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Last update 26 Feb 2026

Azacitidine

Last update 26 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+ [49]

Inactive Indication

Acute Erythroblastic LeukemiaAcute Myeloid Leukemia with FLT3/ITD MutationAdult Acute Myeloblastic Leukemia+ [90]

Originator Organization

Celgene Corp.

Active Organization

Nippon Shinyaku Co., Ltd.

The University of Texas MD Anderson Cancer Center

Takeda Pharmaceutical Co., Ltd.

+ [18]

Inactive Organization

Pfizer Inc.

Millennium Pharmaceuticals, Inc.

The University of Texas Health Science Center at Houston

+ [17]

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

902

Clinical Trials associated with Azacitidine

NCT07425808

/ Not yet recruitingPhase 2/3IIT

Gilteritinib in Combination With Azacitidine and Venetoclax Compared to Induction Chemotherapy "7+3" in Combination With a FLT3-inhibitor in Fit, Newly Diagnosed, FLT3-ITD Mutated Adult AML Patients: a Randomized Trial of the EORTC Leukemia Group and GIMEMA.

This international, multicenter, randomized (1:1), open-label phase II/III trial will evaluate the efficacy and safety of gilteritinib combined with azacitidine and venetoclax (experimental arm) versus standard "7+3" induction plus a FLT3inhibitor (quizartinib or midostaurin) (control arm) in newly diagnosed FLT3-ITD mutated AML patients eligible for intensive chemotherapy.

Start Date01 Dec 2026

Sponsor / Collaborator

European Organisation for Research & Treatment of Cancer

[+2]

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 Jul 2026

Sponsor / Collaborator

National Cancer Institute

NCT07177079

/ Not yet recruitingPhase 1IIT

High-dose Ascorbate (HDA) in Combination With Azacitidine and Venetoclax (Aza/Ven) in Newly Diagnosed Acute Myeloid Leukemia (AML)

This is a randomized, open-label, Phase I clinical study with expansion. It will assess the safety and efficacy of high-dose ascorbate administered concomitantly with azacitidine and venetoclax in newly diagnosed AML.

Start Date31 May 2026

Sponsor / Collaborator

University of Iowa

100 Clinical Results associated with Azacitidine

100 Translational Medicine associated with Azacitidine

100 Patents (Medical) associated with Azacitidine

7,571

Literatures (Medical) associated with Azacitidine

01 Mar 2026·Redox Biology

Methylation reader MBD2-mediated GPX4 transcriptional repression drives ovarian granulosa cell ferroptosis in PCOS

Article

Author: Li, Yin ; Wang, Tingyu ; Wang, Daojuan ; Wang, Yihan ; Ruan, Binjia ; Zhu, Zhengquan ; Wang, Ruizhe ; Yu, Xinye ; Tao, Gaojian ; Chen, Haiyun ; Wang, Yong ; Cao, Wangsen

Arrested follicular development and anovulation are hallmarks of polycystic ovary syndrome (PCOS), in which granulosa cell (GC) ferroptosis is emerging as a potential contributor. However, its precise role and regulation remain largely unknown. Here, we identify a methyl-CpG-binding domain protein 2 (MBD2)-driven ferroptotic program as a central pathogenic mechanism in PCOS. In a dehydroepiandrosterone (DHEA)-induced PCOS mouse model, GCs exhibited marked ferroptotic alterations and transcriptional suppression of glutathione peroxidase 4 (GPX4), a key anti-ferroptotic enzyme. GC-specific Gpx4 knockout exacerbated ferroptosis, impaired follicular maturation, reduced corpora lutea formation, and aggravated PCOS pathology. GPX4 repression was associated with increased DNA methyltransferases (DNMTs), elevated DNA Methyl-reading protein MBD2 and hypermethylation of the Gpx4 promoter. Pharmacological inhibition of MBD2 with KCC-07, or DNMT blockade with 5-Azacytidine, restored GPX4 expression, reduced lipid peroxidation and GC ferroptosis, and alleviated ovarian dysfunction. Integrative ATAC-seq and RNA-seq analyses revealed enhanced Gpx4 promoter accessibility in PCOS ovaries, where MBD2, MAZ, HDAC3 and NCoR assembled into a repressive complex that was interrupted by KCC-07 treatment. Importantly, pharmacologic GPX4 inhibition with RSL3 or GC-specific Gpx4 deletion abrogated the protective effects of MBD2 inhibition, establishing GPX4 repression as the critical downstream effector. Collectively, these findings uncover an MBD2-driven epigenetic program that silences GPX4, triggers GC ferroptosis, and promotes PCOS pathogenesis. Targeting MBD2 to restore epigenetic control of ferroptosis offers a promising therapeutic strategy for PCOS.

01 Mar 2026·CYTOPATHOLOGY

A Case of Mistaken Identity: When All Mimics AML

Article

Author: Amiot, Quentin ; Konopacki, Johanna ; Malfuson, Jean‐Valère ; Dejean, Anne‐Margaux Legland ép. ; Hervet, Phoebé ; Bugier, Sarah ; Arnautou, Pierre

ABSTRACT:

A 39‐year‐old man was diagnosed with acute lymphoblastic leukaemia (B‐ALL) with SYNRG::ZNF384 and P2RY8::CRLF2 gene fusions. He was treated according to the GRAALL 2014 and underwent an allogeneic transplantation, achieving complete remission. Twenty‐five months later, he developed pancytopenia. The bone marrow examination showed blasts with an immunophenotype consistent with minimally differentiated acute myeloid leukaemia (AML M0). Although the P2RY8::CRLF2 fusion persisted, no molecular evidence of a lineage switch was detected. This rare presentation underlines the diagnostic challenges of acute leukaemias. The patient was finally treated with azacitidine and venetoclax.

01 Mar 2026·LEUKEMIA RESEARCH

CPX-351 versus venetoclax plus hypomethylating agents for newly diagnosed acute myeloid leukemia: A systematic review and meta-analysis

Article

Author: Alabbas, Fahad ; Fero, Henri ; Miyashita, Akihiro ; Basendwah, Abdulrahim Mohammed

Acute myeloid leukemia (AML) predominantly affects older adults, with median age at diagnosis being 68-70 years. Selecting an appropriate regimen is important for older patients. CPX-351 demonstrated superior overall survival (OS) compared to 7 + 3 chemotherapy, while venetoclax and azacitidine significantly improved OS and composite remission rate compared to azacitidine alone. However, the optimal regimen in real-world practice remains uncertain. We performed a systematic review and meta-analysis comparing CPX-351 and the combination of venetoclax and hypomethylating agents (Ven/HMA) in newly diagnosed AML. We systematically searched PubMed, Scopus, and Cochrane Central Register of Controlled Trials from inception to January 2026. Eleven retrospective studies comprising 1852 patients comparing CPX-351 and Ven/HMA were included. The weighted mean of median age was 63.5 years in the CPX-351 cohort and 73 years in the Ven/HMA cohort. CPX-351 did not significantly improve OS (hazard ratio [HR] 0.89; 95 % CI 0.76-1.04; p = 0.1486) with median OS being 13.1 months versus 11.6 months in Ven/HMA cohort. There were no differences in the rates of composite remission rate (48.3 % vs 48.4 %; odds ratio [OR] 0.83; 95 % CI 0.58-1.18; p = 0.295), minimal residual disease negative remission (13.5 % vs 33.9 %; OR 0.61; 95 % CI 0.25-1.49; p = 0.281), 30-day mortality, and 60-day mortality. In conclusion, CPX-351 did not demonstrate superiority over Ven/HMA in composite remission rate and OS. Both regimens remain reasonable therapeutic options for older, newly diagnosed AML patients, and prospective comparative studies are needed to better guide treatment selection.

911

News (Medical) associated with Azacitidine

24 Feb 2026

·www.biospace.com

ORYZON Receives European Medicines Agency Approval to Initiate a Phase II Study of iadademstat in Essential Thrombocythemia

MADRID and CAMBRIDGE, Mass., Feb. 24, 2026 (GLOBE NEWSWIRE) -- Oryzon Genomics, S.A. (ISIN Code: ES0167733015, Ticker: ORY), a clinical-stage biopharmaceutical company and a global leader in epigenetics, today announced that the European Medicines Agency (EMA) has authorized its Clinical Trial Application (CTA) to initiate a Phase II study of iadademstat, Oryzon’s potent and selective LSD1 inhibitor currently in clinical development in oncology and hematology, for the treatment of essential thrombocythemia (ET). The study, named IDEAL ( IaDademstat treatment for EssentiAL thrombocythemia ), is a multicenter, single-arm Phase II study to be conducted in Spain in adult patients with ET who are resistant/intolerant to hydroxyurea. The primary objectives of the study are to evaluate the safety and tolerability of iadademstat and to assess its efficacy in reducing the percentage of adult ET patients with abnormal platelet counts. Secondary objectives include assessing the durable clinical hematologic response (DCHR) rate, confirming the pharmacokinetic and pharmacodynamic pro iadademstat in ET patients, and evaluating the duration of hematologic remissions (DHRs). Iadademstat will be administered for up to 24 weeks. An additional 24-week extension phase will be available for patients who are benefiting from treatment and who, in agreement with their physician, elect to continue in the study. Essential thrombocythemia is the most common type of myeloproliferative neoplasm (MPN) and is associated with an increased risk of serious complications such as stroke, heart attack, and pulmonary embolism. The disease affects approximately 200,000 people in the United States. Current treatment strategies focus primarily on reduction of platelets and ET symptom control, aiming to reduce the risk of thrombo-hemorrhagic complications and prevent progression to post-ET myelofibrosis or secondary acute myeloid leukemia (AML). Despite available therapies, a significant proportion of patients develop resistance or intolerance to first-line treatments such as hydroxyurea, highlighting the need for novel therapeutic approaches. Inhibition of LSD1 has been shown to block the terminal differentiation of megakaryocytes into platelets, leading to a steady reduction in circulating platelet counts, supporting the use of LSD1 inhibitors in ET. Positive results with another LSD1 inhibitor have been reported in a Phase II trial in high-risk ET patients, further validating this mechanism in the disease. Dr. Carlos Buesa, Oryzon’s CEO, said, “The initiation of the IDEAL study reinforces our strategy to broaden the clinical utility of iadademstat beyond acute leukemia into additional hematologic indications with significant unmet medical need. Iadademstat is by far the most potent LSD1 inhibitor in clinical development, with more than 100-fold greater potency than any other LSD1 inhibitor currently in development. We believe the mechanistic pro LSD1 inhibition uniquely positions iadademstat to address the underlying biology of essential thrombocythemia, and we look forward to advancing this important program, with the potential to demonstrate efficacy across the full spectrum of myeloproliferative diseases.” Dr Ana Limón, Oryzon’s Senior Vice-president of Clinical Development and Global Medical Affairs added, “LSD1 inhibition alters the natural biology of myeloid diseases by reversing differentiation blocks and reducing the prevalence of leukemic stem cells. Early intervention in ET could potentially prevent progression to post-ET myelofibrosis or secondary AML. The IDEAL study will explore this potential of iadademstat, in addition to assessing its dose-dependent effects on platelet counts and thrombotic events in ET patients who are resistant or intolerant to standard-of-care treatment with hydroxyurea. ” Iadademstat is also being actively investigated in multiple oncology clinical trials, including the Phase Ib ALICE-2 study in combination with venetoclax and azacitidine in first-line AML. Highly encouraging preliminary data were presented at the American Society of Hematology (ASH) 2025 annual meeting, showing a 100% overall response rate (ORR) and 90% strict complete remission (CR) rate. Additional studies include the company-sponsored FRIDA trial evaluating iadademstat in combination with gilteritinib in relapsed/refractory FLT3-mutated AML, for which preliminary positive data were also presented at ASH-2025, as well as several trials conducted under a Cooperative Research and Development Agreement (CRADA) with the U.S. National Cancer Institute in first line AML, myeloproliferative neoplasms and small cell lung cancer, and investigator-initiated studies in myelodysplastic syndrome and small cell lung cancer. In addition, the company is conducting a clinical trial evaluating iadademstat in sickle cell disease. About Oryzon Founded in 2000 and headquartered in Barcelona, Spain, Oryzon (ISIN: ES0167733015) is a clinical-stage biopharmaceutical company and a European leader in epigenetics, with a strong focus on personalized medicine for central nervous system (CNS) disorders and oncology. Oryzon’s team comprises highly experienced pharmaceutical professionals based in Barcelona, Boston, and New Jersey. The Company has an advanced clinical portfolio built around two LSD1 inhibitors: vafidemstat, its lead CNS program, which is Phase III–ready; and iadademstat, its oncology/hematology program, with several ongoing Phase I and II studies and outstanding preliminary results in first-line acute myeloid leukemia, including a 100% overall response rate (ORR) presented at ASH 2025. In addition, Oryzon is advancing a broader epigenetics pipeline targeting other mechanisms, including HDAC6, for which a clinical candidate, ORY-4001, has been nominated for potential development in Charcot–Marie–Tooth disease (CMT) and amyotrophic lateral sclerosis (ALS). The Company also operates a robust platform for biomarker identification and target validation across a range of malignant and neurological diseases. For more information, visit About Iadademstat Iadademstat (ORY-1001) is a small oral molecule, which acts as a highly selective inhibitor of the epigenetic enzyme LSD1 and has a powerful differentiating effect in hematologic cancers (see Maes et al., Cancer Cell 2018 Mar 12; 33 (3): 495-511.e12.doi: 10.1016 / j.ccell.2018.02.002.). A FiM Phase I/IIa clinical trial with iadademstat in R/R AML patients demonstrated the safety and good tolerability of the drug and preliminary signs of antileukemic activity, including a CRi (see Salamero et al, J Clin Oncol, 2020, 38(36): 4260-4273. doi: 10.1200/JCO.19.03250). Iadademstat has shown encouraging safety and strong clinical activity in combination with azacitidine in a Phase IIa trial in elder 1L AML patients (ALICE trial) (see Salamero et al., ASH 2022 oral presentation & The Lancet Haematology, 2024, 11(7):e487-e498). Iadademstat is currently being evaluated in combination with azacitidine and venetoclax in 1L AML in an investigator-initiated study (IIS) led by OHSU and in combination with gilteritinib in the company-sponsored Phase Ib FRIDA trial in relapsed/refractory FLT3-mutant AML, with highly encouraging preliminary safety and efficacy data recently reported at ASH-2025 for both trials: 100% ORR and 90% strict CR in 1L AML, and 67% CCR (at the dose under expansion) in R/R AML. Additional studies in hemato-oncology include an IIS in MDS, and trials in myeloproliferative neoplasms and 1L AML both sponsored and conducted by the U.S. National Cancer Institute (NCI) under a Cooperative Research and Development Agreement (CRADA) signed between Oryzon and the NCI. Beyond hematological cancers, the inhibition of LSD1 has been proposed as a valid therapeutic approach in some solid tumors such as small cell lung cancer (SCLC), neuroendocrine tumors (NET), medulloblastoma and others. In a Phase IIa trial in combination with platinum/etoposide in second line ED-SCLC patients (CLEPSIDRA trial), preliminary activity and safety results have been reported (see Navarro et al., ESMO 2018 poster). Iadademstat is in two trials in ED-SCLC: a Phase I/II randomized trial in 1L in combination with ICI sponsored by NCI and led by the Memorial Sloan Kettering Cancer Center, and an IIS trial in 1L/2L in combination with ICI and radiotherapy. In addition, Oryzon has expanded iadademstat’s clinical development into non-oncological hematology indications, with trials in sickle cell disease (approved by EMA, enrolling) and essential thrombocythemia (approved by EMA). Iadademstat has orphan drug designation for SCLC in the US and for AML in the US and EU. FORWARD-LOOKING STATEMENTS This communication contains, or may contain, forward-looking information and statements about Oryzon, including financial projections and estimates and their underlying assumptions, statements regarding plans, objectives, and expectations with respect to future operations, capital expenditures, synergies, products and services, and statements regarding future performance. Forward-looking statements are statements that are not historical facts and are generally identified by the words “expects,” “anticipates,” “believes,” “intends,” “estimates” and similar expressions. Although Oryzon believes that the expectations reflected in such forward-looking statements are reasonable, investors and holders of Oryzon shares are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Oryzon that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include those discussed or identified in the documents sent by Oryzon to the Spanish Comisión Nacional del Mercado de Valores (CNMV), which are accessible to the public. Forward-looking statements are not guarantees of future performance and have not been reviewed by the auditors of Oryzon. You are cautioned not to place undue reliance on the forward-looking statements, which speak only as of the date they were made. All subsequent oral or written forward-looking statements attributable to Oryzon or any of its members, directors, officers, employees, or any persons acting on its behalf are expressly qualified in their entirety by the cautionary statement above. All forward-looking statements included herein are based on information available to Oryzon on the date hereof. Except as required by applicable law, Oryzon does not undertake any obligation to publicly update or revise any forward‐looking statements, whether as a result of new information, future events, or otherwise. This document does not constitute an offer or invitation to purchase or subscribe shares in accordance with the provisions of Regulation (EU) 2017/1129 of the European Parliament and of the Council of 14 June 2017, and/or the restated text of the Securities Market Law, approved by Law 6/2023 of 17 March, and its implementing regulations. Nothing in this document constitutes investment advice. In addition, this document does not constitute an offer of purchase, sale or exchange, nor a request for an offer of purchase, sale or exchange of securities, nor a request for any vote or approval in any jurisdiction. The shares of Oryzon Genomics, S.A. may not be offered or sold in the United States of America except pursuant to an effective registration statement under the Securities Act of 1933 or pursuant to a valid exemption from registration.. Spain Oryzon IR & Media, Europe & US Patricia Cobo/Mario Cordera Emili Torrell Sandya von der Weid Atrevia Chief BD Officer LifeSci Advisors, LLC +34 91 564 07 25 +34 673 33 97 65 +34 93 515 1313 +41 78 680 05 38 pcobo@atrevia.com mcordera@atrevia.com etorrell@oryzon.com svonderweid@lifesciadvisors.com

Phase 2Clinical ResultPhase 1ASHOrphan Drug

24 Feb 2026

·www.oryzon.com

ORYZON receives European Medicines Agency approval to initiate a Phase II study of iadademstat in essential thrombocythemia

MADRID, SPAIN and CAMBRIDGE, MA, UNITED STATES, February 24th, 2026 - Oryzon Genomics, S.A. (ISIN Code: ES0167733015, Ticker: ORY), a clinical-stage biopharmaceutical company and a global leader in epigenetics, today announced that the European Medicines Agency (EMA) has authorized its Clinical Trial Application (CTA) to initiate a Phase II study of iadademstat, Oryzon’s potent and selective LSD1 inhibitor currently in clinical development in oncology and hematology, for the treatment of essential thrombocythemia (ET). The study, named IDEAL (IaDademstat treatment for EssentiAL thrombocythemia), is a multicenter, single-arm Phase II study to be conducted in Spain in adult patients with ET who are resistant/intolerant to hydroxyurea. The primary objectives of the study are to evaluate the safety and tolerability of iadademstat and to assess its efficacy in reducing the percentage of adult ET patients with abnormal platelet counts. Secondary objectives include assessing the durable clinical hematologic response (DCHR) rate, confirming the pharmacokinetic and pharmacodynamic profile of iadademstat in ET patients, and evaluating the duration of hematologic remissions (DHRs). Iadademstat will be administered for up to 24 weeks. An additional 24-week extension phase will be available for patients who are benefiting from treatment and who, in agreement with their physician, elect to continue in the study. Essential thrombocythemia is the most common type of myeloproliferative neoplasm (MPN) and is associated with an increased risk of serious complications such as stroke, heart attack, and pulmonary embolism. The disease affects approximately 200,000 people in the United States. Current treatment strategies focus primarily on reduction of platelets and ET symptom control, aiming to reduce the risk of thrombo-hemorrhagic complications and prevent progression to post-ET myelofibrosis or secondary acute myeloid leukemia (AML). Despite available therapies, a significant proportion of patients develop resistance or intolerance to first-line treatments such as hydroxyurea, highlighting the need for novel therapeutic approaches. Inhibition of LSD1 has been shown to block the terminal differentiation of megakaryocytes into platelets, leading to a steady reduction in circulating platelet counts, supporting the use of LSD1 inhibitors in ET. Positive results with another LSD1 inhibitor have been reported in a Phase II trial in high-risk ET patients, further validating this mechanism in the disease. Dr. Carlos Buesa, Oryzon’s CEO, said, “The initiation of the IDEAL study reinforces our strategy to broaden the clinical utility of iadademstat beyond acute leukemia into additional hematologic indications with significant unmet medical need. Iadademstat is by far the most potent LSD1 inhibitor in clinical development, with more than 100-fold greater potency than any other LSD1 inhibitor currently in development. We believe the mechanistic profile of LSD1 inhibition uniquely positions iadademstat to address the underlying biology of essential thrombocythemia, and we look forward to advancing this important program, with the potential to demonstrate efficacy across the full spectrum of myeloproliferative diseases.” Dr Ana Limón, Oryzon’s Senior Vice-president of Clinical Development and Global Medical Affairs added, “LSD1 inhibition alters the natural biology of myeloid diseases by reversing differentiation blocks and reducing the prevalence of leukemic stem cells. Early intervention in ET could potentially prevent progression to post-ET myelofibrosis or secondary AML. The IDEAL study will explore this potential of iadademstat, in addition to assessing its dose-dependent effects on platelet counts and thrombotic events in ET patients who are resistant or intolerant to standard-of-care treatment with hydroxyurea.” Iadademstat is also being actively investigated in multiple oncology clinical trials, including the Phase Ib ALICE-2 study in combination with venetoclax and azacitidine in first-line AML. Highly encouraging preliminary data were presented at the American Society of Hematology (ASH) 2025 annual meeting, showing a 100% overall response rate (ORR) and 90% strict complete remission (CR) rate. Additional studies include the company-sponsored FRIDA trial evaluating iadademstat in combination with gilteritinib in relapsed/refractory FLT3-mutated AML, for which preliminary positive data were also presented at ASH-2025, as well as several trials conducted under a Cooperative Research and Development Agreement (CRADA) with the U.S. National Cancer Institute in first line AML, myeloproliferative neoplasms and small cell lung cancer, and investigator-initiated studies in myelodysplastic syndrome and small cell lung cancer. In addition, the company is conducting a clinical trial evaluating iadademstat in sickle cell disease.

Phase 2ASHClinical ResultPhase 1

23 Feb 2026

·www.biospace.com

Faron’s Bexmarilimab Data Accepted for Poster Presentation at the BSH Annual Scientific Meeting 2026

TURKU, FI / ACCESS Newswire / February 23, 2026 / Faron Pharmaceuticals Ltd. (HEL:FARON)(LSE:FARN), a clinical-stage biopharmaceutical company focused on tackling cancers through novel immunotherapies, today announces that data from its Phase I/II BEXMAB trial evaluating bexmarilimab in combination with azacitidine for the treatment of higher-risk myelodysplastic syndromes (MDS) have been accepted for presentation at the 66th Annual Scientific Meeting of the British Society for Haematology (BSH), taking place 19-21 April 2026 in Liverpool, UK. The abstract, titled "Bexmarilimab Plus Azacitidine in Higher Risk Myelodysplastic Syndrome: Updated Results from the BEXMAB Study," has been selected for poster presentation (Poster P02.10) under the theme Myeloid Malignancies and Bone Marrow Failure Syndromes . The acceptance supports Faron's consistent scientific presence at major hematology and oncology meetings. While the BSH poster contains updated figures from the latest data cut, the dataset itself is previously reported. Inclusion at BSH underscores the Company's ongoing engagement with the MDS community and its commitment to transparent, continuous reporting of BEXMAB clinical progress. The poster presents the full dataset included in the accepted abstract based on the 3 November 2025 data cut (55 patients: 21 treatment‑naïve; 34 HMA‑refractory/relapsed). The results support the planned advancement of bexmarilimab into the next development phase in treatment‑naïve higher‑risk MDS. Dr Emma Searle, presenting author from The Christie NHS Foundation Trust and the University of Manchester , commented: "We are pleased to share the BEXMAB data with the hematology community at BSH. The combination of bexmarilimab and azacitidine continues to show a favorable safety pro encouraging response rates across both treatment‑naïve and HMA‑refractory higher‑risk MDS populations. Presenting this data at BSH offers a valuable opportunity to engage with investigators and KOLs who are eager for bexmarilimab 's next HR‑MDS trial, exchange clinical insights, and help shape the field's continued progress." About bexmarilimab Bexmarilimab is Faron's wholly owned, investigational immunotherapy designed to overcome resistance to existing treatments by targeting Clever-1, a receptor on immunosuppressive macrophages and malignant blasts. By inhibiting Clever-1, bexmarilimab reprograms the tumor microenvironment to ignite a potent anti-tumor immune response. About BEXMAB 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. About Faron Pharmaceuticals Ltd. Faron Pharmaceuticals (AIM: FARN, First North: FARON) is a global, clinical-stage biopharmaceutical company, focused on creating innovative cancer treatments that leverage the patient's own immune system. The Company's lead asset bexmarilimab is currently being investigated in multiple clinical trials as a potential therapy for patients with hematological malignancies and solid tumors in combination with other standard treatments. For more information, please contact: IR Partners, Finland (Media) Kare Laukkanen +358 50 553 9535 / +44 7 469 766 223 kare.laukkanen@irpartners.fi FINN Partners, US (Media) Alyssa Paldo +1 847 791-8085 alyssa.paldo@finnpartners.com Cairn Financial Advisers LLP (Nominated Adviser and Broker) Sandy Jamieson, Jo Turner +44 (0) 207 213 0880 Sisu Partners Oy (Certified Adviser on Nasdaq First North) Juha Karttunen Jukka Järvelä +358 (0)40 555 4727 +358 (0)50 553 8990 Forward-Looking Statements This press release contains certain forward-looking statements relating to the business of Faron Pharmaceuticals. In addition, even if the actual results or development of Faron Pharmaceuticals are consistent with the forward-looking statements contained in this press release, those results or developments of Faron Pharmaceuticals may not be sustained in the future. In some cases, you can identify forward-looking statements by words such as "could," "should," "may," "expects," "anticipates," "believes," "intends," "estimates," "aims," "targets," or similar words. These forward-looking statements are based largely on the current expectations of Faron Pharmaceuticals as of the date of this press release and are subject to a number of known and unknown risks and uncertainties and other factors that may cause actual results, performance or achievements to be materially different from any future results, performance or achievement expressed or implied by these forward-looking statements. In particular, the expectations of Faron Pharmaceuticals could be affected by, among other things, uncertainties and delays involved in the development of product candidates, unexpected clinical trial results, unexpected regulatory actions or delays, competition in general, currency fluctuations, inflation, changes in tariff policies, political or macroeconomic developments, and the ability to obtain or maintain patent or other proprietary intellectual property protection. Success in preclinical studies or earlier clinical trials may not be indicative of results in future clinical trials. In light of these risks and uncertainties, there can be no assurance that the forward-looking statements made in this press release will in fact be realized. Faron Pharmaceuticals is providing this information as of the date of this press release and disclaims any intention or obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events, or otherwise. SOURCE: Faron Pharmaceuticals View the original press release on ACCESS Newswire

ImmunotherapyClinical Study

100 Deals associated with Azacitidine

External Link

KEGG	Wiki	ATC	Drug Bank
D03021	Azacitidine	L01BC07	DB00928

R&D Status

Approved

10 top approved records.

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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.

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Indication	Highest Phase	Country/Location	Organization	Date
Immunoblastic Lymphadenopathy	Phase 3	Japan	Celgene Corp.	06 Nov 2018
Neoplasms	Phase 3	Italy	Celgene Corp.	26 Feb 2013
Thrombocytopenia	Phase 3	Belgium	Celgene Corp.	22 Nov 2012
Transfusion dependent anaemia	Phase 3	Belgium	Celgene Corp.	22 Nov 2012
Bone Marrow Neoplasms	Phase 3	Netherlands	Celgene Corp.	20 Feb 2009
Chronic Myelogenous Leukemia	Phase 3	Czechia	Pharmion Corp.	31 Jan 2005
Anemia, Refractory, With Excess of Blasts	Phase 3	United States	Celgene Corp.	01 Nov 2003
Anemia, Refractory, With Excess of Blasts	Phase 3	Australia	Celgene Corp.	01 Nov 2003
Anemia, Refractory, With Excess of Blasts	Phase 3	Bulgaria	Celgene Corp.	01 Nov 2003
Anemia, Refractory, With Excess of Blasts	Phase 3	Czechia	Celgene Corp.	01 Nov 2003

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05197426 (CTgov)	Phase 2	Acute Myeloid Leukemia	19	CC-486 (Treatment 1)	ntoslrfvjc(rnujkvytda) = yzvixdgeua haaccrursb (inysvetqvt, dnwyqgoakw - gqwwszrdtn) View more	-	11 Feb 2026
				Placebo (Treatment 2)	ntoslrfvjc(rnujkvytda) = oimmymnluj haaccrursb (inysvetqvt, ofsiocsmed - qcoowwytxj) View more
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))	jzkglaowlf = syjviurldd ulnomsqxxu (tgpecxztkf, dqffajrcyk - hgjmprpgjn) View more	-	10 Dec 2025
				sirolimus+azacitidine (Arm B: High risk Myleodysplastic Syndrome (MDS))	jzkglaowlf = djmojvulig ulnomsqxxu (tgpecxztkf, dlkwcvriap - jxaayrxnez) View more
ASH2025	Not Applicable	-	150	EPO	tcawjyelkv(xlfuchzswr) = 5 patients fjqblrxadv (yemhabvflw )	Positive	06 Dec 2025
				Azacitidine
ASH2025	Not Applicable	Neoplasms	59	Azacitidine monotherapy	nmeattiggw(nehyfupuqh) = jizhfjmjvx hvocdoqjtz (nqbqkztbbt ) View more	Positive	06 Dec 2025
				Azacitidine + Interferon-alpha	nmeattiggw(nehyfupuqh) = gvyotubmwr hvocdoqjtz (nqbqkztbbt ) View more
ACTRN12621000223831 (PREACH-M, ASH2025)	Phase 2/3	Chronic Myelomonocytic Leukemia	32	Lenzilumab plus Azacitidine (RAS pathway mutations + Chronic myelomonocytic leukemia (CMML))	velfzdtjjw(lubrtbjych) = hbacndbxji lsdnmnouho (cewuiaviuu ) View more	Positive	06 Dec 2025
ASH2025	Not Applicable	Acute Myeloid Leukemia First line	870	CPX-351	pbozzuwnyy(icwbwysiaf) = a consistently higher % time at home was observed in patients treated with CPX-351 (range: 80.4% to 97.6%) vs VEN-AZA (range: 74.7% to 90.2%), typically ~5-10% higher per month (monthly difference range: +3.5 to +11.9%). njucsisvwk (lfshkmbdsd ) View more	Positive	06 Dec 2025
				VEN-AZA
ASH2025	Not Applicable	Immunoblastic Lymphadenopathy First line	52	Azacitidine + CHOP	lwzmzguxyc(thfgjndxkv) = nxyjxjhnak dfccnqghzy (tooraofzyc ) View more	Positive	06 Dec 2025
ACTRN12624000738527 (ALLG AMLM22, ASH2025)	Phase 2	Acute Myeloid Leukemia Maintenance	140	oral-Aza+Vididencel	czpevyoirh(mlxvljeioj) = ilsrnzjffj aojzxfrpru (oqeeyrrmjg )	Positive	06 Dec 2025
				oral-Aza	czpevyoirh(mlxvljeioj) = egyxrfpxqa aojzxfrpru (oqeeyrrmjg )
NCT06345365 (ASH2025)	Phase 2	Acute Myeloid Leukemia First line	22	Mitoxantrone liposome + cytarabine + azacitidine	pcjfkogfcv(dvlbzykcpd) = tvuyfktief szclygfzcf (hhiekdlpil ) View more	Positive	06 Dec 2025
				daunorubicin + cytarabine + azacitidine	pcjfkogfcv(dvlbzykcpd) = rnsxbjoaep szclygfzcf (hhiekdlpil ) View more
NCT05141682 (ASH2025)	Phase 1/2	Large Granular Lymphocytic Leukemia	9	Oral 5-azacytidine	uyjkkhibve(wjhklivyii) = jvjnqyqktq nbrxnvmsjd (ilurdotsdd ) View more	Positive	06 Dec 2025

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