Clevegen (bexmarilimab) / Faron Pharma - LARVOL DELTA

Efficacy of macrophage checkpoint Clever-1 inhibition with bexmarilimab plus azacitidine in myelodysplastic syndrome: Results from the ph1/2 BEXMAB study. (ASCO 2025) - P1/2 | "Clinical Trial Registration Number: NCT05428969 The abstract will be released to the public on May 22, 2025 at 4:00 PM CDT"

Clinical • Hematological Malignancies • Myelodysplastic Syndrome • Oncology

Faron announces acceptance of bexmarilimab Phase II data for oral presentation at ASCO 2025 (Faron Press Release) - "Faron Pharmaceuticals Ltd...announces that new Phase II data from its ongoing BEXMAB study evaluating bexmarilimab in high-risk myelodysplastic syndromes (HR-MDS) will be presented as a part of a Rapid Oral Abstract Session at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting, taking place from 30 May to 3 June 2025, in Chicago, Illinois, USA....The abstract, selected for oral communication, highlights the efficacy and tolerability of bexmarilimab, Faron’s novel humanized anti-Clever-1 antibody, with no dose-limiting toxicities during dose escalation, when used in combination with standard-of-care, azacitidine, treatment in patients with both treatment naïve (frontline) and relapsed/refractory (r/r) HR MDS patients."

P2 data • Myelodysplastic Syndrome

Inside Information: Faron Announces Positive Phase II results in higher-risk myelodysplastic syndrome (Faron Press Release) - P1/2 | N=181 | BEXMAB (NCT05428969) | Sponsor: Faron Pharmaceuticals Ltd | "Faron Pharmaceuticals Ltd...announced positive topline results of the BEXMAB trial, which shows a high overall response rate (ORR) among both frontline as well as relapsed/refractory (r/r) HR MDS patients treated with a combination of bexmarilimab and azacitidine...According to this first fully enrolled Phase II analysis, the treatment continues to be well tolerated without any dose-limiting toxicity in r/r HR MDS patients with no other currently effective treatment options. A high objective response rate of 63% was observed in this initial data cut. The median overall survival remains at the same level as previously reported. Among treatment naïve (frontline phase 1) HR MDS patients, an ORR of 76% was observed...Faron is planning for a Phase III trial, pending U.S Food and Drug Administration (FDA) End of Phase II meeting Feedback"

New P3 trial • P2 data • Myelodysplastic Syndrome

Faron Announces Acceptance of Bexmarilimab Data for Oral Presentation at 2025 Annual MDS Foundation Congress (Faron Press Release) - "Faron Pharmaceuticals Ltd...announces that data from its ongoing BEXMAB study evaluating bexmarilimab in patients with high-risk myelodysplastic syndromes (HR-MDS) have been accepted for an oral presentation on May 10th in a plenary session at the 18th International Congress on Myelodysplastic Syndromes (MDS 2025), taking place on May 7-10, 2025 in Rotterdam, Netherlands."

P1/2 data • Myelodysplastic Syndrome

Inside Information: Faron Receives Positive EMA Opinion on Orphan Drug Designation for Bexmarilimab for the treatment of myelodysplastic syndrome (MDS) (Faron Press Release) - "Faron Pharmaceuticals Ltd...announces that their lead candidate bexmarilimab has been issued positive opinion on Orphan Drug Designation (ODD) for the treatment of myelodysplastic syndrome (MDS) in combination with azacitidine by the Committee for Orphan Medicinal Products (COMP) of the European Medicine Agency (EMA)....'The interim results of bexmarilimab’s ongoing phase I/II BEXMAB MDS trial reviewed by the EMA included also a median Overall Survival estimate'."

Orphan drug • Myelodysplastic Syndrome

Inside Information: FDA Grants Orphan Drug Designation for Bexmarilimab in MDS (Faron Press Release) - "Faron Pharmaceuticals Ltd...announces that their lead candidate bexmarilimab has been granted Orphan Drug Designation for the treatment of myelodysplastic syndromes (MDS) by the USA Food and Drug Administration (the FDA)."

Orphan drug • Myelodysplastic Syndrome

Proposed Issue and Placing (Faron Press Release) - "Faron Pharmaceuticals Ltd...announces a proposed private placement to raise preliminarily approximately EUR 10 million before expenses to a limited number of institutional investors and other investors ('Placing'). Carnegie and Bryan Garnier are acting as Joint Bookrunners in the Placing....The proceeds of the Placing are to be used to advance the development of the Company’s pipeline and to strengthen the financial position of the Company. Bexmarilimab development: Securing topline readout of Phase II BEXMAB trial in April 2025. Continuation of the BEXMAB Phase II trial to generate high-quality follow-up data (duration of response and survival); Preparing for a meeting with FDA after Phase II topline response rate readout."

Financing • P1/2 data • Acute Myelogenous Leukemia • Myelodysplastic Syndrome

Final Patient Identified for the BEXMAB Study (Faron Press Release) - "Faron Pharmaceuticals Ltd...announces that, the final patient has been identified for the BEXMAB Phase II dose optimization study in refractory or relapsed myelodysplastic syndrome (r/r MDS), as well as the BEXMAB Phase I/II study in frontline high risk (HR) MDS. The topline response rate read out is thus expected to become available in April 2025. Detailed data will be presented later at upcoming major medical conferences. The reportable data set will consist of approximately 35 r/r MDS patients and 20 frontline HR MDS patients. The r/r MDS population will consist of 32 patients treated with either 3mg/kg or 6mg/kg dose of bexmarilimab per the U.S. Food and Drug Administration (FDA) Project Optimus guidance, and three r/r MDS patients treated with 1mg/kg dose of bexmarilimab in Phase I....The BEXMAB trial will continue enrolling CMML patients and there is also the possibility to expand the enrollment of r/r MDS patients under the current protocol."

P1/2 data • Trial status • Chronic Myelomonocytic Leukemia • Myelodysplastic Syndrome

Encouraging Efficacy of Bexmarilimab with Azacitidine in Relapsed or Refractory MDS in Bexmab Ph1/2 Study (ASH 2024) - P1/2 | "Preliminary data suggest significant changes in the expression of genes related to blast cell energy production and macrophage activity induced by bexmarilimab and azacitidine treatment. Conclusions Bexmarilimab plus azacitidine is well tolerated and results in promising clinical efficacy in r/r MDS patients after HMA failure."

Clinical • Bone Marrow Transplantation • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Myelodysplastic Syndrome • Neutropenia • Oncology • CD4 • CD8 • TP53

Faron presents BEXMAB data at ASH Annual Meeting (PRNewswire) - P1/2 | N=181 | BEXMAB (NCT05428969) | Sponsor: Faron Pharmaceuticals Ltd | "Overall response rate of 80% (16 out of 20) in refractory or relapsed HMA failed MDS patient population (r/r MDS); Observed responses were primarily deep and durable with 70% (14 out of 20) r/r MDS patients achieving complete response (CR) / marrow complete remission (mCR) / partial response (PR). Four patients have moved on to receive a bone marrow transplant; Estimated median overall survival (mOS) of approximately 13.4 months in r/r MDS population; The combination of bexmarilimab and azacitidine remains well tolerated....Faron Pharmaceuticals Ltd...today announced full analysis of the positive Phase 2 interim readout presented at the 66th American Society of Hematology (ASH) Annual Meeting and Exposition."

P1/2 data • Myelodysplastic Syndrome

Positive Phase 2 Interim Results from BEXMAB Trial (Cision) - P1/2 | N=181 | BEXMAB (NCT05428969) | Sponsor: Faron Pharmaceuticals Ltd | "The treatment has been well tolerated, without any dose-limiting toxicity. The ORR in this otherwise untreatable population is 80% (16/20). Similar size patient cohorts treated with existing alternatives have reported 0-20% ORR, without deep and durable remissions. The estimated median overall survival of the 20 r/r MDS patients remains 13.4 months....The detailed efficacy, safety and biomarker results of the 20 r/r MDS patients treated in the BEXMAB trial will be presented at the 66th American Society of Hematology Annual Meeting. The BEXMAB trial is continuing to enroll patients as planned with the next efficacy data readout for the fully recruited BEXMAB trial patients expected around the end of Q1 2025."

P1/2 data • Myelodysplastic Syndrome

Macrophages as Potential Therapeutic Targets in Acute Myeloid Leukemia. (PubMed, Biomedicines) - "Existing research indicates that targeting macrophages could enhance the effectiveness of current AML treatments. This review addresses the importance of macrophages as therapeutic targets including relevant drugs investigated in clinical trials such as pexidartinib, magrolimab or bexmarilimab, but also provides new insights into lesser-known therapies, like macrophage receptor with a collagenous structure (MACRO) inhibitors and Toll-like receptor (TLR) agonists."

Journal • Review • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

BEXMAB: A Study to Assess Safety, Tolerability and Preliminary Efficacy of Bexmarilimab in Combination With Standard of Care in Patients With Hematological Malignancies (clinicaltrials.gov) - P1/2 | N=181 | Recruiting | Sponsor: Faron Pharmaceuticals Ltd | Trial completion date: Dec 2024 ➔ Dec 2025 | Trial primary completion date: Dec 2024 ➔ Apr 2025

Combination therapy • Trial completion date • Trial primary completion date • Acute Myelogenous Leukemia • Chronic Myelomonocytic Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology

FDA Grants Fast Track Designation for Bexmarilimab (Faron Press Release) - "Faron Pharmaceuticals...announces that their lead candidate bexmarilimab has been granted Fast Track Designation for the treatment of relapsed or refractory myelodysplastic syndrome (r/r MDS) in combination with azacitidine by the USA Food and Drug Administration (the FDA)....Given the previously reported promising results in both Phase 1 and 2 of Faron's BEXMAB trial when treating r/r MDS patients using a combination of bexmarilimab and azacitidine to overcome primary or developed resistance to azacitidine, bexmarilimab has been granted Fast Track Designation subsequent to the accelerated development plan proposed by the FDA in July 2024."

Fast track • Myelodysplastic Syndrome

Faron Pharmaceuticals shares jump 17% on positive FDA feedback (Proactiveinvestors) - "Shares in Faron Pharmaceuticals Limited (AIM:FARN) jumped almost 17% in early trading after receiving 'very positive' feedback from the US Food & Drug Administration on the design of its phase III clinical trial for bexmarilimab, a treatment for myelodysplastic syndrome, a type of bone marrow cancer. The FDA's feedback could streamline the development process, targeting newly diagnosed high-risk patients and eliminating the need for a separate study for resistant cases....The stock was changing hands for 115.55p, up 16.55p."

Stock price • Hematological Malignancies • Myelodysplastic Syndrome • Oncology

Faron gets positive FDA feedback on bexmarilimab study plans (Sharecast) - "Faron Pharmaceuticals announced positive feedback from the FDA regarding the clinical development plan for its drug candidate bexmarilimab, aimed at treating myelodysplastic syndrome (MDS), on Thursday. The AIM-traded firm said that in a formal meeting, the FDA acknowledged the challenges of conducting a randomised study with a comparator in the relapsed/refractory (r/r) setting, and instead proposed a confirmatory phase three study in frontline high-risk MDS (HR MDS). That approach, Faron explained, would eliminate the need for a separate phase three study in r/r MDS....Full approval would be based on survival data from the single phase three study."

FDA event • Hematological Malignancies • Myelodysplastic Syndrome • Oncology

Initial positive data from Phase 2 of BEXMAB (Cision) - P2 | N=181 | BEXMAB (NCT05428969) | Sponsor: Faron Pharmaceuticals Ltd | "Faron Pharmaceuticals Ltd...today provided first data from patients treated during the Phase 2 part of the ongoing BEXMAB trial in myelodysplastic syndrome (MDS) patients that have failed a hypomethylating agent (HMA)...There are now a total of 14 HMA-failed MDS patients treated in both Phase 1 & 2 with this novel combination. The treatment has been well tolerated, without any dose-limiting toxicity...The ORR in this otherwise untreatable population is 79% (11/14).The best responses for these 14 patients are as follows: 1 complete response (CR), 7 marrow complete remissions (mCR), 1 partial response (PR), 2 hematological improvements, 2 stable diseases (SD) and 1 progressive disease (PD)....'With these first results from the Phase 2 continuing the positive results already seen in Phase 1, we are committed to rapidly advancing Bexmarilimab to market, because patients are waiting for treatment options like this.'"

P2 data • Hematological Malignancies • Myelodysplastic Syndrome • Oncology

Faron Founders and bexmarilimab Developers Selected as Finalists for the European Inventor Award 2024 (GlobeNewswire) - "Dr. Markku Jalkanen...and Dr. Sirpa Jalkanen, co-founder and member of Faron’s Scientific Advisory Board, have been nominated in recognition of their research developing Faron’s wholly owned precision cancer immunotherapy candidate, bexmarilimab. One of the most advanced myeloid cell-targeting immunotherapy candidates in development, bexmarilimab is designed to target the Clever-1 receptor present on macrophages, activating anti-tumor immunity in hematological and solid tumor microenvironments to overcome resistance and provide better patient outcomes....Bexmarilimab is currently being investigated in the ongoing BEXMAB trial, evaluating the candidate’s safety and efficacy in combination with standard of care (SoC) in patients with hypomethylating agents (HMAs)-refractory or relapsed myelodysplastic syndrome (MDS), an aggressive myeloid leukemia with very few treatment options."

Clinical • Trial status • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology

Inside Information: Additional Positive Data from the Phase 1 Part of the BEXMAB Study in Both Higher-Risk HMA-Failed MDS and r/r AML (GlobeNewswire) - P1/2 | N=181 | BEXMAB (NCT05428969) | Sponsor: Faron Pharmaceuticals Ltd | "Latest readout of the BEXMAB study shows more responding patients and good durability of remission amongst HR HMA-failed MDS patients. 4/5 of the initial Phase 1 HR HMA-failed MDS patients were still alive after eight months of follow-up....3 additional HMA-failed HR MDS patients have been enrolled in Phase 1 part, leading to a total of 7 out of 8 patients responding, an overall response rate of 87.5%....Faron will be hosting a virtual webinar to discuss these data tomorrow, Tuesday, 19 March, at 11.00 EET/9am GMT."

P1 data • Hematological Malignancies • Myelodysplastic Syndrome • Oncology

Faron to Host Webcast to Discuss latest data from Phase 1 part of the BEXMAB Study of Bexmarilimab (GlobeNewswire) - "Faron Pharmaceuticals Ltd...today announces it will host a virtual BEXMAB update event on Tuesday, March 19 at 11.00 EET/9am GMT, following the most recent data cut-off and scheduled analysis....During the event the Company will discuss latest data from the ongoing BEXMAB trial in acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) patients, with a particular focus on the durability of remission and objective responses following treatment with bexmarilimab in combination with standard of care. This interactive event will also include an introduction to myeloid leukemia, insights into bexmarilimab’s unique mode of action and the Company’s latest understanding of the early and long-term efficacy of bexmarilimab treatment. Faron will also provide an update on bexmarilimab’s future development pathway and business opportunity."

P1 data • Acute Myelogenous Leukemia • Myelodysplastic Syndrome

Faron’s Financial Statement Release January 1 to December 31, 2023 (GlobeNewswire) - "Preparations are ongoing for the initiation of the Phase II BEXCOMBO trial evaluating bexmarilimab with PD-1 blockade, aimed at improving the clinical benefits from standard-of-care PD-1 blockade. The first proof-of-concept cohort under investigation will be head and neck cancer, followed by non-small cell lung cancers. Patient cohorts will comprise between 15 and 40 subjects, with the opportunity for subgroup enrichment....Given the positive results to date, the Company is exploring bexmarilimab's potential in frontline HR MDS, chronic myelomonocytic leukaemia (CMML) patients and considering further development and expansion opportunities with bexmarilimab in hematological cancers in the form of further partnerships."

New P2 trial • Chronic Myelomonocytic Leukemia • Head and Neck Cancer • Myelodysplastic Syndrome • Non Small Cell Lung Cancer

Detailed Analysis of BEXMAB Data Provides Insights into Patient Profiles of Responding HMA-Failed MDS Population (GlobeNewswire) - P1/2 | N=181 | BEXMAB (NCT05428969) | Sponsor: Faron Pharmaceuticals Ltd | "Faron Pharmaceuticals Ltd...today provided details from its further analysis of data from the completed Phase 1 part of the ongoing BEXMAB trial....The new analysis of data from the Phase 1 part of the trial explores the 100% overall response rate (ORR) achieved among both the higher-risk frontline and HMA-failed MDS patients treated with a bexmarilimab/azacitidine combination – 5 out of 5 patients in each population – and examines previous therapies in the patients’ treatment pathways....3 of the 5 patients were refractory to previous HMA-therapy, with progressive disease (PD) or stable disease (SD) being the best responses achieved from that therapy."

P1 data • Hematological Malignancies • Myelodysplastic Syndrome • Oncology

A Study to Assess Safety, Tolerability and Preliminary Efficacy of Bexmarilimab in Combination With Standard of Care in Patients With Hematological Malignancies (BEXMAB) (clinicaltrials.gov) - P1/2 | N=181 | Recruiting | Sponsor: Faron Pharmaceuticals Ltd | Trial primary completion date: Dec 2023 ➔ Dec 2024

Combination therapy • Trial primary completion date • Acute Myelogenous Leukemia • Chronic Myelomonocytic Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology

Inside information: Faron Announces First HMA-failed MDS Patient Dosed with Bexmarilimab as part of Phase 2 of BEXMAB Trial (GlobeNewswire) - "Faron Pharmaceuticals...announced that the first patient has been dosed in Phase 2 of the BEXMAB trial that evaluates the safety and efficacy of bexmarilimab, in combination with standard of care (SoC) in patients with hypomethylating agents (HMAs)-refractory or relapsed myelodysplastic syndrome (MDS), an aggressive myeloid leukemia with very few treatment options. The ongoing, randomized parallel-assigned Phase 2 part is enrolling 32 HMA-failed MDS patients at 3 mg/kg and 6 mg/kg dose levels of bexmarilimab. Dose levels have been selected in accordance with the FDA’s Project Optimus initiative, which aims to reform the paradigm of dose optimization and selection in oncology drug development. Patients are being randomized 1:1 between the doses before moving into a Phase 2/3 study expansion....Faron is currently opening additional sites to speed up the trial’s rapid recruitment, ahead of a future registrational study."

Trial status • Myelodysplastic Syndrome

Encouraging Efficacy Observed in Bexmab Study: A Phase 1/2 Study to Assess Safety and Efficacy of Bexmarilimab in Combination with Standard of Care in Myeloid Malignancies (ASH 2023) - P1/2 | "Treatment of AML bone marrow cells with BEX alone or in combination with azacitidine/venetoclax results in enhanced antigen presentation capacity and increased activation markers on effector T cells with synergistic effect on cell death. Additional clinical and pharmacodynamic data of the completed Ph1 of the study will be presented during the meeting. Ph2 of BEX plus azacitidine will open in the 2nd half of 2023 in HMA-failed r/r AML and/or higher risk MDS patients."

Clinical • Combination therapy • P1/2 data • Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Immunology • Leukemia • Myelodysplastic Syndrome • Oncology • Rare Diseases • CD8