Through transcriptional activation, saRNA therapeutics promise a revolution in our ability to modulate previously undruggable targets.

Through transcriptional activation, saRNA therapeutics promise a revolution in our ability to modulate previously undruggable targets.

DEVELOPMENT PROGRAMS

MTL-CEBPA

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 that is delivered into certain immune cells around the body including subpopulations of myeloid cells.

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 of myeloid cells and other cells types around the body. Myeloid cells are frequently dysregulated in the microenvironment of solid tumours and are understood to be an important resistance hurdle for many anti-cancer medicines. Activating CEBPA expression in the tumour microenvironment is hypothesised to improve the efficacy of cancer therapies in solid tumour malignancies.

MTL-CEBPA DRUG PRODUCT

OUTREACH CLINICAL TRIAL

OUTREACH is a multi-centre Phase Ib clinical study of MTL-CEBPA in combination with Sorafenib in patients with primary liver cancer. The study is assessing the safety, tolerability, pharmacology and anti-tumour activity of MTL-CEBPA in combination with Sorafenib. The study is ongoing in the United Kingdom, Singapore and Taiwan.

To learn more about the OUTREACH clinical trial, please visit ClinicalTrials.gov.

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. The majority of liver cancer patients have limited treatment options, with a five-year survival rate of less than 15%. In 2007 Sorafenib, a tyrosine kinase inhibitor, became the first approved therapy for advanced liver cancer and remains the frontline standard of care.

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