EPI-743 in Friedreich's Ataxia Point Mutations

The primary objective of this study is to investigate whether treatment with EPI-743 has a
discernible impact on visual function—including visual acuity, visual fields and color
vision as well as on any of a number of functional and subject/clinician-rated scales
relevant in the treatment of Friedreich's ataxia, and to determine the safety of treatment
with EPI-743.

Inclusion Criteria:

1. Diagnosis of genetically confirmed Friedreich's ataxia point mutation.

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

3. FARS score of 20 to 90.

4. Male or female between 18 and 65 years of age.

5. Agreement to use contraception if within reproductive years

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

7. Willingness and ability to comply with study procedures.

8. Willingness and ability to arrive at study site metropolitan area day prior to
evaluations.

9. 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.

10. Abstention from foods or beverages or bars fortified with Coenzyme Q10, vitamin E,
super fortified functional foods or beverages at least 30 days prior to initiation of
treatment and for the duration of the study.

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

12. Subject can swallow multiple size 0 capsules.

13. Subject has voluntarily signed an IRB approved informed consent form to participate
in the study after all relevant aspects of the study have been explained and
discussed with the subject.

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

9. Clinically significant arrhythmia within past two years requiring treatment.

10. Surgery planned through the duration of the study, including follow-up.

11. Pregnancy or breastfeeding.

12. Anticoagulant therapy within 30 days of enrollment.