Santhera Pharmaceuticals Holding AG

Good afternoon ladies and gentlemen and welcome to the Sancero Pharmaceuticals half year results investor presentation. Throughout this meeting attendees online will be in listen only mode. Questions are encouraged, they can be submitted at any time via the Q&A tab that's just situated on the right hand corner of your screen. Please just 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 will post your responses where it's appropriate to do so. Before we begin, we would just like to submit the following poll, and if you could give that your kind attention, I'm sure the company would be most grateful. And I would now like to hand you over to the team from Santerra Pharmaceuticals. Catherine Shabir, good afternoon.

Good afternoon, everyone, and welcome again to the interim results for the six months ending the 30th of June, 2025. Giving the presentation today is myself, Catherine Isted, the CFO, as well as our Chief Medical Officer, Dr. Shabir Hassan. Unfortunately, our CEO, Dario Eklund, cannot be with us today as he's actually had a recent operation on his shoulder, but he will be back with us shortly. As was said in the introduction, there will be an opportunity to ask questions at the end of the presentation, so please add these into the box as we go along. To start with, as always, we have a disclaimer. Please feel free to read this at your leisure. While I appreciate this is a results call, we thought it's worthwhile spending a bit of time going through who Sanfera is and what we do, and also a reminder about the D&D market. So I will start off with this small snapshot of the company. So we are a Swiss-listed company, listed on six, with our global headquarters in Basel, with about 110 employees in total, about 150 including contractors. We have a product called a Gamry. This is a differentiated product in the Duchenne muscular dystrophy market, and it is unique because it is a dissociative corticosteroid, and we'll come into that in more details later. Agamri is being rolled out worldwide and we have approvals across the US, Europe, UK, China and Hong Kong. And our own commercialisation in Western Europe is going well with launches in Germany, Austria and the UK. We've also had a good launch in the US from our partner Catalyst. In terms of financing, as we announced this morning, we have a new financing for 20 million Swiss francs. This means that we have a runway through to cash flow break even by the mid-2026 and our cash at the end of June 2025 was £18.4 million. I'll now hand over to Shabir to go through a bit more details about the DMD market.

Thank you, Catherine. Just for those of you who may not be familiar with Duchenne muscular dystrophy, I'm just going to give you a very brief overview and then go into a little bit more detail about the value proposition and why we have such confidence with the Molero. Duchenne is a lifelong neuromuscular disorder characterized by progressive loss of muscle strength and function. These are the main characteristic features. The thing to understand about it is that currently there is no cure. It's such a large gene with so many mutations that current technologies just will not afford a cure. At best, you could try to moderate the disease to be a little bit milder, and that's really the aim of most of the therapies coming to market. Age of onset is very young, three to five years of age. Children often start to present symptoms of weakness and inability to walk and run and keep up with their peers. And what this means is that they actually present very early to physicians and to the health care system. Unfortunately, life expectancy is only limited to the mid 20 to 30 years of age. You get a progressive weakness with the loss of ambulation, no ability to run, walk. You end up in a wheelchair. You get weakening of the upper limbs. But really what's more important to understand is as the disease continues, you get weakness of the heart and lung muscles, leading to cardiac or respiratory failure, which is the predominant cause of death. Current therapies today Let me talk a little bit about corticosteroids. Corticosteroids have been and will remain the cornerstone of treatment with Duchenne muscular dystrophy. They have 20 years of experience and data behind them, a robust data set, perhaps the most robust data set of all therapies at market in the moment. They have been shown to delay progression of disease at all stages, so the ability to run, walk, they delay the time to getting into a wheelchair, they delay the loss of ability to use the upper limb, hands, fingers, fine motor skills. But they also now show an impact in terms of a delay to cardiac function decline and respiratory function decline. So they have shown robustly this ability to slow down the disease progression at all stages. And that's why it's absolutely crucial to try to encourage patients to stay on the drug as long as possible. However, corticosteroids do have limitations. We know that they are relatively toxic. We know from experience across all diseases, all therapy areas where they're used, that these can be limiting in terms of the ability to maintain a high dose and to be able to stay on drugs. The challenge with Duchenne, you're diagnosed at the age of three to five, but you often start a treatment about seven to eight years, and this is because of the toxicity. When you give steroids to children at such a young age, you're impacting really important periods of growth and bone formation and behavior. Children aren't able to stay on therapeutic doses for long enough, often having to down titrate within about two, three, four years of treatment, and then having to stop very soon before you start to see the benefit on the cardiac and respiratory function, which would be really important ones to try to moderate. And of course, they stop very quickly. This is a graph depicting what I think is a very nice way to show the key challenge in terms of how physicians try to keep patients on steroid treatment. If you want to stay on maximum therapeutic dose, over time the toxicity accumulates. When you start steroids, early you see behavioral change in weight gain, which is common to all steroids. As you stay on drug beyond two to three years, you start to see an impact on growth, vertebral fractures, long bone fractures, ophthalmological conditions, eye conditions, and then eventually hypertension and insulin and diabetes. And so really, with the pivotal data that we had some years ago, we were only able to demonstrate the benefits in the short term, the behavior and weight gain. We showed some data showing that we didn't impair growth. But now, of course, the focus is on the longer-term data. Agamory, just in summary, is a dissociative corticosteroid. It's the first dissociative corticosteroid to be approved. We've demonstrated that we have the anti-inflammatory effect associated. the corticosteroid treatment. In the short-term data, we were able to show that we don't impede growth, that we don't have a negative effect in terms of bone health, and that's both bone biomarkers, but some early data on bone density. We showed a benefit in behavior. Now, of course, we're focusing on the longer-term data. We've got children who've been on treatment five to seven years, and we're currently analyzing these data and hope to make an announcement somewhere in quarter four about the longer-term effects and some of the more deleterious side effects. But in essence, because this treatment is tolerated better and the side effect profile doesn't really impact children of a very young age, and this is growth, osteoporosis and bone fractures, we see a gamma ray being adopted earlier in terms of the treatment algorithm. We see patients are maintaining a higher dose for longer, and some of that evidence will be coming out with the data announced in quarter four. And, of course, we see patients are able to maintain treatment for longer than when we compare them to natural history. What makes CMV very attractive, of course, is it's one of the larger of the rare diseases. There are around 300 individuals affected globally. Of those in North America and Europe, 90% are diagnosed because the symptoms are very obvious and parents report readily to healthcare systems. The steroid utilization varies, and that's an opportunity for us to, of course, expand segments where current usage may not be as much. North America and some countries in Europe have a very good standard of care, but there are opportunities in some other European countries where steroid use could be improved. Patients and parents, of course, are often treated within specialized treatment centers, and that's an advantage for a rare disease but also a small company. be efficient to be able to commercialize this product. And of course, physicians have been using this drug for decades. They're very familiar with it. There's nothing very different about the Molarone that it can't be adopted immediately, of course, because there are guidelines. In fact, we're now seeing guidelines clearly adopting a position for the Molarone, which is encouraging and will continue to work with the community to educate them on that. With that, Catherine, I hand back to you.