Mito research around the world


What is Omaveloxone?

Omaveloxone is a drug currently being developed by a U.S based pharmaceutical company called Reata Pharmaceuticals. It is a capsule that is taken by mouth.

How does it work?

The drug works by activating Nrf2, a protein that triggers the expression of genes involved in mitochondrial function. Activation of Nrf2 also protects against tissue damage caused by injury or inflammation.

What is it supposed to do?

Pre-clinical studies have shown that omaveloxone can protect cells against oxidative stress. This can occur when there are high levels of harmful chemicals, known as free radicals, within a cell. Oxidative stress can cause considerable damage to cells and may contribute to some of the symptoms of Mitochondrial Disease. Omaveloxone may also work by increasing the amount of energy the mitochondria can produce and increasing mitochondrial biogenesis (the production of new mitochondria). It is hoped that this will improve muscle function in patients by increasing the number of healthy mitochondria and overall energy production.

Is it a cure?

Unfortunately not. Omaveloxone cannot correct the underlying genetic defect that causes Mitochondrial Disease.

What types of Mitochondrial Disease could it treat?

It is currently being trialed in patients with a diagnosis of Mitochondrial Disease and mitochondrial myopathy (muscle weakness).

Have there been any trials of Omaveloxone?

There is currently one clinical trial taking place across the USA and Denmark, known as the MOTOR study. This is a two-part phase 2 study that aims to evaluate the safety and efficacy of omaveloxolone in 100 patients with mitochondrial myopathy. The first part of the trial is ongoing to assess the safety of omaveloxolone at various doses over a 12-week period. Results are expected in late 2017.

What next?

If the results from the first part of the MOTOR study are positive, Reata expects to initiate the second part of the clinical trial. This will evaluate the safety, efficacy and pharmacodynamics (how the drug affects the body) of omaveloxolone at two different doses over 12 weeks. It is hoped that this will provide additional data to support the use of omaveloxone in patients with mitochondrial myopathy.

Is there more information?

More information can be found using the following links:


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