Syndax Pharmaceuticals Inc. (Nasdaq: SNDX) and Incyte Corporation (Nasdaq: INCY) have announced positive topline results from the pivotal AGAVE-201 trial of axatilimab in chronic graft-versus-host disease (GVHD). The trial successfully met its primary endpoint, demonstrating an overall response rate (ORR) of 74% when administered at a dose of 0.3 mg/kg every two weeks. Furthermore, the data revealed that 60% of patients continued to respond to treatment even after one year, indicating a durable response.
These promising results highlight the potential therapeutic value of axatilimab as a first-in-class CSF-1R monoclonal antibody for chronic GVHD. In light of these findings, Syndax and Incyte have plans to submit a Biologics License Application (BLA) by the end of 2023, further advancing the development and potential availability of this treatment option.
The AGAVE-201 trial enrolled a total of 241 patients from various countries who had previously undergone two or more lines of therapy for