GMI-1359 is a rationally-designed small molecule that antagonizes both E-selectin and a chemokine receptor known as CXCR4. Since E-selectin and CXCR4 are implicated in the retention of cancer cells in the bone and bone marrow, we believe that targeting both E-selectin and CXCR4 with a single compound could improve efficacy in the treatment of cancers that affect the bone and bone marrow, particularly solid tumors that have a propensity to metastasize to bone, such as breast and prostate cancer.
We completed a Phase 1 randomized, double-blind, placebo-controlled, single-dose escalation trial of GMI-1359 in healthy volunteers. In this trial, volunteer participants received a single injection of either GMI-1359 or placebo, after which they were evaluated for safety, tolerability and PK. This trial was conducted at a single site in the United States. GMI-1359 was generally well tolerated in this trial, with no participants experiencing serious adverse events. A Phase 1b trial of GMI-1359 in HR+ breast cancer patients whose tumors have spread to bone is currently ongoing (NCT04197999). The trial is being conducted at Duke University and will evaluate safety, pharmacokinetics and pharmacodynamic markers of biologic activity in these patients.
Additionally, the FDA awarded GMI-1359 a rare pediatric disease priority review designation and orphan drug designation for the treatment of osteosarcoma.
Recent scientific publications and presentations with GMI-1359 include the following:
- In a paper published in Science Translational Medicine in May 2016, researchers at Duke University—together with GlycoMimetics scientists—demonstrated the key roles of both E-selectin and CXCR4 in trafficking of breast cancer cells to bone marrow.
- At the ASH annual meeting in December 2016, we presented preclinical data suggesting that GMI-1359 has a unique tumor cell mobilization profile and enhanced the ability of chemotherapy to target and improve survival from a high-risk form of mutated AML.
- In November 2016, at the annual meeting of the Society for Immunotherapy of Cancer, we presented data from a preclinical study in which GMI-1359, in combination with an antibody against the cancer regulatory programmed death receptor ligand (PD-L1) shortened time to complete tumor regressions in an animal model of colon cancer. In this preclinical study, the combination therapy also selectively reduced regulatory T cells, a class of lymphocytes that suppress immune responses, and created a more favorable immune-mediated anti-tumor environment.
- Most recently, at the 2020 ASH meeting, poster presentations conveyed how GMI-1359 enhances sorafenib’s anti-leukemia effect in pre-clinical AML models; and how GMI-1359 can uniquely generate motility-enhancing signals in AML cells and deplete AML cells from protective vascular niches in the bone marrow.
GMI-1359 has not been approved for use by any health authority anywhere in the world.
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