Exicure Completes Patient Enrollment in Phase 2 Study of GPC-100 for Stem Cell Mobilization in Multiple Myeloma Patients Undergoing Autologous Transplant
Topline results expected H2 2025
Exicure, Inc. (NASDAQ:XCUR), a clinical-stage biotechnology company developing therapeutics for hematologic diseases, today announced it has completed patient enrollment in its ongoing Phase 2 clinical trial (NCT05561751) evaluating the safety and efficacy of GPC-100 (burixafor) in combination with propranolol and G-CSF in multiple myeloma patients undergoing autologous stem cell transplant (ASCT).
The randomized, open-label, multicenter study is designed to assess whether GPC-100, a small molecule CXCR4 antagonist, can improve CD34+ hematopoietic stem cell mobilization from the bone marrow into the peripheral blood, where they can be collected via leukapheresis for use in ASCT. Topline results from the study are expected in Fall 2025.
"Efficient hematopoietic progenitor cell mobilization is critical for successful hematopoietic cell transplantation. Inadequate mobilization can lead to delayed transplant, multiple rounds of apheresis, and increased costs—all of which increase the burden on patients and healthcare systems," said Jack Khouri, MD, FACP, hematologist at Cleveland Clinic and primary investigator on the trial. "GPC-100 could be transformative for patients and providers alike. We have seen effective and efficient mobilization with GPC-100 in combination with G-CSF and propranolol in our multiple myeloma patients, with an excellent safety profile. We look forward to the results of this study as we continue to evaluate GPC-100 across additional indications."
In addition to multiple myeloma, the Company is planning a Phase 1 chemosensitization study in patients with relapsed or refractory acute myeloid leukemia (AML). A previous chemosensitization trial was conducted by Taigen in China, and discussions with Key Opinion Leaders (KOLs) are underway to determine the best combination strategy to consider. The company is also exploring GPC-100's potential applications in other diseases where improved stem cell mobilization could help enable more efficient and effective treatment approaches, such as sickle cell disease, rare diseases requiring autologous transplant, and cell and gene therapy settings.
About GPC-100
GPC-100 (burixafor) is a highly selective small molecule antagonist of CXCR4, a chemokine receptor that plays a central role in retaining hematopoietic stem cells in the bone marrow niche. By blocking CXCR4, GPC-100 may enhance the mobilization of these cells into the peripheral blood for collection and use in autologous stem cell transplant (ASCT) procedures. Originally developed by GPCR Therapeutics, Inc., GPC-100 became part of Exicure's pipeline following the company's acquisition in January 2025. In addition to multiple myeloma, GPC-100 is also being considered in acute myeloid leukemia (AML) and other diseases where improved stem cell mobilization could help enable more efficient and effective treatment approaches, such as sickle cell disease, rare diseases requiring autologous transplant, and cell and gene therapy settings.
About Exicure
Exicure, Inc. (NASDAQ:XCUR) is a clinical-stage biotechnology company developing therapies to address key challenges in hematologic diseases. The company's lead program, GPC-100, is being evaluated for its ability to improve stem cell mobilization in multiple myeloma, sickle cell disease, and in support of cell and gene therapy. It is also being studied as a potential chemosensitizing agent in acute myeloid leukemia (AML). For more information, visit www.exicuretx.com.
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