ESCRS 2024: Approaching a paradigm shift in cell therapy for corneal oedema and endothelial dysfunction

This September, the European Society of Cataract and Refractive Surgeons (ESCRS) holds its annual congress in Barcelona, Spain. At the meeting, Michael Goldstein, MD, president and chief medical officer, Aurion Biotech, presented a from the Escalón trial, titled "12-Month Safety and Efficacy Clinical Trial of Human Corneal Endothelial Cell Therapy for Corneal Oedema Secondary to Endothelial Dysfunction." In this video, he tells Ophthalmology Times Europe about the research and how it represents a paradigm shift in the corneal and refractive surgery space.

Editor's note: This transcript has been lightly edited for clarity.

Hattie Hayes: Hi. I'm Hattie Hayes. Welcome to Ophthalmology Times Europe. This year, the European Society of Cataract and Refractive Surgeons is holding its annual congress in Barcelona, Spain. Dr Michael Goldstein will be there presenting 12-month safety and efficacy data from a clinical trial of human corneal endothelial cell therapy for corneal oedema, secondary to endothelial dysfunction. But today, he's here with me to tell you all about that presentation. Dr Goldstein, thank you so much for joining me.

Michael Goldstein, MD: Thank you, Hattie, great to be here with you.

HH: Now, I know that the data we're talking about today are 12-month results. Give me a little bit of an overview of that patient experience, from day one of the therapy through to that year mark.

MG: Sure. So we're really excited to present this data. This is data from our most recent study in El Salvador, looking at patients who have corneal oedema, and what we're doing is we're treating them with our cell therapy product, which is a combination of a cell therapy and a rho kinase inhibitor. And what we've seen and we're presenting is the data looking at both the anatomical improvement, meaning, Does the cornea get less thick? and the functional improvement, meaning, Do the patients actually see better?

What we show with this data is, by 1 month, patients are noticing a very large improvement in terms of their corneal thickness. And more importantly, they're actually improving their vision. In particular, we're looking at a clinically-meaningful improvement in vision, and we define that as a three-line gain, and so by the 1 month period, we're already starting to see patients that actually have three lines of gain that increases to 3 months, and then pretty much stabilises at 6 months. And we're able to see that the durability for both visual acuity improvement and corneal thickness improvement maintained out to a year. And what's really cool about this data is this replicates prior data that we've done in other trials in El Salvador, as well as data that have been generated in Japan.

HH: I'm glad that you mentioned the previous trials in El Salvador and Japan, because I know that you have been dosing patients all over the world, including in the US and, earlier this year, the first patients in Canada. What can you share about what to expect from these clinical trials going forward?

MG: So it's a little bit different in each country. As you may be aware, in Japan, we actually have an approval for the product, and we recently actually got reimbursement approval in Japan. And we're excited to say that we're going to actually launch the product in the next couple months in Japan. So we'll start the commercial process in Japan.

In El Salvador, we continue to do clinical trials, and should have some additional data coming out of El Salvador, probably early next year. And in the US and in Canada, we have a phase 1/2 trial, which we started in October of last year and finished [enrollment in] April of this year. We look to have data from that trial in the first quarter of next year. That trial was a dose-ranging study, where we looked at different doses of the cell therapy and compared that to each of the compositional elements. So this would be some really interesting data coming coming out of that trial. Once we have the data from that trial, we'll pick one of the doses from that trial and move that forward into pivotal trials, which will take place middle of the next year in in the US and Canada.

HH: Well, that sounds right on time for the ESCRS Winter Meeting, so I'm gonna go ahead and put that in my calendar real quick! Now, you're presenting these data as part of a larger session on advancements in the corneal therapy space. What shifts or trends are you seeing in in that space, and what are you most excited to talk about with colleagues at this year's ESCRS meeting?

MG: Yeah, great question. So I think one of the exciting things about ophthalmology, why a lot of us got into ophthalmology, is innovation, and we're constantly looking at ways to improve the patient experience. I think the biggest example that people are familiar with is cataract surgery. So we started with intercapsular cataract surgery, and that was a good surgery. And then we came to extracapsular cataract surgery, and that was even a better surgery. And then...the paradigm really, really changed with phacoemulsification and all the advances we've had there. I think corneal transplant surgery is at that same point: we've had really, really good surgeries for our patients, starting with full thickness penetrating keratoplasty. And then more recently, going to endothelial keratoplasty, starting with DSEK surgery, and now more recently, DMEK surgery.

But the Holy Grail really is, can we take those endothelial cells, inject them into the eye and get them to reform inside the eye? And what cell therapy offers us is that opportunity to do this. And you know, this is no longer in the science fiction world. This is work that Professor Kinoshita has been doing in Japan, starting in the late 90s, but it's now at a point where it's reality. We now have a drug product and a procedure that we can effectively treat these patients who have corneal oedema with cell therapy. And the data we're presenting here, and the data that Professor Kinoshita has presented, shows that this can actually be the new reality and the new paradigm for how we treat these patients. So I think probably one of the most exciting developments that we have in ophthalmology, certainly one of the most exciting developments in the field of cornea.

HH: Thank you so much for speaking with me and for bringing this data to everyone at this year's ESCRS meeting. And I hope that you have safe travels!

MG: Thank you. You too.