MiNA Therapeutics Announces AASLD Liver Meeting Presentation On Lead Program Demonstrating Reversal Of Liver Failure In Animal Model

LONDON--(BUSINESS WIRE)--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 several markers of liver function in a rat model of liver failure. MTL-CEBPA is the first development candidate to emerge from MiNA’s RNA activation platform and the company is advancing the compound into clinical investigation in 2016 with an initial focus on patients with liver cancer and impaired liver function.

“By restoring endogenous expression of C/EBP-a, MTL-CEBPA achieved reversal and near normalisation of multiple indicators of liver failure after just two weeks of treatment”

The data were presented at the 2015 American Association for the Study of Liver Diseases (AASLD) Liver Meeting on November 16 in San Francisco. The oral presentation was titled, “Systemic administration of a novel development candidate, MTL-CEBPA, up-regulates the liver enriched transcription factor C/EBP-a, and reverses CCl4-induced liver failure in vivo”.

“By restoring endogenous expression of C/EBP-a, MTL-CEBPA achieved reversal and near normalisation of multiple indicators of liver failure after just two weeks of treatment,” commented Robert Habib, CEO of MiNA Therapeutics. “This data illustrates the potential for MTL-CEBPA to treat diseases complicated by liver failure, including liver cancer, liver cirrhosis and non-alcoholic steatohepatitis (NASH).”

MTL-CEBPA is a SMARTICLES^® liposomal formulation of a short activating RNA targeting the CEBPA gene. In the experiments covered by the presentation, MTL-CEBPA was administered systemically for two weeks in male Sprague Dawley rats with CCl4-induced liver failure. In the treated animals, MTL-CEBPA restored CEBPA mRNA levels as compared to normal and negative vehicle controls. The CEBPA gene encodes CCAAT/enhancer binding protein alpha (C/EBP-a), a transcription factor that plays an important role in normal hepatocyte function and response to injury. The treatment regime further reduced fibrosis and fatty infiltration as well as normalised levels of several important liver function markers, such as serum bilirubin, serum transaminase levels and coagulation time. In addition, treatment also led to an increase in the treated animals’ body weight and attenuated hyper-ammonaemia, a marker of impaired liver detoxification.

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.