A First in Human Study of RT001 in Patients With Friedreich's Ataxia

Study RT001-002 is a randomized, double-blind, controlled, ascending dose study to evaluate
the safety, tolerability, pharmacokinetic, disease state, and exploratory endpoints in
patients with Friedreich's ataxia after oral administration. The study includes 2 dose
levels of RT001.

Inclusion Criteria:

1. Male or female 18 to 50 years of age

2. Medical history consistent with the symptoms of FRDA at ≤ 25 years of age

3. Homozygous for GAA repeat expansions in the Frataxin gene in the affected range for
FRDA

4. FARS-Neurological score of 20-90 points

5. Ambulatory (with or without assistive device) and capable of performing
assessments/evaluations

6. Body Mass Index ≤ 29.9 kg/m2

7. Agrees to dietary restrictions and agrees to receive calls from a dietary coach

8. Signed the informed consent form prior to entry into the study

9. Agrees to spend the required number of overnight clinic days

10. Able to provide the necessary repeated blood samples

Exclusion Criteria:

1. Received treatment with other experimental therapies within the last 30 days prior to
the first dose

2. Known point mutation in the FXN gene

3. History of malignancies (other than basal cell carcinomas)

4. Impaired renal function at screening

5. Alanine transaminase (ALT) or aspartate transaminase (AST) laboratory values > 2 x
upper limit of normal (ULN) at screening

6. Known hepatitis B surface antigen (HBsAg)-positive, or known or suspected active
hepatitis C infection, or is known to be human immunodeficiency virus (HIV) positive

7. Female who is breastfeeding or has a positive pregnancy test

8. Male participant or female participant of child bearing potential, who is sexually
active and unwilling/unable to use a medically acceptable and effective double
barrier birth control method throughout the study

9. Unwilling or unable to comply with the requirements of the protocol

10. Clinically significant cardiac abnormalities at screening that, in the opinion of the
Investigator, would make the patient unsuitable for enrollment

11. Diabetes mellitus (Type 1 or 2)

12. Suicidal ideation as determined by the Columbia-Suicide Severity Rating Scale

13. History, within the last 2 years, of alcohol abuse, significant mental illness, or
physical opioid dependence

14. Cannot adhere to the dietary guidance required to be followed by the protocol

15. Cannot take the medication due to impairment in swallowing capsules