Conference Transcript - OCS 2026-04-16

Serge Belanger, Analyst — Needham and Company LLC

Hi, good morning. I'm Serge Belanger, one of the healthcare analysts at Needham and Company. I want to welcome everybody to Needham's 25th Annual Healthcare Conference. And for our next presentation session this morning, we have Oculus Development Stage Company in the ophthalmic disease space. And from the company, we have the CFO, Sylvia Chung. I think Sylvia has a slide presentations, and then we'll proceed to Q&A afterwards. So, Sylvia, thanks for joining us this morning. We're happy to have you with us, and I'll hand it over to you for the presentation.

Sylvia Chung, CFO

Great. Thank you, Serge. Very happy to be here. Thank you for the introduction, and thanks to Needham for the opportunity to present. And thank you, everyone, for joining the virtual conference. I'm excited to share with you the advancements by Oculus across our late-stage assets. Before I begin, I'd like to point out that forward-looking statements made during this presentation are subject to this Safe Harbor disclosure, and I also encourage you to also review our public filings with the SEC. Oculus is a public global biopharma, and we have three registrational-stage assets targeting a market opportunity of over $25 billion. OCS01 is a first non-invasive eye drop treatment for diabetic macular edema. Licaminimab is a first genotype-based development for dry eye disease. And Previceptor is a first-in-class neuroprotective candidate. And we have a pioneer program for optic neuritis with the FDA and recently granted breakthrough therapy. and the EMA granted prime designation to us. The second indication under the Pioneer Program is NAION. In addition to that, we also have plans to expand into broader indications addressing neuroaxonal diseases. On the financial front, we have a strong balance sheet, no debt, and the current cash runway is into 2029. This is excluding a 100 million Swiss francs loan facility. Our business is comprised of two franchises, an ophthalmology franchise with OCS-01 and licaminimab, both are in the ophthalmology franchise, and the previous sector is the candidate for neuro-ophthalmology. You can see from our pipeline that we have a catalyst-rich horizon driven by three transformative assets that are in active registrational programs. Let me highlight for you the following. OCS01 for DME is currently in final stages of two phase three trials, and we expect top-line readout this quarter in June. Licaminimab for dry eye disease, our registrational study called PREDICT-1, is active, and we expect top-line readout around the end of 2026. PrevalSector's Pyrenea program has three trials, two for optic neuritis and one for NA-ION. The first trial, Pioneer 1, has initiated, and Pioneer 2 and 3 will follow later in the year. We'll now take a closer look at each of the core assets, OCS01, Likminimab, and Privil Sector, and we'll start with OCS01 for DME. The current U.S. DME market is roughly about $3 billion. Only invasive treatments are available today. For the 1.8 million diagnosed DME patients, about half a million is treated and well-controlled. OCS01 is developed to transform DME treatment paradigm by enabling early intervention and effectively treat inadequate control patients. OCS01 is a non-invasive self-administered eye drop treatment. It offers significant opportunity to address two segments of the market where there are no solutions today. OCS01 is positioned to be first-line treatment in the diagnosed and untreated segment. It can also address the inadequately controlled patients as a standalone or an action therapy in the diagnosed and treated populations of the market. Together, the two opportunities combined into one is over 1 million patients, so it's a much larger opportunity than what the current market is serving. OCS01 has demonstrated positive and consistent results across four prior studies, including a Phase 3 Stage 1 trial, which is part of our DIAMON registrational program. Slide nine here is our summary of the protocol for the DIAMOND program, which comprises of two stages. Stage one is a 12-week trial, which was completed, and I will go over the data in the following slides. Stage two contains two 52-week trials, which is near completion. Data readout is expected this quarter in June. Over 800 patients were enrolled in stage two, two trials, 400 patients in each of the trials. The trial has a six-week induction phase followed by a 46-week maintenance phase. Primary endpoint is change in BCVA at week 52. Stage one of the Diamond Program was completed successfully and showed substantial and clinically relevant visual improvements. as well as rapid reduction in retinal edema in both naive and previously treated population. From a safety standpoint, OCS01 was well-tolerated with no unexpected AEs. Here you can see OCS01 showed statistically significant visual improvement with 7.2 letter gain with BCVA versus baseline at week 6, increasing to 7.6 at week 12. Greater than 25% of patients gaining more than 15 letters at week 6, increasing to more than 27% at week 12. OCS01 eyedrop showed favorable efficacy profile in Diamond Phase III Stage 1 study, here in comparison with the published data of Orzodex, which is a sterile implant. You can see that Orzodex's result at week 12 was below 15% of letter gains of greater than 15 letters, while OCS01 at the same time point is over 27%. Top-line results from both of the Diamond Program Phase III trials is expected this quarter in June, and if positive, NDA submission is planned for Q4 this year. Potential approval is in Q4 next year. Now let's move on to licaminimab. It is a precision medicine approach for dry eye disease. Licaminimab is a novel anti-TNF alpha eye drop for ocular inflammation with clinically proven mode of action. Licaminimab is a proprietary genetic biomarker. This antibody fragment technology is specifically formulated for topical delivery and it enhances ocular penetration. It is widely known that dry eye market is large and very unsatisfied, with only 13% of patients experiencing lasting relief after 12 months. 85 to 90% of patients discontinue within six months of prescription drugs. Our solution is licaminumab, a precision treatment approach product to provide a preferred option for physicians and payers for TNFR1 genotype patients. We have three positive dry eye phase 2 trials, two on symptoms and one on signs. All phase 2 trials were completed and consistently showed positive results and potential for precision medicine approach. PREDICT-1 is our first registrational trial for dry eye, and it has been initiated, and the top-line results are anticipated around late 2026. Let's now turn our attention to PrevalSector. PrevalSector is a novel neuroprotective candidate with broad potential for neuroexonal diseases. This new molecular entity is a peptoid small molecule that causes blood-brain and retinal barriers. Purvalsector was selected by high-throughput screening for its unique ability to promote neuronal-axonal survivals. It's validated across multiple in vitro injury models, including apoptosis, oxidation, and inflammation. Preclinical data confirmed neuron axonal survival in glaucoma, MS, and optic neuritis models. Prevalsector received FDA breakthrough therapy designation, as well as EMA prime designation for optic neuritis. Prevalsector has very compelling preclinical data showing prevention of retinal ganglion cell damage, damage, reduction of optic nerve axonal loss, and reduction of optic nerve demyeleneation. The neuroprotection benefits of prevalsector can be translated into several indications. Our initial wave of development focuses on acute indications, being optic neuritis and NAION. Beyond that, there are vast opportunities in chronic indications. An abrupt opportunity applies to both ophthalmic and neurological conditions due to lack of neuroprotection therapies. Privilceptor's first wave of development targets two main optic neuropathies, and they're under the same IND. Optic neuritis and NAION are both rare diseases. There are roughly around 30,000 cases annually for each of ON and NAION separately. Using the low end of the pricing analog range, which is roughly about $100,000 to $400,000 per year, the market is estimated to be over $7 billion U.S. dollars. Coldpoint is very focused and efficient due to the highly concentrated neuro-ophthalmologist population of around 400 to 500 here in the U.S. Let's take a look at optic neuritis, which is an acute inflammation of the optic nerve. It's an orthoindication with around 65,000 patients per year in the U.S. and Europe together. Optic neuritis is a type of neuropathy causing vision loss and pain and can lead to permanent visual impairment. The disease is mainly affecting young women with an average onset at age 32. Optic neuritis has a direct link with chronic conditions like MS and other autoimmune diseases. And in fact, in our phase two trial, the ACUITY trial, two-thirds of our patients were MS patients. NAION is a rare disease, which leads to significant and permanent visual impairment in most patients. Today, there is no treatment available. For patients with NAION, their retinal ganglion cells, axons, and optic nerve atrophy is caused by hypoperfusion, leading to vision loss. The disease causes sudden vision loss and lead to substantial visual impairment in over 60% of patients. Here's a trial design. Here's the trial design for the phase two acuity study in optic neuritis. It's a randomized, double-masked, placebo-controlled, multi-center study. Acuity trial is a first in-patient study, and we had safety primary endpoint. Secondary endpoints are on function, anatomy, and biology. The trial had two arms. The active arm is peripheral sector plus steroid, and a comparator arm is placebo plus steroid. Patients would undergo five daily treatments followed by a six-month evaluation period. Let's take a look at the efficacy data. First on function. Patients with prevalsector treatment achieved an improvement of 18 letters, which is clinically meaningful and is sustained through the study period. Functional improvement we saw on the prior stage also correlated with significant preservation of neurons in the retina. The peripheral sector arm showed 43% better preservation of GCIPL thickness comparing to the vehicle arm, and this is the layer with retinal ganglion cells. The functional improvement that we saw in LCVA also correlated with significant preservation of axons, and this was measured by less thickness loss of INFL, which is the axon layer of the retina. Preval sector benefits also was observed in biological sign of neuronal and axonal death. We saw significantly less neurofilaments released into CSF and blood as a result of axonal and neuronal death. Safety profile reported in acuity phase two showed no AEs leading to drug withdrawal or study discontinuation. As I mentioned, Pervilsector recently received breakthrough therapy designation from the FDA and prime designation from EMA. A global registrational program called Pioneer is underway, and there are three registrational trials in the Pioneer program, two for optic neuritis and one for NAION. Pioneer 1 has initiated and top-line results are anticipated in 2027. In conclusion, Oculus has significantly progressed and advanced toward building a global ophthalmology and a neuro-ophthalmology company. We are currently targeting $25 billion of potential markets with highly differentiated products across our core assets. We have a catalyst-rich upcoming quarters with registrational trials readouts for each of the three transformative assets. First is DME Diamond Program, readout this quarter in June, and FDA submission in Q4. Second is Lecominimab Predict 1, readout is anticipated around the end of 2026. For the approval sector, Pioneer 1 is underway, and its readout is anticipated in 2027, with Pioneer 2 and 3 commencing later this year. We're very excited about the upcoming milestone events and look forward to reporting on those in the coming quarters. On behalf of Oculus, I'd like to thank you for your time and participation. and search. I think there may be some questions. Thanks, Sylvia. Appreciate the overview.

Serge Belanger, Analyst — Needham and Company LLC

I guess first question, I guess on the OCS01 DME program, since we're nearing the phase three readout, one of the questions we get from investors, and I assume that question will continue until we get the readout is what is the the the bar for success for for those results um is it the stage one results that you were able to achieve at six weeks and 12 weeks and is that

Sylvia Chung, CFO

what you'll be looking for at 52 weeks yeah thank you thank you serge yes the readout is upcoming very soon. For us, the bar for success, I would say, base case bar for success is similar to Orzodex in terms of efficacy and safety. And as you know, the active is the same, dexamethasone. And with that particular profile, we'll be able to address a market that is potentially eight to 10 times more patients with a topical solution for patients. And in the early intervention space, nobody owned that space but us once approved. Now, a great scenario will be same safety and higher and better efficacy, which we saw in our previous phase three, stage one. this would give us still the large much larger accessible population but most probably also give us a stronger penetration in those segments and the two segments are on one hand half of the diagnosed population who's currently not being treated on the other hand the treated population where 40 percent of the patients are not getting adequate result in our product will be able to either you use as a standalone treatment or in combination with anti-VegF.

Serge Belanger, Analyst — Needham and Company LLC

Okay. And if Ozodex is the bar for success, what is kind of the, I don't know if there's a specific number or range in terms of letter gains or, you know, 15 line responder, 15 letter or three line responders. Is there a specific number or range for those metrics?

Sylvia Chung, CFO

we uh the metric is uh statistical significance in both of the points that you mentioned we are confident in the outcome based on the previous studies that we have and if we can replicate what we saw in the prior study i would say that it's a great outcome and will significantly aid our launch and market penetration okay and assuming you get statistical

Serge Belanger, Analyst — Needham and Company LLC

significant results, what kind of label would the data from the DIAMOND studies support? Do you expect kind of a broad DME label, and how would it be used?

Sylvia Chung, CFO

So we expect a broad DME label. So as you know, this trial is an all-commerce trial. The board label is consistent with our interactions and discussions with the FDA. previously, including the end of phase two meeting. Okay. And we've already discussed

Serge Belanger, Analyst — Needham and Company LLC

Ozodex. Does that represent kind of a good proxy for what OCS01 could achieve commercially?

Sylvia Chung, CFO

We believe that commercially OCS01, if proven successful from our phase three trial, and we're confident in that, we believe OCS01 can address a much broader market than Ozodex. This is due to the topical nature of the product, where today half of the diagnosed population, so 1.8 million of patients are diagnosed, half of that, so 900,000 patients, so close to a million, today are not being treated. And we will be the only product in that segment addressing patients who are currently on observation. And on the other hand, OZODEX currently is a second or third line treatment and a treated population addressing patients who are not getting adequate results from anti-VEGEP, probably because inflammation is a bigger component of the disease. And we're not looking to displace anti-VEGEP, but in that treated segment, we have a couple of different options to aid the retinal specialist to treat their patients better. either through standalone or through combination got it all right so looking forward to seeing

Serge Belanger, Analyst — Needham and Company LLC

those data in june um on the primosector program um like pretty novel to have a neuroprotection agent maybe you know why was uh acute optic neuritis chosen as the initial indication to demonstrate proof of concept?

Sylvia Chung, CFO

Yes. So acute optic neuritis was chosen by the prior sponsor of the study before we took over. So that particular company was a CNS-focused company. I think it made sense for a couple of reasons. So we weren't there when the decision was made, but looking back, I think it makes sense. One is optic neuritis is characterized by acute inflammation of the optic nerve, leading to significant retinal ganglion cell death. And because the damage is acute, quantifiable, and measurable, visible, it allows for faster evaluation of neuroprotective drugs' ability to preserve structural and functional integrity. So I think that's why optic neuritis was selected. And secondly, looking at optic neuritis being an orphan disease with high met need, I think that also supports the decision to test this neuroprotective agent, which turns out to be very promising from the data that we saw in the acuity study.

Serge Belanger, Analyst — Needham and Company LLC

Yeah. And for both AON and NAION, I believe there's no approved treatments. What were physicians using? Was it just corticosteroids to temper inflation, inflammation, or was there other agents also used for these indications?

Sylvia Chung, CFO

Yeah. So NAION, there is zero, no treatment whatsoever, nothing available. For optic neuritis, currently physicians are using steroid and it's a five-day treatment. So our trial was designed to kind of mirror the current standard of care, which is high-dose steroid, five days of treatment, and our product is an IV infusion, so privil sector. So the acuity study that I was talking about earlier, the active arm was privil sector plus steroid, five daily treatments. Both are a high met need because nothing is approved for NAI-ON, and for ON, there is no neuroprotective treatment for optic neuritis.

Serge Belanger, Analyst — Needham and Company LLC

Yeah, and I imagine because there's no approved treatments, that's why you were able to get the breakthrough therapy designation from FDA and the prime designation from EMEA. Yeah, I guess what is that? how does that help going forward for these programs?

Sylvia Chung, CFO

Yeah, I think the breakthrough therapy and the prime designations really validated the clinical strength in addition to the fact that the two diseases are a rare disease. Both agencies looked at the detailed information from the acuity study and granted the breakthrough therapy as well as the prime designations to us. I think the status enables us to have frequent dialogues with the agencies, which will make a more efficient clinical and regulatory development pathway. And it also has the potential to save time and improve the likelihood of approval, ultimately, you know, earlier patient access. So we're very pleased about the outcome of achieving these two, um, statuses, um, which really speaks to the acuity, um, studies, uh,

Serge Belanger, Analyst — Needham and Company LLC

strength. Yeah. And then I think you had your pre-phase three FDA interactions, uh, last year, um, maybe just discuss how interested the FDA is in a neuroprotective agent for, these indications and the discussions around the endpoint since, you know, there hasn't been anything approved and how you're trailblazing the regulatory path here for both of these

Sylvia Chung, CFO

indications? Yep, absolutely. So while I don't have firsthand involvement in those dialogues, my expert regulatory team did. I think the observation that we have, that I have, is the process was extremely engaging. The FDA is very supportive of the novel development approval sector and is very supportive in terms of helping us to see the product through the regulatory pathway. We're very pleased with the interaction. The trial design is clearly discussed with them with their feedback, and we are very excited to execute on the trial and potentially bring top-line readout to physician community, investment community in 2027 from prior year one.

Serge Belanger, Analyst — Needham and Company LLC

Okay. And then maybe just to finish off on the dryout program that will generate additional data by your end um maybe just talk about i know there's been a couple new entrants in this market the dry market um how it's evolving and where do you think a program that offers a biomarker approach would fit within the the treatment yeah yeah so while while there were a couple of

Sylvia Chung, CFO

recently launched products that offer additional options for dry eye patients. The reality is significant unmet needs still remain. And it's really due to the highly heterogeneous dry eye market. And treatment decisions are still driven by trial and error. people having gone through, tried multiple treatments and not getting the results that they want or deserve. And the vast majority of the estimated 10 million patients, this is the moderate to severe population of dry eyes. So 30 million total, 10 million moderate to severe. the vast majority of them are still struggling to find lasting relief. So what we have in hand is really a fundamentally different approach to treating dry eye with a genotype-based development program, licaminumab. In our phase two study, what we saw was the sign and symptom efficacy results were five to seven times greater in the population TNFR1, the genotype population. And this particular genotype genetic biomarker is roughly about 20% of the U.S. population. So we're talking about 2 million of moderate to severe patients, 2% of the 10 million of moderate to severe patients, so 2 million of genotype positive patients who would have really strong response, efficacy response to licaminumab. We have designed a study and got FDA's agreement on the trial design. And with the proven mechanism of action, anti-TNF alpha, we're basically in a position to potentially become the first line treatment for dry eye patients with this particular TNFR1 genotype. So it's a very unique approach. This approach, just from a commercial and economic standpoint, will be highly beneficial from a payer standpoint, from a PNL standpoint. So let's see. The trial is ongoing. Results are anticipated around late this year. And we'll be looking forward to report on that.

Serge Belanger, Analyst — Needham and Company LLC

And remind me again, do you need to develop a genotype assay for this biomarker?

Sylvia Chung, CFO

The test is currently available. It's pretty rapid turnaround. Think of it as a saliva swap COVID type test. Results will be back within a day. So it's a pretty easy process to identify the TNFR1-positive patients.

Serge Belanger, Analyst — Needham and Company LLC

And most of these patients with this genotype with dry eye disease tend to be on the moderate to severe side of dry eye disease?

Sylvia Chung, CFO

That I do not know. What I do know is 20% of U.S. population is TNFR1-positive. Yeah, whether or not that genotype causes frite eye, it's not something that I'm aware of.

Serge Belanger, Analyst — Needham and Company LLC

Maybe I'll let you wrap up and highlight if there's anything you feel is misunderstood or underappreciated by investors or analysts on any of the three assets that Oculus is currently in late-stage development.

Sylvia Chung, CFO

I think analysts and investors are having much better appreciation of where OCS01 will be placed in the market in the two segments, early intervention, where OCS01 will be standalone with no competition. and on the back end, how we can be an additive to anti-VEGF as well as a standalone to address patients who are not getting the adequate results. I think initially people were thinking of Orzodex as being a benchmark or us displacing anti-VEGF. That's not what we're doing. Anti-VEGF will be there. What we're looking to do is to fill in the two voids that currently exists in the market where we can play, but no one else will be able to. So that is one area, and I think appreciation for that has gotten traction. And for approval sector and lecominimab, we have registrational trials ongoing. Each of these are first in class with very strong historical clinical results, and we're looking to replicate. We also have very positive discussions with the FDA on each of those programs and recently received the breakthrough therapy designation and prime designation from FDA and EMA, respectively, for approval sector. So we're very excited to be delivering multiple registrational trial readouts starting this quarter in a couple months and through 2027. So we're in a good position and happy to update you and the rest of the community on our progress in the coming months. Great. Well, thanks for spending

Serge Belanger, Analyst — Needham and Company LLC

time with us this morning. We appreciate the overview. And I think there aren't many companies that I cover that offer three late stage assets like Oculus does. And, you know, two of them are going to have phase three readouts in 2026. So definitely an exciting time for the company.

Sylvia Chung, CFO

Thank you. Thank you very much, Sarge.