Diamyd Medical AB (publ)

Hejsan hejsan allesammans. Välkommen till den rapportpresentationen som handlas av oss här på GV Pondkommission. Linus Svensson heter jag och med oss idag så har vi D&I Medical tillsammans med bolagets vd Ulf Hanelius som ska få presentera rapporten här för det första kvartalet för perioden 2024-2025 som kom ut imorse. Hoppas alla haft en chans att gå igenom den. Kvartalet i sig och även de senaste veckorna har bjudit på en hel del spännande nyheter, både kopplat till studierna som bolaget har på gång och även den kommersiella potentialen. Jag kan säga redan nu att det går bra att börja skicka in frågor via chatten om man skulle vilja göra det. Och så tar vi upp dem efter att Ulf egentligen har gått igenom kvartalet och sin presentation. Jag tänker att vi hoppar direkt in. Så varsågod Ulf!

Tusen tack. Det är roligt att det är så många som loggar in på den här presentationen. Som sagt, jag kommer att dra, jag har inte många slides, men jag kommer att ha två slides som är lite mera ingående och framförallt tala om kanske de två största nyheterna under kvartalet, vilket är både vår alignment med FDA, alltså vad vi kring det här möjligheterna, accelererat godkännande and commercial potential. These two images will be more relevant when we discuss them. We will also be able to ask questions after the presentation. To sum up briefly, the big thing is that we now have the opportunity to move towards an accelerated approval in the USA. Our launch indication is type 1 diabetes, but mainly stage 3, where we are now clinically diagnosed with type 1 diabetes. We focus on the genetic subgroup that we have seen in previous studies, linked to the effect of our treatment. And as I said, in 2024, we got fast track designation, that is, one of their fast track programs for all stages of type 1 diabetes. And as part of that, there was the opportunity to open up discussions with FDA about the accelerated approval, where we had a first meeting in the summer of 2024, where we got the thumbs up for accelerated approval. And then we have a follow-up meeting now in December, where we could still more clearly agree on all of the most important key components before a previous review and an accelerated approval. And then we also have a previous sales status, which is important, especially when you go to the market due to pricing and the like, which we can also discuss. And we now have almost only one year left until the den tidigare avläsningen i pågående fas 3, det vill säga vi förväntar resultatet runt mars 2026, så det är inte långt bort. Om det är positiva, starka resultat, så har vi då möjlighet att börja skicka in en marknadsansökan i USA, vilket är jättestort. Då är vi potentiellt sett först ute i hela världen på den marknaden. Vi är först ute som har möjlighet också i borgerliga taxerier att godkänna en antipettiabetes. Most of you know what we do, our main treatment is scandinavian thiamine, but just briefly to lift up perhaps the three most important points here. It is a simple treatment, which is important. That is to say, it is about three injections, one month in between. It is done in an out-of-the-box way, but you don't go to the hospital or anything like that. There are other treatments of what you do. And then after the three injections, we re-program the immune system, and then we maintain our own insulin production. So we actually change the slope of this curve, the disease progression, so we make it less steep. And one of the most important things we have is our safety profile. We have so far treated with the active drug more than 1,000 people with the different studies of type 1 diabetes without any serious side effects. And it is extremely important, above all also for a potentially accelerated approval, because when you do not have a complete effect data from the whole study, it is absolutely important that you can show that you had a lot of safe treatment, which makes it easier also for authorities to be able to approve something like that compared to if you would have a safety profile that looks different and that they have to make a decision. Do we dare to approve or not approve this? This is very important. And here you have to say that there is no other treatment that has a special status in the United States that has such a large safety database as we have. And that is also important. And precision medicine, we are talking about a genetic subgroup of type 1 diabetes. About 40% of type 1 diabetics have the right genetic profile to get an effect of our drug. And precision medicine is really the way forward now within several different indications. And we have, as the slide shows, the first the first precision medical fast-trace study ever conducted before type 1 diabetes, which is currently being conducted in Europe and the USA. A total of about 60 clinics. In the summer of 2024, we did a so-called futility analysis, that is, a futility analysis that was blind, 70 patients who had been born in six months, and it could then show that green light from the DSMB Security Committee to continue the study without any modifications, which in practice means that there is some kind of effect there yet. It was a blind analysis, so you don't know exactly what the result looks like, but in any case it is not active and possible to be equivalent at that time, but there is some kind of effect, the safety is good, so it reduces the risk further in the study. And now, as it also says in the report, we have randomized more than 200 patients in phase 3, and in November 2024, we would have received more than the 170 patients that are needed to make this earlier assessment with a 15-month review, which can lead to an accelerated approval. So the study is in full swing and very good as well. Then a little more detail about this FDA meeting we had in December. When we had our meeting in July with FDA in Washington D.C., we were given the thumbs up for being taxed and approved, and to use C-peptide as the primary effect parameter. C-peptide is the biomarker for the production of its own insulin. It's the first time FDA has given a thumbs up for that. So this really breaks new ground for type 1 diabetes and immunotherapies. And then the FDA asked for more details about the statistical analysis protocol, how it should look like to be able to do this accelerated reading, and also the clinical protocol, which changes should be made to it. And then we had sent in an updated analysis protocol to the FDA, an updated clinical protocol, and other things to really agree on those points. And here, Again, at this meeting, the FDA said that it is reasonable that we can go for an actual approval based on our FAST-3 study and a previous review. We agreed on these protocol updates on the clinical protocol, the statistical analysis and how the blindness should look. And here it is important to note that when you do a previous review of the ongoing FAST-3 study, you can't break the blindness for patients or doctors, or for the majority of the company. If the blindness is to be broken, it can create some difficulties, which can affect the final result in the complete study. So it is extremely important how you do the blindness, who becomes blind and who does not become blind, whether you should have a clear protocol and procedure for that. So that's what we agreed on, and they approved it too. to ensure that the integrity of the entire study remains the same, even though one has measured the results in a previous chain. Also in agreement with how the security database in the accelerated approval looks like, and then it is the earlier studies we have done with the Interinfantil administration and the earlier placebo-controlled studies with the Sukutan administration, samt den säkerhetsstatus som finns från den pågående fastigheten vid tillfället man gör den här tillgängliga avläsningen. Samma sak, vad är då den bekräftande effekt- evidensen, från vilka studier ska det komma? Det viktigaste här kanske take-home message är att vi behöver inte göra några ytterligare We do not need to fill in the database or the effect database, but what we already have, plus what comes with the earlier reading, it is enough for FDA to be able to give a approval. FDA also confirmed during the meeting our special request status for Diamyd in our launch indication, i.e. STAGE. 3 type 1 diabetes, which is important given that we got this particular study quite a long time ago, many, many years ago, but it was confirmed. And the FDA does not need any further interactions when it comes to this process for a possible accelerated approval, but the next discussion takes place at a so-called pre-BLA meeting, and at a pre-BLA meeting, then you have the results. That is, you go to a pre-BLA meeting DSNB-committee has given a thumbs up that they think the results look good enough to be able to take the discussion with FDI on a marknadsgodkännande, och då har man ett pre-BLA-möte var några, mycket få, från bolaget är med, och sen är det ju från CRO-bolag och från FDI med på det. Men egentligen det är då man diskuterar resultaten, och ingenting behöver egentligen diskuteras innan det, om inte vi vill som bolag stämma av någonting ännu. Sen, om vi går in på den kommersiella potential here, we have done further market analysis in the USA. We have followed up on what we did a year ago, we have done further interviews with both doctors and payers in the USA to understand even more about how exactly the launch would look, where to launch it so that the launch is done in the best possible way. And the most important thing here is that you can see that there is a sales potential over 2 billion dollars for our advanced indication in the USA. And then it's about patients who have stadium 3 type 1 diabetes, are 12 years old or older, have the right type of HLA and who have enough of their own insulin production left to preserve. So it's not just some newly diagnosed, it's about maybe up to 80% of the patients keep quite a lot, including two years after the diagnosis, so you can now count them on, shall we say, the annual available market. And when it comes to pricing, we expect at least a gross price of around $160,000. It can go up to $20,000 or more, it depends in the end on the final three results. Vi förväntar oss ganska små rabatter på priset. Det är ganska vanligt i USA att man ger rabatter. Det finns olika mellanhänder som tar sin del. Bolaget har ett listpris. Sen finns det mellanhänder som tar en del av den kakan. Det slutliga priset är oftast något annat än listpriset. Det är inte alltid helt tydligt vad det slutliga priset är. because there are different intermediaries that take different amounts. Here it is expected that it is a maximum of 20% discount. Some may not take any discount at all, mainly because it is a special drug and there are no other drugs today approved for this disease. Therefore, no large discounts are expected either. It is expected quite large so-called market access because most in the US have have type 1 diabetes, that is, in their insurance for type 1 diabetes, and that most insurance companies will also approve or pay for such a drug. So over 80% of those who have the disease will also be able to get access to this treatment. And then in a base case scenario, we expect at least a 30% market penetration, which is quite standard. It can absolutely go up to almost 50-60% as well. So it depends a little on, with more than 30% market penetration, that is to say that you achieve up to 30% of the target population. And with this pricing, then you achieve an expected sales of around 2 billion dollars. Then there is the possibility of broadening the treatment quite significant. So outside of the USA, if you take an example from the type 2 diabetes market, you can expect that the rest of the world is about 40% of the total. So then the USA would stand for 60% and the rest of the world for 40%. Some sometimes say that the USA can stand for 80% of everything, but in type 2 it is quite normal that it is about 40% of the rest of the world. And then we can also expand to the two earlier stages of type 1 diabetes, stage 1 and stage 2. It is stage 2 where Sanofi has been approved for T-sealed, that is, just before the clinical diagnosis, you have autoantibodies and a delayed blood sugar control, but no symptoms yet. So far, it is also clear in our market analyses that this market is not really mature yet, because there are no screening programs available, so it is extremely difficult to find these stage 2 individuals today, but hopefully the day we get approved there, then this screening will be available. And that's also why it's good to start with stage 3, because there is a market there. And then we have LADA, which is the 10% up to 2 diabetes, which has an autoimmune form of diabetes, and that is a market that exists. These are then diagnosed patients, They are often not insulin-dependent during diagnosis, so they become a bit similar to those treated in stage 2 type 1. And then the concept is to treat them before they become insulin-dependent. There are no disease-modifying treatments approved today, so potentially we should be the first to go out there too. And it is a market that is at least as big as stage 3 type 1, if not bigger. So there is also expected peak sales in the USA of over $2 billion. Then if we look a little at management and board and advisory board, so here, above all, there has been a change, an increase in advisory board, what we have announced, we have added both Emily Sims and Alice Long, who are two young people with a lot of, shall we say, strong and upcoming researchers in the USA who are very clinical. Emily Sims is a child doctor, very well known in the USA and in the world, and Alice Long is a transnational immunologist. Transnational here means how to translate something that comes from pre-clinical study to human. Both are very well known and this is especially important for us to both think about increasing our presence in the USA, and we also increase our presence in something that is very close to the clinic in the USA, because now it's about creating awareness about what is going on, so that on that day, if we get approved, then doctors, nurses, patients should also know what is going on, that the launches should be able to go as well as possible. So this is a lead in that process. And then the last slide. Kort här då om finanserna, market gap just nu runt 1,8 miljarder. Cash hade vi sista november då som ju då vårt Q1an med ungefär 150 miljoner kronor och nu den närliggande nästa finansiella hållpunkten är våra täckningsoptioner i mars som skulle kunna generera efterkostnader runt 100 miljoner kronor innan kostnader blir en affär. 111-112 million kronor, if it were to be 100% calculated. So, that's the next coming financial milestone. Of course, it is important to be able to extend our runway here, and make sure that we can prepare all of them, so that we can now enter the previous reading in March 2026. And with that, I thought we could leave it to questions.

Yes, thank you very much Ulf for a very nice introduction and just as we mentioned earlier, it is excellent to ask any questions that have arisen already now or that appear during the Q&A here in the chat, we will raise them. I think we can jump in directly here and it is when can patients under 12 years be estimated to have access to BMI?