Company Description
Exicure, Inc. (Nasdaq: XCUR) is a clinical-stage biotechnology company focused on developing therapies for hematologic diseases. According to company disclosures, Exicure has historically been an early-stage biotechnology company working on nucleic acid therapies targeting ribonucleic acid against validated targets. Following a restructuring and suspension of its prior clinical and development activities, the company has shifted its focus and is now advancing a lead small-molecule program in hematology while continuing to explore broader strategic alternatives to maximize stockholder value.
Business focus and lead program
Exicure describes itself as a clinical-stage biotechnology company developing therapies to address key challenges in hematologic diseases. Its lead program is burixafor (GPC-100), a highly selective small molecule antagonist of the chemokine receptor CXCR4. CXCR4 plays a central role in retaining hematopoietic stem and progenitor cells in the bone marrow niche. By blocking CXCR4, burixafor is designed to mobilize these cells into the peripheral blood so they can be collected for use in autologous stem cell transplant procedures and potentially in other treatment settings.
Burixafor (also referred to as GPC-100) became part of Exicure’s pipeline through the acquisition of GPCR Therapeutics USA Inc. and related assets in January 2025. Company materials state that GPCR USA conducts all business activities and operations of Exicure, while Exicure Operating Company is dormant and KC Creation Co., Ltd. is a subsidiary in Korea that plans to engage in business described in the company’s registration documents.
Therapeutic areas and development strategy
Exicure reports that its lead program is being evaluated for its ability to improve stem cell mobilization in several settings:
- Multiple myeloma in patients undergoing autologous hematopoietic cell or stem cell transplant (AHCT/ASCT).
- Sickle cell disease, where improved stem cell mobilization may support gene editing and autologous transplant approaches.
- Cell and gene therapy settings and rare diseases requiring autologous transplant, where efficient mobilization of hematopoietic progenitor cells could support treatment procedures.
In addition, Exicure states that burixafor is being studied, or is planned to be studied, as a potential chemosensitizing agent in acute myeloid leukemia (AML). Preclinical data cited by the company suggest that burixafor may mobilize malignant cells from protective bone marrow niches into the peripheral blood, where they may be more effectively targeted by chemotherapy. The company has discussed plans for a Phase 1 chemosensitization study in AML and references prior chemosensitization work by other parties.
Clinical development of burixafor (GPC-100)
Exicure highlights an open-label, multicenter Phase 2 clinical trial (NCT05561751) evaluating burixafor (GPC-100) in combination with propranolol and granulocyte colony-stimulating factor (G-CSF) in patients with multiple myeloma undergoing autologous hematopoietic cell or stem cell transplantation. The study is designed to assess whether this regimen can improve CD34+ hematopoietic progenitor or stem cell mobilization from the bone marrow into the peripheral blood, enabling collection via leukapheresis for transplant.
According to company news, interim and topline data from this Phase 2 trial have shown that a high proportion of enrolled participants achieved the primary endpoint of collecting a target number of CD34+ cells within a limited number of leukapheresis sessions. The company reports that burixafor has rapid mobilization kinetics, with peak peripheral CD34+ cell levels observed within a short period after administration, enabling same-day dosing and leukapheresis. Exicure also notes that many participants had prior exposure to daratumumab, a therapy associated with reduced mobilization in some studies, yet a large majority of those patients still achieved the primary endpoint in the trial.
Across its communications, Exicure emphasizes the observed safety profile of burixafor in combination with G-CSF and propranolol in the Phase 2 multiple myeloma study, stating that the regimen was well tolerated and that no burixafor-related adverse events higher than Grade 2 were reported in the described dataset. The company also notes that all participants who elected to proceed with transplant in the trial were able to undergo autologous transplant and engraft.
Corporate evolution and strategic alternatives
Regulatory filings describe a significant change in Exicure’s business over time. The company explains that it historically focused on nucleic acid therapies targeting RNA, but in 2022 it announced a substantial reduction in force, suspended preclinical activities, and halted all research and development while beginning to explore strategic alternatives. In later disclosures, Exicure reports entering into a licensing agreement for patents related to one of its historical drug candidates, receiving a small one-time payment and potential modest royalties, and selling certain samples related to that product. The company also reports selling additional historical biotechnology assets under an asset purchase agreement.
Exicure states that it obtained financing in late 2024 to continue operations and to support its exploration of strategic alternatives, including possible private company acquisitions, raising additional capital, strategic partnerships, or combinations of these approaches. The acquisition of a clinical-stage biotechnology company developing therapeutics for hematologic diseases, and the integration of burixafor (GPC-100) into its pipeline, represent a key outcome of this strategic process.
Regulatory and listing status
Exicure’s common stock trades on The Nasdaq Capital Market under the ticker symbol XCUR. Company announcements describe periods of non-compliance with Nasdaq’s periodic filing requirements, followed by subsequent filings and notices indicating that Exicure regained compliance with Nasdaq Listing Rule 5250(c)(1) based on the timely submission of required quarterly reports. The company has also filed a registration statement on Form S-1 relating to the resale of shares by selling stockholders, reflecting its status as a smaller reporting company.
Financial condition and going concern disclosures
In multiple quarterly financial releases, Exicure reports that its cash and cash equivalents are not sufficient to continue funding operations over an extended period. The company states that it has implemented significant cost reductions and that its ability to further reduce expenses is limited. As a result, Exicure discloses that it needs substantial additional financing in the short term to pay expenses, fund the ongoing exploration of strategic alternatives, and support any alternatives that may be identified, as well as to fund its operations. The company cautions that there can be no assurance that such financing will be available or, if available, that it can be obtained on acceptable terms.
Corporate governance and stockholder matters
Exicure’s proxy materials and current reports describe typical public-company governance activities, including annual meetings of stockholders, election of directors, ratification of the independent registered public accounting firm, and advisory votes on executive compensation. The company has reported changes in board composition, including the appointment of new directors and the resignation of others, as well as the use of virtual-only formats for its annual meetings. These documents also outline stockholder voting procedures and confirm Exicure’s status as a Delaware corporation.
Summary of Exicure’s positioning
Based on its own public statements, Exicure, Inc. is positioned as a smaller reporting, clinical-stage biotechnology company in the hematology space, centered on the development of burixafor (GPC-100) as a CXCR4 antagonist for stem cell mobilization and as a potential chemosensitizing agent in AML. The company’s disclosures emphasize its transition from historical nucleic acid therapy programs to a hematologic disease–focused pipeline, its ongoing strategic review, and the need for additional capital to support continued operations and clinical development.