Principal Investigator: Daniel J. DeAngelo, MD, PhD


Uproleselan (GMI-1271), a specific E-selectin (E-sel) antagonist, will be evaluated in combination with chemotherapy in a phase 3 trial (NCT03616470) of approximately 380 patients with relapsed or refractory (R/R) acute myeloid leukemia (AML). Adults aged between 18 years and 75 years with R/R disease will be permitted to enroll if they have not received more than 1 prior stem cell transplant or the chemotherapy regimen selected for induction use on this trial.1

Continue Reading

Uproleselan will be administered in combination with either mitoxantrone, etoposide, and cytarabine (MEC) or fludarabine, cytarabine, and idarubicin (FAI) among patients randomly assigned to the experimental arm. Individuals randomly assigned to receive the control therapy will only receive MEC or FAI.

The primary end point is overall survival; secondary end points include the rate of severe oral mucositis and overall response rate. Other outcomes measures include event-free survival, duration of remission, adverse events, and pharmacokinetic exposure.

A novel E-sel antagonist, uproleselan has been shown to disrupt cell survival pathway activation, enhance chemotherapy response, and protect against toxicities such as mucositis with improved survival in vivo. Uproleselan’s anti-E-selectin mechanism is attractive in the AML setting because binding of E-sel to the E-sel ligand on the leukemic cell surface (sialyl Lex) can not only activate malignant cell survival pathways but also promote chemotherapy resistance.2


  1. Study to Determine the Efficacy of Uproleselan (GMI-1271) in Combination With Chemotherapy to Treat Relapsed/Refractory Acute Myeloid Leukemia. NCT03616470. Accessed February 19, 2021.
  2. DeAngelo DJ, Erba HP, Jonas BA, et al. A phase III trial to evaluate the efficacy of uproleselan (GMI-1271) with chemotherapy in patients with relapsed/refractory acute myeloid leukemia. J Clin Oncol. 2019:37(Suppl 15). doi:10.1200/JCO.2019.37.15_suppl.TPS7066