Syndax and Incyte report topline data from the pivotal AGAVE-201 trial

Syndax Pharmaceuticals (SNDX) and Incyte (INCY) announced positive topline data from the pivotal AGAVE-201 trial of axatilimab, an anti-CSF-1R antibody, in adult and pediatric patients with chronic graft-versus-host disease, or GVHD, following two or more prior lines of therapy. The trial achieved its primary endpoint across all cohorts, with patients treated with axatilimab at doses of 0.3 mg/kg every two weeks, 1.0 mg/kg every two weeks and 3.0 mg/kg every four weeks demonstrating overall response rates, or ORR, within the first six months of treatment of 74%, 67% and 50%, respectively. Responses were achieved across key patient subgroups, including those with prior exposure to ruxolitinib, belumosudil and/or ibrutinib. Based on these results, and pending agreement from the FDA, Syndax and Incyte intend to submit a Biologics License Application to the FDA by year-end 2023. The AGAVE-201 pivotal study enrolled a total of 241 patients across 121 sites in 16 countries. Patients enrolled in the trial had received a median of four prior systemic therapies with 74% having previously received ruxolitinib, 23% having previously received belumosudil and 31% having previously received ibrutinib. 54% of these patients had at least four organs involved at baseline including 45% with lung involvement.