Conference Transcript - SANA 2026-06-03

Roger Song, Analyst — Jefferies

okay great morning everyone welcome to uh jeffrey 2026 global healthcare conference my name is roger song senior analyst cover simica biotech it is my pleasure to have our next company center biotechnology we have a ceo steve hart here welcome steve thank you roger and thanks

Steve Harr, CEO

everybody for joining uh both online and in person awesome all right uh maybe steve uh take a moment

Roger Song, Analyst — Jefferies

What's the state of art for Thana? You have an exciting year ahead of you. I think, you know, everyone looking forward to that. Maybe just give us some high level.

Steve Harr, CEO

I'll start, since you asked me, to look at the year ahead of us by making sure everybody knows we'll make forward-looking statements. So, you know, take a look at our disclosures, read about our risk factors. So we built the company around two platforms. One is an ability, we hope, to hide allogeneic cells from immune recognition when transplanted. into patients. We call that our hypoimmune platform. We've done a lot of work to show that this works really across, you know, multiple species, whether that's non-human primates, mice, humanized mice, and we've even done some human work, which we'll get into. And we're applying that, and it's, I think, its most advanced and maybe most valuable form for a potential functional cure for patients with type 1 diabetes. And I'll come back to that in one minute. The other platform was set up around in vivo delivery, so to be able to deliver DNA or RNA or other genetic material directly and specifically to cells in the body. And the most advanced program around that is an in vivo CAR-T platform with two different assets moving forward towards the clinic. That's a place where we've, you know, it's gained a lot of excitement over the course of the last several quarters. I think we have been in the forefront of moving in this space for a while, and we took our time and tried to make bells and whistles that we think will be really important in making a best-in-class therapy. All that being said, the most recent information is highly compelling from people in the field. That excites us. It gives us a high bar to stretch for. We think we can do it or do better, and we'll be able to tell you about that in not too distant future. So to take a step back in type 1 diabetes, and first I'll lay the frameworks. I think it's often a disease that's underestimated. There are about 10 million people in the world with type 1 diabetes, and it's growing at a mid-single-digit rate per year. People who are diagnosed, I always think about this in the context of my daughter. She's 22 years old. If a 22-year-old woman who's diagnosed with type 1 diabetes has a shorter expected lifespan than a 22-year-old woman diagnosed with either HIV or breast cancer. It's pretty astounding when you think about that. And that time is very, very difficult, right? You have a daily burden around insulin and food intake that you have to grapple with, exercise if you're feeling a little bit sick. And, you know, that time is also spent with a number of comorbidities such as blindness, amputation, heart attack, stroke, and hypoglycemia and coma risk and things like that. So we have to And the field has made a lot of progress, and we stand on those shoulders. About 25 years ago, someone named James Shapiro in Canada started taking pancreases from someone who was recently deceased and isolating the islets. And to take a step back, the etiology of type 1 diabetes is super simple. The immune system kills the pancreatic beta cell, and the pancreatic beta cell is the only cell in our body that makes insulin. And so without insulin, sugars just float around in our blood. They're too high in the blood, but our cells starve to death because they can't get any sugar. And so 100 years ago, there was an invention of insulin. And what James Shapiro started taking was pancreatic islets, which think of as a beta cell plus a support structure, and transplanting them into patients with type 1 diabetes. And what he's found is that patients can live for a long, long, long time off insulin, and they do quite well. But there are two problems with that. Number one, cadavers aren't really a great and predictable source for islets. And number two is, like any transplant, these patients have to be on lifelong immunosuppression. And there just aren't that many people for whom lifelong immunosuppression is better than lifelong insulin. So the second step is that several in the field have now taken stem cells and made them into, you can differentiate them into islets. They've shown they can transplant these and patients do quite well. It's much more predictable, probably much more scalable, but you still have to be on lifelong immunosuppression. And so what we did about 18 months ago was gene modify in a proof of concept experiment, on cadaveric islets and transplanted that into a single person in Sweden. And it's great to say that this person is now living and making insulin in his body for the first time since 1987. These islets are living with no immunosuppression and they are functioning and they are persisting. And so you put that all together, gene-modified stem cell-derived islets, and you have a chance for a functional cure, a one-time treatment for type 1 diabetes. And we've been working super hard. It's been a long slog to bring this into humans. Our goal is to have an IND this year, begin our trial this year, and, you know, I'm quite optimistic that in less than 12 months from now we'll know whether or not this is really Similarly, on the in vivo cartoon, I'll be much briefer there, you know, we'll start the study this year, begin to get jaded this year. When and how we share it, we'll see. But again, within 12 months we'll know if that platform is working. So it is something where we have, I think, a pretty meaningful opportunity to really see how these drugs work in people. And once we see how these drugs work in people, to rapidly move them through clinical development and towards the market.

Roger Song, Analyst — Jefferies

Excellent. Yeah, it's a meaningful and transformative trauma for both platforms. Maybe we focus right now for the type 1 and the other hypoimmune platform. So for the type 1, I think you're guiding a couple months, a couple quarters, you say, as early as 2026, you're going to file the I&D. I think recently you changed even update that to start phase 1 in 2026. So what's the remaining steps? I think we talk about this, you know, in terms of a mass cell bank. So just walk us through that a little bit of technical and what's remaining to be done.

Steve Harr, CEO

Yeah. So one of the real challenges that we have accomplished is making a gene-modified master cell bank. A master cell bank is the single cell from which the product starts and grows forever, right? And when you make cells and stem cells are no different, divide, they tend to make one or two mistakes. It's really not a big deal. Mostly it's, you know, it's in DNA that isn't really copied and expressed. But when you're making trillions and trillions of cells, what you can see is that these, and particularly when you're doing that in a growth media that you're trying to make cells grow fast in, right, which is what you're trying to do when you're making the full cells, you can actually select for DNA repair enzymes, mutations, something like P53, use that as an example. And that really can be problematic. And so that took us years, but that's in the rearview mirror. We've made a gene-modified, genomically-stable GMP master cell bank, and we have hundreds of vials of it. We then take each one of those vials, and we make hundreds of vials of a working cell bank, and then you thaw one working cell bank vial, and that's how you start a manufacturing process run. So that's basically in the rearview mirror. So what's left to do is really two things. One is to complete a non-clinical package, and that's generally just around things like GLP toxicology study, you know, efficacy, biodistribution, where does the drug go anywhere else in the body. That's nearing completion, you know, I don't think that's going to end up likely being a problem. It's one of those things where if it does become a problem, it becomes a real problem, but it, you know, we're optimistic it won't be. And then we're also in the middle of transferring a manufacturing process that we utilize in our own labs that we developed in our own company with our own people into something where it's in a GMP facility utilizing, you know, an outsourced group of people. And so that process is ongoing. You know, it's, again, one of those things that it will happen. It's not really an if. There is always a component of when to this. You know, things don't always go perfectly during technology transfer across sites. I'm optimistic. think we have plenty of buffer uh in the guidance we've given you we've eaten into some of that buffer because now it's not like it's super simple uh but we still have buffer and you know we we are

Roger Song, Analyst — Jefferies

you know on track to to do what we said we would do okay uh that's an interesting comment that i i know you have some buffering because that's why you say at the end is 2026 well i always say as

Steve Harr, CEO

early as because i don't want to get into some debate with a lawyer or regulator is it really going to happen in 2026 or could it happen on january 1st then how do you change your disclosures and this was a lot easier to say as early as because i can't i know it's not going to be in

Roger Song, Analyst — Jefferies

2025 it's true right it's already in the it's so yeah got it uh and then uh you did mention some uh you know you do have a buffer but you you think you you eat some or add some a buffer so how much you can say about that or it's just a it's just a comment it's just a comment um it's just a

Steve Harr, CEO

comment to say this is hard uh and while we are optimistic we'll get it done there aren't guarantees yet that it will get done, right, and, but that we still have, you know, some, some work to do, and we still have some, you know, ability to kind of, our team still has some leeway if they need to take a bit more time, so those are things we're working through, and again, optimistic we'll get this done. Got it, and then the, I think it's super straightforward, we can get into, like, what it then takes to understand, does the drug work, right, is that worth jumping into? Yeah, yeah, go So, because once you start this study, there are really, I think there are just three simple questions you want to ask. As we move across to this new technology, do these cells engraft and evade immune detection and function, right? And you should know that within the last time, it was within about four weeks, we were very confident that these cells wouldn't be rejected and they would live. So I think similarly, if, you know, the cells engraft in the first patient, and they're not rejected by the immune system within three to four weeks, they're going to last a long time, right? And so there's just not a component of the immune system that pops up later than that. And so that, I think, would be part one. And so at that point, you know, this technology works. The second thing is we're not actually trying to evade the immune system. That's just part of our goal. What we're trying to do is make a curative therapy, right? And so are our cells potent enough that you get patients off insulin and they're able to, you know, live a normal life? And so, again, that should take a few months longer but not forever. And at that point, you know you have a really viable drug. So then the third question is how generalizable is the result, right? And that's one of those things where if we're six for six, you can be pretty confident it's very generalizable, right? If we're three for six, you might say, you know what, I want to see a little more, right? And so I don't know how long that would take. But those first two will happen pretty quickly. And the third one, I'm optimistic this will work in, you know, maybe not everybody, but most people. And the catchment of people we're going to enroll in the study is more or less all comers. I'm sure we'll get into that. But it's a very broad catchment of patients.

Roger Song, Analyst — Jefferies

Okay, got it. Yeah, you basically set the goal for this kind of phase one to achieve in terms of the rejection and then the curative and the potent enough, and then, obviously, the end, right? So you need to repeat that. Okay, got it. Yeah, let's take a step back. Okay, you want to enroll the patient, and then what's the baseline characteristic you are trying to do? You say all comers, any additional details we can say this is all comers?

Steve Harr, CEO

It's essentially all comers over 18. We're not going to put this into really old people to start, And we're not going to put this into really young people to start, right? It's an experimental therapy, and you want to have Equipos, which is likely benefit outweighing The second is we won't put in someone who had a heart attack yesterday. That could be confusing, right? And so, you know, some element of relative health. But beyond that, there are a few small exclusion criteria, but it's essentially going to be, you know, all comers. Assuming success, we would then look to expand that, you know, as part of a broader phase one program into 16-plus, and then 12-plus, because, you know, teenage years, and ultimately we want to get down to two and six-plus, but that may take a little bit of time. And we'll work on the over-65 population, as I think older people will also want this. And I don't really feel like you're 65 that old anymore, it's just around the corner. But the, anyway, that's a little bit around, you know, kind of what it is. It's most comers. It's not going to be, you know, brittle diabetics or people who have hemoglobin A1Cs over some Frankly, if you do that in a study like this, people will just let their hemoglobin A1Cs drift up so they can hit the entry criteria, and then they'll be part of the study. And that will be really not a good thing for a patient. We get a lot of calls to be in this study. There's a lot of demand. And so we don't need to have a whole bunch of patients going out of control just to try to hit entry criteria.

Roger Song, Analyst — Jefferies

Got it. And then just want to also emphasize, you do have other companies also doing the cell therapy for the type 1, but it seems the inclusion criteria are a lot more restrictive compared to Thana because they require some hypoglycemia event, even more severe event. So that's not the inclusion criteria or exclusion criteria you have.

Steve Harr, CEO

That's not the patient population we're targeting, first off. We're really going after all comers. You know, generally others in the field, and by the way, like, I don't consider this competition, and I don't say that, like, arrogantly. I say that humbly. There are 10 million people with this disease. If we somehow just nail this, and, like, we have the best possible outcome you can think of. It's one-time treatment. It works in every person. We only have to do it. We somehow scale this to 100,000 people per year. which would be spectacular for a cell therapy, we will take the global growth rate from 5% to 4%. Like, there's so much space for so many different approaches here, you know, we need help in conquering this for patients. But the population that we're going after is pretty much all comers. And others, you know, if you have immunosuppression on board, you may need to go after a sicker population. But at the same time, that sicker population has a real need for, you know, a novel therapy. So it's great that you have other people, you know, going in that world.

Roger Song, Analyst — Jefferies

Yeah, got it. And then a little bit more on the phase one design. And then how should we think about the do you what you do like a dose escalation or you start with, you know, potentially therapeutic dose. And then how many patients for those cohorts if you want to do multiple cohorts?

Steve Harr, CEO

I think you know what the right dose is more or less. Right? There's such extensive experience with cadaveric islet transplants. And so our cells may be moderately different in potency, but it's not going to be meaningful. So I do think we'll be at a therapeutic dose out of the gate. You know, we could be wrong by one dose or something, but we should be. And really, if you think about the side effects here, they're not generally linked to dose. because the first thing we worry about is severe hypoglycemia right after the transplant. And that can happen because cells die and they have insulin granules in them and they release them. And it's manageable very simply by just monitoring, seeing it, and if a patient develops it, giving them IV glucose, right, not challenging, and it will be gone within a matter of hours. Obviously, you don't want to miss it because that's something that could be catastrophic for a patient, but it's not difficult to manage. The other is, I think, more of a long-term challenge, which is when you're making stem cells into islets, there's a risk that some of those cells are off-target cells or the wrong cells. And those cells could continue to divide and create a tumor or growth or something else in the patient. That's going to take years, potentially, to see itself and develop. And so it's unlikely, really, that dose-dependent. And so I think we'll be in a dose range to start that, you know, we're optimistic, can be therapeutic. The very first patient that was dosed in this trial from Vertex was in the New York Times, just to give you a sense of it went very well for that person. And, you know, I'd like to hope that similarly we can offer a benefit for a patient right

Roger Song, Analyst — Jefferies

You know, you mentioned Vertex and certainly they are pioneering this. But a completely different setting because they are not hypoimmune and then also different stem cell like you. But I think in terms of data disclosure, so if once you start to enroll, maybe one is how quickly you expect the enrollment going to look like. And then two is how soon we can see the first data you think you're getting around like 12 months. We're going to see that?

Steve Harr, CEO

I think enrollment will start very quickly. I'll start there. there. It's a pretty unique program, I think, in terms of just external interest. When we'll disclose data, we'll let you know when we know something. It doesn't really help you to say we're still learning. We don't really know yet, but if we learn something, we're small enough, and this is important enough, that most early learnings will be material to the company, and I think at that point we'll have an obligation to let you know. time, and so we will do that as it comes about. Exactly predicting when that will happen or what will be material is difficult ahead of time, but I think we have a relatively low bar understanding the importance and external interest of this. Got it. But, you know, on the flip side of that, the long-term view and really the, you know, I presume investors are aligned with this, you know, we need to get these data presented at peer-reviewed scientific conferences, not just in, like, you know, kind of press releases and things like that. And so we will need to kind of balance those two as we go forward.

Roger Song, Analyst — Jefferies

Got it. You know, it's the first inhuman in this product. For sure, you will disclose data as you learn something material. And then how should we think about at what level of the maturity you can have the regulatory discussion and then thinking about even moving to the pivotal stage because, yeah.

Steve Harr, CEO

I don't think it will take much. I think it's about a dozen patients will allow us to move forward. I think the harder part is not the clinical data. The harder part is we have, I was talking about the scientific challenges here, and we've done pretty well in tackling them so far, but we're not done. One is overcoming autoimmune and allergenic rejection. I think we've shown we can do that, right? We've done that in the papers published in the Journal of Medicine. It's done very well. The second is we need to make this gene-modified, genomically stable master cell bank. That was super hard for us. We've done that. The third is make the drug at a purity, potency, and yield to allow us to run a phase one study. We've done that more or less, but we barely did it. I mean, it's like we're hanging on, right? And this isn't a robust, this is not a product we would ever take to market as current is. The third is to really make a, I'm sorry, the fourth, is to make a, you know, a process that is at a scale of purity, potency, and yield that's really commercially important and we can serve an unmet, really unmet need in a large population. We have work to do to do that, to be clear. So I think the, you know, the rate limiter to us moving into a pivotal study is much more likely to be having a locked commercial process than it is to kind of get a dozen patients through an early stage study which should enroll very quickly. And so that, you know, we may get that done in a timeline that there's no delay. way. We may get that done at time that allows us, and I'll take the lemonade side of this, to explore other patient populations like younger patients or older patients before we move into our pivotal study. But I don't think it will be that long to do it. I mean, we've made a lot of progress on it, but we still have a bit of work to do. And you have to assume that when you're done, you know, once you've locked, your process is still another nine months or so at least before you can start enrolling a patient. And so

Roger Song, Analyst — Jefferies

we have work to do to get there yeah yeah you have time and then the as you generating clinical data you continue to lock in the the commercial process and

Steve Harr, CEO

for the pivotal we're working on it already okay yeah good again this is different than some other fields that you're used to where the science is understood and it's a it's a capital investment that needs to take place right hey this is if you kind of think of it as you know you have the scale is number of batches per run times number of runs, we're still in the number of batches per run problem, which is a scientific problem, right? And so that's good from a capital efficiency perspective, but it's a bit more of a scientific journey we're still on and some unknowns that we

Roger Song, Analyst — Jefferies

have to grapple with. Yeah. The foundation of the science is amazing, right? So you just need to figure out a couple technical parts. And then in terms of the current expectation for the Do we expect it also will be the all-comer, or if that's the case, and then how many patients you actually need to enroll compared to type two? I think type one is a different kind of situation.

Steve Harr, CEO

Male Speaker 2- Yeah, I think it's going to be something on the order of, you know, a total of, you know, you have a precedent for another field. It'll be like 50 patients. You know, the precedent is about, you know, 37, total of 50 between phase one and phase I can't imagine we're logarithmically different than that. I could make an argument we should be smaller. or I can make an argument we should expect to be bigger, but it won't be meaningfully So, again, it's relatively straightforward, you know, and you know if this works or not. Patients without, if you don't, if we have a patient who's insulin free, that is implausible, is entirely implausible without the intervention. So there doesn't need to be some control arm around other mechanisms for control, right? Patients will die without this. they will die within a matter of months.

Roger Song, Analyst — Jefferies

Yeah, you know, you're curing those people, giving them the insulin or giving them the beta cell.

Steve Harr, CEO

In some regards, it's like the clinical simplicity and I think actually the patient passion like an orphan disease, right, that you see, or even an ultra-orphan disease, but with the market size and the unmet need that is of a very, very large market.

Roger Song, Analyst — Jefferies

I think you mentioned cell therapy. I cover a lot of the cell therapy, the long-term risk is always the case. But how do you think about the initial approval, right? You may get to six a year follow-up to get to the efficacy, and then you can get approval label, and then you'll keep following them as a post-marketing, or is that the case? Or how long you need to follow them before you can actually get the initial approval?

Steve Harr, CEO

Well, let me just say we've had no discussions with dialogue with regulators around what a pivotal study might look like. So anything that I'm saying is speculation. I don't think we really know. I know we don't know. If it were me making the decision, I would ask us to do a relatively minimal, just looking at what the, you're going to know if it works or not, right? And the safety is likely something that is rare and long-term, right? So there isn't a lot of value in a larger registration study. I'd have a relatively small registration study and very onerous post-marketing follow-up requirements and something like that where we have to really kind of keep an eye on it. And we have to anyway. It's a gene-modified therapy, right? So a 15-year requirement by law for following every patient. So we will have that in our bag of things we need to do regardless.

Roger Song, Analyst — Jefferies

I think we should touch on the in vivo quality platform I think a lot of interest a lot of the you know the but what our chatter out there so I think your in vivo quality is different right you know your combination of many no delivery gene editing and then the MR RNA you know the construct so tell us why you choose the platform you're using now and then and how you think it will be different from others.

Steve Harr, CEO

So, when we started this, we made two really fundamental, you know, assumptions, and they may prove to be right or they may not be, right? The first one was that the signal or the gene that you insert needs to integrate into the target cell's DNA. The reason being is that in the CAR-T space, there's another alternative, which is just put mRNA in, right? But you might make 100 million CAR T cells, something like that, and, you know, if you just look at B cells, that's all you want to get rid of is B cells. We have a few hundred billion of them, right? And so what you see in the CAR T field is this logarithmic expansion of cells, right? Expansion being growth. And so, you know, they divide, and with mRNA, when you divide, the signal can't go with both progeny, right? The mRNA just goes with one. And so when it's in the DNA, the progeny each have the same signal. So you can get that logarithmic expansion. So we believed you had to integrate. That's part one. And I think that's generally proving out so far in the clinic with the really robust data you've seen from other VLP, virus-like particle approaches. The second is that cell specificity matters. And so you only want to go to the cells that you're targeting, the T cells. I think many others would say that's not as important as you just need to get enough cells into the T-cells, right? And the others will take care of itself. And if it turns out both those things are true, we have a best-in-class platform. I think I can be fairly confident in saying that. If it turns out that neither is true, we've made things really complicated, and other things are going to win. And so that's the bet we've made, right? Right, and so we're different, and I think we've shown that in our ability really to target just the T cell. And that's something we think it will help us from a manufacturing, like T cells are rare in your body, so if you go to other cells, you're going to have to make a lot more drug just to get in, because you're going to go to the liver and other cells. It's going to help because when you go to off-target cells, particularly antigen-presenting cells, you run into immunology problems, right? And then the third is I don't think you really want to be in certain DNA and expressing proteins in off-target cells just because there's only bad things that can happen. It may not happen, but nothing good is going to happen. That's why we thought this, and it's taken us some time to really make this work. We almost went in the clinic a few years ago and really kind of ran into some challenges that thought would make it less likely our drug would work. I think we were right in taking our time, and I think now we have a very, very high probability this works not guaranteed and but it should work and we'll have data you know very soon to bear that out. Makes sense. Yeah it's interesting and so

Roger Song, Analyst — Jefferies

seems to you are more targeted and then I would argue it's even more powerful because you're integrated to the DNA versus you know and others say it's more RNA kind of a peripheral and then that how should we think about the initial clinical setting, and then what is the, you know, the data we're going to see in the community?

Steve Harr, CEO

Maybe one other thing that's different. What you see if you look in the literature as these virus-like particles are starting it going is you see a new, you've seen a new side effect emerge, which is a post-infusion toxicity that occurs within hours and lasts for a few days, and it's like a second case of CRS or cytokine release syndrome. So, patients have fever, cardiovascular collapse. It's been manageable. No one's died or anything from it yet, but it's been problematic. And so, that happens most likely partly because it's a virus. It's a virus-like particle. But a lot is because the portal of entry that all the other technologies use is CD3. And when you bind the CD3, you activate the T cells. So, you get this very rapid activation of T cells that is helpful in helping these T cells grow, but it is difficult in terms of a side effect profile. We use a different portal of entry into the targeted T cell. I think that gives us just, again, hopefully something that's a bit more of a safety advantage. I mean, these drugs look pretty good so far, so it's a high competitive bar for us. Our path here, so we have a CD19 that we're going to start in lymphoma, non-Hodgkin lymphoma, large B cell lymphoma. If that works, which, you know, I hope it does, we will continue down the path of expanding oncology and we'll rapidly hopefully move in the autoimmune disease setting as well. We also have a BCMA which will begin human testing, you know, like a year from now or something that is also right in there. We won't do, you know, we're not going to pile failure upon failure. So if the first one doesn't work, we probably don't go forward with the second. But if the first one works, we really unlock a lot of opportunity of, you know, potentially to move into CD19 and, you know, multiple tumors, lymphoma, leukemia, to move into multiple autoimmune diseases, and then to really push rapidly with our BCMA-targeted car as well.

Roger Song, Analyst — Jefferies

Excellent. Awesome. All right. Thank you, Steve, for being with us, and thank you, everyone, for listening and watching.

Steve Harr, CEO

Thank you, Roger, and thanks to everybody else. Have a great afternoon or morning.