Cellectis S.A. ($ALCLS)

Earnings Call Transcript · March 20, 2026

ENXTPA FR Health Care Biotechnology Earnings Calls 46 min

Earnings Call Speaker Segments

Operator

Operator
#1

Hello, and welcome, everyone, to today's Cellectis' Full Year 2025 Earnings Conference Call. [Operator Instructions] Please note this call is being recorded [Operator Instructions]. It is now my pleasure to turn the meeting over to Arthur Stril, Chief Financial Officer and Chief Business Officer. Please go ahead.

Arthur Stril

Executives
#2

Good morning, and welcome, everyone, to Cellectis Fourth Quarter and Full Year 2025 Business Update and Financial Results Conference Call. Joining me on the call today are Dr. Andre Choulika, our Chief Executive Officer; and Dr. Adrian Kilcoyne, our Chief Medical Officer. Yesterday evening, Cellectis issued a 20-F and press release reporting our financial statements for the 12-month period ended December 31, 2025, and a business update. The report and press release are available on our website at cellectis.com. As a reminder, we will make statements regarding Cellectis' financial outlook, including the sufficiency of cash to fund operations in addition to its manufacturing, regulatory and product development status as well as product development status of its license partners. These forward statements, which are based on our management's current expectations and assumptions and on information currently available to management, including information provided or otherwise publicly reported by our licensed partners are subject to risks and uncertainties that may cause actual results to differ from those forecasted. A description of these risks can be found in our most recent Form 20-F filed with the Securities and Exchange Commission, SEC, and the financial report, including the management report for the year ended on December 31, 2025, and subsequent filings Cellectis makes with the SEC from time to time. I would now like to turn the call over to Andre.

André Choulika

Executives
#3

Thank you, Arthur. Good morning, and thank you, everyone, for joining us today. Cellectis was founded with the conviction that gene editing could fundamentally transform how we treat patients in the 21st century. That foundation and ID, we pioneered the allogeneic CAR T approach ready on day 1, built for all patients regardless of their condition. Today, I'm proud to say that Cellectis is the one of the only company running a pivotal Phase II allogeneic CAR T trial, specifically in B-cell acute lymphoblastic leukemia. The past several years have been among the most challenging in recent memory of biotechnology companies. Many programs were shelved and many companies were forced to retreat. While others were stepped back, Cellectis stepped forward. We held the line. We manage our cash with rigor. We invested where it matters, and we kept our team focused entirely on one thing, delivering clinical results for patients who are running out of time with no therapeutic solution. In 2025, that discipline paid off. In October, at our R&D Day, we presented the full Phase I data set for lasme-cel, our allogeneic CAR T candidate targeting CD22 in relapsed or refractory B-cell acute lymphoblastic leukemia. lasme-cel achieved 100% overall response rate in the target Phase II population. Critically, lasme-cel converted all patients in the target population to transplant eligible candidates. These results were achieved in patients in third line and beyond. Most of them have already failed CD19 CAR T, blinatumomab and inotuzumab. Their options were exhausted. This is clinical proof that allogeneic CAR T can deliver deep, durable responses in one of the most difficult cancer to treat. Let me take a moment to explain why our bridge to transplant strategy is medically powerful. For patients with relapsed or refractory BLL, only known path to long-term cure is a bone marrow stem cell transplant. But to be eligible for transplant in a patient, a patient must first achieve deep remission, ideally minimal residual disease negative or MRD negative. The challenge is that heavily pretreated patients often too burdened with disease or too time constrained to wait for an autologous therapy to be manufactured. Window is narrow. This is precisely where our allogeneic CAR T product, lasme-cel becomes a game changer, off the shelf, immediately available. Lasme-cel has the potential to reach a patient in days, not weeks, rapidly eliminate residual disease and open the door to transplant. For the patients, it is the difference between a chance to cure and no chance at all. Without our pivotal Phase II trial now initiated, we will continue site openings in North America and Europe and enroll expansion in 2026. The first interim analysis of the pivotal Phase II trial is expected Q4 2026. Turning to our second product candidate, eti-cel for patients with relapsed or refractory non-Hodgkin lymphoma. Eti-cel is a best-in-class allogeneic dual CAR T targeting CD20 and CD22 simultaneously, 2 differentiated antigens validated in oncology. This dual targeting is a deliberate answer to one of the most stubborn clinical problem in lymphoma, antigen escape. When cancer cells lose on surface marker to evade a single target therapy, they cannot hide from both. Eti-cel was built for that challenge. At the ASH 2025 Annual Meeting in December 2025, Cellectis presented Phase I interim results, which demonstrated an encouraging overall response rate of 88% and a complete response of 63% in heavily pretreated patients. These preliminary data underscore the potential of this innovative approach to transform outcomes for relapsed or refractory non-Hodgkin lymphoma patients. Trial is now investigating any potential impact of low-dose interleukin-2 support to significantly enhance expansion and the persistence of CAR T cells to boost CAR T efficacy without exacerbating toxicity. Cellectis expects to present the full Phase I data set of eti-cel this year. Now a few words on our partners. Cellectis is not operating in isolation. Our gene editing platform has become technological backbone of a broader allogeneic CAR T ecosystem. Two of our key partners are approaching pivotal moment. Servier through Allogene's cema-cel program is currently in a pivotal Phase II study, evaluating it as a consolidation therapy in first-line large B-cell lymphoma patients. Allogene anticipates that an interim futility analysis is on track for Q2 2026. Our partnership with Iovance is another powerful signal to reach any versatility of our platform. Iovance is advancing IOV-4001 in PD-1 inactivated tumor infiltrating lymphocytes or TIL cell therapy. In previously treated advanced melanoma patients, leveraging our gene editing capacity, clinical results of IOV-4001 in melanoma are anticipated this year. R&D activities continue to advance under our research and collaboration agreement with AstraZeneca, which leverages Cellectis' gene editing expertise and manufacturing capabilities to develop up to 10 novel cell and gene therapy products for areas of high unmet medical need, including oncology, immunology and rare genetic disorders. Partnership is a further testament to the industrial credibility of our gene editing platform and our manufacturing capabilities. 2026 will be a year of data of milestones and of momentum for Cellectis. We are grateful for your continued trust and support. We look forward to updating you through the year as we execute against each of these milestones. With that, I would like to turn the call over to Dr. Adrian Kilcoyne, our Chief Medical Officer, who will provide further details on our clinical programs. Adrian, please go ahead.

Adrian Kilcoyne

Executives
#4

Thank you, Andre. I will provide a focused clinical perspective on our lasme-cel and eti-cel programs. The Phase I BALLI-01 study of lasme-cel in third line and beyond acute lymphoblastic leukemia enrolled 40 patients with confirmed at least 70% CD22 expression. These patients were heavily pretreated with a median of 4 prior lines of therapy. The median number of prior lines of therapy at the recommended Phase II dose was higher at 5. These heavily pretreated patients have already relapsed following multiple targeted therapies. Most patients have been previously treated with blinatumomab and relapsed. Approximately 50% have also relapsed following CD19 CAR T and alemtuzumab, a CD22 directed antibody drug conjugate. Therefore, there remains very few, if any, therapeutic options for these patients. At the recommended Phase II dose, lasme-cel achieved an overall response rate of 83% and a CR/CRi rate of 42%. In the target Phase II population with an upper age cutoff of 50 years old, response rates were even higher with 100% overall response and a CR/CRi rate of 56%. Importantly, of those subjects who achieved a CR/CRi, 80% achieved MRD-negative status. Additionally, of the 9 patients in the target Phase II population, all became transplant eligible with 7 of 9 receiving stem cell transplant at the time of data cutoff. This is a very positive outcome for these patients. In patients who achieved MRD-negative , median overall survival was 14.8 months, a meaningful survival benefit in this heavily pretreated population. The safety profile of lasme-cel was favorable and similar or lower than observed with other autologous CAR T therapies. Greater than or equal to Grade 3 cytokine release syndrome occurred in 2.5% of patients and greater than or equal to Grade 3 ICANS occurred in 5% of patients at the recommended Phase II dose. The full Phase I data has been submitted for presentation at the 2026 European Hematology Association Congress to be held in Stockholm in June. The Phase I program also addressed 2 important additional questions. The first is whether our internally manufactured product could result in similar or indeed improved efficacy compared to product manufactured by an external CDMO. The second is whether alemtuzumab as part of the preconditioning lymphodepletion regimen results in superior lasme-cel expansion and therefore, efficacy compared to the standard lymphodepletion regimen. Validating our decision to internalize our manufacturing capabilities, Cellectis' manufactured product demonstrated meaningfully higher response rates than external CDMO manufactured product with overall response rates of 68% and 28%, respectively. Secondly, the data demonstrated that increased alemtuzumab exposure correlates with improved response. These data were outlined at the American Society of Hematology Annual Conference in December 2025. Additional data have also been submitted for presentation at the 2026 European Hematology Association Congress. We are now enrolling in the pivotal Phase II program focused on the target Phase II population of patients aged between 12 and 50 years of age. We are accelerating site opening and are on track to reach our goal of approximately 75 recruiting centers across Europe and North America. As Andre highlighted, we expect to complete the first interim analysis of 40 patients in Q4 2026. These data will be shared publicly in a forum to be determined. I will remind you that as previously disclosed, the anticipated BLA submission is planned for the second half of 2028. Our second ongoing program investigating our dual CAR asset, eti-cel, targets CD20 and CD22 expressing tumors in third line and beyond non-Hodgkin's lymphoma. This highly differentiated product offering important and much needed alternative targets of CD19 continues in Phase I. The preliminary Phase I data presented at ASH 2025 showed an 88% overall response rate and a 63% complete response rate at the current dose level in the 8 evaluable patients. Cellectis believes we can further enhance these already high response rates through the addition of low-dose IL-2 support. The addition of low-dose IL-2 offers the potential to further enhance CAR T expansion, tumor killing and persistence without negatively impacting toxicities. We expect to present the Phase I data set, including the IL-2 cohort results later this year. We plan to progress this program to pivotal Phase II in 2027 and anticipate a BLA submission in H2 2029. As you can see, 2026 promises to be an exciting year for Cellectis with a number of critical milestones and catalysts as we transform into a late-stage development organization. I look forward to sharing our progress later this year. With that, I would like to hand the call over to Arthur Stril, Cellectis' Chief Financial Officer and Chief Business Officer, for an overview of our financials for the fourth quarter and full year 2025. Arthur, over to you.

Arthur Stril

Executives
#5

Thank you, Adrian. Let me now walk you through our financial position. As Andre mentioned, we have managed our cash with discipline over the past year, focusing our spend on what matters most. The development of lasme-cel and eti-cel and the operation of our end-to-end manufacturing facilities in Paris and Raleigh. We believe our current cash position gives us the financial runway to execute on our pivotal Phase II program for lasme-cel and our Phase I for eti-cel and delivered 2 key readouts in Q4 2026. The first interim analysis with 40 patients for lasme-cel and the full Phase I data set for eti-cel . We are well positioned financially to execute on these 2 trials as our cash, cash equivalents and fixed term deposits as of December 31, 2025, remain sufficient to fund our operations into H2 2027. We are also looking forward to the upcoming data readouts for our partner programs, in particular, Servier and Allogene cema-cel in April 2026 and Iovance's IOV-4001 this year as well. Finally, we are excited that activities are progressing under our strategic collaboration with AstraZeneca, which has positively impacted our 2025 revenue. As of December 31, 2025, our cash, cash equivalents, restricted cash and fixed term deposits classified as current financial assets amount to $211 million compared to $264 million as of December 31, 2024. This $53 million decrease is mainly due to $36.9 million cash in from revenue, $8.4 million of interest received from our financial and cash equivalent investments, partially offset by cash payments from Cellectis to suppliers of $50.5 million. Cellectis wages, bonuses and social expenses paid of $40 million, the payments of lease debts of $11 million and the repayment of the PGE loan of $5.4 million. You are invited to refer to our press release for figures related to consolidated net loss attributable to shareholders of Cellectis for the 12 months ended December 31, 2025. We very much look forward to a rich 2026, especially with Servier and Allogene's readout for cema-cel next month and our 2 readouts for lasme-cel and eti-cel later this year. We'll now turn the call over to the operator for questions.

Operator

Operator
#6

[Operator Instructions] Our first question will come from Amin Makarem with Jefferies.

Mohamad Amin Makarem

Analysts
#7

Two from us. First, on the lasme-cel and the BALLI-01 enrollment. How is recruitment tracking in the pivotal study? And when do you expect to complete enrollment for the dose selection portion? And I have a follow-up.

Adrian Kilcoyne

Executives
#8

Yes. Thanks, Amin. I can take that. Currently, we're doing very well. We're engaging many sites. Site opening is on track. We're certainly on track to complete our data analysis by the end of the year. So that's just for the other people on the call, that's the first 40 patients interim analysis, and that's part of the dose optimization phase of the study, looking at 2 alternative doses of alemtuzumab, which is required by CDER. So yes, it's going well, and we're on track for Q4 data sharing.

Mohamad Amin Makarem

Analysts
#9

And for this dose optimization portion, as you evaluate the 2 alemtuzumab dose levels, what are the expectations? What differences should we expect between the 2 arms in terms of efficacy signal? Or are you expecting a meaningful difference in the safety? Or do you think the 2 arms are going to be generally in the same ballpark?

Adrian Kilcoyne

Executives
#10

We don't -- it's very difficult to say, and almost require a crystal ball to decide will there be a big difference in terms of both. We anticipate, however, that we have designed a very strong analysis plan to allow us to differentiate between the 2 components. That will be based on efficacy, safety, but also significantly important translational markers in terms of our CAR T expansion, host T-cell reconstitution, et cetera. So we do believe that we will be able to define an optimized dose by the end of the year. We don't think that will be a significant challenge. However, we know that both of these doses of alemtuzumab, very importantly, they work. It's all about dose optimization now. It's not about finding an effective dose.

Operator

Operator
#11

Our next question will come from Salveen Richter with Goldman Sachs.

Salveen Richter

Analysts
#12

Two questions from us. One is, could you provide more details on the Servier arbitration given the ruling that you have to engage in discussions with Allo regarding the direct licensing of 501? And help us understand how this impacts your eligibility for the $340 million in milestones into this interim data? And then secondly, could you talk about your decision to include CD52 preconditioning in both the studies in the context of safety as we see some of the competitors moving away from CD52.

Arthur Stril

Executives
#13

Salveen, this is Arthur. Thanks for the question. I'll take the question on the arbitration and then hand it over to Adrian for the clinical question. So just as a reminder, so the arbitral decision of December 2025 ruled on the partial termination of the license agreement. with respect to one product, which is UCART19 V1, also named ALLO-501 by Allogene. So this product has been brought back to Cellectis, and we are free to develop it or moving forward. The tribunal did not affect ALLO-501A or cema-cel. So we remain fully eligible for up to $340 million in development and sales milestones. That is obviously under the Servier agreement, which has been then sublicensed to Allogene. So this does not impact any eligibility to upcoming development and sales milestone as well as royalties down the line on cema-cel, but it gave us back UCART19 or ALLO-501 and we're free to do whatever we would like to do with it. And I will hand it over to Adrian for the clinical question.

André Choulika

Executives
#14

One thing I'd like to add, like during our thinking is that once you -- like the pivotal trial is effective, then the milestone should be triggered. So we're definitely expecting to have this $20 million milestone paid one day.

Adrian Kilcoyne

Executives
#15

Okay. I'll answer the alemtuzumab question. It's a great question. There's a few things -- just a few observations from our studies. One is that alemtuzumab is really important to optimize lymphodepletion. And as I said earlier, the more optimal lymphodepletion, the better the outcomes in terms of responses. You get a much deeper response. As part of our Phase I program, we did test a lymphodepletion regimen without alemtuzumab, and we failed to get any MRD-negative responses. So we know that alemtuzumab is very effective. The second part is you're saying some people are moving away from it. Now we believe, as I've said already, alemtuzumab is important, but we also believe that it's critically important that you get the right dose. It's like everything too much is always too much. So we have -- compared to other companies that are using alemtuzumab or alemtuzumab similars, we're using a much lower dose. We spent a lot of time already optimizing our doses. So we believe we've got the right balance between optimal efficacy, mitigating the risks that may be associated with that more enhanced lymphodepletion regimen. But also, let's not forget that alemtuzumab has been widely used in other clinical context. So it's got a very well-characterized safety profile, and we've been able to build into all our protocols very extensive risk mitigation. So we believe we've got the right strategy with our alemtuzumab. I cannot answer why others may have transitioned away from it.

Operator

Operator
#16

Our next question will come from Jack Allen with Baird.

Jack Allen

Analysts
#17

Congrats to the team on all the progress. I guess the first one I wanted to ask was on eti-cel and how we should think about the update in the fourth quarter of this year. Any additional color you're willing to provide as it relates to the breadth of the update and number of patients and the durability of follow-up there would be great. And then I have a quick follow-up as well.

Adrian Kilcoyne

Executives
#18

I can give you a top line update on top of the data we already have. As we've shared already at our current dose level, we're seeing a 63% complete remission rates, 88% overall response rates. By the end of the year, you will see data in cohorts with and without IL-2. Now we would acknowledge that 63% complete remission rate is actually a very strong result. And theoretically, we do not need to enhance that further, but we want to. We believe we can with the addition of low-dose IL-2. So by the end of the year, you will see -- we're currently expanded at our recruiting sites rather significantly we've doubled them. So we anticipate we will be able to get a reasonable -- and I cannot give you a number because I don't know what the recruitment rates are going to be. But we will be towards the latter part of the year also not in the staggering phase. So we would anticipate a significant increase in recruitment. You will see some durability data is what you asked about. But of course, for the patients in the later phase, there will hopefully be good durability signals. We have in our mind the durability levels we want to achieve, and it's around the 6-month mark. But hopefully, by the end of the year, you'll be able to see a very clear picture. I think the efficacy question has already been answered. The question is, can we enhance it even further into the 70s rather than in the mid-60s. But also, it will give us a very good signal is to -- is IL-2 really a potential game changer for allogeneic therapy. We obviously based on our preclinical data, which is going to be presented at AACR and again, some at EHA that we believe it could offer a really, really a fundamental improvement for allogeneic cell therapies.

Jack Allen

Analysts
#19

Great. And then we might be getting ahead of ourselves a little bit here. But on lasme-cel and the commercial opportunity there, I was wondering if you've been looking at Aucatzyl from Autolus. And if you have any thoughts around the initial commercial launch of that CD19 allo therapy in ALL.

Adrian Kilcoyne

Executives
#20

I can give it from a medical perspective, and Arthur, maybe you want to talk a bit more broadly on the commercial opportunity. Let's not forget, our patients are generally post-CD19 failures. So we don't -- we are not seeing this as a competition. We have a very differentiated target CD22. We believe the market is pretty saturated with CD19. It's not to Aucatzyl isn't a very good product. It is. Their data is very strong. But these patients are very difficult to treat. Many of them will relapse and they need to have an alternative target. So we believe that this is not a competing product, but actually a very differentiated one. In terms of market size, you may be aware, we're starting to look at earlier lines of therapy. We will have data starting to be generated next year on that frontline consolidation, very much aligned with what Allogene has done, which we think is a fantastic idea in the non-Hodgkin's lymphoma place. We will be looking at that in the ALL space. So we believe that's a really important part for those incredibly difficult to treat patients frontline.

Arthur Stril

Executives
#21

I can add, I think from a commercial launch perspective, you absolutely cannot compare an autologous launch to an allogeneic launch. And I think the 2 primary differences is, one, you need to have leukapheresis access for the patients, and this is slow. This is very competitive, and this is controlled by hospitals, not by the pharma companies, whereas obviously, with off-the-shelf, we will have manufactured a huge number of doses in advance so that at the time of launch, we can address all our clinical centers immediately without having to set up these cumbersome logistics aspect. And the second thing, obviously, is because of our off-the-shelf nature, we have very significant economies of scale, and we see it even at the pivotal stage, which will allow us to get extremely competitive gross margins and gross margin, which was much more the types of what pharma is now used to with small molecules and antibodies, whereas pricing of autologous has been difficult because of the significant cost of goods that make a very important impact on the margin. So I think the fact that we're off-the-shelf will allow us for a much smoother launch in terms of pure access due to the off-the-shelf nature and also in a much more economically competitive aspect due to the economies of scale and the very favorable gross margins. So I don't think Aucatzyl will be a very good comparator there.

André Choulika

Executives
#22

One thing I'd like to add, Jack, one thing that was interesting. I was like recently visiting a clinical center we're working with for acute lymphoblastic leukemia with Adrian. And there was one ALL patient that was sitting in the backlog waiting for an available apheresis spot since long time, [indiscernible] with non-Hodgkin lymphoma, DLBCL patient, multiple myeloma patient, autoimmune patient, et cetera. And this patient -- like ALL is a very aggressive disease. And still this stage, I'm not going to say which type of product it was about to be given to him because I don't know. But the fact is that this patient was like in real distress waiting for this like apheresis spot, and this is what you would consider as autologous launch of yet another CD19 autologous CAR T.

Operator

Operator
#23

Our next question will come from Silvan Tuerkcan with Citizens.

Silvan Tuerkcan

Analysts
#24

I just wanted to ask if you could just give us a brief recap of what you're expecting at EHA from these 2 programs that you have ongoing? Is it mainly longer follow-up?

Adrian Kilcoyne

Executives
#25

So you will be aware that we shared our data at our R&D Day back in October. We, after that, actually added some more patients in because we wanted to do some level of dose optimization in advance of our Phase II program. We wanted to ensure that, that lower dose is an acceptable dose to give to patients. So there are going to be an updated data set, the whole data set, including those additional patients. So it is -- I would consider it a level of progression from the original Phase I package that we presented. The data are not remarkably different, let me tell you, but it's an important addition to the data. The second thing that you -- we hope -- we're assuming that it will be accepted is really to try and understand what makes a product successful. And again, to build on the question from our Goldman Sachs colleagues, whether we're talking about alemtuzumab is the importance of that preconditioning and that day 0, not only from a level of lymphodepletion, but actually the environment in which you infuse your cells is critically important and in many ways, predicts the outcome. And the fact that we have now identified a really clear picture of optimal lymphodepletion, optimal environment in which you infuse your cells, we are now able to predict very early on our patient is going to respond. So all these data will be shared. So I think we're excited by it so far.

Operator

Operator
#26

Our next question will come from Sebastiaan van der Schoot with VLK.

Sebastiaan van der Schoot

Analysts
#27

So I wanted to ask you how you're looking at the possibility of applying your lymphodepletion procedure in the outpatient setting as well as the allogeneic CAR T. And then maybe can you provide some insight on how the partnership with AstraZeneca is going? Can we expect any updates in the next 24 months?

Adrian Kilcoyne

Executives
#28

I can take the first part of that, Arthur, in terms of outpatient setting. Right now, the regulatory authorities require inpatient delivery of products. And I think that's consistent with most autologous therapies as well in the CAR T space. And once there becomes some clinical confidence in how to use, will that transition into the outpatient setting? I think that may be a natural transition. But for that, you need a body of evidence. So hopefully, that will be provided not only by the Phase II program, but also by clinical usage from that. But again, I think it's a very different offering in that to Arthur's point, we do not leave that leukapheresis, et cetera. So I do think it will take some time for this to be in the outpatient setting. But assuming that we continue to have a fairly reassuring safety profile, there's no real reason why that this cannot in time, transition to the outpatient setting.

Sebastiaan van der Schoot

Analysts
#29

And I will take the question on AstraZeneca. So first of all, we're extremely pleased to count AstraZeneca as a strategic partner. As you have seen, they have continued to invest very heavily in the cell and gene therapy space, and they're one of the few companies that are betting very hard not only on cell and gene therapy, but also on off-the-shelf treatments. And I think we're very fortunate to have them as a key shareholder, but also a strategic R&D partner. The activities are going very well under the collaboration. There's a number of activities ongoing across a range of therapeutic area. Do not expect updates in the short term. This is at the request of AstraZeneca. They are essentially asking us for now to stay reasonably quiet, especially given the competitive nature of certain aspects that we are working on. But definitely, as we continue the dialogue with them around disclosure and as and when the time is right, we will be providing update. And I think you will be interested in seeing what has been brewing with them.

Operator

Operator
#30

[Operator Instructions] Our next question will come from Yanan Zhu with Wells Fargo.

Yanan Zhu

Analysts
#31

So first, regarding the lasme-cel pivotal study. I was wondering for the 4Q readout, is the focus there, the 3-month CRi from the 2 arms in that first 40-patient cohort? And then how do you manage the transition from completing that cohort to the start of the enrollment of the 80-patient portion for the optimal alemtuzumab dose, i.e., the final pivotal cohort. Will you be able to start enrolling before you have the data for the comparison of the 2 alemtuzumab cohorts? Yes. And then I have a follow-up.

Adrian Kilcoyne

Executives
#32

Great. This has some great questions there. First and foremost, no, the decision for dose optimization is not based on the 3-month CR/CRi. It's actually based on an earlier time cutoff of 8 weeks. And that's important. We will still be looking at efficacy, safety, but all the important translational markers predictive of not only short-term but long-term outcomes, we have those answers by that first 8 weeks. So it's a composite of many of those that we will be presenting. In terms of that transition from that to the longer phase of the study, we have built a lot of flexibility into the protocol to allow us to continue recruiting. So importantly, this does not mean our study has to stop while we're engaging with the regulatory authorities. The recruitment will continue for the remaining [indiscernible] patients.

Yanan Zhu

Analysts
#33

Great. Very helpful color. And the follow-up is mainly on the competitive landscape in terms of additional modalities in vivo CAR T specifically. Can you talk about the advantages or limitations for that modality or whether you have any interest in moving in that direction at early stage efforts?

Adrian Kilcoyne

Executives
#34

I can have an initial thoughts on this. And I think I know that Andre and Arthur also have views on the positioning of in vivo CAR. We're looking at incredibly sick patients with incredibly aggressive disease that need a lot of therapy. We don't believe these very difficult-to-treat tumors will be the definition for in vivo CAR. We think in vivo CAR certainly have a place. But will it be the right therapy for incredibly difficult to treat tumors and incredibly difficult to manage patients? We don't believe so. So yes, there will be in vivo CAR in some, maybe autoimmune may be a better destination. But any therapy that requires extensive gene editing and extensive patient management will probably -- maybe it will be a bridge too far for in vivo CAR. And I know, Andre, you have a view on this as well.

André Choulika

Executives
#35

Think the process like in vivo CAR is similar to autologous CAR T. The only thing it allows is essentially try to leapfrog apheresis, which is a great thing. It's like it's a huge market access option that is given here. Nevertheless, it will still be totally linked to the fitness of the cells of the patient or their presence. So if it doesn't respond to blina, if you can't do an autologous, then you go for in vivo, well, if the cells are not really very well functional, it's not going to work anymore because you need the T cells to be fit. That's one point. The second point is it will also be very much related to the current malignancies that are treated, which is essentially liquid tumors, B-cell malignancies or multiple myeloma. But the spread of this into solid tumor is going to be different. And finally, like there's a lot of papers that are coming out, like a lot of cells are transduced by these vectors that are not T cells. So like you need to restrict at the time. I think these technologies are going to come in the coming future, to try to restrict the cells to what needs to be transduced by the in vivo CAR T and not to go and deliver. For example, I've seen a paper recently that shows more vector inside, for example, hepatic cells expressing CAR and then in T cells. So if you do this worldwide like really broadband, then the questions at a time. And also like the genome is only 6.4 billion base pairs, like 23, 2x chromosomes. If you inject trillions and trillions of vectors in hundreds of patients or thousands of patients, it's like I hope that this will be safely integrated. If you go, for example, with autologous CAR T, you can master exactly when the cells are transduced. If you go for an allogeneic CAR, you have all the QC that lasts a long period of time to show that everything is totally square. If you make the product in vivo, which is the case here, then you have to master how the product is made and not to have a lot of byproducts all around the place. So that's like the initial concern. But I'm sure that this will be solved in coming decades.

Operator

Operator
#36

And at this time, there are no further questions in the queue. So I'll turn the meeting back over to our speakers for any additional or closing remarks.

Arthur Stril

Executives
#37

Thank you very much, everyone. I really appreciate all the questions here. As you can see, this will be a very exciting year 2026 with a number of updates from our partners and from ourselves. And I think we are poised for a new dawn of allogeneic CAR T cell therapy. So stay tuned for more updates and looking forward to further discussions as our progress unfolds this year. Thank you very much.

Operator

Operator
#38

Thank you. This brings us to the end of today's meeting. We appreciate your time and participation. You may now disconnect.

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