azacitidine / Generic mfg. - LARVOL DELTA

Potential role of miR-29b from mesenchymal stromal cell-derived extracellular vesicles in leukemic cell progression. (PubMed, PLoS One) - "EVs isolated from HS-5, an immortalized stromal cell line, promoted the proliferation and 5-azacytidine (AZA) resistance of SKM-1 cells...When we transduced miR-29b into leukemic cells, these cells demonstrated greater resistance to AZA and venetoclax than did their parental cells, mirroring the effect observed with HS-5 EVs. The introduction of miR-29b down-regulated DNMT1, DNMT3L, TET1, and TET2, which may underlie the changes in DNA methylation levels, increased chromosomal instability, activation of type I interferon pathways, acquired cell migration, and drug resistance observed in leukemic cells. Overall, we suggest that the sustained influx of miR-29b through EVs may catalyze the evolution of leukemic cells, and its clinical relevance warrants further investigation."

Journal • Hematological Malignancies • Myelodysplastic Syndrome • Oncology • DNMT1 • TET2

Macrophage reprogrammer Bexmarilimab Plus Azacitidine in Myelodysplastic Syndrome: PK/PD and biomarker results from the Phase 1/2 BEXMAB Study (ESMO 2025) - No abstract available

Biomarker • P1/2 data • PK/PD data • Hematological Malignancies • Myelodysplastic Syndrome • Oncology

Successful remission induction therapy with azacitidine and venetoclax for a treatment-naive elderly patient with ETP/myeloid mixed-phenotype acute leukemia: a case report. (PubMed, Front Oncol) - "Consequently, the administration of subsequent doses of venetoclax/azacitidine for induction therapy was delayed, and nirmatrelvir/ritonavir was given as therapy for COVID-19. We report on an elderly patient with MPAL treated with venetoclax combined with azacitidine. This regimen successfully induced complete remission with no adverse side effects, and despite testing positive for COVID-19 multiple times during induction therapy, accompanied by mild dry cough but no radiographic evidence of pneumonia, the patient remained clinically stable."

Journal • Acute Lymphocytic Leukemia • Cough • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Novel Coronavirus Disease • Oncology • Pneumonia • Respiratory Diseases • DNMT3A • JAK3 • MPO • NOTCH1

Pilot Study of Reduced Venetoclax Exposure (clinicaltrials.gov) - P2 | N=41 | Recruiting | Sponsor: Northwell Health

New P2 trial • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

NCI-2018-01919: Enasidenib and Azacitidine in Treating Patients With Recurrent or Refractory Acute Myeloid Leukemia and IDH2 Gene Mutation (clinicaltrials.gov) - P2 | N=50 | Recruiting | Sponsor: M.D. Anderson Cancer Center | Trial completion date: Sep 2025 ➔ Sep 2027 | Trial primary completion date: Sep 2025 ➔ Sep 2027

Trial completion date • Trial primary completion date • Acute Myelogenous Leukemia • Chronic Myelomonocytic Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • IDH2

NCI-2018-00921: Ivosidenib and Venetoclax With or Without Azacitidine in Treating Patients With IDH1 Mutated Hematologic Malignancies (clinicaltrials.gov) - P1/2 | N=96 | Recruiting | Sponsor: M.D. Anderson Cancer Center | Trial completion date: Sep 2025 ➔ Sep 2027 | Trial primary completion date: Sep 2025 ➔ Sep 2027

IO biomarker • Trial completion date • Trial primary completion date • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Myeloproliferative Neoplasm • Oncology • IDH1

NCI-2020-14163: Seclidemstat and Azacitidine for the Treatment of Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia (clinicaltrials.gov) - P1/2 | N=44 | Recruiting | Sponsor: M.D. Anderson Cancer Center | Trial completion date: Sep 2025 ➔ Sep 2027 | Trial primary completion date: Sep 2025 ➔ Sep 2027

Trial completion date • Trial primary completion date • Chronic Myelomonocytic Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • ASXL1 • NRAS • RUNX1 • SETBP1 • TP53

Primary Acute Myeloid Leukemia with Concomitant BCR∷ABL and NPM1 Mutation. (PubMed, Clin Lab) - "AML with BCR∷ABL cases are relatively rare, and the establishment of standardized diagnostic and treatment strategies is still lacking. Our research findings emphasize the importance of including BCR∷ABL detection in the diagnostic examination of AML to enable the provision of the most appropriate risk classification and treatment options for patients."

Biomarker • IO biomarker • Journal • Acute Myelogenous Leukemia • Hematological Malignancies • Infectious Disease • Leukemia • Oncology • ABL1 • ANPEP • BCR • CD123 • CD33 • CD38 • CD7 • CDKN2A • IL3RA • ITGAM • KMT2C • NCAM1 • NOTCH2 • NPM1 • USH2A

Venetoclax Combined With Azacitidine, Chidamide, Vindesine, and Dexamethasone in Newly Diagnosed ETP-ALL Like Patients (clinicaltrials.gov) - P2 | N=27 | Recruiting | Sponsor: The First Affiliated Hospital of Soochow University

New P2 trial • Adult T-Cell Leukemia-Lymphoma • Hematological Malignancies • Leukemia • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology

A Case of Hepatosplenic T-Cell Lymphoma: A Rare and Aggressive Subtype of Peripheral T-Cell Lymphoma (SOHO 2025) - "Interventions: The patient received four cycles of HyperCVAD (cyclophosphamide, vincristine, doxorubicin, dexamethasone alternating with high-dose cytarabine and methotrexate). He had an initial response but had relapsed shortly after and subsequently received one cycle of ICE (ifosfamide, carboplatin, etoposide) with persistent disease...He was started on a combination of romidepsin and azacitidine, which he could not tolerate due to cytopenia... HSTCL is a fatal malignancy. Continued research and therapeutic advancement are needed to improve outcomes."

Clinical • IO biomarker • Hematological Malignancies • Hepatosplenic T-cell Lymphoma • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Peripheral T-cell Lymphoma • T Cell Non-Hodgkin Lymphoma • CD4 • CD5 • CD8 • NCAM1 • STAT5B

Dose-Reduced Venetoclax-Azacitidine in Elderly Acute Myeloid Leukemia: A Real-World Experience (SOHO 2025) - "Four patients required cytoreduction with hydroxyurea ± cytarabine for hyperleukocytosis. In this real-world Latin American cohort, shortened-duration VEN-AZA was feasible and highly effective, reproducing VIALE-A remission rates even in frail elderly patients. Curtailing VEN exposure appeared to lessen infectious complications without jeopardizing remission outcomes. Interpretation is limited by sample size and retrospective design."

Clinical • Real-world • Real-world evidence • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • FLT3 • IDH1 • IDH2 • NPM1 • TP53

Individualized Acute Myeloid Leukemia Management in an Older Adult Patient With a Mechanical Heart Valve: A Case of Targeted Cytoreduction and Modified Maintenance (SOHO 2025) - "Cytoreduction was initiated with hydroxyurea and a single dose of gemtuzumab ozogamicin. Upon control of white blood cells, azacitidine and venetoclax were started... This specific case demonstrates that, with individualized therapy, it is feasible to achieve long-lasting, durable remission in AML in older patients with significant comorbidities. Gemtuzumab-assisted cytoreduction followed by modified decitabine/venetoclax enabled long-term disease control. A successful outcome for this high-risk patient required careful coordination of anticoagulation and supportive care."

Clinical • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • KRAS • NPM1 • SRSF2

New Therapies and Targeted Treatments: MDS/ MPN Overlap Syndromes (SOHO 2025) - "This has led to the use of parenteral azanucleosides — decitabine and azacitidine — and, perhaps most successfully in the United States, the oral agent decitabine/ cedazuridine (Dec-C)...While decitabine led to improved response rates, it failed to show benefit in event-free survival (EFS) or overall survival (OS) over the hydroxyurea arm, 5 raising questions about the role of azanucleosides — at least in MP-CMML — and underscoring the urgent need for disease-specific therapies for patients with MPCMML and other MDS/MPNs...This yielded promising responses, 8 but the arm was closed, as itacitinib was discontinued by the manufacturer...9 Given these findings, in the phase 2 PREACH study in Australia comparing lenzilumab plus azacitidine in untreated, high-risk RAS- mutated CMML, investigators reported a 55% complete response (CR) rate, found to be durable at 18 months...STX-0712 is a cytotoxicity targeting antibody (CyTAC) that links an antibody designed to induce..."

IO biomarker • Acute Myelogenous Leukemia • Chronic Myeloid Leukemia • Chronic Myelomonocytic Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Myeloproliferative Neoplasm • Oncology • ABL1 • BCR • CCL2 • CCR2 • CSF2 • LILRB4 • SF3B1

Moving Beyond Romidepsin: New Strategies for Relapsed/Refractory PTCL (SOHO 2025) - "Current FDA-approved agents include pralatrexate, 2 belinostat, 3 and brentuximab vedotin...In this study, romidepsin plus CHOP (cyclophosphamide, doxorubicin, vincristine, prednisone) was compared to CHOP in untreated T-cell lymphomas (TCLs) but failed to show an improvement in progression-free survival (PFS) — the primary endpoint of this study 6 Brentuximab vedotin has the highest response rate of 87% in R/R anaplastic large cell lymphoma (ALCL), 4 but the response rates are lower in other subtypes...7 Duvelisib — a dual PI3K delta/ gamma inhibitor — has shown an overall response rate (ORR) of 48%, a complete response (CR) rate of 33%, and a median duration of response of 7.89 months...Other PI3K inhibitors include tenalisib 10 and linperlisisb, 11 which demonstrate similar results...Inhibitors of the JAK/ STAT pathway like ruxolitinib are showing promising results in PTCL — both as single agents and in combination...The ORACLE study compared 5-azacitidine against..."

Oncology • Peripheral T-cell Lymphoma • CD5 • CD7 • CD70 • EZH2 • GATA3 • IL2RA • KLRD1 • SIRPA • TNFRSF8

From Low Risk to Blast Crisis: A Case for Mastering Hematological Malignancies (SOHO 2025) - "Leukemia was suspected, and he was treated with hydroxyurea for cytoreduction...His intended treatment was azacitidine, planned to begin 1 week after presentation...A thorough review of CBC results and differential at each presentation is key in the early identification of a potential blast cell crisis. Our case demonstrates the importance of a comprehensive understanding of hematologic malignancies and how recognizing the signs and symptoms of disease progression and crises is a skill relevant to all physicians."

Clinical • Chronic Myelomonocytic Leukemia • Hematological Malignancies • Leukemia • Oncology

Gilteritinib Experience in Relapsed/Refractory FLT3-Mutated AML Patients After Allogeneic Transplant: Two Cases (SOHO 2025) - "After an unsuccessful FLAG-IDA (combination of fludarabine, cytarabine, and granulocyte colony stimulating factor with idarubicin) induction therapy, one course of EMA (etoposide, mitoxantrone, Ara-C) therapy was administered. Following no response to EMA, the patient started azacitidine and gilteritinib 120 mg/day in December 2024...After an unsuccessful FLAG-IDA induction therapy, gemtuzumab therapy followed by gilteritinib 120 mg/day was initiated...The patient developed liver graft-vs-host disease (GVHD), and therapy was paused. She remains in hematological remission."

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

A Rare Presentation of Acute Myeloid Leukemia Associated With Philadelphia Chromosome (SOHO 2025) - "Hematology-oncology recommended leukapheresis and hydroxyurea for cytoreduction...The patient started azacitidine/venetoclax induction but failed to achieve response with increasing peripheral blasts...He started cladribine, low-dose cytarabine (LDAC), and dasatinib...He continued dasatinib with excellent disease control for 2 years before switching to asciminib due to edema...Our case highlights prolonged remission with TKI monotherapy after LDAC induction, suggesting that patients may achieve sustained disease control without transplantation. Further research is needed to evaluate TKI monotherapy outcomes and identify factors predicting durable response in Ph+ AML."

Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • ABL1 • BCR

CULMINATE: A Study of Cusatuzumab Plus Azacitidine in Participants With Newly Diagnosed Acute Myeloid Leukemia Who Are Not Candidates for Intensive Chemotherapy (clinicaltrials.gov) - P2 | N=103 | Active, not recruiting | Sponsor: OncoVerity, Inc. | Trial completion date: Aug 2025 ➔ May 2026

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

Durable response to venetoclax, azacytidine, and ruxolitinib in chronic-phase myelofibrosis resistant to ruxolitinib: a case report and literature review. (PubMed, Front Oncol) - "After two courses of treatment with venetoclax and azacytidine combined with ruxolitinib, the patient has been continuously treated with oral ruxolitinib and has achieved a therapeutic effect for more than 1 year. The treatment response of this patient we reported provides a new safe and effective treatment method for MF-CP patients who are no longer responsive to ruxolitinib."

Journal • Myelofibrosis • Oncology • Polycythemia Vera

Phase II Study of Combination Azacitidine and Entinostat and Nivolumab in Patients With Metastatic Non-Small Cell Lung Cancer (IASLC-WCLC 2025) - P2 | "Further correlative evaluation, including in-depth genomic and transcriptomic analyses, are ongoing to accurately identify patients most likely to benefit from this therapeutic approach. Final integration of these translational studies with clinical outcomes will work to identify the biologic effect of epigenetic therapy in re-shaping the tumor microenvironment to personalize this epigenetic combinatorial approach."

Clinical • Metastases • P2 data • Gene Therapies • Lung Cancer • Non Small Cell Lung Cancer • Oncology • Pulmonary Disease • Solid Tumor

Vorinostat Dose-escalation After Allogeneic Hematopoietic Cell Transplantation (clinicaltrials.gov) - P1 | N=15 | Active, not recruiting | Sponsor: Johns Hopkins All Children's Hospital | Recruiting ➔ Active, not recruiting

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

PEMAZA: MRD-guided Treatment in NPM1mut AML Patients (clinicaltrials.gov) - P2 | N=26 | Terminated | Sponsor: Technische Universität Dresden | Trial completion date: Dec 2026 ➔ Feb 2025 | Recruiting ➔ Terminated | Trial primary completion date: Dec 2025 ➔ Feb 2025; Feasibility

Trial completion date • Trial primary completion date • Trial termination • Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Oncology • NPM1

Checkpoint immunotherapy is associated with preferential activation of tumor antigen-specific CD4+ T cells in MDS. (PubMed, Blood Neoplasia) - P1 | "Based upon this hypothesis, early phase trials combining immune checkpoint inhibitors (ICIs) with azacitidine in patients with myelodysplastic syndrome (MDS)/acute myeloid leukemia (AML) were undertaken, but clinical and immunologic efficacy have proven disappointing...We hypothesized that combining vaccination against the New York esophageal squamous cell carcinoma 1 (NY-ESO-1) tumor antigen with decitabine and an ICI would allow us to understand antigen-specific immune responses in patients with MDS. To test this hypothesis, we developed an investigator-initiated phase 1 trial in transplant-ineligible patients with MDS/low blast count AML incorporating the anti-programmed cell death protein-1 (PD-1) ICI nivolumab...Approaches to augment the number and function of cDC1 populations in myeloid disease might overcome this defect and enhance the efficacy of immunotherapy for patients with MDS. This trial was registered at www.ClinicalTrials.gov as #NCT03358719."

Clinical • IO biomarker • Journal • Acute Myelogenous Leukemia • Esophageal Cancer • Esophageal Squamous Cell Carcinoma • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • Squamous Cell Carcinoma • Transplantation • CD4 • CTAG1B

γ9δ2 T-Cell Activation (γδTCA) With ICT01 and Azacitidine-Venetoclax (Aza-Ven) Induces High Rates of Complete Remission (CR) in Newly Diagnosed (ND) Acute Myeloid Leukemia (AML): Preliminary Results of the Phase 1/2 Study EVICTION (SOHO 2025) - P1/2 | "ICT01 was safe and well tolerated and generated very high CR and CR/CRi rates in older/unfit NDAML patients. The recommended phase 2 dose is 10 mg ICT01 Q4W with Aza-Ven. The high response rates seen in adverse-risk patients warrant further clinical investigation."

Clinical • P1/2 data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • IFNG • KRAS • TNFA • TP53

Co-Targeting in CML (SOHO 2025) - P2 | "Historically, the French SPIRIT and German CML IV studies combined imatinib with either interferon alpha or low-dose cytarabine...More recently, the German TIGER trial 9 and Nordic BosuPEG trial (NCT03831776; www.clinicaltrials.gov ) have combined nilotinib or bosutinib, respectively, with pegylated interferon...The phase 1 dose-escalation study showed promising results in terms of efficacy when asciminib was combined with imatinib, dasatinib, or nilotinib, with rapid achievement of response, but tolerability was inferior to asciminib alone...Combination approaches have included lineage agnostic strategies such as FLAG-IDA chemotherapy in combination with ponatinib, 24 or decitabine, ponatinib, and venetoclax, 25 or ponatinib plus azacitidine for myeloid BP-CML (NCT03895671; www.clinicaltrials.gov )...26,27 These include the use of blinatumomab with TKIs in multi-drug approaches...Clinical research in CML remains very active, but we must be cautious and respect the..."

IO biomarker • Acute Lymphocytic Leukemia • B Cell Lymphoma • Chronic Myeloid Leukemia • Lymphoma • Oncology • ABL1 • MYC