GlycoMimetics has dosed the first patient in a Phase III clinical trial of uproleselan (GMI-1271) to treat relapsed / refractory acute myeloid leukemia (AML).
Uproleselan will be evaluated in combination with mitoxantrone, etoposide and ara-C (MEC) or fludarabine, ara-C and idarubicin (FAI) as part of the single pivotal, randomised, double-blind trial.
Both MEC and FAI are standard of care used for relapsed / refractory AML in the US.
The placebo-controlled trial is expected to enroll around 380 adult patients worldwide, with most trial sites located in the US.
The primary objective of the trial is overall survival, while key secondary objectives include incidence of severe mucositis and remission rate evaluated in a hierarchical design for potential inclusion in the product labeling.
GlycoMimetics clinical development senior vice-president and chief medical officer Helen Thackray said: “This is a rigorously designed Phase III trial that has the potential to bring us one step closer to meeting the significant unmet needs of individuals living with relapsed / refractory AML.
“The trial is an important component of our comprehensive late-stage development programme for uproleselan that positions us to evaluate the use of our product candidate across the spectrum of AML.
“It is the first of three randomised, controlled trials for uproleselan in AML, which we believe should provide clear efficacy and safety outcome measures in each of the settings being assessed.”
The trial will not require censoring for transplant in the primary efficacy analysis.
Uproleselan is developed to block E-selectin, an adhesion molecule on cells in the bone marrow, from binding with blood cancer cells.