Please tell us about your clinical experience. How many patients with friedreich's ataxia do you see on a yearly basis? What is the standard of care and what treatments are available?How much of a need is there for novel treatments ? Where do you see omaveloxolone fitting in this space? What other upcoming therapies are you excited about?

Can you tell us more about the role of mitochondrial protein frataxin in this disease, and how treatments aim to address it?

How often do FA patients experience visual impairment, hearing loss, diabetes, or cardiomyopathy?

Could you please discuss Nrf2 activators and their mechanism of action, restoring mitochondrial function, reducing oxidative stress, and inhibiting pro-inflammatory signaling?

Could you touch on the potential mechanisms of Nrf2-mediated myocardial damage and dysfunction?Alsom ow much truth is in the statement that “Nrf2 activators” are actually “Keap1 inhibitors” ?

Please tell us your opinions on the available stuy results, where:
-In total, 97% (73 of 75) of patients in the Full Analysis Set who completed MOXIe Part 2 enrolled in the extension study, of which 39 were previously randomized to placebo and 34 were previously randomized to omaveloxolone. Similar annualized mFARS slopes were observed between the placebo to omaveloxolone (0.29 points [±0.68]) groups and omaveloxolone to omaveloxolone groups (0.17 points [±0.61]); however, both slopes were less than the expected gain of 1.9 points per year observed in natural history data. After nearly 2 and a half years of treatment, investigators noticed that patients previously randomized to omaveloxolone in MOXIe Part 2 continued to show no worsening in mFARS relative to their original baseline.
-Adverse events (AEs) recorded were generally mild to moderate in intensity. In total, 8 patients (5.4%) discontinued study due to AEs. ALT increase (n = 26; 18%), headache (n = 22; 15%), upper respiratory tract infection (n = 23; 15%), nausea (n = 22; 15%), fatigue (n = 15; 10%), and abdominal pain (n = 15; 10%) were the AEs documented. In total, 9 patients (6.1%) reported serious AEs that were considered treatment-emergent; however, no new safety signals were observed in the extension study to date.Original topline results from MOXIe published in 2019 showed a statistically significant 2.40-point improvement in mFARS score for those treated with the investigational agent compared with placebo (P = .014).2 A mean improvement of 1.55 points in mFARS score from baseline was recorded in the treatment group, while patients treated with placebo experienced a mean worsening of +0.85 points. When patients without pes cavus were included in the analysis (n = 103), a statistically significant mean 1.93-point improvement on mFARS was recorded compared with placebo (P = .034).