Ocular Therapeutix at Needham Conference: Strategic Moves in Eye Care

On Monday, 07 April 2025, Ocular Therapeutix (NASDAQ: OCUL) presented at the 24th Annual Needham Virtual Healthcare Conference. The company, led by CEO Pravin Dugel, shared ambitious plans to transform treatments for wet macular degeneration, despite facing challenges like high patient dropout rates. Their innovative product, Axpaxli, aims to address these issues with advanced drug delivery technology.

Key Takeaways

• Ocular Therapeutix is focused on revolutionizing wet macular degeneration treatment with Axpaxli.
• The company has a strong financial foundation, with $392 million in cash to last through 2028.
• Phase 3 trials, SOUL-1 and SOLAR, are strategically designed to maximize clinical insights.
• Promising early data for Axpaxli extends to diabetic retinopathy and macular edema.
• A collaborative relationship with the FDA supports efficient regulatory progress.

Financial Results

• Ocular Therapeutix ended the last calendar year with $392 million in cash and cash equivalents.
• This financial cushion is projected to sustain operations through 2028, covering the completion of SOLE programs.
• The company maintains financial discipline, ensuring a strategic allocation of resources.

Operational Updates

• The Phase 3 program includes two complementary studies: SOUL-1 (superiority) and SOLAR (non-inferiority).
• A recent amendment to SOUL-1, approved by the FDA, extends the study to 12 months, enhancing long-term data collection.
• Patient rollover between studies improves logistics and enrollment efficiency.
• SOLAR's sample size reduction accelerates the timeline to reach primary endpoints.

Future Outlook

• Ocular Therapeutix plans to submit regulatory approvals based on SOUL-1 and SOLAR results.
• The company aims for a differentiated label, potentially offering flexible treatment intervals.
• Axpaxli's commercial potential is significant, with aspirations to become a first-line treatment.
• Discussions with the FDA for expanding Axpaxli's use in diabetic retinopathy and macular edema are planned.

Q&A Highlights

• Ocular Therapeutix has a collaborative relationship with the FDA, supported by a Special Protocol Assessment and a Type C response.
• The company prioritizes adherence to FDA guidelines to minimize risks in the regulatory pathway.

In conclusion, for a detailed understanding of Ocular Therapeutix's strategic initiatives, refer to the full transcript below.

Full transcript - 24th Annual Needham Virtual Healthcare Conference:

Serge Belanger, Healthcare Analyst, Needham and Company: Good afternoon. I'm Serge Belanger, one of the healthcare analysts at Needham and Company. Wanna welcome everybody to Needham's twenty fourth annual healthcare conference. For next fireside chat session, we have Pravin Dugel, the president and CEO of Ocular Therapeutix with us. Before I hand it over to Praveen to give us an intro and kind of a overview of the company for those who aren't familiar with Ocular, just wanna highlight that we will take q and a.

You can submit questions via the portal that you're watching the presentation on and we'll take them as they come in. So with that being said, I'll hand it over to, to Praveen.

Pravin Dugel, President and CEO, Ocular Therapeutix: Suresh, thank you very much again for for inviting us here. It's it's it's a pleasure and it's an honor to be here. Anytime we get to tell our story, especially when things are turbulent like this is is is a great opportunity. What we are is a retina focused company. This company has changed a great deal in the last year.

And what we what we're doing is to capitalize on an opportunity, that is there, and has proven to be there with with other indications that we've seen, over the last decade or two. The fundamental part of what we do is wet macular degeneration, and the problems that we're trying to solve are two problems. One is, the fact that, as effective as the treatments are right now that are commercially available, the anti VEGFs, The tragedy, which we think is unacceptable, is that forty percent of patients in this country, end up dropping out of treatment and going blind because of sustainability issues. So that's one problem that we're trying to solve. The second problem that we're trying to solve simultaneously is that even those patients that stay on treatment eventually end up getting worse than baseline, and that's not because of rebleeding, but rather because of fibrosis.

And that's felt to be because of the episodic pulsatile nature of our injections, which is on a monthly or bimonthly basis. And that's the second problem we're trying to solve. So what we're trying to do with our product, ex Paxley, is solve the problem of sustainability as well as better long term visual outcomes.

Serge Belanger, Healthcare Analyst, Needham and Company: So as you mentioned off the top, you know, there is a a change in in management at the company last year, kind of this change in focus. So just what brought about those changes and the refocus of the company on on retinal diseases?

Pravin Dugel, President and CEO, Ocular Therapeutix: Well, it's simple. It's it's it's a matter of the opportunity and the technology. I had known this technology from my previous company, where we did a very, very deep dive independently with two different organizations based on the best sustained delivery delivery format and both independently came up with this Elutex technology. So I'd known about the technology. I'd known about the studies that were done by this company prior to me being here, in Australia and The US.

And as someone that has practiced, for more than thirty years, I I certainly understand the need that's out there. And from a commercial point of view, I I certainly saw the you know, historically, what has happened if you look at the success of EYLEA over Lucentis or Bibismo, you know, over over Lucentis and EYLEA, it's not based on increased safety or even increased efficacy. It's really based on increased durability for about a week, couple of weeks, that kind of thing. So the thirst is certainly there for technology that, has has has a position for increased sustainability. And that's the reason that I'm here because I knew the technology, and I certainly recognize the opportunity.

Serge Belanger, Healthcare Analyst, Needham and Company: Maybe let's talk a little more about that the Lutix technology. Obviously, it was developed in house, but, you know, how it differentiates AxePaxly versus some of the other extended duration programs that are in development.

Pravin Dugel, President and CEO, Ocular Therapeutix: Again, there are two components. Right? First is the TKI itself and the second part is the format. The TKI we know scientifically proven is that it is the most potent TKI being investigated by about 200 fold and also the most selective. So so the TKI is well proven and well known.

As far as the format is concerned, the LUTIX technology, this is not new. This has been FDA approved in other indications, systemic indications, and, you know, literally hundreds of thousands of patients have been exposed to this, including in the eye on the eye itself, which is with our, DEXTENZA technology. The the the the the special sauce here is that it's a tunable hydrogel, and it's the same hydrogel, which is DEXTENZA that we use in EXPAXLETE. In DEXTENZA, it's it's tuned to last for a month. And ExPaxly, of course, is tuned to last for ten months.

And also the beauty of this is that the PK and PD are very closely matched so that when the drug is gone, the the the format is gone, the delivery system is gone. In other words, there's nothing left behind. There are no carcasses floating around. It simply is is tuned to be both gone at the same time. As far as safety is concerned, which is really one of the first things that I looked at, and to me, the most important thing before I joined this company, the safety profile has been absolutely spotless.

As I say, it's not just spotless in terms of the ocular technology, but also in the systemic technology. Perhaps to me, what was most important, is actually our glaucoma data, which is the PACSTRAVA data. The hydrogel is exactly the same. And here deliberately PACSTRAVA is placed at what I think is the most susceptible part of the eye, which is the angle, and that's directly behind the corneal endothelium. As you know, the corneal endothelium is a, neuroectodermal monolayer that doesn't regenerate and is exquisitely sensitive to any kind of toxicity, any kind of inflammation.

And once the corneal endothelial layer is lost, there's corneal edema. So with this with PEXTRAVA, we actually measured the number of corneal endothelial cells as well as corneal edema, and there was no difference. And to me, that is the ultimate test of the eye of safety, placing this hydrogel in the most vulnerable position for the eye and seeing a completely clean safety profile. Okay.

Serge Belanger, Healthcare Analyst, Needham and Company: And so far there's been two phase one trials conducted with XPaxli. So, you know, one in The US, another in Australia. So maybe speak to those and what you learned from each one that makes you believe that Xfaxine can be a treatment paradigm shifting product.

Pravin Dugel, President and CEO, Ocular Therapeutix: Well, first of all, I already addressed these, the safety issue and and what we didn't see was any change in that whatsoever. The safety profile, which we didn't expect, of course, in the study in Australia or in The US. I think the take home message on the study in Australia is that this is the first and only time that I know where a sustained delivery TKI has been used as monotherapy in a treatment naive patient. In other words, with nothing at all completely naked. And what we saw were responses from these patients and again, treatment naive patients used as monotherapy that you would expect to see for commercially available anti VEGF.

So in my mind, there was no doubt that there was effectiveness of this that would be comparable, immediately to, the commercially available anti VEGF. In the study in The US, it was used in comparison to, to Eylea, very closely designed to what we're seeing now in the SOLAR study. Although it was done in a completely non enriched patient population, and that's something that I'm sure we'll talk about, later on. And here, what we saw were results that were remarkably encouraging, which is that at six months, a hundred percent of patients, literally a hundred percent of patients were rescue free, and at ten months, eighty percent of patients were rescue free. And we feel very, very good about both studies, but in direct relationship to the SOLAR study, we feel particularly good because remember, in SOLAR, we're doing a similar design in a in a specifically enriched patient population, enriched for stability.

And we're very confident the results based on The US study that has already shown results that are remarkable based on an unselected patient population.

Serge Belanger, Healthcare Analyst, Needham and Company: Yeah. And then for the phase three program, you guys took a very unique approach, at least one unique because it hasn't been adopted by by others in in the space. So maybe just talk about why you chose this path and was it a function of the axSpAxig profile that you would see it in the phase one trials or it was just another opportunity to seek additional differentiation?

Pravin Dugel, President and CEO, Ocular Therapeutix: Yes. So so look, that's probably one of the things that I'm most proud of, and I take absolutely no credit for it because it was not my design. It was the team around me, and I'm very, very, very proud of the team. I think they're the finest team ever assembled in this planet. And I know that sounds arrogant, but I believe that that's true, as far as retina is concerned.

They're the ones that designed this. What I would say is the knee jerk reaction, as you know, for decades and decades and even now is to have two studies because two studies are required for approval, but that that that are the same. Right? But when you think about it, that is terribly inefficient. I know it gets the approval done if both are positive, but the second study really adds no information whatsoever.

It's simply a repeat. So what the team got together and designed is what I believe will be really the standard from this point on where two studies are designed in a complementary fashion. And the FDA loves this because, as you know, what the FDA wants to do is to make sure that whatever conclusion was reached was reached scientifically and not by accident. And there's no better way to do that than to have two different study designs reach the same conclusion. So they love that complementary aspect to this.

That's what we did. We designed a superiority study named SOUL one and a noninferiority study named SOMAR. And the noninferiority study, I think, for a lot of people was a surprise because everybody expected a SOUL two, which we didn't do. The advantages of of doing this are many, and we've already realized most of them. The first one is logistic, which is that we don't lose any patients.

Everybody whose screen failed in SOUL one immediately rolled over to SOLAR, And that's why we had such a bump in enrollment immediately after SOUL one was completely randomized. So logistically, it worked superbly. From a commercial strategic point of view, it really answers all the questions that physicians and patients have. We have the potential, the potential of having the first and only superiority label, that is that is flexible, six months to twelve months of treatment, as well as repeatable. There also answers questions regarding a head to head with Eylea two milligram.

Also, numerically relevant information as to Eylea as to as to head to head with Eylea high dose, which which we should talk about as well. From a regulatory point of view, and I think this is the part that has been underappreciated, remember that no regulatory agency is going to see either study without the other. So the fact that that that the regulatory agencies will see how long this drug lasts based on SOL one and how it compares to the standard of care based on SOLAR is immensely powerful in terms of having complementary information together. So it's I think it's a unique and and absolutely, trend setting and new design. Again, I take no credit for it as the team, And I really believe that this will be the standard henceforth.

Serge Belanger, Healthcare Analyst, Needham and Company: I I agree about the advantages. I'm I'm curious why you think other companies have not adopted the same approach or are they just waiting to see on whether it's successful for Rex Paxley and then will become more of a trend going forward?

Pravin Dugel, President and CEO, Ocular Therapeutix: Well, it's a good question. But, you know, look, there there are certain things that need to happen. First of all, in a time like this, I couldn't be happier with what we've done. Alright? In a a time of uncertainty like this, the fundamentals matter, and we've done everything fundamentally correct.

The most important thing probably is the regulatory pathway, and that matters. The fact that we have a spa, the fact that we have written types of agreement, those kind of things really matter at a time like this. The fact that we didn't use sham, the fact that we've got two complement complementary studies that that help each other out in terms of answering all the questions. All those fundamental things that we have are what's gonna be what's gonna shine, especially in a time like this. Why others have done it?

No. I can't answer. But if you look at the superiority study design, right, you may ask why haven't others done that because exactly what the FDA wants. There is no sham. It's a it's not a design that that we made.

It's a draft guideline that the FDA had to say, look. This is what you should do if you if you want a superiority study. Well, the one thing I know is that you have to be convinced that your drug lasts for nine months. Right? Otherwise, you wouldn't be doing that study, and we certainly are.

Not only are we are we convinced that it lasts for nine months, we're convinced that we that we have the potential of getting a twelve month label. That's why we had the recent amount, announcement, the recent amendment that we spoke about. So there may be many reasons others aren't doing it. But fundamentally, you have to be convinced of your technology to follow these FDA guidelines.

Serge Belanger, Healthcare Analyst, Needham and Company: Yeah. And these guidelines are really just a recommendation or is the FDA pretty rigid for companies to follow them in their clinical development plans?

Pravin Dugel, President and CEO, Ocular Therapeutix: Well, you know, I think that's where there's been a fair amount of confusion, Serge. I mean, I think others have said, look, you know, we've gone to the FDA and they've said, you know, sham is fine and they haven't stopped us. It must be okay. Well, the FDA is not gonna stop you. Right?

The FDA is gonna only stop you if there's a a safety issue. And and having sham, there is no safety issue. It's a masking issue. So, of course, they're not gonna stop you. But the the decision that we've made is that we're simply not gonna do anything at risk.

When you do something that that veers away from the FDA guidelines, then you're doing it at risk. And we're not going to be doing anything at risk. We've made that fundamental decision internally. Now it does mean that you have to believe in your technology. It does mean that maybe harder to recruit, but it also means that risk is removed from the table.

And that's why everything that we've done, with with a spy, as you know, in SOL one, and and with a type c written, response, in SOLAR has been exactly what the FDA wants, We've done that fundamentally without risk.

Serge Belanger, Healthcare Analyst, Needham and Company: While we're on this subject of the FDA, just curious if you're aware of the recent changes at the agency have impacted the, ophthalmology division.

Pravin Dugel, President and CEO, Ocular Therapeutix: You know, so so here's what I know. We are we are very, very, very happy with the collaboration that we continue to get with ophthalmology. Nothing has changed, and we feel particularly good for the reasons that we've mentioned. The fact that we're doing everything exactly the way they want us to do it as validated by SPA, as validated by the Ribbon Type C response makes us feel even better. And if you want proof of that, again, we haven't talked about the recent amendment, and I'm I'm sure we will.

But but but think about how many programs that you know. And, again, all I know is RET and some some a bit of a one trick pony, but you may you may or may not know some, so I'm asking you this. That in the middle of a phase three program like SOAR one, there has been a major amendment in the study design while not changing the SPA whatsoever. For the FDA to be able to do that with with the sponsor, I think a major take home message there is, boy, you must have great collaboration with the FDA in order to allow that to happen. Right?

Remember, in that amendment, we didn't give up anything. We're gonna get to the finish line faster, cheaper, with potentially a better label, and that has to speak to our collaboration with the FDA. Otherwise, I don't think that they would have allowed that change to occur while making sure that the spa was completely protected. So I think if you want proof of our relationship with the FDA about how important it is to make sure that those guidelines are followed in terms of collaboration with the FDA, I think that is it.

Serge Belanger, Healthcare Analyst, Needham and Company: Yeah. Yeah. And to to answer your question, we've I've seen changes to phase three programs, but not as part of a SPA. Feel like the SPAs are kind of we don't we don't see them as often as we we used to. So it's interesting that your program is supported by a spa.

I think it's a positive.

Pravin Dugel, President and CEO, Ocular Therapeutix: I think especially at a time like this. I mean, you know, I one of the things Sergeant Yeah. That feedback that we got and perhaps it was a bit of criticism, before the the the uncertainty see we see now is, look, why are you doing all this? It's just not necessary. You know, why they need to go and get a spa?

Why do you design a similar study so that, you know, you've got this third masking arm? Why do you need to do all those things when it's really not necessary and others aren't doing it? And our response was always that, look, we're just not gonna take any risks. And, you know, as as you know better than anybody else, look, drug development is not linear. You're you're blindsided by things, you know, macroeconomics change.

And this is the time the in in a time like this, this is when we feel very, very good that we've done everything we've done, you know, right down the fairway in terms of checking all the boxes.

Serge Belanger, Healthcare Analyst, Needham and Company: Yeah. I think a spa is a prudent approach when you're taking a a new unique path to to approval. Right? Yeah. Makes sense.

Pravin Dugel, President and CEO, Ocular Therapeutix: So when when when you're taking risks, when you're you're when you're going when you're veering off the guidelines and you're sort of going off the range, doesn't mean you can't do what you do, but it just means that the risks become amplified in a time like this. In a time like this, when you're doing everything per the guidelines, I think you're rewarded, and I think that we feel very good about that.

Serge Belanger, Healthcare Analyst, Needham and Company: Yeah. So so let's talk about the the most recent amendment to SOUL one. I think it kinds of extends, pushes out the readout a little bit, which shortens the regulatory timelines. But yeah. How it came about and why it's important.

Pravin Dugel, President and CEO, Ocular Therapeutix: Oh, let me just get to the bottom line. The bottom line is these amendments allow us to get to the finish line faster, cheaper, and potentially with a with with a better label, the best that I think anybody has ever seen in our field. But let me just tell you how it happened and why it happened. Before this team was here, the the previous team, to their credit, recently received a spot, based on SOL one. And SOL one concluded at month nine.

There was nothing afterwards. And what we heard from the investigators is to say, look. You're putting me in a bit of a difficult position because once sold one is successful, I have nothing for my patients until the drug is in the market. Right? So so I just simply have to tell the patients, look.

You're you're done. And these are patients that volunteered to be in this study because they were so excited about the product. And you're you're you're you're putting in a position of having a difficult conversation with these patients, and they were right. So that's number one. Number two was we saw what happened with drugs such as Vibismo where they received a a a duration in their label, Vibismo four months, as you know, based on very few patients actually crossing the threshold.

So very few patients actually have to cross the threshold to get that on the label. And we looked at that and said, wow, there's no doubt in our mind that with ex Paxley at twelve months, patients that number of patients will clearly cross and certainly more. So that was the second thing. The third thing, and this was the gating factor because one of the things that people ask me all the time is, look, if your team is that smart, why don't you do this right away? Well, there was a gating factor, and the most important gating factor was patient retention.

And all of us who've been doing this for twenty, thirty years in the team, and we've got, really centuries of experience in the team, collectively, we looked at each other and said, we've never seen patient retention. The thirty or so years we've we're doing this that is this good and referring to SOLA-one. So based on those three things, we we had a discussion with the FDA, and we basically said, look. All of SOLA-one, the eight hundred and twenty five patients originally there is based not on powering, but based on the safety requirements for the FDA for unrestricted label according to how many patients they need at year two. Because as you know, in so one, as I said, all the patients are gonna end at month nine.

So that's why we have to have a very large patient population in SOLA one, not because of the powering, but because of the safety requirements. So we asked the FDA, we said, look, we don't wanna jeopardize the STOP. We we don't wanna jeopardize the spa at all. But can we go ahead and have a year or two where SOLO one becomes a very efficient study? First year, with a single injection, we look at the durability of the product.

In the second year, with every six months injections, we look at the maximal exposure and safety of the product. And they said yes, without jeopardizing the spa. They said yes. And then we said, if we can do that, can we then reduce the size of SOLAR from 825 patients to 555 patients because that will satisfy the safety requirements? And they said yes.

So what we have the potential of, and this is this is why the readout for SOUL one is delayed by a quarter, is that if we keep these patients masked, right, we have the potential of including month twelve in the label. And that's why we had to delay it by by a quarter because we wanna have every opportunity possible by keeping it masked. Because if we if we unmask it, as was originally designed, in the end of twenty twenty five, we wouldn't have any opportunity of having month 12 on the label. But because we're keeping these patients masked, we certainly have that potential of month 12 on the label. So again and and and we gave up nothing.

That's important to understand. The the primary endpoint remains exactly the same. The powering remains exactly the same. We gave up absolutely nothing, and we have now the potential of having the best label there ever was in this in this field, getting to the finish line faster and getting to the finish line cheaper.

Serge Belanger, Healthcare Analyst, Needham and Company: Okay. That makes sense. And then for the primary endpoint, if you can just talk about the the powering around that endpoint at the the nine point it's gonna be nine months against EYLEA. Just what kind of difference you're looking for?

Pravin Dugel, President and CEO, Ocular Therapeutix: Yeah. So so so based on our analyses with EYLEA with a single injection, and there's some public, public, information regarding this specific LATALEN study, we expect about twenty percent of patients with Eylea will get to the endpoint. And the endpoint, as you know, is at month nine is the proportion of patients maintain vision. If patients are rescued at any point, they lose 15 letters or more vision, which would be a net of at most five letters of vision. We expect with ex Paxley, again, in a in a non enriched patient population, and this is this is The US study, that that will be at least seventy percent.

So fifty percent delta, and all we need is a 15% delta one five to be statistically significant. Now realize, one of the things that I think that's really important is that we have very carefully enriched our patient population. These are VEGF dependent patients. These patients are designed to be VEGF dependent so that they're designed to fail. And all that matters at the end of the day for the success of this study is the delta.

The delta between a single injection of ex Paxley and a single injection of Eylea in the most VEGF dependent selected patient population that we can find. So, again, a very enriched patient population specifically selected for this study design. Also remember that everybody's counted. There are no patients who are censored. So there will be patients that will be rescued off protocol for sometimes appropriately, obviously, that may not have lost 15 letters of vision.

But everybody who is rescued, our SPA dictates must be counted. Nobody is censored. Obviously, that's very much in our favor, because there will be many more of those patients who are rescued, either on protocol or off protocol who are on Eylea versus axpaxil. And finally, what I have said is that under masking, and obviously we're still masked, what I've said is that the the most important goal of this company is the conduction of these studies, specifically solverone. And what we what we study are three parameters.

One is the number of rescues, the other is the cadence of the rescues, and the third is whether those rescuers on protocol or not. And what I've said under masking is that we couldn't be more thrilled with what we're seeing now. I've also said that the vast, vast, vast majority of patients who are being rescued are being rescued on protocol, and we're thrilled with what we're seeing under masking.

Serge Belanger, Healthcare Analyst, Needham and Company: Okay. Solar. So it's part of the amendment. You were able to reduce the size of it. I don't know if you have any update on where enrollment is or what was the last update?

I think it was in early January.

Pravin Dugel, President and CEO, Ocular Therapeutix: Yes. You're you're right. In early January, what we said was we had slightly over 300 patients in SOLAR through n eleven. These patients, you know, so essentially, we already had a bolus coming in from SOLAR one once SOLAR one finished randomizing. And we are recruiting SOLAR extremely well.

I couldn't be happier. We haven't updated it as the guidance as yet. But clearly, what's happened with our recent amendment is that that delta of the card term between SOL one and SOLAR now has been greatly reduced. And remember, we need both studies in order to submit to the FDA for approval of ex taxly. And the primary endpoint for SOLAR is week fifty six.

And week 56 now will come much, much earlier because we have a reduced sample size. Right? Not 825 patients, but rather a third of that, only 555 patients. So as soon as we hit the primary endpoint in week fifty six, we will go ahead and submit for approval based on both studies, and that timeline has been much accelerated with the recent amendment.

Serge Belanger, Healthcare Analyst, Needham and Company: Okay. But the the sole one primary endpoint readout really will derisk whatever comes from sole r. Is that the right way to

Pravin Dugel, President and CEO, Ocular Therapeutix: We we absolutely believe that. And and there are many reasons for that. But, really, the primary reason that the simplified boil down reason is, look, with a positive SOL one, you absolutely derisk SOLAR because the probability of success for SOLAR is sky high with a positive SOL one. And the the the simple logic is, look. If there if there's a drug that lasts for nine to twelve months, why would it not last for six months?

Serge Belanger, Healthcare Analyst, Needham and Company: Yep. Okay.

Pravin Dugel, President and CEO, Ocular Therapeutix: And remember also that we we we're very fortunate in in not only having a singular nonblended primary endpoint at week fifty six, but we are also very fortunate to have a noninferior margin of the maximal that the FDA allows, which is 4.5 letters.

Serge Belanger, Healthcare Analyst, Needham and Company: Yeah. So from an label standpoint, these two trials, as we talked earlier, could support a differentiated label.

Pravin Dugel, President and CEO, Ocular Therapeutix: Oh, well, absolutely. I mean, you know, I mean, not only a differentiated label, but also the amount of information that the doctor and the patient will have. Right? So, again, obviously, we haven't had I've gotta say this, we haven't we're not we haven't had labeling discussions with the FDA where where this is too early for that. But but, you know, having said that, there's a potential for the only superiority label in this field, the only one, you know, based on SOLO-one, a label that allows flexibility of treatment every six months based on SOLA r to every twelve months based on SOLA one, and repeatability based on SOLA r.

I mean, so I believe that we're in great shape from a strategic point of view for for a very, very impactful and and and and very good outcomes in terms of our labeling discussions. The the other thing that I also wanna mention is, look, and I think this this this has been underemphasized. That is the masking arm of SOLAR. Remember, we have no sham as per the FDA's requirements, right, in either study. So if you don't have sham, and and and the FDA has repeatedly said sham is not proper masking.

And and and if sham is used, it's at risk, and we're not doing anything at risk. So we need a masking arm. We could have chosen anything for the masking arm. It could be water. It just simply has to be the same cadence as your primary drug and the same rescue criteria as your primary drug.

We chose high dose Eylea for competitive advantages. Now that is not for statistical analysis. That's important. It's not for statistical analysis. It's for numeric analysis.

And it will potentially provide us a great commercial advantage in terms of the information that we have from that. Again, for numeric analysis only and for a for a commercial advantage in terms of our comparison with high dose. We're very happy to compare ourselves with high dose. You know, may add another week or two weeks at most. But we're just in a different orbit altogether.

We're talking about nine to twelve months, not just weeks. And if you think about the thirst out there for anything that extends even a little bit, you can start way back by looking at EYLEA versus Lucentis, which may be two weeks to now, Vibismo versus, you know, Lucentis, which may also be two weeks or so. But look at the revenue growth in terms of Vibismo based only on that. I mean, it's not safer. It's not more efficacious.

It simply lasts two weeks longer. And again, we're in a completely different orbit. So we feel very, very, very good about our, commercial potential.

Serge Belanger, Healthcare Analyst, Needham and Company: Speaking of that commercial potential, I guess, with with your label, you have the potential to play a frontline role similar to an EYLEA. Is that where you expect to to play or it's more of a maintenance role post initial EYLEA?

Pravin Dugel, President and CEO, Ocular Therapeutix: Yes. So so, Serge, that's a great question. And, you know, what I would say flippantly, and I'm not saying this right, but but I'm I'm just sort of telling you flippantly, is is to say, like, why do I care? Right? Even even in the worst case scenario, if this is a maintenance drug, it will be the most impactful drug ever in the history of our field.

Having said that, look, I believe it's a first line drug. And and why do I say that? I I say that for two reasons. One is based on the studies that we've some of them we've presented in Arvo, for instance, that steady state occurs within 24 hours. Also, of what I mentioned earlier on, the study in Australia where it was used as monotherapy in treatment naive patients and the results were what you would expect to see from commercially available, anti VEGF.

But I will tell you, look, we have been intensely focused on the ramp with the loading phases and patient selection in both SOLA one and SOLA r. The reason for that is not biologic because we don't feel that we're it's needed to have an anti VEGF for loading. The reason we've done that is for patient selection is because we wanna enrich the patient population and give the trials in a bespoke manner the best chance of success. That's why we've done it. So, again, it's not for biological reasons.

It's for patient selection. And we've done that in, I think, the most thoughtful way possible that nobody else has to have exactly the right patient population for the trials that we've designed. So the bottom line is, look, others or we will do that study to see, you know, is this a first line drug? Is is it comparable to the anti VEGF? But we're not gonna dive on the hill for that.

Our job here is to get this drug approved, and we're very comfortable with the fact that even in the worst case scenario, which we don't think will happen, even in the worst case scenario, if this becomes a maintenance drug, it will still be the most successful drug ever in this field.

Serge Belanger, Healthcare Analyst, Needham and Company: Yeah. Okay. And then thinking beyond wet AMD, I think you've started looking at NPDR and even DME. I think the next step there is to meet with the the FDA. So I'm sure we'll get an update on the the next quarterly update.

But would you go and are those two separate tracks? Do you move to phase two or is it straight to phase three on on those indications?

Pravin Dugel, President and CEO, Ocular Therapeutix: So the first of all, let's look at the data. The Helios data was just phenomenal in my opinion. It's not what I expected. It's not what any of us expected. It was a pure safety study.

It was not a specifically selected patient population. It wasn't an enriched patient population. It was really all comers. And the fact that every single parameter, and I mean every single parameter was aligned in favor of the drug, is absolutely remarkable. I mean, that that's something that I really haven't seen in any study in which patients were quite unselected, particularly in a study that has the most amount of systemic variabilities.

The most important thing from a clinical point of view is preventing these patients from going blind. Right? We know that in advanced non proliferative diabetic retinopathy, the risk of vision threatening complications is twenty percent to forty percent year upon year. In the control arm, that's exactly what it was. It was thirty seven point five percent.

But if you give a single injection, a single injection of Axpaxil at week forty eight, that rate is reduced to literally zero, and that is absolutely remarkable. That means that if this is duplicated in a phase three study and approved, a doctor can sit in front of a patient and say, you know, your risk of going blind or having a blinding, complication is thirty to forty percent year upon year. But if you come to see me once a year, like you would go to see your dentist for teeth cleaning, I can reduce that risk literally to zero. I mean that is remarkable. And by the way, the other thing also is that every single patient that had diabetic macular edema, because these were selectively non sensory involving diabetic macular edema, Every single patient improved, was treated, every single patient in the control arm got worse.

So we absolutely will target non proliferative diabetic retinopathy and diabetic macular edema. How that will look depends on our our discussion with the FDA. We're on track with what we said, which is that we'll have a discussion in the first half of this calendar year. Right now, that field of nonproliferative diabetic retinopathy and diabetic macular edema is wide open. And you know we don't have any competition in non proliferative diabetic retinopathy.

It's a huge market and we absolutely will go after that.

Serge Belanger, Healthcare Analyst, Needham and Company: Okay. We only have a few minutes left so maybe if I can get you to give us an overview of financials.

Pravin Dugel, President and CEO, Ocular Therapeutix: Well, one of the things that we've done, as you know, is to be very, very disciplined from a financial point of view. In the end of the last calendar year, we announced that we had $392,000,000 in cash and cash equivalents. We also announced that we're on track to go to 2028 from a cash point of view. And that's well beyond the completion of the SOLE programs. Now that doesn't include the non proliferative diabetic retinopathy and diabetic macular edema program because we don't have a final, study on that as yet.

But from a cash point of view, I think we're in very, very good shape, which matters a lot, obviously, at a time like this.

Serge Belanger, Healthcare Analyst, Needham and Company: Absolutely. And then maybe just to wrap up, anything you feel is misunderstood or underappreciated about Baxley?

Pravin Dugel, President and CEO, Ocular Therapeutix: I think, you know, and again, thank you so much for allowing us to be here. I really appreciate the invitation. And we touched on a lot of things, but I think the most important thing that anybody can look at here is how thoughtfully these trials have been designed and how thoughtfully patients have been selected. These patients are the most derisked patients that are carefully selected and bespoke for each study that I've ever seen. Patients that are VEGF dependent in the SOL1 study, which is exactly the kind of patients you want, and patients that are stable, and super selected and enriched and derisked in the SOLAR study.

So that I think is the most important thing, way the patients are selected. And we've already touched on the complementary nature of this trial. Again, I'll I'll end with this, which is at a time like this, the fundamentals that we've had, the boxes that we've checked, the fact that we have a clear regulatory path that's in writing is gonna be extremely important. I think this is where the fundamental shine, and we feel very, very good about where we are.

Serge Belanger, Healthcare Analyst, Needham and Company: Well, let's end it here. Wanna thank you for spending time with us this afternoon and telling us more about Ocular Therapeutix. It's an exciting story.

Pravin Dugel, President and CEO, Ocular Therapeutix: Thank you, sir. Thank you for the opportunity, and thank you very much for having us here. I'm very grateful.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.