Hansa Biopharma AB (publ) (HNSA) Earnings Call Transcript & Summary

Hello, and welcome to Hansa Biopharma Interim Report for January to June 2021. [Operator Instructions] Today, I am pleased to present CEO, Søren Tulstrup. Please begin your meeting.

Thank you, moderator. Good afternoon to those of you in Europe, and good morning to those in the U.S. Welcome to the Hansa Biopharma conference call on the first half results of 2021. I'm Søren Tulstrup, CEO of Hansa. With me today, I have our CFO, Donato Spota; as well as our Head of Investor Relations, Klaus Sindahl. Today, we'll review the overall progress and highlights of our business in the first half of 2021 and provide a preview of our near-term milestones. Our presentation should take around 15 minutes. And after that, we'll take your questions. Turn to Slide 2. Please allow me to draw your attention to the fact that we'll be making forward-looking statements during this presentation, and you should therefore apply appropriate caution. Now please turn to Slide 3. Hansa Biopharma's transformation into a fully integrated commercial stage biopharmaceutical company has become a reality in 2021. Our focus remains on delivering on our strategic priorities to build tomorrow's Hansa. This is done through ensuring a successful commercialization of Idefirix in the early launch markets in Europe, advancing our platform in the new indications and therapeutic areas, and lastly, through building our organizational capabilities and expanding our technology platform. During the second quarter, we continued to see good progress across our business and pipeline activities. We were particularly pleased to receive the first national level market access decision in Sweden, which was announced at the end of June by Swedish New Therapies Council who recommends use of Idefirix as a desensitization treatment for highly sensitized kidney transplant patients in Sweden. Beyond Sweden, we continue to roll out our European commercial activities in other early launch countries as planned. We are pleased with the positive interest we have seen so far and the interactions we've had with national reimbursement authorities and leading transplantation clinics. In the U.S., following extensive discussions with the FDA, Hansa recently announced a trial design for a randomized controlled trial targeting 64 highly sensitized kidney transplant patients with the highest unmet medical need in the U.S. As previously guided, the first patient is expected to be included in the second half of 2021. More details around the study design will be shared later in this presentation. In our ongoing Phase II programs for GBS and AMR patient recruitment continues to progress on risk-based site-by-site approach that we implemented due to the COVID-19 pandemic, which continues to impact enrollment. First, data readout in both studies is expected in the second half of 2022, as previously guided. Lastly, we're excited to have now initiated IND-enabling toxicology studies with the lead candidate from our next-generation of antibody-cleaving enzymes, also known as a NiceR program. NiceR is a new set of enzymes developed for repeat dosing scenarios that may potentially open up a broad array of new indications to pursue, including reoccurring AMR, relapsing autoimmune diseases and oncology. Please turn to Slide 4. As I said initially, Hansa Biopharma's commercial launch activities are progressing as planned in early launch countries like the Nordics, Benelux, Germany and the U.K. The company is currently interacting extensively with national reimbursement authorities and leading transplant clinics. And as I previously noted, we received our first national level market access decision by Sweden's New Therapies Council a few weeks ago. This decision is an important milestone for transplant clinics across Sweden, who would like to introduce Idefirix as desensitization treatment to enable highly sensitized patients to qualify for a potentially life-saving and life-altering kidney transplant from a diseased donor. The recommendation follows an earlier health economic assessment by the Swedish Dental and Pharmaceutical Benefits Agency, which concluded that Idefirix treatment would be cost-effective or even cost saving for the society. Moving forward, we expect additional such decisions and agreements around reimbursement, funding and market access to be reached in other early launch countries beginning in the second half of 2021 and onwards. In parallel, with reimbursement negotiation processes, Hansa continues to work with select key centers that potentially have the ability to access funds outside the national reimbursement system for individual patients. On June 30, Hansa hosted our first launch symposium for European key opinion leaders, more than 120 physicians from 80 transplant centers in 13 different countries across Europe attended the symposium. The symposium focused on Idefirix as a potential new standard of care for highly sensitized kidney transplant patients needing desensitization treatment and included multiple sessions where expert speakers discuss specific patient cases to address topics around patient selection, AMR management, patient care and more. We're very pleased to see such a strong interest and active participation in our launch event, which illustrates the need for an innovative therapy like Idefirix to address a high unmet medical need in the area of kidney transportation. Going forward, Hansa will continue to work on increasing awareness, understanding and buying around Idefirix as a desensitization treatment among key opinion leaders, targeted clinics, patient organizations and the broad of transplant healthcare systems. In doing so, we will leverage already existing collaborations and connections with key organizations such as the European Society of Organ Transplantation to put in place updated guidelines, et cetera. Now please turn to Slide 5. As I highlighted earlier, following extensive discussions with the FDA, we recently announced a trial design for a randomized controlled trial of imlifidase in highly synthetized kidney transplant patients. In the study, 64 highly sensitized kidney patients with a cPRA score of 99.9% or above will be enrolled. This represents a subset of very highly sensitized patients that continue to be disadvantaged despite prioritization under the U.S. Kidney Allocation System. When a donor organ becomes available and a positive crossmatch with the intended recipient indicates that the organ is not compatible will be randomized to either imlifidase desensitization treatment or to a control arm that will receive standard of care, which is to remain on the wait list for a compatible kidney offer and/or receive an experimental desensitization treatment, which may include any combination of plasma exchange, IVIg, anti-CD20 antibody and eculizumab. The primary endpoint measured in the form of eGFR at 12 months after randomization will be used to demonstrate the clinical benefit of imlifidase compared to the control group. For randomized patients who do not undergo transplantation or who lose their graft before 12 months, eGFR was set to 0, consistent with kidney failure. We're really pleased to be moving forward with this study as it brings hope to highly sensitized patients on dialysis in the United States, who are waiting for potentially life-saving and life-altering kidney transplant. Preparatory work has been initiated, and we expect to engage with 12 to 15 leading transplantation centers in the U.S. to conduct the study. Among the new centers are Northwestern Memorial Hospital in Chicago and UAB Hospital at Birmingham, Alabama. Robert Montgomery, MD and Professor of Surgery and Director at the Langone Transplant Institute in New York City has been appointed principal investigator. As I noted earlier, the first patient is expected to be included in the second half of 2021, with a recruitment period of 12 to 18 months before a 12-month follow-up is completed in the second half of 2023. Results from this clinical study could support a potential BLA submission under the accelerated approval pathway in the first half of 2024. Now please turn to Slide 6. A week ago, Hansa announced 3-year data from our long-term follow-up study of Phase II patients that have received imlifidase treatment and transplantation. We are very encouraged by the results, which have been published in the American Journal of Transplantation. The 3-year data demonstrated graft survival of 84% after imlifidase treatment and transplantation. Three allograft losses occurred during the first 6 months with 2 further losses between 24 and 36 months after transplantation, while mean eGFR, a measure of kidney function was 55. In a subgroup of patients deemed most highly sensitized and unlikely to be transplanted with a cPRA score of 99.9% and above, data demonstrated a graft survival of 92%. On top, data also showed an improved kidney function with a mean eGFR at 60 at year 3. The frequency of AMR was not substantially different from what is expected from other desensitization protocols and the literature for highly sensitized patients undergoing transplantation and, importantly, none of the AMRs resulted in graft loss. As I said, we're extremely pleased with these results that support the growing evidence indicating imlifidase is a potent option to enable transplantation among patients with the highest medical need that leads to good long-term outcomes in line with what is expected. Please turn to Slide 7. Turning now to our Phase II program in AMR and GBS. Enrollment in these studies is ongoing under a risk-based site-by-site approach. Both studies were reinitiated at the end of 2020 after being temporarily halted for some time during 2020 due to the COVID-19 pendemic. In the AMR trial, 12 patients out of a target of 30 have now been enrolled, while 10 out of a target of 30 patients have been enrolled in the GBS study. The persistent presence of the COVID-19 pandemic in Europe, most recently, the emergence of the Delta variant is causing new waves of the disease in several European countries where important trial centers are located. This has led to negative impact on enrollment rates in our GBS and AMR programs. To increase the rate of enrollment of patients in both studies, we plan to open additional centers, this summer, reaching a total of 14 centers in the AMR study and 10 centers in the GBS trial, compared to the 7 centers currently enrolling in each of the 2 studies. Given the volatility of the situation we are facing, we expand our guidance for completion of enrollment to also include the first half of 2022. We currently, however, do expect to have a first data readout for both studies in the second half of 2022, as previously guided. Moving on to our anti-GBM program. We've started preparations for planned engagements with the FDA and the EMA and expect to have clarity around the regulatory path forward to a BLA or MAA submission towards the end of this year. Last year, we announced very encouraging data in anti-GBM, which is an ultrarare acute autoimmune disease affecting approximately 1.6 in 1 million people globally per year. High-level data from a Phase II study demonstrated that 2/3 of the anti-GBM patients enrolled achieved dialysis independence 6 months after treatment. Please turn to Slide 8 and a summary overview of our pipeline. As depicted on this slide, thanks to continued progress over the past few years, we have developed a broad clinical pipeline in both transplantation and autoimmune diseases. In addition, we have exciting preclinical projects ongoing in cancer and antidrug antibodies, as well as in the very promising field of gene therapy, where preclinical studies with the imlifidase were begun last year by our partner Sarepta Therapeutics, as part of efforts to develop imlifidase as a potential pretreatment ahead of gene therapy in Limb-Girdle and Duchenne dystrophy. With that overview, I will now hand over the call to Donato, who will take us through a review of the half year financials. Donato, please?

Thank you, Søren. Please turn to Slide 9. As Søren outlined, we made continued progress across our business and pipeline activities. Revenue for the first half of 2021 grew to SEK 13.5 million, including SEK 6 million in product sales. In the second quarter, Hansa recorded SEK 4.5 million in revenue, including SEK 3.5 million in revenue recognition from the Sarepta upfront payment received in July 2020. As mentioned earlier, beyond the positive market access position in Sweden, we do expect additional agreements around reimbursement and market access to be reached during the second half of 2021 and onwards, which we expect will pave the way to further revenue growth. Please turn to Slide 10. For the first half of 2021, SG&A expenses amounted to SEK 141 million compared to SEK 88 million for the same period of last year. The increase is in accordance with our plans and mainly reflects Hansa's broadened commercial activities and organizational expansion related to the launch of Idefirix in Europe, including investments in marketing, market access and supply chain activities as part of those efforts. Our investments in R&D amounted to SEK 102 million for the first half of 2021, which is at the same level as our R&D expenses for the same period last year. Investing in R&D and our pipeline activities across all of our therapeutic areas remain a key priority for Hansa as well as for the long-term value creation of the company. As we initiated the U.S. randomized controlled trial as well as the post approval study in Europe in the second half of this year and progress, our ongoing clinical programs, we do expect our R&D investments to increase in line with our previous guidance. The net loss for the first half of 2021 was SEK 237 million compared to SEK 193 million for the same period last year. The increase is primarily driven by increased activities and ramping up our commercial activities and organization. Please turn to Slide 11. Cash flow from operating activities amounted to minus SEK 233 million for the first half of 2021, which compares to minus SEK 199 million in the first half of last year. As of June 30, '21, our cash position, including short-term investments, amounted to SEK 1.14 billion, corresponding to approximately USD 132 million. With our solid cash position and projected burn rates, we do expect Hansa to be financed into 2023 as previously guided. I'll now hand back to Søren to give his final remarks.

Thank you, Donato. Please turn to Slide 12. Hansa Biopharma's transformation into a fully integrated commercial stage biopharmaceutical company has become a reality in 2021 and we continue to demonstrate good progress across the business and our pipeline activities. Looking at the milestones ahead, we expect to include the first patient in the U.S. trial in the second half of the year and to gain clarity around the regulatory path forward for our anti-GBM program. Due to the continued impact from the COVID-19 pandemic and the increased transmission from the new Delta variant, we may see implications on the patient recruitment for our ongoing Phase II studies in GBS and AMR and therefore, expand our guidance for completion of recruitment to also include first half of 2021 -- sorry, 2022. We currently expect, however, to have a first data readout in both studies in the second half of 2022, as previously guided. Lastly, we expect GLP tox studies to be completed in 2022 relating to NiceR, our next-generation enzymes for repeat dosing. Upon successful completion of these studies, we expect to advance the NiceR program into clinical studies. We look forward to keeping you updated on our progress in advancing our mission to bring life-saving and life-altering therapies to patients with rare diseases while generating long-term value to our shareholders and society at large. Please turn to Slide 13. With this, we are now ready to take your questions, and I hand over to the operator. Please begin.

[Operator Instructions] We have a question from the line of Douglas Tsao So from H.C. Wainwright.

How significant revenue contribution from the imlifidase do you expect to come from nonnational level insurance once those get online? And then I have a follow-up question as well.

Yes. So in general, we expect that the vast majority of sales really will come only after you have national level reimbursement. You may be able to actually activate 1 or 2 centers outside of the national reimbursement system even before getting national level reimbursement on an exceptional basis, on a patient-by-patient basis. And that actually, we have had sales also before, but really that is not something that we really count on. We really need kind of the national level reimbursement decision to be put in place. That is critically important. And so therefore, it's great to be on time with the first such decision, the Swedish decision. And the nature of that decision is also really very positive in that download restrictions in that decision, it's spang on label. And it comes at a price, which is in line with what we want and the value that we bring to society.

And then just a question on the GBM and AMR studies and the expansion in terms of the sites, where are the additional sites being located that you're opening. Are those going to be from a geographic standpoint?

The AMR additional centers. So we're expanding both in Europe but also in the U.S. The GBS trial is a European trial primarily. And the reason why we've especially been hit over the past few months is the fact that we've had some key centers in France, and France has been very significantly impacted and now there is a third wave also hitting France.

Our next question comes from the line of Christopher Uhde from SEB.

So just -- my first question is if you could maybe give us a sense of your trial plans for the, I guess, other organs and tissues. And also, if you can give us any color on the pathway for anti-GBM?

Yes. So for other organs and tissues, obviously, as we've discussed, we definitely see potential for heart and lung. We clearly also see the potential bone marrow transplants. So we are looking at all of these. And obviously, we'll update the market when there is news to share. But clearly, we have the same kind of issue and challenge with the sensitization in these areas. Obviously, hard and long, the volume there is significantly lower than for kidney, but these are very critical situations. Bone marrow is a very interesting opportunity, we think, and certainly something that we're looking at and discussing also with key opinion leaders and others. And then you asked about anti-GBM. And here again, we have received following the completion of the Phase II trial with very encouraging results that I talked to as part of my presentation. We have -- had several rounds of discussions with specialists and received advice from various parties, including regulatory. And we're certainly now getting ready to engage further with regulatory authorities to kind of determine the path forward for this disease. And it should not be underway -- I mean obviously, it's not a huge commercial opportunity. We're talking about maybe 1,000 patients or so between the U.S. and Europe. But this is a very, very serious disease, 2 out of 3 patients lose their kidney function and end up on dialysis and having to wait for a kidney transplant. And there is no approved therapy whatsoever. So there is very, very significant interest from the small society and a network of people, who are dealing with this very serious disease. So we definitely think that this is something that also will move forward and hopefully, with a good outcome also for the patients, who are in desperate need of a solution here. And it's not insignificant if we can mobilize a reasonable proportion of those patients.

And I guess, as a follow-up to that, but would you say that it will -- the path forward definitely would involve a trial?

That's what we expect. We -- I mean based on what has been done at this point in time, we definitely think that a trial will be needed in one shape or another, and this is the focus, what is the next step, specifically, how would such a trial look like given the setup and the disease.

Okay. So then if I could ask 1 more follow-up on this. I mean is it really still fair then -- I mean you described on your website or presentation Phase II or potentially pivotal trials. I mean, is it still fair to describe those Phase II trials in, say, GBS and AMR, for example, as well as potentially pivotal?

So I think for both GBS and AMR what again, is most likely to happen is that you will need to have additional trials following this. So the assumption is not -- the base assumption is not that this is it, and this is the pivotal per se.

Our next question comes from the line of Johan Unnerus from Redeye.

First, clarification, national reimbursement will, of course, be very important. Will you -- can we expect that you will announce them as they will be secured going forward?

So good question. We will not be making announcements each time you receive a decision or something happens in a particular country. We have announced the Swedish decision because it's the first such kind of national level decision. There may be a second because the Swedish decision is very special in the setup in Sweden and actually this is the national level decision, the regions make individual allocations and so on. In other countries, it's more of an automatic kind of setup. So there may be 1 more, but we don't expect our intent to make announcements whenever there is such a decision. And we are on track, as I said, during the call. We do expect a near-term similar decisions. I think the Swedish decision is a great harbinger because again, there is no restriction. And b, the health technology assessment that is underlying this decision is very good, comes up with the conclusion of the business either as we discussed, cost-effective or cost savings. So we expect similar decisions to come in during the remainder of this year but also into next year. We have an ongoing process with a number of early launch countries like Germany, there is a pretty rigid process that's ongoing. Also in the U.K., we're in discussions both with NICE and with the National Health Service. We have -- actually, in Finland, just to update you, this is a bit of an outline that there is no national level kind of process. This is a process where we've gone through with the only -- the single center that is really going to deal with these transplants. And we have an agreement in place. So that, again, Finland is a second country where now for real, we're getting ready to see an update.

But we will update obviously within our quarterly report?

Obviously, we'll come in quarterly reports. And maybe I can take this opportunity also because I've seen some chatter a little bit around quarter-to-quarter sales development and so on. For good reasons, we do not provide sales guidance, and we're really not focused on sales at this point in time. This really is a very special launch scenario. This is not the launch of a chronic therapy for a major disease and intercept this market. This is a transformative specialty product, which is high-value and single dosing, targeting relatively few patients and very few prescribers. It's also being launched into a market where there's essentially no experience or very little experience. We've had a couple of centers in Europe involved in our Phase II trials, but desensitization in these situations is not really done typically in Europe at all. So one should not underestimate the work that has to be done in order to really penetrate, we really believe there is significant potential, but this is not a huge sales uptake year 1 and subsequent years. This will be, as we've discussed many times, an S-shaped launch curve. And what we are focused on from a kind of launch metrics perspective to see whether this is a successful launch or not whether we are on track is a set of other things. First of all, of course, getting the right label approved. We can check that box. We've got a very good label approved, which really focuses on the patients with the highest degree of unmet medical need but also without any kind of artificial arbitrary limitation. So it's up to the centers very much also to determine who would benefit in this particular situation, which is great. The second key thing that we're looking at, obviously, is market access, in particular, getting reimbursement. And as I said, at the national level, this is the key box to tick, and this is going to be country by country, of course. And as I said, we will only see real launch after that essentially. And there, the Swedish decision is great, it's on time and the language is as expected and very, very good. I would say. The third kind of launch metric then would be, of course, supply chain. Are we ready to have part of the market? We already have that. We have commercial sales, yes. So that's something where many biotech companies launching their first product, see significant challenges, and we are on the market that we can check that box. Then we have key opinion leader awareness and interest, which is not a given. But in this case, we're really very encouraged. We've obviously interacted very much with our scientific advisers and key opinion leaders over the past couple of years. But only the launch symposium really kind of showed how broad and deep the interest is. And like I said, 120 transplant surgeons from 13 different countries, representing 80 different clinics. That's a fantastic start. And they just did not participate in a 1-hour sing and dance show. This was an afternoon full of intense, in-depth patient case reviews led by world renowned faculty. So that was really great, and we've received excellent feedback after that launch symposium. Then what we're looking at is, are we enabling specific centers. They need to go through several steps. They need to be trained. They need to put protocols in place and have product available and have access to funding and so on. All of that we are monitoring and we're seeing the progress, we want -- and this is going forward, what will be very important for us. If we can get a couple of the key centers in Europe to try it out on the first patient, potentially a second, that should be tried out on 1 patient first and then maybe a second after having seen good outcomes in the first, then we are on track. So that is a key launch metric for us as well. And then clearly, we're also looking at broader support from the society and in Europe, you have the European Society for Organ Transplantation. We're working with them to put guidelines in place. We expect that to happen, which is really at a very early time in this launch, which is great. And then obviously, we'll be looking at a patient outcome. So it's very important to get the right patients treated by the right centers. We're not trying to maximize patient usage upfront. So I hope this kind of just clarified the situation we're in. Don't look for sales progress quarter-by-quarter. I mean that is meaningless in this case. It's really going to be extremely volatile, will go up, go down and go up. Hopefully, over time, as I said, following an S-shaped launch curve, we'll get there, we see very, very significant commercial potential for a company like ours.

That's very useful. And these key initial centers. I suppose there's a challenge at choosing the right patients, patients that are highly sensitized, but not high risk of failing for other reasons not related to you. On the other hand, with these prices is, of course, an expensive treatment as it should be. It's perhaps attendance to choose these patients.

Yes. Absolutely. That is a risk that we're very cognizant of, right, that there is this tendency when the new transformative therapy is brought to the market that the first patients to enrolled into and to be tried out of those are extremely marginal. And obviously, you can sympathize with them. But really, we want to make sure that we have the right patients treating those that have the best chances of kind of long-term benefit. So this is critically important for us in our dialogues with the centers and which is why, again, we're not really focused on having broad patient uptake early on. And that certainly could be a possibility, but that's certainly not what we want.

Excellent. And finally, this long-term data and feedback is very positive, especially perhaps for the very highly sensitized patient group and this 13-patient cohort also shows very early positive response in kidney functionality, and that's also the primary end point for the upcoming U.S. study, of course, that will be 64 patients, not 13, there might be other differences. What -- how much confidence can we take from that?

No, actually that's a great observation and absolutely an important one. And certainly, it's something that we also find very encouraging, right, that you have in these high highly sensitized patients, 99.9% and above, 92% graft survival and mean eGFR of 60%, right? This is really great. So that's also something that we're looking at as a success metric, what actually we're generating in terms of long-term data, and this is good data.

Yes, it's interesting. And most of that effect seems to come already after 6 months.

Yes, that's correct. Thanks for interest.

Our next question comes from the line of Ingrid Gafanhão from Kempen.

I was just hoping if you could remind us, well, are there specific countries that you seek reimbursement first? I think you already mentioned a couple of them, but if you can just give us an overview of that. And I think just a follow-up on the European situation. What is the timing and the plan for the European confirmatory trial?

Thank you, Ingrid, for those questions. First, on the countries, well, we would expect countries like the Netherlands, for instance, to be one of the early launch countries. And actually, the Netherlands is a country that potentially also could be a very good showcase. There are some excellent centers in that country. We've been working with those for a while that have the ability and certainly interest to do something. So gaining market access in the Netherlands is something that will be quite helpful. As I said, Germany, we have a process ongoing. There's a hearing coming after the summer. And then a rigid process in place for decision-making. So Germany could potentially also come later this year. And as we've discussed, we already have some sales out of Germany, if that was from the first quarter. And then we're also looking at the U.K. In the U.K., of course, you have both NICE and then the National Health Service to negotiate with, and this is happening right now. Whether this leads to a decision this year or whether we get it next year, early next year, I can't say at this point in time. But we would hope also that the U.K. comes online. And then you have some other -- typically some of the Nordic countries already. Finland is essentially, as I said, is open now. This is not a national level decision. This is the 1 single center in Finland. And then we have a few other countries, but most countries really come next year, we expect, including the South European countries. Then the second question, sorry, was on the post-approval study. We're running this, as you know, as opposed to approval efficacy study and we are getting ready to get going over summer. We've had interactions with the EMA and the protocol and expect to have first patient in, in the second half of this year. So that's another great way to really actuate some of the key centers in Europe and have them have positive experiences with the right patients. So that is happening then in parallel with the commercial launch as an important way to prepare for later significant uptake.

That's clear. And if I may, just a quick follow-up question. You mentioned some of the progress that has been done on the preclinical front from your partner programs. But I was wondering, in addition to anti-GBM, GBS and AMR, have you been considering to start any sort of early stage proof-of-concept trials by yourself in the short term?

Anti-GBM, GBS, AMR.

[indiscernible]

Okay. Sorry, I thought you are getting that. We are -- I mean, clearly, we're looking at other diseases that would be relevant. In the auto industry space, there is quite a number of IgG-mediated diseases, where you have rapid disease progression. And we're definitely looking at that. We have not made a decision to move forward. But as you know, we are looking at also potential combinations. We are looking at various approaches to this. So definitely, we see a pretty rich landscape in the autoimmune disease space. AMR obviously is -- it's not really autoimmune. It's more a transplant, but it kind of goes back and forth over the border. And that's something we're just moving forward. We will have no more plans at this point in time other than the ongoing trial.

All right. That's very clear.

[Operator Instructions] We have a question from the line of Charles Weston from RBC.

I have 3 questions, please. First, on the U.S. trial. You've said that the patients being randomized, not to the Idefirix arm, we'll be getting -- or could potentially get experimental desensitization treatment and you name a few that could be used. What is the clinical efficacy that I guess the best case, clinical efficacy that could be expected from all of those other experimental desensitization treatments?

Well, in the U.S., a bit unlike in Europe, some -- very few centers, and actually, we work with them, have used IVIg, rituximab in combination, sometimes plasma exchange and so on to try to desensitize patients, primarily when you have enough time to do it, which you typically don't have when you have an organ from a deceased donor. And the outcome is certainly not satisfactory. I mean, this is -- we talked to Stan Jordan at Cedars-Sinai and he's been the lead -- the pioneer in the space, and he is one of the biggest advocates for litigation, getting this on to the market because it's clearly needed. So there is no established kind of efficacy data. I mean, this is not -- this is experimental. No large studies, no approvals, nothing. But we -- obviously, since patients in the U.S. in general have access to this. But sometimes there could be a very specific case where a center who thinks that this is appropriate to try out. You cannot exclude this from the patients that are being enrolled. But we definitely don't think that this is going to play any major role whatsoever. I'm not sure if any patients will be put on this, but at least they're allowed to be put on this.

That's clear. Second question on the Sarepta collaboration, I think you mentioned last quarter that we shouldn't necessarily expect first data from that collaboration to be years away. Are you able to provide any sort of even rough time frame of when you might expect to see something?

Obviously, when you're working with a partner, it's very difficult and this is also for clinical to make any predictions around when data will become available. What I can say is that we have an excellent collaboration with Sarepta, a great team. They're seeing good progress on the clinical work. We're obviously discussing, what a clinical trial should look like and getting ready for that when that decision is going to be made when there is sufficient kind of basis to do that, I can't predict. But I would -- given where we are, and clearly, with the momentum there is, I would say, at some point towards the end of next year, potentially a decision could be made, but this is not for me to predict. So this is just my few pennies worth.

Understood. And the last question, please, on Idefirix in Europe. How complex is it to actually give the patient Idefirix? So obviously, you've talked a lot about doctors requiring training. Quite a lot of that seems to have referred to the patient, the choice of which patients actually get it. But in terms of actually delivering the drug and the additional logistics that might be in place in terms of staffing, in terms of any other facilities that are needed. Can you just give us a sense of how difficult it is to actually give it for Idefirix please?

To give the product is fairly simple. It's a 15-minute infusion. So what is, of course, critically important, as you also noted, is patient selection and it's patient -- broader patient management, right? Making sure that you manage potential AMRs and minimize the risk of AMRs, as you would do in any of these patients, right? So that is what is important. Having a good protocol in place for that, making sure that staff is trained and knows what to do. So those are the key things. And then obviously, again, there's some logistical elements around organ procurement and how that center receives organs and so on in specific situations and with the imlifidase situation. I mean you have -- it's organ from a deceased donor, you have very little time, right? So you need to desensitize the patient and then be ready to perform the intervention. So it's around those parameters primarily.

Our next question is a follow-up question from the line of Christopher uhde from SEB.

So my -- I have 2 follow-ups. The first one is relating to gene therapy partnerships potentially. So would you say that the -- let's say, the number or intensity of -- or, let's say, the stage of talks has advanced or picked up since the path to approval for the U.S. was set with your trial?

No. So the U.S. design announcement per se has not really changed that at all. I think overall, what we're seeing is great interest. Obviously, as you know, over the past year or 2, many of the gene therapy companies have encountered challenges in many different ways, including durability of response and FDA feedback and so on. And I would say that if anything, that has increased the overall interest. So that's a general statement I can come up with. I think it's important to note that we believe that there is very significant potential in the gene therapy space for imlifidase and other enzymes as preconditioning therapy, not only to just enable gene therapies, but also to improve outcomes, potentially reduce safety issues and increase durability of response and so on. And so that is what we're focused on, kind of the long-term vision, how can we get imlifidase there. And importantly, we remain in control of imlifidase also post the Sarepta collaboration and as part of the Sarepta collaboration. Sarepta collaboration is a great way to advance this agenda to create the data that is needed. And for Sarepta, obviously, it's a great way to differentiate their gene therapies, but also ensure fast access to the broadest possible range of the prevalent population, which is what you want if you are a one of gene therapy company. So that is the setup, we definitely expect. We are certainly working and talking to many different parties, to move many different discussions. Because we don't have all the resources needed. We need access to complementary assets. And so that is ongoing, and some of this will lead to some kinds of subtypes of collaborations and agreements, other talks may lead to other types of setups. Obviously, there is a limit to how much we need and want. And so I cannot predict what will happen at what point in time, there will be further announcements and so on. The general agenda is really to move more in the gene therapy space, and we're very encouraged by what we're seeing, including what we're seeing through the collaboration with Sarepta.

Okay. But so I mean, would you say that it's -- I mean, is it fair to say that any of the talks you're having are advancing?

Absolutely, absolutely. So again, we have talks with a broad range of companies and different topics. And absolutely, you're seeing progress overall. It takes time. There's a lot of complexities involved in this. So that's the overall case.

Okay. And my second question is about living donor transplantation. Is this something that you're planning to do a trial for it at some point in the relatively near future?

So our strategy clearly has been to focus on initially the patients with the highest degree of unmet medical need, and that's deceased donor situations with these highly, highly sensitized patients. But clearly, there are also -- a need -- this is what we hear from many key opinion leaders in living donor transplant situations. And so is that something we'll look at, absolutely at some point. But our focus right now is really on the existing indication. But clearly, living donor scenarios could be relevant for some patients at some point, yes.

Our next question comes from the line of Caroline Banér from Danske Bank.

So good to hear that the U.S. study will progress as planned. So on the back of the confirmation on the study design there, would you be able to say anything on what you expect on the future U.S. label compared to the current European one? Would it be wider restricted and so on?

Good question. We currently expect a label that similar to the one we have in Europe. This is a label that makes a lot of sense, really focusing on the right patients, but also giving flexibility to the centers to make individual decisions and an applied judgment. So we expect and certainly would work towards a similar label in the U.S.

Great. And also a question on the gene therapy discussions that are ongoing. Could you say anything about whether these discussions are with larger or smaller players?

It is with a range of players and with different continent, right? So that's all I can say at this point in time. And companies at different stages, different approaches. And as I said, we are pursuing different things, given knowledge and so on. So it's a mixture.

Our next question comes from the line of Erik Hultgård from Carnegie.

A follow on to the previous question regarding the confirmatory trial in Europe. So I was wondering if you could comment a little bit on the scope of the studied number of patients that would be enrolled and also the enrollment time? And second to that, given that the highly specialized approach that you're taking with a high price point. Do you think that this would have -- or should we anticipate that this confirmatory study would have a meaningful negative impact on commercial sales in the short term?

Good questions. So in terms of the scope of the trial, we are looking at approximately 100 patients to be recruited over beginning this fall. And I cannot give you a very specific time line how fast we will enroll these patients. That depends on multiple things, of course. So that's what I can say at this point in time. As I said, it's a great way to generate experience in some of the centers that don't have it at this point in time and also, of course, generate additional data. We do not expect this to have a material impact on the uptake of the product other than the positive one as I talked to namely that we're generating experience in some of these key centers. So that's what I could say. Hopefully, that's helpful.

There are no questions registered. So I hand back to the speakers for any closing remarks.

Well, thank you very much, operator, and thank you, everyone, for your interest in this report. I look very much forward to continuing the dialogue. This is a very exciting time for the company, and we look forward to reconnecting a quarter from now. Thank you very much. Bye-bye.