Welcome everybody. I'm Douglas Tsao, Senior Analyst at H.C. Wainwright & Co. We are thrilled to have Maria Törnsén from Hansa Biopharma up next, and Maria was telling me that she's now celebrating her one-year anniversary at the company.
That is correct.
Maria, maybe as a starting point, for investors who are new to the story, maybe just start by explaining the problem that imlifidase is trying to solve, and the lead indication for highly sensitized kidney transplant patients, and maybe why it is such an unmet need and why these patients are underserved.
Yes, happy to. Imlifidase is an IgG cleaving enzyme that is approved in Europe. We have filed for a U.S. BLA and have a PDUFA in December. The patients that are highly sensitized and are awaiting a kidney transplant, they have a very difficult time finding a matching kidney. The reason being is that they have a very high antibody profile, which sometimes is referred to as a CPRA score. They have a very high antibody profile, which can be due to the fact that they have been pregnant, they've had a previous transplant, or they had a blood transfusion. Therefore, in the matching allocation system, they will never match and find a kidney.
These patients that are highly sensitized, and you tend to speak about patients that are 80%+ CPRA, they stay on this wait list for a long period of time, and the higher your CPRA score is, the longer you wait. For those that are, you know, have 98%+, which today in the U.S. is 7,000 patients, they can wait between five to seven years, many of them more than seven years. What happens is they remain on dialysis. Many of them, either become too sick, so they are removed from the transplant list, they are not eligible for transplant, or they will die while they're waiting for a transplant. For these patients, that is where imlifidase comes into the picture to enable them to receive a transplant.
Maria, I'm just curious though, you know, there are some methods that some centers use to transplant these patients.
Yet they still have sort of bad outcomes.
as you talked about.
Yeah.
Maybe if you could just walk through why things like plasmapheresis aren't ultimately effective, right, or don't solve the need that imlifidase does.
Yeah. There are no approved therapies in the U.S. today, but transplant centers are trying various off-label desensitization approaches. The challenge with all of these approaches, and there are multiple variations that centers use, is that none of them work quickly enough and have the rapid drop in IgG antibodies. What imlifidase does is when you give the product, it will cleave the IgG antibodies very quickly within a couple of hours below 5%, and that will enable a transplant. Why that is critical is that the majority, 80% of kidneys today are deceased donor kidneys. When the kidney has traveled, you know, from one state to another or even within a state, you know, there's only a certain amount of time that it will be still viable for transplantation.
Once you receive that kidney, it is critical that you can deplete those antibodies very quickly. These other therapies would require patients to be on therapy for weeks and months sometimes, which obviously is not something that is suitable if you think, but you never know when a kidney is gonna show up, so you can't stay on these therapies forever. That is why they're not really suitable, and that's something that we also showed in our U.S. ConfIdeS phase III trial.
I think it's worth maybe talking a little bit or Maria just sort of providing an overview of the results from the ConfIdeS trial. I think it's also worth talking about what happened in the control arm-
because I thought that was very interesting. Maybe just high level.
Exactly
you know, imlifidase was able to do, right, for these patients.
Exactly. The ConfIdeS trial was a phase III trial that we ran in the U.S. It was designed in close collaboration with the U.S. FDA. They wanted to see this type of trial design. There were 64 patients enrolled in the trial, 32 in the imlifidase arm and 32 in the control arm. The FDA wanted to see like what a standard of care look like. In reality, there is no standard of care, as I said. In the control arm, the transplant centers could basically use anything they wanted at any time point to try and desensitize these patients. I think what is important to remember is that these are highly motivated patients. These were patients with a CPRA of 99.9%. These patients wait seven years or longer for a transplant.
If you're then offered to participate in a trial and the center is participating, of course, they're gonna do everything they can to get these patients transplanted. What happened in reality is that these centers tried everything that was available to them that they wanted to do in the control arm. At randomization, every single patient was offered an organ. What happened? In the control arm, only three patients proceeded with a transplant. That is, again, you know, as I mentioned before, a sign of the fact that these other experimental off-label therapies are not efficient enough when it comes to depleting the antibodies. That is truly, I think what the ConfIdeS trial showed is that there is really no standard of care in the U.S. There is a high unmet need for these patients.
As I mentioned, when the kidney shows up in real clinical practice, you don't have weeks and months to desensitize a patient. So that is what it showed. I think, you know, going to the outcomes of the ConfIdeS trial, the primary endpoint was eGFR, and we showed that in the control arm, the eGFR after 12 months was 19, and in the imlifidase arm, it was 51. A p-value of 0.001, again demonstrating that not only did imlifidase enable a transplant, but it also led to a good eGFR after 12 months.
You know, even if we compare the data for imlifidase patients to those who received a transplant.
the comparison looks favorable, right?
It does. It looks favorable on the eGFR, which was the data we reported back in September. We are, as I mentioned, we filed to the U.S. FDA, and we will read out more data at ATC in Boston in June. There will be more data released from the secondary endpoints and some of the other safety analysis.
I'm just curious how people or investors should think about the size of the market. You know, do you think that the opportunity is sort of a niche rescue population, or do you think that over time, as centers gain experience with imlifidase, they appreciate that the value that it provides, that this is really something that will be used much more broadly.
That, you know, whereas the patients in the ConfIdeS trial had very, very high CPRAs, you know, eventually you'll start to see that drift down as centers look to transplant more patients.
Yes. To just to look at the U.S. market today, there are 100,000 patients waiting for a kidney transplant in the U.S., 15% of those are highly sensitized, which means having a CPRA over 80%. That is generally what you see in the literature when it comes to how they define a highly sensitized patients. I think if you look at then a more narrow population, 98% CPRA, which I think is, you know, has sort of a higher unmet need, there are 7,000 patients in the U.S. today.
I think, you know, when we look at launching, hopefully, imlifidase, I would expect that the highest usage will be in those higher CPRA scores because if you look at how the kidney allocation system works in the U.S., the higher CPRA score you have, the higher up you are on the wait list. The system wants these patients to be transplanted, but they're never matching with an organ. I think you'll start to see in those higher 99% patients, and then eventually it will go down. As I mentioned, there are thousands of patients waiting. Just in the ConfIdeS population, there are 3,500 patients today in the U.S. You know, I think that's where you'll start to see it.
You know, going back to your questions, I think eventually as they start to gain experience, you may see usage in lower CPRA as well.
I think, you know, one question that we get from investors is how we should think about the U.S. opportunity versus the European opportunity, right?
You know, the product has been on the market in Europe for a number of years now. I think the launch trajectory has sort of been uneven.
It sort of has felt like two steps forward, one step back, three steps forward, two steps, you know, three steps forward, twp steps back. I think the conditions in the U.S. market are very different, where you're gonna be coming to market with very different conditions, both from a structure standpoint as well as clinical experience standpoint. Maybe if you could just talk about that?
Sure. The product was launched back in 2020 in Europe with very limited data set. The EMA approved the product based on phase II data. There were two centers in Europe, one in Sweden, one in France, that had clinical experience. No one else had heard about the product. That was the first hurdle to overcome, that you have transplant centers that have never heard about a product for a population that they've never been able to treat because there's been nothing else available on the market. As we all know, Europe is also very fragmented. It takes time to gain reimbursement. It has taken time to write guidelines in each of the different countries, set up supply agreements with every single hospital in different countries in Europe.
That has taken a few years, and I think that's why the sales in Europe have been a bit up and down quarter to quarter, just based on all of those factors. If you look at the European market, it is a significant opportunity. I think we all recognize that launching in Europe is quite different compared to launching in the U.S. The benefit of the U.S. is obviously that when we come to market, we have significantly more data. We have our phase II data. We have long-term data, five-year outcomes data that shows that graft survival, patient survival is similar to a normal kidney transplant. We will have the ConfIdeS phase III data from the U.S.
We are in the middle of this year, we are going to read out the PAES, the study in Europe that has been run to seek full approval. That will be 50 patients. We will have real-world evidence as well, being published out of France. The clinical knowledge and experience will be significantly different, I think, when we come to the U.S. The other point I would also say is that in our study in the U.S., in the ConfIdeS study, we had 25 centers participating, and those 25 centers represent 25% of the total kidney volume transplant in the U.S. Again, we have a lot of KOLs with experience, obviously our 1 kidney allocation system. I think we are in a different position compared to when we launched in Europe.
in, you know, you mentioned France.
Yeah
That has arguably been, from a commercial standpoint, your bedrock market.
Yes.
Not only are they using it in kidney transplant, I think there is some use in lung transplantation as well. I'm just curious, you know, sort of Stepping away from just the European market.
what was it about France that ultimately saw such good adoption? What is that let you, or how can you apply that to the U.S. launch?
France was one of the two centers that participated in the phase II, and we also had an early access program running in France. That early clinical experience was available in France. What we've also seen over the years is that has sort of led to more and more centers in France using the product, using it in more patients. They've obviously had great outcomes, and we've also started to see off-label use in lung transplant. That actually led to the lung transplant community asking the French ANSM to approve reimbursement for lung transplant. Back in November 2025, lung transplant was reimbursed in France, despite the fact that we don't have a label in lung, obviously. It's being reimbursed for lung as well in France.
That is, you know, again, early clinical experience, participation in clinical trial, early access programs was one of the success factors in France.
Okay. I do have some other questions I wanna ask, but I did wanna touch on the news from this morning.
Yes
in which that you basically monetized the European opportunity by selling imlifidase or selling the commercial rights to imlifidase to SERB.
I guess, for EUR 110 million upfront, as well as EUR 5 million, on full approval.
I'm just curious, about what sort of made you as a company to execute this transaction at this time, and what does this allow you to do?
Yes. This morning we announced an out-licensing agreement with SERB for the European Union, U.K., Liechtenstein, Switzerland, and the MENA, Middle East North Africa region, for the amounts that you mentioned, a total of EUR 150 million. You know, the European market is, you know, a significant market potential. There are between 7 thousand and 11 thousand highly sensitized patients in Europe. As I alluded to before, the launch has been a bit slow due to the way it was launched many years ago. We, you know, we could perfectly have kept the business in-house, but we received multiple inbound, you know, interest in the product, in the opportunity, which led us to sort of consider it and run a process.
That led to the announcement that we made this morning. For Hansa as a company, this is a transformative deal. You know, it gives us non-dilutive financing into the company. We have said that we will invest it sort of in a robust U.S. launch, and we will also invest it in our pipeline, where we have, you know, a follow-on molecule for GBS. You know, subject to U.S. approval in December this year, it will lead us in a path to profitability. It is, you know, a transformative deal for us as a company.
Obviously, we think SERB will be a great partner for us. We look forward to working with them, as they take this on, subject to the normal, you know, regulatory approvals in 60 days.
You know, maybe turning to just what the capital allows you to do.
I think on the call you talked about obviously it relieves some pressure.
for some of the debt obligations you had, funding HNSA-5487, as well as the U.S. launch. You know, in terms of a commercial build-out, does it let you be a little bit more aggressive in terms of the size of your commercial organization and open up some investments that were perhaps ones that you were not sure would sort of meet the threshold given your current, prior financing situation?
I think it definitely gives us an opportunity to have a more robust launch. From sort of a size of the field team, I don't think that's gonna change, you know. The reason I'm saying that is that the transplant market is very condensed in the U.S. There are 200 transplant centers in the U.S. 100 of those centers represent 80% of the volume. My plan to have a field team of between 15 and 20 is not gonna change. I think certain activities we may have, you know, more resources to put behind. I think most importantly, I think it allows us also to progress HNSA-5487, our follow-on molecule.
We have mentioned before that we are in discussions with the FDA on taking that into GBS, with the aim to start a trial by the end of the year. I think that gives us an opportunity, and it also gives us the opportunity to further think about how we're going to invest in our pipeline.
Maria, you know, you had generated some data in GBS with imlifidase. What is it about HNSA-5487, or why did you choose to move ahead with that molecule, which is your next generation molecule? What is it or what are some of the characteristics of that molecule that perhaps people should understand?
Yeah. HNSA-5487 is our follow-on molecule. It's also an IgG cleaving enzyme that we've decided to take into GBS. It's mainly sort of a strategic decision to have two pillars. One pillar being with imlifidase, focused on transplant and gene therapy. We also have some gene therapy partnerships there. Then have a second pillar being in the autoimmune space. You know, HNSA-5487 we think is, you know, there are a lot of similarities between the molecules obviously. We think that, you know, the potency of that product, it's slightly different, slightly less immunogenic compared to imlifidase. We believe that's gonna be suitable for autoimmune diseases where you have these flares, not for chronic treatment, but when you have these type of flares.
You know, we've decided that GBS is our, is our first indication there.
When we think about GBS, 'cause, you know, that was one where you had generated some data with the imlifidase that looked quite strong.
Correct
although patients were only dosed once. When you think about the opportunity or the with HNSA-5487 to redose, is that contemplated for GBS, or is that just something that you think about as optionality for other potential indications?
Well, I think time will tell. We, you know, as we mentioned before, we're in discussions with the FDA on what the clinical trial program will look like for GBS. I think it's a bit too early to say exactly what the dosing is gonna look like, what the comparator is gonna look like. I know that it has been sort of discussed before. Can you redose? You know, how many times can we redose? That's obviously something that will need to be considered.
You know, I did want to maybe looping back to kidney transplant. You know, one question that has come up from investors to just sort of understanding reimbursement and how that might affect the launch trajectory. You've obviously talked about needing an NTAP. You know, the cost of the imlifidase is well above the sort of existing DRG for kidney transplant. How do you think about navigating that for the first few years? You know, because it'll be some time before you get an NTAP, although I think you can apply before approval.
There'll still be a little lag, most likely.
Yeah
as well as eventually having to wait for your DRG to potentially reset.
I mean, I think, this is an inpatient drug that will be covered by DRG codes. As those are historical, they never capture sort of any innovative new products coming to the market. The way we look at this is it's very similar to how the CAR-Ts launched when they came to market. Launching into a DRG where the transplant centers will need to apply for outlier payments to CMS, which is something that, most of them are used to doing today already for other drugs. We will apply for NTAP, new technology add-on payment, in October. When we get that will also then give another reimbursement to the transplant centers.
Obviously, you know, after a few years, you can potentially get your own DRG, which is the pathway that the CAR-Ts took. I think that the way sort of they approached the inpatient DRG NTAP, it's gonna be a very similar path for imlifidase.
I think we're almost out of time, but maybe just a final one for me. You know, do you think the transplant field is going to move with the imlifidase some sort of a philosophical shift from sort of wait for the perfect donor to sort of enabling transplantation for, or, you know, enable transportation safely?
patients?
I think it will. I think it will. I think in general, if you look at what's happening in the transplant market today, there's a lot of efforts from CMS to improve transplant care, improve kidney care in general in the U.S. There are roughly 100 centers in the U.S. that have been designated as IOTA centers, and these centers are being incentivized to do more transplants to achieve better outcomes for patients to use more organs to make sure that not as many organs gets discarded. I think something like imlifidase can be part of sort of achieving those type of goals. I do think, you know, that the transplant market itself is going to improve and move towards that direction for sure.
Great. Well, thank you very much, Maria. With that, I think we're gonna have to conclude for the concluding panel. Thank you.
Great. Thank you.