Onureg (azacitidine oral) / BMS - LARVOL DELTA

Oral azacitidine maintenance therapy for patients with acute myeloid leukaemia and myelodysplasia-related changes: Post hoc analysis of the QUAZAR AML-001 trial. (PubMed, Br J Haematol) - No abstract available

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

Trials in progress: Phase IB/II of CPX-351 as maintenance therapy in AML patients ineligible for bone marrow transplantation (ASH 2025) - "In patients ineligible for allogenic hematopoietic stem cell transplant (HSCT), oral azacitidine is the only FDA approved treatment option...Eligible patients are newly diagnosed with AML in CR that have received any induction regimen with standard consolidation or a hypomethylating agent (HMA) and venetoclax, for at least 6 cycles and no more than 12 cycles...Prior HSCT patients are excluded, as well as cumulative lifetime anthracycline (doxorubicin equivalent) dose equal to or greater than 345 mg/m2, and for patients with prior mediastinal radiation therapy, anthracycline dose equal to or greater than 295 mg/m2...This investigator-sponsored study was supported by a research grant and provision of study drug from Jazz Pharmaceuticals. The study was independently designed and conducted by the investigators."

Clinical • P1/2 data • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Transplantation

Trial in progress: Phase I trial to evaluate safety of oral azacytidine in combination with pomalidomide as maintenance therapy in AML (ASH 2025) - "Oral azacitidine has demonstrated improved relapse-free and overall survival in older, transplant-ineligible patients, establishing a role in AML maintenance (Hunault-Berger M Blood Cancer J. 2017). In resource-limited settings, delivery of high-dose cytarabine or allogeneic transplant is frequently compromised due to multidrug-resistant infections, poor performance status (PS), and lack of donors...Participants must have adequate hematologic recovery, ECOG PS 0–2, with no prior exposure to azacytidine or venetoclax...Participants are enrolled sequentially into each cohort to ensure safety. Dose escalation is ongoing as per protocol."

Clinical • Combination therapy • P1 data • Acute Myelogenous Leukemia • Acute Promyelocytic Leukemia • Heart Failure • Hematological Malignancies • Immunology • Infectious Disease • Leukemia • Liver Failure • Nephrology • Renal Disease • CRBN • IKZF1 • IKZF3

Maintenance therapy in AML: A meta-analysis and a single center retrospective study (ASH 2025) - "Therapeutic interventions were analyzed, including CC-486, sorafenib, gilteritinib, FLT3 inhibitors, azacitidine, and decitabine. The meta-analysis suggests that maintenance therapy in AML patientsis associated with improved OS, DFS, and RFS, with particularly pronounced benefits observed in MRD+ and intermediate-risk subgroups. Although maintenance therapy increases hematologic and dermatologic toxicities, its overall safety is acceptable. The retrospective analysis indicated that the VA regimen showed certain efficacy and safety in unfit or elderly AML patients, but the study is limited in number and needs more cases and a prospective cohort study to further verify."

Retrospective data • Acute Myelogenous Leukemia • Hematological Disorders • Infectious Disease • Neutropenia • Thrombocytopenia

Risk of relapse and mortality after non-myeloablative allogeneic stem cell transplant for Acute Myeloid Leukemia and myelodysplastic syndrome after fludarabine, cyclophosphamide, and 200 centigray total body irradiation with gvhd prophylaxis using post-transplant cyclophosphamide, sirolimus, and mycophenolate mofetil: Impact of maintenance chemotherapy (ASH 2025) - "Oral HMA agents were decitabine/cedazuridine (dec-c) 3d/28-day cycle, and oral azacitidine (Oral-Aza) for 14 days per 28-day cycle...A total of 32 (31%) patients received post-HSCT maintenance chemotherapy: infusional HMA (n=2), recombinant-Granulocyte Colony Stimulating Factor (rhGCSF)/decitabine (n=2), gilteritinib (n=7), dec-c (n=9), oral-aza (2), and venetoclax/Azacitidine (n=8)... NMA conditioning with Flu/Cy/2Gy TBI yields low NRM. Post-transplant maintenance therapy, particularly with HMAs and targeted agents, is associated with reduced relapse in intermediate-risk and adverse-risk TP53 wt AML and MDS. However, outcomes remain poor for patients with TP53 mu t AML and MDS, indicating an urgent need for alternative or intensified maintenance strategies in these high-risk groups."

Clinical • Post-transplantation • Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Chronic Myelomonocytic Leukemia • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Myelodysplastic Syndrome • Transplantation • FLT3 • TP53

Oral azacitidine as maintenance therapy post allogeneic stem cell transplant in patients with myeloid malignancies (ASH 2025) - "Initial induction therapies of patients were low-intensity venetoclax combinations in 6 (55%), intensive chemotherapy without venetoclax in 4 (36%), and hypomethylating agent-based therapy without venetoclax in 1 (9%). Oral azacitidine is a feasible post-transplant maintenance therapy. Gastrointestinal side effects are common and require proactive management to maximize time on therapy. Larger sample sizes are required to define the role of oral azacitidine in the post-SCT maintenance setting."

Clinical • Acute Myelogenous Leukemia • Graft versus Host Disease • Hematological Malignancies • Immunology • Infectious Disease • Leukemia • Myelodysplastic Syndrome • Oncology • Transplantation • TP53

Maintenance with hypomethylating agent + venetoclax after intensive induction chemotherapy in patients with newly diagnosed Acute Myeloid Leukemia (ASH 2025) - "Background Consolidative therapy with high/intermediate-dose cytarabine is standard practice for fit patients with newly diagnosed acute myeloid leukemia (ND-AML) who achieve complete remission (CR) after receiving intensive induction chemotherapy (IC). Oral azacitidine is approved as maintenance therapy in patients who achieve CR after IC but are unable to complete intensive consolidation therapy...Five patients (20%) received a FLT3 inhibitor with HMA and VEN (3 quizartinib, 2 gilteritinib), and 1 patient (4%) received an IDH-2 inhibitor (enasidenib)...These findings must be interpreted in the context of several limitations: a relatively small sample size, a median of one maintenance cycle, and the absence of scheduled repeat disease assessments. Larger, prospective studies are needed to confirm these observations and better define the role of HMA + VEN in this setting."

Clinical • Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • FLT3 • IDH1 • IDH2 • TP53

Trial in progress: A Phase IIb randomized clinical trial combining oral azacitidine and vididencel, a novel immunotherapy, for post-remission / maintenance therapy for adult patients with cytogenetically intermediate and high-risk Acute Myeloid Leukemia (ALLG AMLM22/D4 CADENCE) (ASH 2025) - "We gratefully acknowledge the patients and families. Contact: www.allg.org.au"

Clinical • IO biomarker • P2b data • Acute Myelogenous Leukemia • Hematological Malignancies • Infectious Disease • Leukemia • Lymphoma

Targeting the methylome in T-LGL leukemia: Pre-clinical analysis and Results of a Phase I trial with oral 5-azacytidine (ASH 2025) - P1/2 | "In vivo, NSG huIL-15 mice engrafted with LGLLcells and treated with 5-aza (3 mg/kg) showed significant LGLL reduction via IVIS imaging and improvedoverall survival (n=5 each, p<0.001), supporting the potential of epigenetic therapy in LGLL therapy.Given the strong preclinical impact of 5-aza on T-LGLL cells, we initiated a multi-center Phase I/II trialusing oral 5-aza (CC-486) in relapsed/refractory T-LGLL patients (NCT05141682)...In vitro, in vivo, and Phase I trial datashow that epigenomic targeting with 5-aza yields clinical responses across doses without DLTs. The PhaseII component of the oral 5-aza clinical trial is currently ongoing, reinforcing the critical role of themethylome in T-LGLL pathogenesis."

P1 data • Preclinical • Gene Therapies • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Neutropenia • T-Cell Large Granular Lymphocyte Leukemia • CD5 • CD8 • IL15

A prospective clinical study of venetoclax plus azacitidine for the maintenance treatment of post-remission Acute Myeloid Leukemia (ASH 2025) - "Oralazacitidine (AZA) (CC - 486) maintenance has shown improved overall and relapse-free survival (QUAZARAML - 001 study). The most frequent grade 3–4 TEAEs were neutrophil count decreased (n=22) and plateletcount decreased (n=6). No treatment-related deaths or treatment discontinuations due to severe adverseevents occurred.CONCLUSIONSVEN+AZA maintenance demonstrates promising RFS and manageable toxicity in remission-phase AML.Extended follow-up (target N=114) will further define survival benefits across different patient groups."

Clinical • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia

Oral azacitidine maintenance improved survival for t-AML or AML-MR patients in first remission after CPX-351 and not eligible to HSCT (ASH 2025) - P | "Ann Oncol. 2020"

Clinical • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Disorders • Myelodysplastic Syndrome • ASXL1 • BCOR • DNMT3A • NPM1 • NRAS • SF3B1 • SRSF2 • TET2 • TP53 • U2AF1

Fedratinib in combination with CC-486, an oral hypomethylating agent, in patients with accelerated phase myelofibrosis: Results from the phase I/II famy trial (ASH 2025) - "Combining a parentral hypomethylating agent (HMA) with the JAK inhibitor (JAKi)ruxolitinib is reported to be active in MPN-AP. For the first time, the feasibility of combining 300mg FED with CC-486 in MPN-AP is shown. Consideringthe dismal prognosis and lack of optimal therapies for MPN-AP, the preliminary but encouraging resultsof this regimen warrant further evaluation in an international trial."

Clinical • Combination therapy • P1/2 data • CNS Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Leukopenia • Myelofibrosis • Neutropenia • Thrombocytopenia • ASXL1

PlatΤform: A multicenter, multi-arm, academic platform trial evaluating novel agents and combinations in relapsed or refractory peripheral T-cell lymphomas (ASH 2025) - P1/2 | "Each sub-study has additional and specific inclusion and exclusion criteria.Sub-Studies: Two sub-studies are currently open:GolcAza: evaluates the combination of golcadomide and oral azacitidine in R/R follicular helper T-celllymphoma. PlaTform represents a novel academic initiative dedicated to R/R PTCL, designed toaccelerate therapeutic innovation in this underserved population.Acknowledgement: We wish to thank the patients and their families for their participation in the clinicaltrials, iOnctura for providing access to roginolisib, and Bristol-Myers Squibb for providing access togolcadomide and azacitidine."

Clinical • B Cell Lymphoma • Cutaneous T-cell Lymphoma • Follicular Lymphoma • Hematological Malignancies • Leukemia • Lymphoblastic Lymphoma • Lymphoma • Non-Hodgkin’s Lymphoma • Peripheral T-cell Lymphoma • T Cell Non-Hodgkin Lymphoma • T-Cell Large Granular Lymphocyte Leukemia • PIK3CD

A phase 2 dose confirmation trial of oral ASTX030, a combination of oral azacitidine with cedazuridine among patients with myelodysplastic syndromes, chronic myelomonocytic leukemia, and acute myeloid leukemia (ASH 2025) - P2/3 | "Background :Parenteral hypomethylating agents such as azacitidine (AZA) and decitabine are standard treatments formyelodysplastic syndromes (MDS) and acute myeloid leukemia (AML). Oral ASTX030 showed PK, pharmacodynamic, and clinical profiles comparable to SC AZA,supporting its potential as an oral alternative. The observed variability in drug exposures, influenced bypts' BSA, highlights the value of BSA-based dosing to better align systemic exposure with that of SC AZA.The international Ph3 trial, AZTOUND, which incorporates BSA-based dosing, is currently recruitingparticipants.Clinical trial registration: NCT04256317"

Clinical • P2 data • Acute Myelogenous Leukemia • Chronic Myelomonocytic Leukemia • Constipation • Gastroenterology • Gastrointestinal Disorder • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Myeloproliferative Neoplasm • Neutropenia • Thrombocytopenia • HEY1

Real life treatments and outcomes of older patients aged more than 60 years with FLT3-mutated acute myeloid leukemia: Report from the multicentric french observational ALFA-PPP study (ASH 2025) - P | "Anotheroption for maintenance therapy in these patients is oral azacitidine (AZA) as demonstrated in a post-hocanalysis of the phase 3 QUAZAR AML-001 trial (Döhner et al, Blood, 2022).Methods...In all, 80 (53.7%) receivedintensive therapy (median age 68.5y, 60-78) while 60 (40.3%) underwent a non-intensive treatment,mainly AZA plus venetoclax for 81% of them (median age 77y, 64-98); 3 received best supportive care and6 died before treatment initiation...Intensiveapproaches were as follows: 7+3 for 57 (81%), CPX-351 for 11 (16%), 7+3 plus GO for one patient (1%) andamsacrine/cytarabine for the last patient (1%). Specifically in the 7+3 subcohort, the majority received7+3 with midostaurin (n=39, 68%; idarubicin 28, daunorubicin 11); 17 were treated without midostaurin(30%; ida 11, dauno 6)...In this analysis, allotransplantation (HR, 0.09 [95% CI,0.01-0.78]; p=0.029) or maintenance therapy with midostaurin/gilteritinib (HR, 0.17 [95% CI, 0.03-0.93]; p=0.041) were..."

Clinical • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • FLT3 • NPM1

Oral Azacitidine Combined With Venetoclax in Previously Untreated Higher-risk Myelodysplastic Syndromes (clinicaltrials.gov) - P1/2 | N=36 | Active, not recruiting | Sponsor: Groupe Francophone des Myelodysplasies | Recruiting ➔ Active, not recruiting

Enrollment closed • Hematological Malignancies • Myelodysplastic Syndrome • Oncology • Transplantation

A Study of Lenalidomide and CC-486 With Radiation Therapy in Patients With Plasmacytoma (clinicaltrials.gov) - P2 | N=21 | Active, not recruiting | Sponsor: Memorial Sloan Kettering Cancer Center | Trial completion date: Nov 2025 ➔ Nov 2026 | Trial primary completion date: Nov 2025 ➔ Nov 2026

Trial completion date • Trial primary completion date • Hematological Malignancies • Multiple Myeloma • Oncology • Plasmacytoma

An Efficacy and Safety Study of Oral Azacitidine (CC-486) as Maintenance Therapy in Chinese Participants With Acute Myeloid Leukemia in Complete Remission (clinicaltrials.gov) - P2 | N=34 | Active, not recruiting | Sponsor: Bristol-Myers Squibb | N=78 ➔ 34

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

The Combination of Nivolumab and CC-486 Is Active in Hodgkin Lymphoma Refractory to PD-1 Blockade (ASH 2023) - "The median number of prior therapies was 6 (range 2-14), and 90% had prior brentuximab vedotin (BV). 76% of pts had received pembrolizumab, 67% had received nivolumab, and 43% had received both agents previously... Nivolumab combined with CC-486 is tolerable and elicits a high response rate in a PD-1 refractory cohort. At this time, the study has been amended to enroll 10 more patients at DL2 to provide a better estimation of the efficacy."

IO biomarker • Anemia • Endocrine Disorders • Fatigue • Hematological Disorders • Hematological Malignancies • Hodgkin Lymphoma • Lymphoma • Metabolic Disorders • Neutropenia • Oncology • CTLA4

Subsequent Treatment and Clinical Outcome Following Induction Therapy on a Phase II Study of Oral Azacitidine Plus CHOP for Peripheral T-Cell Lymphoma (PTCL) (ASH 2024) - P2 | "Subsequent salvage regimens for the 10 relapsed PTCL patients included : duvelisib single agent (n=1), duvelisib + romidepsin (n=2, one moved on to alloSCT), duvelisib plus ruxolitinib (n=1), romidepsin + BV followed by alloSCT (n=1), single agent sequences of romidepsin, bendamustine, duvelisib, followed by BV-DICE (n=1), romidepsin plus azacitidine sequenced with single agent bendamustine, followed by BV-DICE (n=1), high dose methotrexate-based regimen with modified MATRIX without rituximab for CNS relapse (n=1), phase 1 clinical trial (n=1), and palliation (n=1). The one patient with relapsed DLBCL was treated with R-DHAX 2 cycles, polatuzumab vedotin/bendamustine/rituximab 2 cycles, followed by CAR-T (Yescarta)...Subsequent treatments following relapses were notable for salvage sequences incorporating novel agents as well as allogeneic stem cell transplant, which may benefit to extend survival in this cohort. These preliminary response and survival outcome data..."

Clinical • Clinical data • IO biomarker • P2 data • Diffuse Large B Cell Lymphoma • Hematological Malignancies • Lymphoma • Oncology • Peripheral T-cell Lymphoma • T Cell Non-Hodgkin Lymphoma • DNMT3A • TET2 • TNFRSF8

Decitabine/Cedazuridine (ASTX727) Combined with a Molecularly-Targeted Agent (Venetoclax, Gilteritinib, Ivosidenib, or Enasidenib) As Personalized Maintenance Therapy in Acute Myeloid Leukemia: First Results from a Phase 1b Study (ASH 2023) - P1 | "Currently, oral azacitidine (CC-486) is the only drug approved in the maintenance setting in patients with AML who are unable to complete standard therapy...Patients having received either intensive induction (at least 2 cycles of therapy based on intermediate to high dose cytarabine) or low-intensity induction (at least 3 cycles based on low-dose cytarabine or a hypomethylating agent) were permitted... A fully oral, targeted maintenance regimen is feasible in AML. Early results show encouraging RFS and OS. Further enrollment and follow-up are required to better assess the safety and efficacy of these regimens."

Clinical • P1 data • Acute Myelogenous Leukemia • Anemia • Cardiovascular • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Hypertension • Infectious Disease • Leukemia • Myelodysplastic Syndrome • Myeloproliferative Neoplasm • Neutropenia • Oncology • Respiratory Diseases • Thrombocytopenia • BCL2 • IDH1

Oral Azacitidine Maintenance after Front-Line Treatment with Azacitidine and Venetoclax in Elderly AML Patients. Single Center Experience (ASH 2024) - "As expected, side effects were mainly gastrointestinal and neutropenia, both easily manageable. This approach can improve the quality of life for patients, with fewer hospital visits and no life-threatening events."

Clinical • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Leukemia • Neutropenia • Oncology • Thrombocytopenia • IDH2 • NPM1

Intensive Chemotherapy (IC) Followed By Oral Azacitidine (AZA) Maintenance Versus Venetoclax (VEN) Plus AZA for Patients (pts) with Acute Myeloid Leukemia (AML): Retrospective Analysis of an Electronic Medical Record (EMR) Database in the United States (ASH 2023) - "Exclusion criteria included diagnosis of acute promyelocytic leukemia, treatment with arsenic trioxide/tretinoin, clinical trial participation, or prior hematopoietic stem cell transplant (HSCT). Pts treated with frontline IC followed by Oral-AZA as maintenance had significantly better survival outcomes than pts who were treated and achieved remission with frontline VEN-AZA. These results suggest that pts with ND AML eligible for frontline IC should receive this regimen followed by Oral-AZA maintenance, if they are not candidates for transplant at remission."

Retrospective data • Acute Myelogenous Leukemia • Acute Promyelocytic Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Myeloproliferative Neoplasm • Oncology • Transplantation

A Prospective Clinical Study of Venetoclax Plus Azacitidine for the Maintenance Treatment of Post-Remission Acute Myeloid Leukemia (ASH 2024) - P2 | "Oral azacitidine (CC-486) maintenance has shown improved overall and relapse-free survival (QUAZAR AML-001 study)...The patient unfortunately relapsed after 1 cycle of maintenance therapy, and then was given a salvage therapy with venetoclax combined with mitoxantrone, etoposide and cytarabine...Conclusions : Preliminary, venetoclax combined with lower dose azacitidine is a feasible and safe maintenance strategy regardless of whether venetoclax has been exposed to previous treatment. We will update the results of this study with a longer follow-up."

Clinical • Acute Myelogenous Leukemia • Hematological Malignancies • Infectious Disease • Leukemia • Oncology

Azacitidine Plus Venetoclax Maintenance in Acute Myeloid Leukemia Produces Sustained Remissions with Low Toxicity: Results of a Phase 2 Study (ASH 2023) - P2 | "The QUAZAR AML-001 study demonstrated improved overall and relapse-free survival with oral azacitidine (CC-486) maintenance...Cohort 1 enrolled patients who had received at least 2 cycles of intensive therapy (defined as containing intermediate to high-dose cytarabine)... Lower dose azacitidine plus venetoclax is a feasible and safe maintenance strategy in AML. This approach yields encouraging results in MRD-negative patients with non-adverse ELN risk (favorable or intermediate risk). A genetic-guided strategy may help select patients who benefit the most from this combination (i.e."

Clinical • P2 data • Acute Myelogenous Leukemia • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Hypotension • Infectious Disease • Leukemia • Neutropenia • Oncology • Thrombocytopenia • IDH1 • IDH2 • NPM1 • TP53