Slide 1: Commitment to a Cure
COMMITMENT TO A CURE cellectis.com
Slide 2: Forward-Looking Statements
FORWARD-LOOKING STATEMENTS This presentation contains “forward-looking” the risk that any one or more of our product statements that are based on our management’s candidates will not be successfully developed and current expectations and assumptions and on commercialized. information currently avail
Slide 3: Writing the History of Allogeneic CAR T-Cells
WRITING THE HISTORY OF ALLOGENEIC CAR T-CELLS 20 years 8 years 6 clinical trials of expertise in of experience in allogeneic ongoing as of 2020; gene editing CAR-T manufacturing 3 Cellectis-sponsored 3 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 INDIVIDUAL CAR-T DECISION PATIENT APHERESIS OF A SINGLE THERAPY PATIENT PRODUCT Allogeneic process: Consistent m
Slide 5: Partnerships with Industry Leaders
PARTNERSHIPS WITH INDUSTRY LEADERS Up to $3.2B in potential milestone payments plus royalties Exclusive license to 15 allogeneic CAR T- Up To $2.8B in Development & Sales Milestones Cell Targets Including UCARTBCMA/ALLO-7151 + High Single-Digit Royalties on Sales Exclusive license to CD19-directed a
Slide 6: Pipeline: Innovative Cancer Therapies for Unmet Needs
PIPELINE: INNOVATIVE CANCER THERAPIES FOR UNMET NEEDS Phase 1 Phase 1 Disease Product Study Preclinical Dose Escalation Dose Expansion Pivotal Phase* ACUTE MYELOID UCART123 AMELI-01 LEUKEMIA ACUTE LYMPHOBLASTIC UCART22 BALLI-01 LEUKEMIA MULTIPLE UCARTCS1 MELANI-01 MYELOMA ACUTE LYMPHOBLASTIC UCART19
Slide 7: Pipeline Targets Multiple Unmet Needs in Cancer
PIPELINE TARGETS MULTIPLE UNMET NEEDS IN CANCER Estimated numbers of new cases in 2020 ALL AML MM 48,300 30,000 32,270 19,940 6,150 7,500 U.S. E.U. U.S. E.U. U.S. E.U. Survival data 20% 5-year OS* in adults 27% 5-year OS in adults 50% 5-year OS in adults median disease-free <6 months survival in ped
Slide 8: Clinical Trial: Design of Phase Studies (Dose Finding)
CLINICAL TRIAL: DESIGN OF PHASE 1 STUDIES (DOSE FINDING) 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 for UCART123 DL:
Slide 9: UCART19: Proof of Concept / First Allogeneic CAR-T
UCART19: PROOF OF CONCEPT / FIRST ALLOGENEIC CAR-T PHASE 1 dose N=21 escalation in R/R ALL CALM PALL Safety – Primary Objective Efficacy – Secondary Objective 82% 0% Grade ≥2 skin Graft vs Host Disease 67% CR/CRi rate with optimal lymphodepletion overall CR/CRi rate 0% Grade 3-4 neurotoxicity 71% of
Slide 10: 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 11: 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 12: CD123 Target: Rationale for Therapy in Acute Myeloid Leukemia
CD123 TARGET: RATIONALE FOR THERAPY IN ACUTE MYELOID LEUKEMIA High expression on malignant cells Limited expression on healthy cells CD123 is expressed >90% on malignant cells in AML AML >90% Total bone marrow cells ~ 7% CD123 positive Normal ~ 7% Only ~ 1% expresses the antigen at high levels CD123
Slide 13: CD22 Target: Rationale for Therapy in Acute Lymphoblastic Leukemia
CD22 TARGET: RATIONALE FOR THERAPY IN ACUTE LYMPHOBLASTIC LEUKEMIA CD22 Expression in B-cells Potential in disease space Flow cytometric analysis of B-cell differentiation CD22 CD19 Relapses following CD19-directed CAR T-cell Pre- therapy can show loss of CD19 antigen but CAR persistent expression o
Slide 14: CS1-SLAMF7 Target: Rationale for Therapy in Multiple Myeloma
CS1-SLAMF7 TARGET: RATIONALE FOR THERAPY IN MULTIPLE MYELOMA High expression on malignant cells Clinical validation >95% expression in MM cells Elotuzumab is a monoclonal antibody targeting CS1 Elotuzumab is safe and effective in MM patients Elotuzumab (in combination with lenalidomide and • CS1 exp
Slide 15: UCART Manufacturing
UCART MANUFACTURING Frozen Thawed PBMCs Lentivector TALEN®-mediated gene leukopaks transduction editing 8 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 16: In-House Manufacturing
IN-HOUSE MANUFACTURING Raw materials Clinical & Commercial UCART Product Candidates Paris, France Raleigh, NC 14,000 sq ft. facility 82,000 sq ft. facility Production of clinical Operational “go-live” Production of clinical & Operational "go-live" starting materials targeted in 2020 commercial UCART
Slide 17: The Cellectis Group
THE CELLECTIS GROUP ~68.9%* ownership NASDAQ: CLLS NASDAQ: CLXT EURONEXT GROWTH: ALCLS $60M cash as of December 31, 2019 ~$304M** cash as of December 31, 2019 Expected to fund operations into mid-2021 Expected to fund operations into 2022 Based in Minnesota, USA Based in Paris, France, New York & Ra
Slide 18: Achieved Milestones in 2019 / Expected Milestones in 2020
ACHIEVED MILESTONES IN 2019 Proprietary clinical programs Partnered clinical programs Manufacturing UCARTCS1: Phase 1 R/R MM ongoing; first UCART19: Phase 1 in R/R ALL ongoing Ongoing construction of 2 in-house patient dosed in Q4 2019 manufacturing plants: UCART19 (ALLO-501): Phase 1 in R/R NHL UCA
Slide 19: Thank You
THANK YOU Reach us at: investor@cellectis.com 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