OUTREACH – The first-in-human clinical study of a saRNA therapeutic

OUTREACH is a first-in-human Phase I clinical study of MTL-CEBPA, our lead saRNA therapeutic, in patients with liver cancer.

The multi-centre, dose-escalation Phase I study will assess the safety and tolerability of MTL-CEBPA in patients with advanced primary or metastatic liver cancer who are ineligible or resistant to standard therapies. MTL-CEBPA will initially be administered as an intravenous infusion once weekly for three weeks followed by one week of rest.

To learn more about the OUTREACH clinical trial, please visit ClinicalTrials.gov or contact us at clinicaltrials@minatx.com

MTL-CEBPA clinical candidate

MTL-CEBPA is the first small activating RNA (saRNA) therapeutic to be tested in a clinical study. Designed to activate the CEBPA gene, MTL-CEBPA comprises a double stranded RNA payload formulated inside a SMARTICLES® 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 liver, myeloid cells and adipose tissue. In the liver, C/EBP-α plays an important role in normal hepatocyte function, response to injury and inhibition of cancer development.

By restoring C/EBP-α mRNA and protein expression to normal levels, MTL-CEBPA has been demonstrated to attenuate or reverse liver disease in a range of pre-clinical studies including models of liver cancer, liver cirrhosis, non-alcoholic fatty liver disease (NAFLD) and non-alcoholic steatohepatitis (NASH). In the future we expect to initiate clinical trials of MTL-CEBPA in a number of diseases beyond liver cancer.

Preliminary Phase I clinical data presented at ASCO 2018
Pre-clinical mechanism of action published in Molecular Therapy
Pre-clinical liver cancer proof of concept published in Hepatology

Small activating RNA targeting CEBPA gene

Active ingredient

NOV340 SMARTICLES® liposomal nanoparticle



Drug Product

Primary liver cancer

Liver cancer is the second leading cause of cancer related deaths worldwide with approximately 745,000 deaths each year. The overwhelming majority of primary liver cancer (HCC) patients also suffer from an underlying liver disease such as alcoholic liver disease, non alcoholic fatty liver disease, Hepatitis C virus or Hepatitis B virus which can all progress to fibrosis and cirrhosis of the liver.

Abnormal liver function is a co-morbidity that independently predicts patient survival and reduces eligibility for standard of care therapy. Due to complicating liver diseases as well as late detection, the majority of liver cancer patients have limited treatment options and poor survival.