Reata Enrolls First Patient in RTA 408 Trial for Mitochondrial Myopathies

Margarida Azevedo, MSc avatar

by Margarida Azevedo, MSc |

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Irving, Texas-based Reata Pharmaceuticals has just announced the enrollment of the first patient in the company’s Phase II MOTOR clinical trial. This study will seek to determine the safety, tolerability and efficacy of Reata’s experimental RTA 408 Oral Capsules as a potential treatment for mitochondrial myopathies (MM).

RTA-408 works to improve mitochondrial function by stimulating the body’s innate anti-oxidative pathways with transcription factor Nrf2. In previous studies on mice models of MM, the drug showed an ability to boost glucose uptake, fatty acid oxidation, and oxygen utilization — all signs of improved cellular metabolism.

The MOTOR trial is being conducted at multiple study sites throughout the United States and plans to enroll about 52 patients diagnosed with MM, a group of rare diseases caused by abnormalities in mitochondrial DNA. Because of these mutations, patients experience respiratory chain deficits and reduced energy production, which can lead to a wide range of health issues, including muscle weakness, fatigue and disability in different organ systems. Many mitochondrial myopathies also lead to shortened lifespan. Today, about 20,000 Americans are estimated to have some type of MM, but there are still no approved treatments for it.

MOTOR will be evaluating RTA 408’s primary efficacy endpoint according to a change in peak workload (Watts/kg) during physical activity. As for the secondary endpoint, evaluation will be done according to patients’ performance of a 6-minute walk distance. Additionally, MOTOR will be analyzing changes in peak oxygen utilization during full exertion, as well as in the Fatigue Severity Scale.

“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,” explained Dr. Colin Meyer, the company’s Chief Medical Officer.  “We are hopeful that RTA 408 will benefit mitochondrial myopathy patients, and we appreciate the guidance and support that Reata has received from the UMDF and the MM patient community.”

“We are very excited this trial will be underway with its first patient,” said Charles A. Mohan, Jr., Executive Director and CEO of the United Mitochondrial Disease Foundation.  “Coordination, communication and collaboration between our industry partners and our patients to promote and support clinical trials is the only way we will accelerate the development of diagnostic tools, therapies, and potential cures for mitochondrial disease.  We are pleased with the role Reata has taken in this endeavor and honored to call them a partner.  We must all remember; no patients no trials, no trials no treatments nor cures.”

To learn more about MOTOR, visit