Cellectis Reports Third Quarter 2025 Financial Results and Provides Business Update

• Presented data underscore the potential of lasme-cel (UCART22) and eti-cel (UCART20x22) to improve outcomes in r/r B-ALL and r/r NHL:

Lasme-cel in r/r B-ALL (BALLI-01)

• • ORR of 68% with lasme-cel Process 2 (n=22), 83% at RP2D (n=12) and 100% in the target Phase 2 population (n=9)
  • Median OS of 14.8 months in patients who achieved MRD-negative CR/CRi
  • First interim analysis for the BALLI-01 trial expected in Q4 2026

Eti-cel in r/r NHL (NATHALI-01)

• • ORR of 86% and 57% CR rate (n=7)
  • Development update to be presented at the ASH 2025 annual meeting
  • Full Phase 1 dataset expected to be shared in 2026
    
    
• Servier arbitration: arbitral decision expected to be rendered on or before December 15, 2025
  
• Cash, cash equivalents and fixed-term deposits of $225 million as of September 30, 2025¹ provides runway into H2 2027

NEW YORK, Nov. 07, 2025 (GLOBE NEWSWIRE) -- Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene editing platform to develop life-saving cell and gene therapies, today provided financial results for the third quarter 2025 ending September 30, 2025 and business updates.

“We are proud of the promising data from our core clinical product candidates. Our lasme-cel program for r/r B-ALL and eti-cel program for r/r NHL demonstrated their ability to induce deep and meaningful responses, underscoring their potential to improve outcomes in diseases with high unmet medical needs” said André Choulika, Ph.D., Chief Executive Officer at Cellectis. “We look forward to sharing an additional development update on eti-cel at the ASH 2025 Annual Meeting and to provide the first interim analysis for the pivotal Phase 2 BALLI-01 trial in Q4 2026. Together, these milestones strengthen our leadership in allogeneic CAR-T innovation and position Cellectis for a transformative year ahead."

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¹ Cash, cash equivalents and fixed-term deposits include restricted cash of $4.4 million as of September 30, 2025 and fixed-term deposits of $168.2 million as of September 30, 2025, of which $137.6 million are classified as current financial assets and $30.6 million are classified as non-current financial assets (due to a fixed bank deposit investment maturing in October 2026, including accrued interest).

Pipeline Highlights

UCART Clinical Programs

BALLI-01 study evaluating lasme-cel (UCART22)

• Clinical data from the Phase 1 BALLI-01 study with lasme-cel for the treatment of relapsed or refractory B-cell acute lymphoblastic leukemia (r/r B-ALL), were presented at the Cellectis’ R&D Day that took place on October 16, 2025. The presented data position lasme-cel as a potentially game-changing therapy for patients with r/r B-ALL.
  
  In the Phase 1 of BALLI-01 study, 40 transplant ineligible third line or beyond (3L+) patients were dosed with lasme-cel: 18 patients (n=18) were dosed with product manufactured by an external CDMO (Process 1, or P1) and 22 patients (n=22) were dosed with Cellectis-manufactured product (Process 2, or P2).
  
  Highlights include:

• • Efficacy: lasme-cel demonstrated an overall response rate (ORR) of 68% with Process 2 product (n=22), and an ORR of 83% at the recommended Phase 2 dose (RP2D; n=12) and 100% in the target Phase 2 population (n=9)
    
    
  • Safety: in Phase 1 (n=40), lasme-cel was generally well tolerated; there was one case of grade 2 immune effector cell–associated hemophagocytic syndrome (IEC-HS), which resolved.
    
    
  • Durability: among patients who achieved minimal residual disease (MRD)-negative complete remission or complete remission with incomplete hematologic recovery (CR/CRi), median overall survival was 14.8 months.
    
    
  • Depth of response in target Phase 2 population: the CR/CRi rate was 56%, with approximately 80% of these patients achieving MRD-negative status.
    
    
  • Transplant eligibility in target Phase 2 population: all patients (100%) became eligible for transplant, and 78% proceeded to transplantation.

• The survival curve for this study suggests a clear benefit: patients who proceeded to hematopoietic stem cell transplantation (HSCT) after lasme-cel therapy showed a trend to longer overall survival than those who did not undergo transplant.
  
  The Phase 1 data showed that lasme-cel maintained its efficacy regardless of the number or type of prior lines of treatments, including CAR-T (60% of subjects), transplant (50% of patients), and blinatumomab (80% of subjects).

• Following successful End-of-Phase 1 meetings with the U.S Food and Drug Administration (FDA) and the European Medicines Agency (EMA), Cellectis provided a registration path for lasme-cel in r/r ALL. The first interim analysis for the Phase 2 of the BALLI-01 trial is expected in Q4 2026. Cellectis anticipates submitting a Biologics License Application (BLA) in 2028.

Commercial Opportunity for Lasme-cel

• As part of the R&D Day presentation, the Company discussed the potential commercial opportunity for lasme-cel in r/r B-ALL.
  
  If approved for commercialization, Cellectis estimates that lasme-cel could achieve up to approximately $700 million in potential peak gross sales across the U.S., EU4 (France, Germany, Italy, Spain) and UK in 2035, corresponding to an estimation of about 1,100 patients treated annually. Furthermore, gross peak sales could increase to up to approximately $1.3 billion with potential label expansion to second line and first line MRD+ consolidation. These estimates highlight that lasme-cel has the potential to drive meaningful growth of the CAR-T market in B-ALL, leading to a robust peak sales potential with attractive margins stemming from the allogeneic approach.

American Society of Hematology (ASH) 2025 annual meeting poster presentation

• On November 3, 2025, Cellectis announced the acceptance of an abstract for lasme-cel for poster presentation at the American Society of Hematology (ASH) 2025 annual congress, that will take place on December 6-9, 2025.
  
  
• The poster highlights the correlation between alemtuzumab exposure and depth of response in the difficult-to-treat r/r ALL patients who have received lasme-cel. Additionally, the data identifies a threshold exposure level of alemtuzumab above which achieving a complete response/complete response with incomplete hematologic recovery (CR/CRi) is more likely without any increase in toxicities.
  
  The poster presentation will occur on December 8, 2025, 6:00 PM - 8:00 PM ET, in Room OCCC - West Halls B3-B4.

NatHaLi-01 study evaluating eti-cel (UCART20x22)

• At the R&D Day, Cellectis unveiled preliminary data on eti-cel, its allogeneic CAR-T product candidate for relapsed or refractory non-Hodgkin lymphoma (r/r NHL), demonstrating an encouraging ORR of 86% and CR rate of 57% at the current dose level (n=7), with 4 out of 7 patients achieving a complete response. The preliminary high rate of complete responses underscores the potential of this innovative approach to transform outcomes for r/r NHL patients. Cellectis expects to present the full Phase 1 dataset for eti-cel, including low-dose IL-2 combination cohorts, in 2026.
  
  
• On November 3, 2025, Cellectis announced the acceptance of an abstract for poster presentation at ASH 2025.
  
  The poster provides a development update on eti-cel for patients with r/r NHL and outlines the addition of low dose interleukin-2 (IL-2) to further deepen and extend anti-tumor activity of eti-cel in patients with r/r NHL, supported by compelling preclinical data.
  
  The poster presentation will occur on December 7, 2025 at 6:00 PM – 8:00 PM ET, in Room OCCC – West Halls B3-B4.

Innovation

Circular single-stranded DNA (CssDNA) as a non-viral template for gene therapy

• In October 2025, Cellectis presented findings in a poster, highlighting the strong potential of circular single-stranded DNA (CssDNA) as a universal, efficient non-viral template for gene therapy, at the European Society of Gene and Cell Therapy (ESGCT) annual congress.
  
  Over the past decade, non-viral DNA template delivery has been used with engineered nucleases to target single-stranded DNA sequences in hematopoietic stem and progenitor cells (HSPCs).
  
  While developed for gene therapy purposes, so far this method has been restricted to gene corrections. To expand this scope, Cellectis developed an editing process using its gene editing technology and kilobase-long circular single-stranded DNA donor templates.
  
  The data presented show that:

• • CssDNA editing process achieved high gene insertion frequency in viable HSPCs.
  • CssDNA-edited HSPCs show a higher propensity to engraft and maintain gene edits in a murine model than adeno-associated viruses (AAV)-edited HSPCs.

TALE base editors (TALEB) off-targets in the nuclear genome

• At ESGCT 2025, the Company presented in a poster a comprehensive study of TALE base editors (TALEB) off-targets in the nuclear genome.
  
  TALE base editors (TALEB) are fusions of a transcription activator-like effector domain (TALE), split-DddA deaminase halves, and an uracil glycosylase inhibitor (UGI).
  
  These recent additions to the genome editing toolbox can directly edit double strand DNA, converting a cytosine (C) to a thymine (T) through the formation of an uracil (U) intermediate without the need of DNA break. Base editing has great potential in therapeutic applications. However, being able to avoid potential off-target effects is key toward this goal.
  
  To evaluate TALEB safety, Cellectis combined advanced bioinformatic predictions with multiple experimental approaches to investigate potential off-target effects in the nuclear genome of primary T cells.
  
  The study found no evidence of biases towards off-site C-to-T editing at sites flanked by CTCF binding sites, a key DNA-binding protein that regulates genome organization and gene expression at genome wide level.

These results provide a strong framework for the safe development of TALEB in therapeutic cell engineering, supporting their potential for future nuclear and mitochondrial applications.

AstraZeneca – Joint Research and Collaboration Agreement

• In its presentation during the Cellectis’ R&D Day held in October, AstraZeneca highlighted the significance of its strategic investment and research collaboration with Cellectis to accelerate its cell therapy and genomic medicine ambitions. The collaboration 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.

Servier arbitration

• With respect to the ongoing arbitration proceeding through the Centre de Médiation et d’Arbitrage de Paris, the arbitral decision is expected to be rendered on or before December 15, 2025.

Iovance

• In November 2025, Iovance reported that clinical results for IOV-4001, a PD-1 inactivated TIL cell therapy, in previously treated advanced melanoma patients are anticipated in the first quarter of 2026. Other potential indications for IOV-4001 are also in development.

Financial Results

Cash, cash equivalent and fixed-term deposits: As of September 30, 2025, Cellectis had $225 million in consolidated cash, cash equivalents, restricted cash and fixed-term deposits classified as current and non-current financial assets. The Company believes its cash, cash equivalents and fixed-term deposits will be sufficient to fund its operations into H2 2027.

This compares to $264 million in consolidated cash, cash equivalents, restricted cash and fixed-term deposits classified as current financial assets as of December 31, 2024, with no fixed-term deposits classified as non-current financial assets as of such date. This $39 million change includes $30.5 million of cash-in from our revenue, $7.1 million of interest received from our financial and cash-equivalent investments, $2.9 million cash-in from credit VAT, $1.5 million cash-in from other financial investments, offset by cash payments from Cellectis to suppliers of $35.5 million, Cellectis’ wages, bonuses and social expenses paid of $32.4 million, the payments of lease debts of $8.1 million, the repayment of the “PGE” loan of $4.0 million and the payments of capital expenditures for $3.0 million.

We currently foresee focusing our cash spending in supporting the development of our pipeline of product candidates, including the manufacturing and clinical development expenses of lasme-cel, eti-cel and potential new product candidates, and operating our state-of-the-art manufacturing capabilities in Paris (France) and Raleigh (North Carolina).

Revenues and Other Income: Consolidated revenues and other income were $67.4 million for the nine-month period ended September 30, 2025, compared to $34.1 million for the nine-month period ended September 30, 2024. This $33.3 million increase between the nine-month period ended September 30, 2024 and 2025 was mainly driven by the evolution of activities performed in connection with the Research Plans and fulfillment of our performance obligations under the AstraZeneca Joint Research and Collaboration Agreement. As a reminder, revenues as recorded in the nine-month period ended September 30, 2024 included a $5.4 million development milestone under the License Agreement with Servier.

R&D Expenses: Consolidated R&D expenses were $69.1 million for the nine-month period ended September 30, 2025, compared to $69.7 million for the nine-month period ended September 30, 2024, down by $0.6 million mainly driven by a decrease in purchases & external expenses of $2.1 million, offset by an increase of $1.7 million in R&D personnel expenses of which non-cash stock-based compensation increase by $1.0 million and wages and salaries increase by $0.7 million.

SG&A Expenses: Consolidated SG&A expenses were $15.0 million for the nine-month period ended September 30, 2025, compared to $14.2 million for the nine-month period ended September 30, 2024. The $0.8 million change is mainly due to a non-cash stock-based compensation increase of $0.7 million and an increase of $0.1 million in purchases and external expenses.

Other operating income and expenses: Other operating income increased slightly by $0.1 million between the nine-month periods ended September 30, 2024, and 2025.

Net financial gain (loss): We had a consolidated net financial loss of $25.6 million for the nine-month period ended September 30, 2025, compared to an $5.7 million net financial gain for the nine-month period ended September 30, 2024. This $31.2 million difference reflects mainly (i) a one-off $14.3 million gain in change in fair value of the derivative instrument component of the Subsequent Investment Agreement dated November 7, 2023 between us and AstraZeneca Holdings (the “SIA”), which was recognized in the nine-month period ended September 30, 2024, (ii) a $5.8 million loss in the fair value remeasurement of the warrants issued to the European Investment Bank ("EIB"), as required by our finance contract entered into with EIB in December 2022 recorded in nine months period as of September 2025 to be compared to $3.9 million fair value gain recorded in 2024, (iii) a $16.7 million increase in foreign exchange loss and a $2.0 million increase in foreign exchange gain over the period due to the USD volatility, partially offset by (iv) a $7.5 million decrease in loss on fair value of our investment in shares of Cibus, Inc., which was entirely sold in the first quarter of 2025.

Net Income (loss) Attributable to Shareholders of Cellectis: Consolidated net loss attributable to shareholders of Cellectis was $41.3 million (or a $0.41 net loss per share) for the nine-month period ended September 30, 2025, compared to a $42.7 million net loss (or a $0.49 net loss per share) for the nine-month period ended September 30, 2024. The $1.4 million change in net loss was primarily driven by (i) an increase in revenues and other income of $33.3 million offset by (ii) a $0.2 million increase in operating expenses and other operating income, (iii) a $31.2 million change from a net financial gain of $5.7 million as of September 30, 2024 to a net financial loss of $25.6 million as of September 30, 2025 and (iv) a decrease in deferred tax asset income of $0.5 million.

Adjusted Net Income (Loss) Attributable to Shareholders of Cellectis: Consolidated adjusted net loss attributable to shareholders of Cellectis was $37.4 million (or a $0.37 loss per share) for the nine-month period ended September 30, 2025, compared to a net loss of $40.4 million (or a $0.46 loss per share) for the nine-month period ended September 30, 2024.

Please see "Note Regarding Use of Non-IFRS Financial Measures" for reconciliation of GAAP net income (loss) attributable to shareholders of Cellectis to adjusted net income (loss) attributable to shareholders of Cellectis.

Note Regarding Use of Non-IFRS Financial Measures

Cellectis S.A. presents adjusted net income (loss) attributable to shareholders of Cellectis in this press release. Adjusted net income (loss) attributable to shareholders of Cellectis is not a measure calculated in accordance with IFRS. We have included in this press release a reconciliation of this figure to net income (loss) attributable to shareholders of Cellectis, which is the most directly comparable financial measure calculated in accordance with IFRS.

Because adjusted net income (loss) attributable to shareholders of Cellectis excludes stock-based compensation expense - a non-cash expense, we believe that this financial measure, when considered together with our IFRS financial statements, can enhance an overall understanding of Cellectis’ financial performance. Moreover, our management views the Company’s operations, and manages its business, based, in part, on this financial measure. In particular, we believe that the elimination of non-cash stock-based expenses from Net income (loss) attributable to shareholders of Cellectis can provide a useful measure for period-to-period comparisons of our core businesses. Our use of adjusted net income (loss) attributable to shareholders of Cellectis has limitations as an analytical tool, and you should not consider it in isolation or as a substitute for analysis of our financial results as reported under IFRS. Some of these limitations are: (a) other companies, including companies in our industry which use similar stock-based compensation, may address the impact of non-cash stock- based compensation expense differently; and (b) other companies may report adjusted net income (loss) attributable to shareholders or similarly titled measures but calculate them differently, which reduces their usefulness as a comparative measure. Because of these and other limitations, you should consider adjusted net income (loss) attributable to shareholders of Cellectis alongside our IFRS financial results, including Net income (loss) attributable to shareholders of Cellectis.

About Cellectis

Cellectis is a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies. The company utilizes an allogeneic approach for CAR T immunotherapies in oncology, pioneering the concept of off-the-shelf and ready-to-use gene-edited CAR T-cells to treat cancer patients, and a platform to develop gene therapies in other therapeutic indications. With its in-house manufacturing capabilities, Cellectis is one of the few end-to-end gene editing companies that controls the cell and gene therapy value chain from start to finish.

Cellectis’ headquarters are in Paris, France, with locations in New York and Raleigh, NC. Cellectis is listed on the Nasdaq Global Market (ticker: CLLS) and on Euronext Growth (ticker: ALCLS). To find out more, visit www.cellectis.com and follow Cellectis on LinkedIn and X.

Cautionary Statement

This press release contains “forward-looking” statements within the meaning of applicable securities laws, including the Private Securities Litigation Reform Act of 1995. Forward-looking statements may be identified by words such as “anticipate,” “believe,” “can,” “could,” “estimate,” “expectation,” “expected,” “illustrative,” “look forward,” “plan,” “potential,” “potentially, “positioned,” “projected,” “suggest,” and “will,” or the negative of these and similar expressions.  These forward-looking statements, which are based on our management’s current expectations and assumptions and on information currently available to management, include statements regarding the market market opportunities with respect to lasme-cel (and the assumptions on which such determinations are based, including with respect to addressable populations and potential pricing), the potential of the Phase 2 BALLI-01 trial to be a registrational phase, the advancement, timing and progress of clinical trials (including with respect to patient enrollment and follow-up), the timing of our presentation of data and submission of regulatory filings (including without limitation, the date of BLA filing), the sufficiency of cash to fund operations, the potential benefit of our product candidates and technologies, and the financial position of Cellectis. These forward-looking statements are made in light of information currently available to us and are subject to significant risks and uncertainties, including with respect to the numerous risks associated with biopharmaceutical product candidate development. Among these are significant risks that the BALLI-01 Phase 1 data may not be validated by data from later stage of clinical trials and that our product candidate may not receive regulatory approval for commercialization. Particular caution should be exercised when interpreting results from Phase 1 studies and results relating to a small number of patients – such results should not be viewed as predictive of future results. With respect to the sufficiency of cash, cash equivalent and fixed-term deposits to fund our operations, which we refer to as our runway, we note that our operating plans, including product development plans, may change as a result of various factors. Furthermore, many other important factors, including those described in our Annual Report on Form 20-F as amended and in our annual financial report (including the management report) for the year ended December 31, 2024 and subsequent filings Cellectis makes with the Securities Exchange Commission from time to time, which are available on the SEC’s website at www.sec.gov, as well as other known and unknown risks and uncertainties may adversely affect such forward-looking statements and cause our actual results, performance or achievements to be materially different from those expressed or implied by the forward-looking statements. Except as required by law, we assume no obligation to update these forward-looking statements publicly, or to update the reasons why actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future.

For further information on Cellectis, please contact:

Media contacts:
Pascalyne Wilson, Director, Communications, + 33 (0)7 76 99 14 33, [email protected]
Patricia Sosa Navarro, Chief of Staff to the CEO, +33 (0)7 76 77 46 93

Investor Relations contact:
Arthur Stril, Chief Financial Officer & Chief Business Officer, [email protected]

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