MSDC-0160 is a novel mTOT Modulator being developed to treat neurodegenerative diseases such as Alzheimer’s and Parkinson’s disease. It is a once-a-day, oral insulin sensitizer that works through a new drug target located in the inner mitochondrial membrane, called mTOT. The mTOT mechanism ties mitochondrial metabolism to aspects of the pathology associated with insulin resistance and certain neurodegenerative diseases.

Alzheimer’s Disease

MSDC-0160 recently completed a successful Phase 2a study conducted by researchers at Rush University Medical Center in Chicago  in patients diagnosed with dementia due to Alzheimer’s disease. In this study, MSDC-0160 prevented the decline in a measure of brain glucose utilization in patients with mild to moderate Alzheimer’s disease.  (Current Alzheimer Research, 2014, 11.)  

Parkinson’s Disease

In research conducted in collaboration with Patrik Brundin, MD, PhD, Head of the Laboratory for Translational Parkinson’s Disease Research at Van Andel Institute (Grand Rapids, Michigan), MSDC-0160 has demonstrated significant neuroprotective effects in the En1+/- mouse model of Parkinson’s disease via modulation of the mTOR-autophagy signaling cascade.   

Type 2 Diabetes

MSDC-0160 was originally developed as a potential drug to treat type 2 diabetes. A 90-day, randomized, double-blind, comparator- and placebo-controlled, multi-dose Phase 2b clinical study in 258 patients with type 2 diabetes was completed in December 2011. Data from this study was published in Clinical Pharmacology & Therapeutics (advance online publication 6 March 2013).