Komzifti (ziftomenib) / Kura Oncology, University of Michigan, Kyowa Kirin - LARVOL DELTA

Co-Mutational Patterns and Transcriptomic Heterogeneity Within NPM1-Mutated Acute Myeloid Leukemia: An Integrative Analysis Using the ASH HematOmics Program (SOHO 2026) - "Two menin inhibitors, revumenib and ziftomenib, were recently FDA-approved and work by blocking the HOX/MEIS1 pathway that drives NPM1-mutated AML. NPM1 co-mutation subgroups exhibit distinct transcriptomic profiles and pathway enrichment patterns, suggesting biologically meaningful heterogeneity beyond mutational classification alone. The enrichment of inflammatory signaling pathways in the NPM1/FLT3/DNMT3A subgroup and heme metabolism in the NPM1/IDH subgroup may have implications for therapeutic targeting. Prospective validation with clinical outcome data is needed to determine whether these transcriptomic differences carry prognostic or predictive significance and can inform trial stratification or treatment selection."

Heterogeneity • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • ASXL1 • BCL6 • BCOR • DNMT3A • EZH2 • FLT3 • IDH1 • IDH2 • IFNG • IL6 • MEIS1 • NPM1 • SF3B1 • SRSF2 • STAG2 • TET2 • TNFA • U2AF1 • ZRSR2

Ziftomenib in combination with venetoclax and azacitidine in relapsed/refractory NPM1-m or KMT2A-r acute myeloid leukemia: Updated phase 1a/b safety and clinical activity results from KOMET-007 (ASH 2025) - P1 | "In the ongoing KOMET-007 study, ziftomenib RP2D of 600 mg QD + Ven/Aza was welltolerated with robust clinical activity in patients with R/R NPM1-m or KMT2A-r AML. No ziftomenib-relatedQTc prolongation was reported. One case of DS (NPM1-m, Gr 3) successfully resolved with protocol-specified mitigation."

Clinical • Combination therapy • P1 data • Acute Myelogenous Leukemia • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Leukemia • Neutropenia • FLT3 • KMT2A • NPM1

Exposure-Response Analysis of Ziftomenib Combined With Venetoclax/Azacitidine or Cytarabine/Daunorubicin in Newly Diagnosed and Relapsed/Refractory NPM1-M or KMT2A-R Acute Myeloid Leukemia (SOHO 2026) - P1 | "No significant ER relationship for efficacy or safety was observed across ziftomenib doses of 200–600 mg QD combined with ven/aza or 7+3 in NPM1-m or KMT2A-r patients, demonstrating a wide therapeutic margin for ziftomenib. The lack of clinically meaningful drug-drug interaction supports coadministration of the combination agents without dose adjustments. Taken together with overall safety, efficacy, PK, and pharmacodynamic data, these findings support ziftomenib 600 mg QD as the optimal dose in combination with standard-of-care ven/aza or 7+3, providing the best benefit/risk outcomes in NPM1-m or KMT2A-r patients."

Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • KMT2A • NPM1

Resistance mechanisms and therapeutic strategies after menin inhibitor failure in acute myeloid leukemia. (PubMed, Expert Rev Hematol) - "Menin inhibitors, including the FDA-approved agents revumenib and ziftomenib, have emerged as transformative targeted therapies in acute myeloid leukemia (AML), particularly in KMT2A-rearranged (KMT2Ar) and NPM1-mutated (NPM1m) disease. No clinical tests are currently available to reliably predict emerging resistance or early therapeutic failure, and treatment options after menin inhibitor failure remain limited and largely empiric, including, if not previously used, venetoclax-based regimens, mutation-directed therapies, and intensive chemotherapy as a bridge to allogeneic HSCT. Emerging preclinical data identify key vulnerabilities, including bypass signaling, apoptotic dependence, and epigenetic escape, providing a rationale for next-generation combination strategies, ideally in the frontline setting to maximize the chance of success and minimize the risk of clonal escape."

Journal • Review • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Leukemia • Oncology • Transplantation • KMT2A • NPM1

Preliminary data from the ongoing Phase 1 study of the menin-MLL inhibitor enzomenib (DSP-5336) in combination with venetoclax and azacitidine in patients with relapsed or refractory Acute Myeloid Leukemia (ASH 2025) - P1/2 | "Median age was 50 yrs (21-76), 56% were female andmedian prior regimens was 2 (1-4); 3 pts (16.7 %) had prior allogeneic stem cell transplant (SCT), 6 pts(33.3%) had prior VEN, and 5 pts (27.8%) received prior menin inhibitor (2 ziftomenib, 1 revumenib, 2enzomenib). Preliminary data show ENZO up to 300 mg BID to be well tolerated in combination withVEN/AZA with no DLTs in 18 pts with R/R KMT2Ar or NPM1m AML. No QT prolongation was reported andthere was 1 report of non-serious DS. Promising preliminary clinical activity has been observed,particularly in pts without prior VEN or menin exposure (100% ORR and 67% CRc rate)."

Clinical • Combination therapy • P1 data • Acute Myelogenous Leukemia • Central Nervous System Leukemia • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Leukopenia • Neutropenia • Septic Shock • Thrombocytopenia • FLT3 • KMT2A • NPM1

Identifying novel ’druggable’ targets via Npm1A-turboid fusion and mass spectrometry to overcome genetic or adaptive resistance to menin inhibitors in mtNPM1 AML (ASH 2025) - "Treatment with revumenib may also cause emergenceof hot spot mutations in menin (e.g., S160T, M327V, M327I, G331D, G331R, and T349M) exhibitingreduced affinity to MI-binding...OCI-AML3 MEN1-M327I cells were resistant toSNDX-50469, ziftomenib, and DS1594b, but sensitive to the second-generation MI, bleximenib, aspreviously reported.To identify novel 'druggable' targets in mtNpm1 AML cells either sensitive or resistant to MI, we knockedin TurboID by CRISPR/Cas9 into the C-terminus of the mtNpm1 gene in OCI-AML3 cells...When combined with a BET inhibitor (pelabresib) ora novel dual BET/HAT inhibitor (NEO2734/EP31670), both RocA and talazoparib induced synergisticlethality in the sensitive OCI-AML3 and OCI-AML2-Npm1A KI, as well as the MI-resistant (OCI-AML3-Menin-M327I or the OCI-AML3 MITR) cells...Exvivo treatment with EP31670 and RocA or talazoparib induced synergistic loss of viability in MI (SNDX-50469)-resistant, patient-derived (N = 3) mtNpm1 AML cells. In the..."

IO biomarker • Acute Myelogenous Leukemia • AURKA • CDK9 • CDKN1A • EIF4A1 • EIF4A2 • FLT3 • HOXA9 • IL7R • IRAK4 • KMT2A • MEIS1 • MEN1 • MYC • NPM1 • PLK1 • S100A8 • SF3B1 • SMARCA2 • TP53

Menin Inhibitors in Acute Myeloid Leukemia: Clinical Integration, Resistance, and the Path Beyond Monotherapy. (PubMed, Cancers (Basel)) - "We explore data from the landmark AUGMENT-101 and KOMET-001 trials, which have led to regulatory approval of revumenib and ziftomenib for select patients...We highlight resistance mechanisms that have emerged from the use of contemporary menin inhibitors and efforts aimed at addressing this resistance, with a focus on emerging clinical data on enzomenib and bleximenib. Finally, we discuss prospects on the putative role of menin inhibitors in the measurable residual disease (MRD)-positive and maintenance settings in AML. Menin inhibitors have successfully transitioned from biological proof-of-concept to approved targeted therapy, and future advances will depend on rational front line combination strategies, MRD-guided treatment, post-transplant maintenance strategies, and therapeutic sequencing informed by mechanisms of resistance."

Journal • Monotherapy • Review • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • Transplantation • HOXA9 • KMT2A • NPM1

ZIFTOMENIB IN RELAPSED/REFRACTORY NPM1-MUTANT ACUTE MYELOID LEUKEMIA: PHASE 1B/2 CLINICAL ACTIVITY AND SAFETY RESULTS FROM THE PIVOTAL KOMET-001 STUDY (EHA 2025) - P1/2 | "In the pivotal KOMET-001 study, the phase 2 primary endpoint was met. Ziftomenib achieved deep and durable responses in R/R NPM1-m AML, with comparable clinical activity regardless of prior venetoclax exposure. Ziftomenib was well tolerated with limited myelosuppression and only 3% ziftomenib-related discontinuations."

Clinical • P1/2 data • Acute Myelogenous Leukemia • Anemia • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Leukemia • Neutropenia • Oncology • Thrombocytopenia • KMT2A • NPM1

Efficacy and Differentiation Syndrome With Menin Inhibitors in Relapsed/Refractory KMT2A Rearranged or NPM1-Mutated Acute Leukemia: An Exploratory Proportional Meta-Analysis (SOHO 2026) - "Although early clinical studies of revumenib and ziftomenib have shown encouraging activity, comprehensive pooled estimates of remission rates and differentiation syndrome incidence remain limited. Menin inhibitors demonstrated consistent CR/CRh rates of approximately 23% across studies, with differentiation syndrome occurring in roughly one-quarter of treated patients and grade ≥3 events in approximately 15%. These findings support menin inhibition as a reproducibly active therapeutic strategy in R/R KMT2A-rearranged and NPM1-mutated acute leukemias, while underscoring the need for vigilant recognition and management of differentiation syndrome. AML: acute myeloid leukemia, CI: confidence interval, CR: complete remission, CRh: complete remission with partial hematologic recovery, HOX/MEIS1: hexose oxidase/meis homeobox 1, KMT2A: lysine methyltransferase 2A, NPM1: nucleophosmin 1, ORR: overall response rate."

Retrospective data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • KMT2A • MEIS1 • NPM1

Postmarketing Safety Signals of Revumenib (Menin-KMT2A Inhibitor) vs Acute Myeloid Leukemia-Targeted Therapies: A Class-Restricted Disproportionality Analysis of the FDA Adverse Event Monitoring System (2016–2026) (SOHO 2026) - " AE reports (January 2016–April 2026) were extracted for revumenib (n = 716) and eight targeted AML therapies (n = 70179): ziftomenib, enasidenib, ivosidenib, olutasidenib, gilteritinib, midostaurin, quizartinib, and venetoclax. Analysis of 716 revumenib reports (median age, 50 years; 50.2% male; 57.5% US-sourced; 55.6% serious) identified AML as the recorded indication in 59.2% of cases. Eleven AEs qualified as robust signals. Established toxicities included differentiation syndrome (n = 32; ROR, 9.12; 95% CI, 6.31–13.20), QTc prolongation (n = 29; ROR, 8.88; 95% CI, 6.03–13.08), and decreased platelet count (n = 113; ROR, 3.91; 95% CI, 3.19–4.79)."

Adverse events • Clinical • P4 data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • FLT3 • KMT2A

Ziftomenib in combination with venetoclax and azacitidine in newly diagnosed NPM1-m acute myeloid leukemia: Phase 1b results from KOMET-007 (ASH 2025) - P1, P3 | "In the ongoing KOMET-007 study, ziftomenib RP2D of 600 mg once daily combined withVen/Aza was well tolerated and demonstrated robust clinical activity in patients with newly diagnosedNPM1-m AML, including 84% CRc after a median of 3.5 weeks and 54% CRc MRD-negativity after amedian of 8.4 weeks. Low rates of ziftomenib-related cytopenia and no additional myelosuppressionwere observed with this combination. One case each of differentiation syndrome (grade 2) andinvestigator-assessed QTc (grade 3) were successfully resolved."

Combination therapy • P1 data • Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • FLT3 • HEY1 • KMT2A • NPM1

Ziftomenib Combined with Venetoclax/Azacitidine in Relapsed/Refractory NPM1-m or KMT2A-r Acute Myeloid Leukemia: Interim Phase 1a Results from KOMET‑007 (ASH 2024) - P1 | "Based on these encouraging initial results, a dose expansion phase evaluating this triplet combination in newly diagnosed and R/R NPM1-m and KMT2A-r AML patients is underway. Updated results will be presented, as data from the 400 mg cohorts continue to mature and 600 mg cohorts are enrolling."

P1 data • Acute Myelogenous Leukemia • Anemia • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Neutropenia • Oncology • Pneumonia • Respiratory Diseases • KMT2A • NPM1

Menin Inhibitors in NPM1 Mutations and KMT2A Rearrangements in Acute Myeloid Leukemia: Differential Efficacy Across Frontline and Relapsed Settings: A Systematic Review and Meta-Analysis (SOHO 2026) - "Regimens included revumenib- and ziftomenib-based monotherapy and combination approaches...In R/R AML, monotherapy achieved CR/CRh rates of approximately 22% to 23%, whereas frontline triplet therapy (azacitidine, venetoclax, and revumenib) demonstrated CRc rates greater than 80%... Menin inhibitors demonstrate meaningful clinical and molecular activity in molecularly defined AML, with markedly higher efficacy in frontline combination regimens than achieved with R/R monotherapy. These findings highlight their potential as a backbone for precision-based combinations. Prospective randomized studies are needed to define optimal sequencing strategies, combination approaches, and the prognostic relevance of MRD and immunophenotypic evolution."

Retrospective data • Review • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • KMT2A • NPM1

Comparative Toxicity of Menin Inhibitor–Based Combination Therapy in Newly Diagnosed Acute Myeloid Leukemia: Venetoclax-Containing Versus Non-Venetoclax Regimens (SOHO 2026) - "BEAT AML trial (revumenib+venetoclax+azacitidine; n = 43; median age, 73 years; unfit for intensive chemotherapy) reported these rates: differentiation syndrome, 19% for any grade, grade ≥3 in 5% (2/43); QTc prolongation, 44% for any grade, grade 3 in 12% (5/43); febrile neutropenia, grade ≥3 in 26% (11/43); treatment discontinuation, 0%; early mortality, 7% (3/43, sepsis/respiratory failure)...Non-venetoclax regimens: KOMET-007, 7+3 (ziftomenib+cytarabine/daunorubicin) group (n = 51; median age, 59 years; fit patients) reported these rates: differentiation syndrome, 0%; febrile neutropenia, grade ≥3 in 47% (24/51)... Venetoclax-based menin inhibitor combinations demonstrated significantly lower febrile neutropenia, compared with intensive chemotherapy, and manageable differentiation syndrome. No differentiation syndrome with 7+3 likely reflects delayed menin inhibitor initiation after cytoreduction. Cross-trial comparisons are limited by heterogeneous populations, small..."

Combination therapy • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • KMT2A • NPM1

A Practical Alternative to Refine the Estimate of fmCYP3A4 and Evaluate Drug-Drug Interaction Potential for Ziftomenib Using PBPK Modeling to Inform Labeling. (PubMed, CPT Pharmacometrics Syst Pharmacol) - "Moderate or weak interaction (2.6-fold and 1.4-fold increase in AUC) was predicted with itraconazole and isavuconazole, respectively. Approximately 80% reduction in ziftomenib AUC was predicted with rifampicin. Ziftomenib was predicted to be a weak CYP3A4 inhibitor, causing a 1.9-fold increase in midazolam exposure. In the absence of data from dedicated DDI clinical trials, these results were used to support regulatory interactions with the US FDA regarding concomitant administration of ziftomenib with other medications such as CYP3A4 modulators. These modeling results ultimately supported a range of DDI language in ziftomenib label."

Journal • Acute Myelogenous Leukemia • Infectious Disease

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. (ASCO 2025) - P1/2 | "Median age was 69 yrs (range 22–86), 56% female, 83% ECOG PS 0–1, median of 2 prior therapies (range 1–7), including 60% prior venetoclax (VEN) and 23% prior transplant... In the pivotal KOMET-001, the phase 2 primary endpoint was met: Ziftomenib achieved deep and durable responses in R/R NPM1-m AML, regardless of prior VEN. Ziftomenib was well tolerated with limited myelosuppression and only 3% ziftomenib-related discontinuations. Taken together, these data support the potential use of ziftomenib monotherapy as a new treatment option for R/R NPM1-m AML."

Clinical • P1/2 data • Acute Myelogenous Leukemia • Anemia • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Leukemia • Neutropenia • Oncology • Thrombocytopenia • KMT2A • NPM1

Can Menin Inhibitors Bridge Patients to Transplant? Response Depth, MRD Negativity, and Differentiation Syndrome in NPM1-Mutated and KMT2A-Rearranged Acute Myeloid Leukemia (SOHO 2026) - "Interventions: Revumenib, ziftomenib, and DS-1594b as monotherapy in relapsed/refractory disease; revumenib combined with azacitidine and venetoclax in newly diagnosed AML. Menin inhibitors produce clinically meaningful, often MRD– deep remissions that may enable HSCT bridging in selected patients with KMT2A-rearranged and NPM1-mutated AML. Differentiation syndrome and QTc prolongation remain key safety considerations requiring protocoled monitoring. AML: acute myeloid leukemia, CR: complete remission, CRh: complete remission with partial hematologic recovery, HSCT: hematopoietic stem cell transplantation, KMT2A: lysine methyltransferase 2A, MRD: measurable residual disease, NPM1: nucleophosmin 1."

Clinical • Minimal residual disease • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • KMT2A • NPM1

Ziftomenib with venetoclax and azacitidine in relapsed/refractory NPM1-mutated acute myeloid leukemia. (PubMed, Blood) - P1 | "The combination of ziftomenib 600 mg with venetoclax/azacitidine was well tolerated with deep and durable clinical activity in R/R NPM1-m AML. This trial was registered at www.ClinicalTrials.gov as #NCT05735184."

Journal • Acute Myelogenous Leukemia • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Leukemia • Leukopenia • Neutropenia • Oncology • Thrombocytopenia • Transplantation • NPM1

Comparative Efficacy and Safety of FDA-Approved Menin Inhibitors in Relapsed/Refractory NPM1-Mutated Acute Myeloid Leukemia: A Systematic Review and Meta-Analysis (SOHO 2026) - "Following the 2025 FDA approvals of revumenib and ziftomenib, comparative evidence regarding efficacy and safety remains limited...Additionally, approximately 20.0% of patients (95% CI, 14.3%–26.8%) successfully bridged to allogeneic stem-cell transplantation, supporting the emerging role of menin inhibitors as effective targeted salvage therapies in post-venetoclax relapsed/refractory AML populations... FDA-approved menin inhibitors demonstrated significant efficacy in relapsed/refractory NPM1-mutated AML, with ziftomenib showing deeper molecular responses and superior cardiac safety, supporting its potential as a next-generation targeted therapeutic backbone in AML. RoB 2.0 assessment demonstrated an overall low-to-moderate risk of bias across the included studies. CI: confidence interval, CR/CRh: complete response/complete response with partial hematologic recovery, FDA: Food and Drug Administration, MRD: measurable residual disease, NPM1: nucleophosmin 1, QTc:..."

Retrospective data • Review • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • NPM1

Menin Inhibitors vs FLT3 Inhibitor-Based Therapy in NPM1-Mutated Relapsed/Refractory AML: Comparative Efficacy, MRD Depth, and Safety Profiles (SOHO 2026) - "Interventions: MENi (revumenib, ziftomenib) targeting menin-KMT2A/HOXA signaling, and FLT3i-based regimens (gilteritinib monotherapy or combinations) targeting FLT3-driven signaling in relapsed/refractory AML. MENi and FLT3i-based regimens demonstrate comparable remission rates in NPM1-mutated relapsed/refractory AML but differ in response depth. MENi shows higher MRD negativity. FLT3i provides broader disease control with higher infectious burden, while MENi is associated with differentiation syndrome."

Clinical • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • FLT3 • KMT2A • NPM1

MRD-Adjusted Therapeutic Index of Menin Inhibitors in NPM1-Mutated and KMT2A- Rearranged Acute Myeloid Leukemia: A Genotype-Stratified Systematic Review and Meta-Analysis (SOHO 2026) - "Revumenib received Food and Drug Administration (FDA) approval in 2024 for relapsed/refractory acute leukemia with KMT2A translocation in 2024 and for relapsed/refractory NPM1-mutated AML in 2025, while ziftomenib was approved in 2025 for relapsed/refractory NPM1-mutated AML. This genotype-stratified meta-analysis will move beyond response-rate reporting by quantifying the clinical trade-off between molecular remission depth and toxicity burden. An MRD-adjusted therapeutic index may help define which AML genotypes and treatment contexts derive the most favorable benefit-risk profile from menin inhibition and inform future sequencing, combination, and transplant-bridging strategies. KMT2A: lysine methyltransferase 2A, NPM1: nucleophosmin 1, QTc: corrected QT interval."

Retrospective data • Review • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • KMT2A • NPM1

Targeting the Menin-KMT2A Axis in AML: Translating Molecular Insights into Therapeutic Success. (PubMed, Clin Lymphoma Myeloma Leuk) - "These findings rapidly led to phase I/II clinical trials with oral MIs such as revumenib and ziftomenib, which demonstrated CR+CRh rates of 22% to 23% in refractory/relapsed patients, with conversion to measurable residual disease (MRD) negativity in 61% to 68% of responders and early use as a bridge to allogeneic transplantation. Key emerging clinical issues include specific adverse events (QTc prolongation in 12%-44%, differentiation syndrome in 10%-29%), drug-drug interactions mediated by CYP3A4 metabolism, resistance dynamics (on-target mutations in 39% of cases), and the definition of the optimal role in combination with venetoclax, hypomethylating agents, and intensive chemotherapy. This review synthesizes molecular mechanisms, preclinical and clinical data, discusses use within MRD-guided and transplant pathways, and proposes translational priorities for integrating MIs into future therapeutic paradigms, pending confirmation from randomized studies."

Journal • Review • Acute Myelogenous Leukemia • Gene Therapies • Hematological Malignancies • Leukemia • Oncology • Transplantation • CYP3A4 • KMT2A • MEIS1 • NPM1

ZIFTOMENIB COMBINED WITH INTENSIVE INDUCTION (7+3) FOR NEWLY DIAGNOSED NPM1‑M OR KMT2A-R ACUTE MYELOID LEUKEMIA (AML): LONG-TERM RESULTS FROM THE KOMET-007 TRIAL (EHA 2026) - P1, P3 | "Aims Here we report updated safety and clinical activity in patients (pts) with ND AML treated with ziftomenib 600 mg once daily (QD) in combination with standard doses of cytarabine/daunorubicin (7+3) from KOMET-007. . Low rates of additive myelosuppression were observed with this combination. These data further support the ongoing Ph3 registrational trial of ziftomenib in combination with intensive chemotherapy (KOMET- 017; NCT07007312 )."

Acute Myelogenous Leukemia • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Leukemia • Leukopenia • Neutropenia • Pruritus • Thrombocytopenia • KMT2A • NPM1

ACTIVITY, TOLERABILITY, AND RESISTANCE PROFILE OF THE MENIN INHIBITOR ZIFTOMENIB IN ADULTS WITH RELAPSED/REFRACTORY NPM1-MUTATED AML (EHA 2023) - P1/2 | "Ziftomenib continues to demonstrate significant clinical activity in heavily pretreated and co-mutated R/R NPM1mAML pts where 35% of pts achieved CR. The safety profile remains consistent, and episodes of DS are clinicallymanageable. Data reveal that remissions are durable, with MRD clearance of NPM1 and key co-mutations.Resistance mutations develop infrequently, and ziftomenib remains effective against a common menin gatekeepermutation."

Clinical • Late-breaking abstract • Acute Myelogenous Leukemia • Anemia • Bone Marrow Transplantation • Hematological Disorders • Hematological Malignancies • Leukemia • Oncology • Thrombocytopenia • Transplantation • FLT3 • IDH1 • IDH2 • KMT2A • MEN1 • NPM1

Ziftomenib in relapsed or refractory acute myeloid leukaemia (KOMET-001): a multicentre, open-label, multi-cohort, phase 1 trial. (PubMed, Lancet Oncol) - P1/2 | "Ziftomenib showed promising clinical activity with manageable toxicity in heavily pretreated patients with relapsed or refractory acute myeloid leukaemia. Phase 2 assessment of ziftomenib combination therapy in the upfront and relapsed or refractory setting is ongoing."

Journal • P1 data • Acute Myelogenous Leukemia • Cardiovascular • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Neutropenia • Oncology • Pneumonia • Respiratory Diseases • Septic Shock • Thrombocytopenia • KMT2A • NPM1