Faron Pharmaceuticals Oy (FARN) Earnings Call Transcript & Summary

Good afternoon, Europe. Good morning U.S.A. Welcome to the Faron Pharmaceuticals EHA 2026 webcast. My name is Juho Jalkanen, the Chief Executive Officer of Faron Pharmaceuticals. On to the next slide, please. Today, with great pleasure, we are happy to present what was presented over the weekend at European Hematology Association Annual Meeting in Stockholm, Sweden. Here presenting the actual data and in the Congress is Dr. Mika Kontro from Helsinki University Hospital, the principal investigator of the BEXMAB Phase I/II trial. Then after that, as many of you may know and are eagerly waiting for the next Phase IIb, our Chief Medical Officer, Dr. Petri Bono, will be presenting on how is the start-up of the trial progressing. Before we go more deeper into the data and upcoming next trial, I would like to lay out a higher level of big picture coming from EHA and the field of high-risk MDS. Next slide, please. As many of you may remember, ASH, end of '25, left the high-risk MDS field, I would say, devastated after the Phase III VERONA failure with venetoclax. It left a vacuum in late-stage programs. But what actually since then happened is a fleet of upcoming new companies, even new drugs, to the field of high-risk MDS, which we are delighted to see. With the recent developments, though, we are happy to be in a lead position going further. And especially what still differentiates us from the crowd is our novel mode of action, having the possibility to really induce hematopoiesis and make the bone marrow healthier for these high-risk MDS patients. A lot of these different programs we're seeing, unfortunately, they are reruns of BCL-2 inhibitors, CD47 inhibitors and kinase inhibitors. Though to mention and highlighting, nice to see also the immune modulation, immunotherapy pickup in this field, want to highlight anti-Galectin-9 from Gallop Oncology and PI3K gamma inhibitor from Stelexis Biosciences. Those are good to be looking for in the future when it comes to immune modulation in high-risk MDS. So this is the current state of the field and coming back on, I would say, maybe some depressed moves at ASH. Now at EHA, I think there is positive enthusiasm generating in the field and looking very much forward to these upcoming data readouts in the field that hopefully, there will be something new for the high-risk MDS population. So with these words, and enthusiasm for the future, let's go see the latest from the BEXMAB trial reported on EHA. Next slide, and over to you, Dr. Kontro.

Thank you, Juho. And can I have a first slide? So we're very pleased about the mode of bexmarilimab. Basically when monocytes and macrophages express CLEVER-1, it's kind of immunosuppressive molecule. And bexmarilimab is monoclonal antibody against CLEVER-1. When a CLEVER-1 is inhibited in monocytes, it leads to kind of enhance the immune activation. But we see the base on samples, we see enhanced expression of antigen-presenting molecules, activation of T cells and also activation of cytokines. And basically, these all make the monocytes more aggressive, and that may lead to better disease control. Actually CLEVER-1 is also expressed on the myeloblast cells, and there is emerging data that when we inhibit with bexmarilimab CLEVER-1 on the blast cells, it may have an effect on the mitochondrial function of these cells and basically enhance the cell killing. Next slide, please. This is actually data that we showed at ESMO last fall. We actually observed that when we have good CLEVER-1 target engagement, we have more complete responses. So meaning that this shows good relationship between target and engagement with bexmarilimab and response. And as mentioned, the more targeted [ engagement ], the better responses we have. Next slide, please. And this is the overview of Phase I/Phase II BEXMAB trial. The trial recruited both treatment nit, high-risk MDS patients. We had 20 patients or 21 patients and also the trial-recruited HMA failed high-risk MDS based on population. The trial was recruiting across 10 sites in U.S. U.K. and Finland. It was a combination of study with azacitidine, which is a hypomethylating agent. In the Phase I, we evaluated 3 different dose levels, 1, 3 and 6 milligrams per kilogram. And for a Phase II, we included patients with HMA failed high-risk MDS and explored 2 dose levels. I want to underline on the right-hand side that actually very many of these patients that were included, had a very high-risk disease as defined by IPSS-R and actually also TP53 mutations, which are commonly associated with very poor outcome, were highly prevalent in this patient population. And we're looking at treatment naive patient population, actually, 57% of the patients were very high IPSSM disease, which is currently more and more used for classification of the MDS. Next slide, please. This is the data from frontline patient population. In summary, we have observed a complete remission rate as per 2006 criteria to be 45% that overall response rate extremely good 85%. What we have already previously shown and the responses continue to be maintain it. With the aza plus bex combination, we have shown that there is high clearance of blast cells. We have also good response rate in TP53 mutated disease. And at this meeting, we also reported the duration of complete remission, and it's currently over 16 months, which is very encouraging in this patient population. Next slide, please. When looking at the data on HMA failed MDS patient population, overall response rate in this patient population is 64%. And we still see good responses and duration of response. Next slide, please. Then discussing briefly about the toxicity. In general, we can say that safety profile remains to be very promising. We have to remind ourselves that this patient population is rather fragile and elderly patient population and even though we haven't seen any Grade 5 bex-related AEs in this patient population. Next slide, please. Adding a bit more granular today when looking at the most common treatment-related adverse events, it's mainly related in hematopoietic toxicity, which is quite common anticipation population. And next slide, please. And then when looking at Grade 3 or higher adverse events, were basically a bit more serious adverse events; again, these are mainly based on hematopoietic toxicity. Febrile Neutropenia was observed in 32.7% of the patients. Perhaps it's good to remind you that actually this was not just the front-line patient population, but also the HMA failed patient population. In addition, the patients were not required to receive prophylactic antibiotic therapy as a study therapy. Next slide, please. Then looking at some of the very interesting data that we actually presented first time at EHA meeting, so this data comes from the patients that have been treated in the BEXMAB trial. And on the left-hand side, you can see the date of the hematopoietic status of the patient. So basically, what we were capable of showing is that when the patient received aza plus bex combination, the number of -- or the proposal of hematopoietic approach to ourselves is increasing, and this might be also relating to less transfusions and hematologic improvement. When looking at the right-hand side, we also observed that while the patients who are receiving aza plus bex mix, we actually had a higher number of cytotoxic CD8 T cells and quite interestingly, also a decrease of exhausted TIM-3-positive T cells. Next slide, please. And this data was also presented first time at EHA. So this is the patients that were evaluated at baseline and achieved response or not. And we observed that there was higher proposal of both CD8 and CD4 central memory T cells, which actually can then be differentiated using bexmarilimab plus azacitidine. And interestingly also, we observed less terminal effector cytotoxic T cells in the patients that did not achieve treatment response. So basically, this shows that the patients that achieved CR had a best capacity to activate their T cell responses while receiving aza plus bex.

Next slide. Thank you, Mika. Very nice biomarker data, really, I would say, capturing and highlighting the effect of the bex has on the immune system and activating the immune system and activating also hematopoiesis in this, again, very frail patients. Next to over to our CMO, Dr. Bono on where are we with starting the upcoming Phase IIb.

Thank you, Juho. Could I get the next slide, please? All right. [ Vegirasima ], the randomized placebo control double-blinded Phase IIb trial shown here. First of all, population, newly diagnosed MDS patients with morphologically confirmed higher-risk MDS and eligible for standard of care azacitidine treatment and not eligible at time of screening the stem cell transplant. And in the trial, patients will be randomized into 3 arms: Arm A, bex 1 mg per kilo plus azacytidine; Arm B, bex 3 mg per kilo plus azacitidine; and then finally, Arm C, placebo plus azacitidine. And patients will be treated with bex with weekly infusions. And patients who receive CR as their best response may proceed after 3 months to biweekly treatment or every other week infusion. And so that means why there is Q2, we, in some way, remember that usually bex is given every week in the BEXMAB trial. But there's a possibility to move over to every 2-week dosing. Primary analysis in the trial, that will happen at 3 months after last patient first. We see and all the results will be stratified according to [ IPSS-M ] risk category that will be used in the trial and also according to baseline bone marrow blast count percentage and TP53 mutational status. Next slide, please. And where are we going currently with the trial preparations? We have earlier announced that Parexel has been selected as a CRO and actively working together with them. And we are well underway with the trial preparations, and we are on track to really have the first patients enrolled to the trial already in early October. We -- protocol is ready, protocol's signed, informed consent forms are ready. Country selection and site feasibilities, they have been done. And first sites have already been qualified and are on track to be activated already in October. A study global PI, Professor [ Annette Seider ] from Yale will be the global PI. And then in EU, Mika Kontro will be the coordinating European PI and also Finland will be the reporting member state in EU. And from U.K., the coordinating investigator will be Dr. Austin Kulasekararaj from King's, London. Next slide, please. And here are the planned sites. If we start with the U.S. sites, the study will first be most likely enrolling first patient from U.S., although U.K. and now is also very fast in opening. We will have here familiar sites from BEXMAB such as Yale, MD Anderson, City of Hope in California. But in addition to that, University of Wisconsin, University of Colorado, Vanderbilt, Mass General for Boston, Harvard Hospital as well as Moffitt Cancer Center in Tampa, Florida. And in Europe, we will have countries, Finland, Spain, Italy, France and also Poland and Czech Republic. And not to forget U.K., there will be sites also 4 sites, probably in U.K. Altogether, the planned number of sites is 35. As a reminder, the study will have altogether 90 patients, 30 patients per arm from these 35 sites. And there's a nice geographical spread across Europe as well as across U.S. Next slide, please. And maybe then as a takeaway from the newest update of BEXMAB trial here at the end. So lots of interest in Mika's presentation, data nicely maturing towards better. With safety, we have -- earlier at ASH when we reported, we have in the front line, a bit longer than 9 months median follow-up of the patients. Now we had a bit more than 14 months median follow-up of the patients for 5 months more follow-up 5 months more time for safety evaluation. Safety profile remains actually numbers very similar to earlier which is good news. So the safety profile remains very good in this longer follow-up. Then the efficacy, strong response rates, strong CR rates. And very importantly, CR rate is 45% in the overall treatment-naive population, 70%, the TP53 mutant. And someone who may remember the ASH waterfall plots, so there were a couple of changes also in the decline in the blast count changes, a couple of deeper bars as well as a bit higher number of 100% decline in the blast numbers. And finally, but very importantly, regulatory agencies take into account that not just the CR rates, but also the duration of CR is important when considering approval of novel drugs for example, in the treatment of higher-risk MDS. So the duration of CR has increased from earlier 12 months and reported at ASH to 16.1 months, now reported by Mika Kontro at the EHA meeting, so about a 4-month improvement in the duration of CR. And importantly, as a summary, the Phase IIb BEXERA trial preparations, they are on track and at the EHA meeting, lots of interest and engagement from principal investigators from both EU and U.S. So we expect to have a rapid start for the trial. And I think this was my last slide, and I give back to Juho.

Yes. Thank you, Petri. And opening up to Q&A as our Investor Relations starts getting in those stores in. I have a couple to start with and now that I have Mika here, we were actually so busy at the [indiscernible] conference, didn't have proper time to discuss with Mika that how do you see now bex in this field compared to others and how much excitement? To me, it felt that this is the readout that everybody will be looking at in this field. How about you?

Yes, I tend to certainly agree. So first of all, I think that what is a really good signal about the upcoming BEXERA trial, is that basically all sites that were involved into earlier big trial are very eager to join. And I think that we have also, during the conference, had very good discussions that people are very interested in joining the trial. So -- and with the current results and with the current CR and overall response rate and also now with longer duration of survival as -- duration of response data, this is getting more and more interesting and very much looking forward to start also BEXERA here in Helsinki.

And as you said, more and more interesting have to do another question for you, Mika. What I hear a lot and what I heard at the Congress is with this response rate in TP53-mutated where usually response rate is very poor, should we focus solely on that? What's your answer?

Well, you perhaps know my answer already beforehand, but I think that it actually would be very feasible option also to do an conducted trial solely on TP53 mutated patient population. Due to the fact that there is no current available treatment options, all patients are receiving basically azacitidine -- azacitidine plus venetoclax, unfortunately, leads to responses, but the responses are quite short literally. So I certainly would be very keen to see the trial perhaps separate trial to be conducted in that patient population as well. We do know that for FDA bar for approval of a compound that improves survival and response rate in TP53-mutated MDS, perhaps also in AML would be highly valuable.

Thank you. Yes. I total agree. Maybe we will expand into that as our resources expand. And like my answer to that is we need further data before we just go do TP53 mutated, we will deliver this next Phase IIb. We will learn more. And if needed, we will focus and also -- and even possibly solely on TP53, that is the route. You learn always as you do and collect more data. Then I have one question to Petri before I let the audience go. You mentioned that there's been good engagement now putting together this trial, [ like I ] said, could you -- what's it been like getting all these sites? How much work has that been?

Yes. Of course, a lot of work, but yes, it's kind of nice that usual at least 4x more feasibilities are sent out and actually went up in the trial. For example, from U.S., so 100% of the size that have been approached, so they have been engaged and want to join our trial. Means that there's a lot of need for new treatments for the patients and the early experience from the existing site. So then what the new sites have heard from presentations at various meetings have been positive. So they are eager to jump as a trial site and be part of the development story. So very good response from various peers.

And correct me if I'm wrong, but I also get request, but are we -- is there already more that we can take long to the trial?

We need to limit the trial sites. So there would be clearly a lot more interest than that -- how many sites we can open. Need to remember that this is a 90-patient trial, so it is not very feasible to open a lot more sites.

Okay. Thank you, Petri. So let's open the floor to the audience and questions.

Thank you, Juho. The first question goes for Dr. Kontro. The median duration of complete remission is 16.1 months in frontline high-risk. For treating this population today, how meaningful is the 16.1 months compared to what's typically seen with azacitidine alone?

Yes. I think that's a great question. And what we have thus far been seen is that the responses have been, as mentioned, quite good. The duration of responses is also related to the fact that how many patients do respond. So for an example, azacitidine response rate is approximately 25% to 30%. And here for frontline patient population. We are looking at complete remission rate of 45%. So we have kind of better number of patients. For azacitidine, we are commonly -- of course, most of the data is a bit outdated, but we are looking perhaps similar numbers with regards of survival instead of complete remission rate. And if Petri actually could update on some of the latest data that you have been now going through regarding the duration of responses for aza.

So it is -- yes, if you ask me, Mika, that how I see the duration of CR, so yes, it is clearly sort what we have seen here. But of course, it depends always compared to early phase trials or randomized contract. But the 16 months compares very, very favorably to any studies with the CR duration report.

Thank you. Then the next question. What's the signal that this combination is doing something that the standard of care doesn't? What's the value that bex add?

Maybe I'll go first from a lean perspective, again, very scientific data presented at EHA and here in this webcast showing -- what is very important for these patients is that these, again, are very anemic, neutropenic patients and the backbone regime of aza adds to that. And what we're seeing from a safety profile and now in the cells, we're presenting here that we actually do not make neutropenia or anemia worse than aza would. It may even be that we make it even better and this happens through the production of these new red and white blood cells. But maybe a more sophisticated answer from Dr. Kontro.

Thanks, Juho. So I think that one perhaps low-hanging fruit in a sense, how to evaluate this is that what is the additional value of bexmarilimab is the Phase II or HMA failed patient population, which already has been receiving azacitidine and have progressed while receiving therapy. And then for this patient population, as I showed previously, we see treatment responses, we see quite good overall survival in this patient population. So perhaps this is also one patient population that illustrates additive value of bexmarilimab on the top of azacitidine.

Your view, Petri?

Yes. Maybe to add on that, Mika, very right that in that our MDS patient population is a good one to see the added benefit of bex. But I also like to highlight that the target engagement slide that Mika showed in the beginning, so that -- we have a very nice relationship. The higher target engagement, the higher response so the CR patients got the highest target engagement. And I think that's probably one of the best pieces of data in addition to that RR-MDS patient population what we can get without a randomized trial. Of course, the upcoming randomized trial will then give like the definitive answer to the contributes under the magnitude of contribution. But yes, I personally like the target engagement side really in addition to the translational results that Mika showed the increase in T-cell infiltration and antigen presentation, of course, also.

Yes, absolutely. Just like to comment for the benefit of the audience. In other words, the more CLEVER you block, the better response you get. Very nice to see.

Safety in an old and high-risk population is always a concern. How would you describe the tolerability so far?

This, I'll hand over to our practicing physician. Over to you, Dr. Kontro.

What we have actually been discussing also previously is that that, as you mentioned, the patient population is rather fragile, very prone to the toxicities. And basically, toxicity profile is very similar to azacitidine therapy only. Of course, when interpreting this data, we have to remind ourselves that this is now from all patient population, all [ 55 ] patients, including de novo patients that haven't been treated before and also patients with HMA failed disease. But also given the fact that what we have been discussing with the -- in the investigators meetings, it really seems that as mentioned, also the gut feeling is that, that the combination is quite well dolerated. And this was also illustrated by the fact that we haven't had any bexmarilimab-related deaths in the trial. So as mentioned, rather well-tolerated therapy option for these patients.

How do you plan to demonstrate the benefit of bex regarding the duration of complete responses and overall survival if patients who undergo bone marrow transplant are censored from the follow-up?

Dr. Bono?

Yes, this is taken into account in the trial design. And actually, it follows exactly new guidance that FDA has provided for industry in the treatment of MDS. So patients who are transferred -- so who get a good response for the treatment to transfer to stem cell transplant will not be censored. And this is how this is handled, and that's taken into account in the trial design. And there's a lot of variation how these patients have been reported earlier in various trials, but now there's a clear FDA guidance that how we should do that. And we are following exactly that guidance.

Thank you. You have described that bexmarilimab is not just reducing cancer cells, but helping the body to produce healthy cells again. How important is this dual mode of action?

Maybe I go first to me, that is actually our, I would say, most important differentiator from other more cytotoxic treatments. And for this population, this is an extremely valuable aspect of the drug. How about you, Dr. Kontro?

Yes, I agree. And really also looking very much for the randomized data on the topic so that actually how much we achieve with combination of bex to the azacitidine, do we have good number of reduced red blood cells and platelet infusions. So I think that will be very crucial for this, again, frail patient population. So if we have that possibility that we need to transfuse patients less it's a huge benefit for the patients and huge benefit also for quality of the life of these patients.

And then the final last question. What are the key milestones between now and end of this year? And what should investors watch for this coming year?

Petri, over to you.

The key milestones are, of course, the launch of the new BEXERA trial, approval from regulatory agencies, submissions in time and then first patient in time and really fast ramp-up of the patient enrollment related to the BEXERA. Another milestone for the -- at ASH meeting, a new updated efficacy will be for the first time told, for example, time-to-event endpoints in the frontline patient population. We plan to submit an abstract there and then to do a November data cut before the ASH meeting. And then, of course, not to forget solid tumor trials. And so we expect that there will be first places also enrolled in those trials actually quite soon.

Thank you, Petri. And then back to you, Juho.

Okay. Thank you, everybody. That is a wrap. I would say, super exciting times ahead for bex, both in high-risk MDS and solid tumors. But definitely for the high-risk MDS field, very happy to see a lot of new attempts entering this field, more immunomodulating therapies into this field. I think they're highly needed and exciting times ahead for everybody. Thank you all, and have a wonderful day.