Welcome everybody, people in the room, people joining us via the web broadcast, to Immunicum's pipeline and strategy outlook. It has been a special year and, I'd like to thank the investors, but also other interested people, our colleagues, for staying with us in a year that was quite challenging for biotech, for the capital markets. We went through a merger process as a company at the time that COVID was still bringing a lot of restrictions in travel and other ways to communicate with our colleagues. Plus we went through a strategic review of our portfolio.
It was quite a busy year, but we believe with a very good outcome, and one that we want to share with you today with a quick review over the past year, but certainly also the outlook for 2022 and beyond. I'm joined today by my colleagues from the management team, Jeroen Rovers , our Chief Medical Officer, Alex, our Chief Scientific Officer, and Lotte, our Chief Financial Officer. I will do a presentation of roughly half an hour with respect to the review and the outlook for the company's pipeline and strategy. Then we will have a Q&A session based on questions that were sent in and also some of the questions that the room may have or that may come in during the event.
Next to the management team, Immunicum is supported by a highly professional board that keeps us sharp and focused. Together with them, we have set out the strategy for the company going forward. As a short agenda, a review of 2021, the clinical pipeline update with also context on why we have taken certain decisions, and then an outlook of 2022 and moving forward. Today, Immunicum is a company that is addressing the two major challenges in the cancer therapy landscape, which is tumor recurrence and hard to treat tumors that are still poorly treated with currently available therapies, including immunotherapies. We believe we have a competitive pipeline of products based on unique expertise that we've built up, first of all, as separate companies, Immunicum, DCprime, but certainly now that we've brought both companies together with a critical mass of expertise.
We have a program in AML that is starting to shape up in a very promising way. There is a big need for maintenance therapies in tumors like AML, which are quite aggressive and lead to rapid recurrence. We believe that we now have data at hand, which we presented end of last year, that provide for a promising basis moving forward. This product is so-called off the shelf, so we make it so that it's readily available for hospitals to use and is based on a proprietary cell line. That's important for two reasons. First of all, we own the cell line, so it's this important starting material we use each time for our manufacturing process. A cell line allows for scalable manufacturing, which is very different from, for example, patient material as a starting material.
We have additional programs in solid tumors. We have a program in ovarian cancer, and we have decided to position ilixadencel, which is an intratumoral primer in gastrointestinal stromal tumors. I will give a bit more background, of course, on why we've made those choices and what you can expect from those clinical programs moving forward. Also, very importantly, we have set up a strong in-house R&D organization. Of course, we are now strengthened with Alex, who is the original founder of Immunicum. He lives in Leiden, he heads the research group. That is important both from, let's say, a fundamental scientific perspective, but certainly also to develop process development expertise, which leads to better manufacturing processes for our lead programs. Finally, of course, we have our headquarters here in Stockholm, and we are listed on Nasdaq Stockholm.
Last year, we were reaping the benefits from the merger with DCprime. First of all, the product that DCprime brought into Immunicum allows us to address tumor recurrence. Tumor recurrence is the most dominant feature actually determining cancer therapy today, and it is one that you can only address with a product that acts as a vaccine, and it carries its own antigens, right? Il ixadencel, as you may remember, which was the product Immunicum has originally started with, is something that uses established tumors, which is also an important therapeutic angle, but you cannot use that product to vaccinate patients in order to prevent tumor recurrence. That's a specific angle, technical angle, I would say, in the cancer therapy landscape that the DCprime merger brought into the company. Then we benefit from an actual lab.
What you see here is a picture of the new headquarters, which are here in the Matchstick Palace in Stockholm, but also the new facilities that we are moving into as we speak in Leiden, which will allow us to further build out our research expertise and process development expertise. We are by now an integrated team. The merger was factually done end of the first quarter. Of course, it really started when people started working together and also had a chance to travel and meet with each other. We really feel like one team now with, of course, a critical mass of expertise.
As a result of the pipeline review of last year, we now have a focused pipeline with, we believe, a very competitive positioning in the cancer therapy landscape. To review the clinical and research milestones in 2021. It's actually quite a lot, but let me summarize the highlights. We continued with ongoing studies, which were the MERECA study in renal cell carcinoma, and also the ILIAD trial, which was a combination trial with checkpoint inhibitor, both involving ilixadencel. But we also initiated a new study in ovarian cancer, and we had an important data readout end of last year of our ongoing phase II study in AML, which we presented at ASH, which is the largest, by the way, hemato-oncology conference in the world. It's the American Society of Hematology.
It was an important achievement that we were able to present the data at that specific conference. The other part was the progress in research. As I said, we onboarded the process development for both of our lead programs in the Leiden facilities. We have a broad research network that we use to as partnering partners, but also to progress our leading position in the field of immunogenic cell death biology, and we presented those data at high-quality conferences. Finally, in December, we published a very important mechanism of action papers describing how one of our lead programs, DCP-001, works, and that was a joint effort in which we were very happy Alex Karlsson-Parra, our CSO, was also participating.
I think one of the first clear examples of the level of expertise that we have established in the company. A topic that raised a lot of attention, particularly with the historic shareholders of Immunicum, what are you going to do with ilixadencel? The key challenge was that most of the data had been generated in renal cell carcinoma in the MERECA study that I just described. With 88 patients treated, we did see a difference, which we also updated in February of last year, between sunitinib, which is a specific small molecule therapy, and sunitinib in combination with ilixadencel. The product shows initial signs of clinical efficacy in that indication. The landscape had changed. It means that there's now new therapies, particularly checkpoint inhibitors in first-line renal cell carcinoma, and they basically put the bar a lot higher for us.
The products that are now first-line treatment have a similar to superior efficacy as the one we observed with ilixadencel. That, combined with the fact that this is a large indication and that you have to show an incremental effect, would mean we would have to engage into a very large clinical study with a smaller chance of success than what we have observed in the phase II study, the MERECA study. This was basically a hurdle we could not take. Then we had a lot of data and other indications, but they were all with smaller indications and with small patient numbers. On the one hand, attractive, but on the other hand, also not yet decisive. As a last important point, ilixadencel is a product that is derived from donor material.
It means that we have to collect donor material, we have to ship it to the place where we do the manufacturing, and then you have to isolate the product, which is quite complicated. It's better than working with patient material, but it's also not a product that you can easily move into larger indications. We took all of that into account, and we decided to move forward in gastrointestinal stromal tumors for two reasons. One is we had promising signs of efficacy. Secondly, also a program in GIST will allow us to get relatively fast to clinical data that should be clear-cut, right?
It's a poorly treatable tumor with current available therapy, so if we can show a clinical signal in GIST, we have something we can immediately push forward, and that will lead to, let's say, more control over the company to establish this efficacy signal. Also in the meantime, we are working on further optimization of our manufacturing process. That is, in the end, the outcome of the strategic review we did not only by ourselves, by the way, we involved both our industry network and also experts in the field, KOLs, and doctors in the different indications to better assess the way forward, and this was an outcome of quite an extensive process actually. On the manufacturing side of things, two platforms. One is the DCOne platform, which is cell-line-based.
It means that we have the cells at hand each time we start the cell cultures, basically with the same material. The game there is to increase cell densities and to work towards more scalable manufacturing platforms. This is a platform that is ultimately suitable for scalability. Then we have ilixadencel, which is donor derived. Dendritic cells, which is the basis of the product, are notoriously difficult to manufacture. They usually show a lot of variability in the manufacturing process. They're a relatively sensitive cell type. Here we focus on two things, which is one, to get to donors that consistently give a better result of our manufacturing process.
Secondly, also to make the manufacturing process itself more robust, to get to a more consistent product, which is what you need to move a product into pivotal stage and commercialization in the end. That's a parallel process. Finally, with the combined research expertise and the ongoing activities both in our own labs and in collaboration, we have a broad possibility to continue to develop the pipeline and to also fuel the clinical pipeline moving forward. On the clinical pipeline, this is visualizing the opportunity we are addressing today. In the past, primary tumors were basically a deadly cause of the disease. By now, because so much has been invested in improving cancer therapies, that picture has completely shifted towards tumor recurrence.
Out of the 950 patients that still die from cancer to date, 90% is actually caused by metastases and is caused by residual disease that causes tumor recurrence. This is the real big picture to address, and that is what we are currently doing with the programs in AML and ovarian cancer, where we try to prolong the time to disease recurrence and basically provide patients with an option to treat the disease in the meantime, which is an important element. One reason is that it has not been addressed so far is that you need to define your clinical endpoints, right? That is something that we have consciously done in two ways. First of all, we look at disease where tumor recurrence is relatively fast.
You also have a relatively fast evidence or readout that your product makes a difference. Also with modern techniques, it's easier today to measure the disease, and you can actually monitor while you are treating patients if you have an effect on the disease. That is something that we are also incorporating in our studies together with so-called immunomonitoring, basically showing that our products have an effect on the immune system of the patient. Ilxaden cel is addressing something else. What we've seen in the cancer therapy landscape is that initial success was relatively fast with so-called checkpoint inhibitors, so antibodies that facilitate the immune system to act against tumors. That was a relatively consistent picture.
What was less consistent or what was more challenging is that after that initial success, it turned out to be very difficult to move the needle beyond that initial success. After many years of research and literally thousands of trials, the conclusion is that hardly anything moves the needle beyond checkpoint inhibitors. A second checkpoint inhibitor helps, but it also tips the balance into toxicity relatively easy. A limited group of tumors, the ones that are most immunogenic, so that quite easily trigger an immune response, are best responding to immunotherapy. That whole field is quite crowded. The hot tumors, the tumors that do raise an immune response are getting a lot of attention, whereas other tumors that are poorly triggering immune responses receive little attention.
This is where we see a positioning for ilixadencel and maybe also potential future immune primers that we are developing that can be administered into the tumor microenvironment, basically breaking the immune tolerance of the tumor towards the immune system. Just as one of those opportunities. If you look into patient numbers, and these are just high-level numbers, based on U.S. cases only, you can see that AML and ovarian cancer are quite substantial in terms of new patients, on an annual basis, but also in the amount of cancer deaths. Actually, AML and ovarian cancer are highly deadly disease, mostly because of the tumor recurrence.
You can see that GIST is a smaller indication, but it's one that receives little attention because it is small, but also it's very suitable, not only for the reasons I just mentioned with respect to immunology, but also for a product like ilixadencel, which is donor-derived, and a company like Immunicum keeping control over the product development going forward. As said, we have an opportunity in GIST to confirm clinical efficacy with a relatively small patient number, which will give us a relatively fast output as compared to the larger indications like renal cell carcinoma, for example. Ovarian cancer is an indication we chose because it is so deadly related to tumor recurrence, but also we see an opportunity there to more or less create a maintenance window.
AML is a disease, it's a blood-borne disease that comes back very quickly, and there is more or less an established need for maintenance therapies. This is a quote from MD Anderson basically emphasizing that last point, right? AML patients face high risk of recurrence. There is something that needs to be done, but you can't keep on treating them with high-dose chemo, for example. You need to find something else. The market is now opening up with an oral formulation of a specific chemotherapy called azacitidine, but we believe that there's a lot of room for improvement and that with DCP-001, we have a product that is well-positioned to address this medical need. What we do with DCP-001 is basically treating patients that have successfully undergone initial treatment.
The disease level is down, but you can still measure it, and that is called MRD, or measurable residual disease. Today, with molecular techniques, you can pick this up, and it makes almost a black and white difference, but certainly it's a predictor of a very poor prognosis for the patients with respect to tumor recurrence. MRD positivity means the disease will come back, and it will come back very quickly. What we try to do is, after that initial successful treatment, provide patients with DCP-001, which is a vaccination, as a way to stabilize the disease and to basically regain immune control of the residual disease. That will lead to a reduction of the recurrence rate and hopefully also some patients that are basically disease-free after the treatment. The market schematically looks as follows.
Oral azacitidine has just been approved, which is opening up the AML maintenance window, which we expect to grow because of the high medical need, but also with room for a product like DCP-001 to be added to the standard of care or to be added to the total, AML maintenance therapy window. This is the data from a very large trial that was done to get oral azacitidine approved. What you can see is that those patients that are MRD positive, which is the panel on the right-hand side, have a relatively poor prognosis as compared to the patients that are MRD free, that are MRD negative. What's also very striking is that when patients are treated with placebo, the median relapse-free survival, so the time before patients get sick, is only 2.7 months.
This gives you an impression on how dramatic the disease evolves if you don't treat it, right? This is the background against which we are testing DCP-001. That can be either in addition to azacitidine, or it can be something that is going to be used next to azacitidine, for example, in combination with upcoming therapies. There's room for improvement. That's the key message. In the phase I study, we did one important observation next to the safety signal, which is that patients that were already sick, that were already no longer in a complete remission and were going through what's often called a florid relapse, so a very rapid progression of the disease, cannot be treated with immunotherapy. It's not something we have observed only by ourselves. Also, others have confirmed this with other immunotherapies.
If you have patients that are developing the disease, you're basically too late to apply an immunotherapy. The encouraging signal was that those patients that were in a complete remission and that had a low burden of blasts, so that basically people look into the blood and look into the bone marrow, and patients that were having low levels of blast did respond with a very encouraging relapse-free survival median of 14 months and an overall survival median of 36 months. This was an encouraging start, but it did not yet incorporate MRD because at this time of the phase I study, it was not yet an established phenomenon in AML treatment.
In the current study, which is a phase II study, we focus on MRD because it is such an important predictor of disease recurrence, but also because it is a good way to monitor the effect of our vaccine. What we published, and this is basically a summary of the data we presented at ASH, or at this large conference, is that the majority of patients responded to the therapy and showed either MRD conversion, so meaning that there was no longer detectable disease, or showed a strong reduction of MRD or a relatively stable MRD. A very encouraging first sign of efficacy and one of course, that we need to follow up. Within the study window, we could already assess that the patients that were MRD converted were stable and relapse-free.
How this data will play out over time and how it will translate into relapse-free and overall survival in the end is of course something that we keep a close eye on and that we will also keep the market updated on. This is factually where we stand today with AML maintenance treatment, again, with a very encouraging initial sign of efficacy. In ovarian cancer, this is the picture I described. After first treatment, which in the case of ovarian cancer is surgery combined with chemotherapy, most patients are seemingly healthy in a complete remission, but the disease comes back very aggressively, very often within the first year, right? This, together with the clinical experts we work with at the University Medical Center Groningen, led us to design a trial specifically addressing maintenance treatment in ovarian cancer. This trial is ongoing.
It's a phase I study. We're testing safety, feasibility, maybe initial signs of immune responses. It is the first and important step into a maintenance window in another indication. Moving to ilixadencel. The pictures as I described, there was a lot of data, of course, from the 88 patients treated in renal cell carcinoma, but also a lot of additional data with one consistent picture in very challenging disease ilixadencel shows clinical efficacy. We've seen it with monotherapy. We've seen it in combination with small molecules like tyrosine kinase inhibitors, and we've seen it with checkpoint inhibitors. Also in the ILIAD study, which we announced to close end of last year because the study basically delivered the safety feasibility that we were looking for.
We saw additional signs of clinical efficacy, for example, in patients that had been heavily pretreated, including with checkpoint inhibitors. They started responding again to the checkpoint inhibitors when we added ilixadencel. The summary of the consistent picture is that in very tough to treat tumors and in tough situations, ilixadencel showed clinical efficacy, right? Out of this, we chose GIST as the main indication going forward for the reasons mentioned. In GIST we saw in six patients that two of those patients responded with an objective response to ilixadencel treatment, and this was after they had undergone multiple rounds of previous treatments. Again, very tough setting and this is the basis to move forward. Of course it's also a signal that we need to confirm because this is relatively small patient numbers.
In the end, that leads to a focused pipeline with which we enter 2022. We have the ongoing ADVANCE II study, which is the AML maintenance study, delivering multiple updates in 2022. We are preparing for the GIST study. Of course, we will provide an update as soon as we have worked out the details, but GIST has been chosen as the prioritized indication for ilixadencel. We have the ongoing ALISON study in ovarian cancer that will deliver its first clinical data in the course of this year. As a summary, 2022 will be dominated by progressing DCP-001 in AML, which means on the one hand additional clinical updates, but also preparing for the next steps in clinical development. We will report also for DCP-001, the first data from the open label study in ovarian cancer.
We are preparing for a study in GIST, which will be a confirmatory efficacy study with ilixadencel. That will of course also be something that we plan for this year. At the same time, our new facilities in Leiden are becoming operational, where we will further continue to optimize our manufacturing processes. Of course, with Alex as CSO, we have additional expertise on board to continue to develop our preclinical pipeline that will in the end also fuel the future clinical pipeline. We will keep the market updated. We started last year after basically the COVID situation seemed to stabilize with an investor event in November. We were very happy with the people that came and also that are here today. We will continue to do that. We appreciate very much your coming to this event.
Sometimes we will be formal, but we will also organize for another more informal event where you can just talk with management and ask questions about what we are doing. We plan to have such an investor meeting in Gothenburg where the company was founded, actually. For historic reasons, we also want to be there. We will provide for a clinical update in the second quarter when we also have more progressing data at hand from the ADVANCE II study, and of course, then we have the regular reporting to follow. With that, I would like to thank you for your attention and open the Q&A. The Q&A will be moderated by Mario Bakouli from Felicity Communications.
Mario, please, let us know what was sent in via the IR at the Immunicum email address and any other questions that may come in.
Okay. I will do. Good evening, everyone. We'll start the Q&A session with the questions that came from the covering analysts of the firm first, and then the emails that came to the IR email. But everyone here in the end has also the chance to ask any questions you like, so feel free to chime in at the end. The first set of questions came from Ingrid Gafvelin from Kempen, one of the covering analysts, and I will read them so that you can listen to them and the team is going to answer them then in due course. The first set of questions was: On the AML program, have you had a chance to dig into the data that you presented at ASH?
Are you confident on moving forward with the MRD status endpoint, or should we start thinking about potential other outcomes such as the duration of the response, relapse-free survival, or overall survival? The second question from her was: What do you see as an important MRD conversion benchmark? We have information about how authorities feel about the MRD conversion in ALL, but little information on AML. Want to tackle?
Maybe to Jeroen.
Yeah. I'm more than happy to take the question. I think it's a lot of questions in one, so we'll try to break it down bit by bit. I think the data which Erik showed was the status which we were able to present end of last year. That means the data represents the status as we have collected it up to, say, beginning of November 2021. We were able to report on the readout of residual disease. That's one of these key parameters which we're monitoring in this study for the majority of these patients, but not all.
By now we know that the last patient has also had, say, readout of MRD and that kind of data we will present in April because the last moment in time when this is read out for the last patient treated is actually towards the end of March. I think I can't give a full update on that data, but that is to come. I think the other important question here raised is you're looking at residual disease, you're looking at potential responses on residual disease, and it's a fair remark. Is that a validated endpoint for regulators? Well, the clear answer is not yet because no product has been approved on the basis of MRD purely as an endpoint.
The standard endpoints, which are always looked at for registering a product in a disease like AML, are the relapse-free or the disease-free kind of survival, overall survival periods, and, say, the actual survival, so how long does somebody survive? That is of course relatively early still. Although having said that, if we look at the ADVANCE II study, we're around about 11 months median survival for all patients. We're progressing nicely towards, say, one year and beyond. That kind of survival data is expected to come towards the end of the year. We have basically two important readouts coming up. One is, do we see responses on residual disease? Can we present more than the four patients who converted to become totally negative on residual disease?
That's something you will learn towards, say, April. By the end of the year we know more about, say the overall or the relapse-free survival periods. I think that's the key. Did I miss on some of the elements of the question there?
No, I think that was plenty. I think we can move on to her second, you know, box of questions which were around the ovarian cancer program. She asked, is the upcoming readout in 2022 focused on antigen-specific responses, and what are you expecting to see? Would you already be able to disclose some of the secondary endpoints as well? And how would a future trial look like? Is this an indication that you can follow up on your own?
Yeah. I think what Erik showed you is a little bit of, say, the natural course of disease also for ovarian cancer. Unfortunately, it's a disease which we see that is diagnosed in most cases late or too late, which means it has spread. It has spread throughout, say, the abdominal cavity. For the majority of women, that means you try to get away as much of the cancer as you can by doing what they call debulking surgery. Every, say, small piece of cancer you see during operation, you try to cut away. On top of that, they are being given chemotherapy. The fact of life unfortunately is that there is always small pockets of cancer which you probably missed, which also maybe the chemotherapy missed.
That's why the natural course of disease is that in a period of one-two years' time, there is often new evidence for disease and tumor growing. Currently we wait and see what happens, and recently new products are being developed and introduced to try and treat patients during that period, so the maintenance there, and that's of course our focus. Initially, the primary focus for this study is to look at the induction of immune responses which we think are relevant to take control of the disease. What we're doing is we're translating the observations in the leukemia program that we are able to trigger responses to some antigens which are common for all tumors, so not only leukemia, but also ovarian cancer or other disease types.
Those signs we wanna see, because these patients are treated with high dose of chemo, and we wanna know that if we then start to vaccinate them, do we still see responses which we intend to see? That's basically the question. We're looking for immune responses as a very early sign of whether this concept is translatable to ovarian cancer. The later time point, so reading out relapses, et cetera, that's to follow. That's something we will have to wait until there's a bit more time progressed in this study. For this year, we are able to see is it safe, and can we actually see whether there's immune responses induced? I think that's the major objective for this study.
Great. Moving on to our last questions, which were all around the GIST development program. She was asking, can you provide more details on the GIST study with respect to patient population, efficacy, safety benchmark, and overall trial design? And is it correct to assume that it is meant to be a pivotal trial?
Maybe address the last bit first. I think what the setup is for the GIST trial is to represent the first signs we saw. In this very limited subset of patients, the six shown also on the presentation here, we saw that two out of those six responded. That is a promising sign. Why? I think if you look at the current standard of care for patients who are suffering from GIST, the initial treatment seems to work in, say, half of the population. Once they progress, once they go to second line, then actually your choices are limited and the responses to the treatments are limited.
Sunitinib is standard second line for GIST patients, but only one in ten show a response, and that one in ten is of course extremely low. The median progression-free survival for those patients is only six months, and this is exactly what we're trying to see now. If we add ilixadencel on top of sunitinib, for which we already have good evidence that we can combine these two treatment modalities through the MERECA data, for instance, can we actually increase that? The six patients where we had two responses, that's 30%. Is that something which we can reproduce if we do it in 20 patients, for instance? That is the aim for this study, so it's not a pivotal study.
It will not lead immediately to registration, but it should lead to a clear-cut sign of efficacy which allows us to progress. I think that's the main answer for that part. How does it look like? Well, a lot of details will have to come, but I think the key is to focus on second-line GISTs. Patients who are in that situation where normally they would get sunitinib and only one in 10 response, and we try to see whether we can increase that to hopefully this 30%, which we saw in this first study. But that's the bar which we have to cross.
Good. Moving on to the next person. We got Christian Binder from Redeye with a couple of questions. First, on ilixadencel, do you still plan to pursue a potential checkpoint inhibitor combination if an interested partner for clinical development can be found? And with regards to the preclinical pipeline, can you elaborate on the kinds of agents you plan to develop and what unmet medical needs and indications do you expect them to address?
I can take that question, Mario. The main choices we've made as a company is to focus, right, with the outcome of the clinical pipeline that we just described. Also, in all honesty, we are a relatively small company, not only with respect to market cap, but also organization. To focus on the key programs is our key priority. That's one part of the answer. That means that we will continue in our research group, led by Alex, to look for differentiated combinations. We've seen, for example, very interesting data with ilixadencel and CTLA-4, which is a specific checkpoint inhibitor. But we've also tested novel combinations, some of which we are patenting. We've published data that, for example, demonstrated the potential synergy with blocking of CD47, which is a novel checkpoint inhibitor.
What we are currently doing is we are creating a basket of opportunities in the preclinical pipeline in order to be able to fuel the deeper clinical pipeline in the end. We haven't made clear-cut decisions there which ones we will prioritize. It's basically investing into the future or continued investment into the future. Of course, we also look for partnering opportunities, right? That is something that we will always balance against the focus where we generate most of our shareholder value at the moment, which is in the clinical pipeline. On the one hand, we do keep a finger on the pulse, basically always, through business development, through our regular contact with the pharmaceutical industry. For now, the main focus is on progressing the clinical programs.
The same thing holds true for the other part of the question on combination therapies. The current focus of the research group led by Alex is to support the ongoing clinical programs and to test them, for example, in combination with novel or existing therapies to make sure that we can potentially capture a broader positioning with those lead programs.
Good. Christian had another set of questions on manufacturing and your activities in the new facility in Leiden. I'll read them. Can you elaborate on the value of your in-house process development capabilities to potential strategic partners value manufacturing know-how? Are the facilities in the Netherlands focused on manufacturing process development and smaller scale production only, or could manufacturing capacity suffice to produce large scale batches for later stage trials?
All right, I will take that one as well. On the one hand, the process development is key. Process development means you know your own product best. You need to invest in process development because that in the end determines what process you bring into a so-called GMP environment, which is the manufacturing environment, because after that it's done. Once you start producing, certainly for pivotal stage trials and commercialization, at a certain point, that's your process. What we are doing today is investing mostly in that expertise for both DCP-001 and ilixadencel. Right now we still have a chance because we are not yet in pivotal stage to optimize those processes. That is the ongoing and most important activity also in our new facilities in Leiden. The question on GMP manufacturing is a more tactical one.
I believe we could opt in the end to set up GMP manufacturing in-house. Certainly when you move to larger studies, including maybe preparations for commercialization, it's not logical to do that in-house. You'd rather do that with a larger and international manufacturing network actually of third party service providers who have already set up the GMP environment. A tactical choice could be that we realize smaller scale facilities in-house so that it could give us additional flexibility in the future. It's not a key priority at this moment. The key priority is to make sure that our processes are robust and that they're ready for the next stage of clinical development and ready for transfer to GMP facilitie
Moving on. The last set of questions from a covering analyst came from Jonas Peciulis from the Edison Group, and he starts with the GIST program. Given GIST is a rare cancer, would you consider bringing ilixadencel in this indication all the way to the market yourself, or would you prefer to be in some kind of partnership deal while still in development?
Well, on the partnering aspect, it's all about timing and, I think for ilixadencel in general, but specifically also in the GIST indication, we need to make sure we stay in a position of strength and we can take this program forward by ourselves and make sure we deliver clear and strong signals of efficacy combined with us having, better and more control over our manufacturing situation, which means that we work towards a process that is delivering good quality ilixadencel moving forward. That is the first step we need to take. In the meantime, of course anything can happen, but we are not actively looking for a partnership because we feel we can do a better deal once we have moved further along the development path.
The second group of questions from Jonas were around DCP-001. Any insights on the dose of DCP-001 in AML? I believe there were two levels used in the ADVANCE II trial, and what other cancers could be suitable to investigate for DCP-001 or what other drug or anti-cancer therapy combinations could be synergistic? That's probably for you.
Yeah, I think to the question on the dose, correct. In the ADVANCE II study, two different doses are used. The difference is not super big. We're using 25 million cells per vaccination or 50. We're not seeing a difference, a major difference there between the two doses. Actually it's known from vaccinations that the higher dose is not a guarantee for a better effect. Sometimes even a lower dose can be better. The booster vaccinations, which we introduced in this program, patients are getting four vaccinations at these doses just described, and then at the later stage, twice they get a booster vaccination. Those are at lower doses. We also see that the boosters give reactions to the vaccinations.
I think carrying forward is most likely to go with the minimum dose of 25 million cells here, incorporating the boosters. I think that further question, can you then further still modify your treatment? The answer is there. We don't have a clear answer now. That is all depending on the readout also of more in-depth immune responses which we are monitoring in this study. But it could well be that additional boosters might have a beneficial effect that could be introduced in any future development in any future study. The other question was?
Well, mostly on all the other combinations or other cancer indications you would go after.
Yeah. I think the whole principle of doing a vaccination could of course be applied to a multitude of tumors. If we look at the acute myeloid leukemia program, our initial focus, of course, is in this maintenance space. Somebody has gone through, for instance, high doses of chemo, has recovered, the bone marrow shows that the number of cancer cells is very low, and that's what you call a complete remission. How do you keep that? That's the biggest challenge. If you don't have the option to go for a bone marrow transplant, you don't have the option basically to have a potential cure. That focus will remain and in that category it is translated to also other AML patients.
In other words, patients who are not getting high doses of chemo, but they are being treated with other modalities, like the hypomethylating agents. Those are also sometimes coming to a stage of complete remission, and there, of course, this can also be applied. That's a more broadening within that space of treatment. Other hemato-oncology indications are also possible. We have preclinical data on multiple myeloma where it works, and it could be translated to other diseases, but that's too early to speculate. I think the focus on acute myeloid leukemia already is broad enough, and that's what we're going for.
Good. We will now move on with the questions that were sent via email, and they were mostly around two big topics, before we then open up the floor for your questions. The first set of questions from via email were about pharma partnerships. What is Immunicum's strategy with regards to partnering your assets?
I'll take that one. Yeah. To make it very clear, we are in constant contact with the pharmaceutical industry, right? We keep a finger on the pulse, not only to basically get a sense of direction of where different programs are moving, also the programs that the pharma companies are developing, but also to keep a feel on what they believe to be important value drivers for their own programs. That is the first and most important thing to do. The other thing is you can't plan for a partnering deal. You can prepare yourself for a partnering deal, right?
You have to do that in a way that it puts you in the strongest possible position, which mean that you have convincing clinical data, which means that you have control of your manufacturing processes, and that you have expertise, basically, that they don't have in-house. That is the part we take care of on a day-to-day basis, and if there's, at the right moment, an opportunity to do a pharma deal, of course, we will consider it as one of the, let's say, many options in the regular course of business. Certainly when it can accelerate our clinical development and contribute in a value-generating way to the development of the company and in the end also the shareholder value, right?
It's all about staying connected, making sure that you are in a strong position to do a deal, and then once you do a deal, make sure that that deal really brings added value to the company. That is what I think I can say about the partnering deal. Of course, we cannot comment on, let's say, individual situations or ongoing negotiations, but I hope this gives you a good sense of the way we strategically deal with partnership opportunities.
Good. The second set of questions were all around financials. Looking at your financials and your cash position and cash guidance. With the market seemingly a bit more cautious, in terms of financing, what are other strategies you are looking at as to extend your cash runway?
Right. Well, the simple answer is all options. I think as a biotech, you should always make sure that you stay funded and that you do it in the best possible way, for all stakeholders. So of course, we are looking into all options available to the company. I think for good programs, there's always funding, and of course, it's best to do it when markets are great, and they were great for a large number of consecutive years, and today they are not. It was a poor market last year, and the start of the year was also quite poor. Then looking at Immunicum, the way we are valued versus the data that are now shaping up and continue to shape up this year, I'm confident we will find a way forward.
Of course, I may not even have to state it, but we have all put money into the company. All of us are shareholders. I've put quite a substantial amount of company money into the company. We are in this, and we are behind this plan, and every time, not only for ourselves, of course, but for all shareholders, we will aim to make the best possible decisions on the financing of the company moving forward.
Thanks, Erik. With that, we're happy to take any questions that come from the audience. Just give me a signal, and I'll run and give you the mic.
Rutger Smith. I do understand that you're not making predictions, but on the spend for this year, will that be in the same magnitude as last year?
Well, the only thing we've said is that we will reach the end of this year with the cash we raised last June, right? In last June, we had the financing round, and that financing round basically was meant to fund the ongoing studies to prepare for new clinical studies and of course, ongoing activities, including research. That is a statement we will stick to. If you look at the current cash position, that should give you also a good indication of the cash burn we expect for this year. I'm not sure if you can add anything to that, Lotta, but I think this is-
No. Yeah
the most concise answer we can give.
Yes. It is.
Anyone else?
Hi. I'm interested in the MERECA study and the long-term survival. Do you have anything to clarify? I think it's one year since the last report and might be something in February right now. We are collecting the data, but we're not analyzing it this year. It's gonna be analyzed when the last patient actually has completed the full follow-up there. That's gonna be beginning of next year.
There will be no report in this quarter?
No.
No. Okay.
Anyone else?
The things that I've been trying to analyze, I tried to look on where we are with the programs and how is the competition. I will be happy to hear comments about this. As you have seen, for instance, the combination of ilixadencel with CTLA-4, it gave like 70% efficacy and no recurrence in the preclinical study, which is like breakthrough. Are you aware of any trials that are in AML or whatever, say ovarian or something like this that could compete with our program?
Absolutely. No, I think it's a good question. For each of the programs where we look for competitive positioning, we of course go through this analysis. We first focused on the clinical programs, and then we will focus on the preclinical programs. The CTLA-4 data, the preclinical data are striking, right? I think in that sense, they could provide for, you know, future studies. It's more a question of priorities. When we look into the indications we are currently pursuing, checkpoint inhibitors have been poorly effective, right? That opens up a whole different window of opportunity. In bloodborne tumors, specifically AML, there has been very little checkpoint inhibitor efficacy, probably because the disease disturbs the immune compartment, right?
There I think we have a competitive positioning, of course, first showing monotherapy data, which are always considered to be the strongest. Also for ovarian cancer, very little checkpoint inhibitor activity and same for GIST. Those were the most logical choices and prioritizations to make now. Like I answered, you know, this is a very promising preclinical signal, absolutely. We're starting to see other more differentiated checkpoint inhibitor combinations as well. I think the name of the game with the checkpoint inhibitors is there have literally been thousands of trials, as you know, and ongoing still, with very little really having an impact in terms of moving the needle on top of checkpoint inhibitors. That's also a given.
That's one of the reasons why we prioritize first what we want to do in the clinic now, and that's already quite a lot actually for a company of our size. Then we will look into more differentiated combinations with checkpoint inhibitors. The study you are referring to, the preclinical study, is a very strong one. Like I said, we have additional data now, also some data that we have recently been able to file for patent application, for example, that are more around specific combinations between our product and checkpoint inhibitors. That is the endgame how we see it with checkpoint inhibitors. You have to look for differentiated combinations first before you jump into the big pool basically of all the ongoing trials. It's certainly not off the table. Thanks for reminding us of that.
Any other questions from the room? That does not seem to be the case, so I'll hand over to Erik for the closing of the meeting.
Thanks. First of all, thank you all for being part of this meeting, both the people in the room and the people that have been following this online. Thanks to my colleagues, both in the management team and the board, but certainly also the colleagues that are following this from their desks in Sweden and in Leiden. I think we went through a very interesting year, with I think now a data package, but also a starting point in the form of a focused clinical pipeline that will make this a very interesting journey. As CEO of this company, I'm extremely proud and happy with the results we achieved.
I'm proud and happy with the team, including also the contribution of Alex particularly in providing more depth and more expertise into the programs that we are pursuing with the key publication already being a clear sign of what we are currently moving towards as experts in the field. But also very excitingly of course, now a starting point with multiple studies planned for progress, including data, significant data progress in 2022. Thank you for now, and we look forward to keeping you updated in the rest of the year.