GMI-1359 is an investigational therapy being developed by GlycoMimetics as a potential treatment for cancers that involve the bone marrow — including acute myeloid leukemia (AML) and myeloma — rare pediatric cancers such as osteosarcoma, and other solid tumors that spread to bones, such as prostate cancer and breast cancer.

The U.S. Patent and Trademark Office has issued a patent for GMI-1359, which will protect the composition of this innovative treatment as well as its pharmaceutical formulations through to 2035.

How does GMI-1359 work?

GMI-1359 is designed to target and block E-selectin and CXCR4 simultaneously. Both E-selectin and CXCR4 are adhesion molecules — molecules that promote interaction between cells — that play important roles in metastasis, or the spread of cancer cells from one site to another.

In the case of breast cancer, the disease can return even after complete treatment. That indicates that there may be cancer cells that hide and remain dormant in the body. A preclinical study by researchers in the U.S. showed that these dormant cells hide within protective sites, including the bone marrow, aided by the simultaneous action of E-selectin and CXCR4.

E-selectin allows the migration and entry of breast cancer cells into the bone marrow. Meanwhile CXCR4 plays a key role in anchoring those cancer cells within that marrow.

Thus, the simultaneous inhibition of E-selectin and CXCR4 by GMI-1359 can potentially prevent breast cancer metastasis as well as remove already disseminated breast cancer cells.

In addition, preclinical studies have shown that GMI-1359 could enhance efficacy in the treatment of prostate cancer, breast cancer, AML, myeloma, and osteosarcoma.

GMI-1359 in clinical trials

The safety and pharmacokinetics (movement in the body) of GMI-1359 has been evaluated in a Phase 1, randomized, double-blind, placebo-controlled, single ascending dose trial (NCT02931214). That study, in Nebraska, enrolled some 60 healthy volunteers, ages 19-60. GlycoMimetics reported that GMI-1359 was safe to administer and its pharmacokinetic parameters were well suited to administration in an acute care setting.

An open-label, dose-escalating Phase 1 clinical trial (NCT04197999) now is recruiting participants with advanced breast cancer in North Carolina. This trial will test the safety, tolerability, pharmacokinetics, and pharmacodynamics (effect on the body) of GMI-1359. Up to 12 patients with metastatic hormone receptor-positive breast cancer will be recruited. The results from the study, which are expected in late 2020, will be used to guide the future development of GMI-1359.


Last updated: Feb. 11, 2020


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