Ocular Therapeutix at BioConnect: Strategic Advances in Wet AMD Treatment

On Tuesday, 20 May 2025, Ocular Therapeutix (NASDAQ:OCUL) presented at the H.C. Wainwright 3rd Annual BioConnect Investor Conference 2025, showcasing strategic advancements and challenges. The company highlighted progress with its lead product, Expaqli, aimed at treating wet age-related macular degeneration (AMD) and diabetic retinopathy. While optimistic about regulatory support and market potential, the company also acknowledged the need for financial prudence in an uncertain macroeconomic climate.

Key Takeaways

• Ocular Therapeutix’s SPA amendment for the SOLO-1 trial allows for a faster, cost-effective path to market.
• Expaqli offers potential for longer treatment duration than existing therapies, enhancing market share prospects.
• The company is financially positioned to continue pivotal trials through 2028.
• Praveen Tugo expressed confidence in the FDA’s support and dismissed gene therapy as a major competitive threat.
• Plans for global commercialization of Expaqli are underway, with a strong commercial team already in place.

Financial Results

• Ocular Therapeutix is capitalized to fund operations through 2028, securing the completion of pivotal SOLE programs.
• Current capitalization excludes the diabetic retinopathy/macular edema trial, pending further planning.
• The company emphasizes financial responsibility to protect shareholders.

Operational Updates

• SOLO-1 Trial: SPA amendment includes redosing every six months in the second year, aiming for a 12-month label. Randomization is complete, with top-line data expected in Q1 2026.
• SOLAR Trial: Enrollment is robust, targeting both treatment-naive and experienced patients. Enrollment numbers will be disclosed when appropriate.
• Diabetic Retinopathy/Macular Edema: Positive HELIOS study results showed no vision-threatening complications in the treatment arm, with discussions ongoing with the FDA.

Future Outlook

• Ocular Therapeutix is confident in FDA support for its trial designs, which include superiority and non-inferiority studies.
• Expaqli’s six-month treatment duration is expected to provide a significant market advantage.
• The company plans to pursue additional indications once the macro environment stabilizes.
• Patents for the drug and hydrogel are secured until 2041, with pending extensions to 2044.

Q&A Highlights

• Praveen Tugo dismissed gene therapy as a significant threat for wet AMD, citing safety, efficacy, and logistical challenges.
• He emphasized the market’s potential, noting a 40% patient dropout rate in the first year, which he believes is underestimated.

For further details, readers are encouraged to refer to the full transcript below.

Full transcript - H.C. Wainwright 3rd Annual BioConnect Investor Conference 2025:

Keith, Host, HCBY CERT / Nasdaq: Good afternoon. Welcome to the HCBY CERT Annual Bell Connect Investor Conference and Nasdaq. For this session, we will have a fireside chat with doctor Praveen Tugo, executive chairman, president, and CEO of Ocular Therapeutics. Thank you for joining us.

Praveen Tugo, Executive Chairman, President, and CEO, Ocular Therapeutics: Keith, thank you for having us. Honored to be here.

Keith, Host, HCBY CERT / Nasdaq: So I understand that the special protocol agreement for SOLO one trial was recently amended to incorporate redosing, right, in wet AMD. Was the the reason that you believe either the FDA or ophthalmologist or retina specialist will like to see redosing being evaluated in two pivotal trials instead of one?

Praveen Tugo, Executive Chairman, President, and CEO, Ocular Therapeutics: No. Actually, it’s got nothing to do with that whatsoever. I think that, look, let me sort of take a little bit of time in describing the amendment because it’s extremely important to us. And let me just get get to the bottom line. I think there are two take home messages from this.

The first take home message is that we gave up absolutely nothing, And, this is a huge win for us because it allows us to get to the finish line faster and cheaper. That that’s number one. Number two is it really speaks to the relationship that we have with the FDA, and I believe the relationship is there because we’ve done everything exactly the way they wanted us to do it that resulted in the SPA, as well as the, the trial designs that we have. But let me back up a little bit and tell you what happened. There are really three things.

The first is that, the team before us really deserves the credit for getting the SPA. The SPA agreement, it was such that the trial, the primary endpoint was at month nine, and the trial ended exactly there. There was nothing after that. The trial simply ended. There are two consequences of that.

The first consequence is that, look, the FDA requires of everybody, not just us, that there are a certain number of patients that we followed for two years for safety purposes. It has nothing to do with efficacy but just for safety purposes. And obviously with the trial ending at month nine, we couldn’t satisfy that with SOLA-one, so we had to way overload SOLAR with eight twenty five patients to be able to satisfy that requirement. The second consequence is really our PIs. Our PIs came to us and said, Look, you’re putting us at a really difficult position because once the study is successful at month nine, there’s nothing we can do for the patients.

They’re done and they’ve got to wait until the drug is approved. And they’re right. I’ve been in that situation before, and it’s not pleasant. The second thing that happened was with that we saw the Bibismo label. They got a four month label, but in actuality, there are very few patients that actually made it to the four month So the threshold for getting it on the label apparently is really quite low.

And we were convinced, we are convinced, that there will be many more patients who will be able to make the twelve month mark with ex Paxley. The third thing that happened, and this is really the gating event because people ask us, well, why didn’t you do this right away when you came in? And this is really significant because this speaks to the quality of patients that we’ve recruited and so on. As you know, in our company, we’re very fortunate to have a bunch of really, really experienced world class retina people, and all of us have practiced for twenty, thirty years. We sat down on a table and think about this, all of us looked at each other and said, we have never in our entire career seen patient retention that is this good.

And that was the gating factor. So when you put all those three things together, we contact the FDA and said, look, can we have a second year? We don’t wanna disturb the spot at all, but can we have a second year for SOLAR-one where we treat every six months? So we maximize the drug exposure. And they said yes.

And then we said, well, can we then add that with this every six months in SOLAR so that there’ll be enough patients that will satisfy your safety requirements? And they said yes. And then we said, well, if we can do that, then can we reduce the number for SOLAR from eight twenty five to five fifty five by a third? And they said yes. And at the same time, we could also keep the masking so that we would mask until month twelve, so we would potentially get month 12 on the label.

So it’s an absolute win for us. It’s we gave up absolutely nothing. The primary endpoint is exactly the same. The powering is the same. We satisfy the FDA’s requirements for safety.

We potentially have a phenomenal label, the best ever with superiority on the label, with dosing flexibility from every twelve months to every six months with repeatability. And remember, in our back pocket, we will have our own data, and this is numeric analysis, not strategic analysis, versus high dose EYLEA for our commercial leverage. So it’s a great win for us, but that’s the reason we did what we did. It’s a lot of stuff, I know. But at the end of the day, it just simply allows us to get to the finish line faster and cheaper.

Keith, Host, HCBY CERT / Nasdaq: Got it. Got it. SOUL one trial is not has not completed enrollment. Right? When do you expect to report top line data and what’s your expectations for the readout?

Praveen Tugo, Executive Chairman, President, and CEO, Ocular Therapeutics: It’s for SOUL one. Right? Yeah. So SOUL one has completed randomization. Right?

And we announced that. And what we said was because we’re gonna do the masking till month twelve to potentially get that on the label, our readout date now is the first quarter of next year, ’20 ’20 ’6.

Keith, Host, HCBY CERT / Nasdaq: For retinal specialist with real world practice, would you say the SOAR trial showing none inferiority of expaxially compared to aflibercept would be more meaningful?

Praveen Tugo, Executive Chairman, President, and CEO, Ocular Therapeutics: So look. It’s it’s it’s an appropriate question, but what what I wanna emphasize here, and I didn’t do this, so I’m not taking the credit for this, is that we’ve got an absolutely revolutionary trial design in each of these these two trials that that confirms and is completely in line with what the FDA wants. But more importantly, we’ve got two complementary trial designs, right? So what everybody expected is for us to have a second trial that’s exactly the same as the first trial because that’s what everybody does. You know, you you look at, I know, view one, view two, Hawk Harrier, Anchor Marina.

It’s the same thing that’s done twice. And when you think about it, the second trial will validate the first trial, and it’s a no brainer. It’s a knee jerk, but it’s a lost opportunity because it adds absolutely no information. That’s not what we did. And this has been, again, validated by the FDA.

We have two complementary trial designs where all retina physicians that have practiced for decades and decades. And the reason we did that is because both trials should be looked at together, and it affords us huge advantages from a logistic, regulatory and also a patient physician point of view. But to answer your question specifically, never should one trial be considered alone, okay? So let me answer that. If I just showed you SOUL I, you would say, Well, how does it do against Eylea?

If I just showed you SOLAR, you would say, Well, how does it how long does it last? But when you look at it together, every question is answered. The doctor will know exactly how long the drug lasts based on SOL1. The doctor will know exactly what the drug does versus regular Eylea based on SOLAR. The doctor will know exactly what the drug does versus high dose Eylea based on the numeric analysis that we’ll have in our masking arm.

So every question that’s relevant that the physician, the clinician will want will be answered by a combination of those two trials.

Keith, Host, HCBY CERT / Nasdaq: Is SOAR currently enrolling both treatment naive and treatment experienced patients?

Praveen Tugo, Executive Chairman, President, and CEO, Ocular Therapeutics: Yes. A great question. The answer is it’s it really is treatment naive patients only with a tiny asterisk. And let me just explain. As you know, it’s designed to have a long ramp deliberately because we want to only enroll patients that are as stable as possible.

And that’s true of both trials where we’ve derisked the patient population. But if there’s a patient, for instance, that you may have that you say, Look, I’ve already treated this patient twice with EYLEA per the label, and I can document the OCTs and everything else, can I enroll this patient? As long as the documentation is there and it’s been properly treated, we will allow that patient to come in. So technically, that may be a previously treated patient, but that’s gonna be a very small special group, but otherwise, it’s treatment naive patients.

Keith, Host, HCBY CERT / Nasdaq: Based on the current enrollment speed, when do you expect to complete enrollment for SOAR?

Praveen Tugo, Executive Chairman, President, and CEO, Ocular Therapeutics: It’s a great question. What we said in January is that we had three eleven patients in SOLAR in various stages of loading and randomization that were enrolled. The reason we gave that number because is that we wanted to show people that, look, our trial design works in a logistic advantage because all the patients that were in SOLO-one now were bolus into SOLAR. And it worked perfectly, and we suddenly got through in 11 patients. Since then, we haven’t given you any updates.

We will when appropriate. We’re thrilled with not just the speed of enrollment of SOLAR, but the quality of the patients that we’re enrolling. And the reason it takes a little bit of time is twofold. And one is because we want to give you accurate numbers, but the other is because to get accurate numbers, we’ve got to go through the ramp process. And the ramp process by design is quite long.

Keith, Host, HCBY CERT / Nasdaq: Based on existing clinical data, what percentage of patients do you expect in the SOLAR trial to remain rescue free during the six months period after expecimen treatment?

Praveen Tugo, Executive Chairman, President, and CEO, Ocular Therapeutics: Yes. So that’s a great question. So the answer is that we are very, very confident, right, with a positive again, all these two studies have to be taken together, right? Nobody is going to see a SOLAR study without a positive SOL-one study, right? So think about the question that you asked in the context of a positive SOUL one study.

Right? So the question to be asked is knowing that this drug lasts for nine months in one study, what are the chances this drug will last for six months in another study? That’s pretty damn good.

Keith, Host, HCBY CERT / Nasdaq: How likely do you think the FDA will require a second SOAR trial?

Praveen Tugo, Executive Chairman, President, and CEO, Ocular Therapeutics: I don’t see why they should. I mean, I I see absolutely no reason. So let me just tell you what happened last week. There was a major conference, I’ll call the Retina World Congress. And Bill Boyd, who has taken over Wiley Chambers’ position, as you know, was the keynote speaker.

And he was asked a few questions. Right? One of the questions that he was asked was about sham. And we’ve been saying all along that we’ve been following the FDA guidelines perfectly. We do not have sham in any of our trials whatsoever.

And Bill reiterated exactly that. He said sham is not recommended. Sham is not proper masking. He even went so far as to say why it’s not proper masking because it forces him to cross trial compare with a study that’s properly masked, which is not scientific. He was also asked if a sponsor like us follows his guidelines, the FDA guidelines, are two studies that are complementary allowed, which is a superiority study and non inferior study?

And the answer was yes, of course. They’ve never ever required the two studies to be exactly the same. They actually like it when the two studies are not the same. Because remember, the job of the FDA at the end of the day is to make sure that something did not occur by accident, right? That it was not done just by accident.

So if you’ve got two different study designs that come to the same conclusion, there’s a lot more power that that didn’t arrive by accident, that was a real result. So I don’t see why another study would be required at all. I don’t think there’s any notion of that whatsoever given the feedback that we’ve got. And remember, we’ve got a SPA agreement and we’ve got a written Type C approval validating that these studies are Phase III studies. And by the way, we also have a written validated letter from them saying that that is all that’s required for approval, these two studies and nothing else.

Keith, Host, HCBY CERT / Nasdaq: Got it. Thanks. Do you believe that the first drug on the market was a six months treatment duration for wet AMD could have a significant advantage in terms of market prospects?

Praveen Tugo, Executive Chairman, President, and CEO, Ocular Therapeutics: Yes. You should ask Genentech that. So I’m old enough to remember this yeast, so not many people a a lot older than you, so but but not not many people here may be old enough. But I say you should ask Genentech that because, look, I lived through this. You’re asking about a six month advantage.

Okay. What about a seven year advantage, right? So you think about where we were with wet macular degeneration. Genentech, a phenomenal company, one of the greatest companies in the world, came up with a miraculous drug named Lucentis. It actually not only preserved vision, it improved vision.

And what we saw, that graph, was absolutely miraculous. Genentech had a seven year head start. Not six months, seven years. And then came a company that nobody knew about that was struggling and about to go bankrupt named Regeneron. They came up with a drug that wasn’t safer, Right?

It wasn’t stronger. It simply lasted very arguably one week longer. And guess what happened after seven years? You talk about six months? Seven years.

Totally dominated. Now what else has happened recently? You look at Vibismo. Right? There comes a Vibismo.

Vibismo is not safer. It’s not stronger. It may last two weeks longer. And guess what that’s done? Phenomenal revenues, overtaking the market.

So when you’re asking me whether six months matters, you know what? The better drug always wins. And there is a huge appetite for anything that is more sustainable, even incrementally more sustainable. Even a week buys you a huge market opportunity. We’re not coming within with an extra week or two.

We’re coming in we’re in a different orbit. We’re coming in with nine to twelve months. So do I care about another drug that’s six months? Absolutely not. If that should happen, history proves me correct.

It’s the better drug that always wins.

Keith, Host, HCBY CERT / Nasdaq: What’s your development plan for diabetic retinopathy? Do you expect expect expect sleep to perform equally well in Doctor?

Praveen Tugo, Executive Chairman, President, and CEO, Ocular Therapeutics: Yeah. So let me tell you what the HELIOS study shows. And, you know, this was a study that was done by the team before us, and we discovered this study, we said, Oh, my goodness. It’s a pure safety study. Nothing else.

The patients were not super selected or as carefully selected at all. Was all comers. It was basically what would happen when this drug got placed in a diabetic eye. That’s it. And I knew there were no safety signals, by the way, under masking.

There never have been. There’s never been a safety issue with this drug whatsoever. And we unmasked the study because it reached its primary endpoint. But I knew that people would extrapolate this into efficacy. And I knew that in the most variable disease we treat, We had simply got a bunch of patients together and just put the drug in the eye that the that the efficacy was gonna be all over the place.

Right? Well, it wasn’t. And what’s absolutely remarkable is that every single parameter that we looked at, I mean, single parameter was in favor of the drug. You know, the most important thing we do in diabetic retinopathy is realize that the natural history of these patients, and these patients are young people in the workforce, they may be mail carriers, they may be lawyers, they may be doctors, they may be accountants, they’re completely asymptomatic in the most productive period of their life. We know that every year, year upon year, thirty to forty percent of them will have vision threatening complications.

Just think about that. Thirty to forty percent. And you know how many are being treated? Nobody. I mean, nobody’s coming in every month to get a needle in the eye with Lucentis or Eylea when they’re asymptomatic.

Nobody. Nobody’s being treated. So what do we see in this small study? We saw that in the control arm, thirty seven point five percent of patients had vision threatening complications, which is exactly in line with the natural history. When you look at the treatment arm with one injection, one injection of expaxly, at week forty eight, vision threatening complications, zero.

Absolutely zero. So as a clinician, that’s remarkable. That means I can sit with a patient and say, missus Smith, your chance of having complication year upon year is thirty to forty percent. But if you come to see me but once a year, like you go to your dentist for teeth cleaning, I can reduce that chance to zero, that’s pretty impactful. So, yes, we’re gonna do diabetic retinopathy.

We have no complication we have no competition whatsoever. What we what we committed to was to go to the FDA and figure out a path forward where we’ve had great, great, discussions with the FDA, fantastic discussions. We will detail that publicly when the time is appropriate. If you and I were sitting here six months ago, I I I promise you, we would be talking about already having started diabetic retinopathy. The only thing that gives me pause at this point, and the reason that that that there is a little bit of pause, is the macro environment.

You know, we’re in a situation where, you know, my job, my only boss other than my wife, are the shareholders. Right? And and at the end of the day, I’ve gotta protect the shareholders. I’ve gotta do what’s financially responsible. And to launch a large phase three study in an unstable environment, especially when you have absolutely no competition and there’s no rush, may not be the most financially responsible thing to do.

But we will do it when it’s a little bit more stable because we’ve got a clear path forward and we’ve got great results and there’s no competition and we will take that market.

Keith, Host, HCBY CERT / Nasdaq: About diabetic macular edema?

Praveen Tugo, Executive Chairman, President, and CEO, Ocular Therapeutics: Same thing and thank you for asking because when I say diabetic macular non proliferative diabetic retinopathy, I should be saying nonproliferative diabetic retinopathy and diabetic macular edema. And the reason for that is that in this study, there were patients with non center involving diabetic macular edema. Every single patient in the treatment arm improved. Every single patient in the control arm got worse. Got it.

Keith, Host, HCBY CERT / Nasdaq: That’s very exciting. Is expressly protected by any issued patents?

Praveen Tugo, Executive Chairman, President, and CEO, Ocular Therapeutics: Yes. So we have two components here, right? One is the drug itself. It’s an FDA approved drug. One is the actual hydrogel, and it’s an FDA approved hydrogel.

Remember, this hydrogel is being used on the eye right now with DEXTENZA. Over five million patients have been exposed systemically. It’s been used in neurosurgery, urology, etcetera. So yes, the patents are well protected. This is done entirely in house.

So the patents not only apply to the composition of matter, but also apply to the entire process, which is in house and proprietary. All the materials as well as the IP is in The US. The IP runs till 02/1941. We do have methods that are currently pending for patents that will take us to 02/1944.

Keith, Host, HCBY CERT / Nasdaq: I guess the the company currently has sufficient cap capital to complete ongoing pivotal trials. Right?

Praveen Tugo, Executive Chairman, President, and CEO, Ocular Therapeutics: Yes. So what we’ve announced, in our last earnings call is that we are, capitalized into 2028. It’s important to note that, that does not include the diabetic retinopathy, diabetic macular edema trial because at this point, we don’t know what we haven’t announced what that’s going to look like, all right? So we have we’re capitalized enough to go ahead and complete the SOLE programs, which is to see the car turn of SOLE-one as well as SOLAR.

Keith, Host, HCBY CERT / Nasdaq: Could you comment on the commercial approach for wet AMD?

Praveen Tugo, Executive Chairman, President, and CEO, Ocular Therapeutics: Yes. You know, there are two things that are really important that I have in this company that I that I’m very fortunate to have that I did not have in the last company in iveric bio. The first thing is that I don’t have to convince anybody that there’s a market out there. Right? With geographic atrophy, I have to tell people there’s a market out there.

Here, we’ve got forty years of experience in knowing that there’s a huge market there. And that market actually is very much underestimated because remember, there’s a forty percent patient dropout in the first year. So we’re seeing just the tip of the iceberg. So it’s a huge market that I don’t have to convince anybody about. The second thing that I have that I didn’t have there is a mature, well developed, proven commercial team because remember, we’re commercializing and selling DEXTENZA.

So given that and given our knowledge of retina, we are in a great position to go ahead and take this product and commercialize not just in The U. S. But globally, and that’s what we intend to do. And that’s where a lot of our effort is going in right now.

Keith, Host, HCBY CERT / Nasdaq: Great. Really looking forward to the SOLO-one trial data first. Any questions from the audience?

Praveen Tugo, Executive Chairman, President, and CEO, Ocular Therapeutics: Do you see any competitive pressure coming from the genetic therapy arena? Yeah. So look. This is you know, I’ll I’ll I’ll tell you what I think in as diplomatic a way as I can, but I will be fairly opinionated, so I apologize. I think gene therapy is great for inherited retinal diseases when there’s a gene that needs to be replaced, etcetera.

Think gene therapy is extraordinarily challenging when you’re talking about infecting a gene as a factory that just pumps out protein. I think it’s very, very problematic in terms of safety, in terms of efficacy and in terms of logistics. So I really don’t view that as competition. Look, what we have that I’m very excited about is that this drug is going to be adopted the very next day that is out there. And the reason is because nothing has to change.

From an economic point of view, the doctors are going be in great shape because the payers are going save a lot of money because we’re going to have fewer people drop out and fewer people go blind. The ASP is going to be higher. They’re going to get paid more for every injection. There’s going to be a much larger patient pool to inject, not just the same patient over and over again because the dropout rate is less and then catchment is going to be much, much bigger. In doing all of this and seeing more patients and happier patients and getting paid more for it, nothing has to change.

They don’t have to buy a single piece of new equipment. The workflow doesn’t change one bit. The experience doesn’t change one bit. And they simply get a better product, and it’s a win win win situation from adaptability point of view.

Keith, Host, HCBY CERT / Nasdaq: Okay. Great. Thank you very

Praveen Tugo, Executive Chairman, President, and CEO, Ocular Therapeutics: much. Thank

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