Exicure's Burixafor Achieves 90% Success in Phase 2 Multiple Myeloma Trial, Showing Rapid and Reliable Stem Cell Mobilization
Positive Topline Data Shows Consistent Mobilization, Even in Challenging Patient Cohorts
Exicure (NASDAQ:XCUR) has delivered encouraging news for patients and clinicians alike with new Phase 2 data showing its lead compound, burixafor, in combination with propranolol and G-CSF, achieved rapid and reliable hematopoietic stem cell mobilization in multiple myeloma. In results presented at the 2025 American Society of Hematology (ASH) Annual Meeting, 17 of 19 trial participants (89.5%) met the primary endpoint—collecting at least 2x106 CD34+ cells/kg within two leukapheresis sessions. Even among those with prior exposure to challenging regimens like daratumumab, burixafor showed consistent performance.
Key Data Points Highlight Burixafor’s Differentiated Profile
The study’s main numbers highlight why the data stand out in the stem cell mobilization space. Not only did 90% of patients reach the stem cell collection target in just two sessions, but rapid mobilization kinetics allowed same-day dosing and apheresis—potentially streamlining patient experience. Most notably, participants heavily pretreated with daratumumab and lenalidomide (known to reduce stem cell yield) saw primary endpoint success rates above 85%.
| Measure | Result |
|---|---|
| Participants Achieving Primary Endpoint | 17 of 19 (89.5%) |
| Prior Daratumumab Recipients Achieving Endpoint | 14 of 16 (87.5%) |
| Median Neutrophil Engraftment | 13 days |
| Median Platelet Engraftment | 17.5 days |
| Peak Mobilization Time | Within 1 hour of administration |
| Burixafor-Related Adverse Events = Grade 3 | None |
Safety and Speed: Clinical Results Indicate a Favorable Profile
Safety was a key highlight, with no burixafor-related adverse events above Grade 2, reinforcing the therapy's tolerability. For clinicians, this means a more predictable and potentially less taxing experience for patients compared to other CXCR4 inhibitors or conventional mobilization regimens. The ability to mobilize sufficient CD34+ cells with a single administration also supports same-day procedures—potentially reducing patient time in the clinic and lowering healthcare resource utilization.
Potential Beyond Multiple Myeloma: Pipeline Opportunities Emerge
While the trial focused on multiple myeloma, Exicure plans to explore burixafor’s utility across other hematologic and rare diseases—such as sickle cell disease, cell and gene therapy support, and even chemosensitization for AML. By mobilizing both healthy and malignant cells, burixafor may help improve access to autologous transplantation or make cancer cells more vulnerable to chemotherapy in AML.
Takeaway: Exicure Sets Stage for Expanded Burixafor Applications
The latest trial results put Exicure’s burixafor squarely in the spotlight for stem cell mobilization—showing reliability, speed, and a clean safety record even in tough-to-treat cohorts. With expansion planned across additional indications and potential use in chemosensitization, investors and clinicians may want to watch how upcoming trials shape the next chapter for XCUR.
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