azacitidine / Generic mfg. - LARVOL DELTA

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

Efficacy and Safety of Venetoclax Plus Azacitidine for Patients With Treatment-Naive High-Risk Myelodysplastic Syndromes. (PubMed, Blood) - P1, P3 | "Overall, venetoclax plus azacitidine at the RP2D was well tolerated and had favorable outcomes. A phase 3 study (NCT04401748) is ongoing to confirm survival benefit of this combination."

Journal • Bone Marrow Transplantation • Constipation • Febrile Neutropenia • Gastroenterology • Gastrointestinal Disorder • Hematological Disorders • Hematological Malignancies • Myelodysplastic Syndrome • Neutropenia • Oncology • Thrombocytopenia • Transplantation

ALL-ORAL DECITABINE-CEDAZURIDINE (DEC-C) + VENETOCLAX (VEN) IN PATIENTS WITH NEWLY DIAGNOSED ACUTE MYELOID LEUKEMIA (AML) INELIGIBLE FOR INDUCTION CHEMOTHERAPY: PHASE 1/2 CLINICAL TRIAL RESULTS (EHA 2025) - P1/2 | "Background: In patients (pts) with AML aged ≥75 years or otherwise ineligible for induction chemotherapy (ICT), the combination of azacitidine (AZA) with the Bcl-2 inhibitor VEN is approved based on the VIALE-A trial (complete remission [CR] rate, 36.7%; median overall survival [mOS], 14.7 months); intravenous (IV) decitabine (DEC) + VEN is also approved in this setting. The all-oral regimen of DEC-C + VEN resulted in comparable safety, response, and survival rates to parenteral AZA + VEN in pts with newly diagnosed AML ineligible for ICT. Compared with standard dosing, early BM examination and subsequent dose reductions in DEC-C and/or VEN during post-remission treatment cycles were associated with improved long-term outcomes and tolerability. These data underscore the importance of regimen optimization and suggest a practice-changing role for oral DEC-C + VEN in this pt population with high unmet need."

Clinical • P1/2 data • Acute Myelogenous Leukemia • Anemia • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Leukemia • Neutropenia • Oncology

Outcomes of Frontline Triplet Regimens With a Hypomethylating Agent, Venetoclax, and Isocitrate Dehydrogenase Inhibitor for Intensive Chemotherapy-Ineligible Patients With Isocitrate Dehydrogenase-Mutated AML. (PubMed, J Clin Oncol) - P1/2 | "Given the excellent outcomes of IDH-triplet therapy for newly diagnosed, IC-ineligible IDH-mutant AML, further prospective studies comparing IDH-triplet versus IDH-doublet regimens are warranted."

Journal • Acute Myelogenous Leukemia • Transplantation • IDH1 • IDH2

Azacitidine and Venetoclax in Previously Untreated Acute Myeloid Leukemia. (PubMed, N Engl J Med) - P3 | "The incidence of febrile neutropenia was higher in the venetoclax-azacitidine group than in the control group. (Funded by AbbVie and Genentech; VIALE-A ClinicalTrials.gov number, NCT02993523.)."

Journal • P3 data • Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Neutropenia • Oncology • Thrombocytopenia

Magrolimab plus azacitidine vs physician's choice for untreated TP53-mutated acute myeloid leukemia: the ENHANCE-2 study. (PubMed, Blood) - P3 | "Patients determined inappropriate for intensive therapy were randomized to receive Magro/Aza or venetoclax plus azacitidine (Ven/Aza); those appropriate for intensive therapy were randomized to receive Magro/Aza or 7+3 induction chemotherapy. ENHANCE-2 did not meet its primary endpoint of OS in TP53-mutated AML but provides important data informing future studies in this challenging population. This trial was registered at www.clinicaltrials.gov as #NCT04778397."

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

AZACITIDINE, VENETOCLAX, AND REVUMENIB FOR NEWLY DIAGNOSED OLDER ADULTS WITH ACUTE MYELOID LEUKEMIA (AML) AND NPM1 MUTATION OR KMT2A REARRANGEMENT: UPDATED RESULTS FROM THE BEAT AML CONSORTIUM (EHA 2025) - P1/2, P3 | "AVR has demonstrable clinical activity with manageable safety in ND older adults with NPM1m or KMT2Ar AML with ORR of 88% in all pts and 100% in response-evaluable pts within 2 cycles of treatment. A randomized phase 3 study (NCT06652438) of AVR vs. AV-placebo in ND older adults with NPM1m is in development based on these findings."

Clinical • Acute Myelogenous Leukemia • Hematological Malignancies • Infectious Disease • Leukemia • Oncology • Respiratory Diseases • Septic Shock • KMT2A • NPM1

Venetoclax (VEN) and azacitidine (AZA) with gilteritinib (GILT) in patients with newly diagnosed (ND) FLT3mut+ Acute Myeloid Leukemia (AML) ineligible for intensive induction chemotherapy (chemo): Interim results from the phase 1/2 VICEROY study (ASH 2025) - P1/2 | "Dosing holds wereclosely monitored during C1 after marrow response, and VEN/AZA/GILT duration was reduced onsubsequent cycles after remission. The phase 2 portion is ongoing and enrolling patients."

Clinical • P1/2 data • Acute Myelogenous Leukemia • Cardiovascular • Febrile Neutropenia • Neutropenia • Thrombocytopenia • FLT3

Macrophage reprogrammer bexmarilimab plus azacitidine in myelodysplastic syndrome: PK/PD and biomarker results from the phase I/II BEXMAB study (ESMO 2025) - P1/2 | "In addition to these translational results, full pharmacokinetic (PK) and pharmacodynamic (PD, including correlation with clinical response) data will be presented for the first time. Conclusions Together, the clinical and biological findings highlight bexmarilimab's potential as a novel therapeutic strategy for HR MDS and support its advancement into phase 3."

Biomarker • P1/2 data • PK/PD data • Hematological Malignancies • Myelodysplastic Syndrome • Oncology • AVEN • CD8

Long-term results from the AGILE study of azacitidine plus ivosidenib vs placebo in newly diagnosed IDH1-mutated AML. (PubMed, Blood Adv) - P3 | "These long-term efficacy and safety results confirm the benefit of ivosidenib-azacitidine in this challenging-to-treat population and support its use as a standard of care with the longest reported survival benefit for intensive chemotherapy-ineligible patients with IDH1-mutated AML. ClinicalTrials.gov registration ID: NCT03173248."

Journal • Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Oncology • IDH1

Efficacy and safety of pivekimab sunirine in combination with venetoclax plus azacitidine in unfit patients with newly diagnosed Acute Myeloid Leukemia (ASH 2025) - "Unfit pts with newly diagnosed CD123-postive AML demonstrated high CR rates withPVEK+VEN+AZA. These robust response rates were observed across the select mutational profiles tested.Follow up with duration of response and survival estimates among all pts, by molecular subgroups andMRD negativity status, are being evaluated. Triplet therapy in unfit 1L pts with CD-123–positive AML waswell-tolerated, and no new safety signals were observed."

Clinical • Combination therapy • Acute Myelogenous Leukemia • Constipation • Gastroenterology • Gastrointestinal Disorder • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Myocardial Infarction • Neutropenia • Respiratory Diseases • Thrombocytopenia • CD123 • IL3RA

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

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

Updated response and safety analyses from a Phase 1 study of ivosidenib combined with intensive chemotherapy in patients with newly diagnosed (ND) Acute Myeloid Leukemia with isocitrate dehydrogenase (IDH)1 mutation (ASH 2025) - P1, P3 | "Introduction Ivosidenib (IVO) is approved as monotherapy and in combination with azacitidine for frontline treatmentof patients (pts) with mIDH1 acute myeloid leukemia (AML) unfit for intensive chemotherapy (chemo)...Pts with ND mIDH1AML received induction therapy: cytarabine 200 mg/m2/d × 7 d and either daunorubicin 60 mg/m2/d oridarubicin 12 mg/m2/d × 3 d (up to 2 cycles of induction were permitted) and IVO 500 mg once dailystarting on d 1 of induction therapy...IVO maintenance has an acceptable safety profile, is associated with stablenormalization of blood counts, and results in durable responses and long-term survival acrosscomutational profiles. The benefit of this frontline regimen is being assessed in a phase 3 randomized,blinded trial (NCT03839771)."

Clinical • P1 data • Acute Kidney Injury • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Disorders • Hematological Malignancies • Leukemia • Leukopenia • Neutropenia • Renal Disease • Thrombocytopenia • TP53

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

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

Prognostic impact of cognitive and other impairments in older adults with acute myeloid leukemia (AML) participating in a geriatric assessment-guided treatment selection trial (ASH 2025) - P2 | "Based on the protocol, eightpatients (11%) received intensive chemotherapy; others received low-intensity chemotherapy: azacitidineor decitabine alone (prior to approval of venetoclax) or in combination with venetoclax. Older adults with newly diagnosed AML and normal cognitive function have lower risk ofmortality. Patients with 0-3 vs. 4 or more impairments in GA domains have lower risk of mortality.Multidimensional assessments including cognitive testing at AML diagnosis can be useful forprognostication."

Clinical • Acute Myelogenous Leukemia • Alzheimer's Disease • CNS Disorders • Cognitive Disorders • Depression • Hematological Malignancies • Leukemia • Solid Tumor

Transfusion independence, hematological improvement and associated safety outcomes with bexmarilimab and azacitidine in HR-MDS: Results of the bexmab Phase 1/2 study (ASH 2025) - "In addition, pre-clinical datafrom Clever-1 knock-out and anti-Clever-1 treated mice suggests improved hematopoiesis andhematological recovery after 5-fluorouracil based chemotherapy. Altogetherthese data suggest that Clever-1 inhibition with the bexmarilimab combination enables hematopoieticrecovery in MDS patients. ConclusionMaintenance of baseline TI status, increased TI rate and increased number of BM progenitor cellsproducing platelets and RBCs, support bexmarilimab's unique mechanism of action in HR MDS,improving the efficacy of HMAs and supporting hematopoietic recovery, associated with lower rate ofadverse events."

Clinical • P1/2 data • Leukopenia • Myelodysplastic Syndrome • Neutropenia • AVEN

A Clinical Trial of Cidabenamine Plus Azacitidine to Prevent Post-Transplant Progression in High-Risk Peripheral T-Cell Lymphoma (clinicaltrials.gov) - P2/3 | N=40 | Recruiting | Sponsor: Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine

New P2/3 trial • Bone Marrow Transplantation • Hematological Malignancies • Hepatosplenic T-cell Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Peripheral T-cell Lymphoma • T Cell Non-Hodgkin Lymphoma • Transplantation

γ9δ2 T-cell activation (γδTCA) with ICT01 combined with azacitidine-venetoclax (AV) for older/unfit adults with newly diagnosed (ND) AML: Preliminary efficacy and dose selection in phase 1/2 study EVICTION. (ASCO 2025) - P1/2 | "For AV combination, the recommended Phase 2 dose is 10 mg ICT01 Q4W. Both ICT01 regimens were safe and very well tolerated and generated very high CR and CR/CRi rates in older/unfit ND-AML pts. The high response rates seen in adverse risk pts warrant further clinical investigation."

Clinical • P1/2 data • Acute Myelogenous Leukemia • Febrile Neutropenia • Hematological Disorders • Neutropenia • ASXL1 • DNMT3A • IDH1 • IDH2 • IFNG • JAK2 • NPM1 • NRAS • RUNX1 • SF3B1 • SRSF2 • STAG2 • TNFA • TP53 • U2AF1

Phase 1b/2 study of lisaftoclax (APG-2575) combined with azacitidine (AZA) in patients (pts) with treatment-naïve (TN) or prior venetoclax (VEN)-exposed myeloid malignancies. (ASCO 2025) - P1/2 | "LISA at different dose regimens combined with AZA provides promising treatment options for pts with HR-MDS or AML. No DLTs occurred. The MTD was not reached."

Clinical • P1/2 data • Acute Myelogenous Leukemia • Chronic Myelomonocytic Leukemia • Febrile Neutropenia • Hematological Malignancies • Infectious Disease • Neutropenia • Oncology • Pneumonia • Respiratory Diseases • Septic Shock • Thrombocytopenia

CA209-9WW: Nivolumab or Nivolumab and Azacitidine in Patients With Recurrent, Resectable Osteosarcoma (clinicaltrials.gov) - P1 | N=21 | Active, not recruiting | Sponsor: H. Lee Moffitt Cancer Center and Research Institute | Trial completion date: Dec 2025 ➔ Apr 2026

Trial completion date • Oncology • Osteosarcoma • Sarcoma • Solid Tumor

Phase I/II Study of Quizartinib, Venetoclax, and Decitabine Triple Combination in FLT3-ITD Mutated AML (ASH 2023) - "These patients have a median overall survival (OS) of 9.9 months when treated with the standard of care regimen (azacitidine and venetoclax). The combination of DAC + VEN + Quiz demonstrated activity in heavily pretreated and prior FLT3i-exposed (including 78% with prior gilteritinib exposure) R/R FLT3-ITDm pts, with a CRc rate of 68% and a median OS of 7.1 months. In the frontline setting, all pts achieved CRc with no early mortality, median count recovery of 40 days, and median OS not reached. The study continues to accrue, and updated results will be reported at the meeting."

P1/2 data • Acute Myelogenous Leukemia • Febrile Neutropenia • Gastrointestinal Disorder • Infectious Disease • Neutropenia • Pneumonia • Respiratory Diseases • Septic Shock • FLT3