Cellectis Provides Business Update and Reports Financial Results for Third Quarter and First Nine Months 2022

-ASH 2022 sponsored programs – UCART123 abstract selected for oral presentation & preclinical data on UCARTCS1 selected for poster presentation, in collaboration with the Amsterdam University Medical Center  

-Preclinical data on TALEN®-edited smart CAR T-cells to be presented at SITC 2022  

-Partner Allogene Therapeutics announced having initiated industry’s first allogeneic CAR T Phase 2 trial 

-Iovance Biotherapeutics, Inc. announced having dosed the first patient and completed safety observation period in its IOV-GM1-201 clinical trial 

-Mark Frattini, M.D., Ph.D., appointed Chief Medical Officer 

-Cash position[1] of $103 million as of September 30, 2022  

-Conference call scheduled for 8AM ET/1PM CET on November 4, 2022 

 

[1] Cash position includes cash, cash equivalent, current financial assets and restricted cash. Restricted cash was $5million as of September 30, 2022.

November 3, 2022 - New York (N.Y.) - Cellectis (Euronext Growth: ALCLS – Nasdaq: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop potentially life-saving cell and gene therapies, announced its results for the three-month and nine-month periods ending September 30, 2022.

“2022 has been a productive year thus far for Cellectis. Today, we were proud to announce that our UCART123 abstract was accepted for an oral presentation at the American Society of Hematology Annual Meeting. Acute myeloid leukemia presents a huge unmet medical need with a lack of cell therapy options, and these encouraging preliminary clinical data are a great step for patients with this condition,” said André Choulika, Ph.D., Chief Executive Officer of Cellectis.  

“We continue to enroll patients in our three Cellectis-sponsored Phase 1 dose escalation clinical trials and are very excited to initiate the dosing of patients with our product candidates UCART22 and UCART20x22 that have been fully manufactured in-house. This quarter, our partnerships continued to be a highlight for Cellectis, as several of our licensed partners disclosed exciting milestones. Our licensed partner, Allogene Therapeutics, announced that it was entering into the potentially pivotal Phase 2 trial, for patients with large B-cell lymphoma. Iovance Biotherapeutics announced that the first patient was dosed and completed the safety observation period in the IOV-GM1-201 clinical trial of Iovance’s first genetically modified TALEN®-edited TIL therapy for the treatment of previously treated advanced melanoma or metastatic NSCLC. These achievements showcase once more that TALEN® is a technology of choice for leading cell and gene therapy companies, and that we are continuing to deliver on our promise of constant innovation in order to advance the efforts of both our and our partner’s clinical trials. Cellectis remains steadfast in its mission to develop innovative cancer therapy product candidates, and I am proud of the progress we have made in our journey this year.” 

Pipeline highlights

UCART Clinical Development Programs 

• BALLI-01 (evaluating UCART22) in relapsed or refractory B-cell acute lymphoblastic leukemia (r/r B-ALL)
• UCART22 is an allogeneic CAR T-cell product candidate that targets CD22 and is being evaluated in the BALLI-01 clinical study, a Phase 1/2a open-label dose-escalation study designed to evaluate the safety and clinical activity of the product candidate in patients with relapsed or refractory B-cell acute lymphoblastic leukemia (r/r B-ALL) 
   
• BALLI-01 is currently enrolling patients at dose level 3 (DL3) (5 × 106 cells/kg) with fludarabine, cyclophosphamide and alemtuzumab (FCA) preconditioning regimen. 
   
• Cellectis plans to initiate dosing patients with UCART22 product candidate manufactured fully in-house. 

 

AMELI-01 (evaluating UCART123) in relapsed or refractory acute myeloid leukemia (r/r AML) 

• UCART123 is an allogeneic CAR T-cell product candidate targeting CD123 and is being evaluated in patients with relapsed or refractory acute myeloid leukemia (r/r AML) in the AMELI-01 Phase 1 dose-escalation clinical study. 
   
• Today, Cellectis announced the release of an abstract, which was accepted for oral presentation at the American Society of Hematology (ASH) 2022 Annual Meeting.  
• The abstract includes preliminary clinical data from the Phase I, open-label, dose-escalation AMELI-01 study in patients with r/r AML, showing that adding alemtuzumab to the fludarabine and cyclophosphamide lymphodepletion regimen was associated with improved lymphodepletion and significantly higher UCART123 cell expansion, which correlated with improved activity.  These encouraging data support the continued enrollment into the study.  
• Oral presentation will occur on December 12 at 5:00PM; Ernest N. Morial Convention Center, Hall E. Link to abstract here. 

MELANI-01 (evaluating UCARTCS1) in relapsed or refractory multiple myeloma (r/r MM) 

• UCARTCS1 is an allogeneic CAR T-cell product candidate targeting CS1 and is being evaluated in patients with relapsed or refractory multiple myeloma (r/r MM) in the MELANI-01 Phase 1 dose-escalation clinical study.
• Cellectis is currently enrolling patients at dose level 1 (DL1) (1 × 106 cells/kg) with fludarabine and cyclophosphamide preconditioning regimen. 

NatHaLi-01 (evaluating UCART20x22) in relapsed or refractory Non-Hogdkin Lymphoma (r/r NHL) 

• UCART20x22 is Cellectis’ first allogeneic dual CAR T-cell product candidate being developed for patients with relapsed or refractory non-Hodgkin lymphoma (r/r NHL).  
   
• UCART20x22 is Cellectis’ first product candidate fully designed, developed and manufactured in-house, showcasing the Company’s transformation into an end-to-end cell and gene therapy platform spanning discovery, product development, manufacturing of starting materials used for genomic engineering and final cell therapy product candidates, in addition to clinical development.  
   
• On August 1st, the U.S. Food and Drug Administration (FDA) allowed Cellectis’ IND to proceed with UCART20x22 in clinical development for patients with r/r NHL.  

 

Research Data & Preclinical Programs 

UCARTCS1 

• Today, Cellectis in collaboration with the Amsterdam University Medical Center (VUmc) announced the release of an abstract on product candidate UCARTCS1, which was accepted for poster presentation at the ASH 2022 Annual Meeting.  
• The abstract includes preclinical data evaluating in vitro activity of UCARTCS1 against multiple myeloma (MM) cell lines and bone marrow samples from MM patients, as well as in vivo activity in a MM mouse model. The potential impact of previous therapy and tumor characteristics on the in vitro efficacy of UCARTCS1 was also investigated. 
• The preclinical data that will be presented demonstrate anti-tumor activity in vitro and in vivo supporting the potential benefit of our UCARTCS1 first in-human study, MELANI-01, a Phase 1, open-label, dose-escalation trial for patients with r/r MM.   
• Presentation will occur on Saturday, December 10, 2022; 5:30 PM - 7:30 PM; Ernest N. Morial Convention Center, Hall D. Link to abstract here. 

TALEN®-edited smart CAR T-cells 

• On October 5, Cellectis announced that preclinical data on TALEN®-edited smart CAR T-cells supporting improved solid tumor targeting will be presented at the Society for Immunotherapy of Cancer’s (SITC) Annual meeting, to be held on November 8-12, 2022 in Boston. 
• Cellectis will present two posters:  

A poster on TALEN®-edited smart CAR T-cells targeting MUC1-expressing solid tumors. MUC1 is a tumor-associated antigen that is overexpressed in a number of solid tumor malignancies including triple-negative breast cancer (TNBC).

A poster on innovative T-cell engineering strategies designed to increase the activity of CAR T-cells for solid tumors while mitigating toxicity risks. 

• Presentations will occur on November 10, from 9:00AM until 9:00PM ET, Hall C. 

 
TALEN®-based gene therapy preclinical programs 

• On October 11, at the European Society of Gene and Cell Therapy (ESGCT), Cellectis presented pre-clinical data that leverages TALEN® gene editing technology to develop a hematopoietic stem and progenitor cell (HSPCs)-based gene therapy for the treatment of sickle cell disease, and a TALEN®-based gene editing approach that reprograms HSPCs to secrete alpha-L-iduronidase (IDUA), a therapeutic enzyme missing in Mucopolysaccharidosis type I (MPS-I). 
• The pre-clinical data presented at ESGCT further demonstrate our ability to leverage TALEN® gene editing technology to potentially address genetic diseases, namely sickle cell disease and lysosomal storage diseases. By correcting a mutation or inserting a corrected gene at the HSPC level, Cellectis aims to provide a lifelong supply of healthy cells in a single intervention. 
• Click here to access the presentations and abstracts. 

 

Licensed Allogeneic CAR T-cell Development Programs 

Allogene Therapeutics, Inc.’s CAR T programs utilize Cellectis technologies.  

ALLO-501 and ALLO-501A are anti-CD19 products being jointly developed under a collaboration agreement between Servier and Allogene based on an exclusive license granted by Cellectis to Servier (the “Servier Agreement”). Servier grants to Allogene exclusive rights to ALLO-501 and ALLO-501A in the U.S. while Servier retains exclusive rights for all other countries. In September 2022, Servier communicated that it was discontinuing its involvement in the development of in-licensed CD19 products and purporting to provide Allogene with the ability to elect to obtain a license to the CD19 Products outside of the United States. We are evaluating all available options and contractual remedies to address the foregoing matters and other performance issues, which we believe may involve material breaches of the Servier Agreement by Servier. Allogene’s anti-BCMA and anti-CD70 programs are licensed exclusively from Cellectis by Allogene and Allogene holds global development and commercial rights to these programs.

Servier and Allogene: anti-CD19 programs  

•  On October 6, 2022, Allogene announced it has initiated potentially pivotal allogeneic CAR T Phase 2 clinical trial of ALLO-501A (ALPHA2 trial) in patients with relapsed/refractory (r/r) large B-cell lymphoma (LBCL). Allogene expects that the ALPHA2 trial will enroll approximately 100 patients who have received at least two prior lines of therapy and have not received prior anti-CD19 therapy. Allogene announced that updated clinical data from the CD19 program will be provided at Allogene’s R&D Showcase on November 29, 2022. 

Allogene: anti-BCMA program 

• On November 2, 2022, Allogene announced that the Phase 1 portion of the UNIVERSAL trial on ALLO-715 continues enrolling patients with r/r multiple myeloma (MM). Allogene also announced it will provide a clinical update from UNIVERSAL focused on a single dose ALLO-715 and discuss next steps for the program at the R&D Showcase. 

Allogene: solid tumor program

• On November 2, 2022, Allogene stated that ALLO-316, which targets CD70, its first AlloCAR T candidate for solid tumors, is being studied in patients with advanced or metastatic clear cell renal cell carcinoma (RCC) in the Phase 1 TRAVERSE trial.   

Gene Editing Partnerships 

Iovance Biotherapeutics, Inc. (“Iovance”)  

• On October 10, 2022, Iovance announced that the first patient was dosed and completed the safety observation period in the IOV-GM1-201 clinical trial of Iovance’s first genetically modified TIL therapy in development, IOV-4001, for the treatment of previously treated advanced melanoma or metastatic NSCLC.
• To inactivate the gene coding for the PD-1 protein, IOV-4001 utilizes the gene-editing TALEN® technology licensed from Cellectis. This single genetic modification in IOV-4001 has the potential to enhance the antitumor activity of the TIL mechanism to directly target and kill tumor cells. Dosing the first patient with IOV-4001 is an important first step in providing proof-of-concept for delivering genetically modified TIL therapy to solid tumor patients with significant unmet needs and few treatment options. 

Cytovia Therapeutics 

• Cytovia announced it will present new preclinical data on TALEN® gene-edited, induced pluripotent stem cells (iPSC)-derived Natural Killer (NK) cells at the Society for Immunotherapy of Cancer’s (SITC) Annual Meeting. The abstract, entitled “TALEN®-Based Gene-Edited iPSC-Derived NK (iNK) Cells Demonstrate Enhanced Antitumor Activity”, was accepted for poster presentation. 
• These data highlight the progress of Cellectis’ research and development collaboration with Cytovia to develop TALEN®-edited iPSC NK and CAR-NK cells. Cellectis has developed custom TALEN® which Cytovia is using to edit iPSCs in a safe and effective manner.  
• The poster presentation will occur on November 11, from 9:00AM until 9:00PM ET, Poster Hall. 

Corporate Updates 

Appointment 

• On September 28, 2022, Cellectis announced the appointment of Mark Frattini, M.D., Ph.D., as Chief Medical Officer. 
• Dr Frattini joined Cellectis in August 2020 as Senior Vice President of Clinical Sciences and has been responsible for Cellectis’ clinical leadership including the clinical development strategy of the Company’s current immune-oncology UCART product candidates. He has also been serving as a core member of the senior clinical team and has been managing a team of physicians and clinical scientists. As Chief Medical Officer, Dr. Frattini oversees clinical research and development for Cellectis’ UCART clinical trial programs. He remains based in Cellectis’ New York office and has joined the Company’s executive committee