Cellectis Reports Financial Results for the First Quarter 2025

• Lasme-cel (UCART22) Phase 1 dataset and late-stage development strategy expected in the third quarter of 2025
• Eti-cel (UCART20x22) Phase 1 study in relapsed or refractory B-cell non-Hodgkin lymphoma (r/r NHL) ongoing with readout expected in late 2025
• AstraZeneca partnership: R&D activities ongoing on three programs – one allogeneic CAR T for hematological malignancies, one allogeneic CAR T for solid tumors, and one in vivo gene therapy for a genetic disorder
• Cellectis will present novel non-viral gene editing and base editing research at the 2025 ASGCT annual meeting
• Cash position of $246 million as of March 31, 2025[1] provides runway into H2 2027

 

New York, NY – May 12, 2025 - 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 first quarter 2025, ending March 31, 2025, and provided a business update.

« We are making progress in our wholly-owned clinical studies and in the three programs under our strategic partnership with AstraZeneca. We will continue to focus our efforts and resources on advancing these core programs and are looking forward to the results expected over the next few months » said André Choulika, Ph.D., Chief Executive Officer at Cellectis.

Pipeline Highlights

UCART Clinical Programs

BALLI-01 study evaluating lasme-cel (UCART22) in relapsed or refractory B-cell acute lymphoblastic leukemia (r/r B-ALL)

• On April 15, 2025, the International Nonproprietary Names (INN) Expert Committee of the World Health Organization (WHO) selected lasmecabtagene timgedleucel (lasme-cel) as recommended international non-proprietary name of UCART22 drug substance.
• Cellectis continues to focus on the enrollment of patients in the BALLI-01 study and expects to present the Phase 1 dataset and late-stage development strategy for lasme-cel in r/r B-ALL in the third quarter of 2025.

 

NATHALI-01 study evaluating eti-cel (UCART20x22) in relapsed or refractory B-cell non-Hodgkin lymphoma (r/r NHL)

• On April 15, 2025, the INN Expert Committee of the WHO selected etivelcabtagene erigedleucel (eti-cel) as recommended international non-proprietary name of UCART20x22 drug substance.
• Cellectis continues to focus on the enrollment of patients in the NATHALI-01 study and expects to present a Phase 1 readout for eti-cel in r/r NHL in late 2025.

Research Data & Preclinical Programs

Novel Non-Viral Gene Editing and Base Editing Research

• On April 28, 2025, Cellectis announced the presentation of research data on TALEN®-mediated non-viral transgene insertion for advancing cellular and gene therapies, and advancements in genetic editing using TALE base editors (TALEB), at the American Society of Gene and Cell Therapy (ASGCT) annual meeting that will be held on May 13-17, 2025 in New Orleans. The data will be presented in two posters:

TALEN®- Mediated non-viral Transgene Insertion for the Advancement of Cellular and Gene Therapies

• In this work, Cellectis combines TALEN®-mediated gene editing with non-viral transgene insertion for advancing cellular and gene therapies and explores gene insertion-efficacy and cellular health using single-stranded DNA (ssDNA) for payload delivery in different cell types.
• This innovative approach has the potential to address the challenges associated with traditional lentiviral viral methods or AAV-mediated transgene insertion such as manufacturing constraints, potential genomic toxicities or limited payload size.

High fidelity C-to-T editing with TALE base editors

• TALE base editors (TALEB) are fusions of a transcription activator-like effector domain (TALE), split-DddA deaminase halves, and an uracil glycosylase inhibitor (UGI). The C-to-T class of TALEB edits double-stranded DNA by converting a cytosine (C) to a thymine (T) and does not involve a DNA strand nick. Cellectis has developed a method to evaluate TALEB activity, analyzing factors affecting its efficiency. Using precise ssODN knock-in in primary T cells, the method assesses how target sequence composition and spacer variations impact TALEB performance.
• Overall, the results of this study enhance the control and use of TALEB, allowing for the design of highly efficient and specific TALEB compatible with future potential therapeutic applications.

The abstracts are live on the ASGCT website. The posters will be available on Cellectis’ website the first day of the event.

Partnerships

AstraZeneca Joint Research and Collaboration Agreement

Research and development activities are ongoing under three cell and gene therapy programs under the joint research and collaboration agreement entered into by Cellectis and AstraZeneca in November 2023: one allogeneic CAR T for hematological malignancies, one allogeneic CAR T for solid tumors, and one in vivo gene therapy for a genetic disorder.