Therapeutic Focus

Mito Biopharma has a proprietary platform technology for developing small- molecule mitochondrial modulators (see Our Technology). The company has a strong focus on liver-based diseases which continue to represent a high unmet need. We have developed leads for the treatment of NASH (non-alcoholic steatohepatitis) and type 2 diabetes. Both diseases have a clear basis in hepatic accumulation of fat, which plays a causal role in type 2 diabetes and NASH development and progression. In addition, as a secondary priority, we are also developing liver-based orphan diseases related to metabolic imbalance and metabolic defect in liver.

Although NASH has been described over 35 years ago, it was only recent that the potential of viable treatments emerged. Yet the hurdle of meeting FDA guidelines on resolution of steatosis as well as impacting on liver fibrosis remains challenging despite many companies entering this therapeutic space. Mito Biopharma’s ability to compete in this challenging therapeutic area relates to our differentiating and patent-protected technology that dissipates lipid accumulation of liver cells as well as inhibits activation of hepatic stellate cell differentiation, which are critical causative factors of NASH and NASH progression.