
Opinion|Videos|June 9, 2025
Safety and Efficacy Results from AGAVE-201 (Axatilimab Therapy in cGVHD)
Panelists discuss how axatilimab showed promising response rates in the AGAVE-201 trial, even in patients with graft-vs-host disease (cGVHD) who had failed other approved therapies.
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Episodes in this series

Belumosudil and Axatilimab Data
This segment presents follow-up data from the ROCKstar study of belumosudil, showing:
- 74%-76% overall response rate across both dosing regimens (200 mg once or twice daily)
- Consistent responses across organ systems
- 57% failure-free survival at 12 months
- 25% of patients successfully discontinued all immunosuppression
- Common adverse events included fatigue, gastrointestinal symptoms, cough, and headache
The panel transitions to axatilimab data from the AGAVE-201 trial, which evaluated 3 different dosing regimens. The FDA-approved dose (0.3 mg/kg every 2 weeks) demonstrated:
- 74% overall response rate
- Median time to response of 1.4-1.7 months
- Median failure-free survival of approximately 11 months
- Main adverse events were laboratory abnormalities related to CSF1R blockade
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