Zimura Compared to Sham in Patients With Autosomal Recessive Stargardt Disease (STGD1)

Inclusion Criteria:

- At least two pathogenic mutations of ABCA4 gene confirmed by a CLIA-certified
laboratory

- Best corrected visual acuity in the study eye between 20/20 - 20/200 Snellen
equivalent, inclusive

Exclusion Criteria:

- Macular atrophy secondary to any condition other than STGD1 in either eye

- Any prior treatment for STGD1 including gene therapy, stem cell therapy or any prior
intravitreal treatment for any indication in either eye

- Participation in an interventional study of a vitamin A derivative ≤ 3 months prior to
screening

- Presence of intraocular inflammation, macular hole, pathologic myopia, epiretinal
membrane, evidence of significant vitreo-macular traction, vitreous hemorrhage or
aphakia

- Any intraocular surgery or thermal laser within 3 months of trial entry. Any prior
thermal laser in the macular region

- Diabetes mellitus

- HbA1c value of ≥6.5%

- Stroke within 12 months of trial entry

- Any major surgical procedure within one month of trial entry or anticipated during the
trial

- Any treatment with an investigational agent in the past 60 days for any condition

- Women who are pregnant or nursing

- Known serious allergies to the fluorescein dye used in angiography, povidone iodine,
or to the components of the Zimura formulation