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Exicure, Inc. (Nasdaq: XCUR), a clinical-stage biotechnology company developing therapeutics for hematologic diseases, today announced that results from its completed Phase 2 study evaluating burixafor, a small molecule CXCR4 antagonist, will be featured in an oral presentation at the 67th American Society of Hematology (ASH) Annual Meeting and Exposition, taking place December 6-9, 2025, in Orlando, Florida.
The presentation will highlight additional patient data from the open-label, multicenter Phase 2 trial (NCT05561751) assessing burixafor in patients with multiple myeloma undergoing autologous hematopoietic cell transplantation (AHCT). By blocking CXCR4, burixafor is designed to mobilize hematopoietic progenitor cells from the bone marrow into the peripheral blood, where they can be collected for use in transplantation procedures.
"The combination of burixafor, G-CSF and propranolol demonstrated an excellent safety profile and was able to ensure adequate and efficient mobilization of hematopoietic progenitor cells, with all patients who chose to undergo transplant (18/19) proceeding to AHCT. These are very encouraging results, especially since the majority of the enrolled patients were treated with the anti-CD38 monoclonal antibody daratumumab, which has been associated with lower HPC mobilization in certain studies. The same day administration of burixafor with leukapheresis is also more convenient for patients and may reduce time toxicity," said Jack Khouri, M.D., Associate Professor of Medicine, Cleveland Clinic Lerner College of Medicine, Case Western Reserve University, study lead investigator, and presenter.
Oral Presentation Details
Abstract Number: 1050
Title: An open-label, multi-center Phase 2 study to assess the safety and efficacy of burixafor (GPC-100) and propranolol with G-CSF for the mobilization of hematopoietic progenitor cells in patients with multiple myeloma
Presenter: Dr. Jack Khouri, Associate Professor of Medicine, Cleveland Clinic Lerner College of Medicine, Case Western Reserve University
Session: 711. Cell Collection and Manufacturing of HSPCs, CAR-T Cells, and Other Cellular Therapy Products: Refining CAR-T Cells and Engineered HSPCs; New Approaches to HSPC mobilization
Date and Time: December 8, 2025, 5:45-6:00pm EST
Location: Hyatt - Regency Ballroom R
About Burixafor (GPC-100)
Burixafor (GPC-100) 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, burixafor 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., burixafor became part of Exicure's pipeline following the company's acquisition in January 2025. In addition to multiple myeloma, burixafor is also being considered 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. A chemosensitization trial in AML is also being planned, leveraging burixafor's mechanism of mobilizing malignant cells from protective bone marrow niches into the peripheral blood, where they may be more effectively targeted by chemotherapy.
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, burixafor (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 .
Contact:
Exicure, Inc.
847.673.1700 (Tel)
847.556.6411 (Fax)
