On record. Okay, okay. Martin.
Martin Welschof.
Welschof.
Yes.
Good afternoon, everyone. As you just heard, my name is Tim Otitola. I will be representing Jefferies Investment Banking here at the Healthcare Global Conference, and it is my great pleasure today to introduce Martin Welschof, CEO of BioInvent International AB. Thank you.
Thank you very much. I will jump right into my presentation. We'll start with a little snapshot of the company. BioInvent is a publicly listed company in Sweden based in Lund. We are roughly 120 people at the moment, an integrated company in that sense. We do target discovery, antibody discovery. We do our own manufacturing up to 1,000 liter GMP. We do also formulations. We will later see that we not only have IV, but also subcutaneous formulation that we have generated in-house. Of course, clinical development. It's actually a powerhouse in immuno-oncology. In that sense, we're currently running six clinical programs. Those are five compounds in various stages of clinical development. The lead compounds are now in phase two, and that's actually where we'll focus mostly on today. I'll give you also a quick overview about the whole portfolio.
We're listed in Sweden, Stockholm OMX, but we have a strong international shareholder base. As you can see, major owners are Redmile, Van Herk, Forbjorn, HBM, Omega, AB4, and Invesco. Those names control roughly 65% of the company. The rest is in the hand of small Swedish and retail investors. Regarding runway, we just did actually a royalty purchase agreement with Zoma that gave us $20 million upfront, the whole deal volume of $30 million. Together with the cash that we already had, we have a runway until the end of next year. The platform that I will also briefly introduce to you that we used for our portfolio has been nicely validated in a number of deals. You see the names there: Pfizer, Daiichi, Bayer, Mitsubishi , Takeda, Genentech. Before I go into the portfolio, just very briefly, introduction to the platform.
It's called FIRST, and it's a reverse phenotypic functional screening that allows us to screen directly on patient material, which in our case, of course, since we are an immuno-oncology company, tumor material. In the first step, what we do is actually only selecting specific binders to the material of interest. Before we even know what the target is that they're binding to, we do strong functional characterization in vitro as well as in vivo. We would focus only on those antibodies that have strong therapeutic effects in a number of different animal models. Then we identify the target. What we get out of this: novel IO targets, as I will explain to you, TNFR2, FcγR2B, but also uniquely functional epitopes on validated targets.
We have our own proprietary anti-CTLA4 that is nicely differentiated from ipilimumab and actually a much stronger T regulatory cell depleter compared to IP. With that platform, we generated this portfolio, which is actually for a company of our size, quite a broad and deep portfolio. No worries, I will not go through everything in detail. On the left-hand side, you see the three different targets: TNFR2, FcγR2B, and CTLA4. TNFR2 we identified in the screening on ovarian cancer tissue as differentially upregulated on T regulatory cells, but only in the tumor microenvironment, not in the periphery. We have generated two different candidates, and the lead, as I will tell you later, has now generated nice efficacy and safety data and is in phase two. FcγR2B is another very interesting target.
It's not only expressed in the tumor microenvironment on dendritic cells, and it's the only inhibitor receptor of those cells. It's also expressed on tumor B -cells on Hodgkin lymphoma, where it basically contributes significantly to the resistance to anti-CD20-based therapy. That's also why we developed this not only in non-Hodgkin lymphoma, but also in solid cancer. Both. We do actually the same for TNFR2 because this is also expressed in a tumor microenvironment, but also in tumoral T -cells. I'll come back to that later in more detail. Last but not least, we have a program targeting CTLA4, which is an oncolytic virus combined with our antibody, also generating good data. Our main focus in the company is really on the TNFR2 and the FcγR2B platform.
Today I will mainly talk about the two lead compounds, BI-1808 and BI-1206. Starting then with the TNF receptor II program. In that screen on ovarian cancer tissue, we identified several hundred specific antibodies for TNF receptor II. Out of those, we selected BL-1808 and 1910 with very different mode of actions. BL-1808 is a first-in-class ligand-blocking Fc receptor engaging antibody. It is an IgG1, whereas BL-1910 is an agonistic antibody, both resulting in anti-cancer activity, but in different ways. In that context, we just submitted a scientific publication to a high-ranking journal describing both mode of action and the clinical data that we have up to date in more detail. Hopefully that will come out soon. On the right-hand side, you see a number of competitors. It is mainly actually China.
For the reason, TNF receptor II plays a role within the anti-PD1 pathway. There are synergies. Since China has probably the most competitive anti-PD1 market that you can imagine, I think there are 15 different anti-PD1s in the market. They all try to differentiate themselves somehow. When we started to work on TNF receptor II, they kind of followed. We are at least one year ahead. We have now, as you will see, efficacy data, safety data, whereas they have just the first safety data sets. Going forward, mainly focusing on BL-1808, that is the clinical trial design. We saw pre-clinically that there was strong single-agent activity as well as strong synergies with pembrolizumab. That is why we developed this in those two arms. We have done successfully the dose escalation. No toxicity issues at all.
Very, very safe even in the combination. Now we are in the dose expansion cohorts focusing on ovarian cancer, lung cancer, GIST, and then TCL, including CTCL. That is what we are looking at at the moment. By the way, I forgot to mention it on the overview portfolio slide. Whenever we work with Pembro, this is always under supply and collaboration agreement with Merck. We have five of those agreements and one with AstraZeneca regarding acalabrutinib. This is the most recent data set out of this study. This is very promising data on CTCL. We had an abstract at EHA, and there will be an update next week when the poster comes out. At that time point, eight available patients, and we had responses eight out of eight, one complete response, three partial responses, and four stable diseases. Super well tolerated.
We have actually two ways of mechanism of action here. The antibody, of course, depletes the tumoral T -cells, but also what we see is an influx of CD8 positive cells into the lesions in the skin. It is also an activation of the immune system that is happening. In the upcoming poster presentation, there will be more detailed and recent data, including also a very severe population within CTCL, PTCL. To give you an overview of what the landscape looks like currently in CTCL, you see there are a number of drugs with bad efficacy and quite some toxicity. We are very well based, even though it is still early days, with efficacy around 50% at the moment, and no toxicity at all. This is, of course, as a single agent. I think this is very compelling and exciting.
The timelines for the development of CTCL monotherapy in the U.S. looks as follows. We got orphan drug designation. We just received fast track designation. We're currently finishing this part, which is the part A monotherapy, and then also will be a little bit of dose optimization. Next year, we'll start the pivotal and hopefully then also get a breakthrough designation. I think this is a very interesting way forward for BL-1808 in CTCL. To summarize then around the positioning of CTCL, I mentioned most of the BL-1808, the CTCL things. Very good safety, as I said. All available therapies so far, as I told you, have limitations in both safety as well as efficacy.
With an overall response of 40% or a little bit higher, along with a very good safety profile we have, I think we have a very good positioning as a frontline treatment of choice in that indication. On the other side, and that's actually where we started, we have the solid tumors, and I will show you also where we're standing there. We saw actually single-agent activity in ovarian cancer, in non-small cell lung cancer, in GIST, and also in TCL. On the next slide, there's a summary of the dose escalation that we already published at ASCO. That was 26 available patients. We had a very nice complete response, partial response, and then a number of stable diseases.
When you look at the data, because this is obviously in patients that are end stage, it looks very much like Pembro, the first data when they were not frontline, but at the end of the treatment. To give you also a little bit of impression there, this is the ovarian cancer patient, 63-year-old. She received all the usual treatment that she should get. Nine weeks into the treatment was a very nice, stable, complete response, single-agent activity again. I think that is important to emphasize. Same for GIST patient. That was actually even more impressive because that we first thought it was progressive disease, but then it was pseudo-progression, went very nicely down. There you can also see the effect on the immune system.
This is pre-dose, and this is five weeks into the study, and all the green dots are CD8 positive cells that have invaded the tumor tissue. That patient now is probably also a complete response already because we did biopsies in the areas where the lesions were. We could not find any tumoral cells anymore, which means this robust partial response is probably now a complete response. This is where we are currently with that program, BL-1808, TNF receptor II. Now I want to switch gears and talk about FcγR2B and then mainly on the lead program, BI-1206, that we are developing in non-Hodgkin lymphoma as well as in solid tumors. Again, the reason for that is because the target is expressed in a tumor microenvironment of solid tumors, plays a big role there, but also on tumoral B -cells in non-Hodgkin lymphoma.
I will start with the non-Hodgkin lymphoma first. There's a little bit of background on the receptor. That's the only inhibitory receptor of the innate immune system, though there are a couple of activating receptors. I think the unique thing about BI-1206 is the specificity for the inhibitory receptor only because these dose receptors, they are quite homologous, and that's why it's very complicated and difficult to get a monospecific antibody. I think we are the only company that achieved that. On the non-Hodgkin lymphoma, this is data that was published in Blood quite a while ago, clinical data looking at mantle cell lymphoma, follicular lymphoma, and diffuse large B cell lymphoma. There you can see whenever FcγR2B is highly expressed, it means bad news for the patients because the outcome is basically bad.
It's associated with shorter survival in those patients receiving rituximab therapy. I also have to provide a little bit of background here. When you talk to KOLs, including Leukemia & Lymphoma Society, even with CAR-Ts as well as bispecifics, anti-CD20-based therapy will remain core and center for at least the next 10 years because it works very well. It's very safe. The only problem with it is that every patient at some time point will develop resistance. Will become resistant and cannot be treated anymore with anti-CD20-based antibodies. In this case, it's rituximab. That's where we position because BI-1206 basically blocks the major resistance mechanism to anti-CD20 and at the same time point also acts single-agent activity because it's an IgG1.
The first data set they have generated, and this is actually patients that do not respond anymore to anti-CD20-based therapy, was actually already quite impressive. This in combination with rituximab, those patients who do not respond anymore, we get a very high overall response rate, high number of complete responses. I think what is also quite interesting and intriguing is that we have long-lasting complete responses, so high quality of complete responses. We now have a number of patients that are in complete response for more than three years after the end of treatment, which I think is super cool. Based on this, in order to get this even higher from roughly 60% overall response to over 70%, we talked to AstraZeneca and got them interested and basically started this study, which is combining not only rituximab and BI-1206, which by the way is sub-Q.
It is an easy five-minute injection, but also then acalabrutinib in a triplet. That study is running. We have had the first data out, and the first eight patients were all responding, two complete responses, three partial responses, three stable diseases. Very nice overall response rate and a disease control rate of 100%, which I think is very good. Of course, and that is part of the important positioning, no toxicity. Absolutely safe. We will move this further on. By first half of next year, we will have 30 patients. This is eight patients. I think we have a significant data set. AstraZeneca obviously is following this very interested because they do not have follicular lymphoma. That is what we are focusing on their label. They just have mantle cell lymphoma. I think there could be some interesting discussions.
The plan for this is obviously then targeting second line, not first line. We're currently finishing this phase 2A. As I said, by early next year, we will have, first half of next year, we'll have the data. We could agree on the pivotal phase 2 design because we already had discussions with the regulators, and they agreed that we can do approval based on the pivotal phase 2. Of course, we also go for breakthrough designation. This is, of course, data dependent, but as long as the data looks like as it looks at the moment, I think that should be a possibility. This is follicular lymphoma. I think I covered all the things that are mentioned here on the left-hand side. That, as said, is done together with AstraZeneca under a collaboration and supply agreement.
On the right-hand side, we target FcγR2B when it's expressed in a tumor microenvironment. There we're targeting patients that do not respond anymore to either anti-PD1 or anti-PDL1. This we do under supply and collaboration agreement with Merck. We have both on the same compound. We're about to finish the phase one dose escalation. We saw some very interesting responses there, complete responses, partial responses, number of stable diseases. I think what is important to remember is that those patients have received at least two or in many cases three IO-containing regimens and do not respond anymore. Just to give you also the overview of the trial design, this is ongoing. We started with the IV. Now we're working also here with the sub-Q, and that's what we're mainly focusing on.
Going forward, we will focus on non-small cell lung cancer, melanoma, and other tumor types. Just to give you a glimpse of the data, this is a very robust partial response, now ongoing for many, many years. That patient is under compassionate use now. That was a lady that basically received everything, multiple lines of ICIs and chemo, no response anymore, developed a very nice robust partial response, is still in partial response, still improving, and now treated under compassionate use. I think very impressive, keeping in mind that oval melanoma is very difficult to treat. The same for cutaneous melanoma, it was male. That person received three lines of previous ICI therapy, developed first partial response that then turned into complete response, which is also still ongoing and which is, I think, also very compelling and interesting.
This is basically a summary of all the responses that we got with BL-1808. We did not discuss today BL-1910, but there will be the first data set, meaningful data set by the second half of this year. Of course, the various BI-1206 studies. I think across the board of the portfolio, we have good progress. The key catalysts for this year, and that is where I will stop. I mentioned this data is out, but there will be an update next week. BL-1808 in CTCL single agent. We have the data out for the triplet combination. The next release will be then probably by the end of this year or early next year when we have closer to 30 patients. There is still data to come, phase one data with the combination with BI-1206 and Pembro, which will be soon.
During the second half, as I mentioned, we'll have further data for BL-1808, but then this time in combination with Pembro, Bl-1910. I mentioned two data sets, and then we didn't discuss that at all, BL-1607. We'll also have the first data set by the end of the year. I'll stop here. Thank you very much.
Thank you so much. We can now transition into general Q&A. Please let us know of any questions now.
Do you mind terribly putting back the glass slide?
No, it's this one.
This one?
Yep. This one?
Thank you.
Okay. Any other questions as we progress here?
Yeah. Would you mind talking a little bit about where those studies are being conducted?
Yeah.
What countries are you looking at?
It's all our studies under IND as well as the approval in Europe. I can't tell you exactly which is where, but CTCL, and maybe I can start with that one first. We are in all the important centers in the U.S. because in the U.S., CTCL patients are treated in centers, not locally. Also, we are now opening sites in Spain and in the U.K. I think also for the other solid cancer, we are in the US and in Europe. BI-1206, I think it's mainly in Europe, but also a couple of sites in the U.S., and BL-1607 and BL-1910 as well. What we normally do is have IND as well as the European approval. Then we try to identify sites either in the U.S. or in Europe where we can do the quickest recruitment, and then we broaden up.
That's basically what we do.
Are all the assets developed internally?
Yes.
Yeah.
Yeah. So nothing has been, there's only one license agreement around BI-1206 for China, Hong Kong, and Macau. Otherwise, it's unpartnered. We have, of course, a quite extensive collaboration with Merck and AstraZeneca, but this is only supply and collaboration agreement. They don't have any rights to the compound.
Making the transition between pre-human study with your resources, how did that go? Sometimes being challenging.
Yeah. We have, as I mentioned at the beginning, we're quite integrated as a company. All the preclinical work we do also in-house. We have access to animal models. We have even experts in-house that can generate, if necessary, transgenic animal models. That's also what we do. It's mainly in-house. Of course, whenever we need support from outside, we take that as well. It's mainly in-house, actually. The company is around for quite some time and has gathered all this experience and knowledge that we use for the development of this portfolio.
Even into the clinic.
Even into the clinic. Exactly.
Thank you.
You're welcome. Ashley, any questions?
She took all of mine.
All right. I think we can wrap up here. Thank you so much, everyone, for attending. I hope you enjoy the rest of the conference.