Faron Pharmaceuticals (FARN) Study update summary

Study design and patient population

• BEXMAB is an open-label phase I/II trial evaluating bexmarilimab plus azacitidine in relapsed/refractory MDS, with a median patient age of 72.5 years and high-risk disease profile.

• 45% of patients had TP53 mutations, 40% were pretreated with HMA plus venetoclax, and 15% had prior immune therapy.

• 70% had received at least one prior therapy, and 50% had at least two prior lines.

• 20 patients with at least one response evaluation were included in the interim analysis; full phase II will enroll 35 patients.

• Data cut-off was November 25, 2024, with ongoing enrollment and additional UK and US sites.

Efficacy results

• Overall response rate was 80%, with 70% achieving remission (CR/PR/mCR), and 55% had ≥50% reduction in bone marrow blasts.

• Median overall survival was 13.4 months, significantly higher than historical 4–6 months.

• 63% response rate in HMA/venetoclax pretreated patients; 56% in TP53-mutant subgroup.

• 20% of patients proceeded to stem cell transplantation, indicating potential long-term benefit.

• Updated results reinforce deep, clinically meaningful responses in a population with limited options.

Safety and tolerability

• BEXMAB was well tolerated, with no grade 3 or higher BEX-related adverse events or dose-limiting toxicities observed.

• No BEX-related discontinuations or dose-level safety concerns reported.

• Majority of patients experienced hematological improvements.