GlycoMimetics (GLYC) Study Result summary

Study design and patient population

• Phase 3 trial enrolled 388 relapsed/refractory AML patients aged 18–75 across 59 sites in 9 countries, randomized 1:1 to uproleselan or placebo with MEC or FAI chemotherapy backbones from 2018–2024.

• Eligible patients had primary refractory or first/second relapse AML, with ≤1 prior HSCT.

• Stratification included age, disease status (primary refractory, early relapse, late relapse), and chemotherapy backbone.

• Enrollment completed in November 2021, with a low discontinuation rate of 3%.

• Data cutoff was at the end of Q1 2024, with topline results in Q2 2024.

Primary and secondary efficacy outcomes

• The primary endpoint of overall survival in the intent-to-treat population was not met: median OS was 13.0 months (uproleselan) vs. 12.3 months (placebo), HR 0.89, p=0.3869.

• Complete remission (CR) rates were 36.1% (uproleselan) vs. 33.5% (placebo); CR/CRh rates were 46.4% vs. 41.2%.

• MRD-negative CR at end of induction: 67.1% (uproleselan) vs. 61.5% (placebo).

• Post-treatment stem cell transplant rates were similar between arms (52.1% uproleselan, 51.0% placebo).

• Additional endpoints, including oral mucositis and response rates, trended in favor of uproleselan but did not reach statistical significance.

Subgroup analyses and key findings

• In primary refractory AML, median OS was 31.2 months (uproleselan) vs. 10.1 months (placebo), HR 0.58.

• Uproleselan plus FAI backbone showed median OS of 30.2 months vs. 12.8 months for FAI alone, HR 0.73.

• MRD-negative patients on uproleselan did not reach median OS, compared to 24.1 months for placebo, HR 0.49.

• Transplanted patients on uproleselan had median OS not reached vs. over 2 years for placebo, HR 0.59.

• Median duration of remission in primary refractory patients was not reached for uproleselan vs. 12.7 months for placebo, HR 0.28.