Irving, Texas based Reata Pharmaceuticals, whose mission is to translate innovative research into breakthrough medicines for difficult-to-treat diseases that have significant unmet needs, recently announced the enrollment of their first patient into MOXIe, their Phase 2 clinical study examining the safety, tolerability, and efficacy of RTA 408 for the treatment of patients with Friedreich’s Ataxia (FA).
- Gene: is a unit of heredity that gives cells a map-like code to create (encode) the body’s proteins.
- Mitochondria: an important cellular structure that is oftentimes called “the powerhouse of the cell” because they generate most of the cell’s supply of energy (ATP).
About Friedreich’s Ataxia:
FA is a rare inherited disorder that is caused by defects in an important gene that encodes for the protein, frataxin. Frataxin is a protein that regulates the amount of iron in the mitochondria, and the defects caused by FA lead to iron overload and irreversible damage to the mitochondria’s DNA. Patients diagnosed with FA have a progressive degeneration of the central and peripheral nervous systems, impaired coordination and gait, and fatigue from energy deprivation and muscle loss. According to the National Institutes of Health (NIH), 20,000 people in the US and Europe have FA. There are currently no FDA approved therapies for FA.
Reata’s RTA 408 For the Treatment of FA:
RTA 408 works by inducing the activity of Nrf2, which regulates many genes that are responsible for the production of cellular energy (ATP) in mitochondria. Activation of Nrf2 increases the proper use of fuel by the mitochondria and has been shown to promote muscle repair and recovery and reduce markers of oxidative stress and muscle injury.
In a recent press release about the announcement, Dr. Colin Meyer MD, Reata’s Chief Medical Officer and Vice President of Product Development, stated, “Our large body of preclinical and clinical data with RTA 408 and related molecules provides strong support for the hypothesis being tested in MOXIe that RTA 408 may be beneficial in patients suffering from FA. We have been productively collaborating with key FA clinicians and patient advocacy, and we look forward to evaluating the results of MOXIe as they become available.”
MOXIe will include up to 52 FA patients that meet the following eligibility criteria:
- Between 18-40 years old
- Be willing to maintain a consistent exercise routine and stable medication doses throughout the study.
- Be willing to discontinue taking all antioxidant supplements and vitamins, or any other medication intended to treat FA, before beginning the study.
Patients who meet the eligibility criteria will be studied over a 5 month period that includes 12 weeks of treatment, with the primary study endpoint being peak work volume during exercise testing, as well as changes in measures of physical activity, fatigue, and biomarkers associated with mitochondrial functioning.
In a previous statement about the importance of this trial for FA patients, Dr. Meyer said, “Our collaborators and I have shown in preclinical studies that genetic or pharmacologic Nrf2 activation positively regulates mitochondrial function and energy production. We hope to translate this effect into improved physical functioning and reduced fatigue in patients with FA and mitochondrial myopathies. These rare, debilitating diseases currently have no approved therapies.”