Autolus Therapeutics plc

Hello, ladies and gentlemen, and welcome to the Autolist Therapeutics call to discuss its full year 2024 financial results and business updates. As a reminder, this conference call is being recorded. I would now like to turn the conference over to your host, Amanda Cray. Please go ahead.

Thank you, LaTanya. 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 Ocassel in the U.S., auto list manufacturing, sales, and marketing plans for Ocassel, the market potential for Ocassel, and the status of clinical trials and development and or regulatory timelines for OB-Cell 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 will then take your questions. With that, I'll turn it over to Christian.

Thank you very much, Amanda, and welcome everybody to our fourth quarter 2024 financial results update. So first off, obviously, we had a very successful 2024, which sets us up well for the 2025 that we're already in a few months now. First of all, I'd like to just highlight kind of the key objectives that we have for the year. So the primary objective clearly is to execute a successful commercial launch for a capsule, and we're doing really well and advancing well on that. We'll provide a bit of an update as we go through the presentation. But also, we see a lot of opportunity for OVCell to actually expand its utility, the excellent profile that we've seen in our current experience with the product, and explore the opportunity and additional indications, as well as look at the opportunity for moving some of our other product candidates forward And at the same time, obviously, we'll be, I think, very selective and very careful in how we're going to place our investments. Now, one of the things that you'll hear us also briefly mention is the fact that we're planning for an R&D event on April 23rd in New York that will actually outline in more detail kind of the plans on how we want to move forward in sort of creating additional opportunities for future growth. So with that, on the next slide, I believe we're going to summarize kind of the key opportunities that we have seen and sort of achieved during the course of 2024. As I mentioned, this has been a remarkably successful year, and we had a very strong start of the year in the first quarter when we were able to add about $600 million to our balance sheet through our collaboration with BioNTech as well as a public financing that we conducted in the first quarter. This set us up very well to really be able to focus fully on making sure that we get OBSEL already to get through the approval process in the US, but also to set us up well for the commercial launch of the product. As we're going through the year, I would say we had continuous activities on the commercial side to actually get the sites ready. so that they actually would be in a position, once the product was approved, to actually start onboarding the product and be in a position to move forward and actually use the product in the commercial space. Now, when we look at the approval itself, we achieved the approval in the US on November 8. And I think what was very encouraging was that not only did we actually achieve the approval slightly ahead of schedule, but also actually had a label which gives us a broad opportunity in the relapsed refractory ALL space for adult patients, and actually having the first product getting through the approval process that actually did not require a REMS program. Very nice setup in terms of the approval itself, the timing of the approval, not having a delay, recognizing that the product had an attractive safety profile, and therefore no obligation for collecting additional information around CRS and ICANNs going forward. And that actually is, I think, a very, very strong foundation. What was then very, very helpful is that we were able to actually get the clinical results published in the New England Journal of medicine in the early part of December and shortly thereafter the product was included in the NCCN guidelines. This is very important as you start launching a product because it gives obviously a lot of confidence and indeed this is a recommended therapy and also will support the decisions that payers have to obviously go through to get the patient signed off for commercial use. When we look at the track that we had as we went through the first three months of the launch, we see a very good development in terms of the number of centers that are actually authorized and are able to deliver the product. We're currently showing 33 centers that are authorized as of March 19. And we expect that that group of centers allows us to reach approximately 60% of the target patient population in the US. We are continuing to move forward, adding additional centers as we go through this year. By the end of the year, expect to have approximately 60 centers that will be ready and in a position to deliver Ocacil to patients. We believe that those 60 centers actually reach the vast majority of the patients in the U.S. and will give us a very strong foundation for a successful delivery of the product to the patients across the U.S. I already mentioned that we obviously have the publication of the study, of the Felix study in the England Journal, but we're also, in addition to obviously getting ready for launch in the U.S., we're also moving through the regulatory steps in the U.K. and in Europe and had filed in the early part of 2024 with EMA and then by middle of the year with the MHRA. And we expect actually regulatory decisions in the second half of 2025. We also started the process to actually establish ultimately the utility of the product in the UK and we're going through the assessment. by the National Institute for Health and Care Excellence, or NICE, which is one of the key processes that you have to run through to get to a position to actually get a product adequately reimbursed in the UK. As we went through the year, there were several data presentations at key conferences that really focused on the properties that we have observed with a capsule or OV cell in the relapsed refractory adult population as we were going through and sort of actually gained more and more data from the Felix study. We could report on the durability of the product, highlighted the safety profile, the impact of having deep molecular responses and persistence as indicators and likely requirements to get to long-term outcomes with the patients. And we also actually were able to show the impact of tumor-verted guided dosing, as well as the safety profile's impact on reducing health economic cost when we look at the cost of treating the patients. So quite a comprehensive data set that we actually were able to share across a range of conferences during the course of last year, including the tandem meeting in February this year. Moving to the next slide, we're looking briefly here at some of the key outcomes that were actually presented and published in the New England Journal publication. And what we're seeing is that the product has a very high level of clinical activity reaching deep molecular responses in the majority of the patients. And that type of activity obviously has come with an attractive safety profile that we can see that overall the level of high-grade cytokine release syndrome and the level of high-grade neurological toxicity or ICANs are low. And in fact, when we look at patients that have low levels of disease burden at the time of dosing, those patients did not experience high-grade events, either CRS or ICANs, which I think gives us a good understanding of patient profile and also kind of from a safety perspective. And as we're looking at the next slide, we also see that the tumor burden also actually gives us important information about the impact of the therapy it can have in terms of longer-term outcome. In this slide, we're looking at event-free survival. As you can see in the top part of the panel, event-free survival stabilizes at around between 45 and 50 percent. And we see that the line actually is starting to go horizontal, indicating that there is a proportion of patients that do not actually seem to progress or actually have events that are occurring. When we look at what the impact is of the disease burden prior to dosing, we can see that patients that have low disease burden, less than 5%, do remarkably well. Whereas most patients with a wide range of disease burden up to 75% actually continue to do very well. And only the patients that had extremely high levels of disease burden of more than 75% of blasts in the bone marrow at the time of dosing obviously have a lower outcome. So overall, I think, gives us a lot of information about the properties of the product. We have just talked about the safety that we've seen across the board, obviously the efficacy from an event-free survival across the board, which looks very attractive, and also there the impact of actually treating patients when the disease burden is still at a lower level, which gives us a very good prognosis for these patients. When we look at the next slide, we see that translating into overall survival, and we see the same overall picture, again, stabilization of overall survival in the overall population, and as well an impact that we see in terms of tumor burden, where patients, again, with lower tumor burden at lymphodepletion, and also patients with extremely high levels of tumor burden actually have a poorer outlook. Overall, remarkable set of data and I think sets us up for a very, very attractive proposition for patients in this field. Moving to the next slide, as we're sort of thinking about sort of the early momentum of the launch in the US, indicators here is the centers that can actually actively deliver products to patients. As I mentioned, we reached 33 centers on the 19th of March, which gives us obviously a very good reach and distribution already across the U.S. and obviously a presence in a lot of the more densely populated parts of the U.S., which is also the reason why we're already reaching more than 60% or around 60% of the patient population. What's also important is we're making good progress in terms of patient access, in terms of lives covered, and we see actually very good momentum there as well as we have been going through the last three months. So that is very encouraging and also actually is a good sign that indeed patients do have really an opportunity here to get access to this therapeutic option. Finally, on the next slide, on the update, you have heard us actually talk quite a bit about the actual manufacturing facility for commercial supply, which is the nucleus facility. I think one of the remarkable things about this field is obviously as you have the first commercial patient come in, that's when you literally start the operation at that facility. And I think at this point we can say that we're off to a really good start and the facility came to life and is humming well and I think will give us a very strong foundation in our ability to reliably deliver product to the centers. So with that, on the next slide, which is really focusing on the expansion of the OB cell opportunity, a key area that obviously we're going to be focusing on in 2025. First off, on the autoimmune side, we've been running obviously the Carlisle study. We have the initial cohort of six patients that are all dosed. We're running through the data cut for the upcoming R&D event on April 23rd. And we're looking forward to updating you on that initial experience with the first six patients. And we also are planning to present the data with long-term follow-up across the patients at a later time point in the second half of the year. On the hematology side, obviously we also have been moving forward with our pediatric study, the PY1 study, and we expect to provide an update also second half of the year. That study has also been evolving very nicely. In terms of the early pipeline, we obviously have a set of activities ongoing with our partners at the University College in London, and we are moving forward or have been active in Auto 122, Auto 6 and G, and Auto 8, and we continue to progress the activities around those programs, collecting more information around the programs and are planning to also give a short update at the R&D day towards the end of April. So with that, I'd like to actually hand over to Rob and give us a summary of the financial results.