Ocular Therapeutix, Inc. Chardan's 10th Annual Healthcare Conference

Andreas, first of all, thank you for having me here. It is an honor to talk with you, and thank you all for being here. I think you first start out with a need here. We have had a very, very effective and safe class of drugs, the anti-VEGFs, now for almost four decades. Yet, in this country alone, in the first year, there is a 40% dropout rate. That is not just tragic, it is intolerable really. All of these patients who drop out, almost half of them end up going blind. So there is a great need for sustainability of treatment so that the dropout rate decreases. We know that is the case historically.

The other part of this is also that because of the pulsatile nature of injections, even in those patients who are able to stay through the therapy, the majority of them, after 2 to 5 years, end up losing vision because of atrophy and fibrosis. So what we are trying to do is to solve both of those problems. We have a tunable hydrogel with a TKI called AXPAXLI. We have shown superiority. We have hit a superiority endpoint versus an anti-VEGF for the first time ever. We are very proud of the results. We think the results are terrific, and we have got a single trial approval pathway formalized with the FDA.

That's a great question. Look, I think it goes without saying to this audience that we're in a very, very tough field. Nine out of 10 drugs fail, and it has an impact on everything, including a lot of employees, their families, and everything else. That's the first thing to be said. There's no joy in seeing somebody else fail. The way that I look at this is there are lessons to be learned here, and we'll talk about that in detail, and these are really important. I'll take a little bit of time. In terms of the overview, we had one competitor in our field, which is that we have a maintenance drug. We don't have a bolus injection. Everything else that you hear about, which are non-inferiority studies, are in a morass of bolus injections.

There are really, as far as we're concerned, two drugs competing for maintenance. One is us and one is EyePoint, and now it's only us. I think there's a clear path forward for us without any kind of competition whatsoever. Now let me get to this really important topic, and this really applies to anybody who's an analyst, who's an investor looking at chronic retinal diseases. One thing that is absolutely misunderstood, in my humble opinion, outside of the retina community, is the variability of our diseases. Let's look at macular degeneration, but quite honestly, it's true for diabetic macular edema, for RVO, for diabetic retinopathy, all of those things. The bottom line is that we are at least 75 years behind oncology and rheumatology and immunology. We have absolutely no idea about disease stratification.

If I were to come to you, Andreas, and say, "Look, I have a drug for colon cancer," you'd just look at me like I was from Mars. You'd say, "Well, what kind of colon cancer? What stage? What grade?" I come to you and say, "Look, there's a drug for wet macular degeneration or diabetic macular edema," and nobody pays mind. They go, "Okay, that's great." We know there's an enormous amount of variability. We know that even with the regular Lucentis, there are patients that require treatment every two weeks. There are patients that require treatment every six months. So the selection of patients and clinical trial design is absolutely key. We're not doing 2,000, 3,000-patient large studies anymore that would negate this, perhaps, or mitigate this at least, like Genentech and Roche and others did. We're doing smaller studies. Great attention has to be paid to patient selection.

Because we don't have any biomarkers of disease. We don't have any genetic markers of disease. What we have done is very, very thoughtfully gone through patient de-risking that in a bespoke manner for each of our studies. It's an entirely different patient population and an appropriate one that we have selected for SOL-1 versus the stable patient population for SOL-R. This is why we're so confident in succeeding with SOL-R. It has nothing to do with retreatment criteria. It's got everything to do with clinical trial design and proper patient selection. I think now EyePoint themselves are actually admitting to that now following the failure. I think you saw some of that news this morning and yesterday.

We emphasize that. I think the reason for their failure is not only what we said earlier on, which is that we believe it's a weaker TKI, but one of the very, very big reasons is the patient selection. Now, it's easy to enroll trials when you allow everybody to come in as they did. They mixed previously treated patients with treatment-naive patients. They randomized, and then they loaded. We're doing the opposite. We're only using treatment-naive patients. We're making sure that we load first, and the loading for us is not necessarily biologic. It's to select the right patient population, and only then are we randomizing. That is the key to why we are so confident in the success and why this is different than what happened with LUGANO and LUCIA.

Well, look, I wish them well. I don't see them as our competitor for reasons that I mentioned earlier on, which is that they're a bolus injection. I think it's important to separate bolus injections from maintenance drugs. We're a maintenance drug. As I said earlier on, our competitor was EyePoint, and now it's only us. Whatever bolus injection one uses, if one feels the need to use that prior to using a maintenance drug, really doesn't matter to us. It could be Avastin, it could be Vabysmo, it could be Lucentis, it could be the Kodiak drug if it makes it that far. But it's the maintenance drug category that we're in. So it's not the same thing. It's important to separate, and we don't consider them competitors at all. Having said that, I think there are a lot of questions to be answered.

Who were the patients that actually lasted 6 months? Where were they? Were they after 6 bolus injections? Were they in the beginning? Do they have to be retreated? How many injections did they actually get? We haven't seen the total number of injections. These are just really basic questions. It's a PRN arm versus an on-label arm. I don't know what the regulatory pathway is for a PRN arm. As far as AEs are concerned, I think we, at best, may have seen 25% of the AEs. I think there's a lot more to be seen. So look, I wish them well. There's a lot of data that we'd still like to see from a clinical trialist point of view. I'm sure that'll be forthcoming. I hope that'll be forthcoming. But again, we don't consider them our competitors.

They're competing with the Lucentis, Avastin, and Vabysmo of this world as a bolus injection.