CT-01 + everolimus

CT-01 + everolimus is an investigational, oral combination therapy in development for the treatment of intermediate or advanced hepatocellular carcinoma (HCC)[1][3][5]. The combination pairs CT-01, a novel small molecule protein degrader, with everolimus, a well-established inhibitor of the mTOR pathway. CT-01 targets and induces the degradation of several proteins involved in tumor cell viability and microenvironment stability, specifically GSPT1, NEK7, and SALL4. Everolimus inhibits the mechanistic target of rapamycin (mTOR), a pathway frequently dysregulated in cancer. Preclinical data and the current phase 1 clinical trial suggest the combination has greater anti-tumor efficacy than either agent alone in liver cancer models. The combination is being evaluated for safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy in patients with HCC who have progressed despite or are intolerant to prior systemic therapies[1][3][5].