Study of RG-012 in Male Subjects With Alport Syndrome

This is a randomized, double-blind, placebo-controlled, multi-center, Phase 2 study of RG-012
in male subjects with Alport syndrome. Eligible subjects will be randomized in a 1:1 ratio to
receive subcutaneous (SC) injections of RG-012 or placebo every other week for 48 weeks.
After completion of this double-blind treatment period, subjects will have the opportunity to
receive RG-012 in a 48 week open-label extension period.

Male subjects with a confirmed diagnosis of Alport syndrome and a baseline GFR between 40 and
90 mL/min/1.73m2 will be eligible for enrollment. Subjects may enter this study directly or
may enroll after participation in the RG012-01 ATHENA Natural History Study.

Subjects may continue treatment with angiotensin converting enzyme (ACE) inhibitors or
angiotensin II receptor blockers (ARBs), but must be on a stable dosing regimen for the 30
days prior to screening.

Inclusion Criteria:

1. Male, age 18 to 60 years (inclusive)

2. Confirmed diagnosis of Alport syndrome (clinical and genetic)

3. eGFR between 40 and 90 mL/min/1.73m2

4. Proteinuria ≥ 300 mg protein/g creatinine at screening

5. For subjects taking an ACE inhibitor and/or ARB, the dosing regimen should be stable
for at least 30 days prior to screening

Exclusion Criteria:

1. Causes of chronic kidney disease aside from Alport syndrome (such as diabetic
nephropathy, hypertensive nephropathy, lupus, or IgA nephropathy)

2. End stage renal disease (ESRD) as evidenced by ongoing dialysis therapy or a history
of renal transplantation

3. Weight > 110 kg (242.5 pounds)

4. Any other condition that may pose a risk to the subject's safety and well-being