GlycoMimetics plummets after uproleselan fails leukemia trial

GlycoMimetics faced a significant setback as its innovative blood cancer treatment, uproleselan (GMI-1271), failed to meet the primary survival endpoint in a late-stage clinical trial. This news caused the company’s shares to plummet by over 79% before the stock market opened on Monday.

Uproleselan is a pioneering E-selectin inhibitor that aims to target endothelial cells, disrupting the adhesion between bone marrow cells and leukaemic cells. The recent study involved 388 patients suffering from relapsed or refractory acute myeloid leukaemia (AML). Unfortunately, the data showed that the combination of uproleselan and chemotherapy did not yield a statistically significant improvement in overall survival (OS) compared to chemotherapy alone. Specifically, the median OS for patients receiving the uproleselan-chemotherapy combination was 13 months, while the placebo group had a median OS of 12.3 months.

Secondary goals of the study included measuring the incidence of severe oral mucositis, complete remission rates, and overall remission rates. The adverse events observed were consistent with the known side effects associated with the chemotherapy regimens used in the trial, according to the company.

CEO Harout Semerjian commented, “We are thoroughly analysing the data…and are committed to submitting a comprehensive data analysis for presentation at an upcoming medical meeting.” During a March earnings call, Semerjian had suggested that uproleselan could significantly impact the company's future direction.

The trial had originally been scheduled for completion in 2020. However, it faced delays, with final results pushed to the end of this quarter. This delay followed the FDA’s approval of a protocol amendment allowing for a time-based analysis of overall survival. Despite the setback, the company noted that a Phase II/III study of uproleselan in newly diagnosed AML patients, conducted in collaboration with the National Cancer Institute, is still ongoing.

Prior to these results, GlycoMimetics had projected a market opportunity for uproleselan in the US to be between $650 million and $850 million. Analysts from H.C. Wainwright had suggested that, assuming positive clinical outcomes and regulatory approvals, uproleselan could be launched as early as 2025. They projected sales of $45.7 million in the launch year, potentially growing to $215.8 million by 2028.

Despite the disappointing trial results, GlycoMimetics remains committed to a thorough data analysis and is looking forward to presenting its findings at an upcoming medical meeting. The ongoing study with the National Cancer Institute offers a glimmer of hope as the company continues its efforts to bring effective treatments for AML patients to the market.