Exicure’s burixafor nears phase 2 data readout in multiple myeloma

Published 06/10/2025, 12:06
Exicure’s burixafor nears phase 2 data readout in multiple myeloma

REDWOOD CITY, Calif. - Exicure, Inc. (NASDAQ:XCUR), a $26.35 million market cap biotechnology company, announced Monday that it is approaching a key data readout for its lead program burixafor (GPC-100) in multiple myeloma, with plans to expand into additional indications including sickle cell disease and acute myeloid leukemia. According to InvestingPro data, the stock has shown significant volatility, with a 54% gain over the past year despite a recent 68% decline over the last six months.

The biotechnology company reported that its Phase 2 trial evaluating burixafor for stem cell mobilization in autologous stem cell transplant procedures has completed patient visits and database lock, with topline results expected in Q4 2025. With a beta of 3.77, the stock’s high volatility suggests significant potential price movements around key clinical milestones. InvestingPro subscribers can access 8 additional key metrics and insights about Exicure’s market performance.

Interim results from the trial have shown that 100% of patients (10/10) achieved the primary endpoint of successful CD34+ stem cell mobilization, including patients previously treated with daratumumab. The company highlighted that burixafor allows for same-day administration and collection, potentially differentiating it from other approved agents.

Burixafor is a small molecule CXCR4 antagonist designed to mobilize hematopoietic stem cells from bone marrow into the bloodstream for collection and use in transplant procedures.

Exicure also announced new leadership appointments, including Josephine Cardarelli as President and Chief Scientific Officer, Niña Caculitan as Head of Clinical, and Devki Sukhtankar as Head of Preclinical Research and Translational Medicine.

"The progress we’ve achieved since integrating GPCR Therapeutics earlier this year reflects both the strength and potential of the burixafor program and the dedication of our team," said Andy Yoo, Chief Executive Officer of Exicure, according to the company’s press release.

The company is preparing for a potential Phase 3 trial in multiple myeloma while simultaneously planning an investigator-sponsored trial in sickle cell disease and a Phase 1 chemosensitization study in acute myeloid leukemia.

Exicure acquired the burixafor program through its acquisition of GPCR Therapeutics USA in January 2025.

In other recent news, Exicure Inc announced the completion of the last patient visit in its Phase 2 clinical trial for GPC-100, aimed at multiple myeloma patients undergoing stem cell transplants. The company plans to release the topline results from this study in the fourth quarter of 2025. The trial is focused on assessing the effectiveness of GPC-100, a small molecule CXCR4 antagonist, in enhancing the mobilization of hematopoietic stem cells from bone marrow into peripheral blood for collection and use in autologous stem cell transplant procedures. These developments mark a significant milestone for Exicure in its ongoing research efforts. The announcement comes as part of Exicure’s broader strategy to advance its clinical-stage biotechnology initiatives. Investors and stakeholders are closely monitoring these updates as they could have implications for the company’s future endeavors.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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