Ocular Therapeutix Q4 2024 Earnings Call Transcript

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Provided by Quartr
March 3, 2025
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Operator

Good morning, and welcome to the Ocular Therapeutics Fourth Quarter twenty twenty four Earnings Conference Call. At this time, all participants are in a listen only mode. After the prepared remarks, we'll conduct a question and answer session. As a reminder, this conference call is being recorded and will be available for replay on the Investor Relations section of the Ocular Therapeutics website. I would now like to turn the call over to Ocular's Vice President of Investor Relations, Bill Slattery, Jr.

Operator

Please go ahead, Mr. Slattery.

Bill Slattery
Bill Slattery
VP - Investor Relations at Ocular Therapeutix

Good morning, everyone, and thank you for joining us today. Earlier this morning, we issued a press release and filed our annual report on Form 10 K outlining our financial results and business updates for the fourth quarter and full year of 2024, along with several updates to our registrational program for ex Paxley and wet AMD. Ocular's Executive Chairman, President and CEO, Doctor. Praveen Dougal will summarize recent business highlights before we move to our question and answer session. Joining Doctor.

Bill Slattery
Bill Slattery
VP - Investor Relations at Ocular Therapeutix

Dougal for the Q and A portion of the call will be Donald Notman, Chief Financial Officer and Chief Operating Officer Sanjay Nayak, Chief Strategy Officer and Steve Myers, Chief Commercial Officer. We refer everyone to this morning's press release and our Form 10 K for a comprehensive update of fourth quarter and full year 2024 financial and business results. During today's call, certain statements we will be making constitute forward looking statements under the Safe Harbor provisions of the Private Securities Litigation Reform Act of 1995. Actual results may differ materially as a result of a variety of factors, including risks and uncertainties identified in the Risk Factors section of our annual report on Form 10 ks and our other SEC filings. With that, I'd like to hand the call over to Doctor.

Bill Slattery
Bill Slattery
VP - Investor Relations at Ocular Therapeutix

Praveen Dougal to review our recent updates. Praveen?

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

Thank you, Bill, and thank you to everyone for joining us today. 2024 was a transformational year for Ocular Therapeutics. We sharpened our focus and embraced a single bold mission to redefine the retina experience. Today disrupting the treatment paradigm and wet AMD is our top priority and we are executing with speed, precision and confidence to bring ex Paxly to patients who need a more sustainable and effective treatment option. Despite established and effective treatments for wet AMD, the burden of frequent dosing leads up to a forty percent of patients discontinuing treatment in the first year alone effectively allowing themselves to go blind.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

Patients deserve better and Paxly has the potential to become the standard of care by offering a long lasting flexible treatment option. And wet AMD is just the beginning. With compelling early clinical results in non proliferative diabetic retinopathy and diabetic macular edema, we see a significant opportunity to expand into these and other highly prevalent retinal indications where millions of patients remain untreated. This morning, we announced several exciting updates that enhance and accelerate our registrational program for ex paclit, positioning us incredibly well as we move toward clinical data and a potential NDA submission. Let's get right to it.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

First, we recently received FDA approval for an amendment to our special protocol agreement for SOL1 that incorporates redosing in all patients at week fifty two and week seventy six. We believe this amendment unlocks the potential for XFAXWAY to secure an unprecedented six to twelve month dosing label in wet AMD showcasing what we believe to be best in class durability. The impact of this cannot be overstated. The most recent approvals in this indication extend durability by about two weeks in most patients, yet they have rapidly gained market share and generated significant revenues in a very short period of time. With ex Paxly, we're talking about a different orbit altogether with the potential to extend durability by months.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

As part of this amendment, the thirty six week primary endpoint for SUL-one remains unchanged, but we're required to maintain masking until week 52 to provide additional information on durability up to and to allow potential label permitting redosing at twelve months. As a result, we now expect to report top line data in the first quarter of twenty twenty six. While this slightly shifts the timeline for SOL-one top line data, we believe the long term benefits of this strategy are tremendous and will be abundantly clear at the end of this call. Most importantly, this strategy should allow us to accelerate our timeline for a comprehensive regulatory submission for Xpaxley and wet AMD as I will describe. Moreover, we expect the additional data for repeat dosing will provide valuable insights into EXPACSLE's extended durability, potentially supporting even greater flexibility on the product label.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

SOL1, a trial that many thought would never be enrolled, completed randomization in December 2024, well ahead of expectations with three forty four subjects randomized across more than 100 clinical sites in The U. S. And Argentina. Trial conduct is now our top priority. We are thrilled to report that subject retention has been exceptional to date and the vast majority of rescue treatments as we have reviewed on a mass basis remain in line with the pre specified criteria established in the SOL-one protocol.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

We have also optimized SOLAR, our non inferiority study to align with the changes in SOL-one. Because redosing is now incorporated into SOL1 and retention in the trial is exceptional to date, we are reducing the size of SOLAR from eight twenty five subjects to approximately five fifty five subjects randomized, while maintaining robust statistical power of 90% to evaluate our primary endpoint base on our expectations of how X Paxly will perform. SOLAR was initially designed to include eight twenty five subjects to meet the minimum FDA requirements to receive an unrestricted label fully dosing. Now that subjects in SOL-one are also being redosed, we're afforded the flexibility to reduce the number of subjects in SOLAR. This reduction should accelerate the SOLAR timeline for reporting data, enhance capital efficiency and maintain the scientific rigor needed for regulatory approval.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

Remember that SOLAR is a non inferiority trial comparing ex Paxly administered every twenty four weeks against two milligram of flivarast administered every eight weeks. The primary endpoint is the mean change in best corrected visual acuity or BCVA at week fifty six and patients will be followed until week one hundred and four for safety. Importantly, as per the protocol agreed to by the FDA, the non inferiority margin for the ex Paxly arm at the lower bound is minus 4.5 letters of mean BCVA when compared to two milligram of flivarastat dosed every eight weeks. This is also in line with the FDA's draft guidance, which we are adhering to by incorporating an eight milligram of flivarast masking compared to our arm with the exact same dosing frequency as the EXPACCELLI arm. Additionally, our pre specified supplemental injection criteria are strategically designed to ensure trial integrity and preserve clinical relevance.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

These criteria are consistent with previous non inferiority studies and include a combination of vision loss and OCT changes. By aligning these standard non inferiority trial rescue protocols, we believe the SOLAR study remains well positioned to demonstrate EXPACSLE's durability and effectiveness in a repeat dosing setting. Solar enrollment continues to be strong. We previously announced three eleven subjects enrolled across various stages of loading and randomization as of 01/10/2025. This momentum puts us in a great position to advance our registrational program efficiently and effectively.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

Here at Ocular, our clinical trial design strategy using the loading phase to select patients for randomization and thereby potentially de risking our patient populations has been a key differentiator. We designed SOL-one and SOLAR to work together with each trial seeking to answer critical questions about ex X Paxley's durability, repeatability and flexibility. These studies were developed to be complementary and not redundant, ensuring that together they provide a robust clinical data set that could support both regulatory approval and commercial adoption. So one is focused on durability, evaluating whether a single dose of EXPACSNE can maintain vision for thirty six weeks compared to a single two milligram of flubrasset injection. This study was designed to establish superiority and to set a new benchmark for long term efficacy in wet AMD treatment.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

With the recent SPA amendment incorporating fifty two week and seventy six week redosing, Sol-one now also provides important insights into extended treatment intervals and long term redosing potential. SOLAR on the other hand is designed to address more frequent repeat dosing and potential real world applicability. By comparing ex Paxlovid every twenty four weeks to two milligram of flivarastat every eight weeks, Solar evaluates how ex Paxlovid performs relative to a common, but burdensome treatment regimen. The study is structured to provide critical evidence supporting the feasibility of an every six months treatment paradigm, one that we believe is aligned to preferences of retinal specialists and that could dramatically reduce the burden of care for patients and caregivers alike. Both studies were thoughtfully aligned with regulatory guidance.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

So one supported by SPA and SOLAR validated by Type C written response received from the FDA in August 2024 along with a subsequent written response received from the FDA in December 2024. Importantly, neither study utilizes sham for masking adhering to the FDA's clear stance that sham can introduce bias expressed through the agency's written feedback and public comments. The FDA previously agreed that together SOL1 and SOLAR could constitute two adequate and well controlled trials to support a potential NDA and label for EXPaxelate in wet AMD. Pending a successful outcome, we intend to submit our NDA after the SOLAR fifty six week primary endpoint is reached. By integrating these complementary approaches, seeking to demonstrate both best in class durability in SOL-one and sustained repeat dosing in SOLAR, we believe we are enhancing the potential for successful trials that support a differentiated commercially attractive label.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

The dual nature of these trials allow us to generate a comprehensive clinical data set that has the potential to support a strong regulatory submission and a compelling value proposition for patients, retina specialists and payers. Beyond wet AMD, we see a tremendous opportunity for EXPACSLE in non proliferative diabetic retinopathy or NPDR and diabetic macular edema or DME. Diabetic retinopathy is the leading cause of blindness in the working age population in The U. S. Despite the availability of anti VEGF therapies, fewer than one percent of the six point three million NPDR patients in The U.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

S. Received treatment today, largely due to the burden of frequent injections. EXPACSLE has the potential to transform this landscape. Our proof of concept HELIOS trial demonstrated compelling results for Xpaxley in both NPDR and DME. In this trial, we showed that a single Xpaxley injection may prevent vision threatening complications for up to twelve months.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

Additionally, all patients in the EXPACSOLA arm that had DME saw improvement at week forty eight. So what does this mean? That means that I as a retina physician can say to a patient, your risk with diabetic retinopathy of developing vision threatening complication year upon year is thirty percent percent to forty percent. However, if you come to see me just once or twice a year, much like you go to a dentist for teeth cleaning, I may be able to reduce that risk to almost zero. That is a powerful message.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

I'm happy to inform you today that we expect to receive FDA feedback on our clinical trial design for NPDR and DME in the first half of this year paving the way for our next steps in these important indications. Following receipt of the FDA feedback, we look forward to sharing with you our clinical trial design for these underserved vision threatening indications. We continue to operate from a position of financial strength with a cash balance of $392,000,000 as of 12/31/2024. We remain laser focused on execution of SolOne and SolR. Our disciplined and prudent approach means that we are well financed and believe we have sufficient cash to fund our planned operating expenses, debt service obligations and capital expenditures into 2028 fully funding our registrational trials in wet AMD and leaving us well positioned to execute on our broader strategic objectives.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

Our current cash projections do not factor in the impact of clinical trial activities in NPDR and DME as the scope of that program is dependent on FDA feedback. That said, and to be very clear, we do not currently intend to raise additional capital this year. 2024 was a year of transformation for Ocular Therapeutics and 2025 is shaping up to be even more impactful. What differentiates us is not just the speed and intensity, but also the precision and exceptionally high quality of our execution. In less than one year, we have built a world class team, advanced a groundbreaking asset and executed a registrational program that puts us in a position of strength as we move toward top line data and a potential regulatory submission and approval.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

Today, we shared several updates designed to enhance and accelerate the Xpaxley registrational program, further derisking our path to approval. To summarize, first, the FDA approved an amendment to the SOL1 SPA to incorporate redosing at weeks fifty two and seventy six, increasing potential label flexibility to every six to twelve months. Second, due to the requirement to maintain masking until redosing at fifty two weeks, We now expect top line data for SOL-one including the thirty six week primary endpoint will be released in the first quarter of twenty twenty six. Third, SOL-one retention has been outstanding to date and the vast majority of rescue treatments reviewed on a mass basis have been in accordance with the pre specified criteria of the protocol. Fourth, as a result of our updated Sol-one strategy for redosing, SolR has been streamlined with a target randomization reduced from eight twenty five to five fifty five subjects.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

This should accelerate the time to both SOLAR data and regulatory filings for ex PAXley and wet AMD, all the while maintaining strong statistical powering of 90% based on our expectations of how ex Paxley will perform. Fifth, the non inferior margin for Solar is set at 4.5 letters and standard rescue criteria have been pre specified to align with regulatory guidance. Sixth, enrollment has been excellent across both trials. SOL-one completed randomization ahead of schedule in December and we previously announced SOLAR enrollment of three eleven subjects across various stages of loading and randomization as of 01/10/2025. Seventh, Xpaxley's opportunity extends beyond wet AMD and we expect FDA feedback on the clinical trial design for NPDR and DME in the first half of this year.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

And eighth, Ocular Therapeutics is well financed and has cash runway into 2028. We do not intend to finance this year. We're maintaining strong capital efficiency as we advance toward key clinical readouts and a potential NDA submission in wet AMD. We are redefining the retina experience, setting a new standard in treatment durability, flexibility and long term outcomes. With strong execution, regulatory alignment and clinical momentum, we're well positioned to become a leading retina company.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

Thank you for your time and continued support. Operator, we are now ready to take questions.

Operator

Thank you. Our first question comes from the line of Tazeen Ahmad with Bank of America. Please proceed with your question.

Tazeen Ahmad
Tazeen Ahmad
Analyst at Bank of America

Hi guys. Good morning. Thanks for the updates. A couple of questions for me. Praveen, just given the stats that you provided about the continued strength of what you're seeing blinded in SOL1 in terms of patients staying in the study as well as the redosing rates coming in as you would have expected from your pre specified criteria.

Tazeen Ahmad
Tazeen Ahmad
Analyst at Bank of America

Can you give us a little bit more color about why you think it makes sense to make these changes, especially right now? And then secondly, in terms of making Solara smaller study, just given the high demand that there has been clearly for patients to want to be in the study, can you talk about the reasons of why it wouldn't make sense anymore to have a study of this size just to add a little bit more cushion to the data set that we're expecting? And then I have one follow-up. Thanks.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

Thank you, Tazeen. Good morning to you. And again, thank you for your question. What you've raised is a really important point and let me try and give a comprehensive answer. And I think this really speaks to the way we've designed our SOL programs and I include both.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

These are complementary trials and I dare say that this is revolutionary and I think really quite historic. I think everyone in the world expected us to have SOL1 and SOL2, which really has been the tradition. And every just about every trial that I know, it's been the same trial done twice. The second trial certainly is good to get the product approved and confirm the first trial. But when you think about it, it's terribly inefficient because it really doesn't add any extra information whatsoever.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

Instead, what we've designed is two trials that are always to be taken together and are absolutely complimentary in the information they provide. And this I think is a unique and I dare say a revolutionary and historic concept that will allow for efficiency both in terms of the logistics of the trials, the commercial applicability of the product, as well as the regulatory pathway. The bottom line in what we announced today, the bottom line is that we will potentially get EXPAC sleeve to patients faster and with a better label. So let me go through the sequence of events. Two things happened.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

The first is that the FDA approved our redosing SPA amendment for SOL1 at week fifty two and fifty six. The second thing that happened is that we noted that the retention in SOL1 was absolutely superb under masking of course. So based on these two events, we're able to increase the efficiency of both studies. In SOWL1, what we're able to do is to get information in the first year on the durability and flexibility of the drug, because now we have information at month nine, which is the primary endpoint that remains unchanged and at month 12. And in the second year with redosing, what we're able to do in SUL-one is to maximize the exposure of the drug to patients and satisfy many of the FDA's safety requirements.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

So immediately, SOV1 became a much more efficient clinical trial. And with that in mind, we're able to pass down that efficiency to SOLAR the way these trials were designed. So no longer did we need eight twenty five patients in SOLAR, we could maximize efficiency in terms of our resources as well as in terms of getting the filing to the FDA by reducing the size from eight twenty five patients to five fifty five patients, while maintaining the integrity of the statistics. The impact of this is that we will potentially have a much better label, a label that is a superiority label with flexibility of six months to twelve months. And the second impact, equally important of course, is that we will get to market potentially faster and more efficiently than we were able to before.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

So both of those things terribly important in terms of the impact of what's happened today. What I want to note very importantly also that the primary endpoint remains unchanged. Remember, the primary endpoint of SOL-one is at nine months. That remains unchanged. The statistics remain unchanged completely.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

What we added today, what we announced today were completely added efficiency bonuses. These are complementary trials designed to work together and I believe they work together absolutely beautifully.

Tazeen Ahmad
Tazeen Ahmad
Analyst at Bank of America

Okay. That's super helpful. And my one follow-up is that given that, as you said, the primary endpoint stays the same. Can you give us a sense of what the FDA might be looking for from redosing now in SOR-one?

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

Well, again, the redosing allows for us in the first year to look to get information regarding flexibility and durability of the drug. Now we'll have information at month nine. Again, that's the primary endpoint, which remains absolutely unchanged. But additionally, we'll also have information at month 12 regarding durability of the drug. So potentially, there's flexibility in the label for month nine as well as month 12.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

And as I mentioned, with the redosing in the second year, there's more exposure of the drug to the patients. Therefore, we'll have even more data in regards to safety. The beauty of this is that when you think about the efficiency of SOL1 with a single injection in the first year, we'll have information for month nine and month twelve and with repeat dosing for the second year, we'll have increased safety information with more exposure of the drug to patients.

Tazeen Ahmad
Tazeen Ahmad
Analyst at Bank of America

Okay. Thank you for all the color.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

Thank you, Tiffany.

Operator

Our next question is from the line of Biren Amin with Piper Sandler. Please proceed with your questions.

Biren Amin
Biren Amin
Managing Director & Senior Research Analyst at Piper Sandler Companies

Yes. Hi guys. Thanks for taking my question and congrats on all the updates. So maybe Praveen, let me just start with SUL-one. What was the rationale for dosing at week seventy six?

Biren Amin
Biren Amin
Managing Director & Senior Research Analyst at Piper Sandler Companies

I understand the week fifty two dosing, but I was trying to understand, I guess, the rationale for week seventy six. And then second question is for SUL-one, will all patients receive redosing at week fifty two and week seventy six regardless of the arm, including the control arm? And also will those patients with prior rescues receive redosing at weeks fifty two and week seventy six?

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

Barry, good morning and thank you for your question. The answer for the first one is very simple. We wanted to maximize again the exposure of the drug to patients to satisfy the FDA safety requirements thus redosing at week fifty six at week fifty two, I'm sorry, week fifty two and week seventy six with a study ending at week one hundred and four. The answer to your second question, yes, every patient will be redosed at week fifty two and at week seventy six. The redosing will occur with the same drug that the patient was randomized to regardless of rescue.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

And that's very important. The answer to your question is, yes, everybody will be redosed at week fifty two, at week seventy six with the original drug that they were randomized to. I hope that answers your question. Thank you, Barry.

Biren Amin
Biren Amin
Managing Director & Senior Research Analyst at Piper Sandler Companies

That does. And I just got a follow-up. So on SOLAR, as a result of these changes, you clearly reduced the size to five fifty five patients, which I think, was that primarily due to the safety data that you're generating from SOL-one? Because I think now you would have another 172 patients that are redosed with EXPaxly from SOL-one. Whereas I think you reduce the need for the patients to be redosed with SolR by about 108 patients within the exaflin arm?

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

And Barry, again, thank you. You're exactly right. Your explanation is exactly correct. What we did was we designed SolR originally to have eight twenty five patients to satisfy the FDA safety requirements. And with the redosing amendment that we were able to get from the FDA, we were able to satisfy the safety requirements with both studies combined with the ability to reduce the number of patients from eight twenty five patients to five fifty five patients, while still maintaining the statistical significance.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

So your answer to your question is exactly correct. Thank you.

Biren Amin
Biren Amin
Managing Director & Senior Research Analyst at Piper Sandler Companies

Got it. And then maybe one last question. I guess from a timeline standpoint, were these changes to Solon and Solr underway or made when the board constructed your performance base package on Docu Oil share price hitting, I think there were four different targets between $15 and $30 So I guess I want to understand if these changes provide increased confidence on hitting those performance targets?

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

Yes. So, Biren, again, thank you very much for the question. The answer, obviously, we have discussions with the FDA that are ongoing and we want to keep those discussions confidential. We don't discuss them publicly as with my discussions with the Board. But suffice it to say that our confidence as a company in the success of these trials are higher than ever.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

And certainly my confidence particularly is higher than ever and that you'll see that everything in this company is aligned to make sure that we ensure the success of both trials. Thank you.

Biren Amin
Biren Amin
Managing Director & Senior Research Analyst at Piper Sandler Companies

Great.

Biren Amin
Biren Amin
Managing Director & Senior Research Analyst at Piper Sandler Companies

Thanks for taking my questions.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

Absolutely.

Operator

The next questions are from the line of Colleen Cusi with Baird. Please proceed with your questions.

Colleen Kusy
Senior Research Analyst at Robert W. Baird & Co

Great. Thanks. Good morning and thanks for taking our questions. So for the just to maybe clarify for the redosing of patients in SOL-one, is that just for the FDA safety requirement for redosing or do you need to show anything for efficacy in that protocol in the second year? And then I have a follow-up.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

Colleen, good morning and thank you for the question. The answer is that it really is for the safety requirement for the FDA. However, what it does allow us to do again is get information given this because remember, it's not just a matter of taking the second year separately, but also taking the second year in regard to how it affects the first year. So what it allows us to do is to get that extra information that we will now have not only at month nine, but also at month 12. So it does allow for the flexibility that we hope to get in the label because we have additional information now at month 12 as well as month nine.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

But again, we will satisfy the safety requirements, but we will also gain additional information in the second year regarding redosing. So this is not a requirement for hitting the primary endpoint. I want to make that very, very clear. It's important to emphasize that nothing about the original primary endpoint changes. The primary endpoint remains at month nine and the statistics remain the same.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

This is added efficiency today.

Colleen Kusy
Senior Research Analyst at Robert W. Baird & Co

Got it. That's helpful. Thank you. And then I know when you first shared the SOLAR trial design last summer, I think it was a forty eight week endpoint and then but that was prior to talking to the FDA. Now you're talking about a fifty six week endpoint.

Colleen Kusy
Senior Research Analyst at Robert W. Baird & Co

So can you just talk about what drove that change and what that means for your dosing schedule, which I believe ex factory is still going to be dose every twenty four weeks. So just kind of talk about that fifty six week endpoint, please?

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

Yes. Thank you. Thank you, Colleen. So you're exactly right. I think the first time you might have heard about the forty eight week endpoint for SOLAR was in our June Investor Day.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

And I want to emphasize, if you go back and check your notes then, we made it absolutely clear that we were proposing the SOLAR clinical trial design at risk. And I think those two words at risk were mentioned quite a few times in that meeting. And the reason we did that is we made it very clear, very transparently that we had not yet gone to the FDA for formal approval regarding this trial design. So that's why it was at risk. Now, we did subsequently go to the FDA and the FDA came back and told us that they preferred a primary endpoint at week fifty two or later.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

And as you know, we have a history of doing everything per the FDA. We wanted to make sure that we did it with a minimal amount of risk possible. In fact, we wouldn't want to take any risk as far as our regulatory pathway is concerned. So then we decided, given their preference to pick week 56 and the FDA has now accepted in writing our proposal to take the primary endpoint for SOLAR at week 56. I hope that answered your question, Colleen.

Colleen Kusy
Senior Research Analyst at Robert W. Baird & Co

Great. Thanks. And last one, just any timelines for SOLAR recruitment now in light of the decrease in the study size?

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

Yes. So Colleen, we did give an update at JPM, as was said in my prepared statements. Since then, what I can tell you is that we've been thrilled with the recruitment of Solar, and we will give you an update when appropriate, but it may be a little bit too early at this point. But thank you for

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

your question.

Colleen Kusy
Senior Research Analyst at Robert W. Baird & Co

Understood. Thanks for taking the questions.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

Thank you.

Operator

The next question is from the line of Kelly

Clara Dong
Clara Dong
Vice President - Biotechnology Equity Research at Jefferies

This is Clara on for Kelly. Congrats on the update. So you mentioned that you intend to submit NDA after fifty six week primary endpoints of SOAR is reached. So I understand that the primary endpoints do not change. But also just trying to understand whether you would expect to have some long term redosing durability data in SOW1 as well by that time?

Clara Dong
Clara Dong
Vice President - Biotechnology Equity Research at Jefferies

And also just want to clarify whether the FDA requires that portion of data to support the redosing flexibility portion on the label? Thank you.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

Clara, good morning and thank you for your question. Very important questions. And I want to make it very clear as to what the FDA requirements are for NDA submission versus the information that we'll gather. To be very, very clear, what the FDA requires for the NDA submission is hitting the primary endpoint in both studies. So again, the primary endpoint for SOL-one at month nine remains the same.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

The primary endpoint for SOLAR is at week fifty six. And our intention is that with presumably a positive study at week fifty six with SOLAR, we will submit the NDA application to the FDA. That is unchanged. We just expect given the announcements today that SOLAR will be recruited with more efficiency given the fact that the sample size is smaller. Now in regards to your other questions regarding other requirements, yes, the FDA does have safety requirements as you're aware.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

They will be fulfilled with the combination of patients in the second year in both SOL-one and SOLAR. In addition to that, we will also have a significant amount of information that we believe we can leverage for commercial success. And some of which you've already talked about and we've already talked about, which is the redosing of this drug that is now available both in SOL1 and in SOLAR. And remember, we also have a numeric analysis that we're able to do with the third arm in SOLAR, which compares this drug to high dose EYLEA. Now that is not for statistical analysis, that's for numeric analysis, but you can imagine we will have between these two studies a significant amount of data that is not necessarily required by the FDA, certainly it's not required for approval or NDA submission, but we can leverage for our commercial advantage and we do intend to do that.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

Thank you for your question, Claire.

Clara Dong
Clara Dong
Vice President - Biotechnology Equity Research at Jefferies

Thank you.

Operator

Our next question is from the line of Sean McCutcheon with Raymond James. Please proceed with your questions.

Sean McCutcheon
Sean McCutcheon
Vice President - Biotechnology Equity Research at Raymond James Financial

Hi guys. Thanks for taking the question. So for SolR, could you give some detail on what you think the FDA expectations are, what your expectations are for what would be an acceptable number of rescues and timing of rescues regardless if you hit the non inferior land within the non inferiority margin? Thanks.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

Sean, good morning and thank you for your question. Your question is a very important question, of course. So let me discuss rescues. We mentioned today, I just want to make clear that the non inferiority margin was 4.5 letters. And I just want to emphasize that that is because we're exactly in guidelines with the FDA.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

We've always done that and we expect to do that with everything that we do in regards to study design. In regards to the rescues, we have not detailed the rescue criteria as yet for competitive reasons, but we will do so. But here's what I can tell you. We will detail those when appropriate. You can expect the rescue criteria to be of no surprise.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

They will be absolutely in line with the traditional rescue criteria that you've seen in the past. The FDA has given very clear guidelines of what they think about rescues, not just for us, but for all studies. The FDA has clearly stated and this was stated most recently by Doctor. Boyd in the American Academy of Ophthalmology meeting that the first rescue in a non inferiority study will be allowed as long as it doesn't affect the primary endpoint. And that is a period of approximately three months or so as a rule of thumb before the primary endpoint.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

All other rescues will be subject to review. And again, that's been stated for a number of years now. That is no different than any other clinical trial and that is a general statement that still stands. However, what I can tell you is that I believe we will have a huge advantage due to the design of our clinical trials. Remember, these are complementary clinical trials and remember that SOLAR will never be reviewed by any regulatory agency without a positive SOLAR-one study.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

And when these rescues for SOLAR are being evaluated, it will be evaluated in the context of a positive SOL-one study. So the regulatory agencies will have a very clear idea that this drug will last for nine to twelve months based on a positive SOL-one result. So again, the rescues will be reviewed in context of a positive SOL-one study, which will define the durability of this drug, which I will think will be very much to our advantage. Again, this also emphasizes the advantage that we have of having these two complementary studies. Thanks for your question, Sean.

Sean McCutcheon
Sean McCutcheon
Vice President - Biotechnology Equity Research at Raymond James Financial

Thank you.

Operator

The next question is from the line of John Woven with Citizens JMP. Please proceed with your question.

Jon Wolleben
Managing Director at JMP Securities LLC

Hey, thanks for taking the questions Praveen. Two from us. Can you discuss the change in powering for solar now for the primary? And then stepping back, it seems like these changes have two big implications. One is the accelerate potential timing to a filing, but then also the new label information.

Jon Wolleben
Managing Director at JMP Securities LLC

But how do you think about the importance of those two? Because my expectation would be, if you get a year on the label, the docs could treat and extend as they do anyway. So how much value is that fifty two weeks in the label versus just getting to market quicker?

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

John, good morning and thank you for your questions. Again, very important question. Look, as far as the solar power ring is concerned, we haven't guided you to that publicly. But what I can tell you is nothing changes. We had the eight twenty five patients patient number to satisfy the FDA safety requirements.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

The powering and the statistics really do not change at all. We're able to reduce the size because of the redosing amendment that was approved by the FDA for SOL1. The statistics remain very robust and unchanged for us. In regards to your second question regarding labeling, look, as a physician, I can tell you as someone who has practiced for the last thirty years, the label becomes very important for several reasons. One is scientific.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

It gives you the scientific data of what this drug does, which would be very important to see. The opportunity is there in this study to find out the durability of this drug in a scientific manner. And that is very valuable information for physicians and for patients and for payers alike. The second reason for the labeling is really for the payers, and it will give us a huge commercial advantage. So our goal strategically is to have the best label that the market has seen, a label that is the superiority label, which will be the first and only of its kind potentially and one with a great deal of flexibility of dosing from six months to twelve months.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

Thanks for your question, John.

Jon Wolleben
Managing Director at JMP Securities LLC

Thanks, Kreen.

Operator

Our next questions are from the line of Yi Chen with H. C. Wainwright. Please proceed with your question.

Yi Chen
Managing Director & Senior Analyst at H.C. Wainwright & Co., LLC

Thank you for taking my question. With respect to the MTBR and BME, once you get the FDA feedback on clinical trial design, how quickly do you anticipate to advance those two indications into clinical trials? And do you intend to advance both indications concurrently? Thank you.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

Good morning. Again, thank you for your question. I'm glad you asked about NPDR and DME. And I'm glad that you put those together because I think it's very important to emphasize that with the HELIO study, we have shown absolutely everything to you, which is every single eye of every single patient. And what you'll see there is not only a remarkable result in non proliferative diabetic retinopathy or NPDR, but also in diabetic macular edema or DME.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

In fact, every single patient who had diabetic macular edema on entry of that study improved. And what you saw in the angiogenesis meeting is a remarkable presentation by Doctor. Eller's where he showed that very clearly that these patients with fluid improved significantly after a single injection after forty eight weeks. And so our goal is to pursue not just NPDR, but also DME. How the trial will be designed, we don't know yet because we will still meet with the FDA formally.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

We committed to doing that in the first half of this calendar year. That has not changed. How quickly we will implement that depends on that meeting. So I can't say now. But what I can say and what we did say is that we are financed very, very efficiently.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

We are very disciplined in our financial expenditures. And our intent is to keep the projections the way they are, which is that we are financed into 2028. And at this point, we do not have any intention of requiring any additional funding this year and that remains. Thank you for your question, Lee.

Operator

Our next question is from the line of Greg Harrison with Scotiabank. Please proceed with your question.

Greg Harrison
Director, Biotechnology at Scotiabank

Hey, good morning and thanks for taking the question. I'm curious what your expectations are for the percentage of patients that could show durability to fifty two weeks without rescue in SUL1 and to what extent do you think it could be used that way in practice?

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

Greg, good morning and thank you for your question. The data, first of all, it's very, very valuable information and we don't shy away from this at all because not only because it's very valuable, but we believe that we're going to win quite honestly. We believe that there are a number of patients that will go to fifty two weeks. And this is based on The U. S.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

Study. In The U. S. Study with sixty percent of patients that went that long with a single injection and that was in a nonenhanced patient population. As you know, in previous discussions, we've gone through how painstakingly and thoughtfully we have selected our patients in a bespoke manner for each study to derisk the patient selection and derisk the outcomes.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

And the sixty percent that I quote to you, it really is in a unselected non enriched patient population. So we're very confident of what we will show at week 12. Remember that there are drugs that are in the market now that are approved on label for a two week or so extension, some going to four months with not very many patients that have actually crossed that threshold in their study. So from our point of view, we're going to provide very valuable information here to physicians, payers and patients alike. And we have the opportunity to have the best in class label as well.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

Thank you, Greg for your question.

Operator

Thank you. This concludes our question and answer session. I'll now turn the call back to Doctor. Praveen Dougal for closing remarks.

Pravin Dugel
Pravin Dugel
Executive Chairman, President & CEO at Ocular Therapeutix

Once again, I'd like to thank everyone for taking the time to join us on our call today. We look forward to updating you on our progress. And if you have any follow-up questions, please reach out to Bill Slattery, our Vice President of Investor Relations. Have a great day, everybody. Thank you.

Operator

This concludes today's conference. You may disconnect your lines at this time. And thank you for your participation.

Executives
    • Bill Slattery
      Bill Slattery
      VP - Investor Relations
    • Pravin Dugel
      Pravin Dugel
      Executive Chairman, President & CEO
Analysts
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Earnings Conference Call
Ocular Therapeutix Q4 2024
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