Mitotherapeutix Announces Positive Results from Pre-Clinical Liver Biopsy Study in Cynomolgus Monkeys

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siRNA Drug Candidates Suppress Novel NASH Target MCJ

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Mitotherapeutix LLC (MITO), a pre-clinical stage company focused on siRNA drug discovery and development based on suppression of the mitochondrial regulator MCJ, today announced that it has successfully completed an in vivo chronic study in cynomolgus monkeys to suppress expression of MCJ, a new and novel target that regulates mitochondrial metabolism. The results of the study showed steady suppression in MCJ expression using a GalNAc conjugated siRNA. The GalNAc technology to deliver the siRNA to the liver as well as the nucleotide sequences were developed and discovered by MITO. The level of MCJ suppression at four weeks was approximately 70%, consistent with the standard set by other siRNA candidates in development as well as FDA approved.

“Demonstrating significant efficacy in non-human primates is an important milestone in siRNA drug discovery and development,” said Jack Talley, Chief Executive Officer, MITO. “We developed the GalNAc delivery platform and siRNA targeted MCJ in-house and are delighted with the outcome of this successful study as a new and novel means to potentially treat NASH. There is a significant unmet medical need in this area that we hope to fill. Based on our assessment of the data, we intend to meet with the FDA for a pre-submission meeting early next year.”

The Company is seeking funding by way of government grants, commercial partnerships or the sale of equity in order to advance the MCJ platform forward.

About NASH:
The liver is an organ which detoxifies various metabolites, synthesizes proteins and produces biochemicals necessary for digestion and growth. The normal liver does not store fat, but fat can be found in individuals that have Nonalcoholic Fatty Liver Disease (NAFLD). If there is hepatitis (inflammation) in addition to fat accumulation, then the disease is recognized as Nonalcoholic Steatohepatitis (NASH). According to the National Institute of Diabetes and Digestive and Kidney Diseases (NDDK) as many as 30% to 40% of Americans have NAFLD and about 3% to 12% have NASH. Dietary changes and weight loss can be of major help in the treatment of NASH but occurs in only a small number of individuals. There is major activity in drug discovery and development for NAFLD and NASH. Thus far no drug has been approved. We believe we may have such a drug. Our approach is enhancing the metabolic activity in the mitochondria. We do this by enhancing the electron transport chain (ETC) in mitochondria and have demonstrated the efficacy of this approach in several animal models that reduce liver fat.

About Mitotherapeutix LLC:
Mitotherapeutix is a US-based pre-clinical stage pharmaceutical company that is developing drugs (siRNA) targeting metabolism to treat disease. Our initial program targets a protein MCJ/DNAJC15, found in the mitochondria and is responsible for enhancing the production of energy. MCJ is unusual because it is a negative regulator of metabolism and knocking out MCJ enhances the electron transport chain (ETC) and the production of ATP without increasing reactive oxygen species (ROS). Enhancing the production of energy corrects the imbalance of fat, inflammation and fibrosis in the liver. MCJ is also present in other tissues besides the liver including the kidney and the heart. For more information, please visit

MITO Disclosure Notice: This press release contains “forward-looking statements” as that term is defined in the Private Securities Litigation Reform Act of 1995. For this purpose, any statements contained herein, or which are otherwise made by or on behalf of the Company that are not statements of historical facts may be deemed forward looking statements. Without limiting the generality of the foregoing, words such as “may,” “will,” “to,” “plan,” “expect,” “believe,” “anticipate,” “intend,” “could,” “should,” “would,” “estimate,” or “continue,” or the negative or other variations thereof or comparable terminology are intended to identify forward looking statements. Investors are cautioned that all forward-looking statements involve risk and uncertainties which may cause results to differ materially from those set forth in the statements. Such risks and uncertainties include, but are not limited to the following: the success of research and development activities and the speed with which regulatory authorizations and product launches may be achieved; government regulation generally; competitive developments; the ability to successfully market products domestically and internationally; difficulties or delays in manufacturing or issues relating to manufacturing capacity; commercial obstacles to the successful introduction of brand products generally; legal defense costs, insurance expenses, settlement costs, and the risk of an adverse decision or settlement relating to product liability, patent protection, governmental investigations, and other legal proceedings; the Company’s ability to acquire and protect patents and other intellectual property both domestically and internationally; the absence of certainty regarding the receipt of required regulatory approval or the timing or terms of such approvals; any changes in business, political and economic conditions; business interruption due to hurricanes or other events outside of the Company’s control.

Investors are cautioned not to place reliance on these forward-looking statements, which are valid only as of the date they were made. The Company undertakes no obligation to update or revise any forward-looking statements to reflect new information or the occurrence of unanticipated events or otherwise, except as expressly required by law.

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Jack V. Talley
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