Research and Development

Development of Mito-99-0103 and Mito-99-0053 for type 2 diabetes and NASH respectively

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.

Mito-99-0103 and Mito-99-0053, exhibited excellent pharmacology, ADME, PK/PD, and toxicology profiles, and have been selected as potential IND leads for type 2 diabetes and NASH respectively.

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 as backup compounds for NASH and type 2 diabetes indications, as well as for potential leads for other indications.

Biology Research

The biological research team is working closely with medicinal chemistry team to expand the drug candidate repertoire. In addition, the biological team is testing the efficacy of some of the lead compounds on other indications, such as metastatic tumors, in rodent models.

– Niclosamide ethanolamine–induced mild mitochondrial uncoupling improves diabetic symptoms in mice

– Mitochondrial uncoupler reverses diabetes

– Antihelminthic drug for T2DM therapy?