Ocular Therapeutix at The Citizens JMP Life Sciences Conference: Strategic Insights

On Wednesday, 07 May 2025, Ocular Therapeutix (NASDAQ:OCUL) presented at The Citizens JMP Life Sciences Conference 2025, outlining its strategic focus on retina-related conditions and the development of its lead product candidate, Ex Paxley. CEO Praveen Duval highlighted both the challenges and opportunities facing the company, particularly in addressing the high patient dropout rates associated with current anti-VEGF therapies.

Key Takeaways

• Ocular Therapeutix is focused on developing sustainable treatments for retina-related conditions, particularly wet AMD.
• The company is conducting two major phase 3 clinical trials, SOUL-1 and SOLAR, to evaluate Ex Paxley.
• CEO Praveen Duval expressed confidence in the regulatory pathway and trial designs, emphasizing adherence to FDA guidelines.
• The company aims to reduce the patient dropout rate by improving the sustainability of treatments.
• Future plans include targeting diabetic retinopathy and diabetic macular edema.

Strategic Focus and Mission

• Ocular Therapeutix aims to improve long-term outcomes for wet AMD patients by minimizing fibrosis, scarring, and atrophy.
• The company seeks to provide sustainable efficacy with Ex Paxley, reducing the need for frequent injections.
• Addressing the high dropout rate of 40% in the US is a key goal, with the potential to significantly impact patient retention.

Regulatory Landscape and Trial Design

• The company is following FDA guidelines meticulously in its clinical trial designs.
• SOUL-1 is a superiority trial targeting VEGF-dependent patients, while SOLAR is a non-inferiority trial for stable patients.
• Recent amendments to the SOUL-1 trial aim to extend it into the second year to meet FDA safety requirements.
• A data readout for SOUL-1 is anticipated in the first quarter of 2026.

Ex Paxley and Clinical Data

• Ex Paxley combines a known TKI with the Elutix drug delivery system.
• Previous phase one studies have shown promising results, comparable to existing anti-VEGF treatments.
• The company reports high patient retention and consistent rescue timing under masking, indicating robust trial management.

Future Outlook

• Ocular Therapeutix plans to expand its focus to diabetic retinopathy and diabetic macular edema, areas with little competition.
• The company is preparing to engage with the FDA on these conditions and expects to complete enrollment in the SOLAR trial ahead of schedule.
• CEO Praveen Duval remains optimistic about the commercial potential of Ex Paxley and its ability to enhance patient outcomes.

For a detailed view of the strategic insights and clinical developments discussed, please refer to the full transcript below.

Full transcript - The Citizens JMP Life Sciences Conference 2025:

John Walden, Analyst: One of the Citizens Life Science Conference for 2025. My name is John Walden, analyst here, and pleased to be kicking off with Ocular Therapeutix, a name we’ve been covering for quite some time. CEO Praveen Duval joining us, And we’re just gonna have a a light discussion here. We’ll save some time for q and a at the end from the audience. But hopefully, something that we can all learn a little bit because this is a very exciting time.

We’re hearing developments in the ophthalmology space, you know, pretty much, you know, at least every month from people developing drugs for, you know, wet AMD, retina specific conditions. But Praveen, maybe to kick off and we can jump into some details is, you know, what is your overall strategy and mission in Ocular Therapeutix?

Praveen Duval, CEO, Ocular Therapeutix: Yeah, John. So first of all, listen. Thank you. Thank you for all the diligence that you do for with with on us, and thank you for all your your support. And then thank you also for inviting us to this this meeting.

So really grateful for that. So the mission is really simple. I mean, look, we wanted to you know, when I came in here, obviously, there’s a lot of things that changed. The company, as you know, changed entirely. But one of the first thing we wanted to do is to have an identity for the company and a mission for the company.

And this is a retina focused company now. It’s not a retina only company, it’s a retina focused company. And we have a drug in ex Paxley and the goal here is is to to have two different things that we do. There are two goals. First, the one that is fairly obvious is we wanna have a drug that’s sustainable as good and the reason is really simple.

As as good as the anti VEGFs are. Right, they’ve been around for almost four decades and they’re phenomenal drugs. What is really tragic, I think, and and should be unacceptable to us is that because of the sustainability issues in this country alone, and it’s just in this country, you know, the richest country on earth really from a medical point of view, forty percent of patients drop out in the first year. Mean just think about it, and those patients go behind. Forty percent.

We have a known target. We have a known treatment, but yet it’s not sustainable. So that’s goal one. Goal two is even the patients that are able to come back and come in on a regular basis, the rest that stay behind, what people don’t realize is that even when they’re able to come back, almost all of them inevitably after two to five years end up getting worse than baseline anyway. And when we saw this, the obvious thing was we think, well, that’s from rebleeding.

It’s not from rebleeding, it’s from fibrosis. It’s from scarring and atrophy. And what we know now is the reason for the scarring and atrophy is that the way we give these medicines in a pulsatile manner, the back of the eye gets thick and thin and thick and thin. It’s neural tissue. It’s kinda like having multiple concussions, right?

Mhmm. And that’s why it scars. So those are the two things that we’re trying to achieve which is to have get better sustainability, right, and more people who are treated and less people who go blind. There’s great opportunity there. And the second one is we’re trying to get better long term outcomes for these patients.

John Walden, Analyst: And it’s hard for me to keep track of time these days. How long ago was it when you joined the company?

Praveen Duval, CEO, Ocular Therapeutix: Yeah. It’s been wow. It’s been a year and a bit. It hasn’t been all that long. It’s it’s it’s a big change in the company.

But again, I think what we’ve done now is to have a identity. We’ve we have a focus. We certainly have the personnel that we want, and it’s all about executing. And and, you know, that’s what we’re focused on. And so far as you know, we’ve been, I think, executing exceptionally well.

John Walden, Analyst: And and there was chatter a little over a year ago when you weren’t with the company where you’d end up. You had a a very successful career in the space and people were wondering, well, what’s next for Praveen? And then you chose Ocular Therapeutix, made a big splash. You know, how’s the report card a year in? How were your priorities?

What drew you to the the company? And then what did you wanna do? And then how’s that been going so far?

Praveen Duval, CEO, Ocular Therapeutix: Yeah. So, John, I mean, mean, honestly, I I had no I I loved what I do. I I love what I do now. I love my previous company. I love every second of the career change I made.

I don’t think there are whole bunch of people that make the career change I made. And remember, for thirty years, you know, I was a practicing retina surgeon. Right? And and so I came into Iveric Bio. I was very happily retired in making wine in that before for all of three months, and I realized that, you know, my wines weren’t gonna be ready till 2027, and I had to find something to do.

So, again, I wasn’t looking but this is this was an opportunity that was just phenomenal and I you know, as confident as I am in the product as I was then, I am even more confident today and I’m sure you’ll ask me seeing what I’m seeing. But the reason is really simple. It’s it’s what I just mentioned to you which is that this is a product that has a it I mean, it’s it’s not something that is a high risk product, believe, because there’s a known target, there’s a known mechanism, it’s proven. And the regulatory pathway, which we’ll talk about I’m sure is completely clear. We’ve done everything exactly according what the FDA wants.

And to the credit of the team before us, they got a spa, the team before us, and they deserve all the credit. So when I saw the product, when I saw the opportunity, having practiced for thirty years before, knowing that this would fit in, you know, like that in the in the office and be adopted like that and knowing the need and knowing that with a spa there was an absolutely clear regulatory pathway forward, it was just an opportunity I couldn’t pass on. And then can you talk a little

John Walden, Analyst: bit about the landscape? You mentioned anti VEGFs, forty percent of people stop, go blind eventually. We have newer drugs in the last couple of years, high dose Alea, Vabismo. Vabismo doing great, high dose Alea trying to catch up. What’s going on in the landscape where we’re seeing these longer duration products?

And then, you know, recently, we just saw a high dose Alea proposed to have a six month label. FDA said, no. Thanks. How important is the longer duration and, you know, what impact do you think that would have on commercial uptake?

Praveen Duval, CEO, Ocular Therapeutix: Yeah. It’s a great question and there are many, many layers to your question. It’s a really profound question. But first of all, let me just again emphasize that 40% number. That is in this country alone.

That that number is way higher outside The US and that 40% number is just is not just made up. It’s been shown over and over and over again in real data. So it’s it’s a verified number. And and to me, it’s astonishing. 40% of people I mean, that’s and and if we can, with our product, increase the sustainability and decrease the dropout rate by even 10%.

That’s a quarter million more people in this country alone that will not go blind. So the potential of the impact of this drug is just absolutely enormous. So I think the main thing is to sort of look at that. The other part also is the regulatory part which I think is what you’re getting to with Bibismo and everything else. The the CRL that Regeneron got is really important from this from the point of view of the statement that the FDA made.

The FDA has made it very clear. These are regulatory guidelines that we want you to follow. If you wanna do a superiority study, this is what you do. If you wanna do a non inferiority study, this is what you do. It couldn’t be clearer.

It doesn’t mean that you can’t go outside the guidelines and if you do, they’re not gonna stop you because it’s not a safety issue at all. It’s it’s it’s a risk issue. Right? It’s it’s a proper masking issue. What that means is that you’re doing a trial at risk and what that what that also means is that even if you hit the primary endpoint, you may not be approved or your bar for success may be much much higher.

We’re doing none of that. We’re taking no risk whatsoever. We have a staff for the first study. We have a written type c validation for the second study. What the CRL shows you, the Regeneron CRL, is to say look, if a study is not properly conducted and properly masked, we’re we’re not gonna approve you.

Mhmm. And I think that actually portends very well for us because we’ve done everything by the books.

John Walden, Analyst: And, you know, if the the bears for ocular, they point to you ran a small phase one study, not a lot of patients, but, know, what gives you confidence that what you’re doing now is, you know, supported by that data? Can you talk

Praveen Duval, CEO, Ocular Therapeutix: a little bit about that phase one dataset? Yeah. Sure. And, John, look, that’s a very fair and very appropriate question. Thanks for asking that.

And and the reason is very simple. It’s the results that you see. Right? If you had enough of this inherited retinal disease, but if you have a inherited disease where kids pass away at six years of age say and you’ve got, I don’t know, 12 kids that have made it to 40, that’s pretty convincing. Right?

Just like that, you know, in wet macular degeneration what happens is that this disease, patients don’t get better by themselves. When we saw what we saw in the phase one studies in US and in Australia and we saw the results that we saw, that doesn’t happen by accident. It’s a binary event. So it’s not like, say, in my previous company, Geographic Atrophy, where you can say, well, you need large numbers there because the changes are micrometers, right? And it can go either way based on patient selection and so on and so forth.

That’s not the case here. Either it works or it doesn’t work. It’s really a binary decision. You can see that right away. And more importantly, the way the studies were conducted and what the studies show is really, really important.

The first study was a dose ranging study and this is the one in Australia and the significance of that is that it’s the only time that I know, the only study that I know, and this is really important, where a sustained delivery medication was used as monotherapy in a treatment naive patient population. Our competitors haven’t done that. They’ve always combined that with a commercially available anti VEGF so it’s hard to tell which one is having what impact. Here, this was entirely naked. It was used by itself in a treatment naive patient population.

What I would say is the results that we got were what you would expect for commercially available anti VEGF. And that doesn’t happen by accident. You know, that’s not something that just happens by chance. And then when we did the study which was a controlled study in The US, what we saw were the rescue squeeze rates which were very very convincing to us versus EYLEA. And I think that’s that’s when we made the decision to say, look, this is enough evidence for us to go to a phase three study.

So that’s really the reason. And when we look at

John Walden, Analyst: the data across kind of the class, the tyrosine kinase inhibitors, it gets difficult. You know, there’s different patients, different criteria, study designs. What you know, to your point that you’re only ones who have shown this data, if we don’t see the data, it’s hard to guess, but what is it about expaxly that could be different than the others in the class?

Praveen Duval, CEO, Ocular Therapeutix: Yeah. So I think I think again, great question. I think there there are two ways that I two ways that I’d like to answer that. First is the product itself, and the other is the study design and patient selection. As far as the product itself is concerned, the TKI is a well known TKI known to be the most selective, most potent TKI.

The Elutix technology again is not something that is new at all. It’s been used, approved by the FDA and over 5,000,000 patients have used it, obviously not just in the eye but elsewhere as well. It’s been used in neurosurgery, urological surgery, So we have a approved format which is the LUVIX technology, an FDA approved format and we have an approved TKI. So we are very, very content and confident that these two products, the way we put them together, are gonna work exactly the way we want them to work. And in fact, what we’re seeing under masking now, which we’ll talk about in just a little bit, validates what I’m saying right now.

So now let me just push the next one, which is to say, how are these studies conducted? I think one of the most important things that’s missed in our field, and this is sort of my biggest talking point that I can give you, is the single most important thing that anybody can ask me is something that very people have what nobody’s really ever asked me directly, is to say, tell me what you have done in your clinical trial design to derisk the patient population and increase the chance of success. And that

John Walden, Analyst: is the most important thing. That was on the list. That was on the list.

Praveen Duval, CEO, Ocular Therapeutix: I saw it. Yeah. I was on the list and that was you. And the reason is really simple. If I sat here with you and said, John, I’ve got a great drug for colon cancer, you wouldn’t let me get away with that.

Right? You say, what do you mean colon cancer? What kind of colon cancer? What stage? What grade?

So I’m here to tell you that wet macular degeneration is no less variable than any of the other oncologic diseases that you know. Be it colon cancer, bladder cancer, breast cancer, lung cancer, all those things. The problem is we are fifty to eighty years behind oncology. We have no genetic marker. We have no anatomic biomarker.

We have no idea how patients are going to behave. And that’s why we do something called Treat and Extend. And treatment extend really is let’s play it by ear. Let’s see how you respond. There are some patients that will require treatment every two weeks.

There are others that may require treatment every six months. That’s being a doctor. Yes, but there’s no way to know. Yeah. Which is fine, being a doctor.

But if you’re doing a clinical trial, hard. Right? And if you and this has happened, by the way, I won’t mention names, but in other other trials where they’ve failed. If if you happen to have unintended, like this group of patients that may be totally VEGF dependent or whatever, it doesn’t need very many, you can completely bias your trial unintentionally and leave a good drug on the table.

John Walden, Analyst: So I think this is a good segue and you alluded to this, your two phase three trials, SOUL one and SOUL r. Yeah. It’s kinda hard to unpack as, you know, one identity, but you guys designed these so they flow into each other. So maybe if you could just talk at a high level between the two and then

Praveen Duval, CEO, Ocular Therapeutix: we can talk about each separately. Absolutely. So let me just talk about the patient selection and how the two are designed. The first trial is a superiority trial. Right?

It’s it’s called SOUL one. Again, both of these are exactly according to FDA guidelines. And in a superiority trial what you want to do is you want to select patients that have the most amount of VEGF receptors and are most VEGF dependent, load them up and allow them to fail once and that’s what was required, right? So we selected not patients with poor vision because there you get fibrosis and atrophy as I said, but patients with exceptionally good vision, twenty, eighty or better and most amount of VEGF receptors. And we tested the VEGF receptors to make sure they function because those patients were required to improve by 10 letters or more for gain twenty twenty.

So perfect patient selection to go ahead and load them up and then allow them to fail once. Right? That’s the patient selection for superiority study. Now that is very very different than a non inferiority study which is the second study which is called SOLAR. In a non inferiority study, that’s not the patient selection you want.

What you want in a non inferiority study is absolute stability definition because stability wins. So here what we have is we have three loading doses of EYLEA and then we have two opportunities to observe. And what are we observing for? We’re observing for those VEGF dependent patients that will fluctuate. We have two opportunities to weed them out.

We have two more loading doses and then we randomize. So in each case we’re super selecting patients, enriching our patient population in a bespoke manner appropriate for each study. Now that’s that’s the patient selection part. So why do we decide to have these two studies the way we did? Well, the knee jerk reaction that everybody has and others are doing as well is to design the same study twice.

Right? It’s the same thing twice. You need two studies for approval. We didn’t do that. When you think about it, that is terribly inefficient.

I mean, it doesn’t require much of that brainpower but it’s terribly inefficient because the second study really provides no additional information, just confirmation. Alright? We wanted to have two studies that complement each other and provide all the information that’s needed so that we have a logistic, regulatory, as well as commercial advantage. So let me talk about each. So logistically, we required that everybody go into SOUL one first.

And those that screen failed SOUL one in the beginning ended up in SOLAAR. So we didn’t lose any patients. And that worked perfectly. And once SOUL one was fully randomized, then we took patients from the outside for SOLAAR. So it is the most efficient, logistic, complementary study design that you can have.

So that worked really well. From a commercial point of view, look, we have the potential of having the best label ever in this field. A label that is a superiority label based on SOL1, only one, right, the first and only one. A label that has the flexibility of dosing of every six months based on SOLAR to every twelve months based on SOL1 and repeatability based on SOLAR. So from a commercial point of view, it’s worked out beautifully.

And we have the numeric analysis. Remember, we talked about the second generation anti VEGFs. We will be going up against high dose Eylea. Not for again this is not for statistical analysis, this is for numeric analysis and SOLAR as a masking arm, but that’s gonna be very very valuable commercial advantage for us. And then finally from a regulatory point of view, and this has not been recognized as much as it should, remember no regulatory agency will see either of these studies in isolation.

You’ll always see it together. So for instance, when somebody looks at a regulator and one of the agencies looks at the number of rescues, say, in SOLAR, which is the non inferiority study, they will look at the rescues in context of SOL-one knowing that this drug lasts for nine to twelve months. That’s a huge amount of power that we have in terms of information distribution between these two studies. So they work in a perfect complementary fashion. They’ve worked perfectly well so far.

And I really do believe that this kind of design for its efficiency is gonna be the standard for the more thoughtful sponsors in the future.

John Walden, Analyst: And with SOL1, can you talk a little bit about that superiority endpoint, the primary endpoint you’re powering, and what gives you confidence that, you know, some of these EYLEA patients won’t go all the way to to nine months?

Praveen Duval, CEO, Ocular Therapeutix: Well, I mean, again, it’s so the the modeling that we’ve talked about is really based on previous studies as well as interim modeling. But for EYLEA, what I can point to, for instance, is the TALEN study. And that was in a non super selected patient population. And even then when patients were treated and extended, meaning the drug was on board, the anti VEGF was on board, even then only twenty percent or so of patients, or less than twenty percent actually made it to that endpoint. So we’re very confident that in a super selective patient population that’s selected to fail, that number is a very conservative number.

For us, we know from The US study, we’re very confident that over seventy percent of patients for ex Paxley will be rescue free. So the biggest thing though is not those numbers. The biggest thing is the delta. Right? We need 15, one five, 15 percent delta to be statistically significant.

And we’re very confident that we’re gonna be well above that. What I’ll also tell you is the reason that I’ve I’ve said, John, earlier on that today I’m more confident than I’ve ever been as confident than I was before is because of what we’re seeing under masking. Now, it’s very important to say that we’re masked. Right? Wear masked.

But we can look at three things, and we look at this obsessively for trial conduct, which is the most important thing in this company, is trial conduct of SOUL one. We look at the number of rescues, we look at the cadence of the rescues, the profile of the rescues, and we look to see if those rescues are on protocol or not. All under masking. So as far as the number of rescues are concerned, if we’ve selected the patients properly to fail, we should be seeing a robust number of rescues. And that’s exactly what we’re seeing.

The other the second part is the cadence or the profile of the rescues. We expect a certain cadence knowing knowing how Aylea behaves. We and what we’re seeing, the timing of the rescues, we’re thrilled with. We’re very, very, very happy with that with that cadence. That’s number two.

And number three is whether we’re seeing those rescues on protocol or not. And what I’ve said earlier on and repeated many times is that the vast, vast, vast, and I’ve said it three times, the vast, vast, vast number of rescues that we’re seeing are absolutely on protocol. So given all that under masking, again, I’m repeat this, this all under masking, I feel more confident today than ever.

John Walden, Analyst: And you guys recently amended that trial design. Can you talk a little bit about what you did and why you did it?

Praveen Duval, CEO, Ocular Therapeutix: Yeah. So this is actually thank you for bringing that up. This is actually really important. So so let me just tell you what what happens if there’s a good historical background here. There are three things that are

The first one is that the team before us, as I mentioned, and they deserve the credit, actually got the SPA which was fantastic for SOUL one, the superiority study. The way it was designed is the primary endpoint is at month nine and the study ended. There was nothing else after that, right? So the FDA requires a certain number of patients to have at least a two year follow-up for safety only, not for efficacy but for safety. So in order to satisfy the FDA requirements we had to put all those patients and overload the second study, SOLAR.

So originally it had eight twenty five patients. And not because we needed them for powering, but we needed them to satisfy the FDA’s second year requirement because SOUL one ended at month nine. So, our investigators came to us and said, Hey, look, you’re putting us in a really difficult position because once the study ends and the study is positive, we’ve gotta go to these patients who volunteer to be in the study and say, hey, look, there’s nothing for you until the drug is approved. And that’s a really difficult conversation. They’re right.

Mean, done this for thirty years, it’s not a great conversation to have. That’s number one. Number two is we saw what happened to Vibismo. Vibismo got a four month label and there are very few patients that actually made it to that mark. Mhmm.

So the threshold for getting something on the label, the number of patients to make it there, is actually quite low. And we were confident, we are confident, that there will be quite a number of patients with with X Factor that will be able to easily make it past the primary endpoint to the twelve month mark. Mhmm. And it’d be great to have the opportunity to get twelve months on the label. That’s number two.

And number three, and this is the gating factor, and people ask why do we wait this long? This is the reason. Many of us have done more clinical trials than anybody else and I’m very proud of the people that we have here. This is the best of the best of the best in terms the retina talent we have. None of us, none of us have ever seen patient retention as good as we’re seeing right now.

You’ve never seen it. So putting that all together, we said, look, let’s just go to the FDA and see if we can extend SOUL one into the second year. And they said, okay. So now we can have the maximum amount of exposure in the second year to satisfy their safety requirements. If that’s the case, we can drastically reduce their recruitment for SOLAR.

So we reduce it from eight twenty five patients to five fifty five patients. So what that does is the recruitment for SOLAR, remember you need both studies, is gonna happen much faster, much more efficiently. The bottom line of all this is we’re gonna get to the finish line much faster, much cheaper, and potentially with a much better label that has the flexibility, a potential superiority label with a flexibility of six month to twelve months of dose.

John Walden, Analyst: And in the last minute, that’ll be a perfect button. Can you talk about timing of Callus over the next, let’s call it twelve point four months? Like, what what should investors be keeping an eye on? Well, I I think I I think there are there are a number of

Praveen Duval, CEO, Ocular Therapeutix: things that are really exciting on board here. You know, obviously, the the completion of SOLAR will be updating you as appropriate. We’ll also

John Walden, Analyst: guidance on SOLAR?

Praveen Duval, CEO, Ocular Therapeutix: Well, you know, we not not as not as yet from the time the last time. You know, in January we said we had enrolled three eleven patients. Remember, now it’s gonna be much faster because the number is gonna be five fifty five patients as opposed to eight twenty five. So all of that is gonna happen a lot faster. Mhmm.

And, you know, when appropriate, we’ll we’ll guide you. But remember, when we give you guidance, we make sure that we do an accurate job and so forth, and we will. You’ll also, I think, hear about our interaction with the FDA regarding diabetic retinopathy and diabetic macular edema, which we’re thrilled with. We’ve made a commitment to go after that target. We have no competition.

Our results of the HELIOS study have been absolutely fantastic. We’re really encouraged you’ll hear about that. And then finally, obviously what everybody’s waiting for, which we’re very confident about is the card churn for so long, which will gonna be in the first quarter of twenty twenty six.

John Walden, Analyst: Okay. Perfect. And I guess that wraps up the time we had. We covered a lot of ground. We’re looking forward to tracking the progress.

This is one of the more compelling stories, I think, in the space, and obviously there’s a lot of commotion, so it’s a fun time to be covering it. And thanks again Praveen for joining us.

Praveen Duval, CEO, Ocular Therapeutix: John, thank you again. Thank you for having us here and for all your diligence and support. Thank you. Thanks. Thanks.

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