Hello, ladies and gentlemen, and welcome to the Autolus Therapeutics' Call to discuss the Full Year 2023 Financial Results and Business Updates. [Operator Instructions] As a reminder, this conference call is being recorded. I would now like to turn the conference over to your host, Olivia Manser. Please go ahead.

Thanks, Michelle. Good morning or good afternoon, everyone. Thanks for joining us on today's call. With me are Dr. Christian Itin, our Chief Executive Officer; and Rob Dolski, our Chief Financial Officer. So, on Slide 2, before we begin, I'd just 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 the status of clinical trials and development and for regulatory timelines for our product candidates and our expectations regarding our cash runway. 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 our SEC filings, both available on the Investors section of our website. On Slide 3, you'll see the agenda. As usual, Christian will provide an overview of our operational highlights, Rob will then discuss the financial results before Christian will conclude with upcoming milestones and closing remarks, and we will then take questions. Over to you, Christian.

Thanks a lot, Olivia, and welcome, everybody, to our full year update. Obviously, it's been a very successful year for us, and we're going to go through the accomplishments during the course of last year, particularly with our lead program, obe-cel, on the following slide. But what we'd like to start out with is obviously the important transactions that we've been able to complete in February this year, obviously, post the reporting period. Obviously, the two transactions we conducted. One was the strategic collaboration with BioNTech, which I think sets us up in a very interesting and attractive way going forward with a very strong partner, and I think a lot of opportunity to realize synergies between the two companies. The focus of this collaboration has been sort of on three key platforms that we have developed. The first one is providing access to BioNTech to our manufacturing platform. Obviously, at the core of that being the Nucleus manufacturing facility, but also, of course, all the systems surrounding the product supply platform. Secondly, the commercial platform that we've been setting up and obviously in the midst of preparing the company for a potential launch at the end of this year for obe-cel. Also the systems we set up, the procedures, the presence and the centers we're building up, all obviously can be leveraged beyond just obe-cel. And I think there's significant opportunity there for additional programs to serve -- be served through that platform. And then, also, obviously, the access to two of our product candidates, AUTO1/22 and AUTO6NG in co-development and co-commercialization options that we have granted to do both of those programs. And I think will allow us to accelerate some of our pipeline programs, and obviously looking forward to the collaboration here with our partner. We also…

Thanks, Christian, and I'm going to be on Slide 26, the financial summary. Good morning or good afternoon to everyone. It's my pleasure to review our financial results for the full year 2023. Our cash and cash equivalents at December 31, 2023, totaled $239.6 million as compared to $382.4 million at December 31, 2022. Our total operating expenses net for the year ended December 31, 2023, were $179.7 million as compared to $143.4 million for the same period in 2022. Our research and development expenses increased from $117.4 million to $130.5 million for the year ended December 31, 2023, compared to the same period in 2022. This change was primarily due to increases in operating costs related to the company's new manufacturing facility, contractual milestone payments and headcount-related costs, as well as a decrease in our U.K. reimbursable R&D tax credits claimed through the U.K. small- and medium-sized entity scheme. These were partly offset by decreases in clinical and manufacturing costs associated with the obe-cel clinical program. Please note, in prior years, Autolus reported the R&D tax credits as income tax benefits on the statement of operations. The company has revised its financial presentation, including the prior years, and will now present such tax credits as a reduction in R&D and development expense. As a result, income tax benefit has reduced by $19.5 million and $24.6 million for the years ending December 31, 2023 and 2022, respectively, with the corresponding reductions in research and development expenses and total operating expenses. Moving on to general admin. Our expenses increased from $31.9 million to $46.7 million for the year ended December 31, 2023 compared to the same period in 2022. This increase was primarily due to an increase in general administrative headcount, supporting the overall growth of the business, primarily related to pre-commercialization activities. Our net loss attributable to ordinary shareholders was $208.4 million for the year ended December 31, 2023, compared to $148.8 million for the same period in 2022. Autolus estimates that with its current cash and cash equivalents and proceeds received from the strategic alliance with BioNTech and our equity financing, we are well capitalized to drive the full launch and commercialization of obe-cel in relapsed/refractory adult ALL as well as advance our pipeline development plans, which includes providing runway to data in our first pivotal study of obe-cel in autoimmune disease. I'll now hand things back to Christian to wrap up with a brief outlook on expected milestones. Christian?

Thanks, Rob. So, a quick look on Slide 28 on the planned news flow. Obviously, clearly, a significant focus on obe-cel delivery. First, on data, as I mentioned, we're planning to update at the ASCO, EHA and ASH this year. This is going to be sort of the June and December timeframes. We're planning to submit to the U.K. MHRA, a marketing authorization application in the second half of this year. We know, obviously, our PDUFA target action date, November 16 this year. And we plan to show the first data from our SLE Phase 1 study in -- towards the end of this year. In addition, obviously, there will be updates on AUTO8 expected for the end of this year, as well as opportunities, certainly, for publications during the course of this year on a number of our programs. So, this is kind of a quick update on the planned news flow. And in terms of summary, going to Slide 30, obviously, we're in a very strong position with the company. We've been executing very consistently during the entirety of the last two years. We've continued on this exact same pace into 2024, and we continue to do so. We have a strong cash position in the business. As indicated, pro forma, we sort of started the year with north of $800 million in the bank between the year-end cash, the BioNTech transaction and the equity raise that we conducted at the beginning of February. And obviously, this gives us a very strong base to not only drive our lead program forward onto the market and through the launch, but also expand the opportunity beyond. In addition to, obviously, opportunity to expand from a pipeline perspective also through the options granted to BioNTech. I think when we think about the capabilities we've built, we are obviously in a very good position with regards to our commercial manufacturing capabilities. That's been an enormous lift over the last -- course of last year. It was also a very significant investment that we have conducted through in that period. Obviously, those costs, obviously, are going down because a lot of the obvious capital expense-related costs obviously have some -- are behind us. And with that, we see sort of a shift of expense moving from the setup costs for the facility, completion of the pivotal study in ALL moving over to launch-related launch preparation-related costs. So that's sort of the key swing that we'll see as we go through the course of this year. And I think we have significant opportunity with the pipeline and also with obe-cel to expand from an indication perspective and also from an overall product opportunity perspective. So, overall, I think we're in a great spot. I think great outlook for this year. And we're looking forward to taking your questions. Thank you.

Thank you. [Operator Instructions] Our first question comes from James Shin with Deutsche Bank. Your line is open.

Hi. Good morning, guys. Can you hear me? I'm on a bus. I apologize if there's any background noise.

No worries, loud and clear. Hi, James.

Hi, Christian. A question on the MHRA approval. Did their inspection have any overlap with the FDA or EMA or any other upcoming inspections? And then, I have a follow-up on obe-cel for lupus and ALL.

Right. So, the MHRA inspection and license is a prerequisite for us as a company to be able to export whether this is going to be to the U.S. or to the EU. So, it is independent and separate from a U.S. FDA perspective, but it is linked to the European filing. In fact, it is a prerequisite for the European filing. And it is actually the fact that the facility has to actually have a certificate from the MHRA to be even to file with the European agency. So that's obviously where it's directly linked to the European side, and it is a necessity for the U.S. side, but independent of the DLA -- of the FDA review of the facility.

Is there any overlapping metrics from the MHRA inspections with FDA inspection by chance?

Well, first of all, obviously, the fundamentals are the same. And this is all around the GMP manufacturer and the guidelines associated with GMP manufacturing. So, the basis for the inspection, the basis for review is the same basis. So, there's no difference there. But there are two independent bodies that actually do their independent reviews, whereas in the Europe, the European agency relies on the MHRA's reviews.

Understood. And then, as you prepare for obe-cel ALL launch, a lot of oncologists we've spoken to is focused on quality products in a timely manner, given the buyer condition of the patient. And if you look at some of the peer cell therapy launches, there's been some pickup. What is Autolus doing to make sure or what have you learned from these peer launches to avoid some of these pitfalls?

Yeah. I mean it's absolutely a relevant question. And in fact, when we talk to physicians and our ad boards, the top question coming back or the top point coming back is, we need access to product. And so, the ability to get access to product, to get slots, to get the product in time is absolutely critical. It's a reflection of some of the challenges that the centers experience with prior launches. So, we spent a lot of time, obviously, optimizing our systems, minimizing the turnaround time will be in terms of delivery time at around 16 days at the time of launch. And we're also going to do, obviously, full runs from each one of the centers through the entire chain to ensure that from every center, actually, the flow and the processes are fully operational before launch, before we're getting to launch. So, there's going to be not only the processes have been adjusted, that be simplified, but also it's going to be a whole bunch of dummy runs from all of those centers to ensure that, indeed, all the aspects of that journey actually are fully vetted and fully tested for each individual center.

Appreciate that. And then finally, on CARLYSLE, have you looked or depleted any of the six or the first six patients? And then what are the gating factors to get the subsequent six patients?

So we haven't guided actually on dosing or not dosing. The study is open. It's enrolling. The study itself is not a dose escalation study, so we don't have DLT periods and we're not limited by sort of the typical Phase 1 dose-finding studies, which they have to go through review processes in the industry. So, we have an ability to actually enroll all of these six patients as they become available. But we don't have limitations from the study design that would sort of actually gate or slow down the process.

Appreciate it. I'll yield to the floor. Thank you.

Thanks a lot, James. Appreciate it.

Thank you. Our next question comes from Asthika Goonewardene with Truist. Your line is open.

Hi, guys. This is Karina for Asthika. Thanks for taking the question. So. I had a question on the FELIX update at ASCO. Besides the longer follow-up, what new data can we expect in this presentation, and also will it be able to be paint a clear picture of what obe-cel can do in terms of like the favorable impact for patient outcomes without a need for transplant?

So, in terms of the -- thanks, Karina. In terms of the FELIX study and what we're expecting to do, obviously, there is quite a significant level of information within the FELIX study that we haven't actually really worked through or presented yet the impact of bridging in more detail, what are the components that matter there. There are other components as well, obviously longer follow-up as well, and there's other risk factors and risk categories that actually start to see as you go through the data that I think are very helpful and I think very informative. So, there's going to be additional sub-analysis, longer-term view, but also additional sub-analysis that we're going to be -- we'll be presenting during the course of this year. So that's the first part. The second part is actually we also have been looking at the impact of transplantation. That's certainly also an area that we're planning to sort of report on as we go through the course of this year. Obviously, an interesting question there is do we think it is actually -- does it look like it's actually improving the outcomes, or do we actually have a different type of outcome here. And those are questions we're evaluating, and we'll certainly are reporting as we go through the course of this year, if not at ASCO then certainly at ASH.

And the data presentations for EHA and ASH later on that's just an encore of the ASCO presentation?

No, the presentations are -- some are related, but there's not -- the abstracts are not identical between the two. They may share some of the data, but in terms of the focus is slightly different.

Okay. Got it. Thank you so much.

Thanks a lot, Karina.

Thank you. Our next question comes from Gil Blum with Needham & Company. Your line is open.

Hey, good morning and good afternoon. Thanks for taking our questions. So first question kind of focusing on the bridging aspect in the study. This is something that has also recently come up in the advisory committee materials for ABECMA and CARVYKTI. It looked like bridging had a pretty important impact on patient survival even before you got there, treatment. In many ways, maybe the fact that you allowed physicians to pick their bridging may have actually assisted you here. And how can you optimize bridging? Is there like a study to be done here?

It's a really good question. I think, first of all, the situation is obviously a bit different between multiple myeloma, adult ALL and multiple myeloma, which is the progression of disease is much less rampant, much less significant in terms of the speed of deterioration as you would have in ALL. ALL obviously can go as you have seen, you have patients go from minimal residual disease to more than 75% tumor burden. I mean, that gives you a sense of the explosiveness of the disease. So, bridging is obviously something that we have to do in these patients. Otherwise, the tumor burden actually gets overwhelming and in of itself becomes a limitation in these patients. But as you could see, obviously, the impact can be that you either have no -- see no impact whatsoever and the patient just go straight over, stays about 75%. These are -- certainly, you would call this refractory in the very functional -- as a very functional determination. But you also may have patients that actually transiently may actually have a significant decrease in tumor burden between involvement and the actual dosing. And that does have an impact in the sense that the level of tumor burden as lymphodepletion. So right before your dosing, obviously, it seems to actually have quite a significant impact and outcome. And this is the data that we have shown and we just walked through a little earlier. So that is absolutely true. That is what you do see. And it seems better correlated than actually the tumor burden at the time of screening or inclusion because that obviously is somewhat arbitrary. It just happens to be when you actually see the patient, determine the level of tumor burden and that obviously could be at any level in the relapse. And so, at the time of bridging, obviously, that gives you much more relevant information. It's part of the therapy. It's always been part of the therapy in ALL. Even when you look at other therapies at times, if you have excessive tumor burden, you would actually first need to with a short course of chemotherapy to push down tumor burden [indiscernible] before you dose. So one of the things we have done during inside the development and certainly was important to also not only improve or have a chance for outcome in that approach, but also to reduce the risk for very severe adverse events. So, it is an integral part. And what we certainly will do is over time is probably look at different types of bridging and whether we might actually see differences there. So that's certainly a part of the analysis we're also still running through the trial, I think we actually post, I think, opportunities for also potentially investigating sponsored study [eventually] (ph).

Okay. And switching to autoimmunity, you mentioned that there are a lot of similarities between obe-cel and the German [ISD] (ph) assets. There are a couple of other companies out there who also have very similar programs to the German ISD. What would you say is a differentiator between your program and other programs? And what do you think is your key advantage?

Well, the first thing, I think, which is interesting is that it's not -- I think, is that we can make a statement to our product as it stands, how that may compare to others, I think, is very difficult because, for the most part, we don't have data to compare to. But we do have data to compare our product to the product that was used in Erlangen and that is relevant because that gives us a very clear understanding of the features that a product in Erlangen had versus ours and with that the predictability for outcome. I think that is relevant. And I think what we do see also compared to the Erlangen program is that as we have seen prior in the pediatric ALL patients is going to compare the CARPALL study to the ELIANA study from a safety perspective, there's a very significant difference there. Where we have none of the cases actual experience high-grade CRS, where compared to 47%. We have patients with high-grade CRS in the ELIANA study, virtualized were conducted within a very short period of time, actually overlapping each other as well. So, the same environment, same way of treating patients and managing safety, et cetera. So, we do know that we have a quite a significant difference there and that difference also was observed and was observable when comparing to the program that was running in Erlangen on the pediatric patients, which obviously was sort of prior to the work that we all know about on the obe-cel side.

All right. And maybe a last one on this topic. So, most discussions that we've had on the use of cell therapy and autoimmune diseases, suggest that only a relatively small window of deep B cell aplasia needed to "reset" the immune system. I mean, I guess that's one kind of hypothesis that is out there, but I'm sure you have maybe a different view on that.

Yeah. It's interesting. There's obviously a lot of hypotheses and that sort of gets me back to actually what we know works. And so what we know works is a product that is a long positioning product in pediatric ALL when used in autoimmune patients, which have a very active immune system as a consequence, the persistence, of course, is shorter than in highly uncompromised pediatric ALL patients. That you do see that, that product with that type of properties gives us the type of outcome we're seeing to suggest that a product that wouldn't actually be active in pediatric ALL would be able to do the -- get a significant outcome in -- of the immune patients, I think it's a postulate you can set up, but certainly, there's no data that would support that at this point. So, what we know is that a long persisting CAR T program, it gives you the right outcome that we were looking for. We know our product has exactly those properties. And I think everything else, people will have to actually run trials and actually figure that out. But it is not, I think, very easily understandable for why you would make that correlation if you start with a product that is not -- may not be really active or substantially active in pediatric ALL and then conclude that it would still work in autoimmune. It may, but I think at this point, it's sort of a statement in the absence of data.

All right. Thanks for taking our questions.

Thanks a lot, Gil.

Our next question comes from Eric Yeung with William Blair. Your line is open.

Hi. Eric on for Matt Phipps. I was wondering, firstly, with the additional cash on the balance sheet, I was wondering how you're thinking about obe-cel development in other lymphoma indications. And if you plan on enrolling any additional patients within NHL or CLL?

Thanks, Eric, for joining. So, we are currently looking at quite a range of indications and we're sort of looking at where we want to actually put our bets down. And we're clear we're going to have at least one pivotal study in autoimmune. And we're looking whether -- where there would be a second autoimmune pivotal study or whether we're going to be running, a, oncology study in one of the non-Hodgkin's indications. And that's currently actually under evaluation, that's not yet decided. But we're probably going to generate some additional data also for obe-cel in the non-Hodgkin's indication to sort of round out the experience that we have made so far.

Great. And then just an additional question. So, I know you've had to do next-generation sequencing on patients for enrollment in AUTO4 and AUTO5 and that paper you guys published in the [Blood Journal Cancer] (ph) on the TRBC1 and 2 staining, I was wondering if you think that paper could be supportive of a potential companion diagnostic for AUTO4 and 5?

It's a really good question. And that's obviously the reason why we've been working together with the parties we had on the paper. That obviously, if you can move away from NGS and go to an antibody that you can actually use and with classical staining, that obviously would simplify the approach and probably also accelerate it, because obviously, if you go with NGS, you actually you need to have the sequences, you need have the right primers, et cetera, specific and then actually run the analysis, it's not trivial. This could be easier, and it also would actually allow you to include the staining procedures in a more standard panel of stains that we'd be using to characterize tumors. It may actually lead to already a determination of TRBC status in patients ahead of even having -- basically include them in a therapeutic approach. We have it like you have CD19 and other markers or standard markers to analyze, have them actually move also in that direction. And of course, as you point out, it gives you the opportunity for companion diagnostic based on staining procedures.

Great. Thank you.

Thanks a lot, Eric.

Thank you. Our next question comes from Yanan Zhu with Wells Fargo. Your line is open.

Great. Thanks for taking our questions. First on SLE, I know you mentioned you're not guiding for dosing or not dosing. I'm curious about the level of interest at your first clinical site. And also, regarding your guidance for data late 2024, I was wondering about the number of patients at the time of readout and the duration of follow-up. Do you have a minimal kind of duration for follow-up? Thanks.

Thanks, Yanan, and thanks for joining. So first of all, the study we're conducting in the U.K. and in Spain. In the U.K., there are actually no competing studies for these types of patients. So that puts us in a good position and one where we think actually we have obviously, a very sort of interesting standout feature around the study and opportunity for patients. In terms of data end of the year, we obviously are expecting that we can involve the six patients, and we are able to report on those six patients, how much follow-up we'll have of them. I think that's premature to sort of point to. But the goal would be to actually have the cohort enrolled and then report on data from that cohort.

Great. Thanks for those color. On FELIX, wondering for the mid-year data update. Would the data be a pooled analysis? Or would it be the pivotal cohort? Also, since you commented on your real-world bridging therapy strategy, I was wondering, does that have any implication for FDA review. Thanks.

Yeah, really good question. So, in terms of the data update, obviously, as I indicated, we're looking, in particular, at different risk groups within the data set, so that's going to be a key focus. And I think we're going to be -- obviously, the general update will be likely across the entire study because from a physicians' perspective, that outlook is actually what's really relevant because that reflects the patients that you would actually see in actual practice. And so, we're probably going to report on that as a reference point referencing back to the ASH data. The bridging itself, obviously, is relevant in the sense that the analysis that you can run is sort of in two different ways and different agencies take different positions on it. You have to look at the analysis based on the tumor burden that's screening, which is basically, frankly, at the time point where any physician can look at the patients and you focus on the patients that have more than 5% tumor burden. Those are the morphological patients that's the intent to treat. That's certainly the position or the primary focus you'll see with the European agency as of the primary view, which is to the physician's view. This is when the physician can make a decision, okay, this is the patient that gets included, what is the outcome related to that. And then, when you look at the review process for Blincyto as well as for Tecartus, the focus the FDA has is more on the patients that actually have 5% tumor burden at the time point of dosing. And so that would be 5% at lymphodepletion, and it would be that category of patient, which is the primary group for assessment. So, the difference is and some different views that are taken in terms of analyzing data, and it really depends on the agency. But that's the fundamental difference on what we're seeing based on prior review history in the space.

Great. Appreciate the color. Thank you.

Thanks a lot. All right. If we have no more -- oh, sorry. Please go ahead.

Thank you. Our next question comes from Kelly Shi with Jefferies. Your line is open.

Hi. This is Dev on for Kelly. I have a couple of questions. One is you did analysis on obe-cel and German product, which is similar. Can you talk about what kind of patient baseline you're thinking about enrolling in your study? And for data presentation, are you thinking of presenting at that medical conference?

Yeah. So, when we look at the patients that we're enrolling in the study, we're rolling patients that have obviously our lupus patients that have or do not have the kidney involvement. So both manifestations, we do see. We do have patients as that's done in the Erlangen study that will have at least one organ involvement in these patients. So, it's very much track alongside what you have seen and read about in the publications from the Erlangen team. So, it's a very comparable patient population that we're enrolling in terms of inclusion/exclusion criteria. The plan would be for us to see that we can have our first data at one of the medical conferences at the end of the year. So that would be the plan.

Great. Thank you.

Thank you very much.

Thank you. Our next question comes from Jacob Mekhael with KBC Securities. Your line is open.

Hi there, and thanks for taking my question. I have a few if that's okay. First, I have a question on the option agreement with BioNTech on AUTO1/22 and AUTO6NG. What do you need to show or reach with those programs to trigger and opt-in from BioNTech? That's my first question. And then perhaps more, a very broad question on autoimmunity here. Given the larger patient population we're talking about, from your point of view, what needs to happen in the CAR T ecosystem to ensure that if approved, those treatments -- or there is enough capacity in the ecosystem to ensure that those patients have access to those treatments? Thank you.

Thanks, Jacob. I think two very good questions. So first of all, with regards to the option agreements on AUTO1/22 and AUTO6NG with BioNTech, the option really is structured such that the options have to be exercised before we start our pivotal studies with those programs. So, it's actually triggered by progression, it's not defined by a defined outcome or a defined certain level of activity. It's the actual decision to move forward into pivotal study. So that's the latest time point for the exercise. It could happen before, but that's the latest time point. So that's the sort of the option exercise question. In terms of the breadth with regards to autoimmune indications, I think the first observation is that we expect that the initial application for CAR T will really be in what you -- often refer to as the more refractory type of population in those indications. So that's intrinsically a small -- a relatively small part of the overall autoimmune indication that you'd be looking at. So, you have the overall lupus population, you may look at a few hundred thousand patients, but actually will be expect to go in and to sort of be amenable for a CAR T approach that may be in the few thousands. So, it is a smaller subset. And we expect that to be true also with some of the other indications. So from that perspective and moving into this disease setting, we would expect that capacity actually and the ability to serve is doable and will be there. One of the questions will be ultimately with some of the other indications is, how overall you would actually be able to go into these indications and have an adequate value proposition for these patients. And I think that's something we still need to learn on how far into these earlier stages or less severe stages of disease, is it sensible to actually bring in a therapy like a CAR T therapy. And I think that's something we'll be frank, that we still need to figure out and we need to generate data in the field, to get a better understanding of sort of where the right place and how broad that position can be. The initial positioning, we believe, is very well servable with the current types of infrastructures and technologies.

All right. Thank you very much. Thank you. Appreciate it.

Thank you. That's all the time we have for questions. I'd like to turn the call back over to Christian Itin for closing remarks.

Well, thank you very much for joining today. Obviously, great to have an opportunity to really review, I think, all the progress we've been able to go through the course of last year and into this year. A lot more to come this year. We appreciate the continued support and interest, and wish you all a great upcoming period and looking forward to connecting in person again. Thank you.

Thank you for your participation. This does conclude the program. You may now disconnect. Everyone, have a great day.