azacitidine / Generic mfg. - LARVOL DELTA

Dosing decitabine and venetoclax for terminal differentiation to improve outcomes in TP53 mutant MDS and AML. (ASCO 2025) - P2 | "Venetoclax (Ven) added to the hypomethylating agents (HMA) of Decitabine or Azacitidine is the current standard of care for elderly patients with AML and is frequently used in high-risk MDS (HR-MDS). In this cohort, of elderly patients with poor risk TP53 mutated MDS and AML the use of a non-cytotoxic dosing schedule of Decitabine and Ven resulted in over half the patients achieving a CR and transfusion independence. The median OS of 11.3 months compares favorably to currently approved cytotoxic dosing of HMA/Ven."

Acute Myelogenous Leukemia • Myelodysplastic Syndrome • TP53

An all-oral regimen of decitabine-cedazuridine (DEC-C) plus venetoclax (VEN) in patients (pts) with newly diagnosed acute myeloid leukemia (AML) ineligible for intensive induction chemotherapy: Results from a phase 2 cohort of 101 pts. (ASCO 2025) - P1/2 | "Clinical Trial Registration Number: NCT04657081 Background: In pts with AML aged ≥75 years and ineligible for induction chemotherapy, the combination of the Bcl-2 inhibitor VEN plus azacitidine (AZA) was approved based on the Phase 3 VIALE-A trial (complete remission [CR] rate, 36.7%; median CR duration, 17.5 months; median overall survival [OS], 14.7 months). The all-oral regimen of DEC-C plus VEN resulted in comparable safety, response, and survival rates to parenteral AZA plus VEN in pts with newly diagnosed AML ineligible for intensive induction chemotherapy. These data support the potential use of DEC-C plus VEN as a treatment option for these pts."

Clinical • P2 data • Acute Myelogenous Leukemia • Anemia • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Leukemia • Neutropenia • Oncology

A phase 1 study of IO-202, an anti-LILRB4 antibody, in chronic myelomonocytic leukemia and acute myeloid leukemia. (PubMed, Blood Neoplasia) - P1 | "Herein, we present the phase 1a dose escalation data of IO-202 as monotherapy and in combination with azacitidine (AZA) in patients with relapsed/refractory (R/R) acute myeloid leukemia (AML) and R/R chronic myelomonocytic leukemia (CMML), and the phase 1b dose expansion data of IO-202 combined with AZA for the treatment of hypomethylating agent (HMA)-naïve CMML. Overall, the data support a future pivotal study of IO-202 + AZA in patients with HMA-naïve CMML. This trial was registered at www.clinicaltrials.gov as #NCT04372433."

Clinical • Journal • P1 data • Acute Myelogenous Leukemia • Chronic Myelomonocytic Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Myeloproliferative Neoplasm • Oncology • Thrombocytopenia • Transplantation • LILRB4

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

Phase I trial of a novel first-in-class drug ONC201 as a post-transplant maintenance for acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) (ASH 2025) - P1 | "GVHD prophylaxis included tacrolimus and methotrexate with or withoutantithymocyte globulin. ONC201 was well tolerated with no observed DLT and a manageable safety profile. The AEprofile was not different from AEs expected in a post-transplant population. Rates of cytopeniaswere low."

Clinical • P1 data • Post-transplantation • Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Hepatology • Immunology • Infectious Disease • Leukemia • Liver Failure • Myelodysplastic Syndrome • Neutropenia • Thrombocytopenia • Transplantation • ASXL1 • BCOR • DRD2 • STAG2 • TP53 • U2AF1

Long-term follow-up of azacitidine, venetoclax, and gilteritinib in patients with newly diagnosed FLT3-mutated Acute Myeloid Leukemia (ASH 2025) - "Continued follow-up of pts with ND FLT3-mutated AML treated with azacitidine, VEN andGILT demonstrates durable responses and encouraging survival. Outcomes appear superior to standardazacitidine and venetoclax, and randomized studies are warranted."

Clinical • Acute Myelogenous Leukemia • Febrile Neutropenia • Infectious Disease • Neutropenia • Septic Shock • BRAF • FLT3 • KRAS • NF1 • NRAS • PTPN11

Phase II study of clinical efficacy of venetoclax in combination with azacitidine in patients with therapy related myelodysplastic syndrome (t-MDS) (ASH 2025) - P2 | "Combination of Aza/Ven was effective in inducing responses in >50% of tMDS pts includingbiallelic TP53 and complex karyotype with no new safety signals. While Aza/Ven did not confer a survivalbenefit in high-risk MDS over Aza alone in the phase 3 VERONA trial, the combination approach may be afeasible strategy to employ as a bridge to transplant in the exceptionally high-risk group of tMDS pts withno other standard of care options."

Clinical • Combination therapy • P2 data • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Infectious Disease • Myelodysplastic Syndrome • Neutropenia • Septic Shock • Thrombocytopenia • TP53

Azacitidine to treat measurable residual disease in patients with MDS/AML: final long-term results of the RELAZA2 trial. (PubMed, Blood) - P2 | "A majority of MRDpos patients can be effectively treated with azacitidine with potential long-term remission even after termination of azacitidine. Clinicaltrials.gov: NCT01462578."

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

Risk stratification of low-dose cytarabine and venetoclax in patients with AML ineligible for intensive chemotherapy. (PubMed, Blood Adv) - P1/2, P3 | "Although the European LeukemiaNet (ELN) classifications (2017, 2022) for AML have been used to stratify outcomes for patients receiving intensive chemotherapy, application to patients receiving less intensive therapy, like azacitidine plus venetoclax, has been less satisfactory. Overall, these data support the importance of molecular sub-classification in defining treatment outcomes to venetoclax-based therapies. NCT02287233; NCT03069352."

Journal • Acute Myelogenous Leukemia • Hematological Malignancies • Infectious Disease • Leukemia • Oncology • FLT3 • KRAS • NPM1 • NRAS • TP53

Impact of venetoclax trough levels on safety and efficacy in the treatment of acute myeloid leukemia. (PubMed, Sci Rep) - "Venetoclax-azacitidine chemotherapy is a key treatment for older or medically unfit patients with acute myeloid leukemia (AML). In the treatment-naïve group, trough levels above 1857.3 ng/mL were associated with a higher composite complete remission rate (77.3% vs. 47.8%, P = 0.042). These findings highlight the need for venetoclax dose optimization based on drug levels to improve both safety and efficacy in AML treatment."

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

Phase 2 Study of Azacitidine plus Pembrolizumab as Second-Line Treatment in Patients with Locally Advanced or Metastatic Pancreatic Ductal Adenocarcinoma. (PubMed, Oncologist) - P2 | "Azacitidine plus pembrolizumab demonstrated limited clinical activity in second line, locally advanced or metastatic PDAC. Biomarker analysis suggests higher baseline CD8+ T-cell infiltration and lower proliferative index may identify patients more likely to benefit. (NCT03264404)."

IO biomarker • Journal • P2 data • Fatigue • Oncology • Pancreatic Cancer • Pancreatic Ductal Adenocarcinoma • Solid Tumor • CD68 • CD8 • GZMB • PD-L1

Phase II Study of Cladribine with Low Dose Cytarabine and Venetoclax Alternating with Azacytidine and Venetoclax for Newly Diagnosed Acute Myeloid Leukemia (ASH 2024) - P2 | "Conclusions : CLAD-LDAC-VEN alternating with AZA + VEN produces an excellent rate of CR/CRi with high rates of MRD negativity , translating into favorable long term OS and EFS in patients aged ≥ 60 yrs or those unfit for intensive chemotherapy. Benefit was seen across most genomically defined subgroups, including those with RAS mutations, where an HMA-Ven alone approach is associated with a mOS of 12 months."

P2 data • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Leukemia • Oncology • DDX41 • DNMT3A • FLT3 • KRAS • NPM1 • TET2 • TP53

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

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

Tagraxofusp, azacitidine, and venetoclax (TAG-AZA-VEN) triplet therapy shows efficacy, tolerability, and transplant potential in patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN): Results of a phase 2 trial (ASH 2025) - P1 | "Triplet therapy with TAG-AZA-VEN is effective and feasible in patients with previouslyuntreated or R/R BPDCN, including in an older patient population. The safety profile is consistent withprior studies of TAG and AZA/VEN, and triplet therapy does not add new AEs not seen with TAG orAZA/VEN. A high proportion of patients (1L and R/R) proceeded to transplant in remission."

Clinical • IO biomarker • P2 data • Atrial Fibrillation • Cardiovascular • CNS Disorders • Diabetes • Febrile Neutropenia • Hematological Malignancies • Infectious Disease • Neutropenia • Renal Disease • Thrombocytopenia • Transplantation • BCL2 • CD123 • IL3RA

Efficacy, molecular and translational analysis of TP53-mutated HR-MDS with bexmarilimab and azacitidine: Updated results from the bexmab Phase 1/2 study (ASH 2025) - "ConclusionBexmarilimab plus azacitidine shows encouraging activity in mTP53 HR MDS and translational datasupports potential for altering the immune dysregulation in mTP53 MDS. Updated clinical andtranslational data will be reported."

Clinical • P1/2 data • Hematological Malignancies • Myelodysplastic Syndrome • AVEN • CD8 • TP53

CA235-0001: A Phase I Study of BMS-986497 alone or in combination with Azacitidine (Aza) and Azacitidine plus Venetoclax (AZA+VEN) in relapsed or refractory (R/R) AML or MDS (clinicaltrialsregister.eu) - P1 | N=35 | Recruiting | Sponsor: Bristol-Myers Squibb Services Unlimited Company | Not yet recruiting ➔ Recruiting

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

Azacitidine and Abatacept in Relapsed or Refractory T-Cell Lymphoma (clinicaltrials.gov) - P1 | N=20 | Recruiting | Sponsor: National Cancer Institute (NCI) | Not yet recruiting ➔ Recruiting

Enrollment open • Adult T-Cell Leukemia-Lymphoma • Follicular Lymphoma • Hematological Malignancies • Leukemia • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Peripheral T-cell Lymphoma • T Cell Non-Hodgkin Lymphoma

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 | Completed | Sponsor: OncoVerity, Inc. | Active, not recruiting ➔ Completed

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

Aclarubicin Plus With Azacitidine and Venetoclax in the Treatment of Acute Myeloid Leukemia (clinicaltrials.gov) - P1/2 | N=112 | Not yet recruiting | Sponsor: Shanghai Jiao Tong University School of Medicine

New P1/2 trial • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • FLT3 • IDH1 • IDH2

A Multicenter RCT of "3+7" vs Venetoclax + CACAG in Newly Diagnosed Mid/High-Risk AML Patients (clinicaltrials.gov) - P2 | N=160 | Active, not recruiting | Sponsor: Chinese PLA General Hospital | Recruiting ➔ Active, not recruiting | Trial primary completion date: Apr 2026 ➔ Sep 2025

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

Venetoclax, Azacitidine, and Mitoxantrone Hydrochloride Liposome Versus Idarubicin and Cytarabine in Newly Diagnosed AML (clinicaltrials.gov) - P3 | N=204 | Not yet recruiting | Sponsor: The First Affiliated Hospital of Soochow University

New P3 trial • Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Oncology

Olutasidenib alone or combined with azacitidine in patients with mutant IDH1 myelodysplastic syndrome. (PubMed, Blood Adv) - P1/2 | "Olutasidenib with or without azacitidine demonstrated encouraging clinical activity and tolerability in patients with higher-risk mIDH1 MDS. NCT02719574."

Journal • Acute Myelogenous Leukemia • Fatigue • Hematological Disorders • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • IDH1

Olutasidenib in Relapsed IDH1 Mutated AML Patients Who Have Previously Received Venetoclax (clinicaltrials.gov) - P2 | N=25 | Not yet recruiting | Sponsor: Timothy Pardee

New P2 trial • Acute Myelogenous Leukemia • IDH1

A CASE REPORT OF MULTI- DRUG COMBINATION THERAPY FOR RELAPSED/REFRACTORY ANGIOIMMUNOBLASTIC T-CELL LYMPHOMA (EBMT 2026) - "The standard first-line treatment for AITL typically involves the CHOP regimen(cyclophosphamide,doxorubicin, vincristine,and prednisone) or CHOP-like protocols... We conducted a single-case retrospective analysis of a 54-year-old male patient with R/R AITL who had progressed following prior treatment with first-line CHOPE (CHOP plus etoposide) and second-line GDP (gemcitabine, doxorubicin liposome and corticosteroid) regimens. After one cycle of chidamide, mitoxantrone, azacitidine and corticosteroid therapy at our linstitution, the original lesions showed significant shrinkage with partial resolution; however, a new lesion emerged, indicating disease progression...The treatment regimen was subsequently modified to a multi-agent combination: chidamide (20 mg twice weekly), decitabine (10 mg d1-5), mitoxantrone liposome (20 mg d2), bevacizumab (600mg day 1), bortezomib (2mg d1, 4, 8) and dexamethasone (20mg d1-4 ), repeated every 21days... Chidamide, a histone deacetylase..."

Case report • Clinical • Combination therapy • IO biomarker • Bone Marrow Transplantation • Immune Modulation • Immunology • Infectious Disease • Lymphoma • Non-Hodgkin’s Lymphoma • Peripheral T-cell Lymphoma • T Cell Non-Hodgkin Lymphoma • Thrombocytopenia • CD7