- Exicure, Inc. (XCUR, Financial) completes patient enrollment for Phase 2 trial of GPC-100 in multiple myeloma.
- Topline results from the study expected in the second half of 2025.
- Study explores GPC-100's potential in stem cell mobilization and other disease applications.
Exicure, Inc. (XCUR), a biotechnology company focused on developing therapies for hematologic diseases, announced the completion of patient enrollment in its Phase 2 clinical trial for GPC-100 (burixafor). This study is investigating the safety and efficacy of GPC-100 in combination with propranolol and G-CSF in patients with multiple myeloma undergoing autologous stem cell transplant (ASCT).
The randomized, open-label, multicenter trial aims to evaluate whether GPC-100, a small molecule CXCR4 antagonist, can enhance CD34+ hematopoietic stem cell mobilization from the bone marrow to the peripheral blood. These cells are then collected via leukapheresis for ASCT use. Results from this study are anticipated in the fall of 2025.
Dr. Jack Khouri, MD, FACP, hematologist at the Cleveland Clinic and principal investigator of the trial, highlighted the importance of efficient stem cell mobilization for successful hematopoietic cell transplantation. Issues with mobilization can delay transplants, necessitate multiple apheresis rounds, and increase patient and healthcare system burdens. Dr. Khouri noted the promising safety profile and effectiveness of GPC-100 when used with G-CSF and propranolol.
Beyond multiple myeloma, Exicure is also planning a Phase 1 study to assess GPC-100 as a chemosensitizing agent for patients with relapsed or refractory acute myeloid leukemia (AML). Further applications of GPC-100 are being explored in diseases where improved stem cell mobilization could advance treatment, such as sickle cell disease and conditions requiring autologous transplant, as well as in cell and gene therapy settings.
