1. XCUR's burixafor achieved 90% success in mobilizing stem cells for myeloma. 2. Results were presented at the ASH Annual Meeting, enhancing visibility. 3. Burixafor shows potential in treating multiple myeloma and other conditions. 4. Strong safety profile noted; no severe side effects reported. 5. Future applications may extend to rare diseases and AML treatments.
FAQ
Why Bullish?
Positive trial results typically lead to investor optimism; historic examples like CRISPR Therapeutics show similar trends.
How important is it?
High likelihood of trial success influencing XCUR’s stock; similar biotech results often yield significant price movements.
Why Short Term?
Immediate market attention expected post-presentation; historical responses to trial results support this.
Exicure Reports Promising Phase 2 Trial Results for Burixafor in Multiple Myeloma at ASH Annual Meeting
REDWOOD CITY, Calif., December 08, 2025 — Exicure, Inc. (Nasdaq: XCUR), a clinical-stage biotechnology company focused on therapies for hematologic diseases, unveiled positive findings from its Phase 2 trial of burixafor (GPC-100). This investigational treatment, tested with propranolol and granulocyte colony-stimulating factor (G-CSF), aims to facilitate the mobilization of hematopoietic progenitor cells (HPCs) for patients with multiple myeloma undergoing autologous hematopoietic cell transplantation (AHCT). The results, showing that approximately 90% of participants met the primary endpoint, were presented today at the 67th American Society of Hematology (ASH) Annual Meeting in Orlando, Florida.
Trial Overview and Key Findings
The Phase 2 trial, registered under NCT05561751, was an open-label, multicenter study designed to evaluate the efficacy of burixafor in conjunction with G-CSF and propranolol. A total of 19 participants were included in the analysis, out of which 17 (or 89.5%) successfully collected ≥2 × 10⁶ CD34+ cells/kg required for transplantation. While two others needed an additional leukapheresis session, the trial's results underline the rapid mobilization capabilities of burixafor.
- Median time to neutrophil engraftment: 13 days
- Median time to platelet engraftment: 17.5 days
- Peak CD34+ cell levels observed within one hour of burixafor administration
Interestingly, 16 out of 19 participants had previously received daratumumab, a treatment known to reduce mobilization efficacy. Yet, 14 of these individuals (or 87.5%) achieved the primary endpoint, including 12 out of 14 who also underwent treatment with lenalidomide. This suggests that burixafor demonstrates resilience in patient populations typically expected to respond poorly.
Safety and Tolerability
The combination therapy was well tolerated, exhibiting a commendable safety profile. Notably, there were no burixafor-related adverse events exceeding Grade 2 severity, reinforcing its safety in clinical use.
Insight from the Lead Investigator
Dr. Jack Khouri, Associate Professor of Medicine at the Cleveland Clinic Lerner College of Medicine and lead investigator of the study, commented on the findings: “The combination of burixafor, G-CSF, and propranolol exhibited an excellent safety profile and effectively mobilized hematopoietic progenitor cells. This allowed all participants who chose to proceed with transplantation to successfully engraft. We are particularly encouraged by the results from participants previously treated with daratumumab, as they may ordinarily experience reduced stem cell yields.”
Presentation Details
The results were shared in an oral presentation, with the following specifics:
- 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
- Session: 711. Cell Collection and Manufacturing of HSPCs, CAR-T Cells, and Other Cellular Therapy Products
- Date and Time: December 8, 2025, from 5:45-6:00 PM EST
- Location: Hyatt - Regency Ballroom R
About Burixafor (GPC-100)
Burixafor is a highly selective small molecule antagonist of CXCR4, a critical chemokine receptor involved in the retention of hematopoietic stem cells within the bone marrow niche. By blocking CXCR4, burixafor may enhance the mobilization of these vital cells into the peripheral blood, facilitating autologous stem cell transplant (ASCT) procedures.
Originally developed by GPCR Therapeutics, Inc., burixafor joined Exicure’s portfolio following its acquisition in January 2025. Beyond its application in multiple myeloma, burixafor's potential is being explored in various conditions requiring improved stem cell mobilization, including sickle cell disease, other rare diseases, and cell and gene therapy spaces. Furthermore, a chemosensitization study in acute myeloid leukemia (AML) is also planned.
About Exicure
Exicure, Inc. (Nasdaq: XCUR) is dedicated to developing innovative therapies to tackle critical challenges in hematologic diseases. The company’s primary program, burixafor (GPC-100), aims to improve stem cell mobilization not only in multiple myeloma but also in sickle cell disease, as well as to support cell and gene therapy initiatives. For additional information, please visit www.exicuretx.com.
Media Contact
Sarah Ellinwood, PhD
Kendall Investor Relations
sellinwood@kendallir.com