On Monday, 10 March 2025, Ocular Therapeutix (NASDAQ: OCUL) presented at the Leerink Global Healthcare Conference 2025, offering a strategic overview of its lead product candidate, AXPAXLI. The company’s CEO, Pravin Dugel, discussed the promising potential of AXPAXLI in treating retinal diseases, highlighting both opportunities and challenges. The focus was on clinical trials, market adoption strategies, and financial stability.
Key Takeaways
- Ocular Therapeutix is well-capitalized to continue operations through 2028, supporting key clinical trials.
- AXPAXLI is positioned as a first-line treatment for retinal diseases, with a unique formulation and potential for premium pricing.
- Amendments to the SPA with the FDA are expected to expedite the regulatory submission process.
- The company anticipates seamless market adoption due to minimal changes required in clinical practice.
- Positive results from the SOL-1 study could significantly enhance the success of subsequent trials.
Financial Results
- Cash Runway: Ocular Therapeutix is financially secure through 2028, ensuring funding for the completion of the SOL-1 and SOL-R studies.
- Potential Premium Pricing: AXPAXLI is expected to command a higher price due to its clinical benefits.
- ASP (Average Sales Price): Physicians may receive higher compensation per injection, reflecting the drug’s value.
Operational Updates
- SOL-1 Study: Randomization was completed in December, with results anticipated in early 2026.
- SOL-R Study: An amendment has reduced the study size, accelerating its completion, expected in the first quarter of 2026.
- NPDR Program: A meeting with the FDA is scheduled for the first half of the year to discuss study design.
- Manufacturing and Logistics: AXPAXLI’s adoption is expected to be straightforward, requiring no changes in doctor’s offices.
Future Outlook
- FDA Submission: The company plans a faster FDA submission following positive outcomes from the amended SPA.
- Regulatory Path: The path to approval is considered derisked due to compliance with FDA guidelines.
- Market Adoption: A shift to a fixed treatment regimen, with doses every six months, is anticipated.
Q&A Highlights
- SOL-1 and SOL-R Studies: Success in SOL-1 is expected to bolster the SOL-R study’s outcomes.
- Rescue Protocols: All rescue interventions will be included in the primary analysis, benefiting Ocular Therapeutix.
- Patient Selection: Careful patient selection aims to minimize variability and enhance trial success.
- Differentiation from Competition: AXPAXLI stands out among TKIs for its potency, selectivity, and unique formulation.
For further details, readers are encouraged to refer to the full transcript provided below.
Full transcript - Leerink Global Healthcare Conference 2025:
Mark Goodman, Biopharma Analyst, Leerink: Okay. Alright. We’re gonna get started with our next session. Thank you everybody for joining us at the Leerink Global Healthcare Conference. I’m Mark Goodman, one of the biopharma analysts, and we’re lucky to have Ocular Therapeutics with us, Pravin Dugel, who is the CEO.
Thank you for joining us. So maybe I’ll let you just make a quick just opening comment. It’s been just a lot going on over the past year and joining the company. And just give us a sense of why this company? I guess you could have done a lot of different things, and it’s always an interesting question to kind of kick start.
Pravin Dugel, CEO, Ocular Therapeutics: So Mark, first of all, thanks for having us here. It’s really an honor and a delight to be here. And when we ask you to open a new question like that, unless you pull the plug on me, I may keep going for the entire half hour.
Unidentified speaker: I’ll I’ll pull
Pravin Dugel, CEO, Ocular Therapeutics: I’ll stop. I’ll stop. But but but it’s really simple. You know, look, as a as a person that practiced for thirty years, it to me, it’s absolutely tragic that we have a treatment that we know works, a target that we know is valid. And yet in this country and in this country alone, in the first is that even in patients who stay, after about two to five years, inevitably end up losing vision anyway, even with this treatment.
And the reason for that, now we understand it, is because of the way we treat, which also makes it not sustainable, causes oscillations in the back of the eye. Because we treat in a pulsatile way, the back of the eye, which is nerve cells, end up getting thicker and thinner and thicker and thicker and thinner. It’s like having multiple concussions, and they end up having fibrosis and atrophy and company with Ax Paxil is solving two problems. The obvious one, which is that of a sustainable treatment, but the other one, which may not be quite as obvious, but is equally important, which is better long term outcomes by reducing the amount of fibrosis.
Mark Goodman, Biopharma Analyst, Leerink: Right, right, right. Okay. So just high level for a second, just explain the goal of the product for a second before we jump into it. So I don’t want to get into the details
Pravin Dugel, CEO, Ocular Therapeutics: One is a superiority study called Sol one. The other is a non inferiority study called SOL-R. And you’ll notice immediately that what we’re not doing is we’re not repeating the same study twice, which is what most people do. And this is very deliberate, and we believe it’s quite revolutionary in a positive way, which adds even more information having two different studies, a superiority study and a non inferiority study, reach the same conclusion. So we’re targeting wet macular degeneration with a product called X Paxly, which is a TKI, a tyrosine kinase inhibitor.
We’ve also said publicly that our plan, sometime in this calendar year is also to target non proliferative diabetic retinopathy for which there is really effectively, there is no used treatment at this point.
Mark Goodman, Biopharma Analyst, Leerink: Okay, good. Thank you. So during the earnings call, there was some changes to the study and stuff. So take us through, here’s what we were doing and here’s what we changed and here’s why we’re doing this.
Pravin Dugel, CEO, Ocular Therapeutics: Yeah. The bottom line of what we the change, which is an amendment to the SPA, is that it allows us to get to the finish line much faster, much cheaper, and with a better label. And let me explain that. What we had in the superiority study, the SOL-one study, was a primary endpoint at month nine. We didn’t have anything else after that.
And as you know, what the FDA requires is a certain number of patients to be followed for two years for safety reasons. So we had to overpower our second study named SOL-R in order to satisfy that FDA requirement. And what we did, was we requested an amendment that was approved to extend SOL one to satisfy the FDA’s requirement, we also could now reduce the size of SolR. So effectively, what we did was to reduce that delta between the card churn of Sol-one and SolR. And remember, we need both studies to be positive before we can submit to the FDA.
And effectively, we can now submit to the FDA with two positive studies a lot faster, than we could have without the amendments. So and by the way, I’m sorry, Mark. No. We gave up absolutely nothing. And that’s the really important part.
Part. We gave up absolutely nothing. The primary endpoint remains exactly the same, which is nine months and so on. Yeah. And the statistics have not changed.
So again, recapping the the end result of this, we’ll get to the finish line a lot faster, a lot cheaper, and with a much better label.
Mark Goodman, Biopharma Analyst, Leerink: So it’s basically numbers of patients is what we’re talking about.
Pravin Dugel, CEO, Ocular Therapeutics: We shifted from every all the safety follow-up being in the second study to some being in the first study. Therefore, we could reduce the size of the second study. Why now? Yeah. That’s a great question.
So, you know, and that’s been asked of me often, and which is which is to say, look, why why why’d you do it now? If if you knew that, why do it now? Why not do it in the very beginning? And the answer is very simple. In this, what I’m very proud of and what we did in this company is to have what we call an all planet team.
We’ve got a whole bunch of people here that have been doing clinical trials for twenty to thirty years. In our experience in retina and doing clinical trials, what we noticed, under masking, of course, is is retention rates like we’ve never seen before. The best retention rates that we’ve ever seen, which has been phenomenal. We got fantastic retention in in in Solon. And without having fantastic retention, we would have never proposed this amendment because we needed to have the confidence of saying, we absolutely will get that number of patients to the finish line of two years.
So why now is because we needed to see that retention number.
Mark Goodman, Biopharma Analyst, Leerink: I see. Okay. That makes sense. So, maybe it’s a good time to talk about what the market needs, which is kind of where you started, which is this duration issue. So talk about how you think the product will be used in the real world.
And then also talk about, you know, there’s some other TKIs. We didn’t talk about it’s a TKI, but talk about, you know, what we have with TKI, there’s some other TKIs and how you differentiate your product from the others in the market.
Pravin Dugel, CEO, Ocular Therapeutics: Okay. There are a lot of questions there. But let me just answer the first one. First is in terms of how it will be used. First of all, one of the really important things is that it’ll be adopted the very next day after it’s approved.
There’s there’s there’s gonna be a very simple and very quick and very seamless adoption, and that’s because of logistics and it’s also because of economics. The logistic part is very simple. Nothing needs to change for the doctor. There’s not a single piece of new equipment. There’s absolutely nothing that needs to change that patient flow doesn’t need to change.
A doctor simply reaches out and gets a better medicine without having to change one thing in his or her office whatsoever. So it’ll be very, very easily and seamlessly adopted. The whole experience for the doctor and the patient is gonna be no different than injecting ILA or Lucentis. It’s just a better drug with a better outcome that lasts longer. Now from an economic point of view, it’s a win win win for everybody involved.
As I said, let’s talk about payers first. As I said earlier on, forty percent of patients
Unidentified speaker: in this country alone are actually dropping out of treatment.
Pravin Dugel, CEO, Ocular Therapeutics: Those patients don’t drop out of the costliest types of patients, and I know this sounds very cold, are blind patients. They use an enormous amount of resources, have all kinds of accidents, things like that. And to be able to reduce that by even ten percent means because because now we have more sustainable treatment, means that there are about quarter million more patients that are being treated in this country alone. That’s a huge cost savings for payers and much better for patients, obviously. The payers will love this.
That means they will be able to certainly be eligible for premium pricing. That also means that the ASP will be higher, so the doctors will get paid more per injection. So from an economics point of view, it makes complete sense. Doctors oftentimes don’t, you know, know economics as well as they should. And if you ask a lot of them, they’ll say, well, it’s a better drug.
It’s going to be much better for patients. But, you know, I make my money by injecting, and I may be injecting less. That’s absolutely not true. They’ll actually be injecting a heck of a lot more. Not the same patients, but the of patients will be much greater because the dropout, we’re convinced, will be less.
Again, even if the dropout is reduced by 10%, that’s two hundred and fifty thousand more patients in this country that’ll be eligible for treatment. So they can inject all they want. They’ll inject with a better drug. They get paid more. And they potentially will have other targets for which there is no injection at this point, which is non proliferative.
Mark Goodman, Biopharma Analyst, Leerink: So somebody understands. So how are the docs making money? And how’s that changed? I mean, the
Pravin Dugel, CEO, Ocular Therapeutics: Well, the ASP changes because the higher the price of any drug. And what’s the ASP? That’s the actual pricing.
Mark Goodman, Biopharma Analyst, Leerink: No, no, what is it plus now though? It’s ASP plus?
Pravin Dugel, CEO, Ocular Therapeutics: Right now it’s 2%. Two %.
Mark Goodman, Biopharma Analyst, Leerink: And it used to be higher.
Pravin Dugel, CEO, Ocular Therapeutics: Yes, exactly. Because the pricing will be higher.
Mark Goodman, Biopharma Analyst, Leerink: Yes. So I’m just saying that it used to be ASP plus six and now
Pravin Dugel, CEO, Ocular Therapeutics: it’s ASP plus six. It’s used
Mark Goodman, Biopharma Analyst, Leerink: down to 2.5. So they like right.
Pravin Dugel CEO, Ocular Therapeutics: So I’m saying they
Mark Goodman, Biopharma Analyst, Leerink: like the fact that the drug could be higher. Right. Because on an absolute basis that’s what they’re yes, yes, yes. Okay, so let’s come back to how you see this product in the real world. So it’s launched, it’s okay.
Pravin Dugel, CEO, Ocular Therapeutics: Yes. So again, it will be adopted very quickly for the reasons that I mentioned. And I think the way that it will be used in the very beginning is what we call treatment extend. And for those of you who are not familiar with treatment extend, what we do is to go ahead and treat a patient with, say, Eylea, Lucentis, what have you, vibezbo. And then for every patient, we wait to see when that drug loses effectiveness.
For some patients, it may be after a month. For some, maybe after two months or three months. And then we and then we go ahead and keep doing essentially trial and error until we find the proper cadence for that particular patient. That’s treatment extent. And I think that’s exactly what we’ll do with this drug.
And the reason for that is very simple. We’re used to doing it. We’re very familiar with it. However, the treatment extend will be moved. The needle will be moved very much to the right.
And what I mean by that is if there’s a patient that requires ILEA, say, every month, that patient may now require XFAXLA every six months. If somebody requires ILEA every two months, it may be every ten months with XFAXLA. So I think it still will be treatment extent, but it’ll be moved very much to the right.
Unidentified speaker: My prediction, and this is just my prediction, is that after a
Pravin Dugel, CEO, Ocular Therapeutics: little bit of time, I think My prediction, and this is just my prediction, is that after a little bit of time, I think we’ll all move to to fixed treatment. And the reason for that is, again, quite simple. It’s because treat and extend as as as as useful as it is now,
Unidentified speaker: is very, very difficult on the patient and scheduling for the doctor.
Pravin Dugel, CEO, Ocular Therapeutics: It’s it’s it’s it’s a on the patient and scheduling for the doctors. A lot of experiments. It’s a lot of exactly. And scheduling wise, it’s a nightmare for both. Yeah.
And I think what people realize is to say, look, this is a great drug, and it lasts for, you know, this long a period of time. It’s it’s perfectly safe, and it’s very effective. And most of us like to see patients with chronic diseases every six months. And I think they’ll come in every six months. The burden is coming in.
It’s not the injection. Right? They’ll already have taken the burden of coming in. And I think they’ll be just treated every six months on a fixed basis. The scheduling will be easy for the doctor and will be easier for the patient as well.
Mark Goodman, Biopharma Analyst, Leerink: So today, it’s not Jeff, obviously, what you’re talking about the treatment extends. So the goal here is maybe someone’s been on EYLEA for a certain amount of time. They’ve had the loading dose, maybe someone’s been on EYLEA for a certain amount of time, they’ve had the loading dose, they’re comfortable with this one month or two month or whatever it is that they’re comfortable on in year one and then why not bring your product in kind of is that how you see this like later in year one or is it a year two or even out of the gate, why not just how do you
Pravin Dugel, CEO, Ocular Therapeutics: So Mark, quite honestly, I do believe that I think what you’re asking me is, is this a first line drug or is this a maintenance? That’s And it’s a fair question. And what I would say is really simple. I mean, my flippant answer is, you know, why would I care? Because, you know, if somebody has to use ILA once or twice and then uses EXPASLI for the next twenty years, it’s still going to be the most impactful drug.
And that’s the worst case scenario. However, I firmly believe that this is the only drug that will be needed. I believe it is a first line drug. And why do I say that is simple. One is because we’ve got great studies to show that steady state is established within twenty four hours.
And we have patients in our study in Australia where this drug was used as monotherapy in treatment naive patients. The only thing that was used. And what we saw were responses that were akin to what you’d expect to with commercial grade ILA or Lucentis.
Mark Goodman, Biopharma Analyst, Leerink: As quickly as
Pravin Dugel, CEO, Ocular Therapeutics: As quickly as
Unidentified speaker: As quickly as As quickly as As quickly as
Pravin Dugel, CEO, Ocular Therapeutics: Exactly. So
Mark Goodman, Biopharma Analyst, Leerink: there’s no reason to even wait.
Pravin Dugel, CEO, Ocular Therapeutics: That’s what I think. Now, having said that, even the worst case scenario, if it wasn’t, this still will be by far the most impactful drug in our field. So somebody will do that study, maybe it will be us, maybe it will be others, but I believe that this will be the only drug that will be required.
Mark Goodman, Biopharma Analyst, Leerink: Yes. So I think a frequently asked question, I’m sure, as well, you have competition going after your space a little bit as well doing with the same we’ll call it the same business model, same six months. Talk about just the differences between your product and their product.
Pravin Dugel, CEO, Ocular Therapeutics: Well, look, people ask me about the biosimilars for the anti VEGFs and VIBISMA and so on and so forth. As far as the anti VEGFs are concerned, we’re agnostic to that. It really doesn’t matter because we’re in a different orbit. When we talk about a VIBISMO or high dose ILA, we’re talking about adding another two weeks or what have you. And by the way, we will have data that we will have that’s not non statistical data because there is a masking arm versus high dose ILA in the SOL-R trial, the third arm.
And it’s non statistic numeric data that we’ll have. But nonetheless, again, these drugs add another couple of weeks. We’re talking about a different orbit. We’re talking about, you know, nine to twelve months. So we’re not worried about that fight whatsoever.
As far as other TKIs are concerned, look, we’re very different. Our TKI is known to be the most potent by 200 fold or so, the most selective by about 100 fold. Our data is completely different than others. And very importantly, the format is completely different. We have a tunable hydrogel.
There are no carcasses floating around. There’s nothing left behind. When the drug is gone, the hydrogel is gone. So it’s an entirely different product.
Mark Goodman, Biopharma Analyst, Leerink: And what about the clinical studies that everybody is pursuing? Like this is the part that I didn’t quite understand, the sham versus the not sham. Can can you just explain to people?
Pravin Dugel, CEO, Ocular Therapeutics: Yeah. Absolutely. So what what people don’t realize is that when you numb the eye to give an injection, you just numb the skin of the eye, what we call the conjunctiva. That’s all you know. You don’t numb the optic nerve.
And and the consequence of that is the vision doesn’t change at all. And, you can imagine people notice tiny, tiny floaters. They certainly notice when a huge amount of water or or drug is coming in. So typically, in in clinical trials, and this happens all the time, patients would say, oh, I saw it come in. I see the drug come in.
Because they see a whole bunch of stuff coming in because the optic nerve, again, functions just as well as it always did. And in what you do is you touch the skin of the eye with the hub of the syringe. There’s nothing going in at all. So patients can easily, you know, say, look, doc, you didn’t do anything. I don’t see anything.
And or they’ll say, oh, you injected. I can see the whole thing coming in. So the FDA knew this all along and said, look, sham is not proper masking, period. And they set very clear guidelines in what they what they want. So if you want to go down a regulatory pathway that is risk free, this is what you do for non inferiority.
This is what you do for superiority.
Mark Goodman, Biopharma Analyst, Leerink: Yep.
Pravin Dugell, CEO, Ocular Therapeutics: And we followed the guidelines exactly. And we were rewarded with a SPA with the SOL-one study and written Type C letter for SOL-R. So we’re doing exactly what the FDA wants us to do. We believe that our path for regulatory approval is completely derisked.
Mark Goodman, Biopharma Analyst, Leerink: Right. You nailed both studies, you’re there. Right. Let’s talk about Sol-one. Looking at the percent of patients who make it out to this nine months without rescue, I think this would be interesting.
There’s no rescues. I guess this is kind of an interesting question, right? Because you worry about, I guess, what’s the right word, off protocol rescues, is that the right word? Just somebody who’s just because it’s the right thing for the I mean, how does that work here in this study?
Pravin Dugel, CEO, Ocular Therapeutics: So, let me start with the statistics and the powering, and then we’ll talk about what we see right now. As far as the powering is concerned with very good modeling as well as with studies that are publicly available, such as Talend, for instance, we believe that with a single injection of EYLEA, at most twenty percent of patients will get to that nine month mark. Remember, the primary endpoint remains at nine months, and it’s the percentage of patients who maintain vision, right? We believe that most with EYLEA, that’s going to be twenty percent. Based on our US study, we believe that that’s going to be at least seventy percent with the ex Paxil group.
So the delta is fifty percent, and all we need is a 15% delta to be statistically significant. So we’re quite well powered as far as that’s concerned. On top SPA, what the FDA has said is that as far as rescues are concerned, nobody will be censored. That’s really important. So every rescue, whether on protocol or off protocol, has to be counted for the primary analysis.
And that’s very much in our favor because there’ll be many more of those in the
Unidentified speaker: in the ILEA. Does that
Mark Goodman, Biopharma Analyst, Leerink: work in your favor? Yeah. Is that even fair?
Pravin Dugel, CEO, Ocular Therapeutics: Well, we’re we’re happy to have that. But, you know, there’ll be many more in the ILEA arm than in in the EXPACLE arm. So clearly, as far as the powering is concerned and the statistics are concerned, we are very much favored, I believe. So we’re in very good shape. Now what I have said publicly is, obviously, with SOL one, we’re masked.
But we are our number one priority in this company is the conduct of the trials. And the way we look at the conduct of the trials is by looking at the number of rescues, the cadence of the rescues, and whether the rescues are on protocol or not. And what I’ve said publicly is when we look at those three parameters, we couldn’t be happier. We’re absolutely thrilled. I’ve also said publicly that the vast, vast, vast majority of rescues, and I’ve said that three vast, are absolutely on protocol.
Mark Goodman, Biopharma Analyst, Leerink: Interesting. Okay. And just remind us, like, where are we in enrollment with these studies?
Pravin Dugel, CEO, Ocular Therapeutics: So with Sol one, we have completed randomization in December, and we announced that publicly. With SOL-R, we haven’t guided you as to the most recent updates. What we did say at the time of JPM, which was in January, is that we had three eleven patients who are enrolled in various stages of loading and randomization. Now obviously, with a third of the patients now being reduced, right, because we’ve gone from eight twenty five patients to five fifty five patients in SOL-R based on the recent amendment, we believe that, that randomization, the completion of SOL-R is going to be much faster.
Mark Goodman, Biopharma Analyst, Leerink: Right. Okay. So the timing now of when we’re going to get data for these studies is what?
Pravin Dugel, CEO, Ocular Therapeutics: So again, it’s a little bit too early to say for SOL-R. We will guide you when appropriate, but we haven’t done so as yet.
Mark Goodman, Biopharma Analyst, Leerink: Okay. And Sol one?
Pravin Dugel, CEO, Ocular Therapeutics: And Sol one, what we’ve said because of the amendment, remember, we want to we need to remain masked in order to get the twelve month data. Now what we will have is the card churn in the first quarter of twenty twenty six.
Mark Goodman, Biopharma Analyst, Leerink: So in a sense, Sol one is going to take a little longer and Sol R is going to take a little shorter. And at the end of the game, it doesn’t really matter because they both have to be done to file.
Pravin Dugel, CEO, Ocular Therapeutics: Well, not quite. So Sol R, because remember, we have to keep masking, right, is going to be, instead of being in the fourth quarter of of twenty twenty five, is gonna be in the first quarter of twenty twenty six. So a very slight delay on that. That’s so SOL-R. r.
Mark Goodman, Biopharma Analyst, Leerink: SOL-R, SOL-R, SOL-R, SOL-R, SOL-R
Pravin Dugel, CEO, Ocular Therapeutics: SOL-R. SOL-R.
Unidentified speaker: Right. Right.
Pravin Dugel, CEO, Ocular Therapeutics: Whereas SOL-R now will be reduced in size and will be a lot faster. We haven’t got any as to when that’ll be. However, we need both studies to be able to apply to the FDA.
Mark Goodman, Biopharma Analyst, Leerink: Right.
Pravin Dugel, CEO, Ocular Therapeutics: And the application process because SOL-R now is going to be so much faster, is also going to be a lot faster.
Mark Goodman, Biopharma Analyst, Leerink: Okay, good. NPDR, talk about that program and just a little bit of a saga with it, and big picture and then kind of come down to your program and where we are?
Pravin Dugel, CEO, Ocular Therapeutics: So non proliferative diabetic retinopathy is a huge unmet need. There are about six point five million patients in this country. What we know with that is that these are patients that are effective treatment that’s never used, which is the anti VEGS. That has to be given every month to every other month. And it’s in less than one percent of patients are being treated, less than one percent.
So there’s a humongous need, there’s a well known target, and there’s and, you know, unfortunately, patients continue to go blind.
Mark Goodman, Biopharma Analyst, Leerink: And they’re not getting that And
Pravin Dugel, CEO, Ocular Therapeutics: they’re not getting that It’s not approved. It is approved, but patients just can’t come in every month. And these are asymptomatic patients, and they’re not going to be leaving their work to come in every month or every other month. So effectively, nobody’s getting treated, although there’s a very well known target. Right?
So it’s a fantastic opportunity. What we did was a study called HELIOS, which is primarily a safety study. What What we saw there at week forty eight was that in the control arm, thirty seven point five percent of patients developed what we call vision threatening complications. Right? This is diabetic macular edema or proliferative diabetic retinopathy, which is right in line with the natural history.
I said thirty to forty percent. However, what to me was absolutely remarkable was that in the X Paxley arm, with a single injection of X Paxley, at week forty eight, zero percent of patients, zero percent of patients had vision threatening complications. I mean, that is remarkable. Mhmm. As a physician, I can sit there with a patient and say, if you come in to see me once a year, like you go to your dentist for teeth cleaning, I can reduce your chance of having a potentially blinding condition from thirty percent to forty percent to absolutely zero.
I mean, that is remarkable. So we absolutely will go after non proliferative diabetic retinopathy and diabetic macular edema. And I say and diabetic macular edema because every patient who was enrolled, every single patient who had diabetic macular edema with a single injection improved at week forty eight. And what we’ve done is to show you, not just every single patient, we’ve shown you every single eye. And the more we analyze this, the better and better and better that data gets.
As you saw maybe in the angiogenesis presentation, which was a few weeks ago, when we looked at different layers of fluid, there was clearly a separation in favor of X. Paxley. So we will go after non proliferative diabetic retinopathy and diabetic macular edema. We will meet with the FDA in the first half of this year. We’ve said we’ll do that and we’ll guide you as to the study when appropriate.
It’s a clear and open target for us, and there’s clearly a huge need as well.
Mark Goodman, Biopharma Analyst, Leerink: So there was confusion, you had the forty week data, and then you showed the forty eight week data, right? So what was the confusion?
Pravin Dugel, CEO, Ocular Therapeutics: Yes. So I don’t know there was much confusion.
Mark Goodman, Biopharma Analyst, Leerink: What was the debate? What was the question?
Pravin Dugel, CEO, Ocular Therapeutics: Yes. We unmasked the trial at week forty.
Mark Goodman, Biopharma Analyst, Leerink: Right.
Pravin Dugel, CEO, Ocular Therapeutics: Right? Because the primary endpoint was reached. It was a safety study, and there are absolutely no safety issues, and there have never been safety issues.
Mark Goodman, Biopharma Analyst, Leerink: Yeah.
Pravin Dugel, CEO, Ocular Therapeutics: The data was so good. We said, look, let’s try and follow the patients as much as we can. And the most once you unmask the trial, it’s difficult to follow the patients, right? So we went ahead and followed the patients up to week forty eight and we showed everything.
Mark Goodman, Biopharma Analyst, Leerink: Right. That’s the data that you were just talking. Yes.
Pravin Dugel, CEO, Ocular Therapeutics: Yes. I mean, the week forty is just as good as well. The week forty eight gets even better.
Mark Goodman, Biopharma Analyst, Leerink: Yes. It’s just interesting how like, I don’t know, I guess the response was different, I suppose, people understanding the data better.
Pravin Dugel, CEO, Ocular Therapeutics: I think what you’re talking about is the share price, right? That’s what we’re talking about. Okay. So that’s the confusion. So, yeah, so what ended up happening is very simple.
This was a safety study. Right? And the patient population was really not controlled and was not super selected. And I think what people did was to look at that and say they compared it to what’s called the PANORAMA study, where ILA was given every month or every other month. And they looked at something called a two step difference, right, which is an arbitrary profile that we have in terms of response.
And they looked at that and they said, wow, yours is different. Yours is not as good as Panorama’s. Well, it wasn’t because of a safety study. It wasn’t designed for that. It was it was a small safety study.
Now if it was patients were selected specifically for that, we’re convinced with the data that we have that we can go ahead and and and and be as good. But again, it wasn’t selected for that. That. One should really look at the effect of a single injection over forty eight weeks and the vision threatening complications, as well as if you look at the DRSS changes, everything also there is in favor of
Mark Goodman, Biopharma Analyst, Leerink: the drug. Yes. Let’s come back to Solon and SolR. And obviously, you need both studies to be successful to file whatever. So talk about, okay, the first one comes out, how do we think about the second one with respect to de risking and
Pravin Dugel, CEO, Ocular Therapeutics: Yeah, it’s a great question. Success. So we are convinced with a lot of work internally that with a positive Sol one study, the chance of SOL-R being successful is through the is through the roof. And we’re absolutely confident of that. And there are many reasons why, but the what it really boils down to is if the drug is gonna last for nine to twelve months, why would it not last for six months?
And that’s really what it is, it’s repeating every six months in SOL-R. So we’re very confident that with a positive SOL-R one, we’ll have a positive SOL-R.
Mark Goodman, Biopharma Analyst, Leerink: Yes. And so I guess right now, you’re thinking the second study will be when again? Because the first study will get the data, as you said.
Pravin Dugel, CEO, Ocular Therapeutics: So the first study, SOL-R, will have the card turn in the first quarter of twenty twenty six. And the second study, SOL-R, we haven’t guided you that as yet. But we certainly feel they’ll be much faster now with the amendment.
Mark Goodman, Biopharma Analyst, Leerink: Right. Okay. Got it. Got it. What have we not hit on in our last couple of minutes?
What else?
Pravin Dugel, CEO, Ocular Therapeutics: Well, I think the most I feel the most misunderstood or not understood part of our trial designs is the patient selection. What we do not do in retina is we don’t talk about patient selection all that much, but we do realize that we have in wet macular generation a extremely variable diverse disease population. Some patients are treated every month, some patients are treated every six months. We do something called treatment extend. When you’re doing massive studies, like we used to do, with 2,000, three thousand patients, you know, it comes awash.
But when you’re doing studies of our size, it’s extraordinarily important to pick the proper patients to allow the best chance of success in your study. And I think that’s what we’ve done with a great deal of thought and in a bespoke manner for each study. In a superiority study, what we’ve done is pick patients with very good vision, who have the most amount of VEGF receptors. We’ve tested those receptors to make sure they function by requiring an improvement of 10 letters or vision visual acute of twenty twenty.
Unidentified speaker: Mhmm.
Pravin Dugel, CEO, Ocular Therapeutics: So it’s the perfect patient population to go ahead and load up and allow the drug to fail once. Right? Perfectly selected for that. It’s very different than SOL-R, which is a non inferiority study. Here, what you want is absolute stability.
And what we have is three loading doses. And then we’ve put in something that nobody else has, which is two opportunities to observe the patient. And what are we observing for? We’re observing for fluctuations. Because we want stability.
We have two opportunities to weed out unstable patients. We have two more, injections of loading, and then we randomize. It’s much harder to go through that kind of patient selection. It’s much easier to just let everybody come in. But what we’ve done, and what I hope people understand, is that we’ve reduced all these variabilities painstakingly, derisked the patient population and increased the chance of success of the study in a bespoke manner for each study.
Mark Goodman, Biopharma Analyst, Leerink: Yes, I’m glad you brought that up. And then just lastly, just give us the cash position, just so we understand. What we understand.
Pravin Dugel, CEO, Ocular Therapeutics: What we said in our earnings call is that we’re well capitalized into 2028. So certainly, the completion
Mark Goodman, Biopharma Analyst, Leerink: of both studies and okay, good. Thank you. Thanks for joining us.
Pravin Dugel, CEO, Ocular Therapeutics: Thank you, Matt. Thank you for the opportunity.
Mark Goodman, Biopharma Analyst, Leerink: Thank
Pravin Dugel, CEO, Ocular Therapeutics: you so much. Yes. Good to see
Unidentified speaker: you.
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