azacitidine / Generic mfg. - LARVOL DELTA

Long-Term Outcomes of Azacitidine, Venetoclax and Gilteritinib in Newly Diagnosed FLT3-Mutated AML. (PubMed, Blood Adv) - P1/2 | "Randomized studies comparing this regimen to standard of care approaches are warranted. This trial is registered at www.clinicaltrials.gov #NCT04140487."

Journal • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Disorders • Transplantation • FLT3

Treatment of Higher-Risk Myelodysplastic Syndrome With High-Dose Chemotherapy Followed by Lenalidomide and Azacitidine: A 4-Year Survival Update (SOHO 2026) - "Standard therapies include the hypomethylating agents azacitidine and decitabine...We offered our patients induction chemotherapy including cytarabine 3 g every week for 8 consecutive weeks followed by azacitadine 75 mg/m2 per day for 5 consecutive days per 28-day cycle, concomitant with lenalidomide 10 mg/day continuously... The combination of induction chemotherapy followed by hypomethylating agents plus immunomodulators seems to be a good choice in low income countries in treatment of higher-risk MDS patients."

Hematological Malignancies • Myelodysplastic Syndrome • Oncology

Real-World Practice With Generic Venetoclax and Generic Azacitidine in Acute Myeloid Leukemia With Different Risk Groups (SOHO 2026) - "Those with high white blood cell counts received subcutaneous cytarabine for cytoreduction before starting our treatment. The results of our study show that new generic drugs can be employed in patients with newly diagnosed AML with evident response and survival advantage in low-income countries. AML1-ETO: RUNX family transcription factor 1–RUNX1 partner transcriptional co-repressor 1, FLT3: fms related receptor tyrosine kinase 3, NPM1: nucleophosmin 1."

Clinical • Real-world • Real-world evidence • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • Renal Cell Carcinoma • NPM1 • RUNX1 • RUNX1T1

Survival and Toxicity Outcomes With Hypomethylating Agent Plus Venetoclax vs Hypomethylating Agent Alone in Higher-Risk Myelodysplastic Syndrome: A Propensity-Matched Retrospective Cohort Study (SOHO 2026) - "Patients/Participants: Adults with HR-MDS receiving azacitidine, decitabine, or decitabine-cedazuridine. In this propensity score–matched real-world cohort of HR-MDS patients, HMA plus venetoclax was not associated with improved survival compared with HMA alone and carried higher mortality and infectious/myelosuppressive toxicity across all three time horizons. These findings support careful patient selection and prospective validation before broader adoption of this combination in HR-MDS."

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

Phase 1b/2 Study of Magrolimab (Magro), Azacitidine (AZA) and Venetoclax (VEN) in Patients (pts) with Newly Diagnosed (ND) Older/Unfit or High Risk Acute Myeloid Leukemia (AML) and Relapsed Refractory (R/R) AML: Final Clinical Data and Genomic Markers of Resistance/Relapse (ASH 2024) - "Interestingly, pts with TP53mut AML who underwent alloSCT had med EFS 12 mos and med OS NR. Potential mechanisms of resistance/relapse included erythroid differentiation, inflammatory tumor microenvironment, and CD47 upregulation."

Clinical data • P1/2 data • Acute Myelogenous Leukemia • Anemia • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Leukemia • Neutropenia • Oncology • CD47 • IFNG • TNFA • TP53

Modulatory effect of resveratrol on 5-azacytidine-induced cardiac differentiation of human adipose-derived mesenchymal stem cells. (PubMed, Differentiation) - "Resveratrol improved the cardiac induction profile by enhancing the expression of cardiac-related genes. Although it showed a trend toward reducing 5-azacytidine-induced cytotoxicity, this effect was not statistically significant."

Journal • Cardiovascular • Congestive Heart Failure • Heart Failure • MYOD1

Olutasidenib in post-venetoclax patients with mIDH1 AML. (ASCO 2023) - P1/2 | "VEN was used with azacytidine (AZA) in 8 patients, after AZA (1), and with decitabine (5), cytarabine +/- idarubicin (5), and dinaciclib (2). Olutasidenib induced durable remissions in patients with mIDH1 R/R AML, including those failing prior treatment with a venetoclax-based regimen. Clinical trial information: NCT02719574."

Clinical • Acute Myelogenous Leukemia • Hematological Disorders • IDH1

Venetoclax-azacitidine treatment in TTMV::RARA-driven acute myeloid leukemia mimicking acute promyelocytic leukemia. (PubMed, Haematologica) - "Not available."

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

VERDI: Venetoclax + Azacitidine in Patients With Acute Myeloid Leukemia (clinicaltrials.gov) - P2 | N=28 | Active, not recruiting | Sponsor: Grupo Cooperativo de Estudio y Tratamiento de las Leucemias Agudas y Mielodisplasias | Recruiting ➔ Active, not recruiting

Enrollment closed • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • Transplantation • ABL1 • NPM1

Real-world outcomes of venetoclax plus hypomethylating agents in unfit acute myeloid leukaemia: Results of the GIMEMA AML2320 trial. (PubMed, Br J Haematol) - P, P3 | "The Gruppo Italiano Malattie Ematologiche dell'Adulto (GIMEMA) AML2320 is a prospective, multicentre, observational study (NCT04589728) including 193 newly diagnosed unfit AML patients receiving VEN plus azacitidine or decitabine between November 2020 and December 2021. The GIMEMA AML2320 trial confirmed the efficacy of VEN/HMAs in a prospective, real-world unfit population. Early remission was associated with improved outcomes."

Clinical • Journal • Real-world evidence • 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

Safety and efficacy of lisaftoclax, a novel BCL-2 inhibitor, in combination with azacitidine in patients with treatment-naïve or relapsed or refractory acute myeloid leukemia. (ASCO 2024) - P1/2 | "Our data support an emerging role for this new BCL-2 inhibitor Lisa combined with AZA for the treatment of elderly/unfit TN and R/R AML pts, especially with low early mortality and promising mPFS. A phase 3, randomized, double-blind clinical study is in progress to determine whether Lisa + AZA improves OS in elderly/unfit pts with AML. *HW, XW, and YL are co-first authors."

Clinical • Combination therapy • Acute Myelogenous Leukemia • Anemia • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Neutropenia • Oncology • Pneumonia • Respiratory Diseases • Thrombocytopenia

Venetoclax-Azacitidine in Combination With Chidamide and CAG in Fit Older Patients With Acute Myeloid Leukaemia (clinicaltrials.gov) - P3 | N=120 | Recruiting | Sponsor: Chinese PLA General Hospital | Phase classification: P2 ➔ P3

Phase classification • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Venetoclax and Azacitidine Therapy in Acute Myeloid Leukemia Patients With Severe Renal Impairment: A Retrospective Cohort Propensity Score–Matched Analysis (SOHO 2026) - " Using the TriNetX Global Network, we identified adults with AML who received VEN with azacitidine or decitabine (initiated within 14 days). In this large PSM analysis, AML patients with severe CKD/end-stage renal disease treated with VEN/HMA demonstrated comparable OS, TLS, and sepsis rates to those without severe CKD. These real-world findings align with pharmacokinetic data indicating that no dose adjustment is required, supporting the feasibility of VEN/HMA in this underrepresented population. AKI surveillance remains important given the observed trend toward higher incidence."

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

Outcomes of patients treated with venetoclax plus azacitidine versus azacitidine alone stratified by advanced age and acute myeloid leukemia composite model. (PubMed, Leukemia) - P1b, P3 | "Taken together, these data suggest that patients benefit from venetoclax plus azacitidine regardless of age or degree of frailty and the combination may be considered for patients with AML who may be deemed frail. Clinical trial information NCT02993523; NCT02203773."

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

Overall survival by IDH2 mutant allele (R140 or R172) in patients with late-stage mutant-IDH2 relapsed or refractory acute myeloid leukemia treated with enasidenib or conventional care regimens in the phase 3 IDHENTIFY trial. (ASCO 2022) - P3 | "Pts were preselected to a CCR (azacitidine, intermediate- or low-dose Ara-C, or supportive care), and were then randomized 1:1 to ENA 100 mg/d or CCR in 28d cycles. Mutational burden and co-mutational profiles differed between pts with mIDH2-R140 and mIDH2-R172 R/R AML. ENA improved survival outcomes for pts with IDH2-R172 mutations, with median OS and 1-year survival rate approximately double those in the CCR arm."

Clinical • P3 data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • DNMT3A • FLT3 • IDH2 • JAK2 • NPM1 • RUNX1 • SRSF2 • TMB • TP53

KIT Mutation-Driven Resistance to Venetoclax-Based Therapy in Acute Myeloid Leukemia: Superior Outcomes With Intensive Chemotherapy in Newly Diagnosed Patients (SOHO 2026) - "KIT mutations, particularly exon 17 alterations, are associated with resistance to venetoclax-based therapy in AML. Intensive chemotherapy demonstrated superior survival and response outcomes, supporting its preferential use in medically fit patients. These findings highlight KIT mutation status as an important biomarker for treatment selection and risk stratification."

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

Phase Ib/II trial of anti-CD33 monoclonal antibody BI 836858 and azacitidine in previously untreated older acute myeloid leukemia patients: Beat AML S2 sub-study results. (PubMed, Haematologica) - "Not available."

Journal • P1/2 data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Phase 2a Study of SLS009, a Highly Selective CDK9 Inhibitor, in Combination with Azacitidine and Venetoclax for Relapsed/Refractory Acute Myeloid Leukemia after Prior Venetoclax Treatment (ASH 2024) - "Clinical activity was seen particularly in pts with ASXL1 mutation which may be a subpopulation of patients with preferential sensitivity to SLS009 + AZA/VEN. Further development will be focused on AML-MR patients with and without ASXL1 mutations."

Combination therapy • P2a data • Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Metabolic Disorders • Myelodysplastic Syndrome • Nephrology • Neutropenia • Oncology • Renal Disease • ASXL1 • FLT3 • IDH1 • IDH2 • MCL1 • RUNX1 • SRSF2 • TET2 • TP53

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

Venetoclax-Based Combinations in Higher-Risk Myelodysplastic Syndromes: Outcomes in TP53-Mutated Disease and Post Hypomethylating Agent Failure (2019–2025) (SOHO 2026) - "A phase 1b study of venetoclax plus azacitidine in relapsed/refractory MDS after prior HMA exposure demonstrated modest activity, with marrow complete remission rates of roughly one-third and median overall survival under 1 year. In summary, although venetoclax-based combinations represent an important milestone, emerging evidence suggests that they may be insufficient as presently deployed for the highest-risk MDS subsets. Future progress will likely require a focus on biomarker-directed selection and optimization of dosing and schedule to reduce myelosuppression."

IO biomarker • Hematological Malignancies • Myelodysplastic Syndrome • Oncology • HAVCR2 • TP53

Administration of TAA-Specific CTLs; Hodgkin or Non-Hodgkin Lymphoma; TACTAL (clinicaltrials.gov) - P1 | N=36 | Completed | Sponsor: Baylor College of Medicine | Active, not recruiting ➔ Completed | Trial completion date: Sep 2027 ➔ Jul 2026

Trial completion • Trial completion date • Hematological Malignancies • Hodgkin Lymphoma • Indolent Lymphoma • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology

PRELIMINARY SAFETY AND ANTILEUKEMIC ACTIVITY OF SONROTOCLAX (BGB-11417), A POTENT AND SELECTIVE BCL2 INHIBITOR, IN PATIENTS WITH RELAPSED/REFRACTORY ACUTE MYELOID LEUKEMIA (EHA 2024) - P1/2, P1b/2 | "Although treatment with the BCL2 inhibitor venetoclax has improved outcomes in some patients (pts) withnewly diagnosed acute myeloid leukemia (AML), it is not approved in relapsed/refractory (R/R) AML. In this ongoing dose escalation phase 1b/2 trial, sonrotoclax + azacitidine was generally well tolerated. Thiscombination demonstrated promising antileukemic activity in pts with R/R AML including in the lowest dosecohorts. Further evaluation in pts with R/R AML is ongoing."

Clinical • IO biomarker • Acute Myelogenous Leukemia • Anemia • Bone Marrow Transplantation • Febrile Neutropenia • Infectious Disease • Lymphoma • Myelodysplastic Syndrome • Myeloproliferative Neoplasm • Neutropenia • Thrombocytopenia • 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

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