Captor Therapeutics S.A. has commenced a clinical trial that could revolutionize the treatment of hepatocellular carcinoma (HCC).
The mechanism of action of CT-01 is based on Targeted Protein Degradation (TPD) technology. The investigational drug causes the removal of two proteins: GSPT1 and NEK7. Degradation of the GSTP1protein triggers the activation of programmed cell death, apoptosis, while removal of the NEK7 protein targets blocks tumor-promoting inflammation in the Tumor MicroEnvironment (TME). As a result, tumor growth is inhibited and the tumor regression is observed in preclinical models of liver cancer.
This innovative approach coud potentially represent a breakthrough in treating patients with advanced hepatocellular carcinoma. For these patients, current treatment options are very limited, and only a small fraction of patients responds to existing therapies.
The Phase 1 trial has began in 19 European clinics across Spain, Germany, and France. It is being conducted in collaboration with the global company ICON, a Contract Research Organization (CRO) specialized in conducting clinical trials. Patient safety is being overseen by an independent committee of experts, an Independent Data Monitoring Committee (IDMC), which monitors the study’s progress.
The first patient has been dosed with the CT-01 drug candidate at the renowned Barcelona Clinic Liver Cancer (BCLC) – where the standard prognostic staging system and a globally adopted treatment strategy for liver cancer were originally developed.
The CT-01 trial is being conducted as an open-label study (meaning both the patient and the physician know that the active drug is being administered, with no placebo control) and features escalating doses of CT-01 in the first part of the trial. The aim of the study is to evaluate safety and to determine the drug dose that will be used in later stages of the trial.
Why is this so important? Hepatocellular carcinoma (HCC) is the sixth most commonly diagnosed cancer and the third leading cause of cancer-related deaths worldwide. It accounts for about 90% of primary liver tumor cases. Most cases of HCC develop on a background of liver cirrhosis, which further complicates treatment and worsens the prognosis.
According to statistics, at advanced stages of the disease (which is how HCC is most frequently diagnosed), only one in five patients will survive 5 years. Projections indicate that by 2030, the number of deaths due to HCC may increase by over 50% compared to 2020.
“CT-01 represents an entirely different treatment approach from those used to date, one based on targeted protein degradation therapy that removes proteins critical for tumor survival. For patients with HCC, this could mean the only real chance for a cure. The project has been enthusiastically received by the hepatology community. Physicians who treat hepatocellular carcinoma are hopeful for the emergence of a new therapeutic option that is unmatched by current treatments. Through its unique mechanism of action, CT-01 may help achieve a treatment response in the majority of patients,” said Robert Dyjas, Medical and Clinical Development Director at CTX.
