US-based biotechnology company GlycoMimetics has reported positive results from the Phase I portion of a Phase I/II clinical trial of its new E-selectin antagonist, GMI-1271, in conjunction with induction chemotherapy, to treat relapsed / refractory acute myeloid leukemia (AML).
GMI-1271 acts as an E-selectin (an adhesion molecule on cells in the bone marrow) inhibitor, which blocks it from binding with AML cells to resist the well-established mechanisms of leukemic cell resistance within the bone marrow microenvironment.
Conducted at medical institutions in the US, Ireland and Australia, the 44 day multinational open-trial is designed to evaluate the safety, pharmacokinetic (PK) data and efficacy of GMI-1271, in combination with induction chemotherapy to treat high-risk AML.
The trial’s primary objective is to assess safety, while additional endpoints include overall response rate, biomarkers of activity, durability of response and overall survival.
The study exhibited an overall response rate combined complete remission (CR) and remission with incomplete recovery (CRi) of 47% among 19 patients, including those who were older than 60 years, with primary refractory or relapsed disease, poor cytogenetic risk factors including FLT-3 ITDs, and / or extramedullary disease.
Out of 19 patients, eight achieved the best clinical response of CR with one patient displaying CRi, and one patient achieved morphologic leukemia-free state (MLFS).
Five out of the 19 patients were subjected to hematopoietic stem cell transplant.
The study had reported no mortality and no dose-limiting toxic reactions in the patients.
Harvard Medical School associate professor of medicine Dana-Farber Cancer Institute department of medical oncology and adult leukemia clinical and translational research director Daniel DeAngelo said: "The data from the Phase I portion of this Phase I/II trial are very encouraging, demonstrating a high remission rate, and the combination of GMI-1271 and chemotherapy seems to be extremely well tolerated.
"We believe GMI-1271, when combined with chemotherapy, has the potential to address the unmet therapeutic needs of AML patients, and we look forward to gathering data from study of additional patients."
The company will initiate the Phase II of the trial, where approximately 25 patients will be eligible to be subjected to more than one cycle of treatment.
Image: Bone marrow aspirate reveals acute myeloid leukemia. Photo: courtesy of VashiDonsk.