- CT-01 compounds induce degradation of GSPT1, SALL4 and another as yet undisclosed neo-substrate
- The unique degradation profile supports the strong competitive potential of the program
- Compelling pre-clinical data generated demonstrating HCC tumor regression
- CT-01 on track to enter clinical development in 2023
WROCLAW,
Compelling in-vitro and in-vivo preclinical data demonstrate that CT-01 candidate compounds induce degradation of Eukaryotic peptide chain release factor GTP-binding subunit ERF3A (GSPT1), Sal-like protein 4 (SALL4) and another undisclosed neo-substrate with essential function in tumorigenesis. GSPT1 is a protein involved in the termination of translation, a process in which ribosomes synthesize proteins after the transcription of DNA to RNA. There is a demonstrated link between GSPT1 degradation and antitumor activity. SALL4 is a transcription factor expressed in the human fetal liver and silenced in adults but often re-expressed in HCC patients, which correlates with poor prognosis.
"The CT-01 targets fundamentally contribute to cancer development and their elimination has potent therapeutic potential in the treatment hepatocellular carcinoma as well as several other malignances," said Dr
About Project CT-01 and HCC
The purpose of Project CT-01 is to develop, based on targeted protein degradation technology, a drug candidate which will stop the progress of hepatocellular carcinoma (HCC) and potentially offer significant benefits for patients. HCC, a form of liver cancer, constitutes a significant unmet medical need since most patients are diagnosed at a late stage of the disease, and present treatments bring limited benefits in terms of overall survival rate. With ~700,000 new cases each year, HCC constitutes the second most common cause of cancer mortality. In patients diagnosed early, surgical removal of the tumour remains the only effective therapy. In unresectable HCC, the best reported outcome is the combination of Atezolizumab (Tecentriq®) plus Bevacizumab (Avastin®), where 19.2 months median Overall Survival (OS) and 29.8% Overall Response Rate (ORR) were reported in the IMbrave150 study, indicating that there remains a dramatic need for new treatments.
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