Cellectis Provides Financial Results for the Second Quarter 2024

• ODD and RPDD granted by the FDA and ODD granted by the European Commission to UCART22 for the treatment of ALL

 

• ODD granted by the FDA to CLLS52 (alemtuzumab) for ALL treatment

 

• Cash position of $273 million as of June 30, 2024[1]; cash runway projection into 2026

 

New York, NY – August 6, 2024 - 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 business updates and reported financial results for the six-month period ending June 30, 2024.

"Over the past months, we have achieved a significant milestone with the granting of ODD designations by the Food and Drug Administration and the European Commission, complemented by the FDA’S Rare Pediatric Disease Designation. We have overcome major challenges, which reflects our ongoing commitment to innovation. Driven by an unwavering belief in our ability to revolutionize the healthcare field, we continue our pursuit of advancement with the confidence that our work will lead to the launch of a life-saving drug product. Our determination is the engine of our future success” said André Choulika, Ph.D., Chief Executive Officer at Cellectis.

 

Pipeline Highlights

UCART Clinical Programs

• On June 4, 2024, Cellectis received Orphan Drug Designation (ODD) from the European Commission (EC) for UCART22, for the treatment of acute lymphoblastic leukemia (ALL). The Orphan Drug Designation in the European Union is granted by the EC based on a positive opinion issued by the European Medicines Agency (EMA) Committee for Orphan Medicinal Products. This designation may allow certain regulatory, financial, and commercial incentives to develop medicines for rare diseases where there are no satisfactory treatment options.
• On July 25, 2024, the FDA designated UCART22 as a drug for a Rare Pediatric Disease (RPDD). This designation may allow to obtain a “Priority Review Voucher” at the time of Biologics License Application (BLA). The FDA also granted ODD to UCART22 product candidate for ALL treatment. Receiving ODD by the FDA may help to expedite and reduce the cost of development, approval, and commercialization of a therapeutic agent.
• Patients with relapsed/refractory ALL have limited, if any, treatment options, especially for those who have failed prior CD19 directed CAR T-cell therapy and allogeneic stem cell transplant. These designations for UCART22 mark an important step towards developing allogeneic CAR T products that would be readily available for all patients.
• On August 1, 2024, the FDA granted ODD to Cellectis’ CLLS52 (alemtuzumab), an Investigational Medicinal Product (IMP) used as part of the lymphodepletion regimen associated with UCART22, evaluated in the BALLI-01 clinical trial. 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.
• Cellectis continues to focus on the enrollment of patients in the BALLI-01 study, evaluating UCART22 in relapsed or refractory B-cell acute lymphoblastic leukemia, in the NatHaLi-01 study, evaluating UCART20x22 in relapsed or refractory B-cell non-Hodgkin lymphoma, and in the AMELI-01 study, evaluating UCART123 in relapsed or refractory acute myeloid leukemia. We expect to provide updates in the advancements of BALLI-01 til the end of the year 2024.

 

Research Data & Preclinical Programs

Non-viral Gene Therapy Approach for Sickle Cell Disease

• On June 12, 2024, Cellectis announces the publication of a scientific article in Nature Communications.
• Cellectis leverages TALEN® technology and a non-viral gene repair template delivery to develop a clinically relevant gene editing process in hematopoietic stem and progenitor cells (HSPCs). This process enables efficient HBB gene correction with high precision, specificity and minimal genomic adverse events.
• Applying this HBB gene correction process to SCD patient-HSPCs results in over 50% expression of normal adult hemoglobin in mature red blood cells and in the correction of sickle phenotype, without inducing β-thalassemic phenotype. Edited HSPCs engraft efficiently in an immunodeficient murine model and maintain clinically relevant levels of HBB gene correction events. This comprehensive preclinical data package sets the stage for the therapeutic application of autologous gene corrected HSPCs to address SCD.

 

Partnerships

Licensed Allogeneic CAR T-cell Development Programs

Anti-CD19 Programs

Allogene’s investigational oncology products utilize Cellectis technologies.

We have initiated an arbitration proceeding through the Centre de Médiation et d'Arbitrage de Paris. We are requesting that the arbitral tribunal issue a decision (i) terminating the Servier License Agreement, and (ii) requiring Servier to pay us fair financial compensation for losses incurred due to the lack of development of the licensed products and for non-payment of milestone payments for milestones that have been achieved under the Servier License Agreement.

In May 2024, Allogene announced the execution of an Amendment and Settlement Agreement (the "Servier Amendment"), which amended the license agreement between Servier and Allogene, under which Servier exclusively sublicensed to Allogene its rights under the License Agreement between Cellectis and Servier (the "Servier License"), for the development and commercialization of allogeneic anti-CD19 CAR T cell product candidates in the U.S. (the "Allogene Sublicense"). Allogene disclosed that, pursuant to the Servier Amendment to the Allogene Sublicense, the licensed territory was expanded to include the European Union and the United Kingdom, and Allogene was granted an option to further extend its licensed territory to include China and Japan subject to certain conditions.

 

Corporate Updates

Collaboration and Investment Agreements with AstraZeneca

• On May 6, 2024, Cellectis announced the completion of the subsequent investment of $140M in Cellectis by AstraZeneca (LSE/STO/Nasdaq: AZN) (the “Additional Investment”).
• AstraZeneca subscribed for 10,000,000 “class A” convertible preferred shares and 18,000,000 “class B” convertible preferred shares, in each case at a price of $5.00 per convertible preferred share, issued by the Board of Directors of Cellectis.
• On the completion date of the Additional Investment, AstraZeneca owned approximately 44% of the share capital and 30% of the voting rights of the Company (based on the number of voting rights outstanding at the time).
• Following the Additional Investment, Mr. Marc Dunoyer and Dr. Tyrell Rivers have been nominated members of the board of directors of Cellectis, designated by AstraZeneca.

 

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/