A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia

Inclusion Criteria:

• mFARS ≥20 to ≤70 at baseline
• Must be able to ambulate at least 10 feet in 1 minute with or without assistance (non-wheelchair).
• Friedreich ataxia diagnosis (homozygous for guanine-adenine-adenine [GAA] repeatexpansion in intron-1 of frataxin [FXN] gene), confirmed by clinical testing (Note:size of GAA repeat is not required for eligibility)
• Consent to comply with study procedures. For participants under the age of 18 (or ageof consent), parent(s)/legal guardian(s) of the participant must agree to comply withthe requirements of the study, including the need for frequent and prolonged followup; parent(s)/legal guardian(s) with custody of the participant must give theirconsent for participant to enroll in the study.
• Difference in the mFARS at screening and baseline of no more than 4 points.
• Must be able to abstain from anticoagulants and any aspirin (including 81 mg) for 30days prior to the baseline visit and for the duration of the study; any possiblediscontinuation of anticoagulants should be monitored and indicated by a specialist (for example, cardiologist, neurologist, or hematologist) and discontinuation will benoted by the prescribing physician.
• Must be able to abstain from potent cytochrome P450 (CYP) 3A4 inducers/inhibitors (forexample, ketoconazole, rifampin, St. John's wort, grapefruit juice or any grapefruitproduct) for at least 30 days prior to enrollment
• Must be able to swallow capsules
• Males and females of childbearing potential must be willing to use an effective methodof contraception from the time consent is signed until 30 days after the last dose ofstudy drug or early termination visit. Male participants must agree not to donatesperm during the study and for at least 30 days after the last dose of study drug orearly termination visit.

Exclusion Criteria:

• Individuals with clinical diagnosis of FA who have point mutations or deletions orother non-GAA expansion mutations
• Previous treatment with vatiquinone
• Allergy to vatiquinone, sesame oil, gelatin (bovine and/or porcine), titanium dioxide,or red iron oxide
• Ejection fraction <50%
• Uncontrolled diabetes (glycated hemoglobin [HbA1c] >7.0%) at the time of screening
• Has current suicidal ideation based on Columbia-Suicide Severity Rating Scale (C-SSRS)within 3 months prior to screening or between screening and baseline at the baselinevisit or suicidal behavior within the last year at the screening visit or betweenscreening and baseline at the baseline visit
• Pregnant or lactating participants or those sexually active participants who areunwilling to comply with proper birth control methods; females of childbearingpotential must have a negative pregnancy test at screening and during the baselinevisit
• Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) ≥2 * upper limit ofnormal (ULN) at time of screening
• International normalized ratio (INR) ≥1.5 * ULN at time of screening or clinicallysignificant (CS) bleeding, as determined by the investigator
• Serum creatinine ≥1.5 * ULN at time of screening
• Comorbidities that may confound study results (for example, fat malabsorptionsyndrome, other mitochondrial disorder) in the opinion of the investigator
• Participation in any other interventional clinical trial or received anyinvestigational drug in any other clinical trial within 60 days prior to the baselinevisit. Participants may be rescreened after the exclusionary period of 60 days haspassed.
• Concomitant use of interventional coenzyme Q10 (CoQ10), vitamin E, or any approved ornon-approved medication for FA within 30 days prior to the screening visit. Theseprohibited medications can be discontinued at the screening visit; if this is thecase, the mFARS assessment must be repeated to confirm inclusion eligibility after aminimum of 30 days post-discontinuation and there must be no more than a 4-pointdifference in mFARS assessed from the post-discontinuation visit to the baselinevisit.
• Illicit drug use 30 days prior to screening and during the study is prohibited.