Research and Development

NASH and cancer

Proof-of-concept studies of Mito Biopharma technology on NASH and type 2 diabetes have been validated in multiple mouse model systems. For testing efficacy on NASH, HFD (high-fat diet) model (for efficacy on steatosis), HFD and high cholesterol diet model (for efficacy on steatosis, inflammation, and fibrosis), and CCl4model (for efficacy on fibrosis) have been evaluated. For testing efficacy on type 2 diabetes, HFD and db/db mouse models have been used.

Showing excellent pharmacology, ADME, PK/PD, and toxicology profiles, MT-238 and MT-514 have been selected as IND leads NASH and cancer indications.


Medicinal Chemistry Research for Expanding Drug Candidate Pipeline

Mito Biopharma has developed a number of chemical classes with distinct pharmacodynamic properties, including their impact on oxygen consumption, mitochondrial membrane potential, ATP synthesis and ATP level, and plasma membrane potential. Moreover, Mito Biopharma has proprietary know-how in SAR and is producing a repertoire of leads in each PD group with diverse ADME, PK, toxicology properties. These leads are being tested and validated as potential leads for other indications, including HCC (hepatocellular carcinoma), metastatic colon cancer, metastatic pancreatic cancer, ALL (acute leukocytic leukemia), and AML (acute myeloid leukemia).


Biology Research

The biological research team has performed extensive proof-of-concept studies to validate the mechanism of action on the various indications in clinically relevant animal models. Below are the published example studies that demonstrated the effectiveness of the mechanism of action on type 2 diabetes and metastatic colon cancer. In addition, the biological research team is working closely with medicinal chemistry team to expand the drug candidate repertoire and testing new leads. Similar comprehensive efficacy studies have been conducted with various testing compounds to select leads in various indications, including NASH, type 2 diabetes, metastatic colon cancer, metastatic pancreatic cancer, and ALL. 


Publications (click to review)

Mild mitochondrial uncoupling improves diabetic symptoms in mice (Nature Medicine)

Mitochondrial uncouplers are efficacious in treating metastatic colon cancer in mice (Cell Death and Disease)

– Mitochondrial uncoupler reverses diabetes (Nature Drug Discovery Review)

– Anthelminthic drug for T2DM therapy? (Nature Endocrinology Review)