The following transcript has been edited for clarity.
Hi, my name is Sun Young Lee, MD, PhD. I'm a retina specialist and principal investigator at the USC Roski Eye Institute. I’m here to explain the recently launched phase 2B clinical trial testing [Regenerative Patch Technologies’] retinal pigment epithelial (RPE) cell implant for geographic atrophy (GA) patients. This implant was designed to provide healthy RPE cells to the area of GA for severe vision loss in advanced age-related macular degeneration. We have a great safety profile that we have learned from prior phase 1 and 2a trials and we just have launched phase 2b. We’re very excited to be recruiting patients and also study more about this technology.
Retinal Physician: Can you explain how the stem cell–derived RPE implant is designed to work in eyes with GA?
That's a very important question. We believe that loss and dysfunction of RPE cells play a central role in the development of geographic atrophy. As we all know, RPE cells are a very important for photoreceptor survival because they support the daily phagocytosis, metabolism, and visual cycle, and so on. This implant is designed to replace lost RPE cells by delivering healthy a fully differentiated stem cell–derived RPE cell implant directly to the affected area. One of the many key innovations in this technology is the ultrathin scaffold, which mimics Bruch’s membrane and helps the implanted RPE cell maintain proper orientation and structural stability within the eye.
RP: What are the key efficacy and safety endpoints you will be watching most closely over the one-year follow-up?
As I mentioned, in our earlier phase 1/2a trial, safety was a primary focus and we have observed a favorable safety profile. To our surprise, about 27% of the patients also showed a visual improvement. Building upon that research, the current phase 2B study as a focused on efficacy particularly functional vision a measured by microperimetry, along with anatomical outcomes such as changes in GA lesion size. Of course, we’re going to observe very carefully about any potential safety issues in this trial as well.
RP: At the end of this study, what would you consider to be a meaningful or practice-changing outcome?
As we know, right now treatment options for GA are quite limited and largely aimed to slow disease progression. A truly meaningful practice-changing outcome would be showing that this stem cell–derived RPE cell implant can actually improve visual function rather than only slowing vision loss. We're looking forward to learning from this technology at the end of our first-year outcome measurement. RP
