Santhera Pharmaceuticals CFO on AGAMREE growth in 2026 - One2One Investor Forum

Well, I'm very pleased to speak to you today. Um, my name is Catherine Istead. I am the CFO of Santhera. um, and over the next 20 minutes, I just want to tell you a bit more about Santhera. Um, to start with, as always, we have a disclaimer, um, please feel free to read this at your leisure.So let's give sort of some uh high level details about Santhera. We are a fully integrated commercial stage specialty pharma company. We are listed in Switzerland on the six exchange and have about 110 employees.We have a product called Agamri. Uh, this is a differentiated product in Duchenne muscular dystrophy, um, a rare genetic disease. Um, the product itself is a steroid, which might sound maybe not so exciting, but, uh, it's what we call a dissociative corticosteroid.So has all the power of the normal er steroids but doesn't have the er the safety or the issues with the safety profile that you normally get with traditional steroids.In terms in terms of our commercial launch, er, that is well underway. er we have approvals in er multiple of geographies, including the US, Europe, UK, China, Hong Kong, um, Switzerland as well as Canada.We have a model where we commercialize the product ourselves in what I call Western European markets. We then have a licensing deals with uh the US and China and now Japan, as well as distribution partners.Financially, we're in a very strong position. Um, our sales for 2026 are expected to be 80 to 90 million Swiss francs, and we're guiding for that to grow to 250 million Swiss francs by 2030.And cash at the 30th of June

was just under42 million Swissfrancs.

So to start with, let me just talk a little bit more around the er the Duchenne muscular dystrophy.Um, as you, you might not have come across this. Basically, this is an inherited disease. About 2/3 of these boys, um, are, have inherited it from, uh, the mother's side of the family. Um, otherwise it does actually appear, um, just randomly as well. It is a progressive loss of muscle strength and function. And unfortunately there is no cure, um, and there's a real high medical need, um, for solutions in this area.The onset of age is around 3 to 5, and you sort of can see the progression of the disease, um, in this slide. So the boy, when they're younger, maybe they seem a little bit clumsy, around 3 to 4 years old. The, uh, the parents then sort of take it, uh, the boy to the doctor and say, so why isn't he able to run as fast or he's falling over, he doesn't seem quite like other little boys at nursery.Um, they then, uh, diagnose, uh, Duchenne muscular dystrophy. It is actually quite easy to diagnose. As the boy gets older, the muscle weakness continues, and then unfortunately by the time they're in their teens, they're in a wheelchair.Um, and then as the disease progresses, it impacts other muscles such as the, uh, the diaphragm for the lungs and the heart, and it does mean that they're then on a respirator, um, in their, their later teens. Life expectancy is around sort of late 20s to 30s.So if I now look at what is current standard of care, well, before our product came onto the market, um, it's corticosteroids. So steroids are really good at reducing inflammation and in the end this, the, the, the progression of the disease is due to the muscles, um, over time causing scar tissue, which means they don't work as well. So in theory, from the day that a.A young boy gets diagnosed through to their their their last last breath, they should be on steroids because this is helping reduce the inflammation in the muscles and this is delaying disease progression. So it's delaying time to a wheelchair, it's delaying time to a res respirator.The problem is with steroids, and you know, you may be aware of this, but while they're very effective, um, there are many serious side effects, so it will come onto that. And because of these side effects, the traditional route is to start them a bit later on. Um, they then can't dose as high as you ideally would like, and then you stop them soon because of just the buildup of side effects are so bad.Looking at the side effects, we've sort of list them here. So from very early on, you get behavioral changes, and you can see this is affecting over a third of boys. You have weight gain, you have gross stunting, vertebral fractures, long bone fractures, cataracts and glaucoma. That's just to name a few.And over time these side effects are cumulative, so they build up in your body and in the end, the parents say, and the boys say, I can't, you know, I can't live with the extent of these side effects. So while they know that the drug is beneficial for their muscles and actually helping extend the time they can use their muscles, the extent of the side effects, uh, mean that they have to stop or lower the dose um of the steroid they're um that they are using.So this is where Agamri comes in. Now, interestingly, for a steroid, now while many er companies and large pharma companies have looked at steroids, they've looked at how can we get, make a better steroid. Well, actually, steroids are very effective.It's the side effects. So when Eric Hoffman discovered a gamma ray, he actually worked with the Duchenne, uh, patient organizations, and they said, we don't need a better steroid, we need one with a better safety profile.So the difference for his screening of compounds was that he looked at where can we have the benefits in terms of the side effect profile. And due to that, he actually came up with a, with a gamma ray. So the great thing about a gammari, R steroid, is that it retains the anti-inflammatory action and efficacy. So that was number one, it has to be as good as the existing steroids. But in terms of the side effect profile, and we'll come onto the actual clinical data.We've already shown that it improves bone health. This means it improves growth, we have improvements in behavior, um, as well as other benefits such as reduction in cataracts and glaucoma. But we'll come onto that that data, uh, in a minute. We've got 8 year data on this now.Why is this important? With the lower side effect profile, this means you can start boys as soon as they're diagnosed. The, the, the, the parents aren't worried um about the the side effects building up.You also can then stay on maximum dose or high doses for longer. That means the drug is efficacious for longer and then you can stay on treatment for longer. So it really is um a game changer um in the DMD space.So looking at Duchenne muscular dystrophy, you may not have come across it, but it is one of the largest rare diseases um that is out there. So there's about 300,000 individuals affected globally.It is one of these diseases that is actually very easy to diagnose. So actually in uh North America and and and Western Europe, about 90% of patients are diagnosed. Now that's not the same, many rare diseases, it takes a long time to actually get that diagnosis. So we know where the patients are. And of those patients, about between 50 and 75% of those are on a steroid treatment. In theory, they should all be on a steroid treatment.But the reason they're not is because of these side effects er profiles.Finding these patients is, is pretty easy too, er, they are treated in specialist centers. So, um, it is, it's a, it's an easier way to, um, to actually find the right doctors to get to the right patients. Uh, and on this slide you can see the number of, uh, patients in the different geographies.So going through to the market size and how we monetize a gamri and how we get our drug to patients, you can see here the total global market size is, is in excess of $700 million. And we break this down into 3 different models of how we generate revenue from a gamri.At the top, we look at Europe. So in Europe, we have our own uh commercial organization. Uh, we're based in Switzerland, but we have teams, uh, throughout what I'll describe as Western Europe. Um, uh, marketing at Agamri, uh, of which we started just over 2.5 years ago in our, our first launch country, Germany.We're continuing to roll out. We actually have 80% of the major markets now covered, and we're now continuing on to roll out in the, uh, the other major market as well as uh some of the small and mid-size markets. Um, in those markets, we obviously, um, uh, our revenue is at 100% of net sales.In 3 other large markets, so that's the US, China, and Japan, we have a licensing agreement. So, a typical agreement there, you have an upfront payment, you then receive milestones on regulatory approval, um, as well as sales milestones, and then we get a double digit royalty income.In the US this has now been also on the market since 2025. It's been doing incredibly well. In China, they launched just over a year ago in the private payer market, and that private payer market is actually already bigger than, uh, than Germany and Austria put together. And in Japan, we signed the deal earlier on this year, so they're working towards, uh, gaining regulatory approval.The size of these markets, uh, as you can see in the US, about $350 million. Uh, China, we estimate around $100 million and, and the same for, uh, the Japan and

South Korea, Australia and

New Zealand.In the rest of the world, we run a distributor market, uh, so a distributor model. So we have a partner in, uh, in what I call Eastern Europe, which is Genesis, and then we have multiple other partners in the rest of the world, and we'll, we'll come on to that. In those markets, we book around about 60 to 70% of net sales, but all of the marketing costs are, are covered by our partner.

I'd now like to go on to talk about H12026. We've had a really exciting first half of the year. Um, so let's go through a few of the, the, the major achievements that we've had.So to start off with, in terms of our commercial launch, we now have 4 out of the 5 major markets, uh, where we have sales. We added Spain and Italy in the first half of the year in terms of the pricing and reimbursement agreements. Um, and we now have sales coming as, as of, uh, July.In the markets where we've been launched longer, er, for example, Germany, um, and the UK and Austria, they are doing incredibly well as well.So in Germany, we had volumes of bottles up 50% if you look at H126 over H125. And in the UK where we've only been launched for five quarters, if you're looking on a quarter by quarter basis, we were actually up 40% in terms of um bottle numbers from Q2 26 to Q126.If I now look at uh the other uh major or one of the other major achievements, um, this was our licensing deal with Nextera. Uh, this, uh, I mentioned this in the previous slide, so this covers Japan, South Korea, Australia and New Zealand.The deal value there is $215 million and included a total of actually a $40 million cash payment upfront, um, of which $10 million was a cash payment itself and then $10 million was an equity stake.We've also continued to work on other countries within Europe, um, some of the smaller countries, so, uh, we've achieved Swiss medic approval in Switzerland, and we now have our first, um, pricing and reimbursement in the Benelux region, um, in Luxembourg that happened, uh, just back in August.We've continued to expand the distributor market, um, we have already have a, an agreement with Bomedica for Russia and we've now included, er, various other countries such as Georgia, Kazakhstan, um, into that agreement.And we've also um got it um our first marketing um authorization in Serbia as well.Looking at 0.5 we generated some very exciting long-term data, which I'll come on to a bit later. So this is data up to 8 years of a gamma use, again, really showing that profile of a great efficacy as well as a huge improvement in terms of safety profile.We've also managed to increase our label. So originally a gammari was approved for um boys aged or or children aged 4 and above. Um, we've now managed to extend that down to 2 years. I think really showing that safety profile. And we also have some real world data um in adults because obviously over time these boys turn into teenagers and into adults and, uh, adding additional data there is really important.We've had some changes to our board, um, and exec team, so we have a new CEO as of July, Orlando Oliveira and Mark Klauser joined as our chief commercial Officer in the summer too. And, uh, Doctor Shrishti Gupta, uh, was elected to the board in May 26.So looking at the, the European market, and as a reminder, um, the, what we call Western Europe is where we sell directly. As I mentioned, we have 4 out of 5 major markets here, and we're working on the other mid-size countries. The last major market, um, is, uh, France.Um, we need to, uh, add additional data to the submission package there, and we're getting an additional cut of our long-term data in the first quarter of 27, and we'll be using that in France. The key point in these geographies is we have a very strong uh pricing discipline. Um, so when we're selling our product, we're selling it for around 300,000 to €45,000 a year.Um, and in places like France, we needed, we need to add additional data to make sure that we can price within that range.Looking outside of Western Europe, I mentioned earlier Genesis, um, our product Agammari is available in 13 countries, um, in that Western European area through one route or another. And actually what was great news is earlier this month, uh, we added Poland to that. Now, Poland is actually the 6th largest country, um, in Europe in terms of population. So you could say we now have 5 out of 6, largest countries now, um, having a gammari available.Looking at some of the successes um in er the Western er European markets.And this just really I think shows how well a gamma ray is being taken up by doctors and patients.So I'd mentioned in Germany that volumes were up 50% over the first half of 25, but I think more importantly, we already have switched 40% of DMD patients onto a gamri from the older steroids.I think for any company to have a 40% switch within 2.5 years of launch um is, is an amazing achievement and I think it really shows the benefit that agamri brings, um, and the demand that's there from the patient groups, the parents, um, and the physicians to give something that doesn't have the extent of side effects of these older corticosteroids.Um, we've also, um, in the UK, um, had, you know, good growth there as well, as I've mentioned. Um, we're now getting the benefit of being updated, uh, on treatment guidelines, and we're getting increased adoption as well.If I look at Austria, which has been launched around the same time as Germany, so about 2.5 years, we're actually already at about 50%, um, of all steroid patients are now using a gammari, so that's, that's the highest percentage, um, but we've already achieved that in 2.5 years.So, to go through some numbers, um, in one of the earlier slides, we talked about how we see Western, um, Western Europe or our Western markets being around about €150 million of sales, and over the next two slides, I want to explain how we get to that.So on the left hand side you see the number of DMD patients, that's around 13,000 in Western Europe, of which 50 to 75% are on steroids. As a reminder, really all of them should be on steroids, but it's been the side effect profile, um, that has stopped patients either continuing on steroids or not starting steroids.Of that 8000, we're targeting about 60%. So we, we think of the current steroid market, we should be able to gain at least 5000 patients. I don't think that's unrealistic at all, bearing in mind that in Austria we're already at 50% of the market share and uh in Germany at 40% market share.But there's certainly room to grow.To move on to the next slide here,

so

at thebottom here you can see the 5000 patients. And to explain how we get to €150 million of peak sales, we then take into account the annual cost. This is based on the weight of the patient and therefore from that you can get the number of bottles a year. On average, for our average patient at the moment, that is 15 bottles a year.In this calculation, we've assumed that on average, the cost is €2000 a bottle, and that's how you get to the annual cost of 30,000 a year. However, if I look at Germany, we're at €3000 a bottle, so that is €45,000 a year. And in our other European markets, um, and Spain and Italy, which we've just, uh, received approval, we're in that 2000 to 3000 range. So, I think being have looking at 3000.A year is certainly on the conservative side.So looking at the full market side, I certainly think that we can achieve more than 5000 patients with us, our superior side effect profile, and I think our initial assumptions of €2000 a bottle a year, I think is on the Conservative side too.In terms of expanding into the rest of the world markets, just geographically, we've had a lot of uh agreements signed over the last 1415 months. So this just shows the number of countries that we've added um over the last 15 months. Uh, that's in addition to Israel and Qatar that we already had signed pre uh mid 2025.The one area that we still have to sign up is the Latin America region and particularly Brazil, and we are working on an announcement there before the end of the year. So we certainly have, we're in the final stages of discussions. Once we have that,

we reallydo have,

er, the rest of the world um covered in terms of being able to provide a gangway to patients.So just going back to the agreement we signed in January with Nextera, so as a reminder, that was a 250 million um buy world deal um for Agamri in Japan, South Korea, Australia and New Zealand.Uh, 40 million upfront and up to 175 million in sales and regulatory milestones. There's an excess of 2000 DMD patients, um, in Japan and about 60-70% of those on steroids. So, uh, Nextther is now working on studies to actually be able to gain approval, uh, and we're looking at that, um, over the, the coming few years to actually move ahead in that area.In terms of Catalyst, so Catalyst, um, that has now been acquired by Angelini, um, in the middle of the year, reported very strong, um, growth. Um, they had revenues in, uh, the first quarter of, of 36 million. Um, that was up from, uh, uh, 22 million if you look at the same quarter in the previous year.Um, they have been guiding to 140 to €150 million so 140 to $150 million for the full year 2026. Um, and as a reminder, we get royalties on that in the double digit range. Uh, Catalyst is now in about 90% of the DMD centers of excellence, so they've, uh, had a really good expansion.Um, so, yeah, we're delighted of how Catalyst is doing, and, uh, we look forward to them continuing on their growth.In China, um, we have, uh, a slightly different situation that, that, uh, Spirogenics, our partner, um, is in the private payer market. However, already with only about 1 year in that market, they have 1000 patients treated. So that is actually more than, than Germany and Austria put together, um, since launch, um, 2.5 years ago. They may well look to get what they call NRDR listing, which is the government listing.Um, and they're working towards that potentially over the next year or two. There is, however, a, uh, a, a, a counter to that, that the price will need to go down. However, obviously the number of patients potentially treated will grow significantly. So we watch as, uh, they continue to progress in China.So just quickly going on to some of the, the data that I mentioned earlier. So this is data that came out in March 2026, so just earlier on this year. We have already seen an uptick, quite a significant uptick from Q1 to Q2 on the back of this data.So this is data from patients, um, up to 8 years of treatment with a medium follow-up of 5 years. And what it showed was this comparable efficacy over the long term to what are called classical corticosteroids, but with far fewer, uh, side effects. And we'll go into that and I'll show you some of the slides, um, um, over the next 2 slides.So I just quickly look at vertebral fractures. So what you can see here is on the red slide, you can see that nearly 42% of patients had a vertebral fracture. This was reduced by about 80% taking a gammari. So this is a huge reduction in vertebral fractures.fractures, and the reason for this is that we have normal bone turnover. So this is something that we'd noted earlier on, but now we have that data showing, uh, this big improvement in fractures and also in terms of the severity as well, um, you can see this big reduction, so that's the slide on the right hand side.The next two slides, the one on the left shows efficacy, and actually what you want here is an overlap of, uh, the classical corticosteroids, which are in red with a gammari, which is in the blue. And what we see again over the long term.Is that we have equivalent efficacy, and that's really important. I think early on when we launched, there was a question that our people were happy that happier that we had a better safety profile. They wondered if we were as efficacious over the long term, and we've now shown that.On the right hand side is a chart which shows standard deviation from a normal growth um of of of these boys. So actually a flat line means that you're growing normally. A line that goes down shows that you're increasing the number of standard deviations from normal height, so that you're becoming shorter and shorter compared to the an average boy of that age. So we're delighted to see that on a gamri, our boys grow normally.Um, and unfortunately, as is well documented, um, if you're on steroids because of the reduced bone bone turnover, these boys don't grow as well, so they are very short. Now, while having a, uh, a boy of normal height is, is obviously, you know, very good for self-esteem.The other thing that has been noted by our KOLs is if you have normal height, the chances are you have a normal um lung volume because your lung size is related to your height. And with that, we're now looking at whether with the increased lung volume that increases the time to going onto a respirator, and that's some of the data that we're working out, uh, working through over the coming 6 to 9 months.So to move on to some numbers, um, these are the numbers

for

the

first half of the year to the 30th of June.

So our revenues grew incredibly well, up, uh, just

over 100% to

48.3 million Swiss francs. This was due to the very strong growth in a gammari sales, um, as well as the upfront from the Next era licensing deal.

Looking at product

sales, so this is from our distributors as well as ourown product sales,that they grew nearly 50% in the first half of the year. And as a reminder, that doesn't include any sales from either Spain or Italy. They started in July, so we're expecting that certainly to continue on strongly in the second half of the year.Milestones and royalties increased to

29 million,

particularly driven by the upfront payment from Nexthera of just over 24 million. Um, the only area where wewere slightly lower is oursupply of products and services to partners. So this is where we actually sell bottles to our licensing partners. Um, we knew that Catalyst was going to transition to direct sourcing.Um, after Q1, uh, 26, and that is what they've done, so that will be lower. However, this is a very low margin business. Um, we, we charge a small markup on these products, so between 10 and 20%. Um, so, uh, so this doesn't really have a, a major impact on, on our cash flow, but that is slightly lower, um, in, uh, will, will be for the full year 26.In terms of global Agari sales, they exceeded $175 million. Um, this has triggered, uh, a milestone payment to the originator company, um, of $20 million. Um, but in terms of operating expenses, they were down 8.4% to $25 million. So well on track, and I'll come back to our, our guidance, but we're guiding for OPEC to be, um, in the 50 to $55 million for the full year.Um, in terms of cash, uh, as I mentioned, I think at the very start, we had just under 42 million Swiss francs of cash at the 30th of June.So

looking at guidance for

the

full year

and longer term guidance, uh,

we expect product sales to continue togrow,

uh, strongly, so up by more than 50%, uh, for the full year, with total revenues at the 80 to 90 million range. Uh, we expect royalty income to increase year on year, and milestone income, of course, will be higher due to the upfront payment that we receive from Nexttera.And as previously discussed, our revenues, or low margin revenues from products and supply and services will be down, but that was as planned.Operating expenses in the 50

to 55 million

range, although from our first half, uh, we are looking like we're gonna be at the lower end of that range. And in terms of our cash, I think this is important that we're gonna be approximately cash stable through to the end of the year from our, our current levels. There will be a slight decline.Um, in the first, uh, quarter of 27 due to some inventory purchases, and, and also there is a, a German pricing reduction that impacts us, um, from the start of 27. Um, but as we go into

the second half of the

year, we expect that, uh, um, the cash profile to move to cash accretive, and we do not see any requirement for any additional funding.Longer term, uh, the outlook that we've talked about is total revenues, excluding milestones of 140 million by 2028, and that grows to 250 million Swiss francs by 2030.We're expecting quite a bit more

news flow, um, as we go into the final quarter of theyear

and into 27. So further pricing agreements and commercial launches in the mid and small size EU markets. Um, a distribution agreement, um, in South America,

obviously focusing on

Brazil.

Additionaldata, and long-termdata from our, our

Guardian

study,

as well as updates in terms of indication expansion from our licensing partners.Um, we know for example, Next there is also targeting, uh, Japan regulatory filing in the second half of 27.So the final slide, um, talks about strategy and particularly what happens beyond a gammari.

So the four,buckets here, um,

I think are very important.

The first one, talking about our commercializationin Europe,

yes, we need to continue on, um, uh, expanding our sales in Europe, getting into those smaller markets. Um, and we'll continue on doing that, and you can see how well we've done the first half of the year.

In terms of geographicalexpansion, the major one here is

Brazil and er Latin America, and we look to have an announcement shortly on that.The red one I think iskey.

We have an infrastructure um across um

across the Western European countriesthat

can

add one or two products to it.

So

we are lookingto, uh, make synergies from that coststructure

by adding.

One or two latestage or commercialstage products to our rep's bag.

So we're not going to be looking to take on commercial risk, but for example,

ifit's a US company, um, that wants to launch in the US but just sees Europe is a bit too complicated.We'll be very happy to take on their products to launch that through Europe. Um, we're looking also to China as well for potential, um, products as well,

so that, that's a core focus of what

happens beyond a gamri.

In

terms

of additional indications, um, our partners are looking at additional indications.

We have the opportunityto opt into those indications at a later stage, so we won't be spending our R&D money on that, but we will follow very carefully what our partners are doing, and that again potentially could expand the Agamri market to beyond D&D.

Um, withthat, I, I hope that has given you,uh, a nice

overview of, um, of Satherra, uh, and what we're looking to do.

We've hada, a very exciting 2026, and, uh,please reach out to

the

team here, um,if you'd like to learn more about us. Thank you.