Our lead candidate - a small molecule SkQ1 - is a purposely designed cardiolipin peroxidation inhibitor. SkQ1 was developed to deliver a highly active antioxidant plastoquinone into mitochondria. SkQ1 has been shown to accumulate in mitochondria in an extremely targeted and efficient manner. The physics of the mitochondrial membrane and the unique properties of SkQ1 direct it into the inner leaflet of the inner mitochondrial membrane with an incredibly high precision. Once positioned in mitochondria, the molecule acts as a mitochondria-targeted ROS scavenger in general and as a very efficient cardiolipin protector in particular.
Why is ROS scavenging in mitochondria important?
The accumulation of SkQ1 in mitochondrial membrane breaks the chain reaction of lipid destruction. Mitochondrial ROS (mtROS) have been shown to cause damage in mitochondrial membrane leading to mitochondrial dysfunction and apoptosis. At the same time mtROS have been linked to uncontrolled progression of inflammation. Accumulating evidence suggests that mtROS contribute largely to the aging process as mitochondrial dysfunction due to oxidative stress is considered one of the contributors to aging. That is why ability of our lead molecule to protect cells against oxidative stress can become a solution against various age-related disorders such as autoimmune, cardiovascular, neurodegenerative and eye diseases. SkQ1 is also well-positioned to help patients suffering from several rare mitochondrial disorders.
SkQ1 and the challenge of drug development
Development of effective delivery methods for mitochondria-targeted antioxidants is another challenging task. Mitotech has successfully solved this complex problem for a variety of therapeutic areas and has designed several SkQ1-based pharmaceutical products currently going through various stages of clinical development: from pre-clinical to late-stage clinical. Visomitin® is an eye drop formulation of SkQ1 that has been developed to target ophthalmic disorders like dry eye syndrome, uveitis and age-related macular degeneration. Plastomitin™ is a systemic formulation engineered for treatment of neurodegenerative, cardiovascular and metabolic diseases. We also conducted a great volume of safety studies in our pre-clinical and clinical programs in the U.S. and demonstrated an excellent safety and tolerability profile of SkQ1.
We at Mitotech are determined to keep expanding our pipeline with therapeutic areas that are important for millions of patients worldwide, including both common age-related diseases and rare mitochondrial disorders.