Good afternoon and welcome to the Poolbeg Pharma plc investor presentation. Throughout this recorded presentation, investors will be in listen only mode. Questions are encouraged and can be submitted at any time via the Q&A tab situated on the right-hand corner of your screen. Please just simply type in your questions and press send. Before we begin, I'd like to submit the following poll. I'd like to hand you over to Jeremy Skillington, CEO. Good afternoon, sir.
Good afternoon and thank you for the introduction. Thank you everybody for joining us this afternoon for the Poolbeg presentation. Of course, on the back of our RNS this morning talking about the interim results. Again, we have a fantastic presentation to go through to update you on that. I'm delighted to be joined by my colleagues, Liam Tremble and Ian O'Connell, and we'll kick off, and let's go through this. As I say, we've got the A-team with us today. I'll talk about the background, talk about the company to set the scene. Liam will talk more about the clinical trial, the TOPICAL clinical trial. As I say, we've announced the positive interim data this morning. We're all delighted with that just as things are moving. We've got some terrific momentum now, so we're looking to execute.
Ian will speak more about the commercial opportunity that we have here, because ultimately we're looking for partners and partnering the program. We'll talk a little bit about that. Again, to set the scene, Poolbeg Pharma are a clinical stage company. We're developing POLB 001 to potentially transform the lives of cancer patients, to allow them to have their cancer immunotherapy treatments safe and locally. By doing that, we're preventing CRS is the plan. That's our goal, to prevent cytokine release syndrome. That means then they can take their drugs from home or in the community. We also have an oral GLP-1 program that we may touch upon later on. Many of you know we have a fantastic team here, a proven track record of deals and transactions, executing deals with big pharma companies, selling companies, et cetera.
Very excited about the clinical trials that are pushing forward. As I say, the focus today will be on POLB 001 and the TOPICAL trial and these interim data. Again, clearly the goal of the company is to partner this program. We have a very clear path forward what a phase III looks like and ultimately getting on the market. We talked in recent times about meeting with the FDA earlier this year, so that lays out that groundwork. Again, we have some terrific ongoing discussions with both big and mid-size pharma companies about the program and what it would take to move that forward. As I say, we're building nice momentum in that setting as well. Importantly then, we had a fundraise earlier this summer, so we've got a financial runway into Q2 2028. So that gives us a nice solid foundation for those negotiations.
POLB 001, potentially the first approved preventative therapy for cancer immunotherapy-induced CRS. We have mentioned this in the past that cancer immunotherapy are essentially wonder drugs. They are driving cures of certain cancers that did not exist 10 years ago. In essence, cytokine release syndrome did not exist in this context. It is a large and growing issue for these cancer immunotherapies, and we believe that we have a drug that could potentially prevent CRS from occurring in these situations. As I mentioned, ultimately getting these drugs into the community care, patients can take them at home. There is a big unmet need there and a lot of interest in what we are doing. As a reminder, CRS or cytokine release syndrome, we see ourselves as having that potential solution. It is a systemic inflammatory side effect for these cancer immunotherapies.
Again, in a nutshell, cancer immunotherapies are redirecting your own immune system to go after your tumors, but you have these nasty side effects including CRS. What does that mean? Patients get fevers, they get increased heart rates, they get low blood oxygen, low blood pressure, and then that can escalate into severe issues around kidney injury, neurotoxicity, capillary leaks, et cetera. Again, that is a severe issue. If it starts, it is a bit like a runaway train.
There is an issue where you want to put your foot on the brake of that train and bring it back to normal. As I say, with POLB 001, we believe we can stop that from happening in the first place. It is an oral agent. It is given orally. It is an inhibitor of an important immune control system called p38 MAP kinase. It is a gatekeeper of that inflammatory response.
As I said, we want to inhibit these cytokines that drive that kind of inflammatory response. As a master regulator of that inflammatory response, p38 MAP kinase is an excellent target in that context. Again, this drug has been in humans previously. We have done our standard phase I, single ascending, multiple ascending dose. So it has been in many people, many humans. As many of you know, we did an LPS challenge study a few years ago and saw excellent results there to prevent inflammation that is caused by a bacterial fragment, LPS. Again, it is important to look that one of our significant advantages is we are given orals. Patients can take our drug from home. They do not have to come into hospital and get an infusion or an injection of a protein-based drug, for example.
As I say, what we are happiest with here is that the coverage, and you can see here in the lower box, the red dots, admittedly the font is quite small, but what we are seeing here is POLB 001 can inhibit a whole host of cytokines and far more than other drugs in this section can as well. Tocilizumab is given as a standard of care once CRS develops, and you can see the red dots there. There is only a handful of these cytokines that it inhibits, and far less than POLB 001 can. So again, there are other drugs like dexamethasone, which docs do not like at all because that can have an immune suppressant side effect. Then JAK kinase inhibitor as well, that is going to inhibit some, but not a lot of these cytokines. So we see advantages here for POLB 001 in that setting.
I mentioned about this large and growing market of cancer immunotherapies. The graph on the left shows the expected growth in revenue of these many approved T-cell engagers they are called, and they come in two different classes, the bispecific antibodies, which we are using in our clinical trial, as well as CAR T-cell therapies. As I mentioned, the efficacy they are seeing, particularly in the blood cancers such as multiple myeloma, is very impressive. As you can see in the table on the right-hand side, these approved drugs, you can see the big pharma companies, Johnson & Johnson, who we are collaborating with on this TOPICAL trial. Other companies like AbbVie and Roche, they have these bispecific antibodies approved and on the market. As you can see on the right-hand side, they have significant issues around CRS.
These are noted that teclistamab, or TECVAYLI, as it is here in our clinical trial, 72% of patients develop cytokine release syndrome. I would say for our TOPICAL trial, we are looking to reduce that number. It goes across the board. They know it is an issue. They know it is coming. They know patients have challenge with CRS. It is an added burden to the healthcare system who are there to look after the patients and their cancers, but now knowing that a large majority of them can and will develop cytokine release syndrome. So they are looking for solutions as well. As you will note when we talk to, particularly clinicians in the U.K. about our work, there is a lot of interest in participating in our clinical trial. There is a video here I want to show.
Bob Munro is an actual multiple myeloma patient, and Bob talks here, about a minute and a half, about his issues that he had in the past with CRS. So I want to bring our story to life and what the challenge and issues are for patients.
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Appreciate Bob's comment on there, because again, I think it highlights the issue of CRS and the fact that in a real-world setting, as I say, our goal here is to allow patients to get their drugs and then, as I say, go home and then not have to kind of stop or start their treatments or as I say, develop CRS, these nasty side effects. That's kind of me in an intro. I will be back later on. I am going to pass across to Liam Tremble now to talk more about the clinical trial, as I say, from the RNS this morning. Liam, I will step to the left.
Cheers. Thank you, Jeremy. Really excited to speak to you all today with this really exciting news for the company. I guess just as background for those of you that maybe are not so familiar with the story of the company, this is really the transformation that POLB 001 is really going to make for these cancer patients. These bispecific antibodies, if you look in the left-hand side of this slide, typically this is done in hospital. They have to be hospitalized for up to a week. What happens is you give this immunotherapy, and it activates T-cells. This drives the production of cytokines, results in cytokine release syndrome, and this happens through p38 MAP kinase signaling.
This is a potentially life-threatening side effect that needs to be managed quickly so that it does not progress, where if it progresses, it can affect virtually any organ system in the body. In addition to this, if cytokine release syndrome does happen, that week of hospitalization could be extended. If they have cytokine release syndrome, they cannot get the next dose. What they are in for a week because they have to get two or three doses, and these are small doses before they get a full dose. This is purely to get the body used to it so that we do not have overwhelming cytokine release syndrome in these patients. If cytokine release syndrome does occur, then they need to delay these step-up doses. That week can turn into a much longer period.
And obviously, this is a challenge not just in the existing centers to administer, but also in making it more widely accessible to centers that don't have that resource utilization to have beds for these bispecific antibodies. Alternatively, the hypothesis with POLB 001 and the solution that it will offer is that we can actually give this orally at home. They can take it before their cancer immunotherapy happens. It doesn't affect the cancer immunotherapy. They still get the benefit of the therapy. They don't get this cytokine release. They can get this in an outpatient or at-home setting. P38 blocks the CRS, and by preventing CRS, then we actually reduce the hospital stay. We enable administration, particularly in community hospitals, and improve patient quality of life.
And that community hospital piece is so important because, as I'll talk to in a moment, particularly in the U.S., a lot of patients actually can't access treatment in what we refer to as high tech or centralized hospitals. In America, up to 75% of late-stage relapse myeloma patients only get treated in the community care, which is very different to what we're used to. So getting into this community setting, critically important that we get this treatment out of the hospital setting. Just a little bit more on the TOPICAL trial itself. Obviously, we're excited to share really good news on this. Just before we go into some of that, what is the trial? So it's a first inpatient trial, as I mentioned, in multiple myeloma patients, relapse refractory, so quite ill patients who are receiving the bispecific antibody teclistamab.
It's been led by Dr. Emma Searle, who's a Consultant Haematologist at The Christie Hospital in Manchester, and it's being conducted by a blood cancer specialist organization, Accelerating Clinical Trials, who are really equipped to deliver this as quickly. And we've made a lot of effort to design this trial in a way where it can be delivered quickly and equipped with teams that are able to make that happen as well. So the trial is planning to recruit about 30 patients.
And down the bottom here, as Bob Munro just spoke to, Emma Searle, the chief investigator, experiences this day to day and the challenge that it presents to patients, obviously on their quality of life, but also from her perspective that to get these treatments to more patients, you really need something that removes CRS, removes this bottleneck, and that's how you will access a broader population and get these life-saving immunotherapies to the patients who need them. So the trial. This on the left-hand side is the design of what's happening in the trial, designed to be conducted quickly. So cytokine release syndrome, as I mentioned, happens when these patients are getting step-up doses. So we have it here as tech dose one, tech dose two. That's teclistamab dose one and two. After these first two small doses, they get what we call a full dose.
It is during this period where we step up the level of the dosing that cytokine release syndrome occurs. We give the patients POLB 001 to take twice daily orally at home during this period, then they can get these bispecific antibodies without cytokine release syndrome happening. The trial itself is single arm, which means that everybody in the trial is getting our study drug, POLB 001, and it is open label, so the data is available for the investigators to read as it goes along. As I mentioned, it is going to recruit approximately 30 patients. There is a partnership with Johnson & Johnson there for the provision of teclistamab, which we think is a really important endorsement to the program, that they see the promise of this cytokine release preventative strategy as well. Endpoints in the study, what are we looking at?
Obviously, the safety of POLB 001 is critically important. We are going into a late-stage cancer population who are severely unwell, and it is the first time the drug is going into that population. So safety is a key focus. Related to that, we are obviously looking at the incidence and the severity of cytokine release syndrome. Then we are also looking at other things from a drug development perspective, like confirming the safety and the pharmacokinetics of the drug itself. That pharmacokinetics refers to the level of drug in the body before it is metabolized and it is eliminated from the body. This is really important information as we go forward to make sure that these patients have an adequate amount of drug in the body.
We are also looking at things like how is CRS managed and how is tocilizumab used, because this tocilizumab is an IL-6 receptor, quite often given by IV. There is a lot of pieces that go into CRS management that is quite burdensome from the hospital. This is reported in a lot of the biggest trials that happen across the world with these bispecific antibodies that have revolutionized cytokine release, or, multiple myeloma treatment. Seeing all of that fall away is quite an important aspect of what we are doing. We are not just looking at the cytokine release, we are also looking at a lot of that administrative burden and burden to the patient falling away as well. Just to quickly mention, this is not a typical oncology trial.
Obviously we are looking at multiple myeloma patients, and typically when you are doing a trial in this population, you are giving a disease-modifying drug. You are trying to prevent their cancer growing. You have to follow them for a long time. While cytokine release syndrome would happen at the start of their therapy, you have to wait months or years to see how these patients respond. As long as they continue responding, they stay on protocol.
That is why trials in this setting can typically take many, many years to complete. What we are looking at with the TOPICAL trial is actually we are only looking at that first section of the patient journey of cytokine release syndrome and when it occurs. Basically, that is going to happen in the first couple of weeks. The patients will only stay on protocol for a month or two, and after that, they will come off.
So as soon as we can get the patients through this process, that is the trial done. As I mentioned, short-term monitoring, one to two months. Open label, as I previously mentioned, really important. That's rapid visibility for the investigators that they know whether or not this drug is working. As I mentioned, single arm. So every one of those 30 patients is receiving POLB 001. So not like a typical trial where only half of your patients will get the study drug. Just to give you background of what we've done to date, obviously a lot of effort went into designing this trial so that we could deliver it rapidly. We announced earlier this year MHRA approval. That was a long time ago, it feels at this stage. A lot of work has gone in since.
After that, we made sure we had the right sites on board, that they had the right volume of patients, and also they had the right quality systems to conduct this trial and collect the data in a high-quality manner. That was validated by going to site initiation visits where we went and checked, really these sites are fully equipped to deliver everything they do and to hold them to delivering a certain amount of patients. If they want to partake on the trial, there's an obligation there where they're meant to try recruit as gone. As we've seen, as we're announcing today, that designing the trial to recruit quickly has been validated now. We're seeing certain sites really able to deliver patients quickly, which we're really excited about. Site activation occurs after all of that.
Once site activation happens, this is all CRO, so Accelerating Clinical Trials led activities with the chief investigator. We really move into site level pieces, and this is patient identification. Typically what happens is you'll have a multidisciplinary team of specialists who sit once every couple of weeks in every hospital, and they'll go through all of their patients, and they'll identify patients who need to go onto a new line of treatment, and also who's suitable for a clinical trial. After that, you go to the patients and see if they want to actually participate on the trial. We've got really good feedback that a lot of patients are really happy to partake in this trial, that it's attractive to them as well, which is a really important part of trial design.
If they're willing to take part, the patients are screened to make sure that they align with the formal eligibility criteria of the trial, and then they can be recruited and dosed into the trial. This is really where the substance of the trial happens. It's where we're at now. It's where we're rapidly gaining momentum as more and more sites come on board. We're continuing to hopefully get that momentum. Obviously, as we're announcing today, the recruitment, dosing, results in interim data. We're really enthused with what we've received. The clinical trial interim data, we're super excited. This is a major milestone for the company. Obviously, these are seriously unwell patients, and the Safety Review Committee have reviewed the data. They have announced that the study can continue, no modifications to the protocol.
So obviously they have a lot of faith that this is safe to continue in and that they have a lot of optimism that the study can go forward as it is. For us obviously, cytokine release syndrome is itself a safety endpoint. So these are all really positive milestones. The fact that the study drug can be administered as we expected in this really sick population is a really encouraging signal for us at this stage, and we're enthused by this. So, as I mentioned, this is a really positive sign for the program as we position it that it can be scaled up quickly, that it's attractive for partners, and that it can get, as we mentioned earlier this year, after the pre-IND feedback from the FDA, that there is a clear path to market here that could happen quite quickly.
This is a key de-risking point in the program that gives a lot of confidence in the program and will also instill a lot of confidence in potential partners to really move forward with it. So we have a lot of momentum. The recruitment is continuing. Even since having this news, we've already got news that there's another patient being dosed, which is just fantastic. So we really are thankful to Accelerating Clinical Trials and to all the sites involved in the trial- to- date, and the feedback so far has been really good. So we're super excited. Obviously, we'll be sharing this with partners, but I'll let Jeremy speak more about that.
Yeah. Thank you for that, Liam. Yeah, appreciate that. So listen, we kind of have nice momentum on a lot of fronts. So on the business development front, we mentioned this previously, we've had some really productive discussions with several different companies that are interested obviously, to see the outcomes of this interim analysis and as the clinical trial develops. But I think what's important here is that, as I say, we see this, and they see this as a de-risking issue. So, interim data we have now, the Safety Review Committee has reviewed that, told us to go forward, keep the same dose. Everything is going according to plan. As Liam said, recruitment is going exceptionally well across the sites. So we're excited with that. As I said, we're building that data package from a business development standpoint.
I think what's interesting in that front is that, in the background, we've looked at the commercial opportunity that this provides as well. That's one of the key questions I ask. Liam is going to talk us through that aspect, and then I'll be back later on just to wrap up. Ian, over to you.
Thanks, Jeremy. Following on from Liam on the clinical side, as Jeremy said, we want to spend just a few moments to run through the recent commercial work which we've done, which is strongly supportive of the program. Earlier this year, we commissioned Acumetis to run independent payer research in the U.S. This was conducted then through by means of structured interviews with current commercial payers covering around 75 million lives in the U.S. a pretty broad cross-section of the market, right the way across commercial insurance, Medicare, Medicaid. This was complemented by cost offset modeling, whereby Acumetis were able to quantify the cost savings from avoiding the CRS-related costs, such as hospitalization, that Liam already spoke to.
The first thing that came back loud and clear from the payers was that CRS, for them, is an expensive problem, and one that they recognize. Hospitalization, ICU use, length of stay, and acuity are key drivers of this cost. Secondly, the research found that an effective preventative therapy could support the broader outpatient delivery of these cancer immunotherapies. There would be meaningful access and cost implications for the payers if this was able to be effected. On POLB 001 specifically, they think that it's positioned as a commercially meaningful CRS prophylactic, or as what we'd say, preventative therapy, driven by reduced hospital burden and broader outpatient use. I think most significantly, the key insight was really that on the payer side, because of all these issues, there was a clear willingness to pay at commercially meaningful price points.
On that then, we'll flick onto the next slide, and you can see the conclusion of Chris Grimes-Crompton, who led the research at Acumetis. I think it's there pretty direct. POLB 001 is positioned as a compelling CRS solution with significant market potential. Because payers are willing to pay at commercially meaningful price points because of all the factors that we've talked about, such as hospitalization, they concluded that taking into account the patient flows and the epidemiology of the condition, that there is multi-billion dollar peak sales potential in the U.S. alone for POLB 001. Again, really significant commercial product, and one piece that really resonates with potential partners. Now that we've covered the validation of the commercial opportunity, I'll hand you back to Jeremy to talk about the partnering in a little more detail.
Great. Ian, thank you so much for that, because I think it's an important component. For those of you, some of you might have been at the Investor Summit last Friday. We had a terrific panel there, that included Michiel Bröker at UBS. He talked about what pharma look for. What pharma look for in the deals that they send their scouts out to scour the world essentially to look for. This is kind of summarized here quite accurately, scientific rationale. We put this in the context of POLB 001, like are we checking these boxes? Do we meet the needs that pharma look for when it comes to partnering? I think from our standpoint, scientific rationale, absolutely. p38 MAP kinase is a great target. There's nothing out there to prevent CRS right now, and we've got a terrific preclinical and clinical data set.
The clinical data is obviously a very important component, and obviously that's where the TOPICAL trial is moving forward. Happily today, as I say, we've passed muster when it comes to that first interim safety analysis, which is terrific. As we mentioned, recruitment is going, and we're keeping at that same dose, the 150 mgs twice a day dose that we have data generated from. We were also encouraged to see that recruitment is pushing on, and we've been approached by other clinical trial sites in the U.K. that they want to join this trial as well. Great enthusiasm from the field because there's such an unmet need. From a regulatory standpoint, how do you get this drug through the clinic and onto the market? That's where the regulators come in, the FDA, the EMA.
We mentioned we sat down with the FDA back in May this year and talked about the program, talked about what a phase III would look like, and they gave us fantastic feedback on guidance, what a label would look like, where exactly you can sell it, and the endpoints for phase III. That gave us great clarity or confidence we're on the right track, and we share this with pharma all the time. This is where the FDA thinks we're going. As Liam mentioned particularly with the TOPICAL trial, these are short clinical trials. Getting this to the market quite rapidly is very exciting to a lot of people. Intellectual property, key component in this industry. We've had several announcements this year of patent grants across Canada, Europe, South Africa, Australia, and notice of allowance in Israel.
There's great confidence that we have protection, we have exclusivity when it comes to once we get it on the market. Ian spoke about the commercial validation. It's an unmet need, and I said earlier on, it's a relatively new space because cancer immunotherapy drugs are relatively new. They're growing. Big market, large unmet need, multi-billion dollars in the U.S. As I say, we have a lot of compelling discussions with big pharma and some mid-size pharma about the program, and just moving that forward. I think we're excited where we are right now, say from the clinical trial standpoint, and building up that bit of competitive tension about getting the partners into our data room to do their diligence. This is a slide, again, I borrowed from Michiel from the conference last Friday, but pharma is under pressure.
They have a patent cliff coming where a lot of their largest-selling drugs are going off patent. There's a list here of when they go off patent from 2026 to 2033. About $400 billion in revenue for big pharma goes off patent, so they lose exclusivity. They need to find other drugs to fill those gaps, fill those holes. As I say, our discussions with partners is like, "POLB 001 can do that." We can generate revenues. We mentioned the market is $ 10 billion in size and over $ 2 billion in revenue, so it'll plug a little bit of that hole. Big unmet need for pharma. As I mentioned, we have diligence ongoing. Pharma companies are in our data room. The font size here is quite small, but we've got all of our documentation in these virtual data rooms online they can access and review.
We call it they're doing diligence on the program to understand everything we've done so far. As I say, we're going to build up that story, build up that competitive tension. Ultimately, the goal is to transact on POLB 001. We'll wrap it up there. I do want to leave some time for questions. Again, appreciate those who've been sending questions in. In summary, very experienced team here at Poolbeg executing. I think that should come through in this presentation. There's been fantastic execution of the clinical trial in 2026. Again, with thanks to Liam, Meena Arora, and Paul, and the team for driving that forward along with our collaborators such as Accelerating Clinical Trials. Again, very exciting with the stage of the programs and moving those forward. Again, high value with large unmet needs. Again, partnering focused.
Again, a reminder, we have cash runway into Q2 2028, which again gives us that buffer period where we can negotiate strong deals, attractive deals for Poolbeg and our investors, and looking forward to the next stage of the company. Again, appreciate all of your time this afternoon. We'll wrap it up there from the presentation standpoint, but I do want to get to questions. Again, appreciate those that have submitted. We'll go through as many as we can in the next 10 or 15 minutes or so. Again, appreciate input in advance from Liam and Ian as I read out these questions. All right. The first question came in. I like it short and to the point. "Is this what you expected?" Well, personally, I'd say it is.
We're happy because things move forward, but again, maybe I'll point to Liam to give in a bit more detail to what the expectations were and what this readout is.
Yeah. Thanks, Jeremy . At this stage of the trial, we're really excited by this news that we're going into this sick population, and the trial can continue with the Safety Review Committee. This is really positive. It's a major milestone for us. I think it's a major milestone for the program, and it gives us a lot of encouragement pushing forward. So yeah, it has to be considered in the context of where we're at. This is the first time going into late stage relapse refractory multiple myeloma patients. These patients can be severely unwell, and being able to deliver the drug in a way in which the committee feel is safe is a strong endorsement to the program. So we're really excited as the program moves forward, and it gives us a lot of encouragement.
Super. All right. I'm going around in circles. I should say Liam and I are in Glasgow right now. The International Myeloma Society meeting's on, so a lot of good discussions here as well. I appreciate that. The next question is quite lengthy, but I'll read through. "Today's RNS describes the interim safety review as positive," correct? "Without asking Poolbeg to disclose anything commercially sensitive, can you give investors more detail on what the interim cohort actually showed?" Particularly the number of patients treated, CRS incidence and severity, and whether there were any early signs of POLB 001 having the intended biological effect. I'm aware this phase is focused on safety, but often there's additional clinical indications to share. Thank you so much for writing out that question. Again, I'm going to look to Liam to address that.
But obviously, the commercially sensitive aspect as well, we're very deliberative in what we're allowed to disclose. Liam, if you can address some of that.
Yeah, absolutely, Jeremy. The investigators are, as Jeremy mentioned, we're actually at the International Myeloma Society meeting now, and a lot of the investigators in the TOPICAL trial are here. They will be meeting tomorrow. They'll be going through all of the data in detail, the same as the Safety Review Committee went through. This is the patients going in. We have really good momentum. We're going to give updates as that enrollment picks up. We'll keep everybody up to date on what goes. But the fact there's no change to the protocol, and cytokine release syndrome is essentially a safety endpoint as well, which needs to be borne in mind. So that it's safe in this population is really positive. This is a major milestone, as I mentioned. I'm really encouraged to move forward.
Thank you for that, Liam. Again, going around in circles. Why did you run this study in the U.K.? That's a really good question. I'll go back, something maybe historical. We had some really good interactions with Emma Searle a few years ago. Emma, as you know, is the co-principal investigator on this study. When we brought our idea to Emma, she kind of seized on it immediately. She saw, again, she's at the coalface, she's seeing these multiple myeloma patients, giving them these bispecific antibodies, seeing the CRS develops. She saw straight away the potential. You can give a small molecule or already available drug to patients, that would be great to reduce CRS. I think one of the reasons we have them in the U.K. is because there's such a really good, tight network of myeloma or hematologist or myeloma docs.
Emma reached out to Rakesh Popat down in the UCLH in London, and he's a key opinion leader in the space. He wanted to join this study. They ended reaching out to Charlotte Pawlyn in The Royal Marsden. We kind of built this early momentum, and we recognized from a trial design standpoint, we needed 30 patients. It was realistic that we could achieve that goal relatively rapidly. Liam went through that this is kind of a rapid study in the U.K. with Emma and her colleagues to collaborate. We have the six sites in the U.K., as we mentioned. As I said, we've been approached by others because the network in the U.K. is so tight. I think it's fantastic for this particular trial.
I will comment, for the phase III design that we have mapped out and discussed with the FDA, and indeed kind of introduced to pharma companies, that will be a global study. We're looking at many more patients. Obviously, you want to go global and test it in other centers. I will say, once we kind of introduce that concept or idea to other clinicians in other hospitals across the globe, particularly in the U.S., there's strong interest to participate. Again, we're trying to be seamless as we're kind of moving through this clinical trial plan, the path forwards, and build that. Again, appreciate the question there. Let's ask that. Question, how many patients have been dosed with POLB 001 to date? Again, that's a good question. When you think back to for this particular study, the numbers are relatively small.
As I say, we're going to dose upwards of 30. I think the plan from a timing standpoint, we'll have all that done, wrapped up, completed in the first half of next year. That's what we believe as things are kind of moving forward, particularly if other trial sites come online. I think it's moving along quickly in that sense. Again, we're trying to get this into as many patients as possible. Of course, worth highlighting, it's been in many human subjects, healthy volunteers over the last number of years for the various studies that were done with POLB 001. Next question. Again, appreciate the questions. Are discussions taking place with potential partners now, and is a deal possible before the full results? I'll take that one. Ian can weigh in as well if he'd like, but for that one, absolutely.
I've been in this industry for 23, 24 years. They always talk about how deals get done. It's not an overnight discussion. It's building confidence in the program, sharing details, sharing information. Every patent that's granted, we notify our potential partners on that, and we do have a long list of people who are engaged, people who are kind of reaching out. As I say, we want to time it right that we have sufficient clinical data in the TOPICAL trial that de-risk the program, and also kind of syncing that up with the actual value of the program, what other parties are willing to pay. There's a nice kind of, as I say, negotiations going on in that sense, and when is the right timing. There's always discussions going on. We're always updating them.
I think before the full results, this is where the competitive tension comes in. People realize that if they do not pull the trigger now, it will be gone soon. There will be good discussions, as I say, from various companies, and each company has a different perspective. Some of the big pharma companies might want to pair this with their drug and their drug alone, but then you have cancer supportive care companies who would like to get POLB 001 on the market and to be used with all T-cell engagers, bispecifics, and CAR T-cell therapies. So a nice little bit dynamic. As Ian went through, it is a significant market, and I think we have the potential to be the first orally available small molecule drug to prevent CRS. Again, appreciate that. Next question. When are the full results due?
Again, I talked about that is linked to patient enrollment. We are happy with the rate of enrollment right now as the sites progress, and as I mentioned, potentially quicker if additional sites come on board, and we are doing diligence on that. Again, appreciate the work that Meena, Liam, and Paul are doing to drive those forward as well. Another business development question here: What is the ideal? deal structure? One partnership, multiple partnerships, or a buyout? Ian, do you want to take that so I can rest my vocal cords a little bit?
Yeah, no, good question. Appreciate that. The best deal for Poolbeg Pharma is really the one that yields the most value for the shareholders. We are, as Jeremy said, continuing to engage with the potential partners. We are fortunate in that the bits are nicely falling into place. We got the clinical data. We have the path to market now validated with the FDA. We have the commercial work done. So what we will be doing now is really beginning to push on and engage to see which path will yield the most value. I think we are somewhat agnostic whether it is one partnership, multiple partnerships. It is really about maximizing the value of what we have, which we think is quite an exciting package in POLB 001. Jeremy?
Well said, Ian. Could not have said it better myself, and again, appreciate the water break. Very good. Next question. Again, appreciate these coming in, and again, we will try to tackle as many as we can. Is Poolbeg interested in targeting other problem areas affected by CRS, such as COVID and sepsis? What other areas can POLB 001 potentially address, and is there a role to play in autoimmune conditions? Listen. Wow, we could spend quite a while talking about that. I think that the short answer is yes. We are very much focused on, as I say, the cancer immunotherapy-induced CRS. I think that is where we have our best knowledge. We have filed intellectual property. We do know, and I think a lot of people remember back in COVID where cytokine storms was also an issue. The sickest patients had cytokine storm.
The difference is that we're preventing something from happening with the cancer immunotherapies. Patients know when they're going to get their cancer immunotherapy drug, so it's a much more better-controlled clinical trial. But you could see potentially down the line in the past, giving it after the event, and say the cytokine storm has started. That's planned for the future, and of course, that could open up a whole potential other market area. I think somewhat ironically, some people may remember we started in the infectious disease space looking at severe influenza. But from a commercial and market standpoint, the unmet need in cancer immunotherapy-induced CRS is far greater, so that's where our focus is right now. Again, appreciate the questions. Autoimmune conditions, yes, we know there's inflammatory or autoinflammatory.
Again, a lot of work needs to be done there from that standpoint, but very focused on executing on a TOPICAL trial. Again, deal work, collaborations, partnerships, and then that's probably a discussion that we'll have with our partners down the line. Again, appreciate the question. Next question. Following the recent U.S. trip, Jeremy alluded to pharma having discussions with banks. Can Jeremy tell us a little more about this and what type of pharma companies? Again, appreciate the question. I mentioned earlier on, so we had some good discussions. Ian and I were at the H.C. Wainwright conference in New York last week. A lot of investors in the room. There was pharma companies present. There was biotech companies presenting. What we've found, and again, this came from a discussion we had with Michiel Bröker at UBS, is that they're in constant discussion.
The banks are in constant discussion with pharma companies about what they're looking for. What does pharma need? As I say, we showed the slide earlier on about the $400 bi llion that's falling off their balance sheets when it comes to patent expiries that they're concerned about. So they look to what companies are out there, what companies have impressive data, clinical stage, could fill a gap. So really good discussions around what pharma needs. Now we're lining that up, as I mentioned in one of the slides, about understanding that is important. Obviously we're having that dialogue as well with the big pharma companies who have the cancer immunotherapies. Again, it's nice. It's kind of fascinating. When they question what type of pharma companies, I would say all. Everybody has a hole to fill when it comes to patent expiries.
Our plan is obviously POLB 001 will fill some of that, the hole that's developing. Appreciate that. When is the GLP-1 oral study due to begin? I was speaking recently this week with our colleagues at AnaBio. As you know, it's their encapsulation technology that is encapsulating the oral GLP-1. Given orally, it's protected from the stomach acids and enzymes, and then it's released into the small intestine. There's discussions going on with the manufacturers, with the principal investigators about mapping that out. So we still have H2 on the critical path, the second half of this year to get that trial, moving that. Again, I won't ask Ian to comment on this, but we've had fantastic discussions with partners or potential partners late who are inquiring about oral GLP-1. These are large companies that are in the injectable GLP-1 space are looking for oral alternatives.
So we have, again, maybe you could say similar to POLB 001, we're kind of lining up interested parties before we have the data in hand, and then when we have the data, things will accelerate from there. Again, appreciate that. Next question. I guess this will go back to Liam, and I think we may have addressed this already. How many clinical sites are now live or planning to go live with patients recruited for a TOPICAL trial? Why are they appearing to take so long to go live? Okay.
Yeah, happy to take that, Jeremy. We announced earlier in the year there's going to be six sites. We already have a number of sites activated. There's two or three still to activate. That's very normal for a trial like this. They're always phased. What we try to do is we try to get the most engaged, motivated sites open first that are going to recruit most strongly, and then once they're open, the resource goes into opening some of those other sites. There's a combination of just a practical perspective that we want to get this recruited as quickly as possible. There's a little bit of strategy behind how we do that. It's not unusual with six sites that there's normally one. We are hopeful that everything's going to get sorted now in the next month.
I think that the momentum that we have here at the moment in this announcement, we have the investigators meeting tomorrow. This is competitive recruitment for the trial, meaning whatever site recruits them first, recruits them first. There's no quota for every site per se that'll be held back. It's whoever recruits first will conduct the trial, and everybody really wants to be part of this trial. All of the investigators, we've got a great relationship with them. They really want to open their sites as quickly as possible. We're really encouraged that this momentum is going to translate, and obviously, as those sites come on board, we expect the recruitment rate to get even faster as we go forward. We'll definitely keep you guys updated as that progresses.
But for today, we're really happy with the momentum we've had, and we continue to engage the investigators exceptionally well, and I think they're really enthused to be a part of the trial and the results to date, and this Safety Review Committee review. Obviously that they can continue the trial uninterrupted from a protocol perspective, again, gives them reason to be really enthused about the trial and want to get this done as quickly as possible. They know our perspective that there is, as I mentioned earlier, there's a quick path to approval here. Jeremy just was asked a question about other indications, and of course, this is an anti-inflammatory drug. There's lots of places you could say where that might be beneficial.
Obviously, it's not always in shareholders' interest for us to say all of that, because if it's an announcement later, but there's definitely Look, there's a lot of discussions that we're having here. You could even look at something like this if it was to get later as a product in a pipeline or the other way around. But definitely these are things that we think there's a lot of potential in this drug if we can get it through this gateway. We've seen the value proposition for you guys that we really want to position this optimally, but it doesn't mean that that's the full extent of where we see this drug going.
Really encouraged, and I think a lot of the investigators are sharing great ideas with us as well of how we accelerate this and how we get more done quicker and how we increase the value for you guys.
Yep. I like that answer. I like that answer. That's good. Again, I'm cognizant of time. We're 45 minutes in, but we'll keep going for a few more and we'll, as I say, try and answer as many questions as possible here. "What intellectual property do you have on encapsulated GLP-1, and can it prevent it from being copied?" Again, appreciate the question. Our collaborators, AnaBio, have filed and granted patents around the encapsulation technology. We mentioned in the past, they have marketed products in more of the nutraceutical space, encapsulating probiotics and iron and vitamins. That's kind of on the market. They believe they have a strong patent portfolio, as I say, with granted patents, and the GLP-1 is kind of a separate section.
I think when we started collaborating with AnaBio, the GLP-1 we used was off patent, so that means we could kind of freely use it. But I envisage that as we move forward with the programs that taking other peptides and encapsulating them, that creates new intellectual property as we go itself. And they have a large, even belong besides granted patents, they've got a lot of technological knowhow, secret sources almost that they can kind of protect and nobody else can copy because of their strategic approaches. So I think that's in pretty good shape for that. Next question again. "Considering today's positive news, can you give us a ballpark figure of what pharma company would have to pay to totally buy out Poolbeg?" That's a great question. That's a really good question. I'd love it to be multiples. As I say, obviously we're a public company.
The market cap is out there. There is obviously premiums come with a lot of these deals as well, and that is all part of the joy of negotiating. We have all negotiated strong deals in the past. I think what will drive this is the competitive tension I mentioned earlier on, where you will have a series or a host of companies who each want a technology and they can kind of start bidding against each other.
While we never write a number or announce a number because you do not want to draw a line in the sand, we will continue to drive the program forward to de-risk the program, and there is a direct correlation between de-risking and value creation, and that is the direction we are taking right now. Again, we will keep kind of pushing, and as I say, the ultimate goal is getting the best deal for shareholders.
As I say, we have pulled a lot of the aspects together of a deal to make it compelling. We will push hard there on the valuation of that. A question here, "Are there any other patent applications outstanding for POLB 001, and if so, which ones are they?" I think maybe this is referring back to the influenza story that we talked about, and I will say that we are kind of working on another aspect. Again, we won't announce, but we do see 001 having potential in other areas, and we are kind of drafting in that area right now. Again, the goal is, like Liam's kind of comment about the pipeline and a product. You can use the same drug for many different indications, and obviously we will see where we go from there. Again, appreciate the question.
When do you envisage" I think we have answered this already, the whole topic of trial be completed. From Mark O., "How many of the 30 patients are actually in the mentioned one to two months dosing trial process?" I am not sure I know. "How many of the 30 patients are actually in the mentioned one to two month dosing trial process, and when will the first patient trial result data be announced and released?" I think I understand. I mean, the trial has started. We have reached that interim. We do not announce individual patient data, although many of you might have said or might have seen. The Daily Mail, I think it was, did a profile of the first patient in, The Christie in Manchester, who had a very positive experience on a trial.
Again, the whole point here is, as I say, from a statistical standpoint, to take the data as a whole rather than individual and build it forward. As I say, we are making good progress, as Liam talked about recruitment. We are very happy with that and pushing on from there. Another question, Adam. "You continue to reference strong engagement from investigators in trial sites, correct? Can you elaborate on the level of interest you are seeing from the hematology community, and whether that has influenced site activation plans?" Liam, I know you kind of addressed some of that. I can comment that, I mentioned this earlier on, that as Emma started the ball rolling in reaching out to our hematology colleagues, we have got the pieces together. Once we got MHRA approval, then we had inbound inquiries, other investigators in the U.K. asking to participate in this trial.
Some of which we've done diligence on the sites. Some of them take longer to get up to speed, up to get the site approval, et cetera. But we've been pleasantly surprised by the additional sites that have come to us, and one of which we think will come on board relatively soon, all going well. We'll keep working, and again, that'll help with recruitment and pushing on from there. Question from Kevin. "What is the evidence that dampening the CRS response will not also impair the anti-tumor efficacy?" Really good question, and I'm going to get Liam to address that one. But that's a really clever question that we've talked a lot about.
What was the question? Sorry, Jeremy.
Sorry. Which one? The one up here. It was about dampening the efficacy of the anti-tumor.
Ah, okay. Sorry, I missed that. Obviously, that's a key piece of this development program. There's no point preventing cytokine release syndrome if you blunt the efficacy of the immunotherapy. So, it's a really good question. What I can say is we've done a lot of preclinical work around this. And obviously, it's a key question that investigators ask before you go anywhere near a patient. Obviously, these patients are late stage relapse patients. They need these treatments to work. They're not going to go on a clinical trial for CRS preventative unless there's a high level of confidence that we're not going to negatively impair that. Obviously, we're early in development, so we're phase II. There will be more evidence generated, so I'm not going to put anything definitive out there. But we're very optimistic that we don't think this is a major issue at the moment.
But look, that's a different question. That's a regulatory question. From our perspective, we started this program as well because we saw signs. We realized that this would be a really good fit as a preventative for CRS, specifically because there's actually a lot out there to show that if you inhibit p38 MAP kinase, you can dampen down cytokines without having any impact on T-cell killing, and that's a key part of the hypothesis. So if in T-cell engaging therapies, obviously you need the T cells to actually bind and kill those tumor cells, or in the case of a CAR T-cell therapy, the T cell is the therapy. So that's essentially important. So that's something that we've considered from the earliest days of this program. It's evidence that we have collected. We have presented some of it at conferences.
So it's something that we have answered before. I'd welcome people to look up our data on it, but we're in a strong position there.
Cool. Thank you for that. Yeah, really good question. Appreciate that. Next question. "Will POLB 001 be given as a single course prior to first anti-cancer treatment, or will multiple courses be given with each immunotherapy course?" Again, really good question. The design of the trial, we've kind of mapped this out. We've talked about it before. The patients receive, they take POLB 001. It's twice a day. You want to inhibit the target before you get the cancer immunotherapy. So you take the drug for several days in advance of the immunotherapy. And this actually works well because patients take it from home. Let's say on a Friday, they'll take the POLB 001. They'll take it twice a day, and then they'll come in on the following Tuesday or so to take the cancer immunotherapy. And then they stay on that.
As I said, they stay on our drug for another 10 days or so to keep the immune system calm and under control, while they're also receiving their cancer immunotherapy. So I think it's a balance, and then we're done. We say it's like just a short course of treatment for 001 to inhibit p38 MAP kinase for a short period of time, and then the cancer treatment goes and does its work. So, again, appreciate the question there. I think we've answered that one. Yeah. I think just around the number of people being dosed, as I said, we're happy with the progress. The results are there. We can't say specific numbers, but I think, as I say, we'll have the full 30 all done and dusted. As I said, the plan is in the first half of next year. So you can extrapolate backwards from there.
In order to access the U.S. market, would you have to carry out larger clinical trials in the U.S.? And how long will this take, months or years?" Again, really good question. Now we are looking at the phase III trial here. Assuming success of this TOPICAL trial, we have, and this is what we discussed with the FDA, what that phase III looked like, how many patients that it looks like. It will be, as I mentioned earlier on, a global study. We want to get it into as many patients but as quickly as possible. That was the feedback from the FDA. As I say, we have talked to contract research groups who run these clinical trials, so we have a good understanding of budgets and timelines.
Again, I would reiterate that we are only looking at that first 1-2 months of patients when they receive our drug, and then we are done from that patient's perspective. They will remain on the cancer drug, so that will go into a few years. I think that it is a very short period of time compared to other clinical trials, and I think that is what is very attractive from a partnering standpoint as well, because we could have this drug on the market in a relatively short space of time. Okay. Now my voice is about to go, but again, appreciate everyone who stayed online. I know we are way over time, but I did want to address as many questions as possible. "Who are your main competitors?" This is from Bruno.
Who are your main competitors in this field, and how far ahead do you think you are currently?" I will kind of start. Or do you want to? Liam can take that.
Yeah.
I will rest my voice a little.
Look, in terms of what is in development for cytokine release syndrome at the moment, there is actually an incredibly sparse competitive landscape out there, so we are definitely the preeminent piece. There are some things that are being used in the clinic, but there is no development pathway for them, so they are not on the way to an approval. So this, if the TOPICAL trial is able to push on, we are able to get positive results and push ahead with our program, we really will be the first preventative for cytokine release syndrome. There are a couple of things in the clinic, but they are all actually investigator-led clinics for the most part, unless I am mistaken. So being investigator-led generally means that there is not a financed company behind them to really drive them forward. Sometimes these are repositioned things, and that they might be available for a different indication, being used off-label.
But quite often, the company manufacturing those drugs is not necessarily going to push forward from the IIT. It can be done fully by the investigator. So from a company perspective of a development program with cytokine release syndrome, and again, this is because you need the right drug to combat cytokine release syndrome, that you need to be able to prevent cytokine release, and you obviously need to not impair the therapy, so have no negative impact on T-cells, and you need an exceptional safety profile. The whole hypothesis here of this drug is actually to transition this to an outpatient to at home. We can only do that if you have a drug with an exceptional safety profile.
There have been other things that have looked at this area before, like people looked at one of our slides that we commonly use as the cytokines, the POLB 001 inhibits compared to tocilizumab steroids, but the third one on that list is often Ruxolitinib, which is a JAK inhibitor. JAK inhibitors can blunt inflammation, but actually you get high levels of cytopenias, which means your immune cells in the blood go down really low, and then you are actually exposed to infection when that happens. So again, this is something where it could be effective, but particularly when you are looking at that preventative approach, those drugs are not suitable. So we have got a really strong position here at the moment to kick on, and that is why I think a lot of people are really interested in this program, and a lot of partners would love to be a part of it.
Super. All right, we will wrap it up there. Again, appreciate for those who stayed on. We did cover as many questions as we could. Again, happy to address questions offline if people have something that is on their mind or maybe something that kind of comes up after the call or after the discussion. Again, a great day for Poolbeg. Very happy where we are at right now from the TOPICAL trial standpoint. Great to get the go-ahead, as I say, from the Safety Review Committee. Staying on the same dose that we have, terrific. As I say, it is great for patients as well. So looking forward to updating you on more of our activity as it progresses. Again, I want to appreciate or acknowledge Ian and Liam's presentation aspects as well, because we covered a lot in this clinical trial or in this presentation.
Again, appreciate your time for that. But looking forward to updating you more in the future. Thank you.
Cheers.
Perfect. Thank you to the team.
Thanks
For updating investors today. Could I please ask investors not to close this session, as you will now be automatically redirected to provide your feedback. On behalf of management team of Poolbeg Pharma plc, we would like to thank you for attending today's presentation, and good-