Latest news with #G-CSF
Business Wire
01-08-2025
- Business
- Business Wire
Exicure Completes the Last Patient, Last Visit in Phase 2 Study of GPC-100 for Stem Cell Mobilization in Multiple Myeloma Patients Undergoing Autologous Transplant
REDWOOD CITY, Calif.--(BUSINESS WIRE)--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
Yahoo
14-04-2025
- Business
- Yahoo
Exicure, Inc. (Nasdaq: XCUR) Nears Completion of Phase 2 Study of GPC-100 in Multiple Myeloma
REDWOOD CITY, Calif., April 14, 2025--(BUSINESS WIRE)--Exicure, Inc. (Nasdaq: XCUR) today announced that GPCR Therapeutics USA, a subsidiary of Exicure Inc., has dosed the 19th patient in its ongoing Phase 2 clinical trial evaluating GPC-100 (burixafor), a small molecule CXCR4 inhibitor, for the mobilization of stem cells in multiple myeloma (MM) patients undergoing autologous stem cell transplant (ASCT) (NCT05561751). The study is an open-label, multi-center trial evaluating the safety and efficacy of GPC-100 and propranolol in combination with G-CSF. Data from an interim analysis of 10 patients were previously presented at the 2024 American Society of Hematology (ASH) Annual Meeting. Preliminary results are encouraging, with 100% of patients achieving the primary endpoint of successful CD34+ stem cell mobilization, including patients previously treated with daratumumab. Notably, GPC-100 enabled same-day administration of the mobilizing agent and leukapheresis, offering faster kinetics compared to FDA-approved agents such as plerixafor and motixafortide, which require overnight pre-treatment. Median times to neutrophil and platelet engraftment were 11 and 14 days, respectively, which is consistent with standard of care. This trial is expected to finish patient recruitment at the end of April. About Exicure, Inc. Exicure, Inc. has historically been an early-stage biotechnology company focused on developing nucleic acid therapies targeting ribonucleic acid against validated targets. Following its restructuring and suspension of clinical and development activities, the Company is exploring strategic alternatives to maximize stockholder value. For further information, see View source version on Contacts Media Contact: Sarah Ellinwood, PhDKendall Investor Relationssellinwood@ Sign in to access your portfolio
