The next company presenting today is Hansa Biopharma, a commercial stage biotech working in the space of treatments for patients with very immunological conditions. With us today, we have here Søren Tulstrup, the CEO of the company. Søren, the stage is yours.
Thanks so much for the introduction. Thank you, everyone, for your interest in Hansa Biopharma. I'm first gonna give a brief overview of the company, then hopefully there should be time to take your questions. Before I start the presentation, though, first, the usual warning that my presentation today contains forward-looking statements, therefore you should apply appropriate caution. For those of you who are not familiar with Hansa Biopharma, here's a very brief high-level overview. Founded in 2007, we sit on top of a validated platform, a platform that has been validated in at least three ways. First, we have an approved product in the exciting area of transplantation.
We've also generated a strong proof of concept in the large area of autoimmune diseases through a disease called anti-GBM in a phase II trial. We have made a forceful entry into gene therapy through now three collaborative partnerships in that space. We have an exciting and broad pipeline, not just in the transplant space and in the autoimmune disease space and gene therapy space, but even in the oncology field. Our organization counts approximately 160 FTEs. Even though it's a young organization, as you know, a collection of individuals, it's a very experienced organization, where the average tenure in the industry is more than 20 years, and where more than half of our employees have relevant PhDs.
It's also a very international organization, with people representing more than 30 different nationalities, based in Sweden, across Europe and in the U.S. Last year, we had three financing transactions, raising $110 million, and we're financed into 2025. Just a few words on the platform, which consists of a number of enzymes that all share the property that they very fast and effectively Cleave Immunoglobulin G. They're specific to IgG. They do not touch other immunoglobulins, and they cover all the four subtypes of IgG.
What they do is that they essentially decapitate the IgG molecule just below the so-called hinge section, creating a Fab2 and an Fc component, and through this, they inactivate the IgG molecule so that within two hours from a 15-minute infusion, as you see on the right-hand side of the slide here. The levels of IgG drop down below detectable, and then they stay down there for approximately seven days before bouncing back. Then within a couple of months, you're back to normal. This is different from what you see from for instance, plasma exchange therapy and FcRn inhibitor therapy, where you also see IgG levels drop, but only down to maybe 20% or so, the starting level, and it takes approximately one week to get there.
This is immediate, and it's complete. Based on this platform, we have built a an exciting and broad pipeline of valuable drug candidates in transplantation. First, autoimmune diseases, gene therapy, and oncology, as I said, initially. Our approved product is to enable kidney transplants in so-called highly sensitized patients. This has been approved in Europe. We're running a pivotal trial in the U.S., and we're also running a post-approval efficacy study in Europe. We have a phase II program that has been completed in another transplant indication called AMR. These are episodes of rejection following a transplant, and we've done it specifically for kidney transplants.
We reported on the primary endpoint late last year, and we'll report the total results of the study later this year. We have just completed enrolling a GBS study, a phase II trial in GBS with 30 patients, and we expect to have a readout later this year. As I said initially, we have generated strong POC in anti-GBM, an ultra-rare autoimmune disease also affecting the kidney. We've just started a pivotal trial where the aim is to include 50 patients across approximately 50 centers. In the gene therapy space, we have, as I said initially, 3 collaborative agreements. The most advanced is with Sarepta in Duchenne muscular dystrophy, but we also work with them in the area of limb-girdle muscular dystrophies.
Here, Sarepta has communicated that they expect to take imlifidase into the clinic later this year. The Pompe disease program with Bayer is still at the preclinical stage, as is the most recently concluded partnership with a French company, Genethon, for Crigler-Najjar. The oncology space, we're looking at both improving the efficacy, potentially of immuno-oncology therapies, as well as hematopoietic stem cell transplantation, currently at the preclinical stage. Finally, we have the next generation of enzymes for more frequent dosing moving forward, through the lead candidate, HNSA-5487, which has been taken into the clinic and is currently in a phase I trial in healthy volunteers. I think this really depicts better than the standard pipeline chart, the kind of the growth avenues and the potential of our pipeline.
As you see here, at the core, we have imlifidase, which is very efficacious and well-tolerated, but which can also in addition to being used alone, can be combined with other modalities to deal with IgG, pathogenic IgG in your body. For instance, we're looking at a potential combination with FcRn inhibitors. Outside of this, we have the next generation of enzymes, with HNSA-5487 being the lead molecule, where we're looking at developing drug candidates that can be dosed multiple times, and through this, essentially, opening up a universe of more chronic diseases. We have at least four overall growth avenues here in transplantation.
We have targeted the kidney, first, but there are other organs, obviously, that are relevant for transplant enablement, like the lung, and heart. We're also looking at post-transplant situations with AMR, as I talked to. In the gene therapy space, initially, we focused on limb-girdle Duchenne, but we're also looking at Pompe and some other diseases, Crigler-Najjar, and there's a host of other diseases in the gene therapy space that are relevant, and I'll touch on that briefly. In autoimmune diseases, again, we're active in anti-GBM and Guillain-Barré Syndrome, but there are many other IgG-driven autoimmune diseases, both monophasic, acute autoimmune diseases, as well as more chronic autoimmune diseases. So that is overall the broad growth universe that we have.
Many stepping stones ahead. Let me now turn to the most advanced project we have, which is imlifidase in kidney transplantation. Looking at Europe and the U.S., are approximately 180,000 patients on the wait list. 10%-15% of these patients are what we call highly sensitized, meaning that because of a previous transplant, a blood transfusion, or multiple pregnancies, the immune system has been primed so that it's very difficult to find a matching kidney. Looking at the numbers here, obviously, there is an undersupply of kidneys, of organs, and therefore, the addressable patient population is, we estimate, 4,000-6,000 between the U.S. and Europe, but that's a substantial number that we're looking at here.
We have launched in Europe, and obviously, one of the first hurdles to pass is to get market access. We have executed very well here. We most recently communicated that we have gotten access in Spain, so we now have full access in the top five markets in Europe, representing 2/3 of all kidney transplants. In addition to this, we're also in a situation where we have access in a number of smaller and medium-sized countries. Turning to the pipeline, and first, the autoimmune disease space. Initially, we focused on anti-GBM and Guillain-Barré Syndrome. Guillain-Barré Syndrome is in the nervous system category. Anti-GBM is a system or a disease that affects the kidney, as I said, sometimes the lung.
There's a host of a long list, I would say, of other tissue and organs that are relevant if you look at the overall autoimmune disease space, from blood to the GI tract, lung, nerves, brain, the hormonal system, kidney, as I mentioned, and the bone and muscle set up. Just looking at the numbers, as you can see, we've just started to really scratch the surface here through our anti-GBM and GBS programs. This indicates the number of patients in each of the disease areas here. As you can see, in all of these four categories, there is substantial growth potential. Anti-GBM, very solid data, generated in POC in a phase II trial, albeit in a limited number of patients, given that it's an ultra-rare disease.
In this phase II trial with 15 patients, we demonstrated that imlifidase can prevent all of the patients that had not progressed to dialysis from progressing to dialysis and even taking some of the patients that had gone on dialysis off of dialysis. That's really very solid and, as I said initially, we now started a pivotal trial in 50 patients and recently communicated that we have the first patient in here. Guillain-Barré Syndrome just completed enrollment for that trial and expect readout later this year. Approximately 10,000 patients are affected annually between the U.S. and Europe. IVIG is essentially the standard of care.
Mortality, though, remains relatively high, 4%-5%, and up to 40% and up in respiratory support, and it takes up to 6 months-12 months to get back to normal. There's a high degree of unmet medical needs, and we're really looking forward to the readout later this year on safety tolerability, and next year on efficacy versus a matched cohort from a patient registry. Looking at the exciting area of gene therapy, obviously, there's been much progress over the past decades, and especially the past few years, with approved products. What remains a major challenge for essentially all of the AAV-based programs is the issue of neutralizing antibodies against the AAV vectors used.
What we can offer is essentially, again, an approach that can neutralize these neutralizing antibodies and create a window of opportunity where the transduction of the healthy gene can take place. We demonstrated that in non-human primates, and that data set was most recently presented at a large conference in the U.S. very exciting times. As I said initially, Sarepta has indicated that they will take imlifidase into the clinic as conditioning therapy ahead of their own gene therapy program in Duchenne. Looking at the numbers and the growth opportunity, there are very substantial patients out there to capture.
We've again, only started to scratch the surface through the Duchenne, Pompe, limb-girdle programs, and we've even now put the Crigler-Najjar program here onto the slides. Again, if you look at some of the other disease areas in this space, there is really a substantial number of patients. Again, depending on the specific vector used and the specific indication, up to 70% or so of the patients may have issues with too high titers of neutralizing antibodies. The way we've gone about the gene therapy space is that we've not out licensed our molecules. What we have done is we have made an arrangement so that we will essentially book the sales. That's the case in Sarepta, where we negotiated downstream economics as well.
Sarepta will finance the clinical and also the preclinical development program. In the case of AskBio, we have just negotiated kind of the feasibility, both the preclinical and clinical feasibility study, and then they have a first right to negotiate for downstream economics if they want to commercialize the product and take it all the way to the market. Genethon is again using the same model. Overall, we still have control of our own molecule here in the ultra-rare disease called Crigler-Najjar. Many things to do given our platform, the versatility and flexibility of the platform. In this situation, obviously, it's important to have clear strategic priorities, and we do.
Clearly, top of the list is to continue the successful launch of Idefirix for kidney transplantation in Europe, get the approval in the U.S. There's a significant opportunity in the U.S., continue progress in our late-stage pipeline and get our molecules in GBS and anti-GBM approved, and then move our programs in gene therapy and potentially hematopoietic stem cell transplantation forward. Importantly, also move our next generation enzyme, HNSA-5487, forward. Last slide here before questions. Just to say that obviously, we are very much focused on ESG, and we have a number of specific objectives set up in three broad buckets. We wanna contribute to making people healthy, to create a healthy business, and also a healthy planet.
We have a separate report on all our ESG goals and initiatives. With this, thanks so much, and I'll hand back to you, Gonzalo.
Great. Thank you very much, Søren, for the nice presentation. We have a few minutes for questions now and a discussion, and I will start touching base on the commercial uptake of imlifidase that you have in front of you. I mean, now you're mentioning that the five core markets in Europe have granted reimbursement decision, very positive. I was wondering, how big is, let's say, if we have to weight this compared to doctors actually deciding to allocate organs to highly sensitized patients, I mean, where would you put, what's more important in terms of potential revenues for you?
Well, getting access obviously is a precondition, right? We're very pleased with the progress we've seen here, having essentially gotten access to the vast majority of the market potential now at what we think is a price point that adequately reflects the value we're bringing to the table. As you say, obviously, you also need to look at putting protocols in place and making sure that the organ allocation systems are tweaked so that there is a steady flow of organs to these difficult-to-transplant patients. Here we've seen a good progress now with the Eurotransplant system being tweaked now. It's being implemented in June. That's an organ allocation system that covers Germany, Benelux, and a few other countries.
They have put in place a system where some patients will be identified, initially 20, and then they will be allowed to receive organ offers, and they will evaluate the progress on an ongoing basis. That's great to see. We also have a good system in place in the U.K., and we hope to see that starting to kick in later this year. Spain, we got reimbursement recently, but it will take a little while before that has been kind of cascaded down through the regions. That's a big opportunity for us as well.
Great. The second question is on the second indication that you have there, it's AMR. You scored positive results, and they were announced, like, a few months ago. We haven't seen data yet. Hopefully, we'll be able to see it soon. Independently of you file for accelerated approval or if you have to run a larger trial after, once it's approved, in terms of market uptake, do you see a faster market uptake compared with imlifidase in the transplant setting? Probably it's a little bit more complicated in terms of logistics, for the AMR would be a little bit more simple. What's your view on that?
Yeah, I mean, obviously, the prescribing situation or utilization situation is different in AMR because you have a patient that has been transplanted and suddenly has an issue, and there's a high risk that he or she will lose the kidney. You need to do something acutely, right? You don't have the whole issue of organ allocation and so on, and patient selection and so on. It's a much easier market, if you will. Having said that, it is also a challenging market to generate data in, right?
Yeah.
We're very pleased with what we saw from our phase II. We've released the data on the primary endpoint. We think that it's important to add, you know, another approach to dealing with these difficult situations.
Yeah. Great. Next question it's on the gene therapy segment, I mean, that you are collaborating with Sarepta.
Yep.
Sarepta will probably, I mean, we don't know yet, but probably by the end of June 22nd, I think it's the decision for their gene therapy for DMD, for a specific group of patients, 4 years- 5 years old, probably. What is your view on this potential approval, and how much does it impact your development if it gets approved or not approved in your development way with them?
Obviously, we would want the product to be approved and sooner rather than later. Having said this, you know, actually the development of imlifidase is not dependent on this molecule getting approved. Again, the collaboration we have with Sarepta is around all of their Duchenne programs in gene therapy and all of their programs in limb-girdle. This is the most advanced one and really looks like they will get it approved, you know, get an accelerated approval based on a surrogate marker of efficacy, which is great, not just for Sarepta, but for the entire field of gene therapy. We have high hopes that this will happen on the 22nd of June.
It looks like initially it'll be with a certain limitation on the age, 4 years- 5 years, but they will generate data relatively soon afterwards, and then potentially the label can be expanded.
Yeah. No, great. I wanted to ask you about something more like early stage. I mean, a few weeks ago, I don't know if you saw it, there was a paper that was published showing that in MS, in multiple sclerosis, it seems that IgG aggregates can contribute to neuronal and neuron death. I was wondering if it's this kind of degeneration or, yeah, neuronal death, if it's something you are considering exploring in the future?
I mean, this is early days, right? I mean, I think on a continuous basis, without being specific, we look at all the different potential applications and where there is an unmet medical needs, right? I think, as I said, initially, it is really a versatile and flexible platform that can be used in multiple different therapeutic areas.
Yeah. Oh, great. One last question is on your new collaboration with Genethon, also gene therapy. I was wondering, now you will start running early stage experiment or preclinical work. How much can you use from your Sarepta, like, collaboration for this part? I mean, how much time, let's say, or resources can you save in that part?
In general, obviously, I mean, there's a learning curve, right? There's also data set that can be used in different areas. Obviously, we expect that we'll be able to cut the timeline as we progress. Specifically with Genethon, we do think that there is an opportunity to get into the clinic relatively quickly because we have experience in this space. They have extensive experience, actually, one of the leaders, and they even have experience specifically with imlifidase. We have high hopes here. It's a very exciting program, and, you know, an area with huge unmet medical need, even though it's an ultra-rare disease, right? Yeah, very exciting.
Yeah. Genethon, could you explain us how this works? I mean, Genethon would take the project at the beginning, do they bring it towards the market?
That's not their model, right? This is a nonprofit organization, even though they're one of the leaders in the gene therapy space, right? They were founded by a patient association decades ago. They played a huge role in sequencing the human genome back in the beginning of the 90s. They have been involved in both the LUXTURNA and ZOLGENSMA programs. Some approved gene therapies. Really a leading player, but their model is not to commercialize their own programs, right? They find a partner.
Yeah.
In this case, you know, with the Crigler-Najjar program, which is really exciting, right? As a standalone proposition. You know, we even have a first right to negotiate, you know, should we want to commercialize that program.
Great. Thank you very much, Søren, for being here with us today and for your presentation.
Been a pleasure. Thank you.