Hello everyone. I'm glad to welcome Renée Aguiar-Lucander, CEO of Hansa Biopharma, a Sweden-based and listed biopharma company. Just a quick intro to the company itself. So its lead asset is the Imlifidase. It's an antibody designed to eliminate pathogenic IgGs that play a central role in transplant rejection and multiple autoimmune disorders. The product is already approved and commercial in Europe for kidney transplantation, and it recently met the primary endpoint for its phase three trial in the U.S. and is now planning for BLA submission before the end of 2025. Renée, the floor is yours.
Thank you. Hopefully you can hear me. Does work. Here we go. Welcome to this presentation of Hansa Biopharma. Just want to draw your attention, obviously, to using forward-looking statements in terms of referring to any filings that the company has made. So IgG, as was mentioned, so obviously we have a Hansa has really built around a novel first-in-class IgG cleaving platform. The lead product candidate, Imlifidase, as was said, actually is the only thing out there that can actually reduce IgG down to more than 95% from baseline within two to six hours of administration. It's a very simple 15-minute infusion. This drug, as was mentioned before, obviously has already got commercial approval or got approval from EMA in Europe. We just read out very successfully a phase three program here in the U.S. There is also a follow-up enzyme that's been named Hansa 5487.
We are going to bring that into one of the autoimmune diseases that we have previously run a phase two trial, namely in GBS. In terms of actually looking at this, this is just a very brief kind of summary of what kind of differentiates this type of platform from, for example, the FCRNs. As you can see, this really creates an immediate, really kind of like significant reduction of IgG. Obviously you will then have a kind of, it'll start, B cells will start reproducing kind of antibodies post that cleaving, but it cleaves it intravascularly, extravascularly, and it's a highly, highly targeted mechanism. It can really cleave through very, very high levels of IgG very, very effectively. The indications that we have target is basically desensitization and autoimmune diseases. In terms of desensitization, the first program there is really in kidney transplant.
This is a drug that will enable kidney transplantation of highly sensitized patients. These are patients that have very, very high levels of antibodies. It is very difficult for these patients to find an organ that is not a positive cross-match versus their profile. By using this cleaving technology, you can basically almost remove antibody as an issue for any kind of kidney transplant patient. The other area where we are looking at this is obviously to enable gene therapy. You have anywhere, you know, there is a variation in terms of the delivery mechanism for gene therapy, but most of them use AAV vectors. There are a lot of patients out there that have high levels of antibodies against AAV vectors, and therefore, even if they are eligible under label, they are not able to be dosed or included in a clinical trial for that reason.
We've also, companies also run two very successful trials in both anti-GBM, which is an ultra-rare kind of kidney-related disease, and GBS, which is a neurology-related rare disease as well. There is obviously been approved in Europe, and as I said, we've just read out a very successful phase three. We have a very experienced leadership team, so pretty much everyone that has a senior position in the organization, it has recently been restructured. I joined Hansa at the end of April of this year, and I have actually added to the senior management team quite significantly over the last several months. Everyone on this kind of team has experience in terms of launching drugs in the U.S. into the renal space, getting approvals from the FDA, et cetera. Let's focus a little bit on the transplantation market to start with.
There is a really high-end medical need with a very significant market potential here. This is truly a pioneering approach for this kind of patient category that previously really has had very, very few alternatives. In terms of these highly sensitized patients, they do remain on the waiting list for a very long time, and a lot of them do not necessarily even get referred to the waiting list. As we know, there are over 550,000 patients on dialysis in the U.S. The waiting list consists of 100,000 patients. Obviously, there is another kind of large number of patients who do not even figure on the actual waiting list.
In terms of this kind of organ, you know, overall kind of organ availability and the organ allocation system in the U.S. has kind of realized this and is actually kind of putting a premium on trying to get as many kind of organs, you know, offered to this type of patient population through the structure of the national organ allocation system. Despite that, it's a very difficult kind of situation for a lot of these patients. As we can see, as I said, there are about 100,000 patients on the waiting list today. If we look at those who are kind of defined generally as highly sensitized, those patients with a CPRA score above 80%. You will also see there are about 15,000 patients who are in that category today on the wait list.
If you then narrow it further, you have about 7,000 patients with a CPRA score above 98% and 3,500 patients with a CPRA score of 99.9% or higher. As I'm sure you can imagine, these patients will stay on this waiting list much, much longer than the average patient, and it'll be very difficult for them to find a matching organ with that type of antibody profile. In terms of the U.S. market, as I said, we have a very experienced team. This is a very kind of small, I would say relatively small kind of commercial footprint that we need to build. The target is certainly to kind of seek, as we've said, BLA to submit the BLA before the end of the year. We will ask for priority review.
If we get that, obviously we'll be looking at a potential approval at the end of August of next year. This is a tight call point. There are only about 100 centers that represent about 80% of all kind of adult transplants in the U.S. The phase three trial that I recently mentioned, 25 out of those centers were part of that phase three trial. Half of all of those kind of centers have substantial experience at this point in time with using the drug and understanding what it can do for patients. We believe that we need maybe 15-20 salespeople really to cover this effectively. It's a very medical affairs-driven, obviously kind of in the beginning.
This is obviously also why we have brought on board, you know, very significantly, very kind of experienced people, both from market access and medical affairs to help drive that kind of preparation of the market. In terms of, this is obviously an inpatient procedure. We would be falling into the DRG category. We would be following on from kind of good experiences already kind of paved the way for us here in terms of CAR-T, for example. We would apply for an NTAP, and we will then move to our own kind of DRG code. During that time, there will be some outlier payments from hospitals, and we will be able to apply for that NTAP next year when we are in review for a potential approval. We have not set a price in the U.S.
We are going to conduct some pricing research here, just as a kind of comparison. In Europe, obviously, as I said, the drug is commercially available. In Europe, companies do not set pricing independently. It is done in negotiation on a country level. These countries will run health economic analysis to establish what price would be either kind of cost-beneficial or potentially cost-neutral for them in that country. On the basis of that analysis, price points in Europe, the list price is about $300,000-$350,000 per treatment. This is the recent kind of phase three study that we read out. I think that this was basically a, the FDA really wanted to have a control arm in this trial, which is quite an unusual setup in terms of how do you do a control arm for a transplantation population.
Actually, there were 64 patients in all one-to-one. The primary endpoint was eGFR, so kidney function at 12 months post-randomization. We've looked at that in a variety of different kinds of statistical models. What you can see is that obviously what happened in this trial is that all the patients in this trial were on dialysis, they were highly sensitized, and they came in obviously then all as they came into the trial, you basically, they delisted some of their kind of antigens. You made them look as if they were less highly sensitized than what they were. This would enable all of them to actually open up that kind of organ allocation kind of probability. All the patients actually had an organ offer at the time of randomization. They were then randomized either to active or control.
All of them had that, obviously, just because you changed their kind of synthetic profile did not mean that they had less actual kind of antibodies. The choice in the control arm was the FDA had allowed the physicians there to basically use whatever was on the market whenever they wanted, however they wanted. Actually, it was a very, very broad and allowed control arm. This is really because, obviously, the FDA wanted to give these patients any kind of opportunity to get a transplant as part of this trial. Despite the fact that that was a definition, you can see that less than 10% of the actual control arm could go ahead with an actual transplant, despite the fact that they had been on the waiting list for a long time, were on dialysis, were highly sensitized.
That I think really speaks to the really significant unmet medical need of this patient population. This trial, I believe, actually shows that there's very little options available for these patients today, apart from obviously waiting for, you know, a match that is as kind of close to a natural match as possible in order to limit the risks of proceeding with a transplantation for these patients. As you can see, the p-value for this trial was 0.0001. That again, I think, reflects both the kind of robustness of this trial and also the significant market opportunity and really significant medical unmet need that exists in this patient population. We've also shown from a long-term perspective, so what is a long-term outcome from the patients who are actually then used Imlifidase for a transplantation?
As you can see, this is a kind of a pool of all the kind of phase two trials that actually will tell you that actually the long-term outcome is virtually identical, whether you actually use Imlifidase and you're therefore able to implant kind of a positive cross-match organ, or if you actually just are lucky enough to find a kind of a matched organ, a negative cross-match. Europe, in contrast to the U.S., EMA and Europe took a different view in terms of how to bring this product to market. In Europe, there was basically a small phase two trial upon which EMA decided that because of the significant unmet medical need, EMA was willing to actually give a conditional approval very early on and actually have the phase three trial run after kind of a conditional approval.
In Europe, there was quite a limited amount of exposure and kind of experience from a KOL perspective. As we know, Europe is a fairly kind of fragmented market in itself. A lot of, you know, different nations obviously have different rules. They all have different organ allocation systems. This patient pool, as we've just shown in that phase three, was obviously a patient pool that physicians do consider is a difficult patient population to deal with without having something like Imlifidase at hand. Despite all of these issues, which I think are kind of inherent to the European market to some extent, on top of that, obviously the company has been running a phase three trial, a large phase three trial enrolling 50 patients. There is no control arm in Europe. EMA did not find that that would be appropriate.
Those 50 patients, that trial was enrolled, fully enrolled in Q1 of this year. That will read out in the middle of next year, that kind of full, and that will become the basis for a full approval application in Europe. Despite having these kind of issues in the backdrop, we're certainly kind of seeing, you know, continued commercial traction in Europe. Nine months to date is about $15 million of revenues. We see a continued kind of growth. I believe that with the phase three data that we've just reported out, this is really the first kind of large control data set that will become available to physicians in Europe.
I think also the fact that they're now obviously enrolled and have seen some of that benefit from the phase three trial that they've been running, we're expecting Europe to kind of have a significant, you know, commercial growth, certainly kind of towards also through 2026 and 2027. What we're doing is we're certainly kind of going through and looking at, as I said, we've kind of brought on board some more senior people in the team as well that have a lot of experience also from European commercialization. We are addressing some of the things that we believe we can do fairly quickly in terms of system, you know, systems and real kind of follow-up KPIs, et cetera, to further accelerate the European growth trajectory. In terms of gene therapy, obviously it's a compelling opportunity.
There are lots and lots of kind of activity in gene therapy area as a whole. As I said, obviously from that perspective, we have a couple of partnerships. This is not an area where we will run trials ourselves. We do this in partnership with companies that have existing either kind of clinical trials or commercial products in order to kind of look at. We have actually in the last couple of months generated the very first clinical data from these partnerships. What we have shown is that with Imlifidase we are able to again cleave through, you know, over 95% from baseline and to bring these kind of treatments into line in label so that all of these patients that are eligible under the label can actually be treated with that gene therapy.
The antibody profile of those patients should not exclude them either from a clinical trial or from an actual kind of commercial product being available. With this actually on Genethon, this was also a long-term follow-up with an ultra-rare indication called Krigler-Najar. This is something, an inability to break down bilirubin. That patient has now for a long-term follow-up been kind of off light therapy ever since dosing. This patient would've been excluded from any kind of treatment had it not been from a treatment with imlifidase. Moving on a little bit to autoimmune diseases. These are IgG mediated autoimmune diseases that the company really is focused on. There have been very successful phase two trials that have been run both in anti-GBM and in GBS, Guillain-Barré.
What we've known is obviously we have an anti-GBM phase three trial, which is reading out in Q4 of this year. As you know, there's not a lot left of Q4, but we will read out before the year end. That is our second phase three that we will then read out this year. Anti-GBM is rare. It's an acute inflammatory disease. It is very much driven by IgG. This is really kind of a disease where the vast majority, if you look at the historical kind of outcomes for these patients, there's nothing approved today. What you'll find is that, you know, generally 80% or more of these patients historically will lose their, will have lost their kidney function within six months of being actually diagnosed.
This is certainly an area where there was a phase two study that was done so we could look at instead of 18%, which was the kind of historical comparable, the actual 65% of the patients in this trial retained their kidney function six months after diagnosis. A very significant difference. There is a phase three trial, 50 patients that is ongoing. Primary endpoint is eGFR at six months and dialysis independence. What we're looking for, of course, is to read this out in, as I said before, the end of the year. We would look to most likely, subject to positive data, file an sBLA for this. This again is obviously a nephrology kind of oriented kind of call point.
For us, this would really be kind of a second product that we could roll out with the same kind of infrastructure in the U.S. to have kind of actually a follow-on product very kind of rapidly after our potential kidney approval. The second area where the company has been running clinical trials is in the area of Guillain-Barré. There was a phase two study, just under 30 patients, also an open study, which really saw a very rapid overall improvement in functional status. These patients, obviously, it is a neurological peripheral kind of neurology disease, but it can also result in just paralysis, but also kind of breathing problems. You have to have mechanical ventilation a lot for these patients. Again, there is nothing approved for this kind of patient group today.
What we saw in the actual open trial is that there was a significant improvement in terms of patients being able to walk independently, leave the hospital, not end up in mechanical ventilation, et cetera, in that trial. Actually, this was presented in a European conference a couple of months ago. There was a lot of KOL support. I think this is not an ultra-rare disease. It's a rare disease, but not ultra-rare. I think there's a lot of interest from KOLs and we've had a lot of interest, I would say, to try to move this forward and actually provide a solution for these patients.
This one is where we have just recently decided that we're going to go forward in this disease with our follow-up enzyme 5487. This is a non-human derived kind of enzyme in terms of a host. It's been through phase I clinical data. We have quite a lot of safety data on this enzyme. It has a very similar effect to imlifidase, still very kind of robust. It's probably more durable, more potent. It's far less immunogenic than what our lead product is. This is the one that we're going to hopefully have interactions with the FDA in the first half of next year with a view to having a fairly, hopefully fairly accelerated program in GBS that we can then launch next year. On this basis, in terms of financials, as I already mentioned, obviously we have commercial sales in Europe.
I would expect the European business on a standalone basis, including all of the kind of regulatory and other kind of costs, to actually be cashflow positive next year. We do have a very good reimbursement, very broad reimbursement in Europe. Obviously, on the basis of this phase three data. I think the actual kind of clinical experience that's coming from them participating, there are about 23 sites in Europe participating, mainly academic institutions all across Europe participating in this phase three trial. I think that that, as I said, is going to read out next year. I think we're seeing a lot of interest, you know, commercially now as that kind of trial is fully enrolled. In terms of cash, we recently did an equity raise.
Q3 we had about $25 million of cash on the balance sheet, but pro forma, that is about $90 million of capital at the end of Q3 with a capital raise that we closed on October 1. This cash will bring us in, you know, well into 2027. It'll get us past our filing, hopefully our approval and commercial launch in the U.S. as a consequence. In terms of the pipeline and some of the kind of actual catalysts that are coming up, obviously we have the anti-GBM phase 3 data coming up in December. Also we are going to file or the target is certainly file our BLA before the end of the year.
We will be asking for priority review in terms of then sometime hopefully in February timeline, we would then hopefully get a BLA acceptance and that would provide us with a PDUFA date. If we do get priority review, that would be at the end of August that we could expect a decision. We also have targeted the ATC as a place to actually provide the full phase three data of the Confidus trial, which will take place in June of next year. We will also have five-year European study follow-up that we also hope will be published shortly that can also be then presented. The Genethon phase two trial, the Krigler-Najar, will be hopefully they can get the, all the three patients, they can report at interim data on all of those patients.
We also then hope to have clarity from the FDA in terms of the design of this kind of, of the GBS program that we intend to take forward. In the second half, this is when we would then launch into the U.S. and this is also when we would expect the phase three in Europe to read out. This would be the basis for filing for full approval in the EU. We would also then be able to file for an sBLA in the U.S. as a follow-on to an approval here. We could also then in Europe move forward post a full approval there. We could do variation that would also include anti-GBM. With that, that really is the end of the presentation.
I think I have five minutes left in the case that there are any particular questions. Otherwise, I thank you very much for your attention. Yes. How relevant is the European trial readout for commercial? Is there an inflection point then in Europe? Also, is there any particular learnings from European commercial so far that might then be something to think about in the U.S.? Absolutely. I think in terms of the learnings in Europe that we've kind of had is that, you know, really working, having that kind of KOL experience with the drug and having that kind of community understand how this works and having had kind of experience is very important. I think that what we have learned is, you know, we have to take that and be very medical affairs focused.
I think also focusing on things like guidelines, you know, putting guidelines in place, having that kind of collaboration with KOLs. I think in the U.S. making sure that, you know, patients are aware of the fact that this actually kind of exists. I think that those are certainly some of the learnings. I think that there are a lot of differences between Europe and the U.S., both in terms of organ allocations, how that works, how that system works, which I think we can't really kind of draw a lot of conclusions from or a lot of help from. I do think the fact that, you know, the U.S. has had so much more and longer exposure and experience with this drug than what has ever been in Europe.
I think it's really about kind of having everybody understand really, you know, this is a 15-minute infusion. You take a very simple test in terms of saying, you know, has this turned from positive cross-match to negative cross-match? You can then proceed with the actual kind of transplantation. You actually post-transplantation, you will treat these patients with kind of a normal autoimmune suppression regimen as you do with all transplant patients to ensure that, you know, these antibodies, you know, don't kind of, you know, go out of control. I think that really kind of having that understood and having the delisting process understood, that I think is another learning that we know that this is very much of an account selling.
It's very much of kind of getting that kind of team in and around the kind of transplant surgeon to understand, you know, the delisting, you know, the, you know, how does this work? What do we do? I mean, I think that is important. That I think is something we've definitely learned from Europe. I think in terms of the PAS, I would say that actually, you know, if you ask physicians what they want, the answer is always more data, right? Do you have more data? Do you have more clinical data? I think that the benefit will be that obviously we'll have that June, hopefully that June presentation of the full phase three. Because another thing that I've certainly learned is that physicians don't really listen to kind of company, you know, presentations or read press releases.
They want to do things in kind of, you know, conferences, congresses and, you know, peer review journals. I think that that will be really important. I think that the readout of the PAS will be important, particularly for Europe, because I think this is when you're really going to see, you know, a complete picture rather than the kind of each nation might only have like two or three patients. I think having that whole group of 50 patients, which is a very significant number, will be really kind of impactful for the European transplant kind of community or transplant surgeons.
I do think that in the U.S., because of the fact that there has already been a lot of experience with this drug and they've been kind of running the phase three for the last two years, I think that'll be a, you know, it'll all, you know, again, more data is always better. I think that they will absolutely be very curious and they'd love to see that data. I think that the U.S. is kind of ready to kind of go already now with the dataset that they have themselves. This will be kind of an added benefit, but I'm not sure it's going to be as much of a trigger as it's probably going to be for Europe. Yes. Hi.
Is there going to be a certain CPRA that's going to be on the label or that doctors are going to look at to see if it's well enough for? The second question is, is it the same drug as that's looking at different?
In terms of the label, I'll get back to you on that when I've had that negotiation with the FDA. I would say that actually the, you know, we of the trial was run in 99.9% patients in the most, you know, severe patient population. Actually in Europe, the label just says highly sensitized, right? A lot of those patients were actually kind of 98% and above. This drug is obviously not a drug that's kind of dose dependent, right?
It doesn't necessarily matter whether you give it to a patient that has 99.9% or 89.5%, or it, you know, it will cleave with the available IgG that's there. I think from a kind of dosing safety security, I mean, I don't think that physicians are going to be concerned about whether they use it in patients that have 99.9% or 98.2%, or, you know, I just don't think that that's going to be part of their, the way that they think about this. I also think that CPRA scores is something that does vary. I think let's, you know, that it's not necessarily a constant number. I think that, you know, over time as this, you know, gets approved, it will be a combination obviously of what's on the label.
Also, I think in terms of medical practice, what do the different, you know, how confident and how, you know, which patients do really the physician believe should be treated with this? Because as we know, I mean, a lot of people have been on dialysis for a long time, that, you know, you run out of veins, you run out of options. I mean, you run out of actually you can't, you can't be on dialysis any longer. I think that there will be kind of some of those decisions in terms of which patients do these transplant surgeons believe are the best, you know, is the best profile for this. It is the same drug. Yes. So we're at around.