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RGT-264 is a small molecule, orally administered, highly potent and selective inhibitor of hematopoietic progenitor kinase 1 (HPK1), also known as mitogen-activated protein kinase kinase kinase kinase 1. HPK1 acts as a negative regulator of immune cell function, particularly in T cells and dendritic cells. By inhibiting HPK1 activity, RGT-264 aims to enhance anti-tumor immune responses, making it a promising candidate for cancer immunotherapy. The drug was initially developed by Shanghai Qilu Ruige Pharmaceutical R&D Co., Ltd., with current development led by Regor Therapeutics Group/Regor Pharmaceuticals Inc. Its primary indication is for the treatment of solid tumors (neoplasms). As of the latest updates in early 2025, RGT-264 has reached Phase 1 clinical trials for advanced solid tumors but has not yet received regulatory approval[1][2][3][4][5].
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