Incyte and Syndax Pharmaceuticals announced the FDA approval of their jointly developed anti-CSF-1R antibody, branded Niktimvo, for treating chronic graft-versus-host disease (GVHD) in patients who have not responded to at least two prior treatments.
Niktimvo is the first approved anti-CSF-1R antibody designed to target inflammation and fibrosis in chronic GVHD, offering a new treatment option. GVHD is a serious complication of allogeneic stem cell transplants where the donor's immune cells attack the recipient's organs, leading to inflammation, fibrosis and potentially life-threatening damage.
The approval was based on the AGAVE-201 study, which demonstrated that Niktimvo provided durable responses across multiple organs and patient subgroups. In the study, 75% of patients treated with Niktimvo achieved an overall response rate within six months, with 60% maintaining the response at 12 months. The trial also showed significant improvements in the modified Lee Symptom Scale, which measures chronic GVHD symptoms.
The partners inked a worldwide collaboration and license agreement to develop and commercialize Niktimvo back in 2021, with Syndax receiving $152 million upfront plus a $35 million equity investment, with potential for $450 million in additional milestone payments.
Incyte and Syndax plan to launch the new treatment in the U.S. no later than early 2025, with plans to introduce smaller vial sizes to optimize patient dosing and minimize waste.
