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Sunday, April 13, 2025

Exicure Planning for a New Clinical Trial in Acute Myeloid Leukemia



Exicure (XCUR) has announced plans for a new clinical trial of its lead asset GPC-100 (burixafor) in Acute Myeloid Leukemia (AML). The CXCR4 inhibitor has shown promise when combined with modern AML treatments. A previous Phase 1 study with 15 relapsed/refractory AML patients evaluated GPC-100's safety and preliminary efficacy in combination with fludarabine and cytarabine.

Recent preclinical data presented at the 2024 ASH Annual Meeting demonstrated improved chemotherapy response through dual inhibition of CXCR4 with GPC-100 and beta-2 adrenergic receptor blockade. The company has secured patents for this approach in multiple countries.

GPC-100 is currently in a Phase 2 trial for multiple myeloma patients undergoing autologous stem cell transplant, with results expected in Q4 2025. Exicure is also exploring potential expansions into sickle cell disease, pediatric oncology, and cell and gene therapy applications.

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