Cellectis Reports Financial Results for the Fourth Quarter and Full Year 2024 and Provides a Business Update

[1] Cash position includes cash, cash equivalents, restricted cash and fixed-term deposits classified as current -financial assets. Restricted cash was $4.6 million as of December 31, 2024. Fixed-term deposits classified as current-financial assets was $115.8 million as of December 31, 2024.

 

New York, NY – March 13, 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 fourth quarter and full year 2024, ending December 31, 2024, and provided a business update.

“2024 has been an important year for Cellectis: we are now developing three programs in collaboration with AstraZeneca. So far, we announced the start of one allogeneic CAR T for hematological malignancies, one program of an allogeneic CAR T for solid tumors, and first program of an in vivo gene therapy for a genetic disorder. 

We are thrilled to grow this strategic collaboration with AstraZeneca, a top leader of the pharmaceutical industry, aimed at shaping the future of next generation of cell and gene therapy. We are excited about the huge opportunities this partnership will bring in the months ahead” said André Choulika, Ph.D., Chief Executive Officer at Cellectis.

“AstraZeneca’s additional equity investment of $140M in Cellectis and the drawdown of the final tranche of the finance contract with the European Investment Bank (EIB) give us confidence that our cash runway is funded until mid-2027.

In 2025, Cellectis will continue to focus its efforts and expenses on advancing its core clinical trials BALLI-01 and NATHALI-01, while building the next generation of genomic medicines to address areas of high unmet patient needs within, our partnership with AstraZeneca, and within our proprietary preclinical pipeline. 

We expect to present the Phase 1 data set and late-stage development strategy in the third quarter of 2025 for UCART22, for the treatment of r/r ALL. For our product candidate UCART20x22, in r/r NHL, we continue to focus on the enrollment of patients and expects readout in late 2025.”

Pipeline Highlights

UCART Clinical Programs

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

• In July 2024, the FDA designated UCART22 as a drug for a Rare Pediatric Disease (RPDD). This designation may allow a “Priority Review Voucher” at the time of Biologics License Application (BLA). The FDA also granted Orphan Drug Designation (ODD) to UCART22 product candidate for the treatment of ALL. In June 2024, Cellectis received Orphan Drug Designation (ODD) from the European Commission (EC) for UCART22, for the treatment of ALL.
• In August 2024, the FDA granted ODD to Cellectis’ CLLS52 (alemtuzumab), an Investigational Medicinal Product (IMP) used as part of the lymphodepletion regimen associated with UCART22. The importance of adding alemtuzumab to the lymphodepletion regimen has been demonstrated in Cellectis’ BALLI-01 study, where the addition of this lymphodepletion agent to the fludarabine and cyclophosphamide regimen was associated with sustained lymphodepletion and significantly higher UCART22 cell expansion allowing for greater clinical activity.
• These designations for UCART22 and CLLS52 mark an important step towards developing allogeneic CAR T products that would be readily available for all patients.
• 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 UCART22 in relapsed or refractory ALL in the third quarter of 2025.

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

• Cellectis continues to focus on the enrollment of patients in the NATHALI-01 study and expects to present the Phase 1 dataset and late-stage development strategy for UCART20x22 in relapsed or refractory NHL in late 2025.

UCART123 in relapsed or refractory acute myeloid leukemia (r/r AML)

• In November 2024, the Company decided to focus its current development efforts on the BALLI-01 and NATHALI-01 studies and therefore to deprioritize the development of UCART123. Up to now, this study has provided important insights into the role of CD123-targeted allogeneic CAR T therapy in relapsed refractory acute myeloid leukemia and the future development of our allogeneic CAR-T platform.

Research Data & Preclinical Programs

Innovative strategy for T cell engineering to enhance efficacy against solid tumors

• In February 2025, Cellectis presented a poster ‘SMART CAR T’ strategy to enhance efficacy against solid tumors at the American Association for Cancer Research – Immuno-oncology (AACR-IO). Leveraging its proprietary TALEN® gene editing technology, Cellectis has developed CAR T cells capable of expressing a tumor-specific, inducible IL-2 variant immunocytokine. This novel approach aims to enhance CAR T cell efficacy against solid tumors by localizing IL-2 activity within the tumor microenvironment, potentially offering a safer and more effective method to boost CAR T cell expansion and anti-tumor activity.

Breaking barriers in solid tumors with SMART allogeneic CAR T-cells

• In November 2024, Cellectis presented pre-clinical data to enhance CAR T cell activity against solid tumors while preventing potential toxicity at the Society for Immunotherapy of Cancer (SITC) Annual Meeting. Utilizing TALEN® gene editing, Cellectis developed allogeneic CAR T-cells that leverage tumor microenvironment cues to target cancer cells effectively. Key findings include tethering cytotoxic activity to the tumor area and confining IL-12 to the tumor microenvironment, which enhances CAR T-cell proliferation and reduces side effects. This innovative approach aims to improve the safety and efficacy of CAR T-cell therapies for solid tumors.

Controlling C-to-T editing with TALE base editors

• In October 2024, Cellectis showcased pre-clinical data that permit the design of an efficient and specific TALE base editors (TALEB) at the European Society of Gene and Cell Therapy (ESGCT) annual congress.
• This novel strategy characterizes C-to-T conversion efficiencies and assesses TALEB activity using TALEN®-mediated ssODN knock-in in primary T cells. This research has led to improved understanding of TALEB, enabling the design of more efficient and specific tools with potential therapeutic applications.

MUC1 CAR T-cells for treating Triple-Negative Breast Cancer

• In September 2024, Cellectis published a scientific article in Science Advances suggesting that TALEN®-edited MUC1 CAR T-cells could be a potential treatment option for advance-stage triple negative breast cancer (TNBC) patients with limited therapeutic options. Cellectis described its CAR T-cell engineering strategy using TALEN® and synthetic biology to multi-armor CAR T-cells with synergistic functionalities to overcome the immunosuppressive tumor microenvironment of solid tumors.

SMART DUAL CAR T-cell approach for treating recalcitrant solid tumors

• In August 2024, Cellectis published a Molecular Therapy article on SMART DUAL CAR T-cell approach for recalcitrant solid tumors, while mitigating potential safety risks. This innovative strategy uses allogeneic CAR T-cells with a dual targeting mechanism to effectively infiltrate and target triple-negative breast tumors while minimizing on-target, off-tumor toxicity. This approach would address key challenges in solid tumor therapy, including low CAR T-cell infiltration and antigen heterogeneity.

Partnerships

Servier and Allogene – Allogeneic CAR T

Allogene’s investigational oncology products utilize Cellectis technologies.

cema-cel: ALPHA3 Trial in Large B-Cell Lymphoma (LBCL)

• Allogene announced that the pivotal Phase 2 ALPHA3 trial, which initiated in June 2024, now has 40 sites activated and continues to generate strong enthusiasm from both community cancer centers and academic institutions. This groundbreaking study is evaluating consolidation treatment with cema-cel as part of the 1L treatment regimen for patients with LBCL with minimal residual disease (MRD) after standard 1L treatment with R-CHOP or other chemoimmunotherapy. This randomized trial will enroll approximately 240 patients and is designed to demonstrate a meaningful improvement in event free survival (EFS) in patients treated with cema-cel relative to patients who receive the current standard of care (observation). Allogene announced that the lymphodepletion selection and futility analysis are anticipated around mid-2025, that efficacy analyses from the ALPHA3 trial are expected to occur in 2026 and will include an interim EFS analysis monitored by the independent Data Safety Monitoring Board in 1H 2026 and the data readout of the primary EFS analysis around YE 2026, and that a potential biologics license application (BLA) submission is targeted for 2027.
• In February 2025, the Journal of Clinical Oncology published data from Allogene’s Phase 1 ALPHA/ALPHA2 trials of cema-cel in relapsed/refractory LBCL, demonstrating durable responses comparable to approved autologous CD19 CAR T therapies.

ALLO-316: TRAVERSE Trial in Renal Cell Carcinoma (RCC)

• In November 2024, Allogene announced positive Phase 1 data from the TRAVERSE trial highlighting a manageable safety profile and significant anti-tumor activity of ALLO-316 in heavily pretreated patients with advanced or metastatic renal cell carcinoma. Allogene further announced that additional data from the Phase 1b expansion cohort, which is evaluating safety and efficacy of ALLO-316 at DL2 (80M CAR T cells), is expected to be announced in mid-2025.

AstraZeneca – Joint Research and Collaboration Agreement

• Research and development activities under three cell and gene therapy programs have started under the joint research and collaboration agreement entered into by Cellectis and AstraZeneca in November 2023 (the “AZ JRCA”): one allogeneic CAR T for hematological malignancies, one allogeneic CAR T for solid tumors, and one in vivo gene therapy for a genetic disorder.
• Under the AZ JRCA, $47m have been paid to Cellectis up to December 31, 2024 (of which $25m upfront and $22m reached development milestones for the three initial programs), in addition to reimbursement of research costs incurred under the AZ JRCA.

Corporate Updates

AstraZeneca’s Additional Investment

• Following clearance from the French Ministry of Economy and satisfaction of all other closing conditions, AZ Holdings completed in May 2024 the additional equity investment of $140 million in Cellectis, as previously announced by Cellectis on November 1 and 15, 2023. Under the subsequent investment agreement, AZ Holdings agreed to make a further equity investment in Cellectis of $140 million by subscribing for two newly created classes of convertible preferred shares of Cellectis: 10,000,000 Class A Preferred Shares and 18,000,000 Class B Preferred Shares, in each case at a price of $5.00 per share (the “Additional Investment”). The Additional Investment closed on May 6, 2024.
• Until they convert into ordinary shares, the Class A Preferred Shares have single voting rights and will not be eligible for double voting right under any circumstances, and the Class B Preferred Shares do not carry voting rights for a period of 74 years, except with respect to any distribution of dividends or reserves. Both class of preferred shares have a liquidation preference (if any liquidation surplus remains after repayment of Cellectis’ creditors and of par value to all shareholders) and are convertible at any time, at AstraZeneca's election (and with 12 months’ prior notice in the case of the Class B Preferred Shares), into the same number of ordinary shares with the same rights as the outstanding ordinary shares.
• As of December 31, 2024, considering the ordinary shares held by AZ Holdings as well as all Class A Preferred Shares, which AZ Holdings has the right to acquire within the next 60 days, AZ Holdings beneficially owns approximately 32% of our ordinary shares. As of December 31, 2024, considering the ordinary shares held by AZ Holdings and giving effect to the conversion of all Class A Preferred Shares and Class B Preferred Shares without regarding for when they may first be converted, AZ Holdings would beneficially own approximately 44% of our ordinary shares. As of December 31, 2024, AZ Holdings may exercise voting power with respect to approximately 30% of the voting rights outstanding with respect to our share capital (inclusive of (i) the ordinary shares held by AZ Holdings and (i) the voting rights of the Class A Preferred Shares, which vote together with our ordinary shares).

Drawdown of the second and third tranche of the European Investment Bank financing

• In January 2024, Cellectis drew down the second tranche of €15 million (“Tranche B”) under the credit facility agreement for up to €40 million entered into with the European Investment Bank (“EIB”) on December 28, 2022.
• In December 2024, Cellectis drew down the third tranche of €5 million (“Tranche C”) under the credit facility agreement for up to €40 million entered into with the EIB on December 28, 2022. With the drawdown of Tranche C, Cellectis has drawn down the full €40 million available under the Finance Contract.

Appointments

• In May 2024, pursuant to the SIA and implemented by the Company's shareholders decision dated December 22, 2023, Mr. Marc Dunoyer and Mr. Tyrell Rivers serve on the Company's board of directors as members designated by AZ Holdings.
• In May 2024, Cellectis announced the appointment of Mr. Arthur Stril as Interim Chief Financial Officer, following the resignation of Bing Wang, Ph.D. Mr. Arthur Stril was appointed Chief Financial Officer and Chief Business Officer in January 2025.
• In August 2024, Cellectis announced the appointment of Adrian Kilcoyne, M.D., MPH, MBA as its Chief Medical Officer.

Annual Shareholders Meeting

• On June 28, 2024, Cellectis held a shareholders’ general meeting at the Biopark auditorium in Paris, France.
• At the meeting, during which approximately 40% of shares were exercised, resolutions 1 through 28 were adopted and resolution 29 was rejected, consistent with the recommendations of the management. The detailed results of the vote and the resolutions are available on Cellectis’ website: https://www.cellectis.com/en/investors/general-meetings/

2024 Financial Results

(download the PDF below to access Financial Results)