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STX002 is a next-generation antibody-drug conjugate (ADC) designed for the treatment of hepatocellular carcinoma (HCC). It consists of a humanized monoclonal antibody targeting Glypican-3 (GPC3) site-specifically conjugated to a potent topoisomerase I inhibitor payload via a cleavable linker. GPC3 is an oncofetal antigen highly expressed in HCC but minimally present in normal adult tissues, facilitating targeted delivery of the cytotoxic agent. Upon binding to GPC3-positive cells, STX002 is internalized, releasing the payload to inhibit DNA topoisomerase I, which leads to DNA damage and apoptosis. Preclinical studies have demonstrated that STX002 induces deep and durable tumor regressions in various HCC models, including those resistant to standard-of-care tyrosine kinase inhibitors, while maintaining a wide therapeutic index and favorable pharmacokinetic profile.
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