Topline results expected H2 2025

Exicure, Inc. (Nasdaq: XCUR), a clinical-stage biotechnology company developing therapeutics for hematologic diseases, today announced it has completed the last patient, last visit in its ongoing Phase 2 clinical trial (NCT05561751) evaluating the safety and efficacy of GPC-100 (burixafor) in combination with propranolol and G-CSF in multiple myeloma patients undergoing autologous stem cell transplant (ASCT). 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 (AML) 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.

About GPC-100

GPC-100 (burixafor) 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, GPC-100 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., GPC-100 became part of Exicure’s pipeline following the company’s acquisition in January 2025. In addition to multiple myeloma, GPC-100 is also being considered in acute myeloid leukemia (AML) and 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.

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, 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.

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