Welcome to the Faron Pharmaceuticals Rights Offering webcast. My name is Juho Jalkanen. I am the CEO of Faron. As a public company, standard disclaimer as we will be making forward-looking statements. To today's agenda. It gives me great pleasure to bring to you today basically the entire management team. Today is all about Faron as a business case, and then the rights offering, explaining what it is, 'cause not that many are used to one. Let's move on. Starting with me here today is our Chief Scientific Officer, Dr. Maija Hollmén. Please, Maija, a couple of words on yourself.
Thank you, Juho. My name is Dr. Maija Hollmén. I'm the Chief Scientific Officer at Faron. I have over 25 years of experience in cancer tumor immunology, drug development, antibodies, and a special interest in macrophages, and that's why we are here now.
Yeah, I've heard you're a macrophage expert. Is that true?
I could say that.
Good. Nice to be next to one. What's the problem? Why does cancer kill? Treatment resistance. We have hundreds of cancer drugs. The pharma industry spends billions of dollars getting for the new groundbreaking drug, treatment resistance, that's the problem. The source of treatment resistance, or what are we here to do is we want to give immunotherapy, turning our immune system against cancer through harnessing the power of myeloid cells. Myeloid cells, for many of you, are known as macrophages. Maija, why are macrophages so important?
Thank you for the question, Juho. You're in the heart of modern immuno-oncology. Macrophages are very important for our wellbeing, as they destroy pathogens, help in immune responses, and maintaining tolerance. However, cancers normally hijack the system, and actually they give macrophages the possibility to support them rather than killing them. It's in fact known nowadays that cancer needs 14 hallmarks to grow. These hallmarks could be sustained proliferation, metastasis to distant organs, increased angiogenesis, immune evasion, and so forth. What is actually known about macrophages is that they can support 13 of these 14 hallmarks.
13 out of 14. That's a lot. That's crazy.
Yeah, that's crazy, and that's why they are so important.
Okay
Why it's so important to target them in cancer.
I get it. Macrophages in cancer, what's so special about CLEC? That's our target. That's what I know, but tell us about CLEC.
CLEC-1 is expressed on these very immunosuppressive macrophage populations that you can find in tumors, and they are called tumor-associated macrophages. These are the cells that can do many things to support cancers to grow. What we actually know about how cancer cells utilize macrophages is a biological normal system that we have in our bodies when women get pregnant. The placenta is filled with these CLEC-1 positive macrophages that suppress the immune reaction towards the fetus so that it can grow due to the genetic difference that the father's genes bring to this fetus, and this supports the fetus growth because CLEC-1 is suppressing the immunity against the fetus. This is a very interesting observation, that cancers can use this system to grow as well.
CLEC allows cancers to grow and metastasize.
Yeah.
What we have developed is an antibody against CLEC-1 named bexmarilimab. We call it BEX. Maija, how does BEX work?
BEX has multiple actions, and I'm first explaining number one, which is CLEC-1 is expressed on monocytes and macrophages. Here we have a monocyte in the picture. When bexmarilimab binds to CLEC-1 on the monocytes, it can actually activate the monocyte from an immunosuppressive monocyte to an immune activating monocyte. This increases the possibility to educate the adaptive immune system T cells, cytotoxic T cells, to attack cancer cells. This is the like main immunological function that we have.
Right
For bexmarilimab. As you can see from the slides, that number two is also there when we are in regards of myeloid hematological malignancies, for example, myelodysplastic syndrome, where these malignant blast cells also express CLEC-1. There, when bexmarilimab can bind to CLEC there, we can see an imbalance in metabolic functioning of these cells that then sensitizes them to standard of care treatments such as azacitidine, for example.
We're overcoming treatment resistance.
Exactly, yes.
Wonderful. You touched upon the point myeloid malignancies, for example, myelodysplastic syndrome. Why is high-risk MDS so important for us as a company?
It's important for us because it's a macrophage myeloid-driven disease, and because since we're targeting macrophages' myeloid cells that express CLEC, and in HR-MDS, CLEC is very abundantly expressed on these malignant blasts. Therefore, this is so the right target for us as an indication because this is the one where we get the best responses, because they're CLEC positive.
Okay, is CLEC-1 possibly the reason why these are so difficult to treat cancers?
This could be possible because it's super immunosuppressive, so you don't get any immune activating drugs to work if you're not suppressing CLEC as well. Because there is a very high unmet need with MDS due to the fact that there are no new treatments in over 15 years with this indication.
Thank you, Maija.
Thank you.
Next up here with me is our Chief Medical Officer gonna come and talk about what have we achieved in the treatment of high-risk MDS. Over to you, Dr. Bono.
Hello, everyone. Good afternoon. My name is Petri Bono, Chief Medical Officer at Faron. I'm a medical oncologist by my basic training. I've had a career about 25 years in clinical oncology trials first, early 2000 as a junior investigator up to global PI and been in approximately 60 different trials involved before joining Faron.
To high-risk MDS and what have we accomplished.
Yeah. Maija introduced the disease. High-risk MDS, so it's something where we haven't really got anything new since late 2000 or 2009 when azacitidine came to the market. Many attempts to increase the efficacy and to treat patients better, but it has been very difficult. This is a heterogeneous group of cancers and, for example, relapsed high-risk MDS, it resembles pancreatic cancer what comes to the overall survival rates. You can see down here, in relapsed or refractory R or RR MDS, survival estimate with salvage treatment or palliative care or supportive care, it's five-six months only, so really an aggressive tumor type. New treatments are needed.
In the upper part of the slide, you can see that first BEXMAB result. How does that look? First of all, BEXMAB with that we can get a 45% complete remission, which means disappearance of cancer from the patients, improvements or normalization of the blood values. When we compare this to the only existing standard treatment, the azacitidine alone, that provides 16%-18% CR rate. We are more than doubling the CR rates in the frontline setting.
Sorry to interrupt there, but could you explain to the audience how important a CR is for, especially for these patients?
It's very important. First from patient's point of view is that when you get a CR response, you think that I'm free from cancer, although they get treated, so mentally it's super important. We know that it's very well correlated with the overall survival of the patients, and not just the absolute CR rate, but also the duration of CR. Our duration of CR in the frontline is 12 months, which is a very good number in this aggressive patient population that we enroll to the frontline trial. These results are from the phase 2a BEXMAB trial. The phase I trial results were published a year ago in The Lancet Oncology that included MDS patient as well as AML patients. Today, we are focusing solely on MDS results as shown in the slide.
The lower part of the slide, that's the RR MDS population. We had 34 patients that were presented at the ASH meeting in December, and you can see there that the overall survival is 14.5 months there. With salvage therapy, it's just that five-six months. That's a really high number. It's 2.5 times longer than what you would expect. This is a combination of azacitidine plus BEX. Need to remember, the patient had failed AZA when they entered the trial, so by continuing the AZA, how come could one get benefit and responses still?
What kind of reactions have you heard from the field of MDS with these results?
Excuse me?
What kind of reactions have you heard from physicians or.
Physicians are super excited. It was a shock to the field that venetoclax phase III failed, especially after that. Everyone is saying that we are the hope for the MDS patients. This is what the leading physicians in the best U.S. sites, for example, tell, but of course also in the European hospitals. They say that we want to be part of the clinical trials. We want to treat our patients already at this stage with BEX-AZA combo because they've seen the result. Good safety profile, good efficacy, results, and they believe that this is to the benefit of the patients.
Yes, you touched upon safety there 'cause drug approval is only, it's not only about efficacy, it's also safety. Tell us about safety.
Yeah. Yeah, it's always a balance and this is the safety result of bexmarilimab + azacitidine shown in the dark blue bars and then azacitidine alone in the gray bars. As you can see in every parameter, so actually it's not just that we are not increasing the toxicity of the azacitidine treatment. Actually, what we're seeing is increased tolerability, and there's a good rationale also for that. We have shown that for example, hematopoietic progenitor cells, they get increased when patients are treated with bexmarilimab, and that's what we believe is the reason behind these improved safety results. What does it mean again for the patients?
Less neutropenia, less neutropenic fevers that are treated in the hospitals, less need for red blood cell transfusions, less need for growth factors to treat low leukocyte numbers, and less platelet infusions. Really clinically meaningful reductions in these endpoints. Finally the last bar there is probably one of the reasons sometimes combination trials fail is that it's the increased toxicity that leads to the reduction of the dose intensity in the other part of the regimen. Here we can see that when the patients were treated with BEX and AZA , there was a lot less dose reductions for the azacitidine. This is also another ultimate proof that this is a well-tolerated combination.
Yes, it looks pretty remarkable that the combination of BEX -AZA looks even better than AZA, but we'll see in the randomized trial to come. Talk about that.
Yeah. It's shown here. The frontline development plan per FDA's advice. On the left side, you can see the randomized phase IIb design. It's a randomized double-blinded placebo controlled trial to compare the combination of BEX 1 mg per kilo +AZA or BEX 3 +AZA or placebo +AZA , and in a double-blinded fashion. After 90 patients have been treated, 30 patients per arm, the data will be released and thereafter we're gonna go back to FDA to show the data and of course then to get formal approval for the dose for the phase III.
Knowing that we have the very good BEXMAB overall survival data, we have an upcoming City of Hope RR MDS new trial to be launched in the fall, and then having here the randomized data showing the contribution of BEX in the combo for the efficacy, so very likely we can also discuss with FDA the possibility for accelerated approval for RR MDS, but that's of course a matter of that meeting.
Of course.
After that one, the phase 3, the primary endpoint is something that is that FDA approves. It's CR rate and the duration of CR and the beauty of the upcoming phase III is that actually there's a possibility for resizing the trial size in case, for example, AZA alone comparator arm or AZA placebo would do better than one has anticipated in the power calculators. We can do some adaptation. Of course, we need to show that it's not detrimental, this treatment for patients, so follow survival and the survival follow-up, it's confirmatory in this one. The approval endpoint is the CR plus duration of CR.
Yes. Would you say this is a modern adaptive trial? That's what I've understood myself, that a lot of these past failures in the field have been very rigid standard classical phase III without this adaptivity along the way.
Yeah. The adaptivity is one of the key features for the upcoming registrational trial. Actually some of the earlier trials have been quite close to be positive also, and if they had had an adaptive design, they could have been also positive. They were with different agent and with different targets.
Okay. Tell us what else is happening.
A lot. Medical oncologist telling about solid tumor pipeline is shown here.
Don't take too long now.
I won't. Three trials, they are shown in the blue bars, BLAZE, BEXAR, FINPROVE. Metastatic BLAZE, metastatic melanoma, lung cancer, BEXAR, metastatic soft tissue sarcoma, FINPROVE, metastatic frontline, hormone receptor positive, breast cancer. They all gonna launch first patient in H1 this year, and first stage one results are expected, H2 2027. In addition to these three trials where we have actually contracts in place, they are waiting regulatory approvals. We have there in the slide also two other trials.
One is that City of Hope IIT in relapsed MDS together with an oral azacitidine, and then another one is by the Nordic AML Group that is after allo stem cell transplant patients, when they do have a molecular relapse, they are retreated not with azacitidine, that is the standard treatment there, but rather AZA + BEX to increase the molecular remission rates and to really have permanent cure still for these AML patients after their first relapse after the transplant. Very excited about these new trials, and we are moving forward with all these ones. For example, the one on the top, this phase 2b BEXMAB-02, the randomized trial. We have already the trial synopsis. Site selection has been started now in U.S. first, and then the trial full protocol.
It's close to be ready. I would say it's 90% ready at the moment. Nice progress, so that first patient in the fall.
Wonderful. There'll be a lot of stuff happening, a big news flow. I just have to ask you, so what are your favorites from this upcoming news flow?
Yeah. It's the BEX is about to.
Of course, something else.
Yeah.
The solid tumor guy.
Now of course, yeah, the solid ones. I think it's difficult. I kind of like BLAZE a lot because actually it was suggested by the PI at The Royal Marsden, Anna Minchom, who was a very active investigator in the MATINS trial where BEX was used as single agent for 216 solid tumor patients. We had their melanoma patients, 25 melanomas. It's kinda follow-up directly, and we know from Maija's work that that was published already some years ago in Clinical Cancer Research, BEX plus anti-PD-1 antibody is synergistic, very nicely synergistic in a mouse cancer model. We are now testing that in patients.
These patients have failed anti-PD-1 treatment when they entered the trial, and we know that macrophages, as you talked with Maija, they are part of the resistance mechanisms, probably the most important part. With BEX, we trying to overcome the resistance. How does it work? First shot of BEX a week before the combination starts. They are challenged first little bit with single agent Bex. Thereafter, the combo BEX plus PD-1.
We're priming the tumor microenvironment?
Exactly.
Wonderful. Love it. Thanks a lot, Petri. We could talk all day about this, but we have to move on. Next up to the stage is our Chief Business Officer, Ralph Hughes. Welcome, Ralph. Couple of words on yourself.
Hi, Juho. Thank you and very happy to be here. My name's Ralph Hughes. I'm the Chief Business Officer here at Faron Pharmaceuticals. In a former life I was with PharmaVentures, Pfizer, and Mundipharma. I'm here today to talk a little bit about the market landscape that is facing bexmarilimab and Faron. The global oncology market continues to grow at a very significant pace. This is being driven by underlying factors such as an aging population, increasing awareness, and increasing early diagnosis. It's also being driven by factors such as new products coming to the market. A really good example of this is the immune checkpoint inhibitors. They make up $60 billion worth of the market. Keytruda, one of these immune checkpoint inhibitor, makes up half of that.
It's a brilliant example of what can be achieved with a single drug over multiple indications. We've talked about MDS already.
Yes.
MDS is a market that's expected to grow significantly in the coming years as well. Again, it's a disease of older people, so the aging population has a disproportionate effect on this indication. We also see greater awareness of this indication, which is in part being driven by low-risk MDS, which is a space where there have been lots of new treatments. But that also raises awareness of the higher-risk indications. When people move beyond low-risk into higher-risk, we haven't had a new treatment for the past 20 years. When speaking to payers and KOLs about bexmarilimab in the higher-risk indication, we ask them, "Do you think that you're gonna see a lot of faster uptake? Do you think you're gonna see high pricing potential?" There is always a resounding yes.
The lack of treatments in this space make this an enormous commercial open goal for the drug and the company that's able to crack this indication. A target that a lot of people have been aiming to achieve for a long time.
You think we could take a big portion of that growing MDS market?
I won't say precisely the amount, but yes. I think the KOLs and the clinicians that we're speaking to are talking about very large percentage of that market for bexmarilimab if we're able to get approval. There just isn't anything out there, Juho. The market is crying out for a new drug in this space.
Well, that's music to ears of a CEO.
A CBO as well. Biopharma deal-making has had a very soft 2024 and for the first half of 2025 as well. Really low figures. Really hard work for BD people out there. However, in the last quarter of the last year, we saw a very significant uptick in deal making. This is driven by a few large deals of de-risked late-stage assets with randomized clinical data. This has primarily been driven by the fact that we've got an enormous patent cliff ahead of us. Something like $300 billion worth of value is gonna be taken off the market within the next 10 years.
That's a crazy amount.
It's a stupid amount of money.
Yeah.
These pharma companies are looking for mid- to late-stage assets, de-risked, randomized clinical data, that are able to plug that gap, and bring new products to the market in the future. We know, and this is nothing new here, but we know that big pharma are looking for later stage assets, and they are willing to pay a disproportionately high premium versus early stage assets. I say nothing new here, but it's never been truer than it was as a result of that patent cliff I just mentioned. Overall, immuno-oncology market is large and continues to grow. MDS is an area of higher unmet need, a commercial open goal that for the product that's able to crack it. Deal making is experiencing positive sentiment, kind of coming back from the dead a little bit.
However, drug companies are looking for de-risked late-stage clinical assets to fill in the gap that's gonna be left by that patent cliff. This proposed raise, this discussion we're having today, we are opening ourselves up to a much more significant deal potential than anything we've considered in the past.
Absolutely great. I've heard this threshold of what pharma is looking for from a product, what big pharma is. Annual sales of $2 billion-$3 billion. How does BEX fit that projection or what pharma is looking for?
Yeah. A historical number was EUR 1 billion. That's what was considered to be a blockbuster. These days it's more like EUR 2 or 3 billion as a result of inflationary pressure and various other things. In MDS alone, our projections are very close to that kind of number. With the addition of multiple other indications, we're well into that category of a modern blockbuster. Yeah, absolutely something to look forward to.
Wonderful. Thanks a lot, Ralph. Great having you. Thanks a lot. Next to the technical features of our rights offering and to the stage, our CFO, Jurriaan Dekkers. Welcome, Jurriaan.
Hi, Juho. Thanks for having me.
My pleasure.
Jurriaan Dekkers, CFO, started in December last year, so December 2025, replacing Yrjö Wichmann, who decided to retire. More than 25 years experience in finance. Worked as a CFO in healthcare and biotech. Worked at AstraZeneca, CFO Netherlands, CEO of Acerta Pharma, and the last couple of years, CFO of ProQR Therapeutics, a Dutch-based Nasdaq-listed biotech.
Take it away.
The rights offering, couple of highlights. The offering is up to 80 million offer shares, targeting to raise approximately EUR 40 million in gross proceeds. The good thing is that we already have received subscription commitments and cornerstone commitments for the amount of EUR 11.8 million. Together with the commitments from the core guarantors, EUR 28.3 million, we can say that this rights offering is 100% guaranteed and 100% committed. The total offering shares are approximately 67% of the existing shares in the company. The subscription price is set at EUR 0.50, which is a discount of 7.5% to the TERP based on the closing price on the ninth of March. The ratio is 13 to nine.
There's a lot of information on our website with calculation how that works, but maybe in short, if you have 1,300 Faron shares, you'll get 1,300 subscription rights, which entitles you to 900 offer shares. At a subscription price of EUR 0.50, you'll pay EUR 450, and then after the subscription you have 1,300+ 900 is 2,200 shares in Faron. It's important to understand for the existing shareholders, but of course also for new shareholders, that action is needed. There are a couple of things which you can do. You can use your subscription rights you have received and subscribe for the offer shares. You can also buy on the stock exchange additional subscription rights, but you can also sell a part or all of your subscription rights.
If you're a new investor, if you're not yet a shareholder, you can purchase subscription rights on the stock exchange. Also, if you wanna do a secondary subscription for the offer shares that have not been subscribed for, that is also of course possible. Important to understand as an existing shareholder, you need to take action. What are we going to do with the gross proceeds? Why are we doing the rights offering? It's important to understand that we needed to strengthen our financial position to extend our cash runway, to be able to continue the development of BEX, to drive it to the next set of milestones. Important, as Petri already alluded to, the 90-patient phase IIb trial with BEX in combination with AZA in frontline high-risk MDS, with the readout scheduled for November 2027.
A lot of other important value inflection points. We're going to support up to five IITs, five investigator-initiated trials, to validate BEX potential in combination trials. Those are all the reasons why we did the offering and where we are going to use the proceeds. This slide shows the key dates. There's a lot of information also on the website. Please make sure you're comfortable with all of these dates, but I'll just only highlight a couple. Today, 17th of March, is where the subscription period starts and trading of the subscription rights starts on the stock exchange. The second of April is when the subscription period ends, so it's important to take action before that date.
On the 9th of April, there's the announcement of the final results of the offering, and then the listing and trading of the offer shares starts on the 15th of April. There's a lot of information on our Faron corporate website. There's Q&As, there's materials, but there's also the prospectus. Please make sure you also have a look at the prospectus. It includes the process, it includes the timelines, but it also includes summary of the risks. It's important to understand that the prospectus is the official document approved by the Finnish Financial Supervisory Authority, and important to understand the impact of the rights offering. The prospectus includes all of the terms of the transactions. We have already talked about it in this webcast, but there's more information in the prospectus.
Please have a good look at the prospectus as well.
Thank you, Jurriaan. Very technical, but please, everybody, take your time, make yourself comfortable with these data and aspects, and calling for action. To sum up, again, what are we looking at here? With the recent developments in the field of high-risk MDS, Faron has become, I would say, the leading company in developing the next potential drug in this space. We have completed with outstanding results an open-label phase I/II, and now are moving on into a randomized, blinded phase IIb for regulatory and business purposes. We're raising EUR 40 million to do that, and then also supporting investigator-initiated trials in up to five different cancers to show that BEX is not only an MDS drug, but it has potential in a number of different cancers, which will support us in our business negotiations.
These are the highlights, and then we are moving on to Q&A, and I invite everybody back on the stage, please.
Thank you for the excellent presentation. Let's kick off. How are you planning to use the EUR 40 million, and what does it mean that this is fully committed or fully guaranteed, this transaction?
With the EUR 40 million, we're actually extending our cash runway, which was initially mid-April 2026. We've now extended it to November 2027, and with that, we can continue all of our clinical programs. As Petri already talked about, we will extend our clinical trial, the phase 2b trial with readout in November, but also support all of the IITs during that period. Your question about the commitments, the fact that the rights offering is fully committed means that we've actually secured EUR 40 million gross proceeds. If there are existing shareholders who do not subscribe for their offer shares, there are new investors, but also the guarantors who are then taking up these shares, and actually that's because they're extremely interested in Faron.
Thank you. What are the important milestones before November 2027? What are the most important milestones that investors should be focusing for?
I will hand that over to our Chief Medical Officer who will be delivering those milestones.
Thanks, Juho. Yeah. The, of course, very important is to get the phase 2b trial enrolling patients to get all the preparations ready. We are well on track with those. Then, of course, during the first half of 2027 to really make certain we are proceeding as planned so that we will have the actual results then in November 2027. The primary endpoint takes three months after the last patient has started the treatment. Why three months? It's because we know from the BEXMAB trial that it took 93 days on average to get the CR response.
Thank you. What is new data that you're planning to produce on the phase 2b randomized data? What's new compared to the data that we have produced so far?
It's very different. Now we get combination data that we can directly compare to placebo arm, meaning that all the efficacy benefit, so it's really showing the contribution of BEX in the regimen. On the other hand, also similarly in the safety. So, I showed that we have improved safety profile, so that's also possible to show in this randomized controlled placebo trial. This is a huge change also how the world is looking at the data. It's a true game changer, not just for patients, clinicians, but also for pharma and pharmacy interest.
Yeah. Maybe we touch upon that. How does pharma see a randomized dataset versus an open label dataset, Ralph?
It's completely different. I mean, I think what we've heard from a lot of pharma, a lot of the sort of partnering discussions and so on that we've been having, is that they want to see this contribution of components. They want to see the randomized data. It's the same thing essentially that the FDA want to see. For them, that's one of the key elements that's gonna make them want to move forward in some kind of partnership or decision around bexmarilimab. It's absolute game changer. You used completely the right word there. Definitely something that pharma want to see.
Well, does this mean that the partner discussions are somehow gonna change compared to what we've been having?
Partner.
Are there partner candidates gonna change?
Partnering discussions take
A long time. These aren't things that just happen, you get the data, and all of a sudden you've got a partnership. They can take years. It's developing relationships. It's also putting together those kind of relationships. Over the next few years, over the course of this trial, we'll be talking to all of the people that we've already been speaking to, of which there are. Well, it's basically everyone you can think of. We will continue those conversations. We'll be presenting the new plan. We'll be discussing the new data. We'll be priming them and getting them ready for the data release at the moment that it comes so that they are ready, interested, excited, paying attention.
That's really the role of business development and what we're gonna be doing over the next period.
Thank you, Ralph.
Thank you.
May I still comment?
Yeah, definitely.
One additional thing. Also regulatory agencies, so they think this, that, there's a big difference. What actually FDA has advised us is that, do a randomized trial to show the contribution of BEX in the regimen. They say that, the data that we have produced, it's really encouraging, looks really good. Please do also the randomized trial.
Yeah. You would say that's like the ultimate test for a drug?
Yeah. That's the ultimate test. Yeah.
Thank you. There is a lot of discussion about competition, like looking beyond current competitors like CD47, TIM-3. Is there something new coming on the market that are like these non-toxic agents?
Well, one to mention is the AbbVie drug venetoclax, which was a BCL-2 inhibitor. There's now a Chinese company developing a next-generation BCL-2 inhibitor that is entering phase III. That is, I would say, the biggest competitor at the moment. Other competitors are mainly in phase I, so. Having the recent failure of BCL-2 inhibitors in this space, at least physicians do not seem to be very excited about that, and there is toxicity issues with those drugs.
Thank you. What gives you the conviction today that bexmarilimab can become a new standard of care treatment in high-risk MDS? Why you are confident about that?
To me, it's the efficacy safety profile, the risk-benefit ratio, and of course, all the contacts coming from the field, from the leading conferences, cancer centers in U.S., basically telling that we want to join your development program. We want to treat our patients with your drug. Really highlighting that we are not just BCL-2, another BCL-2 inhibitor. We are first in class, completely novel mode of action. They are excited, and that's, of course, fantastic to know this also.
Well, let's also ask Maija, 'cause Maija sees actually patient samples under her eyes. What do you see, and why do you believe?
I also believe very strongly with this drug because what we see in our models that we use in the lab, cell models that recapitulate what we know about MDS and their MDS cells, we see those same changes if we treat them in the lab with bexmarilimab in the patient samples that we receive from the clinical trial. I'm super confident that the biology is right, the research hypothesis is right, and we believe in success due to these reasons.
Now it's down to a randomized placebo-controlled trial.
Yes
To prove it.
Thank you. Why did you choose the specific IIT indication, such as melanoma, soft tissue sarcoma, and so on?
It's a logical continuation what we learned in the first-in-man single-arm MATINS trial. We had 25 melanoma patients, for example, there. We saw clinical activity there. That's why melanoma has been chosen to be further investigated together with the standard of care that is the PD-1 inhibitor there. Good rationale to combine also with chemo. There's mouse tumor data showing that anti-CLEVER-1 antibody plus chemo is actually synergistic in those models, and this is then translated to both the BEXMAB, the sarcomas soft tissue sarcoma trial, as well as the FINPROVE that is the frontline metastatic breast cancer trial in ER-positive breast cancer patients.
It's really something that lab science from the lab from different labs have been combined, and that's why we've also chosen. Of course also what is a disease where there's a need for new treatments. For example, soft tissue sarcoma, it is one of the highest CLEVER expression in tumors, so there hasn't been a new drug approved. Doxorubicin has been for sure 30 years at least there, and if not 40. There's really a need, high unmet need for novel treatments.
Again, let's ask Maija, 'cause you've treated a lot of mouse models. What do you feel about the solid tumor trials that are starting or the indications?
Yeah, I think they're very important indications, in regard to what we know about the tumor microenvironment and how the bexmarilimab treatments can induce efficacy in those specific ones. We have used patient-derived explant models, MATINS trial samples to understand which patients benefit from the treatment. It seems that these tumor types that now have been selected for the IITs have actually those tumor microenvironments where we see bexmarilimab activity.
That makes me a believer.
Yeah.
What kind of results we would need to have that we can say that IIT is successful?
It depends on the tumor type. It's well known with the standard of care chemo that what kind of response rate you get in the frontline treatment in sarcoma or in breast cancer and then, for example, in melanoma. We are aiming to get 30% overall response rate in patients who have failed within three months their PD-1 treatment. One would expect, for sure less than 10% responses there. Even in the stage I, that the results we will have in our hands in H2 2027, if we have more than one response in the stage I, that's over 10%. That is in the statistical estimation that is required to move to the stage II in the trial. That, that's even enough in the first stage.
30% is something we're aiming at, for example, in that trial. There's a detailed statistical design for every single trial, but probably not worth going through them in this webcast.
Thank you. How confident are you that complete response is approvable endpoint for the FDA?
Over to you, Petri.
Yeah, that's FDA released in early July last year a guidance for industry for the MDS treatment development of those treatments. CR is there very nicely mentioned as one of the main endpoints, and not just the overall CR rate, but also the duration of CR. Of course, they want to see that it's not detrimental for the patients that you don't have an inferior overall survival. Really, the field is moving towards CR, duration of CR. If one goes to FDA's webpages where all the approval endpoints are listed, so also CR is there mentioned as an endpoint for MDS.
Thank you. There is one analyst question. How confident are you in the timelines that you can follow all of these timelines? How confident you are?
I'll maybe comment first that they're tight, but I'll be watching these guys that they deliver. Over to you, Petri.
This is, if we talk about, for example, the phase 2b randomized MDS trial, to have the results in hand in November means that we need to execute very effectively the trial. For that purpose, we need also a good CRO company to work with. It's part of the detailed trial planning so that we can trust the CRO. They work together with us, and I can tell that all these activities are ongoing already. As I mentioned, we are on track to really have the first patient in the fall 2026 to the trial.
Yeah. Thank you. One final question. There is a lot of positive comments about this presentation and that you all are on the stage. Are you planning to have a R&D day or capital markets day later this year? Final question.
Yes, we are, 'cause we'll introduce these trials in more detail. Hopefully we'll have even some physicians saying something about why they are doing them. Yes, definitely coming up late spring, early summer.
Thank you.
Thank you. Have a nice day, everyone. [crosstalk]
Thank you.