Onureg (azacitidine oral) / BMS - LARVOL DELTA

CC486 as a safe and effective bridge to transplant in MDS-del5q with transfusion-dependent anemia following lenalidomide relapse: A case report. (PubMed, Curr Res Transl Med) - No abstract available

Journal • Anemia • Hematological Disorders • Transplantation

AMLM22/D4: The International AML Platform Consortium (IAPC) trial – Combining Oral Azacitidine and Dendritic Cell Vaccination Vididencel in maintenance (CADENCE) (ANZCTR) - P2 | N=140 | Recruiting | Sponsor: Australian Leukaemia and Lymphoma Group (ALLG) | Not yet recruiting ➔ Recruiting

Enrollment open • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • NPM1

Histamine dihydrochloride and low-dose interleukin-2 in an emerging landscape of relapse prevention in acute myeloid leukemia. (PubMed, Ther Adv Hematol) - "More recent trials have identified efficacious remission maintenance strategies, including (1) midostaurin or quizartinib for patients with FLT3-mutated AML, (2) oral azacitidine for older AML patients, and (3) immunotherapy with histamine dihydrochloride and low-dose interleukin-2 (HDC/IL-2) for younger patients. We discuss clinical efficacy in relation to patient age and anti-leukemic immunity as well as leukemic cell chemosensitivity, chromosomal integrity, and mutational profiles. Finally, we propose a role for HDC/IL-2 within an evolving landscape of strategies to achieve durable remission in a broader population of AML patients."

IO biomarker • Journal • Review • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • FLT3

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: Aug 2024 ➔ Jan 2026

Trial primary completion date • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Neutropenia • Oncology • T-Cell Large Granular Lymphocyte Leukemia • CD8 • IL15

VIALE-M: A Study of Oral Venetoclax Tablets and Oral Azacitidine as Maintenance Therapy in Adult Participants With Acute Myeloid Leukemia in First Remission After Conventional Chemotherapy (clinicaltrials.gov) - P3 | N=112 | Active, not recruiting | Sponsor: AbbVie | Trial completion date: May 2025 ➔ Jun 2026

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

A Study to Evaluate CC-486/Onureg in Participants With Moderate or Severe Hepatic Impairment Compared With Normal Hepatic Function in Participants With Myeloid Malignancies (clinicaltrials.gov) - P1 | N=32 | Active, not recruiting | Sponsor: Bristol-Myers Squibb | Recruiting ➔ Active, not recruiting

Enrollment closed • Hepatitis C • Hepatology • Liver Failure • Myelodysplastic Syndrome • Oncology

RESULTS FROM A CLINICAL STUDY OF THE ALL-ORAL REGIMEN OF CC-486 (ORAL AZACITIDINE) AND VENETOCLAX FOR NEWLY DIAGNOSED AND RELAPSED AND REFRACTORY ACUTE MYELOID LEUKEMIA (EHA 2025) - "Oral aza/ven is a regimen currently being investigated for the treatment of ND and R/R AML. High response rates have been seen in the newly-diagnosed cohort with 9 achieving a CR/CRi, including 4/5 who harbor a TP53 mutation. Final results with genomic annotation, as well as further translational studies exploring mechanistic insights will be presented."

Clinical • IO biomarker • Acute Myelogenous Leukemia • Anemia • Fatigue • Febrile Neutropenia • Metabolic Disorders • Neutropenia • Thrombocytopenia • BCL2 • TP53

REAL-WORLD OUTCOMES OF ORAL AZACITIDINE BASED MAINTENANCE THERAPY FOR ACUTE MYELOID LEUKEMIA IN REMISSION: A SINGLE CENTRE EXPERIENCE IN INDIAN POPULATION (EHA 2025) - "Most received the 3+7 induction regimen (92%) with Daunorubicin and Cytarabine, while 8% received hypomethylating agents...Those with FLT3-ITD or FLT3-TKD (28%) also received Midostaurin in combination with oral Azacitidine...One patient who had received 6 cycles of Decitabine as induction, received 11 cycles of Decitabine as consolidation before starting maintenance therapy.Relapse-free survival at 1 year was 95% (95%CI 99,86.2) and at 2 years was 54% (95%CI 87.4,22.9), with median RFS not reached... This review analyses the characteristics and outcomes of pts treated with oral Azacitidine maintenance after intensive induction and consolidation therapy. More than half of the pts were relapse free at the end of 2 years and median overall survival after start of oral Azacitidine was 36.9 months. Our results support the use of oral Azacitidine maintenance therapy in pts with AML not proceeding to transplant at remission."

Clinical • Real-world • Real-world evidence • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Leukemia • Oncology • CEBPA • CREBBP • NPM1 • PHF6

NCI-2021-13436: CC-486 and Nivolumab for the Treatment of Hodgkin Lymphoma Refractory to PD-1 Therapy or Relapsed (clinicaltrials.gov) - P1 | N=33 | Active, not recruiting | Sponsor: City of Hope Medical Center | Trial completion date: May 2025 ➔ May 2026 | Trial primary completion date: May 2025 ➔ May 2026

Trial completion date • Trial primary completion date • Classical Hodgkin Lymphoma • Hematological Malignancies • Hodgkin Lymphoma • Lymphoma • Oncology • PD-L1

Romidepsin, CC-486 (5-azacitidine), Dexamethasone, and Lenalidomide (RAdR) for Relapsed/Refractory T-cell Malignancies (clinicaltrials.gov) - P1 | N=26 | Active, not recruiting | Sponsor: National Cancer Institute (NCI) | N=20 ➔ 26

Enrollment change • Cutaneous T-cell Lymphoma • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Peripheral T-cell Lymphoma • T Cell Non-Hodgkin Lymphoma

Retrospective review of relapse after allogeneic stem cell transplant for myeloid malignancy. (ASCO 2025) - "3 received gilteritinib, 1 decitabine and GCSF, and 1 oral azacitidine. Patients with myeloid malignancies who experienced relapse after alloHSCT had poor prognosis. Development of GVHD prior to relapse trended toward improved survival after relapse. In this cohort, maintenance chemotherapy prior increased time to relapse and DLI improved survival after relapse."

Retrospective data • Review • Bone Marrow Transplantation • Graft versus Host Disease • Hematological Malignancies • Immunology • Oncology • Transplantation

A Study to Evaluate Long-term Safety of CC-486 (Oral Azacitidine) in Subjects With Hematological Disorders (clinicaltrials.gov) - P2 | N=5 | Completed | Sponsor: Celgene | Active, not recruiting ➔ Completed | Trial completion date: Jan 2026 ➔ Apr 2025 | Trial primary completion date: Jan 2026 ➔ Apr 2025

Trial completion • Trial completion date • Trial primary completion date • Hematological Malignancies • Oncology

Therapeutic Agents in Myelodysplastic Syndromes – Too Few and Not Effective Enough: We Can Do Better (MDS 2025) - "Reports remind us that statistics can be tricky, p-value depends on the sample size and is not necessarily the optimal verdict regarding the efficacy of a drug, and that binary interpretation of p-values can lead to misinterpretations of trial results. Several examples of misinterpreted trials are provided, including those involving erythropoietin, Pevonedistat, thrombomimetics, Roxadustat and CC-486. 7) Poor pharmacokinetics."

Hematological Malignancies • Myelodysplastic Syndrome • Oncology

Clinical response to azacitidine in MDS is associated with distinct DNA methylation changes in HSPCs. (PubMed, Nat Commun) - "We conducted a phase 2 trial comparing injectable and oral azacitidine (AZA) administered over one or three weeks per four-week cycle, with the primary objective of investigating whether response is linked to in vivo drug incorporation or DNA hypomethylation...By cycle six-when clinical responses typically emerge-further differential hypomethylation in responder HSPCs suggests marrow adaptation as a driver of improved hematopoiesis. These findings indicate that intrinsic baseline and early drug-induced epigenetic differences in HSPCs may underlie the variable clinical response to AZA in MDS."

Journal • Hematological Disorders • Hematological Malignancies • Myelodysplastic Syndrome • Oncology

CC-486, Lenalidomide, and Obinutuzumab for the Treatment of Recurrent or Refractory CD20 Positive B-cell Lymphoma (clinicaltrials.gov) - P1 | N=8 | Completed | Sponsor: Joseph Tuscano | Active, not recruiting ➔ Completed | Trial completion date: Jul 2025 ➔ Feb 2025

Trial completion • Trial completion date • B Cell Lymphoma • B Cell Non-Hodgkin Lymphoma • Chronic Lymphocytic Leukemia • Extranodal Marginal Zone Lymphoma • Follicular Lymphoma • Hairy Cell Leukemia • Hematological Malignancies • Indolent Lymphoma • Leukemia • Lymphoma • Lymphoplasmacytic Lymphoma • Mantle Cell Lymphoma • Marginal Zone Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Small Lymphocytic Lymphoma • Waldenstrom Macroglobulinemia • CD20 • CD4

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