On June 15, 2023 GlycoMimetics, Inc. (Nasdaq: GLYC) a late clinical-stage biotechnology company discovering and developing glycobiology-based therapies for cancers and inflammatory diseases, reported that the U.S. Food and Drug Administration (FDA) cleared the addition of a protocol amendment to the company’s pivotal Phase 3 study of uproleselan for relapsed/refractory (R/R) Acute Myeloid Leukemia (AML) (Press release, GlycoMimetics, JUN 15, 2023, View Source [SID1234632725]). The amendment will allow a time-based analysis of the primary endpoint of overall survival to be conducted following a defined cutoff date if the 295 survival events originally planned for an event driven analysis have not been observed. With the addition of a time-based analysis, topline results are expected to be reported by the end Q2 2024.
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"We are pleased the FDA cleared the addition of a time-based pathway to final analysis as it provides the opportunity to evaluate the effect of uproleselan on R/R AML based on a clinically mature database with more than three years median follow-up," said Harout Semerjian, Chief Executive Officer of GlycoMimetics. "This approach is consistent with regulatory precedent adopted for a prior pivotal AML study for an approved drug and reflects our commitment to science-driven analysis as we seek to deliver uproleselan to R/R AML patients in need of new treatment options as soon as possible."
The Phase 3 trial completed enrollment of 388 patients across 70 sites in nine countries in November 2021. An event based final analysis of overall survival was previously expected after year end 2022, but the number of events has slowed, resulting in the projected timeline being extended.
Based on blinded pooled data observed to date, a time-based final analysis in Q2 2024 is expected to yield a clinically mature dataset to evaluate uproleselan in R/R AML. This dataset is expected to reflect a median follow-up of greater than three years, including at least two years of post-transplant data. The majority of surviving patients in the study received hematopoietic stem cell transplantation (HSCT). As a result, the company believes the capture rate of survival events for this study beyond Q2 2024 would provide limited additional value for the primary analysis.
Two years post-transplant is generally considered an important milestone in AML because most patients who survive at least two years post-transplant without experiencing disease relapse are typically deemed to be long-term survivors. For patients over two years post-transplant, disease relapse is less likely.1
As part of the protocol amendment, the FDA also cleared the addition of landmark EFS and overall survival analyses as secondary endpoints.
About Uproleselan
Discovered and developed by GlycoMimetics, uproleselan is an investigational first-in-class, E-selectin antagonist. Uproleselan (yoo’ pro le’se lan), currently in a comprehensive Phase 3 development program in acute myeloid leukemia (AML), has received Breakthrough Therapy and Fast Track designations from the U.S. FDA and Breakthrough Therapy designation from the Chinese National Medical Products Administration for the treatment of adult AML patients with relapsed or refractory disease. Uproleselan is designed to block E-selectin binding and stimulation of myeloid cells. E-selectin is expressed on the surface of blood vessels, and its binding to myeloid cells confers a pro-survival effect. Uproleselan intends to provide a novel approach to disrupting established mechanisms of leukemic cell resistance.