On 17th March 2017, the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation to GMI-1271, a novel E-selectin (E-sel) antagonist , for the treatment of patients with Relapsed or Refractory (R/R) Acute Myeloid Leukemia (AML).1
E-sel is a cell adhesion molecule expressed constitutively in the bone marrow endothelium and it is involved in cell signaling and chemotaxis. Binding of AML blasts to E-selectin (E-sel), can lead to activation of leukemic cell survival pathways, thereby contributing to chemotherapy resistance. GMI-1271 is a novel antagonist of E-sel, which acts by disrupting leukemia cell survival pathways thus enhancing chemotherapy response.2,3
Currently, GMI-1271 is being explored in the phase 2 portion of the phase 1/2 study (NCT02306291), which is aiming to assess the safety, efficacy and pharmacokinetics of this agent in combination with standard chemotherapy in patients with R/R AML and also in AML patients 60 years of age and older with newly diagnosed disease.