Incyte: Phase 2 Trial Results Of Niktimvo In Chronic Graft-Versus-Host Disease Meet Primary Goal

(RTTNews) - Incyte (INCY) and Syndax Pharmaceuticals (SNDX) announced that results from the pivotal Phase 2 AGAVE-201 trial of Niktimvo(axatilimab-csfr), 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 AGAVE-201 pivotal trial enrolled 241 patients with recurrent or refractory chronic GVHD who had received two or more prior systemic therapies, with 74% having previously received ruxolitinib, 31% having previously received ibrutinib and 23% having previously received belumosudil. Patients were enrolled across 121 sites in 16 countries.

The results show that 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 (ORR) 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. Among the patients who had a response in the 0.3 mg/kg dose cohort, an estimated 60% of patients maintained a response at 12 months.

Clinically meaningful reduction in chronic GVHD symptoms was reported by 60% of patients in the 0.3 mg/kg dose cohort. In the same cohort, organ-specific responses, including complete responses (CRs), were seen across all organs studied, including lower gastrointestinal (GI), upper GI, esophagus, joints/fascia, mouth, lungs, liver, eyes and skin.

Additionally, responses were notable in fibrosis-dominated organs, including the esophagus (78%), joints and fascia (76%), lungs (47%) and skin (26%). Responses were observed across key patient subgroups, including objective response rates =75% in the 0.3 mg/kg cohort who received prior ibrutinib, ruxolitinib, and/or belumosudil.

On August 14, 2024, Incyte and Syndax announced the U.S. Food and Drug Administration's approval of 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 (88.2 lbs), with a recommended dosage of 0.3 mg/kg, up to a maximum dose of 35 mg, as an intravenous infusion over 30 minutes every two weeks until progression or unacceptable toxicity.

In the United States, Niktimvo will be co-commercialized by Incyte and Syndax Pharmaceuticals. 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 first quarter 2025.

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