Study of EO2002 in Subjects With Corneal Edema Secondary to Corneal Endothelial Dysfunction

This study is designed to evaluate the safety and efficacy of EO2002, an investigational magnetic human corneal endothelial cell (mHCEC) therapy, in adults with corneal edema secondary to corneal endothelial dysfunction. Participants will be randomized to receive a single intracameral injection of one of two doses of EO2002 (150,000 or 500,000 cells) or placebo.

The study will compare the effects of EO2002 on visual acuity and corneal thickness, while also evaluating safety over a 52-week follow-up period. Approximately 105 participants will be treated at multiple sites in the United States.

• 150K cells (Biological): Intracameral injection - 150K dose
• 500K cells (Biological): Intracameral injection - 500K dose
• Placebo (Procedure): Intracameral injection of vehicle

Primary outcomes

• BCVA [26 weeks]: Proportion of study eyes achieving ≥ 15-letter best-corrected visual acuity (BCVA) improvement at 26 weeks post-treatment as compared to baseline.

Secondary outcomes

• BCVA [26 weeks]: Mean change in BCVA compared to baseline
• CCT [26 weeks]: Mean change in CCT compared to baseline
• BCVA [26 weeks]: Proportion of subjects achieving ≥10-letter BCVA improvement from baseline
• BCVA [26 weeks]: Proportion of subjects achieving ≥70 letters (ETDRS)

Sex: All, ages: 21 Years to any

Key Inclusion Criteria:

1. Symptomatic central corneal edema that is primarily responsible for loss of vision associated with endothelial dysfunction which may be secondary to Fuchs corneal dystrophy or pseudophakic bullous keratopathy.
2. Early Treatment Diabetic Retinopathy Study (ETDRS)-best-corrected distance visual acuity between 20 and 65 letters inclusive (≥ 20 and ≤ 65 letters)
3. Central corneal thickness ≥ 600 μm and ≤ 1,000 μm, OR Medical Monitor review and approval based on either:

   1. Historical CCT increased by ≥ 50 μm OR
   2. Findings of edema by corneal tomography or Anterior Segment Optical Coherence Tomography (AS-OCT).
4. Participant is considered a surgical candidate for full-thickness corneal transplantation or endothelial (partial thickness) keratoplasty (EK) consistent with standard-of-care indications.

Key Exclusion Criteria:

1. Corneal disease in either eye (other than corneal endothelial dysfunction secondary to Fuchs dystrophy or pseudophakic bullous keratopathy) including active or prior herpetic ocular infection, severe dry eye disease (e.g., Sjogren's), or active inflammation.
2. Central corneal scarring from trauma, burns, or infection, or band keratopathy
3. History of keratoconus or other corneal ectasia (e.g., keratoglobus and pellucid marginal degeneration).
4. Visually significant cataract, that may limit the participant's ability to demonstrate treatment-related improvement in BCVA.
5. Presence of an anterior chamber, sutured, or scleral-fixated intraocular lens.
6. Presence of a multifocal or diffractive intraocular lens.
7. Prior vitrectomy.
8. Corneal refractive surgery.
9. Descemet dettachment
10. Minimally invasive glaucoma surgery (MIGS), intraocular pressure (IOP)- lowering implant (e.g., Durysta® or iDose®), or any incisional glaucoma surgery (e.g., trabeculectomy, glaucoma drainage implant).

• Noelia Kunzevitzky, PhD, Study director, Emmecell