Dose-ranging Study of PRX-102 in Adult Fabry Disease Patients

Inclusion Criteria:

- Symptomatic adult Fabry patients (≥18 yrs)

- Males: plasma and/or leucocyte alpha galactosidase activity (by activity assay) less
than lower limit of normal (LLN in plasma=3.2 nmol/hr/ml, LLN in leucocytes=32
nmol/hr/mg/protein)

- Females: historical genetic test results consistent with Fabry mutations

- Globotriaosylceramide (Gb3) concentration in urine > 1.5 times upper normal limit

- Patients who have never received enzyme replacement therapy (ERT) in the past, or
patients who have not received ERT in the past 6 months and have a negative anti
alpha galactosidase antibody test

- eGFR ≥ 60 mL/min/1.73m2

- The patient signs informed consent

- Female patients and male patients whose co-partners are of child-bearing potential
agree to use a medically acceptable method of contraception, not including the rhythm
method

Exclusion Criteria:

- Participation in any trial of an investigational drug within 30 days prior to study
screening

- Chronic kidney disease stages 3-5 (CKD 3-5) (Appendix 7)

- History of dialysis or renal transplantation

- Angiotensin converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB)
therapy initiated or dose changed in the 4 weeks prior to screening

- Severe myocardial fibrosis by MRI (≥2 late-enhancement [LE] positive left ventricular
segments) (Weidemann et al. 2009)

- History of clinical stroke

- Pregnant or nursing

- Presence of HIV and/or HBsAg and/or Hepatitis C infections

- Known allergies to ERT

- Known allergy to Gadolinium based contrast agents

- Presence of any medical, emotional, behavioral or psychological condition that, in
the judgment of the Investigator and/or Medical Director, would interfere with the
patient's compliance with the requirements of the study