Hello, ladies and gentlemen, and welcome to the Autolus Therapeutics Third Quarter 2023 Financial Results Conference Call and Business Update. At this time, all participants are in listen-only mode. After the speakers’ presentation, there will be a question-and-answer session. [Operator Instructions] Please be advised that today's conference is being recorded. I would now like to turn the conference over to your host, Julia Wilson. Please go ahead.

Thank you, and good morning or good afternoon, everyone, and thank you for joining us to take part in today's call for Autolus' third quarter 2023 financial results and business update. I'm Julia Wilson, Communications Consultant for Autolus. With me today are Dr. Christian Itin, our Chief Executive Officer; and Rob Dolski, our Chief Financial Officer. Before we begin, I would 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/or regulatory time lines 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 will see the agenda for today's call, which is as follows: Christian will provide an overview of our operational highlights for the third quarter of 2023. Rob will then discuss the company's third quarter 2023 financial results, before Christian will conclude with upcoming milestones and closing remarks. Finally, we will, of course, welcome your questions. Over to you, Christian.

Thank you, Julia, and good morning to you all. Thank you for joining us. It's my pleasure to review our progress for the third quarter in 2023. Moving to Slide number 4. We'll give you here a quick summary of the progress we've made with obe-cel during the third quarter. Key focus has been on preparing the BLA filing, which is on track for a filing by the end of the year. Building on the data we presented at ASCO and EHA early in the year for our pivotal FELIX study in adult patients with relapsed/refractory acute lymphoblastic leukemia, we look forward to presenting longer follow-up and subgroup analysis at ASH in December. The ASH abstract embargo has just lifted and we have an oral presentation giving longer follow-up data from the FELIX study on Saturday, December 9. In addition, we will, in a poster presentation, provide long-term follow-up data from our prior ALLCAR19 study in the FELIX 1b study on both ALLCAR19 and FELIX 1b, we're approaching up to five years and up to three years of follow-up, respectively. I'd also like to highlight that before the end of the year, we'll be initiating a pediatric Phase 1 study of obe-cel to support our filing with the European Medicines Agency. Which is expected to start in the first half of – sorry, will be filed in the first half of 2024. Critical for the successful outcome of the FELIX study was our robust product delivery platform with high manufacturing success rate. Short range to delivery time and high percentage of patients dosed with obe-cel. During the third quarter, we successfully completed the process performance qualification at the Nucleus manufacturing facility set up for commercial supply and are on track to support a biologics license application with the FDA by…

Thanks, Christian, and good morning or afternoon to everyone. It’s my pleasure to review our financial results for the third quarter ended September 30, 2023. Our cash and cash equivalents at September 30 totaled $256.4 million, as compared to $382.4 million in December 31, 2022. Total operating expenses net for the three months ended September 30 were $47.8 million, as compared to $43.5 million for the same period in 2022. Our research and development expenses decreased by $0.4 million to $37.2 million for the three months ended September 30 compared to the same period in 2022. This was primarily due to decreases in clinical trial and supply costs related to our obe-cel clinical program, and these were partially offset by increases in operating costs related to our new manufacturing facility, as well as salaries and employee related costs driven by increased headcount. On the G&A side, general and administrative expenses increased by $2.4 million to $10.6 million for the three months ended September 30 compared to the same period in 2022. This increase was primarily due to salaries and other employee related costs, driven by an increase in general administrative headcount supporting the overall business growth primarily related to pre-commercialization activities. Our net loss attributable to ordinary shareholders was $45.8 million for the three months ended September 30, 2023, compared to $42.8 million for the same period in 2022. Autolus estimates that our current cash and cash equivalents on hand and anticipated future milestone payment from Blackstone will extend the company’s cash runway into 2025. And just as a reminder, in our projections, we do anticipate an R&D tax refund from the UK HMRC in Q4 of this year, and would anticipate being eligible for a tax refund in a similar fashion next year as well. I’ll now hand back to Christian to wrap up with a brief outlook on expected milestones. Christian?

Thank you, Rob. And so we’re moving to Slide 21. This is a quick summary of kind of the planned news flow. And as you can see, there’s a lot of activity coming up towards the end of the year and into the early part of next year. As mentioned before, big item of focus and clearly where most of the organization is working towards is get the BLA filing in before the end of the year. We obviously talked about the obe-cel FELIX data update as well as the updates around ALLCAR19 and the FELIX 1b study, which gives us longer-term information and I think a very nice proxy for in terms of what to expect in terms of longer-term outcome from the larger pivotal FELIX study. Then obviously we’ll have the update on AUTO8, which is a program that is shaping up very nicely and I think will give us sort of a first interesting look at the profile of that program. And then as mentioned just before the start of the AUTO6NG Phase 1 trial in children with neuroblastoma, which we expect to actually get the first child in before the year end. Importantly, as we go into early next year, we expect to get the Phase 1 up and running and the first patient in, in the early part of next year. Treating patients with autoimmune disease, particularly focus obviously here is SLE in this initial study and the study is designed to confirm the dose and with that then be able to have a basis for the regulatory discussions around the pivotal study design. And then as indicated also in the earlier part of next year, we’re expecting to get the filing in Europe, the MAA application with the European Agency as well. So a lot of activity in the upcoming month, I think a lot of very significant value steps for the company. I think we’re very well set up. And we have a fantastic team also on the – now building up on the commercial side that I think will put us in a very strong position to get to the next steps once we’re through the actual interactions on the regulatory side. So a lot of good stuff ahead of us, really looking forward to seeing many of you probably at ASH and happy to take questions now.

[Operator Instructions] Our first question comes from the line of Matthew Phipps of William Blair. Your line is now open.

Hi. Thanks for taking my questions and all the updates. I guess first on ASH, will the obe-cel FELIX update, did you think of sufficient follow-up to really show EFS curves at ASH? And then for AUTO8, another six IO patients were those just from the BCMA alone CAR in cohort 1, or were there some of those with cohort 2 that had that CD19 as well? And I guess maybe how many patients do you expect from cohort 2 at ASH?

Yes. Very good questions. Thanks for joining, Matt. So first question is, are we expecting to update or present event free survival data? And the answer is yes, on the FELIX study. And then with regards to the AUTO8 program, what Matt was referring to is that we started the Phase 1 clinical study dosing patients with the BCMA CAR alone to establish the base profile for the BCMA CAR and then actually after that was initially established added also the CD19 CAR component to the product. And we’re actually going to have data from both patients with BCMA only as well as with both CARs present. And we’re at a point where we’re probably going to be in the range of about 10 patients, maybe slightly above that.

And if I can ask just one other one on the autoimmune side again. Can you remind me – I can’t remember if this was discussed last week. But is there a safety kind of DLT period like in between patients for the initial, like you have to wait a month before you can treat a subsequent patient or anything like that.

We would expect that there is a gap between probably the initial patients and then as soon as you actually have initial data, you can then actually enroll at that level. Obviously, we do have quite a number – quite a good level of data at this level of dose across particularly the ALL population. So there’s a lot safety data already in place, so we should be able to enroll relatively quickly.

Great. Thanks for taking my questions.

Thanks, Matt.

One moment for our next question. Our next question comes from the line of Dev Prasad of Jefferies. Your line is now open.

Hi. Thank you for taking our question. This is Dev on for Kelly at Jefferies. So I have a quick question on SLE. How do you think about the cell persistence that can have an impact on efficacy and safety in contrast to the oncology indications that we have seen so far?

Hi, Dev. Very good question. And so as mentioned before, what you look to sort of achieve is a very deep cut into the compartment to remove both the autoreactive precursor cells and memory cells, as well as the autoreactive plasma cells. So deep cut is what you need given that this is a cell based therapy that will take some time to be effective and complete. So you will need a certain level of persistence to be able to do that. What we’ve seen and what I think is very encouraging is that the program in Erlangen showed very nicely that they did get a very deep response. They got obviously a good transition in terms of translation from a clinical efficacy perspective. And when you look at the persistence of the CAR T cells, you see that the CAR T cells are pretty much around for about six months. And after that you do start to see the B cell compartment regenerate. You see the maturation of B cells from the various subtypes – antibody subtypes perspective, but what you do not see is actually a recurrence of autoreactivity. So there is a certain amount of persistence that clearly will be important. What we do see is that our product, and in fact the product that was used in Erlangen, in pediatric ALL patients, has quite very similar behavior in terms of persistence. Also in their hands they do see that they have children with long-term persistence. We’re talking two years. That’s at least the current experience, and that is similar to what we have seen with obe-cel. And so we think actually that the profile should match very nicely.

Thank you for taking our question.

Thank you.

One moment for our next question. Our next question comes from the line of Gil Blum of Needham and Company. Your line is now open.

Good morning and good afternoon and thanks for taking our questions. So a couple of items from us. First of all, for AUTO8, it looks like the data is really interesting, but it’s a super crowded market and there’s a lot of assets going into it. What do you think is going to be a key differentiator, maybe on the angle of safety?

First of all, thanks Gil for joining. Very interesting question. And I think one that that we sort of see actually quite a bit of an evolution in the field as well. So in order for what we’re designed the product for is to be able to get, on the one hand, deep responses. So we want to actually see that indeed we can get to very low levels of disease burden in these patients. And obviously you can measure in multi myeloma, similar to ALL you can measure minimal residual disease levels. And that gives you one parameter I think that we’re clearly very interested in looking at and pursuing. The second aspect was a bit of a conundrum, I think, from – in the field, in the sense that what we’re seeing with regards to cell dynamics, CAR T cell dynamics in multi myeloma patients is that the CAR T cells tend to actually not persist well. And so one of the things we wanted to achieve is see that indeed we can actually get a longer-term persistence through the CD19 CAR component with the basic rationale that you do have, obviously new CD19 positive cells being formed that can actually help drive a certain level of baseline activation and maintain that for the CAR T cells. The other aspect, the third aspect I think goes actually links quite nicely with the conversation we just had on the autoimmune side, which is that there is at an early stage of the plasma cell compartment is CD19 positive. And certainly it’s been sort of postulated for probably more than 25 years then that the driver cells from multi myeloma might in fact have a CD19 expression. So what it also will allow us to do, the combination is to obviously get into that part of the compartment and may actually also help us get at some of the driver cells. So that’s sort of the three hypotheses. In terms of the data what we’re looking for is want to see a high level of responses in these patients. We want to see whether these responses get deep over time, usually depending on what you need to score for, can take a bit of time to develop fully. And secondly, we want to see the cellular dynamics of the products in the patients as a consequence of adding the CD19 component to the product. So those are probably the key things. And I think once we have that data in and with a reasonable number of patients backing it, I think that’s been the time for us to sort of think about the approach that would be suitable for the development for the program going forward. But in terms of the hurdle for development, clearly you need to have an exceptional profile of the product to develop that in multi myeloma. So that’s sort of where we are and our current thinking is.

Makes sense. I do want to go back to a point that you made during the SLE event. So why does the company believe that other commercial stage CAR T’s would need to maybe redo their production and do more clinical studies in order to move into SLE despite the fact that they already have commercial stage assets?

That’s a really good question. And I think what we’re seeing with two programs that are being moved forward is that the companies decided to massively change their manufacturing. So in one case it’s moving from a manual, very lengthy manufacturing process to a highly automated, probably two day process. That’s a massive change in terms of the actual manufacturing process. It will lead to, frankly, different products. And particularly if you go to this very early stage or very short manufacturing process, you run the additional issue that at that point in time, the CARs, the chimeric antigen receptors are not yet expressed properly on the surface. And with that, the cells are not yet functional, biologically functional from a potency perspective or an ability to kill other cells perspective. So it creates clearly a very, very different type of product, different set of product properties. And with that you have a new product and that means that you have to obviously redo or generate new clinical safety data, et cetera, build that database. And from a commercial manufacturing base, it’s a fundamentally different setup from the manual setup, which requires, as an example, a high grade clean room environment versus the semi or the automated platform as we use it, where you basically go to a low grade clean room environment. So it’s a very different environment to be optimal. The way you have the flows in a facility and be optimal for that from a cost perspective looks different. And we would expect it will require pretty significant investments to actually change or build new manufacturing facilities to sort of accommodate this very different manufacturing process. The second company in this space also has made very significant changes, calls the product differently as well in terms of its coding. And we do not believe that the product will be comparable. And there’s also going to be certainly some level of adjustment that will have to be made to the commercial manufacturing infrastructure. To adjust to the new process and trying to be effective with a decision with it. So those are probably some of the key considerations. What they obviously do have is the base set of systems for commercialization to a cell orchestration and so on. That's obviously straight, usable and can be straight applied. So there is clearly an advantage there of being able to rely on that as we will rely on the systems we're setting up now for ALM.

All right, thanks for taking my questions.

Thanks a lot, Gil.

One moment for our next question. Our next question comes from the line of Yanan Zhu of Wells Fargo. Your line is now open.

Hi, thanks. Thanks for taking our questions. I have a couple for the SLE and a couple for FELIX. Perhaps let's start with the SLE. I was wondering, Christian, you were talking about the benefit of us having – one of the benefits of having the same product as your other soon-to-be commercial product is that may be discharged of some of the safety database size requirements in your new indication. I was just wondering is that something that you have had some preliminary agreements with the agencies on? Or is that your general expectation? Also, I was wondering if you have any thoughts on Novartis' YTB323 abstracts at ACR? Any comment on their dose level early efficacy and also early safety? And how – does that have any implication for your confidence for obe-cel in SLE? And I'll ask the FELIX question afterwards. Thanks.

Okay. Well, thanks, Yanan, for joining. Really good questions. So first off, on the safety. Also, this is based on sort of the regulatory guidance you have in terms of the general size of safety databases to get after discharge, the respective level of risk. And that's actually consistent when you look at the various indications and the way you can actually use information gained in one indication applied to another for as long as you're working with the same product. So this is not specific guidance. This is the general guidance that you have in terms of safety requirements and discharging safety business. The second aspect – a second part of the question was related to the abstract from the Novartis' program, which I think things look good. I think it's very encouraging because overall, it looks like they were able to replicate the data from the airline and team. I think that's great news. I think for the active, for the approaches, that we're working on, and particularly also for the approach we're working on. In terms of the dose, the dose is relatively low, and that is actually, I think, quite consistent with our own experience where we have been able to obviously show in adult patients, we can go as low as 10 million cells, and we get high degrees of activity and responses. So we know that the cells are active at that level, and they're operating at a somewhat lower level that was used to the airline. But again, it's a different product that they're using. So there's going to be some differences. And it may also be guided and to some extent from needing sub-safety data at the respective dose levels. So I think overall, I think, very encouraging and I think good news because we start to get, I think, a clear view that the data can be replicated that the airline have generated, I think that's frankly good news for everyone in the field.

Great. Thanks. So on FELIX and the ASH update, I was wondering, is the data cutoff for the abstract which we are seeing now, would that be the same as the actual presentation? And also, I think we noted the median DOR is now stated as not reached. I think back at ASCO, there was a median DOR. So it feels like the curve has probably fluctuated around the median and was bumped up somewhat with newer patients and additional patients or longer follow-up. I was just wondering if that the right understanding? And also if you can comment on how much it was bumped up, that will be super helpful. Thank you for the question.

Yes. So in terms of the data cut, obviously, the data cut that we're going to have at the – for the ASH meeting will give us about the six months additional view on the patients. If any case that the median follow-up at ASH was nine months, we expect the median follow-up – at ASCO was nine months, we expect the median follow-up for ASH to be at around 15 months. So it's essentially the data cut. And it's also different from the abstract, the data cut that went through to the abstract. I think one of the things that we're pointing out in the context of the ASCO data, in the ASCO presentation was the fact that the time-dependent measures clearly were not mature by any stretch of the imagination. We have most of the patients still in the very early part of the curves when we presented at ASCO, which means that there's still a significant amount of variability that we had in the curve, and we're seeing obviously some of that play out. And usually, it's one patient up or down that can move the curve, particularly if you're focused on the median and that's almost arbitrary. So you need to – clearly, we're going to have more follow-ups, six additional months of follow-up that obviously will give us more stability into the curve. But clearly, compared to where we were at ASCO, was predominantly driven by the fact that most of the data were still very early.

Great, very helpful. Thank you, Christian.

All right, thank you very much.

One moment of our next question. Our next question comes from the line of Asthika Goonewardene of Truist. Your line is now open.

Hi, this is Karina for Asthika, congrats on the progress. I had a question on – a few questions on AUTO8. So what kind of data can we expect at ASH? Will this have like baseline characteristics and percentage of patients with EMD and other high-risk agnostic factors? And I think you said it's going to be eight patients worth of data at ASH, can you just confirm that?

So the data, obviously, this is a Phase 1 study. So yes, we'll obviously, we'll have a review of the patients that were included, the state they were in, condition they were in and we'll also give a full characterization of those patients on the treatment. Overall, we think we're probably going to be in the range of 10-plus patients as mentioned before, so this is still as indicated, a Phase 1 study.

Okay. And will there be enough follow-up for six months, nine month PFS?

No, that's – well, there is some follow-up, but I think we need more patients and more follow-up to sort of be firm around those types of information.

Okay, thank you.

Thank you, Karina.

One moment for our next question. Our next question comes from the line of Simon Baker of Redburn Atlantic. Your line is now open.

Thank you for taking my questions. Two, if I may, please. Firstly, on SLE. As you say, you have an advantage over most, if not all, competitors in the space in terms of the size of the safety database and the suitability of the manufacturing process. So I'm just wondering if you could give us some idea of how much in terms of time that advantage is or alternatively, and I suspect it probably won't, but some more idea on when we should think about pivotal trial starting and potential commercialization, should it be successful? And then secondly, on the manufacturing capacity and the Nucleus, could you give us an idea of the validated capacity here and now of Nucleus. I know it's a modular facility that can be expanded, but some idea of where – what the capacities at the moment will be very handy? Thanks so much.

All right. Well, thanks, Simon. Thanks for joining. On the SLE side, the question was when do we expect a pivotal trial to be rerun, obviously, that's slightly premature, but the basic notion is that we're planning to, the dose confirmation during the course of next year. With that data, we'll then approach the regulators, which would sort of point to the following year as the start point than the starting point for a pivotal study. I think it is too early to point to kind of what the duration of a trial, a pivotal trial will be in the absence of having an agreement on the trial design with the agency. So that's probably too early. . With regards to the capacity question at the Nucleus. So the facility is – basically, it's a modular setup, as indicated, it has, for the initial setup for ALL 3 clean rooms. Each one of those scaled for 700 products a year. And we're obviously going to be launching out of the first clean room. So that gives us an immediate 700 capacity. And that also will bring online the second and the third clean room because it doesn't make sense to operate clean rooms that are not being utilized from a capacity perspective yet. But the facility is fully built and obviously validated. But in terms of the actual ramp and how you run that is that you make sure that you have the right level of capacity at the right point in time. But the important thing is here is we have no limitation to actually ramp ahead of demand here. And I think that sets us apart from all the other launches that we've seen over the last few years.

All right, thanks so much.

Thank you, Simon.

This concludes our question-and-answer session. I would now like to turn it back to Christian Itin for closing remarks.

Well, first of all, thanks all for joining. Looking forward, obviously, to seeing you at ASH with the additional data updates. And for those of you who happen to be in London middle of November, we're looking to – or having a visit at the actual manufacturing facility. So if either on the analyst or investor side, there is interest, do visit the facility, please contact Susan Noonan, to join us for that visit. But you might find it interesting and helpful to sort of understand what the reality looks like on the CAR T therapy side and what you need to have in place to be able to launch. So with that, I'd like to thank you all for joining. I appreciate your time and speak to you soon. Thank you.

Thank you for your participation in today's conference. This does conclude the program. You may now disconnect.