During Friday’s extended trading session, Exicure, Inc. (NASDAQ: XCUR) saw a significant increase, closing at $7.80 after rising 13.28%. Following the company’s confirmation of the accomplishment of a crucial clinical research milestone, this increase occurred.
Phase 2 Clinical Trial Milestone Completed
The biotechnology business reported that the final patient visit in its current Phase 2 clinical study (NCT05561751) was successfully completed. The safety and effectiveness of GPC-100 (burixafor) in conjunction with propranolol and G-CSF for patients with multiple myeloma undergoing autologous stem cell transplantation (ASCT) are assessed in this research. Exicure anticipates this trial’s topline findings in the fourth quarter of 2025.
GPC-100’s Potential for Stem Cell Mobilization
A very specific small molecule antagonist of the chemokine receptor CXCR4, which is essential for keeping hematopoietic stem cells in the bone marrow niche, is GPC-100 (burixafor). GPC-100 may help mobilize these stem cells into peripheral circulation for collection and further ASCT operations by inhibiting CXCR4. GPCR Therapeutics, Inc. was the original developer of the chemical, which Exicure acquired in January 2025 and added to their pipeline.
Beyond multiple myeloma, GPC-100 is under consideration for acute myeloid leukemia (AML) and other medical conditions where improved stem cell mobilization could enhance treatment outcomes. This includes applications in sickle cell disease, rare disorders requiring autologous transplant, and in cell and gene therapy programs.
Differentiated Clinical Performance and Patient Benefits
The ongoing randomized, open-label, multicenter study is designed to determine whether GPC-100 can improve CD34+ hematopoietic stem cell mobilization from the bone marrow into peripheral blood for ASCT procedures. Data presented at ASH 2024 indicated that GPC-100 offers a faster mobilization rate compared to currently approved agents.
Typically, mobilization requires patients to receive injections the night before stem cell collection, creating logistical burdens. In contrast, GPC-100 can be administered just 45 minutes before the procedure, resulting in efficient mobilization and enhancing patient experience.
Exicure is also advancing plans for a Phase 1 AML study and engaging in discussions for potential collaborations to expand GPC-100’s use in cell and gene therapy applications.