Exicure completes last patient in Phase 2 GPC-100 combo trial

Exicure (XCUR) announced it has completed the last patient, last visit in its ongoing Phase 2 clinical trial evaluating the safety and efficacy of GPC-100 in combination with propranolol and G-CSF in multiple myeloma patients undergoing autologous stem cell transplant. Topline results from the study are expected in the fourth quarter of 2025. 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. In data presented at ASH 2024, GPC-100 appears to be well differentiated in terms of the speed with which it mobilizes stem cells compared to currently approved stem cell mobilization treatments. Dr. Muthalagu Ramanathan, Director of the Myeloma Program and Medical Co-Director of the Blood and Marrow Transplant and CAR-T Program at U Mass Memorial Health noted that “traditionally, patients undergoing mobilization are required to come, as an outpatient, the night before their procedure to receive the mobilization injection – a process that adds logistical challenges. The study drug GPC100 needed to be administered just 45 minutes before stem cell collection and resulted in a successful mobilization, significantly improving the patient experience. This is a true blessing for our frail myeloma patients.” In addition to multiple myeloma and a planned Phase 1 study in acute myeloid leukemia patients, the company is also in discussions for potential collaborations that would utilize GPC-100’s rapid and safe mobilization of stem cells in cell and gene therapy settings.