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Exicure, Inc., an early-stage biotechnology company focused on developing nucleic acid therapies targeting ribonucleic acid against validated targets, is planning for a clinical trial in acute myeloid leukaemia (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 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, paediatric oncology, and cell and gene therapy settings.
Exicure, Inc. has historically been an early-stage biotechnology company focused on developing nucleic acid therapies targeting ribonucleic acid against validated targets. Following its restructuring and suspension of clinical and development activities, the company is exploring strategic alternatives to maximize stockholder value.
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