ASRS 2024: 100-week results from the RESTORE trial showcase retinitis pigmentosa outcomes

At this year's American Society of Retina Specialists (ASRS) meeting, Michael Singer, MD, presented 100-week results from the RESTORE trial. In this video, Dr Singer, who is a clinical professor of ophthalmology at UT Health San Antonio and director of clinical research at Medical Center Ophthalmology, both in Texas, shares key takeaways from his presentation.

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

Michael Singer, MD: My name is Michael Singer. I am clinical professor of ophthalmology at UT Health San Antonio and director of clinical research at Medical Center Ophthalmology. Okay, so talking about the RESTORE trial today, I was lucky enough to present the RESTORE trial on behalf of Nanoscope Technology.

And the RESTORE trial was, was looking at this thing called a multichromatic opsin in the treatment of retinitis pigmentosa. What's a multichromatic opsin, you say? It's something that can take care of all types of retinitis pigmentosa. As you probably know, inherited retinal diseases have as many as 275 genes causing this disease. And a lot of therapies are tailored to those specific genes. What's nice about the MCO product is that it's an AAV vector intravitreally injected that targets bipolar cells. And what it does, it transforms bipolar cells into photoreceptors. And as a result, it doesn't need to have a specific genotype.

What's interesting to know is today I presented the data on the 100-week results of the RESTORE trial, which was a Phase 2b3 trial looking at two different doses of MCO-10. It was a high dose, a low dose and sham. Primary endpoint was visual acuity at 1 year, secondary endpoint was virtual acuity at 76 weeks. And this was the end of study at 100. But the good news is that we're going to follow these patients and a different study, but same patients called the REMAIN study for another for a 5-year overall or 2.5 years from now.

So what did it show? What it showed was, essentially, that you had a statistically significant improvement in best-corrected visual acuity in both doses at Week 52, with 40% of people being three-line gainers. At week 76, the high dose was still statistically significant in terms of vision, and there were 56 three-line gainers. And at 100 weeks, there was still a statistically significant improvement in visual acuity and 30% three-line gainers. But I need to explain to what three-line gainers mean. In this population of people who essentially have no vision at all. The EDTRS trial basically breaks dow, and it breaks down because. It's not made for that ... it's made for 2800 or better. So, we need to choose a new visual acuity type test called the Freiberg Visual Acuity Test.

The Freiberg Visual Acuity Test is specifically made for these low vision patients. It's a new test, however it has been independently verified by independent agencies as well as been used in other clinical trials. So, in this patient population that potentially can't see, we need a special test. The fact that the vision was so good, make it very encouraging. There were secondary testsm like a mobility tested, you know, an IRDS shape discrimination tests. And patients were able to get improvement in at least two out of three of these different tests. Obviously, you inject an AAV vector, what the concern is, is that you're going to get inflammation. So there was a steroid prophylaxis at the beginning there, but there was no occlusive retinitis or vasculitis, or hypotony, or all the things that keep us up at night, to be honest.

The good news is that the vast majority of people were able to be tapered off steroids. And by week 100, there was only one patient on topical steroid once a day. So you know, in conclusion, the drug seems to work, has a good safety profile, has a good duration of activity,. and we're gonna follow the long term safety for another 2.5 years, which is 5 years total, and I'm honored to be able to deliver this information. Thanks.