Kura Oncology(US:KURA)2024-11-07 21:15Clinical 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].