Cellectis S.a. · EURONEXT:ALCLS
Cellectis — Investor Presentation (January 2021)
19 slides from Cellectis's investor presentation, January 2021
Slide 1: Commitment to a Cure: Investor Presentation January 2021
COMMITMENT TO A CURE Investor Presentation January 2021 cellectis.com
Slide 2: Forward-Looking Statements
FORWARD-LOOKING STATEMENTS This presentation contains “forward-looking” statements that manufacturing delays problems; inability to achieve enrollment are based on our management’s current expectations and disagreements with our collaboration partners of collaboration assumptions and on information
Slide 3: Writing the History of Allogeneic CAR T-Cells
WRITING THE HISTORY OF ALLOGENEIC CAR T-CELLS 21 years 9 years 7 clinical trials of expertise in of experience in allogeneic ongoing as of 2020; gene editing CAR-T manufacturing 3 Cellectis-sponsored 4 partnered INVENTORS / PIONEERS OF GENE EDITING & ALLOGENEIC CAR T-CELLS In 2012 . . In 2015 . . Mi
Slide 4: Advantages of Allogeneic vs. Autologous CAR-T
ADVANTAGES OF ALLOGENEIC VS. AUTOLOGOUS CAR-T Autologous process: Manufacturing variability + several weeks before treatment is available 1. 2. 3. 4. CANCER TREATMENT CANCER MANUFACTURING OF A INDIVIDUAL CAR-T DECISION PATIENT APHERESIS SINGLE PATIENT THERAPY PRODUCT Allogeneic process: Consistent m
Slide 5: UCARTs – Allogeneic CAR T-Cells Through Precision Gene Editing
UCARTs – ALLOGENEIC CAR T-CELLS THROUGH PRECISION GENE EDITING RATIONAL Engineering EFFECTIVE via accurate insertion of Persistence best CAR via knock-out of CD52 or b2-microglobulin UCART Universal Chimeric Antigen Receptor T-cell TUMOR CELL SMART SAFE Action Administration CAR linked to suicide sw
Slide 6: TALEN Gene Editing – Advantages
TALEN® GENE EDITING – ADVANTAGES TALEN®: Driven by protein/DNA interactions to work on potential off- Our nucleases act like DNA scissors to edit genes at precise target sites: site cleavage + Releases DNA ends accessible to DNA repair mechanisms to DNA sequence perform gene insertions and correction
Slide 7: UCART Manufacturing
UCART MANUFACTURING Frozen Thawed PBMCs Lentivector TALEN®-mediated gene leukopaks transduction editing 9 years of experience in allogeneic CAR-T 1 2 3 4 manufacturing Validated gene editing technology for cell manufacturing 4 UCART product candidates manufactured so far Cell expansion Purification
Slide 8: Partnerships with Industry Leaders
PARTNERSHIPS WITH INDUSTRY LEADERS Up to $3.2B in potential milestone payments plus royalties Partner License Geography Most Advanced Targets Status Economics to Cellectis Exclusive license to CD19- UCART19 Up To $410M In directed allogeneic CAR T- Ex-US Ph1 (Anti-CD19) Development & Cells Sales Mil
Slide 9: Pipeline: Innovative Cancer Therapies for Unmet Needs
PIPELINE: INNOVATIVE CANCER THERAPIES FOR UNMET NEEDS Product Disease Study Phase 1 Phase 1 Pivotal Phase2 Dose Escalation Dose Expansion ACUTE MYELOID UCART123 AMELI-01 LEUKEMIA ACUTE UCART22 LYMPHOBLASTIC BALLI-01 LEUKEMIA MULTIPLE UCARTCS1 MELANI-01 MYELOMA ACUTE UCART193 LYMPHOBLASTIC CALM/PALL
Slide 10: Clinical Trial: Design of Phase 1 Dose Escalation Studies
CLINICAL TRIAL: DESIGN OF PHASE 1 DOSE ESCALATION STUDIES Primary Objectives: Secondary Objectives: Safety and Identification Efficacy and Correlative of Optimal Dose Studies Dose Escalation: Optimal dose definition DL1 DL2 DL3 DL4 2-4 patients 2-4 patients 2-4 patients 2-4 patients DL: Dose Level P
Slide 11: ALLO-501: Cellectis-Licensed Allogeneic CAR-T
ALLO-501*: CELLECTIS-LICENSED ALLOGENEIC CAR-T PHASE 1 dose escalation in N=22 (safety) R/R Non-Hodgkin Lymphoma N=19 (efficacy) ALPHA Study Safety – Primary Objective Efficacy – Secondary Objective 0% Graft vs Host Disease 63% Overall Response Rate 37% Complete Response Rate 0% ICANS (Immune Effect
Slide 12: UCART22: Initial Anti-Leukemic Activity in BALLI-01
UCART22: Initial Anti-Leukemic Activity in BALLI-01 Phase 1 in R/R B-ALL PHASE 1 dose escalation in N=5 (safety) R/R Adult B-Acute N=5 (efficacy) Lymphoblastic Leukemia BALLI-01 Study Efficacy – Secondary Objective Preliminary data from 5 patients who received UCART22 at DL1 or DL2 Patients at DL1 a
Slide 13: UCART22 in Acute Lymphoblastic Leukemia
UCART22 IN ACUTE LYMPHOBLASTIC LEUKEMIA ALL Incidence Rates & Survival Data 6,150 20% <6 Estimated new cases of ALL in 5-year OS in adults Months median disease-free survival the US for 2020 in R/R pediatric patients CD22 Expression in B-cells Treatment potential for CD19-negative patients Flow cyto
Slide 14: UCART123 in Acute Myeloid Leukemia
UCART123 IN ACUTE MYELOID LEUKEMIA AML Incidence Rates & Survival Data 19,940 27% 6% Estimated new cases of AML in the US for 2020 5-year OS in adults 5-year OS in adults >55 years old High CD123 expression on malignant cells Limited CD123 expression on healthy cells CD123 is expressed >90% on malig
Slide 15: UCARTCS1 in Multiple Myeloma
UCARTCS1 IN MULTIPLE MYELOMA MM Incidence Rates & Survival Data 32,270 43-83 50% Estimated new cases of MM in the US for 2020 Months is median OS for stages 2-3 5-year OS in adults High expression on malignant cells Treatment alternative to BCMA-targeted therapies >95% Many BCMA-targeted cell therap
Slide 16: A Cross Atlantic Organization
A CROSS ATLANTIC ORGANIZATION US Footprint EU Footprint New York, NY - Gene Editing Platform - Innovation - Clinical Development Paris, France Paris - Process & Analytical Development - Starting Materials Manufacturing • 14,000 sq ft. facility • Manufacturing operations went live in 2020 Raleigh, No
Slide 17: The Cellectis Group
THE CELLECTIS GROUP ~64.7%* ownership NASDAQ: CLLS NASDAQ: CLXT EURONEXT GROWTH: ALCLS • $30M cash and cash equivalents as of September 30, 2020 • ~$278M** cash and cash equivalents (before the $15M Registered Direct as of September 30, 2020 Offering in October 2020) • Expected to fund operations in
Slide 18: Achieved Milestones and Expected Future Milestones
ACHIEVED MILESTONES Proprietary clinical programs Partnered clinical programs Manufacturing UCART22: Phase 1 in R/R ALL ongoing; data UCART191: Phase 1 in R/R ALL near 2 in-house GMP manufacturing plants: presented at ASH 2020 completion Paris, France facility has started manufacturing UCARTCS1: Pha
Slide 19: Thank You
THANK YOU Reach us at: [email protected] Cellectis Paris Cellectis New York Cellectis Raleigh 8, rue de la Croix Jarry 430 East 29th Street 2500 Sumner Boulevard 75013 Paris – France 10016 New York, NY – USA 27616 Raleigh, NC – USA P19