Here at Evercore ISI and very pleased to be here with Ellie from Aura. She's actually the founder and CEO, which is so nice to talk to a founder. Tell us about the history of bel-sar.
Yes, so the technology was discovered at NIH, and yes, in those early days, and with a very young Ellie, we partnered with the scientific founder, so I would spin out the technology. And I hired the first employee out of NIH as the postdoc that had developed the early proof of concept. So yes, exciting days at that time. We had only an N of two employees, and we've had a great agreement with NIH for now over 10 years. So we've seen it all from the bench to really, hopefully, to approval very soon.
That's so exciting. Tell us about how bel-sar works, as I think it's a very interesting technology platform.
Yes, so bel-sar is a virus-like drug conjugate, so basically, it's a virus-like particle that is uniquely targeted to cancer cells, and it's targeting a receptor that is expressed on the cancer cell surface early on in malignant transformation from epithelial to mesenchymal transition, and it's something that uniquely differentiates this type of drugs because the biologic part of the drug, you don't have to change it. You can target a rare ocular oncology indication or a much more prevalent urologic oncology indication without changing the virus-like particle. The second thing is that you can conjugate to this structure, which is a protein structure, 200 molecules or more than 200 molecules of cytotoxic payload. If you compare it to an ADC, these are VDCs, so the closest would be an ADC that is five to seven molecules, so the potency per virus-like particle is really, really high.
And then it has what we call a dual mechanism. It's not only a targeted cytotoxicity because you're delivering a cytotoxic payload, but you have an immune activation because you have a virus in the tumor microenvironment. So very unique, very novel, and first in class, hopefully, for many indications.
So why did you decide to focus on these kind of smaller ocular cancers? Start there.
Yes, ocular oncology, so the key driver for me personally was to make a difference, not just to make a business, so the key was to find a group of a medical field that had no innovation where we could show the value of our technology and get it approved very quickly, where we would not necessarily need to invest in marketing dollars, but really there would be no real competition. We did a whole evaluation of a map of diseases where our technology could work and be highly differentiated. Neuro-oncology and ocular oncology were the two that we thought were perfect, and we decided for ocular just because usually, as you know, viruses, gene therapy has a lot of precedent in the eye for being areas that the eye can tolerate the viral administration, so that's why we started, but as you've seen this year, that's just the beginning.
We can transform the medical field of ocular oncology and then go beyond.
OK, great. So tell us, let's walk through what you've shown so far in uveal melanoma or choroidal melanoma. I think you've called it uveal melanoma now. Actually, what's the distinction here?
It's nearly semantics. So the uvea is the layer that goes from the ciliary body all the way to the back of the eye. It's a constant layer, but the part that's just on the back is the choroid. What happens is that these tumors, 90% are in the choroid. So you can call them uveal melanomas or choroidal melanomas. It's semantics nearly because 90% of the times they overlap. You can also have a ciliary body melanoma or an iris melanoma. Those are really rare, less than 10%. So that's the word, that's the semantics. So primary uveal melanoma is appropriate, so is primary choroidal melanoma. So what data do we have? The key for us is to go early stage because our technology can actually prevent metastatic disease. So pre-metastatic is the right way to go as early as possible.
What happens is that in ocular cancers, in ocular melanoma, we diagnose these patients really early before they are metastatic and not even known micrometastases. They cannot be detected with a CT scan. So the opportunity is, can we treat in a way that we can preserve vision and cure the disease? It's a local cure, and if we can, it could be transformational because right now, 5% of Caucasians have a nevi. Those are hundreds of thousands. That's not a rare disease, but nevus transform into early melanomas, and right now, there is no tool for treating these patients because there's no treatment except for radiotherapy or enucleation, blinding you or removing the eye. No one wants to do that.
But what we believe is that when there's a drug approved for particularly these early stage patients, a lot of these there will be hopefully underdiagnosed because a lot of these nevus will no longer be in observation and will be actually early melanomas that should be treated. So in this patient population of early stage disease where we can make a difference, the data shows that we can basically locally cure 80% of the patients with a three-cycle treatment. So it's an acute treatment for three months, and then we follow them for 12 months, but they remain basically cured. We don't have longer durability of response, but we think it's a curative treatment. And in this particular phase two, we also showed that with suprachoroidal administration, 90% of the patients preserve vision. So it is one of those things.
You don't have to give up vision to be treated. Hopefully, that allows many more patients to choose to be treated versus just observe or not be treated at all.
How many patients are in this pool of indeterminate to small melanoma?
Yes, so 80% of the patients. If you calculate the market, including these early stage diseases, is 11,000 patients. Well, 8,000 between the U.S. and Europe, 5,000 are in this bucket where we could treat them without needing a systemic treatment, without having side effects, without having to go through surgery, sorry, radiotherapy. Basically, as a frontline approach, it is a great market opportunity, right? For patients, it's transformational. For us, we could have a first in class without competition. We're competing with literally observing a disease that's life-threatening or blinding with radiotherapy. That's not real gold.
You've gone up to size of, is it three millimeters?
Yeah, up to 2.5 millimeters. That's the study design because that's a red carpet from the FDA to be able to compare in a randomized manner to sham or observation. You have to have a very small tumor, right? Because if you have a tumor that's bigger than 2.5 millimeters, you would treat it with radiotherapy.
But in real world, do you think some of those people with three or it's still small, a single tumor?
They will all choose bel-sar. What we want, though, is to go earlier and earlier, right? We're not so concerned about the bigger than three. While we could treat them, obviously, we just want to treat all the early stage ones. We want more referrals. Now for the phase three, we're going to these nevus clinics. Well, there are patients with nevus that are actually already identified, and retina specialists see them every three to six months. We have hundreds of thousands of patients in nevus clinics, and so to be able to have a drug that first is going to be ocular oncologists as a very efficient market launch, then we can go to retina specialists and start getting these referral patterns from the nevus clinics. That's where we win. That's where patients win.
Why did you start with uveal melanoma? There are other forms of eye cancer as well, right?
Yes, so there are up to four indications we can have in ocular oncology. Remember, our drug is not particularly targeted to melanoma. It's targeted to solid tumors. We have identified that this receptor that we bind to, it's across the board. So we can treat. We've identified up to four different types of tumors in the eye, and each indication is bigger than the one before. So we're really excited we can treat metastasis to the choroid, meaning breast cancer patients. That I'm really passionate about because imagine surviving breast cancer and then being blinded both eyes because of radiotherapy. We should do better than that. So mets to the choroid is a phase two that we're about to start, but then cancers of the ocular surface, like conjunctival melanomas, squamous cell carcinomas, like people who do drops of 5-FU or mitomycin in the eye.
Forget mitomycin in the bladder. Imagine mitomycin in the eye. That's how we treat these patients. We even have perforations of the eye because of the chemo. So that's the kind of like you feel like you elevate even to have the possibility of offering something different that doesn't have those kind of toxicities. So lots of opportunity. Collectively, ocular oncology is 66,000 patients. We think we can treat them all.
So we've talked about. You've talked a little bit about some of the phase two data you've seen. You've talked about preserving 90% of patients, preserving vision, and some of your kind of responses out through beyond actually even 12 months. Tell us about the phase three study, kind of the design of that, what you're hoping to see.
Yes, so it was a big win because innovation is always a beautiful word, but it means that no one's done it before. So for us, to have the first time ever a drug being in a phase 3 to start in ocular oncology was an important milestone. And we wanted to make sure that the design of the study and the endpoints were going to be supportive for approval. And so we did a SPA agreement for that reason. The study is designed as a three-arm, the high dose, low dose sham. Again, red carpet because of the sham because obviously with something that cures 80% of the patients to compare to sham, I think we're going to do all right.
But nonetheless, we have to randomize the patients and make sure that we have. It's a time to event analysis, meaning that every event is an event of tumor progression. We're hoping to see very few events of tumor progression in our high dose and a lot of events of tumor progression in the sham. That means that patients will be treated with radiotherapy very quickly in the sham. Any patient that fails is managed, so they'll be treated with radiotherapy. And the number of events in a log-rank test is going to drive the p-value for approval at 15 months. So that's the design of the phase three. It also has a time to composite where it's not just tumor progression, but visual acuity failure too, which is also something that the FDA offered to us because they said, like, it's great that you have this vision preservation.
If you don't have a visual acuity endpoint in the study, you're not going to be able to put in a claim. So how do we have versus sham? We couldn't have superiority on vision, but including it in the composite was an excellent way, and so again, a big win from the FDA, and only 100 patients. We're not talking about 400 or 500. It's 100 patients sufficient to support the registrational purpose for the study, so we're excited. It's a global study. We just had earnings. We said, like, we finally got EUCTR. I'm from Europe, and I'm like, oh, it's tough to go through EUCTR, but it's really nice when you have it, right? Because then you have all the countries, and it's the first time that we're running this study in Europe. It's always been US-based.
So, to now have all the countries have sites active, it makes me really happy. I always thought that our drug should be approved globally. And unmet need is global. And the way physicians treat these diseases is the same everywhere. So it should be approved globally. So it's one step closer.
How is enrollment going?
So we're not guiding on enrollment, but we have guided that enrollment should be finished in 2025. The BLA submission should be in 2026 and approval hopefully 2027. So that's what we're guiding towards. And we're very diligent, hoping to get that flag of approval soon.
And your vision is to commercialize this yourself in Europe as well. Maybe you can speak to that.
Yes, so look, right now, diluting the asset is the last thing that we want to do. It's such a valuable asset, especially now that we have bladder cancer data. But ocular oncology, we can launch ourselves. There's 50 ocular oncologists in the U.S., 50 in Europe. This is an easy indication or even a field, a group of rare oncology indications that are easy for us to commercialize with 20 MSLs, sales force, we could do it. Bladder cancer is a different story, right? It's a much larger indication. But right now, there is no need to partner. Right now, there's only a need to generate value.
Great. Let's talk through bladder cancer a little bit. I think you had some fairly provocative data there. Walk us through the bladder cancer data.
Yes, bladder cancer, we're excited. It was always my dream to say, like, these should work for many other cancers, right, and we've seen it in over 15 different animal models, so we discussed with the FDA the opportunity to do a window of opportunity. What does it mean? It means that patients that are scheduled to go to surgery in the eye, it's radiotherapy. In bladder is surgery. You have these bladder lesions. You go through TURBT. They remove the lesions, and the big problem is recurrence, well, the FDA said, OK, yes, you have fast track, you don't have a lot of tox data in bladder, but you can do this window of opportunity, just treat and then do the surgery anyways, standard of care, so the patient is not at risk, so we did that, and really, the idea was single dose.
Single dose, not just single dose, single low volume, low dose from the eye study. Going to the bladder, these tumors were 10 times bigger, and I was like, look, it's great because we'll have clinical data early on rather than spending time and effort in tox studies, but we treated, and [in] the patients four out of five in the intermediate risk, which is a large, like it's probably the largest in non-muscle invasive bladder cancer with more than 80,000 patients. And we treated once, and when the physician went to do the surgery, there was nothing. And they still went to do the surgery, and there was a little bit of edema where the tumor used to be. They removed tissue, so we now have the highest level of complete response, which is histopathological evaluation that there was nothing left.
There was a clear 2-centimeter urothelial carcinoma before. Such a great thing. Then, because we have tissue, because of this design of the study, we have this whole panel of immune profiling where we can see that, look, the tumor is gone because we necrotized the base of the tumor. There was an immune response that was actually not just on the tumor itself, but a field effect because we were actually having complete responses in other distant lesions that had not been treated. It's driven by a CD4, CD8 because look at the staining before and after. It's just picture perfect. Sometimes you have to.
Analogy in real time.
Yes, so clearly a drug for bladder cancer that can be treated without the need for surgery, can be treated in the office. We can really show durability of response, which we should with this kind of T cell response. And again, this is just single dose. Now we're expanding the study to treat multiple doses, higher volume. So we should have something remarkable for these patients.
So when will be the next kind of point of visibility on bladder cancer?
It will be next year. So not only we'll have the full we provided the first eight patients because it was so remarkable that we had.
So walk us through those eight patients.
Yes, so we had, of the eight patients, we had the four that had. Okay, so 13 patients. The first five were just for safety. They were just patients that were with drug without light activation. It was a request from the FDA, right? And those had basically nothing, no response, but no safety issues. Those were the first five. And then we presented data of eight, of which we classified into two groups, the low-grade intermediate risk and the high grade. Although they were in different cohorts, it's easier to report the data that way. So for the intermediate risk, that's the low-grade intermediate risk, the large bucket of patients, of those, four out of the five patients had a complete clinical response. And there were, sorry, five patients with intermediate risk, four out of five clinical complete response. And then we had three patients with high risk.
Two out of these three had the tumors smaller after a single dose, and you know the high-risk patients are patients that are a little bit bulkier. It doesn't mean that the drug doesn't work in those. It just means that we treat with a low-volume single dose, but still, we had response across the board, but the intriguing thing is this kind of like intermediate risk patient population because seeing those kind of responses in that patient population, we think that's, again, if we focus there, we could have a very expedited path to it.
When will we see longer-term follow-up from these eight patients? Because that's the key with bladder cancer, right? Like the treatments work today. They just have recurrences. So I think the key is you don't want to see recurrences, right?
Correct, yes. And you know those eight patients, because the window of opportunity study, even though they had complete responses, they all went through surgery. And in the study, they are only followed for 50 days. So even if I told you 100% were clean, it's like, OK, surgery or your treatment, you don't know. So that's unfortunate. But it was a win to have the window of opportunity study anyway. So now we're amending, and now we're going to be enrolling new patients. And hopefully, we'll come at the beginning of the year at J.P. Morgan with the design of the study. But we're hoping for an endorsement to be able to show the durability of response. And that will be important because with those two data sets, then we can really think about, OK, the next step is really a phase two or three for approval, right?
And that could be a very, very big milestone. But next year, durability of response for sure from this amended study.
OK, so we talked about 2025, which is mainly going to be this kind of new bladder cancer.
Additional bladder cancer data.
And then just tell us about your cash runway?
Yes, so we have a really good cash runway. We've covered for the studies that we're running, including this expansion of the bladder study. And we have capital until the second half of 2026. So a very good cash position that allows us to really generate the data that we want to decide whether we expedite them for approval studies.
Great. Well, I think we're out of time, but thank you so much.
Yes, thank you, Liisa. Thank you, everyone.
Here at Evercore ISI.
Technology was discovered at NIH, and yes, in those early days, and with a very young Ellie, we partnered with the scientific founders, so I would spin out the technology. And I hired the first employee out of NIH as the postdoc that had developed the early proof of concept, so yes, exciting days at that time. We had only an N of two employees, and we've had a great agreement with NIH for now over 10 years, so we've seen it all from the bench to really, hopefully, to approval very soon.
That's so exciting. Tell us about how bel-sar works. I think it's a very interesting technology platform.
Yes, so bel-sar is a virus-like drug conjugate. So basically, it's a virus-like particle that is uniquely targeted to cancer cells. And it's targeting a receptor that is expressed on the cancer cell surface early on in malignant transformation from epithelial to mesenchymal transition. And you know it's something that uniquely differentiates this type of drugs because the biologic part of the drug, you don't have to change it. You can target a rare ocular oncology indication or a much more prevalent urologic oncology indication without changing the virus-like particle. The second thing is that you can conjugate to this structure, which is a protein structure, 200 molecules or more than 200 molecules of cytotoxic payload. If you compare it to an ADC, these are VDCs. So the closest would be an ADC that is five to seven molecules. So the potency for virus-like particle is really, really high.
And then it has what we call a dual mechanism. It's not only a targeted cytotoxicity because you're delivering a cytotoxic payload, but you have an immune activation because you have a virus in the tumor microenvironment. So very unique, very novel, and first in class, hopefully, for many indications.
So why did you decide to focus on these kind of smaller ocular cancers? Start there.
Yes, ocular oncology. So the key driver for me personally was to make a difference, not just to make a business. So the key was to find a group of a medical field that had no innovation where we could show the value of our technology and get it approved very quickly, where we would not necessarily need to invest in marketing dollars, but really, there would be no real competition. We did a whole evaluation of a map of diseases where our technology could work and be highly differentiated. Neuro-oncology and ocular oncology were the two that we thought it's perfect. And we decided for ocular just because usually, as you know, viruses, gene therapy has a lot of precedent in the eye for being areas that the eye can tolerate the viral administration. So that's why we started. But as you've seen this year, that's just the beginning.
We can transform the medical field of ocular oncology and then go beyond.
OK, great. So tell us, let's walk through what you've shown so far in uveal melanoma or choroidal melanoma. I think you've called it uveal melanoma now. Actually, what's the distinction here?
It's nearly semantics. So the uvea is the layer that goes from the ciliary body all the way to the back of the eye. It's a constant layer. But the part that's just on the back is the choroid. Well, it happens that these tumors, 90% are in the choroid. So you can call them uveal melanomas or choroidal melanomas. It's semantics nearly because 90% of the times they overlap. You can also have a ciliary body melanoma or an iris melanoma. Those are really rare, less than 10%. So that's the word, that's the semantics. So primary uveal melanoma is appropriate. So is primary choroidal melanoma. So what data do we have? The key for us is to go early stage because our technology can actually prevent metastatic disease. So pre-metastatic is the right way to go as early as possible.
What happens that in ocular cancers, in ocular melanoma, we diagnose these patients really early before they are metastatic and not even known micrometastases. They cannot be detected with a CT scan, so the opportunity is, can we treat in a way that we can preserve vision and cure the disease? It's a local cure, and if we can, it could be transformational because right now, 5% of Caucasians have a nevus. Those are hundreds of thousands. That's not a rare disease, but nevus transform into early melanomas, and right now, there is no tool for treating these patients because there's no treatment except for radiotherapy or enucleation, blinding you or removing the eye. No one wants to do that.
But what we believe is that when there's a drug approved for particularly these early-stage patients, a lot of these there will be hopefully underdiagnosed because a lot of these nevi will no longer be in observation and will be actually early melanomas that should be treated. So in this patient population of early-stage disease where we can make a difference, the data shows that we can basically locally cure 80% of the patients with a three-cycle treatment. So it's an acute treatment for three months. And then we follow them for 12 months, but they remain basically cured. We don't have longer durability of response, but we think it's a curative treatment. And in this particular phase two, we also showed that with suprachoroidal administration, 90% of the patients preserve vision. So it is one of those things. You don't have to give up vision to be treated.
Hopefully, that allows many more patients to choose to be treated versus just observe or not be treated at all.
So how many patients are in this pool of indeterminate to small melanoma?
Yes, so 80% of the patients, so if you calculate the market, including these early-stage diseases, is 11,000 patients, well, 8,000 between the U.S. and Europe, 5,000 are in this bucket where we could treat them without needing a systemic treatment, without having side effects, without having to go through surgery, sorry, radiotherapy. Basically, as a frontline approach, it is a great market opportunity, right? For patients, it's transformational. For us, we could have a first-in-class without competition. We're competing with literally observing a disease that's life-threatening or blinding with radiotherapy. That's not real go.
You've gone up to size of, is it three millimeters?
Yeah, up to 2.5 millimeters. That's the study design because that's a red carpet from the FDA to be able to compare in a randomized manner to sham or observation. You have to have a very small tumor, right? Because if you have more than it's a tumor that's bigger than 2.5 millimeters, you would treat it with radiotherapy.
But in real world, do you think some of those people with three or it's still small, a single tumor?
They will all choose bel-sar. What we want, though, is to go earlier and earlier, right? We're not so concerned about the bigger than three. While we could treat them, obviously, we just want to treat all the early-stage ones. We want more referrals. Now for the phase three, we're going to these nevus clinics. Well, there are like patients with nevus that are actually already identified, and retina specialists see them every three to six months. We have like hundreds of thousands of patients in nevus clinics. And so to be able to have a drug that first is going to be ocular oncologists as a very efficient market launch, then we can go to retina specialists and start getting these referral patterns from the nevus clinics. That's where we win. That's where patients win.
Why did you start with uveal melanoma? There are other forms of eye cancer as well, right?
Yeah, so there are up to four indications we can have in ocular oncology. Remember, our drug is not particularly targeted to melanoma. It's targeted to solid tumors. We have identified that this receptor that we bind to. It's across the board. So we can treat. We've identified up to four different types of tumors in the eye. And each indication is bigger than the one before. So we're really excited. We can treat metastasis to the choroid, meaning breast cancer patients. That I'm really passionate about because imagine surviving breast cancer and then being blinded both eyes because of radiotherapy. We should do better than that. So mets to the choroid is a phase two that we're about to start. But then cancers of the ocular surface, like conjunctival melanomas, squamous cell carcinomas, like people who do drops of 5-FU or mitomycin in the eye.
Forget mitomycin in the bladder. Imagine mitomycin in the eye. That's how we treat these patients. We even have perforations of the eye because of the chemo. So that's the kind of like you feel like you elevate even to have the possibility of offering something different that doesn't have those kind of toxicities. So lots of opportunity. Collectively, ocular oncology is 66,000 patients. We think we can treat them all.
So we've talked a little bit about some of the phase 2 data you've seen. You've talked about preserving 90% of patients, preserving vision, and some of your kind of responses out through beyond actually even 12 months. Tell us about the phase 3 study, kind of the design of that, what you're hoping to see.
Yeah, so it was a big win because innovation is always a beautiful word, but it means that no one's done it before. So for us, to have the first time ever a drug being in a phase three to start in ocular oncology was an important milestone. And we wanted to make sure that the design of the study and the endpoints were going to be supported for approval. And so we did a SPA agreement for that reason. The study is designed as a three-arm, the high-dose, low-dose sham. Again, red carpet because of the sham because obviously with something that cures 80% of the patients to compare to sham, I think we're going to do all right. But nonetheless, we have to randomize the patients and make sure that we have. It's a time-to-event analysis, meaning that every event is an event of tumor progression.
We're hoping to see very few events of tumor progression in our high dose and a lot of events of tumor progression in the sham. That means that patients will be treated with radiotherapy very quickly in the sham. Any patient that fails the sham, so will be treated with radiotherapy. And those number of events in a log-rank test is going to drive the p-value for approval at 15 months. So that's the design of the phase 3. It also has a time-to-composite where it's not just tumor progression, but visual acuity failure too, which is also something that the FDA offered to us because they said, like, it's great that you have this vision preservation. If you don't have a visual acuity endpoint in the study, you're not going to be able to put it in a claim. So how do we have versus sham?
We couldn't have superiority on vision, but including it in the composite was an excellent way, and so again, a big win from the FDA, and only 100 patients. We're not talking about 400 or 500. It's 100 patients sufficient to support the registrational purpose for the study, so we're excited. It's a global study. We just had earnings. We said, like, we finally got EUCTR. I'm from Europe, and I'm like, oh, it's tough to go through EUCTR, but it's really nice when you have it, right? Because then you have all the countries, and it's the first time that we're running this study in Europe. It's always been US-based, so to have now all the countries have sites activated, it makes me really happy. I always thought that our drug should be approved globally, and unmet need is global.
The way physicians treat these diseases is the same everywhere. It should be approved globally. It's one step closer, we'll say.
How is enrollment going?
So we're not guiding on enrollment, but we have guided that enrollment should be finished in 2025. The BLA submission should be in 2026 and approval hopefully 2027. So that's what we're guiding towards. And we're very diligent hoping to get that flag of approval soon.
Your vision is to commercialize this yourself in Europe as well. Maybe you can speak to that.
Yeah, so look, right now, diluting the asset is the last thing that we want to do with such a valuable asset, especially now that we have bladder cancer data. But ocular oncology, we can launch ourselves. There's 50 ocular oncologists in the U.S., 50 in Europe. This is an easy indication or even a field, a group of rare oncology indications that are easy for us to commercialize with 20 MSLs, sales force, we could do it. Bladder cancer is a different story, right? It's a much larger indication. But right now, there is no need to partner. Right now, there's only a need to generate value.
Great. Well, let's talk through bladder cancer a little bit. I think you had some fairly provocative data there. So walk us through the bladder cancer data.
Yes, bladder cancer, we're excited. It was always my dream to say, like, these should work for many other cancers, right? And we've seen it in like over 15 different animal models. So we discussed with the FDA the opportunity to do a window of opportunity. What does it mean? It means that patients that are scheduled to go to surgery in the eye, it's radiotherapy. In bladder is surgery. You have these bladder lesions. You go through TURBT. They remove the lesions, and the big problem is recurrence. Well, the FDA said, OK, yes, you have fast track. You don't have a lot of tox data in bladder, but you can do this window of opportunity, just treat and then do the surgery anyways, standard of care. So the patient is not at risk. So we did that. And really, the idea was single dose.
Single dose, not just single dose, single low volume, low dose from the eye study going to the bladder. These tumors were 10 times bigger. And I was like, look, it's great because we'll have clinical data early on rather than spending time and effort in tox studies. But we treated, and when the patients 4 out of 5 in the intermediate risk, which is a large, like it's one of the probably the largest in non-muscle-invasive bladder cancer with more than 80,000 patients. And we treated once, and when the physician went to do the surgery, there was nothing. And they still went to do the surgery, and there was a little bit of edema where the tumor used to be. They removed tissue. So we now have the highest level of complete response, which is histopathological evaluation that there was nothing left.
There was a clear 2-centimeter urothelial carcinoma before. Such a great thing. Then because we have tissue, because of this design of the study, we have this whole panel of immune profiling where we can see that, look, the tumor is gone because we necrotized the base of the tumor. There was an immune response that was actually not just on the tumor itself, but a field effect because we were actually having complete responses in other distant lesions that had not been treated. It's driven by a CD4, CD8 because look at the staining before and after. It's just picture perfect. Sometimes you have lot.
Analogy in real time.
Yeah, so clearly a drug for bladder cancer that can be treated without the need for surgery, can be treated in the office. We can really show durability of response, which we should with this kind of T cell response, and again, this is just single dose. Now we're expanding the study to treat multiple doses, higher volume, so we should have something remarkable for these patients.
When will be the next kind of point of visibility on bladder cancer?
It will be next year, so not only we'll have the full we provided the first eight patients because it was so remarkable that we got.
So walk us through those eight patients.
Yeah, so okay, so 13 patients. The first five were just for safety. They were just patients that were with drug without light activation. It was a request from the FDA, right? And those had basically nothing, no response, but no safety issues. Those were the first five. And then we presented data of eight, of which we classified into two groups, the low-grade intermediate risk and the high-grade. Although they were in different cohorts, it's easier to report the data that way. So for the intermediate risk, that's the low-grade intermediate risk, the large bucket of patients, of those four out of the five patients had a complete clinical response. And there were five patients with intermediate risk, four out of five clinical complete response. And then we had three patients with high risk.
Two out of these three had the tumors smaller after a single dose, and the high-risk patients are patients that are a little bit bulkier. It doesn't mean that the drug doesn't work in those. It just means that we treat with a low-volume single dose, but still, we had response across the board. But the intriguing thing is this kind of like intermediate risk patient population because, seeing those kind of responses in that patient population, we think that's, again, if we focus there, we could have a very expedited path to it.
When will we see longer-term follow-up from these eight patients? Because that's the key with bladder cancer, right? Like the treatments work today. They just have recurrences. So I think the key is you don't want to see recurrences, right?
Correct, yes. And those eight patients, because the window of opportunity study, even though they had complete responses, they all went through surgery. And in the study, they are only followed for 50 days. So even if I told you 100% were clean, it's like, OK, surgery or your treatment, you don't know. So that's unfortunate, but it was a win to have the window of opportunity study anyway. So now we're amending, and now we're going to be enrolling new patients. And hopefully, we'll come at the beginning of the year, J.P. Morgan, with the design of the study. But we're hoping for an endorsement to be able to show the durability of response. And that will be important because with those two data sets, then we can really think about, OK, the next step is really a phase two or three for approval, right?
That could be a very, very big milestone. Next year, durability of response for sure from this amended study.
OK, so we talked about 2025, which is mainly going to be this kind of new bladder cancer.
Additional bladder cancer data.
And then just tell us about your cash runway?
Yeah, so we have a really good cash runway. We've covered for the studies that we're running, including this expansion of the bladder study. And we have capital until the second half of 2026. So a very good cash position that allows us to really generate the data that we want to decide whether we expedite them for approval studies.
Great. Well, I think we're out of time, but thank you so much.
Yes, thank you, Liisa. Thank you, everyone.