Vyxeos (cytarabine/daunorubicin liposomal formulation) / Jazz, Nippon Shinyaku - LARVOL DELTA

CPX-351 in combination with ruxolitinib for advanced-phase myeloproliferative neoplasms: results of a phase 1 study. (PubMed, Blood Neoplasia) - No abstract available

Journal • P1 data • Myeloproliferative Neoplasm • Oncology

Results from paradigm - a phase 2 randomized multi-center study comparing azacitidine and venetoclax to conventional induction chemotherapy for newly diagnosed fit adults with acute myeloid leukemia (ASH 2025) - "The study has met its primary endpoint. Aza-ven was associated with significantly improved EFS, as wellas higher rates of OR and CCR, vs IC in younger, IC-eligible pts. Overall survival data continue to mature."

Clinical • P2 data • Acute Myelogenous Leukemia • CNS Disorders • Depression • Hematological Malignancies • Infectious Disease • Leukemia • Psychiatry • Respiratory Diseases • Septic Shock • FLT3 • IDH1 • IDH2 • NPM1 • TP53

Outcomes after Transplant in Relapsed/Refractory KMT2Ar (MLLr) and mNPM1 (NPM1c) leukemia Patients Achieving Remissions after Menin Inhibition: SNDX-5613 (revumenib) Ph1 Experience (ASH 2022) - P1/2 | "1 was among the 12 patients described in Table 1: a 40 yo F with KMT2Ar AML with FLT3 TKD, and 3 prior lines of therapy including 7+3+midostaurin and HSCT...She then had a molecular relapse and received CPX-351, gemtuzumab, venetoclax, and azacytidine, a 3rd allo-HSCT, and due to persistent positive MRD by flow, she received a donor lymphocyte infusion... In SNDX-5613 patients proceeding to transplant, durable remissions occurred across a range of heavily pre-treated patients. In addition to patients achieving CR/CRh, two patients with CRp also had ongoing remissions post-transplant. AUGMENT-101 continues to enroll patients, agnostic of transplant eligibility, with the option for SNDX-5613 post-transplant maintenance."

Clinical • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Infectious Disease • Leukemia • Oncology • Septic Shock • Transplantation • FLT3 • KMT2A • NPM1

A randomized phase II study of selinexor added to standard induction and consolidation chemotherapy in adults with acute myeloid leukemia (AML). (ASCO 2026) - P2 | "In this randomized phase II study, the addition of selinexor to standard induction and consolidation chemotherapy was associated with numerically higher response rates and longer median overall survival compared with chemotherapy alone. Small study size and early termination limit definitive conclusions. However, these findings suggest biological activity of nuclear export inhibition in AML and support further evaluation of selinexor-containing combinations."

Clinical • P2 data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • FLT3

Lower-intensity CPX-351 plus venetoclax induction for adults with newly diagnosed AML unfit for intensive chemotherapy. (PubMed, Blood Adv) - P1b | "This study highlights that lower-intensity therapy of CPX-351 plus venetoclax as induction therapy provides a well-tolerated treatment option in adults with AML deemed unfit for intensive chemotherapy. This trial was registered at www.clinicaltrials.gov as #NCT04038437."

IO biomarker • Journal • Acute Myelogenous Leukemia • B Cell Non-Hodgkin Lymphoma • Hematological Disorders • Leukemia • Lymphoma • Oncology • TP53

Mitoxantrone liposome, subcutaneous cytarabine and G-CSF (CMG) combined with venetoclax as first-line treatment in secondary Acute Myeloid Leukemia (sAML) or elderly AML patients: A multicenter, prospective, single-arm clinical trial with concurrent control (ASH 2025) - P=N/A | "CMG+VEN represents a more safe alternative regimen in sAML and elderly AML patients,showing higher MRD negative rate and deserving further investigation."

Clinical • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Myeloproliferative Neoplasm

CPX-351 vs Standard of Care as Bridging to AlloHCT in Patients With Higher-Risk Myelodysplastic Neoplasms or Oligoblastic Acute Myeloid Leukemia: Results of the Randomized Phase 2 PALOMA Study (SOHO 2026) - "PALOMA met its primary endpoint in interim analysis showing that CPX-351 resulted in improved EFS when compared with CCR among patients with HR-MDS or oligoblastic AML scheduled for alloHCT. alloHCT: allogeneic hematopoietic stem cell transplantation, AML: acute myeloid leukemia, AZA: azacytidine, CCR: conventional care regimens, CI: confidence interval, CPX-351: liposomal cytarabine and daunorubicin, EFS: event-free survival, FAS: full analysis set, HR-MDS: higher-risk myelodysplastic neoplasms, IC: induction chemotherapy, NR: not reached, OS: overall survival."

Clinical • P2 data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology

Molecular Features, Treatments and Outcomes for Pediatric AML Patients from APAL2020SC Pediatric Acute Leukemia (PedAL) Screening Trial (ASH 2024) - P1/2 | "The most common regimens were combinations including fludarabine/cytarabine (FLA); 18/40 (45%) and 12/18 (67%) achieved remission...In the prAML group, 10 of 23 with cycle 1 treatment and response data achieved remission : 2 with a FLA regimen, 5 with a venetoclax regimen, and 3 with CPX-351. Conclusion The APAL2020SC protocol is collecting valuable real-world treatment and response data, as well as comprehensive molecular characterization, for children with relapsed and refractory AML. These data will fill gaps in knowledge regarding this patient population and will inform the design of future therapeutic trials."

Clinical • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • Pediatrics • AFDN • CBFA2T3 • FLT3 • GLIS2 • KMT2A • KRAS • MLLT10 • MLLT3 • NRAS • NSD1 • NUP98 • PTPN11 • RUNX1 • RUNX1T1 • WT1

PALOMA: Comparison of Therapies Before Stem Cell Transplantation in Patients With Higher Risk MDS and Oligoblastic AML (clinicaltrials.gov) - P2 | N=150 | Completed | Sponsor: GWT-TUD GmbH | Recruiting ➔ Completed | Trial primary completion date: Mar 2026 ➔ Aug 2026

Trial completion • Trial primary completion date • Acute Myelogenous Leukemia • Myelodysplastic Syndrome • Transplantation

Lower-intensity CPX-351 + venetoclax for patients with newly diagnosed AML who are unfit for intensive chemotherapy. (ASCO 2022) - P1b | "Lower-intensity CPX-351 + VEN was generally well tolerated in adults with newly diagnosed AML who are unfit for IC and showed promising initial efficacy, with CR or CRi in the majority of pts."

Clinical • IO biomarker • Acute Myelogenous Leukemia • Cardiovascular • Cough • Infectious Disease • Myelodysplastic Syndrome • Myocardial Infarction • Pulmonary Disease • Respiratory Diseases • TP53

RISK STRATIFICATION OF NEWLY DIAGNOSED ACUTE MYELOID LEUKEMIA TREATED WITH VENETOCLAX IN COMBINATION WITH INTENSIVE CHEMOTHERAPY (EHA 2025) - P1/2, P2 | "While IC+VEN may abrogate certain ELN22 adverse features, pts with TP53mut and MECOMr still do poorly and require novel therapies. These findings require independent validation."

Combination therapy • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • FLT3 • IDH1 • IDH2 • KMT2A • KRAS • NRAS • PTPN11 • TP53

Fitness-Based Induction Selection and Outcomes in Adult Acute Myeloid Leukemia at a Tertiary Cancer Center in Southern California (SOHO 2026) - "Lower-intensity regimens included HMA ± venetoclax or low-dose cytarabine ± venetoclax... In a contemporary Southern California AML cohort, prespecified fitness classification strongly aligned with induction intensity selection. Low early mortality across fitness groups may reflect fitness-adapted treatment selection and modern supportive care. These findings provide regional real-world practice patterns for AML induction counseling and supportive care planning."

Clinical • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Combination of CPX-351 and Gemtuzumab Ozogamicin (GO) in Relapsed Refractory (R/R) Acute Myeloid Leukemia and Post Hypomethylating Agent (HMA) Failure High-Risk Myelodysplastic Syndrome (HR-MDS) (ASH 2024) - P2 | "Background : Outcomes of patients (pts) with HR-MDS or AML who are refractory to or progress after HMA ± Venetoclax (Ven) based therapy is dismal, warranting evaluation of newer agents...OS was meaningful in responders, considering multiple prior lines of therapy. Infection related events were the most common serious adverse events."

Acute Myelogenous Leukemia • Bone Marrow Transplantation • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Myelodysplastic Syndrome • Myeloproliferative Neoplasm • Neutropenia • Oncology • Pediatrics • Pneumonia • Respiratory Diseases • Septic Shock • CD33 • KMT2A • MECOM • TP53

Real-World Outcomes of Acute Myeloid Leukemia, Myelodysplasia-Related: A Single Center Study From Pakistan (SOHO 2026) - "In a resource-constrained setting, access to molecular diagnostics and HSCT consolidation is required to improve outcomes. CI: confidence interval, CR: complete response, CRh: complete remission with partial hematologic recovery, CRi: complete remission with incomplete hematologic recovery, DFS: disease-free survival, ELN: European LeukemiaNet, HiDAC: high-dose cytarabine, HMA: hypomethylating agent, HMA-Ven: hypomethylating agent plus venetoclax, HSCT: hematopoietic stem cell transplantation, IDAC: intermediate-dose cytarabine, IQR: interquartile range, PR: partial remission."

Clinical • Real-world • Real-world evidence • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology

Updated Results of CPX-351 in Combination with Gemtuzumab Ozogamicin (GO) in Relapsed Refractory (R/R) Acute Myeloid Leukemia (AML) and Post-Hypomethylating Agent (Post-HMA) Failure High-Risk Myelodysplastic Syndrome (HR-MDS) (ASH 2022) - P2 | "Background: Treatment outcomes in patients (pts) with AML and HR-MDS who progress after HMA +/- venetoclax (VEN) based regimen remains poor and warrants new therapeutic strategies... In a cohort composed predominantly of heavily pre-treated and VEN exposed adverse risk AML and HR-MDS pts, CPX-GO led to overall response (CR/CRh/MLFS/PR) rates of 52% and year-long median survival in responders (CR/CRh/MLFS) with no major non-hematological adverse event."

Combination therapy • Acute Myelogenous Leukemia • Bone Marrow Transplantation • CNS Disorders • Epilepsy • Hematological Malignancies • Infectious Disease • Mucositis • Myelodysplastic Syndrome • Transplantation • CD33 • NPM1

Comparative Efficacy and Safety of FLT3 Inhibitor–Based Frontline Regimens in Newly Diagnosed FLT3-Mutated Acute Myeloid Leukemia: A Systematic Review and Network Meta-Analysis (SOHO 2026) - "Introduction: The rapid expansion of FLT3 inhibitors (FLT3i) and venetoclax (VEN)-based combinations has transformed the frontline landscape for FLT3-mutated AML...Gilteritinib-based triplets achieved the highest complete response/ complete remission with incomplete count recovery (CR/Cri) rates (OR, 3.15; 95% CI, 2.10–4.72) and measurable residual disease negativity (OR, 2.80; 95% CI, 1.65–4.75) vs midostaurin + 7+3, although OS benefit was partially offset by early mortality. For hypomethylating agent backbones, VEN + FLT3i triplets outperformed VEN + AZA (azacitidine) doublets in event-free survival (HR, 0.68; 95% CI, 0.49–0.94)... While triplet therapies offer the deepest molecular responses, quizartinib-based intensive induction currently represents the most favorable balance of survival and safety for fit patients; notably, the overall risk of bias across included studies was low. 7+3: 7 days of cytarabine and an anthracycline for the first 3 days, ASH: American..."

Retrospective data • Review • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • FLT3

CPX-351 VERSUS STANDARD OF CARE AS BRIDGING TO ALLOHCT IN PATIENTS WITH HIGHER-RISK MYELODYSPLASTIC NEOPLASMS OR OLIGOBLASTIC ACUTE MYELOID LEUKEMIA: RESULTS OF THE RANDOMIZED PHASE 2 PALOMA STUDY (EHA 2026) - P2 | "Pts are commonly bridged from diagnosis to alloHCT via hypomethylating agents such as azacitidine (AZA) or with conventional induction chemotherapy (IC)...Summary/Conclusion PALOMA met its primary endpoint in interim analysis showing that CPX-351 resulted in improved EFS when compared to CCR among pts with HR-MDS or oligoblastic AML scheduled for alloHCT. Figure 1: Event-free survival from the date of randomization in the full analysis set"

Clinical • P2 data • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Leukemia • Leukopenia • Myelodysplastic Syndrome • Neutropenia • Thrombocytopenia

A Phase 2 Trial of CPX-351 Combined With Venetoclax in Relapsed or Refractory Acute Myeloid Leukemia. (PubMed, Am J Hematol) - P2 | "CPX + VEN has activity in RR AML, particularly when used in first salvage and in patients who do not harbor TP53 mutations. ClinicalTrials.gov Identifier: NCT03629171."

Journal • P2 data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • Transplantation • TP53

Impact of Cytogenetics, Induction Chemotherapy and MRD on Outcomes of Intensively Treated AML with KMT2A Rearrangements – Experience from UK NCRI AML17 and AML19 (ASH 2024) - "Menin inhibitors have shown encouraging activity in relapsed/refractory disease, and trials combining these agents with intensive chemotherapy or venetoclax-azacitidine are ongoing...We also show that molecular MRD after course 2 is a strong predictor of long-term survival. These data are important to inform the design of future trials incorporating menin inhibitors in front-line therapy, and suggest that intensified induction and RT-qPCR MRD should be incorporated into these studies."

Acute Myelogenous Leukemia • Hematological Malignancies • FLT3 • KMT2A • MLLT3

Lower-Intensity CPX-351 + Venetoclax for Patients with Newly Diagnosed Acute Myeloid Leukemia Who Are Unfit for Intensive Chemotherapy: Post Hoc Analysis By Disease Risk Subgroups (ASH 2022) - P1b | "The combination of lower-intensity CPX-351 + VEN demonstrated efficacy in both the favorable/intermediate and poor disease risk subgroups, without excessive myelosuppression. These data also provide further insights into outcomes on CPX-351 combination therapies for the treatment of AML."

Clinical • IO biomarker • Retrospective data • Acute Myelogenous Leukemia • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Neutropenia • Oncology • Thrombocytopenia • TP53

V-FAST Master Trial: Subgroup Analysis of Outcomes with CPX-351 Plus Midostaurin in Adults with Newly Diagnosed Acute Myeloid Leukemia By FLT3 Mutation Type (ASH 2022) - P1b | "V-FAST (Vyxeos – First Phase Assessment with Targeted Agents) is an open-label, multicenter, multi-arm, nonrandomized, phase 1b master trial (NCT04075747) to evaluate the safety and preliminary efficacy of CPX-351 combined with targeted agents (MID, venetoclax, enasidenib)...In conclusion, preliminary results from the V-FAST trial suggest the combination of CPX-351 + MID is feasible with a manageable safety profile and promising remission rates in adults with newly diagnosed, FLT3-mutated AML. Although conclusions are limited by the small numbers of patients in each subgroup, results are generally consistent for patients with FLT3 ITD and TKD mutations."

Clinical • Acute Myelogenous Leukemia • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Leukopenia • Myelodysplastic Syndrome • Neutropenia • Oncology • Thrombocytopenia • Transplantation • FLT3

Salvage Chemotherapy Intensity Does Not Impact Survival in Relapsed/Refractory Acute Myeloid Leukemia: A Single-Center Retrospective Analysis (SOHO 2026) - "Salvage chemotherapy intensity did not significantly impact PFS or OS in R/R AML, regardless of induction regimen. Notably, IC→LIC patients achieved numerically longer PFS and OS than IC→IC patients despite older age, suggesting LIC-based salvage may offer comparable efficacy. Limitations include the retrospective single-center design, small subgroup sizes, and absence of molecular stratification."

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

Comparison of venetoclax and azacitidine versus a cytarabine-based treatment for patients with acute myeloid leukaemia in first relapse after chemotherapy. A real-world study of the DATAML-IPC-AURAML consortium. (PubMed, Br J Haematol) - "In multivariable analysis, relapsing patients ≥60 years with low/intermediate-risk cytogenetics and VEN-AZA had a better OS (p = 0.002), while I/HDAC was more efficient for those with poor risk cytogenetics and < 60-year-old (p = 0.002). VEN-AZA salvage therapy thus appears promising compared with conventional I/HDAC-based IC; this question warrants evaluation in randomized studies."

Journal • Real-world evidence • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Leukemia • Oncology • Transplantation

CPX-351 in Down syndrome-associated Myeloid Leukemia: Results and Prognostic Factors from the Phase 3 ML-DS 2018 Trial. (PubMed, Blood) - P3 | "Intensity-reduced induction and reinduction therapy with cytarabine, idarubicin ± etoposide of the ML-DS 2006 trial was replaced with CPX-351 (66 U/m² on three days in course 1 and two days in course 2)...In conclusion, replacing intensity-reduced induction therapy with CPX-351 in ML-DS resulted in significantly lower event-free survival, highlighting the need for dose optimization to balance efficacy and toxicity in this sensitive patient population. EudraCT: 2018-002988-25."

Biomarker • Journal • P3 data • Developmental Disorders • Genetic Disorders • Hematological Malignancies • Leukemia • Oncology • GATA1

RANDOMIZED PHASE 2 STUDY OF CPX-351 + POMALIDOMIDE VS CPX-351 ALONE IN NEWLY DIAGNOSED AML WITH MDS-RELATED CHANGES: RESULTS OF SAFETY RUN-IN EVALUATION (EHA 2024) - P2 | "The MTD and RP2D of Pom was determined to be 4 mg for 14 days starting on day 21 of CPX-351 induction. There was one incidence of grade 3 AST/ALT elevation seen without any other grade 3 toxicities related toPom. The randomized phase of this study is currently ongoing comparing CPX-351 plus Pom versus CPX-351alone in newly diagnosed AML-MR."

Clinical • P2 data • Acute Myelogenous Leukemia • Cardiovascular • Chronic Myelomonocytic Leukemia • Fibrosis • Hepatology • Immunology • Myelodysplastic Syndrome • Targeted Protein Degradation • Thrombosis • Venous Thromboembolism • CRBN • IKZF1 • IKZF3 • IL2