Cellectis - Earnings Call - Q2 2025
August 5, 2025
Transcript
Speaker 3
Good morning and welcome, everyone, to Cellectis's second quarter 2025 business update and financial results conference call. Joining me on the call today are Dr. André 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 six-month 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's financial outlook, including the presentation of our BALLI-01 and Natalie01 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 André.
Speaker 4
Thank you, Arthur. Good morning, and thank you, everyone, for joining us today. I'd 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 1 dataset and late-stage development strategy for 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, a recording of the event will be made available in the following days after the event. Despite the challenges of the biotech markets, our teams have remained focused on advancing research and developing solutions for patients with unmet medical needs. In July 2025, Cellectis completed the end of Phase 1 discussion with both the U.S.
Food and Drug Administration and the European Medicines Agency. We are excited to prepare for the initiation of pivotal Phase 2 trials for UCART22 in relapsed or refractory acute lymphoblastic leukemia in the second half of this year. Regarding the NATHALI-01 study, which is assessing UCART20x22 in relapsed or refractory non-Hodgkin’s lymphoma, Cellectis anticipates presenting data from Phase 1 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 its sublicensee, Allogene, and following Servier's decision in September 2022 to cease 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 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 Müller 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 Bastide and Mr. Axel Sven-Malkomes, who have terminated the directorship for their esteemed 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.
Speaker 3
Thank you, André. As André 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 1 BALLI-01 study, which is evaluating UCART22 in relapsed 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 1 dataset has been shared with both FDA and EMA as part of the end of Phase 1 and scientific advice meetings. Following productive interactions with both agencies, we now have a clear path to commence our pivotal Phase 2 study later this year.
We have set up additional trial sites in order to accelerate accrual into the Phase 2 study and will 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 2 study by the end of the year. We are also planning to publicly share the full Phase 1 dose escalation dataset during our R&D Day, as highlighted by André earlier. Additional data from the Phase 1 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 refractory non-Hodgkin’s lymphoma. This study is addressing 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 2 preparation in 2026. With that, I would like to hand the call over to Arthur Stril, Cellectis’s Chief Financial Officer and Chief Business Officer, for an overview of our financials for the second quarter 2025. Arthur, please go ahead.
Speaker 7
Thank you, Adrian. We're pleased about the progress of our wholly owned product candidates, UCART22 and UCART20x22, as well as of 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 1 dataset and late-stage development strategy of UCART22 as we prepare to launch a pivotal Phase 2 in H2 2025. We also expect to provide an update on UCART20x22 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, amount 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's wages, bonuses, and social expenses paid of $23.6 million, the payments of lease debts 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 six 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.
I would like to turn the call over to André for closing remarks.
Speaker 4
Thank you, Arthur. To close out this call, I would like to reiterate how excited we are to have one of our first products moving into Phase 2 pivotal trial powered registration and 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 at the forefront of this promising medical and scientific field. With that, I'd like to open the call for Q&A.
Speaker 8
Thank you. At this time, if you would like to ask a question, please press star one on your telephone keypad. You may remove yourself from the queue at any time by pressing star two. Once again, that is star one to ask a question. We will pause for just a moment to allow questions to queue. Thank you. Our first question will come from Gina Wang with Barclays Bank PLC. Your line is open.
Speaker 0
Thank you for taking my questions and congrats on the progress. 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 UCART22. You already met with both FDA and the EMA regarding the pivotal Phase 2 trial design. Could you share a little bit high-level thoughts with us? What could be the path forward there?
Speaker 3
Hi, Gina. This is André speaking. I would 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 that things are going to go. I don't want to draw here any kind of scenario, knowing that any kind of scenario can happen from one side or another. 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 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 types of arbitrations. I'd like to keep the door open as much as possible for any kind of scenarios.
We believe that we're totally right in our position, knowing that there is nothing that has been happening so far. The 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. For the questions for like the interaction with the FDA and the EMA, I'd like to defer this to Adrian. Adrian?
Yes, thanks, André. Yeah, great question. Yes, we've interacted with both EMA and FDA. Just in the spirit of transparency, the EMA, it was a written feedback. They felt our submission was clear and therefore they could give us very clear guidance on that and very productive feedback that it was. The FDA was a face-to-face meeting. While I can't go into the details, at the R&D day on October 16, we will share a lot more detail around the study design, et cetera. A few top-level take-homes is we got clear agreement on endpoints. There were no concerns raised around our statistical plan. There were no issues raised about the size of the database we would have as we go to BLA based on what we had submitted. Overall, in our view, there is a very clear path forward for our Phase 2 program.
I'd reiterate very productive meetings with both regulatory authorities who are generally aligned in their feedback, which is always a bonus, I feel.
Speaker 0
Thank you.
Speaker 8
Thank you. Our next question comes from Kelly Shi with Jefferies LLC. Your line is open.
Speaker 5
Hi, this is Anjali on behalf of Kelly Shee from Jefferies LLC. Thank you for taking our questions. I have two questions. For the R&D Day, what data points should we expect? Supposing we don't have the trial design at this point, you will disclose on the R&D Day. How do you consider the dynamic in the FDA that could potentially have any impacts on the pivotal trials? Thank you.
Speaker 7
Good morning. This is Arthur. I will start on the R&D day and then let Adrian add any points and also on the FDA question. The purpose of the R&D day is really twofold. We want to be presenting the full Phase 1 dataset for UCART22 that will include all patients that have been dosed so far and a particular focus on the patients at the recommended Phase 2 dose. That will be safety, efficacy, and durability dataset. The second point is we want to present what we call the late-stage development strategy, which indeed will include the Phase 2 design, the patient population, and then provide some color on the path to BLA. It will be both a look into the past at all the Phase 1 set, but also a look into the future as to the plans for the product all the way to commercialization.
I'll hand it over to Adrian if you want to add any color and address the FDA question.
Speaker 3
Yeah, 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 2 and pivotal? Absolutely not. I think the regulatory authorities, both of them, acknowledged the high unmet need that exists within the space we're going to position in that product. I think it was very clear from the tone of these meetings that they're broadly supportive of what we're doing. 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. Alignment between ourselves and the regulatory authorities. We, as Arthur said, will be presenting the study design in detail and discussing through those endpoints. I would restate that the registration path for this product seems relatively clear.
Of course, everything is dependent on ultimately the Phase 2 data.
Speaker 5
Great. Thank you.
Speaker 8
Thank you. Our next question will come from Jack Kilgannon Allen with Robert W. Baird & Co. Incorporated. Your line is open.
Speaker 6
Great. Thanks so much for taking the questions. Thanks. Congratulations on the progress. I guess maybe I'll start with one on UCART22 and the upcoming R&D day. I wanted to ask how the team is thinking about the bar for success in relapsed refractory ALL. I know there's some data about best to know out there and also some autologous CD19 CAR-Ts. As you move towards the pivotal study, what are you looking at as the bar for success? What kind of expectations do you have for durability of response from UCART22? How much follow-up should we expect on Phase 1 patients when we get that data update in the back half of this year and, I guess, in mid-October? I have a quick follow-up as well.
Speaker 7
Thanks, Jack, for the great questions. Adrian, do you want to take this?
Speaker 3
Sure. There are many questions in there. Suffice to say, more detail on the endpoints and the timing of those endpoints will be shared in detail during the October 16th R&D Day. Durability of response in the allogeneic setting is really important. On sharing the data with both regulatory authorities, they're very clear on the approach we're taking and will give you the adequate data in order to support a registration, assuming the data is positive based on what we're showing. Of course, there will be a longer-term follow-up in all these. We're committed to a 15-year follow-up for these patients. Also, within the trial, we will have a longer overall survival follow-up for a number of years, but that is not part of the primary analysis. The primary analysis, as we will share, is a much more short-term surrogate endpoint, which has been agreed with the regulatory authorities.
Speaker 6
That's very helpful. Maybe for Adrian, again, I'll let Arthur take that 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? Would you anticipate including alemtuzumab in a potential pivotal study of UCART22?
Speaker 3
I can start on this, Arthur and André. We've been following the Allogene story very closely. We believe that alimtuzumab is really important as part of the lymphoid depletion measurement. 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, which is much earlier. We also don't know very much around the pharmacokinetics of the Allogene product. It appears to be at a much higher dose than we give within our clinical trials. The third thing, which is really, really important, is that everything we do is based on risk-benefit. The risk-benefit assessment within the Allogene program is very different to the risk-benefit assessment we see in our programs.
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 program with alimtuzumab. We believe that all adds a critical improvement in responses, which we're fairly confident in our approach moving forward.
Speaker 7
Yeah. If I can add something, I think, Adrian, you're spot on, obviously, on the dose. It's also important to remember that these are different products. 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. We know we have access to actual alemtuzumab. ALLO-647 is a different product. Honestly, we're not exactly aware as to how it compares, what are the glycosylation patterns, what is the structure of the antibody. It's very difficult to compare apples to oranges. We're very pleased to be moving forward with actual alemtuzumab.
Speaker 6
Great. Thank you so much. 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 protocols you could put into place to mitigate infection risk?
Speaker 3
Yeah, I mean, as part of all our trials, we have a mandatory prophylaxis. That's already built in. We have significant risk mitigation strategies already built into our trials. I think we've already addressed much of that. Of course, we remain vigilant.
Speaker 6
Got it. Great. Thank you so much for taking the questions. Congratulations again on the program.
Speaker 8
Thank you. Our next question comes from Yanan Zhu with Wells Fargo Securities. Your line is now open.
Speaker 2
Oh, great. Thanks for taking our questions. Maybe a follow-up on the FDA discussion for the BALL program. I know you will provide more details at the R&D day. I'm wondering if the population for the pivotal trial, is that a specific population, like post-CD19 CAR-T, or is that a broader population? 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? Thanks.
Speaker 3
Yeah, good questions. Excuse me. As you're probably aware, we're looking at a very late-stage treatment. 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. You'll see a detailed breakdown of this at the R&D day. I'd 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 two is the requirement of the safety database, which often takes precedence.
I think your suggestion that looking at other autologous CD19 CARs in this space will give you, while not entirely accurate, a ballpark as to what kind of numbers we need to have in these kind of trials. It's driven by the size of the safety database rather than the assumed statistical powering.
Speaker 2
Great. That's super helpful. 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? Thank you.
Speaker 7
Yeah, thanks, Yanan. Unfortunately, as this is an ongoing legal matter, we're not going to be able to give more details on this. Sorry.
Speaker 2
No problem. Thanks for taking the question and congrats on the progress.
Speaker 7
Thank you so much.
Speaker 8
Thank you. Our next question will come from Sebastiaan van der Schoot with Jefferies LLC. Your line is open.
Speaker 1
Hi, team. Congrats on the progress. Thank you for taking my questions. Just from my side, the first one is a follow-up on the 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 on inclusion of the anti-CD52 in your treatment regimen? Do you expect that there might be another conversation with the FDA after these findings from Allogene? I'll follow up.
Speaker 7
Thanks, Sebastiaan. Adrian, you want to take this one?
Speaker 3
Sebastiaan, I just want to make sure I've understood the question. Could you just repeat it?
Speaker 7
Sure. It comes down to whether you expect that this decision from Allogene that came after your conversation with the regulator, whether that can still influence the design of the pivotal study with the use of the anti-CD52 antibody.
Speaker 3
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, the Allogene product is different. Even if we assume they may be even similar, our dosing levels are significantly lower than we see with the Allogene product. The regulatory authorities have reviewed our full end-of-phase-1 package, including a detailed look at the safety profiles. I don't see that there will be, we don't foresee any changes based on what we've seen with Allogene.
Speaker 7
Got it. Thank you. Very clear. Maybe regarding the cash runway and cash position, could you indicate whether that incorporates the entire completion of the pivotal study for UCART22?
Speaker 3
Yeah, great question. The cash runway into H2-2027 does include pivotal studies. Actually, we've made assumptions both for UCART22 and for UCART20x22. 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-diluted funding. These ones have been probabilized. There is potential for upside there if they do materialize. We will provide the full detailed timelines of the Phase 2 at the R&D Day. To answer your question, yes, the runway does include the pivotal study.
Speaker 7
Great. Maybe the last one, it's on the dataset for UCART20x22 by year end. Can you just give some color on the size of that same dataset? Will it be similar in patient size? I understand that there will not be enough follow-up. In terms of patient size, will it be similar to the UCART22 disclosure back in October?
Speaker 3
Yeah, I'll take that, Arthur. UCART22 is a much more, it's our lead candidate with much more patients within the Phase 1 program than UCART20x22. You will be anticipating smaller patient numbers. You'll see, based on our submissions to ASH, you'll see much more of that data then.
Speaker 7
Great. Thank you so much.
Speaker 8
Thank you. Our next question will come from Salveen Reitzer with Goldman Sachs. Your line is open.
Speaker 6
Hey, good morning. This is Mark on for Salveen. Thank you so much for taking our question and congrats on the quarter. I have a, I guess, a follow-up on UCART20x22. There's been a lot of news from autologous dual-targeting CAR-Ts recently. What are your thoughts? How do you view that data? Is there any read-through to UCART20x22? Beyond the allogeneic autologous sort of difference, how do you think UCART20x22 is differentiated in the dual-targeting space? Thanks.
Speaker 3
Yeah, it's a great question. We believe we've got a very well-differentiated product with UCART20x22. We believe our positioning is very clear. It's a later line diffuse large B-cell lymphoma, most likely, certainly within the non-Hodgkin's lymphoma space. We think our, and again, when you see what's presented at ASH, it'll 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. I can't really say much more until you see what we will be hopefully presenting at ASH.
Speaker 7
If I can add, and thanks, Mark, for the great question. I think the, and sorry, I'm going to state the obvious, but the big important piece about UCART20x22 is it does not target 19. A lot of the dual-targeting data we've seen is like 19, 20, 19, 22. I think it's great, but it's yet again hitting 19. The primary competitors to a 19, 20 or a 19, 22 are going to be the approved autologous 19 and potentially some of the Allogene 19. I think where UCART20x22 is pretty unique to our knowledge at this stage is that it is a 20 and 22. It will be particularly relevant to physicians who have hit 19 once and will then want to, or twice, and will want to alternate the targets. The other important thing to remember is right now 19 is firmly entrenched in the second line.
This is primarily Yescarta and Brionzy. 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. There will be a very, very strong need to hit orthogonal targets. Coming at it with an off-the-shelf alternative that does not require an additional round of leukapheresis, harvesting of the patient's slots, et cetera. A pure off-the-shelf alternative that does not target 19, I think in our universe of NHL and LBCL is pretty differentiated and unique.
Speaker 6
That is it. Thank you.
Speaker 8
Thank you. It appears we have no further questions at this time. I'll now like to turn the program back over to our presenters for any additional or closing remarks.
Speaker 4
Thank you all. There is a very rich second half this year for Cellectis. Please stay tuned. We hope that you will be all at our R&D Day event on October 16th and probably wait to see what's going to happen for Cellectis by the end of the year. A lot of rich event things will come up. Thank you very much for your attention and have a good day.
Speaker 8
Thank you, ladies and gentlemen. This concludes today's event. You may now disconnect.