Reata Pharmaceuticals, Inc., a company based in Irving, Texas, recently announced the enrollment of the first patient in the company’s Phase 2 MOTOR clinical trial to assess the safety, tolerability and efficacy of its experimental RTA 408 Oral Capsules as a therapy for patients with mitochondrial myopathies.
Mitochondria are the energy factories within cells and their dysfunction can lead to impairment in cellular energy metabolism. Mitochondrial myopathies are a group of inherited diseases that result from a defect in mitochondrial genes and that can cause impaired energy production, muscle weakness, fatigue and disability in different organ systems. Patients with these disorders often have reduced lifespans. It is estimated that around 20,000 individuals in the United States are affected by a mitochondrial myopathy, and there are currently no approved therapies for any of these disorders.
RTA 408 is a synthetic oleanane triterpenoid compound with anti-oxidative and anti-inflammatory properties. It is able to induce the body’s anti-oxidative pathways through the activation of Nrf2 and inhibit the pro-inflammatory factor NK-kappaB, leading to an increase in the production of several antioxidants, anti-inflammatory genes and detoxification. RTA 408 can also improve mitochondrial function and consequently muscle function. Studies in animal models have shown that RTA 408 can promote a healthier cellular metabolism.
“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 Reata’s Chief Medical Officer Dr. Colin Meyer in a press release. “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 [United Mitochondrial Disease Foundation] and the MM [mitochondrial myopathy] patient community.”
The multi-center Phase 2 MOTOR trial (NCT02255422) is expected to enroll 52 patients with mitochondrial myopathies. Patients will receive either a placebo drug or RTA 408 oral capsules at 2.5 mg, 5 mg and 10 mg dose once daily for 12 weeks. The trial’s primary efficacy endpoint is the change in peak workload (Watts/kg) during exercise, and the secondary endpoint a change in the 6-minute walk distance test. Changes in peak oxygen use during maximal exercise testing and in Fatigue Severity Scale will also be assessed.
“We are very excited this trial will be underway with its first patient,” said the Executive Director and CEO of the UMDF Charles A. Mohan, Jr. “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.”
Reata is currently recruiting participants for the MOTOR clinical trial. If you are interested in participating or want to know more about it, please visit this link or the list of contacts and locations.