Incyte and Syndax Pharmaceuticals’ Niktimvo (axatilimab-csfr) has been approved by the US Food and Drug Administration (FDA) to treat chronic graft versus host disease (GVHD).
The anti-CSF-1R antibody has been specifically authorised for use in adult and paediatric patients who weigh at least 40kg and have failed on at least two prior lines of systemic therapy.
Approximately 42% of patients receiving an allogeneic stem cell transplant will develop GVHD, which occurs when the donated cells initiate an immune response and attack the transplant recipient’s organs.
There are two main types of GVHD: acute and chronic. Almost 50% of patients who develop chronic disease, which begins more than 100 days after transplant, will require at least three lines of treatment.
Niktimvo is designed to target key drivers of inflammation and fibrosis in chronic GVHD and the FDA’s approval was supported by data from the phase 2 AGAVE-201 study, in which treatment with the drug resulted in durable responses across all organs studied and patient subgroups.
Among those who received Niktimvo at the approved dose of 0.3mg/kg every two weeks, 75% achieved an overall response rate within the first six months of treatment and 60% maintained a response at one year.
The trial also met a key exploratory endpoint, with 56% of patients achieving at least a seven-point improvement in the modified Lee Symptom Scale score.
Syndax’s chief executive officer, Michael Metzger, said the approval represents a “significant treatment advancement” for this patient population.
“We look forward to bringing this first-in-class anti-CSF-1R antibody to patients in need of new treatment options, while also continuing to explore [Niktimvo’s] potential in combination with other standard of care therapies for chronic GVHD and in other indications.”
A phase 2 trial evaluating Niktimvo alongside ruxolitinib and a late-stage trial of the drug in combination with steroids are expected to begin by the end of the year. Niktimvo is also being studied in an ongoing phase 2 trial in patients with idiopathic pulmonary fibrosis.
Niktimvo will be co-commercialised by Incyte and Syndax in the US, while Incyte has exclusive commercialisation rights for Niktimvo outside the country.