Santhera Pharmaceuticals Holding AG

Good afternoon and welcome to the Santera Pharmaceuticals investor presentation. Throughout this recorded presentation, investors will be in a synonym mode. Questions are encouraged and could be submitted at any time via the Q&A tab situated on the right-hand corner of your screen. Simply type in your questions and press send. The company may not be in a position to answer every question it receives during the meeting itself. However, the company can review all questions submitted today and publish responses where it's appropriate to do so. Before we begin, I'd like to submit the following poll. I'd now like to hand you over to the management team. Dario, good morning, sir.

Thank you, Lily. Good morning. And for those in the US who are joining us, a good afternoon for those in here in Europe. And welcome to this full year 2025 investor call. As usual, I'm today joined by Catherine Eastead, who's our chief financial officer, as well as Shabir Hasham, who's our chief medical officer. They will both give their respective parts of the presentation and be here for the Q&A as well. So can I have the next slide, please? Can we have the next slide after that? Here we go. No, the disclaimer, please. Can we go back? So I just want to draw your attention to the greetings from our legal department here where you should at your own leisure read this if you're not familiar with the text. And with that, let's move into the actual presentation. So 2025 was a real exciting year with expansion and global momentum continuing. These six points that you can see on this slide here really are the summary of our presentation today. We will have deeper dives on each of the six subjects. But I just want to highlight a few things here that, first of all, in Austria and Germany, we are continuing to make great progress. In Q1 of this year, we actually had 50% growth. over last year, which means that in Germany, we're well over 40% penetrated in the glucocorticoid steroid market. And in Austria, we're actually already hitting numbers above 50% market share. This is in the first year or shortly after the first full year of launch. We're also making good progress with reimbursement across Europe. A few weeks ago, we announced that we had reached an agreement with the Spanish authorities for a reimbursement in Spain. We're expecting to launch there in June or July. And in Italy, we announced today that we have an agreement with AIFA. It's still pending the publication in the official Gazette, which is expected in six to eight weeks, after which we can officially launch also in Italy. We've also seen fantastic progress with our partner Catalyst in the US who delivered 117 million of US dollars of sales in the first full year and in China our partner Spirogenics is also advancing nicely with the self-pay soft launch which I'll speak to in a few minutes. At the end of last year, we were very busy with partnering negotiations for Japan and Korea, and we decided to also wrap in Australia and New Zealand into a deal. I'll speak more about that in a minute, but that's a great achievement to complement our global footprint. And speaking of global footprint, we also last year signed up the five remaining countries of the GCC, India, Turkey and Russia for distribution partnerships. And we're currently in negotiations, late stage negotiations with our partners, potential partners for Latin America. And then finally, Shabir will speak about this later in the presentation, the Agamri Guardian study has had its first tranche of analysis presented at the MDA conference in March in Florida, and the results are truly spectacular and a real testimony to the power of Agamri differentiation. Moving to the next slide, please. So let's start with a quick update on Germany. I want to remind everybody that Germany was a market or is a market where we had no pivotal clinical trial sites. So when we launched in Germany, we had zero clinical experience in the market with Agamri. And yet the uptake is beyond impressive. We have now, as I said, 40% plus of the patients switched. The initial patients in the first 12 to 18 months were switchers aged six to 12, but we start to see now a broadening of the patient population where patients older who have either stopped taking steroids altogether or have never started are switching to or starting Agamri, as well as the newly diagnosed patients for five-year-old boys who, as a rule, almost now all get Agamri in Germany. The price in Germany, it's worth mentioning, was originally 3,600 euros per bottle. That was agreed with the German government for an 18-month period. So the January 1st, 2026 price has dropped down to 3,000 euros per bottle. That was a pre-agreed price reduction that was anticipated from our side. As you can see now, we are almost halfway through the patient population, the combined market, Germany and Austria. Those are both markets with a relatively low penetration of glucocorticoids in the DND population. 50% to 55% of patients only are taking steroids as a standard. In other European markets like the UK, Spain, Italy, as well as in the US, that percentage, the equivalent percentage, is well above 70%. So we believe that in Germany and Austria, as we start seeing more and more data come out from the Guardian and the long-term extension studies, we believe that this could also be a market growth opportunity because many of those patients who are not taking steroids in these two markets should medically be on steroids, but are avoiding it because of the toxicities and the side effects. Can I get the next slide, please? So here's a quick overview of our launch progress. It's obviously driven by the access in the various countries. Germany and Austria, as well as the UK and Spain are check marks. We have reimbursement there and in Spain, the launch is imminent. In Italy, we announced today that we have a price agreement with the AIFA. And as I said, in six to eight weeks, when the Gazette publishes the official notification, we'll be able to launch. In the Nordics, we're somewhat delayed. We had hoped to be able to wrap up all the Nordic countries by mid-year, but it now looks like it could be delayed by three to six months. In the Nordics, I will not go into a lot of details on the negotiations there because it's different from country to country. But as a whole, we want to include the Guardian data into the health economic modeling that is being done in the Nordic countries to make sure that the price that we achieve in the Nordics is in line with the price that we have achieved in other markets. The same goes for Benelux as well. In France, we have decided to withdraw our application and resubmit entirely. Our initial ASMR5 was just not sufficient for a reasonable price agreement with the French government. We believe that with this long-term extension data that we have now, we can get an ASMR4 in the next round, which would then allow us to negotiate a price that is in line with pricing in other European countries. The resubmission is expected in Q3 of this year, and as many of you know, that process could take up to a year, so we probably won't have reimbursement in France until the second half of next year, and France is likely going to be the last country in Europe to have reimbursement for Gammerin. In Switzerland, we're in the process of negotiating with the BEAGE, and we're expecting to have reimbursement in the third quarter of this year. Next slide, please. A few words about our licensed partner markets. Catalyst in the US is making excellent progress with the drug in the US. We had 117 million of US dollars in revenue last year above the guidance that they had originally given. This also triggered a milestone payment to us, which had its cash flow early in the year. And the guidance they provide for this year is 140 to 150 million. I want to remind everybody that Catalyst has their first quarter earnings call next week on Wednesday. In the US, it's a very concentrated market. They have about 90 centers of excellence in the US, where more than 90% of the patients are treated. And to continue the theme of 90, about 90% of those centers have actually started prescribing Agamri as a routine drug for their boys and young men. Moving to the next slide, the other licensing partner in China, which is Spirogenics. They have an early access program slash out-of-pocket pay soft launch ongoing right now. There are about 800 patients cumulatively that have been treated with Agamben in China. This is obviously more than we have in Germany already, and this is just the self-pay portion of the launch. They're submitting for what's called an NRD, a National Reimbursed Drug Listing, in China in the fall of this year. with the expectation that the reimbursement will be granted as of early 2027. Next slide, please. And our final licensing partner of the three licensing deals that we have, the new one, which is Nexera. This is a mid-sized Japanese company. It has management sitting both in Japan as well as in London. They are having their own affiliates in both Japan and Korea, and they're looking to find partners for Australia and New Zealand where they can either sub-licensed or distributed for Nexera. Nexera is a fully integrated company, so they have their own regulatory commercial and manufacturing activities and capabilities. And they will be basically manufacturing the product themselves in Japan. Well, they will have either manufacturing done through a third party in Europe or manufactured locally in Japan. And what is worth mentioning here is that this team is very familiar with the drug. The Japanese and Korean affiliates of Nexera were actually acquired by Idorsia, which obviously was the company that had the initial rights to Vamoran. And the CNC group, the technical group that is now part of Nexera, is actually the former Edorcia team, and they're sitting here near us in Basel. So that collaboration is functioning extremely well. There are two options for the launch timing in Japan. If Nexera is not required to do a registrational bridging study in Japanese patients, We could be looking at a launch towards the end of 27, so the end of next year. Should they have to do a bridging study, which we don't expect, the launch would then be a year later in around end of 28. Next slide, please. So this takes us to the geographical overview of where Agamri is now partnered and where it is already available or will soon be available through our partners and our own organization. So we signed up Turkey, GCC, India and Russia in the summer and fall of last year. We signed up through Nexera, South Korea, Australia, New Zealand and Japan early this year. And we're expecting a partnership announcement for Brazil slash Latin America sometime this summer, so in a few months time. So that will cover then most of the globe. And we continue to work closely with these both distribution partners and licensing partners to get product locally either reimbursed or through other methods paid for so that the revenues from that start flowing back to us. Just as a reminder to everybody, for distribution partnerships, we typically retain about 60 to 70 percent of the partner's net sales. It depends on the contract, but 60 to 70, so about two thirds of the net sales of these partners come to us. And obviously, that's a very high incentive for us to support them and work closely with them. So with that, I'll change tact and we'll move to a clinical update. And I'll pass it on to Shabir to give you a quick update on the Guardian data and why we believe this is very significant for the differentiation, market access, and uptake of the drug. Shabir. Thank you, Darian.

Last month, we were delighted to be able to present the outcomes of our long-term data collection at the MDA meeting. It's a prestigious meeting based in the US, where many of the world's experts in Duchenne, both physicians and patient advocacy communities, convene. We had an extremely positive reaction to the data, both from the clinical community and, as Dario has alluded to, we've had a very positive reaction to the data from market access and payers. We will continue to dig and learn more about the Mall Run and its unique attributes. But for now, I'm going to present to you just the highlights of the data that were presented last month and indicate where we think these will continue in terms of our strategic growth. The long-term data collection is an open label study where we followed up 110 boys who originally started the Molarone in the clinical development program. So they started when they were young. They are now being followed up for up to eight years on average five, which is a really great period to learn about where differentiation occurs because many of the side effects of course occur with continued use of steroids around the three to four years of age. In terms of efficacy, we were very clearly able to show comparable long term effectiveness to classical corticosteroids, both prednisone and deflazocort. And this has very important implications in terms of how we think market penetration will continue. In terms of tolerability and safety, we were very pleased to show an 80 percent reduction in the risk of spinal fractures. And now this is actually the second study in which we've been able to do this. We were able to show maintenance of height. As you know, Duchenne is a terrible disease and then inflicting growth stunting on these boys causes a huge social issue. So I'll go into that a little bit more. We were also able to show cataracts are significantly lower than deflazocort and no glaucoma has been reported to date. And we've shown that there are no new safety signals emerging, which again gives us continued confidence in terms of the benefit risk profile. Now that the data have been presented, we are aggressively submitting manuscripts, which will be submitted probably in the next few weeks so that the data will be available both to the clinical community, but also, more importantly, for our colleagues in the field to be able to educate the physicians and patient groups. And we're planning additional readouts from the long-term data collection over the next three years annually, so there will be continued data flow. Next slide, please. This is an extremely impressive graph. Here you can see the impact of the Molerone in terms of reduction in spine fractures. What you will see in the panel on the right in red are the number of patients who experienced a fracture on deflazacort. Just a reminder deflazacort is perceived currently to be the more efficacious corticosteroid, but we have now shown absolutely clearly that we have comparable efficacy, but the Flascord is also associated with some of the more severe side effect profiles. Here we can show very clearly an 80% reduction in the number of patients who have fractures, but also on the panel on the right where you see the blue and orange, we have not only fewer patients, but we have also very importantly, less severe fractures. We show also in the next slide, On the first panel on the left, this is a Kaplan-Meier plot where we show time to loss of ambulation. This is an extremely important milestone in terms of disease progression when children lose the ability to walk. It's one of the more robust ways to measure efficacy. We've shown here in red the time to loss of ambulation for prednisone and deflazocort together pooled, and in blue for vermorin. You'll see there's absolutely no difference between the two. We've also shown similar outcomes when we separate the individual corticosteroids. And so now we are able to confidently communicate to our clinical community that there is very comparable and durable efficacy for both. The reason this is really important is not only does this give confidence in the long-term outcomes of the Molyneux, but it also now gives physicians the confidence to be able to switch patients who are currently on a classical corticosteroid with the confidence that they will maintain efficacy. On the right hand side is a graph showing growth. And you'll see in blue that it's a flat line. This is a Z score. It means that height for age is kept. So children on vermolorin grow as would be expected as if they were not on a corticosteroid. In red, you'll see the same comparison for the pooled prednisone and deflazocort cohort. You'll see that there is a significant widening of the curve, about 12.1 centimeters at five years. This is a huge change in the mindset of the physicians. There was a dogma previously that you had to keep children short to be able to maintain efficacy. So physicians in some ways were forcing children to have a stature comparing a 12 year old looking like a six to seven year old. We have now shown definitively that height has no impact on efficacy, that children can grow normally, appear normal and retain efficacy. This is also another aspect where we will be in terms of future research focusing. If children are taller, it means that physiologically that they will probably also have larger lung volumes because lung volume and thoracic cavity are associated with height. We're very excited and we have already started amending protocols to collect respiratory function data retrospectively which means that somewhere second half of next year we should be able to do an analysis to show that not only do we maintain efficacy and we have normal height but we may actually for the first time ever be able to define a new efficacy outcome measure in terms of respiratory function if it holds true so that's something that we're adamantly um focusing on and very excited to to bring to market in the next few months with that i'll hand over to catherine just to talk about um how we're expanding our manufacturing capabilities and some financial highlights