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REDWOOD CITY, Calif., Feb. 05, 2026 (GLOBE NEWSWIRE) -- Exicure, Inc. (Nasdaq: XCUR), a clinical-stage biotechnology company developing therapeutics for hematologic diseases, today announced the publication of results from a prior Phase 2 clinical study evaluating burixafor (GPC-100/TG-0054), a highly selective CXCR4 inhibitor, in the journalAnnals of Hematology.
The manuscript, titled “Burixafor, a CXCR4 Inhibitor with a Differentiated Kinetics Profile: Results of a Phase 2 Study for Rapid Cell Mobilization in Multiple Myeloma and Lymphoma Patients Undergoing Transplant,” reports results from a 12-participant, multi-center, open-label Phase 2 study (NCT02104427) evaluating burixafor in combination with granulocyte colony-stimulating factor (G-CSF) in patients with multiple myeloma (MM), non-Hodgkin’s lymphoma (NHL), and Hodgkin disease (HD) undergoing autologous stem cell transplantation (ASCT).
In the study, 11 of 12 participants (92%) met the primary endpoint of collecting ≥5.0 × 10⁶ CD34+ cells/kg within two leukapheresis sessions, with six participants achieving the target in a single session. Median time to neutrophil and platelet engraftment was 12 and 22 days, respectively. Burixafor was generally well tolerated, with only two treatment-related adverse events reported, both low grade.
Notably, peak mobilization of CD34+ cells occurred within one hour of burixafor administration, substantially faster than currently approved CXCR4 inhibitors, which typically peak 10-14 hours after dosing. This rapid mobilization profile enables same-day leukapheresis, offering the potential to simplify treatment logistics, reduce hospital resource utilization, and minimize patient burden.
In addition to robust stem cell mobilization, burixafor in combination with G-CSF led to marked increases in circulating white blood cell subsets, including lymphocytes. In participants with MM, lymphocyte counts increased by up to 11-fold within hours of burixafor administration, supporting potential applicability in gene therapy, CAR-T, and other gene-editing workflows that require efficient peripheral blood cell collection.
“ASCT remains a treatment cornerstone for multiple myeloma and certain lymphomas. Efficient mobilization of peripheral blood progenitor cells is critical to achieving reliable engraftment while minimizing patient burden and overall healthcare costs,” said Michael Schuster, MD, Clinical Professor of Medicine at Stony Brook University and a study author. “In this study, burixafor demonstrated enhanced mobilization kinetics that led to significantly faster attainment of stem cell collection goals, highlighting its differentiated profile and potential to meaningfully improve the efficiency of cell collection for ASCT. Beyond transplant, rapid and predictable mobilization of CD34-positive cells and lymphocytes may also be highly relevant for emerging gene-based and gene-editing approaches, including those being explored for conditions such as sickle cell disease.”
Exicure continues to advance burixafor’s clinical development and has recently completed an additional Phase 2 study evaluating burixafor in combination with G-CSF and propranolol in patients with multiple myeloma undergoing autologous hematopoietic cell transplantation (NCT05561751). The addition of propranolol was informed bypreviously published preclinical datademonstrating functional crosstalk between CXCR4 and beta-adrenergic receptors, suggesting the potential for enhanced mobilization through dual pathway blockade. Positive topline data from this recent study were presented at the American Society of Hematology (ASH) Annual Meeting in December 2025 and the Tandem Meetings in February 2026, further supporting burixafor’s differentiated and rapid mobilization profile in the transplant setting.
About ExicureExicure, 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 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 stem cell mobilization into the peripheral blood to support collection and use in autologous stem cell transplantation (ASCT).
Burixafor is being evaluated for its potential to improve stem cell mobilization in multiple myeloma, sickle cell disease, and in support of cell and gene therapy. In addition, Exicure is planning a chemosensitization trial in acute myeloid leukemia (AML), leveraging burixafor’s ability to mobilize malignant cells from protective bone marrow niches into the peripheral blood, where they may be more effectively targeted by chemotherapy. Burixafor became part of Exicure’s pipeline following the company’s acquisition of GPCR Therapeutics, Inc. in January 2025. For more information, visitwww.exicuretx.com.