Autolus Therapeutics plc

Good day. Thank you for standing by. Welcome to Autoless Therapeutics' third quarter 2025 financial results conference call. At this time, all participants are in listen-only mode. After the speaker's presentation, there'll be a question and answer session. To ask a question during the session, you will need to press star one one on your telephone. You will then hear an automated message advising your hand is raised. Please note that today's conference is being recorded. I will now hand the conference over to your first speaker, Amanda Cray, Executive Director of Investillations.

Please go ahead. Thank you, Livia. Good morning or good afternoon, everyone, and thank you for joining us on today's call. With me, our Chief Executive Officer, Dr. Christian Eiten, and Chief Financial Officer, Rob Dolski. I'd like to remind you that during today's call, we will make statements related to our business that are forward-looking under federal securities laws and the Safe Harbor provisions of the Private Securities Litigation Reform Act of 1995. These may include, but are not limited to, statements regarding status of the ongoing commercial launch of OCSEL in the U.S., Autolist's manufacturing, sales, and marketing plans for OCSEL, the market potential for OCSEL, and the status of clinical trials, development, and or regulatory timelines and market opportunities for OBSEL and our other product candidates. These statements are subject to a variety of risks and uncertainties that could cause actual results to differ materially from expectations and reflect our views only as of today. We assume no obligation to update any such forward-looking statements. For a discussion of the material risks and uncertainties that could affect our actual results, please refer to the risks identified in today's press release and in our SEC filings, both available on the investor section of our website. On slide three, you'll see the agenda for today's call. As usual, Christian will provide an overview of our operational highlights. Rob will then discuss the financial results, and Christian will conclude with upcoming milestones and closing remarks. We'll then take questions. With that, I'll turn it over to Christian.

Thank you very much, Amanda, and let's move to slide number four. Welcome, everybody, and thank you for joining us at the third quarter financial results. We had a very good quarter, and I think we're off to an excellent launch with Ocatzel in the U.S., treating or targeting patients with relapsed refractory BLL. We managed in the first nine months to achieve market leadership, broad market access and coverage, and established reliable product delivery. We also believe that there is a significant opportunity to grow the CAR T market in this indication. And we're very pleased with the feedback we're receiving from physicians using the product and the interest expressed in conducting investigator sponsored trials in frontline settings in ALL as well. We're now at the point where we have achieved a significant amount of stability in our operation and our ability to deliver product in a timely and high quality manner. And we're now actually focusing on optimizing our operation and really leveraging the investments that we've made in our infrastructure and our systems and have now an opportunity with a lot of data that we collected along the way in these first nine months to optimize our processes and with that drive efficiencies going forward. Importantly, in parallel, we're expanding the opportunity that we believe we have with OBSEL or a capsule, and are looking to build a pipeline in a product, and are working on three current indications. The first one is in pediatric ALL, where we're initiating a potentially pivotal study, a phase two study in lupus nephritis, also with an intent to drive towards a label, and an exploratory phase one study in progressive multiple sclerosis. In short, we're focusing on driving market share in ALL, improve the margins, and expand beyond ALL. Moving to slide number five, quick view on the performance that we have to date in our launch of a capsule in the US. We have achieved 21.1 million in net sales in the third quarter, and we have deferred revenue of 7.6 million indicating that there is a good number of products currently sitting at the centers at the end of the third quarter, not yet infused and ready for infusion in the fourth quarter. The nine months ended September 30, we achieved 51 million in sales for the product. When we look at how we're executing on product delivery and patient access, we're tracking very well. We had at the beginning of the year told you that we're targeting to authorize 60 centers. By the end of this year, we achieved that goal. So we're at 60 centers today. And we will keep adding additional centers to fill out the geographic gaps that we're having across the U.S. And with that, minimize the travel distances for patients to access therapy. The manufacturing success rate is well above 90%. And we have attained patient access for more than 90% of U.S. covered lives. So this gives us a very strong foundation, both from a rollout perspective, but also from a dynamic perspective as we're sort of looking into the fourth quarter and into 2026. Moving to slide six. We believe there's a significant opportunity to increase the penetration and grow the CAR T market share. When we look at the level of CAR T market, CAR T use, Before our product entered commercialization in 2024, we're seeing about 15% market share for CAR-T therapies in this indication. When we look in the treatment centers, the 60 treatment centers that we're active in now, which actually cover the vast majority of the relapsed refractory ALL patients, we believe that the market share within that group of 60 centers at this point is probably around 20%. This gives us an opportunity for substantial growth within the centers that we're already present in and the footprint we already built. This is about relationship building and deepening those relationships to really drive the uptake further and actually drive towards the types of levels of uptake that we've seen a few years ago BlindSight to achieve in this particular indication. The importance, I think, of the experience that the physicians are making with the product really cannot be overstated. The actual hands-on experience is critical here, and we believe that the way that we're seeing the product perform and also what we're seeing being reported or planned to be reported at ASH from the Rocket Consortium indicates that there is a very positive experience that the physicians are making with the product consistent with the experience that we have gained through our development and in particular also within our registration study, the Felix study. Moving to slide number seven. We're going now, focusing, we had a quick look at the launch. We're now focusing on the key approaches here that we're looking to optimize our operation and really improve the overall margins and efficiencies. When we look at slide number eight, first a quick look at changes that we have made within the leadership team. We have three new key members in the team that joined us during the course of the third quarter. First off, Miranda Neville, who some of you may actually have met in the past, has been actually with the company for about five years and initially worked on the planning and setup of the Nucleus facility. then actually took over the overall program team for OBSEL and drove the program through the registrations and getting us the approvals in the US, in Europe, and also in the UK. And now actually returns back to product delivery in the role of chief technology officer leading the entire team. Cynthia Pagina has taken over from Brent Rice as the US chief commercial officer and country manager. and has an extensive set of experience in oncology, in immunology, but most importantly for us, in cell and gene therapy. She has an extensive experience launching products in this space and we believe is extremely well positioned to actually grow the opportunity for our council from here forward. And then Patrick Milveni, who's taken over from Andrew Merzika as the Chief Accounting Officer reporting into Rob Dalsky, Patrick joins us from Horizon Pharma, where he's been going through the very significant growth that that company went through, and also increasing complexity and belief has an excellent background to really help us actually go through the next phase of optimization and also of growth for the company. Now, I would like to sort of highlight also the three members of our team that actually handed over the responsibilities to Miranda, Cynthia, and Patrick. Dave Brochu, who's been the chief technical officer handing over to Miranda, has done an excellent job building our product delivery team and building also the nucleus facility, taking it into operation and launching the product. It was a huge accomplishment to actually go through that entire growth phase and building that entire part of the operation from the ground up. And there's a huge thanks that goes to Dave for that incredible achievement. Brent Rice, a predecessor of Cynthia, has done a fantastic job building a very strong commercial team. The systems required and actually executed an excellent launch of the product and getting us really rolling now, I think, in a way that is quite remarkable and gives us, I think, a great outlook as we're sort of looking into 2026 now under the leadership of Cynthia. And also Andrew Merzika had built obviously a lot of the infrastructure pieces from a corporate side perspective that Patrick's now taken over. And also a great thanks going to Andrew as well. So with that, I'd like to move to slide number nine. And just a few thoughts in terms of the ability to really drive efficiencies and cost savings. I think where we are is that we have now built a very strong foundation through the strong launch performance with a highly reliable product supply. That's been the critical piece that we had to actually build and establish this year. And frankly, has been a very significant challenge for most companies launching cell therapy and in particular CAR T products. Having established that now gives us an ability also with all the learnings that come out of that experience to really streamline the processes. The training wheels can go off and we can now focus on making sure that we're just simple, that we're as simple as possible, as straightforward as possible in terms of the processes that we run. Take those processes that we decided that are necessary, optimize them, and automate them to the extent we can. And importantly, innovate. And one of the two key areas that we're planning to and we're focusing to innovate on is really on manufacturing, where there's a lot of activity going on, on technology, on automation, but also on the biology side and on market access to make sure that we can actually increase the footprint, the geographic footprint for the product and the ability to actually serve a substantially larger group of patients going forward. So with that, I'd like to go to slide number 10. And we're now actually moving forward looking at the opportunities to expand the potential for and realize the potential of OB cell in additional sets of indications. With that, heading to slide 11, a quick overview of the clinical trials that are ongoing to expand the use of OB cell in additional sets of indications. We're starting with the pediatric BALL study called Catalyst that is ongoing. We are reporting the phase one portion of that trial at ASH, and we also have just received a few weeks ago an RMAT designation for the program as well. We're in the process of starting up the phase two portion of this trial, and we're excited about the opportunity and obviously the opportunity to share the initial experience from our phase one. The second study is the Carlyle study in severe systemic lupus patients. We had initial data presented at ACR, and I'll briefly talk about that in the upcoming slides. And we are planning for an oral presentation at ASH with a slightly expanded data set. Lupus nephritis is the focus for our first pivotal study in autoimmune disease. The study is called Lumina and we expect to have the first patient in the study before the end of the year. And then finally, our exploratory phase one study in progressive multiple sclerosis called Bobcat. Bobcat started in the third quarter and we had our first patient dosed in October. Now, in addition to the internal studies, we obviously are going to support the investigator-sponsored trials, exploring the use of a capsule in frontline settings, but also our OC will follow the real-world experience that the ROCA Consortium is collecting for OB-Cell in relapsed refractory adult ALL, and it's truly a reflection of the real-world experience of our customers, collected by them and analyzed by them. So with that, we're going to slide number 12 and just a few words on the data that we actually presented at the ACR conference in Chicago just at the end of October. We did present the experience that we had with the product at the 50 million fixed dose level that we have evaluated in six patients. These patients are patients with very advanced disease, very high, slight high scores at inclusion, very significant impact on their kidney function. We have at least six months of follow-up with the patients that we've reported on. And we have seen that five out of the six patients achieved Doris remission. We have 50% of three of six achieved a complete renal remission. And we have no evidence of new disease activity up to 14 months of follow-up. And the patients do not receive any lupus-directed therapy. Also, obviously, when you have urinodorus remission, the steroid levels that patients may receive are at or below 5 milligrams per day. The safety profile with the product was very positive. The patients had no ICANS, no high-grade cytokine release syndrome, no DLTs at the 50 million cell dose level. PK and biomarkers, we showed a quick B cell depletion after infusion. We saw then obviously that at the point when the CAR T cells stopped persisting, that the B cells started to recover. And we do see a predominance of naive B cells and reconstitution. More of that data is expected to be shown at ASH. When we look in terms of now the next steps with the study, there are two directions that we're sort of exploring further. One is actually at 50 million cell dose adolescent patients, age 12 to 17. For a lot of these patients with early onset of lupus, they have a particularly challenging course of the disease, and there's a very significant medical need. In fact, when you look at the population, the highest medical need is in the youngest patients. So this will be explored at 50 million cell dose, which is obviously equivalent to, as you may remember, the pediatric level that we would use and just translate it into a fixed dose level. And we're also exploring one additional dose level on the adult side to sort of round out the experience in the SLE patients. The recommended phase two dose is already defined as 50 million cells. Moving to the next slide, slide 13. This is a more detailed look at the safety for the product. And as you can see, it's a very good safety profile. No eye cans. We only had observed one grade one CRS in half of the patients. A relatively short period of neutropenia following lymphodepletion, which was resolved by day 22. No high-grade infections that we have observed in the patients. And not unusual for patients that have significant kidney involvement. We have seen five patients with transient hypertension. Three had pre-existing hypertension of the five. When we now look on slide 14, this is a view of the SLED-I scores and the progression of the SLED-I scores over time, looking at each individual patient. And what we're looking at, if you look at the screening bars, which are on the left-hand side of the respective charts, you can see that every one of these patients had a significant kidney component. This is the blue part of the bar. And then the other colors actually relate in part to increased DNA binding, as well as low complement, which is the black and the yellow. And the other colors actually are linked to other forms of autoimmune manifestation. and inflammatory processes, whether this would be mucosal ulcers, rashes, arthritis, alopecia that we have seen in these patients. So as you can see, these patients have not just actually a renal component, they have a multitude of disease manifestations. And you can see as these patients progress over time, these manifestations actually are very quickly reduced. And we then actually see with a bit of a lag also in the majority of the patients, a reduction of the renal signal as well. So all very encouraging. What's important is, again, none of these patients actually had any form of relapse or flare. So moving to the next slide, this is slide number 15. This is a quick look at the DORIS assessment that you can see that Five out of six patients had achieved a doris remission with a median onset of five months. And again, in terms of the steroid dose, that by month six, all patients had steroids tapered to less than or equal of five milligrams per day. And no other lupus medication, of course. Now, when we look at the upcoming data presentations on slide 16 in oncology, pediatric ALL, poster presentation, phase one experience from the CATALYST study, adult ALL first, an oral presentation from the FELIX trial looking at the impact of the product cell phenotypes or the actual features of the product and the linkage to the longer-term outcome in these patients. And then second, a poster, which is looking at CAR T-cell persistence at month three, and the ability to actually predict outcome based on that data for these patients, the longer-term outcomes. Again, experience from the Felix study. On the autoimmune side, as mentioned, we have an oral presentation for the CAR-LIL trial. And we just want to highlight the fact that the ROCA Consortium has several presentations, one of which looks at the patient characteristics, toxicity response after real-world administration of both Obicel and Brexicel alongside the same time horizon within their centers, which at least to my knowledge is probably the first time we're seeing basically data collection of two CAR-Ts in the real-world setting in parallel. With that, we're heading to financial results and heading over to Rob.