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

V-FAST: a phase 1b master trial to investigate CPX-351 combined with targeted agents in adults with newly diagnosed AML. (PubMed, Blood Neoplasia) - P1b | "Although few patients received CPX-351 with enasidenib, these results suggest that CPX-351 may be safely combined with venetoclax or midostaurin. This trial was registered at www.clinicaltrials.gov as #NCT04075747."

Journal • P1 data • Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology

How to Select Intensive vs. Lower Intensity Therapy in Younger Patients (SOHO 2025) - "When combined with hypomethylating agent (HMA) therapies (decitabine and azacitidine) in single-arm clinical trials, rates of response among older patients were remarkable, with a composite remission rate (CCR) of 67% and a reported median overall survival (OS) of 17.5 months...These data would include response rates, MRD clearance, bridging to transplant, and survival outcomes. It is very possible that in the not-too-distant future, HMA-venetoclax will serve as the therapeutic backbone for the majority of treatment options offered to AML patients, regardless of age or functional status."

Clinical • IO biomarker • Acute Myelogenous Leukemia • Oncology • FLT3 • IDH1 • IDH2 • NPM1 • TP53

Updates on Therapy Options in Fit and Unfit Patients with Newly Diagnosed AML. (PubMed, Curr Treat Options Oncol) - "In our practice, we continue to use 7 + 3 induction for fit patients, adding midostaurin or quizartinib for FLT3-mutated AML, or gemtuzumab ozogamicin for core binding factor (CBF) AML expressing CD33...In the presence of IDH1 mutations, we consider azacitidine combined with ivosidenib. If venetoclax is contraindicated or not tolerated, targeted therapies such as gilteritinib, ivosidenib, or enasidenib may be appropriate based on mutation profile...When feasible, clinical trial enrollment should be considered for newly diagnosed patients with these alterations. As the therapeutic landscape for AML continues to evolve, timely molecular characterization is more essential than ever to optimize outcomes and select the most appropriate frontline strategy."

Journal • Review • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • CD33 • FLT3 • IDH1 • IDH2 • KMT2A • NPM1

IDH Inhibitor Combination Strategies in IDH Mutated AML (SOHO 2025) - P1/2, P2, P3 | "4–6 Selective inhibitors targeting mutant IDH — including the IDH1 inhibitors ivosidenib (IVO) and olutasidenib (OLU), and the IDH2 inhibitor enasidenib (ENA) — are FDA-approved as single agents for the treatment of relapsed or refractory (R/R) IDH -mutated AML...IDH Inhibitors in Combination with Hypomethylating Agents Given the hypermethylated phenotype induced by mutant IDH enzymes, combining an IDH inhibitor with a hypomethylating agent (HMA), such as azacitidine (AZA), represents a rational therapeutic approach for elderly or unfit individuals with IDH -mutated AML...It is noteworthy that the AZA monotherapy arm demonstrated an impressive median OS, likely due to subsequent AML-directed therapy during follow-up, including the use of ENA and/or venetoclax (VEN) in a substantial percentage of patients...Another promising triplet regimen combines oral decitabine/ cedazuridine (DEC-C) with VEN and either IVO or ENA for IDH1- or IDH2 -mutated AML,..."

IO biomarker • Acute Myelogenous Leukemia • Oncology • IDH1

Evolving Paradigms in Acute Myeloid Leukemia: Personalized Approaches to Therapy Across Age and Risk Groups. (PubMed, Cancers (Basel)) - "Older or unfit individuals benefit from low-intensity treatments such as hypomethylating agents combined with venetoclax, now considered a frontline standard of care...Emerging therapies, including menin inhibitors, antibody-drug conjugates, and immunotherapies like CAR-T cells and vaccines, offer additional options, especially in relapsed/refractory settings. This comprehensive review outlines the current landscape and future directions in AML therapy, emphasizing the transition toward individualized, mutation-driven treatment strategies."

IO biomarker • Journal • Review • Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Oncology • FLT3

NCI-2019-03712: Testing the Effect of Taking Ruxolitinib and CPX-351 in Combination for the Treatment of Advanced Phase Myeloproliferative Neoplasms (clinicaltrials.gov) - P1/2 | N=12 | Completed | Sponsor: Ohio State University Comprehensive Cancer Center | Active, not recruiting ➔ Completed | Trial completion date: Jun 2025 ➔ Feb 2025

Trial completion • Trial completion date • Acute Myelogenous Leukemia • Essential Thrombocythemia • Hematological Disorders • Hematological Malignancies • Leukemia • Myelofibrosis • Myeloproliferative Neoplasm • Oncology • Polycythemia Vera • Thrombocytosis

Optimising Transplant Potential in older AML Patients: Insights from Real-World Data (ICBMT 2025) - "In the actual lecture, we will review the data on CPX-351 with a special focus on the sequence of CPX-351-based induction followed by allo-SCT. We will further give an update on our real-world analysis from Germany and practical advice, how to use CPX-351 in clinical routine."

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

NCI-2018-01812: Liposome-encapsulated Daunorubicin-Cytarabine and Gemtuzumab Ozogamicin in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia (AML) or High Risk Myelodysplastic Syndrome (clinicaltrials.gov) - P2 | N=50 | Active, not recruiting | Sponsor: M.D. Anderson Cancer Center | Recruiting ➔ Active, not recruiting

Enrollment closed • Acute Myelogenous Leukemia • Chronic Myelomonocytic Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • CD33

CPX-351 in Combination With Ruxolitinib for the Treatment of Myeloproliferative Neoplasm-Accelerated/Blast Phase (SOHO 2025) - "CPX-351+Rux appears tolerable with no new safety signals. Median OS compares favorably to historical 7+3 data, but response rates remain modest, highlighting the need for novel therapeutics in MPN-AP/BP."

Combination therapy • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Myeloproliferative Neoplasm • Oncology

Case of CPX-351-Induced Purpuric Rash Occurred in a Patient With Pure Erythroid Leukemia. (PubMed, J Dermatol) - No abstract available

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

VyClo: Vyxeos® With Clofarabine for Pediatric AML (clinicaltrials.gov) - P1 | N=25 | Recruiting | Sponsor: Princess Maxima Center for Pediatric Oncology

New P1 trial • Acute Myelogenous Leukemia • Pediatrics

V-RULES: Impact of Treatment Setting on CPX-351 Safety and Effectiveness in Secondary Acute Myeloid Leukemia (SOHO 2025) - "Outpatient delivery of CPX-351 was feasible, especially after first induction, with a reduction in hospitalization incidence and duration, and was not associated with increased adverse events compared to inpatient treatment. These data provide insights into real-world CPX-351 use in U.S. patients with t-AML or AMLMRC, highlighting an opportunity for outpatient treatment for some patients."

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

Future Role of CPX-351 and CPX-351-Based Combinations (SOHO 2025) - No abstract available

Acute Myelogenous Leukemia • Oncology

Safety of liposomal daunorubicin-cytarabine (CPX-351) in secondary AML: Japanese phase 1/2 study and global phase 3 study. (PubMed, Int J Hematol) - "In the global study, univariate analysis was performed after one cycle of induction therapy to investigate factors that delay neutrophil and platelet recovery, but no specific factors were identified. In these analyses, the pattern and frequency of adverse events were similar in Japanese and non-Japanese patients, with no need for race- or region-specific management."

Journal • P1/2 data • P3 data • Acute Myelogenous Leukemia • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Neutropenia • Oncology • Pneumonia • Respiratory Diseases

Future treatment strategies for acute myeloid leukemia (PubMed, Rinsho Ketsueki) - "This review outlines the significant transformation in AML management after the introduction of azacitidine plus venetoclax, FLT3 inhibitors, and CPX-351...The current paradigm in AML treatment has shifted toward strategic personalization, encompassing molecular abnormality identification, measurable residual disease assessment, and treatment adaptation based on these findings. To achieve wider adoption of precision medicine in clinical practice, Japan must continue strengthening its diagnostic systems, streamlining genomic testing in routine practice, and integrating these strategies with novel therapeutic development."

Journal • Review • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • Transplantation • FLT3 • MECOM • TP53

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

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

Postbiotics: Modulation of the Gut Microbiota and Potential for Association with Nanotechnology. (PubMed, Probiotics Antimicrob Proteins) - "In the pharmaceutical industry, several nanotechnology-based delivery systems have already been approved by the FDA and successfully marketed, demonstrating their clinical and industrial viability, such as Abraxane®, Doxil® (or Caelyx®), Onivyde®, Marqibo®, and Vyxeos®...However, although nanotechnology in the delivery of postbiotics is an emerging and innovative field with promising preclinical studies, the lack of consolidated clinical or commercial data means that this topic needs to be expanded in order to achieve standardization and clinical validation. This review aims to highlight the importance of postbiotics in the modulation of gut microbiota and in the prevention/treatment of disease, as well as the potential of using nanotechnology to facilitate the targeting of these compounds and the limitations of using these systems for such applications."

Journal • Review • Gastrointestinal Disorder

Palbociclib or Tazemetostat in Combination With CPX-351 for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia (clinicaltrials.gov) - P1 | N=24 | Suspended | Sponsor: Thomas Jefferson University | Recruiting ➔ Suspended

Trial suspension • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

CPX-351 as a Novel Approach for the Treatment of Older Patients With AML and MDS (clinicaltrials.gov) - P2 | N=13 | Active, not recruiting | Sponsor: Case Comprehensive Cancer Center | Trial completion date: Jun 2027 ➔ Oct 2026

Trial completion date • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology

CPX-351 as a Novel Approach for the Treatment of Older Patients With AML and MDS (clinicaltrials.gov) - P2 | N=13 | Active, not recruiting | Sponsor: Case Comprehensive Cancer Center | Trial primary completion date: Jun 2025 ➔ Sep 2025

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

Phase I/II Clinical Trial of Proteasome Inhibitor in Combination With CPX-351 for the Treatment of Newly-Diagnosed TP53-mutated Acute Myeloid Leukemia (AML) (clinicaltrials.gov) - P1/2 | N=32 | Recruiting | Sponsor: Masonic Cancer Center, University of Minnesota | Not yet recruiting ➔ Recruiting

Enrollment open • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Study of Standard Intensive Chemotherapy Versus Intensive Chemotherapy With CPX-351 in Adult Patients With Newly Diagnosed AML and Intermediate- or Adverse Genetics (clinicaltrials.gov) - P3 | N=882 | Active, not recruiting | Sponsor: University of Ulm | Recruiting ➔ Active, not recruiting

Enrollment closed • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology

Induction chemotherapy with CPX-351 in acute myeloid leukemia: revisiting the role of early bone marrow assessment. (PubMed, Leukemia) - No abstract available

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

MYCOTIC ANEURYSM AS A FATAL COMPLICATION IN A PATIENT WITH ACUTE LEUKEMIA: A CASE REPORT (EHA 2025) - "However, blood cultures showed growth of the filamentous fungus Scedosporium (Lomentospora) prolificans, prompting the initiation of antifungal therapy with Isavuconazole.Subsequently, the patient developed neurological symptoms, including headache, dysarthria, deviation of the labial commissure, and a non-reactive right pupil... Patients with Acute Myeloid Leukemia (AML) may develop life-threatening complications, particularly in the context of neutropenia following intensive chemotherapy.Mycotic aneurysm is a critical complication that should be considered in immunocompromised patients presenting with infectious and neurological symptoms.A high index of clinical suspicion and detailed radiological review are essential for early diagnosis and appropriate management."

Case report • Clinical • Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Neutropenia • Ocular Infections • Ocular Inflammation • Oncology • Ophthalmology • Pain • Subarachnoid Hemorrhage

A PHASE I/II, OPEN-LABEL, MULTI-CENTER STUDY EVALUATING THE SAFETY AND EFFICACY OF RUXOLITINIB AND CPX-351 IN COMBINATION FOR THE TREATMENT OF ADVANCED PHASE MYELOPROLIFERATIVE NEOPLASMS (EHA 2025) - "Use of intensive induction chemotherapy in MPN-BP is associated with poor overall survival (OS) (median OS 3.9 months (mo); Mesa et al, Blood 2005), whereas decitabine with ruxolitinib (Rux) has shown early efficacy with a median OS of 9.4 mo in a phase 2 study (Mascarenhas et al, Blood Adv 2020)... The combination of CPX-351 + Rux was feasible and well tolerated with a safety profile similar to CPX-351 alone. Serious bleeding event was the main DLT at both rux dose levels which is a known risk associated with CPX-351. Median OS compares favorably to historical controls treated with "7+3" induction alone, but the study is limited by small numbers."

Clinical • Metastases • P1/2 data • Anemia • Cardiovascular • Cerebral Hemorrhage • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Myelodysplastic Syndrome • Myeloproliferative Neoplasm • Neutropenia • Oncology • Thrombocytopenia