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EpCAM-SN-38 is a preclinical-stage targeted therapeutic being developed by RNA NanoBiotics for the treatment of EpCAM-positive metastatic colorectal cancer. The drug utilizes a programmable, self-assembling RNA nanostructure platform (specifically an aptamer-guided 4-way junction RNA nanocarrier) to selectively deliver SN-38, the active metabolite of the chemotherapeutic irinotecan, directly to EpCAM-expressing tumor cells. By targeting the epithelial cell adhesion molecule (EpCAM) on cancer cells, the platform aims to enhance tumor specificity, facilitate intracellular delivery, and enable controlled release of the cytotoxic payload, thereby reducing systemic toxicities associated with conventional chemotherapy. RNA NanoBiotics is leveraging the FDA's 505(b)(2) regulatory pathway to accelerate the clinical development of this candidate.
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