Exicure announces near-term strategic priorities

Exicure’s (XCUR) ongoing Phase 2 study is a randomized, open-label, multicenter trial evaluating burixafor, a small molecule CXCR4 antagonist, in autologous stem cell transplant for multiple myeloma. By blocking CXCR4, burixafor is designed to mobilize hematopoietic stem cells out of the bone marrow and into the bloodstream, where they can be collected for use in transplant procedures. Interim results to date have been highly encouraging, with 100% of patients achieving the primary endpoint of successful CD34+ stem cell mobilization, including patients previously treated with daratumumab. Burixafor has a faster kinetics of mobilization with a well-tolerated safety profile, enabling same-day administration of the mobilizing agent and leukapheresis. This differentiates burixafor from FDA-approved agents such as plerixafor and motixafortide, which require overnight pre-treatment. In August, the company announced that the final patient had completed their last study visit. The clinical database has since been locked, and Exicure remains on track to report topline data in Q4 2025. Preparations are also underway for a potential Phase 3 trial. A full data publication from the ongoing Phase 2 trial is anticipated in 2026. In addition, a publication from a previous Phase 2 study evaluating burixafor in combination with G-CSF in patients with multiple myeloma, non-Hodgkin lymphoma, and Hodgkin disease, is currently under peer review. Building on progress in multiple myeloma, Exicure is preparing to expand burixafor into additional indications: Sickle Cell Disease: Exicure is in discussions with key clinicians at leading institutions to initiate an investigator-sponsored trial evaluating burixafor for improving stem cell mobilization in patients undergoing gene editing and autologous transplant. Acute Myeloid Leukemia: The company is also planning a Phase 1 chemosensitization study in AML. Preclinical data suggest that burixafor may mobilize tumor cells from protective bone marrow niches, potentially enhancing the efficacy of chemotherapy.