AST-201

**AST-201** is an investigational glypican-3-targeted aptamer-drug conjugate developed by Aptamer Sciences for GPC3-positive advanced solid tumors. The synthetic aptamer selectively binds glypican-3 on tumor cells and is designed to deliver an activated gemcitabine payload through receptor-mediated endocytosis, potentially bypassing gemcitabine-resistance mechanisms involving reduced nucleoside-transporter uptake and cytidine deaminase-mediated inactivation. AST-201 is being evaluated as intravenous monotherapy in the first-in-human Phase 1 AST-201-01 study in GPC3-positive advanced solid tumors, including hepatocellular carcinoma and squamous non-small cell lung cancer. ([clinicaltrials.gov](https://clinicaltrials.gov/study/NCT06687941))