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

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