The following transcript has been edited for clarity.
Hi, I’m Diana V. Do, MD, at Retinal Physician. Today I’m joined by a leading retina expert, Arshad M. Khanani, MD, FASRS, who is discussing the latest clinical trial data from the ELAAVATE clinical study at the American Academy of Ophthalmology Retina Subspecialty Day. Tell us about the exciting news.
Arshad M. Khanani, MD, FASRS: Thanks, Diana. It’s always a pleasure to present at the subspecialty day and thanks to you and the program cochairs for allowing me to present the data on the ELAAVATE study, which is actually an investigator-initiated phase 2 study looking at safety and efficacy of sura-vec (ABBV-RGX-314; AbbVie/Regenxbio) delivered subretinally in patients with center-involving diabetic macular edema (DME). And Diana, this is the first time we’re actually studying subretinal gene therapy for DME because we want to know: Is this treatment safe? Is it doable? Meaning, diabetic patients are younger, and we are enrolling both phakic and pseudophakic patients, and trying to see if we can reduce the treatment burden, we can control the disease in terms of improvement or maintenance of visual acuity as well as disease control. So that’s why I initiated the study, and I’m really excited to present the results.
Dr. Do: Can you give us a little sneak peek? Is it efficacious and safe?
Dr. Khanani. Yes. To date, I’ve performed 9 surgeries in the ELAAVATE study with subretinal sura-vec, and I’m happy to say that we have not seen any new safety signals. We performed surgery in both phakic and pseudophakic patients. We have not had any complications. We have seen retinal pigmentary changes that we see with the subretinal gene therapy. But Diana, the interesting thing is that they’re happening much later and the intensity is much less compared to neovascular AMD patients. And we have that long-term experience.
The other thing is that many of the patients that were enrolled were previously treated with anti-VEGF agents. That was a criterion. These are previously treated patients and many of them were on second-generation agents. And what I was impressed to see is that actually we are able to maintain the good disease control without the need for injections, especially with the high dose. We have seen a dose-dependent response and the majority of patients in the trial have received 0 or 1 supplemental injections.
Of course, the data are evolving. We are still enrolling, but to me, the questions that you were asking about safety and efficacy seem to be answered in this early data cohort. There’s a signal of efficacy and disease control as well as safety that’s comparable to the wet AMD program.
Dr. Do: That’s exciting. Is there any need for local or systemic immunosuppression in this patient population?
Dr. Khanani: I think that’s a really good question. With subretinal gene therapy, we have not seen immune responses. We use the topical steroid taper similar to what we do with vitrectomy; I usually do a 4-3-2-1 taper over a month. That’s a really important question because subretinal gene therapy, we have not seen any immune responses, so we don't have to give oral or long-term topical drops.
Dr. Do: Thank you for sharing this exciting data. It may be something new to offer our patients in the near future. Thank you for leading that.
Dr. Khanani: Thank you so much. I'm looking forward to learning more, and hopefully we can take it forward into a larger trial to confirm the findings and possibly have an option for patients to move away from repeated injections toward more sustained delivery with gene therapy. Thank you, Diana.
Dr. Do: Thank you, Arshad. RP
