Fig. 2: Efficacy of OT-82 in different xenograft models of hematopoietic malignancies.

a OT-82 efficacy in SC xenograft model of MLL-rearranged AML. Mice (SCID) were inoculated SC with 2 × 106 MV4–11 cells on day 0. Starting on day 14, mice were treated PO with vehicle (30% HPBCD) or OT-82 (25 or 50 mg/kg) on 6 consecutive days per week for 3 weeks (“1st treatment”, days 14–33; n = 13–15 mice/group). For secondary treatment, mice treated with 25 mg/kg on days 14–33 were divided into two groups (n = 9–13 mice/group) and treated with vehicle or 50 mg/kg OT-82 according to the same schedule on days 35–54. Mean tumor volume ± SE is shown and was compared between groups using Students’ t-test. b OT-82 efficacy in SC xenograft model of erythroleukemia. Mice (SCID) were inoculated SC with 1 × 106 HEL92.1.7 cells on day 0. Starting on day 13, mice were treated PO with vehicle (30% HPBCD) or OT-82 (10, 25, or 50 mg/kg) on 6 consecutive days per week for 3 weeks. Mean tumor volume ± SE is shown (n = 14–20 tumors/group). Differences from the vehicle-treated group were statistically significant (p < 0.05, Students’ t-test) for the 50 mg/kg group from day 20 to 34 (end of the study). c OT-82 efficacy in systemic xenograft models of MLL-rearranged AML (MV4–11), erythroleukemia (HEL92.1.7) and infant MLL-rearranged ALL (MLL-2) [24]. Mice were inoculated IV with 107, 5 × 106 and 2.5 × 106 cells, respectively, and treated with the indicated doses of OT-82 PO 6 days per week during the indicated time periods. Treatment was started on day 5 (MV4–11) or day 4 (HEL92.1.7) post inoculation or when the median percentage of huCD45 + cells in the peripheral blood of the cohort reached >1% (MLL-2).