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Last update 04 Feb 2026

Azacitidine

Last update 04 Feb 2026

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

Drug Type

Small molecule drug

Synonyms

4-amino-1-beta-D-ribofuranosyl-s-triazin-2(1H)-one, 5-azacytidine, 5-AZC

+ [20]

Target

DNMT1

Action

inhibitors

Mechanism

DNMT1 inhibitors(DNA (cytosine-5)-methyltransferase 1 inhibitors), DNA methylation inhibitors, Epigenetic drug

Therapeutic Areas

NeoplasmsHemic and Lymphatic DiseasesOther Diseases+ [4]

Active Indication

Juvenile Myelomonocytic LeukemiaMyeloproliferative DisordersPhiladelphia chromosome positive chronic myelogenous leukemia+ [46]

Inactive Indication

Acute Erythroblastic LeukemiaAcute Myeloid Leukemia with FLT3/ITD MutationAdult Acute Myeloblastic Leukemia+ [83]

Originator Organization

Celgene Corp.

Active Organization

betapharm Arzneimittel GmbH

Takeda Pharmaceutical Co., Ltd.Bristol-Myers Squibb Pharma EEIG

+ [17]

Inactive Organization

Pfizer Inc.Celgene Europe BV

Millennium Pharmaceuticals, Inc.

+ [13]

License Organization

Agios Pharmaceuticals, Inc.

Knight Therapeutics, Inc.

The University of Texas MD Anderson Cancer Center

+ [1]

Drug Highest PhaseApproved

First Approval Date

United States (19 May 2004),

Myelodysplastic Syndromes

RegulationOrphan Drug (United States), Orphan Drug (Japan), Orphan Drug (South Korea), Accelerated assessment (European Union), Priority Review (China)

Structure/Sequence

Molecular FormulaC8H12N4O5

InChIKeyNMUSYJAQQFHJEW-KVTDHHQDSA-N

CAS Registry320-67-2

888

Clinical Trials associated with Azacitidine

NCT07283900

/ Not yet recruitingPhase 2IIT

Title: A Phase II Trial of High Dose Ascorbate in Combination With Azacitidine in Adults With Myelodysplastic Syndrome

This is an open-label, phase II clinical trial with safety run-in evaluating the safety, tolerability, and efficacy of IV HDA in combination with azacitidine for participants with MDS.

Start Date01 May 2026

Sponsor / Collaborator

University of Iowa

NCT05554419

/ Not yet recruitingPhase 2IIT

Eradicating Measurable Residual Disease in Patients With Acute Myeloid Leukemia (AML) Prior to StEm Cell Transplantation (ERASE): A MyeloMATCH Treatment Trial

This phase II MyeloMATCH treatment trial compares cytarabine versus (vs.) cytarabine and venetoclax vs. liposome-encapsulated daunorubicin-cytarabine and venetoclax vs. azacitidine and venetoclax for treating patients who have residual disease after treatment for acute myeloid leukemia (AML). Cytarabine is in a class of medications called antimetabolites. It works by slowing or stopping the growth of cancer cells in the body. Venetoclax is in a class of medications called B-cell lymphoma-2 (BCL-2) inhibitors. It may stop the growth of cancer cells by blocking Bcl-2, a protein needed for cancer cell survival. Liposome-encapsulated daunorubicin-cytarabine is a drug formulation that delivers daunorubicin and cytarabine in small spheres called liposomes, which may make the drugs safer or more effective. Azacitidine is a drug that interacts with DNA and leads to the activation of tumor suppressor genes, which are genes that help control cell growth. This study may help the study doctors find out if the different drug combinations are equally effective to the usual approach of cytarabine alone while requiring a shorter duration of treatment. To decide if they are better, the study doctors will be looking to see if the study drugs lead to a higher percentage of patients achieving a deeper remission compared to cytarabine alone.

Start Date01 Mar 2026

Sponsor / Collaborator

National Cancer Institute

NCT06694454

/ Not yet recruitingPhase 1/2IIT

Phase I/II Study of Neoadjuvant Inhaled Azacytidine With Platinum-Based Chemotherapy and Durvalumab (MEDI4736) - a Combined Epigenetic-Immunotherapy (AZA-AEGEAN) Regimen for Operable Early-Stage Non-Small Cell Lung Cancer (NSCLC)

Background:
Lung cancer is the leading cause of cancer-related death worldwide. Non-small cell lung cancer (NSCLC) is the most common type of lung cancer. Surgery to remove the tumors is the standard treatment for people diagnosed with early stages of NSCLC. Despite complete removal of these tumors, many recur (happen again). An FDA-approved drug combination to treat early-stage NSCLC prior to the surgery is durvalumab plus standard chemotherapy. The FDA approved infusion drug azacytidine [AZA] is used to treat several diseases because it can rapidly kill dividing cells (including cancer cells) but it is not approved for NSCLC. An inhaled (aerosolized) form of AZA is also not approved for NSCLC. However, researchers want to know if an inhaled version of AZA can help improve treatment of people with NSCLC because inhaled AZA goes directly into the lungs with limited absorption into the bloodstream.
Objective:
To find the safest and most effective dose of inhaled AZA in participants with early-stage non-small cell lung cancer (NSCLC) that can still be removed by surgery.
Eligibility:
Adults aged 18 and older with operable early-stage NSCLC. Participants will be required to also enroll in NIH protocol 06C0014 which allows for pre- and post-treatment biopsies and bloodwork to be obtained for additional research studies.
Design:
Participants will be screened. They will have a physical exam with blood tests. Their medical records will be reviewed. They will have imaging scans and tests of their heart and lung functions. Participants will be required to have a tissue sample (biopsy) taken of their tumor prior to receiving study drug and again during surgery after Cycle 3; airway tissue biopsies and collection of collect bronchial (lung) fluid may also be done.
Participants will receive the study treatment for 3 cycles. Each cycle is 21 days. They will need to come to the NIH Clinical Center (CC) on days 1-4 of Cycles 1-3.
AZA will be given as a drug mist that can be inhaled (like the type of mist in an asthma inhaler) using a nebulizer at the NIH Clinical Center (CC) for 3 days in a row (consecutive days) during the first week of each cycle. The participant will inhale the AZA drug mist for 20 to 30 minutes each time. Participants will also receive durvalumab and a specific 2-drug assigned chemotherapy by intravenous (IV) infusion on day 4 of each cycle.
Participants will have a follow-up visit 2 weeks after their last dose of study drugs. Then they will have planned surgery to remove the tumors.
Participants will have additional follow-up visits at the NIH CC about 1 and 3 months after the surgery, and then for every 3 months for up to 3 years.
...

Start Date03 Feb 2026

Sponsor / Collaborator

National Cancer Institute

100 Clinical Results associated with Azacitidine

100 Translational Medicine associated with Azacitidine

100 Patents (Medical) associated with Azacitidine

7,358

Literatures (Medical) associated with Azacitidine

01 Feb 2026·COMPUTATIONAL BIOLOGY AND CHEMISTRY

GC/MS analysis and computational evaluation of C. cinerea EO constituents for colorectal cancer: Molecular docking, dynamics, and quantum chemical insights into Cis-Verbenyl acetate

Article

Author: Bekkar, Yahia ; Bouraoui, Rania ; Lanez, Touhami ; Neghmouche Nacer, Salah ; Bourougaa, Lotfi ; Lanez, Elhafnaoui ; Garzoli, Stefania ; Larbi Benamor, Mohammed ; Samah, Bouchagra

The essential oil of Cotula cinerea was subjected to gas chromatography-mass spectrometry (GC-MS) analysis, leading to the identification of 31 chemical constituents. The major compounds were selected for comprehensive computational investigations to explore their potential anti-colorectal cancer activity. Among the identified constituents, cis-verbenyl acetate (CVA) exhibited the most favorable binding affinities across a panel of fifteen protein targets implicated in colorectal cancer. Notably, Tankyrase1 (PDB ID: 4OA7) and Tankyrase2 (PDB ID: 4UFU) emerged as the most promising targets, with docking scores of -9.49 and -8.46 kcal/mol, respectively. Molecular dynamics simulations conducted over 300 nanoseconds demonstrated the structural stability and sustained interactions of the CVA-protein complexes, characterized by low root mean square deviation (RMSD) values and limited conformational fluctuations. These findings were corroborated by molecular mechanics Poisson-Boltzmann surface area (MM-PBSA) calculations, which revealed highly favorable binding free energies, particularly in the CVA- TNKS2 complex (ΔGtotal = -45.64 kcal/mol). In silico ADMET profiling indicated high oral bioavailability and low predicted toxicity for CVA compared to native ligands. Furthermore, density functional theory (DFT) analysis provided insight into the electronic properties of CVA, revealing a stable electronic configuration, suitable frontier molecular orbital energies, and a well-distributed electrostatic potential surface. Thermodynamic evaluations supported its chemical stability, showing a consistent increase in entropy, enthalpy, and heat capacity with rising temperature. Collectively, these computational findings suggest that CVA is a thermodynamically stable, pharmacokinetically favorable, and potentially bioactive compound with dual-target affinity against colorectal cancer-related proteins, meriting further experimental validation.

01 Feb 2026·Redox Biology

Redox-dependent protein S-glutathionylation governs azacitidine sensitivity and resistance in AML

Article

Author: Pimková, Kristýna Gloc ; Stopka, Tomáš ; Myšáková, Michaela ; Minařík, Lubomír ; Nemes, Dušan ; Jonášová, Anna

Disruption of redox metabolism is a hallmark of drug-resistant cancer cells, representing a major obstacle to the effective treatment of acute myeloid leukemia (AML). While recent studies have highlighted the importance of redox balance in AML therapy, the specific contribution of protein redox signaling to resistance remains poorly understood. Defining these mechanisms could uncover therapeutic vulnerabilities of resistant AML cells and guide the development of novel combination strategies. Here, we performed comprehensive mass spectrometry-based redox and quantitative proteomic profiling of AML cell lines and patient samples sensitive or resistant to the hypomethylating agent azacitidine (AZA). We demonstrate that AZA disrupts redox homeostasis, which inactivates the glyoxalase system and DNA damage response, and thereby induces cell death. In contrast, AZA resistance is associated with a redox reset characterized by elevated glutathione levels and diminished protein S-glutathionylation. Importantly, AZA failed to induce oxidation of proteins in these pathways in resistant cells and patient-derived AML samples. Pharmacological inhibition of glutathione synthesis restored protein S-glutathionylation and resensitized resistant AML cells to AZA.

01 Feb 2026·Blood neoplasia

Targeting dysregulated epigenetic and transcription factor networks in KMT2A-rearranged AML using iPSC models

Article

Author: Kaczkowski, Bogumił ; Zhong, Xiangfu ; Hofmann, Sophia ; Arner, Erik ; Lehmann, Sören ; Lundin, Vanessa ; Thier, Jonas ; Cabrerizo Granados, David ; Lennartsson, Andreas ; Tae-Jun Kwon, Andrew ; Palau, Anna ; Naughton, Aonghus

KMT2A chromosomal rearrangements (KMT2A-r) are frequent in pediatric acute myeloid leukemia (AML) and are associated with poor prognosis. The KMT2A gene encodes a histone lysine methyltransferase responsible for maintaining active chromatin marks (H3K4me3) at gene promoters and enhancers. KMT2A-r lead to the formation of oncogenic KMT2A fusion proteins with over 70 potential partners, disrupting normal hematopoiesis and driving leukemogenesis. Among these, KMT2A::MLLT3, a fusion of KMT2A and MLLT3, is one of the most prevalent in AML. Disruption of the epigenome is a hallmark of AML, with recurrent abnormalities in epigenetic regulators. These alterations often occur early as disease-initiating events, making epigenetic-targeted therapeutics a promising avenue for treatment. Induced pluripotent stem cells (iPSCs) have emerged as faithful models of human AML pathogenesis, recapitulating the underlying genomic lesions and epigenetic profiles. We investigated transcriptional regulation of hematopoietic development using iPSCs derived from a patient with AML with the KMT2A::MLLT3 rearrangement. Our analysis identified key transcriptional activators and repressors that contribute to the altered regulatory landscape in KMT2A::MLLT3 AML. Further analysis of chromatin immunoprecipitation sequencing data indicated that a significant subset of genes, whose expression was downregulated in AML iPSC-derived hematopoietic stem and progenitor cells (AML-HSPCs), were direct targets of Polycomb Repressive Complex 2 (PRC2). Treatment with the dual EZH1/2 inhibitor UNC1999 and 5-azacytidine reactivated these PRC2 target genes, specifically in AML-HSPCs, toward normal gene expression patterns. These findings suggest that targeting Polycomb repression offers a promising epigenetic strategy for improving outcomes in KMT2A-rearranged AML.

904

News (Medical) associated with Azacitidine

30 Jan 2026

·www.biospace.com

New Review Published in Immunotherapy Highlights the Potential for Clever-1 Inhibition with bexmarilimab to Become the Cornerstone of Next-Generation, Multi-Indication Cancer Immunotherapy

Comprehensive review synthesizes clinical learnings from BEXMAB and MATINS trials, detailing the potential of bexmarilimab in overcoming immune resistance in cancer January 29, 2026 / Faron Pharmaceuticals Ltd. (AIM:FARN)(First North:FARON), a clinical-stage biopharmaceutical company focused on tackling cancers through novel immunotherapies, today announces the publication of a comprehensive review article in the prestigious peer-reviewed journal, Immunotherapy. The article, titled ‘ Clinical optimization of bexmarilimab as a myeloid checkpoint therapy,' provides a definitive scientific and translational analysis of Clever-1, a scavenger receptor expressed on immunosuppressive macrophages. It positions Clever-1 as a ‘foundational myeloid checkpoint,' essential for maintaining the immune-evasive environment that many aggressive cancers rely on to survive. Coming on the heels of the positive clinical data from the pivotal BEXMAB trial presented across five leading conferences in 2025, ending with American Society of Hematology (ASH) Annual Meeting in December, this review serves as a cornerstone bringing together years of research, clinical data, and future strategic positioning across multiple tumour types for Faron's lead candidate, bexmarilimab . "This peer-reviewed publication is the culmination of our deep-rooted scientific understanding of the myeloid cell's role in cancer," said Dr. Maija Hollmén, senior author and Chief Scientific Officer of Faron Pharmaceuticals, and Adjunct Professor of Tumour Immunology, Group Leader, MediCity Research Laboratory and InFLAMES Flagship, University of Turku, Turku, Finland. "By highlighting how bexmarilimab specifically reprograms Clever-1-positive macrophages from a pro-tumoral to a pro-inflammatory state, we are demonstrating that the immune system can be re-ignited. This review clarifies that targeting myeloid checkpoints is not just a secondary strategy, but a primary requirement for overcoming the immunosuppressive microenvironment that current therapies often fail to penetrate." The article offers a detailed exploration into the role of Clever-1 as a master regulator of immune suppression, providing a detailed mechanistic blueprint of how bexmarilimab functions. By binding to the extracellular domain of Clever-1, bexmarilimab effectively blocks its scavenger and recycling functions, triggering a metabolic shift within the macrophage. This conversion from a ‘cold' M2-like phenotype to a ‘hot' pro-inflammatory state effectively strips away the tumor's protective shield, facilitating the recruitment and activation of anti-tumor T-cells. This fundamental reprogramming is showcased as a critical step in converting non-responsive (or cold) tumors into immune-active (or hot) environments. Beyond the science, it provides a comprehensive clinical synthesis of Faron's journey with bexmarilimab to date, integrating pivotal longitudinal data from the MATINS trial in late-stage solid tumors and the BEXMAB trial in hematological malignancies. These findings illustrate a consistent safety pro significant clinical activity across diverse and difficult-to-treat indications, underscoring the universal nature of Clever-1 as a myeloid checkpoint. Looking toward the future landscape, the authors define a clear, de-risked roadmap centered on a biomarker-driven strategy to identify patients whose tumors show high Clever‑1 expressionBy positioning Clever-1 blockade as a foundational backbone therapy, the review outlines how bexmarilimab can be strategically combined with standard chemotherapies or existing PD-1/L1 inhibitors to overcome treatment resistance and redefine the current immunotherapeutic options. "As we move toward registrational trials in higher-risk MDS and expand our footprint into metastatic solid tumors like sarcoma, having this level of peer-reviewed validation is vital," said Dr. Petri Bono, Chief Medical Officer of Faron. "The data presented at ASH 2025 demonstrated very encouraging clinical activity. This article brings that data together with the underlying biology, reinforcing our belief that bexmarilimab may become a transformative therapy for patients who have exhausted current options." The publication further discusses the positioning of Clever-1 blockade within the broader immunotherapy landscape, suggesting that myeloid-targeted agents represent the next wave of checkpoints that will complement existing PD-1/PD-L1 inhibitors. The BEXMAB study is an open-label Phase I/II clinical trial investigating bexmarilimab in combination with standard of care (SoC) in the aggressive hematological malignancies of acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). The primary objective is to determine the safety and tolerability of bexmarilimab in combination with SoC (azacitidine) treatment. Directly targeting Clever-1 could limit the replication capacity of cancer cells, increase antigen presentation, ignite an immune response, and allow current treatments to be more effective. Clever-1 is highly expressed in both AML and MDS and associated with therapy resistance, limited T cell activation and poor outcomes. Bexmarilimab is Faron's wholly owned, investigational immunotherapy designed to overcome resistance to existing treatments and optimize clinical outcomes, by targeting myeloid cell function and igniting the immune system. Bexmarilimab binds to Clever-1, an immunosuppressive receptor found on macrophages leading to tumor growth and metastases (i.e. helps cancer evade the immune system). By targeting the Clever-1 receptor on macrophages, bexmarilimab alters the tumor microenvironment, reprogramming macrophages from an immunosuppressive (M2) state to an immunostimulatory (M1) one, upregulating interferon production and priming the immune system to attack tumors and sensitizing cancer cells to standard of care. Faron (AIM: FARN, First North: FARON) is a global, clinical-stage biopharmaceutical company, focused on tackling cancers via novel immunotherapies. Its mission is to bring the promise of immunotherapy to a broader population by uncovering novel ways to control and harness the power of the immune system. The Company's lead asset is bexmarilimab , a novel anti-Clever-1 humanized antibody, with the potential to remove immunosuppression of cancers through reprogramming myeloid cell function. Bexmarilimab is being investigated in Phase I/II clinical trials as a potential therapy for patients with hematological cancers in combination with other standard treatments. Further information is available at . The content above comes from the network. if any infringement, please contact us to modify.

ImmunotherapyClinical StudyASHASCOAACR

17 Jan 2026

·www.globenewswire.com

SELLAS Life Sciences Enters Agreement with IMPACT-AML to Expand SLS009 Clinical Program into Europe

Supports capital-efficient expansion of the SLS009 clinical program into frontline AML, enabling broader U.S. and European patient enrollment U.S. enrollment evaluating SLS009 in combination with AZA/VEN in newly diagnosed AML with high-risk features is planned for Q1 2026, with European enrollment anticipated in Q2 2026 Jan. 14, 2026 -- SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) (“SELLAS’’ or the “Company”), a late-stage clinical biopharmaceutical company focused on the development of novel therapies for a broad range of cancer indications, today announced that it has entered into an agreement with IMPACT-AML, a European collaborative initiative dedicated to advancing innovative treatments for patients with acute myeloid leukemia (AML). Under the agreement, the IMPACT-AML network will conduct a clinical study evaluating SLS009, a highly selective CDK9 inhibitor, enabling access to multiple European clinical sites and patients. IMPACT-AML is a pan-European project and builds an inclusive clinical network (STREAM platform) that connects patients, clinicians, and researchers to test novel AML therapies and improve patient outcomes (https://impactaml.eu/). It is part of the prestigious EU Mission Cancer program and a top-tier scientific cluster. The IMPACT-AML project is led by a consortium of major research and clinical institutions in Europe, including IRST (IRCCS Istituto Romagnolo per lo Studio dei Tumori “Dino Amadori”), the University of Bologna, IIS LA FE (Health Research Institute Hospital La Fe), several European AML collaborative groups, and supranational organizations under the umbrella of the European Leukemia Net (ELN), as well as various university hospitals across Europe. By leveraging IMPACT-AML’s existing infrastructure and expertise, SELLAS expects to expand European patient access to SLS009 in a highly cost-efficient manner while supporting broader participation across the clinical program. “This is a highly meaningful milestone for SELLAS and for the SLS009 program,” said Angelos Stergiou, M.D., Sc.D., President and Chief Executive Officer of SELLAS. “Gaining access to the IMPACT-AML framework represents strong external validation of SLS009 and reflects the growing recognition of SLS009’s potential in addressing critical unmet needs in AML. Importantly, this collaboration allows us to efficiently expand our clinical program into Europe by leveraging an established infrastructure, significantly improving capital efficiency while supporting broader and faster patient enrollment as we advance the program into frontline AML.”The collaboration is expected to support the continued execution of the SLS009 clinical program as SELLAS advances into frontline AML. The study in Europe is planned to enroll approximately 40 patients to evaluate SLS009 in combination with azacitidine and venetoclax (AZA/VEN) in patients with newly diagnosed AML with high-risk features. Enrollment in the first part of the trial for newly diagnosed patients is expected to begin at U.S. sites in Q1 2026, followed by initiation at European sites in Q2 2026, subject to regulatory and site readiness. “IMPACT-AML is committed to accelerating access to promising new therapeutic approaches for patients with AML who face limited treatment options,” said IMPACT-AML Scientific Coordinator, Dr. Giovanni Martinelli. “We are pleased to collaborate with SELLAS and support the evaluation of SLS009 within our European network, consistent with our mission to facilitate efficient, high-quality clinical research in AML.” SELLAS is a late-stage clinical biopharmaceutical company focused on the development of novel therapeutics for a broad range of cancer indications. SELLAS’ lead product candidate, GPS, is licensed from Memorial Sloan Kettering Cancer Center and targets the WT1 protein, which is present in an array of tumor types. GPS has the potential as a monotherapy and combination with other therapies to address a broad spectrum of hematologic malignancies and solid tumor indications. The Company is also developing SLS009 (tambiciclib) - potentially the first and best-in-class differentiated small molecule CDK9 inhibitor with reduced toxicity and increased potency compared to other CDK9 inhibitors. Data suggests that SLS009 demonstrated a high response rate in AML patients with unfavorable prognostic factors including ASXL1 mutation, commonly associated with poor prognosis in various myeloid diseases. The content above comes from the network. if any infringement, please contact us to modify.

Clinical Study

14 Jan 2026

·www.prnewswire.com

AVEO Oncology, an LG Chem company, Announces First Patient Dosed in Front-Line AML Combination Study of Ficlatuzumab

– Collaboration with Blood Cancer United® in its Beat AML® Master Clinical Trial – – AVEO's ficlatuzumab compound to be studied in combination with azacitidine and venetoclax in patients with AML in a Phase 1b/2 clinical trial – – First patient dosed into the Phase 1b/2 clinical trial – BOSTON, Jan. 14, 2026 /PRNewswire/ -- AVEO Oncology, an LG Chem company, (AVEO) announced today that the first patient has been successfully dosed in a Phase 1b/2 clinical trial evaluating ficlatuzumab in combination with azacitidine and venetoclax in patients that are 60 years of age or older with untreated acute myeloid leukemia (AML) through a Master Clinical Trial Collaboration Agreement with Blood Cancer United®, formerly the Leukemia & Lymphoma Society. As part of this strategic collaboration with Blood Cancer United, a global nonprofit focused on blood cancer patient support, research, and advocacy, Blood Cancer United will be sponsoring the clinical trial as a sub-study in their Beat AML® Master Clinical Trial, the first collaborative precision medicine clinical trial in a blood cancer, which tests multiple therapies in multiple study arms simultaneously. The Phase 1b/2 clinical trial is designed to evaluate the safety, pharmacokinetics, pharmacodynamics, and preliminary efficacy of the combination as a potential treatment for AML. Ficlatuzumab is AVEO's investigational humanized IgG1 monoclonal antibody that is designed to bind to the hepatocyte growth factor (HGF) ligand and prevent it from signaling via the c-Met receptor. Early phase clinical trials evaluating ficlatuzumab in combination with cytarabine in AML reported a favorable safety profile with promising clinical activity, supporting ficlatuzumab for further study in AML patients, particularly in those who are considered unfit for standard intensive first-line therapy. "This is a significant milestone for AVEO and Blood Cancer United, who are both dedicated to improving patient outcomes through novel clinical research," said Michael P. Bailey, President and CEO of AVEO. "We welcome this partnership and are excited to collaborate on this important clinical trial as we continue to expand our efforts to address high unmet medical needs with our novel clinical stage portfolio of therapies." "The Beat AML Master Clinical Trial seeks to change the paradigm for how this deadly cancer is treated, using an innovative precision medicine protocol and implementing strategic partnerships," said Lore Gruenbaum, Ph.D, Chief Scientific Officer of Blood Cancer United. "Our collaboration with AVEO is a key next step for the trial and transforming patient care for adults with AML." About AVEO Pharmaceuticals, Inc. AVEO Pharmaceuticals, Inc., an LG Chem company, (AVEO) is an oncology-focused biopharmaceutical company committed to delivering medicines that provide a better life for patients with cancer. AVEO currently markets FOTIVDA® (tivozanib) in the U.S. for the treatment of adult patients with relapsed or refractory advanced renal cell carcinoma (RCC) following two or more prior systemic therapies. AVEO and its strategic partners continue to develop FOTIVDA in other novel targeted combinations in RCC. The company also has investigational programs in other areas of high unmet need, including ficlatuzumab in HPV-negative refractory head and neck squamous cell carcinoma and rilogrotug (also known as AV-380) in cancer cachexia. AVEO became a wholly owned subsidiary of LG Chem Life Sciences USA, Inc. on January 19, 2023. AVEO continues to operate under the AVEO Oncology, an LG Chem company, name. About LG Chem, Ltd. and LG Chem Life Sciences LG Chem is a leading global chemical company with a diversified business portfolio spanning across petrochemicals, advanced materials, and life sciences. LG Chem Life Sciences, the life sciences business division of LG Chem, is dedicated to developing and delivering innovative medicines across a broad range of therapeutic areas. Guided by its mission to transform people's lives through inspiring science and leading innovation, LG Chem Life Sciences is offering differentiated solutions to its customers. For more information, please visit . About Blood Cancer United® and the Beat AML® Master Clinical Trial Blood Cancer United (formerly The Leukemia & Lymphoma Society) is the largest global nonprofit focused on blood cancer patient support, research, and advocacy. The organization's mission is to cure blood cancer and improve the quality of life of all patients and their families. To achieve it, Blood Cancer United brings together a community of people—patients and their families, volunteers, healthcare providers, scientists, staff, partners, fundraisers, and philanthropists—who believe all blood cancer patients deserve longer, fuller lives. For support and to learn more, visit . The Beat AML® Master Clinical Trial (NCT03013998), launched in 2016 by Blood Cancer United, is the first collaborative precision medicine clinical trial in a blood cancer. The trial uses advanced genomic technology to match patients to the most promising targeted treatment based on their unique genetic mutations. The trial tests multiple therapies in multiple sub-studies simultaneously and has already generated strong results, demonstrating positive survival rates and better quality of life. Over the past decade, Beat AML has grown into a powerful ecosystem of pharma companies, genetic testing experts, data specialists, regulatory agencies, and other industry partners working toward better and safer treatments for patients with AML. For more information, visit . About Ficlatuzumab Ficlatuzumab (formerly known as AV-299) is a potent hepatocyte growth factor (HGF) immunoglobulin G1 inhibitory antibody that binds to the HGF ligand with high affinity and specificity. HGF is the natural ligand of c-Met and blocking HGF inhibits signaling through the HGF/c-Met signaling pathway. The U.S. Food and Drug Administration designated as a Fast Track development program the investigation of ficlatuzumab and ERBITUX® (cetuximab) for relapsed/recurrent HNSCC in September 2021, which is currently in a Phase 3 clinical study. Contacts Media: John F. Kouten JFK Communications, Inc. 908-227-4714 [email protected] SOURCE AVEO, an LG Chem company 21% more press release views with Request a Demo

Phase 3Fast Track

100 Deals associated with Azacitidine

External Link

KEGG	Wiki	ATC	Drug Bank
D03021	Azacitidine	L01BC07	DB00928

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Juvenile Myelomonocytic Leukemia	United States	Bristol Myers Squibb Co.	20 May 2022
Myeloproliferative Disorders	European Union	betapharm Arzneimittel GmbH	24 Mar 2020
Myeloproliferative Disorders	Iceland	betapharm Arzneimittel GmbH	24 Mar 2020
Myeloproliferative Disorders	Liechtenstein	betapharm Arzneimittel GmbH	24 Mar 2020
Myeloproliferative Disorders	Norway	betapharm Arzneimittel GmbH	24 Mar 2020
Philadelphia chromosome positive chronic myelogenous leukemia	China	Bristol-Myers Squibb Pharma EEIG	24 Apr 2017
High Risk Myelodysplastic Syndrome	Australia	Celgene Pty Ltd.	30 Nov 2009
Acute Myeloid Leukemia	European Union	Bristol-Myers Squibb Pharma EEIG	17 Dec 2008
Acute Myeloid Leukemia	Iceland	Bristol-Myers Squibb Pharma EEIG	17 Dec 2008
Acute Myeloid Leukemia	Liechtenstein	Bristol-Myers Squibb Pharma EEIG	17 Dec 2008
Acute Myeloid Leukemia	Norway	Bristol-Myers Squibb Pharma EEIG	17 Dec 2008
Chronic Myelomonocytic Leukemia	European Union	Bristol-Myers Squibb Pharma EEIG	17 Dec 2008
Chronic Myelomonocytic Leukemia	Iceland	Bristol-Myers Squibb Pharma EEIG	17 Dec 2008
Chronic Myelomonocytic Leukemia	Liechtenstein	Bristol-Myers Squibb Pharma EEIG	17 Dec 2008
Chronic Myelomonocytic Leukemia	Norway	Bristol-Myers Squibb Pharma EEIG	17 Dec 2008
Myelodysplastic Syndromes	United States	Bristol Myers Squibb Co.	19 May 2004

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Immunoblastic Lymphadenopathy	Phase 3	Japan	Celgene Corp.	06 Nov 2018
Thrombocytopenia	Phase 3	United States	Celgene Corp.	26 Apr 2013
Thrombocytopenia	Phase 3	Australia	Celgene Corp.	26 Apr 2013
Thrombocytopenia	Phase 3	Belgium	Celgene Corp.	26 Apr 2013
Thrombocytopenia	Phase 3	Brazil	Celgene Corp.	26 Apr 2013
Thrombocytopenia	Phase 3	Canada	Celgene Corp.	26 Apr 2013
Thrombocytopenia	Phase 3	Czechia	Celgene Corp.	26 Apr 2013
Thrombocytopenia	Phase 3	Denmark	Celgene Corp.	26 Apr 2013
Thrombocytopenia	Phase 3	Finland	Celgene Corp.	26 Apr 2013
Thrombocytopenia	Phase 3	France	Celgene Corp.	26 Apr 2013

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT01869114 (CTgov)	Phase 2	Adult acute myeloid leukemia with del(5q) \| Myelodysplastic Syndromes \| Acute Promyelocytic Leukemia	57	sirolimus+azacitidine (Arm A: Acute Myeloid Leukemia (AML))	crelargfgs = zvfganvqzq ofvvfeihrj (mfbucqcbsl, mticwymmaa - wsmlumheta) View more	-	10 Dec 2025
NCT01869114 (CTgov)	Phase 2		57	sirolimus+azacitidine (Arm B: High risk Myleodysplastic Syndrome (MDS))	crelargfgs = fqbsrbeebf ofvvfeihrj (mfbucqcbsl, zlatalpvlv - vwlpsztmnn) View more	-	10 Dec 2025
NCT06656494 (ICP-CL-01205, ASH2025)	Phase 1	Myeloid Tumor \| Acute Myeloid Leukemia	24	Mesutoclax + Azacitidine	qskowmhmen(qhypisaekq) = gnhqcxrocv gyrwvdyxfg (zvyblyctsc ) View more	Positive	06 Dec 2025
NCT06068621 (ASH2025)	Phase 3	Acute Myeloid Leukemia First line	170	Venetoclax-azacitidine in combination with chidamide and CAG (cytarabine, aclarubicin, granulocyte colony-stimulating factor)	fkqsmjbeqk(klbydxkorb) = fxonxdutpl xahepdyqdf (cmaqnzrvgn ) View more	Positive	06 Dec 2025
NCT06068621 (ASH2025)	Phase 3	Acute Myeloid Leukemia First line	170	daunorubicin and cytarabine	fkqsmjbeqk(klbydxkorb) = uomeoezjwi xahepdyqdf (cmaqnzrvgn ) View more	Positive	06 Dec 2025
NCT06345365 (ASH2025)	Phase 2	Acute Myeloid Leukemia First line	22	Mitoxantrone liposome + cytarabine + azacitidine	swqfwtztia(klihfvuqjs) = vrvqcwdjhe lqlxtlbuhp (vzdxgnzfxo ) View more	Positive	06 Dec 2025
NCT06345365 (ASH2025)	Phase 2	Acute Myeloid Leukemia First line	22	daunorubicin + cytarabine + azacitidine	swqfwtztia(klihfvuqjs) = vycexdngbg lqlxtlbuhp (vzdxgnzfxo ) View more	Positive	06 Dec 2025
ASH2025	Not Applicable	Acute Myeloid Leukemia	155	AZA-VEN	dyxfcmmrzw(plicbcokdc) = tsfxqfdyrl owrentiiiz (kftxfcfsae ) View more	Positive	06 Dec 2025
NCT06030089 (VENETACIBLE, ASH2025)	Phase 1	Acute Myeloid Leukemia First line Bcl-2 expression	20	Azacitidine + Venetoclax	qwadfimdvp(dypqiedidc) = BH3 profiling predicted VEN resistance as early as day 5 of treatment. In the overall cohort, a significant increase in sensitivity to Mcl-1 (MS-1) (p < 0.01) and Bcl-xL (HRK) (p < 0.05) peptides was observed at day 5 post-treatment. Among responders, no significant changes were detected, whereas in non-responders, a significant increase in sensitivity to the Mcl-1 (MS-1) peptide was observed at day 5 post-treatment (p < 0.01) nfnogvkyxq (pxnwedqfbg ) View more	Positive	06 Dec 2025
ASH2025	Phase 2	FLT3-wild type \| TP53 mutations	15	Azacitidine + Venetoclax + Gilteritinib	zxdwhkkaam(kkghmcxfpg) = zznjakzzfs qlflisnwfs (xroeghhivc ) View more	Positive	06 Dec 2025
ASH2025	Phase 2	FLT3-wild type \| TP53 mutations	15	Azacitidine + Venetoclax + Gilteritinib (TP53 mut)	zxdwhkkaam(kkghmcxfpg) = vgyhoziarr qlflisnwfs (xroeghhivc )	Positive	06 Dec 2025
ASH2025	Not Applicable	Acute Myeloid Leukemia with FLT3/ITD Mutation FLT3-ITD mutation	48	AzacitidineVenetoclaxGilteritinib + AzacitidineVenetoclaxGilteritinib + AzacitidineVenetoclaxGilteritinib (FLT3-ITD mutated AML + Refractory/Relapsed disease or persistent MRD)	leuyfggswi(pueisgjcdj) = knzxkgtrzm unjzdbngiw (ucalfcjvyw, 3.8 - 21.0) View more	Positive	06 Dec 2025
ASH2025	Not Applicable	ANKRD26 pathogenic variants	27	Venetoclax combined with cladribine, idarubicin, and cytarabine	bywvgsnflt(tmzisrisvt) = xftudykxyi jssomikawq (mvwqhpqiby ) View more	Positive	06 Dec 2025
ChiCTR2400093555 (ASH2025)	Phase 4	Acute Myeloid Leukemia First line	51	Venetoclax plus mitoxantrone hydrochloride liposome and Azacitidine	yewdediduu(wmufvaosjf) = yxlafpofbq nagezsgpgj (uueipirdcg ) View more	Positive	06 Dec 2025

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