Good day and welcome to Q2 Investor Summit Virtual. We appreciate your participation in today's virtual event. Up next, we're pleased to introduce Barinthus Biotherapeutics. If you would like to ask a question during the webcast, you may click the Q&A icon button on the right side of your screen. Please type your question into the box and click Send to submit it. At this time, it is my pleasure to hand over the session to Bill Enright, CEO at Barinthus Biotherapeutics, who will lead the presentation. Sir, the floor is yours.
You can now start, Sir Bill.
Oh, okay. Thank you very much, sir.
Thank you.
Thank you for joining. I will walk through the Barinthus Biotherapeutics, story. First, a little word from our lawyers. We are a publicly listed company, and there may be some forward-looking statements. You can find more information on sec.gov, or our website. Barinthus, is really about the next generation of immunotherapies, and we're focused in the autoimmune and inflammatory disease, space, trying to bring new developments to treat patients. We're developing immunotherapies, for autoimmunity and other inflammatory diseases. We're really focused on a platform that we have called SNAP-TI. This is a self-assembling nanoparticle platform that really allows us to help restore the balance to the immune system. We've got clinical programs also in infectious disease and oncology, with a viral-based platform that we're winding down right now and looking to find partners to advance those programs.
The SNAP-TI, platform is really where we're focused. This is a really differentiated platform that we think is a potentially more effective and more patient-friendly approach to antigen-specific immune tolerance. The lead candidate is in celiac disease. We call that VTP-1000. We're in phase I clinical trials right now with an expected data readout towards the end of Q3, early Q4. We've got additional undisclosed preclinical candidates. We've got about $100 million, on the balance sheet, which gives us a cash runway into 2027, with no debt or outstanding warrants. In the I and I disease, these are diseases, where we're really resulting from an imbalance of the immune system. We get our immune system, wrongly attacking our own tissues. This is a result of T cells, that are inappropriately activated. The T effect, or cells induce a pro-inflammatory response.
These can be prevalent in a number of diseases like autoimmune diseases, allergies, transplant rejections. There are a number of evolving therapies that are pretty effective. Like T cell, and B cell, depletion, which has been pretty effective in MS, there are anti-cytokine antibodies, and other Treg cell therapies, that are being developed for these things. These are very broad, overreaching technologies. We are trying to take a more directed approach using this antigen-specific immune tolerance approach, and as are a number of others. That is really again trying to restore this balance between the T regulatory cells, and the T effector cells. Some of the challenges that people have seen so far are with some of the current technologies that are available to go after this antigen-specific approach, you have very limited antigen coverage because of the way these particles are put together.
It often requires IV administration, which is not particularly patient-friendly, especially in some of the diseases that we'll be talking about. Because the body is already reacting to these antigens, we know there's a pro-inflammatory response. Some of these approaches also have a lot of reactogenicity, when you initially dose these patients. It's unclear whether or not they will get an adequate T cell response, to kind of, again, rebalance that immune system. We've developed a very specific approach to try and address some of these issues. We call it SNAP-TI. Again, this is a self-assembling nanoparticle. We're co-delivering multiple disease-associated antigens, together with an immunomodulator. This is particularly important because this immunomodulator, is important from helping to promote that rebalancing of the Treg and T effectors.
It also helps when you're administering the initial doses in helping to mask that pro-inflammatory response, that you would otherwise see to these antigens. These nanoparticles, are really precise composition, so they're really easy to manufacture. We can control the size of these things. That's really important because we're able to inject these either IM or sub Q. The ongoing phase one study is doing it IM, but we have preclinical data that suggests we could do this sub Q. That allows you to get into different tissues than you could otherwise get into. This small size allows us to get into not only the liver and spleen, where many particles are able to reach, but also into the draining lymph nodes. You get a broader distribution of the drug. It enables very broad antigen coverage.
We can incorporate numerous antigens, again, in a patient-friendly administration using IM or sub Q. And then based on the preclinical data that we have, we do believe there's going to be improved tolerability and a difference in this Treg to T effector ratio. What we're doing here is the injection, you stimulate the APCs, and you're modifying this ratio here because you're inducing transdifferentiation of these T effectors, into Tregs. You're actually stimulating Tregs, and you're depleting T effectors. You're doing this in multiple ways that allows you to kind of help rebalance the immune system. Here's some of the preclinical data that we have. This first graph is looking at, so there's no real good models in celiac disease. We're looking at a mouse EAE model, of multiple sclerosis.
In this model, what many people do is you're looking to show that you can prevent disease onset. You are giving the drug at the same time you are giving the animals the disease. You can show that in those animals that have our particles either with or without the immunomodulator, you are able to protect those animals, where in particles that have irrelevant antigens, or if you are looking at a vehicle control, those animals are not protected. The tougher experiment really is, can you actually treat animals that already have established disease? In this middle graph here, we are looking at animals that have been treated after they have peak disease symptoms. You can see we do get a nice reduction in disease score, in those animals that have our particles with the immunomodulator, versus those that are only treated with the vehicle control.
The last graph here, is really looking at that mechanism of action. It's looking at the Treg to T effector ratio, that I've talked about. You see the best ratio, here is when you have that nanoparticle, with the immunomodulator. Vehicle controls or recombinant IL-2, or irrelevant antigens, really do not change that ratio. Again, the lead candidate is in celiac disease, and I'll dive into celiac disease, a little bit here. I think most people know somebody suffering from celiac disease. It's really a significant issue. About 1%, of the worldwide population is estimated to have celiac disease. Many of those are not able to adhere to a strict gluten-free diet. The only treatment right now is the strict gluten-free diet. Even the patients that are able to get on gluten-free diets, many of those are non-responsive.
Even if you are responsive, you're always worried about, can I go out to eat? Can I eat at my friend's house? Getting on an airplane, other things where there may be some cross-contamination issues, because this is a significant burden to people as far as being ill after eating anything with gluten, in it. We designed a candidate here that allows us to go after this and change the Treg to T effector ratio. In celiac disease, the T effector cells, attack the lining of the small intestine. They overwhelm the regulatory T cells that normally prevent autoimmunity and the unwanted inflammation. The VTP-1000, allows you to reduce that T effector, and stimulate the T regulatory cells, really change that ratio, and rebalance the immune system, kind of restoring homeostasis.
Bill, excuse me. I don't think this screen is moving for your presentation.
Oh, it's not.
Yeah.
It's changing on my screen anyhow. That's not helpful.
We're very sorry to interrupt you.
No, no, that's fine. I don't know.
Yeah, the audience are messaging us on the chat that they only see the first slide. Sorry about that.
No, that's. Yeah, that's not that either. Apologies, folks.
No worries. Take your time.
Did you choose the entire screen or a window to share your screen?
That's what I'm trying. Yep, there we go.
Okay, we can try one more time.
Is that better?
Yep. Could you check that one? That's good.
Yep. Good. All right. Sorry about that.
There we go. Thank you.
Yep. All right. So VTP-1000, as I've mentioned, incorporates these peptides. So each of these peptide strings here is an individual antigen. It's got a hydrophilic side, that says water-friendly and a hydrophobic side. The hydrophobic side, actually, in the presence of water, they self-assemble into these nanoparticles. When you combine with an immunomodulator, the immunomodulator, actually gets incorporated into these particles in a very systematic way. The immunomodulator, that we're using in VTP-1000, is an mTOR inhibitor, called rapamycin. This helps, as I said, it allows us to dampen the pro-inflammatory response, at injection, but also helps promote the change from T effectors, to Treg cells, in that improved skewing, if you will. The phase I, trial is ongoing. This is a two-part study. It's a pretty stringent study for a phase I, design. It's a double-blind placebo-controlled study.
Part A, is 18 patients, and a single ascending dose. We're looking at three different doses, six patients, in each dose cohort, four, that will be treated and two placebos. Even though this is a safety study, the safety is obviously pretty important here given what we're seeing from this disease in that, as I mentioned, some of the competitive technologies, out there, you still get significant reactogenicity, because of this pro-inflammatory response, to the antigen itself that you're using in your drug. We'll be looking at safety from this study as well as taking whole blood from these patients because there are a number of good biomarkers here. We'll look at whole blood before and after dosing to see if we can see any change in some of these biomarkers.
We will be reporting on those data, as I mentioned, towards the end of the third quarter, early fourth quarter. We are also proceeding with the multiple ascending dose portion. We have just gotten FDA approval, to move forward with this part of the study as well. This is in 24 patients. Each of these patients will get three, different doses at each dose cohort, two weeks apart. We will do a gluten challenge. We will be able to discern a hint of efficacy here. It is not powered statistically to determine efficacy, but we should be able to see if they were making the right changes here. The data from the multiple ascending dose portion should be available towards the middle of the year next year.
We looked at a number of in vivo things in this VTP-1000, as well, looking to make sure that we could stimulate the right T cell response. As I mentioned, VTP-1000, traffics to the draining lymph nodes, and it remains detectable for upwards of two weeks, which is why the dosing schedule here. You can see that after two weeks, that falls away. Whereas if you're just giving peptides alone, it falls away very, very quickly. Those get trafficked and cleared pretty quickly. It allows the drug to stay around a while. The middle graph here is showing that we actually do see a nice T cell, response here with the VTP-1000, or a positive control, whereas if you have irrelevant antigens in the nanoparticle, you don't see that T cell, stimulated.
In the last one here, we're looking at a gluten challenge, gluten peptide challenge in whole blood, as I was mentioning that we're going to do for the SAD, portion here, where you stimulate the whole blood of the celiac patients, with gluten, and then you can see whether or not you're having an impact. You can see in some of the things that we did ahead of the clinical studies in the celiac patients, you can see that we're getting a response with VTP-1000, whereas not with the gluten peptides. Key things here, we designed this to address a number of the issues that we've seen in some of the earlier technology approaches, optimal design for getting into draining lymph nodes, as well as the spleen and liver. We can add multiple antigens. The CMC, the manufacturing, is very, very consistent.
You get the same size particles with the same number of agents, and we're co-delivering this with the immunomodulator, which enhances both efficacy as well as safety from a reactogenicity perspective. Because we're getting so much antigen into each particle, we can also administer IM, as well. We have the celiac program, in the clinic. We showed data in multiple sclerosis, and vitiligo and type 1 diabetes, and other indications. We presented some of that data at ACIT, last year. We are looking to continue to develop some of these other indications to move our next candidate into the clinic. We will guide on that probably next year. I will spend a few minutes just talking about our viral-based platform. Again, these are programs that have been in the clinic and are now finished. We are looking for partners for these.
We had a program called VTP-300, potential functional cure for chronic hepatitis B, where we've recently presented data at ESOL, from a couple of phase II, studies, as well as VTP-850, for prostate cancer, where we recently finished up a phase I, study. Those data are being analyzed as we speak. Chronic hepatitis B, is a huge market opportunity. Over 250 million, people chronically infected with hepatitis B, about 1.2 million, new infections every year, about a million, people dying from cirrhosis, and cancer of the liver, as a direct result of hepatitis B infections. There really aren't any very good treatments. The standard of care right now is going on nucleoside, what we call NUCs, and they have very low cure rates and take a long time. That includes pegylated interferon. With typical NUCs, you're getting maybe 1-2%, functional cure rates.
With pegylated interferon, it may get up 8 or 9%, but there's significant side effects with the pegylated interferon, as well. Again, the chronic HBV infection, the exposure to the virus leads to T cell, exhaustion because the virus pumps, out this decoy protein called surface antigen, the HBsAg. Over time, these T cells, lose their function, and you get decreased secretion of cytokines, and the killing molecules that you need to get rid of the virus. In severe stages, essentially, you have no T cells, that can recognize the virus any longer. You have no control over the disease. That's really what VTP-300, was designed to overcome. It's stimulating de novo T cells, and tired T cells, exhausted T cells, so that they can fight off the virus. We know now that the combination approach is needed to get to a functional cure.
A functional cure has got a regulatory definition. It is defined as surface antigen loss when you are off NUCs, for at least six months. You have to get to surface antigen loss, take people off their NUCs, and then show that they can maintain surface antigen loss after being off their NUCs, for six months. As I said, initially, people thought that if we inhibited viral replication with NUCs, the body would be able to take over. That did not pan out. Only 1-2%, of patients get to a functional cure. They thought maybe if we got rid of the decoy protein, if we found ways to directly lower the surface antigen burden, maybe then the body would be able to take over and control the virus. A lot of really nice surface antigen reducers were developed using RNAi, antisense, monoclonals.
They do a good job at getting the surface antigen levels down. However, the body is still not able to take over and get control of the virus. You need a surface antigen stimulator. That is where VTP-300, comes in. We have designed this to induce sustained surface antigen reduction. We have shown that now in multiple clinical trials. HBV003, are some of the data that we presented at ESOL, recently. This was the trial design, essentially looking at two different viral-based platforms, ChAdOx, which is an adenovirus, and MVA, which is a pox virus in combination with low-dose nivolumab, and looking at repeat dosing as well as timing of when we deliver the low-dose nivolumab. You can see here that we get surface antigen declines across all groups soon after that day 29, dose regimen. It was maintained out to day 169.
We get stronger responses in the folks that had the combination of low-dose and EVO, when they were given at the same time, in groups one and two. You can see that 71%, of participants met the criteria to get off their NUCs. In this study, getting off your NUCs, was optional, so not everyone chose to come off their NUCs. Of those that did, we got two of the participants achieve functional cure. We actually have a couple of others that got to functional cure and then rebounded for a time point and came back down to surface antigen loss again. Very encouraging data. The other surface antigen reducers are not able to show that you get this sustained surface antigen decline. When you stop giving those molecules, the surface antigen levels rebound.
The initial study in HBV003, was focused on patients that have low starting levels of surface antigen, so 200, international units or less. In this study that we did with a collaboration with Arbutus, we actually started with patients that had 5,000, international units per mL, or less. We know from previous studies that Arbutus, has done that when they use their siRNA, Imdusiran, if they give patients a six-month course, they're able to get 90-95%, of those patients down below 100, international units per mL. As I said, they don't stay down. The idea here is to pre-treat with the Imdusiran, come back with VTP-300, either alone or in combination with low-dose Nivolumab, to see if we can get that sustained response again, get to that functional cure.
Some of the data that we presented at ESOL, are illustrated here. In group C, which is where we're looking at the imdusiran and then coming back with VTP-300, in combination with the low-dose nivolumab, that's where we saw the best response. You get to about 23%, of patients getting to non-detectable levels of surface antigen, at week 48. All of those participants actually seroconverted. They now show antibody response against the virus. Two of those participants at starting baseline levels less than 1,000, reached functional cure. Very encouraging data in both of these studies showing that we're getting in the neighborhood of 20-25%, functional cure rate, significantly higher than standard of care currently or anything else that we are aware of that's in development, to be honest.
To wrap up, we have about $100 million, in cash, as was reported at the end of Q1. No debt or outstanding warrants. Estimated cash runway into 2027, with some near-term milestones in both the single ascending dose data and then the multiple ascending dose data later next year in our celiac program. Thank you very much for listening, and I apologize for the technical difficulties.
Thank you for the presentation, Bill. For the audience, you can now ask questions at the chat box.
As far as fundraising goes, again, we have $100 million, in cash right now, and that gets us into 2027. No immediate plans to do fundraising. We'll wait to see if we get some reasonable data here from our celiac program. To go back to one of the questions on the readouts, we are expecting data from the SAD, portion of the celiac study, towards the end of the third quarter, early fourth quarter of this year, and then from the MAD, portion of the celiac study, around the middle of next year.