Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia

Inclusion Criteria:

1. Diagnosis of genetically confirmed Friedreich's ataxia

2. Visual acuity at baseline more than 15 letters on EDTRS at four meters

3. FARS score of 20 to 90

4. Agreement to use contraception if within reproductive years (see specifics in section
D1, page 21)

5. Hormone replacement therapy, if used, must remain stable for the duration of the
study

6. Willingness and ability to comply with study procedures

7. Willingness and ability to arrive at study site day prior to evaluations

8. Abstention from use of dietary supplements and non-prescribed medications at least 30
days prior to initiation of treatment and for the duration of the study. This would
specifically include idebenone, Coenzyme Q10 and vitamin E

9. Abstention from use of other investigative or non-approved drugs within 30 days of
enrollment and for the duration of the study

Exclusion Criteria:

1. Allergy to EPI-743 or sesame oil or nuts

2. Clinically significant bleeding condition or abnormal PT/PTT INR (INR > two; PTT >
two-times normal)

3. Liver insufficiency with LFTs greater than three-times upper normal limit at
screening

4. Renal insufficiency with creatinine > 1.5 at screening

5. Fat malabsorption syndromes

6. Any other respiratory chain diseases of the mitochondria or inborn errors of
metabolism

7. Any other ophthalmologic conditions

8. History of alcohol or drug abuse

9. Clinically significant cardiomyopathy with ejection fraction < 40 percent at
screening

10. Clinically significant arrhythmia within past two years requiring treatment

11. Anticoagulant therapy within 30 days of enrollment.