RPE Cell Therapy Shows Improvements in Early GA With AMD
RG6501/OpRegen was well-tolerated, with no serious treatment-related AEs reported in follow-up data from a phase 1/2 trial.
David Telander, MD, PhD
Credit: University of Minnesota
RG6501 (OpRegen) allogeneic retinal pigment epithelium (RPE) cell therapy has continued to be well-tolerated in patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD) in extended follow-up data from a phase 1/2 trial (NCT02286089). Patients with less advanced GA also experienced gains in best-corrected visual acuity (BCVA).1
Data from the trial were presented at the 2024 Retinal Cell & Gene Therapy Innovation Summit, held on May 3, 2024, in Seattle, Washington, by David Telander, MD, PhD, a staff physician at Retinal Consultants Medical Group, in Sacramento, California.
“The products approved recently for the treatment of GA secondary to AMD have not demonstrated a compelling functional benefit, so there remains a need for treatments that can positively affect patient vision,” Brian M. Culley, the CEO of Lineage, said in a statement.2 “We are encouraged by the apparent durability of visual performance being achieved by patients in this study, notably in the 5 patients who received a thorough coverage ofOpRegen cells across the majority of their atrophic areas and who had experienced a cessation or reversal of their areas of atrophy, evidenced by outer retinal structural improvements on optical coherence tomography (OCT). Dry AMD has traditionally been considered to be an irreversible and progressively degenerative disease leading to vision loss. However, these data suggest thatOpRegen RPE cells may provide a one-time treatment which could slow or even reverse this damage by way of direct support to the patients’ remaining retinal cells, including those near or within atrophic areas. We look forward to additional, future clinical data updates on the OpRegen program from our partners, Roche and Genentech.”
Investigators observed improvements in BCVA, specifically, in 10 patients in cohort 4 with less advanced GA 12 months after treatment, which persisted through 24 months. These participants had a mean change in BCVA of +5.5 letters on Early Treatment Diabetic Retinopathy Study (ETDRS) assessment at 24 months post-treatment. In 5 participants with extensive OpRegen bleb coverage of their GA area, the mean change in BCVA was +7.4 ETDRS letters at 24 months.1
These participants also maintained or improved external limiting membrane (ELM) and RPE drusen complex (RPEDC) structure on optical coherence tomography OCT. RPEDC area improvements were maintained compared with baseline in treated eyes from 12 months (+2.6 mm2; n = 5) to 24 months (+2.6 mm2; n = 4). In untreated fellow eyes, RPEDC area had mean decreases at 12 months (–1.1 mm2; n = 5) to 24 months (–2.8 mm2; n = 4). Similarly, ELM mean area increased in treated eyes from 12 months (+0.4 mm2; n = 5) to 24 months (+0.8 mm2; n = 4) compared with decreases in untreated fellow eyes from 12 months (–1.3 mm2; n = 5) to 24 months (–1.9 mm2; n = 4).1
No serious treatment-related adverse events (AEs) were reported. The most frequent ocular AEs reported in all patients on study treatment were conjunctival hemorrhage/hyperemia (71%) and ERM (67%), and most AEs were mild (87%).1
“These data suggest that OpRegen RPE cells may counteract RPE cell dysfunction and loss in GA by providing support to the remaining retinal cells within atrophic areas; such effects are durable through at least 24 months after a single administration,” Telander said during his presentation.1
