Welcome everybody. We switch gears completely, so from bathroom to the hospital, cancer therapy. I will try to give you a quick summary what we are trying to do at BioInvent. At the end, I understand we have a Q&A, such that we can go a little bit deeper and hopefully then explain things that might be not completely clear. This is our forward-looking statement since we are listed at the Stockholm Stock Exchange. I would like to start with a short summary or snapshot of the company. As you can see from the headline, we are trying to invent or develop new cancer therapies, and that's obviously quite necessary, and I'll come back to that later in more detail.
So there are good therapies out there, but there's still a so-called high unmet medical need. And specifically, we are trying to address resistance to current cancer therapies, because as I said, there are a lot of good cancer therapies out there, but at some time point in their life, all patients will become resistant to the current standard of care, and what do you do then? And that's what we're trying to address. And we have a specific focus on cancer biology, and we have a lot of knowledge around immunology. So basically, what we're trying to develop, and we had good progress during the last couple of months, is innovative immuno-oncology therapies. And as of today, we currently have five programs in early stages of clinical development.
First programs are already showing signs of efficacy. The platform that we're using, that I also will explain to you, briefly, has been nicely validated through a number of partnerships with pharma companies. We're currently around 100 people. They're all located in lovely Lund, and we're currently having roughly around $150 million in our bank account, which give us a very nice runway to do the work that we want to do until the end of 2025. I come back later to the programs just on the technology. So the company is relatively integrated. That means we do the discovery piece. That means we identify new ways of therapies, then we characterize them first in animals. That's pre-clinical development, so-called.
Then we move it into clinical development, and we also do actually our own manufacturing, so we can produce the materials that we're using clinically ourselves. As I said, so that's the second bullet. The technology that we're using has been validated now through a number of deals, and some of the names you will recognize, companies such as Daiichi, Bayer, Mitsubishi Tanabe, Takeda. They have compounds from our platform in clinical development, either phase I and phase II. And this gives us good confidence that not only BioInvent is using the platform to develop new therapies, but also international pharma companies, too. We are listed in Stockholm. I mentioned that already at the beginning, but we have a very strong international shareholder base.
And basically, just to mention the names you will see there, these are high-quality U.S. investors, European investors, really, really large funds that support our development, including Redmile, Van Herk, Forbion, HBM, Omega. But then we also have a high-quality Swedish and Nordic investors such as AP4, Swedbank Robur, Handelsbanken. Those were names, will be names that you're probably very familiar with. I mentioned already the solid cash position. I think that's quite important because, as you probably know, due to macroeconomics, especially companies in the biotech sector, they're actually suffering. And if you don't have a strong balance sheet, so then you have a problem, and we don't have that problem, luckily. So, to set the stage a little bit, so you might have heard about immune checkpoint inhibitors. So the number one product there is KEYTRUDA from Merck.
That's a $1 billion product per year, and that is used actually quite broadly. But just to mention, without going into much detail, so basically 10%-15%, in some cases, 20% of the patient respond. So there's a remaining part of 80, at least 80% of patients that are not responding, and that's what we're trying to target. So as I said, there's still a huge unmet medical need. And one reason for this is that the number of specific targets for therapy that are, you know, useful for therapy, is still very limited. So all the companies that are in the space basically work on a number of target structure that is a little bit more than 5, maybe, at the most, 10, with different compounds.
What we have at BioInvent is a technology that allows us to screen for new target structures and for new mode of actions, consequently. This platform is called Function First, has been developed at BioInvent. We have it IP protected, and this is a different way of identifying new mode of actions. And what we do, we have a very close collaboration with the local hospital in Lund, such that we receive fresh patient material on a regular basis, and we screen on that patient material. On that patient material, we try to identify structures that could be useful for cancer therapy. And then a very important part of the screening is that we actually, very early in that process, we also go after functionality.
So that means at some time point, out of our compound library, which is an antibody library, we find then antibodies that specifically bind to this, those new structures. And before we actually really spend a lot of time in characterizing them, is really that we check immediately at that stage in animal models whether they have therapeutic effects. And then we would only select those that show strong therapeutic effects in a number of different animal models, and for those, then we at the end, also identify the target structure. So that's the platform that we use, and that's also the platform that has been used by the partners that I mentioned on my previous slide, so Daiichi, Takeda, Mitsubishi, et cetera. So we are in very good company in using that platform ourselves.
So with that platform, we have built a portfolio of different potential product or drug candidates. And you can see that we also had actually quite some speed, and that also differentiates us from the rest of the biotech companies in the Nordics. I would say even in Europe. Because normally what a biotech company does, it would focus on one program and then try to run it all the way through. We have chosen a different strategy. That means we are really going after a portfolio, which is also risk diversification, because, as you know, clinical development is quite risky. So even if you do a good, very good characterization in animals, it doesn't translate one-to-one in clinical success, and that's why we have a number of programs.
And since we have a very integrated company, I mentioned discovery, development, as well as manufacturing, we can grow very quickly. So 2018, you see that on the left-hand side, we had 1 program. Last year we had 5 programs, and by the end of this year, we'll have 6 programs. And they're all so-called first-in-class, which means that they're completely new target structures and compounds that we are developing, which of course is some risk because we are the first one really trying to do that. But of course, there's also very high potential success, because if we are the first one showing that, for instance, one of our compounds is working, then that will translate into, you know, a potential huge commercial upside.
So on the programs that we're running, it's all targets that are related to the immune system. On the top, you see a target, which is called FcγRIIB. That's a target that we basically were among the first ones that discovered this target, and there we have two different compounds, as you can see, BI-1206 and BI-1607, that are currently in clinical development, and I will come back later to a little bit into more detail. Just to mention it already here, so the BI-1206, we already have partnered with an American Chinese biotech pharma company called CASI for China, so they received an exclusive license for China, Hong Kong, Macau, and Taiwan. And that shows also a little bit the model that we have.
So currently, we don't have the ambition to commercialize ourselves. So what we are doing is identifying new potential ways of treating cancer patients, showing that that works in the clinic, and then we try to find a pharma partner that could then more broadly develop it, after we have shown the proof of concept and then, also can do, the marketing and production. So that's kind of our business model, which I think also is a, a nice, way of sharing risk and, and, and upside. Then the lower panel, this is on, on, two different targets. One is TNF receptor 2, which is a very new target that we have discovered among, one other group in Boston, and there's already quite some activity around it.
So Novartis has a program, then BeiGene, which is the largest Chinese pharma company, has a program, and some other companies as well. But we are still leading at least one year those companies. We're the first ones putting it into the clinic. And there we also have two candidates. One is called 1808, and the other one is 1910. And then the other target that we're working on is CTLA-4, and that's actually a target which is already known. So there's an antibody called ipilimumab that is already used in the clinic, but the problem with ipilimumab, it's very efficacious, so it works well, but it's also very toxic, so you cannot use it for a long time. And we have a different version that we use locally, and by this reducing the toxicity.
So now I will go a little bit program by program to give you some more details. So starting with 1206, which is our lead program, and that's a clinical trial design. Looks a little bit complicated, but I will try to simplify it. So basically, what is important for you to remember, is that we're focusing on patients who do not respond anymore to the standard of care, which in this case is a compound which is called rituximab. And then what you do, you move through a path which is called dose escalation, where you know, on a constant basis, increase the dose until you find the dose that is the right dose in order to achieve efficacy, and then you go into dose expansion.
So we have done the dose escalation part, and I see already good results that I will summarize to you in the next slide. And we're now in Part B, the dose expansion part. That means a later stage of clinical development. So the data that we have seen, and keep in mind, those are patients that did not respond anymore to the standard of care. They have received a number of lines, standard of care, no response anymore. That means those patients normally would have no treatment options as of today. And in this patient pool, we saw four so-called complete responses. Those are responders that are basically disease-free, and they have been now disease-free for a couple of years. So long-lasting complete responses, that's what the regulatory agency want to see.
Then we also had a couple of partial responses and what you then also call stable disease. So it's a very good response rate that we have seen. And we are now moving into the dose expansion part, where we then basically recruit more patients at the dose that we have selected. And just to mention it, we are also studying another formulation, so this is currently used intravenously. It's called the IV formulation, but we also have developed something which is called subcutaneous formulation, which is much easier because then you don't have to go to a transfusion center. You can do it at your doctor's office. It's just under the skin. So it just takes you know, 15 minutes or whatever, and some waiting period, and then you can go back home.
So it's also a very convenient way of treating. So that's basically a summary of what I have told you. So it's unique. It's first-in-class. That means we're the first one really developing this type of therapy currently in hematology, and we also are developing it in solid cancer, and I'll come back to that in a minute. As I mentioned, so we're targeting patient population with a very high unmet need for safer. So those are patients that may basically at the end of their treatment, and they don't tolerate any toxicity, but of course, you want to still see a good efficacy, and that's what we're trying to provide. And it can be combined with the current standard of care.
I think what is very important, what you see below, is that you have to generate long-lasting, complete response, because you don't want to see a response maybe for another half year, which might be nice, but what you want to see is really years. Years of stable, basically, stability without any further disease progression. That's what we are seeming to achieve. Then the other part of BI-1206 is in solid cancers, and there we're targeting the so-called tumor microenvironment, which is also quite important. And there we're a little bit earlier, so it's the same setup. We are also, again, targeting patients that do not respond anymore to the standard of care. We're still in dose escalation, so we're not that advanced yet in this study.
But we have seen already very, very nice first signs of efficacy, especially in patients that have very, very difficult to treat, cancer diseases such as uveal melanoma, for example, or sarcomas. Very difficult to treat, and those are patients that have received already three times the therapy, which is standard of care. They don't see any response anymore, that means that is the last option actually for those patients. And we see some first responders, which is quite nice, but we have to go a little bit deeper into dose escalation before we move forward. But it looks quite promising here as well. That's a summary. I think I mentioned that to you already.
Then, the second candidate, so we have a second candidate against the same target, BI-1607, which has a different way of working. And this has just started last year the clinical development, and we are targeting here especially also metastatic solid tumors. And, we're currently almost at the end of dose escalation, so that data package will probably read out at some time point during the second half of this year. Looks also quite promising, but just stay tuned when we have the readout that will be published, as I said, during the second half of this year. Then going to the TNFR2 program, so that was FcγRIIB, TNFR2. As I mentioned, we have two candidates. BI-1808 is our lead.
BI-1910 will go into the clinic also during the second half of this year, with a different way of working. But BI-1808 is already quite advanced, and we have tested this antibody not only in combination but also as a single agent, and we have seen preclinically that it works both in combination with the standard of care as well as a single agent. And, as you can see, we have two arms in the clinical development. So we have finished the dose escalation for the single agent, which means that's the antibody, the product alone. So very nice response, first signs of responses.
It's safe because that's what you do during that part of development, during dose escalation, that you want to see it's safe, patient can tolerate it, and you want to see first signs of efficacy before you then move to the next step. We have done that for BI-1808 as a single agent, and that's now will move into the so-called dose expansion. That will happen during the second half of this year, and you see the indication on the right-hand side. So it's non-small cell lung cancer, which is a very important and difficult to treat cancer indication. Ovarian cancer is quite interesting because there's no good immuno-oncology product approved yet, so this could be the first one.
Then we're also targeting a very rare T-cell lymphoma, which is also quite interesting, since it's a smaller patient population and for us, more easy to develop in that area. In parallel, that's the lower part. We're also developing it in combination with the standard of care, which is KEYTRUDA . And you've seen probably also on the other slides a couple of times, MSD. So we run a lot of our studies in collaboration with Merck, who are the leader in immuno-oncology, and they find our programs very interesting. That's why they participate also in our clinical development. So basically, the next step here is that we have the move from dose escalation for the single agent into dose expansion. So that will be the next important milestone for BI-1808.
So then, to BT-001, and this is actually even more complicated, but I will try to explain it as simple as possible. So this is a combination of two things. And we run this in collaboration with, you see the name, with Transgene. This is a company that works on oncolytic viruses. Those are viruses that can destroy tumor cells. And what we have done is we have developed this very unique anti-CTLA-4 antibody, and we have cloned the genes of this anti-CTLA-4 antibody into the oncolytic virus. So what happens is that the oncolytic virus is used and will be injected into tumor lesions. There, the oncolytic virus starts to replicate, and also during this process, it will produce the antibody.
So what you achieve with that, and that's the important thing, you get a local treatment rather than a systemic treatment. And that's important if you have an agent, which is very potent, very efficacious, but also might be very toxic. And what you do, you target very specifically just the solid tumor environment, but have no detection actually possible in the bloodstream or in other peripheral organs. So that's basically what we try to achieve, and it looks actually quite well. We even won a prize for this program, and we have finished the first part of the clinical development, which is the dose escalation as a single agent of that compound.
Could show it's safe, we could show first signs of efficacy, and now we're more or less ready to move in the second part. That's the Part B, where we combine it also again with pembrolizumab, because pembrolizumab is the leading product. But as I said, only up to 20% of patients are responding, so there's a huge demand to make that product even better. So that leads me to, yeah, there's just one thing before I go to the milestones for the end of this year. So I mentioned the collaboration partners that we have. So besides our own portfolio that I tried to explain to you, you know, at the beginning of the presentation, we have also a portfolio with collaborators. So I mentioned Daiichi, Takeda, Mitsubishi, Bayer.
Those are those programs, so they run those programs themselves at their cost. We have provided the candidate, which means we have received already some upfront. We'll get milestone payments during the clinical development. We get, once it becomes commercial, commercial milestones as well as royalties. So that's part of our portfolio as well, but we don't drive it directly. It is driven by those companies that I've mentioned. And in addition, just to mention, Exelixis. Exelixis is a company close to San Francisco. It's a mid-size pharma company. They have a very interesting billion-dollar product in the market, but they're interested in new modalities, new agents, and that's also something that we can provide with our platform. So this is an early development discovery deal. And just also to give you a little bit the economics.
So when we signed the deal, that was summer last year, we got $25 million upfront. They cover our FTEs, the work that we basically do inside of BioInvent, and once the programs are moving along the value chain, so they pick a lead at some time point, then it's a clinical development candidate. We had milestones, single-digit to double-digit dollar million per milestone, so it's actually quite interesting. And then at the end, it would be also commercial milestones and royalties. So we do this kind of activities as well, but our main focus is really on our own portfolio because that will be going forward the main value driver for the company. Then, as I said, so the expected key milestones for the remainder of the year.
So for our lead program, I mentioned that we will have, during the second half of this year, the preliminary results for the Sub-Q formulation, which is very important because, that's a very important milestone in that sense, because it makes the product more versatile and more compatible with, products that are already out there. Then we will also introduce that, Sub-Q into the solid cancer study for, BI-1206 in combination with pembrolizumab. Then we have, BI-1808. I mentioned our TNF receptor two program, where we have the single agent data, already available for the dose escalation. We want to move now into dose expansion, which is the, phase IIa , Part A. BT-001, this is the oncolytic virus in combination with our antibody. They will start soon the combination with KEYTRUDA , which is pembrolizumab.
And then we have another dataset that's reading out also during this second half, which is then BI-1607, our second FcγRIIB program. That will be the first dataset covering the dose escalation that will also be during this half of the year. And then BI-1910 is our second TNF receptor 2 that will start clinical development. So I think I just maybe stop then here. So basically, what we're doing is we are identifying new potential activities that can be used or mode of action, so-called, in the clinic addressing a high unmet need. And currently, we're not, you know, thinking or targeting ourselves to commercialize it, but we do that with partners, as already done with our lead program with CASI Pharmaceuticals. And we will do it going further as well.
As soon as we have established proof of concept, we will try to identify partners. Those collaborations and deals that you can do, they can be actually quite lucrative. So you're talking there double- and triple-digit million dollars. So, you know, $100 million and more upfront. And then you have, of course, clinical development milestones, and at the end, also can contribute in the commercial upside as well as royalties. And I think that is quite interesting, and also from our perspective, it's a risk diversification, so we're not carrying all the risk. We're trying to cover really the first part. And I think also one last sentence, which is important: pharma companies, they're not good in discovery.
So they're good in, you know, production, marketing, and then obviously also later stage broad clinical development, but they lack really the skills to identify new structures, new targets, and that's why there's such a lack of targets. And there's a lot of, when you look at the various pharma companies, whether it's Merck or Pfizer, they have a gap in their portfolio. So they have products that pay well, and then they have late stage, but they lack really this part that we are addressing, and that's why we, you know, BioInvent is in existence and got good support from notable large investors. Thank you very much.
Thank you for an enlightening presentation. I wouldn't dare to ask too much questions about the scientific side, but in this forum, I think maybe the commercial side, which you sort of elaborated on now, is very important as well. You have quite a number of catalysts this year, six, if I can count them.
Mm-hmm.
Could you perhaps rank them in terms of commercial importance, or is it something that you are more, more excited about than something else? Is it the subcutaneous-
Yeah, so the subcutaneous definitely, because, you know, for BI-1206, we already have shown proof of concept.
Yeah.
So we know it works. But I, as an IV formulation-
Ah.
which is nice. But of course, Sub-Q route would be the much better product for the reason that I mentioned, because it will be used in combination with one other product, which is called rituximab, which is already subcu. And then also the leader in immuno-oncology, pembrolizumab or KEYTRUDA , will be developed also as a Sub-Q version. And if you want to combine, then it makes sense to have it as a subcu. So that will be an important milestone. And then the other one, I think, since it's also a data readout, is then the BI-1607.
Yeah, of course.
Which is the second FcγRIIB, to see how it behaves during dose escalation.
Mm.
To set the stage a little bit, so we're still in early stages of clinical development. We have to go through that, which means the really interesting data is coming next year, because then we are in later stages of clinical development, and then we will really reach very, very interesting value inflection points that could drive the stock very nicely.
Yeah. So that would be the phase II data-
Exactly.
- and
Exactly. Exactly.
We have a question from the internet, so I will read this. It's from Matthias. BioInvent, great to hear positive vibes. But looking at the stock price, it's trading at a very low and below cash balance, which means there's like a negative value on the operations. And it's been so since May, so no one-off. Do you see any options or maybe some changes to the business model to perhaps remedy this, like buybacks? You have a strong cash position or perhaps scaling down the portfolio. Any thoughts?
No, I think, we have the clear support for pushing the portfolio that we have.
Mm-hmm.
We have very large shareholders that are, that are interested that we, that we move ahead, and those are very specialized-
Mm.
- Very knowledgeable investors. And those are even investors from the U.S. Redmile is one of the largest investors in the U.S. They see the entire U.S. universe. They picked us here in Sweden.
Mm.
To put it into context, yes, absolutely. So the share price, you know, excuse my word, sucks.
Mm.
But that's something we share with all the other companies in the sector, with maybe few exceptions, but all the earlier stage companies, whether they are preclinical or discovery stage or early stage clinical development, are completely undervalued at the moment. And that's not something only here in Sweden and for BioInvent, it's in the U.S., it's everywhere. Because all the non-specialists, they moved out of the sector and who's remaining are the specialists, and they're not trading. So Redmile, you know, the names that I mentioned-
Yeah
they don't trade. They sit there for the long term. They know, you know, good data is coming next year, and that will be interesting catalyst, and then the share price will go up, and then who knows what happens. So of course, the company could continue, but I think it also will be, at some time point, a very interesting target for M&A. So there's no plans to be very clear on scaling down because we have exciting data. For all the programs, we see the story intact. That means it's safe. We have first signs of efficacy, so it's full speed ahead.
Yeah.
The macroeconomics, they will change at some time point, and we see first lights, you know, already in the U.S. They always go down first, but they also come up earlier, so things are changing. And then once we reach those value triggers, anyway, you know, will just happen. So, I'm actually super bullish for the company.
That's nice to hear. In terms of regulatory affairs, there's high unmet need, there's first in class and so forth and so on.
Mm.
So does that entail any opportunities for fast track, orphan drug designation?
Absolutely
something like that, to cut down on the-
Absolutely.
But maybe that's a later stage?
Yes.
Okay.
We have discussions ongoing regarding BI-1206, but also regarding BI-1808, just to mention, because there we have a little bit more data.
Yeah.
For that, you need some more data, so there's exactly this possibility. Since non-Hodgkin lymphoma, we have orphan designation-
Mm
for subgroups in non-Hodgkin lymphoma, and we will probably also go for orphan designation for the rare T-cell lymphoma. So there's definitely the possibility to, you know, run smaller studies, and I mentioned also briefly during my presentation. But then still, you know, our aim is not to really then go all the way and, and commercialize ourselves. We were looking for partners, but then also for the partners, it's of course much more interesting if we're closer to market-
Mm
and we would have that possibility. Absolutely.
All right. I'm afraid our time is up, so thank you once again for a very nice presentation. And we're moving to one of your industry colleagues now. To-