Ocular Therapeutix, Inc. 2026 Global Healthcare Conference

Jack, thank you for having us here.

Ocular Therapeutix is a company based in Bedford, Massachusetts. What we have is a TKI or a tyrosine kinase inhibitor on a proprietary hydrogel platform. The idea is to block a receptor, VEGF, which we know works very well in diseases such as wet macular degeneration and diabetic retinopathy. There's more than 40 years of data showing that, and tries to solve two problems. The first problem is that of sustainability. We know that despite having a very effective treatment in this country alone, more than 40% of patients drop out in the first year. That's the more obvious problem that we're trying to solve.

The second problem that we're trying to solve is that even in those patients who continue to get treated and happen to be able to come back, after about 2 to 5 years, they inevitably lose vision, and this is probably because of the pulsatile nature of treatment where we give injections every month or every other month, and that causes a thickening and thinning in the back of the eye. Much like a concussion in your brain, which causes scarring and fibrosis. We're trying to achieve a greater degree of sustainability as well as a better long-term outcome by suppressing a known and validated target.

Sure. What we've done from the time that this team came in about 2 and a half years ago, is we've been in constant touch with the FDA. Everything we've done is exactly according to FDA guidelines. That's why for SOL-1, our successful phase III study, we have a SPA, a Special Protocol Assessment. From the very beginning, we have discussed with them the possibility of a single trial approval. What they've said has been very clear and very consistent, which is that you need 2 evidentiary standards before the CHART turn and 2 evidentiary standards after the CHART turn to allow for a single trial submission. Those evidentiary standards have never changed in the last few decades. First, you need to have a study. This is before the CHART turn that is properly masked. That means no sham. It doesn't mean that sham can't get approved, but just not with one trial.

So properly masked. The second one is that it has to be properly powered. Now, both of those boxes have been checked officially because we have a SPA, obviously. After the CHART turn, the 2 boxes are, one, the P value has to be less than 0.001, and ours is less than 0.0006. The second is that you have to be able to supply the FDA at the time of the submission with at least 300 patients who've been exposed to the drug for safety reasons only, and we're able to do that. So we checked all the boxes. We are in line with all the evidentiary standards, and in a Type C meeting, we have official confirmation by the FDA that we're able to submit with a single trial.

Part of that process was a pre-NDA meeting, which is really an operational meeting that we said that we would get completed by the third quarter of this year. The press release this morning shows not only that we're doing it in time, but that we're completely aligned with the FDA in order to have our complete submission in the fourth quarter of this year.

Yeah. That's a fantastic question. Look, SOL-R, the second study, the non-inferiority study, is very important for us for OUS regulatory agencies. As you mentioned, it's vital for us to protect the integrity of SOL-R. The FDA simply wants 300 patients that have been exposed to the drug, and that's true of any study. It doesn't have to be the same target. It could be any other target, any other disease target. It doesn't even have to be the same dose. It can't be a lower dose, but it can be a higher dose. What we needed to do was to get the safety data from approximately 130 patients from SOL-R. Now, we wanted to do that but protect the integrity of the study.