Exicure Presents Positive Topline Phase 2 Data for Burixafor in Multiple Myeloma at 2025 ASH Annual Meeting

Core Insights - Exicure, Inc. announced positive results from its Phase 2 trial of burixafor (GPC-100) in combination with propranolol and G-CSF for mobilizing hematopoietic progenitor cells in multiple myeloma patients undergoing autologous hematopoietic cell transplantation, with approximately 90% of participants achieving the primary endpoint [1][3][6] Company Overview - Exicure, Inc. is a clinical-stage biotechnology company focused on developing therapies for hematologic diseases, with its lead program being burixafor (GPC-100) [10] - Burixafor is a small molecule antagonist of CXCR4, designed to enhance the mobilization of hematopoietic stem cells for autologous stem cell transplant procedures [8] Trial Details - The Phase 2 trial was an open-label, multicenter study involving 19 participants, where 17 achieved the primary endpoint of collecting ≥2 × 10⁶ CD34+ cells/kg [3] - The median time to neutrophil engraftment was 13 days, and the median time to platelet engraftment was 17.5 days, indicating rapid mobilization kinetics [3] Efficacy and Safety - The combination treatment was well tolerated, with no burixafor-related adverse events exceeding Grade 2, demonstrating an excellent safety profile [5][6] - Notably, 84.2% of participants had prior exposure to daratumumab, yet 87.5% of these still achieved the primary endpoint, suggesting the treatment's effectiveness even in challenging cases [4][6] Future Applications - Burixafor is being considered for other diseases requiring improved stem cell mobilization, including sickle cell disease and acute myeloid leukemia, indicating its potential for broader therapeutic applications [9][10]