GMI-1271, a Potent E-Selectin Antagonist, in Combination with Chemotherapy in Relapsed/Refractory AML: A Novel, Well-Tolerated Regimen with a High Remission Rate

摘要: MethodsA Phase 1/2 open-label trial evaluated GMI-1271 together with cytotoxic induction and consolidation chemotherapy in adult patients with R/R AML. Objectives included assessment of safety, tolerability, PK, biomarkers and anti-leukemic activity.Phase 1 dose escalated single cycle GMI-1271 across 3 pharmacologically active dose levels (5-20 mg/kg) combined with MEC (mitoxantrone, etoposide, cytarabine). Once the recommended phase 2 dose (RP2D, 10 mg/kg) was selected, Phase 2 dose expansion continued enrolling patients with R/R AML and added the option for responders to proceed to 1 cycle of consolidation with GMI-1271 combined with a reduced course of MEC.