Safety and Efficacy Results from AGAVE-201 (Axatilimab Therapy in cGVHD)

Opinion
Video

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.

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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