[Foreign language] Erik Penser Bank [Foreign language] Hansa Biopharma [Foreign language] Head of IR, Klaus Sindahl [Foreign language] .
[Foreign language] , Ludvig. I hope it's okay that I shift to English, just to make it more specific. My name is Klaus Sindahl , and I'm head of Investor Relations since four years back, and very excited to be here today to meet you all. For those of you who are not very familiar with Hansa Biopharma, I'm presenting here a recap of the company today. Hansa Biopharma is a Swedish company, commercial stage biopharmaceutical company. We are based in Lund, in Sweden, and we are listed on Nasdaq. We have a very validated technology with imlifidase, our first generation of antibody cleaving enzyme, which has approval in so-called highly sensitized patients for kidney transplantation.
But we also have proof of concept in the first autoimmune indication, namely anti-GBM. Then thirdly, we have also three partnerships within gene therapy, which I will address later on in my presentation. So from a lot of parameters, we have a validated technology, and as I mentioned before, we're also commercial stage and are ramping up sales in Europe, where we got approval three years ago, and also planning on getting approval in the U.S., where we are running a phase three study. Beyond kidney transplantation, where we have approval, we have a tail of clinical development programs. Actually, we have seven ongoing programs, soon to be eight. And mostly it's in kidney transplantation and in autoimmune indications.
We have also now started to move into the clinic with our second generation enzyme technology, the HNSA-5487, which is intended for repeat dosing. We are around 160- 170 people in Lund. We have our own commercial infrastructure in place in Europe, and we have a very purpose-driven culture, and many of our employees have a long background within life science, with PhD backgrounds, and we have operations both in Europe and in the U.S. From a financial perspective, we are financed into 2025. We had end of the second quarter cash of roughly SEK 1.1 billion. And as I mentioned, we are listed here on Nasdaq in Stockholm and have around 20,000 shareholders.
Going back to how the company was established and developed, we are like a spin out from Lund University, and the company is based on a technology which was developed by Professor Björck and his team in Lund, and with a antibody cleaving enzymes, which stem from a bacteria called Streptococcus pyogenes. And what this enzyme does is it's fast and effectively cleaves IgG antibodies. So from a 15 minute infusion, you see it here, you can basically inactivate your IgG level, which is roughly 80% of your immune system. And that's supposed to have good effects and address multiple indications.
First and foremost, of course, in transplantation, because you create a 5-7 day window to do a transplantation in incompatible patients, but also to deal with acute autoimmune attacks, so in some cases, in some autoimmune diseases, you see patients, for various reasons, be attacked by the immune system, and you want to rapidly knock that attack down, and imlifidase is, at least what we have seen so far, a strong compound to do that in a safe way. As I mentioned in my introduction, we have currently seven clinical-stage programs. We have three programs in phase III, and then we also have a number of preclinical programs, especially within gene therapy, so we have a broad pipeline, mainly focused around transplantation and autoimmune indications initially.
But what you also see is that we have three partnerships in gene therapy, namely with Sarepta, Genethon, and AskBio, in covering four indications. So this is, you know, a new thing for Hansa to pursue, and we see a big opportunity within gene therapy, and I will address that later on in my presentation. If we move into our lead compounds and the commercialization of imlifidase or the commercial with the commercial name Idefirix in kidney transplantation, then we got approved or conditionally approved in Europe for so-called highly sensitized patients. Those patients are patients who for various reasons, it could be multiple pregnancies, it could be previous blood transfusions or transplantations. They have too high levels of donor-specific antibodies to find a matched kidney in the allocation systems.
So they are basically waiting against hope, many, many years. They have a higher likelihood of actually dying than finding a match. So this is represented in the orange box on this slide, and constitutes around 10%-15% of patients on the wait list. So if you look at Europe or and the U.S. combined, you have roughly 170,000 patients on the kidney wait list, and 10%-15% of those cannot really access a kidney, but are just waiting, as I said, against hope. But now with our technology, our commercial product. We actually believe we can deal with this issue, because we have proved or demonstrated in four phase II trials that we have enabled kidney transplantation in 46 patients with 100% success.
Very, very unique, and that's also why we could get approved based on four phase II studies, which is very rare. But it also leads to a conditional approval, so we have to take out this drug in the market in a diligent manner, and we are also supposed to run a post-approval study in 50 patients, actually at no charge, which is also a you can say an overhang here in the first phase of the launch. But we have good evidence that the drug works. We have the first patient cases being published by hospitals. I'm referencing two cases here on this slide. So for instance, we have a 54-year-old male in Barcelona who had been on dialysis since 1984.
38 years of dialysis, where he had to go to the hospital, get his blood cleaned for antibodies, 3-4 times a week, sitting there in a chair 4-6 hours. He basically had no life, no energy to take care of his family, take care of a job, and he was actually successfully transplanted here last year and is doing good today. We also had a 20, excuse me, a 29-year-old lady from the Netherlands, who was transplanted at Erasmus in Rotterdam, also last year, after she's been facing kidney disease since childhood and had undergoing two failed transplantations, back in the 1990s, sorry, back six years ago. She was also successfully transplanted and is doing good today.
We had similar cases in Italy, and more recently, a case in Austria was also published. So very, very good real patient experiences. I want to show you this slide because what it requires to come out or launch a transformative new technology like imlifidase actually requires a lot of steps or a lot of milestones to form a new market because there was no real treatment options for these type of patients. So what we have spent the last 2-3 years is actually building the foundation for future sales to grow. And as you can see to the far left, we have actually now checked off all these boxes. So we have commercialized in the early launch countries, targeting leading clinics and centers.
We have secured market access now in 13 markets, including the Big Five, so UK, Germany, Spain, Italy, and France. We have also ensured that these clinics we are targeting, they are ready, they are clinically ready for taking on patients. We have engaged with a high number of KOLs to increase the awareness around our drug. And then we have also initiated this post-approval study, which we are supposed to do until 2025. And finally, what's also important to acknowledge is that we have finally seen the allocation systems, such as Eurotransplant, allowing for a new tier of patients to be allocated organs.
So, these sophisticated allocation systems, in the past, it was not really possible to allocate kidneys to incompatible patients, but now with the change we have seen, specific allocations are now carried out to imlifidase specific tier of patients. And initially, Eurotransplant is targeting 20 patients for this, which we hope to demonstrate in our product sales this fall. And then going forward, we should see more accelerated growth because we have now built the foundation, we have now seen the first repeat business, good outcome, et cetera, so we should see a much more steep ramp-up. It's been all about having these foundations, these good cases, case reports in the hospitals, and now it's about expanding commercially. Yeah, just a slide on Eurotransplant.
Eurotransplant is like a mediator in Europe. It covers eight or nine countries. As you see, it's the Benelux, it's Germany, it's select geographies in Eastern Europe. Eurotransplant has, up until now, had an Acceptable Mismatch program, which is depicted here in the orange box. They have now established this new pilot, initially targeting 20 patients, which can be treated with our drug. That will definitely support the pickup in countries like Belgium, the Netherlands, and Germany going forward. There's actually 100 patients on this program, but initially the focus will be on 20 patients. Mid-July, we also announced that we had the first provisional approval in Australia, so this is a complete new market for us.
What is very interesting about Australia is that we actually got the first approval in living donor transplantations. In Europe, it's been around deceased donor transplantation, which is not necessarily expanding the market, but living donor transplantations, here you have relative. You can get an incentive to actually donate your kidney to a relative, right? That's actually something we believe will expand the markets of organs. Australia as such is also a very interesting market, since more than 15,000 patients suffer from end-stage renal disease receiving dialysis today. Around 857 patients carry out transplantation every year, and 21% of those patients have a CPRA score of 95 and above, so really fits our definition of patients for imlifidase.
Here in the last part, I will move into the autoimmune space. In the autoimmune indication area, there's over a hundred different types of autoimmune disorders, and we will be targeting those which are more acute, because imlifidase is intended for monophasic diseases with an acute need to take down the antibody level. Around 10% of these diseases we can treat with our first generation, and then with our second generation, we can potentially address even more in the chronic space.
More specifically, we have ongoing programs in anti-GBM and in GBS, where we have demonstrated very good data in anti-GBM, where 2/3 of our patients could actually become dialysis independent versus normal standard of care, which lead to 70% of patients actually become dialysis independent or even die after six months. So a very, very severe indication. Even though it's ultra rare, we actually believe it's an interesting opportunity where we could take large markets here. GBS is another program where we have just completed an enrollment in 30 patients this spring, and expect to announce high-level data later this fall. What is also very interesting is that we have a new investigator-initiated trial in ANCA-associated vasculitis, which is carried out at a German hospital, Charité in Berlin.
Here, the intention is to treat 10 patients with severe ANCA-associated vasculitis. Vasculitis, ANCA, ANCA-associated vasculitis is inflammation in the bloodstream, which leads to damages of the blood vessels and leads to kidney failure in 25% of the cases. So it's also a very severe indication with incidences of to the tune of three patients in 100,000 people annually. So very interesting third program in the autoimmune space. If we move into gene therapy, there's actually a large number of programs being launched.
More recently, Sarepta got the product launched in Duchenne muscular dystrophy in 4-5 year-old boys, and we have, as you know, a partnership with Sarepta, and we'll commence a clinical study here later this fall in these patients who are suffering with neutralizing or high titers of neutralizing antibodies. And the issue as you can see here on the slide is that antibodies will prevent this viral vector to carry out the gene therapy. It will simply prevent, so it will be a barrier when you are supposed to replace the infected gene so through the cell.
So it's basically the same mode of action as we have seen in other indications, where you can cleave the antibodies fast and effectively, and then allow for transduction to happen. So we have today three collaborations or partnerships with leading gene therapy players. First and foremost, Sarepta, where we have two programs in Duchenne muscular dystrophy and limb-girdle. And as I mentioned before, we have now moved into or will move into the clinical phase with Sarepta in supposedly a small trials. We're talking about less than 10 patients, and hopefully, we have data from that study already sometime next year, which then could lead to a path forward and potential commercialization in a not too distant future.
Another collaboration is with AskBio in a disease called Pompe disease. This is still in preclinical phase, and with AskBio, we have an exclusive option for the downstream economics, which has not been settled yet. So it's more around feasibility. When it comes to Sarepta, we have actually agreed all the different phases, the milestones, but also commercialization. Here we are liable for up to $400 million in milestones, and on the sales, Sarepta can address with our compound. We are liable for up to mid-teens in royalties and can also book the sales of imlifidase for those patients. So a very attractive deal.
Genethon is also built around feasibility initially, with an option to determine downstream economics later on, and that's in a small indication called Crigler-Najjar. So we started out that collaboration in the second quarter, and hopefully, we can move into the clinic already early next year. Yeah, just a slide here before we wrap up on Duchenne muscular dystrophy, where Sarepta recently got approval. And this is a very severe indication. So it's mostly boys with an incidence of one birth in 3,500-5,000 , and these boys are typically diagnosed at the age of 3-5 , and if they're not treated, they would typically end in wheelchair at the age of 12, and most would actually die at the age of 26-30 years.
It's a very severe indication. Here, Sarepta now got approval in 4-5 year-olds to actually replace that infected gene with a healthy gene, and 14% of those patients cannot be treated today because of these neutralizing antibodies. This is what we intend to address within Idefirix later this fall, and hopefully we have the first data out next year. Just a summary slide on our platform, because we have these multiple spaces, these multiple opportunities. First and foremost, of course, we have commercial here in transplantation, but as you understand, we have many opportunities in other spaces, autoimmune indication, with two phase II programs and one phase III program.
And then we have a number of opportunities in gene therapy, and are also looking into oncology, potentially hematopoietic stem cell transplantation. And then we have not even addressed our second-generation enzyme, the NiceR program, or what today is called HNSA-5487, where we have actually now completed enrollment in 36 healthy volunteers and are looking on a path forward, later this fall once we have the data from that study. So with that, I will hand it back to Ludvig in the Q&A.
Thank you for a nice presentation, Klaus, as always. So my first question is related to Australia, and you received a conditional approval there. What is the plan with commercialization here? Are you gonna do it your own, with your own sales force, or are you gonna have a partner? What's the plan?
Yeah, actually, Australia is an exception. In Europe, we have built up our own commercial infrastructure. We have territory managers in a number of countries, and it's also the intention to do similar in Australia. We already have an affiliate there and build out a commercial infrastructure for that specific market. But having said that, we actually want to address some of these rest-of-the-world markets through partners because we don't necessarily have the local expertise and understanding of these markets. So that's actually the intention in other markets. But for Australia, we will do it ourselves, and it's a very nice market. It's also the first time we got living donor transplantation-
Mm.
On label. During the four phase two studies, there were a number of living donor transplantations carried out, but we focused initially only on deceased donor transplantation to get that label and approval.
Mm.
For Australia, we now got it, and hopefully we can go back to EMA and even FDA later on and discuss that opportunity because it's also very interesting given that you can expand the pool of organs.
Yeah, absolutely. It's a big opportunity-
Absolutely.
in living donors.
Absolutely.
And are you planning to conduct any studies on living donors alone?
I mean, we already had a number of living donor transplantations-
Right
... during the four phase II studies, which is also the basis for Australia to approve on.
Mm.
The full approval, it's right now a provisional approval. The full approval is also based on, you know, the kind of data we'll generate with the post-approval study in Europe, but also the U.S. ConfIdeS study, so they are actually only deceased donor.
Mm.
So it's not like we necessarily have to focus on a specific living donor study, but of course, they will be followed long-term.
Right. Yeah. I don't know how much you can say about this, but you mentioned that you are seeing repeated sales now at the-
Mm
... clinics in Europe. How many clinics have you reached out to so far, and how many of these are you seeing repeated business at?
Yeah, we don't really disclose sales on a clinical level or country level, but we can say that France has actually been driving sales so far. It's been a great opportunity. It was the first market of the Big Five to take on imlifidase. They had experience from the clinical programs, and we got an early access through our market access, right? So there's a number of clinics in France who have already seen repeated business. But the intention is still, when you go to new markets, to secure a good outcome in the first patient, then they will typically monitor that patient for 4-6 months before they will take on the next one.
Mm.
You need to generate good experience in order to have that repeat business and generate comfortability around our drug, because, I mean, we are still in a conditional approval mode, right?
Yeah.
So we don't want anything to go wrong in this phase.
Yeah, that makes sense. And Eurotransplant, you mentioned this is an important driver for the sales going forward.
Yes
... now with this accelerating phase that's coming, hopefully. Can you tell us what it means, the new guidelines? What does it say?
Yeah, it's more the allocation system which has been adjusted, right? Because previously, there was an acceptable mismatch program, which is still existing, but that mismatch program did not allow for incompatible kidney transplantation, right? I mean, because of these high level of donor-specific antibodies. But we have a disruptive technology which actually deal with that issue. So now they have started a pilot program, initially targeting 20 patients, which will be included in rounds of five patients during this fall. And then they will monitor how it goes, and hopefully they will be able to address hundreds of patients afterwards if successful. But we see it as a significant driver for our business going forward. Yeah.
Okay, great. I see that you only have 30 seconds left, but the phase III trial in Europe, can you tell us something about the status there?
Mm.
You haven't disclosed very much about it.
The phase III-
The confirmatory in Europe.
Yeah. So, we are well ahead, according to plan. So we use this to generate experience in a number of new clinics. But the thing is also, it's also used in some of the commercial clinics, so it's also an overhang.
Oh.
I mean, there is some cannibalization between some clinics, of course, because they treat one or two patients, you know, at no charge. But it will also help, you know, build investment and experience. So, clinics, you know, are more comfortable around the drug going forward and which will lead to sales, more sales eventually.
Yeah, but short term, it's kind of stealing, and it's from-
Yeah, you can-
... commercial.
You can say that, but it's also-
Yeah
... you know, generating experience-
Yeah
... which is good because it's necessary.
Yeah. Thank you very much for this presentation and Q&A.
Thank you. Always a pleasure.