The following transcript has been edited for clarity.
Diana V. Do, MD: Hi, I’m joined by my friend and colleague, Carl Danzig, MD, and you’re presenting exciting data on gene therapy for wet age-related macular degeneration (AMD), the OPTIC trial. Give us a summary of the key findings.
Carl Danzig, MD: Thank you. It is really great being here. The OPTIC trial is a phase 1 multicenter trial of hard-to-treat wet AMD patients. They received, on average, 10 injections prior to enrolling. Ixo-vec (ixoberogene soroparvovec, formerly ADVM-022; Adverum Biotechnologies/Eli Lilly) is a capsid that transduces neuroretinal cells to produce aflibercept for sustained CNV suppression. And [in the trial], they looked at 2 different doses. The OPTIC trial was 2 years, the extension is year 3, and I’m presenting the 4-year data. What we looked at was an 86% on average annualized reduction of anti-VEGF injections in years 2, 3, and 4 following a single intravitreal injection of ixo-vec.
Dr. Do: That’s really impressive data, because we’re always looking for durability of treatment. How about safety? People are always wanting to know more about safety in gene therapy. Are there any safety signals?
Dr. Danzig: Excellent question. That is the number one concern anytime we talk about gene therapy; you’re spot on. There were 2 doses—2E11 was the lower of the 2 doses, and inflammation was dose dependent. So the 2E11 dose, which is what I’m presenting and analyzing, did not have any issues with hypotony, vasculitis, vascular occlusion—none of that. There are a few mild cases of minor intraocular inflammation (IOI) treated with topical corticosteroids. And what they also found, not just with the minimal inflammation in this lower dose, they found that this lower dose had similar aqueous protein levels compared to the higher dose. So they moved on to their phase 2 trial with this lower dose 2E11.
Dr. Do: Great. Well, this is very exciting. Look forward to more data in the future. Thank you so much for joining us at Retinal Physician.
Dr. Danzig: Thank you for having me. RP
