Axatilimab meets ORR end point in chronic GVHD after 2 or more prior lines of therapy

The phase 2 AGAVE-201 trial—of the anti–CSF-R1 antibody axatilimab in adult and pediatric patients with chronic GVHD who received 2 or more lines of therapy—met its primary end point in all cohorts of patients, according to recent topline data. Overall response rates of 74% (95% CI: 63%-83%), 67% (95% CI: 55%-77%), and 50% (95% CI: 39%-61%) were seen among patients treated with axatilimab at 0.3 mg/kg every 2 weeks, 1.0 mg/kg every 2 weeks, and 3.0 mg/kg every 4 weeks, respectively. Responses were also observed across key subgroups, including those who had previously received ruxolitinib, belumosudil, and/or ibrutinib.