Cellectis S.a. · EURONEXT:ALCLS
Cellectis — Investor Presentation (March 2025)
25 slides from Cellectis's investor presentation, March 2025
Slide 1: Commitment to a Cure
Commitment to a Cure Corporate Presentation March 2025 NASDAQ: CLLS EURONEXT GROWTH: ALCLS.PA
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 2 Clinical Trials* Global GMP Facilities Near-Term Clinical Catalysts $264M** as of December 31, 2024 75+ patients dosed in End-to-end manufacturing Multiple near-term UCART Provides runway into Cellectis-sponsored trials*** autonomy clinical data updates mid-2027 Diversified P
Slide 4: Strategic Partnership with AstraZeneca
Strategic Partnership with AstraZeneca Cell & Gene Therapy R&D Collaboration Investment Agreements • Develop up to 10 novel products in oncology, • $220M equity investment (subscribed for 16 immunology and rare diseases million ordinary shares and 28 million convertible preferred shares at $5.0 per
Slide 5: A Highly-Experienced Executive Committee
A Highly-Experienced Executive Committee André Choulika, Ph.D. Steven Doares, Ph.D. Phillippe Duchateau, Ph.D. Adrian Kilcoyne M.D., MPH, MBA Kyung Nam-Wortman Founder & CEO SVP, US Manufacturing Chief Scientific Officer Chief Medical Officer EVP, Chief Human & Site Head Resources Officer Stephan Re
Slide 6: Integrated and Controlled Manufacturing
Integrated and Controlled Manufacturing Discover, Develop, Manufacture, Test and Release Paris 6
Slide 7: Cellectis' End-to-End Cell & Gene Therapy Platform
Cellectis’ End-to-End Cell & Gene Therapy Platform New York, NY Paris, France Raleigh, NC Allogeneic In Vivo Gene CAR-T Therapy Platform Innovation & CMC Development, UCART – Clinical & Clinical Development Starting Materials Paris Intended Commercial Site 25,000 sq ft. facility 55,000 sq ft. facili
Slide 8: Differentiated Targets & Near-Term Catalysts
Differentiated Targets & Near-Term Catalysts Target Indication Study Preclinical Phase 1 Phase 21 Upcoming expected milestone Phase 1 dataset & late-stage UCART22 BALLI-01 Fully Owned ALL development strategy expected in Q3 CD22 NCT04150497 2025 UCART20x22 NatHaLi-01 Phase 1 ongoing with readout NHL
Slide 9: Cellectis' UCART Platform
Cellectis’ UCART Platform 9
Slide 10: UCART Designed with Cellectis' Differentiated Gene Editing Platform
UCART are Designed with Cellectis’ Differentiated Gene Editing Platform CAR-T cell CD52 KO TRAC KO Resistance to Minimizes risk of TALEN® Powered Gene Editing: anti-CD52 Abs GvHD PRECISE Targets desired site with a maximum range of 7 base pairs SAFE Protein/DNA interaction with 32 base pairs recogni
Slide 11: UCART22 and UCART20x22 Positioning
UCART22 and UCART20x22 Positioning LCAR-AIO CD19/CD20/CD22 Post-CD19 CAR-T KITE-363/ KITE-753 autologous CD19/CD20 • CD20 & CD22: Key targets validated in treatments oncology and autoimmune diseases C-CARO39 • UCART22: First-in-class allogeneic CD19/CD20 CD22 CAR-T for ALL. Plan to advance into pote
Slide 12: UCART22 Study Design
1 2 UCART22 Study Design Administering UCART22-P2 (cells manufactured by Cellectis) with FCA Key inclusion criteria: lymphodepletion: • Age 15–70 years, adequate organ function, ECOG PS ≤1 Dose Escalation Dose Expansion • B-ALL blast CD22 expression ≥70% mTPI design • 2-4 pts/cohort • Received ≥1 st
Slide 13: Outcomes of UCART22 Patients Treated at DL2
Outcomes of UCART22 Patients Treated at DL2 3 patients were enrolled into the first UCART22 cohort at DL2: Patient 1 Patient 2 Patient 3 MRD negative MLFS MRD negative CR Refractory 17-year old 68-year old 27-year old • Ph-negative B-ALL • Ph-negative B-ALL • B-ALL with ABL2 fusion • Hypodiploid kar
Slide 14: Expansion of UCART22 Cells in Peripheral Blood Correlates with Serum Ferritin
Expansion of UCART22 Cells in Peripheral Blood Correlates with an Increase in Serum Ferritin Levels Patient 1 Patient 2 Patient 3 100 8000 100 4000 1.0 1500 UCART22 cell/uL UCART22 cell/uL 80 80 UCART22 cell/uL 0.8 6000 3000 Ferritin ng/ml Ferritin ng/ml Ferritin ng/ml 1000 60 60 0.6 4000 2000 40 40
Slide 15: Summary of UCART22 Patients Treated at DL2
Summary of UCART22 Patients Treated at DL2 Safety • No dose-limiting toxicities (DLT) • No immune effector cell-associated neurotoxicity syndrome (ICANS) • No GvHD • CRS in 2/3 (67%) patients with one G1 that resolved without treatment and one G2 that resolved after tocilizumab x1 • Patient 1 had a
Slide 16: UCART20x22 Study Design
1 6 UCART20x22 Study Design Key inclusion criteria: • Age 18–80 years Dose Escalation Dose Expansion • Mature B-cell NHL except CLL/SLL, BOIN design • 2-4 pts/cohort Richter’s from CLL/SLL, Burkitt’s lymphoma, or Waldenstrom’s macroglobulinemia 450 × 106 cells DL 3 • Tumor positive for CD20 and/or C
Slide 17: UCART20x22 Baseline Characteristics
UCART20x22 Baseline Characteristics As of 28 July 2023, 3 patients received LD and were treated with UCART20x22 at Dose Level 1 (50 × 106 cells) Pt 1 Pt 2 Pt 3 Age 76 65 18 NHL Subtype DLBCL Transformed FL Transformed MZL Genetic/Molecular Double expressor (BCL2, MYC) Triple-hit NOTCH1, PLCG2, CCND3
Slide 18: UCART20x22 Safety Summary
1 8 UCART20x22 Safety Summary • No UCART20x22-related DLTs • No ICANS or GVHD was observed • One CLLS52-related DLT of bone marrow aplasia after Day 42 thought to be due to cumulative chemotherapy exposure in a patient with baseline Grade 1/2 cytopenias and bone marrow hypocellularity at screening •
Slide 19: Outcomes of UCART20x22 Patients Treated at DL1
Outcomes of UCART20x22 Patients Treated at DL1 3 patients were treated at dose level 1 (50 × 106 cells) and were evaluable for response: Patient 1 Patient 2 Patient 3 Partial metabolic Complete metabolic Complete metabolic response response response 76-year old 65-year-old 18-year-old • Double-expre
Slide 20: Robust Expansion of UCART20x22 Cells in Peripheral Blood
Robust Expansion of UCART20x22 Cells in Peripheral Blood Correlates with an Increase in Serum Ferritin Levels Patient 1 Patient 2 Patient 3 800 3000 500 2000 20 15000 UCART20x22 cells/uL UCART20x22 cells/uL UCART20x2 cells/uL 400 600 1500 15 Ferritin ng/ml Ferritin ng/ml Ferritin ng/ml 2000 10000 30
Slide 21: UCART20x22 Cells Detected in Day 9 Post-Treatment Biopsy for Patient 3
UCART20x22 Cells Detected in Day 9 Post-Treatment Biopsy for Patient 3 Negative Control Positive Control UCART20x22 Baseline UCART20x22 Day 9 UCART20x22 cells were measured by RNAscope using a probe set specific for the UCART22 targeted sequence, at 20X magnification. No staining was observed with t
Slide 22: Key Takeaways – Why Cellectis?
Key Takeaways – Why Cellectis? Innovative Allogeneic Breaking Paradigms with Life-Saving Therapies CAR T End-to-End In-House Owning Manufacturing is Owning the Product Manufacturing Best-In-Class Gene Designed to be Safe, Precise & Efficient, Backed by Editing Platform Strong IP Strong Partnerships
Slide 23: Why TALEN?
Why TALEN ®? Maturity Genome Recognition Chromotrypsis Precision Vectorization IP Outreach Site # base pairs In clinic since Euchromatin & Every 7 Strong for TALEN® 2015 heterochromatin 32 Not reported base pairs mRNA CLLS Every 64 In clinic since Euchromatin ~20 RNP Scattered CRISPR 2018 only Yes b
Slide 24: Diversified Partnerships with Industry Leaders
Diversified Partnerships with Industry Leaders CAR-T CAR-T Mitochondrial Cell and gene BCMA, CD70 + 13 TILs CD19 DNA editing therapies targets Exclusive worldwide U.S. rights Exclusive worldwide Research Collaboration Joint Research and license to CD19- sublicensed to license to 15 collaboration and
Slide 25: 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 25 Cellectis®, TALEN® and our corpora te logo s are reg iste