Published 30 January 2015
Reata announces enrollment of the first patient in a Phase 2 dose-ranging study examining the safety, tolerability, and efficacy of RTA 408 Oral Capsules versus placebo for the treatment of patients with Friedreich's ataxia.
MOXIe (A two-part, randomized, placebo-controlled Phase 2/3 Study of the Safety, Efficacy, and Pharmacodynamics of RTA 408 in the Treatment of Friedreich's Ataxia) is a multi-center study in approximately 52 patients and is designed to support a potential NDA submission. Part 1 of the study is a randomized, placebo-controlled, double-blind, dose-escalation study to evaluate the safety of RTA 408 at 2.5 mg, 5 mg, and 10 mg in patients with Friedreich's ataxia.
Part 2 of the study is a randomized, placebo-controlled, double-blind, parallel-group study to evaluate the safety, efficacy, and pharmacodynamics of up to 2 dose levels of RTA 408. The study is designed to evaluate a variety of clinical, and biochemical endpoints, including exercise capacity and quality of life measures.
Friedreich's ataxia (FA) is an inherited disorder caused by defects in the gene for frataxin, a protein that regulates iron levels in the mitochondria. Defects in frataxin result in mitochondrial iron overload, causing impaired metabolism, oxidative stress, and damage to mitochondrial DNA.
Patients with FA suffer progressive degeneration of the central and peripheral nervous systems, impaired motion and gait, and eventually may develop cardiomyopathy and diabetes. FA patients have substantially lowered quality of life, with most becoming wheelchair bound by their mid-20s and surviving only into their 30s. Approximately 20,000 people in the US and Europe have FA. There are currently no approved therapies for FA.
"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 Friedreich's ataxia," noted Dr. Colin Meyer, Reata's Chief Medical Officer.
"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."
Source: Company Press Release
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Reata enrolls first patient in Phase II/III MOXIe study of RTA 408 in friedreich's ataxia patients