Core Insights - Kura Oncology is a clinical-stage biopharmaceutical company focused on precision medicines for cancer treatment [2] - The company has an investigational therapy, ziftomenib, which has received Breakthrough Therapy Designation from the FDA for relapsed/refractory NPM1-mutant acute myeloid leukemia [2] - Kura Oncology has entered a global strategic collaboration with Kyowa Kirin to develop and commercialize ziftomenib for AML and other hematologic malignancies [2] Company Pipeline - Ziftomenib is the first and only menin inhibitor to receive Breakthrough Therapy Designation for R/R NPM1-mutant AML [2] - Enrollment in a Phase 2 registration-directed trial of ziftomenib in R/R NPM1-mutant AML has been completed [2] - KO-2806, a next-generation farnesyl transferase inhibitor, is currently in a Phase 1 dose-escalation trial [2] - Tipifarnib is in a Phase 1/2 trial in combination with alpelisib for PIK3CA-dependent head and neck squamous cell carcinoma [2] Upcoming Events - Troy Wilson, CEO, will participate in three investor conferences in March 2025 [3] - The events include the TD Cowen Health Care Conference, Barclays Global Healthcare Conference, and Leerink Partners Global Biopharma Conference [3] - Live audio webcasts will be available on Kura's website, with archived replays following the events [1][3]

Core Insights - Kura Oncology, Inc. will report its fourth quarter and full year 2024 financial results on February 26, 2025, after U.S. market close [1] - A conference call and webcast will be held at 4:30 p.m. ET / 1:30 p.m. PT to discuss the financial results and provide a corporate update [1] Company Overview - Kura Oncology is a clinical-stage biopharmaceutical company focused on precision medicines for cancer treatment [3] - The company's pipeline includes small molecule drug candidates targeting cancer signaling pathways, with ziftomenib being a notable investigational therapy for relapsed/refractory NPM1-mutant acute myeloid leukemia [3] - Kura entered a global strategic collaboration with Kyowa Kirin Co., Ltd. to develop and commercialize ziftomenib for AML and other hematologic malignancies [3] - Enrollment in a Phase 2 registration-directed trial of ziftomenib for R/R NPM1-mutant AML has been completed [3] - Kura is also conducting clinical trials for ziftomenib in combination with current standards of care for newly diagnosed and R/R NPM1-mutant and KMT2A-rearranged AML [3] - KO-2806, a next-generation farnesyl transferase inhibitor, is in a Phase 1 dose-escalation trial [3] - Tipifarnib, another farnesyl transferase inhibitor, is currently in a Phase 1/2 trial in combination with alpelisib for PIK3CA-dependent head and neck squamous cell carcinoma [3]

Core Insights - Kura Oncology and Kyowa Kirin announced positive topline results from the KOMET-001 trial for ziftomenib in R/R NPM1-mutant AML, achieving the primary endpoint of complete response (CR) plus CR with partial hematological recovery (CRh) [1][4][5] - The companies are on track to submit a New Drug Application (NDA) for ziftomenib to the FDA in the second quarter of 2025 [1][5] - Two Phase 3 trials, KOMET-017-IC and KOMET-017-NIC, are expected to initiate in the second half of 2025 to evaluate ziftomenib in combination with different chemotherapy regimens [2][8] Company Developments - Kura and Kyowa Kirin's collaboration aims to commercialize ziftomenib, with plans for two registrational Phase 3 trials to support potential accelerated approval [2][8] - The KOMET-001 trial is the only investigational therapy to receive Breakthrough Therapy Designation (BTD) from the FDA for R/R NPM1-mutant AML [5][16] - Kura's management expressed confidence in ziftomenib's potential to transform treatment for AML patients, particularly given the positive FDA interactions [3][11] Clinical Trial Insights - The KOMET-017-IC trial will assess ziftomenib combined with intensive chemotherapy, while the KOMET-017-NIC trial will evaluate ziftomenib with venetoclax and azacitidine for patients unfit for intensive chemotherapy [9][10] - Both trials will have dual-primary endpoints to support potential U.S. accelerated approval and full approval [7][10] - The focus on achieving minimum residual disease (MRD) negative CR as a primary endpoint is seen as innovative and may expedite therapy availability [12][13] Market Context - AML is a challenging blood cancer with a high relapse rate, affecting approximately 20,000 Americans annually, with significant unmet medical needs in the treatment landscape [15] - The 5-year survival rate for AML is reported at 31.9%, highlighting the urgency for effective therapies [11][15] - Ziftomenib is positioned to address a substantial portion of AML patients, particularly those with NPM1 mutations [3][11]

Executive Leadership Changes - Mollie Leoni, MD, promoted to Chief Medical Officer after serving as Executive Vice President of Clinical Development and clinical lead for the ziftomenib program since 2020 [1] - Francis Burrows, PhD, promoted to Chief Scientific Officer after leading translational research efforts for the past nine years [1] - Stephen Dale, MD, steps down as Chief Medical Officer and Head of R&D to prioritize personal health, effective January 2, 2025 [1] Leadership Contributions and Expertise - Stephen Dale played a key role in advancing ziftomenib through a first registration-enabling study and laying the foundation for KO-2806 as a combination partner across solid tumor indications [2] - Mollie Leoni has a robust background in oncology and orphan drug development, previously leading clinical efforts for mogamulizumab at Kyowa Kirin, which achieved international registration for cutaneous T-cell lymphoma [2] - Francis Burrows has over 20 years of experience in drug discovery and development, focusing on cancer biology and small molecule drugs, and founded two biotech startups, including Conforma Therapeutics, acquired by Biogen in 2006 [3] Company Overview and Pipeline - Kura Oncology is a clinical-stage biopharmaceutical company focused on precision medicines for cancer treatment, with a pipeline of small molecule drug candidates targeting cancer signaling pathways [4] - Ziftomenib, a once-daily oral menin inhibitor, is the first investigational therapy to receive Breakthrough Therapy Designation from the FDA for relapsed/refractory NPM1-mutant AML [4] - Kura entered a global strategic collaboration with Kyowa Kirin in November 2024 to develop and commercialize ziftomenib for AML and other hematologic malignancies [4] - Enrollment in a Phase 2 registration-directed trial of ziftomenib in R/R NPM1-mutant AML has been completed, with a New Drug Application anticipated in 2025 [5] - KO-2806, a next-generation farnesyl transferase inhibitor, is in a Phase 1 dose-escalation trial as a monotherapy and in combination with targeted therapies [5] - Tipifarnib, a selective FTI, is in a Phase 1/2 trial in combination with alpelisib for PIK3CA-dependent head and neck squamous cell carcinoma [5]

Company Overview - Kura Oncology, Inc. is a clinically staged biopharma company established in 2014 and headquartered in San Diego, California [1] - The company focuses exclusively on the development of small-molecule drugs informed by patient selection biomarkers [1] Leadership and Background - The company has a co-founder with a Ph.D. in organic synthesis from Stanford University and experience working for major pharmaceutical companies like Merck [1] - The co-founder has also worked in biotech startups and was the first employee of 1200 Pharma, which garnered significant investment [1] Investment Focus - The company is noted for its focus on market trends, particularly in biotechnology stocks [1]

Financial Data and Key Metrics Changes - Research and development expenses for Q3 2024 were $41.7 million, up from $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 $18.2 million in Q3 2024 from $13.1 million in Q3 2023 [32] - Net loss for Q3 2024 was $54.4 million, compared to a net loss of $38.6 million in Q3 2023, including non-cash share-based compensation of $8.3 million [32] - Cash, cash equivalents, and short-term investments totaled $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]

Clinical Development and Efficacy - Ziftomenib demonstrated a complete remission (CR) rate of 35% (7/20) in patients with NPM1-mutant acute myeloid leukemia (AML) at the recommended Phase 2 dose of 600 mg[51]. - The overall response rate (ORR) for NPM1-mutant AML patients treated with ziftomenib at 600 mg was 45%[52]. - In the KOMET-007 study, newly diagnosed patients treated with ziftomenib and 7+3 achieved a CR rate of 100% (5/5)[60]. - Among relapsed or refractory patients treated with ziftomenib and venetoclax/azacitidine, the ORR was 53% (8/15)[60]. - Ziftomenib received Breakthrough Therapy Designation from the FDA for relapsed or refractory NPM1-mutant AML based on KOMET-001 trial data[56]. - The median duration of response for NPM1-mutant patients treated with ziftomenib was 8.2 months[52]. - The company plans to report topline results from the Phase 2 registration-directed portion of the KOMET-001 trial in early 2025[57]. - The company has initiated studies to evaluate ziftomenib in combination with current standards of care for AML[58]. - The company is supporting an ongoing investigator-sponsored study to evaluate ziftomenib as maintenance therapy following hematopoietic cell transplantation (HCT)[63]. - The Phase 1b expansion study of KOMET-007 is expected to enroll at least 20 patients per cohort, with ongoing dosing at 600 mg[61]. - In December 2023, the company announced a clinical collaboration with The Leukemia & Lymphoma Society to evaluate ziftomenib in pediatric patients with specific types of acute leukemia[64]. - The FDA cleared an investigational new drug application for ziftomenib for the treatment of advanced gastrointestinal stromal tumors (GIST) in combination with imatinib, with a proof-of-concept study expected to start in the first half of 2025[65]. Financial Performance and Funding - As of September 30, 2024, the company had cash, cash equivalents, and short-term investments totaling $455.3 million[77]. - In January 2024, the company completed a private placement, raising approximately $145.8 million from the sale of 1,376,813 shares at $17.25 per share[78]. - The company has not generated any revenues from product sales and has funded operations primarily through equity and debt financings[83]. - The company anticipates significant additional financing will be required in the future to continue funding operations[84]. - Research and development expenses for the three months ended September 30, 2024, were $41.7 million, an increase of 42.2% compared to $29.3 million in the same period of 2023[90]. - General and administrative expenses for the three months ended September 30, 2024, were $18.2 million, up 38.3% from $13.1 million in the same period of 2023[90]. - Total research and development expenses for the nine months ended September 30, 2024, reached $117.7 million, a 42.3% increase from $82.7 million in the same period of 2023[95]. - Ziftomenib-related costs for the nine months ended September 30, 2024, were $52.3 million, an increase of 121% compared to $23.7 million in the same period of 2023[95]. - The company raised approximately $93.6 million from a public offering in June 2023, selling 5,660,871 shares at $11.50 per share[97]. - As of September 30, 2024, the company had an accumulated deficit of $876.2 million[102]. - The company expects research and development expenses to increase in future periods as it continues clinical development activities for its product candidates[93]. - Other income, net for the three months ended September 30, 2024, increased to $5.5 million, up 41.5% from $3.9 million in the same period of 2023[90]. - The company has not sold any shares under the ATM Facility, which allows for an aggregate offering price of up to $150.0 million[100]. - The net cash used in operating activities for the nine months ended September 30, 2024, was $134.8 million, an increase of $44.3 million compared to $90.5 million in the same period of 2023[106]. - The net cash provided by financing activities for the nine months ended September 30, 2024, was $153.6 million, up from $94.1 million in the same period of 2023, primarily due to a private placement[108]. - The company has not generated any revenues from product sales and does not expect to do so until regulatory approval and commercialization of product candidates[104]. - The company anticipates needing substantial additional funding for ongoing operations, with potential financing through stock offerings, debt financings, or collaborations[105]. - The company has borrowed $10.0 million under a term loan facility, which requires principal and interest payments, with variable interest rates[109]. - The company may be required to pay up to approximately $80.0 million in milestone payments under in-license agreements if regulatory and commercial milestones are achieved[112]. - The company does not believe that a 10.0% change in interest rates would have a material effect on the fair value of its investment portfolio as of September 30, 2024[114]. - Inflation has not had a material effect on the company's business, financial condition, or results of operations during the periods presented[116]. Research and Development Initiatives - Preclinical data presented in June 2024 indicated that ziftomenib improved insulin sensitivity and production in a type 2 diabetes model, suggesting its potential as a therapeutic target[66]. - The company plans to nominate its first next-generation menin inhibitor drug candidate aimed at diabetes in the first half of 2025[67]. - The company is evaluating the safety and tolerability of KO-2806 in a Phase 1 trial, with the first patient dosed in October 2023[73].

Financial Performance - Net loss for Q3 2024 was $54.4 million, compared to a net loss of $38.6 million in Q3 2023, indicating an increase of 41%[10] - Research and development expenses for Q3 2024 were $41.7 million, up from $29.3 million in Q3 2023, representing a 42% increase[9] - General and administrative expenses for Q3 2024 were $18.2 million, compared to $13.1 million in Q3 2023, reflecting a 39% increase[9] - As of September 30, 2024, cash, cash equivalents, and short-term investments totaled $455.3 million, up from $424.0 million as of December 31, 2023[10] - The company expects to fund current operations into 2027 based on its operating plan[11] Clinical Development - Topline results from the registration-directed trial of ziftomenib in relapsed/refractory NPM1-mutant AML are expected in early 2025[1] - The Phase 1b expansion study of ziftomenib is currently enrolling at a dose of 600 mg across all cohorts, with 45 patients already enrolled since August 2024[5] - Kura plans to initiate a proof-of-concept study for ziftomenib in advanced gastrointestinal stromal tumors (GIST) in the first half of 2025[6] - The company aims to present updated data from the KOMET-007 trial at the ASH Annual Meeting in December 2024[12] - Kura has received Breakthrough Therapy Designation for ziftomenib, which targets approximately 30% of new AML cases annually[3]

Core Insights - Kura Oncology, Inc. will report its third quarter 2024 financial results on November 7, 2024, after U.S. market close [1] - A conference call and webcast will be held at 4:30 p.m. ET / 1:30 p.m. PT to discuss the financial results and provide a corporate update [1][2] Company Overview - Kura Oncology is a clinical-stage biopharmaceutical company focused on precision medicines for cancer treatment [3] - The company's pipeline includes small molecule drug candidates targeting cancer signaling pathways, with ziftomenib being a key candidate for relapsed/refractory NPM1-mutant acute myeloid leukemia (AML) [3] - Ziftomenib has received Breakthrough Therapy Designation and is currently in a Phase 2 registration-directed trial (KOMET-001) [3] - Kura is also evaluating KO-2806, a next-generation farnesyl transferase inhibitor, in a Phase 1 dose-escalation trial [3] - Tipifarnib is in a Phase 1/2 trial in combination with alpelisib for patients with PIK3CA-dependent head and neck squamous cell carcinoma [3]

Core Insights - Kura Oncology's menin inhibitor, ziftomenib, demonstrates strong and lasting antitumor activity when combined with imatinib in both imatinib-sensitive and resistant GIST models, indicating a promising therapeutic strategy for advanced gastrointestinal stromal tumors (GIST) [1][3][4] Group 1: Preclinical Data and Mechanism - Preclinical studies show that the combination of ziftomenib and imatinib significantly outperforms imatinib alone in patient-derived xenograft models of GIST [3] - The mechanism of action involves a synthetic lethal approach where ziftomenib targets an epigenetic vulnerability in GIST tumors, leading to reduced KIT expression and activity, and effectively silencing ERK and AKT/mTOR signaling pathways [3] Group 2: Clinical Development Plans - Kura Oncology plans to initiate a proof-of-concept study for ziftomenib in combination with imatinib in patients with advanced GIST who have failed imatinib treatment, expected to start in the first half of 2025 [1][4] - The FDA has cleared the Investigational New Drug (IND) application for ziftomenib, facilitating its clinical development for advanced GIST [4] Group 3: Background on GIST and Ziftomenib - GISTs are the most common form of sarcoma, primarily driven by KIT mutations, and while imatinib is the standard treatment, resistance often develops, necessitating new therapeutic options [5] - Ziftomenib is a selective oral menin inhibitor also being developed for acute myeloid leukemia (AML) and has received Breakthrough Therapy Designation from the FDA for this indication [6]