GlycoMimetics AML drug fails phase 3 trial

This week, Maryland-based GlycoMimetics announced that its phase 3 clinical trial for the drug uproleselan, aimed at treating relapsed/refractory acute myeloid leukemia (R/R AML), did not achieve its primary objective of extending overall survival. The phase 3 study was a randomized, double-blind, placebo-controlled trial that included 388 patients. It assessed the effectiveness of uproleselan in conjunction with chemotherapy for individuals battling R/R AML, with overall survival without transplant censoring as the primary endpoint.

Despite high hopes, the trial results revealed that the combination of uproleselan and chemotherapy did not show a statistically significant improvement in overall survival when compared to chemotherapy alone. Participants receiving uproleselan had a median overall survival of 13 months, which was only marginally better than the 12.3 months observed in the placebo group. This slight difference indicates that uproleselan did not provide the expected benefit in prolonging the lives of the patients.

Uproleselan is a unique e-selectin antagonist, a novel drug class. In the context of acute myeloid leukemia, e-selectin is known to facilitate the survival of leukemic cells and protect them from treatments. By inhibiting e-selectin, uproleselan aims to interfere with this protective mechanism, potentially making leukemic cells more susceptible to chemotherapy.

Despite the disappointing phase 3 trial results, GlycoMimetics remains committed to exploring the potential of uproleselan in other clinical settings. A separate phase 2/3 study is currently underway, examining the effects of uproleselan in older adults newly diagnosed with AML who are suitable for intensive chemotherapy. This study is being conducted by the National Cancer Institute (NCI) and the Alliance for Clinical Trials in Oncology, and it continues to assess whether uproleselan might offer benefits in this different patient population.

The ongoing investigation in newly diagnosed AML patients underscores GlycoMimetics' dedication to understanding the potential roles and therapeutic benefits of uproleselan. The company believes that further research and data could reveal specific contexts in which uproleselan is more effective, offering new hope for patients with acute myeloid leukemia.

In summary, the phase 3 trial involving uproleselan and chemotherapy for relapsed/refractory acute myeloid leukemia fell short of its goal to significantly extend overall survival. Nevertheless, GlycoMimetics is pressing forward with additional studies to explore the drug's potential in other AML patient populations, reflecting an ongoing effort to find more effective treatment options for this aggressive form of leukemia.