Kura Oncology Inc 2024年度报告

BUILT FOR PATIENTS Kura is dedicated to helping patients with cancer lead better, longerlivesthrough the discovery and development of targeted smallmolecule drug candidates for disease indications of unmet medicalneed. Our patient-first approach guides every decision as we builduponextensive preclinical and clinical rationale to develop newprecision medicines with the goal of addressing patient needs andoffering improvements to safety, tolerability and efficacy. BUILDING FOR THE FUTURE FARNESYL TRANSFERASEINHIBITORS (FTIS) TOADDRESS RESISTANCEIN LARGE SOLID TUMORINDICATIONS EXPANSION OPPORTUNITYFOR MENIN INHIBITORSINTO SOLID TUMORS ANDMETABOLIC DISEASES ZIFTOMENIB, MENININHIBITOR: POTENTIAL TOADDRESS UNMET NEEDSACROSS THE CONTINUUMOF CARE IN AML Ziftomenib offers potentialto shift the treatmentparadigm in gastrointestinalstromal tumors (GIST),an indication wheretherapeutic approachesare limited to targetedKIT inhibition via tyrosinekinase inhibitors (TKIs).Despite currently availabletherapies, resistanceremains a challenge. Thepotential market in GISTrepresents an additionalopportunity up to $1B inthe United States. While targeted therapiesoffer clinical benefit in awide range of indications,innate and adaptiveresistance mechanismsremain a challenge formany classes of targetedtherapies in cancer. FTIsoffer potential as idealcombination agents toovercome resistance inlarge solid tumor indications,including renal cellcarcinomas. Relapsed/refractoryNPM1-m settingrepresents the first stepin Kura’s developmentof menin inhibitors forthe treatment of acutemyeloid leukemia (AML)patients. Unprecedenteddevelopment andregulatory pathways forfrontline (1L) AML treatmentset the stage for ziftomenib’simpact across the treatmentcontinuum. Relapsed/refractory and 1L AMLmarket opportunities couldexceed $7B per year in theUnited States. BUILT FOR OPPORTUNITY GLOBAL STRATEGIC COLLABORATION WITH KYOWA KIRIN ALLOWS KURA TO REALIZE THE EXPANSIVEOPPORTUNITY FOR ZIFTOMENIB AND OTHER PIPELINE PROGRAMS Kura is well-capitalized and resourced to prepare for potential first approval for ziftomenib in adultpatients with relapsed or refractoryNPM1-mutant AML and execution of a successful launch. Kura retains leadership and key strategic rights to ziftomenib in the U.S. under a 50/50 profit share andwill book all U.S. sales. Kura retains pipeline programs, which we expect to be funded through key value inflection points in2025-2026, including next-generation menin inhibitors targeting oncology, diabetes and other metabolicdiseases and farnesyl transferase inhibitor combinations. Collaboration withKyowa Kirin pluscash balance as ofDecember 31, 2024anticipatedto fund ziftomenibAML programto potentialcommercialization infrontline combinations $420M $1.2B $727.4M in potential near-term milestones,including paymentsupon NDA submissionand launch ofziftomenib in therelapsed/refractorymonotherapy setting total in potentialdevelopment,regulatory andcommercial milestonepayments in cash, cashequivalents and short-term investments as ofDecember 31, 2024* *Includes $727.4M in cash, cash equivalents and short-term investments as of 12/31/24, including the upfront payment of $330M fromstrategic collaboration with Kyowa Kirin BUILT FOR LONG-TERM PIPELINE GROWTH INVESTIGATING ZIFTOMENIB ACROSS THE AML IN UP TO 50% OF PATIENTS KO-2806 AND TIPIFARNIB UNDER CLINICAL INVESTIGATION TO ADDRESS INNATE ANDADAPTIVE RESISTANCE MECHANISMS TO VARIOUS CLASSES OF TARGETED THERAPIES Tipifarnib in combination with alpelisib inrelapsed/metastatic PIK3CA-dependenthead and neck squamous cell carcinoma(HNSCC) KO-2806 as a monotherapy in solid tumorsthat carry an HRAS or KRAS mutation KO-2806 in combination with cabozantinibin clear cell renal cell carcinoma (ccRCC) KO-2806 in combination with adagrasibin KRASG12C-mutated non-small cell lungcancer (NSCLC) BUILT TO DELIVER ZIFTOMENIB POISED TO ADDRESS A SIGNIFICANT UNMET NEED IN RELAPSED/REFRACTORY(R/R)NPM1-MUTANT AML KOMET-001 registrational trial achieved primary CR/CRh endpoint, the benefit-risk profile for ziftomenib ishighly encouraging, and safety and tolerability were consistent with previous reports Full data analyses have been submitted for presentation at 2025 ASCO Annual Meeting Facilitated by the Breakthrough Therapy Designation status of ziftomenib in R/RNPM1-mutant AML,the pre-NDA meeting with FDA was completed and submission of the new drug application (NDA)was announced in the second quarter of 2025 High Unmet Need in R/RNPM1-m AML 20%are primary refractory;~50%will relapse who achievedan initial CR1-4 Fewer than10%of all patients withR/R AML are aliveat 5 years5 TO OUR SHAREHOLDERS: At Kura, our guiding principle is simple: “We work as one for patients.” This commitmentshapes every decision, no matter how challenging, and we’re dedicated to sharingour insights with you. By putting patients first, we believe we can deliver substantialvalue to them and to you, our shareholde