Slide 1: Commitment to a Cure: Cellectis Clinical Update December 2022
Commitment to a Cure Cellectis Clinical Update December 2022 NASDAQ: CLLS EURONEXT GROWTH: ALCLS.PA Exhibit 99.1
Slide 2: Forward-looking statements
This presentation (together with the corresponding webcast, this “Presentation”) contains “forward-looking” statements within the meaning of applicable securities laws, including the Private Securities Litigation Reform Act of 1995. This Presentation shall not constitute an offer to sell, or the sol
Slide 3: Legal Notices
References in this Presentation to Cellectis’ product candidates as “off-the-shelf” refers to the fact that our CAR T-cells are allogeneic, meaning they are derived from healthy donors rather than the patients themselves, which we believe allows for the development of cost-effective product candidat
Slide 4: Cellectis at a Glance
Cellectis at a Glance 4 Ongoing Clinical Trials Operational since 2021 End-to-end manufacturing autonomy Near-Term Clinical Catalyst 200+ patients dosed to date Potential revenues > $4B in milestones + royalties 6 trials sponsored by Cellectis’ licensed partners Diversified Partnerships with Industr
Slide 5: UCARTs are Off-The-Shelf
UCARTs are “Off-The-Shelf” Reduced cost Scalable manufacturing: 1 batch = 100s of doses The goal is to provide potency and consistency to each patient Immediately available to eligible patients Market Access Robustness Scalable Manufacturing
Slide 6: TALEN Powered UCART Candidate Platform
TALEN® Powered TRAC KO Reduces risk of GvHD CS1 KO Self-lymphodepleting CD52 KO Resistance to alemtuzumab or Cellectis’ UCART Candidate Platform CAR20 CAR22 CAR123 CARCS1 Targeting CD20 & CD22 For B-cell Malignancies Targeting CD22 For B-cell ALL Targeting CD123 For AML Targeting CS1/SLAMF7 For MM C
Slide 7: AMELI-01: UCART123
AMELI-01: UCART123
Slide 8: AMELI-01: Preliminary Results from a Phase I Trial of UCART123v1.2 in R/R AML
AMELI-01:Preliminary Results from A Phase I Trial of UCART123v1.2, an Anti-CD123 Allogeneic CAR-T Cell Product, in Adult Patients with Relapsed or Refractory (R/R) CD123+ Acute Myeloid Leukemia David A. Sallman1, Daniel J. DeAngelo2, Naveen Pemmaraju3, Shira Dinner4, Saar Gill5, Rebecca Olin6, Eunic
Slide 9: Background and Introduction
Background and Introduction It is estimated that 20,050 new cases and 11,540 deaths related to AML will occur in the US in 20221 Outcomes for patients with R/R AML remain poor, with response rates <30% and an expected 5-year overall survival of <15%2,3 AMELI-01 (NCT03190278) is a phase I, open-label
Slide 10: UCART123v1.2: Allogeneic Off-the-Shelf T-cell Product
UCART123v1.2 (anti-CD123 scFv-41BB-CD3ζ): CD123 is a validated therapeutic target in AML Genetically modified allogeneic T-cell product manufactured from non-HLA–matched healthy donor cells TRAC disrupted using TALEN® to eliminate TCRαβ from the cell surface and reduce risk of GvHD CD52 disrupted us
Slide 11: AMELI-01 Study Design
AMELI-01 Study Design Key inclusion criteria Relapsed or primary refractory AML (>5% bone marrow blasts) Blasts expressing CD123 PS of ≤1 and adequate organ function Primary objective Safety, tolerability, & MTD/RP2D of UCART123v1.2 Additional objectives Investigator-assessed response UCART123v1.2 e
Slide 12: Baseline Characteristics
Characteristic Total (N = 18*) Age, median (range), years 57 (18-64) Female, n (%) 8 (44) ECOG PS 1, n (%) 17 (94) ELN 2017 Classification, n (%) Adverse risk Intermediate risk 14 (78) 3 (17) Median baseline bone marrow blasts % (range) 37 (0-88) Number of prior treatments, median (range) 4 (3-9) Pr
Slide 13: UCART123v1.2 - Related TEAEs (FC + FCA)
UCART123v1.2 - Related TEAEs (FC + FCA) FC FCA FC + FCA TEAE, n FC Total [n=8] DL1=2; DL2=3; DL2i=2; DL3=1 FCA Total [n=9] DL2=8; DL2i=1 All patients N=17* Any grade Gr ≥3 Any grade Gr ≥3 Any grade Gr ≥3 CRS 8 2 9 2 17 4 HLH 1 1 1 0 2 1 ICANS 1 1 1 0 2 1 ALT increased 4 1 1 1 5 2 AST increased 4 1 1
Slide 14: UCART123v1.2 - Serious TEAEs (All Cause)
UCART123v1.2 - Serious TEAEs (All Cause – FC + FCA) FC FCA FC + FCA Serious TEAE, n (%) FC Total [n=8] DL1=2; DL2=3; DL2i=2; DL3=1 FCA Total [n=9] DL2=8; DL2i=1 Total patients N=17* Any grade Gr ≥3 Any grade Gr ≥3 Any grade Gr ≥3 CRS 3 2 2 2 5 4 ICANS 1 1 0 0 1 1 Pneumonia 1 1 1 1 2 2 Pneumonia fung
Slide 15: Anti-Leukemic Activity Observed in 4/17 Patients
Anti-Leukemic Activity Observed in 4/17 Patients FC arm Patient 101-219 (DL2i): SD Patient 103-223 (DL2i): MLFS FCA arm Patient 114-229 (DL2): SD Achieved greater than 90% BM blast reduction (60% to 5%) at D28 Patient 104-226 (DL2): MRD negative CR Achieved CRi at D28 followed by MRD negative CR at
Slide 16: Absolute Lymphocyte Counts
Absolute Lymphocyte Counts FC Arm FCA Arm Addition of Alemtuzumab Results in Prolonged Lymphodepletion
Slide 17: Addition of Alemtuzumab Results in Increased UCART123v1.2 Expansion
Addition of Alemtuzumab Results in Increased UCART123v1.2 Expansion UCART123 Cell Expansion in WB by VCN VCN AUC(0-28days) FC Arm FCA Arm p=0.04
Slide 18: Cytokine Profile of All Patients from FCA Arm
Cytokine Profile of All Patients from FCA Arm Ferritin vs VCN in Select Patients from FCA Arm Cytokine Secretion and Ferritin Levels Correlated with UCART123v1.2 Cell Expansion
Slide 19: Patient 104-226 Achieved a Durable MRD Negative CR
Patient 104-226 Achieved a Durable MRD Negative CR 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;p13)[5]; IDH1,
Slide 20: Translational Data Supports Two-Dose Regimen
Translational Data From Patient 104-226 and Others Supports Use of a Two-Dose Regimen of UCART123v1.2 UCART123v1.2 expansion correlates with reduction in tumor burden at DL2 (6.25 x 105 cells/kg) but at this dose, UCART123v1.2 cell function is not sufficient for sustained anti-leukemic activity in a
Slide 21: AMELI-01 Amended Protocol with Two-Dose Regimen
AMELI-01 Amended Protocol with Two-Dose Regimen Design Dose-Escalation UCART123.1.2 mTPI design; 2-4 pts/cohort FC Arm UCART123v1.2 (single dose) FCA Arm UCART123v1.2 (single dose) UCART123v1.2 (2-dose regimen) DL-1 DL 1 DL 2 DL 2i DL 2 DL 2i DL 3 DL 2 x 2 DL 2i x 2 DL 3 x 2 DL 1 x 2 UCART123.v1.2 D
Slide 22: Conclusions
Conclusions Adding alemtuzumab to the FC regimen was associated with improved LD and significantly higher UCART123v1.2 cell expansion, which correlated with improved activity and cytokine profiles One patient in the DL2 FCA arm achieved >90% blast reduction at Day 28 One patient in the DL2 FCA arm a
Slide 23: BALLI-01: UCART22
BALLI-01: UCART22
Slide 24: UCART22 – BALLI-01 Trial Design
UCART22 – BALLI-01 Trial Design NCT04150497 MTD: Maximum Tolerated Dose; RP2D: Recommended Phase 2 Dose; LD: Lymphodepletion; DL: Dose Level; F: Fludarabine; C: Cyclophosphamide; FC: Fludarabine + Cyclophosphamide ; FCA: Fludarabine + Cyclophosphamide + Alemtuzumab; NCCN: National Comprehensive Canc
Slide 25: UCART22 Administration Shows Promising Safety Profile
UCART22 Administration Shows Promising Safety Profile Data Source: ASH 2021 Conference Presentation CRFL2: Cytokine Receptor-Like Factor 2 ; FCA: Fludarabine, Cyclophosphamide, Alemtuzumab ; ICANS: Immune effector Cell-Associated Neurotoxicity Syndrome ; TEAE: Treatment Emergent Adverse Event CRS: C
Slide 26: Patient case: MRD-negative CRi
Patient 1 P Age 34 Sex Male Prior Therapies 1: Induction: GRALL 2014 (Intensive chemotherapy) – Daunorubicin; vincristine; cyclophosphamide; L-asparaginase; mercaptopurine; methotrexate; etoposide; cytarabine; prednisone/dexamethasone 2: Salvage: Vincristine; HiDAC; vindesine; ifofsamide; thioguanin
Slide 27: Patient case: MRD-negative MLFS
Patient 2 P Age 24 Sex Female Prior Therapies 1: AALL1131 high-risk arm (Intensive chemotherapy) – Vincristine; daunorubicin; prednisone/dexamethasone; cyclophosphamide; cytarabine; mercaptopurine; PEG-asparginase; methotrexate 2: Salvage: Vincristine; daunorubicin followed by autologous CD19 CART 3
Slide 28: Patient case: MRD-negative CR
Patient 3 P Age 57 Sex Male Prior Therapies 1: Induction: Vincristine; daunorubicin; cyclophosphamide; PEG-asparaginase; prednisone + consolidation: blinatumomab; cytarabine; methotrexate; vincristine; mercaptopurine; dexamethasone; followed by allogeneic HSCT (haploidentical donor) 2: Salvage: Vinc
Slide 29: Summary of UCART22 DL3 data
Summary of UCART22 DL3 data Five subjects were dosed at DL3 (5 x 106 cells/kg) with UCART22 Process 1 (P1) using FCA LD: No safety issues, no Grade 2 or higher CRS 3 out of 5 clinical responses (60% ORR): 1 MRD neg. CR, 1 MRD neg. CRi, 1 MLFS All 3 of the responders failed multiple lines of prior th
Slide 30: Next steps for BALLI-01
Next steps for BALLI-01 Dosing started with UCART22 Process 2 (P2) made by Cellectis UCART22 P2 product candidate has shown a significantly higher potency in vitro than P1 1st subject dosing at DL2 (1 x 106 cells/kg) Next data set with P2 expected in 2023
Slide 31: Summary
SUMMARY AMELI-01 (UCART123) Adding alemtuzumab to the FC lymphodepletion regimen was associated with improved LD and significantly higher UCART123v1.2 cell expansion, which correlated with improved activity One patient in the DL2 FCA arm achieved >90% blast reduction at Day 28 One patient in the DL2
Slide 32: Discover, Create, Develop, Produce and Test
Discover, Create, Develop, Produce and Test Paris Paris, France ALPHA Study HQ, PD/AD, Starting Materials New York, New York Innovation, Clinical Development ̴25,000 sq ft. facility Gene Editing platform – TALEN® I/O discovery platform Gene therapy discovery platform Clinical development Raleigh, No
Slide 33: Expected 2023 Milestones
Expected 2023 Milestones ALL, acute lymphoblastic leukemia; AML, acute myeloid leukemia; DL, dose level; FC, fludarabine + cyclophosphamide; FCA, fludarabine + cyclophosphamide + alemtuzumab; MM, multiple myeloma; NHL, non-Hodgkin’s lymphoma; RP2D, recommended Phase 2 dose; r/r, relapsed/refractory.
Slide 34: Thank You
Thank You Cellectis Paris 8, rue de la Croix Jarry 75013 Paris – France Cellectis New York 430 East 29th Street New York, NY, 10016 – USA Cellectis Raleigh 2500 Sumner Boulevard Raleigh, NC, 27616 – USA Reach us at: investors@cellectis.com