Recent research has highlighted mitochondrial stress and endoplasmic reticulum (ER) stress as key mediators of both the nerve cell death and neuroinflammatory processes. The mitochondrion is the energy production center of the cell, while the ER is the quality control center. These two organelles are in constant communication, and are in fact physically connected by a membrane, and their health is vital to cell survival. When either of these cellular processes go awry, the resulting stress can either kill the cell and/or create inflammation. The brain is extremely sensitive to both mitochondrial stress and ER stress, and both of these pathways have been strongly implicated in causing neurodegenerative disease. We believe that only therapeutically targeting both organelles simultaneously will enact a significant and lasting benefit.
AMX0035 is a proprietary combination of two compounds, dosed in a particular synergistic ratio to simultaneously block mitochondrial and ER stress. Both compounds have been used clinically. We have shown that while each component has some efficacy, the two components together are synergistically more effective. AMX0035 has demonstrated strong efficacy in blocking nerve cell death and neuroinflammation in disease-specific models of ALS, Alzheimer’s, and mitochondrial diseases, as well as non-specific models of nerve cell death and inflammation. These studies have all been performed by industry-leading Contract Research Organizations or at top academic institutions.