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This is an interactive study with them, and that will be done in Finnish, but all the slides will be in English. The reason why we do this first part in Finnish is the request by our shareholders and investors that they would listen once at least to all this exciting data in their home language, and we will be doing that today. But don't worry, at the end of the seminar or event, then Juho Jalkanen, our COO, comes in and then summarizes everything in English. So you will eventually get pretty much the same messaging from that talk. And I also remind you that you are free to do all the questions during the event. Just send them to that address that was provided to you earlier. So with these opening remarks, I will then switch my language to my own home language, and that is Finnish.
Then just invite Mika Kontro and Maija Hollmén to come on the podium, and then we start to really talk about myeloid leukemia, the most aggressive leukemia we still have around us, and it's probably time really to put them on hold and get proper treatment for those patients. Maija and Mika, welcome here. The floor is yours, please.
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[Foreign language] I'm now going to switch to English because the next speaker is our Chief Operating Officer, Juho Jalkanen. He will now summarize these same findings we have been presenting in Finnish. It's pretty much new data we announced yesterday. Juho, please.
Thank you, Markku. Thank you, Mika. Thank you, Maija. Lovely presentations. First of all, everybody, so grateful to be here and present this new data to you. First of all, Mika talked about how dreadful AML and MDS are as a disease. We've been working in the medical field over 20 years with HMAs and azacitidine, and still no improvements. The big problem is being refractory, being non-responsive to it, and we desperately need something for these patients. Now, across literature, it's been known that if you are Clever-1 positive, you usually do not respond to standard of care, that being a hypomethylating agent, and you have a pretty bad outcome. Our non-clinical work that Maija presented has shown that we can overcome resistance to HMAs. I'm going to look at a slide after this one on that topic.
Now, for the first time on this planet, the phase I data shows that we can really overcome resistance to HMAs and get responses in patients that otherwise would be non-responsive. On top of that, the ongoing follow-up shows that it is very likely that we will clearly succeed in the overall survival of these patients. In other words, lifting the Kaplan-Meier survival curve another bar up. That's what you ultimately want to do in cancer care. So far, in these non-responsive patient populations in HMA-failed MDS, the response rate is now at 7 out of 8. And the only one that wasn't a clear response was unfortunately dropped out due to a serious unrelated adverse event while being stable and already early in cycle 2. So I would say that patient even didn't have a chance to respond yet.
So overall, we're still looking at an exceptional response rate at the moment. A bit early to really state what will the likely survival be, but it will be good. This is a new slide Maija presented. I just wanted to pop it up, talk a little bit about it. Why is Bex effective in AML and MDS? First of all, the upper row here, that's a Clever-1 positive happy AML MDS cell, a blast cell. That network map, that's everything going on in the cell regulated by Clever-1. So it's like a vibrant city, lively, vibrant city, and the blast cell is a happy camper there. You can, we are at war against cancer. You can throw in HMAs, you can bomb it with HMAs, it's not going to work for this cancer cell. But give it Bex and look at the network map.
All that vibrant city infrastructure is destroyed. It's going to cripple that vibrant, happy camper blast cell. And then throw in the HMAs and get rid of the blast. So that's ultimately what Bex will accomplish in layman's terms. Because even I can't understand that science. Then to the clinical data. So this is a waterfall plot of all patients treated so far. And you see the blast reductions in the bone marrow. So again, highlighting the majority of treated patients get a blast reduction. And what Mika covered there, a lot of these patients are high risk, refractory, usually non-responsive, and now we see responses in these patients. Another way to look at this data, these are very busy slides. Again, swim lane plot on everybody treated so far. You see the responses, you see the duration. If there's an arrow, they're ongoing.
So we have a number of these patients, some almost up to 2 years, some over 1 year. The thing in AML and MDS, even with AZAs, that if you get a response, they're usually short-lived. But here it clearly seems that we can get duration as well, leading to the survival benefit we are likely to see at the end. So it's looking very good for the drug at the moment. But maybe to put it in perspective here for everybody in the audience. So we're hitting very high response rates in especially high-risk MDS, refractory and frontline. Ultimately, for a treating physician, the objective is to get these patients into remission so that they could go for a transplant, which is the ultimate cure. If we can't get them into remission, let's give them a drug that gives them improved survival, a survival benefit.
So I get asked, if you look at the numbers here, we do a lot better in MDS than in AML. So I get asked, doesn't it work in AML? What's happening in AML? It's the same stuff we saw in the MATINS trials. For those who have followed us over the years, MATINS was highly advanced solid tumors. We can get responses, but they come in the form of stable disease and survival benefit. And that's what we're seeing in AML. AML is so far into the disease, the blast counts are so high that it's very hard to win the game at that point anymore. We can hold back the cancer, we can improve survival. We've shown that the likely survival rate, median overall survival, is going to be above 8 months, which gives us ground to run possibly a randomized study against the comparator in AML.
But the low-hanging fruit here is, again, in high-risk MDS and especially refractory MDS that has failed HMAs because the response rate is very poor, the survival is extremely poor, and that's what we can significantly improve. So key learnings with the drug, and again, what we see in AML, MDS, and what we saw in MATINS: getting early. In MDS, we're right on time still to intervene with the course of the disease. And like we saw in MATINS, treat Clever-1 positive patients. AML and MDS, they're Clever-1 positive patients, almost all of them. So this is why we're seeing these great results. And if we do this broadly applicable, cancer in the war against cancer, cancer is going to suffer another defeat. You know, the big defeats for cancer came with radiation, chemo, checkpoint inhibitors.
We feel Bex is going to deliver one more defeat to cancer. Our vision as a company is to get that phase II data. We're going to talk a little bit about partnering. Then partner to get more resources. Take Bex to its first approval in refractory MDS. With the data, the stuff we've been talking about, we feel that Bex could become a cornerstone of cancer care. Because given the MATINS data, it's proof of principle that it's likely that we can overcome resistance also to checkpoint inhibitors. We have data now from AML and MDS. We can improve hematological recovery. We know from our preclinical studies that in combination with chemo, we can improve hematological recovery and likely also survival, chemo being the most used drug in cancer.
But to get there, to establish Bex as a cornerstone of cancer care, this is where we need to produce clinical data. But we start focusing and getting the first approval in refractory MDS.
Thank you, Juho.
My pleasure.
Let me ask right away, you know, what is Big Pharma waiting from us and how they have reacted to the data we have now provided from the BEXMAB study? Are they interested in moving on with the partnerships?
I get asked that question a lot. So when are you going to partner? That's a question I hear all the time. And let me take you through how Big Pharma works a bit. You know, 80% of their pipeline comes from outside of their own company: small biotech, secondary research, 80%. So most of their pipeline comes out of their internal resources. The bar for producing enough data has risen during the recent years. They made bad bets along the line. Some of the bets have been bad bets. So the bar for proof of concept has risen. Ultimately, Big Pharma is looking for phase II data. For them, it's a matter of de-risking the drug they're looking at, being Bex here, de-risking it to a certain point that they feel confident that it's going to make it to a drug.
Now, if you put the math together on where we are at the response rate and the increasing number of patients we've treated so far, the likelihood of Bex becoming a drug is coming pretty obvious. As I mentioned, ultimately, Big Pharma is looking for phase II data. But at the moment, if you do the math, it's pretty de-risked already. If you look at, for example, MDS, the refractory and the frontline, you can't necessarily combine the both populations. But altogether, we're looking at 12 out of 13 patients have responded, and the one that didn't dropped out early. So in MDS, as a total, it's looking pretty obvious that there is a drug here.
All right. Has the anti-CD47, CD anti-CD47 failure, so to say, impacted on this field?
Yes, it has. The macrophage field is complex. It's been difficult. There's a lot of failures in the macrophage field. 47's recent late-stage failure with Gilead didn't help our case, unfortunately. But I actually feel very strongly that we would be compared to 47 as our mode of action completely differs. First of all, targeting 47 will allow macrophages to eat what they encounter, hence the cancer. But we will allow the macrophage to present what it eats to the immune system. So totally different. And targeting, for example, 47 will kill red blood cells. Anemia is a big problem for that drug. And as Mika presented, here's what I don't get as a physician. You got a vulnerable patient population like AML and MDS who suffer from neutropenia, anemia.
You're going to throw in a cancer drug like anti-47 together with another one like azacitidine, venetoclax, and they're all going to kill cells. They're already anemic. They're going to kill cells. What do you get? Tox issues. Surprise, surprise. Easy to say, you know, hindsight here, but it was clearly obvious this is going to happen. We, again, as I mentioned, and the data shows, we're likely to improve hematological recovery, improve anemia, neutropenia, and all that. So it's totally opposite. The tox issues seen with 47, in any way, cannot be compared to us.
Do you think that one day Bex could be even used to help the chemo-treated patients who have a loss of bone marrow activity?
Exactly. That's how we believe. So Bex could be so widely used, possibly in the future, but it's going to need a lot of clinical work behind it to prove that to the world. But ultimately, yes, I do.
Thank you, Juho. So I will now thank you, all the people who have also now hopefully sent some questions to us, and invite Maija and Mika to come back over here. I will then guide to the proper questions. We will do this first in English, so questions in English first, and then at the end also the Finnish questions. Paavo and David, please.
Thanks, Markku. First question here is probably for Mika. So venetoclax plus AZAs combination trials are ongoing in HMA-failed MDS. How would you compare that treatment with the Bex plus AZAs combination?
I think that's an excellent question. There actually have been also several ongoing trials in the newly diagnosed MDS, azacitidine plus venetoclax. We currently are still waiting for the readout for those trials. With regard to newly diagnosed AML, we do know that toxicity has been issued for venetoclax. For that reason, we actually are currently running our Pan-Nordic LD-VenEx trial, where we try to de-escalate the dose of venetoclax to avoid toxicities. The concern for MDS has been, as Juho pointed out earlier, that we see patients with cytopenias, and then with venetoclax, we even deepen those cytopenias. There is, for that reason, certainly room for bexmarilimab.
I think that what we really need to do is wait for the data to see what kind of toxicities are related to AZAs plus VEN, especially in relapse setting, which is, as mentioned, very difficult to treat patient population.
Thank you. Next question. What does the competitive pipeline in MDS currently look like?
Maybe I'll take that question. But you know the study pretty well. Well, actually, in refractory MDS, there's not really much ongoing. I'm pretty confident saying we are the leading program. There is also, as mentioned there, AZAs and VEN in the refractory, but that's mainly driven actually by an investigator-initiated trial from MD Anderson. AbbVie, as the company sponsoring venetoclax, they're doing frontline high-risk MDS in a phase III. So the refractory setting is pretty clear. There's also a new trial with bispecific CD123/CD3. It had tox issues. It's a drug from Novartis originally. It's been pulled out. But again, it's an investigator-initiated trial from the U.S. in refractory. So the refractory is pretty, I would say, clear from competition still at the moment. So it's such a hard disease to treat.
In the frontline high-risk setting, as mentioned, there's a recent setback with CD47, then recent setback with sabatolimab, the anti-TIM-3 from Novartis. The only thing really that's going to possibly change the landscape is the azacitidine and VEN in the frontline high-risk MDS, at least on my radar. How about yours, Mika? Yeah, as mentioned, we still are waiting for the data. It has been kind of a long waiting road because the readout has been delayed and delayed. Of course, that was the same thing for VIALE-A trial, which ultimately showed the survival benefit in AML. As mentioned, we are still lacking the data for frontline MDS. We really should see that prior to making any conclusions.
I would like to add that hopefully you noticed that among those 8 patients we have now in this HMA-failed MDS group, 2 of them had previous magrolimab anti-CD47 treatments. They were refractory in there. Then they came to our trial, and we have a response. So that's just indicating how different the mode of action between these two molecules really are.
Maybe to touch upon that, because there's also a lot of patients in the trial that had received AZAs and VEN previously in AML and MDS and still being refractory to that. They were responsive to Bex and AZAs. So how I see even being a bit premature, we don't know the data from the phase III in frontline high-risk MDS. But even though AZAs and VEN would become a new frontline in high-risk MDS, there's still going to be the refractory population. Totally wide open space for Bex and AZAs to play in. Because people are still going to refract. Bex is still going to work for them.
Next one, please.
It's a market-sized question. So what is the size of the patient populations that you're looking at for your indications?
Juho, this is for you.
This is probably a market, I'm thinking trial size or market size. Maybe just touch upon the trial size then is, since there's no treatment approved in refractory MDS and it's an orphan condition, the trial size is likely to be small. We have sized them. I'm not going to state them out here. But we're talking even a registrational phase III kind of trial could be 150, 200 patients. So we're not talking big trials.
If I may comment on the kind of patient population size, so currently, as has been discussed previously, we do not have treatment options for those patients that fail HMA. Ultimately, all patients that will not receive allogeneic transplantation, which is the majority of the population, will ultimately fail. Of course, the question is then that should the bexmarilimab be in the first line already? But still, that hasn't been the case. So for that reason, we actually have quite a large patient population considering all those patients that will fail HMA treatment.
Yes, for the market and target population to be treated, it's relatively big in the field of high-risk MDS. But the way we see it as a company, we're going to work our way through first from the refractory, get the first label, get that population drug to market, start revenues, then move up to the frontline. And again, there's plenty of room. There's plenty of patients that are not suitable for transplant. AZAs and VEN, I hear it's not so popular with physicians due to the tox. You want to reduce the dose. And there's a lot of stuff to do there.
Yeah, yeah, certainly. Just to summarize, even this refractory population is tens of thousands of patients which are annually available.
We're still talking a significant market. Recently, GlobalData estimated that in 2028, the global market for high-risk MDS would be $2 billion. And 2038, estimated would be $3 billion. So it's roughly there. If that's the market size, Bex would play a significant role in that market.
Anything else in English?
Last one so far, Markku. What type of partner would be ideal for Faron? So only hematological cancer or solid cancers as well?
A rich one would be my favorite. But that's. You can answer.
So there's a couple of aspects. We look at partners and discuss with a lot of them. Ultimately, it would be perfect if the partner would play in AML and MDS, for example, have a hypomethylating agent and have an anti-PD-1 we could work with in solids. Ultimately, that would be ideal. Another scenario, if a partner were more hem-focused and not playing solids, is that we partner with him only, and we continue with the revenues from there. We continue the solid tumor development. So we'll need a lot of significant value in the company also that way.
Thank you very much. Paavo, are there any questions in Finnish? [Foreign language] ?
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Seuraava, kiitos.
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Thank you very much for really good questions. Thank you for following us. We keep informing as soon as we make next steps and progress with the program. Obviously, we will also come back to our shareholders during the AGM, where hopefully we have further discussions on this topic. Thank you very much for joining us. I also want to thank the speakers for the excellent talks today. Maija made everything clear because there were no questions.
That's good.
Thank you. Bye now.
Thank you.