Good morning, everyone. Wainwright 27th Annual Global Investment Conference 2025. My name is Daniel Smith, and I'm an H.C. Wainwright Equity Research Associate in Biotechnology. With that said, let me introduce our presenter for the session. I'd like to welcome Dr. Elisabet de los Pinos, Founder and CEO of Aura Biosciences, who are developing virus-like drug conjugates for the treatment of cancer. Aura is traded on the NASDAQ under the ticker AURA. Elisabet, the floor is yours.
Thank you, Daniel. It's a pleasure to be here. Aura is well-positioned for continued clinical program execution, and we can show here across four pillars how we are building value for investors. The first is innovation. We are developing a novel class of drugs. The second, we are in clinical trials and it's late-stage development. We're currently running a phase 3 that has a SPA agreement that supports registration in the treatment of early choroidal melanoma. We also have clinical trials in development for bladder cancer. Both areas, ocular oncology and urologic oncology, are, first of all, areas of high-end medical need. We can really transform the treatment paradigm for these patients.
As a publicly traded company, we have key upcoming milestones that you should be paying attention to, especially, our next milestone is going to be the completion of enrollment of our phase 3 study in early choroidal melanoma, as well as data of our bladder cancer study. We have current cash operations well into the first half of 2027. If you look at our pipeline, at our two therapeutic areas, ocular oncology and urologic oncology, one thing that I want to highlight for you is that ocular oncology has over three indications, and all of those are in clinical development, and all of those we can target with the exact same virus-like drug conjugate. The first one, as I said, is in phase 3, enrolling currently.
The second one, metastasis to the choroid, is about to start a phase 2 study, and cancers of the ocular surface are going to be in phase 1 before the end of the year. All collectively, these are 66,000 patients that have currently no treatment options, and we can truly disrupt this market and position not only as a first-in-class but first-to-market opportunity. In bladder cancer, it's a very exciting development. I'm going to cover some of the data of our phase 1 study and our current ongoing phase 2 study, which is enrolling. What is this novel class of drugs? What is a virus-like drug conjugate? Our first VDC is AU-011 or bel-sar. First of all, it's a virus-like particle that's conjugated to a photosensitizer. This is a highly targeted molecule. First of all, it's targeted because the virus-like particle is only binding melanoma cells or cancer cells.
In this case, you can envision a cancer with a tumor in the back of the eye. The virus-like particle is only going to bind the tumor in the back of the eye and not any of the other eye structures. You only activate the cytotoxic payload once you activate it with infrared light. It's extremely selective. The second advantage is that it's very potent with a mechanism that's ablative, meaning that it's independent of any genetic mutation. As long as the virus-like particle binds the cancer cell, you consistently will kill the cancer in a way that's highly reproducible, and it's very difficult to generate resistance. You have a virus-like particle in the tumor microenvironment, and you have a robust way of generating immune cell death. These generate CD4 and CD8 T cells, and it's a unique differentiation to provide durability of response.
You'll see that we have already generated data in ocular oncology showing great durability of response. We're very close to being able to show that in bladder cancer as well. What we believe we have is the opportunity to really develop this as a platform. As you can see, ocular oncology falls within rare oncology, with rare oncology, ultra-rare pricing opportunity across over three, if not four indications. The first one in phase 3, and the possibility not only to be in bladder cancer, but with a separate formulation that we're currently developing to have an ocular franchise and a non-ocular franchise that can expand well beyond bladder cancer into other solid tumors. Let's focus in ocular oncology. 66,000 patients. This is not just ocular cancer or one ocular cancer. There are over four different types of cancers, and we can treat them all with the exact same technology.
Remember, as long as our virus-like particle binds the cancer, we're going to ablate the tumor the same way. Either it's a metastasis in the choroid, it's a primary melanoma, or it's a cancer of the ocular surface. None of these treatments, they basically treat it with surgical intervention or radiotherapy, we have high comorbidities. The opportunity is really to own this therapeutic area in a way no one else can. Now, if we specifically go to ocular melanoma or choroidal melanoma, what is our opportunity? What you can see here are the different stages of the disease, and it's important because you've probably heard of metastatic uveal melanoma, which is the orange part of this graph. When patients are, unfortunately, the tumor has gone outside of the eye and metastasized to the body, there are drugs that are approved there, like KIMTRAC, sold by Immunocore.
What we're trying to do is at the area opposite the spectrum. Most of these patients, 90% of the patients, when they go to an ophthalmologist, they are diagnosed with an early disease, meaning that it's an early choroidal melanoma. It's not yet metastatic. It's a tiny lesion in the back of the eye. The only option they currently have is a surgical intervention, what you see here. What happens is that the majority of those patients that you were seeing in the blue box are in this very long watchful wait. Why? Because they don't want to receive this plaque radiotherapy. They don't want to be blinded, and the disease is very early. Unfortunately for these patients, this is not the right choice. Watchful wait with cancer is never the right choice, and they have a very high risk of the disease either progressing or becoming metastatic.
What we are offering is something that's going to be transformational because rather than having radiotherapy as their only choice or waiting for a year to get radiotherapy, we're going to be offering a frontline therapy that works just as well as radiotherapy, but without blindness, without radioactive intervention, without any of the comorbidities that radiotherapy has. Obviously, a patient in that early stage of the disease would always choose a therapy that has that type of safety profile. That's why we believe that we can really be first to market and first in class to really capture all of this patient population and avoid the use of radiotherapy. We are not a therapy that will still need a little bit of radiotherapy, 10% or 20% less radiotherapy. We're here to disrupt this blue box and really avoid the need for radiotherapy for the majority of patients.
That is the value proposition. With an in-office procedure, you have an early-stage cancer in the eye. You're going to go to the ocular oncologist, and basically, instead of having to go through that surgery and radioactive intervention, you're going to have a superchoroidal injection. It's simply done. It takes a couple of minutes. It doesn't require anesthesia. Then a light activation three to four hours later. This treatment, repeated in three cycles, is all it will need, and that's what we've shown. I'll show you the data, that it has the 80% tumor control with durability to 12 months. This durability is key because we're only treating the first three months, and we've shown great data up to 12 months. We're currently running this phase 3 study. The study is supported and endorsed with a SPA agreement that actually is the highest endorsement from the agency to support registration.
It's only 100 patients where we're going to randomize 2 to 1 to 2, meaning that we have most patients in the treatment arm or a sham arm. Why sham? These patients, again, there are no drugs approved. The FDA has allowed us to compare to what's the standard of care for this early-stage disease, which is observation. The primary endpoint is time to tumor progression, and the key secondary endpoint will be time to tumor progression or visual acuity failure. I'll show you the data in phase 2 that endorses why this study has high probability of success, a very high probability of success with FastTrack, an orphan drug designation, and a SPA. This is the phase 2 data that supports that high probability of success. This is the phase 2 study.
What you see here are events of tumor progression in the first graph, and what you see in blue is patients treated with the high dose of bel-sar, exactly what we're doing in phase 3. What you see in red is patients treated with a very low dose. If the high dose is three cycles here, we're treating with just one injection, less than a cycle. This red arm represents really what we're going to likely see in the sham in phase 3. This is just 10 patients per arm, and you can see how efficacious our therapy is. We basically have only two events of tumor progression. Most patients had the tumor arrested, and in the low dose, very few patients had their tumor arrested. You can see the p-value with just 10 patients per arm is 0.005. Imagine what we're going to get when we dose 100 patients.
The time to composite endpoint is very similar. It's just that there's one additional event of visual acuity loss that happened in a different patient in the blue arm. That's 90% visual acuity preservation. Only one patient lost and only lost 18 letters, meaning that despite having the very, very good growth arrest, the visual acuity preservation was preserved in the majority of patients. Actually, this patient only had a little bit of fluid that went into the macula. It really was not related to the treatment. As you can see, a profile not just in terms of efficacy, but visual acuity preservation, not a single enucleation. Everyone preserved their eye, 90% preserved their vision, and 80% were treated effectively. What does this mean from a commercial launch? We are enrolling the phase 3. We're going to have the phase 3 in 2027.
This is the most efficient commercial launch that you could probably have. There are only 50 ocular oncologists in the U.S. and 50 in Europe. In our phase 3, we have 70 of these ocular oncologists, and we, as I said, will be the first to market in early choroidal melanoma with the possibility to have supplemental VLA and an extension of the indications into the other two indications, meaning that this is probably the most value generation that the company can have in the near term. Is this the only? No, ocular oncology is our main focus, but we're very excited about the opportunity in bladder cancer. In the last few minutes of the presentation, let me walk you through why this is exciting for us. First of all, because the unmet medical need is very high. We're not talking about a rare disease.
This is the ninth most common cancer worldwide. We're looking at 600 patients that have this disease globally. It's a very difficult patient to treat. Patients recur constantly, and BCG is known to be very poorly tolerated. If you look at the three stages of the disease in non-muscle invasive bladder cancer, there is intermediate risk, there is high-risk papillary, and they're very well known to all of us with the recent approvals of high-risk CIS. Most of the approvals, don't get confused, are in these very late stages before the patient goes to cystectomy. 4,000 patients. The majority of the patients are diagnosed much earlier in intermediate risk.
What we've developed is the same composition of matter, the same drug that we know works so well with highly de-risked, as I said, in phase 3 for the eye, but with a different formulation that allows us to have the absolute best product for urologists. Why? Because we've been able to do it at a stable, meaning that we don't need the minus 70 refrigeration that we need for the ocular. We have developed a formulation that it's stable at 2 to 8 degrees. That's ideal. In this case, also, we differentiate highly to any other drug in development that is in the IO space because we don't have a replicating virus. We have something that is as good as a virus to get the immune system activated, but you do not need any biosafety. You have no risk. This is not replicating. You don't need general anesthesia.
This is simple. Simplicity is very important for the urologist's office because the volume is very high. What is our mechanism of action? You'll also see that the current treatment paradigm for bladder cancer is adjuvant treatment. The majority of drugs in development start with, let's remove the tumor and then give maybe eight times or eight times plus six other times, more than 15 times of any type of adjuvant treatment, be an oncolytic virus or be a chemotherapy with a slow release. That's the standard. Our therapy is different. Why? Because all our immune response is driven towards the tumor, so you need the tumor. It makes no sense to give an IO agent if you remove the tumor because what you want is the immune system to respond towards tumor antigens.
We are the only one who's going to be, by default, by mechanism of action, be given first. That's a huge advantage for this disease. If you have something that, by default, you have to give first, that gives you a very, with very good safety, an immune response with durability, why wouldn't you do that for every patient first? The alternative, you can always do later. You can always do surgery and adjuvant treatment later. You're going to have 80,000 patients coming through the urologist's office that they don't want to have general anesthesia right away, where you can offer this first. If we can show that by doing this first, you have the durability that we've already demonstrated in bladder, in the eye, this is the most disruptive therapy for a $6 billion market that you currently will have in the next 12 months. That's why we're excited.
We're excited also because we already have data to show it. With a single low dose, just single dose of the volume that we were giving in the eye, we did a small proof of concept, and we had an intermediate risk. Remember that big bucket with 80,000 patients where most of the patients are, not CIS with 4,000, but the big 80,000 bucket, four out of five treated tumors had a complete response with just a single low volume of our drug. Imagine now that we're dose escalating and we're going to have 10 times this dose. We had three of five patients that not only responded to the treated tumor, but untreated tumors, meaning that with our immune system, just with a little tiny bit of drug, we have a urothelial field effect.
That's what you want because we can all treat cancer if you give a chemotherapeutic agent for years. Do you want chemotherapy in your bladder for more than 12 months or maybe 24 months? No. What you want is something that you can give first that will have that memory in your urothelia. If the tumor comes back, it's the immune system that's going to treat it and not a chemotherapeutic agent being loaded into your bladder. That's the opportunity. In high risk, we also had good response. With this very low dose, we were able to shrink tumors, have complete responses, and all of the lesions were highly, highly immune infiltrated. That is the promise of this potential in bladder cancer. With a completely different formulation, we have really an opportunity. Now, let me show you what it looks like.
What is it to have a complete response with a single dose? It means that you go and with just literally a cystoscope, you can inject this without general anesthesia, a tiny injection into the base of this tumor, and seven days later, the tumor is gone. Not only the tumor is gone, but you have this response. These are T cells. These are B cells. These are dendritic cells that are a clear, clear sign of an adaptive immune response happening. These are the lymphoid cells moving into the base of the tumor. There's no tumor here, but these are your surveillance cells that are going to be scavengers waiting for the tumor if it ever comes back to kill it. That is the power of this technology. We are where we are. This is where we are. All I've shown you is from a tiny low dose.
Imagine if you now treat multiple tumors multiple times and you dose escalate with an impeccable safety profile. You give it first. You can always give anything else later. We don't compete with any adjuvant treatment, but they will want to give us first. The urologists are really excited. We are enrolling, and we're enrolling really, really fast. There's only one reason to enroll fast. When a drug works, physicians want to be behind the drug, especially if it's an early mechanism of action that's so disruptive. That's where we are. We have a very exciting program in phase 3, a very exciting program that can disrupt the market in bladder cancer with the opportunity to partner and a very strong cash to take us to the readouts. Thank you very much.