Kura Oncology(KURA)2024-11-09 10:54Financial Data and Key Metrics Changes - Research and development expenses for Q3 2024 were 29.3 million in Q3 2023, primarily due to increased clinical trial costs related to ziftomenib and KO-2806 programs [31] - General and administrative expenses rose to 13.1 million in Q3 2023 [32] - Net loss for Q3 2024 was 38.6 million in Q3 2023, including non-cash share-based compensation of 455.3 million as of September 30, 2024, compared to $424 million as of December 31, 2023, expected to fund operations into 2027 [33] Business Line Data and Key Metrics Changes - The menin inhibitor program, led by ziftomenib, is positioned to transform treatment for menin-dependent AML, with ongoing trials showing promising safety and efficacy [6][17] - The Phase 1a dose escalation of the KOMET-007 study demonstrated ziftomenib's tolerability and activity in relapsed/refractory patients, with no dose-limiting toxicities reported [8][10] - The Phase 1b expansion study of ziftomenib is set to include multiple combination cohorts, aiming to redefine standards of care for newly diagnosed AML patients [13][14] Market Data and Key Metrics Changes - Ziftomenib is the first investigational therapy to receive breakthrough therapy designation for relapsed and refractory NPM1-mutant AML, which represents about 30% of new AML cases annually [17] - The company is also exploring ziftomenib's potential in treating advanced gastrointestinal stromal tumors (GIST), with promising preclinical data reported [20][22] Company Strategy and Development Direction - The company aims to establish ziftomenib as a cornerstone therapy for acute leukemias driven by the menin pathway, with plans to expand its use beyond AML to solid tumors and metabolic diseases [16][24] - The development of next-generation menin inhibitors targeting diabetes is underway, with a candidate expected to be nominated in the first half of 2025 [24] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the ongoing clinical trials and the potential for ziftomenib to provide significant clinical benefits, particularly in the frontline setting [52][54] - The company anticipates sharing updated data from the KOMET-007 trial at the ASH meeting in December 2024 and top-line results from the KOMET-001 trial in early 2025 [36] Other Important Information - The company welcomed Dr. Michael Vasconcelles to its Board of Directors, bringing extensive experience in oncology drug development [34][35] - The company is preparing for regulatory discussions regarding pivotal studies for ziftomenib, with a goal to start these studies in mid-2025 [72] Q&A Session Summary Question: How is the company thinking about using MRD negativity as part of frontline endpoints? - Management indicated that while survival is the primary endpoint, there may be an opportunity to use MRD negativity as a surrogate endpoint in future discussions with health authorities [39][40] Question: What are the key factors determining how long patients can stay on treatment? - Management highlighted the importance of intercepting patients early in their treatment journey to provide clinical benefits, which could significantly impact the commercial opportunity [52][54] Question: What should be expected in terms of benchmarking safety and efficacy at ASH? - Management noted that safety remains a focus, but they expect to gain key insights into efficacy as data matures [55][56] Question: What is the expected timeline for pivotal study initiation? - Management plans to kick off pivotal studies in mid-2025, with ongoing discussions with health authorities regarding trial design and endpoints [72] Question: What proportion of patients are considered adverse risk? - Management estimated that about 30% of patients fall into the adverse risk category, which includes older patients with complex cytogenetics [76] Question: Is there a mechanistic rationale for improved safety at higher doses? - Management explained that faster count recoveries at higher doses lead to reduced susceptibility to infections and other complications, contributing to improved safety [88][89]