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New Burixafor Clinical Data to be Presented as an Oral Presentation at the 2025 ASH Annual Meeting

MWN-AI** Summary

Exicure, Inc. (Nasdaq: XCUR) has announced an upcoming oral presentation regarding burixafor, a small molecule CXCR4 antagonist, at the 67th American Society of Hematology (ASH) Annual Meeting, scheduled for December 6-9, 2025, in Orlando, Florida. This presentation will highlight findings from an open-label, multicenter Phase 2 study (NCT05561751) that assesses the safety and efficacy of burixafor in patients with multiple myeloma undergoing autologous hematopoietic cell transplantation (AHCT).

Burixafor functions by blocking the CXCR4 receptor, which is essential for retaining hematopoietic progenitor cells in the bone marrow. This action facilitates the mobilization of these cells into peripheral blood, making them available for collection during transplantation. The trial results revealed that the combination of burixafor, G-CSF, and propranolol exhibited an excellent safety profile and effectively mobilized hematopoietic progenitor cells. Encouragingly, nearly all patients (18 out of 19) who opted for transplant proceeded to AHCT, despite the patients predominantly being treated with daratumumab, an anti-CD38 monoclonal antibody linked to lower cell mobilization rates in previous studies.

The oral presentation, led by Dr. Jack Khouri from the Cleveland Clinic Lerner College of Medicine, will occur on December 8, 2025, from 5:45 to 6:00 PM EST in Hyatt - Regency Ballroom R. Burixafor is not only being studied in multiple myeloma but also has potential applications in treating sickle cell disease, rare disorders requiring autologous transplants, and enhancing outcomes in cell and gene therapies. Future investigations include a chemosensitization trial in acute myeloid leukemia (AML), leveraging burixafor’s ability to mobilize malignant cells into circulation for improved chemotherapy targeting.

MWN-AI** Analysis

Exicure, Inc. (Nasdaq: XCUR) is poised for a significant inflection point as it presents pivotal data on burixafor at the upcoming 2025 ASH Annual Meeting. Given the Phase 2 trial's positive outcomes regarding the safety and efficacy of burixafor in mobilizing hematopoietic progenitor cells (HPCs) for patients with multiple myeloma, the market is closely monitoring investor sentiment.

The encouraging results, particularly the high transplantation rate (18 out of 19 patients) and the favorable safety profile of burixafor combined with G-CSF and propranolol, suggest a robust value proposition for Exicure. The fact that patients previously treated with daratumumab, known for complicating HPC mobilization, still achieved successful outcomes, further underscores the potential of burixafor to improve clinical efficacy in challenging populations.

From an investment perspective, the data release during ASH could catalyze bullish momentum for Exicure’s stock. Should the presentation be well-received, it may lead to heightened interest from institutional investors and analysts, potentially driving the stock price upward. Furthermore, the company's strategy to explore burixafor in broader applications—including sickle cell disease and acute myeloid leukemia (AML)—could diversify its therapeutic portfolio and enhance long-term valuation prospects.

However, investors should remain cautious and aware of the inherent risks of clinical-stage biotech investments, including regulatory hurdles and market competition. It may be wise to adopt a wait-and-see approach following the ASH presentation, assessing both market reactions and subsequent analyst reports.

In summary, Exicure represents a compelling opportunity following the release of favorable burixafor data, but careful monitoring of developments is essential for a balanced investment strategy. As the market digests the upcoming presentation, potential investors should weigh both the promising data and the broader market landscape before making decisions.

**MWN-AI Summary and Analysis is based on asking OpenAI to summarize and analyze this news release.

Source: GlobeNewswire

REDWOOD CITY, Calif., Nov. 03, 2025 (GLOBE NEWSWIRE) -- Exicure, Inc. (Nasdaq: XCUR), a clinical-stage biotechnology company developing therapeutics for hematologic diseases, today announced that results from its completed Phase 2 study evaluating burixafor, a small molecule CXCR4 antagonist, will be featured in an oral presentation at the 67th American Society of Hematology (ASH) Annual Meeting and Exposition, taking place December 6-9, 2025, in Orlando, Florida.

The presentation will highlight additional patient data from the open-label, multicenter Phase 2 trial (NCT05561751) assessing burixafor in patients with multiple myeloma undergoing autologous hematopoietic cell transplantation (AHCT). By blocking CXCR4, burixafor is designed to mobilize hematopoietic progenitor cells from the bone marrow into the peripheral blood, where they can be collected for use in transplantation procedures.

“The combination of burixafor, G-CSF and propranolol demonstrated an excellent safety profile and was able to ensure adequate and efficient mobilization of hematopoietic progenitor cells, with all patients who chose to undergo transplant (18/19) proceeding to AHCT. These are very encouraging results, especially since the majority of the enrolled patients were treated with the anti-CD38 monoclonal antibody daratumumab, which has been associated with lower HPC mobilization in certain studies. The same day administration of burixafor with leukapheresis is also more convenient for patients and may reduce time toxicity,” said Jack Khouri, M.D., Associate Professor of Medicine, Cleveland Clinic Lerner College of Medicine, Case Western Reserve University, study lead investigator, and presenter.

Oral Presentation Details
Abstract Number: 1050
Title: An open-label, multi-center Phase 2 study to assess the safety and efficacy of burixafor (GPC-100) and propranolol with G-CSF for the mobilization of hematopoietic progenitor cells in patients with multiple myeloma
Presenter: Dr. Jack Khouri, Associate Professor of Medicine, Cleveland Clinic Lerner College of Medicine, Case Western Reserve University
Session: 711. Cell Collection and Manufacturing of HSPCs, CAR-T Cells, and Other Cellular Therapy Products: Refining CAR-T Cells and Engineered HSPCs; New Approaches to HSPC mobilization
Date and Time: December 8, 2025, 5:45-6:00pm EST
Location: Hyatt - Regency Ballroom R

About Burixafor (GPC-100)
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 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., burixafor became part of Exicure’s pipeline following the company’s acquisition in January 2025. In addition to multiple myeloma, burixafor is also being considered in 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. A chemosensitization trial in AML is also being planned, leveraging burixafor’s mechanism of mobilizing malignant cells from protective bone marrow niches into the peripheral blood, where they may be more effectively targeted by chemotherapy.

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 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 .

Contact:
Exicure, Inc.
847.673.1700 (Tel)
847.556.6411 (Fax)


FAQ**

What potential market impact could the results of the Phase 2 study of burixafor by Exicure Inc. XCUR have on the treatment landscape for multiple myeloma and other hematologic diseases?

The Phase 2 results of burixafor by Exicure Inc. (XCUR) could significantly enhance treatment options for multiple myeloma and hematologic diseases, potentially leading to improved patient outcomes and a shift in clinical practices within the oncology landscape.

How might the safety profile and efficacy of burixafor influence investor confidence in Exicure Inc. XCUR's pipeline and overall company valuation going forward?

The safety and efficacy of burixafor could significantly bolster investor confidence in Exicure Inc.'s pipeline and overall valuation by demonstrating the potential for successful clinical outcomes and market approval, thereby enhancing the company’s perceived growth prospects.

With burixafor's possible applications beyond multiple myeloma, such as sickle cell disease and AML, how does Exicure Inc. XCUR plan to prioritize these research areas in terms of funding and resource allocation?

Exicure Inc. (XCUR) plans to prioritize funding and resource allocation for burixafor's applications in sickle cell disease and AML by evaluating potential market impact, aligning with strategic partnerships, and assessing scientific merit to optimize development pathways.

Can you elaborate on Exicure Inc. XCUR's strategy for leveraging the data presented at the ASH Annual Meeting to attract partnerships or collaborations in the biotechnology space?

Exicure Inc. aims to leverage data from the ASH Annual Meeting by showcasing its innovative RNA-based therapeutics and their potential impact on patient outcomes, thereby attracting strategic partnerships and collaborations with biotechnology firms interested in advancing gene-targeted treatments.

**MWN-AI FAQ is based on asking OpenAI questions about Exicure Inc. (NASDAQ: XCUR).

Exicure Inc.

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