OpRegen® is currently being tested in a Phase I/IIa clinical trial for the treatment of dry AMD with GA. OpRegen® is a cell-based product composed of retinal pigment epithelial (RPE) cells, derived from human embryonic stem cells (hESC) and administered as a cell suspension in ophthalmic Balanced Salt Solution Plus (BSS Plus).
Phase I/IIa Clinical Trial Highlights
- OpRegen® has been well-tolerated, shows signs of structural improvement in the retina, and decreases in drusen density in some patients
- Improvements or possible restorations of the ellipsoid zone and RPE layers have persisted.
- The photoreceptor layer and ellipsoid zone assumed a more regular structural appearance in areas of the transition zone where OpRegen® was administered
- Early data from patients with earlier-stage dry AMD is encouraging
- Structural improvement within the retina
- Evidence of the continued presence of the transplanted cells
- Some improvements in visual acuity recorded
Importantly, in this safety-focused study, no unexpected ocular adverse effects have been observed and those events expected to occur based on the procedures involved in OpRegen administration, such as vitrectomy, have been mild in severity. For more details about the Phase I/IIa study, please see data presented at the 2018 American Academy of Ophthalmology Annual Meeting.
This is a dose-escalating Phase I/IIa clinical study, designed to evaluate the safety and tolerability of OpRegen® transplantation to patients. The study includes also initial exploration of efficacy. A total of approximately 24 subjects will be enrolled, aged 50 years and older, with non-neovascular (dry) AMD, who have funduscopic findings of GA in the macula, with absence of additional concomitant ocular disorders. Subjects are divided into four cohorts, according to their best corrected visual acuity (BCVA) and administered OpRegen dose. Twelve legally blind subjects with best corrected visual acuity of 20/200 or have been enrolled in first three cohorts and twelve subjects with best corrected visual acuity of 20/64 and 20/250 will be enrolled into the fourth cohort of the study.
Primary outcome measures in the study include: (i) incidence and frequency of treatment emergent adverse events (evaluated 12 months post transplantation); and (ii) treatment emergent changes of clinical and ophthalmological parameters (evaluated 12 months post transplantation). The parameters will be measured via different modalities, such as vital signs and ocular imaging and captured as adverse events. Secondary outcome measures in the study include: (i) change in GA lesion area (evaluated 12 months post transplantation), performed based on available imaging data by a central reading center; change in visual acuity (evaluated 12 months post transplantation), measured by ETDRS chart; (iii) change in Quality of Life (evaluated 12 months post transplantation), measured from baseline.