Faron Pharmaceuticals (FARN) Study result summary

Study background and rationale

• Bexmarilimab is a first-in-class antibody targeting Clever-1/CLEVER-1, a receptor highly expressed in immunosuppressive macrophages and associated with poor outcomes in high-risk MDS and AML.

• High-risk MDS patients have limited treatment options, with bone marrow transplant feasible for only 5-10% and median survival after standard therapies as low as 4-6 months; survival after HMA failure is even shorter.

• The study used updated 2023 response criteria, emphasizing blood count recovery and patient-centric outcomes.

• Bexmarilimab aims to activate macrophages and enhance anti-tumor immunity, with promising preclinical and early clinical data.

• Limited treatment options for relapsed/refractory HR MDS highlight the need for novel therapies.

Mechanism of action and target validation

• CLEVER-1 is highly expressed on malignant cells in MDS and AML, associated with poor prognosis and treatment resistance.

• Bexmarilimab inhibits CLEVER-1, reprogramming macrophages to a pro-inflammatory state and enhancing T cell response.

• Baseline CLEVER-1 expression may predict response to therapy.

Study design and patient population

• BEXMAB is an open-label Phase 1/2 trial evaluating bexmarilimab plus azacitidine in high-risk MDS and AML, enrolling both frontline and relapsed/refractory patients.

• Dose escalation tested 1, 3, and 6 mg/kg; phase 2 focused on 3 and 6 mg/kg for dose optimization.

• 21 frontline and 32 relapsed/refractory patients enrolled, median ages 72 and 74, with a significant proportion harboring TP53 mutations (44% frontline, 41% r/r).

• Most patients were high or very high risk at baseline.

• The trial was presented at ASCO 2025 in a Rapid Oral Abstract Session.