Diamyd Medical AB (publ) (DMYDB) Earnings Call Transcript

Yes, I had the honor a couple of weeks ago publishing this article in the Lancet Diabetes and Endocrinology that everybody that's on a co-author list here, if I added up the years that they've been like myself in type 1 diabetes research, I'm sure it would be a combined 150 or more years of research experience. And what we felt like after looking, where we are in type 1 diabetes now, it's time to make for our feel that is trying either delay type 1 diabetes onset or preserve insulin production in those recently onset with the disease. We need to take a pause and readdress and have an honest look at how we perform trials. And if you can go to the next slide, please. And right now, it's the -- I made this quote a while ago: "It's never good to have type 1 diabetes. But if you had to have it, now is the time." In terms of the disease history, the -- thanks in large part to efforts of the JDRF and industry disease management tools in terms of allowing for continuous glucose monitoring, insulin pumps, new insulin analogs. And as also was mentioned, insurance programs, there's been a lot of improvements. But still, it's a disease that we need a way to prevent or cure. And right now, again, we're in a pivotal time, as I mentioned, because actually, in the last couple of years, there has been the identification of multiple drugs that seem to have a benefit in recent onset individuals. But as -- again, as Josh mentioned, not everybody responds, and we need to do a better job in this population of understanding, who might benefit or who's going through a clinical trial where there's little chance of them seeing benefit from going into a trial. And the other thing is, of this list that we gave in the article of agents that show benefit, most of them are not specific for type 1 diabetes or I should say the autoimmune is not specific for the autoimmune attack in type 1 diabetes. They'll target varying levels of the immune system that are not, again, not specific for type 1 diabetes. So you're having benefit, but you're essentially attacking the whole immune response. And that has challenges. And then some of these agents -- and again, I'm not -- this is great research. This is advance that JDRF has been working for many, many years to try and see. But some of these agents, they're -- they have issues of what I call pragmatism. So for example, you would have to treat every 2 weeks for 26 weeks or every 2 weeks for up to a year or possibly you just continually and that -- in order to see some sort of benefit. And that's a challenge. Also some of these drugs, once you provide them, even though they may be beneficial, yes, they have challenges in terms of retreating with the same drug, because it can cause problems both down the road if you need further treatments. Then -- and some of them, it may be even beneficial if you combine them. But if one drug has an impact on the immune response, if you start combining them, you reach -- have additional questions of safety and benefit, which go down to the bottom issue, which is that there are patients, when they're fazed, it's a shock often, when they're recently diagnosed with the disease. And then now how you're coming in and saying that you could use this drug. And it forms a challenge, when people will go on the Internet and explore, and then they see some of the side effects that are common to these drugs. And then they have to weigh the benefit of that versus just continuing on with managing diabetes. So these are some of the challenges that we put in the article. But if I could go to the next slide, we do believe that there are some solutions, and these were all quoted in the article. And one is, again, as a notion that both Josh and Ulf mentioned, precision-based medicine approaches, meaning identifying patients that are most likely to benefit from a given treatment. And I think Diamyd is, if not first in class, one of the first drug trials in therapeutics to try and usually utilize this, meaning by selecting people with the right genetic risk, you're increasing the chances dramatically that you're going to -- that those individuals will have benefit. The second thing is more -- this is on self antigen-based methods. Again, as Josh noted, using an autoantigen would have so many more benefits in terms of disease treatment than these agents that work forward. And so we're encouraged that JDRF is taking a look at these self-antigens and is continuing to support these areas in many ways. The other notion that we thought is that if we --there were ways that we could retreat people with the agents and that, that would have potential benefit, meaning if you give a drug to a patient and they show progress but then you begin to see signs of weakening benefit, would that therapy that you gave them what it be is good to retreat them? And again, one of the advantages of using a drug like Diamyd and an antigen is we believe that retreatment will be safe. And actually, there was just a press release I saw this morning before coming into work where this was shown again with Diamyd. And again, there's good -- as Ulf mentioned, there's now decades of safety data associated with Diamyd that -- where its track record has been stellar. The other thing is, again, I mentioned a problem in the last slide is a word called pragmatic, meaning is this really feasible and acceptable and easy. And I think that the protocol for Diamyd, where it's an in-and-out procedure and it's not so many times, that has benefit versus some of these other therapies that it require continual treatment or long hospitalization stays. And again, just closing in back again on that patient choice in autonomy. I think that the protocol with Diamyd does offer a considered a number of advantages to the patient and/or the family that should be considered, and I think it will be seen as very favorable. So again, I'm very excited of where we are in the type 1 diabetes community. And I think that we're on the cusp of seeing a self-antigen that being Diamyd seeing use in terms of population to help those with type 1 diabetes. Thank you.

And if we move on and I address all 3 of you, saying patient access is an important question and central in making sure that novel treatments reach as many patients as possible. Can you describe the thinking here around Diamyd?

Yes. And I can start off and then probably Josh and Mark will probably have wise words to say about patient advocacy and generally as well. But -- so patient advocacy, as I understand, it's all about -- or access, it's about really making sure that not only that you get an effective treatment to market. I mean that's really a requirement, it needs to work. But obviously, then it works, then it's a bit alluding to what Mark was saying as well is, well, patient choice as well and how -- what are the -- what's the side effect profile and what's the cost, which is guiding a lot of the access. And we, as a company, I know many other companies in this field really have the vision that -- long-term vision should be to cure the disease. But that means also that you don't only want to cure the individuals, who have to -- happen to have a lot of money or have the right insurance companies, something like that, that you need to also think about that, which I think is important that we want to be able to offer these kinds of treatment to as many patients as possible around the globe. It shouldn't only be an exclusive treatment that only a few lucky can access. So that's why it also comes back to a bit of this pragmatist model treatment. That's one part. It should be pragmatic. Obviously, the cost-benefit is very important, when it comes to this. And obviously, the payers, the ones who pay for this, either insurance companies, if it's out of pocket or in a more European health care societies, the taxpayers, that cost-benefit equation needs to work out. Because in the end, if it doesn't work out, it will not get accessed by everyone in the world, and we want to make sure that. I mean, otherwise, our vision won't be able to come true in the end, if we can't have that on our road map, that -- how will this actually be accessed by everyone, who is indeed in the world. So that's really important for us as a company.

Josh, did you have any comment on this?

Not much I can add to what Ulf already said, but I can tell you that JDRF approach is really kind of a holistic look, like I said, across the pipeline. So the accessibility question is just as important as the research going on in the laboratory and the trials going on in the clinic, and we focused on this in a number of ways. I mean, we have a regulatory team that works to make sure that, that access is available. We have to talk to payers to make sure that these are going to be affordable and are going to end up in the hands of patients. But really, there's 2 things that I think are most important here. The first being screening. We have to screen more people to find out, who is at risk of type 1 diabetes in order to identify the patients that can benefit from these therapies that are being developed. We don't have, at least in the U.S., a nationwide automatic screening for type 1 diabetes yet. We need to do more to reach out and find the people that can benefit. And then the second part, it always comes back to education. And that's really the key point here. Once we have a drug approved, we need to educate the community about, who it's appropriate for, who can benefit, and we need to -- and educate the clinicians to start using it in their clinics.

Did you want to add anything, Mark?

My co-presenters here are doing a great job of answering the questions, but all I'll add is one thing just so it might not be confusing as much to the audience, we're really talking about 2 different populations here. Let's say, in the new onset case or individuals with recent onset disease, there's, again, studies dating back to decades that the longer that a person can produce their own insulin or the longer that they can -- better they can manage their disease and keep their blood glucose levels low, the more likely they are to avoid disease-associated complications. And we don't speak as much about disease-associated complications these days. But again, diabetes in general remains a leading cause of blindness in adults. It's a major factor for kidney transplant. Almost 60% of people with type 1 diabetes die of cardiovascular disease, so you have a marked increase risk for that. So any therapy that can avoid these complications or help reduce the risks for them in a recent onset case is a promising benefit. The second group that we've been mentioning and talking about screening is this those at risk for the disease. And thanks, in large parts the efforts of the JDRF and some others, there are now test that can identify risk for diabetes development over short periods of time. And this has benefit in avoiding or helping reduce the chances of what we call diabetic ketoacidosis, or DKA, which is a life-threatening situation. And again, it's key to note that almost 90% of individuals, who develop type 1 diabetes have no family history of the disease. That's another misnomer is that people think type 1 diabetes only occurs in certain families. But let me again repeat, 90% of the time it occurs in people without a family history. So screening affords the ability to know what symptoms to look for and watch out. And then the final thing I'll say on the therapy is, again, if you talk to families, when we talk about advocacy, if you could delay the diabetes onset in a 5-year-old to, say, 10 years or if you could delay the diabetes in a teenage or to adult. Those patients and their family members would say that would mean all the world to them to push the diagnosis as far away as possible and maybe even prevent the disease from occurring at all. That's a big deal.

And to wrap this up then with one last point of discussion or question, we see a significant activity in the type 1 diabetes field regarding advances in science, new approvals as well as acquisitions and licensing deals. Why is this happening now? And what does this mean for the field as a whole and Diamyd specifically?

Yes. I can start off, but I will definitely hand over to Josh and Mark so it's not only me speaking here. Obviously, as a company, for us, it's very important that there is -- there is like, let's say, commercial activity in the field and also that the regulatory bodies like FDA and EMA on the U.S. and who approve new therapies in the U.S. and the European market that things are happening. Because in general, that's one of the main challenges or has been one of the main challenges in this field for many years. Because if there is an unknown, you don't really know what the regulators can approve based on what kind of criteria. It obviously adds a major challenge. You don't really -- you want to know that this trial can actually lead to an approval, if you hit the end point. The second thing is that the commercial thing. You want to know that if you get approval that there is -- that it can actually go all the way to market and sell this. Because if you don't have the -- see the commercial opportunity, especially from the larger pharmaceutical companies, it's not enough to get approval. You need to be able to sell them, and then patients can get access to it. And what has been happening, especially this year, is that you've had both regulatory approvals and acquisitions and licensing deals, which is very important for any of -- the whole field and especially for companies like us because it reduces the risk for us, the development risk, when the commercial opportunity has been identified by larger players and the regulators start approving novel therapies in the field. So that's what I -- it's a great thing what's happening in the field right now.

Who wants to follow Ulf here?

Go ahead, Josh.

All right. I'll just add one small thing. I'll say that there's a tendency to think about when you see an approval of another drug or things going on in this space that it's somehow restricting the market ability. And I think it's the exact opposite in the case of type 1 diabetes. We're in an exciting time, and these advances that other companies are making, that other investigators are making, are really paving the way for what Diamyd and others are trying to do. We know things from -- as both I and Mark talked about earlier that there's people that respond to certain therapies and not others. And we're going to have to identify which therapy is best for which patients. And that creates a very large market, right? And that creates a large opportunity. And if we can catalyze on the success of others and kind of lean into this exciting time, I think it's good for everybody.

Right. And all I'll add is, again, the one interesting thing here is that type 1 diabetes knows no geographic boundaries. And again, it's -- so whatever you're hearing about in the U.S. or Europe and even globally, where different forms of study are taking a look at the disease and finding the global nature of it. So the impact of these efforts on screening and in disease therapy, they're not going to be restricted to one country, but they have the potential to provide benefit again at a global level.

Okay. So I think that will be it for today then. So thank you, Ulf. Thank you, Josh, and Mark for participating, and we are all looking forward to all this progress within the type 1 diabetes field. And thank you for watching.