Exicure reported that its Phase 2 study of burixafor (GPC‑100) combined with propranolol and granulocyte colony‑stimulating factor mobilized hematopoietic progenitor cells in 90 % of patients with multiple myeloma. The 19‑patient trial met the primary endpoint of collecting ≥2 × 10⁶ CD34⁺ cells per kilogram of body weight in 17 participants, with two additional patients requiring a third leukapheresis session.
The study’s patient cohort included 16 individuals who had previously received daratumumab, a drug that can impair stem‑cell mobilization. Of those 16 patients, 14—an 87.5 % success rate—achieved the primary endpoint, underscoring burixafor’s effectiveness in a challenging, heavily pre‑treated population. Safety data were favorable; no burixafor‑related adverse events exceeded Grade 2 severity, and median neutrophil and platelet engraftment occurred 13 and 17.5 days post‑transplant, respectively, indicating a rapid and efficient mobilization process.
Burixafor’s rapid mobilization kinetics differentiate it from existing CXCR4 antagonists such as plerixafor, which require overnight pre‑treatment. The ability to mobilize stem cells within the same day of administration could streamline clinical workflows, reduce patient burden, and provide a competitive advantage in the stem‑cell mobilization market. This operational benefit, combined with the high success rate in daratumumab‑exposed patients, positions burixafor as a potentially superior alternative to current agents.
The positive Phase 2 data strengthen Exicure’s pipeline and support the company’s plan to advance burixafor into Phase 3 trials for multiple myeloma. Exicure also intends to explore the drug in other hematologic indications, including sickle cell disease and acute myeloid leukemia, leveraging the same rapid‑mobilization platform. Successful Phase 3 outcomes could open pathways to regulatory approval and broaden the commercial potential of the GPC‑100 platform.
Dr. Jack Khouri, the study’s lead investigator, noted that the combination of burixafor, G‑CSF, and propranolol produced an excellent safety profile and reliable mobilization, enabling all participants who proceeded to transplant to achieve successful engraftment. CEO Andy Yoo added that the results “reflect the strength of the burixafor program and the dedication of our team,” and that the company remains focused on advancing the platform across multiple indications.
Investors responded positively to the announcement, reflecting confidence in the clinical and commercial potential of burixafor and the broader GPC‑100 platform.
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