Conference Transcript - AUTL 2026-06-02

Matt Phipps, Analyst — William Blair

Hi, everyone. My name is Matt Phipps, a biotechnology analyst here at William Blair.

Christian Aiton, CEO

Thank you for joining us at this year's Growth Stock Conference.

Matt Phipps, Analyst — William Blair

Before we get to our presentation, our next presentation, I have to, of course, say please visit williamblair.com for any and all disclosures. With that, I'm happy to have Christian Knighton, the CEO of Autolis Therapeutics. The company is still in the early stages of launch of, I think, the fifth CAR-T approved for lymphoma setting. So, you know, not a whole lot of these. and we'll hear about the progress of the Ketzel and some of the pipeline.

Christian Aiton, CEO

Very good. Thank you very much. Thanks for having us and really appreciate you all here and I hope you found some food on the way so that we're kind of making it through the presentation. Obviously, also the usual disclaimers. We're a publicly listed company. Please look at our SEC filings and the risk factors in there. Just as a brief summary, kind of where we are. As Matt pointed out, we have our lead product that has been approved in relapsed refractory acute leukemia. And it has actually been launched at the beginning of last year in the U.S. and at the beginning of this year in the U.K. The product is also approved in Europe. We had last year revenues of $74 million. And what we were able to do as we went through the first year of launch is actually reach market leadership in the indication. And we have seen a very significant level of physician interest, on the one hand, reported in real-world outcomes that I'll talk about a little later, but also in interest in moving the product already in exploring its utility in a frontline setting, which obviously is one of the key areas that ultimately we'd like to see the product be present in. Overall, this product has very unusual properties. it allows us to reset the b-cell compartment and with that obviously address the leukemia give us long-term outcomes but it also allows us to look at autoimmune diseases which we now know to be largely if not exclusively driven by b-cells and autoreactive antibodies and we have a very unique ability with this product to actually eradicate those b-cells not just in the periphery but also in the central nervous system and that gives us a very unique positioning and a very unique opportunity with a range of autoimmune diseases I'll talk about that in a little bit what you see on the right hand side there are few indications that we're active in one is obviously in the acute lymphoblastic leukemia setting where we are on the market but we also are running a pivotal study in pediatric patients with acute leukemia that study is up and running we expect data end of next year We're active in a pivotal study in lupus nephritis, that data readout expected for mid-2028, a single arm study designed for full approval and with the endorsement of the agency on the approach. And obviously the final one is in progressive multiple sclerosis called the Bobcat study, which is a study that will also expect to have data, early glimpse at the end of the year then full data during the course of next year so we're really leveraging a very unique profile that the product has established an acute leukemia and now transfer that into additional set of indications and opposite building on our ability to produce the product commercially and also supply it and distribute it commercially as well in the US and in the UK at this point in time so with that I think a few words to the types of approach that we're using here this is a autologous CAR T cell therapy which means that we're producing we're manufacturing individually for each patient so it's a different type of manufacturing that he would normally do it's not large vessels large volumes but it's truly individual for that we actually established our own commercial manufacturing site which is located north of London in the UK and it gives us an ability to serve all of the relapsed refractory ALL patients in the US and in Europe but also has the capacity to allow us to accommodate lupus nephritis as well out of the current facility and with that have an ability to really provide high quality product and also add attractive cost of goods what's very important is because it is an individualized therapy is that the manufacturing success rate has to be very high because if you cannot supply for the patient you failed the patient so and there is no way of going back from there so our manufacturing success rate is well above 90 percent which is a very high hurdle to actually get to for a an individualized product it's fast reliable and we do not have capacity limitations where we are now is we're the process of really optimizing our operating model that we're running and we will be able as we look into this the course of this year we will actually double the output of the facility with the same footprint we have and actually with somewhat less personnel than we had last year and that gives you a very clear indication that obviously this has a big impact on cost of goods and allows us to reduce cost and goods substantially as we go through the course of this year We are really focusing on driving the program to profitability in the ALL, on the ALL side of our business. We've done an adjustment. As you can imagine, when you establish a new facility and new operation, there's a lot of things you have to actually put in place. That is a different part of operation, a different part of setup that you need to actually operate efficiently as we go forward. And so we're going through a transition a few months ago and we'll see a significant impact in terms of our overall cost that we have in the business. When we look at our ability to then actually generate cash flow into the business and retain it in the business, we obviously have to drive our gross margin and we expect our gross profit margin to be in the mature stage for the indication of about 65 to 70 percent. at this point have actually transitioned from obviously the initial launch phase established that we have high reliability on the delivery and the product quality and also are now actually reaching have reached in the first quarter a positive gross profit margin and will continue to build on that as we go through the course of the year driving this business towards profitability in its own for its own standing when we look in terms of okatzel as a product what we're targeting is a structure on the surface of b cells called cd19 when we look at the most recent data that we have out for the first quarter in 2026 we had 26 million in revenue from the first quarter we also had very nice dynamic in terms of the market there was key data released at the astct meeting the tandem meeting beginning of february in salt lake that actually summarized the experience of the physicians in the first year of launch and i'll talk about that in a minute we also obviously are in the early stages of the launch in the uk which is running actually very nicely we're one of the very few products that actually was deemed cost efficient and was also going directly into routine commissioning so it is no need to actually generate additional data for the the product to justify its price in the system. Our guidance for this year is about 120 to 135 million in revenue, so significant step up year over year. Our current presence is in about 76 centers. We expect to be above 80 centers by the end of this year. This allows us to reach the majority of patients in the US and we expect to increase only in a limited way beyond the 80 centers, maybe reaching 90, 95 centers over time. As I mentioned, obviously we did shift to a positive gross margin, which is important and will continue to build as we go through the course of this year. Now, with regards to the regulatory approvals and presence, I did talk about the UK process. In the EU, we're also running through individual market access. The EU have to go country by country. The regulatory approval is central, but then the market access is on a country by country basis. We also have been very careful kind of monitoring the most favorite nation provisions and the topics around that. I think what is positive to see is that gene and cell therapy are currently excluded in the current proposals and pilot and also should give us, I think, ample, I think, differentiation as we go forward. It's an area we're watching carefully. We certainly will make our decisions based on a healthy economic case in each one of the countries before we launch. This is a summary of the initial experience for the Roca Consortium. The Roca Consortium currently captures about 50 centers of the top centers in the US treating patients with acute leukemia. We had about close to 60% of our patients captured in that database. This is completely independent from the company, this is not our data, this is data from the consortium. What they were basically having at the, after one year of launch was 96 patients that they actually had A4East, 91 got dosed, the five that did not get dosed actually were either progressing with disease and might have passed away, or they actually had an infection or lost the structure on the surface that made it recognizable for the therapy. So five patients were not treated, everyone else was treated, everybody actually had product that was InSpec, which tells you something about the reliability of the manufacturing and delivery for these patients. So when we look at the summary outcome, and we're starting on the left-hand side in the middle, which is really looking at the immunological toxicity, which is one of the key aspects that you actually need to manage with these therapies. What we're seeing is in Rockgeist, the data set, in the real-world setting. FELIX is the approval data set that we had to get us, which was published in the New England Journal, which was the basis for the approval by the FDA and the other regulatory agencies. What we're seeing is we had no high-grade cytokine release syndrome in the real-world setting, and we only had 3% of patients that experienced higher levels of neurological toxicity, which is transient. We had no treatment-related mortality. This is an unusual profile in a very difficult patient population patients that do have a lot of comorbidities we had relatively a lot of elderly patients a lot of patients with poor ECOG status which is typically a sign that indeed these patients are difficult to treat and may actually be prone to significant toxicity when we look at the right hand side overall we had more than 90 percent complete remission rate in the in the real world setting and we had very very deep remissions that we actually expect even to increase over time based on the continued activity of the product. So remarkable confirmation of the data that we actually had that led to the approval of the product. Now, when we look at the opportunity beyond acute lymphoblastic leukemia, the first on the adult side is really to add on the pediatric patients. Obviously, there are many kids that do get diagnosed with acute leukemia. The good thing in pediatric acute leukemia is that a lot of the patients can be cured with the standard of care and the frontline treatment. The frontline treatment is high-dose chemotherapy, very, very intense therapy for the kids. In fact, the intensity is beyond what we as adults can actually, frankly, get through. But it allows you to get many of these kids actually into a long-term outcome and cures. It has issues, and the issues are certainly related to the fact that the toxicity has negative impact on the development of the kids, and that actually is something that becomes more visible in the space that many of these children, while they're surviving, they had actually a significant impact on quality of life, also mental faculties and growth, where you see significant impact of the treatment. We are treating patients that actually have relapsed from that initial therapy and are now in a relapsed or refractory state. That is what the population we're including. And we have currently a pivotal study, a study designed for an approval in that patient population, expect data by the end of next year, and would then actually go, obviously, in an expedited review process for the product. In addition, we have two additional indications that we're going in, and that now actually take this remarkable property that the product has that allows us to actually eliminate all the B cells in these patients and actually transfer that into the autoimmune disease setting. What we learned in the autoimmune disease setting is that many of these diseases are truly driven by the B cells in the body and by autoreactive antibodies, and that the cells that actually produce these antibodies are actually cells that have the CD19 antigen on its surface. So they have a structure on its surface that allows us to actually recognize the entirety of the cells that have the memory for autoimmunity, including the producing cells for those autoreactive antibodies, cover the same structure. Our therapy now allows us to take all of these cells out. and because it's a cell therapy it is not only active in systemically in the broader part in our bodies the bone marrow and other tissue but it can also actually cross the blood-brain barrier and get into the central nervous system there are many autoimmune diseases like MS that actually have an inflammatory reaction not just in the periphery but also in the CNS in the brain itself. And that obviously is very hard to treat because most of the therapeutics that we would actually use are systemically given and can actually not cross the blood-brain barrier. So small molecules typically cannot cross, proteins cannot cross. And different to that, the cells actually can. They can actually move through tissue and through these barriers in the body. We know that because there are many patients with acute leukemia that also have leukemia in the brain. And we had a recent report from one of our physicians, and it's online available, you can listen to the call, where she described a patient that had massive disease burden in the bone marrow, the disease was exploding, and was very heavily infiltrating the brain to a point where the disease was basically constricting the spinal cord and rendered the patient paraplegic. she decided to treat that patient and actually got the patient not only free of leukemia but actually regaining control over her body so it tells you in a very I think clear way of the ability of the product to get where the disease is and remove the disease and do it in a way that actually is compatible for that patient and frankly with a safety profile that is appropriate and adequate So we're leveraging this type of a feature and we can do it both in more broad autoimmune disease settings and what talked about lupus nephritis but also in the for the MS setting where we're evaluating this approach as well. In addition we have additional earlier programs we're working on with University College in London to generate early clinical data and a number of them actually we have data that is already published. We expect the next data point to come from a product which is called Auto8 in light chain amyloidosis, which is a plasma cell disorder, data expected at the end of the year. So the opportunity here and why we're so interested in these disease areas outside of oncology and outside of leukemia is that leukemia is a rare form of a very difficult to treat oncological disease. And so we have about between the adult population, the pediatric population, about 2,000 patients in the U.S. that are suffering from that disease and we expect to be able to capture a significant portion of those U.S. patients with our product on the market now. But then as you go into autoimmune diseases, be it lupus nephritis or also progressive multiple sclerosis, you start to look at substantially bigger patient groups which still have very very high medical need so a very significant health economic challenge that needs to be addressed now when we look at pediatric all we're already moving very nicely in a pivotal study I did mention that the cat the phase one study actually is complete that was published or presented at the ash meeting at the end of last year showing more than 90% complete remission rate good safety profile and we're building on that with an extension of the study going adding additional 30 patients to get the data set sufficient for a filing with the FDA so that's ongoing data expected end of next year the initial data here is just summarized just on the top is kind of the adverse event profile with cytokine release and ICANS children have slightly elevated levels compared to the adults but very well tolerated and when we look into the swimmer's plot it's literally you've got every individual patient and you see that almost all of these patients have a green arrow which means they're in continued remission. So looks very strong and obviously with a very strong level of activity and that patient group. The differentiation we believe will be that we will then if we're successful with the study have an ability to really offer this treatment across all age groups and across all phenotypes of the disease and that differentiates us from some of the other products that have been developed for the space which address a part of the population but not the full population. When we then look into the mechanism of action, I already mentioned that a lot of the autoimmune disease settings are going back to B cells, the CD19 positive, they can be treated with our approach and that gives us an ability to really tackle a number of them in lupus we're going through with have data from the carlisle study there is a data presented in a few days at the ular meeting and then a longer update a longer follow-up will be presented at acr at the towards the end of the year and the lumina study obviously is up and running with sites active in the u.s and certain european countries and also additional countries outside those territories as well the idea is obviously with the first approval as a fast to market strategy with the current study with the lumina study and then actually look to broaden the opportunity with a follow-up follow-on study on the back of that this is just a brief summary of the key data just a very few things i want to point out first off on the safety table on the left hand upper side there is a limited amount of cytokine release syndrome these are low grade cytokine release syndrome typically fever and chills but no high grade events that were recorded and no neurological toxicity whatsoever which is important in these patients if you look at the bottom on the left hand side what we're looking at are sled eye scores. Sled eye scores actually look at various manifestations of auto immune disease. This could include arthritis, it could be puritis, it could be other parameters that we have that actually describe you know skin disorders and so on so forth. All of that gets scored and included as well as structural damage to organs which is recorded as well. In blue it's basically structural damage in the kidney and what you can see as you see from left to right is that all of these patients each one is an individual patient you see that these patients actually lose the very colorful components which are typically the manifestation of the disease that is actually experienceable as a patient and what's left is the darker blue which is sort of the actual organ function typically the kidney organ function which is obviously impact by some of the damage that the disease did that's not something you can feel, you can measure but you cannot feel that. What you can see is that across all of these patients there is a marked and quick improvement. Now remember these patients, many of them had 20-25 years of ongoing disease. It's the first time in 20-25 years that they actually feel free of any symptoms. That's a huge transition. What we're also seeing is that most of these patients actually have very deep responses and there was an ability to actually even take the steroid levels which are typically used steroids in these patients down to physiological levels that's a very hard thing to do it's very rarely seen we have seen it in in in five out of six patients and then we did see that the product actually persisted for a few months and once the the product actually sort of basically cleared the body. We also then had a recovery of the B cells and that will be part of the presentations that are coming up at the upcoming conferences where we can show that the cells are coming back but there's no autoimmunity coming back. So the system basically recalibrates, it doesn't induce or re-induce autoimmunity. With regards to multiple sclerosis, We're focusing on patients that have an ongoing gradual decrease of capability in these patients, which are so-called Pira or primary or secondary progressive patients. You have two different types of disease progression. One is the relapse remitting. That's when you kind of typically think about it. The patients have sort of a spurt of activity and a drop in sort of capability and then it's stable for a while and it kind of drops again. is different these patients despite treatment are just continuously coming down and just get worse on a continuous basis and the key driver is that there's an ongoing inflammatory component autoimmune component that's really driving it that's what we're focusing on we're now in the phase one part of this study with a positive outcome would move into a pivotal study focused on this patient group and this just actually illustrates kind of the way that these patients typically progress there is a clinical score called EDSS that as you get worse you kind of gradually the numbers get bigger for the score you get closer and closer to a wheelchair and and eventually obviously you might actually lose your ability organ function as well very tough disease setting we're actually in a set in the situation where the standard of care stops working and that's when we enroll the patients and what we want to see is whether we can actually indeed either slow down or stop the progression and that's what we're right well that's what we're scoring including a lot of other parameters in the in the process so when we look at the upcoming milestones as we have obviously the upcoming two upcoming meetings with data presentations at ULAR for the lupus data and at the European Hematology association meeting additional data analysis that we run there that we're going to present there from our overall pivotal study looking at the impact of bridging therapy in outcome and for the outcome of patients that was an oral presentation there and then we're planning for towards the year end to have an update longer-term update on the SLE study the Carlisle study and and initial data from the MS study, moving then as well as initial data from the amyloidosis study as well. And then as we go to next year, more definitive data on MS, as well as the final pivotal dataset for the Catalyst trial. And finally, the Luminar trial will read out in 2028. So that's kind of where we are, and that leaves us with an opportunity for a few questions.

Matt Phipps, Analyst — William Blair

Thanks, Christian. We have a couple minutes, and then, of course, there will be a breakout session. You know, maybe I'll start. You showed one of the slides with 1,600-ish patients with adult ALL in the U.S. currently. You know, when we've talked to doctors about a capsule, they talk about the ability to actually treat patients that they would have never treated with CAR-T before. So I'm curious, of that 1,600, how many do you think were previously getting or kind of eligible for Tocardis, and where do you think that number could go to as far as what percentage of patients might actually be able to get a capsule from that 6-year?

Christian Aiton, CEO

It's a really good question. So the thing is that the data for Tecardis is not shown for acute lymphoblastic leukemia, but a combination of acute leukemia and mantle cell lymphoma, where there are two labels for the product. It's never been broken out. So that's an estimate we have to make. what we do know is and what the sense we have is that the probably the penetration that for CAR-T penetration at all patients may have been around 10 percent before we know we reached about 10 percent in our first year of launch and that actually started to actually increase the scope of the patients that clearly were treatable before and we could see it because there's real-world data collection for the CAR-T is the real-world data collection for our product and And what we see is that in the patient composition, we see patients, the median age actually go up from about below 40 to 50 years of age. That tells you one dimension, so age is a risk factor. We have patients that have poor ECOG status. That's another dimension where you would normally not treat because it's difficult to treat. And what we see in both situations is that patients with low disease burden are included as well, which makes a lot of sense because that's where you have the highest chance of a long-term outcome. So we start to see, at least based on age and based on ECOG status, as well as some of the other prior histories of the patients, that we're starting to go beyond of what was initially treated with the CAR-T therapy. What has been very encouraging is that the physicians are basically telling us, and this goes back to the same report that's available and you can listen to at our website, where the physicians are talking about the fact that, indeed, it's the profile for the product, the safety profile, allows you to consider treating patients you couldn't possibly treat before. The patient I mentioned with this, you know, basically becoming paraplegic, you would be hard-pressed with any therapy to do that, to actually treat that patient, because it looks like anything you do is probably making things worse. And she decided still to go forward and do it, and it did work really well. When we think about where we can go, I think this is what we actually will deduce from where Blin-Cyto could go, the T-cell-engaging antibody that Amgen is commercializing. And what that product basically achieved before it went into the frontline setting is about a 60% penetration of the relapse refractory setting. We believe we have a good chance of actually doing that.

Matt Phipps, Analyst — William Blair

And then, you know, first year was obviously a lot of just ramping up clinical trial sites, commercial sites now. You were able to get to $74 million in revenue. This year, you know, first quarter, about $100 million run rate right in the first quarter. A little bit more site expansion, but, you know, getting to that $120 to $135. Do you see that as just kind of continued ramp over the year as you, you know, get to those last centers, get more physicians familiar? Are there any other kind of gating things that could lead to any other inflection?

Christian Aiton, CEO

Right, so with the centers that we have active now, we reach approximately 80 plus percent of all patients in the US. So the geographic coverage is there. What we really need to do is we need to get for each center to a point where all the physicians that are treating ALL patients start to using the product. So the expansion is actually not adding more centers. There's a reason for that, why we do that, but that's not the primary reason. The expansion is really driven by the broader adoption within a center, and then for each physician to start using the product across the entire range of the label. I'll give you an example. If you're a transplanter, stem cell transplanter, that's what you have experience with, and you would be using that because you know there's long-term outcomes possible. Not often, but it's possible. So that's your primary go-to situation. So for that type of physician, quite often, the first patient that that physician would treat is a patient that is ineligible for transplant because what I normally want to do is not applicable. I cannot use that. So, okay, I'm going to use actually this product for a patient I couldn't otherwise actually support. And you start gaining experience and then actually gradually build from there. And we've seen that actually play out. So one of the physicians who was involved in our pivotal study He was, you know, a transplanter with, you know, a few years older than myself, and he's been doing that for the most part of his career. Started to treat an 80-plus-year-old patient. He couldn't possibly do anything else because the patient couldn't take more toxicity. Had a good outcome, and then started treating patients at the broader range. When I saw him last, he was basically telling us that he's been, you know, actually having treated close to 10 patients at that point in time, and not transplanted a single one of them so it just shows the behavioral change and so this is what you do in this in these indications what you need to do is really a change in behavior and the change in it and with that kind of moving and broader get to a broader adoption of the product so that's the focus is really in that engagement and broadening the additional centers we're adding has a lot more to do with um either geographic gaps where the distances for travel is just too that doesn't seem reasonable or there are centers feeding into larger treatment centers that on their own actually have substantial patient flow and it makes sense to start treating patients actually closer to where they live and that's sort of kind of the expansion we do but right now you can see it on otolith assist you can see actually on there the distribution of the centers and they pretty much follow frankly the the population density of the u.s pretty much if if you look at the map.

Matt Phipps, Analyst — William Blair

Great, well with that, I think that's our time. Hope you can join us in Burnham B in 10 minutes for the breakout.

Christian Aiton, CEO

All right, thank you much.

Matt Phipps, Analyst — William Blair

Thank you.