Faron Pharmaceuticals (FARN) Study Update summary

Study design and patient population

• BEXMAB is an open-label phase I/II trial enrolling relapsed/refractory MDS patients with high unmet need, median age 72.5 years, and high-risk disease profile.

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

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

• 50% of patients had received at least two prior lines of therapy.

• Bexmarilimab targets Clever-1 on macrophages, aiming to reprogram the tumor microenvironment and enhance immune response.

Efficacy results

• Overall response rate was 80%, with 5% complete response, 15% partial remission, and 50% marrow CR in relapsed/refractory MDS patients.

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

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

• 20% of patients proceeded to stem cell transplantation.

• Over 50% of patients had more than 50% reduction in bone marrow blasts.

Safety and tolerability

• BEXMAB was well tolerated, with only 35% experiencing BEX-related adverse events, all grade 1 or 2.

• No grade 3 or higher BEX-related adverse events or dose-limiting toxicities observed.

• Safety profile supports combination with other agents, including azacitidine and immunotherapies.

• The primary objective is to determine safety and tolerability of the combination therapy.