Fig. 3: PMD-026 alleviates disease burden in FLT3-ITD in vivo models.

A Efficacy in the MV4-11 xenograft model. Percentage of hCD45+ cells in the PB of mice transplanted with MV4-11 cells. Mice were treated with vehicle (n = 5) or PMD-026 (n = 5) for four weeks. %PB hCD45+ statistics were assessed by two-way ANOVA. B Percentage of hCD45+ cells in the BM of xenografted animals at endpoint. Vehicle (n = 5); PMD-026 (n = 3). Statistics were assessed by two-tailed Student’s t-test. C Kaplan–Meier survival analysis of xenografted animals assessed by log-rank test. D–I Efficacy in the syngeneic Vav-Cre Flt3ITDTet2KO model. Chimerism in the peripheral blood (D) and bone marrow (E) following vehicle (n = 6 at Start, n = 4 at Endpoint) or 100 mg/kg PMD-026 (n = 6) treatment at endpoint. F White blood cells in vehicle (n = 4) and PMD-026 (n = 6) treated mice at endpoint. Gray bars denote normal reference ranges. Spleen (G) and liver (H) weights (n = 6 per group) were normalized to animal body weight. Statistics were assessed by two-tailed Student’s t-test. (I) Kaplan-Meier survival analysis assessed by log-rank test (n = 6 per group, gray bars represent treatment).