Onureg (azacitidine oral) / BMS - LARVOL DELTA

Real-world treatment patterns and outcomes with oral azacitidine maintenance therapy in patients with acute myeloid leukemia. (PubMed, Cancer) - "These findings provide real-world evidence further supporting the use of oral-AZA as a standard-of-care maintenance therapy in current routine clinical practice for patients with AML in remission who do not receive hematopoietic stem cell transplantation. These results may inform a broader clinical audience because of the inclusion of patients with diverse demographic and clinical characteristics."

HEOR • Journal • Observational data • Real-world evidence • Retrospective data • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Leukemia • Oncology • Transplantation

PREVENTION OF RELAPSE WITH ORAL AZACITIDINE AND VENETOCLAX AFTER ALLO-HCT FOR AML AND MDS (EBMT 2025) - P3 | "Firstly, patients received: CC-486 200mg/day with venetoclax 20mg/day on day 1 to 7 in 28-day cycles (VOG7) with administration of continuous posaconazole 300mg/day. In this preliminary report, CC-486 combined with venetoclax for the prevention of relapse after allo-HCT appears feasible. Use of VOG7 was associated with a better hematological tolerance compared to VOG14. In this cohort of very high-risk AML/MDS/CMML patients, the cumulative incidence of relapse was very low compared to similar historical cohorts."

Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Chronic Graft versus Host Disease • Chronic Myelomonocytic Leukemia • Graft versus Host Disease • Hematological Disorders • Immunology • Infectious Disease • Myelodysplastic Syndrome

PREVENTION OF RELAPSE WITH ORAL AZACITIDINE AND VENETOCLAX AFTER ALLO-HCT FOR AML AND MDS (EBMT 2025) - P3 | "Firstly, patients received: CC-486 200mg/day with venetoclax 20mg/day on day 1 to 7 in 28-day cycles (VOG7) with administration of continuous posaconazole 300mg/day. In this preliminary report, CC-486 combined with venetoclax for the prevention of relapse after allo-HCT appears feasible. Use of VOG7 was associated with a better hematological tolerance compared to VOG14. In this cohort of very high-risk AML/MDS/CMML patients, the cumulative incidence of relapse was very low compared to similar historical cohorts."

Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Chronic Graft versus Host Disease • Chronic Myelomonocytic Leukemia • Graft versus Host Disease • Hematological Disorders • Immunology • Infectious Disease • Myelodysplastic Syndrome

POST-TRANSPLANT MAINTENANCE THERAPY IN WILD-TYPE FLT3 ACUTE MYELOID LEUKEMIA AND MYELODYSPLASTIC SYNDROMES: A BICENTRIC REAL-LIFE EXPERIENCE (EBMT 2025) - "This bicentric real-life study, with the limits due to its retrospective design, highlights that maintenance therapy post-HSCT in wild-type FLT3 AML or MDS significantly improves LFS.Future prospective studies are essential to validate these findings and to identify patient subgroups that might derive the greatest benefit from post-HSCT maintenance."

Clinical • Post-transplantation • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Hepatology • Immunology • Infectious Disease • Leukemia • Myelodysplastic Syndrome • Neutropenia • Oncology • Thrombocytopenia • Transplantation • IDH1 • IDH2

POST-TRANSPLANT MAINTENANCE THERAPY IN WILD-TYPE FLT3 ACUTE MYELOID LEUKEMIA AND MYELODYSPLASTIC SYNDROMES: A BICENTRIC REAL-LIFE EXPERIENCE (EBMT 2025) - "This bicentric real-life study, with the limits due to its retrospective design, highlights that maintenance therapy post-HSCT in wild-type FLT3 AML or MDS significantly improves LFS.Future prospective studies are essential to validate these findings and to identify patient subgroups that might derive the greatest benefit from post-HSCT maintenance."

Clinical • Post-transplantation • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Hepatology • Immunology • Infectious Disease • Leukemia • Myelodysplastic Syndrome • Neutropenia • Oncology • Thrombocytopenia • Transplantation • IDH1 • IDH2

302: Maintenance Therapy in AML: A Trailblazing Slam Dunk, or a Flagrant Foul (HOPA 2025) - "While oral azacitidine gained approval based on the QUAZAR study, unanswered questions remain regarding the data and its real-world application. Similarly, conflicting data on FLT3 inhibitors (gilteritinib, sorafenib, midostaurin, quizartinib) in the pre- and post-transplant settings fuel ongoing debates over agent selection, toxicity, and quality of life...Compare the current standard of continuous venetoclax-based therapy to alternative approaches, such as fixed-duration treatment or measurable residual disease-guided discontinuation5. Identify toxicity management strategies for maintenance therapies used in AML"

Clinical • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • IDH1 • IDH2

GS-US-546-5920: Study of Magrolimab Combinations in Participants With Myeloid Malignancies (clinicaltrials.gov) - P2 | N=54 | Terminated | Sponsor: Gilead Sciences | Completed ➔ Terminated; Sponsor decision to terminate the study.

Trial termination • Acute Myelogenous Leukemia • Hematological Malignancies • Oncology

OGILAR: Study Investigating the Efficacy and Safety of the Addition of Oral-azacitidine to Salvage Treatment by Gilteritinib in Subjects ≥18 Years of Age With Relapsed/Refractory FLT3-mutated Acute Myeloid Leukemia (clinicaltrials.gov) - P2 | N=33 | Recruiting | Sponsor: French Innovative Leukemia Organisation | Trial completion date: Apr 2026 ➔ Oct 2027 | Trial primary completion date: Oct 2025 ➔ Oct 2026

Trial completion date • Trial primary completion date • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • FLT3

Safety and Efficacy Study of CC-486 With MK-3475 to Treat Locally Advanced or Metastatic Non-small Cell Lung Cancer (clinicaltrials.gov) - P2 | N=100 | Active, not recruiting | Sponsor: Celgene | Trial completion date: Apr 2024 ➔ Jun 2025

Trial completion date • Lung Cancer • Lung Non-Squamous Non-Small Cell Cancer • Non Small Cell Lung Cancer • Oncology • Solid Tumor

Duvelisib in Combination With BMS-986345 in Lymphoid Malignancy (clinicaltrials.gov) - P1 | N=14 | Completed | Sponsor: H. Lee Moffitt Cancer Center and Research Institute | Active, not recruiting ➔ Completed | Trial completion date: Jun 2025 ➔ Apr 2024

Trial completion • Trial completion date • B Cell Lymphoma • Hematological Malignancies • Hepatosplenic T-cell Lymphoma • Hodgkin Lymphoma • Leukemia • Lymphoma • Multiple Myeloma • Natural Killer/T-cell Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Primary Mediastinal Large B-Cell Lymphoma • ALK • BCL2 • BCL6 • CD8 • MYC • TNFRSF8

Oracle: Efficacy and Safety of Oral Azacitidine Compared to Investigator's Choice Therapy in Patients with Relapsed or Refractory AITL (clinicaltrials.gov) - P3 | N=86 | Active, not recruiting | Sponsor: The Lymphoma Academic Research Organisation | Trial completion date: Dec 2024 ➔ Dec 2025

Trial completion date • Follicular Lymphoma • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • T Cell Non-Hodgkin Lymphoma • BCL6 • CXCL13 • ICOS • MME • PD-1

T-LGLL: Oral Azacitidine for the Treatment of Relapsed or Refractory T-cell Large Granular Lymphocytic Leukemia (clinicaltrials.gov) - P1/2 | N=27 | Recruiting | Sponsor: Jonathan Brammer | Trial primary completion date: Dec 2024 ➔ Aug 2024

Trial primary completion date • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Neutropenia • Oncology • T-Cell Large Granular Lymphocyte Leukemia • CD8

Study of Oral Azacitidine (CC-486) in Combination With Pembrolizumab (MK-3475) in Patients With Metastatic Melanoma (clinicaltrials.gov) - P2 | N=24 | Active, not recruiting | Sponsor: M.D. Anderson Cancer Center | Trial primary completion date: Feb 2025 ➔ Feb 2026

Trial primary completion date • Melanoma • Oncology • Skin Cancer • Solid Tumor

A051902: Testing the Addition of Duvelisib or CC-486 to the Usual Treatment for Peripheral T-Cell Lymphoma (clinicaltrials.gov) - P2 | N=170 | Recruiting | Sponsor: Alliance for Clinical Trials in Oncology | Trial completion date: Jan 2026 ➔ Jun 2026 | Trial primary completion date: Jun 2025 ➔ Jun 2026

Trial completion date • Trial primary completion date • Follicular Lymphoma • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Peripheral T-cell Lymphoma • T Cell Non-Hodgkin Lymphoma • BCL6 • CXCL13 • ICOS • MME • PD-1 • TNFRSF8

Duvelisib in Combination With BMS-986345 in Lymphoid Malignancy (clinicaltrials.gov) - P1 | N=14 | Active, not recruiting | Sponsor: H. Lee Moffitt Cancer Center and Research Institute | Trial completion date: Jan 2025 ➔ Jun 2025

Trial completion date • B Cell Lymphoma • Hematological Malignancies • Hepatosplenic T-cell Lymphoma • Hodgkin Lymphoma • Leukemia • Lymphoma • Multiple Myeloma • Natural Killer/T-cell Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Primary Mediastinal Large B-Cell Lymphoma • ALK • BCL2 • BCL6 • CD8 • MYC • TNFRSF8

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=78 | Active, not recruiting | Sponsor: Bristol-Myers Squibb | Trial primary completion date: Jan 2025 ➔ Oct 2025

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

An Open-Label Phase II Trial of Pembrolizumab, an Immune Checkpoint Inhibitor Alone or in Combination With Oral Azacitidine as Second-Line Therapy for Advanced Head and Neck Squamous Cell Cancers. (PubMed, Health Sci Rep) - "The secondary outcomes assessed at 2 years include progression-free survival, time to progression, overall survival, and incidence of treatment-emergent adverse events. The findings of this trial will have translational implications, in terms of immune reprogramming induced by epigenetic therapy among a subset of advanced H & N cancer patients in a clinical setting."

Checkpoint inhibition • IO biomarker • Journal • P2 data • Gene Therapies • Head and Neck Cancer • Oncology • Solid Tumor • Squamous Cell Carcinoma • Squamous Cell Carcinoma of Head and Neck • CD8

Oral Azacitidine Use after Stem Cell Transplantation for Myeloid Malignancies: A Single Center Experience (TCT-ASTCT-CIBMTR 2025) - P3 | "Maintenance with Oral-AZA post-allogeneic HSCT is feasible, fairly well-tolerated, and the encouraging 1-y survival outcomes despite high-risk disease factors warrant further research to estimate long-term clinical benefits. The randomized study AMADEUS (NCT04173533), examining the efficacy of Oral-AZA following HSCT, may provide additional insights in this area of unmet need."

Clinical • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Chronic Myelomonocytic Leukemia • Gastrointestinal Disorder • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Myelodysplastic Syndrome • Oncology • Transplantation • TP53

20-1319.cc: CC-486 and Venetoclax for Acute Myeloid Leukemia (clinicaltrials.gov) - P1 | N=35 | Recruiting | Sponsor: University of Colorado, Denver | N=22 ➔ 35

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

A phase I trial of CC-486, lenalidomide, obinutuzumab in relapsed/refractory indolent non-Hodgkin lymphoma. (PubMed, Leuk Lymphoma) - No abstract available

Journal • P1 data • Hematological Malignancies • Indolent Lymphoma • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology

Results from a Phase 1 Open-Label Dose Escalation and Expansion Trial of Oral Azacitidine + Cedazuridine (ASTX030) in Patients with Myelodysplastic Syndromes (MDS) and MDS/Myeloproliferative Neoplasms (MPN) (ASH 2024) - P2/3 | "Background : Azacitidine (AZA) and decitabine (DEC) are parenteral DNA methyltransferase inhibitors (DNMTis) approved for the treatment of patients with MDS and acute myeloid leukemia...Median age was 72 years (range, 26–87), 35% (n=31) of patients were female, prior treatments included DNMTis (9% [n=8]), luspatercept (3% [n=3]), lenalidomide (3% [n=3]), and erythropoiesis‑stimulating agents (3% [n=2])...Based on the results of the phase 1 trial, 140/20 mg AZA/CED was selected as the RP2D. Enrollment for the phase 2 randomized, crossover (oral vs SC) trial is ongoing and combination dosing is in preparation."

Clinical • P1 data • Acute Myelogenous Leukemia • Chronic Myelomonocytic Leukemia • Gastrointestinal Disorder • Hematological Disorders • Hematological Malignancies • Leukemia • Leukopenia • Myelodysplastic Syndrome • Myeloproliferative Neoplasm • Neutropenia • Oncology • Thrombocytopenia

Acute myeloid leukemia management and research in 2025. (PubMed, CA Cancer J Clin) - "Since 2017, a turning point in AML research, 12 agents have received regulatory approval for AML in the United States: venetoclax (BCL2 inhibitor); gemtuzumab ozogamicin (CD33 antibody-drug conjugate); midostaurin, gilteritinib, and quizartinib (fms-like tyrosine kinase 3 inhibitors); ivosidenib, olutasidenib, and enasidenib (isocitrate dehydrogenase 1 and 2 inhibitors); oral azacitidine (a partially absorbable formulation); CPX351 (liposomal encapsulation of cytarabine:daunorubicin at a molar ratio of 5:1); glasdegib (hedgehog inhibitor); and recently revumenib (menin inhibitor; approved November 2024). Oral decitabine-cedazuridine, which is approved as a bioequivalent alternative to parenteral hypomethylating agents in myelodysplastic syndrome, can be used for the same purpose in AML. Menin inhibitors, CD123 antibody-drug conjugates, and other antibodies targeting CD123, CD33, and other surface markers are showing promising results. Herein, the authors review the..."

IO biomarker • Journal • Review • Acute Myelogenous Leukemia • Acute Promyelocytic Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • Transplantation • CD123 • CD33 • FLT3 • IDH1 • IL3RA

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 • Bone Marrow Transplantation • Diffuse Large B Cell Lymphoma • Hematological Malignancies • Lymphoma • Oncology • Peripheral T-cell Lymphoma • T Cell Non-Hodgkin Lymphoma • DNMT3A • TET2 • TNFRSF8

Personalized Oral Maintenance Therapy with Decitabine/Cedazuridine (ASTX727) Combined with a Molecularly Targeted Agent (Venetoclax, Gilteritinib, Ivosidenib, or Enasidenib) in Acute Myeloid Leukemia in First Remission (ASH 2024) - P1 | "Background : Most patients with acute myeloid leukemia (AML) experience relapse after initial remission, especially when unable to complete standard consolidation strategies such as high-dose cytarabine or allogeneic stem cell transplantation (SCT)...Oral azacitidine (QUAZAR AML-001) and 3-day IV decitabine (ECOG ACRIN E2906) have demonstrated efficacy as maintenance...Prophylactic anti-infectives are encouraged. Further enrollment exploring dose/schedule modifications of ASTX727 on subsequent cycles and longer follow-up are required to confirm the efficacy of these regimens."

Clinical • Acute Myelogenous Leukemia • Anemia • Bone Marrow Transplantation • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Leukemia • Leukopenia • Neutropenia • Oncology • Thrombocytopenia • TP53

Navigating AML treatment in vascular Ehlers-Danlos syndrome: achieving deeper remission with oral azacitidine-a first case report. (PubMed, Leuk Lymphoma) - No abstract available

Journal • Acute Myelogenous Leukemia • Genetic Disorders