Kura Oncology(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 145.8 million from the sale of 1,376,813 shares at 41.7 million, an increase of 42.2% compared to 18.2 million, up 38.3% from 117.7 million, a 42.3% increase from 52.3 million, an increase of 121% compared to 93.6 million from a public offering in June 2023, selling 5,660,871 shares at 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 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 134.8 million, an increase of 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 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 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].