Ireland
--:--IST
Recruiting Phase 2

Bexmarilimab + Azacitidine Versus Placebo + Azacitidine in Participants With Treatment-naïve Higher-risk Myelodysplastic Syndromes

NCT07672769 · tracked via the Priya Life Science UK tracker
Sponsor
Faron Pharmaceuticals Ltd
Phase
Phase 2
Started
2026-10
Last updated
2026-10-20

Condition(s) studied

Higher Risk Myelodysplastic Syndromes

Investigational drug(s) / intervention(s)

bexmarilimab (1mg/kg) →azacitidine →Placebobexmarilimab (3mg/kg) →

bexmarilimab (1mg/kg): 1mg/kg. Administered weekly (Q1W) with the opportunity to reduce frequency to biweekly (Q2W) based on time on treatment and disease response. Intravenous (IV) administration

azacitidine: Standard of care medication, administered per institutional guidelines/label

Placebo: Participants will receive saline placebo, prepared by the local site pharmacy to match bexmarilimab at point of dispensation. Administered on a schedule to match bexmarilimab

bexmarilimab (3mg/kg): 3mg/kg. Administered weekly (Q1W) with the opportunity to reduce frequency to biweekly (Q2W) based on time on treatment and disease response. Intravenous (IV) administration

Study summary

This Phase IIb study (BEXERA) will evaluate the safety and efficacy of bexmarilimab (FP-1305), an antibody targeting Clever-1, given in combination with azacitidine compared with azacitidine plus placebo in adults with treatment-naïve higher-risk myelodysplastic syndromes (HR-MDS). Participants will be randomized to receive bexmarilimab at one of two dose levels (1 mg/kg or 3 mg/kg) plus azacitidine, or placebo plus azacitidine. The primary aim is to select the recommended dose of bexmarilimab for subsequent development based on a predefined integration of clinical response and safety/tolerability.

Eligibility

Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
Inclusion Criteria: 1. Participant provides written informed consent. 2. Participant is ≥18 years of age. 3. Participant has newly diagnosed MDS with morphologically confirmed HR-MDS as defined according to 2022 World Health Organization classification (5th Edition, Annex 7). 1. IPSS-M classification of moderately high risk, high risk, and very high risk. 2. \<20% bone marrow blasts per bone marrow biopsy/aspirate at screening 4. Participant is eligible for azacitidine per local practice and willing to initiate trial therapy. 5. Participant has ECOG performance score 0 to 2. 6. Participant has life expectancy ≥3 months. 7. Participant has adequate organ function: creatinine clearance ≥30 mL/min (Cockcroft-Gault); indirect (unconjugated) bilirubin ≤1.5 times the upper limit of normal (ULN) (unless related to Gilbert's syndrome, in which case the indirect bilirubin levels must be \<3×ULN for inclusion); aspartate aminotransferase (AST) or alanine aminotransferase (ALT) ≤3×ULN. 8. Participant has baseline leukocyte count of \<20×109/L. Hydroxycarbamide use is permitted to meet this criterion. 9. Women of childbearing potential have a negative pregnancy test; participants of childbearing potential (and their partners) agree to use highly effective contraception during treatment and for ≥6 months after last dose. 10. Participant is willing and able to comply with protocol procedures and follow up. Exclusion Criteria: 1. Participant has a previous diagnosis of AML, has transformed to AML, or has MDS subtypes outside the scope or overlapping myeloid neoplasms: MDS evolved from pre-existing myeloproliferative neoplasms (MPN); MDS/MPN overlap (e.g., chronic myelomonocytic leukemia, acute chronic myeloid leukemia, juvenile myelomonocytic leukemia, unclassifiable); advanced myelofibrosis (MF Grade ≥3); or severe autoimmune hemolysis. 2. Participants who are considered appropriate candidates for immediate allogeneic haematopoietic stem cell transplantation (HSCT) at the time of screening are excluded, irrespective of transplant timing, donor availability, or planned bridging therapy. Determination of transplant candidacy should be based on institutional standards and routine clinical practice, including assessment of individual clinical factors such as age, performance status, comorbidities, organ function, disease risk, and donor suitability. 3. Participants with ≥20% blasts in peripheral blood or bone marrow or evidence of myeloid sarcoma (extramedullary AML). 4. Participant has a lack of screening cytogenetic data or demonstrated normal karyotype per local or central analysis, should the patient have \<5% blasts at screening. 5. Participant has received previous lines of anticancer therapy for MDS (disease-modifying therapy), including HMAs (e.g., azacitidine, decitabine), chemotherapy, or HSCT. Supportive care (e.g., transfusions, growth factors) is permitted. 6. Participant has clinically significant cardiac disease: recent myocardial infarction within 12 months; symptomatic congestive heart failure (New York Heart Association Class III or IV) or left ventricular ejection fraction (LVEF) \<40%; uncontrolled clinically significant arrhythmias; or congenital/familial long-QT syndrome or pre-excitation syndrome. 7. Participant has active, uncontrolled infection requiring IV antimicrobials; known active invasive fungal infection; uncontrolled severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection per local standards. 8. Participant has known active central nervous system (CNS) involvement by myeloid malignancy 9. All prior allo-HSCT within 6 months before Screening; ongoing clinically significant graft versus host disease (GVHD) requiring systemic immunosuppression. 10. Participant has active autoimmune disease requiring systemic therapy or requiring ≥10 mg/day prednisone (or equivalent) within 14 days prior to first dose; topical/inhaled/ophthalmic steroids permitted. (Immune conditions such as type 1 diabetes, controlled thyroid disease, vitiligo, psoriasis, alopecia are not exclusions.) 11. Participant has clinically relevant hepatic disease (e.g., Child-Pugh C) or ALT/AST \>3×ULN and bilirubin exceeding inclusion thresholds (indirect \[unconjugated\] bilirubin \>1.5×ULN \[unless related to Gilbert's syndrome, in which case the indirect bilirubin levels must be \>3×ULN\]); persistent chronic ulcers with high risk of infection per investigator's assessment. 12. Participant has received recent non-MDS related anticancer therapy or investigational agents within drug-specified washout periods (e.g., \<21 days from last IV/SC cytotoxic, \<14 days or \<5 half-lives for small-molecule therapy, \<4 weeks for other immunotherapies). 13. Participant has a history of another malignancy that is active, progressing, or has required systemic treatment within the past 2 years of screening, excluding non-melanoma skin cancer, carcinoma in situ treated with curative intent. 14. Participant has known uncontrolled human immunodeficiency virus, active hepatitis B virus, or hepatitis C virus with high-level viremia; participants with controlled viral infections on stable therapy may be eligible per local guidance. 15. Participant is pregnant or lactating. 16. Participant has prior exposure to bexmarilimab. 17. Participant has any condition, including psychiatric or substance-use disorder, that in the investigator's judgment would compromise informed consent, compliance, or interpretation of trial results. 18. Participant has a history of hypersensitivity to compounds related to immunotherapy or to any of their excipients. 19. Participant has undergone major surgery within 4 weeks of Cycle 1 Day 1.

Primary outcome measure(s)

  • Dose selection utility score utilising efficacy and safety components. — 3-4 months from last participant enrollment
    Dose-selection utility formula at the end of the primary dose-selection assessment window, calculated per dose using a pre-specified algorithm that integrates: * Efficacy component (achievement of CR + CReq per International Working Group \[IWG\] 2023 criteria). * Safety/tolerability component (rate of treatment-emergent Grade 3-5 adverse events or rate of treatment-related Grade 3-5 adverse events). For each arm, there will be an observed efficacy rate (PE) and toxicity rate (PT) and the utility calculation will be defined as U = PE - ωPT where the ω is a pre-specified weight.

Trial sites (35)

FacilityCityRegionStatus
City of Hope Medical Center Duarte California Not Yet Recruiting
University of Colorado Cancer Center Aurora Colorado Not Yet Recruiting
Yale Cancer Center New Haven Connecticut Not Yet Recruiting
Moffitt Malignant Hematology & Cellular Therapy at Memorial Healthcare System Pembroke Pines Florida Not Yet Recruiting
Mass General Brigham Cancer Institute Boston Massachusetts Not Yet Recruiting
University of North Carolina Lineberger Comprehensive Cancer Center Chapel Hill North Carolina Not Yet Recruiting
Vanderbilt University Medical Center Nashville Tennessee Not Yet Recruiting
The University of Texas MD Anderson Cancer Center Houston Texas Not Yet Recruiting
Medical College of Wisconsin - Clinical Cancer Center - Froedtert Hospital Milwaukee Wisconsin Not Yet Recruiting
Fakultní nemocnice Brno Brno Czechia Not Yet Recruiting
Fakultní nemocnice Hradec Králové Hradec Králové Czechia Not Yet Recruiting
Fakultni nemocnice Ostrava Ostrava-Poruba Czechia Not Yet Recruiting
Ustav hematologie a krevni transfuze Prague Czechia Not Yet Recruiting
Vseobecna fakultni nemocnice v Praze Prague Czechia Not Yet Recruiting
Helsinki University Hospital Comprehensive Cancer Center Helsinki Finland Not Yet Recruiting
Oulu University Hospital Oulu Finland Not Yet Recruiting
Tampere University Hospital Tampere Finland Not Yet Recruiting
Turku University Hospital Turku Finland Not Yet Recruiting
CHU de Bordeaux Bordeaux France Not Yet Recruiting
Hôpital Saint-Louis Paris France Not Yet Recruiting
CHU de Poitiers Poitiers France Not Yet Recruiting
The Institut Gustave Roussy Villejuif France Not Yet Recruiting
AOU SS. Antonio e Biagio e Cesare Arrigo Alessandria Italy Not Yet Recruiting
IRCCS Azienda Ospedaliero-Universitaria di Bologna Bologna Italy Not Yet Recruiting
INOC - Istituto Nazionale Oncologico Candiolo Candiolo Italy Not Yet Recruiting
IRCCS Istituto Europeo di Oncologia Milan Italy Not Yet Recruiting
Pratia Onkologia Katowice Katowice Poland Not Yet Recruiting
Aidport Clinical Trials Hospital Skórzewo Poland Not Yet Recruiting
MTZ Clinical Research Powered by Pratia Warsaw Poland Not Yet Recruiting
King's College Hospital London Greater London Recruiting
The Christie NHS Foundation Trust Manchester Greater Manchester Not Yet Recruiting
Maidstone and Tunbridge Wells NHS Trust Maidstone Kent Not Yet Recruiting
University Hospital Birmingham Nhs Trust Birmingham United Kingdom Not Yet Recruiting
The Beatson West of Scotland Cancer Centre Glasgow United Kingdom Not Yet Recruiting
Royal Cornwall Hospitals NHS Trust Truro United Kingdom Not Yet Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07672769 on ClinicalTrials.gov ↗ ← All trials in the UK