Good day and welcome to the Hansa Biopharma Confide S Topline Results Conference Call. All participants will be in listen-only mode. Should you need assistance, please signal a conference specialist by pressing the star key followed by zero. After today's presentation, there will be an opportunity to ask questions. To ask a question, you may press star, then one on a touch-tone phone. To withdraw your question, please press star, then two. Please note, this event is being recorded. I would now like to turn the conference over to Hansa Biopharma CEO, Renée Aguiar-Lucander. Please go ahead.
Thank you. Good morning, good afternoon, and welcome to the Hansa Biopharma Conference Call to present the pivotal U.S. Phase III Confide S trial topline results. This is a very exciting day for all of us here at the company, but also, I believe, for many highly sensitized patients who have limited options apart from dialysis today. We are indeed very proud to be able to bring you the encouraging results of our Confide S Phase III trial. I'm Renée Aguiar-Lucander, CEO for Hansa Biopharma, and joining me today is Richard Philipson, Chief Medical Officer, and Maria Törnsén, Chief Operating Officer and President of the U.S. We will also have Angela Maldonado, Senior Medical Director, who's worked on the trial since its inception, and Evan Ballantyne, CFO, join us for the Q&A session. Please turn to slide two.
Please allow me to draw your attention to the fact that we will be making forward-looking statements during the presentation, and you should therefore apply appropriate caution. Please turn to slide three. This is today's agenda, where I will initially provide a very brief introduction of imlifidase and a snapshot of the U.S. individual disease situation. Our CMO will then present the topline results for the pivotal trial, followed by our President of the U.S., who will give an overview of the U.S. market and our commercial readiness. Finally, we'll have a brief overview of the next steps, followed by a Q&A session. Please turn to slide four. Hansa Biopharma is a publicly listed company on OMX in Sweden and is a pioneer in the area of IgG cleaving and desensitization of highly sensitized patients awaiting a potential kidney transplant based on its novel and first-in-class enzyme platform.
Its lead product candidate, imlifidase, is a conditionally approved substance in Europe and in the U.K. and has established reimbursement in 20 European countries to date. Other potential indications for imlifidase include desensitization for out-of-dosing of gene therapies, where initial clinical data is now starting to become available, as well as IgG-mediated autoimmune diseases such as anti-GBM, in which Hansa is conducting a Phase III study, which is expected to read out topline results in Q4 2025. Finally, the company has also developed a second-generation enzyme, which we plan to have interactions with the FDA regarding a clinical development program in Q1 of 2026, subsequent to which we will be in a position to give you an update on potential indications and plans for that candidate. Please turn to the next page.
In terms of the US, we know today that over 800,000 people have end-stage renal disease in the US. This is a medical condition in which kidneys no longer adequately filter waste products from the blood. We also know the majority of people with end-stage renal disease are on dialysis. In 2020, Medicare spent over $30 billion on dialysis, and it does represent a significant cost to the US healthcare system. In terms of treatment of choice for end-stage renal disease, kidney transplant does provide a higher quality of life, less societal impact, and lower mortality than chronic hemodialysis. Unfortunately, every year there's a rise in adult candidates added to the kidney waitlist, and in fact, many patients, as you can see here, 12.4% have been on the waitlist five years or longer, and almost 16% have been on the waitlist for six years or more.
This obviously reflects the fact that the demand for transplant organs is significantly higher than the supply. For those of you who have friends, family, relationships with someone who actually has been in dialysis, you are all aware of how burdensome and difficult it is to actually be dependent on dialysis treatment. With that, I'd like to just go to the next page, please. With this brief introduction, I'd like to hand over to our Chief Medical Officer, Richard Philipson, to take you through the very exciting topline data from our Confide S trial. Richard?
Thank you very much, Renée. I'm going to present a summary of the efficacy and safety outcomes of the Confide S study, which is a Phase III open-label, randomized, controlled study evaluating kidney function at 12 months, as measured by estimated glomerular filtration rate, which I will now call eGFR, in highly sensitized kidney transplant patients treated with imlifidase prior to transplantation compared to a control group. Next slide. I'm going to start by briefly describing the study's design. Next slide. Patients considered potential candidates for the study were consented and entered a pre-screening period. One or more unacceptable antigens were delisted from the patient's HLA profile to increase the likelihood of the patient receiving an organ offer. When an organ offer was received, patients entered screening and underwent final evaluation of eligibility. Eligible patients were then randomized to the imlifidase arm or the control arm in a one-to-one ratio.
The period of follow-up in the study was 12 months from the time of randomization. Patients randomized to the imlifidase arm accepted the organ offer and were treated with imlifidase. If treatment resulted in cross-match conversion from positive to negative, patients were transplanted and entered follow-up. Patients randomized to the control arm either accepted the organ offer, were treated with non-approved desensitization, and then proceeded to transplant, or the organ offer was rejected and the patient waited for a more compatible organ offer or organ offers later in the 12-month follow-up period. Next slide. Moving on now to patient disposition and demographics with enrollment. Next slide. A total of 64 patients were randomized in equal numbers to either treatment with imlifidase or the control arm, so there were 32 patients in each arm of the study. Two patients randomized to the imlifidase arm did not proceed to treatment.
In one case, the organ offer was refused. In the other case, the patient withdrew consent to be treated with imlifidase. The overall rate of completion of the study was excellent. A total of 58 patients, or just over 90% of the population in the study, completed the 12-month follow-up period. The treatment groups were balanced with respect to sex and age, and overall, there were almost equal numbers of males and females in the study, with a mean age of the study population of 45.3 years. Next slide. The treatment groups were balanced with respect to race and ethnicity and representative of a highly sensitized kidney transplant waitlist population. Most patients enrolled were either white or Black or African American. The minority of patients were Hispanic or Latino. Next slide. It's worth noting that there are two key analysis sets.
The full analysis set is used for efficacy analyses and comprises all patients randomized in the study. The safety analysis set excludes patients who did not receive randomized treatment. Hence, there are 30 patients in the imlifidase arm in this analysis set, as explained on the earlier slide. Next slide. Turning to the primary efficacy outcome, at 12 months, mean eGFR was 51.5 mL/min in the imlifidase arm compared to 19.3 mL/min in the control arm, with a statistically significant and clinically meaningful difference between the two groups of patients of 32.2 mL/min with a p-value less than 0.0001. When looking at two of the supportive analyses at the primary endpoint, these provide outcomes consistent with the primary analysis. When we do an analysis of 12-month eGFR using a non-parametric test that doesn't assume normally distributed data, the outcome remains statistically significant.
Similarly, when we look at 12-month eGFR in patients transplanted based on organ offer at randomization, again, the outcome remains statistically significant. These supportive analyses at the primary endpoint give us additional confidence in the robustness of the primary analysis. Also of note, a key secondary endpoint of dialysis dependency at 12 months was statistically significant with a p-value of 0.0007 in favor of imlifidase. Regarding the other key secondary endpoint relating to graft and patient survival, we won't be sharing additional information on that outcome today as it is a longer-term measure and has complexities relating to the major surgical procedure and multiple concomitant medications in patients with complex medical history. We'll provide this as part of the full data presentation at a future conference but won't attempt to address this today. I can, however, confirm that the endpoint was not statistically significant. Next slide. Tolerability.
I'm turning now to the safety outcomes, and I'm focusing here on imlifidase treated patients. The tolerability of imlifidase was good. There was a low incidence of infusion reactions, and no infusions were interrupted due to an infusion reaction. Infections observed in imlifidase treated patients were typically not related to treatment, and the adverse event and serious adverse event profile of imlifidase reflected a population of patients undergoing kidney transplantation. Most serious adverse events were considered unrelated to imlifidase treatment. Next slide. In conclusion, the treatment arms were well balanced at baseline, and the demographic characteristics reflected a highly sensitized, dialysis-dependent population waitlisted for transplantation. Retention in the study was excellent, with just over 90% of patients completing the study. Primary endpoints were statistically significant and showed a clinically relevant difference.
At 12 months, the mean eGFR was 51.5 mL/min in the imlifidase arm versus 19.3 mL/min in the control arm, with a p-value of less than 0.0001. Tolerability of imlifidase was good, and the safety profile was consistent with previous clinical trial experience, reflecting a population of patients undergoing kidney transplantation. Next slide. Now I'd like to hand over to Maria Törnsén, the COO and President US, who will be talking about their market opportunity.
Great. Thank you very much, Richard. With this positive topline data in hand, we are very excited about the opportunity that lies ahead of us. The unmet needs in the US kidney transplant market are significant. There are approximately 100,000 patients on the transplant list, and highly sensitized patients represent 10% to 15% of those. For these highly sensitized patients, we find their CPRA over 80%. They often remain on the transplant list for multiple years. For patients with the highest CPRA, the wait time can far exceed seven years, and thousands of them will never get a matching organ and will either pass away or become too sick to transplant while waiting. We also know that approximately 45,000 patients are added each year to the transplant list, and 27,000 patients are transplanted each year.
Out of those that receive a life-saving transplant, 80% of those organs come from deceased donors. As you heard from Richard, that was also the inclusion criteria in Confide S. Next slide. The US transplant market is highly concentrated, with approximately 200 centers that perform kidney transplants for adults. On this map, you can also see that the Confide S centers were spread across the US, covering multiple large, well-known transplant centers with world-leading key opinion leaders. This creates a great foundation as we prepare for a potential FDA approval and US launch. Next slide. As I mentioned, the market is highly concentrated. Among the 200 centers, half of them represent 80% of the total transplant volume in the US. The Confide S centers that we know have experience from imlifidase represent 25% of the US transplant market.
We believe this significant clinical experience will create a great foundation for commercial launch. From a pricing and reimbursement perspective, the majority of kidney transplants are paid by Medicare. Kidney transplants are inpatient care covered by DRG codes. Therapies that are covered by Medicare inpatient reimbursement can also apply for NTAP, New Technology Add-on Payment, with the criteria being a new therapy with substantial clinical improvement over existing options and exceeding the current DRG payment rate. We expect to apply for NTAP in 2026, and there are multiple examples of other new therapies that have successfully applied for and been granted NTAP. We're also building a world-class US team to lead this launch. Our medical affairs team, who will play an important role in educating centers and multidisciplinary teams, have multiple years of experience in the transplant market and recent US launch experience.
Our VP of Market Access has launched multiple products in the US market, including in nephrology, and we also have in-house analytical expertise, which will enable us to refine our targeting and field force structure. As you've seen, this is a very focused cold point, and we expect to hire a field team of approximately 20 account managers closer to launch. In summary, we are very confident that we have the right commercialization strategy in place to enable a successful launch in the US of imlifidase assuming FDA approval. With that, I will hand it back to Renée for closing remarks and Q&A.
Thank you, Maria. With that, if we can turn to just the next page, please. We will discuss next steps very briefly. Following this highly statistically significant outcome of the Phase III trial, we're now working across the organization to enable a filing of a BLA, a Biologics License Application, with the FDA prior to year-end. We will be requesting priority review based on the rare condition and high unmet medical need, and we will, subject to the acceptance of the filing by the FDA, communicate the resulting PDUFA date. As the slide outlines, this would result in a potential approval in either Q3 or Q4 2026, depending on the chosen FDA review period. Finally, we are targeting to report additional data from this Phase III trial at the American Transplant Congress, which is being held in June 2026.
This is obviously subject to us being able to submit in time and being accepted for a presentation at that conference. With regard to that, this really concludes our presentation, and we will now be happy to take any questions from the audience.
We will now begin the question and answer session. To ask a question, you may press star, then one on your touch-tone phone. If you are using a speaker phone, please pick up your handset before pressing the keys. If at any time your question has been addressed and you would like to withdraw your question, please press star, then two. Please limit yourselves to two questions. At this time, we will pause momentarily to assemble our roster. Our first question comes from Christopher Udd with SEB.
Hi there, Christopher Udd at SEB. Thank you for taking my questions and congratulations on a momentous readout. I have two questions. First, around the trial design, can you provide some clarity on exactly which endpoints were powered and I guess how much power was it or alpha was allocated? In particular, I'm wondering about the organ survival endpoint. For my second question, what can you tell us about the, you know, let's say, ease of market access or the challenges, perhaps more appropriately, of market access that you have to overcome to get this? Can we expect a quick readout launch or is it obviously not as slow as Europe, but something like slower, let's say?
Richard, do you want to take the first question? Maria maybe takes the second one.
Sure. From a statistical point of view, the study had a usual statistical framework for the analysis. The primary endpoint had an alpha level of 0.05. There was a hierarchical testing strategy. Given that we achieved statistical significance for that outcome, we were then permitted, as per the statistical framework, to proceed to the key secondary endpoints to test those. As we've already said, the outcome with respect to kidney dialysis was statistically significant. Maria?
Yes. A very good question on the market access side. The big difference between Europe and the U.S. is that, as you know, in Europe, it can take several months and sometimes years to gain local reimbursement. In the U.S., you can really launch day one. As I mentioned before, this is an inpatient care. We know that the majority of these patients have Medicare as their payer, and the payment will be covered by DRG codes. We are very experienced in terms of how to manage that with our market access team. I am confident that with the clinical experience that we have already in the U.S. through Confide S, we will be able to gain market access rapidly. Thank you so much.
Our next question comes from Sushila Hernandez with Van Lanschot Kempen. Please go ahead.
Yes. Congratulations on the data, and thank you for taking our questions. Could you walk us through what the data package will look like that you will discuss with the FDA? Will you also discuss patient survival and graft survival data with the FDA? Are there also any indicators that the dynamics in the US transplant market might change in favor of highly sensitized patients and imlifidase? Thank you.
Sure. I'll have Maria kind of talk a bit about the second piece. With regards to the discussion with the FDA, obviously, all relevant data will be discussed with the FDA, including any supportive key or other kind of supportive data and information. The complete data package will obviously be available to discuss with the FDA. I think, you know, as I think Richard already alluded to, I think there's no kind of particular concerns or issues with regards to kind of any of the kind of, you know, with the other kind of key secondary endpoint. It just happens to be a slightly more complex and longer-term kind of outcome with regards to that. We don't have any concerns about it whatsoever at all. It's just not something that is easily kind of provided in this kind of a setting.
That will be presented both as part of any kind of full data as well as obviously be discussed with the FDA as well. Maria, would you like to take the second part?
Yes, happy to. I would say, in general, in the US, there are many positive policy changes that are going on currently in terms of modernizing the organ allocation system that overall are very supportive of accelerating kidney care for patients. That is very positive for us. When it comes to the kidney allocation system in the US, they already today favor highly sensitized patients. You can get different scores depending on how sensitized you are. The challenge for these patients is obviously that today there is no approved therapy if you are highly sensitized.
So imlifidase will really fit in nicely there in terms of supporting these patients to actually get access to an organ and be able to be transplanted. I think overall that the market dynamics are evolving in a very favorable way for kidney care in general, but also for highly sensitized patients.
Okay. That's clear. Thank you.
Our next question comes from Matthew Phipps with William Blair & Company L.L.C. Please go ahead.
Hi, on the team. Let me offer my congrats as well. I'm great. I'll come here. I was wondering if you could give us maybe how many or what % of patients in the control arm receive the transplant. We estimate maybe 30 to 40%. How did this compare to maybe what you expected going in and maybe what types of, do you know what types of desensitization techniques might have been used? I don't know if you can comment on this, but do you have anything on just kind of general rates of delayed graft function and AMR within that 12-month follow-up or at least maybe how that compared to previous trials? Richard, would you like to take these questions?
Sure. I think in terms of the % of patients receiving a transplant in the control arm, we did see a number of patients receiving transplants in the control arm, a mix of compatible and non-compatible. We're going to be looking at that in a lot more detail, that rate of transplants in the control arm, and comparing that to the kinds of rates that we usually see in normal clinical practice compared to what we're seeing in this clinical trial setting. Patients can receive quite a number of different desensitization techniques, experimental techniques, non-approved procedures, including things like IVIG, PLEX, rituximab, and eculizumab. We don't have a detailed analysis of that as yet for the control arm patients. In terms of DGF and AMR, I'm going to hand over to my colleague, Angela Maldonado, who's going to give you a bit more color around those two particular outcomes.
DGF was reported for both the control arm and the imlifidase arm in the Confide S data.
What we did see is that there are many contributors to the rates of DGF. For imlifidase, obviously, it's the administration of imlifidase and the cross-match conversion time. For the control arm, all of the experimental or unapproved therapies in the arm require extensive timing. In the setting of a deceased donor transplant, these could definitely contribute to the rates of DGF seen in the trial. As for AMR, AMR is an expected outcome after an HLA incompatible transplant. We are still looking at the causes and the types of AMR that were reported in the trial. Those details will come out from there.
Thanks, Angela. I think it's worth saying that in terms of delayed graft function, we did not see anything that was not in keeping with what we expected. Similarly, really a similar comment for AMR, and particularly of note, there were no transplant losses due to AMR. I think that covers the questions.
Can I ask one quick follow-up for maybe for Maria? Sorry if I missed it. Is your, and maybe you can't comment yet, but is your expectation that some kind of a PRA cutoff might be included in the actual labeled indication or just a kind of highly sensitized type label? Again, I realize you might have to still have these discussions with the FDA.
Renée, do you want to end these discussions?
Yeah, I think, with regards to this, obviously, this is going to be a review issue. We've obviously had no interactions on this, and the FDA will not provide any insight on this until we're kind of in the review setting. We're not able to provide any details on that. Obviously, any clinical data will be included as part of any kind of approval. With regards to the specific label, that's going to have to be a later conversation once the FDA has had a chance to review and look at the actual data.
Great. Thank you for taking my questions. Congrats again. Thanks.
Again, if you have a question, please press star, then one. Our next question comes from Douglas Tsao with H.C. Wainwright & Co. Please go ahead.
Hi. Good morning. Thanks for taking the questions and congratulations on the data. I'm just curious. I know that obviously this data is very fresh. Perspectives that you might have gotten from any of the investigators or KOLs that you've had the opportunity to interact with based on the topline data. I have a follow-up.
Yeah. Obviously, at this point in time, we've had very little opportunity to do this because in the US versus Europe, we have slightly different structures under which we have to report this kind of data. Once we actually receive the topline data, we really have to put this out in the public domain immediately. We cannot confer and discuss and choose a time to do this. Unfortunately, this is why the data, we haven't had a chance to do all analysis or provide a deeper conversation with KOLs. Having said that, I will say that there is a quote in the press release, and I think from one of the primary investigators that I think reflects very well the actual perception for these patients. In this setting, very little progress has been made or innovation has been seen for these patients for a very, very long time.
It's certainly my experience in interacting with KOLs prior to this data that they are extremely excited about having the potential opportunity to use this drug in practice. I don't know if Richard or Maria, if you want to comment any further interactions that you might have had or Angela for that matter.
I would just reiterate your comments, Renée. We've had very little time, but I think it's clear from the comments from Professor Montgomery, who's been a leading light in this study over a number of years. He's very excited by the outcomes. I think that reflects the nature of the outcomes that we've seen, the kind of highly positive differences that we've seen in patients who've received imlifidase.
I'm just curious in terms of the imlifidase patients, if you can provide any color in terms of the amount of variability of the response that you've seen, especially around that eGFR level, or was that sort of consistently sort of the outcome that patients were achieving?
I think it's difficult to provide kind of very detailed information. We are going to, obviously, we want to submit this data both for kind of presentations at conferences, publications, etc. Partly of this is, as I said, we don't have kind of all full, full, full data that we've been able to kind of do an analysis on. I would just caution that in terms of the level of detail that we can share at this point in time, per definition, it has to be a little bit limited. Having said that, if there is something you want to say, Richard or Angela, I will leave it to you to decide.
Yeah. No, I mean, I think it's clear from the outcomes that we've presented. We have seen a very good response to imlifidase. We can see that in the outcomes at 12 months because of where we're seeing the eGFR at 12 months in the patients who received imlifidase and were transplanted. I think that really speaks to the benefits that we're seeing in terms of response to the treatment.
Okay. That's it for me. Thank you.
This concludes our question and answer session. I would like to turn the conference back over to CEO Renée Aguiar-Lucander for any closing remarks. Thank you.
Thank you very much for everybody who participated in this presentation. As I mentioned, we are super excited about the outcome of this trial. Obviously, it can be shown also by the very highly significant outcome in terms of p-value with regards to the primary endpoint. I think this is hopefully the beginning of a very exciting journey for patients really who have, as I said in the beginning, very limited options today. We are very excited about bringing this to the FDA, and we look forward to keeping you updated as that process evolves. Thank you very much for listening.
The conference is now concluded. Thank you for attending today's presentation. You may now.