Diamyd Medical AB (publ)

Hello everyone, my name is Linus Svensson from GOV and we would like to welcome you all to this quarter's update together with Diamyd Medical. The book house communiqué was released this morning, I hope everyone had a chance to look through it and read a little bit. And just like the latest report we did here with GOV, we have with us the company's CEO Ulf Hanelius who will tell us a little about the company's status and What has happened in the last quarter, there have been several positive news related to the development of both the company as a large and phase three. And just about a week later, it turned out to be a success with Teo 3, which gave about 50 million in the cash register. And of course it's good to send questions via chat at any time during the presentation, then we take them up after Ulf has passed the quarter. So I think that with that said, we leave the floor to you Ulf, so have a good one.

Tusen tack och välkomna alla, 32 stycken hittills, det är fantastiskt. Ska vi säga, jag börjar med att dela min powerpoint här. Och det syns bra.

Det syns bra.

Fantastiskt. Då sätter vi igång. Den här är en ganska kort koncilspresentation med framförallt fokus på vad som har hänt under kvartalet och sen var det ju en årsbok som vi går Årets bokslutet också sa att det är bra att backa bandet lite och se allt positivt som har hänt under året. Men det ska finnas mycket till för frågor här också. Men om vi börjar idag här så är det ju verkligen ett starkt momentum för tillfället för oss och för hela typ 1 diabetesfältet. I fältet är det ju det riktigt stora som har hänt, som ni vet, de senaste åren i EU när Sanofi köpte upp Prevention Bio som hade fått approved for its drug to reduce the time for type 1 diabetes, to diagnose type 1 diabetes. And then Sanofi bought it for almost three billion dollars. And that was the first time ever that FDA has approved anything other than insulin and technical aids for type 1 diabetes. So it opened, it reduced the regulatory risk significantly. It also showed commercial potential when Sanofi bought Prevention Buy for three billion. And it has really been a big boost for the entire field. And now, as we are going to go through, we are the only company in the world that may have had three developments with a disease-modifying drug within type 1. So that puts us in an incredibly strong position. And in February this year, we got our first flashtrack designation for stage 3 H3 type 1 diabetes. So that's the indication that even our fast 3 is in progress, that is to say clinically diagnosed type 1 diabetics who still have their own insulin production left and the purpose is to be able to maintain as much of it as possible. It slows down the disease and it makes it much easier for the patient to handle the disease and will reduce their complications as well from the disease. And we got that in February and that's actually where the ice release with FDI in the beginning. We have had our challenges with FDI, we and the type 1 diabetes field. But this was a real turning point in the regulatory climate and for us actually. And we continued on that, and in June we got fast track even for the two previous stages of the type 1 diabetes, stage 1 and stage 2, which are then prevention indications, that you go in before clinical to prevent or shorten the time for clinical diagnosis. And it's good to know about fast track. It's not like FDA just knocks on the door and sends an email and says that we want to give fast track. This is something you apply for. So we have sent in applications. We have also sent in our raw data to FDA, so if you look at it, it's quite a bit of diligence that FDA does before you get anything like this. And in July this year, we had We had a physical meeting with FDI, which is the second time in the company's history that we have met physically. FDI was the first time, as I heard, in 2020-2009. And it's not often that FDI gives such physical meetings. Of course, during Covid, everything was virtual. They started to come back to physical meetings. But often you have a written answer or a virtual meeting. We had the opportunity to meet FDI in Washington DC And during that meeting, we asked the FDA if we could use C-peptide, which is the marker for the egg and insulin production, as a marker to support a potentially accelerated approval. And the FDA said yes, which is the first time they've ever said that to anyone. And now the plan, which is also being discussed with the FDA, to make a prior review of the ongoing study, whether to use C-peptide as the primary effect parameter, and with stronger results, would then be able to support an application for accelerated approval in the USA. And in July this year, we finally got an interim analysis, a so-called futility analysis, which was positive, which meant that it was a thumbs up from the Security Committee in the study to continue the study enligt plan, utan några förändringar. Alternativet skulle vara att det är tummen ner. Det ser inte bra ut. Det här visar på att det finns goda chanser för att det här kommer att gå hela vägen i hand, så det minskar risken ytterligare i det kliniska programmet. Nu alldeles senast har vi också informerat rent officiellt att vi kommer att gå vidare med det accelererat godkännande förfarandet, var vi planerar en tidigare avläsning i den pågående fas 3, var vi förväntar oss resultaten runt March 2026. So it's about about 170 patients who have been followed up for 15 months. And then you look at the CPAP time as the primary effect parameter. And with strong results in the previous slide, to be able to support an accelerated market approval in the USA. If we then look at the progress and the ongoing activities during the quarter, Precisely as Linus already mentioned in the beginning, we have strengthened our finances, where we got 95% of the use of the TH3s, which is actually, you can always have 100%, but in practice, you never get 100%. I think we had a development option many years ago when we did our phase 2, and then, if I'm not mistaken, it was about 92% of the use, which was extremely positive as well. And now it's 95%, so this is a lot of trust from our shareholders, which we are extremely grateful for. And this was part of a previous remission, as these cover options are shared. And then we also received a milestone payment from Breakthrough T1D, which is part of our collaboration with them. Breakthrough T1D is the largest patient organization in the USA, in type 1 diabetes, and we have a collaboration with them. And we also received money in compensation from this previous sale of Companion Medical, which at least a few years ago, which generates a whole lot, around 150 million kronor, we got from it. But there is a number of such different compensation as part of it, and here it still happens to be money to actually get. So we also got some money from that. Operationally, as we update the report, we have about 160 patients recruited in phase 3. So, patient recruitment has taken off, and it's going well for all 60 clinics in the USA and Europe. This also means that we are quite close to having the number of patients required for this previous release. Then we will follow up in 15 months so that we have about 170 patients who have data that can be used in 15 months. So it is also important that we are close to that milestone. And in Umeå, our facility for the manufacture of biological substances, we have manufactured large-scale technical batches, and we can show that we can produce large-scale protein, and it looks good, it seems to be the best, and also the yield, as they say, that is, the exchange seems to be quite good. And there are a lot of activities going on in Umeå, and it is full up there. All focus is now on preparing for the previous reading of the study. Given the strong results of the previous reading, to be able to have as much as possible in place, to then be able to move on to send in a market search in the USA. There is a lot on the manufacturing side, both our own facility and then the whole formulation of the drug and everything it means to be able to to show quality and stability, if possible, before a commercialization. So there is a lot going on, but it goes forward. Regulatory, as it has been mentioned, with fast track and green light, in fact, the approved procedure, we are really sitting in a good-sits today as a home. There is no one else who has achieved what we have done in type 1 diabetes. And as I said, we have an earlier avläsning då så vi har flyttat fram våra positioner ganska rejält nu mot marknad bara de senaste månaderna. Om vi då summerar då de mynd för att verken hamra hem med det här budskapet så sitter vi absolut i framkant inom fältet och vi kan se att vi har kommit andra längst inom hela fältet då vid sidan om vi kan säga T-sidan som är godkänt av Exxon Sanofi nu för tiden. Vi är den enda pågående fastighetsstudien med sjukdomsmodifierande behandling på gång inom typ 1. Det finns inget annat bolag i världen som har en fastighet på gång inom typ 1. Vi är också det enda läkemedlet som någonsin har fått fast track för alla stadier av sjukdomen, vilket också sätter oss i en extremt stark ställning. Vi är det enda läkemedlet som har pågående studier inom alla de här stadierna. De flesta brukar börja i When you go to your own clinic, it's usually either healthy volunteers or, for type 1, it's quite often adults who have had a disease for a number of years. Because it takes a long, long time before you are approved to be able to go down in age and be able to go down into healthy individuals who have risked being sick. So most of them never get that far, and it takes a long time to achieve such status that we have with a very good safety profile, and we have been in four-year-old children with stage 1 type 1 diabetes also with subcutaneous injections, so there are not many who have progressed this far in development. And there is, as I said, no one else who has undergone studies in all stages of the disease. And last but not least, again, we are in the first week or so that we have been given the opportunity to go for an accelerated approval based on C-peptide, i.e. the egg with the insulin production, as a surrogate marker. And this also shows that FDA has now prioritized type 1 diabetes and they prioritize diabetes. And it seems to be a real change from the FDA's point of view on how they look at both us and type 1 diabetes. And with that, I think that was my last slide. then we can jump to questions.

Thank you very much. Thank you very much Ulf for that nice introduction. We have actually received a lot of questions during the presentation, so I think we'll jump right into them. And if anyone has more questions, of course, just ask them and we'll take them up in order. We can start a little with something that you may not have mentioned so much during the presentation, but there has been a question about DiaPrecise and its meaning in relation to FastTracker, where we can expect the first results from that study.

Precisely. There is precisely a smaller study that takes place in these stage 1 and stage 2, that is to say the two earlier stages of diabetes, when you do not yet have any symptoms or complications, but you have autoantibodies and in stage 2 you also have a weak blood sugar control, but as I said, no symptoms. And here is where Precisely does something that we also do together with Diarrhea Union, which is an organization in southern Sweden and Denmark that has screening for children with risk of type 1 diabetes. So that's where we recruit the individuals for this study. And how this relates, we got, it's based on the data we have from the previous prevention studies called Diaprevit, which had the support to get Faststrike from FDA for stage 1 and stage 2. And this DiapreCise is the first study that evaluates intra-lymphatic injections in i barn med stage 1 och stage 2, och vi går även ner i ålder. Det vill säga att vi visar att man kan ge intrafatiska injektioner även i 112-åringar. FastTrack öppnar upp dialogen med FDA om hur vi på bästa och snabbaste möjliga sätt ska kunna ta fram behandlingen, att få det fortsatt även för stage 1 och stage 2 då i USA. Och det är precis en viktig studie, även om den är liten, den är en öppen studie, det finns ingen placebo här, så det är viktigt att kunna visa på att vi samlar in data och säkerhet, men inte den fattigaste det här. Och vår förhoppning är ju såklart att det inte ska krävas några large, very long studies on stage 1 and stage 2 to get approval. We probably don't know, but we will need long-term understanding, of course, but there are many discussions in the field about whether we could also get approval for stage 1 and stage 2, because it will be a bit difficult for both shareholders and companies and others, for example, GRF or Briggs & T&D, to study very long studies for 10 years. But with FastTrack, we have the opportunity to start such discussions with more. Then we believe, and we have also heard a little, that if we get approved for stage 3, then it is very possible that the drug can be used off-label in the USA for stage 1 and stage 2. So before you get approved, it is possible that the drug then also starts to be used for this purpose. But the most important thing is, as I said, FastTrack. How can we in the best possible way go to the market without having to do extremely expensive studies?

I think we've got two more questions about Stage 1 and Stage 2 that you might have answered a little bit in your answer to the previous question. The first one was, why did you decide to apply for FastTracker? Have you received indications from the FDA that it might be a good idea to apply for it? And then also a little thank you for the good performance so far. And then the other question again, if a new study will be needed to be able to treat stage 1 and stage 2, or if you can use the ongoing phase 3.

The purpose of why we applied is actually up to us. All companies should have the opportunity. You have nothing to lose by testing. But we see that stage 1 and stage 2 in the future will be the biggest for us. You can go in with a simple and safe treatment to preventive treatment of type 1 diabetes. There are at least 10 times more people who have risked getting sick in type 1 than who get sick every year, so it's a huge market. So, it is our own decision to move on, and we have analyzed our previous prevention studies with this genetic subgroup. What we see is that even with only two subcutaneous reactions, there is a fairly strong trend for us to reduce the time quite significantly to a better diagnosis. So, it was our own decision to say that now the FDA gave us stage 3 We have clinical data from stage 1 and stage 2 that shows that it is absolutely going in the right direction. And then we send it in too, because it will be the biggest thing for us in the future. And we know that it is stage 2 that applies to those with T-Sield. And they take a lot of time and invest a lot of money in screening and awareness. And just because of that, it is good for us to show that we are is not the only tool with fast track for stage 2 in the world. Now I'm talking so much that I forgot your other question here.

Yes, the other question was actually just, you answered it a little earlier, but it will take a new study to be able to handle stage 1 and stage 2.

Det vet vi ju inte ännu liksom. Best case skulle vara att det räcker med det vi har idag, och att vi får godkänd till stage 3, och med hela den safetyn också, med die precise, och med fast track, att det på något sätt skulle räcka. Men det vet vi inte. Man kan hoppas på det, men man skulle inte räcka med att det är så. Jag tog mera på att det kan bli off-label användning om vi får godkänd till stage 3, die precise, data will be important to put in place what extra we need to get officially approved for stage 1 and stage 2. The hope is that it can be an accelerated approval, so that it does not have to follow up for many years, but that it becomes a shorter follow-up, and then you have some kind of post-market commitments, that is, what you should also follow up for a longer time, the patients who are treated. But this is something that we have to discuss with the authorities. The most important thing is that we do not Att vi inte går med på att tvingas göra en studie vi inte vill göra, utan bättre då att spendera tid på diskussionerna så att vi kan göra någonting som är några år kortare än vad vi annars skulle kanske behöva göra. Men vi får se helt enkelt.

Det är ju viktigast att vi har fans track där också nu.

Regarding pro-insulin, there have been two questions here. Is it possible to try to make a vaccine of pro-insulin? Is vaccine research clinical or antigen-connected to another autoimmunity? And the second question was if Diamnid has a patent for the intralymphatic administration of antigen-diabetes.

If we start with intra-lymphatic, then we have patents that have been approved and suspended according to the administration method for GAD and also for other diabetes at the same time. And you usually get approved one antigen taken often. So we have approved in several countries for intra-lymphatic generation of GAD. We have in some countries also approved, if I remember correctly, intra-lymphatic injection of insulin and pro-insulin or pre-pro-insulin, different variants of insulin. And then it is mentioned in the same patent applications as the other types of diabetes have always been mentioned. But it is actually GAD and insulin that are the most important, I would say, in terms of therapy. And then when it comes to the patent on the treatment itself with insulin or proinsulin as an antigen, we have also sent in applications, and we have approved some of them to treat the other large HLA group, that is, those who have DR4 and DQ8 with a proinsulin or similar antigen. And there, and there it is now for the time being too, you have mentioned some other presentation, an important publication came out this summer where they had analyzed a a study with oral insulin that was in these stage 1 and stage 2 type 1s. The original study did not show any effect, but if you analyze it from the genetic perspective, it shows that it works. And that supports exactly our patent applications. That is, if you have HLA-DR4-DK, you should treat with insulin as always. If you have DR3-DQ2, you should treat with GARD. And those who have combinations of both HLA, you should use a combination most of the time. So we have patent applications for that as well, and in some countries we have approved it as well. If there are others, there is one Belgian company that actually went into competition that had something similar, that is, they had insulin as an antigen, but then they had a different type of molecular construction around it, a slightly different type of mechanism, as they evaluate in type 1. But they didn't see any effect, so they actually went into competition here this spring. And they were only two. So on the competitive side, we had also made our positions really good. Then there are companies that have other types of nanoparticles that they fill with antigen. Or also on the mRNA side, that is, a little more on the COVID vaccine side, who was used. type 1 anti-diabetes, which is the sequence of these mRNA molecules. But they are in a much earlier phase. And then there is always the question of how much they affect our patents as well. But there is no such thing as solvents, they have been introduced year after year when it comes to development.

If we go back to phase 3, we have a question about the fast-forward program. Can you explain why you are doing a fast-track with C-peptide after just 15 months and on participants as 170? You have previously said, which research also shows, that you see big differences already after six months. Would it be possible to shorten the time for reading and therefore be able to get to the market faster?

So why 15 months? I've often been asked why we don't do it for 12 months, for example. But 15 is because today we collect, we do these meal-stimulating tests, so when you collect CPT data, we start the study at six months, the study at 15 months and at 24 months, so we can't do it at 12 months. Now we didn't get to that question right here, but at 15 months we have it and at six months we have it. Att kunna göra det vid sex månader är ingenting som myndigheterna skulle godkänna idag, utan de vill ju ha, för att de ska kunna godkänna, även för accelererade godkännande, måste man ju ha starka resultat och övertygande resultat, för de vill inte godkänna någonting, bara de sedan hamnar i en potentiell situation, att när man ska ha den fulla studien, tvåårsuppföljningen, som ska vara den bekräftande delen, att det då blir ett frågetecken kring att, vänta, nu ser det annorlunda ut, och då blir de, to take it back from the market, and it's easier said than done when something is approved. So they must feel extremely comfortable with the results that come with an early diagnosis. And there are a lot of regulations on how you should do an early diagnosis with blindness, and you must not affect the studies, like operational, introduce some kind of bias and things like that. So there are clear rules there, and that someone has opened up for an early diagnosis at 15 months and fewer patients is a huge paradigm. Men resultat kan man ju redan se. Det är väl mer ur ett utvecklingsperspektiv om man skulle varit i en tidigare stadie så vet vi ju nu att redan vid sex månader vet du. Alltså ser du ganska tydligt, verkar det här funka eller funkar det inte. Så det betyder ju för framtiden att man kanske kan göra betydligt kortare, mer effektivare fast tvåårsstudier för att inte behöva göra ett års uppföljning till exempel utan redan efter sex månader vet du hur det ser ut.

When we got to the SNAP sports program, we got a question about when the application takes place. Is it already now or when you have the finished results in March 2026?

The application consists of sending in all the data and a report on all the non-clinical packages, i.e. preclinic, the entire clinical package, including both effect and safety data. och hela tillverkningspaketet, det vill säga både mellan aktiva substanser, det formulerade läkemedlet och visa på all kvalitet och all stabilitet och allt möjligt som ingår där. Vissa förberedelser kan man ju göra, alltså man kan, det är ju också en resursfråga, det är ju mycket resurskrävande att skriva ihop en marknadsansökan, fullständigt sådant, och det kostar pengar, men vissa förberedelser kan man göra inför en avläsning så att man är to keep the ball rolling. Then there is a lot on the manufacturing side. Above all, it is what the bottles usually are. When you go for a product, it is almost always approval for all companies. But there is a lot that must be prepared and then it should always be written together and summarized. And we try to optimize the timetable in the best possible way so that we Vi har så pass mycket på plats som möjligt, den tidigare utläsningen görs, och ifall det ska vara övertygande resultat, att det inte ska ta för lång tid att efter det kunna skicka in den kompletta marknadsansökan. Sen har vi ju möjlighet att ansöka om så kallad priority review som en del av FastTrack-programmet. Det vill säga att man kan börja skicka in delar av en ansökan. This is done over a one-year period. You don't have to wait until everything is in place, but you can send in non-clinical manuals when you feel that it's ready, and you can at least do a check to make sure that it looks okay. They won't be able to read through everything, but it makes it a little more agile from the company's point of view. So we also have that possibility. But it's hard to say today. We want to be able to do it as quickly as possible, but you should also remind yourself that it's extremely much work. Nu har vi flyttat fram våra positioner så vi är ett och ett halvt år närmare med allting än vad vi tidigare hade. Time to market är extremt viktigt. Varje månad man inte är på marknaden så är det försäljning som man går miste om.

Om allting går bra så säljer man mycket per månad.

Regarding the facility in Umeå, you mentioned it a bit, both in the previous report and today. There are also questions about how the development is going there and the future for it. So if we start with when the calculation is that the first commercial batch can be produced in the factory?

Is the first commercial, it's also good to have one commercial batch. It's complex, so I'm not an expert on the manufacturing side. But what is happening now is technical batches. And what technical batches mean is that it is on a large scale as it is done. And then it is important that you, under technical batches, it is a lot of search. You make sure that everything goes on or, as always in a lab, you discover challenges that you have to figure out and explain so that you see that everything works. Then it goes on in parallel with that is all this with GMP readiness, that all quality systems, everything should be in place, all routines should be in place. So that when the company feels that we are ready to become GMP certified, then you can start the certification process. And the day you have a certification and the manufacturing is in place, then you can start manufacturing according to GMP. And that would mean that such batches can be used for clinical studies. In order to be able to use it for commercial use, you now have to do a complete validation, process characterization, validation, can do a number of large-scale batches after each other to show that they are basically identical, meet all the requirements. So it's a job in itself that's done. It's not enough that you just produce large-scale and then the GMP is finished and such, but it's a lot. Because the day you produce for commercial use, the authorities should also know that this is a drug that is 100% safe, it can be produced all the time in such a way that we dare give it to patients on a large scale. So it's not the same thing as manufacturing for a clinic. But all such preparations are ongoing, and it will continue all the way to the previous review as well, and I think a little past the review as well, because there is so much work, but that's for all companies in this. It's easy to forget what manufacturing means in medical development, it's often is not visible on the surface. But now, when we have taken over the production of this substance, we are in a completely different position to be able to see what it means, instead of just clinging to an external manufacturer and wondering why it takes so long for them. But this is something that we also want to tell more about, because it is extremely interesting, and when we build up this competence, i Umeå. Så det är ju vad man brukar säga high barriers to enter. Vi bygger ju upp ett helt kvalitetssystem, rutiner, kompetens som ger oss möjlighet att börja bli ett tillverkande bolag även för andra grejer. Och det är ju det som vi också, vi har tagit in en business development director i Umeå, vars syftet är både för att hjälpa upp för hela bolagets affärsutveckling, för det är så mycket på gång där, men and also start sondering the terrain for potential external production, potential customers for the future. What needs are there? How are we going to position ourselves correctly within the limits of feasibility? We have a certain technical platform. And also understand even more about the internal competence, if you can start, for example, on the consultant side first. innan man tar an externa tillverkningsuppgifter. Största fokuset är ju vår, vi måste få allt med GAD och infokommersialisering på plats, men vi sonderar nu den terrängen för externa möjligheter, vilket också är jätteviktigt.

Ja, och just kopplat till kontrakttillverkningen där också så vi ser att ni är lite för tidig fas för att redan nu ha tänkt på, men om det är om ni har redan nu budgeterat för några potentiella intäkter eller start looking at when the previous customer would start producing for external customers, if that shows there is a great interest in it?

Nothing we have budgeted for, but it is a survey that is ongoing now, and as I said, the first thing I think we could do is on the consulting side. We as a company have gone through so much, we know both what it means with production, what it means to set up a GMP facility, not many have done that. FastTrack, Designation, and everything. Many small companies have the same questions as we have, but we have actually gone through everything you can think of as a biotech company, so we have a lot of competence to consult on. And if it's the right type of drug, the right type of indication, we don't have the world's largest facility in Hungary, because it's not the one that does it on thousands of liters. So, depending on the type of indication and drugs, there may be a possibility to manufacture in the future for external. Or we can help the company to find the right one, more on the consulting side. But it is nothing we have yet budgeted, because it is a little too early. There is so much on the bond side that we also have to think about how we use our time in the best way.

Yes, and as you mentioned a little bit during your presentation, you have also started to develop large-scale test batches. And there we have got a question if you have been able to show that it works, so that you show continuity in the presentation with good exchange, and then also what will be the next step. And there we have talked a little about getting the facility GMP ready at the end of the year.

Exactly, and as I said during the presentation, we have done technical batches. We can see that we can produce the protein. And it seems to look pretty good. There are a lot of tests and assets that you can do to test the protein compared to earlier production. And it seems to be a pretty good change from what we have seen so far, which is better than what we have seen before, which is extremely positive. Because it is also important for commercial production that you understand how many batches per year we need to manufacture in order to to meet the commercial needs of the active substance. Then you will be formulated into a drug as well, and create that. But so far we look good, and there are always challenges when you are in a lab. It was a lot of years ago when I was in a lab. Nothing works 100% ever in the beginning, but that's the whole point here. When you do it for a drug, you should have so many routines and all these procedures in place so that there is no chance of errors anymore. And that's what's being taken forward, that you have to do it in exactly the same way every time. When it comes to GMP readiness, as I said, it's when we as an organization know that we are ready, that we are certified, then we assume that we are GMP ready. Then it's up to the authorities to make a survey when they come and visit the facilities. You send in the application and they ask questions and then you get You always get comments on different things. We haven't done it on the GMP, but on the GCP side, we've actually done it in the company as well, for FAST 3, that you do the same survey, the authority. So we know what that means too. Lots of questions, you go through something, you always get comments. The most important thing is that you don't get any of the most serious comments. And it can happen that you get a backslash, and you need to say, you have to correct this, and then just a survey again to get the GMP. Vårt mål har varit att känna att vi är färdiga i år med att vara GMP ready. Efter det ska man börja skicka in och göra själva certifieringen. Det är nästa steg. Det är lite av en lite mer abstrakt, lite lösare milestone, för att GMP readiness innebär lite mer att känner vi själva att vi skulle kunna vara redo för en granskning. But I see that the certification is the really big thing then.

Yes, there has been a question here regarding your collaboration with Breakthrough T1D. It was previously JDRF, if there is someone who would be unknown with that name. They changed the name this year, if I remember correctly. And there has been a question regarding milestone payments. In addition, what do you have that is in the pipeline from there and what can you expect from that point of view?

Exactly, we have got them Most of the milestones we have received from them, because it has been quite promising, if we just think about the financial milestones, the greatest value from them is actually their network and everything that it means to help on the regulatory side and to spread information about the study and then to introduce commercialization and to send in an application, then it is extremely important with these collaborations with patient organizations. But in terms of milestones, we have received the most, but there are still when it comes to both a fully recruited study and others that come when everything is ready for the whole package. But there are a lot of recruiting-based milestones that we have had before, when you write down numbers and things like that. But it has been a success, so we have got a majority, if I'm not mistaken, of the milestones so far, but there are still more.

I think both you and many other pharmaceutical companies, there is a lot of focus on just the USA and Europe, and here you have come to the question of whether, for example, partnership or licensing could be faster to go into Asia as well and quickly be able to start a way into that market and maybe also strengthen the cash a little bit before the end of phase 3 and market financing in the USA in the coming year.

Exactly, and Asia is absolutely a possibility, and in Asia, as we say, Japan is perhaps the Traditionally, the third largest market in Europe and the USA has a well-functioning system, which also means high pricing. China has also come into force in the past decade. Of course, it is an extremely large country. Korea is also a country that has a lot of focus. For us, we haven't done studies in Asia, and now when we focus on a genetic subgroup, we know in China, for example, in the largest Chinese population, which is probably Chinese, if I'm not mistaken, our HLA type according to publications is around 20% of type 1. In Europe and the USA, it's around 40%, so it's still a fairly large group. Then there is another HLA background in Japan, for example, much more common with our HLA group, but they probably have another type of HLA group that responds in the same way. So then you will need to do a study to find it. Right now, we have the absolute largest focus in the USA. That's where you will earn up to 80% of everything you earn in the whole world. And that's what the global partners are most interested in. They often have their own local, if they don't cover some parts of Asia themselves, they have their own local partners that they use. Men det finns absolut en möjlighet att licensiera ut, till exempel, till separat i vissa asiatiska länder. Det enda man ska vara försiktig med då är att man inte minskar intresset från global partner, för de vill oftast sitta på full kontroll i hela världen på alla indikationer för läkemedlet, så de inte känner att det är en annan partner som använder samma läkemedel som kan börja smitta- och säkerhetsprofilen som påverkar deras egen försäljning i andra territorier. Så det är mera en sådan diskussion man ska ha också. Men störst fokus har vi på Global Partners och USA är ju det som alla tittar på.

Yes, du skrev i presentationen här som du hade uppe och pratade om att det var 161 stycken patienter i fas 3. When is that figure from? And then there has also been a follow-up question on that, regardless of which, we need 170 patients to be recruited in October. And then we also need the results after the 15-month follow-up to be able to be completed before March 2026.

For the first 161, it's only a few days ago, so it's a pretty solid figure. When it comes to about 170 patients, it is important that we know that 170 patients will have 15-month data. When we talk about data, we prefer to have both a baseline data, i.e. a data point at the start of the study and at least a data point in 15 months. Then there are always patients who miss certain data points due to the fact that it was either a holiday at the lab or that they forgot to do something to come to a visit and things like that. So 170 patients, we want to have 15 months of data on 170 patients and then we will take in a little more than 170 before we say now we have 170. So we should be sure that it is at least 170. And then we have calculated that March is probably one month when it will happen. It's a 15-month update. It is estimated that in a month, the database will be locked and the clinic will be monitored to get all the data. But around March is a pretty good estimate now, and with 161 patients in now, and it's going pretty well for patient recruitment, we will achieve the number we want in the year series. And 15 months after that, plus a few months on the database, you will also end up in about March. But it can definitely come earlier as well. Vi ska inte lova för mycket, men 5 mars är en ganska bra uppskattning.

Om man skulle se då att vi tar det till kommersialisering, så har vi en fråga här. I en tidigare presentation har du nämnt att ett kommersialiseringsbolag skulle ta cirka 20 % av försäljningen, och BMI skulle behålla cirka 80. Skulle dessa nivåer vara snarare, kan vi avtala med Big Pharma, eller hur ser det ut på ett ungefär?

Det är en bra fråga. Traditionally, and this is in the end a matter of negotiation, but if you license the rights, then it is both upfront, some of the fees are paid through approval and through the first sale or a sale over a certain amount, and then royalties on the global sale that is used. be step by step, but you can get up to 20% or 20% in royalties. So if we just look at these royalties, it usually ends up that the biotech company that licenses RUT sits at around 20% of the sales and the big pharma company at 80%. So this is about 80-20%. Because in the end, the pharmaceutical company takes over all costs, the entire logistics, everything around sales, what that means, and then they keep most of it in sales. But the positive thing about a biotech company is that you get upfront payments and maintenance payments that reduce the financial risk in the future, that it takes away the financial risk. If you go with a commercialization company, then it usually is like that, it's actually like a a consulting company that is on the total infrastructure to be able to commercialize a drug, everything that is needed, both to the sales organization, to what is called pharmacovigilance, to be able to always follow up on the safety and on the medical side, that is, the people who are supposed to sit and answer questions from doctors about how you should not use drugs, etc. And then they pay for the sales. And in that case, the biotech company usually keeps roughly 80% of the total sales and 20% goes to pay for the commercialization company. And then, of course, before commercialization, it is preparations that need to be taken into account to go to the market, which is also expensive. which the commercialization company can help with counter-payment and potentially de-risk to a certain financial level by taking the sales back paid when you are on the market. But if you can commercialize yourself, then you assume that you commercialize as well as a big pharma company. If everything else is the same, then it is of course better to do it yourself in the long term. And then you have the opportunity to build up and become a large commercial company. than to leave the rights to a very large company. But it is absolutely more common with a license agreement or purchase than a commercialization company, but it has become more common with commercialization companies now, so it is a possibility for biotech companies to do it themselves. And we have an indication that we are not talking about type 2 diabetes or obesity here, but type 1 genetic subgroup, so it does not require huge supply organizations to cover the entire USA, for example. So it is absolutely a possibility for us too.

Ja, och just med tanke på vägen till marknadsvideos och FastTrack och Accelerator approval i USA, CPT-tid, bordsändelse av surrogatmått, har ni några indikationer på att även IMI skulle öppna upp för en snabbare väg till marknaden i Europa?

Där finns det ju en möjlighet, vi har inte haft någon diskussionerna, men vi kommer ju absolut den dagen vi gör en tidigare avläsning och då ger vi såklart övertygande starka resultat att ta även den diskussionen med den europeiska myndigheten för att se, skulle det här kunna i Europa kallas det då en conditional marketing approval. Så det vet vi inte ännu. Vi har ju haft stort stort fokus på FDA, för det är det som inte rent krasst kommersiellt betyder allra mest, och det som Partners också vill veta om. Men vi kommer att även kolla upp det då med Europa den dagen vi får våra resultat för att se. Annars vet vi ju att Europa har sagt att en veldesignad studie räcker. to be able to go to the market. And of course, good results as well.

Yes, a few questions here related to the company as a whole. And then we start with an owner in Excel Pharma. What is the strategic intention with that? And do you know them or is it just a pure owner?

The simplest answer right now is that it is a pure owner. Just now, as a company, we put almost all of our resources in GAD, Diabyn, and everything it means for the market. So we don't even have the opportunity to do much else. And we don't have the opportunity either, and the rationale is not really that we should invest so much in external projects. Everything should be driven by our internal projects right now, what that means. But it's an ownership, and we also monitor the stem cell market. It happens a lot in the cell therapy sector, within type 1 and other indications. So it's extremely interesting, and they also have a potential drug that has shown good results, even though there aren't as many as we have, and it hasn't come that far. But there are also opportunities for potential synergies in the future. It will be independent of a combination treatment for type 1 diabetes in the future. One combination is that you follow up, you start with, there is T-SIL today, for example, which is given to delay the diagnosis for a number of years. But there are also discussions where you can start with such a type of treatment, follow up with an antigen-specific immunotherapy to create tolerance long-term and potentially continue to boost. with something that is a much nicer treatment. But the same thing, there is a possibility for Nexia and also their treatment to be able to, in the best case, I would say for them, to be able to do something similar like T-SILD, but with a nicer BFX profit, for example. But we have an owner, of course, if it goes well for Nexia, it goes well for us too, then we have money to earn there. And of course, there are opportunities to collaborate, but both companies have so much going on internally now, so we both have to focus on what creates the most value for both companies.

And just related to ownership and collaboration and the like, we have talked earlier about Break from T1 doing a collaboration with them. Skulle det finnas möjlighet att få in ytter kapital från dem genom liknande dator som ni har nu eller om de har visat intresse för att bli ägare i bolaget genom exempelvis att gå in i riktade emissioner? Ni har ju tidigare nämnt att de investerar i lite andra bolag som de är involverade i. Just Breakthrough T1D, alltså det finns

Breakthrough T1D is a patient organization that often has rich individuals, who often have some kind of connection to type 1 diabetes, who donate money to them. And they donate the money and put it into type 1 diabetes research and this type of collaboration. So, Breakthrough T1D does not go in and invest equity shares in companies. There is something called T1D Fund, which is a kind of evergreen fund, who can invest in FTB's companies, often very early stages, not public companies. So we have become quite large, we have come a long way. But there is a possibility, but they never invest alone. They do it in a syndicate and with others. To get more money from Breakthrough T1D, there is always a possibility, and we know them very well, of course. And I think it's going pretty well for Breakthrough T1D right now, when it comes to the donor side as well. Möjligheterna finns alltid och vi har ju såklart diskussioner på gång med dem också men stora värden från dem absolut är ju deras medverk och att ha dem på vår sida.

Yes, jag tänker vi har en avslutande fråga här om det inte är någon nyttjare som har en fråga sen så passa på och skriv i chatten om man har någonting. Men annars så har ni ju nämnts i många år att ni sitter i nära samtal med olika partners. Har ni fått något bud eller någonting om samarbete eller köp av hela bolaget från någon potentiell partner?

Of course, I can't say much about that. Discussions are held under confidentiality. Of course, there have been talks for a long time, and now with the news that we have received and the progress we have made, it is clear that those discussions have reached a new level as well. But it's hard for me to say more, and I know it's so chatty to say all the time that there are of course discussions, but we are in discussions. Det har gått till en ny nivå, givet de framsteg vi har gjort. Vårt största fokus är den tidigare avläsningen nu, klart, om man kan ingå i ett samarbete innan det är billigt att göra det. Det viktigaste är att vi inte... Vi sitter i en extremt bra sits nu, men en klart time partner på vår sida inför förberedelse för marknad skulle vara extremt bra, men vi ska inte sälja oss för billigt heller.

Yes, vi fick faktiskt in en till fråga här också, ett par till, and that the money will be enough by 2026. What do you think about the financing after that? Is it a partner or an investment? What is the financial plan?

The financial plan for us, like all other biotech companies, is that we always look for opportunities to get money. You have to do that in this industry. Unfortunately, there is never too much money in biotech, but I don't think there are any companies in the world that have too much money. Det viktigaste för oss är att vi ska kunna använda den situationen vi nu har, vi själva har satt oss i. Alltså vi har så pass mycket positiva framsteg vi har gjort att vi har satt oss i en position var vi har kommit närmare marknaden med ett till två år plötsligt. Och då ska vi göra det bästa möjligt av det vi kan med den the financial structure that exists today and the other possibilities that may exist. So the only thing I can say is that there is always different discussions on the table on how we should be able to make money in the best possible way, both in the interests of the shareholders and to be able to drive the operational work in the best possible way. The most important thing here is that if there are strong results, around March 2026, that you really should be able to go to the market as soon as possible after that, because that is when the real big value will come.

Yes, and just discussions about partners and so on, we have had a couple of questions here that are related to whether it is a goal you have to get a partner together sometime in 2025, or would you say that it is desirable that you have a partner to inspire on the development work and perhaps also prepared with new studies? further.

I would say that it is desirable to have a partner, a right partner, in good time, just before the market application and commercialization, because they have so much knowledge about that. And of course, financially, they do not lack money. That's the small thing for them. The big thing for a partner is that they take a decision to strategically invest in a game media and area, and then they tie a lot of resources in their organization around a long-term strategy. So that's often why they really have to think about it. In terms of money, they usually rely on quite a lot of resources. And for us, it is absolutely of great interest to be able to have them as early as possible. But at the same time, we also make our own preparations and should be able to have the opportunity to go to the market, wait for results, that is, with stronger results by March 2026, we will be in an even stronger position. It will be an even more expensive contract, so it is always a balancing act. Vi som bolag ska aldrig hamna i en situation var vi blir beroende av en extern part. Vi ska kunna framförallt med den situation vi har satt oss i, kunna gå hela vägen.

Men det är klart det innebär, det kräver resurser och mycket för att gå hela vägen.

Men möjligheten ska finnas. Jag tror faktiskt inte att det kommit in någon mer fråga så jag tänker att vi kan avrunda där och vi tackar dig Ulf för en jättefin presentation och bra svar på alla frågor och så tackar vi även alla ni åhörare som har varit med och ställt bra frågor så får vi önska både en fin onsdag här och sen en fortsatt trevlig vecka.

Tusen tack.

Tack så mycket. Hejdå.