GlycoMimetics has dosed the first patient in its Phase 3 clinical trial of uproleselan (GMI-1271) in relapsed/refractory acute myeloid leukemia (AML).
In the trail, the clinical-stage biotechnology company will evaluate uproleselan in combination with MEC (mitoxantrone, etoposide and ara-C) or in combination with FAI (fludarabine, ara-C and idarubicin).
Both MEC and FAI are standard of care for relapsed/refractory acute myeloid leukemia.
According to GlycoMimetics, uproleselan has been designed to prevent the E-selectin molecule from binding with blood cancer cells. E-selectin is known to play a key role in binding cancer cells within vascular niches in the bone marrow, which blocks the cells from entering circulation where they can be killed more readily by chemotherapy.
Uproleselan was granted Breakthrough Therapy Designation by the US Food and Drug Administration (FDA) in 2017 for its use in the AML patient population.
GlycoMimetics is looking to take in nearly 380 adult patients across the world in the single pivotal randomized, double-blind, placebo-controlled phase 3 trial.
The late-stage trial’s primary endpoint has been defined to be overall survival. The biotechnology firm said that censoring for transplant in the primary efficacy analysis will not be needed.
Among the main secondary endpoints of the trial are incidence of severe mucositis and remission rate, which will be studied in a hierarchical fashion for potential inclusion in the product labeling.
While most of the study sites will be in the US, there will be meaningful participation from clinical centers across certain countries to support regulatory filings in major markets, said GlycoMimetics.
GlycoMimetics CEO Rachel King said: “Our clinical development pipeline sets us up for multiple, value-creating clinical data readouts.
“During 2019, we anticipate topline data from the Phase 3 study of rivipansel being conducted by Pfizer in patients with sickle cell disease. Then, beginning at the end of 2020, we expect to generate topline data from the several trials that we will have underway in AML.”
GlycoMimetics expects to initiate two complementary phase 2/3 trials for uproleselan from two clinical consortia in early 2019.