Syndax Pharmaceuticals and Incyte Corporation have announced positive topline data from the pivotal AGAVE-201 trial of axatilimab in adult and paediatric patients with chronic graft-versus-host disease (cGVHD).
The Phase II AGAVE-201 study (NCT04710576) uses axatilimab, an anti-CSF-1R antibody, at three different doses in patients with cGVHD following two or more prior lines of therapy. All 241 patients enrolled in the trial which took place across 16 countries, had a median of four prior systemic therapies. In all, 74% received ruxolitinib, 23% were administered belumosudil and 31% received brutinib. In addition, 54% of the patients also had at least four organs involved, including 45% with lung involvement.
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Topline results
The companies have reported that the trial achieved its primary endpoint across all cohorts. Patients treated at all doses, 0.3mg/kg, 1mg/kg of axatilimab every two weeks, and 3mg every four weeks demonstrated overall response rates (ORR) within six months of commencing treatment. The cohorts demonstrated ORR of 74%, 67% and 50%. Responses were achieved by patients who had prior exposure to ruxolitinib, belumosudil and/or ibrutinib.
Adverse events occurred in more than 20% of patients, including an increase in aspartate aminotransferase, blood creatine phosphokinase, lipase, blood lactate dehydrogenase, alanine aminotransferase and fatigue. Of the 241 patients, 101 suffered serious events, with 37 discontinuing study treatment. In the 0.3 mg group, fatigue was the only serious adverse event in 20% of patients. Serious events occurred in 30 patients, with five patients discontinuing treatment.
Based on the results Syndax and Incyte intend to submit a Biologics License Application (BLA) to the FDA by the end of 2023.
Chief executive officer of Syndax, Michael Metzger said: “The AGAVE-201 data demonstrates the potentially pronounced impact this mechanism, alone or in combination with standard of care therapies already available for the management of this disease, may have on patients suffering from chronic GVHD. These results underscore our belief that axatilimab could provide a valuable and highly differentiated therapeutic option for this devastating disease.”
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By GlobalDataChief medical officer of Incyte, Dr Steven Stein said: “The results from the AGAVE-201 study are extremely compelling and underscore the potential benefits axatilimab may offer appropriate patients facing the serious complications associated with chronic GVHD.”
What is axatilimab?
Axatilimab is an investigational monoclonal antibody that targets CSF-1R. Inhibition of signalling through the CSF-1 receptor has been shown during pre-clinical trials to reduce the number of disease-mediating macrophages and monocyte precursors. The reduction of these plays a key role in the fibrotic disease process underlying diseases such as chronic GVHD and IPF. In September 2021, Syndax and Incyte entered a co-development and co-commercialisation licence agreement for axatilimab.
