The US Food and Drug Administration (FDA) has approved Incyte and Syndax Pharmaceuticals’ antibody therapy, Niktimvo (axatilimab-csfr), as a third-line treatment for chronic graft versus host disease (GvHD) in adults and children who weigh at least 40kg.
Niktimvo is the second US FDA-approved therapy for chronic GvHD in Incyte’s portfolio. The other product, Jakafi (ruxolitinib), raked in $2.6bn in sales last year, as per Incyte’s financials. GlobalData expects Niktimvo to generate sales of over $305m by 2030.
GlobalData is the parent company of Pharmaceutical Technology.
The companies plan to seek FDA approval for two smaller vial sizes for Niktimvo before a US launch, which is expected in Q1 2025. Niktimvo is a colony-stimulating factor 1 receptor (CSF1R)-targeting monoclonal antibody. It is co-marketed by Incyte and Syndax in the US, with the former having exclusive commercialisation rights outside the US. Syndax acquired the worldwide rights for Nikimvo from UCB in 2016.
The FDA approval was based on the positive data from the Phase II AGAVE-201 trial (NCT04710576). The study met its primary endpoint and had a 75% overall response rate within six months of receiving Niktimvo. The therapy had a median time to response of 1.5 months.
The commonly observed side effects in the Phase II study included increased enzymatic levels of aspartate aminotransferase, blood creatine phosphokinase, lipase, blood lactate dehydrogenase, and alanine aminotransferase, indicative of tissue injuries. Serious adverse events were observed in 42.3% of the participants, with 37 participants discontinuing treatment due to these.
There are multiple therapies currently in development for treating GvHD. Pfizer’s sirolimus, a macrolide lactams derivative drug, and Eli Lily’s Olumiant (baricitinib) are currently being investigated in Phase II trials. Equillium’s IgG1 monoclonal antibody, itolizumab, is currently being evaluated in a Phase III clinical trial (NCT05263999).
Mesoblast is also developing a cell therapy for steroid-refractory acute GvHD. However, the company has had two FDA rejections for remestemcel-L, with the agency requesting additional data to approve the therapy.
Incyte is also evaluating Nikitimo as a first-line combination treatment with Jakafi for chronic GvHD in a Phase II trial (NCT06388564), which is expected to start recruitment by the end of this year. A Phase III trial investigating Nikitimo and steroids in the same indication is also slated for initiation by the end of 2024.
Syndax is also investigating Nikitimo in patients with idiopathic pulmonary fibrosis. The Phase II trial (NCT06132256) is expected to enrol approximately 135 participants, with the study’s completion estimated in Q2 2025.
