The following transcript has been edited for clarity.

Hello, my name is Christine Nichols Kay, MD. I’m a vitreoretinal surgeon and IRD specialist. I'm at Vitreoretinal Associates in Gainesville, Florida. I’m here in Los Angeles at the Retina Society, and today I had the honor of giving the presentation of the MCO-010 (Mogenry; Nanoscope Therapeutics) optogenetics update from the REMAIN 4-year clinical trial data and the RESTORE phase 2b/3 clinical trial update for patients with retinitis pigmentosa (RP).

So let’s start with optogenetics. Optogenetics is a gene-agnostic—actually disease-agnostic—therapy targeting patients with low vision: with RP or with any disease theoretically. This mechanism can bypass patients with severe photoreceptor loss. And so optogenetics, specifically the MCO-010 program, is a multicharacteristic opsin, and that is delivered intravitreally with a single intravitreal injection of an adeno-associated viral vector, serotype 2 (AAV2), delivering a bipolar cell–mediated target, the mGluR6 promoter.

And so the idea is to be able to bypass a patient’s photoreceptors when they have severe photoreceptor loss and be able to target the healthy remaining inner retinal cells, specifically the bipolar cells in this program, to make them have the ability to sense light. And so this is a restoration treatment as opposed to just hoping for stability.

So the clinical trial that I spoke about today is the RESTORE trial, and the REMAIN trial is the long-term data. This was a study in patients with RP with a clinical diagnosis with low vision. So these are patients that already have severe photoreceptor degeneration. In the study, there were 3 arms. There was a low-dose intravitreal treatment, there was a high-dose intravitreal treatment, and there was a sham arm, and there were 9 patients in each arm. We treated the patients with 20 days of oral prednisone to begin, and then we followed those patients out to week 100 in the RESTORE trial. And so there was a statistically significant effect. The primary efficacy endpoint was met, which was looking at visual acuity at week 52 in the treated patients vs the untreated patients. And that actually held true for both arms, both the high-dose arm and the low-dose arm. There was a statistically significant difference and actually a clinically significant difference of 0.3 logMAR visual acuity of improvement in the patients who were treated in either arm vs the sham-arm patients.

The safety profile was excellent. The intraocular inflammation was mild to moderate, and patients, if they had to go back onto steroids, these were typically topical steroids for short term, and patients were able to be tapered off those steroids so that there were no SAEs and no severe vision loss in these patients.

And the REMAIN data was to look at the 4-year durability data. And so that actually did hold durable, that treatment effect of those 2 arms, both the high-dose and the low-dose arm remained in that 0.3 logMAR visual acuity gain. We use logMAR in these patients because these are off-chart patients with visual acuity in the 2.25 logMAR, which is 20/1600 visual acuity. So these are low-vision patients. We use Freiburg, which is off-chart measurement of visual acuity in low-vision patients. And so that did remain durable out to 4 years, which is the data presented today.

So it's very exciting to have an optogenetic therapy as a potential therapy for patients with low vision with RP to be able to look at the mechanism of optogenetics, which might be able to offer restoration or improvement in vision in patients with severe photoreceptor loss.

In conclusion, Nanoscope has completed the biologics license application (BLA) submission, and that was accepted and is now under review by the US Food and Drug Administration (FDA). So hopefully we’ll hear soon in the future regarding the future development of this program. RP