Cellectis S.A. (CLLS) Q2 2025 Earnings Report, Transcript and Summary

Good day, everyone, and welcome to today's Cellectis' Second Quarter 2025 Earnings Call. [Operator Instructions] Please note this call may be recorded. And I will be standing by, if you should need any assistance. It is now my pleasure to turn the conference over to Arthur Stril, Interim Chief Financial Officer. Please go ahead.

Good morning. and welcome, everyone, to Cellectis' Second Quarter 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 6-K and press release reporting our financial statements for the 6 months period ended June 30, 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 presentation of our BALLI-01 and NatHaLi-01 clinical trials, the timing and ability to progress our clinical trial into a later phase, the progress of our R&D activities under the AstraZeneca partnership, the timing and outcome of our arbitration with Servier and sufficiency of cash to fund operations. 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, 2024, and subsequent filings Cellectis makes with the SEC from time to time. I would now like to turn the call over to Andre.

Thank you, Arthur. Good morning, and thank you, everyone, for joining us today. I would like to begin this call with an important announcement. On October 16, 2025, Cellectis will be hosting an Investors R&D Day in New York City. Our leadership team, along with key opinion leaders will present the Phase I data set and late-stage development strategy for lasme-cel, UCART22, in relapsed or refractory acute lymphoblastic leukemia, and will share insights on the Company's vision and differentiated capabilities. Cellectis' Investors R&D Day is scheduled as an in-person only event. However the recording of the event will be made available in the following days after the event. Despite the challenges of the biotech market, our teams have remained focused on advancing research and developing solutions for patients with unmet medical needs. In July 2025, Cellectis completed end of Phase I discussion with both the United States Food and Drug Administration and European Medicines Agency. We are excited to prepare for the initiation of the pivotal Phase II trial for lasme-cel, UCART22, in relapsed or refractory acute lymphoblastic leukemia in the second half of this year. Regarding the NatHaLi-01 study, which is assessing eti-cel program, UCART20x22, in relapsed or refractory non-Hodgkin lymphoma. Cellectis anticipates presenting data from Phase I and outlining its late-stage development strategy late 2025. On the partnership front, Research and development activities are ongoing under the three cell and gene therapy programs under our Joint Research and Collaboration Agreement with AstraZeneca: one allogeneic CAR-T for hematological malignancies, one allogeneic CAR-T for solid tumors, and one in vivo gene therapy for a genetic disorder. Regarding our licensing agreement involving Servier and sublicensee, Allogene, and following Servier's decision in September 2022 to seize the development of the licensed CD19 Products. We've initiated an arbitration before the Paris Mediation and Arbitration Center to protect our interests. We're asking the tribunal to terminate the agreement with Servier and to award fair compensation for the losses incurred due to the lack of development of the licensed products and the payment of the milestone which we consider due under the agreement. The arbitral decision is expected to be rendered on December 15, 2025 or before. Earlier this quarter, Cellectis announced that during its Annual Shareholders Meeting, Mr. André Muller has been appointed as a member of the company's Board of Directors. I'm pleased to welcome André at Cellectis Board. His extensive experience will be an invaluable asset to the company. We would also like to express our gratitude to Mr. Pierre Bastid and Mr. Axel-Sven Malkomes, who have terminated the directorship for their esteem commitment to Cellectis. It has been a huge honor and a pleasure for us all to work with them during their term. Their contribution over the past years has been exceptional and their precious support has greatly contributed to the advancement of the company's strategy. With that, I would like to turn the call over to Dr. Adrian Kilcoyne, our Chief Medical Officer, who will give an overview of our clinical trials. Adrian, please go ahead.

Thank you, Andre. As Andre mentioned, Cellectis continues to focus its development efforts on the BALLI-01 and NatHaLi-01 studies. Recruitment to the dose escalation component of the Phase I BALLI-01 study which is evaluating UCART22 in relapsed or refractory B-cell acute lymphoblastic leukemia has been completed. This study addresses an important unmet need for patients who have relapsed following multiple prior lines of therapy, including a CD19 bispecific or autologous CAR-T and a few, if any, other treatment options. The Phase I data set has been shared with both FDA and EMA as part of the end of Phase I and scientific advice meetings. Following productive interactions with both agencies, we now have a clear path to commence our pivotal Phase II study later this year. We have set up additional trial sites in order to accelerate accrual into the Phase II study, and we'll continue to focus on expanding sites in the United States and Europe, including the United Kingdom. We anticipate having sites open for recruitment into our Phase II study by the end of the year. We are also planning to publicly share the full Phase I dose escalation data set during our R&D Day, as highlighted by Andre earlier. Additional data from the Phase I study has also been submitted for consideration of presentation at the ASH Annual Conference in the fourth quarter. We also continue to enroll in the NatHaLi-01 study of our dual CAR-T asset, UCART20x22 in relapsed or refractory non-Hodgkin's lymphoma. This study is addressing an important unmet needs for patients who have relapsed following previous lines of therapy, including when available, an autologous CD19 CAR-T. Data from this program has also been submitted for presentation at the ASH Annual Conference in the fourth quarter. We are expanding our clinical trial sites to accelerate recruitment and we hope to transition to Phase II preparation in 2026. 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 second quarter 2025. Arthur, please go ahead.

Thank you, Adrian. We are pleased about the progress of our wholly owned product candidates, lasme-cel and eti-cel, as well as over the three cell and gene therapy programs in partnership with AstraZeneca. In this context, we are excited to be hosting an R&D Day on October 16 to focus on the Phase I data set and late-stage development strategy of lasme-cel as we prepare to launch a pivotal Phase II in H2 2025. We also expect to provide an update on eti-cel by the end of the year. Finally, the arbitral decision in our arbitration with Servier is expected to be rendered on or before December 15, 2025. Importantly, we are well positioned financially to execute on our pipeline as our cash, cash equivalents and fixed-term deposits as of June 30, 2025, remain sufficient to fund our operations into H2 2027. Our cash. Cash equivalents, restricted cash and fixed-term deposits classified as current and non-current financial assets as of June 30, 2025, amounts to $230 million compared to $264 million as of December 31, 2024. This $33.2 million decrease is mainly due to $13.4 million of cash in from our revenue and $5.1 million of interest income, offset by cash payments from Cellectis to suppliers of $23.2 million. Cellectis' wages, bonuses and social expenses paid of $23.6 million. The payments of lease debt of $5.4 million and the repayment of the PGE loan of $2.6 million. You are invited to refer to our press release for figures related to consolidated net loss attributable to shareholders of Cellectis for the 6 months ended June 30, 2025. We're very much looking forward to welcoming you at our Investor R&D Day as well as to providing further updates later this year. And now I would like to turn the call over to Andre for closing remarks.

Thank you, Arthur. To close out this call, I would like to reiterate how excited we are to have one of our first product moving into Phase II pivotal trial powered registration. I'm confident about the continued progress of our ongoing clinical trials in hematological malignancies as well as how excited we are about our strategic collaboration with AstraZeneca. At Cellectis, we strongly believe that our product candidates, our technologies and our in-house manufacturing capabilities will lead us and our partners to a paradigm shift for patients with hard-to-treat cancer and genetic disorders, positioning us the forefront of this promising medical and scientific field. With that, I would like to open the call for Q&A.

[Operator Instructions] Our first question will come from Gena Wang with Barclays.

Congrats on the progress. So I have two questions. One is regarding the Servier arbitration decision by December 15. Could you contextualize the different scenarios and your likely actions? The second question is regarding the lasme-cel. You already met with both FDA and EMA regarding the pivotal Phase II trial design. Could you share a bit high-level thoughts with us? What could be the path forward there?

Gena, this is Andre speaking. I'd like to answer the first question. It's complicated to answer your question for the simple reason that, I guess that probably there is still thinking on the way the things are going to go. And I don't want to draw here any kind of scenario, knowing that any kind of scenario can happen from one side or another. And I personally hope that we're going to prevail in this arbitration, it means that we're going to get back our CD19 products and that we're going to have the compensation for like the loss incurred by the non-development by Servier. However, it's very difficult to forecast exactly what could be the decision at the end in these type of arbitration. So I'd like to keep the door open as much as possible for any kind of scenarios. But we believe that we're totally right in our position knowing that there is nothing has been happening so far. A question mark on, -- if I start like putting some scenario, it means, I already thought about the fact that we can have some back position, but we don't have any back position. And for the question for, like the interaction with the FDA and the EMA, I'd like to defer this to Adrian. Adrian?

Yes. Thanks, Andre. Great question. Yes, we've interacted with both EMA and FDA. And just in the spirit of transparency, the EMA, it was a written feedback. They felt the, our submission was clear, and therefore, they could give us very clear guidance on that and a very productive feedback that was. The FDA was a face-to-face meeting. And while I can't go into the details, but certainly at the R&D Day on October 16, we will share a lot more detail around the study design, et cetera. But a few top-level take homes is, we got clear agreement on end points. We had -- there was no concerns raised around our statistical plan. There was no issues raised about the size of the database we would have as we go to BLA with -- based on what we had submitted. So overall, in our view, there is a very clear path forward for our Phase II program. So again, I'd reiterate very productive meetings with both regulatory authorities, who are generally aligned in their feedback, which is always a bonus, I feel.

Our next question comes from Kelly Shi with Jefferies.

This is Anqi Yu on behalf of Kelly Shi from Jefferies. I have two questions. For the R&D Day, what data points should we expect? And supposing we don't have the trial design at this point, you will disclose on the R&D Day. Then how do you consider the dynamic in the FDA that could potentially have any impacts on the pivotal trials?

This is Arthur. I will start on the R&D Day and then let Adrian add any points and also on the FDA question. So the purpose of the R&D Day is really twofold. We want to be presenting the full Phase I data set for lasme-cel that will include all patients that have been dosed so far and a particular focus on the patients at the recommended Phase II dose, and that will be safety, efficacy and durability data set. And the second point is, we want to present what we call the late-stage development strategy, which indeed will include the Phase II design, the patient population and then provide some color on the path to BLA. So it will be both a look into the path that all the Phase I set, but also look into the future as to the plans for the product all the way to commercialization. And then, I'll hand it over to Adrian, if you want to add any color and address the FDA question.

I mean, I think, if I can answer with a question. Are there any barriers that became evident in our interactions with either regulatory authorities to progressing to Phase II and pivotal? Absolutely not. I think the regulatory authorities, both of them acknowledged the high unmet need that is -- that exists within the space, we're going -- positioning that product. But also, I think it was very clear from the tone of these meetings that they're broadly supportive of what we're doing. So again, to get alignment on endpoints, to get alignment broadly on the study design as we had presented it to them and patient numbers, overall, we don't see any significant roadblocks. So again, alignment between ourselves and the regulatory authorities, we -- and again, as Arthur said, we will be presenting the study design in detail, discussing through those endpoints. But I think, again, I would restate that the registration path for this product seems relatively clear. And of course, everything is dependent on ultimately Phase II data.

Our next question will come from Jack Allen with Baird.

Great. Congratulations on the progress. I guess maybe I'll start with one on UCART22 and the upcoming R&D Day. I wanted to ask what -- how the team is thinking about the bar for success in relapsed or refractory. I know, there's some data about best response out there and also some autologous CD19 CAR-Ts. But as you move toward the pivotal study, what are you looking at as the bar for success? And what kind of expectations do you have for durability of response from UCART22? And how much follow-up should we expect on Phase I patients when we get that data update in the back half of this year and then, I guess, in mid-October? And then I have a quick follow-up as well.

Thanks, Jack, for the great questions. Adrian, do you want to take this?

Sure. So there's many questions in there. Suffice to say, again, more detail on the endpoints, the timing of those endpoints will be shared in detail during the October 16, R&D Day. But durability of response in the allogeneic setting is really important. And on sharing the data with both regulatory authorities, they are very clear on the approach we're taking will give you the adequate data in order to support a registration, assuming the data is positive based on our -- what we're showing. Of course, this is also, there will be a longer-term follow-up in all these. You know we're committed to a 15-year follow-up with these patients. But also within the trial, we have -- we will have a longer survival. It's a long overall survival follow-up for a number of years. So -- but that is not part of the primary analysis. So the primary analysis, as we will share is a much more short-term surrogate endpoint, which, again, has been agreed with the regulatory authorities.

Got it. That's very helpful. And then maybe for Adrian, again, with ALLO, correct it, as he sees fit. I'd love to hear any high-level thoughts you have on the recent decision by Allogene to move away from the CD52 lymphodepletion. How are you thinking about that as it relates to your programs moving forward? And would you anticipate including alemtuzumab in a potential pivotal study of UCART22?

Well, I can start on that, Arthur and Andre. Yes, we've been following the Allogene story very closely. We believe that alemtuzumab is really important as part of the lymphodepletion regimen. And we want to be really cautious in how we interpret our approach in the context of Allogene's approach. I want to stress that these are very different positionings of the products. We're talking very late-stage ALL, and we're talking very late stage NHL compared to the Allogene approach is much earlier. While we -- and we also don't know very much around the pharmacokinetics of the ALLO product. But it appears to be at a much higher dose than we gave within our clinical trials. And of course, the third thing, which is really, really important is that everything we do is based on risk benefits. So the risk-benefit assessment within the Allogene program is very different to the risk-benefit assessment we see in our programs. So all in all, very difficult. I think it would be unfair to draw any comparisons between these programs. We are fairly confident about our risk-benefit assessment across our programs with alemtuzumab. And we believe that adds a critical improvement in responses, which we're fairly confident in our approach moving forward.

Yes. And if I can add something, I think, Adrian, you're spot on, obviously, on the dose, but it's also important to -- remember that these are different products. And I think our strategy has always been from the get-go to secure direct access to alemtuzumab, which is something that we've done with Sanofi a few years back. So we know we have access to actual alemtuzumab. ALLO-647 is a different product. And honestly, we're not exactly aware as to how it compares, what are the glycosylation patterns, what are the -- what is the structure of the antibody. And so it's very difficult to compare apples to oranges. And we're very pleased to be moving forward with actual alemtuzumab.

Great. I guess just maybe one last one on the topic. Any high-level thoughts on ways you could mitigate potential infection risk in your study? I know the CD52 lymphodepletion can lead to a longer depletion of T cells. Have you given any further thought on the protocols you can put in place to mitigate infection risk?

Yes. I mean, it's part of all our trials. We have a mandatory prophylaxis. So that's already built in. We have significant risk mitigation strategies already built into our trials. So yes, I think we've already addressed much of that. But of course, we remain vigilant.

Our next question comes from Yanan Zhu with Wells Fargo Securities.

Great. Just maybe a follow-up on the FDA discussion for the BALL program. Wondering -- I know you will provide more details at the R&D Day. Wondering if the population for the pivotal trial, is that specific population like post CD19 CAR-T? Or is that a more broader population? And also in terms of the patient number, could we look to the most recent CD19 autologous CAR-T programs for BALL for the rough range? Or could the trial be smaller than that?

Yes. Good questions. So as you're probably aware, we're looking at a very late stage treatment. So -- but we're going for a fairly broad patient population in terms of age cutoff. And again, we'll share the details with that, we would anticipate there will be a significant number of lines of therapy for most patients in our clinical trial. And that will include, obviously, CD19, CARs, et cetera. So you'll see detailed breakdown of this at the R&D Day. So I would encourage you to come along. In terms of the number of patients, I think the number of patients in our trial is driven by two things. One is the powering of our trials. And the two is the requirement of the safety database, which often takes precedence. So I think, your suggestion that, looking at other autologous CD19, CARs in this space will give you, while not entirely accurate, it will give you a ballpark as to what kind of numbers we need to have in these kind of trials. And it's driven by the size of the safety database rather than the assumed statistical powering.

Great. That's super helpful. And then as a follow-up for the Servier program arbitration discussion. I was just wondering, could you give us a sense of the size of the milestone payments that potentially could be awarded at that point?

Yes. Thanks, Yanan. Unfortunately, as this is an ongoing legal matter, we're not going to be able to give more details on this. Sorry.

No problem. Congrats on the progress.

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

Just two from my side. The first one is a follow-up on Allogene decision to not move forward with the anti-CD2 or CD52 antibody. Can you maybe give some insight whether there's been any feedback from the regulators from inclusion of the anti-CD52 in your treatment regimen? And do you expect that there might be another conversation with the FDA after these filings from Allogene? And then, I have a follow-up.

Thanks, Sebastiaan. Adrian, do you want to take this one?

Sebastiaan, I just want to make sure I've understood the question. Could you just repeat it?

Sure. So it comes down to whether you expect that this decision from Allogene that came after your conversation with the regulator, but that can still influence the design of the pivotal study with the use of the anti-CD52 antibody.

No. We -- honestly, we don't believe so. Again, we have an established safety profile and an established risk-benefit assessment. As Arthur already stated, this is -- the Allogene product is different. And if we assume they may be even similar, our dosing levels are significantly lower than we see with the Allogene product. And again, the regulatory authorities have reviewed our full in the Phase I package including detailed look at the safety profile. So I don't see that there will be -- we don't foresee any changes, based on what we've seen with Allogene.

Got it. Very clear. And then, maybe regarding the cash runway and cash position. Could you indicate whether that incorporates the entire completion of the pivotal study for lasme-cel?

Yes. Great question. So the cash runway into H2 2027 does include pivotal studies. Actually, we've made assumptions both for lasme- cel and for eti-cel. So all the costs are fully loaded in that front. We've also been very prudent, as we've always done on cash in from milestones and non-dilutive funding. So this one have been probabilized, so there is potential for upside there if they do materialize. And we will provide the full detailed time lines of the Phase II at the R&D Day. But to answer your question, yes, the runway does include the pivotal studies.

Great. And then maybe a last one, it's on the data set for eti-cel by year-end. Can you just give some color on the size of that data set? Will it be similar in patient size? I understand that there will not be enough follow-up, but in terms of patient size, will be similar to the lasme-cel disclosure back in October.

Yes, I'll take that, Arthur. So obviously, lasme-cel is a much more -- it's early candidate with much more patients within the Phase I program than eti-cel. So you will be anticipating smaller patient numbers. And again, we will -- you'll see based on our submissions to ASH, you'll see much more of that data then.

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

This is Mark on for Salveen. I have, I guess, a follow-up on eti-cel. There's been a lot of news from autologous dual-targeting CAR- Ts recently. And -- what are your thoughts? How do you view that data? Is there any read-through to eti-cel? And sort of beyond the allogeneic autologous, sort of, difference? How do you think eti-cel is differentiated in the dual targeting space?

Yes, it's a great question. We believe we've got a very well differentiated product with eti-cel. We believe our positioning is very clear. It's a later line diffuse large B-cell lymphoma, most likely, certainly within the non-Hodgkin lymphoma space. We think our -- and again, when you see what's presented at ASH, it will be assuming it's accepted. You'll see a clear strategy, which I think differentiates the product significantly from the current batch of autologous products. So I can't really say much more until we -- you see what we will be hopefully presenting at ASH.

If I can add -- and thanks, Mark, for the great question. I think the -- I'm sorry, I'm going to state the obvious, but the big important piece about eti-cel is, it does not target 19. A lot of the dual targeting data we've seen is like 19, 20, 19, 22. So I think it's great, but it's yet again hitting 19. So the primary competitors to 19, 20 or 19, 22 are going to be the approved autologous 19 and potentially some of the ALLO-19. I think where eti-cel is pretty unique to our knowledge at this stage is that it is a 20 and 22. And so it will be particularly relevant to physicians who have hit 19 once and will then want to -- or twice. And we want to alternate the targets. I think, the other important thing to remember is, right now, 19 is firmly entrenched in the second line. This is primarily YESCARTA and BREYANZI. If Allogene is successful with this first-line consolidation approach, to which, by the way, we have a vested interest in. There is a potential for 19 to come already up to the first-line consolidation. And then there will be a very, very strong need to hit orthogonal targets. So coming at it with an off-the-shelf alternative that does not require an additional round of leukapheresis, harvesting of the patient, slots, et cetera. So pure off-the-shelf alternative that does not target 19, I think in our universe of NHL and LBCL is pretty differentiated and unique.

It appears we have no further questions at this time. I would now like to turn the program back over to our presenters for any additional or closing remarks.

Well, we would like to thank you all. And we definitely -- there is a very rich second half this year for Cellectis. So please stay tuned and we hope that you will be all at our R&D Day event on October 16 and probably wait to what's going to happen for Cellectis by the end of the year. So a lot of like rich event things that will come up. Thank you very much for your attention, and have a good day.

Thank you, ladies and gentlemen. This concludes today's event. You may now disconnect.