Full Press Release Details
June 18, 2025 2025 EHA ANALYST AND
INVESTOR EVENT 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, expectations regarding the relative benefits of our product candidates versus competitive therapies, expectations regarding the therapeutic and commercial potential of our product candidates, market opportunities and expectations
regarding our collaboration with Kyowa Kirin. 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; we may not be able
to obtain additional financing; and our collaboration with Kyowa Kirin may not be successful. 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 may also contain statistical, preclinical and clinical 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.
ZIFTOMENIB Targeted investigational
menin inhibitor for relapsed/refractory and newly diagnosed acute myeloid leukemia (AML) New Drug Application (NDA) based on positive results from the Phase 2 KOMET-001 trial NDA granted Priority Review and assigned Prescription Drug User Fee Act
(PDUFA) target action date of November 30, 2025 Kyowa Kirin partnership funds expansive AML development program through 1L U.S. commercialization
AGENDA Ziftomenib Combined with
Intensive Induction Chemotherapy (7+3) in Newly Diagnosed NPM1-m or KMT2A-r AML: Updated Phase 1a/b Results from KOMET-007 Unmet Need in Newly Diagnosed AML Ziftomenib Global Development Plan and KOMET-017 Phase 3 Clinical Trials Ziftomenib Market
Opportunity in Newly Diagnosed NPM1-m and KMT2A-r AML
KEY OPINION LEADERS AND INVITED
PARTICIPANTS Harry Erba, M.D., Ph.D. Director of Leukemia Program at the Duke Cancer Institute Ghayas C. Issa, M.D. Associate Professor of Leukemia at The University of Texas MD Anderson Cancer Center
UNMET NEED IN NEWLY DIAGNOSED AML
Ghayas C. Issa, M.D.
SIGNIFICANT UNMET NEED REMAINS FOR AML
PATIENTS AML, acute myeloid leukemia; CR, complete response. 1. National Cancer Institute. Accessed May 25, 2025. https://seer.cancer.gov/statfacts/html/amyl.html 2. Kumar CC. Genes Cancer. 2011;2(2):95-107. doi:10.1177/1947601911408076. ~ 70% Up to
70% of patients who achieve a first CR will see AML return within 3 years2 5-year survival rate for AML is 33% and as low as 8.6% for patients aged 65 years1 An estimated 22,000 new cases of AML diagnosed each year in the United States1
Median age at diagnosis is 69 years; majority of diagnoses made in patients aged 65 to 74 years.1 Current FDA approved therapies include combination chemotherapy regimens such as 7+3, venetoclax and hypomethylating agents (HMAs) and FLT3 inhibitors
like midostaurin or quizartinib 33%
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 KMT2Ar1-2 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
ZIFTOMENIB TARGETS THE MENIN PATHWAY,
A FOUNDATIONAL TARGET IN AML Inhibiting the menin-KMT2A complex downregulates HOXA9/MEIS1, leading to differentiation of leukemic blasts5 KMT2A (MLL) and NPM1 sit upstream from major AML targets (i.e., FLT3, BCL2 and IDH1/2)4 In ~35-40% of
AML, leukemogenesis is driven by NPM1 mutations or KMT2A rearrangements,1,2 which cause AML by blocking differentiation of blasts3 1. Papaemmanuil et al. N Engl J Med 2016; 375: 900-1; 2. Issa GC et al. Leukemia 2021;3:2482-95; 3. Collins and
Hess. Curr Opin Hematol 2016;23(4)354-61; 4. Matthews AH et al. Cancers (Basel) 2022 Nov 29;14(23):5906. 5. Thomas. Oncol Ther 2024;12(1):57-72; 6. Lu et al. Cancer Cell 2016;30(1):92-107; 7. Ferreira et al. Oncogene
2016;35(23):3079-82; 8. Jeong et al. Nat Genet 2014;46(1):17-23; 9. Wang et al. Blood 2005;106(1):254-64; 10. Chowdhury et al. EMBO Rep 2011;12(5):463-9; 11. Schmidt et al. Leukemia 2019;33(7):1608-19; 12. Xu et al. Cancer Cell
2016;30(6):863-78; 13. Brunetti et al. Cancer Cell 2018; 34(3):499-512; 14. Wang et al. Cancer Discov. 2023; 13(3):724-45. Ziftomenib Mechanism of Action3,4,6-14
S136 1Duke Cancer Institute,
Durham, NC, USA; 2Roswell Park Comprehensive Cancer Center, Buffalo, NY, USA; 3Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA; 4Weill Cornell Medicine and The New York Presbyterian Hospital, New York, NY, USA; 5University of
Texas Southwestern Medical Center, Dallas, TX, USA; 6SCRI at TriStar Centennial, Nashville, TN, USA; 7Karmanos Cancer Institute, Wayne State University, Detroit, MI, USA; 8Moores Cancer Center, University of California, San Diego, La Jolla, CA, USA;
9Abramson Cancer Center, University of Pennsylvania, Philadelphia, PA, USA; 10Cleveland Clinic, Cleveland, OH, USA; 11Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine, Baltimore, MD, USA; 12Robert H. Lurie
Comprehensive Cancer Center, Northwestern University, Chicago, IL, USA; 13Colorado Blood Cancer Institute, Denver, CO, USA; 14Chao Family Comprehensive Cancer Center, University of California Irvine Health, Orange, CA, USA; 15Georgia Cancer Center,
Augusta, GA, USA; 16Department of Hematology, University of Minnesota, Minneapolis, MN, USA; 17Ochsner MD Anderson Cancer Center, New Orleans, LA, USA; 18The Ohio State University Comprehensive Cancer Center, Columbus, OH, USA; 19Tisch Cancer
Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA; 20Rutgers Cancer Institute of New Jersey, New Brunswick, NJ, USA; 21Carbone Cancer Center, University of Wisconsin-Madison, Madison, WI, USA; 22David Geffen School of Medicine at
UCLA, Los Angeles, CA, USA; 23The University of Kansas Cancer Center, Kansas City, KS, USA; 24University of Oklahoma Health Sciences Center, Stephenson Cancer Center, Oklahoma, OK, USA; 25Stony Brook University Hospital Cancer Center, Stony Brook,
NY, USA; 26Mayo Clinic, Jacksonville, FL, USA; 27Kura Oncology, Inc., San Diego, CA, USA; 28Yale University and Yale Cancer Center, New Haven, CT, USA Ziftomenib combined with intensive induction chemotherapy (7+3) in newly diagnosed NPM1-m or
KMT2A-r acute myeloid leukemia: Updated phase 1a/b results from KOMET-007 Harry Erba1, Eunice S. Wang2, Amir T. Fathi3, Gail J. Roboz4, Yazan F. Madanat5, Stephen A. Strickland6, Suresh Balasubramanian7, James K. Mangan8, Keith Pratz9, Anjali
Advani10, Ivana Gojo11, Jessica K. Altman12, Marcello Rotta13, Kiran Naqvi14, Jorge Cortes15, Mark Juckett16, Leonard C. Alsfeld17, James S. Blachly18, Marina Kremyanskaya19, Neil Palmisiano20, Kalyan V. Nadiminti21, Gary Schiller22, Tara L. Lin23,
Mohamad Khawandanah24, Michael W. Schuster25, Talha Badar26, Julie Mackey Ahsan27, Tianle Chen27, Marcie Riches27, Daniel Corum27, Mollie Leoni27, and Amer M. Zeidan28
KOMET-007: ONGOING COMBINATION
TRIAL OF ZIFTOMENIB IN NEWLY DIAGNOSED AML Ziftomenib started on Cycle 1 Day 8 and administered continuously thereafter. Cytarabine administered on Cycle 1 Days 1-7; daunorubicin on Cycle 1 Days 1-3; re-induction cycles allowed based on
bone marrow biopsy results Here we present updated safety and clinical activity in all newly diagnosed AML patients treated at the ziftomenib RP2D of 600 mg QD in combination with standard doses of 7+3 across phase 1a/b Ziftomenib / 7+3 Combination
Adult AML with NPM1-m or KMT2A-r enroll independently Newly Diagnosed AML (N=82) Phase 1a: High-Riska AML Dose Escalation Dose Selection Ziftomenib / 7+3 Dose level 1: 100 mg Dose level 3: 600 mg Dose level 2: 400 mg Dose level 1: 200 mg
Phase 1b: All IC-eligible AML Dose Expansion / RP2D Determination Ziftomenib / 7+3 Selected dose from Phase 1a Validated RP2D RP2D Validation: Endpoints: Primary AEs DLTs (Phase 1a) CR Secondary CRcb ORRc DoR aHigh-risk is defined as KMT2A-r AML, or
NPM1-m with adverse-risk cytogenetics per ELN criteria, age 60 yrs and/or treatment-related AML regardless of age. bCR, CRh, or CRi. cCRc or MLFS. AE, adverse event; CR / CRh / CRi, complete remission with full / partial / incomplete
hematologic recovery; CRc, composite complete remission; DLT, dose limiting toxicity; DoR, duration of remission; IC, intensive chemotherapy; MLFS, morphologic leukemia-free state; QD, once daily; ORR, objective response rate; RP2D, recommended
KOMET-007 in 1L AML: SAFETY AND
EFFICACY POPULATIONS 82 Enrolled and treated with Ziftomenib 600 mg / 7+3 combination 49 NPM1-m 44 NPM1-m 30 On-treatmentb 2 Received HSCT 14 Discontinued 3 Relapsed/Refractory 1 Withdrew 2 Deathc 8 Otherd 33 KMT2A-r 27 KMT2A-r 19 On-treatmentb 6
Received HSCT, with 1 on ziftomenib maintenance 8 Discontinued 1 AE 3 Relapsed/Refractory 3 Deatha,c 1 Otherd ITT (Safety) Population: Response-Evaluablea (Efficacy) Population: aPatients who had 1 response assessment or who had died - one
KMT2A-r patient had no response assessment and had died before the data cut (not evaluable). bPatients who had not discontinued ziftomenib as of the data cutoff date. cDeaths included: NPM1-m: ischemic enteritis (n=1), cerebral hemorrhage (n=1);
KMT2A-r: bowel perforation (n=1), angioinvasive mucormycosis (n=1), sepsis (n=1). dOther reasons included: NPM1-m: physician decision (n=3), completed planned therapy (n=1), joint pain (n=1), planned for other maintenance study (n=2), patient
decision (n=1); KMT2A-r: physician decision (n=1). Data cutoff: Mar 21, 2025. AE, adverse event; ITT, intention-to-treat; HSCT, hematopoietic stem cell transplant. 6 KMT2A-r - no response assessment as of data cut 5 NPM1-m - no response
assessment as of data cut
BASELINE CHARACTERISTICS AND
DISPOSITION: 1L AML (N=82) Data cutoff: Mar 21, 2025. ECOG PS, Eastern Cooperative Oncology Group performance status; ITD, internal tandem duplication. NPM1-m KMT2A-r All Patients 600 mg (n=49) 600 mg (n=33) 600 mg (N=82) Median age, years (range)
60 (30-71) 43 (18-70) 56 (18-71) Female, n (%) 25 (51) 18 (55) 43 (52) Race, n (%) White Non-White 35 (71) 14 (29) 20 (61) 13 (39) 55 (67) 27 (33) ECOG PS 0-1, n (%) 43 (88) 31 (94) 74 (90) Co-mutations, n (%) FLT3 IDH1/2 6
(12)a 13 (27) 5 (15)a 2 (6) 11 (13)a 15 (18) Therapy-related AML, n (%) 2 (4) 8 (24) 10 (12) Patients on-treatment, n (%) 35 (71) 25 (76) 60 (73) Patients on-studyb, n (%) 47 (96) 29 (88) 76 (93) Median follow-up, weeks (range) 24.9 (4.3-47.1)
15.7 (1.1-40.3) 18.4 (1.1-47.1) aFLT3-ITD allelic ratio <0.05 (3 NPM1-m) or considered ineligible for FLT3 inhibitor (3 NPM1-m, 5 KMT2A-r). bPatients on-treatment or in long-term follow-up.
SAFETY AND TOLERABILITY OF
ZIFTOMENIB IN COMBINATION WITH 7+3 in 1L AML (N=82) TEAEs in 25% of All Patients Data cutoff: Mar 21, 2025. 1. Lin et al. Blood Adv. 2021;5(6):1719-28. TEAE, treatment-emergent adverse event. Ziftomenib safety profile in combination with
intensive chemotherapy was similar to that reported for newly diagnosed AML patients treated with 7+3 alone1 n (%) NPM1-m KMT2A-r All Patients 600 mg (n=49) 600 mg (n=33) 600 mg (N=82) Any Grade 46 (94) 31 (94) 77 (94) Febrile neutropenia 26 (53) 23
(70) 49 (60) Diarrhea 22 (45) 17 (52) 39 (48) Platelet count decreased 24 (49) 13 (39) 37 (45) Pruritus 19 (39) 13 (39) 32 (39) Nausea 18 (37) 8 (24) 26 (32) Hypokalemia 16 (33) 10 (30) 26 (32) Anemia 16 (33) 8 (24) 24 (29) Stomatitis 12 (24) 12
(36) 24 (29) Alanine aminotransferase increased 13 (27) 9 (27) 22 (27) Constipation 15 (31) 6 (18) 21 (26)
SAFETY AND TOLERABILITY OF
ZIFTOMENIB IN COMBINATION WITH 7+3 in 1L AML (N=82) Grade 3 TEAEs in 10% of All Patients n (%) NPM1-m KMT2A-r All Patients 600 mg (n=49) 600 mg (n=33) 600 mg (N=82) Grade 3 42 (86) 29 (88) 71 (87) Febrile neutropenia 25 (51) 20
(61) 45 (55) Platelet count decreased 23 (47) 12 (36) 35 (43) Anemia 16 (33) 8 (24) 24 (29) Neutrophil count decreased 14 (29) 6 (18) 20 (24) White blood cell count decreased 10 (20) 7 (21) 17 (21) Sepsis 8 (16) 5 (15) 13 (16) Lymphocyte count
decreased 5 (10) 4 (12) 9 (11) *Both patients were on other medications (posaconazole and/or piperacillin/tazobactam) at time of QT assessment. Data cutoff: Mar 21, 2025. QTc, QT corrected; TEAE, treatment-emergent adverse event. Grade 3
Ziftomenib-related Adverse Events of Interest 29 Patients (35%) had Grade 3 ziftomenib-related adverse events: Most common ( 10%) were febrile neutropenia (15%), decreased platelet count (15%), anemia (11%), and decreased neutrophil
count (11%) 1 case of differentiation syndrome (KMT2A-r, Gr3), which was successfully managed 2 cases of investigator-assessed QTc prolongation (both KMT2A-r, Gr3)*
CLINICAL ACTIVITY IN ALL
RESPONSE-EVALUABLEa 1L PATIENTS (N=71) a Patients who had 1 response assessment or who had died. b Among evaluable responders tested for MRD per local assay (NGS, RT-qPCR, FISH, flow cytometry). Preliminary central testing also shows
concordance with local MRD-negative rates. Data cutoff: Mar 21, 2025. Per ELN 2022: CR / CRh / CRi, complete remission with full / partial / incomplete hematologic recovery; CRc, composite complete remission; FISH, fluorescence in situ
hybridization; MLFS, morphologic leukemia-free state; MRD, measurable residual disease; NE, not evaluable; NGS, next-generation sequencing; NR, no response; ORR, objective response rate; PR, partial remission; RT-qPCR, quantitative reverse
transcription polymerase chain reaction. n (%) NPM1-m KMT2A-r All Patients 600 mg (n=44) 600 mg (n=27) 600 mg (N=71) CRc 41 (93) 24 (89) 65 (92) ORR CR CRh CRi MLFS PR NR NE 43 (98) 37 (84) 1 (2) 3 (7) 2 (5) 0 1 (2) 0 24 (89) 20 (74) 0 4 (15) 0 0 2
(7) 1 (4) 67 (94) 57 (80) 1 (1) 7 (10) 2 (3) 0 3 (4) 1 (1) CR MRD-negativity, n/N (%)b CRc MRD-negativity, n/N (%)b 24/34 (71) 26/38 (68) 14/16 (88) 15/18 (83) 38/50 (76) 41/56 (73) Median time to CR MRD-negativity, weeks (range) Median time to CRc
MRD-negativity, weeks (range) 4.7 (2-17) 4.7 (2-17) 4.4 (3-12) 4.1 (3-12) 4.5 (2-17) 4.3 (2-17)
DURATION OF TREATMENT &
PRELIMINARY CLINICAL OUTCOMES IN NPM1-m 1L AML Data cutoff: Mar 21, 2025. aAmong response-evaluable patients. bPatients on-treatment or in long-term follow-up. CR / CRh / CRi, complete remission with full / partial / incomplete hematologic recovery;
HSCT, hematopoietic stem cell transplant; MLFS, morphologic leukemia-free state OS, overall survival. For NPM1-m, after a median follow-up of 24.9 weeks (range 4.3-47.1): Median duration of CR was not reacheda Median OS was not reacheda 2
NPM1-m patients received HSCT 3 Discontinuations due to relapse 96% (47/49) of patients remained alive and continued on-studyb Duration of Treatment (weeks)