Lineage Cell Therapeutics, Inc. has announced the launch of a phase 2a study of RG6501 (OpRegen) by Genentech for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD).
Tell me about RG6501.
RG6501 is a retinal pigment epithelial (RPE) cell therapy derived from human embryonic stem cells and administered subretinally as a cell suspension. It was previously studied in a phase 1/2a trial, which concluded earlier this month.
What happened in the Phase 1/2a study?
A total of 24 participants reported one or more ocular adverse events. The majority of adverse events reported were mild and related to the method of subretinal delivery.
Data results suggested the treatment might potentially slow, stop, or even reverse the progression of GA. Further studies are needed to confirm these data and optimize the delivery methods, location, and timing.
What will the phase 2a trial entail?
The study will focus on optimizing the subretinal delivery method in 30 to 60 patients diagnosed with GA secondary to AMD. Primary objectives include evaluating the proportion of participants receiving RG6501 and assessing the safety RG5601 administered via a subretinal surgical delivery, which will be measured by potential procedure-related adverse events 3 months post-surgery.
A secondary objective includes using optical coherence tomography (SD-OCT) to assess any patients who exhibit qualitative improvement in the retinal structure 3 months post-surgery. (via)
The take home
There are currently no FDA-approved therapies for GA secondary to AMD. With several studies currently in progress for RPE cell-based therapies for macular degeneration, largely in early phase trials, RG6501 has the potential to become a new therapy for a patient base with few existing options.