Multiple Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia

Exclusion Criteria

• •1. Subjects who had a serious adverse event (SAE), an adverse event (AE) that is Grade 3 or higher according to the Common Terminology Criteria for Adverse Events (CTCAE), version 5.0 (or higher), or an AE considered clinically significant during participation in CLIN-1601-101 (NCT04176991).
• •2. Subjects who are confirmed as compound heterozygous (GAA repeat expansion on only one allele) for Friedreich's ataxia.
• •3. Subject use of investigational drug (other than CTI-1601) or device within 90 days prior to screening.
• •4. Subject requires use of amiodarone.
• •5. Subject used erythropoietin, etravirine, or gamma interferon within 3 months prior to screening.
• •6. Subject use of daily biotin supplementation that exceeds 30 mcg/day, either as part of a multivitamin or as a standalone supplement, within 7 days prior to study drug administration and/or throughout the entire study.
• •7. Subject has clinically significant arrhythmia on electrocardiogram (ECG), or evidence of predisposition to significant ventricular arrhythmia on ECG, or evidence of active and unstable coronary artery disease.
• •8. Male subject who has a QT interval corrected for heart rate using Fridericia's formula (QTcF) \> 450 milliseconds or female subject who has a QTcF \> 470 milliseconds on an ECG.
• •9. Subject has a screening echocardiogram left ventricular ejection fraction \< 45 percent.
• •10. Subject has a history of aspiration, aspiration pneumonia, or recurrent episodes of pneumonia (greater than or equal to 2 episodes of pneumonia) within the last 12 months.