GMI-1271 Granted Two Review Designations by FDA and EU for AML

The E-selectin inhibitor GMI-1271 was granted breakthrough therapy designation by the U.S. Food and Drug Administration (FDA) and orphan drug designation by the European Commission (EC) for the treatment of patients with relapsed/refractory acute myeloid leukemia (AML).

The FDA’s decision was based on results from an ongoing, phase I/II trial of 47 adults with relapsed/refractory AML who were treated with GMI-1271 in combination with MEC chemotherapy (mitoxantrone, etoposide, and cytarabine). The EC’s orphan drug designation was based on these results, as well as a favorable recommendation by the European Medicines Agency Committee for Orphan Medicinal Products.

The ongoing trial is enrolling patients to receive escalating doses of GMI-1271 (5-20 mg/kg) plus MEC. GMI-1271 was administered 24 hours prior to MEC, every 12 hours during, then 48 hours post-induction or consolidation.

The overall response rate (evaluable in 21 patients) was 45 percent.  At a median follow-up of 11 months, the overall survival was 7.6 months.

The most common grade 3/4 adverse events observed were:

  • febrile neutropenia (36%)
  • sepsis (26%)
  • bacteremia (13%)
  • hypoxia (13%)

The FDA previously granted GMI-1271 orphan drug designation for AML in March 2015.

Sources: GlycoMimetics press release, May 17, 2017; GlycoMimetics press release, May 25, 2017; DeAngelo DJ, Jonas BA, Liesveld J. GMI-1271, a novel E-selectin antagonist, in combination with chemotherapy in relapsed/refractory AML. Abstract #2520. Presented at the 2017 ASCO Annual Meeting, June 5, 2017, Chicago, Illinois.