Cellectis S.a. · EURONEXT:ALCLS
Cellectis — Investor Presentation (June 2023)
31 slides from Cellectis's investor presentation, June 2023
Slide 1: Commitment to a Cure
Commitment to a Cure Corporate Presentation June 2023 NASDAQ: CLLS EURONEXT GROWTH: ALCLS.PA 1
Slide 2: Forward-Looking Statements
Forward-Looking Statements This presentation contains “forward-looking” statements within These forward-looking statements are made in light of the meaning of applicable securities laws, including the Private information currently available to us and are subject to Securities Litigation Reform Act o
Slide 3: Cellectis at a Glance
Cellectis at a Glance 3 Clinical Trials1 Global GMP Facilities Near-Term Clinical Catalysts $88M as of March 31, 2023 50+ patients dosed in • End-to-end manufacturing autonomy • UCART clinical data updates Cash Runway into Q3 20242 Cellectis-sponsored trials Diversified Partnerships with Industry Le
Slide 4: A Highly-Experienced Executive Committee
A Highly-Experienced Executive Committee André Choulika, Ph.D. Steven Doares, Ph.D. Phillippe Duchateau, Ph.D. Mark Frattini, M.D., Ph.D. Kyung Nam-Wortman Founder & CEO SVP, US Manufacturing & Site Head Chief Scientific Officer Chief Medical Officer EVP, Chief Human Resources Officer Stephan Reynie
Slide 5: UCARTs are designed to be Off-The-Shelf
UCARTs are designed to be “Off-The-Shelf” Scalable Robustness Market Access Manufacturing Reduced cost The goal is to provide Immediately available Scalable manufacturing: potency and consistency to all eligible patients 1 batch = 100s doses to each patient Control Production & Costs for Patients Sa
Slide 6: Why TALEN?
Experts in Gene-Editing Use TALEN® High rates of gene editing for knock outs and knock ins Efficient In clinic since 2015 Protein / DNA interaction with Mature Safe ~220 patients dosed 32 base pairs recognition WHY TALEN®? Euchromatin & Genome Targets desired site with a Precise maximum 7 bps range
Slide 7: Cellectis' UCART Candidate Platform
Cellectis’ UCART Candidate Platform Targeting CD20 & CD22 TALEN® Powered CAR20 CAR22 For B-cell Malignancies TRAC KO CAR(s) Minimizes risk of GvHD Targeting CD22 CAR22 For B-cell ALL CD52 KO Resistance to alemtuzumab Targeting CD123 CAR123 For AML CD52 ALL, acute lymphoblastic leukemia; AML, acute m
Slide 8: Differentiated Targets & Near-Term Catalysts
Differentiated Targets & Near-Term Catalysts Candidate / Phase 1 Dose Phase 1 Dose Pivotal Target Indication Study Preclinical Escalation Expansion Phase2 Upcoming Expected Milestones UCART22 BALLI-01 Data update with in-house ALL Fully Owned CD22 NCT04150497 manufactured product UCART123 AML AMELI-
Slide 9: Cellectis' UCART Platform
Cellectis’ UCART Platform 9
Slide 10: UCART22 – BALLI-01 Trial Design
UCART22 – BALLI-01 Trial Design Phase I/IIa, Open Label Dose-escalation and Dose-expansion Study to Evaluate the Safety, Expansion, Persistence and Clinical Activity of UCART22 in Patients with Relapsed or Refractory CD22+ B-cell Acute Lymphoblastic Leukemia Determine MTD Dose Expansion Dose Escalat
Slide 11: UCART22 Administration Shows Promising Tolerable Safety Profile
UCART22 Administration Shows Promising Tolerable Safety Profile Patient Characteristics (N=19) Safety: FCA Cohorts (N=13) Median age: 28 (17-61) 0 dose limiting toxicity WHO classification: 0 ICANS (immune effector cell associated • B-ALL with recurrent genetic neurotoxicity) abnormalities: 8 (42%);
Slide 12: Promising Clinical Responses with UCART22 and FCA Lymphodepletion
Promising Clinical Responses with UCART22 and FCA Lymphodepletion • Host lymphocytes remained suppressed using FCA lymphodepletion • 2/7 patients in DL2 and DL2i achieved blast reductions to < 5% by day 28 using FCA lymphodepletion • 1 patient in DL2: MRD negative CRi • 1 patient in DL2i: MLFS • 50%
Slide 13: UCART123 – AMELI-01 Trial Design
UCART123 – AMELI-01 Trial Design Phase I, Open Label Dose-escalation and Dose-expansion Dose-Escalation UCART123 Study to Evaluate the Safety, Expansion, Persistence and Clinical Activity of UCART123 in Patients with Relapsed or mTPI design; 2-4pts/cohort Refractory Acute Myeloid Leukemia FC Arm FCA
Slide 14: Addition of Alemtuzumab Results in Prolonged Host Lymphodepletion
Addition of Alemtuzumab Results in Prolonged Host Lymphodepletion in AMELI-01 Absolute Lymphocyte Counts Data Source: ASH 2022 Conference Presentation FC: Fludarabine + Cyclophosphamide; FCA: Fludarabine + Cyclophosphamide + Alemtuzumab; LD: Lymphodepletion; DL2: Dose Level 2; DL2i: Dose Level 2 Int
Slide 15: Anti-Leukemic Activity and Robust CAR T-Cell Expansion Observed using FCA
Anti-Leukemic Activity and Robust CAR T-Cell Expansion Observed using FCA Lymphodepletion 2 responses observed in FC arm • Patient 101-219 (DL2i): SD VCN AUC(0-28days) • Patient 103-223 (DL2i): MLFS p = 0.04 2 responses observed in FCA arm Addition of • Patient 114-229 (DL2): SD alemtuzumab Ø Achiev
Slide 16: Patient 104-226 Achieved a Durable MRD Negative Complete Response
Patient 104-226 Achieved a Durable MRD Negative Complete Response Clinical Characteristics Age, Race, Sex 64-year-old white female ECOG PS 1 ELN 2017 Classification; WHO Classification Adverse risk; AML with myelodysplasia-related changes Cytogenetic and Molecular Abnormalities 45,XX,-7,t(10;12)(q24
Slide 17: Translational Data Supports Use of a Two-Dose Regimen of UCART123
Translational Data Supports Use of a Two-Dose Regimen of UCART123 AMELI-01 Amended Protocol with Two-Dose Regimen Design § UCART123 expansion correlates with reduction in tumor burden at DL2 (6.25 x 105 cells/kg) but at this dose, UCART123 cell function is not sufficient for sustained anti-leukemic
Slide 18: UCART20x22: Overcoming CAR T Challenges with Dual Antigen Target
UCART20x22: Overcoming CAR T Challenges with Next Generation Dual Antigen Target UCART20x22 • Strong alternative to CD19 (highly competitive/crowded/CD19 negative relapses) • CD22 and CD20 are validated targets in B-cell malignancies • Dual targeting designed for better killing & prevent escape • St
Slide 19: UCART20x22 – NATHALI-01 Trial Design
UCART20x22 – NATHALI-01 Trial Design Open-Label Dose-Finding and Dose-Expansion Study to Evaluate the Safety, Expansion, Persistence, and Clinical Activity of UCART20x22 In Subjects with Relapsed or Refractory B-Cell Non-Hodgkin Lymphoma (B-NHL) Dose Escalation Determine MTD Dose Expansion LD regime
Slide 20: UCART20x22 – Efficient Activity in vivo Against Multiple Target Combinations
UCART20x22 – Efficient Activity in vivo Against Multiple Target Combinations 100 8x106 UCART20x22 3x106 UCART20x22 Percent survival 8x106 UCART22 50 10x106 NTD Vehicle 0 Subcutaneous lymphoma tumors expressing 0 50 100 different antigen combinations in a single mouse Days • CD20–CD22+ • CD20+CD22– •
Slide 21: Efficient B-NHL Primary Sample Targeting with UCART20x22
Efficient B-NHL Primary Sample Targeting with UCART20x22 Primary B-NHL tumors expressing CD20 and CD22 Cytotoxic activity against B-NHL primary samples 5/1 100 1/1 % Lysis 50 0 P7 P8 P9 0 1 2 3 4 5 4 6 0 2 3 2 O di 3 P1 P1 P1 P1 P1 P1 P2 P2 P3 P3 P3 P4 -1 au LM D M UCART20x22 efficiently targets pri
Slide 22: UCART Platform Takeaways from ~220 Patients
UCART Platform Takeaways from ~220 Patients* GvHD TRAC KO results in safe, non-alloreactive UCART cells Expansion CD52 KO + alemtuzumab use in LD has the potential to provide a safe, effective & controllable therapeutic window Persistence Encouraging clinical activity in ALL, AML, and NHL Safety Pro
Slide 23: Discover, Create, Develop, Produce and Test
Discover, Create, Develop, Produce and Test ALPHA Study Paris New York, New York Paris, France Raleigh, North Carolina Innovation, Clinical Development HQ, PD/AD, Starting Materials UCART – Clinical & potential for Commercial 25,000 sq ft. facility 55,000 sq ft. facility 82,000 sq ft. facility ü Gen
Slide 24: Expected Milestones
Expected Milestones UCART22 UCART123 UCART20x22 r/r B-ALL r/r AML r/r B-NHL Data update with in- Data update with First in-human data house manufactured 2-dose regimen update product Partnerships Updates from licensed partners (Servier/Allogene, Iovance, Cytovia and Primera) 24 ALL, acute lymphoblas
Slide 25: Key Takeaways – Why Cellectis?
Key Takeaways – Why Cellectis? Innovative Breaking Paradigms with Life-Saving Therapies Allogeneic CAR T End-to-End In-House Owning Manufacturing is Owning the Product Manufacturing Best-In-Class Gene Safe, Precise & Efficient, Backed by Strong IP Editing Platform Strong Partnerships Anticipated Mil
Slide 26: Thank You
Thank You Reach us at: [email protected] Cellectis Paris Cellectis New York Cellectis Raleigh 8, rue de la Croix Jarry 75013 430 East 29th Street 2500 Sumner Boulevard Paris – France New York, NY, 10016 – USA Raleigh, NC, 27616 – USA 26
Slide 27: Appendix
Appendix 27
Slide 28: Powerful and Comprehensive Gene Editing Platform
Powerful and Comprehensive Gene Editing Platform Gene Knockout Gene Repair Gene Replacement Gene Insertion Nucleases Experts in Gene Editing: Proprietary ü 30+ years for Cellectis’ electroporation technology founders Base editors ü 22 years doing gene editing ü 10 years developing TALEN® Gene Knocko
Slide 29: Why TALEN?
Why TALEN®? Maturity Genome Recognition Chromotrypsis Precision Vectorization IP Outreach Site # base pairs Strong In clinic Euchromatin & Every 7 TALEN® since 2015 heterochromatin 32 Not reported base pairs mRNA for CLLS Every 64 Scatter In clinic Euchromatin ~20 RNP CRISPR since 2018 only Yes base
Slide 30: Diversified Partnerships with Industry Leaders (1/2)
Diversified Partnerships with Industry Leaders (1/2) Exclusive worldwide license to CD19- 2014 Up to $410M in Development & Sales directed allogeneic CAR T-cells CAR T Milestones CD19 + Low Double-Digit Royalties on Sales U.S. rights sublicensed to 20151 Allogene by Servier CAR T Up to $2.8B in Deve
Slide 31: Diversified Partnerships with Industry Leaders (2/2)
Diversified Partnerships with Industry Leaders (2/2) Collaboration agreement to develop mtDNA gene editing for mitochondrial 19% equity upfront mtDNA Option for up to $750M in Development & 2022 diseases + option for exclusive worldwide license agreement on up to editing Sales Milestones + High Sing