Syndax and Incyte announce publication of positive Niktimvo results in GVHD

Incyte (INCY) and Syndax (SNDX) announced that results from the pivotal Phase 2 AGAVE-201 trial of Niktimvo, an anti-CSF-1R antibody, in adult and pediatric patients with recurrent/refractory active chronic graft-versus-host disease, or GVHD, who had received at least two prior lines of systemic therapy were published in The New England Journal of Medicine. The trial enrolled 241 patients with recurrent or refractory chronic GVHD who had received two or more prior systemic therapies. The trial met the primary endpoint across all cohorts receiving Niktimvo 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. Patients in the 0.3 mg/kg every two weeks cohort achieved the highest overall response rate of 74% within the first six months of treatment. Patients in this cohort experienced a median time to response to Niktimvo of 1.7 months. Clinically meaningful reduction in chronic GVHD symptoms was reported by 60% of patients in the 0.3 mg/kg dose cohort. Responses were notable in fibrosis-dominated organs. Responses were observed across key patient subgroups. The most common treatment-emergent adverse events were consistent with the on-target effects of CSF-1R inhibition and with what was previously observed with Niktimvo treatment. On August 14, the FDA approved Niktimvo for the treatment of chronic GVHD after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg. On August 30, Niktimvo was added to the latest NCCN Clinical Practice Guidelines in Oncology. In the U.S., Niktimvo will be co-commercialized by Incyte and Syndax. Incyte has exclusive commercialization rights for Niktimvo outside of the U.S. The companies anticipate launching Niktimvo in the U.S. no later than early 1Q25.