Through transcriptional activation, saRNA therapeutics promise a revolution in our ability to modulate previously undruggable targets.
MTL-CEBPA is a new medicine initially being developed as a combination therapy in cancer. The first small activating RNA (saRNA) therapeutic to be tested in a clinical study, MTL-CEBPA is designed to activate the CEBPA gene. MTL-CEBPA comprises a double stranded RNA payload formulated inside a liposomal nanoparticle.
The CEBPA gene encodes CCAAT/enhancer binding protein alpha (C/EBP-α), a transcription factor that acts as a master regulator of cell lineage determination and differentiation in several tissues including myeloid cells, liver cells and adipose tissue. In cancer, C/EBP-α plays an important role in regulating tumour growth and the tumour microenvironment. Activating CEBPA expression in the tumour microenvironment is hypothesised to improve the efficacy of cancer therapies in solid tumour malignancies.
For more information on MTL-CEBPA check out these publications:
MTL-CEBPA DRUG PRODUCT
THE FIRST-IN-HUMAN CLINICAL STUDY OF A saRNA THERAPEUTIC
OUTREACH is a Phase Ib clinical study of MTL-CEBPA, our lead saRNA therapeutic, in patients with liver cancer.
The multi-centre, Phase Ib study is assessing the safety, tolerability and anti-tumour activity of MTL-CEBPA in combination with Sorafenib in patients with advanced primary liver cancer. Sorafenib is the 1st line standard of care in liver cancer. The study is recruiting patients in the United Kingdom, Singapore and Taiwan.
PRIMARY LIVER CANCER
Liver cancer is the second leading cause of cancer-related deaths worldwide with approximately 745,000 deaths each year and the fastest growing cause of cancer deaths in the United States.
Due to complicating liver diseases as well as late detection, the majority of liver cancer patients have limited treatment options, with a five-year survival rate of less than 15%.