azacitidine / Generic mfg. - LARVOL DELTA

DNA methyltransferase inhibition by 5-azacytidine promotes thermogenic programming in beige adipocytes. (PubMed, Biochem Biophys Res Commun) - "Our findings indicate that 5-AzaC does not significantly affect adipocyte differentiation but enhances thermogenic gene expression, including Ucp1 and Cox7a1, and increases mitochondrial function and fatty acid uptake. These results suggest that DNA demethylation modulates beige adipocyte function and may have implications for metabolic disorders such as obesity and type 2 diabetes."

Journal • Diabetes • Genetic Disorders • Metabolic Disorders • Obesity • Type 2 Diabetes Mellitus

AZACITIDINE REDUCES SYMPTOMS AND UBA1 MUTATION VARIANT ALLELE FREQUENCY IN PATIENTS WITH VEXAS SYNDROME: A RETROSPECTIVE COHORT STUDY (EULAR 2025) - "Azacitidine treatment reduces the cVAF of the UBA1 -mutation, and induces recovery of inflammatory symptoms in most VEXAS patients. If CR is achieved azacitidine can be stopped successfully."

Retrospective data

IL-1 inhibition in patients with VEXAS syndrome – a retrospective international multicenter study (EULAR 2025) - "Demographic and clinical data, including genetic data, medication use and dose (prednisone, immunosuppressive, biologics and targeted synthetic medications), response to treatment and adverse events were collected anonymously from medical files. Eventhough VEXAS was first described more than 4 years ago, the best treatment approach remains unknown. Drug survival of canakinumab was higher than that of anakinra – with survival in patients receiving the 300 mg/month dose of more than 3 years on average, while in the 150 mg/month dose and anakinra group – survival was ~6 months. This is probably due to a lower adverse event rate and a higher rate of response in the canakinumab group."

Retrospective data • Anemia • Dermatology • Hematological Disorders • Hematological Malignancies • Infectious Disease • Inflammation • Myelodysplastic Syndrome • Neutropenia • Novel Coronavirus Disease • Pneumonia • Respiratory Diseases • CRP

Erythroid Stimulating Agents in Vexas Syndrome: A Multicenter Retrospective Study of an Italian VEXAS cohort (EULAR 2025) - "After ESAs discontinuation, 53% of patients were treated with therapies aimed at suppressing the UBA1-mutant clone (either azacitidine or allo-heamatopoietic stem cell transplantation), 18% received bDMARDS and 24% supportive therapy, including red blood cell transfusion. This study confirms the efficacy and safety of ESAs in VEXAS patients. Further investigation is required to elucidate the mechanisms underlying anemia in VEXAS syndrome. Prospective studies are needed to confirm our retrospective findings and to explore the use of therapies such as luspatercept, which has been approved for anemia in low-risk MDS, in order to identify the most effective treatment options for anemia in VEXAS patients."

Retrospective data • Anemia • Cardiovascular • Hematological Disorders • Hematological Malignancies • Myelodysplastic Syndrome • Oncology • Thrombosis

Emerging treatment strategies for VEXAS syndrome: A systematic review and meta-analysis (EULAR 2025) - "Azacitidine and JAK inhibitors may be the treatment of choice for patients with VEXAS syndrome and concomitant MDS. IL-6 inhibitors are favorable for patients with high inflammatory activity and no hematological involvement. Anti-IL-1 and anti-TNF agents should not be considered until treatment with the aforementioned alternatives has failed."

Retrospective data • Review • Cardiovascular • Fatigue • Hematological Malignancies • Infectious Disease • Musculoskeletal Pain • Myelodysplastic Syndrome • Oncology • Thrombosis

The MAGIC of VEXAS : A case of overlap between Bechet's disease and VEXAS syndrome. (EULAR 2025) - "He responded well to prednisone and methotrexate (MTX)...MTX was discontinued, colchicine and azathioprine were initiated achieving remission. In 2019, he was diagnosed with pulmonary embolism, treated with high-dose corticosteroids and cyclophosphamide, followed by a maintenance therapy with infliximab and azathioprine, achieving remission...Treatment included corticosteroids and Azacitidine reducing transfusion dependency...Tocilizumab was attempted but discontinued due to an anaphylaxis-like reaction. Sarilumab was initiated after ruxolitinib was denied by health care provider...Ongoing management focuses on systemic disease control and addressing hematologic complications. Learning points for clinical practice: Key lesson from this case was the decision to further explore VEXAS in an already diagnosed Bechet’s patient, as well as treatment consequences of this diagnosis."

Clinical • Anemia • Cardiovascular • Dermatitis • Dermatology • Hematological Malignancies • Hypertension • Immunology • Infectious Disease • Musculoskeletal Diseases • Myelodysplastic Syndrome • Ocular Inflammation • Oncology • Ophthalmology • Pulmonary Embolism • Rare Diseases • Respiratory Diseases • Rheumatology • Uveitis • Vasculitis

Revumenib for Relapsed or Refractory Acute Leukemia With a KMT2A Translocation. (PubMed, Ann Pharmacother) - P1/2 | "Revumenib is an innovative targeted treatment with promising activity in r/r acute leukemia with KMT2Ar."

Journal • Review • Acute Myelogenous Leukemia • Cardiovascular • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Neutropenia • Oncology • Pediatrics • Thrombosis • Transplantation • KMT2A

GSK-3484862, a DNMT1 degrader, promotes DNMT3B expression in lung cancer cells. (PubMed, NAR Cancer) - "The FDA-approved nucleoside analogs azacytidine and decitabine are effective demethylating agents for hematologic malignancies but their general use has been limited by their toxicity and ineffectiveness against solid tumors. GSK-3484862, a dicyanopyridine-containing, DNMT1-selective inhibitor and degrader, offers a promising lead for developing novel demethylating therapeutics. These demethylation events correlate with upregulation of DNMT3B expression. These findings suggest that combining inhibitors targeting DNMT1, the maintenance methyltransferase, with those targeting DNMT3A/3B, the de novo methyltransferases, or using pan-DNMT inhibitors, could enhance anticancer efficacy and reduce resistance."

Journal • Hematological Disorders • Hematological Malignancies • Lung Cancer • Oncology • Solid Tumor • DNMT1 • DNMT3A • DNMT3B • TERT

Prognostic impact of methylation-related gene mutations in elderly acute myeloid leukemia: a real-world retrospective analysis. (PubMed, Front Med (Lausanne)) - "Concurrent mutations in DNMT3A and TET2 were associated with significantly poorer treatment response and survival outcomes. These common methylation-related gene mutations did not influence the choice between IC and azacitidine plus venetoclax combination therapy in elderly AML patients."

Journal • Real-world evidence • Retrospective data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • DNMT3A • IDH1 • IDH2 • TET2

In Vitro Drug Profiling to Guide Treatment for Relapse/Refractory AML (ASH 2024) - "Significant correlation was observed among drugs of the same classes, for example between inhibitors of PARP (e.g. niraparib-talazoparib, r=0.78, p=1.3e-22), proteasome (e.g. bortezomib-ixazomib, r=0.90, p=4.2e-36), JAK (ruxolitinib-tofacitinib, r=0.91, p=8.3e-35), MEK (cobimetinib-trametinib, p=0.93, p=8.8e-47) and CDK (abemaciclib-palbociclib, p=0.56, p=2.7e-10), confirming that the readout is biologically meaningful. Intriguingly, there were unexpected correlations between specific pairs of drugs of different classes, for instance homoharringtonine (protein translation inhibitor)-abemaciclib (CDK inhibitor) (r=0.65, p=4.3e-17) and between specific gene mutations and drug sensitivity was observed, e.g. sensitivity of CEBPAbZIP mutated samples to PARP inhibitors (p=0.00156), and of AML with inv(16) to MEK inhibitors (p=0.0016)...Drug response to daunorubicin showed good prediction of chemo-resistance in patients who had non-remission after "7+3" (ROC curve AUC..."

Preclinical • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Leukemia • Oncology • ANXA5 • FLT3

LYT-200-2022-02: A Phase 1 Study With LYT-200 in Patients With Relapsed/Refractory Acute Myeloid Leukemia (AML), or With Relapsed/Refractory, High-risk Myelodysplastic Syndrome (MDS) (clinicaltrials.gov) - P1 | N=90 | Recruiting | Sponsor: PureTech | Trial completion date: May 2025 ➔ Mar 2026 | Trial primary completion date: Feb 2025 ➔ Dec 2025

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

Comparison of the efficacy of continuous VA chemotherapy and I/HDAC consolidation in postremission therapy for acute myeloid leukemia fit for standard chemotherapy (PubMed, Zhonghua Xue Ye Xue Za Zhi) - "Objective: To compare the efficacy and safety of continuous venetoclax combined azacitidine (VA) chemotherapy and intermedium/high-dose cytarabine (I/HDAC) consolidation in patients with acute myeloid leukemia (AML) fit for standard chemotherapy (transform from UNFIT) . I/HDAC consolidation was more likely to help get survival benefits for patients with ELN favorable-risk, positive MRD, wild type FLT3, or IDH1/2 mutation. Continuous VA chemotherapy exhibited superior safety than I/HDAC consolidation."

Clinical • Journal • Retrospective data • Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Neutropenia • Oncology • Thrombocytopenia • FLT3 • IDH1 • IDH2

INDIVIDUALIZED AND INCLUSIVE STRATEGIES FOR TREATING JEHOVAH’S WITNESSES WITH ACUTE LEUKEMIA AND HIGH-GRADE MYELOID NEOPLASMS (EHA 2025) - "Treatments included: 7+3, attenuated decitabine+venetoclax, and azacitidine and sorafenib...One, treated with dasatinib+HyperCVAD, relapsed after 9 mos, then received Ponatinib+Blinatumomab, and underwent stem cell transplant...The third pt was treated with cyclophosphamide/topotecan for a co-occurring neuroblastoma without response, switching to blinatumomab then hospice due to neuroblastoma progression.For MDS - 1 pt received luspatercept for 8 mos before cessation due to cytopenia (Hgb of 2.9 g/dL)...TPO agonists included romiplostim at 2 to 4 mcg/kg weekly or daily eltrombopag, while G-CSF (Filgrastim, 5 mcg/kg) was administered based on ANC levels. Our study of Jehovah's Witness pts with acute leukemia and high-grade MDS underscores the possibility of individualized leukemia-directed care despite transfusion limitations. Despite significant baseline cytopenias, individualized treatment approaches and the use of hematopoietic growth factors can facilitate the..."

Clinical • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Chronic Myeloid Leukemia • Chronic Myelomonocytic Leukemia • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Myelodysplastic Syndrome • Myelofibrosis • Neuroblastoma • Oncology • Septic Shock • Solid Tumor • NPM1

Regorafenib in Combination With Venetoclax and Azacitidine for the Treatment of Patients With Relapsed or Refractory Acute Myeloid Leukemia (clinicaltrials.gov) - P1 | N=20 | Suspended | Sponsor: City of Hope Medical Center | Trial completion date: Feb 2027 ➔ Sep 2030 | Not yet recruiting ➔ Suspended | Trial primary completion date: Feb 2027 ➔ Sep 2030

Trial completion date • Trial primary completion date • Trial suspension • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • BCL2

MENIN inhibitor-based therapy in acute leukemia: latest updates from the 2024 ASH annual meeting. (PubMed, Exp Hematol Oncol) - "At the 2024 ASH annual meeting, updated clinical data regarding monotherapy with MENINis in AL, including revumenib, bleximenib, enzomenib and BN104, were presented. Furthermore, the therapeutic effects of venetoclax plus azacitidine or "3 + 7" regimens were further enhanced by the addition of MENINis in KMT2Ar- and NPM1m-AML patients. Therefore, MENINis offer new therapeutic prospects for AML patients, particularly for those with high-risky and poor-prognostic on-target subtypes."

Journal • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • HOXA9 • KMT2A • MEIS1 • NPM1

Phase 3 study of either ivosidenib monotherapy or azacitidine monotherapy in patients with IDH1-mutant myelodysplastic syndromes who are hypomethylating agent naive (PyramIDH). (ASCO 2025) - P3 | "Key secondary endpoints include duration of CR+PR per IWG 2006 criteria, time to CR+PR per IWG 2006 criteria, transfusion independence rate, AML transformation rate, and number of patients going to transplant. Other secondary endpoints are CR+PR at 6 months per IWG 2006 criteria; CR+PR at 4 and 6 months per IWG 2023 criteria; overall response rate per IWG 2023 criteria, duration of response, EFS, OS, duration of transfusion independence (TI), time to TI, AML transformation, quality of life, PK/PD, and safety."

Clinical • Monotherapy • P3 data • Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • IDH1

Phase II study evaluating olutasidenib and azacitidine in patients with IDH1-mutated higher-risk myelodysplastic syndromes, chronic myelomonocytic leukemia, or advanced myeloproliferative neoplasms. (ASCO 2025) - P2 | "Despite the use of IDH1 inhibitors in AML and the recent FDA approval of ivosidenib for relapsed/refractory IDH1-mutated MDS, no IDH1-directed therapies are approved in MPN or treatment-naïve MDS/CMML, and no combination treatment regimens are commercially available. The goal enrollment is 45 patients (25 treatment-naïve and 20 previously-treated with no more than 5 MPN patients in each arm) across 5-6 centers in the United States. The study was activated and enrollment began in January 2025."

Clinical • Metastases • P2 data • Acute Myelogenous Leukemia • Chronic Myelomonocytic Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Myeloproliferative Neoplasm • Oncology • IDH1

Budget Impact Analysis of Venetoclax Combination Therapies for the Treatment of Newly Diagnosed Acute Myeloid Leukemia Patients Who Are Aged 75 Years or Older, or Who Have Comorbidities That Preclude Use of Intensive Induction Chemotherapy (ISPOR 2025) - "The most up-to-date market share, reflecting the current treatment landscape projected venetoclax + azacitidine or decitabine to capture 53% and 15% market share, respectively, from existing treatments (azacitidine, low-dose cytarabine [LDAC], decitabine, ivosidenib, gemtuzumab ozogamicin, glasdegib + LDAC, and ivosidenib + azacitidine). Use of venetoclax combinations for the treatment of FDA-approved indication of ND AML reduced the budget impact and provides potential financial benefit for US payers."

Clinical • Combination therapy • HEOR • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Rational Design and Synthesis of Highly Stable Haloflavanone DNA Methyltransferase Inhibitors Inducing Tumor Suppressor Gene Re-expression in Cancer Cells. (PubMed, J Med Chem) - "The clinically approved nucleoside DNA methyltransferase (DNMT) inhibitors 5-azacytidine and 5-aza-2'-deoxycytidine lack selectivity and stability, resulting in high toxicity...34b competes for the same DNMT catalytic pocket as confirmed by saturation transfer difference-nuclear magnetic resonance, but assuming different docking poses as shown by computational studies. Overall, the high stability and activity of 34b make it a promising DNMT inhibitor for anticancer research and therapy."

Journal • Colorectal Cancer • Oncology • Solid Tumor • DNMT3A

From Hidden to Critical: The Emergence of Acute Myeloid Leukemia in Blast Crisis (ATS 2025) - "He received rasburicase and was started on continuous renal replacement therapy. He required vasopressors and was started on cefepime and vancomycin while awaiting the results of blood cultures...He was continued on hydroxyurea and started on azacitidine with Venclexta. He was started on meropenem by infectious disease specialists...It can be used for a presumptive diagnosis to initiate early treatment to improve patient outcomes. Patients with skin lesions and blasts seen on blood work should have a high suspicion of AML."

Acute Kidney Injury • Acute Myelogenous Leukemia • Acute Promyelocytic Leukemia • Atrial Fibrillation • Cardiovascular • Congestive Heart Failure • Diabetes • Heart Failure • Hematological Malignancies • Hypertension • Infectious Disease • Leukemia • Metabolic Disorders • Nephrology • Oncology • Renal Disease • Respiratory Diseases • Type 2 Diabetes Mellitus

Low-Risk Myelodysplastic Syndrome in Lung Transplant Candidates: Pause at Candidate Selection? (ATS 2025) - "Posttransplant, he was placed on tacrolimus, mycophenolate, and prednisone, but developed leukopenia and anemia, necessitating discontinuation of mycophenolate...He began chemotherapy with decitabine, venetoclax, and vincristine...His condition worsened despite transfusions and luspatercept-aamt, progressing to AML with 24.1% blasts and mutations in U2AF1, NRAS, and SETBP1 and MECOM rearrangement. Chemotherapy with azacitidine and venetoclax was initiated, but he succumbed to diffuse alveolar hemorrhage. Despite low risk MDS features, both patients in our report progressed to AML after lung transplant; their disease was resistant to conventional therapies. Further research on predictors of MDS progression after solid organ transplant and the impact of immunosuppressive medications on MDS disease progression is needed to help risk stratify older lung transplant candidates with low-risk MDS."

Acute Myelogenous Leukemia • Anemia • Antibody-mediated Rejection • Chronic Obstructive Pulmonary Disease • Hematological Disorders • Hematological Malignancies • Immunology • Infectious Disease • Leukemia • Leukopenia • Myelodysplastic Syndrome • Nephrology • Oncology • Pulmonary Disease • Renal Disease • Respiratory Diseases • Septic Shock • Solid Organ Transplantation • Transplantation • CD34 • MECOM • NCAM1 • NRAS • SETBP1 • TP53 • U2AF1

Invasive Aspergillosis Laryngitis Causing a Difficult Intubation (ATS 2025) - "Limited data is available on treatment strategies given the rarity of the disease, but patients typically respond well to typical antifungals, with surgery rarely being needed.Case PresentationThe patient presented here is a 56-year-old woman with acute myeloid leukemia on chemotherapy with azacitidine and venetoclax presented to the emergency department with complaints of hoarseness of voice and dyspnea for two weeks...Arterial blood gas while on High flow nasal cannula 60 liters, 60% was significant for pH of 7.10, pCO2 of 68, and PO2 of of 76, consistent with acute hypoxemic and hypercapnic respiratory failure.The patient was placed under moderate sedation with Midazolam and Fentanyl...The biopsy cultures grew Aspergillus fumigatus, and treatment was initiated with Amphotericin B. The patient was extubated on day five of intubation and, after finishing her fourteen-day course of Amphotericin, was discharged on Isavuconazonium with infectious disease outpatient..."

Intubation • Acute Myelogenous Leukemia • Anesthesia • Dysphonia • Hematological Malignancies • Infectious Disease • Laryngeal Cancer • Leukemia • Oncology • Otorhinolaryngology • Pulmonary Disease • Rare Diseases • Respiratory Diseases

A Case of Massive Pleural Effusion Due to Azacitidine Therapy (ATS 2025) - "Past medical history was notable only for MDS for which she recently started treatment with venetoclax and azacitidine...Given the results of the fluid studies and high output, we started treatment for azacitidine-associated pleural effusion with 20 mg of intravenous methylprednisolone every twelve hours...She was discharged on a prednisone taper...2019; 25(5):1248-1252. doi:10.1177/1078155218784762"

Clinical • Pleural effusion • Cardiovascular • Hematological Malignancies • Myelodysplastic Syndrome • Oncology • Pulmonary Disease • Respiratory Diseases

DIAGNOSIS AND TREATMENT OF ACUTE ERYTHROID LEUKAEMIA (EHA 2025) - "The median number of mutations detected was 2.5 (range 1-5).Treatment regimens used were venetoclax-azacitidine (2 patients), fludarabine, cytarabine, idarubicin (FLA-Ida, 1 patient) and azacitidine-magrolimab (1 patient), two patients received best supportive care. This cohort reaffirms that AEL patients have very adverse risk genetics, poor clinical outcomes following treatment with existing therapeutic options and short survival. Future studies are urgently needed to further improve the management of patients with AEL."

Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Oncology • ASXL1 • KMT2A • MECOM • PTPN11 • RUNX1 • RUNX1T1 • SRSF2 • TET2 • TP53

Little to show for much effort and investment: An industry perspective on MDS drug development (MDS 2025) - "Randomized studies incorporated lenalidomide, vorinostat, entinostat (MS-275), durvalumab, valproic acid, idarubicin, eltrombopag, pevonedistat (MLN4924), eprenetapopt (APR-246), sabatolimab (MBG453), magrolimab (Hu5F9-G4), or tamibarotene (SY-1425)...While the approval of luspatercept and imetelstat for lower-risk patients provides a glimmer of hope, this track record of failure in higher-risk MDS is enough to make a prudent investor or senior pharmaceutical executive think twice before committing further resources to development in this difficult group of diseases...Nor is it because the existing therapies are so good that they're hard to beat, although the practice of giving a few cycles of azacitidine or decitabine in local clinics before referring a patient to a trial center has been an unfortunate barrier to accrual...In this session I will discuss some potential fixes for a few of these problems. But solutions to other barriers are less clear, and will require..."

Acute Myelogenous Leukemia • Hematological Malignancies • Multiple Myeloma • Myelodysplastic Syndrome • Oncology • FLT3 • TP53