Reata Pharmaceuticals has enrolled the first patient in its Phase II dose-ranging MOTOR study, which will evaluate the safety, tolerability and efficacy of RTA 408 Oral Capsules to treat patients with mitochondrial myopathies (MM).
In the trial, the company plans to recruit approximately 52 patients with MM, which is a collective term for a group of individual rare diseases associated with mitochondrial DNA mutations.
MM will cause respiratory chain deficits and impaired energy production and most patients with these defects share a similar phenotype with skeletal muscle weakness and fatigue.
RTA 408 is said to activate the body's anti-oxidative pathways through transcription factor Nrf2 and it can improve mitochondrial function.
Reata Pharmaceuticals chief medical officer Dr Colin Meyer said: "Emerging translational research demonstrates that activation of Nrf2 (the target of RTA 408) can improve mitochondrial function and cellular energy production.
"These observations underlie our hypothesis that RTA 408 may improve exercise capacity and quality of life in patients with mitochondrial myopathies."
According to the firm, the primary efficacy endpoint of the trial is the change in peak workload (Watts / kg) during exercise testing, while the secondary points will include six-minute walk distance.
In addition, the study will explore the change in peak oxygen utilisation during maximal exercise testing and changes in the fatigue severity scale.
RTA 408 showed ability to increase glucose uptake, fatty acid oxidation and oxygen consumption and direct signs of healthier cellular metabolism in mouse models of bioenergetic disease.