Hansa Biopharma AB (HNSA) March 2026 Conference Transcript

My name is Tom Smith. I'm one of the senior biotech analysts here at Leerink, and I'm happy to welcome you all to day one of the Leerink Partners Global Healthcare Conference. It's my pleasure to introduce our next company, Hansa Biopharma, where I'm joined on stage by CEO Renee Aguiar-Lukander and President of U.S. Operations, Maria Tornson.

Thank you both so much for joining us. Looking forward to the discussion. Thank you.

Renee, maybe you could just kick us off. It is a really busy time for the company. You've had a lot of data here over the last 12 months. We have a BLA submission that's on file in the U.S. Maybe just walk us through some of the highlights of the last year and what we're looking forward to here in 2026.

So I joined the company in April of last year. Maria joined shortly thereafter. So really what we've been doing with the company are a couple of things. We did a bit of a restructuring in order to kind of bring down the burn rate. We restructured the debt facility That was outstanding with the company And we did two capital raises one in June and one in kind of on October 1st In order to really kind of strengthen the foundation of the business I've also brought on board quite a lot of Experienced people particularly with a kind of US background in terms of medical affairs market access commercial As we kind of prepare as a company for a commercial launch in the US. So the most important kind of event really was our phase three readout in September. The confiders trial with a p-value less than 0.0001 and then the subsequent BLA filing following that. So this year obviously we're looking forward to hopefully an approval in December of this year. We also have another phase three trial reading out in the middle of this year which is a European phase three trial with 50 patients being transplanted in that trial which was fully recruited in March of last year as we're looking forward to that that will lead to then filing for full approval in Europe which we will do hopefully then in Q4 of this year and then we have interactions with the FDA with regards to our second generation enzyme related to Guillain-Barré which we're hoping to have an agreement of feedback from the FDA by mid-year and so an ability to start that trial before the year-end and obviously we'll also hope to present the phase 3 data at the

American Transplant Congress in June awesome so quite busy yes very busy let's start kind of high level level setting the product profile for limpidase I mean one of the key differentiating aspects here is speed of onset maybe just walk us through why that matters in highly sensitized kidney transplant patients and is how the onset compares to some of the other desensitization approaches none of which are approved but are used in clinical practice sure so here obviously 80% of all kind of kidney transplants are done with donated donors I mean that disease donors donor organs and so that basically means that you have a limited amount of time to actually transplant the organ to keep it viable so this

basically means that in this case a lot of these patients who are considered to be highly sensitized these are patients with a so-called CPRA score above 80 they a lot of times will be on dialysis on the waitlist for very very very many years the median kind of year that they're on waitlist is seven so I mean they wait for a very long period of time and quite a few of them also actually die on the waitlist or get moved off the waitlist because they are no longer transplantable so in this case obviously it's finding that it's it's for the the actual kind of clinical trial we ran these were really patients with a 0.01 percent chance of ever finding a kind of a natural match so as you can imagine that's a you know very poor outlook for these patients so in this case obviously you don't have that much time and you really you know you know you have an organ you're going to call the patient get them into the hospital and you have very limited time in order to actually do the transplantation in order to keep the organ viable. So in this case Imlifidase really kind of reduces IgA IgG all four classes intramasculally extravascularly within two to six hours by more than 95% from baseline. So you really kind of very very quickly and rapidly cleave all IgG across all parts and actually kind of end up in a situation where you can turn that cross match positive organ to a negative cross match and thereby actually proceed with a transplantation. You cannot really proceed with a cross match positive transplant as that will kind of really have a hyper acute rejection. So this is actually why it's very important in this case to be able to do this in a predictable consistent and safe way and very rapidly in order to enable this transplant to go ahead. That makes a lot sense you mentioned that confide us pivotal data maybe just touch on highlight like key takeaways from that data set what do you feel is the the most underappreciated part of that data we don't and that really goes back to part of your kind of question before which is so what are what is being used today and I think this control arm really shows that you know there is no real standard of care today there is no way of really kind of desensitizing these patients and so if you look at actually the control arm physicians there could use pretty much anything that's available today IVIG, Plex, rituximab, cluzumab etc to kind of like before or after during so there was a fair amount of kind of you know ability for physicians to try to kind of have exploratory protocols but out of those 32 patients in the control group only three of them actually decided that they could progress to transplant at randomization. And you can imagine these are patients with a very low probability of ever getting an organ offer. They're on dialysis and despite that they're in a clinical trial which we know also generate you know even higher kind of levels of kind of results generally. So I think the fact that you only had three kind of out of 32 patients who did that despite being so highly kind of motivated I think really shows that this is this is really something that is a significant and unmet medical need, and there's really nothing out there today. And if you look at the outcomes, because obviously the primary endpoint was kidney function at 12 months after randomization, and in this case, if you look at those 27 patients who actually all progressed to kind of transplant randomization in the amlifidase arm, their EGFR was around 59.3 mils per minute at 12 months. So obviously that would indicate that that was a very, very good performance. from a kind of kidney function perspective if you look at those three who basically kind of were randomized with other types of desensitization approaches their EGFR was 23.1 mils per minute so I think it points to the clinical point trial points to two things one is that you know there really is no standard of care there's no kind of consistent predictable and safe way of doing this and if you try to do some of these things you are I think you run a high risk of having unsatisfactory outcomes and I think that really kind of points to both the unmet medical need as well as the you know significant

opportunity that's out there for emlifidase I want to follow up on the outcomes part of that story because I durability here is really important there's a product that has been approved conditionally and used in Europe now for for quite quite a while maybe just remind us of I guess the kind of the most important long-term follow-up evidence that you have that supports that treatment with lymphidase actually results in better long-term graph function and outcomes ultimately?

Sure so the companies run you know like 10 different kind of clinical studies prior to this so quite a lot of kind of smaller phase two trials and if you actually pool those patients they were pooled and followed for a five-year period and what actually that shows is that in terms of performance this is virtually identical to the performance as they would expect for, you know, a non-highly sensitized or natural matched kind of transplant patient. So there's really no difference in terms of the long-term outcome for these patients compared to those who find a natural match, which I think is very important. And we know that obviously in nephrology in general, you would say that actually the 12-month EGFR has, you know, very, very high level of predictability in terms of long-term outcomes, and that's exactly what we've seen here as well. so in terms of the actual kind of confiders trial we will obviously continue to follow all these patients up until five years but we feel very confident about that long-term outcome considering the excellent kind of 12 month data that we saw in the trial great I want to talk for a minute about regulatory and obviously the FDA we've all seen the headlines it seems to be fairly tumultuous environment at the moment maybe just high level just describe like your regulatory interactions I guess level of alignment with the agency around confidus and approvability sure so the trial was actually very I think the FDA was very influential in terms of designing this trial so obviously I wasn't here but just like looking kind of have the history and the minutes there was a quite a lot of debate particularly with KOLs in the area who felt that having a control arm when there is no standard of care that's accepted was kind of a very odd choice and there was quite a lot of conversations about this but I think the FDA was very adamant about the fact that there needed to be a control arm and so I would say that actually this is something where the FDA has you know consistently been very engaged in this and I think has had a very kind of strong voice in terms of how they wanted this kind of clinical trial to be run and designed and I think what we've certainly found in the very kind of you know the interactions that we've had you know recently I would say that those interactions have been very positive and I think it is you know so far I think all that we've heard from the agencies that they're very supportive and they are you know they are you know they're impressed by the results that you know the clinical study has generated got it and we submitted the BLA.

We learned on, I think it was February 18th, BLA acceptance. I think you learned with your day 74 letter, standard review versus priority review. Maybe you could talk about like, I guess like any insights into why we landed with standard review rather than priority review?

I think we don't know exactly. I mean, I do think that it was clear kind of at the day 60 that you know there was clearly some conversations ongoing internally in the actual in the FDA and I think that you know the the fact is what they were not really willing to be drawn either way on whether they had decided on on what kind of you know review kind of pathway and so I think it's clear to us that there was some internal debate and discussion why and how and what that related to is a little bit unclear to us but I think that you know they have kind of kept on you know it reiterating to us that you know they want to try to be as collaborative and helpful as possible they want to maximize the chances for this drug to be approved and so we don't really have any reason or

any kind of information to believe that this has anything to do with the clinical package or anything that we've seen and so you know we're we don't really know I guess is the answer okay that's fair and one of the other things we typically learn with BLA acceptance and subsequent communication is whether they intend to convene an adcom I guess like any indication from the agency that they would like to form an adcom to discuss the application so they have made it clear on the letter that they don't do not foresee the need for an adcom okay we do not expect that okay cool let's talk about commercial opportunity and you've highlighted relatively concentrated set of centers in the US that we could initially target that do a high volume of kidney

transplants how do you think about I guess like initial launch footprint and resourcing around the launch sure so in the US we have roughly 200 centers that do adult kidney transplants and out of those 200 centers 100 centers represent 80% of the volume so if you think from that perspective it's a very concentrated call point that we estimate would require sort of a field team of 15 to 20 people on the commercial side. The other thing I would highlight is that among those 100 centers, 25 of those participated in the confidus trial that Rene just described. So there's a high level of clinical knowledge and clinical experience of the product, of the mode of action, of how to treat this highly sensitized patient.

That's great. And maybe if you could just walk through some of your other pre-commercial activities, payer engagement, how are we thinking about the potential to price I guess within the procedure code there's obviously add-on payment potential but yeah like what walk us through the other work that you've done preparing for a commercial launch.

Sure so the pre-commercial work is really focused on the medical affairs aspect and the market access work that we're doing. From a medical affairs perspective we're focusing on identifying who are stakeholders within each of these centers particularly the top 100 centers and getting to know the the multidisciplinary team that will take care of these patients you know once the product hopefully is approved from a market access perspective we know that this is an inpatient treatment so kidney transplant is a DRG code today what the transplant centers do is that they also submit them for outlier payments to CMS if there's anything in addition to that what the DRG code is And we expect that we will be treated in the same way to fall into that DRG code and outlier payment The other thing that we are doing is that we are applying for we will apply for NTAP new technology add-on payment

Later this year, you know, we think we have a great chance of getting that if you look at the criteria for NTAP And that will then give these transplant centers an additional reimbursement in addition to the DRG code Awesome, okay want to come back to the conditional approval in Europe and maybe you could just comment on some of the learnings from that initial commercial experience and it's obviously it's very different this would be you know not conditional approval in the US but just talk about some learnings from the Europe commercialization how you plan to apply that sure I think there's some some structures in Europe they were quite different I'll have Maria dress kind of the learnings that we can draw about I think you know what you have to remember

in Europe was that when the company took this to EMA it was basically on the basis of a single arm open label 14 to 16 patient trial and only two centers in Europe was were actually kind of ever involved in kind of phase two trials and the rest was kind of really in the U.S. so there was very very limited clinical experience and clinical understanding because you really hadn't involved there was no kind of European KOL advisory board or kind of that clinical understanding and this is obviously a patient population as we've just shown and confide us that you know physicians don't really have an opportunity to treat these patients today so it's a little bit like you're trying almost to avoid doing this because there really is no way of doing this safely so it's a new kind of way of really kind of addressing a patient population with with kind of a high unmet need and generally what you would expect is that you'd have some fairly robust clinical data in order to kind of you know convince physicians and have them had gone through that kind of clinical experience so it was kind of an unusual setup from that perspective but I think the company's done a great job in terms of creating guidelines getting reimbursement very broadly reimbursed in Europe you know the list price in Europe is about $350,000 per treatment so I think that from that perspective you know the revenues that have been generated in Europe to date over the last three years I would say is more like a pre-launch or kind of a soft launch because really the you know the clinicians really learned about the product through the phase 3 trial. That was really kind of the first time that they were often introduced to the drug or used the drug and that trial was fully recruited in March of last year and we'll read out in the middle of this year so I think it's a little bit of an unusual kind of setup in Europe to say the least but we have certainly some learnings from there.

I think one of the key learnings is that clinical experience matters as Renee mentioned there were two centers in Europe that had experience when we got conditional approval many years ago and that's quite different compared to the situation in the US today so the 25 centers that have been participating confidus they represent 25 percent of the transplant volume in the US so you already have a lot of clinical experience not only with the surgeons but with that multidisciplinary team that includes your nurses nephrologists HLA directors and I think that puts us in a better position compared to the situation in europe and i would say the other thing relates to reimbursement and we all know how fragmented europe is and the challenges and the company's done an excellent job in terms of gaining reimbursement and actually proving that it is cost effective or cost neutral in many cases to to use in this population the difference in the u.s is although pricing and reimbursement always is complex it's a it's you know it's in the u.s it's covered through medicare it's a DRG code and we know that that pathway has been well established with other inpatient drugs before such as the CARTES so we feel sort of confident from market access perspective and the clinical experience perspective that we are in a better position in the U.S. and I would say the third thing is also as Renee said you know since both of us joined we brought in people in the U.S. organizations that have experience that have recently launched in the U.S. that have transplant experience that have experience in nephrology. I have many members of my team that have actually worked in a transplant center. I've been doing this job on the other side so I think that is going to help us as we look to bring imlifidase to the US market.

That makes a lot of sense. I want to come back to the confirmatory study that you mentioned that reads out in the middle of the year.

Maybe just remind us of the design of that study and help kind of set expectations ahead of that Rita sure so this is a kind of it is a single arm so basically it's a 50 patient transplant across Europe so it's about 23 centers kind of mainly academic a large academic institutions in Europe and so EMA really required this to be kind of a pan-european study with 50 kind of patients being transplanted that are highly sensitized so there isn't a particular cutoff in this in this trial it's really kind of up to each center I need to kind of like to practice medicine and to include those patients who they think you know would warrant to be included and transplanted so in that way slightly different clearly kind of from the US trial it doesn't have a control arm it doesn't have any specificity in terms of exactly what kind of patients need to be included I think there's already recognition in Europe of the fact that there is a high unmet medical need here what the other thing is though that it there is a kind of collection of data at the same centers of other transplant patients who are not highly sensitized so it's a bit of an unmatched kind of like so there is data for the EMA to look at in terms of kind of what's been the outcomes in general and kind of transplant for these centers so there is something for them to compare to but it's not kind of a matched arm I would say and so there'll be about a hundred patients in that kind of in that kind of data collection and the end point is very similar to that of the the one in the US so it's basically graft failure free survival or EGFR at 12 months so obviously to the extent that if you think about the US trial to the extent that patients were on dialysis in either arm but in that trial that would be counted as zero right so that would basically kind of say that you know obviously if you graft wasn't working or you had graft failure then obviously you'd be back on dialysis and that would be counted as zero so in this case it's very similar and that's kind of again that kind of 12 month um period uh when they're looking at kind of graph free uh failure graph failure free interesting uh which is the same really as egfr and it's essentially uh like a non-inferiority margin versus the external control or like so there hasn't because it's not a matched kind of they haven't really kind of set it up in terms of so it's more kind of a as a reference I would say that EMI is looking at it as a kind of reference to kind of look at risk benefit broadly across the two groups but there's no kind of exact number that you know needs to be reached because it's not it's more yeah it's not like a matched arm if that makes any sense it makes sense okay wanted to ask about potential use outside of kidney transplant and you had I think with the last earnings call very interesting anecdote of use I believe as a lung transplant patient yeah maybe you could just talk about I guess your expectations for use kind of outside of this core kidney transplant setting and how you think particularly in the US where it seems like transplant centers have quite a bit of freedom in terms of how they manage their patients like how how should we be thinking about potential use in like outside of kidney yeah so it's interesting because there has been kind of case studies that also been published both in lung and in heart and liver kind of transplants because obviously the drug per se is obviously organ agnostic there's nothing that makes this drug specific to one solid organ transplant versus another and this drug only does one thing it's very efficiently cleaves IgG very rapidly and so I think that's why what we've seen is we've seen kind of compassionate use requests and we've seen kind of physicians who published case studies on other kind of solid organ transplants. So in Europe, there was some of these kind of, and in France, I would say, they were one of the two centers that were part of the phase two. So they had, was one of the countries that actually had some clinical experience early on, and what we've seen is that that country is one of the ones who clearly has a very broad use of the drug for the reason that they felt already quite comfortable to actually use the drug and knew how it worked. And so we've had kind of physicians there who've been conducting lung transplants, and they actually independently went to the French government because obviously this is a socialized medicine structure. So they went to the government to actually ask for full reimbursement of Imlifidase to be used in lung transplants as well. And the French government granted this in November of last year, so it's almost like you know the government is actually kind of fully reimbursing lung transplants as well being you know in the use with with the use of Imlifidase. and so that will obviously be very interesting to to follow because that will obviously also be you know will be tracked by the government there you know they will know exactly how much they're spending on on lung transplants as well so I think that that is something that's not so surprising and we do get obviously we've had also here kind of you know compassionate use requests for other kind of solid organ transplants and we've also had I mean the company previously ran a investigative study in AMR so antibody mediated rejection which again I think is something that we get a lot of questions from physicians in terms of if you have something where you already you have a transplant you have a patient and you're seeing that this is running a high risk of antibody mediated rejection would you then actually use this drug to kind of control that patient preserve the organ and actually kind of have a higher probability of retaining that kind of graft. So I think that there are quite a lot of kind of different areas where this could be used but obviously our focus at the moment is to get this approved in kidney. Once we have that approved we obviously we can have plans to go back to the FDA and explore with them what would they require for us to potentially have a label expansion into other solid organs but you know we have to take one step at a time obviously but there is there's clearly a lot of requests coming to the company at this point in time so it's great makes sense let's switch gears and talk about your next-gen IgG protease lots of 5487 and you generated some phase one data maybe just kind of remind us of like the TPP product goal for this next-gen candidate and then what you saw out of the phase one experience sure so this is an enzyme that's derived from a non-human host, so therefore significantly less immunogenic, which means that there is far less AIDAS being generated and also that it can cleave through, you know, it's also more potent and durable so it can actually cleave through very high levels of titers. And so this was really kind of developed with the view to kind of, you know, having that kind of less immunogenic profile. And so what we can see in the actual kind of phase 1b trial that was run is that you know after administration it was actually effectively cleaving through so after six months or so it was actually kind of cleaving through effectively a hundred percent of all AIDA so it would enable kind of that redosing potential so I think that originally when this was kind of designed I think this was obviously before you ended up having quite a lot of maintenance related treatments in some of those kind of crisis and flare type autoimmune diseases and so I think that was probably the original kind of thought of this profile. So I think what we're actually doing now is we're taking this into rare autoimmune diseases with a focus on neurology and so starting with Guillain-Barre where there's really there's a significant unmet medical need and there's really nothing approved today and it's a fairly substantial rare disease and this obviously gives us kind of you know another enzyme with those kind of characteristics um that provides us with an ability to build a completely separate franchise from emlifidase so keeping emlifidase really in the kind of kidney transplant and gene therapy space um and really kind of creating a separate kind of with different ip different pricing abilities different partnering opportunities in kind of you know neurology and rare disease and i think that from a kind of market kind of addressable market situation obviously with 100,000 patients on the waiting list in the U.S. out of which about 15,000 are as a kind of stand-alone market opportunity is obviously very substantial just in kind of kidney transplant if you can think about that type of pricing per treatment with these type of kind of patient numbers that is already, I mean, I think a very substantial kind of product opportunity So instead of just having it kind of be, you know, broader, we've just chosen to kind of create a different franchise with more kind of flexibility and partnering opportunities in autoimmune disease.

That makes sense. You have some experience with amelipidase and Giandora A. Yes. I guess in this engagement with FDA that we're expecting next quarter, like, is there a potential for a more streamlined development path? I'm thinking about in this construct of trying to get novel therapies in the hands of rare disease patients and maybe only one study needed for approval, like how are you thinking about the path forward?

So I think, I mean, because we have had that kind of strong proof of concept already in a phase two and we've obviously had quite a lot of kind of KOLs who are very supportive of this mode of action and the safety profile that they've seen in that study, they've been extremely kind of helpful in terms of trying to really help us design a trial that is this kind of streamlined and kind of you know constructive as possible so it'll be very interesting to see kind of what the FDA's feedback is again in light of a lot of these kind of discussions and things that we hear from the FDA I don't really know if that's going to translate into actual action from the FDA but so this will be an interesting kind of you know case to see what kind of feedback we get and if they are indeed willing to kind of support what I think is an innovative and interesting kind of design. So we will know more in the next quarter we will know more from that feedback and interaction.

We'll stay tuned on that front. Maybe just in the last 30 seconds or so you're also exploring in lymphidase in a gene therapy enabling setting. You have a number of different partnerships. Maybe you could just give us quick soundbite update on that aspect of the amlifidase program.

Sure so this is the same thing obviously it's the amlifidase cleaves kind of very rapidly let you know down to less than kind of 5% of those existing AAV antibodies which is a challenge for a lot of gene therapy companies particularly on the AAV8, AAV9 kind of areas which really prevents them from kind of including patients in clinical trials or or actually having a full kind of commercial opportunity because there's a high percentage of patients who know whose antibody profile really you know prevents that and they ends up being excluded so we have some clinical data towards the end of last year with our partners both of our partnerships are Epta and Genathon and I think actually what we're seeing is as these gene therapy companies get kind of closer to you know kind of bigger trials phase two trials to becoming like bigger into kind of commercial there is a lot of interest to try and obviously make sure that they can reach these patients both in a clinical setting as well as in a commercial setting so there are quite a lot of kind of conversations ongoing and I think that will to some extent also be driven by you know how the regulatory environment ends up for these companies in gene therapy but I think we're certainly having quite a lot of conversations and would hope to you know during this year hopefully kind of strike some more partnerships in the gene therapy space that's great all right perfect well we're up against time but thank you Renee and Maria for joining us and sharing the insights and

be tuned in here in 2026 exciting year great thank you