SkQ1: a cardiolipin peroxidation inhibitor

A few years ago a small molecule SkQ1 was synthesized in the Moscow State University. One part of SkQ1 functions as a molecular "tow truck", carrying the other part of the molecule – an extremely 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 incredibly high precision. The molecule was designed to act as a mitochondria-targeted ROS scavenger in general and as a very efficient cardiolipin peroxidation inhibitor in particular

Why is ROS scavenging in mitochondria important?

The presence of SkQ1 in mitochondrial membrane enables mitochondria to protect itself from reactive oxygen species (ROS) by breaking the chain reaction of lipid destruction. ROS have been shown to cause damage in mitochondrial membrane leading to mitochondrial dysfunction, they can also induce apoptosis. At the same time ROS have been linked to tissue injury during inflammation and to uncontrolled progression of inflammation. That's why ability of our lead molecule to protect cells against oxidative stress can become a solution against various age-related disorders such as autoimmune conditions. cardiovascular diseases, neurodegenerative disorders and ophthalmic conditions. SkQ1 is also well-positioned to help patients suffering from several rare genetic disorders resulting in mitochondrial dysfunction.

SkQ1 and the challenge of drug development

Developing methods for effective delivery of mitochondria-targeted antioxidants into the organism is another challenging task. Mitotech successfully solved this complex problem for a variety of therapeutic areas and designed several SkQ1-based pharmaceutical products currently going through various stages of clinical development. Visomitin™ is an eye drop formulation of SkQ1 that has been developed to target ophthalmic disorders like dry eye syndrome, uveitis and 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 Russia that showed no side effects for regimen and dose intended for use in further human studies. For instance, during clinical trials in dry eye patients SkQ1 ophthalmic solution demonstrated levels of safety and comfort that are not different from those of an artificial tear.

Mitotech is determined to keep adding therapeutic indications that are important for millions of patients worldwide as well as rare genetic disorders to its development pipeline to make sure the pharmaceutical potential of SkQ1 technology is realized to the fullest extent.

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