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Metformin, a naturally-occurring biguanide, is an FDA-approved high-safety profile diabetes drug that has been used extensively for decades. Researchers at Iowa State University and Medical College of Wisconsin synthesized Mito-Met, a mitochondria-targeted analog of metformin and then evaluated the preclinical efficacy of Mito-Met in cell culture and MitoPark animal models of Parkinson’s disease. It was demonstrated that Mito-Met improved motor deficits in MitoPark mice and that it was a neuroprotective agent that exceedingly qualifies for in-depth preclinical efficacy and toxicology evaluations for treating mitochondrial diseases including Parkinson’s disease.

• Increases the survival of dopaminergic neurons in cell culture and in a mouse model of Parkinson’s disease

• Based on FDA-approved low-toxicity drug

• Chemical modifications increased its affinity to the intended mitochondrial target

• Addresses a high value market

• Effective at very low doses

Human Therapeutics, Treatment of Parkinson’s disease