Cellectis Provides Business Updates and Financial Results for Third Quarter 2024

• UCART22 and UCART20x22: enrollment ongoing, Phase 1 dataset and late-stage development strategy to be presented in 2025

• AstraZeneca partnership: R&D activities are 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

• Appointed Adrian Kilcoyne, M.D., MPH, MBA, an industry leader in the advancement of cell therapy treatment, as Chief Medical Officer

• Cash position of $264 million as of September 30, 2024[1]; cash runway projection into 2027

• Conference call scheduled for 8:00 am ET / 2:00 pm CET on November 5, 2024

 

New York, NY – November 4, 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 nine-month period ending September 30, 2024.

“This quarter, we were thrilled to welcome Dr. Kilcoyne to Cellectis as Chief Medical Officer. Dr. Kilcoyne joins us at a pivotal time for the Company, bringing extensive experience in drug development as we are progressing in our clinical programs. We expect to present Phase 1 dataset and late-stage development strategy in 2025 for UCART22 in ALL and UCART20x22 for NHL” said André Choulika, Ph.D., Chief Executive Officer at Cellectis. 

“Additionally, we are excited to announce that research and development activities have started for three programs under our collaboration and research agreement with AstraZeneca: 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 is confident about the continued progress of its ongoing clinical trials in hematological malignancies and is excited about our strategic collaboration with AstraZeneca, with whom we continue to advance our ambition in cell and gene therapy to bring potentially lifesaving therapies to patients with unmet medical needs."

 

Pipeline Highlights

UCART Clinical Programs

• 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. We expect to present the Phase 1 dataset and late-stage development strategy in 2025.
• Cellectis continues to focus on the enrollment of patients in the NATHALI-01 study, evaluating UCART20x22 in relapsed or refractory B-cell non-Hodgkin lymphoma. We expect to present the Phase 1 dataset and late-stage development strategy in 2025.
• 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, currently evaluated in relapsed or refractory acute myeloid leukemia. 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.

 

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

• On September 3, 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. In this article, 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.

Partnerships

Servier and Allogene – Allogeneic CAR-T

Allogene’s investigational oncology products utilize Cellectis technologies.

• Allogene announced that the pivotal Phase 2 ALPHA3 trial was initiated in June 2024. This study is evaluating the use of cemacabtagene ansegedleucel (cema-cel) as part of the first line (1L) treatment regimen for patients with LBCL who are likely to relapse after standard 1L treatment. Allogene announced that ALPHA3 is the first pivotal trial to offer CAR T as part of 1L treatment consolidation.
• Allogene announced that enrollment is ongoing in the relapsed/refractory (r/r) CLL cohort of the Phase 1 ALPHA2 trial of cema-cel, and that initial data readout from the CLL cohort is projected by early 2025.
• Allogene announced that a Phase 1 data update of the TRAVERSE trial of ALLO-316 from approximately 20 patients with CD70 positive RCC is planned by YE 2024. In October 2024, Allogene announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to ALLO-316 for the treatment of adult patients with CD70 positive advanced or metastatic renal cell carcinoma (RCC).

AstraZeneca – Joint Research and Collaboration Agreement

• Under the terms of the joint research and collaboration agreement entered into by Cellectis and AstraZeneca Ireland Limited (“AstraZeneca”) on November 1, 2023 (the “AZ JRCA”), AstraZeneca is leveraging Cellectis’ proprietary gene editing technologies and manufacturing capabilities to design novel cell and gene therapy candidate products. As part of the AZ JRCA, 25 genetic targets have been exclusively reserved for AstraZeneca, from which up to 10 candidate products could be explored for development. AstraZeneca has an option for a worldwide exclusive license on the candidate products, to be exercised before IND filing.
• Research and development activities under three cell and gene therapy programs have already started under 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 triggered so far (of which $25m upfront and $22m reached development milestones for the three initial projects), in addition to reimbursement of research costs incurred under the AZ JRCA.

Appointment

• On August 7, 2024, Cellectis announced the appointment of Adrian Kilcoyne, M.D., MPH, MBA as its Chief Medical Officer.
• Before joining Cellectis, Dr. Kilcoyne was Chief Medical Officer and Head of Research and Development at Celularity, advancing their oncology allogeneic CAR-T and NK Cell therapy programs. Prior to this, he was Chief Medical Officer at Humanigen. He has held numerous Oncology leadership roles across Research and Development, Medical Affairs, Commercial, Health Economic Outcome Research and Evidence Generation in both large pharmaceutical and biotechnology companies such as AstraZeneca and Celgene. Dr. Kilcoyne graduated from Trinity College, Dublin Medical School. He initially trained in Gynecological Oncology at the Hammersmith Hospital in London and subsequently in Public Health Medicine at Oxford during which time he completed a Master’s in Public Health. Dr. Kilcoyne then trained in pharmaceutical medicine and completed his MBA.