Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease

The primary objective of this prospective, multicenter, double-blind, randomized,
placebo-controlled, parallel group, Phase 3 study is to determine the effect of oral
lucerastat monotherapy on neuropathic pain in subjects with Fabry disease (FD) through daily
collection of patient-reported outcomes with an electronic diary.

Inclusion Criteria:

1. Signed and dated ICF prior to any study-mandated procedure;

2. Male or female adult subjects;

3. FD diagnosis confirmed with local genetic test results;

4. Fabry-associated neuropathic pain, as defined by the subject, in the last 3 months
prior to screening;

5. Enzyme replacement therapy (ERT) status:

1. Subject never treated with ERT; or

2. Subject has not received ERT for at least 6 months prior to screening; or

3. Subject treated with ERT since at least 12 months at the time of the screening
visit, and agreeing to stop ERT for approximately 8 months.

6. A woman of childbearing potential is eligible only under certain conditions, e.g.
taking contraceptive measures.

7. Subjects with moderate or severe neuropathic pain during the screening period.

Exclusion Criteria:

1. Pregnant, planning to be become pregnant, or lactating subject.

2. Severe renal insufficiency (eGFR < 30 mL/min/1.73 m2) at screening.

3. Subject on regular dialysis for the treatment of chronic kidney disease.

4. Known and documented transient ischemic attack, stroke, unstable angina, or myocardial
infarction within 6 months prior to screening.

5. Clinically significant unstable cardiac disease (e.g. uncontrolled symptomatic
arrhythmia, congestive heart failure NYHA class III or IV).

6. Any known factor or disease that might interfere with treatment compliance, study
conduct or interpretation of the results.