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Press Release

MiNA Therapeutics Presents Data Demonstrating Survival Benefit of MTL-CEBPA in Animal Model of Liver Failure at AASLD Liver Meeting

MiNA Therapeutics
Posted on: 14 Nov 16

MiNA Therapeutics, the pioneer in RNA activation therapeutics, today announced the presentation of pre-clinical data on its MTL-CEBPA program in which the compound was shown to improve survival in a rat model of liver failure. MTL-CEBPA is the first development candidate to emerge from MiNA’s RNA activation platform and is currently being evaluated in a Phase I clinical study in patients with liver cancer.

The data were presented at the 2016 American Association for the Study of Liver Diseases (AASLD) Liver Meeting on November 14 in Boston, USA in a poster titled “The clinical candidate MTL-CEBPA leads to significant reduction in ascites and improvement in overall survival in a CCl4-induced liver failure model”. MTL-CEBPA is a SMARTICLES® liposomal formulation of a small activating RNA targeting the CEBPA gene.

“The clear and sustained survival benefit we see in the animal model of chronic liver failure illustrates the great potential of MTL-CEBPA to treat severe and late-stage liver diseases which represent significant medical burdens to both patients and society,” commented Robert Habib, CEO of MiNA Therapeutics. “Although the reported data are pre-clinical, we are currently in the clinic testing this compound in patients, many of whom have a background of liver disease. We intend to present the results of our Phase I study in 2017.”

In the experiments covered by the presentation, MTL-CEBPA was administered systemically for twelve weeks in male Sprague Dawley rats with CCl4-induced liver failure. In the treated animals, MTL-CEBPA attenuated hyper-ammonaemia and ameliorated ascites, two hallmarks of liver decompensation. These benefit were maintained in animals up to 14 weeks following completion of treatment. Treated animals demonstrated a very significantly improved survival rate compared to the control group at completion of the study.

The poster presented at the AASLD Liver Meeting is available on the Company’s website in the publication section under “Media”.


MTL-CEBPA consists of a double stranded RNA formulated into a SMARTICLES® liposomal nanoparticle and is designed to activate the CEBPA gene. By restoring CEBPA 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). MTL-CEBPA is currently under evaluation in OUTREACH, a first-in-human Phase I clinical study in patients with severe liver cancer. The multi-centre 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. To learn more about the OUTREACH clinical study, please visit our listing at

About MiNA Therapeutics

Harnessing the innate mechanism of gene activation, MiNA Therapeutics’ platform enables the development of new medicines that restore normal function to patients’ cells. We are applying our technology and clinical know-how to transform the therapy landscape of severe liver diseases. Our initial product candidate will achieve clinical proof of concept in 2017.

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Last updated on: 14/11/2016

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