Kura Oncology, Inc. · NASDAQ:KURA
Kura Oncology — ASCO 2025
14 slides from Kura Oncology's conference presentation, 2025
Slide 1: Ziftomenib in R/R NPM1-mutant AML: Phase 1b/2 Results from Pivotal KOMET-001 Study
#6506 Ziftomenib in Relapsed / Refractory (R/R) NPM1-mutant Acute Myeloid Leukemia (AML): Phase 1b/2 Clinical Activity and Safety Results from the Pivotal KOMET-001 Study Eunice S. Wang1, Pau Montesinos2, Ghayas C. Issa3, James M. Foran4, Harry Erba5, Eduardo Rodríguez-Arbolí6, Kateryna Fedorov7, Ma
Slide 2: Ziftomenib Targets the Menin Pathway, a Foundational Target in AML
Ziftomenib Targets the Menin Pathway, a Foundational Target in AML • NPM1-m drives leukemogenesis in ~30% of AML 1 • Despite current risk stratification, nearly half will have R/R disease within a year, after which outcomes are poor for high-risk patients with < 10% complete remission following vene
Slide 3: KOMET-001: Pivotal Trial of Ziftomenib Monotherapy in R/R NPM1-m AML
KOMET-001: Pivotal Trial of Ziftomenib Monotherapy in R/R NPM1-m AML Phase 1a Phase 1b Phase 1b Phase 2 Dose Escalation Validation Cohorts Expansion Registration-Enabling Completed Completed Completed Enrollment Completed Expansion of 600 mg QD 50 mg 100 mg 1000 mg Cohort 1: 200 mg QD 600 mg ~ ~ QD
Slide 4: KOMET-001: Efficacy & Safety Populations
KOMET-001 in R/R NPM1-m AML: Efficacy & Safety Populations 112 Enrolled and treated with ziftomenib RP2D 600 mg QD • Patients enrolled from January 26, 2023 to May 13, 2024 – 40 Sites, 7 countries 20 Phase 1b 92 Phase 2 (mITT) • Pooled analyses: 112 patients (92 Phase 2 + 20 Phase 1b) 20 Discontinue
Slide 5: Baseline Characteristics: R/R NPM1-m AML
Baseline Characteristics: R/R NPM1-m AML Ziftomenib RP2D 600 mg QD Ziftomenib RP2D 600 mg QD Phase 2 Pooled Phase 1b/2 Phase 2 Pooled Phase 1b/2 n (%) (N = 92) (N = 112) n (%) (N = 92) (N = 112) Median age, yrs (range) 69 (33–84) 69 (22–86) Co-mutations, n/Na 18–64 yrs 33 (36) 42 (38) FLT3-ITD 38/84
Slide 6: Response & Duration of Response: R/R NPM1-m AML
Response & Duration of Response: R/R NPM1-m AML Primary Phase 2 endpoint was met (P-value = 0.0058)* vs. 12% historical control rate1 Ziftomenib RP2D 600 mg QD Phase 2 Pooled Phase 1b/2 (N = 92) (N = 112) For Phase 2 patients, after a median n (%) CR/CRh 21 (23) 28 (25) follow-up of 4.1 months (rang
Slide 7: Comparable CR/CRh Across Pre-specified Subgroups: R/R NPM1-m AML
7 Comparable CR/CRh Across Pre-specified Subgroups: R/R NPM1-m AML • Comparable CR/CRh rates across pre-specified subgroups, regardless of prior HSCT, venetoclax, or FLT3/IDH co-mutations Data cutoff: Dec 20, 2024. PRESENTED BY: CR/CRh, complete remission with full or partial hematologic recovery; E
Slide 8: Transfusion Independence: R/R NPM1-m AML
8 Transfusion Independence: R/R NPM1-m AML Additional benefit beyond CR/CRh Ziftomenib RP2D 600 mg QD Phase 2 (N = 92) Total post-baseline transfusion independence Transfusion conversion (TD to TI) ratea, n/N (%) 17/82 (21) 95% CIb 13–31 Maintenance of transfusion independence (TI to TI) rate c, n/N
Slide 9: Overall Survival: R/R NPM1-m AML
Overall Survival: R/R NPM1-m AML All Patients (Pooled Phase 1b/2) Responders* vs. Non-Responders • Median OS: 6.1 months (95% CI, 3.8–8.4) • Note: 24 patients remained alive on-study, with 9 patients on-treatment *Responders included CRc, MLFS, and PR. PRESENTED BY: Data cutoff: Dec 20, 2024. CRc, c
Slide 10: Safety & Tolerability of Ziftomenib: Treatment-Emergent AEs
Safety & Tolerability of Ziftomenib in R/R NPM1-m AML (Safety Population) Treatment-Emergent AEs in ≥ 20% of All Patients Ziftomenib RP2D 600 mg QD Ziftomenib was well tolerated, with a safety Phase 2 Pooled Phase 1b/2 (N = 92) (N = 112) profile consistent with previous studies, 1,2 Event, n (%) Any
Slide 11: Safety & Tolerability of Ziftomenib: Related AEs
Safety & Tolerability of Ziftomenib in R/R NPM1-m AML (Safety Population) Ziftomenib-Related AEs in ≥ 5% of All Patients Ziftomenib RP2D 600 mg QD Ziftomenib was well tolerated, with a safety Phase 2 Pooled Phase 1b/2 (N = 92) (N = 112) profile consistent with previous studies, 1,2 Event, n (%) Any
Slide 12: Conclusions
Conclusions • In the pivotal KOMET-001 Phase 2 study, the primary endpoint was met − Ziftomenib achieved clinically meaningful, MRD-negative responses in this heavily pretreated R/R NPM1-m AML population − Similar response rates were seen, regardless of prior therapies, including HSCT and venetoclax
Slide 13: KOMET-017: Phase 3 Ziftomenib Pivotal 1L Combination Studies
KOMET-017: Phase 3 Ziftomenib Pivotal 1L Combination Studies • Two independently KOMET-017-NIC: Non-intensive therapy – Ziftomenib + venetoclax/azacitidine combo powered, registration- Patients Arm A Ziftomenib Primary endpoints enabling, randomized • NPM1-m AML R Venetoclax + Azacitidine • CR 1:1 P
Slide 14: Acknowledgements
Acknowledgements • The patients, their families, and their caregivers • KOMET-001 study investigators and their study teams • This study was sponsored by Kura Oncology, Inc. • Medical writing support was provided by Oxford PharmaGenesis Inc., with funding from Kura Oncology, Inc. Copies of this pres