CPT-6281

CPT-6281 is a first-in-class, orally administered molecular glue degrader that targets three previously "undruggable" proteins implicated in cancer progression and immune regulation: GSPT1 (G1 to S phase transition 1), SALL4 (Sal-like protein 4), and NEK7 (NIMA-related kinase 7). GSPT1 is involved in translation termination, SALL4 is a transcription factor overexpressed in hepatocellular carcinoma and associated with poor prognosis, while NEK7 plays a role in inflammasome activation and pro-carcinogenic IL-1β production. By inducing the proteasomal degradation of these proteins, CPT-6281 triggers apoptosis via integrated stress response pathways, reduces tumor-promoting inflammation, and potentially restores anti-tumor immunity. The drug is designed as a prodrug activated by enzymes highly expressed in the liver, lungs, and certain gastrointestinal tumors—making it particularly suitable for treating hepatocellular carcinoma (HCC), lung cancer, neuroendocrine tumors, and possibly other solid tumors. Developed by Captor Therapeutics using targeted protein degradation technology (molecular glue modality), CPT-6281 represents an innovative approach to address multiple oncogenic drivers simultaneously[1][2][3].

Other names

CSPT1/NEK7/SALL4 degrader