On April 11, 2025 Exicure, Inc. (Nasdaq: XCUR) reported updates on its lead asset, GPC-100 (burixafor), a small molecule CXCR4 inhibitor (Press release, Exicure, APR 11, 2025, View Source [SID1234651894]).

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Exicure, Inc. ("Exicure") is planning for a clinical trial in Acute Myeloid Leukemia (AML) with GPC-100. The company believes that GPC-100, when paired with modern AML treatment regimens, can further improve outcomes in this high unmet need clinical indication. A Phase 1 chemosensitization study involving 15 patients with relapsed or refractory AML was previously conducted by Taigen, the original developer of GPC-100. In that study, GPC-100 was combined with fludarabine and cytarabine to evaluate safety and preliminary efficacy. Preclinical data published by Dr. Pam Becker at City of Hope in collaboration with GPCR Therapeutics USA, a subsidiary of Exicure, demonstrated that dual inhibition of CXCR4 with GPC-100 and beta-2 adrenergic receptor blockade led to improved chemotherapy response (2024 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual meeting : Poster #2758). This therapeutic approach is protected by an expanded patent portfolio granted in the United States, Japan, Australia, and Taiwan.

In addition to AML, GPC-100 is currently being evaluated in an ongoing Phase 2 trial in patients with multiple myeloma (MM) undergoing autologous stem cell transplant (ASCT; NCT05561751). Clinical trial results for this study are expected in Q4 2025.

Current Evaluating Potential Indication Expansions – In addition to AML and MM, Exicure is evaluating a range of potential indications for GPC-100, including sickle cell disease, pediatric oncology, and cell and gene therapy settings.