Exicure’s Phase 2 Data for Burixafor in Multiple Myeloma Highlights High Success Rate and Rapid Stem Cell Mobilization
89.5% of Participants Hit Stem Cell Collection Goals—What Makes Burixafor Stand Out?
Exicure (NASDAQ:XCUR) is making headlines after presenting positive topline results from its Phase 2 trial evaluating burixafor in combination with propranolol and G-CSF for stem cell mobilization in multiple myeloma patients. Data were unveiled at the 2025 ASH Annual Meeting, showing 89.5% of study participants achieved the primary endpoint of collecting at least 2x106 CD34+ cells/kg within just two leukapheresis sessions. The therapy’s rapid kinetics—peaking within one hour—differentiates it from other CXCR4 inhibitors, allowing for same-day administration and apheresis.
Data Table: Burixafor Phase 2 Outcomes Stand Out Among Heavily Treated Patients
| Parameter | Value |
|---|---|
| Participants (N) | 19 |
| Met Primary Endpoint in =2 Sessions | 89.5% |
| Prior Daratumumab Exposure | 84.2% |
| Success in Prior Daratumumab Group | 87.5% |
| Median Time to Neutrophil Engraftment | 13 days |
| Median Time to Platelet Engraftment | 17.5 days |
| Peak CD34+ Levels Post-Administration | <1 hour |
| Serious Adverse Events (>Grade 2) | None |
Safety Profile Remains Strong Even in Difficult-to-Treat Cohorts
Burixafor’s combination with propranolol and G-CSF was well tolerated. Importantly, there were no burixafor-related adverse events above Grade 2, even among participants with prior exposure to daratumumab—a therapy known to make stem cell collection harder. Notably, 87.5% of patients who had been treated with daratumumab still reached the cell collection target.
Clinical Significance: Differentiated, Rapid Kinetics Offer Efficiency and Convenience
The standout feature is the speed at which stem cells can be mobilized for collection—peak circulating CD34+ cells are observed within an hour of dosing. This opens the door for a streamlined process that could lessen both clinical complexity and patient burden during transplant preparation.
Burixafor’s Pipeline Expands Beyond Multiple Myeloma
While the Phase 2 trial focused on multiple myeloma, Exicure is eyeing broader indications—including sickle cell disease and chemosensitization in acute myeloid leukemia (AML). This positions burixafor as a potentially valuable tool in several hematologic settings, particularly for patients with challenging mobilization histories.
What’s Next for Investors and Patients?
The Phase 2 success signals meaningful clinical progress for Exicure’s burixafor program. Investors and clinicians alike will want to monitor the transition into additional studies and other disease applications. With rapid mobilization kinetics, a clean safety record, and encouraging data in previously hard-to-mobilize patients, burixafor could soon redefine expectations in autologous stem cell transplant preparation. Will these promising results translate into a broader transformation for stem cell collection standards? Only time—and larger trials—will tell.
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