FDA Approval and Market Opportunity - OJEMDA received FDA approval in April 2024 for treating pediatric patients aged 6 months and older with relapsed or refractory pediatric low-grade glioma (pLGG) harboring a BRAF fusion or rearrangement, or BRAF V600 mutation[7]. - Up to 75% of pLGGs have a BRAF alteration, with approximately 80% being BRAF fusions and 20% BRAF V600 mutations, indicating a significant market opportunity for OJEMDA[20]. - OJEMDA's estimated addressable U.S. opportunity is approximately 2,000-3,000 patients with pediatric low-grade glioma (pLGG)[32]. Financial Performance - As of December 31, 2024, the company reported a strong balance sheet with approximately $532 million in cash and no debt[14]. - For Q4 2024, OJEMDA net product revenues are projected to be $29 million, with an estimated total of $57.2 million for 2024[38]. - Total revenue for the three months ended September 30, 2024, was $93.8 million, with OJEMDA net revenue at $20.1 million[60]. - The company reported a net income of $37 million for the three months ended September 30, 2024, compared to a loss of $46.2 million in the same period of 2023[60]. Clinical Development and Pipeline - The acquisition of DAY301, a PTK7-targeted antibody-drug conjugate, significantly expands the company's pipeline and therapeutic offerings[14]. - The company aims to generate clinical proof of concept data in the DAY301 program and fund pipeline expansion through focused capital allocation[14]. - DAY301, a next-generation antibody-drug conjugate targeting PTK7, is expected to enter its first dose cohort in January 2025[46]. - The pivotal Phase 3 trial for tovorafenib in front-line pLGG is designed to assess the objective response rate based on RAPNO-LGG criteria[44]. - The pivotal Phase 2 trial of tovorafenib in relapsed or progressive pLGG (FIREFLY-1) includes three arms with a total of 77 patients in Arm 1, which targets KIAA1549-BRAF fusion or BRAF V600E mutation[72]. Treatment Efficacy - The overall response rate (ORR) for OJEMDA in patients with BRAF fusion or rearrangement was 51%, with a median duration of response (DOR) of 13.8 months[27]. - In clinical trials, 51% of children experienced tumor shrinkage of at least 25%, and 82% saw their tumors shrink or remain stable[34]. - In the FIREFLY-1 trial, the overall response rate (ORR) for Arm 1 was 51% (39 out of 76 patients) based on RAPNO-LGG criteria[77]. - The median duration of response (DOR) for patients in the FIREFLY-1 trial was 13.8 months, with a 95% confidence interval of 11.3-NR[85]. - The median time to response for patients in the FIREFLY-1 trial was 5.3 months, indicating a relatively quick therapeutic effect[79]. Safety and Adverse Events - 137 patients were evaluated for Tovorafenib safety data, with 100% experiencing any adverse events (AEs) and 98% experiencing treatment-related adverse events (TRAEs)[86]. - The most common TRAE was hair color changes, reported in 76% of patients, followed by anemia at 49% and elevated CPK at 56%[86]. - 33 patients (24%) experienced TRAEs leading to dose reduction, while 50 patients (37%) had TRAEs resulting in dose interruption[87]. - The median duration of dose interruption was 2 weeks, with 9 patients (7%) discontinuing treatment due to TRAEs[87]. - 63% of patients experienced Grade 2-3 AEs, with 42% experiencing Grade 2-3 TRAEs[86]. - Fatigue was reported in 55% of patients, with 4% experiencing Grade 2-3 fatigue[86]. - Vomiting occurred in 50% of patients, with 20% experiencing Grade 2-3 vomiting[86]. - 47% of patients experienced hypophosphatemia, with no Grade 2-3 cases reported[86]. - 34 patients (25%) reported COVID-19 as an adverse event, with no Grade 2-3 cases[86]. Strategic Focus and Growth - The company has established an ex-U.S. commercial partnership with Ipsen for OJEMDA, indicating a strategy for global market expansion[14]. - The company emphasizes the importance of minimizing the burden of conventional treatments, which can have significant long-term consequences for pediatric patients[22]. - Day One is well positioned for sustainable growth and long-term success, focusing on revenue growth and clinical development pipeline execution for FIREFLY-2 and DAY301[62]. - The company aims to leverage its development and commercialization expertise to expand its multiple asset portfolio[62]. - The company is focused on executing its clinical development pipeline while exploring new strategies for market expansion and potential acquisitions[62]. - The company maintains a strong capital position while investing in its pipeline, indicating a commitment to future growth[62].

Core Insights - Day One Biopharmaceuticals reported Q4 2024 net product revenues of $29.0 million and full year revenues of $57.2 million for OJEMDA (tovorafenib) [1][5] - The company ended 2024 with a strong cash position of $531.7 million [1][7] - The CEO highlighted the approval of OJEMDA as a significant growth catalyst and expressed confidence in the company's long-term growth trajectory [2] Financial Highlights - OJEMDA net product revenues increased by 44% from Q3 to Q4 2024 [5] - The company wrote over 1,600 OJEMDA prescriptions within eight months of its launch in April 2024 [5] - License revenue from the sale of ex-U.S. commercial rights for tovorafenib was $0.2 million for Q4 and $73.9 million for the full year [6] - Research and development expenses rose to $61.8 million for Q4 and $227.7 million for the full year, compared to $37.3 million and $130.5 million in 2023 [6] - Selling, general and administrative expenses were $29.8 million for Q4 and $115.5 million for the full year, up from $22.2 million and $75.6 million in 2023 [6] - The net loss for Q4 was $65.7 million and $95.5 million for the full year, with non-cash stock-based compensation expenses of $11.0 million and $48.3 million respectively [6][7] Corporate Developments - OJEMDA received the Exclusively Pediatric designation, reducing its Medicaid and 340B minimum rebate percentage from 23.1% to 17.1% [5] - The DAY301 program, a PTK7-targeted ADC, successfully cleared the first dose cohort in its Phase 1a clinical trial [5] - Enrollment in the pivotal Phase 3 FIREFLY-2 clinical trial is advancing, with full enrollment expected in the first half of 2026 [5]

Group 1 - Shares of Day One Biopharmaceuticals, Inc. (DAWN) have increased by 2.2% over the past four weeks, closing at $12.13, with a mean price target of $35.56 indicating a potential upside of 193.2% [1] - The average price target consists of nine estimates ranging from a low of $25 to a high of $43, with a standard deviation of $5.50, suggesting a potential increase of 106.1% to 254.5% from the current price [2] - Analysts show strong agreement on DAWN's ability to report better earnings than previously predicted, which supports the view of potential upside [4][9] Group 2 - The Zacks Consensus Estimate for DAWN has increased by 4.2% due to two upward revisions in earnings estimates over the last 30 days, with no negative revisions [10] - DAWN holds a Zacks Rank 1 (Strong Buy), placing it in the top 5% of over 4,000 ranked stocks based on earnings estimate factors, indicating strong potential for near-term upside [11] - While consensus price targets may not be reliable for predicting exact gains, they can provide a directional guide for price movement [12]

Core Insights - Day One Biopharmaceuticals will host a live conference call and webcast on February 25, 2025, at 4:30 p.m. ET to report financial results and discuss corporate progress for Q4 and full-year 2024 [1] - The company focuses on developing targeted therapies for life-threatening diseases in both children and adults, addressing the gap in new therapies due to traditional drug development models [3][4] Company Overview - Day One Biopharmaceuticals is a commercial-stage company dedicated to advancing first- or best-in-class medicines for serious diseases affecting all ages [3] - The company's pipeline includes tovorafenib (OJEMDA™), DAY301, and a VRK1 inhibitor program [5] - The company utilizes its Investor Relations website and social media platforms for disseminating news, financial performance updates, and compliance with disclosure obligations [6]

Core Insights - Day One Biopharmaceuticals reported preliminary unaudited net product revenue of approximately $57.2 million for 2024, with $29.0 million generated in the fourth quarter [2][6] - The company ended 2024 with approximately $531.7 million in cash, cash equivalents, and short-term investments, an increase from $366.3 million at the end of 2023 [2][6] - OJEMDA received FDA accelerated approval in April 2024, becoming the first FDA-approved therapy for pediatric low-grade glioma [5] - The company is focused on driving OJEMDA revenue, advancing its pipeline, and maintaining a strong financial position in 2025 [1][13] Financial Performance - Preliminary net product revenues for 2024 were approximately $57.2 million, with the fourth quarter contributing around $29.0 million [2] - Cash, cash equivalents, and short-term investments totaled approximately $531.7 million as of December 31, 2024, compared to $366.3 million on December 31, 2023 [2] Regulatory and Clinical Developments - OJEMDA received FDA accelerated approval for treating patients aged 6 months and older with relapsed or refractory pediatric low-grade glioma [5] - The Centers for Medicare & Medicaid Services approved OJEMDA exclusively for pediatric indications, reducing its Medicaid and 340B minimum rebate percentage from 23.1% to 17.1% [5] - Enrollment in the pivotal Phase 3 FIREFLY-2 clinical trial for tovorafenib is progressing, with over 100 sites activated globally [5] Strategic Initiatives - The company aims to continue growing OJEMDA revenue and advance its existing pipeline, including the FIREFLY-2 Phase 3 trial and DAY301 Phase 1a/b trial [13] - Day One has entered into an exclusive licensing agreement with Ipsen to commercialize tovorafenib outside the U.S. [5] - The company is pursuing business development opportunities to expand its pipeline while maintaining a self-sustaining financial position [13]

Core Insights - Day One Biopharmaceuticals reported preliminary unaudited net product revenue of approximately $57.2 million for 2024, with $29.0 million generated in the fourth quarter alone [2][3] - The company ended 2024 with approximately $531.7 million in cash, cash equivalents, and short-term investments, a significant increase from $366.3 million at the end of 2023 [3] - OJEMDA received FDA accelerated approval in April 2024, becoming the first FDA-approved therapy for pediatric low-grade glioma [6] Financial Highlights - Preliminary 2024 OJEMDA net product revenue was approximately $57.2 million, with $29.0 million in the fourth quarter [2][7] - Cash, cash equivalents, and short-term investments totaled approximately $531.7 million as of December 31, 2024, compared to $366.3 million on December 31, 2023 [3] Business Updates - The company aims to drive OJEMDA revenue and invest in programs leveraging its expertise in development and commercialization [2] - Enrollment in the pivotal Phase 3 FIREFLY-2 clinical trial for tovorafenib is expected to be completed in the first half of 2026 [6] - Day One has expanded its pipeline with the in-licensing of DAY301 and has cleared the first cohort in the Phase 1a portion of the DAY301 Phase 1a/b clinical trial [6] Upcoming Priorities - The company plans to continue growing OJEMDA revenue and advancing its existing pipeline, including the FIREFLY-2 Phase 3 clinical trial and DAY301 [6] - Day One will pursue business development opportunities to expand its pipeline while maintaining a self-sustaining financial position [6]

Since they bottomed in October 2022, the main stock indices have skyrocketed, The S&P 500 recording back-to-back gains of over 20% in 2023-2024, and the tech/growth-oriented Nasdaq-100 has performed even better.They lead the investing group Wheel of Fortune where they share actionable trading ideas across all asset-classes, sectors and industries. The goal of the service is to provide a one-stop-shop for investment and portfolio ideas, while educating the vibrant community of subscribers. Features of the se ...

Day One Biopharmaceuticals (NASDAQ: DAWN ) is a commercial-stage biotech focused on the development and marketing of mostly one drug: an RAF kinase inhibitor indicated for children with certain forms of brain cancer. The company has vacillated between $1 and $2 billion inI have my PhD in biochemistry and have worked for years analyzing clinical trials and biotech companies. It is my passion to educate everyone possible on the science behind the businesses that we invest in, and it's my mission to help you d ...

Company Overview - Day One Biopharmaceuticals is a biopharmaceutical company focused on developing and commercializing targeted therapies for life-threatening diseases across all age groups [1] - The company was founded to address the critical unmet need in pediatric cancer therapeutic development [3] - Day One aims to re-envision cancer drug development and redefine treatment possibilities for all cancer patients, inspired by "The Day One Talk" between physicians and patients [3] Pipeline and Partnerships - The company's pipeline includes tovorafenib (OJEMDA™), DAY301, and a VRK1 inhibitor program [4] - Day One collaborates with leading clinical oncologists, families, and scientists to identify, acquire, and develop targeted cancer treatments [4] Upcoming Event - Dr Jeremy Bender, CEO of Day One Biopharmaceuticals, will present at the 43rd Annual J P Morgan Healthcare Conference on January 13, 2025, at 3:45 PM Pacific Time / 6:45 PM Eastern Time [1] - A live audio webcast of the presentation will be available on the company's website, with an archived replay accessible for 30 days post-presentation [2] Corporate Information - Day One Biopharmaceuticals is headquartered in Brisbane, California [4] - The company utilizes its Investor Relations website, X handle, and LinkedIn page for disseminating business and financial information, including news, announcements, and regulatory disclosures [5]

Dr. Blackman’s departure planned for end of 2024 Company’s search for a new Head of R&D is ongoing BRISBANE, Calif., Nov. 20, 2024 (GLOBE NEWSWIRE) -- Day One Biopharmaceuticals, Inc. (Nasdaq: DAWN) (“Day One” or the “Company”), a biopharmaceutical company dedicated to developing and commercializing targeted therapies for people of all ages with life-threatening diseases, today announced that Samuel Blackman, M.D., Ph.D, co-founder and Head of Research & Development (R&D) of the Company, will be retiring ef ...