Good morning, everyone. Welcome to the 2026 J.P. Morgan Healthcare Conference. My name is Varun, and it is my absolute pleasure to invite on stage Hansa Biopharma, represented by their CEO, Renée Aguiar-Lucander, and to talk about the company and their vision moving forward. Please put your hands together for Renée.
Welcome, everybody. We're going to walk through a presentation that has some forward-looking statements, so I refer you to our filings, including those related to risk factors. So in terms of Hansa Biopharma, we have a novel, unique, kind of first-in-class IgG cleaving enzyme. It's a proprietary technology platform where we have two different kinds of enzymes that we've generated from that platform. It is an incredibly targeted, effective, and efficient IgG cleaving enzyme. It basically cleaves all classes of IgG intravascularly, extravascularly, and basically reduces it to less than 5% of baseline within two to six hours. In terms of benefit, obviously, this is really kind of in order to address kind of acute or serious kind of immune-mediated diseases.
And as you can see, this kind of profile is radically different from some of the FcRn inhibitors, and it really is looking to kind of address things like desensitization, for example, of highly sensitized patients who are on the waiting list today to receive a kidney transplant. Having a very, very broad and significant kind of antibody profile, these patients have a really hard time ever getting a natural kind of organ match, and it's a significant unmet medical need. We've also kind of generated clinical data in gene therapy, where we're looking to basically cleave antibodies related to AAV antibodies to basically enable those patients to either be included in clinical trials or broadening kind of commercial access. In terms of our kind of second- generation, we're looking to really take that into autoimmune diseases and neurology.
And so we're looking to have a late-stage trial initiated, hopefully this year, in GBS, Guillain-Barré syndrome, where we previously have run a successful open-label phase II trial with Imlifidase. The actual compound is conditionally approved in Europe. We have a commercial operation in Europe, and so we are selling this product, but also at the same time actually running a phase III trial. That phase III trial in Europe was fully recruited in March of last year and will be reading out basically kind of mid this year. And the sales generated up through nine months of 2025 was around $15 million, and we would expect this commercial franchise to be cash flow positive this year for the company. In terms of the U.S., we had a phase III, which we read out in September of last year.
I'm going to talk about that in a little bit more detail later on in the presentation. But we submitted a BLA in December, and we would expect to have hopefully an acceptance and a communicated PDUFA date in February. We have asked for priority review. We have fast track, and there's obviously an orphan indication, and there is nothing approved for these patients today. We are listed in Europe on the Swedish Nasdaq exchange, and we recently raised about $70 million on the back of the phase III results in September. So we're going to talk specifically about the transplantation market of kidneys. Obviously, as I mentioned, it's a very high unmet medical need with a significant market potential. Actually, these highly sensitized patients, they're on the wait list for a very long time. It's very difficult to find a matching organ naturally.
Even if, in the kind of U.S., they are actually given what you would say extra points in order to kind of get them high up on the list and actually therefore get a significant number of offers, it's still a very, very significant challenge for these patients to actually kind of find a matching organ. And there is certainly a need for something that is consistent, predictable, efficacious, and safe in order to enable these patients to be transplanted. In the U.S., the wait list is around 100,000 patients. The definition of highly sensitized is approximately anything with a CPRA score of 80 or above. And if you look at those patients that have a total of 15,000 patients in that category, and if you look at those patients that have a CPRA score of 98% or above, there are 7,000 of those patients on this waiting list.
What does it really mean to have a CPRA score of 98? That means that you have a 2% probability of ever finding an actual matched organ through the kind of organ allocation system. Obviously, in terms of having a CPRA score of 99.9, you can imagine that the probability for these patients is quite elusive to find an actual natural organ. In terms of transplantation, this is a transplant list that keeps on growing over the year. There is addition, obviously, to the wait list. Actually, every year, there are about 10,000 patients who fall off this list, i.e., they are no longer candidates for transplantation because of compromised state that they're in, comorbidities that they have developed on dialysis, or they basically have passed away on dialysis. Out of those 10,000 patients every year, 25% of those are highly sensitized.
That means that 2,500 patients today are basically every year either dying or getting to the point where they can no longer be transplanted. The median wait time for these highly sensitized patients, that category, is seven years. That means that 50% of these patients wait longer than seven years. The actual kind of projected survival rate for patients on dialysis, the five-year survival rate is considered to be around 40%. So you can imagine that these patients, from a quality of life as well as obviously from just a pure kind of survival, this is why there is a very significant unmet medical need in this patient population. In terms of the actual kind of, this is a highly concentrated call point.
There are about 200 transplant centers in the U.S. that carry out adult transplants, and about 100 of those sites represent 80% of the volume of all transplants conducted in the U.S. The phase III trial that we read out in September of last year, we had about 25 sites participate in that phase III, and that represents about 25% of the overall market opportunity, and so in terms of kind of our building up for kind of a commercial launch, we will launch this product ourselves in the U.S. Obviously, due to the fact that this is a very kind of targeted and concentrated call point, we will only need about maybe 15-20 actual kind of account managers to address this market, so it's highly efficient for a company of our size and scale, and this is obviously also quite a scientific account kind of driven sale.
We have a lot of medical affairs and market access, field-based staff that we can actually deploy in order to kind of start preparing the market for understanding the profile of this drug. I did mention that we just read out a phase III trial. This was a trial involving 64 patients in total, one-to-one. The control arm in this trial was actually highly interesting. Basically, all patients that were recruited and randomized into this trial, at the time that they were actually recruited into the trial, what happened was that you delisted some of their antigens because obviously this is, per definition, a population that does not get organ allocations easily. By removing some of those antigens, you are now kind of presenting a profile of these patients that are more likely to actually receive organ offers.
Now, obviously, this is a bit of a synthetic way of getting an organ offer. And so obviously, once those organs have been received, they are still cross-matched positive to that actual patient. But all of these patients in the trial had an organ offer at randomization, and so they were then randomized either to the Imlifidase arm or to the control arm. In the control arm, the physicians were able to use things that are kind of generally available, such as IVIG, Plex, rituximab, et cetera, eculizumab. So I mean, there was a range of kind of options for those in that control arm. So what we've actually seen in this trial, which had the primary endpoint was eGFR 12 months after randomization. So that's obviously kidney function at 12 months after randomization.
What we've seen is, despite obviously this being a highly motivated patient group, all of these patients had basically a CPRA score of 99.9, they'd obviously been on dialysis, highly motivated to try and get a transplant. They all had an organ offer at randomization, and what we saw is that only three out of 32 patients, their physicians and patients decided that they would risk going forward with a transplantation without the Imlifidase approach. While in the Imlifidase arm, the vast majority of patients obviously proceeded with the transplantation, leading to that p-value of 0.0001, so what this shows us is obviously that today, the options for this patient population is extremely limited.
There is really nothing out there today that would give physicians and patients the comfort of knowing that this is consistent, predictable, efficacious, and safe to use in order to enable these kind of transplants for this patient group. There was also a secondary endpoint in the trial looking at kind of dialysis dependency at 12 months, and that obviously also strongly favored the Imlifidase arm with a p-value of 0.0007. The trial showed good tolerability with consistent safety profile to other kind of trials that have been conducted. And in total, there's been about 11 clinical trials now conducted with this agent. Importantly, obviously, it's not just to be able to facilitate the transplantation, but obviously also the long-term outcome for these patients. There have been follow-up of these patients in the phase II trial, and what that shows is actually a durable graft and patient survival.
Where actually that outcome in terms of transplanting a cross-matched positive organ into these patients is virtually identical to the outcome of those patients who find a natural match. And so this is another kind of data point that's obviously extremely important that goes to kind of long-term clinical benefit for this patient group. As I mentioned, in terms of Europe, so this was the company had conducted a phase II trial, open-label, quite small trial, 16 patients. They took that to the EMA, and actually EMA did recognize that this was a high kind of unmet medical need and gave the company a conditional approval at that time on the basis of that trial. That phase II trial was majorly kind of like really carried out in the U.S. There were really only two European sites that were included in that.
There was a very limited experience, kind of clinical experience of this drug in Europe, while in the U.S., actually, we have far more clinical experience in the kind of transplant community. But at the same time, EMA wanted to see a phase III trial. And so there is a phase III trial that's been ongoing in parallel with any commercial efforts in 22 centers across Europe in all of the major kind of academic centers. There is 50 patients who were included in that trial to be transplanted. That does not have a control arm, but rather basically is looking at kind of contemporaneous kind of outcomes across those same kind of centers in non-highly sensitized patients. This trial was fully recruited in March of last year, and this will be reading out, we'll be reading out this data in the middle of 2026.
So we're really kind of the company is really generating a significant amount of clinical data and proof, if you can imagine, 64 patients in a randomized control trial, 50 patients transplanted in Europe, and there's also preparations and drafts for a significant publication of around 30 patients' real-world experience and outcomes that's going to come and also into the publication realm looking at kind of that experience in Europe, so this really, as you can imagine, what we've also seen in the phase III, there's quite a pioneering approach for this patient pool. Generally, this is a patient pool that without imlifidase obviously is considered extremely challenging, difficult for physicians to deal with, so what has been happening in Europe, obviously, is that the phase III has been ongoing, but also there's been a lot of work on reimbursement.
This drug is now reimbursed in 20 countries across Europe. The average list price of this drug in Europe is around $350,000. This obviously, as you may know, this is not a price that's set by the company. This is based on health economic analysis by the various kind of countries, and so that pricing of $300,000-$350,000 per treatment really reflects kind of a cost-neutral or cost-benefit that's been assessed by those various countries. There's also been a fair amount of work that's been taking place on drafting guidelines because, again, this is really a patient population group that's not really had a lot of other options, and so there's also guidelines that are continuously being worked on, drafted, and published across Europe, and Europe obviously is quite different from the U.S. It's quite fragmented. You have a lot of variations.
You don't have a centralized kind of organ allocation system. So there are clear kind of regional, local kind of complexities in Europe. But I do think that now that we're actually getting significant kind of clinical data being presented and also the clinical trial being concluded in Europe, that will see a very significant kind of increased traction in terms of the commercial performance in Europe going forward. We would obviously file for full approval with EMA on the basis of that data reading out in the middle of this year. We also saw some clinical data coming from gene therapy this year. So obviously, gene therapy doesn't really matter what you look at in terms of gene therapy. There are lots of different estimates out there in terms of how big this market is going to be.
We're not going to sit and predict what that's going to be. There's a lot of different options. But what we do know is that there are a lot of clinical trials ongoing. There is a real kind of unmet medical need. And we're working with a variety of partners in order to actually look at kind of cleaving AAV-related antibodies to enable dosing of gene therapy in patients who otherwise would have been excluded. We have a partnership with Sarepta, and we have a partnership with Genéthon. And in September- October of this year, we could present clinical data from both of those. So these are two different vectors, two different tissues. And what we could show is actually that Imlifidase very successfully cleaved through high titers, bringing that down again to less than 5% of baseline, thereby enabling dosing of these gene therapies.
And also, kind of, we are now, obviously, these companies are following these patients long-term. And I think, on the basis of that kind of very supportive clinical data, this is an area that we will be focusing on this year in terms of actually increasing the kind of conversations and discussions that we have in the gene therapy field with a variety of kind of gene therapy companies with a view to hopefully bringing about some additional collaborations in that area going forward. I did mention we have a next-generation compound, which we call 5487. This is an enzyme from a non-human host. And what we've demonstrated in kind of a phase I trial is significantly lower immunogenicity, again, very similar kind of rapid and robust IgG reduction.
And what we are going to do, as I mentioned, is really kind of interact with the FDA in the next kind of month or two in order to really look at GBS. So GBS is a rare disease. It's a neurological disease. It's called Guillain-Barré, and it's really kind of a peripheral nervous system-related disease. So these patients actually get paralysis, difficulty walking, and it can also get to the point where you end up having to be on ventilation. So this is, and in this rare disease, there is actually nothing approved today. We have run a phase II with Imlifidase in this indication, and we saw a rapid overall improvement in functional status, including expedited muscle recovery. And we saw 37% of patients able to walk independently already at week one, and 67% of patients able to walk independently at week eight.
We also looked at this, but this was an open-label trial where we had the opportunity to actually kind of look at this in a matched kind of in a cohort from a database called IGOS. It's an extremely well-reputed kind of database collecting data from Guillain-Barré patients, and what we could see there is actually that we had a statistically significant improvement over kind of the standard of care that we could at least detect in that database, so what we are hoping to do, as I said, is interact with the FDA and get minutes, hopefully in Q2, and with a view of moving forward towards the end of this year with a late-stage clinical program in order to kind of be able to address this population.
In terms of kind of commercial traction, financial highlights, as I've already mentioned, obviously, we have a commercial operation in Europe that is generating revenues. Through nine months, it's about $15 million. That is equivalent to what we did for the full year calendar year of 2024. As I said, we have reimbursement across all major countries in Europe. We are seeing a large number of kind of reutilization of clinics in Europe. We have about $93 million of cash at the end of Q3 following the cash raise that I mentioned that we conducted on the back of the phase III data. We had a very successful addition of well-known investors coming into the cap table at that raise. These are some of the kind of milestones that we can see going forward.
So obviously, we are looking for targeting February for BLA acceptance and a communication of a PDUFA date. We're going to get feedback, obviously, in the GBS clinical development program. We're going to read out the top-line data in the European phase III trial. And in June, there is an American Transplant Congress that's going to be held in June between the 20th and the 24th of June. And this is where we're hoping to present the full phase III data from the US Confides study. We're also looking to file for full approval, obviously, with EMA in Europe. And as well as then if we do get priority review and we successfully get an approval, we would expect that to take place in August of this year, enabling a commercial launch in Q4. So these are really some of the programs in a pipeline.
We also have some investigator-led studies ongoing in ANCA, and so obviously, this is what we're hoping to also add to this in terms of having some additional collaborations in gene therapy going forward. In terms of the team, we've been very lucky to bring together a team that recently actually has been launching products in the renal space in the U.S., so we have a highly experienced team with very relevant background, both from kind of transplant as well as kind of the renal space, and so we're very excited about what we're going to be able to see this year. It's going to be a very important year for the company with lots of kind of data readouts and lots of activities, which we are looking forward to, so that concludes the presentation. Thank you very much for listening.
Let's see if we have any potential questions from anyone.
Thank you so much, Renée. We actually have a couple of questions coming in online. There's a question specifically about the U.S. KOLs. Can you comment on the response Hansa has had from the U.S. KOLs based on its phase III Imlifidase trial results?
Yes, absolutely, so actually, we did kind of conduct a webinar towards the end of last year following the kind of phase III data with a couple of KOLs here in the U.S., and I think that what was really kind of revealing from that is really kind of how they look at this as saying, they see these patients all the time in their clinics, and they really feel like when these patients come up to them and say, "What can you do for me?" They really feel like they don't have an answer for these patients today, they have been extensively trying to use things like IVIG, Plex, even in living donors, etc., and they've kind of published some of those results, and they say that the outcomes are not really helpful for this patient population.
So they are extremely excited about having something that has been so kind of well-tested in a variety of situations. And obviously, they have also been part of a lot of the phase II trials and have the ability to really follow these patients long-term. So I think there's a lot of excitement really that we're getting from the U.S. community. And we're also getting a lot of requests at this point in time for compassionate use. And we're also getting a lot of kind of requests in general, I would say, from kind of other transplant situations. So I think that it's really a confirmation of the fact that there is a significant unmet medical need out there. And I think that we're lucky to have some of those kind of KOLs be great supporters of this kind of drug development program.
And just a quick follow-up to that. You mentioned the pricing in Europe being around 300-350 for the drug. But could you comment on the pricing in the U.S. and the market opportunity around that?
Sure. So as I mentioned, obviously, the kind of number of patients that are on kind of basically considered to be highly sensitized is about 15,000 patients on the wait list today. In terms of the actual pricing in the U.S., we are conducting pricing research at the moment. So we have not kind of concluded that. And obviously, we will communicate a pricing upon potential approval. But obviously, what we've seen is the way that, as I mentioned in Europe, obviously, they would look at this in terms of health economic benefit. And as we know, cost of dialysis in the U.S. is very substantial. And these patients really stay on dialysis for a very, very long time and also therefore develop other comorbidities, etc.
I think that this is something where we feel quite comfortable in terms of the health economic benefit that this drug could bring to the system. But in terms of how it will be priced here, we're going to have to wait for the outcome of the pricing research. We'll obviously kind of communicate that in an appropriate time.
Before I go to the last question from online, is there a question from the audience in the room?
Hi there. Well, congratulations on the GBS data. I think it's really exciting. We had read about that before and also seen a lot of excitement reading about it. If you compare it to previous treatments in GBS, so congratulations. The question is, I guess, why hadn't you gone there earlier? It's such an exciting, it's like a really nice application. And yeah, just your thoughts on whether there were scientific or clinical reasons before?
Right. So it's an excellent question. And I think that I joined at the end of April last year, so I don't have the history, unfortunately, of kind of why some of these decisions were made or not made. But I would agree with you that it is a very kind of, it's kind of like an obvious kind of target, I think, for this type of kind of profile for the drug. There was obviously a phase II trial that was run. I think that, as in many kind of pharma companies of this size, there's always a matter of kind of like how much money does the company have in order to be able to kind of advance several kind of clinical programs in parallel.
I think my assumption is really that it's really kind of a question of having sufficient resources in order to bring all of those kind of clinical programs forward in a reasonable time in parallel. But I agree with you. It is exciting.
Can you talk about the dosing duration for the transplant indication? What safety questions do the KOLs tend to ask? And how does your long-term data reflect that?
Sure. So the way that the actual dosing happens, actually, when you actually have an organ, let's say you've gone through the process of delisting, you have an organ, you have a patient, this really is a 15-minute infusion, which will then have that effect of really kind of cleaving IgG very effectively within that kind of two to six-hour period. So what happens is the physicians will dose the patient with Imlifidase and will then kind of, there's a rapid test that's available that you basically just make sure that you've taken that kind of organ from a cross-match positive to a cross-match negative, after which time you actually then can proceed into the transplantation. There is an opportunity to obviously give an additional dose if that would be required.
You can obviously redose within that kind of period of time or those days, even that the actual kind of low levels of IgG exists. There is an opportunity to do that. We've only seen that in about and this is across all kind of clinical trials, I would say. Also in real- world, I think we see that that might happen in 10% or less of the cases. There would be a kind of a perceived need by the physician to rather kind of do a second dose. That's clearly kind of a possibility. What we've seen, and this is actually that it's a highly targeted effect. There's really no kind of off-target issues. This drug really only does one thing, and it does it really effectively, which is cleave IgG.
The risks associated with this drug is obviously there's really no issues on tox or those kind of issues. It's really a matter of like the risk here is obviously what you're doing is you're bringing down your immune system quite substantially. So you do know that that's kind of the inherent kind of risk in terms of what you would be looking at is to make sure that you might have some kind of, we have some prophylactic kind of upper respiratory antibiotics being administered during those kind of days, etc. So that's really kind of the well-known kind of potential concern that the physicians would have to just be aware of, that that's the function of this kind of dosing. But obviously, that's also what enables the actual kind of transplant to take place or gene therapy to be administered. But that's really kind of what we've seen generally.
And I think that's what physicians are aware of. This is obviously not a B-cell modulator. So these IgG, they will be kind of generated once they've been cleaved. So obviously, you have that kind of maybe five- to seven-day period when IgG remains very low. But we would expect, obviously, the antibodies to come back. And so from a transplant perspective, you would do exactly the same thing that you would do with any transplant patient, which is that post-transplant, you would put these patients on an autoimmune suppression regimen. And there's no difference in that regimen whether you have a highly sensitized patient or a normal transplant patient. So that will obviously be what will keep those kind of antibodies under control. And that's also what we've seen is that once you kind of put that in place, that kind of ensures for the long-term performance.
That's what we've seen in that kind of five-year data set is that you have that kind of performance that is very similar to what you'd have from a matched organ transplant.
We've just got another new question from the online audience. What is your supply strategy for Idefirix?
So we have, I mean, obviously, so we are commercializing in Europe. And so we're going to use the same type of supply chain so that we don't see any changes in terms of kind of doing that in the U.S. versus Europe. So we have a kind of where we're working through all of this kind of in terms of Specialty Pharma 3PL kind of system in the U.S. And that's part of the kind of pre-commercial preparations that we're going through at the moment.
Okay, I guess. And the final question from the online audience is, could you please comment on how you're strengthening Hansa's presence in the European markets at the moment?
Sure. So obviously, as I mentioned, I joined the company towards the end of April last year, so I've had an opportunity to kind of come in and assess some of the business parts. One of the things we did do is I brought in quite a lot of significant expertise from the commercial side, both in terms of Europe and the U.S., and so Maria Törnsén, who is our kind of COO and President of the U.S., has significant kind of commercial experience also from Europe, so she's been really kind of analyzing and looking at this, which we kind of mentioned in our Q3 call, and so we've started that kind of rollout. We've made some changes to the leadership situation. We've reorganized some of the kind of reporting lines. We're rolling out some kind of significant system support in Europe.
And we're also looking over the kind of overall kind of incentive programs that are in place. So I think we're kind of just investing in kind of improving the actual kind of operating performance of that business. And so this is something that we're going to roll out over kind of Q1. And so we would expect to kind of see some benefits from that kind of enhanced transparency, accountability, and really kind of data collection so that we can actually run a kind of slightly tighter, more kind of well-defined and have some more visibility in terms of kind of that European business as it kind of grows on the back of kind of data that we hope to read out.
Thank you so much, Renée. Thank you for your time.
Thank you very much for listening.