AB Science will hold a virtual conference on Thursday, October 16, 2025 from 2pm to 3pm CET to provide an update on the Phase 1 study with AB8939
PRESS RELEASE
AB SCIENCE RELEASES INITIAL DATA ON THE COMBINATION OF AB8939 WITH VENETOCLAX FOR THE TREATMENT OF REFRACTORY OR RELAPSED ACUTE MYELOID LEUKEMIA (AML): THE FIRST THREE PATIENTS, ALL OF WHOM HAVE AML WITH VERY UNFAVORABLE GENETIC PROFILES, ARE RESPONDING TO TREATMENT
- The combination treatment is well-tolerated, with no hematological toxicity
- The disease control rate is 100% (3/3)
- The partial response rate is 100% (3/3), including one patient in complete remission
- These results were obtained after the first cycle of treatment (14 days of treatment) in patients receiving third- or fourth-line treatment, two of whom had previously progressed on venetoclax in combination with other chemotherapies
- These three patients all have very difficult to treat cytogenetic profiles, including TP53 mutation and complex karyotypes, gene mutations that typically have a poor prognosis due to their aggressive disease course and treatment resistance
- Phase 1 continues with a new stage that will evaluate a higher dose of AB8939, still in combination with venetoclax
- These results corroborate the mechanism of action of AB8939, which is capable of destabilizing microtubules by evading multi-drug resistance and targeting cancer stem cells without eliminating non tumoral stem cells
AB SCIENCE WILL HOLD A VIRTUAL CONFERENCE ON THIS TOPIC ON THURSDAY, OCTOBER 16, 2025, FROM 2PM TO 3PM CET WITH THREE RENOWNED EXPERTS IN AML
- Prof. Nicholas J. Short, MD, Associate Professor and Co-Lead of Section of Developmental Therapeutics, Department of Leukemia, MD Anderson Cancer Center
- Prof. Olivier Hermine, MD, PhD, Head of the Hematology Department at Necker-Enfants Malades Hospital, Paris, France
- Prof. Christian Auclair, (PharmD, PhD), Emeritus Professor
Paris, October 14, 2025, 8.30am
AB Science SA (Euronext - FR0010557264 - AB) today provides an update on the Phase 1 study of the molecule AB8939 and, in particular, on the initial clinical data for the combination of AB8939 + Venetoclax in the first three patients with acute myeloid leukemia (AML) associated with a very unfavorable genetic profile.
AB8939 is a drug candidate that jointly targets cancer cells by destabilizing microtubules, which are essential for cell division, and also targeting cancer stem cells by inhibiting enzymes (ALDH1A1 and ALDH2) that are essential for maintaining their physiological state and survival.
AB8939 is currently being evaluated in a Phase 1 clinical trial (study AB18001, NCT05211570) in patients with refractory and relapsed AML.
The Phase 1 clinical trial of AB8939 has completed its first two stages, which consisted of determining the maximum tolerated dose (MTD) after 3 and 14 consecutive days of monotherapy, respectively. In both cases, the MTD was 21.3 mg/m².
The third stage, currently underway, involves evaluating the combination of AB8939 + Venetoclax. Three patients were evaluated at the first dose level (AB8939 14 days at a dose of 16 mg/m² + Venetoclax 14 days).
Nicholas J. Short, MD, Associate Professor and Co-Lead of Section of Developmental Therapeutics, Department of Leukemia, MD Anderson Cancer Center, said, "The results observed in these high-risk AML patients are impressive, particularly in the two patients whose leukemia had progressed on venetoclax. These initial results are very encouraging and justify the continuation of patient treatment with additional cycles, particularly in view of the mechanism of AB8939 on cancerous hematopoietic stem cells."
At the end of this third stage, an AB8939 + venetoclax expansion phase will be initiated in a group of about 15 patients with a more homogeneous profile than in the previous stages of phase 1, namely patients in their second- or third-line of treatment and with a poor prognosis (TP53 mutant, MECOM, NRAS mutant) in order to confirm the initial promising clinical data before initiating a registration clinical trial.
Virtual conference
AB Science will hold a virtual conference on Thursday, October 16, 2025, from 2pm to 3pm CET.
ZOOM link to the conference (audio + presentation): Access to the conference.
The purpose of this virtual conference will be to present in more detail the initial clinical data on the combination of AB8939 + venetoclax in the first three patients with refractory and relapsed AML associated with a very unfavorable genetic profile.
The following individuals will participate in the virtual conference:
- Nicholas J. Short, MD, Associate Professor and Co-Lead of Section of Developmental Therapeutics, Department of Leukemia, MD Anderson Cancer Center
Professor Short is a clinical and translational investigator in adult acute leukemias, with a particular emphasis on the development of phase I and II investigator-initiated clinical trials of novel agents and combinations for patients with acute myeloid leukemia (AML) and acute lymphoblastic leukemia (ALL). His major contributions to leukemia research include: developing new immunotherapy-based frontline regimens in Philadelphia chromosome-negative B-cell ALL, developing chemotherapy-free regimens in Philadelphia chromosome-positive ALL, establishing the clinical utility of high-sensitivity next-generation sequencing-based MRD assays in ALL, developing novel MRD-directed therapies in AML and ALL, and developing novel regimens for older adults with FLT3-mutated AML. He serves as principal investigator or co-principal investigator on over a dozen phase I and II clinical trials and has authored over 250 peer-reviewed manuscripts in the field of leukemia. For his clinical and translational accomplishments in the field of leukemia, he has been awarded the ASCO Young Investigator Award and the ASH Junior Faculty Scholar in Clinical Research.
- Olivier Hermine, MD, PhD, Head of the Hematology Department at Necker-Enfants Malades Hospital, Paris, France
Olivier Hermine is Professor of Hematology at Paris Descartes University, Head of the Hematology Department at Necker-Enfants Malades Hospital, member of LYSA, and Director of the CALYM team "Cellular and Molecular Mechanisms of Hematological Disorders and Therapeutic Implications" at the IMAGINE Inserm U 116 CNRS ERL 8654 institute. He is also coordinator of the Reference Center for Mastocytosis (CeReMast), co-founder and director of the scientific committee of AB Science.
His research topics include lymphoproliferative disorders linked to the hepatitis C virus, mantle cell lymphomas, and the regulation of erythropoiesis. He is the author or co-author of more than 900 scientific publications.
- Christian Auclair, PharmD, PhD, Emeritus Professor
Professor Auclair holds a doctorate in pharmaceutical sciences. He is co-founder and former director of the doctoral school of oncology at the Faculty of Medicine of Paris-Saclay University. He is former director of the biology department at the École Normale Supérieure de Cachan (now ENS Paris-Saclay) and director of UMR 8113 at the CNRS. He was also deputy scientific director of the CNRS's life sciences department. He is the author of more than 120 publications in the field of antitumor pharmacology and virology. He is co-founder and scientific advisor to AB Science.
Orphan Drug Status
In April 2025, AB Science announced that the molecule AB8939 had been granted orphan drug designation by the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) for the treatment of acute myeloid leukemia (AML).
The molecule AB8939 had already obtained orphan drug designation from the US Food and Drug Administration (FDA) for AML.
About AB8939
AB8939 is a new synthetic molecule which jointly targets cancer cells, by destabilizing the microtubules essential for cell division, and cancer stem cells, by inhibiting enzymes (ALDH1A1 and ALDH2) essential for maintaining their physiological state and survival. The molecule ‘1-{4-[2-(5-ethoxymethyl-2-methylphenylamino)-oxazol-5-yl]phenyl}imidazolidin-2-one’ is the chemical name of AB8939. The intellectual property of AB8939 is 100% owned by AB Science.
About AB Science
Founded in 2001, AB Science is a pharmaceutical company specializing in the research, development and commercialization of protein kinase inhibitors (PKIs), a class of targeted proteins whose action are key in signaling pathways within cells. Our programs target only diseases with high unmet medical needs, often lethal with short term survival or rare or refractory to previous line of treatment.
AB Science has developed a proprietary portfolio of molecules and the Company’s lead compound, masitinib, has already been registered for veterinary medicine and is developed in human medicine in oncology, neurological diseases, inflammatory diseases and viral diseases. The company is headquartered in Paris, France, and listed on Euronext Paris (ticker: AB).
Further information is available on AB Science’s website: www.ab-science.com.
Forward-looking Statements - AB Science
This press release contains forward-looking statements. These statements are not historical facts. These statements include projections and estimates as well as the assumptions on which they are based, statements based on projects, objectives, intentions and expectations regarding financial results, events, operations, future services, product development and their potential or future performance.
These forward-looking statements can often be identified by the words "expect", "anticipate", "believe", "intend", "estimate" or "plan" as well as other similar terms. While AB Science believes these forward-looking statements are reasonable, investors are cautioned that these forward-looking statements are subject to numerous risks and uncertainties that are difficult to predict and generally beyond the control of AB Science and which may imply that results and actual events significantly differ from those expressed, induced or anticipated in the forward-looking information and statements. These risks and uncertainties include the uncertainties related to product development of the Company which may not be successful or to the marketing authorizations granted by competent authorities or, more generally, any factors that may affect marketing capacity of the products developed by AB Science, as well as those developed or identified in the public documents published by AB Science. AB Science disclaims any obligation or undertaking to update the forward-looking information and statements, subject to the applicable regulations, in particular articles 223-1 et seq. of the AMF General Regulations.
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AB Science
Financial Communication & Media Relations
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