GlycoMimetics Reports Phase 3 Uproleselan Results in R/R AML

ROCKVILLE, Md.-- GlycoMimetics, Inc. (Nasdaq: GLYC), a biotechnology company in the late stages of clinical development, has revealed the topline results from its global Phase 3 study of uproleselan in 388 patients with relapsed/refractory acute myeloid leukemia (R/R AML). Unfortunately, the study did not meet its primary endpoint as the combination of uproleselan and chemotherapy failed to show a statistically significant improvement in overall survival compared to chemotherapy alone.

Patients who received uproleselan had a median overall survival of 13 months, while those in the placebo group had a median overall survival of 12.3 months. Adverse events recorded were consistent with the known side effects of the chemotherapy drugs used in the trial.

GlycoMimetics' CEO, Harout Semerjian, expressed his gratitude to the investigators, patients, and their families for their participation in the study despite the less-than-expected outcome. He emphasized that the company is conducting a thorough data analysis with medical, statistical, and regulatory experts. The findings will be submitted for presentation at a future medical meeting.

The Phase 3 trial was a randomized, double-blind, placebo-controlled study assessing the efficacy of uproleselan in combination with either MEC (mitoxantrone, etoposide, and cytarabine) or FAI (fludarabine, cytarabine, and idarubicin) in R/R AML patients. The trial involved 388 patients across 70 sites in nine countries, randomized equally between the treatment and placebo groups. The primary endpoint was overall survival, with secondary endpoints including the incidence of severe oral mucositis, complete remission rate, and overall remission rate.

In addition to this study, GlycoMimetics is involved in an ongoing National Cancer Institute (NCI) Phase 2/3 study investigating uproleselan in newly diagnosed AML patients aged 60 years or older who are fit for intensive chemotherapy. This study evaluates the addition of uproleselan to the standard cytarabine/daunorubicin regimen versus chemotherapy alone. The Phase 2 portion of this trial completed enrollment of 267 patients in December 2021, and the interim analysis results for event-free survival will be reported when available.

GlycoMimetics also shared preliminary financial results for the first quarter of 2024. As of March 31, 2024, the company had $31.3 million in cash and cash equivalents, compared to $41.8 million as of December 31, 2023. Research and development expenses for the quarter increased to $6.0 million from $5.4 million in the same period in 2023, primarily due to costs associated with raw material acquisition for future manufacturing batches. General and administrative expenses decreased to $5.1 million from $5.5 million, attributed to lower personnel-related and external consulting costs. The number of outstanding common stock shares as of March 31, 2024, was 64,450,835.

Acute Myeloid Leukemia (AML) represents the most common acute leukemia among adults, with around 21,000 new cases diagnosed annually in the United States. Despite existing treatments, the prognosis remains poor, necessitating new therapeutic options. The five-year survival rate for newly diagnosed AML is 31.7%, while it drops to only 10% for relapsed or refractory cases.

Uproleselan, developed by GlycoMimetics, is a first-in-class E-selectin antagonist, designed to disrupt E-selectin binding which is believed to support the survival of leukemic cells in the bone marrow microenvironment. The drug has received Breakthrough Therapy and Fast Track designations from the U.S. FDA and a Breakthrough Therapy designation from China’s National Medical Products Administration for treating adult patients with relapsed or refractory AML.

GlycoMimetics, based in Rockville, Maryland, specializes in developing glycobiology-based therapies for cancers and inflammatory diseases, aiming to create transformative treatments for diseases with high unmet needs.

How to obtain the latest research advancements in the field of biopharmaceuticals?

In the Synapse database, you can keep abreast of the latest research and development advances in drugs, targets, indications, organizations, etc., anywhere and anytime, on a daily or weekly basis. Click on the image below to embark on a brand new journey of drug discovery!