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

Treatment of therapy-related acute myeloid leukemia and acute myeloid leukemia with myelodysplasia-related changes: a comparative analysis of higher-dose intensive 7+3 induction chemotherapy versus liposomal cytarabine and daunorubicin. (PubMed, Haematologica) - "Thirty-day mortality (4.4% for higher-intensity 7+3 versus 5.9% for CPX-351) and adverse events, including febrile neutropenia (61% for higher-intensity 7+3 versus 68% for CPX-351), were comparable. The data suggest that obligatory double-induction may achieve outcomes similar to CPX-351 in these patients and provide a strong rationale for ongoing clinical trials comparing these regimens."

Journal • Acute Myelogenous Leukemia • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Neutropenia • Oncology

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

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

Nanotechnology-Driven Cancer Therapies for Precision Oncology: Advances and Clinical Outlook. (PubMed, Int J Nanomedicine) - "Although multiple nano-formulations such as Doxil®, Abraxane®, and Vyxeos® have reached clinical use, challenges remain including large-scale manufacturing, regulatory pathways, long-term safety evaluation, and cost-effective global accessibility. This review provides a critical appraisal of current evidence, translational bottlenecks, and emerging opportunities to guide future nanomedicine development. Nanotechnology is poised to become a cornerstone of precision oncology, enabling personalized, safe, and effective cancer treatment paradigms."

Journal • Review • Oncology

Phase IB/II of CPX-351 for Relapse Prevention in AML (clinicaltrials.gov) - P1/2 | N=24 | Recruiting | Sponsor: Georgetown University | Trial completion date: Dec 2025 ➔ Dec 2026 | Trial primary completion date: Dec 2025 ➔ Dec 2026

Trial completion date • Trial primary completion date • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • Transplantation

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

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

CPX-351 Selectively Benefits Patients with AML and Myelodysplasia-Related Mutations in the Pivotal Randomized Trial. (PubMed, Blood Adv) - P3 | "The OS benefit of CPX-351 observed in the trial was driven by AML-MR with no benefit of CPX-351 in TP53-AML, where the primary prognostic factor was allelic state. Clinical Trial Information: NCT01696084."

Journal • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • Transplantation • DDX41 • TP53

Νοvel Therapies in High-Risk Myelodysplastic Syndromes. (PubMed, Eur J Haematol) - "Recent advances have led to the development of novel therapeutic strategies, such as BCL-2 inhibitors (venetoclax), IDH1/2 inhibitors (ivosidenib, enasidenib), CD47 inhibitors (magrolimab), TIM-3 inhibitors (sabatolimab), XPO1 inhibitors (eltanexor), NEDD8-activating enzyme inhibitors (pevonedistat), TP53-targeted agents (eprenetapopt), liposomal chemotherapy (CPX-351), and oral HMA formulations. Combinations of hypomethylating agents with these new drugs, as first-line treatment, have to date not proven more efficacious than HMA monotherapy. This review summarizes the current therapeutic landscape on novel therapies for HR-MDS, highlighting their mechanism of action, efficacy, and demonstrates the unmet clinical need for more effective therapies."

Journal • Review • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Disorders • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • Transplantation • HAVCR2 • IDH1 • IDH2

Safety and Efficacy of Combining Midostaurin and Gemtuzumab Ozogamicin with Induction Chemotherapy in FLT3 mutated AML. (PubMed, Blood Adv) - "We evaluated the safety and efficacy of the combination of daunorubicin, cytarabine (DA), gemtuzumab ozogamicin (GO) and midostaurin (DAGO+m) for younger patients with newly diagnosed FLT3mut AML in the UK NCRI AML19 trial...DAGO2+m will now be evaluated in a randomised study (OPTIMISE-FLT3, ISRCTN 34016918). Trial: ISRCTN78449203."

Journal • Acute Myelogenous Leukemia • Transplantation • FLT3 • NPM1

HYPOMETHYLATING AGENTS PLUS VENETOCLAX VS INTENSIVE CHEMOTHERAPY PRIOR TO TRANSPLANT IN HIGH-RISK ACUTE MYELOID LEUKEMIA (EBMT 2026) - "The less-intensive regimen combining the hypomethylating agent (HMA) azacitidine (AZA) and the BCL-2 inhibitor venetoclax (VEN) has changed the treatment paradigm for older subjects with newly-diagnosed AML... Our study provides evidence supporting the use of HMA-VEN as an alternative, less toxic remission induction strategy for patients with ELN-2022 adverse-risk AML who are intended to proceed to allo-SCT."

IO biomarker • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Transplantation • TP53

SEQUENTIAL CONDITIONING USING INDUCTION WITH CPX-351 FOLLOWED BY DOSE REDUCED CONDITIONING BEFORE ALLOGENEIC STEM CELL TRANSPLANTATION FOR HIGH-RISK ACUTE MYELOID LEUKEMIA (EBMT 2026) - "Dose-reduced conditioning regimens consisted of Thiotepa, Busulfan and Fludarabin (Flud) (n=8), Treosulfan and Flud (n=4), or Melphalan, BCNU and Flud (n=1)...GVHD prophylaxis included a calcineurin inhibitor and MMF in all pts, with 5 pts receiving additional ATG, 2 pts additional everolimus/sirolimus, and 5 pts additional ptCy... Sequential conditioning combining CPX-351 with dose-reduced conditioning regimen leads to promising outcome in 1st alloSCT for high-risk AML, but requires further evaluation within randomized clinical trials."

Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Biliary Cancer • Bone Marrow Transplantation • Cholangiocarcinoma • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Solid Tumor • Transplantation

HYPOMETHYLATING AGENTS PLUS VENETOCLAX VS INTENSIVE CHEMOTHERAPY PRIOR TO TRANSPLANT IN HIGH-RISK ACUTE MYELOID LEUKEMIA (EBMT 2026) - "The less-intensive regimen combining the hypomethylating agent (HMA) azacitidine (AZA) and the BCL-2 inhibitor venetoclax (VEN) has changed the treatment paradigm for older subjects with newly-diagnosed AML... Our study provides evidence supporting the use of HMA-VEN as an alternative, less toxic remission induction strategy for patients with ELN-2022 adverse-risk AML who are intended to proceed to allo-SCT."

IO biomarker • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Transplantation • TP53

SEQUENTIAL CONDITIONING USING INDUCTION WITH CPX-351 FOLLOWED BY DOSE REDUCED CONDITIONING BEFORE ALLOGENEIC STEM CELL TRANSPLANTATION FOR HIGH-RISK ACUTE MYELOID LEUKEMIA (EBMT 2026) - "Dose-reduced conditioning regimens consisted of Thiotepa, Busulfan and Fludarabin (Flud) (n=8), Treosulfan and Flud (n=4), or Melphalan, BCNU and Flud (n=1)...GVHD prophylaxis included a calcineurin inhibitor and MMF in all pts, with 5 pts receiving additional ATG, 2 pts additional everolimus/sirolimus, and 5 pts additional ptCy... Sequential conditioning combining CPX-351 with dose-reduced conditioning regimen leads to promising outcome in 1st alloSCT for high-risk AML, but requires further evaluation within randomized clinical trials."

Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Biliary Cancer • Bone Marrow Transplantation • Cholangiocarcinoma • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Solid Tumor • 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

Impact of fitness categorization according to SIE/SIES/GITMO criteria in therapy-related and AML-MRC receiving CPX-351. (PubMed, Blood Adv) - "In conclusion, the SIE/SIES/GITMO criteria distinguished patient subgroups with different short- and long-term outcomes after treatment with CPX-351. The update or design of dedicated fitness criteria could represent a future and valid strategy to optimize the use of this specific treatment."

Journal • Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology

Tazemetostat and Palbociclib With CPX-351for R/R AML (clinicaltrials.gov) - P1 | N=24 | Recruiting | Sponsor: Thomas Jefferson University | Suspended ➔ Recruiting | Trial completion date: Jan 2026 ➔ Jan 2029 | Trial primary completion date: Jan 2026 ➔ Jan 2028

Enrollment open • Trial completion date • Trial primary completion date • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Case Report: Urinary tract isolation of Cronobacter sakazakii in an oncohaematological patient in Southern Italy. (PubMed, Front Oncol) - "Empirical therapy with piperacillin/tazobactam was initiated, leading to rapid resolution of fever and urinary symptoms...Prompt empirical antimicrobial therapy resulted in rapid clinical improvement and complete recovery. Reporting such cases contributes to awareness of this emerging pathogen in adult oncological patients and underscores the importance of culture-based diagnosis to guide effective management."

Journal • Acute Myelogenous Leukemia • Bladder Cancer • Genito-urinary Cancer • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Nephrology • Oncology • Pain • Solid Tumor

Intermediate doses cytarabine after induction with CPX-351 for patients with secondary AML: safety and efficacy. (PubMed, Ann Hematol) - No abstract available

Journal • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Adverse karyotype amplifies risk in secondary acute myeloid leukemia (AML) and AML with myelodysplasia-related changes. (PubMed, Hematology) - "sAML/AML-MRC with an adverse karyotype had a dismal outcome. These findings provide a benchmark for risk stratification in the pre- CPX-351 era."

Journal • Retrospective data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • 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

A Phase II Study of Combination Daunorubicin, Cytarabine (Ara-c) and Nilotinib (TAsigna) (DATA) in Patients Newly Diagnosed with Acute Myeloid Leukemia with KIT Expression. (PubMed, Am J Hematol) - "Only one patient (3%) died in induction due to liver failure, which was thought secondary to daunorubicin. Our current study reveals good outcomes in patients who received HCT and may warrant a larger study to confirm our findings in that specific population."

Journal • P2 data • Acute Myelogenous Leukemia • Cardiovascular • Febrile Neutropenia • Hematological Malignancies • Hepatology • Hypertension • Liver Failure • Neutropenia • Renal Disease • Transplantation • KIT

CPX-351 versus venetoclax plus hypomethylating agents for newly diagnosed acute myeloid leukemia: A systematic review and meta-analysis. (PubMed, Leuk Res) - "CPX-351 demonstrated superior overall survival (OS) compared to 7 + 3 chemotherapy, while venetoclax and azacitidine significantly improved OS and composite remission rate compared to azacitidine alone...In conclusion, CPX-351 did not demonstrate superiority over Ven/HMA in composite remission rate and OS. Both regimens remain reasonable therapeutic options for older, newly diagnosed AML patients, and prospective comparative studies are needed to better guide treatment selection."

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

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

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