Onureg (azacitidine oral) / BMS - LARVOL DELTA

Real-World Treatment Patterns and Outcomes with Oral Azacitidine Maintenance Therapy in Patients with Acute Myeloid Leukemia (ICBMT 2025) - "This presentation will review the evidence for maintenance therapy in AML management, both with and without alloSCT, with an emphasis on oral azacitidine. Real-world data will be presented and maintenance therapy uptake issues will be discussed."

Clinical • HEOR • Real-world • Real-world evidence • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • FLT3

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

Trial suspension • 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

Acute myeloid leukemia: a comprehensive update. (PubMed, Curr Opin Hematol) - "The integration of genomic insights with emerging therapies is transforming AML management. These developments are paving the way toward more personalized care, improved outcomes, and new opportunities for long-term disease control and cure."

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

Oral Azacitidine (Oral-AZA) Maintenance Therapy for Acute Myeloid Leukemia (AML): A Systematic Review (SOHO 2025) - "Our results show the survival benefits of oral-AZA maintenance therapy following complete remission after intensive chemotherapy. The benefit of this therapy needs to be validated with a larger dataset. Long-term follow-up is also essential to assess the durability of response and late adverse effects."

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

Pleural Twist in Leukemia Care: Purpureocillium Infection in AML (SOHO 2025) - "He was subsequently diagnosed with AML and started on induction chemotherapy with a modified 3+7 regimen (daunorubicin and cytarabine)...After a month of induction, the patient underwent two cycles of low-dose oral azacitidine post-induction and showed clinical recovery... This case highlights the challenge posed by Purpureocillium as a cause of pulmonary infection, given its similarity in presentation to common opportunistic fungi such as Candida and Aspergillus. This can complicate diagnosis, and timely identification is essential for effective treatment. We also seek to raise awareness about this emerging pathogen, as current knowledge regarding its optimal treatment and prognosis remains limited."

Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

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 | Completed | Sponsor: Celgene | Active, not recruiting ➔ Completed

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

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) | Trial completion date: Jul 2025 ➔ May 2026

Trial completion date • Cutaneous T-cell Lymphoma • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Peripheral T-cell Lymphoma • T Cell Non-Hodgkin Lymphoma

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