Ocular Therapeutix Q3 2026 Earnings Call Transcript

Ocular Therapeutix (NASDAQ:OCUL) reported third-quarter financial results on Monday. The transcript from the company’s third-quarter earnings call has been provided below.

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Summary

Ocular Therapeutix reported significant progress in 2026, highlighting successful phase 3 results for Xpaxly (OTX-TKI) in wet AMD and positioning it for potential regulatory approval.

The company plans to submit an NDA for Xpaxly in wet AMD in Q4 2026, with a comprehensive package including SOL-1 efficacy and SOLR safety data, aiming for a potential accelerated review via the 505(b)(2) pathway.

Financially, Ocular Therapeutix ended the quarter with $530.6 million in cash, providing a runway into 2028, excluding commercialization expenses for Xpaxly.

Strategically, Ocular Therapeutix is focused on translating Xpaxly’s clinical profile into commercial success, emphasizing its role as a long-term maintenance therapy distinct from existing anti-VEGF treatments.

The company is preparing for a potential launch with ongoing engagement with payers and building the necessary commercial infrastructure to support Xpaxly’s adoption post-approval.

Full Transcript

OPERATOR

Good morning and welcome to the Ocular Therapeutix third quarter 2026 earnings conference call. At this time, all participants are in a listen-only mode. After the prepared remarks, we will conduct a question-and-answer session. If you would like to ask a question, please press star one on your telephone. As a reminder, this conference call is being recorded and will be available for replay on the Investor Relations section of the Ocular Therapeutix website.

I would now like to turn the call over to Ocular’s Vice President of Investor Relations, Bill Slattery Jr. Please go ahead, Mr. Slattery.

Bill Slattery Jr (Vice President of Investor Relations)

Good morning everyone and thank you for joining us today. Earlier this morning we issued a press release and filed our quarterly report on Form 10-Q outlining our financial results and business updates for the third quarter of 2026. We will begin today’s call with prepared remarks summarizing recent business highlights from Dr. Pravin Dugel, Ocular’s Executive Chairman, Chief Medical Officer Dr. Nadia Waheed, and David Robinson, Ocular’s Global Chief Commercial Officer.

Donald Notman, Ocular’s Chief Operating Officer, will join for the question-and-answer session. We refer everyone to this morning’s press release and our Form 10-Q for a comprehensive update of third quarter 2026 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-K and our other SEC filings. On today’s call we will discuss our investigational product candidate Xpaxly, also known as OTX-TKI, which is in development for wet age-related macular degeneration and non-proliferative diabetic retinopathy.

As a reminder, the safety and efficacy of Xpaxly have not been established by the FDA or any other regulatory authority and Xpaxly has not been approved for marketing. With that, I’d like to hand the call over to Dr. Pravin Dugel to review our recent updates. Pravin.

Pravin U. Dugel (Executive Chairman)

Thanks, Bill, and good morning everyone. 2026 has been an extraordinary and truly transformational year for Ocular Therapeutix. We’ve achieved landmark phase 3 superiority results in SOL-1, established a clear regulatory path forward toward our first potential approval for Xpaxly in wet AMD, and continue to generate compelling clinical evidence showing Xpaxly’s durability and disease control. As we approach the end of the year, I believe we are in the strongest position in our company’s 20-year history with an extraordinary opportunity to fundamentally transform the treatment paradigm for retinal diseases.

Today, while several bolus treatment agents are either approved or in development, Xpaxly stands alone as the only intravitreal sustained-release maintenance agent with positive phase 3 data in wet AMD. With a competitive landscape evolving significantly in recent months, we believe our opportunity has never been greater. I want to begin by addressing recent competitive developments because they reinforce the importance of the fundamental principles that have guided our Xpaxly development strategy from the onset.

These include the importance of TKI potency, drug delivery vehicle, and thoughtful clinical trial design including rigorous patient selection. In drug development, molecular potency matters, the drug’s delivery vehicle matters, rigorous clinical trial design matters, and patient selection matters. First, on potency. We have seen potency make a huge difference in wet AMD studies before. For example, Lucentis was considerably more potent than Macugen. Similarly, axitinib, the TKI used in Xpaxly, has demonstrated the greatest potency of the TKIs currently in development for retinal diseases. Axitinib has approximately 100-fold greater potency against VEGF receptor 2 when compared to vorolanib, as well as greater potency at the other VEGF receptors.

We believe this distinction is critical to achieving sustained VEGF suppression over extended treatment intervals. Next, delivery matters. As we know from preclinical studies, our bioresorbable hydrogel is capable of delivering consistent daily release of axitinib. The 9- to 12-month durability observed in SOL-1 provides compelling evidence that the hydrogel performs similarly in humans to what we saw preclinically. Equally important as TKI potency and the bioresorbable hydrogel selected to deliver VEGF suppression is the clinical trial design and patient selection of each trial being evaluated. SOL-1 and SOLR randomized different groups of treatment-naïve patients, since the trials were deliberately designed to answer very distinct clinical questions. In SOL-1, our objective was to demonstrate superiority in vision maintenance over a single administration of aflibercept 2 mg.

Accordingly, we prioritized a challenging population specifically selected to lose vision. Nonetheless, approximately 75% of patients receiving Xpaxly maintained vision at week 36, the primary endpoint, and approximately two-thirds through week 52 following a single injection. In SOLR, our objective is non-inferiority in BCVA to aflibercept 2 mg administered every eight weeks. Accordingly, we incorporated a differentiating six-month screening and loading period to exclude from randomization patients with early persistent fluid or significant fluid variability.

These patients are known to have negatively impacted historical non-inferiority trials. Contrast this with recent developments from a peer that utilized a TKI that is less potent against VEGF receptor 2, in trials that combined previously treated and treatment-naïve patients and randomized patients before the loading phase. We believe our peer’s results do not reflect a limitation of the TKI class, but rather they underscore the importance of getting all three elements right: the TKI and the delivery vehicle, the trial design, and the patient population.

Turning to our regulatory process, our highest near-term priority remains the planned submission of our NDA for Xpaxly in wet AMD in the fourth quarter of this year. Following our positive pre-NDA meeting in September, we remain aligned with the FDA on our planned submission package. Our NDA will be supported by SOL-1 week 52 efficacy data, SOL-1 and interim SOLR week 52 safety data, and a comprehensive body of confirmatory evidence. Importantly, we also plan to submit repeat-dosing safety data from patients who have received multiple Xpaxly injections in both phase 3 trials as part of the NDA review process.

Together, our safety database at the time of submission will include more than 300 patients with at least one year follow-up after Xpaxly treatment, consistent with FDA requirements. We remain on track to conduct the interim SOLR safety analysis this quarter, with these results to be incorporated into our planned NDA submission. Throughout this process we have maintained consistent engagement with the FDA, including our Special Protocol Assessment agreement for SOL-1, our Type C meeting earlier this year, and most recently our positive pre-NDA meeting in September. We also remain aligned with the Agency on pursuing approval through the 505(b)(2) regulatory pathway, which has the potential to shorten the review timeline by up to 60 days compared with the traditional 505(b)(1) pathway for a new molecular entity.

This will be an incredibly important milestone for our organization and one that reflects years of disciplined clinical development, rigorous execution, and extensive collaboration with the FDA. The incredible progress that we’ve achieved only occurs when you have a tremendous team in place. Today you will hear from some of those team members, starting with Dr. Nadia Waheed, Ocular’s Chief Medical Officer, who will speak to recent updates regarding our clinical programs. David Robinson, Ocular’s Global Chief Commercial Officer, will then review the transformative commercial opportunity ahead. Nadia, please go ahead.

Nadia Waheed (Chief Medical Officer)

Thanks, Pravin. Let’s start with SOLAR, our second Phase 3 trial in wet AMD. I want to emphasize that our confidence in this important trial remains very strong and continues to grow. While SOLAR efficacy data are not required for our planned NDA submission, we believe positive results could provide additional commercial advantages in the US and support regulatory approvals internationally. At the recent American Academy of Ophthalmology meeting, we presented a post hoc analysis using an AI-based assessment of retinal fluid. This analysis demonstrated that approximately 71% of Expaxly-treated subjects were quantitatively dry at week 8, meaning they had less than or equal to 10 nanoliters of total fluid volume in the 6-by-6 millimeter scan under this stringent definition of dryness. Remarkably, approximately 70% of these subjects remained quantitatively dry through week 36 following a single Expaxly injection.

We believe these findings provide more than just another illustration of Expaxly’s potential durability. They offer clinicians a potential roadmap for treatment: achieve initial disease control with a couple of bolus anti-VEGF injections, then transition to Expaxly for long-term maintenance treatment. Importantly, recent SOL-1 post hoc analyses have also provided compelling insights into the relationship between early retinal fluid control durability and long-term outcomes, reinforcing our confidence in the SOLAR study design. At last week’s Empire Forum in New York City, we presented a separate post hoc analysis reporting that approximately 28% of Expaxly-treated subjects in SOL-1 experienced early persistent retinal fluid or fluid variability, defined as a greater than 30-micron increase in central subfield thickness at week eight.

These patients had worse visual outcomes, a 32-week earlier time to supplemental injection, and an increased frequency of needing supplemental injections compared to subjects without early persistent fluid in the same period, and this was consistent across both Expaxly- and aflibercept-treated patients, reinforcing the importance of early disease stability independent of treatment assignment. And this is precisely where the design of SOLAR becomes so important. SOLAR incorporates a six-month prerandomization period specifically designed to screen out patients with early persistent retinal fluid or significant fluid variability. Our preliminary analysis indicates approximately 20% of patients otherwise eligible for SOLAR were excluded from randomization because of these characteristics. This is also consistent with historical expectations and closely aligns with the findings from SOL-1.

In short, SOLAR is designed to randomize the very population in which we observed the strongest durability profile for Expaxly in SOL-1. As it relates to SOLAR, I’d also like to address an important refinement to the primary endpoint, which reflects our priority to protect the integrity of our primary analysis. As part of our NDA planning, we will conduct an interim safety analysis on a subset of SOLAR patients to meet the FDA’s requirement of at least 4,300 subjects with one year of Expaxly follow-up. We selected week 52 for the interim SOLAR safety analysis because it’s consistent with the FDA guidance, aligns with the 52-week follow-up period in SOL-1, and it enables our planned fourth-quarter NDA submission, with the original primary efficacy endpoint scheduled for week 56.

Our interim safety analysis would have required unmasking the safety data before all patients completed their primary efficacy assessments. Rather than risk compromising the primary endpoint’s statistical integrity, we proactively initiated a process in May after our Type C meeting with the FDA to move the primary endpoint from week 56 to week 40. This change ensures all patients will have completed their primary efficacy assessments before the planned interim safety analysis.

Importantly, the new week 40 primary endpoint exceeds the FDA’s minimum 36-week requirement outlined in its draft guidance for neovascular AMD and is within the treatment effect observed in SOL-1. Due to the interim safety unmasking, we will incur only a minimal statistical alpha penalty of 0.0001 towards our primary endpoint. Equally important, we have preserved our key secondary and other secondary endpoints at week 56 and 96, including longer-term evaluations of superiority to aflibercept 8 mg and the potential prevention of fibrosis and atrophy relative to aflibercept 2 mg.

It was important for us to complete the rigorous process of changing the primary endpoint time point, including protocol implementation, IRB approvals, and FDA interactions, before conducting the interim safety analysis in SOLAR. As per our prior guidance, SOLAR will remain masked through week 96 under our FDA-aligned masking plan, with top-line results on track for the first quarter of 2028. I want to emphasize that the foundation of these changes is to preserve the integrity of SOLAR, and they are aligned with the FDA.

We believe this approach accomplishes two critically important objectives, simultaneously advancing our near-term NDA submission while preserving the long-term clinical, regulatory, and commercial value of SOLAR. Beyond SOL-1 and SOLAR, we are encouraged by the continued momentum in SOL-EX, our long-term open-label extension study, with the vast majority of investigators and eligible patients choosing to participate. Importantly, based on our recent analysis of the up to 12-month durability demonstrated in SOL-1, we are modifying SOL-EX’s dosing cadence from 24-week treatment intervals to 48-week treatment intervals. Simply put, we believe SOL-1 demonstrated that many patients do not require Expaxly treatment every six months, and this change to SOL-EX reflects our confidence in Expaxly’s durability.

This modification is expected to provide commercially valuable evidence supporting Expaxly’s potential for an annual dosing while also evaluating whether sustained VEGF suppression and earlier initiation of Expaxly treatment may reduce fibrosis and atrophy and improve long-term visual outcomes. Stepping back, our three wet AMD trials are strategically designed to provide clinicians with a comprehensive roadmap for Expaxly’s use in clinical practice. SOL-1 established superiority over a single injection of aflibercept 2 mg, demonstrated durability extending up to 12 months, and characterized performance in a particularly challenging patient population. SOLAR is designed to demonstrate noninferiority to on-label aflibercept 2 mg administered every eight weeks and to establish the safety of repeat Expaxly treatment every six months in all patients.

SOL-EX is designed to evaluate 12-month maintenance dosing and potential long-term visual and anatomic benefits of initiating Expaxly earlier in the disease course. Together, these studies are designed to establish a comprehensive clinical profile supporting Expaxly’s efficacy, durability, and flexibility, further strengthening its potential to become the preferred sustained-release maintenance therapy in wet AMD. As we advance Expaxly towards potential approval, we are increasingly focused on translating its differentiated clinical profile into meaningful benefits for physicians and patients. David will discuss how we are preparing to deliver on that opportunity. David.

David Wayne Robinson (Global Chief Commercial Officer)

Thank you, Nadia. Besides the opportunity to work with this tremendous team, I was drawn to Ocular Therapeutix for a simple reason: to redefine the retina disease treatment paradigm and improve patients’ lives with Expaxly. Our differentiated clinical profile shown in SOL-1, combined with the significant unmet need in the wet AMD market, positions us to redefine the standard of care and deliver meaningful benefits to patients, physicians, and the broader health care system.

Central to realizing this opportunity is our belief that Expaxly addresses a fundamentally different treatment need than the anti-VEGF therapies available today. We believe that it’s critical to distinguish between bolus anti-VEGF therapies used to achieve initial disease control and Expaxly’s intended role as a long-term maintenance therapy. Retina specialists have numerous bolus therapies available today, with additional products in development.

Physicians can choose the bolus therapy appropriate for their patients to dry the retina over a brief initiation period before transitioning to Expaxly for long-term maintenance, if approved. We view the anti-VEGF therapies as complementary to Expaxly rather than direct competitors for our intended maintenance opportunity. And importantly, maintenance represents more than 90% of the wet AMD treatment market. We believe a long-duration treatment like Expaxly will be broadly welcomed, as today’s frequent bolus dosing leads to over 40% of patients being lost to therapy in the first year alone.

Furthermore, following the recent discontinuation of a peer TKI program in wet AMD, we believe Expaxly has no direct late-stage intravitreal sustained-release competitor. Our successful SOL-1 superiority outcome also provides a compelling basis for commercial differentiation. If Expaxly were to receive a superiority label, it could also support premium pricing and potentially help avoid traditional step-edit requirements. Our market research demonstrates strong physician interest based on SOL-1 data alone, including our recent post hoc analysis demonstrating almost 70% of patients remain dry on the strict AI-based algorithm at 36 weeks.

We continue to invest in commercial infrastructure, market access, and launch execution capabilities. Our payer team has conducted, and continues to advance, meaningful preapproval information exchange discussions with 100% of our top-priority tier national and regional payer accounts, covering roughly 84% of commercial lives. These conversations have been well received, with clear interest in Expaxly’s potential to deliver fewer injections and stronger disease control through superior durability.

Our intention is to be fully prepared for Day One following potential approval, with the capabilities and infrastructure necessary to support rapid physician adoption and broad patient access. I look forward to speaking more about our launch preparation activities in the coming months. With that, I’d like to turn the call back over to Pravin.

Pravin U. Dugel (Executive Chairman)

Thanks, David. As you heard today, we are in an exceptional position as we prepare for our planned NDA submission and potential approval in wet AMD. In preparing for launch, today we also announced a change in our board composition as we shift to prioritize commercial expertise. Dr. Riyadh Sharif has joined our board of directors and Dr. Sung Se Hong will retire from the board at the end of the year. We are incredibly thankful to Dr. Hong for his contributions to Ocular during his board tenure.

We are thrilled to welcome Dr. Sharif to Ocular’s board at such an important inflection point in our company’s evolution. He is a highly accomplished leader with a rare combination of deep ophthalmology experience, global commercial experience, and a proven ability to build and lead successful organizations. Having served in senior leadership roles at Alcon, Novartis and Sanofi, and now as the CEO of Oculis, Dr. Sharif brings decades of invaluable experience navigating complex global markets, developing commercial strategies and driving organizational growth.

As we advance Axpaxli toward a potential regulatory approval and prepare to establish Ocular as a global leader in retina, his strategic insights and commercial perspective will be tremendous assets to our board. We’re delighted that he shares our conviction in the transformative potential of Axpaxli and has chosen to contribute his experience to Ocular’s next chapter. In our conversations, Dr. Sharif has shared with me that his conviction only starts with our opportunity in wet AMD, extending to the opportunity for Axpaxli in non-proliferative diabetic retinopathy as well.

In the United States alone, approximately 6.3 million people suffer from this vision-threatening disease and less than 1% are actually treated. Axpaxli has the opportunity to drastically change this unfortunate reality. Enrollment continues to progress in HELIOS 3, our Phase 3 trial evaluating once-yearly Axpaxli across diabetic retinal disease including patients with non-center-involving diabetic macular edema. We remain enthusiastic about this substantial opportunity and you can expect to hear much more about our diabetic retinal disease strategy in 2027.

Financially, we are well positioned to capitalize on the opportunities ahead. We ended the third quarter with $530.6 million in cash and cash equivalents, providing an expected runway into 2028 based on our current assumptions but excluding the full anticipated expenses of commercializing Axpaxli. We remain disciplined in our capital allocation while investing strategically in our regulatory, clinical and commercial priorities. Before we move to Q&A, I want to leave you with several important takeaways from today’s call. First, we remain on track to submit our NDA this quarter, supported by a comprehensive NDA package and FDA alignment. Second, recent competitive developments reinforce that TKI potency, delivery vehicle, thoughtful clinical trial design and patient selection are essential for success. These principles have been foundational to our Axpaxli development strategy and are reflected in our landmark SOL-1 superiority results.

Third, new SOL-1 post hoc analyses provide a potential clinical roadmap for Axpaxli maintenance treatment and reinforce our confidence in SOLAR, with preliminary SOLAR screening data supporting our patient selection strategy. Fourth, our proactive modification of SOLAR’s primary endpoint preserves trial integrity and enables our near-term NDA strategy while protecting important longer-term efficacy assessments and remaining in alignment with FDA guidance.

Fifth, our three wet AMD trials are designed to establish a compelling and comprehensive clinical profile. SOL-1 demonstrated superiority and durability. SOLAR is designed to demonstrate non-inferiority to on-label aflibercept 2 mg and safety of every-six-month repeat dosing, and SOLE-X will evaluate potential long-term benefits of annual Axpaxli maintenance treatment. Sixth, we believe Axpaxli has an extraordinary opportunity to establish a new standard of maintenance treatment in wet AMD and we’re building our organization to capitalize on that opportunity.

And finally, we continue to advance our broader development program, including our registrational HELIOS 3 study in diabetic retinal disease, positioning Ocular for significant long-term growth. We have never been more confident in our differentiated approach, our execution and the opportunity ahead. We remain laser-focused on bringing Axpaxli, if approved, to patients as quickly as possible and delivering on what we believe is a truly transformational opportunity.

Thank you. And with that we would be happy to take your questions.

OPERATOR

Thank you. As a reminder, if you would like to ask a question, please press star-one. Our first question comes from Biren Amin with Piper Sandler. Please go ahead, your line is open.

Biren Amin (Analyst at Piper Sandler)

Yeah, hi guys, thanks for taking my questions. Maybe on SOLE-X and the change in the treatment interval to 48 weeks versus 24 weeks. What new data led to this change? And given wet AMD is a heterogeneous disease, will you offer 48-week frequency to all patients in SOLE-X or will treatment be guided by functional and anatomic endpoints in SOLE-X? Thank you.

Pravin U. Dugel (Executive Chairman)

Good morning, Biren, thank you very much for your question. Let me take a crack at that. And I’m fortunate enough to have Nadia here with me and I’ll let her give you the complete and full answer. So what I would like to share with you is sort of the package that we have in terms of the information we provided to clinicians in SOLE-X. This change in SOLE-X is very much a part of that. And I want to also add that that is a reflection of our confidence in the data that we continue to analyze in SOL-1 and the data that we continue to analyze in a patient population that’s specifically designed to lose vision, and people forget that. Remember this is a very select patient population that’s specifically selected to lose vision.

The fact that two-thirds of these patients were rescue-free with a single injection at 12 months gives us an enormous amount of confidence. And now you’ve heard our presentation in Academy and more recently New York that shows the level of disease control that this drug is able to obtain. So the decision that was made to prolong the interval is made out of that level of confidence based on the data that we’ve analyzed. But think of the data set that we’ll be presenting to clinicians. So there are three of them. First of all, what does SOL-1 answer? Well, SOL-1 tells us first of all the safety of this drug. We had provided unprecedented patient-level safety data from the onset. I think very few, if anybody, has done that from the onset.

SOL-1 also gives you an insight into disease control in this very difficult patient population. What we’ve shown is that with a single injection of Axpaxli at month nine, the OCT is within a stability of 30 microns. The other thing that SOL-1 shows, as I mentioned, is the durability of this drug, which is with a single injection in this difficult patient population, two-thirds of patients are rescue-free at month 12. And now at SOLAR, what we’ll be able to show is the repeatability of every six-month dosing versus aflibercept on-label. With SOLE-X, what we didn’t want to do was to simply duplicate that same information with every six-month dosing. Given the amount of confidence that we now have in this drug, we wanted to provide evidence that this drug can last for a year, especially in a patient population that is already stable. So for that reason we’ll provide the every-one-year maintenance data.

And that’s really the logic behind extending SOLE-X. We’ve really given the clinicians, I believe, a roadmap of how to use this drug. We’ve shown that you can use two bolus agents twice or two bolus injections. And if the eye is dry, you can go ahead and use Axpaxli once. And there’s an over 70% chance that that eye will remain dry for another nine months. And remember, our definition of dryness is really the most stringent definition—that’s within 10 nanoliters of volume—that’s ever been used.

So putting that all together, I think we’ve really given the clinicians a roadmap of how to use this drug. Let me pass this over now to Nadia who will also give you her view of these three different studies. Nadia.

Nadia Waheed (Chief Medical Officer)

Thank you, Pravin. And thank you, Biren. So, Pravin, I think you’ve laid that out really nicely and I completely agree with what you’re saying over here. Let me emphasize, the more we’ve looked at SOL-1 data, and the more we’ve looked at the various different patient subgroups within the SOL-1 data, the more our confidence in the durability of Axpaxli has grown, especially in a cohort of patients that are now in the SOLE-X study two years out from when they started having active disease.

And so part of this change is driven by that confidence in the durability and stability that this medication gives us. And I think also our goal all along has been, while each one of the studies selects—in the SOL-1 study we had a broad coordination that were designed to fail; in the SOLAR study where we have a cohort of patients that are designed to be stable—together these represent the majority of patients of wet macular degeneration that clinicians will see in clinic. And so our goal is to provide a roadmap for clinicians to treat these patients. And SOLE-X, I think, gives us the opportunity to do that.

We would like not to just recapitulate the data that we’re producing in SOLAR that shows stability with every six-month dosing. Over here, now we’re going to show you, backed with the data that we have from the SOL-1 study, how you can go to once-a-year dosing. And I think that this is also reinforced by the decisions that we’ve taken in our diabetic studies of putting these patients on once-a-year dosing. So really as we go along, our confidence in the durability and its ability to maintain stability grows for Axpaxli.

And that’s what we’re hoping to demonstrate with SOLE-X.

Pravin U. Dugel (Executive Chairman)

Thank you. Biren, thank you for your question.

OPERATOR

Thank you. Our next question comes from Taseen Ahmad with Bank of America. Please go ahead.

Taseen Ahmad (Analyst at Bank of America)

Hi, good morning, and thank you for taking my questions. Just a couple to clarify, Praveen, on the SOLE-X extension study, have you already discussed with FDA that you want to make this change to the endpoint? And then just to confirm, is there any read-through to any potential label that you would have with the upcoming filing that you’re going to do in the first quarter? And then also can you just remind us how many patients have received multiple doses from SOL-1 and SOLAR and at what intervals those have happened?

Pravin U. Dugel (Executive Chairman)

Tazeen, good morning, and thank you for your question. Everything that we do is aligned with regulatory agencies. We really don’t do anything at all, as you know, without informing, discussing, and being aligned with regulatory agencies. So any changes that are made, including the extension for solex — which again is made out of utter confidence in this drug — has certainly been discussed with and aligned with regulatory agencies. That’s very important to say as far as the label is concerned.

Tazeen, obviously, it’s way too early to have labeling discussions, but we expect our label to be what I’ve been saying from the very beginning. Again, this is our expectation. We aren’t in discussions, obviously, with the FDA as yet. We expect to have really the best label that exists in this field. We expect the first and only superiority label. We expect the flexibility of dosing every 6 to 12 months with repeatability. As far as repeat dosing is concerned, remember that every patient in SOL1 at week 52 underwent repeat dosing, and everybody in SOLAR obviously is undergoing repeat dosing.

And when the FDA does look at our complete filing, they’ll have plenty of data on repeat dosing based on the SOL1 patients as well as the SOLAR safety patients that we’re going to provide. Nadia, anything else to add?

Nadia Waheed (Chief Medical Officer)

Tazeen, thank you for the question. And Pravin, I 100% agree. I mean, I think all of the 300 or more than 300 patients that we submit as part of our regulatory safety package, either at baseline or at the safety update, will have received repeat dosing. And indeed, at this point, all of the patients that continue to remain enrolled in our trial, whether that be SOL1 or SOLAR or the ones that roll over into solex, all of those patients have received repeat dosing.

So we have a wealth of data on repeat dosing at this point.

Pravin U. Dugel (Executive Chairman)

Thank you, Nadia. And Tazeen, I think the emphasis here is that the FDA will have more than enough repeat dosing data to analyze, and we’re very happy to provide that to them. Tazeen, again, thank you very much for your question.

OPERATOR

Thank you. We will move next with Tara Bancroft with TD Cowen. Please go ahead.

Sam (Analyst at TD Cowen)

Hi, this is Sam on for Tara. Thanks for the update and taking our questions. So on the SOLAR primary endpoint change, did the FDA request that you make this change from week 52 to week 40? And it would be helpful if you could walk us through the timeline of your regulatory interactions with the agency on this matter, starting from after the Type C meeting until it was finalized. Thank you.

Pravin U. Dugel (Executive Chairman)

Good morning, Sam, and thank you for your question. So what I would like to say is, look, we said this from the very beginning. We obviously don’t talk about the details of our interactions with regulatory agencies other than to say that there’s nothing we do without being aligned with them. Remember what we said from the very beginning regarding SOLAR, we continue to believe that the SOLAR efficacy data will not be required for approval in the U.S. We continue to believe, more confidently than ever — and I emphasize more confidently than ever — that expaxly will get approved by the U.S. FDA based on a single trial, which is SOL1, as we’ve always said, with our complete package.

However, we’ve also stated that we believe that SOLAR will be important for some regulatory agencies outside the U.S. We’ve also stated from the onset that we will do everything possible to protect the integrity of SOLAR. And this is a consistent theme that you have heard. And the third thing is we’ve always said that everything that we do, we do with discussion with and alignment from regulatory agencies. And what I’ll tell you, Sam, is that the discussion regarding the primary endpoint began immediately after our Type C meeting, which was in May, as you know.

And the discussion ranged around how to make sure, above all, that the integrity of SOLAR is protected and that there are no questions whatsoever regarding any operational bias. As you also know, the U.S. FDA regards week 36 as an acceptable one-year primary endpoint if you’re using treatment-naive patients. And you know this because that’s what we have in SOL1. It made complete sense, and it was completely logical then to say that we would have an endpoint which would not necessitate any questions about operational bias if we picked an endpoint where patients had already crossed. And at week 40, which is well beyond the week 36 requirement, patients would have already crossed, and there would be absolutely no question about operational bias whatsoever.

And again, this is in line with what we have done to protect the integrity of SOLAR and makes complete sense and is completely aligned with regulatory agencies. Having said that, let me ask Nadia to give us some of her thoughts. Nadia?

Nadia Waheed (Chief Medical Officer)

Thank you, Praveen, and thank you, Sam, for that question. So first and foremost, everything that we do, we are in lockstep and aligned with the regulatory agencies. And everything that we do is focused on, first, ensuring that we have a successful regulatory pathway and, second, ensuring that we have a de-risked regulatory pathway so that we can get axfaxi out to patients as soon as we possibly can. And this change is also focused on that very thing.

So the reason for the 40-week change for the primary endpoint, as Praveen mentioned, was to ensure that at the time of unmasking of a subset of patients for the FDA for the NDA submission to the FDA, we wanted to ensure that each and every one of the patients in SOLAR had already crossed their primary endpoint time and that the database for that — for the primary endpoint — was frozen and locked, so that there was no question whatsoever of operational bias impacting our primary endpoint.

And that is what drove this decision to change the primary endpoint to 40 weeks. And, of course, the week 56 remains one of our key secondary endpoints that we will also look at. And I think part of this change is to ensure — well, all of this change is to ensure — the integrity of the trial. But part of this is also driven by the fact that when we look at the data in SOL1, we’re increasingly confident about both the efficacy as well as the durability of ax pacsi, no matter what time point along that 56-week or one-year pathway we look at. At every single time point along those lines in SOL1, we were seeing exceptionally good results.

And you saw that presented this weekend when we presented our data, for example, at the Empire Forum, where we saw that patients who were dry at the eight-week time point continued to be dry up to the nine-month time point. And so that increasing confidence drove our ability to make these changes and to ensure integrity of our clinical trials and to have a minimal alpha impact on our primary endpoint as a result. So I’m going to stop over there. Praveen.

Pravin U. Dugel (Executive Chairman)

Thank you, Nadia. And Sam, I want to emphasize what Nadia said. We spent a lot of time before talking about how the 56-week primary endpoint was optimal based on peaks and troughs and so on and so forth. You know, we’re beyond that at this point, and we’re beyond that because we are so confident in the expaxolide data based on our SOL1 analyses that it really doesn’t matter, as Nadia said, what time point we use. This drug lasts easily for 12 months in the most difficult patient population that you can possibly select.

And these are patients who are designed to lose vision. There is no doubt whatsoever in our mind that in a patient population that’s super de-risked and super selected to be stable, as we have with a six-month ramp in SOLAR, at every time point this drug will succeed. So at this point, rather than trying to optimize the primary endpoint, the more important thing for us really is to protect the integrity of the trial, to have no discussion whatsoever regarding operational bias, and to be completely aligned with regulatory agencies.

And that’s the reason behind the decision.

Nadia Waheed (Chief Medical Officer)

I’d just like to add one additional thing over here. I think we’ve shown very nice data on fluid that demonstrates the zero-order kinetics and the extended release and sustainability of our medication. And what we’ve shown is that once you have that drying in this population of stable patients, you have rock-solid control all the way out to nine months, one year. And so, you know, we’re much less prone to fluctuations than bolus therapies like aflibercept, where there is some up and down with the eight-week bolus dosing.

And that confidence is really what’s driving our ability to make this change.

Pravin U. Dugel (Executive Chairman)

And to add on to that, you know, look, when Nadia talks about rock-solid stability, Sam, I think it’s important to emphasize that the criteria that we’re using, which is 10 nanoliters, is the most stringent that I know, and it’s about as dry as you can get. And it’s important, when people talk about dryness in other studies, to ask them, what exactly do you mean by dryness? I don’t know of any other analysis, any other study, that has used a criterion of 10 nanoliters, because it’s extraordinarily stringent.

So when we talk about dryness and rock-solid stability, we really mean this. And remember, this is in the most difficult patient population. This is patients that are specifically selected to lose vision. So if we can have that kind of disease control in patients who are specifically chosen to lose vision, we are more confident than ever that we’ll have superb control, even better control, in patients that are specifically de-risked to be as stable as possible.

Sam, thank you for your question.

OPERATOR

Thank you. We will move next with Lisa Walter with RBC Capital Markets. Please go ahead.

Lisa Walter (Analyst at RBC Capital Markets)

Good morning, and thanks for taking your questions. Maybe just another one on the SOLAR primary endpoint change. Just curious if you considered changing the safety interim analysis to week 56 instead to be in line with the timing of the prior primary endpoint. And given the change to 40 weeks for commercial viability, do you think you will need to hit the secondary endpoint at one year for commercial uptake? Any color here will be helpful. Thanks so much.

Pravin U. Dugel (Executive Chairman)

Lisa, good morning, and thank you for those questions. You know, first of all, look, the 52-week safety data analysis absolutely coincides with our timing for the full NDA package submission. So, you know, again, our discussions with regulatory agencies are something that we don’t make public, but it completely also aligns with our discussions with the regulatory agencies as far as data is concerned for, you know, week 40 versus week 56. Look, in SolX we’ll have data for week 48; we’ll have more than sufficient data to show that this drug will last for a year in most patients if we can.

Again, it’s easy to sometimes overlook the patient population in Sol 1. I want to also again emphasize, and I know I’ve said this before, this is the most difficult patient population. These are patients that were specifically anti-VEGF dependent. We selected for them and then we took away the anti-VEGF and we allowed them to lose vision. That’s exactly what we did. And in this patient population, if we can have a durability in two-thirds of patients for a year, I think that provides more than enough data based on the longevity of this drug, and we believe that that will be even greater when you look at the patient population that’s specifically chosen in Solar, who are the most stable patients that you can possibly have.

I want to emphasize what I said earlier on, which is that it’s important to look at the totality of our program. Essentially three different studies here in wet AMD, right? We’ve got Sol1, we’ve got Solar, and we have SolX. And there was absolutely no reason to repeat the data that we would have in Solar by treating every six months, so we extended that to every 48 weeks. And given together in that triad of programs, I think the clinician will have more than enough information as to the safety, the disease control, as well as the durability of this drug.

Nadia, your thoughts?

Nadia Waheed (Chief Medical Officer)

Completely agree, and thank you, Lisa, for that question. I mean, I think the week 52 time point, by the way, for the safety submission for the FDA makes a lot of sense because it’s also consistent with the Sol 1 safety data that we have at week 52. So it just makes it so that all of our patients have that safety cutoff at the same time. So I think there’s that operational component to it that enables us that, you know, partly drove this decision.

Having said that, you know, we will have obviously safety data not only at the one-year time point, but also at the two-year time point with the Solar study, which, as you know, remains masked all the way out to two years. I think Solar will provide us a wealth of information that is going to be very useful for clinicians, including superiority against 8 mg and including data on long-term outcomes such as fibrosis at the two-year time point. So I think that Solar is a critically important study from that perspective.

Having said that, and I’m sure you’re familiar with the data that we just went ahead and presented in Sol 1, I think that we are using post hoc SOL1 analysis to really provide a roadmap for clinicians to adopt XPAXI and to provide guidelines around adoption of XPAXI in clinic. And this is data that, you know, it’s a gift that keeps on giving. You know, as you saw in the analysis that we presented, if we treat patients initially to dryness, which is generally one or two or three injections, we then maintain this dryness with a single injection of XPAXI all the way out to the nine-month time point.

And this is data that we presented at both AAO as well as did some additional analyses at the Empire forum that we presented. And I think that this is a really nice roadmap around durability and maintenance therapy that we’re now providing to clinicians, which I think is going to be really important, especially in this landscape where we may well be the only in-clinic administered durable maintenance therapy available to clinicians. So I’m going to end with that. And Praveen, back to you.

Pravin U. Dugel (Executive Chairman)

Thank you. Nadia, Lisa, thank you so much for your question.

OPERATOR

Thank you. We will move next with Jack Allen with Baird. Please go ahead. Your line is open.

Nick

Hey everyone, it’s Nick on for Jack. Thanks for taking the question. One I had was just on what communication would you all get surrounding the 52-week safety analysis on Solar and would a failure on the primary endpoint halt the study? So, in other words, if you get past the primary endpoint and we don’t hear any news, can we assume that the study is positive? And then also on the 52-week Solar on the NDA submission, are there other gating factors on the submission besides hitting that 52-week Solar endpoint?

Thank you.

Pravin U. Dugel (Executive Chairman)

So Nick, again, thank you. Good morning, and thank you for your question. Just to be very clear, as part of the complete NDA package, we are only providing safety data from Solar at week 52. That is all that’s been requested. And what that does is to fulfill the FDA’s requirement of having at least 300 patients who are exposed to the drug. They don’t necessarily require the same target. Usually they would ask you for the same population of patients.

Age-wise they don’t even require the same dose. It can’t be a lower dose, but it can be a higher dose. And that’s why we’re providing that safety exposure only. No efficacy, just the safety exposure only from Solar as part of our complete NDA submission. What we don’t want to do is during the review process, we don’t want to be adding new data that would trigger a major amendment because, as you know, with a major amendment, the clock starts all over again and we’ll lose the advantage that we have currently being in an accelerated track with the 505 designation. As you know, the card turn for Solar will be at week 96 and that has not changed. So up to that point, the study will be completely masked and all we’ll be providing is simply the safety data up to week 52.

And that’s it. Nothing else. No efficacy data, just the safety data. With what we’ve done today, what we’ve ensured is that the integrity of Solar will be completely protected. Because when we provide that week 52 safety data, all the patients will have already crossed what is now the 40-week primary endpoint. So there’s going to be no risk whatsoever of any operational bias and there’s going to be complete protection of the integrity of Solar. Let me see if Nadia has some thoughts on this as well.

Nadia Waheed (Chief Medical Officer)

So thank you, Nick, for that question, and exactly aligned with what you’re saying, Praveen. The Solar study, I just want to emphasize, remains masked. It remains masked all the way up to 96 weeks while we freeze the database for the primary endpoint at the 40-week time point before we do the safety unmasking. Keep in mind that even within the company, even the safety data is actually masked for the company, and that safety data is getting submitted to the FDA.

But overall, for the study itself, we remain completely masked out to week 96. And there are several good reasons for that, including we need to maintain the masking to be able to produce the superiority data against 8 milligrams as well as to be able to produce the fibrosis data that we hope to produce, which are all key secondary endpoints for us in the Solar study. Of course, what this enables us to do is leverage the patients in the Solar study to provide us with those things which we believe will be very significant advantages, both adoption-wise as well as commercially, as well as significant advantages of durable maintenance therapy. And just based on the—going back to what you were saying—just based on the data that we’re looking at for patients that don’t have early persistent fluid, which, as you know, was a patient cohort that was removed from the Solar study, we only have patients who had no evidence of early persistent fluid.

We’ve presented data at this point that shows us that, you know, there’s almost no credible paradigm under which Solar would not be successful, whether we look at week 40, whether we look at week 56, or whether we look all the way out to week 96. So our confidence in the success of Solar continues to grow the more we look at our data coming out of Sol 1 and, you know, the more we look at the data that we’ve been able to produce out of Sol 1 at this point.

Praveen, anything?

Pravin U. Dugel (Executive Chairman)

Thank you. And Nick, thank you so much for your question.

OPERATOR

Thank you. This concludes our question and answer session. I will now turn the call back to Dr. Pravin Dugel for closing remarks.

Pravin U. Dugel (Executive Chairman)

Thank you once again. Thank you all for joining us today and for your continued support. We have an incredible opportunity ahead, and we are doing everything in our power to execute on this exciting vision that we have outlined. If you have any questions, please reach out to Bill Slattery, our Vice President of Investor Relations. Have a great day, everyone. Thank you.

OPERATOR

Thank you. This brings us to the end of today’s meeting. We appreciate your time and participation. You may now disconnect.

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