Welcome to Mendus Q4 report 2025 presentation. During the Q&A session, participants are able to ask questions by dialing pound key five on their telephone keypad. Now I will hand the conference over to the speakers: CEO Erik Manting and CFO Lotta Ferm, please go ahead.
Thank you everybody for joining. Welcome to the Q4 finance and business update of Mendus on behalf of myself and our CFO Lotta Ferm.
Hi everyone.
Let's start with a summary of Q4. We presented continued positive data of our phase II trial, the ADVANCE II trial, in MRD-positive AML at ASH, the American Society of Hematology Conference, end of last year. The updated clinical development strategy in acute myeloid leukemia and AML aligns with the evolution of first-line treatment, and we will also say a few words about how that first-line treatment is shaping up, including recent updates that took place at the ASH conference last December. Our clinical trial strategy is established to not only position vididencel and AML but also as an active immunotherapy in chronic myeloid leukemia, or CML. We have established together with our manufacturing partner NorthX Biologics a large-scale GMP production, which will support our late-stage clinical development and, in the end, also registration and commercial launch of the product.
It was a very significant milestone that we accomplished in Q4, to make sure that we have a good balance between the costs of the company, and the additional trials that we anticipate to start in 2026. We have completed a reorganization, which included the reduction of staff, including the reduction of the management team, and we financed the company through a directed issue with significant insider support, from members of the board and myself and, of course, our larger shareholders, combined with a SEK 50 million loan facility, that we closed with Fenja, on which Lotta will comment, it when we do the financial update.
The long-term survival data of the ALISON trial in ovarian cancer, which we presented, first at ASCO mid last year but then also updated two-year survival, in Q4, confirmed the safety and feasibility of vididencel as a potential combination therapy in high-risk ovarian cancer. Let me move to the financial slide and let Lotta summarize the financial situation.
Yep. Hi everyone. As you can see in the report, the net loss for the period for Q4 was SEK 38.7 million for the operational loss, but the cash burn for the period was SEK 20.4 million for the period, and the difference is, of course, mainly related to the tech transfer to NorthX Biologics, which we have prepaid in 2023. So therefore there is just effect on the cost but not on the cash flow. The cash position at the end of the quarter and end of the year was SEK 64.7 million, and that's due to that we successfully raised SEK 52.5 million in a directed issue, as Erik mentioned before, and it was supported by the main shareholders, by Erik and board members.
We also secured a loan facility with Fenja Capital of in total SEK 50 million, and the reason for entering into this agreement was because we wanted to minimize the dilution for the shareholders if we had raised more capital in the directed and then there has been a higher dilution. The cash runway with this injection of cash will cover the full 2020s. It will take us into Q1 in 2027 and if we use the additional loans, where we have used the first tranche of SEK 30 million in January. That's everything from me.
Thanks. Let me move to the data we presented at ASH. You are familiar with this picture. We have, of course, provided continued updates on the long-term survival of the patients that participated in the ADVANCE II phase II trial. These were all patients that had undergone high-intensity chemotherapy after being diagnosed with acute myeloid leukemia and were diagnosed with measurable residual disease, which is a high-risk factor in predicting, let's say, the probability of relapse and, in the end, also overall survival. The positive news is that the patients are still doing very well. The patients in long-term follow-up continue to do well. Eight patients have now passed five-year survival, and we have not reached median overall or relapse-free survival.
If we look at the projected five-year survival of 63%, that compares to historical controls, including, for example, the registration trial with oral azacitidine, which is now an approved drug in this setting in acute myeloid leukemia, of less than 30%. So this not only, let's say, in terms of numbers but also particularly what you call a plateau so that you see that you really have a patient population that is stable over a longer period of time is indicative of the active immunotherapy effect, that we, of course, aim to introduce to the benefit of patients. And the data also combined with the general very benign safety profile of the product will allow us to establish a very broad positioning of vididencel as a post-remission therapy across different AML subtypes. So last remark about immunotherapy versus targeted therapies, which are typically directed towards specific mutations.
Both in the phase I trial and in the phase II trial, we have seen no association between the outcome of the treatment and specific mutations, which means that also we can position this product independent of particular mutations in AML, and that makes it next to its safety profile highly competitive in this indication. Now, what was the update at ASH that was relevant in the evolution of first-line treatment in AML? The patients that is are representing roughly half of the patient population in AML, which are either elderly patients above 75 years of age or that have other risk factors that make them a patient population which is described as unfit for intensive chemotherapy until recently had very poor prognosis with also a very poor probability of even accomplishing complete remissions.
That has changed dramatically with the development of a new drug called venetoclax, which is in clinical practice combined with injectable azacitidine, and that combination is called VenAza. Sometimes also said the other way around, Azaven, but it's always the combination of oral venetoclax combined with injectable azacitidine. What was interesting is that in the paradigm trial which was presented at ASH, this is a trial, a large trial, with fit patients, so patients that in principle could undergo high-intensity chemotherapy, but here they were randomized to either high-intensity chemotherapy or VenAza. The outcome of that trial, and this is a phase II trial, was that the fit patients actually benefited from being treated with VenAza versus high-intensity chemotherapy.
There was certainly, in the first year, a significant difference between event-free survival that was better in the VenAza-treated group as compared to the high-intensity chemotherapy, and also there was less severe side effects, so less hospitalizations, less ICU usage, less infections. So this means that there will be a continued drive to position venetoclax as a first-line treatment in AML beyond the patient population that was traditionally classified as unfit. And of course, it's important for us as a post-remission treatment that we anticipate the evolution of the field, including the broader use of venetoclax driven by its clinical success. So what we are currently doing is the ADVANCE II trial was a single-agent trial, and we had already started the CADENCE phase IIb trial, which is a randomized trial in which we combine vididencel with oral azacitidine following high-intensity chemotherapy.
But we will now also engage in the DIVA phase Ib trial, which we are preparing, with Andrew Wei, who is also the principal investigator of the CADENCE trial. Professor Wei has been very influential, particularly also in the development of venetoclax. And what we will do is, in the phase Ib trial, combine vididencel with venetoclax and azacitidine as a first-line treatment, and this will allow us to much more broadly position vididencel as a post-remission therapy in AML. Then chronic myeloid leukemia is an indication that we had considered over a longer period of time, but the development of our clinical development strategy accelerated significantly when Tarek Mughal joined us, as Chief Medical Officer mid last year in 2025.
Tarek has a long background in chronic myeloid leukemia, and he has helped us basically put in place the strategy which will allow us to test vididencel as an immunotherapy in CML. Now, what's the relevance of this strategy in CML? Chronic myeloid leukemia, as the name says, is a chronic indication. In the past it was, like AML, treated with hematopoietic stem cell transplants or bone marrow transplants, but in the past decades after the discovery of so-called tyrosine kinase inhibitors that are suppressing the BCR-ABL oncogene, which is a specific driver mutation of CML, the disease is more or less under control as long as patients keep on taking the TKIs. So we are seeing a fast-growing patient population.
Because of the chronic nature of the disease, we are now already talking about roughly 300,000 patients in Europe and the U.S. alone, and all these patients have to take TKIs on a daily basis. On the one hand, the overall survival of CML patients is now close to that of the general population, but on the other hand, and you can imagine, it's a huge burden they have to take their tyrosine kinase inhibitors treatment for the rest of their lives. So from a immediate disease control question, the treatment of CML has shifted to a quality of life question, and of course, with all the costs associated with treatment, also a significant reduction of costs if we would be able to allow more patients to stop their TKI treatment.
So this new barrier in the treatment of CML is called treatment-free remission, or TFR, and it has become a key therapy goal. The problem is, as soon as you stop treating patients with TKIs, the disease comes back, and that's called a molecular relapse, and it's also called a TFR failure. So patients have to then be put on either their original TKI or a next-generation TKI. There has been an evolution in the development of TKIs, and that continues. The next-generation TKIs is already being developed. There's large companies, first of all Novartis, but then also in the U.S. companies like Terns and Enliven that are developing more effective and more specific TKIs.
But the next wave of innovation will come from compounds that will allow patients to have a more successful TFR attempt, and this is where we believe immunotherapy can play a significant role. If the immune system is able to control the residual disease, the probability of a successful TFR attempt will be higher. Now, this hypothesis we will first test in the VITAL-CML phase I trial, which we are currently preparing, and we hope to start this trial in the second quarter of this year subject to timely regulatory approval of the trial. We have filed for all the regulatory approvals, but of course now we have to wait for that approval to start the trial.
The recruitment in CML we expect will be a lot quicker than in AML because of the larger number of patients and also the patients already having been prescreened to take part in this trial. So in that sense, we can also quite good predict that in a good way we will have initial data from the phase I trial confirming the safety of vididencel in this indication, and then in parallel we will start a phase II trial, which is called the VITAL-TFR trial. And that trial will specifically focus on patients that will attempt to achieve treatment-free remission, but they will do that after they had already failed an earlier TFR attempt.
Now, this patient population has a relatively high probability of around 75% to have, already in the first year, a TFR failure, so in this patient population we hope to see, in the quickest possible way an initial effect of the treatment of vididencel. So with these two trials, we have set out a strategy to enter CML, as a very large and, and also, of course, from a patient's perspective, a high medical need indication, and we hope, assuming that there will be a good safety readout, initial safety readout of the phase I trial to start both of these trials, this year in 2026. So with that, I would like to, summarize where we currently stand. We have the data from the ADVANCE II trial to support a broader positioning of, vididencel in AML.
So we have now already engaged in the CADENCE trial, and we will engage in the course of this year in the DIVA trial, which will combine vididencel with azacitidine and venetoclax, that combined with, of course, keeping a close eye on how first-line treatment of AML will evolve, if we will continue to see separate patient populations which are classified as fit and unfit, or whether we will start to see a larger shift where the decision between high-intensity chemotherapy and venetoclax will become more granular. That is something we keep a close eye on, and those developments combined with our own data from the trials that we have now set out will inform us, of course, about the best possible registration trial for vididencel in AML.
And then we are very excited, of course, to enter CML as a new field, again starting with the VITAL-CML phase I trial and then in parallel, after the initial safety data have been confirmed, the start of the VITAL-TFR phase II trial. And then to conclude the ALISON trial, we are very happy with the data we have collected in the trial that confirms safety feasibility of vididencel in this indication, including the induction of tumor-directed immune responses that were associated with durable clinical responses. We do believe that ovarian cancer, related to the nature of the disease, which is a solid tumor with a very demanding intratumoral environment, will probably need a combination with other drugs that will make the combination more effective in treating this disease.
So we see the ALISON trial currently as a good basis for potential partnering and developing combination therapies in this indication. With that, I would like to conclude. So the positive ADVANCE II phase II data supports a broad positioning of vididencel as a post-remission therapy in AML, so I think the course in this direction that we, of course, implemented with the updated clinical strategy we put in place last year was supported also by the data that were presented at ASH about the evolving first-line treatment landscape in AML. So I think we now have an up-to-date and attractive development strategy in AML, which is basically positioning vididencel as broadly as possible as a post-remission treatment.
We are very excited to add CML as a new indication, which is a very large indication, but also, of course, with a good clinical rationale, to use vididencel as an active immunotherapy to allow patients to have a better chance of treatment-free remission. And for the ALISON trial data, we are happy that we have them currently at hand as the basis for potential combination therapy and potential collaboration. So with that, we have an exciting outlook for this year with multiple clinical milestones anticipated in 2026, and particularly, of course, the first-in-human data in chronic myeloid leukemia, which we expect in the second half of this year. With that, I would like to open up the session for questions.
If you wish to ask a question, please dial pound key 5 on your telephone keypad to enter the queue.
If you wish to withdraw your question, please dial pound key 6 on your telephone keypad. The next question comes from Jyoti Prakash from Edison Group. Please go ahead.
Hi, good afternoon, and thank you for the presentation. I'm calling for Aaron Atker today. My first question relates to the phase Ib DIVA trial. Is it fair to assume that ALLG will act as a primary sponsor for the study? And, given that you're planning the study to start in mid-2026, are we still on track for interim readouts in the second half of the year? And if yes, what kind of recruitment pace and dosing schedule is built in to meet this timeline?
Sure. Thanks, Jyoti, for your question. So related to the DIVA trial, it will be an investigator-sponsored trial.
As you know, we work with Professor Andrew Wei in both the CADENCE trial and the DIVA trial, and it's a very efficient way to explore the broader positioning of the product in AML. And of course, when you work with people like him and the whole infrastructure around it, it's perfectly fine to do it as an investigator-sponsored trial. We are currently working with another CRO to support that trial, but that's just, let's say, a practical element of execution. The most important part is that the collaboration with Professor Andrew Wei allows us to explore the broader positioning in the AML landscape in a very cost-efficient way.
As you know, we've also set up a daughter company called Mendus Australia, that will allow us also to benefit from some of the tax benefits that the Australian government has in place to support clinical trials that take place in Australia. So I think this is a very efficient way to establish vididencel as a broader post-remission treatment in the AML landscape. And of course, we're very happy, you know, to work with one of the best people and an acknowledged global KOL in both of these trials. With respect to the preparations, they are on track. With respect to recruitment, we do expect to recruit around two patients per month in this trial, which is relatively quicker as compared to the recruitment we've seen in the ADVANCE II trial and the CADENCE trial.
The reason is that venetoclax and azacitidine are simply used more and more, so that it's relatively more predictable with respect to the recruitment rate in this setting versus the post-chemotherapy setting. So this answers my questions with respect to the DIVA trial, Jyoti. I'm not sure if you have any other questions.
Yeah, just another question from my side, and this relates to the CML studies. If you can outline the plan design, scale, and endpoints for the two studies, and what could be the expected clinical trial-related costs for both the trials.
Sure. So in CML, like in AML, actually, with the ADVANCE II trial, that was a company-sponsored trial. So also in CML, the phase I trial, the VITAL-CML trial, will be a company-sponsored trial.
We will run that trial together with Professor Bjørn Gjertsen in Bergen, Norway, who was also very active, contributed to the ADVANCE II trial, actually, so he's already quite experienced with the product in the AML setting. For those of you interested, by the way, there's also a number of interviews with patients in AML and CML, including a patient that Professor Gjertsen treated as part of the ADVANCE II trial. So we're very happy with that collaboration. What we will do in the VITAL-CML trial, Jyoti, is that we will first focus on patients that have what you call a suboptimal response to TKIs.
So for patients to even be eligible to try and stop their TKIs, they have to have what's called a deep molecular response, and quite a significant portion of patients don't reach that very deep levels of response of the disease to the TKI treatment, and they will never be able to attempt a treatment-free remission. But also that patient population will benefit maybe very strongly from an alternative modality to suppress the disease. So what we will first look for is safety and tolerability of the product, but also what we expect to see is, over time, what you call early molecular responses, because CML can be followed very specifically based on the BCR-ABL oncogene.
You can quite quickly see whether the therapy has an effect on the levels of disease, and that's why this phase I trial is not only very relevant for the initial safety data, but it's also very relevant in terms of what we may see as an effect of vididencel as an immunotherapy on the disease levels. Now, as soon as we have established the safety signal in the first eight patients, we are ready to start the phase II trial, and this will be a trial with Professor Tim Hughes in Australia. The TFR II setting I just explained when we were going over the CML slide is a different one. Here, patients have accomplished a very deep complete remission, and they will try for a second time after having this deep molecular remission over multiple years to stop their TKI treatment.
Now, in a first TFR attempt, the failure rate is roughly 50%. In a second TFR attempt, the failure rate is a lot higher, like I said, close to 75%. So if we start to see improvement of the outcomes in the TFR II trial, we know we can apply the product maybe also in the first-line TFR setting and, and, and basically from both trials have an initial proof of concept of the use of vididencel in CML. The phase I trial will be with 24 patients. We expect roughly also there a recruitment of about a recruitment time of about a year, but it may be more with bigger groups of patients at the same time because with AML, you're treating newly diagnosed patients, and with CML, you're basically treating patients that have already been screened prior to entering the trial.
The phase II trial will be with 36 patients, and it will take longer because the readout of a TFR signal takes longer.
Thank you. That's, that's very helpful. No further questions from me. Thank you again.
Thank you, Jyoti.
The next question comes from Qian Li from Pareto. Please go ahead.
Hi, good afternoon, and thanks for the update. Just two quick questions from me. So regarding the, the CADENCE trial, obviously it's investigator-led, but can you provide more flavors on the current pace, on enrollment, and what factors are driving the, the timeline? Second question is, regarding the, the future financing strategy. Mm-hmm. Would you consider other non-dilutive functions like partnership or and under what kind of, milestones would be a good execution point? Thank you.
Thanks, Qian.
Yeah, so with respect to the CADENCE trial, we don't provide, let's say, quarter-to-quarter updates, but the recruitment is continuing as planned. We are currently at 15 patients in the trial. We hope to accomplish 20 patients in the first half of this year, 2026, which will also allow us to do an initial readout in the first 10 patients of the treatment arm. I must say that the recruitment rate goes up and down from month to month. Sometimes that has to do with, strangely enough, seasonal effects, but you know, sometimes patients are just less willing to enter into trials. We had a good boost of patients coming in end of last year, and now we are starting to see the first patients coming back into the trial.
There is competition always in AML, and we also see in first-line treatment of AML new drugs coming in, including menin inhibitors. So, you know, you can never be sure that your recruitment goes fully as planned, but so far we are on track, and again, with that initial readout anticipated based on the first 20 patients into the trial. With respect to the future financing strategy, yeah, it's clear that what we try to accomplish and what Lotta also emphasized is in the last round to avoid too much dilution, and stay close to the share price and combine the equity financing with the loan facility that we have negotiated with Fenja Capital.
You can never be sure, of course, when you can do a partnering deal, but when we look at the way we have adjusted the AML strategy, we have listened closely to the feedback from the pharma companies that we are in touch with on a regular basis. And basically, they did not like the very narrow positioning we had originally proposed in the phase III trial that was based on the design of the ADVANCE II trial, which was an MRD-positive patients only. So now we have adjusted the strategy, and of course, also with the data that were presented at ASH, it's becoming more and more clear that, you know, you have to take venetoclax and azacitidine into account, as a major factor in the shift in first-line treatment of AML. So I think with the new strategy, we are on track to establish this broader positioning.
When it's enough, you never know, but at least we are now on track to deliver the data that vididencel can be combined with basically any first-line treatment, and that will help our partnering discussions. With CML, of course, we need to always be conservative. We first have to have the initial first-in-human data, but CML is a field that is really heating up, on the one hand because there's new classes of TKIs being discovered. Novartis is now the leading party with a drug called Scemblix or asciminib as a generic name, in that area, but there's also competition from the companies that I just mentioned, including Terns and Enliven. And so there's a lot of innovation currently going on in the TKIs, and that also, of course, generates a lot of strategic interest and also investor interest, in that space.
Next will be the treatment-free remission paradigm. And I think if we can show in the course of this year that vididencel is an immunotherapy modality that can deliver that support of patients to achieve more successful TFR attempts, that could be a very big shift in the broader positioning of the product in the myeloid blood cancer space. Of course, we first have to make sure we get the green light to start the phase I and then deliver the data to start also the phase II, in the course of this year.
Okay. Thank you very much, Erik. No further questions.
Thanks for joining, Qian.
There are no more phone questions at this time, so I hand the conference back to the speakers for any written questions and closing comments. Thank you.
Yeah, Richard Ramanius from Redeye was unable to join, but he sent in a few written questions. His main questions were about the approval routes both in CML and AML. In AML, like I said, we first need to collect the data in the broader patient populations, being post-chemo and post-stem cell. Then, of course, we need to see how the first-line treatment landscape shapes up. We expect to be in a position to make that assessment in the course of 2027, and that will also guide our registration trial strategy in AML.
Of course, we have done everything to prepare for that, including the large-scale manufacturing of the product, but I think it was a tactical correct decision that we took last year to first focus on the broadening of the positioning of the product rather than try to get it registered on the basis of the post-chemo setting and specifically for MRD-positive patients only. So that is one part of the answer. I can't say too much currently about the registration trial strategy in CML because it's so early days, but what will help in CML is that the historical basis, including the data that are quite predictive of outcome, are all there. So it will be relatively more straightforward as compared to AML to pick up initial signals of efficacy. But of course, it's a different setting.
You can't design a trial in CML based on overall survival, for example. You have to design different trials that actually look at the molecular levels of the disease and, in our specific case, of course, the improved TFR success rate. So we are shaping up this strategy, but I won't go as far as to already predict what the registration trial strategy would look like, in CML. Then the last questions from Rick are related to how much of the AML market we would capture with the positioning of vididencel and also the specific relation between vididencel and hematopoietic stem cell transplants. To put it simply, with venetoclax, azacitidine, and intensive chemotherapy as part of our first-line treatment after which we position vididencel as a post-remission therapy, we basically cover the broader patient population in AML.
Of course, transplants are still the only curative approach in AML, and I don't expect, and we also currently don't have, as a primary objective, to compete with transplants. Actually, what we have seen is that a number of the patients that were treated in the ADVANCE II trial with vididencel later on in the trial were transplanted and also transplanted successfully. So, the potential of vididencel to initially treat patients that are not eligible or not able to find a transplant after post-remission has been accomplished, I think, will cover the broader AML landscape, but whether or not it also makes a transplant no longer necessary will always be, at least in the near future, doctors' choice.
So we're not directly competing with transplant, but we do expect to capture the broader AML landscape, treatment landscape, and also actually that the product will make transplants prep potentially more positive initially we're not able to undergo a transplant. So it would be more a synergistic interaction than a competing interaction. So that is, I think, the way we currently look at the positioning of the product. Of course, when the success of vididencel is more established in the end also registration trial, there could be a shift towards this kind of immunotherapy, which is a lot more safer as compared to hematopoietic stem cell transplant. But at this point in time, that's not part of our strategy. I think that's it for this call.
I haven't seen any other written questions, so thank you, everybody, for joining, and we're looking forward to keeping you updated of the progress of the company this year.
Okay. Thank you.