Incyte and Syndax's immunotherapy for transplant complications receives FDA approval

Incyte and Syndax Pharmaceuticals have been granted approval for Niktimvo (axatilimab-csfr) for the treatment of chronic graft-versus-host disease (GVHD) after failure of at least two prior lines of systemic therapy. The partners, which will co-commercialize the antibody drug in the US, anticipate a launch in the first quarter of 2025.

Chronic GVHD is a complication that can occur after an allogeneic stem cell transplant, where the immune cells from a donor reject and attack the transplant recipient’s organs. It is estimated that approximately 42% of recipients of this type of transplant develop GVHD, which is the leading cause of death for patients who receive bone marrow transplants. 

Currently, nearly 50% of patients who develop chronic GVHD require at least three lines of treatment to stabilize their immune system.

“With the approval of Niktimvo, patients with chronic GVHD whose disease has progressed after prior therapies now have a new treatment option with a novel mechanism of action to help address the serious and devastating complications associated with this disease,” said Hervé Hoppenot, Chief Executive Officer of Incyte.

Niktimvo is the first approved antibody targeting the colony stimulating factor 1 receptor (CSF-1R), a protein that is involved in the processes of inflammation and fibrosis seen in patients with chronic GVHD.

The approval was based on positive results from the AGAVE-201 study, a phase 2 clinical trial that recruited 241 patients with chronic GVHD who received at least two prior lines of systemic therapy. Data from the trial showed that patients who received Niktimvo had durable responses across all organs. Among those who received the antibody drug at the approved dose, 60% maintained a response after 12 months.

“Advanced chronic GVHD is characterized by the development of fibrotic tissue across multiple organ systems, (...) and can be extremely difficult to treat, leading to high rates of morbidity and mortality,” said Daniel Wolff, Head of the GVHD Center at the University Hospital Regensburg. “I am highly encouraged by the robust responses observed across all organs and patient subgroups within the heavily pre-treated population enrolled in the AGAVE-201 trial.

”Syndax Pharmaceuticals first acquired the exclusive worldwide rights to develop and commercialize axatilimab in 2016 from Belgian pharmaceutical company UCB. In 2021, Syndax and Incyte entered a license agreement for the co-development and co-commercialization of the antibody drug in the US. Outside of the US, Incyte has exclusive commercialization rights for Niktimvo.

For Incyte, Niktimvo is the second therapy to receive approval for chronic GVHD. The first one was Jakafi (ruxolitinib), which was approved by the FDA in 2021. Niktimvo is currently being evaluated as a frontline therapy for chronic GVHD in combination with other drugs. Incyte and Syndax expect to initiate phase 2 and phase 3 combination trials with Jakafi and with steroids, respectively, by the end of the year.