CT-01

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.

Captor Therapeutics has signed an agreement for the implementation and funding of the Stage II of the CT-01 Project Captor Therapeutics, a biopharmaceutical company specialized in the development of drugs based on Targeted Protein Degradation ("TPD"), on 12.06.2024 signed an agreement for the implementation and funding of the second stage of its most advanced project, CT-01: "Discovery and development of a new clinical drug candidate for the eradication of cancer stem cel in the treatment of hepatocellular carcinoma, through degradation of oncofetal transcription factor. - Stage II". The agreement approves the extension of the project until March 31, 2026. The decision received is in response to a protest filed in November 2023 by the Company against the originally negative evaluation of the project application. Further Cofinancing in the amount of PLN 6,766,157.95 will be used by the Company for ongoing work on the final formulation of the drug candidate and the start of multi-center clinical trials, still scheduled to begin in 2024. CT-01 is the Company's second implemented in phases Project next to CT-03. The basis of both projects is the application of an innovative approach, i.e. targeted protein degradation (TPD) to fight cancer. In the case of CT-01, the Company has developed a molecule - a triple degrader against proteins: GSPT-1, NEK7 and SALL4 with high anti-cancer activity in xenogeneic models of HCC (Hepatocellular carcinoma), the most common liver cancer worldwide. The therapy being developed by the Company is a response to the very limited treatment options for patients diagnosed with Hepatocellular carcinoma, Currently, the only methods that offer the possibility of a complete cure for this type of cancer are surgery or liver transplantation for which the eligibility criteria are very restrictive. We are confident in the success of Project CT-01 and look forward to the first positive results after administration of our drug. We will keep you informed about the results of the ongoing research