February 5, 2025 ZIFTOMENIB PROGRAM UPDATES Our goal is to develop transformative therapies to extend and improve the lives of patients with cancer Exhibit 99.1
FORWARD-LOOKING STATEMENTS This presentation contains forward-looking statements. Such statements include, but are not limited to, statements regarding our research, preclinical and clinical development activities, plans and projected timelines for ziftomenib, KO-2806 and tipifarnib, plans regarding regulatory filings, our expectations regarding the relative benefits of our product candidates versus competitive therapies, and our expectations regarding the therapeutic and commercial potential of our product candidates. The words “believe,” “may,” “should,” “will,” “estimate,” “promise,” “plan”, “continue,” “anticipate,” “intend,” “expect,” “potential” and similar expressions (including the negative thereof) are intended to identify forward-looking statements. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Risks that contribute to the uncertain nature of the forward-looking statements include: our preclinical studies and clinical trials may not be successful; the U.S. Food and Drug Administration (FDA) may not agree with our interpretation of the data from clinical trials of our product candidates; we may decide, or the FDA may require us, to conduct additional clinical trials or to modify our ongoing clinical trials; we may experience delays in the commencement, enrollment, completion or analysis of clinical testing for our product candidates, or in the reporting of data from such clinical testing, or significant issues regarding the adequacy of our clinical trial designs or the execution of our clinical trials may arise, which could result in increased costs and delays, or limit our ability to obtain regulatory approval; our product candidates may not receive regulatory approval or be successfully commercialized; unexpected adverse side effects or inadequate therapeutic efficacy of our product candidates could delay or prevent regulatory approval or commercialization; and we may not be able to obtain additional financing. Additional risks and uncertainties may emerge from time to time, and it is not possible for Kura’s management to predict all risk factors and uncertainties. All forward-looking statements contained in this presentation speak only as of the date on which they were made. Other risks and uncertainties affecting us are described more fully in our filings with the Securities and Exchange Commission. We undertake no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made. This presentation also contains statistical data obtained from and prepared by third parties. The recipient is cautioned not to give undue weight to such disclosures. Neither the Company nor any other person makes any representation as to the accuracy or completeness of such data or undertakes any obligation to update such data after the date of this presentation.
KURA IS ADVANCING A ROBUST PIPELINE OF THERAPEUTIC PRODUCT CANDIDATES Ziftomenib: Potentially Best-in-Class Menin Inhibitor for AML Relapsed/refractory (R/R) and frontline acute myeloid leukemia (AML) market opportunity could exceed $7B per year in the U.S. Positive topline results from KOMET-001 study in R/R NPM1-m AML; NDA submission anticipated Q2 2025 Kyowa Kirin collaboration funds expansive AML development program through 1L U.S. commercialization Target indications include HNSCC, lung, colorectal, pancreatic and renal cell carcinomas Farnesyl Transferase Inhibitors (FTIs) in Large Solid Tumor Indications FTIs may overcome innate and adaptive resistance to PI3Ka inhibitors, KRAS inhibitors and tyrosine kinase inhibitors (TKIs) in certain indications Clinical data for KO-2806 and tipifarnib in combination expected in 2H 2025 Additional Therapeutic Opportunities for Menin Inhibitors Encouraging preclinical data for menin inhibitors in type 2 diabetes; development candidate nomination anticipated 1H 2025 Phase 1 study of ziftomenib + imatinib in gastrointestinal stromal tumors (GIST) expected to start in 1H 2025; additional potential $1B opportunity
AGENDA Positive FDA Feedback for Upcoming Frontline Combination Trial Designs Positive KOMET-001 Ziftomenib Monotherapy Trial in R/R NPM1-m AML 2025 Anticipated Milestones for Ziftomenib and Pipeline Programs
PREVALENCE OF ZIFTOMENIB-ELIGIBLE PATIENTS UP TO 50% OF AML PATIENTS MAY BENEFIT FROM MENIN INHIBITOR THERAPY AML, acute myeloid leukemia; KMT2Ar, lysine methyltransferase 2A rearrangement; NPM1-m, mutated nucleophosmin 1; NPM1m, nucleophosmin 1 mutation; FLT3m, FMS‐like tyrosine kinase 3 mutation; IDH1/2, mutations in isocitrate dehydrogenases types 1 and 2. 1. Papaemmanuil E et al. N Engl J Med. 2016;374(23):2209-2221. doi:10.1056/NEJMoa1516192 2. The Cancer Genome Atlas Research Network. N Engl J Med. 2013;368(22):2059-2074. doi:10.1056/NEJMoa1301689 3. Issa GC et al. Leukemia. 2021;35(9):2482-2495. doi:10.1038/s41375-021-01309-y 4. Candoni A, Coppola G. Hematol Rep. 2024;16(2):244-254. doi:10.3390/hematolrep16020024 5. Bertrums EJM et al. Haematologica. 2023;108(8):2044-2058. doi:10.3324/haematol.2022.281653 6. National Cancer Institute. Accessed October 16, 2024. https://seer.cancer.gov/seertools/hemelymph/ 51f6cf59e3e27c3994bd547d/ 7. National Cancer Institute. Accessed October 16, 2024. https://seer.cancer.gov/seertools/hemelymph/5a7e288d1ef557f9c8636d31/ 8. Burrows F et al. Poster presented at: AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics: Discovery, Biology, and Clinical Applications; October 26-30, 2017; Philadelphia, PA. 9. Falini B, Dillon R. Blood Cancer Discov. 2024;5(1):8-20. doi:10.1158/2643-3230.BCD-23-0144 AML is characterized by significant genetic heterogeneity due to driver mutations, including NPM1m, FLT3m, IDH1/2m and KMT2Ar 1-2 Up to 50% of AML cases may be menin-dependent, including those driven by NPM1m and KMT2Ar3-7 NPM1 mutations are observed in 30% to 35% of cases and are an important upstream driver mutation that uses the menin pathway8,9 10-15% 5-10% Up to 50% of AML 30% NPM1-m KMT2A-r Other mutations AML MENIN
AGENDA Positive FDA Feedback for Upcoming Frontline Combination Trial Designs Positive KOMET-001 Ziftomenib Monotherapy Trial in R/R NPM1-m AML 2025 Anticipated Milestones for Ziftomenib and Pipeline Programs
Phase 2 registrational trial in relapsed/refractory NPM1-m AML achieved its primary CR/CRh endpoint, and the primary endpoint was statistically significant The benefit-risk profile for ziftomenib is highly encouraging, and safety and tolerability were consistent with previous reports Topline data submitted for presentation at an upcoming medical meeting Pre-NDA meeting with FDA completed; NDA submission for ziftomenib on track for 2Q 2025 POSITIVE TOPLINE RESULTS FROM KOMET-001 REGISTRATIONAL TRIAL IN R/R NPM1-m AML R/R AML MENIN
ZIFTOMENIB MARKET POTENTIAL IN R/R AML R/R AML MENIN High Unmet Need in R/R NPM1-m AML Potential for Sustained Treatment Attractive Market Opportunity 20% are primary refractory; ~50% will relapse who achieved an initial CR1-5 20% ~50% Fewer than 10% of all patients with R/R AML are alive at 5 years6 <10% Combination of encouraging clinical activity and safety in a once-daily oral medication supports an attractive R/R opportunity AML, acute myeloid leukemia; CR, complete response. 1. Issa GC, et al. Blood Cancer J. 2021;11(9):162. 2. Bertoli S, et al. Blood. 2018;132(suppl 1):2802. 3. Hubmann M, et al. Haematologica. 2014;99(8):1317-1325. 4. SEER Cancer Stat Facts: Acute Myeloid Leukemia. National Cancer Institute. Bethesda, MD. Accessed March 14, 2023. https://seer.cancer.gov/statfacts/html/amyl.html. 5 Issa G, et al. Blood Adv. 2023;7(6):933-942. 6. DeWolf et. al, Blood 2020; 136 (9) 1023-1032. $36-40k /month Analog pricing, including for recently approved product $350-400M/yr Market opportunity in R/R NPM1-m AML NPM1-m patients eligible for treatment in R/R AML1 ~1,645 Potential for safe and well-tolerated, targeted Tx to support sustained treatment ~6 mo Duration of Treatment
AGENDA Positive FDA Feedback for Upcoming Frontline Combination Trial Designs Positive KOMET-001 Ziftomenib Monotherapy Trial in R/R NPM1-m AML 2025 Anticipated Milestones for Ziftomenib and Pipeline Programs
Single protocol encompassing two independent, randomized, placebo-controlled Phase 3 trials Single protocol designed to facilitate study start-up and execution Single protocol is attractive to sites as it provides treatment options to the broadest potential patient pool FDA aligned on endpoints, providing pathways for potential accelerated approval in both trials Continuing to work with global health authorities to gain alignment on protocol Both trials expected to initiate in 2H 2025 POSITIVE FDA FEEDBACK ON KOMET-017 REGISTRATIONAL TRIALS IN FRONTLINE INTENSIVE AND NON-INTENSIVE AML FRONTLINE AML MENIN MRD= Minimal Residual Disease
KOMET-017: TWO PHASE 3 TRIALS UNDER A SINGLE PROTOCOL Provides Treatment Options to the Broadest Frontline AML Patient Pool Ziftomenib Venetoclax + Azacitidine Placebo Venetoclax + Azacitidine Randomized Non-Intensive Therapy Trial NPM1-m CR OS KOMET-017-NIC (Non-Intensive Combination) 7+3 Induction Randomized Intensive Therapy Trial NPM1-m or KMT2A-r Arm C Arm A Arm B Ziftomenib Ziftomenib Consolidation 7+3 Induction Consolidation Placebo Placebo 7+3 Induction Consolidation CR 7+3 Induction Consolidation EFS MRD- KOMET-017-IC (Intensive Combination) Ziftomenib SOC Backbone Placebo 7+3 means seven days of cytarabine and 3 days of daunorubicin CR, complete response; OS, overall survival; CR MRD-, complete response with minimal residual disease; EFS, event-free survival FRONTLINE AML MENIN
Patient Population: AML with NPM1 mutation Age 75+ or < 75 with medical comorbidities NON-INTENSIVE CHEMOTHERAPY (NIC) TRIAL Randomized, Double-Blind, Placebo-Controlled Study: Venetoclax + Azacitidine + Ziftomenib or Placebo FRONTLINE AML MENIN Dual Primary Endpoints: Complete Response Overall Survival Ziftomenib Venetoclax + Azacitidine Placebo Ziftomenib SOC Backbone Placebo Venetoclax + Azacitidine Randomized Non-intensive Therapy Trial NPM1-m CR OS
Known myelosuppression with venetoclax + azacitidine from indicated dose and schedule has evolved current clinical practice Standard practice has moved towards minimizing venetoclax exposure upon response Proposed dose modifications for use of venetoclax are based on long-term follow-up exposure data from VIALE-A trial1 and real-world evidence (RWE) cohort results published by Othman et al 2024.2 FDA agreement to allow modified dosing to incorporate real world practice Cycles 1 and 2: venetoclax days 1-28 per label Cycles 3 and beyond: days 1-21 Further dose reductions for toxicity per label NON-INTENSIVE CHEMOTHERAPY (NIC) TRIAL FDA Aligned Around Modified Venetoclax Dosing Regimen FRONTLINE AML MENIN 1. Pratz KW, Jonas BA, Pullarkat V, et al. Long-term follow-up of VIALE-A: Venetoclax and azacitidine in chemotherapy-ineligible untreated acute myeloid leukemia. Am J Hematol 2024;99(4):615-624. 2. Othman J, Lam HPJ, Leong S, et al. Real-world outcomes of newly diagnosed AML treated with venetoclax and azacitidine or low-dose cytarabine in the UK NHS. Blood Neoplasia. 2024;1(3):2950-3280.
Proposed 3-Arm Design to Capture Maintenance Indication in the Label for Newly Diagnosed AML INTENSIVE CHEMOTHERAPY (IC) TRIAL Randomized, Double-Blind, Placebo-Controlled Study: 7+3/Consolidation + Ziftomenib or Placebo FRONTLINE AML MENIN Primary Endpoints: CRMRD- and EFS 7+3 Induction Randomized Intensive Therapy Trial NPM1-m or KMT2A-r Ziftomenib SOC Backbone Placebo Arm C Arm A Arm B Ziftomenib Ziftomenib Consolidation 7+3 Induction Consolidation Placebo Placebo 7+3 Induction Consolidation CR 7+3 Induction Consolidation EFS MRD- CR MRD- as potential accelerated approval endpoint with accompanying supportive/external evidence on correlation of CR MRD- and EFS/OS EFS powered to demonstrate additional benefit of post-consolidation maintenance
INTENSIVE CHEMOTHERAPY (IC) TRIAL Importance of the CRMRD- Endpoint FRONTLINE AML MENIN Published data suggest CR MRD- may correlate better with long-term survival than morphologic CR alone Literature highlights significant impact of NPM1-m MRD negativity on long-term survival and the implication on consolidation therapy choices Higher rates of CR MRD negativity with ziftomenib have potential to diminish the need for allogeneic stem cell transplantation Utilizing this endpoint in KOMET-017-IC: Provides potential to pave the way in the field to establish this new surrogate endpoint Othman J, et al. Postinduction molecular MRD identifies patients with NPM1 AML who benefit from allogeneic transplant in first remission. Blood (2024) 143 (19): 1931–1936. Figure 2.
Expansive Market Opportunity ZIFTOMENIB MARKET POTENTIAL IN NEWLY DIAGNOSED AML AML, acute myeloid leukemia; CR, complete response. 1. American Cancer Society. Updated June 5, 2024. Accessed August 27, 2024. https://www.cancer.org/cancer/types/acute-myeloid-leukemia/about/key-statistics.html 2. Kumar CC. Genes Cancer. 2011;2(2):95-107. doi:10.1177/1947601911408076 3. National Cancer Institute. Accessed August 27, 2024. https://seer.cancer.gov/statisticsnetwork/explorer/application. Potential for benefit / risk to support sustained treatment 12-24 months Analog pricing, including for recently approved product $36-40k months Potential peak sales for menin inhibitors in 1L AML >$7B/yr Combination of encouraging clinical activity and safety in a once-daily oral medication could unlock a large market opportunity Potential for benefit / risk to support sustained treatment 12-24 months Newly-diagnosed cases of AML each year in the U.S.1 ~20,800 Analog pricing, including for recently approved product $36-40k /month Large Population & Potential for Sustained Benefit High Unmet Medical Need of patients who achieve a first CR will relapse within 3 years2 ~70% 5-year survival rate for AML patients aged ≥ 65 years3 ~11% >$7B/yr Annual U.S. market opportunity in 1L AML FRONTLINE AML MENIN
AGENDA Positive FDA Feedback for Upcoming Frontline Combination Trial Designs Positive KOMET-001 Ziftomenib Monotherapy Trial in R/R NPM1-m AML 2025 Anticipated Milestones for Ziftomenib and Pipeline Programs
Ziftomenib ANTICIPATED UPCOMING MILESTONES: SEVERAL EXPECTED 2025 DATA READ-OUTS ACROSS MULTIPLE PROGRAMS Report topline results from KOMET-001 Phase 2 registration-directed trial in R/R NPM1-m AML ü FDA feedback on KOMET-017 registration-enabling protocol in 1L NPM1-m and KMT2A-r intensive and non-intensive AML ü NDA submission for ziftomenib in R/R NPM1-m AML 2Q 2025 Present topline data from KOMET-001 Phase 2 registration-directed trial in R/R NPM1-m AML 2Q 2025 Present preliminary clinical data from KOMET-007 Phase 1b trial in 1L intensive AML 2Q 2025 Initiate KOMET-015 Phase 1 trial of ziftomenib in combination with imatinib in patients with advanced GIST 1H 2025 Initiate KOMET-017 Phase 3 registration-enabling trials in 1L NPM1-m and KMT2A-r intensive and non-intensive AML 2H 2025 Present preliminary clinical data from Phase 1b expansion of KOMET-007 in 1L non-intensive AML 2H 2025 KO-2806 / tipifarnib Initiate one or more expansion cohorts in combination with cabozantinib in RCC 1H 2025 Present preliminary clinical data from FIT-001 trial for KO-2806 as monotherapy and combo with cabozantinib in RCC 2H 2025 Present full clinical data from KURRENT-HN trial of tipifarnib in combination with alpelisib in PIK3CA-m HNSCC 2H 2025 Next-gen Menin Nominate a development candidate for next-generation menin inhibitor program for diabetes 1H 2025
FINANCIAL HIGHLIGHTS (NASDAQ: KURA) Cash, Cash Equivalents and Marketable Securities Anticipated Significant Near-Term Milestones Shares Outstanding in pro forma cash as of September 30, 2024* Kura anticipates collaboration plus cash balance as of September 30, 2024 to fund ziftomenib AML program to potential commercialization in frontline combinations in potential near-term milestones, including payment upon NDA submission and launch of ziftomenib in the monotherapy R/R setting 24.5M options, RSUs, PSUs, warrants & pre-funded warrants as of September 30, 2024 * Includes $455.3M in cash, cash equivalents and short-term investments as of 9/30/24 and upfront payment of $330M from strategic collaboration with Kyowa Kirin $785.3M $420M 77.7M
February 2025 ZIFTOMENIB PROGRAM UPDATES Our goal is to develop transformative therapies to extend and improve the lives of patients with cancer