Exicure Announces Publication in Annals of Hematology Highlighting Rapid Stem Cell Mobilization with Burixafor in Patients with Multiple Myeloma and Lymphoma Undergoing Transplant
MWN-AI** Summary
Exicure, Inc. (Nasdaq: XCUR), a clinical-stage biotechnology company, announced significant findings published in the **Annals of Hematology** regarding its investigational drug, burixafor, a selective CXCR4 inhibitor. This study focused on the rapid mobilization of stem cells in patients with multiple myeloma (MM) and lymphoma undergoing autologous stem cell transplantation (ASCT). In a Phase 2 clinical trial involving 12 participants, burixafor demonstrated substantial efficacy alongside granulocyte colony-stimulating factor (G-CSF), achieving the primary endpoint of collecting at least 5.0 × 10^6 CD34+ cells/kg in 92% of participants, with half achieving this milestone in a single leukapheresis session.
The research highlighted burixafor’s ability to peak CD34+ cell mobilization within one hour of administration, significantly faster than existing therapies that typically see peak effects 10 to 14 hours later. This rapid response suggests that burixafor could streamline the stem cell collection process, improve patient experience, and reduce healthcare costs. Furthermore, the treatment was well tolerated, with minimal and low-grade adverse events reported.
Importantly, the study observed a remarkable increase in circulating lymphocyte subsets, indicating that burixafor may also have applications in gene therapy and CAR-T cell therapies, which require efficient blood cell collection.
With ongoing and upcoming clinical studies, including a recent trial combining burixafor with propranolol to enhance mobilization, Exicure is poised to address significant challenges in hematologic diseases. The potential of burixafor not only to improve engraftment outcomes in ASCT but also to facilitate advanced therapies marks a pivotal development in stem cell mobilization strategies.
MWN-AI** Analysis
Exicure, Inc. (NASDAQ: XCUR) has made significant strides with its lead drug, burixafor, following promising results from a Phase 2 clinical study published in the *Annals of Hematology*. The study demonstrated a high success rate (92%) in rapidly mobilizing stem cells in patients with multiple myeloma (MM) and lymphoma undergoing autologous stem cell transplantation (ASCT). These advancements position Exicure favorably within the biotechnology sector, particularly as it aims to address critical challenges in hematologic diseases.
The rapid mobilization capability of burixafor, which peaks within one hour of administration—far faster than existing CXCR4 inhibitors—suggests an innovative approach to streamline treatment protocols and reduce healthcare costs. This increased efficiency not only enhances patient outcomes but also applies to emerging gene therapies, making burixafor a versatile asset in Exicure's pipeline.
The positive clinical outcomes, coupled with support from significant presentations at notable industry conferences, could enhance investor confidence, potentially driving XCUR's market performance upward. As the biopharmaceutical landscape increasingly values efficient therapeutic solutions, Exicure's commitment to advancing burixafor’s development presents a compelling investment narrative.
Investors should closely monitor the company’s upcoming trials and data releases, especially as they evaluate burixafor's efficacy in other conditions, such as sickle cell disease and acute myeloid leukemia (AML). Given the complexity and competitiveness of the biotechnology field, while the recent news creates an optimistic outlook, prudent investors should also assess the broader market conditions and potential risks associated with clinical-stage investments.
In summary, Exicure represents an intriguing investment opportunity in the biotech sector, primarily driven by the innovative potential of burixafor, making it worthy of attention for those focused on healthcare advancements.
**MWN-AI Summary and Analysis is based on asking OpenAI to summarize and analyze this news release.
REDWOOD CITY, Calif., Feb. 05, 2026 (GLOBE NEWSWIRE) -- Exicure, Inc. (Nasdaq: XCUR), a clinical-stage biotechnology company developing therapeutics for hematologic diseases, today announced the publication of results from a prior Phase 2 clinical study evaluating burixafor (GPC-100/TG-0054), a highly selective CXCR4 inhibitor, in the journal Annals of Hematology.
The manuscript, titled “Burixafor, a CXCR4 Inhibitor with a Differentiated Kinetics Profile: Results of a Phase 2 Study for Rapid Cell Mobilization in Multiple Myeloma and Lymphoma Patients Undergoing Transplant,” reports results from a 12-participant, multi-center, open-label Phase 2 study (NCT02104427) evaluating burixafor in combination with granulocyte colony-stimulating factor (G-CSF) in patients with multiple myeloma (MM), non-Hodgkin’s lymphoma (NHL), and Hodgkin disease (HD) undergoing autologous stem cell transplantation (ASCT).
In the study, 11 of 12 participants (92%) met the primary endpoint of collecting ?5.0 × 10? CD34+ cells/kg within two leukapheresis sessions, with six participants achieving the target in a single session. Median time to neutrophil and platelet engraftment was 12 and 22 days, respectively. Burixafor was generally well tolerated, with only two treatment-related adverse events reported, both low grade.
Notably, peak mobilization of CD34+ cells occurred within one hour of burixafor administration, substantially faster than currently approved CXCR4 inhibitors, which typically peak 10-14 hours after dosing. This rapid mobilization profile enables same-day leukapheresis, offering the potential to simplify treatment logistics, reduce hospital resource utilization, and minimize patient burden.
In addition to robust stem cell mobilization, burixafor in combination with G-CSF led to marked increases in circulating white blood cell subsets, including lymphocytes. In participants with MM, lymphocyte counts increased by up to 11-fold within hours of burixafor administration, supporting potential applicability in gene therapy, CAR-T, and other gene-editing workflows that require efficient peripheral blood cell collection.
“ASCT remains a treatment cornerstone for multiple myeloma and certain lymphomas. Efficient mobilization of peripheral blood progenitor cells is critical to achieving reliable engraftment while minimizing patient burden and overall healthcare costs,” said Michael Schuster, MD, Clinical Professor of Medicine at Stony Brook University and a study author. “In this study, burixafor demonstrated enhanced mobilization kinetics that led to significantly faster attainment of stem cell collection goals, highlighting its differentiated profile and potential to meaningfully improve the efficiency of cell collection for ASCT. Beyond transplant, rapid and predictable mobilization of CD34-positive cells and lymphocytes may also be highly relevant for emerging gene-based and gene-editing approaches, including those being explored for conditions such as sickle cell disease.”
Exicure continues to advance burixafor’s clinical development and has recently completed an additional Phase 2 study evaluating burixafor in combination with G-CSF and propranolol in patients with multiple myeloma undergoing autologous hematopoietic cell transplantation (NCT05561751). The addition of propranolol was informed by previously published preclinical data demonstrating functional crosstalk between CXCR4 and beta-adrenergic receptors, suggesting the potential for enhanced mobilization through dual pathway blockade. Positive topline data from this recent study were presented at the American Society of Hematology (ASH) Annual Meeting in December 2025 and the Tandem Meetings in February 2026, further supporting burixafor’s differentiated and rapid mobilization profile in the transplant setting.
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, burixafor (GPC-100), 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, burixafor may enhance stem cell mobilization into the peripheral blood to support collection and use in autologous stem cell transplantation (ASCT).
Burixafor is being evaluated for its potential to improve stem cell mobilization in multiple myeloma, sickle cell disease, and in support of cell and gene therapy. In addition, Exicure is planning a chemosensitization trial in acute myeloid leukemia (AML), leveraging burixafor’s ability to mobilize malignant cells from protective bone marrow niches into the peripheral blood, where they may be more effectively targeted by chemotherapy. Burixafor became part of Exicure’s pipeline following the company’s acquisition of GPCR Therapeutics, Inc. in January 2025. For more information, visit www.exicuretx.com.
Media Contact:
Sarah Ellinwood, PhD
Kendall Investor Relations
sellinwood@kendallir.com
FAQ**
What are the implications of the Phase 2 study results for Exicure Inc. (XCUR) and its future clinical trials, particularly in the context of multiple myeloma and lymphoma treatments?
How does the differentiated kinetics profile of burixafor contribute to Exicure Inc. (XCUR)'s competitive positioning within the biotechnology sector?
What potential opportunities exist for Exicure Inc. (XCUR) to expand the application of burixafor in gene therapy and other innovative treatments in hematologic diseases?
Given the positive topline data presented by Exicure Inc. (XCUR) at recent medical meetings, what are the projected next steps for the company's clinical development of burixafor?
**MWN-AI FAQ is based on asking OpenAI questions about Exicure Inc. (NASDAQ: XCUR).
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