient starts and ongoing patients remain similar to previous discussions, with approximately 90% on-label usage [33][34] Question: What headwinds did you see in Q1 regarding gross to net and reimbursement? - Management acknowledged seasonality effects in January due to delays in scans and appointments, but noted a rebound in new patient starts in April [35][37] Question: How should we think about new patient starts from here? - Management expects a consistent and steady ramp of new patients over time, emphasizing that this is akin to a rare disease launch rather than typical oncology trajectories [43][44] Question: Can you discuss the penetration within each priority center? - Management reported that 100% of priority one accounts have tried Ogemda, with ongoing efforts to deepen prescribing among these accounts [51][52]

Financial Performance - Since launch, OJEMDA net product revenue reached $87.7 million[10, 14] - In Q1 2025, OJEMDA net product revenue was $30.5 million[10, 14, 19] - Q1 2025 saw a $3 million (+11%) increase in U S net product revenue compared to Q4 2024[14, 19] - Total revenue for Q1 2025 was $30.8 million[29] - The company holds a strong financial position with $473 million in cash[10, 29] - Net loss for the company was ($360) million[29] Commercial Performance - Cumulative prescriptions since launch totaled 2,571[14, 21] - Prescription growth in Q1 2025 was driven by new patient starts and continued therapy among on-label patients[21] Market Opportunity - Priority 1 centers manage approximately 4,000 estimated patients[24] - Priority 2 centers manage approximately 5,000 estimated patients[24] - Priority 3 centers manage approximately 4,000 estimated patients[24]

Financial Data and Key Metrics Changes - In Q1 2025, DayOne Biopharmaceuticals reported net product revenue of $30.5 million, reflecting an 11% increase compared to the previous quarter, driven by a 16% growth in quarterly prescriptions [7][22]. - Operating expenses, excluding cost of sales, were $68.9 million, which included $12.9 million in non-cash stock-based compensation. This represented approximately a 4% decline in operating expenses compared to the previous quarter when excluding a one-time charge of $20 million [23]. Business Line Data and Key Metrics Changes - Ogemda's quarterly prescriptions increased to over 900, marking a 16% growth over the prior quarter, contributing to the overall revenue growth [7][12]. - Cumulative U.S. Ogemda prescriptions reached over 2,500 since launch, indicating growing physician confidence and the drug's critical role in treating pediatric low-grade glioma [12][15]. Market Data and Key Metrics Changes - The estimated prevalent pool of approximately 26,000 relapsed or refractory BRAF altered PLGG patients in the U.S. suggests significant growth potential for Ogemda, with about half of these patients likely in long-term remission [16][20]. - The company noted that prescriber adoption continues to increase, with high payer approval rates and patients experiencing longer treatment periods on Ogemda [8][11]. Company Strategy and Development Direction - The company aims to establish Ogemda as the standard of care in second-line plus pediatric low-grade glioma and to drive revenue growth through clinical development and portfolio expansion [6][10]. - DayOne is focused on advancing its clinical development pipeline, particularly the FIREFLY two and DAY three zero one programs, while also exploring business development opportunities to expand its multi-program clinical stage portfolio [10][11]. Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the steady increase in Ogemda revenue, citing strong market dynamics and a solid financial foundation that allows for strategic investments despite macroeconomic challenges [11][24]. - The company does not project the need for additional financing in the future, emphasizing disciplined financial management alongside growth opportunities [24]. Other Important Information - The company reported a cash balance of $473 million and no debt at the end of Q1 2025, positioning it well for future growth [23][24]. - Management highlighted the importance of educating physicians on the long-term effects and benefits of Ogemda to enhance adoption rates [55][58]. Q&A Session Summary Question: What are you seeing in terms of duration of therapy in the marketplace and how docs plan to use the drug long term? - Management noted that it is early to comment on duration but observed a high percentage of patients continuing therapy month after month, consistent with expectations from the FIREFLY one study [27][29]. Question: Can you provide a rough percentage of revenue from new patients versus ongoing patients? - Management indicated that the dynamics of new patient starts and ongoing patients remain similar to previous discussions, with approximately 90% on-label and 10% off-label usage [33][34]. Question: What headwinds did you see in Q1 regarding gross to net and reimbursement? - Management acknowledged seasonality in January due to delays in scans and appointments but noted a strong rebound in new patient starts in April [35][36]. Question: How should we think about new patient starts from here? - Management expects a consistent and steady ramp of new patients over time, emphasizing that this is akin to a rare disease launch rather than typical oncology trajectories [44][45]. Question: Can you talk about penetration within each of the priority centers? - Management reported that 100% of priority one accounts have tried Ogemda, with ongoing efforts to deepen prescribing and expand usage among physicians [51][52].