On March 9, 2018 MiNA Therapeutics, the pioneer in RNA activation therapeutics, reported the publication of pre-clinical data on its MTL-CEBPA program in which the compound was shown to promote disease reversal in several models of liver disease and to reduce tumour burden in a model of liver cancer. MTL-CEBPA consists of CEBPA-51 small activating RNAs encapsulated in SMARTICLES nanoparticles (Press release, , SEP 9, 2018, View Source [SID1234524616]). It 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 underscore the potential of this compound to address both severe liver disease indications as well as earlier disease stages to enhance chances of survival.
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"Gene activation of CEBPA using saRNA: Preclinical studies of the first in human saRNA drug candidate for liver cancer"
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"The combination of beneficial effects observed across a range of liver disease models supports a unique and promising role for MTL-CEBPA therapy in the treatment of liver diseases and liver cancer," commented Robert Habib, CEO of MiNA Therapeutics. "This exciting pre-clinical data highlights the potential of saRNAs to up-regulate gene expression and treat disease in radically new ways compared to conventional medicines."
In the publication, researchers investigated the potential benefits of MTL-CEBPA in a set of in vivo severe liver disease models representing advanced liver cirrhosis, non-alcoholic steatohepatitis (NASH) and liver cancer. Overall, MTL-CEBPA was shown to restore the expression of CEBPA, a master regulator of liver function. In the advanced liver cirrhosis model, MTL-CEBPA significantly reversed liver fibrosis and liver dysfunction and enhanced survival. In a model of NASH, MTL-CEBPA reversed liver steatosis. Improved liver function as well as a large reduction in tumour burden was also seen in a model of primary liver cancer. Together, these findings validate the beneficial role of up-regulating CEBPA expression in liver disease and liver cancer and are consistent with externally published data using genetic models of liver disease.
The publication titled "Gene activation of CEBPA using saRNA: Preclinical studies of the first in human saRNA drug candidate for liver cancer", was published in the latest issue of Oncogene by researchers at MiNA Therapeutics in collaboration with several well-regarded academic institutions including scientists at Imperial College London and National Taiwan University Hospital. The paper is available on the Company’s website in the publications section under "Media".