Captor Therapeutics, a biopharmaceutical company developing drugs based on Targeted Protein Degradation (TPD), has applied for approval to conduct a first-in-human Phase 1 clinical trial with CT-01, a compound developed as part of a FENG-funded project entitled: "Discovery and development of a drug candidate for the treatment of hepatocellular carcinoma to eliminate cancer stem cells by induced degradation of an oncogenic transcription factor". The trial will be conducted by ICON PLC under the supervision of Captor Therapeutics.
- We have been preparing for many months to make the most important announcement in the CT-01 project, the start of clinical trials. We do so with great satisfaction and a sense of responsibility for the next steps that could bring new treatment options to many patients around the world. Despite significant progress in recent years, treatment options for advanced HCC remain limited. Available systemic treatments are characterized by a moderate response rate and impact on survival, and most patients experience disease progression despite available treatments. As a result, the median survival for patients with metastatic disease is approximately 20 months. Therefore, the treatment of patients with advanced hepatocellular carcinoma remains an unmet medical need," commented Dr. Tom Shepherd, CEO of Captor Therapeutics.
The Company's submission is for a planned multi-center, open-label, dose escalation and expansion Phase 1 study. The study is designed to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of CT-01, both as monotherapy and in combination with everolimus, in patients with moderately to highly advanced HCC (BCLC grade B or C) and preserved liver function (Child-Pugh grade A). The multicenter clinical trial will be conducted at leading European liver cancer centers in Spain, Germany and France. Patients will receive escalating doses of CT-01 orally.
The primary objective of the study is to evaluate safety and tolerability following experimental treatment with the drug candidate in patients diagnosed with primary liver cancer and to determine the maximum tolerated dose and/or recommended dose for further phases of clinical trials in monotherapy and combination treatment. In addition, the anti-tumor activity using radiological imaging and serum tumor markers as well as the pharmacokinetic and pharmacodynamic profile of the drug candidate after monotherapy and in combination treatment will be evaluated.
- CT-01 is a small molecule protein degrader of GSPT1, NEK7 and SALL4 that has demonstrated anti-tumor activity in several liver cancer models in preclinical studies. In combination with everolimus (an inhibitor of the mTOR pathway, which is often dysregulated in cancer cells), CT-01 demonstrated greater anti-tumor efficacy than CT-01 alone or everolimus as monotherapy. The potential anti-tumor efficacy of CT-01 is based on its novel mechanism of action, which involves the removal of factors that affect tumor cell viability (GSPT1) and potentially stabilize the tumor microenvironment (NEK7). Unlike current therapies that rely on inhibiting angiogenesis or activating the immune system, CT-01 works directly by preventing efficient protein synthesis by cancer cells. This is also important in a mechanism that is independent of the presence of an unfavorable mutation of the TP53 protein," said Michal Walczak, Board Member and CSO of Captor Therapeutics.
The goal of the CT-01 project is to develop a drug candidate using targeted protein degradation technology that will halt the progression of hepatocellular carcinoma and provide significant clinical benefit to patients. Primary liver cancer is the sixth most common cancer and the fourth leading cause of cancer-related deaths worldwide. The majority (80-90%) of liver cancers are hepatocellular carcinomas (HCC) that arise from chronic liver disease.
