Core Insights - Aptose Biosciences is developing a unique triple drug therapy (TUS+VEN+AZA) for newly diagnosed acute myeloid leukemia (AML) patients, aiming for a safe and mutation-agnostic frontline treatment [1][6] - Initial data from the Phase 1/2 TUSCANY trial shows promising safety and efficacy, with complete remissions and measurable residual disease (MRD) negativity observed in patients with diverse mutations [1][4] Group 1: Clinical Trial Details - The TUSCANY trial has initiated dosing with tuspetinib (TUS) at 40 mg and 80 mg in combination with venetoclax (VEN) and azacitidine (AZA), demonstrating safety and antileukemic activity [2][4] - The trial is designed to evaluate various doses and schedules of TUS for AML patients who cannot receive induction chemotherapy, with a target enrollment of 18-24 patients by mid-late 2025 [7] Group 2: Patient Outcomes - In the first cohort, a patient with biallelic TP53 mutations achieved complete remission and MRD-negative status, while another FLT3-wildtype patient also achieved complete remission [4] - In the second cohort receiving 80 mg of TUS, all three patients showed blast reductions meeting criteria for complete remissions or complete remission with incomplete blood count recovery (CRi) [4] Group 3: Company Overview - Aptose Biosciences focuses on developing precision medicines for oncology, with tuspetinib as a lead candidate showing activity in relapsed or refractory AML and being advanced as a frontline therapy [9]

Core Viewpoint - Aptose Biosciences Inc. is participating in the 2025 Bloom Burton & Co. Healthcare Investor Conference to present its developments in precision oncology, specifically focusing on its lead compound tuspetinib for treating acute myeloid leukemia [1][4]. Company Overview - Aptose Biosciences is a clinical-stage biotechnology company focused on developing precision medicines for oncology, with an emphasis on hematology [5]. - The company's lead product, tuspetinib (TUS), is an oral kinase inhibitor that has shown efficacy as both a monotherapy and in combination therapy for relapsed or refractory acute myeloid leukemia (AML) [5]. - Tuspetinib is being developed as a frontline triplet therapy for newly diagnosed AML patients [5]. Conference Details - The 2025 Bloom Burton & Co. Healthcare Investor Conference will take place on May 5-6, 2025, in Toronto [1][4]. - Dr. William G. Rice, Chairman, President, and CEO of Aptose, will present on May 5, 2025, at 3:00 p.m. EDT [2][3]. - The presentation will be available via webcast, and further details can be found on the Aptose website [3]. Investor Engagement - The conference provides an opportunity for investors to receive corporate updates from various Canadian healthcare companies through presentations and one-on-one meetings [4]. - Aptose is open to scheduling one-on-one meetings with interested investors during the conference [2].

Core Insights - Aptose Biosciences Inc. is a clinical-stage precision oncology company focused on developing tuspetinib (TUS) as a frontline therapy for newly diagnosed acute myeloid leukemia (AML) [1][5] - The company will participate in the 2025 Bloom Burton & Co. Healthcare Investor Conference in Toronto on May 5-6, 2025, where Dr. William G. Rice will present and host one-on-one meetings [1][2][4] Company Overview - Aptose is committed to developing precision medicines to address unmet medical needs in oncology, with a focus on hematology [5] - The company's pipeline includes small molecule cancer therapeutics designed for single-agent efficacy and to enhance the efficacy of other anti-cancer therapies without overlapping toxicities [5] - Tuspetinib (TUS) is an oral kinase inhibitor that has shown activity as both a monotherapy and in combination therapy for patients with relapsed or refractory AML, and is being developed as a triplet therapy for newly diagnosed AML [5] Conference Details - The 2025 Bloom Burton & Co. Healthcare Investor Conference will feature corporate updates from various Canadian healthcare companies, providing opportunities for investors to engage with company representatives [4] - Dr. William G. Rice's corporate presentation is scheduled for May 5, 2025, at 3:00 p.m. EDT, and will be available via webcast [3]

Financial Data and Key Metrics Changes - The company reported a net loss of approximately $5.9 million for Q4 2024, compared to a net loss of approximately $4.2 million in Q4 2023, reflecting a loss per share increase from $0.39 to $0.54 [62] - For the full year 2024, R&D expenses were approximately $16.1 million, up from approximately $11.9 million in 2023, primarily due to increased research studies and payroll expenses [63] - The total cash position as of December 31, 2024, was approximately $24 million, which is expected to fund operations for at least 12 months [65] Business Line Data and Key Metrics Changes - All clinical stage drug candidates are now in Phase 1 and Phase 2 trials, with significant progress reported in the clinical pipeline and AI platform [12][14] - The Phase 2 asset LP-300 has seen accelerated enrollment, particularly in Japan and Taiwan, where the incidence of non-small cell lung cancer among never smokers is significantly higher [17][70] - The lead cohort for LP-300 achieved an 86% clinical benefit rate and a 43% objective response rate in never smoker non-small cell lung cancer patients [15] Market Data and Key Metrics Changes - The company is expanding its geographic strategy to Japan and Taiwan, where 33% to 40% of non-small cell lung cancer cases occur in never smokers, compared to just 15% in the U.S. [17] - The market potential for LP-300 is estimated to be between $4 billion to $5 billion annually, with a growing opportunity in the targeted population [14] Company Strategy and Development Direction - The company emphasizes the innovative use of AI and machine learning to transform oncology drug discovery and development, aiming for significant returns for investors and patients [8] - The strategic expansion into Asia is expected to enhance enrollment rates and generate robust data sets for clinical trials [17][70] - The company is focused on advancing its product candidates and pipeline, with a strong emphasis on R&D expenses exceeding G&A expenses [63] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the transformative potential of AI in drug development, highlighting the efficiency and cost-effectiveness of their approach [68][76] - The company anticipates multiple value-creating milestones throughout the year, with the potential to deliver transformative therapies for patients with limited treatment options [60][87] - Management acknowledged the need for substantial additional funding in the near future to support ongoing operations and clinical trials [65] Other Important Information - The company has received two FDA Fast Track designations for LP-184 in glioblastoma and triple-negative breast cancer, which could expedite clinical development timelines [18] - The RADR platform has expanded beyond $100 billion in oncology-specific data points, enabling sophisticated analysis and biomarker discovery initiatives [56][58] Q&A Session Summary Question: How is the pace and quality of enrollment in Asia compared to the U.S.? - The enrollment pace in Asia is about 2x to 4x faster than in the U.S., with significant output observed in recent months [90] Question: What are the opportunities for ADCs that substitute the toxic payload with another immunotherapy? - The design of multi-payload ADCs is seen as a future direction, with potential for combining immunomodulating agents with toxic payloads [91][92] Question: What should be expected from the HARMONIC update later this year? - The company expects to have a significant amount of data from enrolled patients, with key insights anticipated by mid to late Q2 [96][100] Question: When will results for LP-184 be provided? - The results for LP-184 have been delayed due to higher dose levels being reached, with therapeutic levels of efficacy beginning to be observed [107] Question: When will the pediatric trial for STAR begin? - The company is close to finalizing a protocol for pediatric brain cancers and expects to launch the trial mid to late this year [111]

Financial Data and Key Metrics Changes - The company reported a net loss of approximately $5.9 million for Q4 2024, compared to a net loss of approximately $4.2 million in Q4 2023, reflecting an increase in loss per share from $0.39 to $0.54 [62] - For the full year 2024, R&D expenses were approximately $16.1 million, up from approximately $11.9 million in 2023, primarily due to increased research studies and payroll expenses [63] - The total cash position as of December 31, 2024, was approximately $24 million, which is expected to fund operations for at least 12 months [65] Business Line Data and Key Metrics Changes - All clinical stage drug candidates are now in Phase 1 and Phase 2 trials, with significant progress reported in the clinical pipeline and AI platform [12][14] - The Phase 2 asset LP-300 has seen accelerated enrollment, particularly in Japan and Taiwan, where the incidence of non-small cell lung cancer among never smokers is significantly higher [17][70] - The lead cohort for LP-300 achieved an 86% clinical benefit rate and a 43% objective response rate in never smoker non-small cell lung cancer patients [15] Market Data and Key Metrics Changes - The market potential for LP-300 is estimated to be between $4 billion to $5 billion annually, with a growing opportunity due to the increasing incidence of non-small cell lung cancer in specific populations [14] - The market potential across indications for LP-184 exceeds $10 billion annually, addressing over 150,000 patients with limited therapeutic options [20] Company Strategy and Development Direction - The company is focused on leveraging AI and machine learning to transform oncology drug discovery and development, aiming for significant returns for investors and patients [8] - The strategic expansion into Asia is expected to enhance enrollment rates and generate robust data sets, positioning the company for compelling readouts in 2025 [17][70] - The company is developing an agentic AI platform that will enhance drug development efficiency and precision, potentially reducing preclinical development costs by 60% to 80% [80] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the transformative potential of AI in oncology, emphasizing the importance of data-driven approaches in drug development [87] - The company anticipates multiple value-creating milestones throughout the year, with a focus on delivering transformative therapies for patients with limited treatment options [60] Other Important Information - The company has received two FDA Fast Track designations for LP-184 in glioblastoma and triple negative breast cancer, which could expedite clinical development timelines [18] - The RADR platform has expanded beyond $100 billion oncology-specific data points, enabling sophisticated analysis that traditional approaches cannot match [56] Q&A Session Summary Question: How is the pace and quality of enrollment in Asia compared to the U.S.? - The pace of enrollment in Asia is about 2x to 4x faster than in the U.S., with significant output observed in recent months [90] Question: What are the opportunities for ADCs that substitute the toxic payload with another immunotherapy? - The company sees potential in using small molecule immunomodulating agents in ADCs, and there is ongoing exploration of multi-payload designs [91][92] Question: What should be expected in the upcoming HARMONIC update? - The company expects to have a significant amount of data from enrolled patients, with key readouts anticipated in mid to late Q2 [100] Question: What is the status of LP-184 data? - The LP-184 data was delayed due to higher dose levels being reached, which has extended the timeline for results [107] Question: When will pediatric trials for STAR begin? - The company is close to finalizing a protocol for pediatric brain cancers and expects to launch trials mid to late this year [111]

Industry Overview - Artificial Intelligence (AI) and Machine Learning (ML) are crucial in precision oncology, enhancing decision-making through the analysis of large datasets and digital pathology [1] - The global AI in oncology market is valued at $1.92 billion in 2023, with a projected CAGR of 29.4%, reaching $11.52 billion by 2030, indicating rising investor interest [3] AI Initiatives and Developments - The National Institutes of Health (NIH) is evaluating the PRIMED-AI initiative to enhance disease diagnosis and treatment through coordinated AI/ML efforts [2] - AI models developed by NIH aim to predict cancer patients' responses to therapies, showcasing the growing integration of AI in healthcare [2] Key Players in AI-Driven Precision Oncology - NVIDIA is a significant player, providing powerful GPUs essential for AI models in imaging and drug development [5] - Palantir's Foundry platform enables comprehensive data analysis across laboratories, enhancing research capabilities [5] - Google's DeepMind, IBM Watson Health, and Microsoft's AI initiatives are also shaping the competitive landscape in healthcare [6] Investment Opportunities - BioXcel Therapeutics utilizes its proprietary AI platform, NovareAI, to expedite drug development, with a long-term expected earnings growth rate of 37.9% and projected revenue growth of 71.2% by 2025 [8][9] - Tempus AI enhances precision medicine through its algorithmic test platform, with a long-term expected earnings growth rate of 42% and projected revenue growth of 78.4% by 2025 [10][12] - Quest Diagnostics is integrating AI to improve operational efficiency and has acquired PathAI Diagnostics to advance digital pathology innovations, with a long-term expected earnings growth rate of 8.2% [13][14]