Q4 2024 Autolus Therapeutics PLC Earnings Call
Hello, Ladies and gentlemen, and welcome to the ATA list Therapeutics call to discuss its full year 2024 financial results and business updates.
Speaker Change: As a reminder, this conference call is being recorded I would now like to turn the conference over to your host Amanda Gray. Please go ahead.
Amanda Gray: Thank you Latanya good morning, or good afternoon, everyone and thank you for joining us on today's call with me are Chief Executive Officer, Dr. Christian <unk>, and Chief Financial Officer, Rob Dulski.
Amanda Gray: I'd like to remind you that during today's call you will make statements related to our business that are forward looking under federal securities laws and the safe Harbor provision.
Amanda Gray: Provisions of the private Securities Litigation Reform Act of 1995.
Amanda Gray: These may include but are not limited to statements regarding the status of the ongoing commercial launch of a capsule in the U S. Ottawa.
Amanda Gray: <unk> manufacturing sales and marketing plans for a castle the market potential for our cattle and the status of clinical trials and development and our regulatory timelines for Ob salad and our other product candidates.
Amanda Gray: 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 and we assume no obligation to update any such forward looking statements.
Amanda Gray: 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 investors section of our website.
Amanda Gray: On slide three you'll see the agenda for today's call as usual Christian will provide an overview of our operational highlights.
Rob: Rob will then discuss the financial results.
Rob: Christian will conclude with upcoming milestones and closing remarks.
Christian: We'll then take your questions with that I'll turn it over to Christian.
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.
Christian: A few months now.
Christian: First of all I'd like to just highlight kind of the key objectives that we have for the year.
Christian: Primary objective clearly is to execute a successful commercial launch for a castle and we're doing really well and advancing well on that will provide a bit of an update as we go through the presentation.
Christian: But also we see a lot of opportunity for Ob sale to actually expand its utility the excellent profile that we see.
Christian: In our prior <unk> in our current experience with the product and explore the opportunity in additional indications as well as look at the opportunity for moving some of our other product candidates forward and at the same time, obviously it will be I think very selective and very careful in how are we going to place our investments now.
Christian: One of the things that you'll hear US also briefly mentioned is the fact that we're planning for an R&D event in April 2030 in New York that will actually outlined 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 slide on the next slide.
Christian: Briefly going to summarize kind of the key opportunities that we have seen and achieved during the course of 2024 as I mentioned this has been a remarkably successful year and we had a very strong I started the year in the first quarter, when we're able to add about $600 million jar balance sheet.
Christian: Through our collaboration with biotech as well as a public financing that we conducted in the first quarter. They set us up very well to really be able to focus fully on making sure that we get ob sell already and to get through the approval process in the U S. But also to set us up well.
Christian: For the commercial launch of the product.
Christian: As we're going through the.
Christian: The year I would say the we had continuous activities on the commercial side, you actually get the sites ready so that they actually would be in a position once the product was approved to actually start onboarding their products and and be in a position to move forward and actually use the product.
Christian: In the commercial space.
Christian: Now when we look at the approval itself, we achieved the approval in the U S on November eight.
Christian: And I think what was very encouraging was that it not only did we actually achieved approval slightly ahead of schedule, but also actually had a label, which gives us a broad opportunity in the relapsed refractory a L. L. A space for adult patients and not and actually having the first products getting through.
Christian: The approval process.
Christian: That actually did not require a rems program. So very nice setup in terms of the approval itself the timing of the approval not having a delay are recognizing that the product had an attractive safety profile and therefore no obligation for collecting additional information around.
Christian: Crs and icons.
Christian: Forward and that actually is I think a very very strong foundation.
Christian: What was then a very very helpful is that we were able to actually get a 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 <unk> guidelines.
Christian: 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.
Christian: And also will support the decisions that payers have to obviously go through.
Christian: To get the patient signed offer for commercial use.
Christian: Now when we look at the track that we had as we went through the first three months.
Christian: 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 were 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%.
Christian: The target patient population in the U S.
Christian: We have continued to move forward, adding additional centers as we go through this year by the end of the year expects to have approximately 60 centers that'd be ready and in a position to deliver a capsule to patients. We believe that those 60 centers actually reach the vast majority of the patients.
Christian: In the U S and will give us a very strong foundation.
Christian: For a successful delivery of the product to the patients.
Christian: Ah patients across the U S.
Christian: I already mentioned that we obviously had the publication of the study of the Felix studying their England Journal, but we're also in addition to I'll say getting.
Christian: Ready for launch in the U S. We're also moving through the regulatory steps in the UK and in Europe and had filed in the early part of 2024 with the image with EMA and then by middle of the year with an HRA and we expect actually rich regulatory decisions in the second half of 2025.
Christian: <unk>.
Christian: We also started the process to actually establish a ultimately the utility of the product in the U K and we're going through the assessments.
Christian: By the National Institute for Health and care excellent are nice, which is one of the key processes that you have to run through to get to a position.
Christian: To actually get a product adequately reimbursed in the U K.
As we went through the year there were several data presentations at key conferences are that really focused on the properties that we have observed with a capsule or b cell.
Christian: In the relapsed refractory adult population as we were going through and sort of actually gain more and more data from the Felix study.
Christian: We could report on the durability of the product highlighted.
Christian: The safety profile.
Christian: 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're also actually were able to show the impact of tumor burden guided dosing as well as the safety profiles impact reducing.
Christian: <unk> health economic cost.
Christian: 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 comprehensive during the course of last year, including the tandem meeting in February this year moved.
Christian: Moving to the next slide we're looking briefly here at some of the key outcomes that were actually.
Christian: 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 and the majority of the patients and that type of activity I would say has come with an attractive.
Christian: Safety profile that we can see that overall the level of high grade cytokine release syndrome, and the level of high grade neurological.
Christian: Toxicity or icons are low and in fact, when we looked at patients that have low levels of disease burden at the time of dosing those patients did not experience a high grade events, either crs or icons, which I think gives us a good understanding of.
Christian: Patient profiles 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.
Christian: As you can see in the top part of the panels are event free survival stabilizes.
Christian: Between 45, and 50% and we see that 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 our actually have events that are occurring.
Christian: When we look at what the impact is of the disease burden prior to dosing, we can see their patients that have low disease burden of less than 5% to remarkably well, whereas most patients with a wide range of disease burden up to 75% actually continued to do very well and only the patients that had extremely high levels of disease burden.
Christian: More than 75% of two of blasts in the bone marrow at the time of dosing obviously, they 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 the I'll say the efficacy for many.
Christian: And 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.
Christian: Picture again stabilization of overall survival.
Christian: 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 lengths of depletion.
Christian: And obviously patient 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 in this field.
Christian: Moving to the next slide as we sort of thinking about sort of the early momentum off the launch in the U S.
Christian: We are now seeing indicators here as the centers that can actually actively deliver product to patients.
Christian: As I mentioned, we reached 33 centers.
Christian: 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.
Christian: More densely parts of that are populated parts of the U S. A which is also the reason viable 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 our lives covered and we see actually very good momentum there.
Christian: Well as we go through the App have been going through the last three months. So that is very encouraging and also actually is a good sign that indeed pay.
Christian: Patients do half a billion opportunity here too.
Christian: Get access to this therapeutic option.
Christian: Finally on the next slides on the update you have heard us actually talk quite a bit about the actual manufacturing facility for commercial supply, which is the new cliffs facility.
Christian: I think one of the remarkable things about this field is obviously as you have the first commercial patient coming that's when you literally start the operation at that facility and I think at this point, we can say that we.
Christian: We had we're off to a really good start and the facility came to life and it's humming well and I think.
Christian: It will be a very strong give us a very strong foundation in our ability to reliably deliver product.
Christian: To the centers.
Christian: 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.
Christian: First off on the autoimmune side, we've been running obviously the carlyle's study we have.
Christian: The initial cohort that we.
Christian: Oh six patients that are all dosed, we're running through the data cut.
Christian: For the upcoming R&D event on April 23rd.
Christian: And we're looking forward to updating you on that initial experience with the first six patients and.
Christian: We're also are planning to present, the data with long term follow up.
Christian: Across the patients at a later time point in the second half of the year on the hematology side. Obviously, we're also have been moving forward with our pediatric study the <unk> study and.
Christian: And we expect to provide an update also second half of the year that study is also it's been enrolling very nicely.
Christian: In terms of the early pipeline, we obviously have a set of activities ongoing.
Christian: With our partners at University College in London.
Christian: And we are moving.
Christian: Moving forward or have been active in.
Christian: <unk> 22, Autosave century, and auto eight number continued to progress.
Christian: The activities around those programs are collecting more information.
Christian: The programs and are planning to also give a short update at the R&D day at the.
Christian: And our four towards the end of April.
Rob: So with that I'd like to actually hand over to Rob and give us a summary of the financial results.
Rob: Thanks, Christian and good morning, or good afternoon, everyone. It's my pleasure to review our financial results for the full year 2024.
Rob: Cash cash equivalence and marketable securities at year end 2024 totaled.
Rob: <unk> totaled $588 million as compared to $239 million.
Rob: At December 31, 223 time frame.
Rob: This increase was primarily a result of our strategic collaboration with biotech.
Rob: And concurrent equity financing for a combined $600 million in gross proceeds.
To bolster the balance sheet ahead of our U S commercial launch for a capsule.
Rob: Loss from operations for the year ending December 31, 2024 was $241 4 million as compared to $179 7 million for the year ended 2023.
Rob: Cost of sales totaled 11.4 million following the BLA approval for Ob cell.
Rob: This amount represents the cost of commercially available plant capacity that can no longer be classified as R&D expense as of November 8th Ob South approval date.
Rob: Even though it was not associated with product sales in the fourth quarter.
Rob: Our research and development expenses increased from 138 increased to $138 4 million for the year ended December 31, 2024 compared to $135 million in the same period 2023.
Rob: This change was primarily driven by increases in employee salaries related costs manufacturing costs related to Ob cell and then partly offset by some decreases in professional fees and facility costs.
Rob: Our selling general and administrative expenses increased to $101 1 million for the year compared to $46 seven for the same period in 2020 for 2023.
Rob: This increase was primarily due to the salaries and other employee related costs driven by the overall increase in head count supporting commercialization activities.
Rob: And finally net loss was $220 7 million for the year ended December 31 2024.
Rob: Compared to $208 4 million for the same period in 2023.
Rob: With the recent approval of a cat fall in the U S. I'd also like to.
Rob: To note two financial milestones that were triggered in the fourth quarter.
Rob: As a result of the FDA approval <unk> received a 30 million dollar milestone payment from Blackstone based on the terms of our previously announced Blackstone collaboration agreement.
Rob: In addition, the company made a regulatory milestone payment of 10 million pound Sterling in accordance with our U C. L. B license agreement.
Rob: These are both detailed.
Rob: More specifically in our 10-K filing.
Rob: We continue to believe that with our current cash cash equivalents and marketable securities. We are well capitalized to drive the full launch and commercialization of Ob cell and relapse.
Rob: Refractory adult E L F.
Rob: As well as to advance our pipeline development plans, which includes adequate runway to reaching data in the first pivotal study of Ob sell in autoimmune disease.
Rob: And as Christian noted, we look forward to providing further detail on these plans at our upcoming R&D event.
Christian: I'll now hand back to Christian to wrap up with a brief outlook unexpected milestones Christian.
Christian: Thank you very much.
Christian: Moving to the upcoming milestones so when we look into 2025.
Christian: We are starting the year with obviously the update on the SL one.
Christian: Carlyle's study.
Christian: Which we are planning to provide on April 23rd at the R&D Company R&D event in New York.
Christian: We then when we look at the pediatric study expected to provide the update on the pediatric study second half of the year as well as obviously longer term follow up on the Carlisle study as well second half of the year and we do expect also during the second half that we received the notifications are ranked the regulatory process.
Christian: For the U K as well as for the EU regarding potential approvals in those two <unk> jurisdictions for Ob sale.
Christian: I think a very interesting year for us in terms of the progression of opportunity that we actually starting to execute on where outline at the company R&D day, but and also obviously, we will be able to provide more and more evidence and protections as well with regards to the.
The launch progress that we're making for a capsule in the U S.
Christian: So with that we're happy to take questions.
Christian: We will take over and over to the operator. Thank you certainly as a reminder to ask a question. Please press star one on your telephone and wait for your name to be announced to withdraw. Your question. Please press star one again and please standby while we compile the Q&A roster one moment for your first question.
Speaker Change: Our first question will be coming from James Sheehan of Deutsche Bank. Your line is open James.
James Sheehan: Hey, good morning, guys. Thank you for the question.
James Sheehan: On capital Wash and I know, it's a progressively evolving situation.
James Sheehan: Can you provide any color on the initial demand.
James Sheehan: Orders, thus far and then secondly.
James Sheehan: Oncologists have mentioned that the split dosing and the delayed onset of Crs oral capsule can be useful for outpatient ministration can you say, whether outpatient use is happening plan thus far.
James Sheehan: And I'll just leave it there.
James Sheehan: Yes, well first of all thanks, a lot for joining James.
James Sheehan: The launch I think what we can say is that we have obviously seen a very nice dynamic around the actual activation of the centers, which are really critical and it's very much driven by the level of interest enthusiasm by the centers to actually do that work and actually and get the product.
James Sheehan: Get the product access.
James Sheehan: That also is often driven by actually having patients that are in need of therapy.
Sort of usually actually the key driver in that in that process. So I think that gives us gives us a very good level of confidence in terms of.
James Sheehan: The how the product is viewed the level of interest and I think the dynamic we've been seeing it's been very encouraging obviously would provide.
James Sheehan: Obviously, a fuller picture at the Q1 update but I think what we can say is that we see a very good dynamic and I think.
James Sheehan: A very significant level of interest and I think a recognition that the product has attractive properties for these patients with regards to the outpatient question I think there is an opportunity as you've seen from the safety data that I highlighted during the presentation is that.
James Sheehan: Ah patients that have very low levels of tumor burden Midland depletion tend to have a very limited amount of hematological.
James Sheehan: Safety events and I think that is certainly attractive and has led to a number of physicians to suggest that they might be interested to actually explore.
James Sheehan: Also bleed tube administered product instead of a hospital outpatient setting will need to see how that evolves to a lot of that has to do with the actual experience gained with the product on the commercial setting of the confidence physicians are building up and I think we will see that they will also with the upcoming of the upcoming periods I don't think at this point in time I think we can.
James Sheehan: Guide you in any way at this point I think it's too early in the launch, but it's clearly an observation that a number of the physicians certainly has made.
James Sheehan: It will be interesting to sort of follow that as we go through the year.
James Sheehan: Thank you.
Speaker Change: One moment partners.
Speaker Change: And one moment our next question.
Speaker Change: Our next question will be coming from Rajiv Sharma of Goldman Sachs. Your line is open.
Speaker Change: Hi.
Rajiv Sharma: Thanks for taking my questions just a couple on logistics actually site.
Speaker Change: Obviously, you have the target to have 60 authorized centers.
Rajiv Sharma: By the end of the year.
Speaker Change: Interested in Nebraska to get to that from where you are right now should we expect that to be linear or are there kind of.
Speaker Change: Could this potentially come in and blocks of centers coming online and then the second one just on Antara.
Speaker Change: You've been a focus for investors and the fact that given the headlines are saying.
Speaker Change: Could you maybe just talk to how you think that could potentially impact given the manufacturing in the UK. Thank you.
Speaker Change: Yeah. Thanks, a lot for joining so the question. The first question is related to the ramp up of the centers getting online.
Speaker Change: I think we've seen in the first 30 centers I think are pretty steady.
Speaker Change: So that trajectory.
Speaker Change: Of these centers actually getting online, which I think has been.
Speaker Change: Expect it because we do have a we didn't have the 30 centers prepared for Onboarding by the time of approval and then that's the activation step that we're running through and you could see that basically that we had early January as we have reported around 20 centers that were active and then that obviously built in through the quarter to middle of March.
Speaker Change: Goodbye thirty-three so is it pretty continuous kind of flow of movement that we've seen where the next 30 centers I think we are.
Speaker Change: We'll see that.
I think build gradually as we go through the year.
Speaker Change: Obviously very much a lot of that is really driven by the individual centers and the the time.
Speaker Change: The speed at which the Onboarding process is sort of conducted a lot of that has to do with legal reviews and so on.
Speaker Change: <unk>.
Speaker Change: With the capacity of the respective hospitals to sort of manage that that workload. So there is an element of variability, but we would assume that we have set a reasonably steady process as we go through the year and we obviously will be able to keep updating you on a quarter by quarter basis and also the actual.
Speaker Change: Centers, obviously are visible.
Speaker Change: For physicians and patients.
Speaker Change: On the <unk> website. So it is something that can actually be followed.
Speaker Change: But in.
Speaker Change: In terms of projection, we would expect that to go relatively steady as we go through the course of the year.
Speaker Change: With regards to tariffs I think very much an open question at this point in time.
Speaker Change: Whether there will be tariffs what it might be what types of products might be impacted et cetera, I think very hard to speculate.
Speaker Change: In most other.
Speaker Change: I think in the past if we look at Paris.
Speaker Change: Imposed on pharmaceutical products.
Speaker Change: They tend to actually be Barry.
Speaker Change: Well thought through and typically minimal.
Speaker Change: Because nobody wants to actually have an impact on the supply of medications for a population that is not a good thing to do from a health perspective, but also overall perspective clearly not the.
I think something that is really attractive.
Speaker Change: To impose.
Speaker Change: Typically block products have been excluded from tariffs if you look at historically at that asset.
Speaker Change: Mr. Harris I think at this point I think way too early to actually have a have a real view.
Speaker Change: I don't think we have visibility in the space.
Speaker Change: So at this point in time, I don't think Theres more that we can really comment on that.
Speaker Change: Okay. Thank you.
Speaker Change: And one moment for our next question.
Speaker Change: Our next question comes from Mr. <unk> of <unk>. Your line is open.
Speaker Change: Hey, good bundle and good afternoon, guys. Thanks for taking my questions.
Among the centers that have treated patients in the commercial setting without capsule can you give us a little bit of color on what the spread is.
Speaker Change: This amount of time from site activation to get enough presentation David.
Speaker Change: Follow up.
Speaker Change: Yes, so thanks for joining us ticker.
Speaker Change: It's quite it's quite variable.
Speaker Change: In terms of the time it takes to actually go from activation to actually having a patient at all.
Speaker Change: For many of the centers a lot of the activation process and the speed at which the activation process kind of went through has to do with a patient actually.
Speaker Change: Being suitable for the therapy and considered to be stood at suitable for the therapy and the momentum arrived that so theres some centers, where if there is a relatively short period in between but you could also have situations, where if the process took a certain amount of time that maybe the patient had to move on to other therapy, and then there might be somewhat of a.
Speaker Change: A bit of a lag until the next patient might actually come along I think the short answer is it's variable I don't think its.
Speaker Change: I don't think we could easily project that within X amount of time. The patients are all were seeing in general that the.
Speaker Change: That we see centers to actually get into sort of a repeat mode and actually.
Speaker Change: Get get more than one patient, obviously on which is really good because one of the things we'd like to see that overtime centers actually start using the product more rapidly and with that obviously it will start to see some element of momentum build overtime. So early signs I think are very positive, but it's still early days.
Speaker Change: Okay. Thanks, and then.
Speaker Change: I know, it's very early days.
Speaker Change: The launch right now, but what are the intriguing things about our cat's always that perhaps.
Speaker Change: Perhaps patients.
Speaker Change: Do better by not having.
Speaker Change: Big picture transplant.
Speaker Change: And you've had several opportunities to talk about the data and present the data.
Speaker Change: And in medical journals since approval I'm, just wondering with your initial experience right out what seems to be the sentiment among prescribers.
Speaker Change: Trading positions.
Speaker Change: There've been some sort of an appreciation trend or do you think you'll still need more time and to give them more hands on experience to feel comfortable saying I don't want to take this patient to transplant after a capsule.
Speaker Change: Yes, I think the question with regards to is there a need for sort of subsequent therapy.
Speaker Change: When we look at the trial experience, we had about 18%.
Speaker Change: The patients move to a subsequent.
Speaker Change: Stem cell transplants are relatively low percentage of the patients.
Speaker Change: A lot of the patients if you had a prior transplant actually wouldn't be eligible again for a transplant.
Speaker Change: Or they might have other.
Speaker Change: Comorbidities et cetera that would sort of prevent.
Speaker Change: A transplant or make it unlikely to be successful.
Speaker Change: To some extent on the nature of the patients coming in but overall, obviously, even in the trial, we had seen the relatively limited use of transplant.
Speaker Change: We'll need to see kind of how that develops I don't think at this point, we have we can even tell because it's just too early.
Speaker Change: But we also based on the experience that physicians had with the product through the development that we would see it probably initially a similar picture in that as there is more.
Speaker Change: Experienced gains we've seen it to see where that where that's headed but overall, obviously very encouraging and and also an option for.
Speaker Change: Patients that currently you would have very limited treatment options for.
Speaker Change: Sure.
Speaker Change: And I think gives us an opportunity here to really position the product across the entire range of risk factors of H.
Speaker Change: And I think Thats really where there is a significant appeal for this product because they see it is actually can actually.
Speaker Change: Deliver.
Speaker Change: Positive outcomes across the entire range of the patients.
Speaker Change: That we have in the relapsed refractory setting.
Speaker Change: I would always say.
Speaker Change: What we have seen this debt.
Speaker Change: The patients that were receiving transplant didn't appear to actually do better.
Speaker Change: And then patients who did not receive transplants and that certainly has resonated with a lot of the physicians.
Speaker Change: And so we'll see how it develops I think too early to tell but overall I think a lot of conviction that there is a real opportunity for the product to be a standalone therapy without the need for subsequent consolidation.
Speaker Change: And one moment our next question.
Our next question will be coming from Matthew Phipps of William Blair. Your line is open Matthew.
Matthew Phipps: Hi, guys. Thanks for taking my question I was wondering if you could just maybe comment on any of the manufacturing success rate our turnaround time, so far and the commercial launch maybe just.
Matthew Phipps: I guess, maybe all in line with some of the clinical experience and then I assume that biotech is not yet made any decision on auto <unk> and G. As far as opting in is there a timeline for when that might occur.
Speaker Change: Yes, thanks for joining Matt in terms of sort of the production.
Speaker Change: Experienced that we're gaining now on the commercial side, we see that actually mirroring very nicely what the experience was during the conduct of the pivotal study. So that's very reassuring.
Speaker Change: And obviously, we're starting to get more and more experience as per cent of running more product through the facility, but we are seeing.
Speaker Change: The data to track very nicely both our prior experience was so that's the first observation.
Speaker Change: Second question was related to the options that.
Speaker Change: Balanced deck has upheld that has an option on our solid tumor program or <unk>.
Speaker Change: That's an option that actually is still running and I think it's obviously going to be.
Speaker Change: The option as we had indicated before that will be triggered prior to moving the product into a pivotal study. So we're still obviously rolling through the current clinical study, we're conducting with UCL.
Speaker Change: And so we would expect that that trial to actually.
Speaker Change: Deliver results and then after we have the results in that the path is clear for the path forward is clear that that's sort of actually would sort of be the time point for an option exercise. So it's still a little bit ahead of us here.
Speaker Change: Okay.
Speaker Change: And one moment our next question.
Speaker Change: Our next question will be coming from Gil Blum of Needham <unk> Company. Your line is open.
Gil Blum: Good afternoon, good morning, and thanks for taking our question just a couple from us.
Speaker Change: No.
Speaker Change: We're going to see some of the <unk> data and your upcoming R&D day.
Speaker Change: And maybe maybe you can put that data in context, I mean six patients.
Speaker Change: What kind of data are we going to see here is safety.
Speaker Change: Safety Phillips.
Speaker Change: And my second question is what would you say.
Speaker Change: Most of your resources as it relates to the launch are being invested.
Speaker Change: Rollout or is there more to it thank you.
Speaker Change: Yes. Thanks.
Speaker Change: Much appreciate it go so with regards to the <unk> data as I indicated this is a cohort of six patients dosed at 50 million cells.
Speaker Change: There's a set of parameters. We're looking at the first set of parameters really understanding the properties of the product we've seen quite a bit of variability across the various programs in terms of the ability of the product to actually expand in vivo as the.
Speaker Change: The persistence was observed with these products. So that's the first observation because that gives us a sense of the quality of the product and how it actually performs and it also gives us an ability to sort of look at.
Speaker Change: And compare back to as an example, the experienced staff.
Speaker Change: Aerojet, Steve Hatten, and Airlines, which I think is still kind of the key data point or set of data points I think the field is comparing to so first is product property from.
Speaker Change: Behavior percentage of the product itself. The second is clearly around safety.
Speaker Change: We've seen variability in terms of the safety events. So we're going to look obviously at the.
Speaker Change: <unk>.
Speaker Change: Crs, we're going to look like there was anything that we would pick up a neurological talks.
Speaker Change: But that gives us sort of the next picture element of the picture in terms of the behavior of the product in these autoimmune patients.
Speaker Change: The third area is around the actual expected action of the product and that actually goes to the depletion of b cells. So we're going to look at B cell depletion. We're also going to look at the impact on auto reactive antibodies, we're going to look at disease scores and in particular, we're going to also look at not just the disease score as.
Speaker Change: Generic score, but also to look at the individual components of the disease score, which I think is important and has certainly led to a bit of confusion during the last year when I think.
Speaker Change: Different datasets were compare to each other or people were trying to do that in general I think the patient population we're looking at.
Speaker Change: Sort of a more advanced and more real world population that we're looking at.
Speaker Change: In our Carlisle study.
Speaker Change: These are patients that do have actually significant impact on.
Speaker Change: On the kidney and so it is a population that has.
Speaker Change: Actual level of.
Speaker Change: Tissue damage to the actually do see an impact from kidney function. So it's a more.
Speaker Change: Real world population compared to the very young and early in their disease population that we've seen an airline but I think it gives us a very good view to sort of accurately compare to some of those early experiences, but also gives us an understanding of the profile of the product the follow up obviously is limited.
Speaker Change: That's also where we're pointing to the second half of the year for longer term follow up full reconstitution of.
Speaker Change: B cell compartment post.
Speaker Change: Persistence stops.
Speaker Change: Stops in those patients.
Speaker Change: So that will be sort of a second data set later in the year, but I think we're going to get a very good feel for the products and the properties that we were expected.
Speaker Change: We expect to see in these patients.
Speaker Change: And then in terms of so the resources going into the launch.
Speaker Change: I think what's important the bag from a commercial perspective.
Speaker Change: What we are delivering this with these types of therapies Israeli a set of services to support the centers. So it's different from your conventional commercial model.
Speaker Change: And it's much more a focused on delivering services and providing support.
Speaker Change: We do that through.
Speaker Change: A single point of access that actually Triaging use and supports the sent the triage as the needs and requests to support and actually it's directly engaging with the centers and <unk>.
Speaker Change: <unk> partners with the center very closely.
Speaker Change: That's sort of the model that we're running and that's obviously been supported by medical Affairs.
Speaker Change: As well as obviously the support on the reimbursement side.
Speaker Change: And the engagement from a market access perspective, so that's sort of the distribution. We have so it's much more on the service side than it would be so that the more classical.
Speaker Change: Sales and marketing functions that you would have with a conventional launch of a product.
Speaker Change: So maybe that.
Speaker Change: It could be helpful.
Speaker Change: Thank you.
Speaker Change: Our next question.
Speaker Change: Our next question will be coming from Kelly <unk> of Jefferies. Your line is open Kelly.
Speaker Change: Congrats on the progress.
Kelly: Thank you for taking my questions have two thanks.
Kelly: Galka Allo program.
Kelly: The first wave of training.
Kelly: Adoption included a centuries had no prior.
Kelly: Sharon.
Kelly: Further Sanjay has some hesitation to make a switch from well with beta customers.
Kelly: Sure. Thank you.
Kelly: Yeah. Thanks, Thanks for the questions Kelly.
I think what's been very interesting to see is that we had both centers with prior experience with Ob cells as part of <unk>.
Kelly: The Felix study as well as sensors that were not part of the CLEC study that we saw actually very early on.
Kelly: Onboarding and activate and getting activated so it was very interesting to see that we have.
Kelly: Both types of centers actually moving very quickly.
Kelly: And we do see that there's clearly a substantial.
Kelly: Need there in terms of patients that were considered by those physicians to be very suitable for further treatment with with Ob cell.
Kelly: And.
Kelly: And they're in their view apparently felt that that was the appropriate treatment for these patients. So it was very interesting to see the dynamic of both.
Kelly: The types of centers to get on very quickly.
Kelly: And I think we're seeing very good.
Kelly: Some interest levels for using the <unk> for using the product early on and across quite a wide range of patients in terms of the conditions of the patients were in.
Kelly: So I think overall I think.
Kelly: Looking very positive sensitive.
Kelly: To kind of a range of experience I think that we're having in the pivotal study.
Speaker Change: Thanks for the color also saw Michele you have mentioned that you could add a few more patients at ethane assistant Milan.
Kelly: This higher.
Speaker Change: Just curious what kind of efficacy signals.
Speaker Change: Yes.
Speaker Change: And to dose higher than 50 Amelia.
Speaker Change: Alternative question is Jeff.
Speaker Change: After several datasets job.
Speaker Change: Last year for cell therapy here.
Speaker Change: Can you share your insights.
Speaker Change: Uh huh.
Speaker Change: The goal of car T.
Speaker Change: Okay.
Speaker Change: At this moment.
Speaker Change: Yeah. So so obviously the way we set up the study is too.
Speaker Change: Going with a fixed dose, which is a translation of the pediatric ALLL dose use that as the basis for the fixed dose. That's how we arrived at the 50 billion cell dose. We then had an opportunity or designed the trial in a way that would allow us to actually step up or step down.
Speaker Change: And we.
Speaker Change: We also have an opportunity to jeff's they expand expand the cohort.
Speaker Change: We'll provide more information obviously at the R&D day, and also to give you a sense of kind of how we're moving forward and what the next steps are and where we're going to go.
Speaker Change: In that space and I think that will sort of answer and actually I think your question and much more deeply.
Speaker Change: Fundamentally.
Speaker Change: Obviously I think we're at a good place in terms of.
Speaker Change: The experience that we had and also the safety data that we already have with.
Speaker Change: With the product at that dose level as well as the efficacy level then it's the ability to really remove b cells very very deeply.
Speaker Change: Eight patients based on the pediatric and El al as well as due to wholesale experience. So we think we're in a good spot there we have a good understanding of what our product does and how it behaves.
Speaker Change: And we're looking forward to giving you the update in terms of where we're going to go next at the April 23rd R&D day.
Speaker Change: Thank you.
Speaker Change: I think you had a question related to <unk> and the positioning of car T and SLE did I hear that right.
Speaker Change: Yes.
Speaker Change: Okay. So I think with that I'll review actually in terms of the positioning of car Ts in SME I don't think has really changed.
Speaker Change: Obviously, when you look at <unk> in the U S. You've got about 400000 patients overall, so it's a very large population an incidence basis, but many of these patients can be managed with standard of care. The reason at a reasonably sensible level.
Speaker Change: But you do have a small proportion of patients to do actually progress.
Speaker Change: Two very severe stages of the disease.
Speaker Change: There the level of intensity of therapy that we're obviously, having with the car T therapy. We believe is very well positioned very well warranted and that's actually a much smaller population than we had indicated that this is somewhere between 10020 thousand patients out of the 400000. So it's a much smaller population of the true high medical need segment.
Speaker Change: Of the disease, where we think this type of a therapy is well suited to be used and I think also where you have a compelling health economic argument to treat those patients with the car T therapy.
Speaker Change: Thank you very much.
Speaker Change: Thank you.
Speaker Change: Our next question.
Speaker Change: Our next question will be coming from Yanan Zhu.
Speaker Change: Wells Fargo. Your line is open.
Speaker Change: Great. Thanks for taking our questions.
Speaker Change: So first.
Speaker Change: Was wondering.
Speaker Change: Could you comment on in terms of the sense of the centers on boarded.
Speaker Change: Have you on board at the top 10 centers in terms of.
Speaker Change: Our patient volume.
Speaker Change: There is still work to be down there.
Speaker Change: And also wondering.
Speaker Change: Are you in a position to comment on a number of apheresis.
Speaker Change: To date or how has that been evolving and lastly on Qatar.
Speaker Change: Qatar.
I'm wondering if you.
Speaker Change: Whether you plan to provide sales guidance it sound plant and if so when might be.
Speaker Change: The right time to sell.
Speaker Change: Thank you.
Speaker Change: Thanks, a lot.
Speaker Change: <unk>.
Speaker Change: I do understand your questions.
Speaker Change: So let me go through first of all have the top 10 top census being included.
Speaker Change: When you go onto the elements of this website you can actually see the census, there on there you do see that.
Speaker Change: And very consistent high levels of patient flows. So we do believe that within those first 33 centers that we have.
Speaker Change: Many.
Speaker Change: Of the of the very high volume centers, they're probably only few additional.
Speaker Change: The high volume centers that may not yet be active at this point.
Speaker Change: And this is why we are guiding to already within that population to cover a very substantial proportion of the.
Speaker Change: Of provided.
Speaker Change: Large proportion of access to.
Speaker Change: So the U S patients already.
Speaker Change: And that also coincides when you look at the distribution also with.
Speaker Change: The big.
Speaker Change: The areas of the states with a lot of with high levels of population.
Speaker Change: Across as well, which is the best way to look at this and so to get a feel for that.
Speaker Change: With regards to <unk>, we do not guide on Ace races.
Speaker Change: And I think there has been <unk>.
Speaker Change: Some companies that have tried to do that we don't think it's going to be helpful to do that because there's obviously a time difference between eight races collection and actually dosing of the patients, which actually then gets I think a very complex way of reporting and sort of following the story. So we got a report actually on the patients that were dosed.
Speaker Change: Of which we think is the cleanest way of doing that because that is also the time points. When in fact, the revenue you recognized revenue. So thats importance that we're going to have a very consistent by reporting that and we're not going to give a leading edge.
Speaker Change: Based on.
Speaker Change: The number of <unk> sites.
And then the last point was around sales guidance, we certainly for this year, we will not give any sales guidance. So we don't believe that that's actually a very sensible thing for us to.
Speaker Change: Due to give guidance.
Speaker Change: Simply because you basically have multiple.
Speaker Change: Developments that you're actually going to go through that each one of them will be all the trajectory.
Speaker Change: Profit zone. So you have obviously the number of centers that are active and you will see that the report on that in this publicly visible. So that is obviously the basis to even have an ability to deliver therapy, so that momentum and the onboarding and the addition of centers obviously, it will sort of increase your footprint and your ability to sort of drive sales the <unk>.
Speaker Change: Second aspect is.
Speaker Change: That obviously within <unk>.
Speaker Change: Each one of the centers there are multiple physicians typically do deliver therapies. They have different backgrounds, and you often start with an individual physicians starting to use the product in overtime and experience gained more of the physicians, we expect to start using the product. That's in other kind of ramp that youre going through and the third has a lot to.
Speaker Change: Do with obviously the experience made with the program per se with that the ability to actually.
Speaker Change: Get access and provide access to a larger proportion of patients who today may not actually have considered access to car T. As well. So you have several.
Speaker Change: Elements that each of those actually are an integral curve tried to actually overlay that and project is actually very challenging and we're not planning to do that so we're going to actually stick with reality, which is patients dosed AD revenue recognizable and I think that gives us a very clear very clean the communication I think will allow everybody to.
Speaker Change: Follow exactly where we are.
Speaker Change: Okay, Great makes a lot of sense and finally have a very quick follow up on the SL E second half.
Speaker Change: Data update I was wondering.
Speaker Change: Beyond the additional follow up for the first six patients.
Speaker Change: Might be the incremental data new data.
Speaker Change: At that update thank you.
Speaker Change: Yes, so I think the most important update will be obviously the follow up because one of the key questions that you have with this type of a therapy is whether indeed, you had a full reset of the compartment. So whether you were able to actually have an impact on the disease removed the auto reactive antibodies and then actually wants to therapy stops working.
Speaker Change: And the patients you see the recurrence of the B cells and what do you want to see at that point is an absence of auto reactive T cells that gives you. The information that indeed, you were able to do a reset in the compartment. So I think that's really important information because it gives you information about the actual clinical impact and true impact that their therapy bolthouse.
Speaker Change: So that's the most important information.
Speaker Change: And then we're going to guide or stay at the April meeting and how we're going to plan going forward, but I think the primary is the most valuable piece of the information will be sharing will certainly be arrived the long term follow up.
Speaker Change: Great. Thank you.
Speaker Change: Thank you.
Speaker Change: And our next question will come from Jacob Macau of KBC Securities. Your line is open Jacob.
Jacob Macau: Hi, there and thanks for taking my question I just wanted to zoom in again on the initial experience with a capsule and the conventional setting, particularly in terms of the vein to vein time that you have been able to achieve have you been able to achieve your target of 16 days with the first few batches and perhaps more broadly.
Speaker Change: What has been going well and are there any learnings from the first few and yet.
Jacob Macau: <unk> batches that you will implement going forward.
Speaker Change: Yes, so thanks for joining Jacob.
Jacob Macau: So what we see actually in.
Jacob Macau: During the initial phase of the launch in terms of the turnaround time for the product is that we see a high degree of consistency.
Jacob Macau: With our prior experience and tracking.
Jacob Macau: Within our expectations that we had for the guidance that we had for the product itself. So we feel very confident that the consistency, we're seeing actually builds very nicely and we see those processes work very well.
Jacob Macau: In terms of the learnings I think one of the interesting things of course is is that I've mentioned that in the context of the manufacturing side its little that patient number one coming through the door basically that's when he actually.
Jacob Macau: Turn the engine on that and you start to actually have.
Jacob Macau: Every aspect of the process.
Jacob Macau: From supporting Percenters tube with information to support actually and secure reimbursement all the way through to the.
Jacob Macau: The actual handling of the product handling FTF reset the whole logistics chain the manufacturing process itself.
Jacob Macau: And that obviously.
Jacob Macau: The additional support to the St. Joe in terms of training et cetera. So you have actually all of those items that actually start to actually well. It helps to have the rollout time they have to be consistent.
Jacob Macau: All of the systems, they have to be robust and stable.
Jacob Macau: That's an interesting transition so.
Jacob Macau: I think a lot of.
Jacob Macau: Yes.
Jacob Macau: Good input that we had working very closely with the centers to really focus on making sure that the experience for the centers are positive and that we're also taking as much of the workload off the centers and I think that's been a key focus as you can imagine each center works a bit differently and so theres a lot of.
Jacob Macau: <unk> that obviously, we're going through with each 100% just to make sure that this is positive and experience as possible and that we're continuously improving our platform.
Jacob Macau: For the engagement.
Jacob Macau: The booking.
Jacob Macau: All the way through in terms of the delivery of the product. So this is an ongoing process and I think we're seeing really good collaboration.
Jacob Macau: With all of our centers very valuable feedback and were continuously looking and strive to actually improve the experience and that's actually the process we're in and.
We're convinced we're going to be in that for the duration of the delivery of this product because we want to be as easy to use as possible for the centers is easy to access as possible and we're going to continue to work on that to make sure that that's going to be an experience that we can actually continuously improves over time, but great start.
Jacob Macau: Good level of engagement great feedback.
Jacob Macau: <unk> puts us in a very strong position.
Speaker Change: Okay. Thank you I just have one more and if I may on your conversations with the EMA on the EU approval and based on those conversations where in the second half do you expect approval to come and perhaps are you able to comment on any preparations that you are working on for the EU launch and how does that.
Jacob Macau: As compared to the U S in terms of center Onboarding.
Speaker Change: Yes, so the.
Speaker Change: Interactions with the European agency, it's going through the normal process, which is a very well described.
Speaker Change: And also from a timeline perspective et cetera, well defined process to go through.
Speaker Change: So that's where.
Speaker Change: That's running we started that process in the end of March last year. So we're kind of getting to the latter later part of the process, which is a series of questions answers additional review final questions again.
Speaker Change: Answers a final review and then you go into the final steps.
Speaker Change: That ultimately resulted in approval or no approvals. So so we're in that second half of the process.
Speaker Change: But I don't think it makes sense for us to actually try to pinpoint date that wouldn't be it would be.
Speaker Change: The whole thing for the for us to do because frankly.
Speaker Change: We don't we don't round the clock.
Speaker Change: So with regards to the preparation I mean very similar too.
Speaker Change: What we did in the U S. In terms of preparation we are working with the clinical centers in the U K and we're working with.
Speaker Change: Clinical centers.
Speaker Change: Earlier stage.
Speaker Change: In Germany is the first country that we're interested in.
Speaker Change: To potentially be in a position to launch so we're in conversation with the center, who start to do the preparatory work as we've done in the U S very similar process.
Speaker Change: That we're running through and a lot of the learnings that we have made in the U S. Obviously very applicable also for the U K as well as these.
Speaker Change: Clickable four four European centers, so that's ongoing.
Speaker Change: Obviously, it gives us a very strong basis, because obviously a lot of the systems have been worked out.
Speaker Change: Already for the U S launch in <unk>.
Speaker Change: Required typically only minimal habitation.
Speaker Change: For the jurisdictions in Europe as well. So we think we're in a very good spot there and can do that and actually frankly do that work in a very efficient way.
Speaker Change: Okay. Thank you that's very clear.
Speaker Change: Thank you very much thank you.
Speaker Change: One moment for our next question and our last question will be coming from Simon Baker of Redburn Atlantic Your line is open.
Simon Baker: Thank you very much for taking my question two if I may please firstly, just continuing on <unk> and.
Speaker Change: And the U K.
Speaker Change: Typically we see the importance of a nice.
Speaker Change: Assessment as having relevance far beyond the borders of the U K. So just wondering if you could give us an idea on the expected timeline of a decision by nice or capsule.
Speaker Change: Then moving on to the Carlisle study.
Speaker Change: Could you give us an idea of the duration of exposure that we will.
Speaker Change: See that interim analysis for the six patients you started dosing in February 2004, So I just wonder if you could give us some flavor.
Speaker Change: How much follow up that will be a cost synergy initiatives six patients. Thanks, so much.
Simon Baker: Thanks, Simon So I'll start with the second question, which is sort of the follow up time.
Simon Baker: Out of the six patients were going to have one patients with more than six months of follow up to patients with more than three months and three patients that are somewhere between one and three months of follow up so that's sort of the nature of the follow up and that's also why I indicated the importance of going through the second half of the year for longer follow up but notwithstanding.
Simon Baker: The overall profile in terms of the reconstitution of the of the visa compartment.
Simon Baker: With regards to to the to the UK.
Simon Baker: As you point out to the two processes the development in the U K Youll have on the one hand, the regulatory process, which has gone through the NHRA and has its own.
Simon Baker: <unk> through its own timeline.
Simon Baker: And then there's the second set of engagement. So just great interest and have actually demonstrated.
Simon Baker: The economic benefit of your product as part of the.
Simon Baker: As part of the assessment that ultimately will lead them to a determination of what the price of the U k's.
Both processes take not insignificant amount of time.
Simon Baker: Both are processes that are ongoing.
Obviously, it is not entirely under our control in terms of the timing.
Simon Baker: But I think as both are.
Simon Baker: Well, where they need to be relative to each other.
Simon Baker: So we're looking forward to updating you once we're sort of factually cleared.
Simon Baker: The regulatory hurdle and then obviously can talk about.
Simon Baker: The final steps that you have to go through before you can actually deliver product in the UK commercially.
Simon Baker: Okay. Thanks, so much.
Simon Baker: Thank you very much.
And I would now like to hand, the call back to Christian for closing remarks.
Speaker Change: Yes first of all thanks, a lot for joining.
Speaker Change: Exciting 2024, I think we're in a really good start for 2025, we're excited about where we are with the with the launch the opportunity to broaden the utility.
Speaker Change: Obi sell in the future as well.
Speaker Change: And we're looking forward to updating you at the 20 <unk> of April at the R&D Day in New York and OSA would love to see many of you actually be able to join us in person.
Speaker Change: So thank you very much and thanks for your questions have a great day and this concludes today's conference call. Thank you for participating you may now disconnect.
Speaker Change: Yeah.
[music].
Speaker Change: Okay.
Speaker Change: [music].
Speaker Change: Okay.
Speaker Change: [music].