Shots:

• The P-I/II trial evaluates axatilimab in 40 patients with recurrent or refractory cGVHD which showed an ORR (82%) in patients dosed at 1mg/kg, q2w in the P-II cohort by cycle 7 day 1; 67% in all patients, the best ORR (CR+PR) was 69%
• m-DoR (not reached); 33% sustained response @≥20wks., 52% reduction in glucocorticoids doses with multi-organ clinical benefits in a heavily pretreated patient population, 58% disease improvement, was well tolerated with a favorable safety profile
• AEs were consistent with on-target effects of CSF-1R inhibition, 2 DLT with no ≥grade 3 on-target toxicities of CSF-1R blockade & no incidence of cytomegalovirus or other viral reactivation, no apparent increases in risk for inf., SAEs (40%), AEs leads to treatment discontinuation (18%)

Ref: Syndax | Image: Incyte