Cellectis Reports Full Year 2025 Financial Results and Provides a Business Update

Pivotal Phase 2 with lasme-cel in r/r B-ALL (BALLI-01 trial) ongoing

• Phase 1: 83% ORR at RP2D and 100% ORR in the target Phase 2 population
• In target Phase 2 population: 100% of patients became eligible to transplant
• Pivotal Phase 2 first interim analysis expected in Q4 2026
• BLA submission anticipated in 2028

Phase 1 with eti-cel in r/r NHL (NATHALI-01 trial) ongoing

• Best-in-class dual allogeneic CAR-T cell product targeting CD20 & CD22
• At current dose level, 88% ORR; 63% CR rate after 2+ prior lines of therapy
• 93% of subjects had prior CD19 CAR-T
• Low-dose IL-2 cohort to be included in; Full Phase 1 dataset expected in Q4 2026

Partnerships

• Servier (through Allogene): Pivotal randomized Phase 2 ALPHA3 trial with cema-cel in 1L consolidation in LBCL: interim futility analysis evaluating MRD clearance and early safety results planned for April 2026
• AstraZeneca: Activities progressing under the Joint Research and Collaboration Agreement

 

• Cash, cash equivalents and fixed-term deposits of $211 million as of December 31, 2025[1] provides runway into H2 2027
• Conference call scheduled on March 20, 2026 at 8:00 am ET / 1:00 pm CET

 

 

New York, NY – March 19, 2026 - 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 fourth quarter and full year 2025, ending December 31, 2025 and provided a business update.

“Lasme-cel demonstrated a potentially transformative efficacy profile in one of oncology’s most challenging settings, achieving 100% overall response rate in the target Phase 2 population. Critically, lasme-cel converted all patients in the target population into transplant-eligible candidates. The pivotal Phase 2 is now enrolling, and with a BLA submission anticipated in 2028, lasme-cel is on a clear regulatory path to potentially becoming the first off-the-shelf CAR-T therapy to address this high unmet medical need” said André Choulika, Ph.D., Co-Founder and Chief Executive Officer of Cellectis. “With interim Phase 2 data for lasme-cel in r/r B-ALL, and full Phase 1 data for eti-cel in r/r NHL, both expected in Q4 2026, we are entering an important year for Cellectis, as we advance our ambition to bring life-saving off-the-shelf CAR-T therapies to patients who have run out of options”.

Allogeneic CAR-T Pipeline

Lasme-cel in relapsed or refractory B-cell acute lymphoblastic leukemia (r/r B-ALL) - BALLI-01

• In October 2025, Cellectis presented full Phase 1 lasme-cel clinical data at the Cellectis’ R&D Day. The presented data position lasme-cel as a potentially game-changing therapy for patients with r/r B-ALL. Data highlighted:

Strong efficacy:

• 68% overall response rate (ORR) with lasme-cel Process 2, manufactured internally (n=22)
• 83% ORR at the recommended Phase 2 dose (RP2D) (n=12)
• 100% ORR in the target Phase 2 population (n=9)

In the target Phase 2 population, the complete response or complete remission with incomplete hematology recovery (CR/Cri) rate was 56%, with approximately 80% of these patients achieving minimum residual disease (MRD)-negative status

Favorable safety profile:

• Low rates of ≥ grade 3 cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS) at 2.5% and 5% respectively

Transplant eligibility in target Phase 2 population:

• All patients became eligible for transplant

Strong survival benefit:

• 14.8 months median overall survival (OS) in patients who achieved MRD-negative CR/CRi

The first interim analysis for the pivotal Phase 2 of the BALLI-01 trial is expected in Q4 2026 (n=40). Cellectis anticipates submitting a Biologics License Application (BLA) in 2028.

 

Eti-cel in relapsed or refractory non-Hodgkin lymphoma (r/r NHL) – NATHALI-01

• In December 2025, Cellectis presented encouraging Phase 1 preliminary data of eti-cel at the American Society of Hematology (ASH) annual meeting. The data showcased the potential of eti-cel in r/r NHL patients who have relapsed following multiple lines of therapy including, for 93% of patients, an autologous CD19 CAR-T, with an 88% ORR and 63% CR at the current dose level (n=8).
• Cellectis is initiating patient enrollment in the cohort with low dose interleukin-2 (IL-2) support to evaluate the potential to further enhance the already high response rates and durability of response in patients with r/r NHL.
• Cellectis expects to present the full Phase 1 dataset in 2026, including results from the IL-2 combination.

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

• In November 2025, Cellectis published a Nature Communications article establishing cssDNA as a highly efficient non-viral DNA donor template, for gene insertion in hematopoietic stem and progenitor cells (HSPCs).

While viral vectors such as AAV6 are commonly used for gene insertion, they raise safety and efficacy concerns. Over the past decade, non-viral DNA templates delivery has emerged as promising alternatives.

Cellectis’ research results mark a pivotal advance toward next-generation non-viral cell and gene therapies.

Key findings:

• Superior efficiency: cssDNA achieved over 40% knock-in efficiency, outperforming linear DNA by 3-5 times.
• Versatility: the process successfully targets multiple loci in HSPCs and primary T cells.
• Better persistence: in murine models, cssDNA-edited cells showed superior engraftment and edit maintenance compared to AAV6-edited cells.

TALE base editors (TALEB) safety and precision

• At ESGCT 2025, Cellectis presented a comprehensive safety study on TALE base editors (TALEB), which enable precise C-to-T DNA editing without causing double-strand breaks.

Key study highlights:

• Safety assessment: researchers used advanced bioinformatics and experimental models to track potential off-target effects in the nuclear genome of primary T cells.
• No bias detected: the study found no evidence of unintended editing at CTCF binding sites, which are critical for genome organization and gene expression.

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

Partnerships

AstraZeneca – Joint Research and Collaboration Agreement

• Activities are progressing under the Joint 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.

Servier (through its sublicensee Allogene) – Anti-CD19 CAR-T

• Under the Servier Agreement, Cellectis is eligible to up to $340 million in development and sales milestones as well as low double-digit royalties on sales.
• In December 2025, an arbitral tribunal has issued its decision in the arbitration proceedings against Les Laboratoires Servier and Institut de Recherches Internationales Servier IRIS SARL (“Servier”), relating to the License, Development and Commercialization Agreement entered into between Servier and Cellectis on March 6, 2019, as amended (the “Servier Agreement”). The Tribunal ruled on a partial termination of the License Agreement with respect to product UCART19 V1 (also referred to as “ALLO-501” by Allogene) and provided that Cellectis shall, at Allogene’s request, engage in good-faith discussions regarding the granting of a direct license to product UCART19 V1. All other claims brought by the parties were dismissed.

Allogene – Anti-CD70 CAR-T

• According to Allogene, the TRAVERSE trial in renal cell carcinoma has completed enrollment in its Phase 1b cohort, and Allogene is currently exploring partnering opportunities to advance the asset.

Iovance

• According to Iovance, new data across several pipeline programs is anticipated throughout 2026, including a Phase 1/2 trial investigating IOV-4001, a PD-1 inactivated TIL therapy, in previously treated advanced melanoma and NSCLC.

Corporate

Annual Shareholders Meeting

• On June 26, 2025, Cellectis held a Shareholders General Meeting. At the meeting, during which approximately 57% of voting rights were exercised, resolutions 1 through 23 and resolutions 25 and 26 were adopted, while resolution 24 was rejected, consistent with the recommendations of the Board of Directors. The detailed results of the vote and the resolutions are available on Cellectis’ website: https://www.cellectis.com/en/investors/general-meetings/

Board composition

• The Cellectis Shareholders' Meeting appointed Mr. André Muller as a director of the Company's Board of Directors. At the close of this meeting, the term of Mr. Axel-Sven Malkomes expired, and the previously announced resignation of Mr. Pierre Bastid became effective. In connection with these changes to the Board of Directors, the Board of Directors appointed Mr. André Muller, Dr. Donald Bergstrom, and Dr. Rainer Boehm as the members of the Company’s Audit Committee.