Celularity Inc. · NASDAQ:CELU
Celularity — Investor Presentation (July 2021)
51 slides from Celularity's investor presentation, July 2021
Slide 1: The Next Evolution in Cellular Medicine
THE NEXT EVOLUTION IN CELLULAR MEDICINE July 2021
Slide 2: Disclaimer
Disclaimer This presentation has been prepared for use by Celularity, Inc. (“Celularity”). This presentation, and any information disclosed, whether in writing or orally, to you as part of (or otherwise in connection with) this presentation, is for information purposes only and is being provided to
Slide 3: Our Vision
OUR VISION Next Evolution in Off-the-shelf Allogeneic Cellular Therapies, at Greater Scale & Quality with Attractive Economics To harness the placenta’s unique biology and ready availability to develop therapeutic solutions Lead the evolution in placental-derived therapeutics: advance the discovery
Slide 4: Key Investment Company Overview & Highlights
KEY INVESTMENT Company Overview & Highlights 1 Robust preclinical differentiation, encouraging clinical data and rapid path to approval Broad pipeline of novel, investigational product candidates across therapeutic areas and 2 indications of high unmet need 3 Proprietary placenta-based platform deve
Slide 5: Celularity: Company History
CELULARITY: COMPANY HISTORY Celgene Spin-out (2017) Leveraging 20+ Years of Cellular Therapeutics Innovation Celularity and Celularity GX Acquisition and GX FIH Genetically Corp. Announce close cryopreserved Allogeneic -modified Merger merger and Allogeneic Celgene & allogeneic Placental NK cell Agr
Slide 6: Celularity IMPACT Platform
CELULARITY IMPACT™ PLATFORM Capitalizing on the Benefits of Placental-Derived Cells to Target Multiple Diseases INHERENT ADVANTAGES OF PLACENTAL-DERIVED CELLS HEMATOLOGICAL ü Abundant and evergreen starting cell source for MALIGNANCIES allogeneic off-the-shelf therapies ü High expandability, persist
Slide 7: Single-Source, Placenta-Based Platform Driving Broad Pipeline
SINGLE-SOURCE, PLACENTA-BASED PLATFORM DRIVING BROAD PIPELINE Four Key Cell Types Driving Six Initial Indications and Potential for Further Expansion FUTURE OPPORTUNITIES AND INDICATIONS SINGLE SOURCE 4 ALLOGENEIC 6 INITIAL 4 KEY PROGRAMS MATERIAL CELL TYPES INDICATIONS Potential Future CAR Construc
Slide 8: Pipeline Program Milestones
PIPELINE Overview CELL TYPE PROGRAM INDICATION 2021 2022 Program Milestones B-Cell IND CAR-T CyCART-19 Phase I Phase II Malignancies Submission CYNK-001 § 2H21: Dose Selection & Initiation of Expansion Cohorts (AML) Unmodified § 2H21: Establish Phase II Dose CYNK-001 Acute Myeloid Natural Phase I Ph
Slide 9: Manufacturing Overview
MANUFACTURING OVERVIEW Fully Integrated, Purpose Built Commercial Scale Manufacturing Site Including Translational Research & Biorepository PURPOSE BUILT FACILITY FOR STAFFED BY OVER 100 HIGHLY COMMERCIAL SCALE, GMP-READY COMMERCIAL-SCALE CELLULAR SPECIALIZED SCIENTISTS, ENGINEERS THERAPEUTIC MANUFA
Slide 10: Placenta Derived Cells
PLACENTA DERIVED CELLS Biological Characteristics Suggest Greater Therapeutic Potential
Slide 11: Stemness: The Placental Advantage
STEMNESS: The Placental Advantage At the core of Celularity’s approach is the innate stemness of its starting material Greatest Proliferative Potential Extended Persistence: and Sustained Activity: As nature’s universal donor tissues, Placental-derived pluripotent stem cells placental-derived plurip
Slide 12: Greater T Stem Cell Memory Characteristics
Greater T Stem Cell Memory Characteristics Stem Cell Memory = Greatest Proliferative Potential, Persistence and Survival Benefit Day 0 P-T cells Day 15 CD19 CAR+ T cells P -T c e ll D iffe r e n tia tio n S ta tu s C y C A R T -1 9 C A R + M a r k e r E x p r e s s io n C D 1 9 C A R + T c e ll D if
Slide 13: Enhanced Efficacy & Persistence, Prolonged Immune Attack upon Tumor Recharging
Enhanced Efficacy & Persistence, Prolonged Immune Attack upon Tumor Recharging Survival CyCART: Durable Persistence 1 0 0 R e - c h a lle n g e 8 0 (d 1 2 2 ) S u r v iv a l 6 0 4 0 2 0 0 0 3 0 6 0 9 0 1 2 0 1 5 0 1 8 0 2 1 0 D a y s § CyCART-19 demonstrates significantly reduced tumor burden and su
Slide 14: Persistence, Maturation and Proliferation with Absence of Allo-HLA Antibodies
Persistence, Maturation and Proliferation with Absence of Allo-HLA Antibodies CYNK-001 demonstrated persistence up to 28 days (mean=11days) Persistent CYNK-001 cells matured and proliferated CYNK-001 demonstrated effector Absence of allo-HLA antibodies in No detectable exhaustion on CYNK-001 cells f
Slide 15: CYNK-001 AML & GBM
CYNK-001 AML & GBM
Slide 16: CYNK-001 Overview: Manufacturing Complexity
CYNK-001(unmodified NK cellular therapy) Overview RATIONALE NK CELL THERAPIES § NK cells are natural immune cells that eradicate both cancer and virus- infected cells ADULT CELULARITY Cell Therapy – Key mediators of antibody-dependent cellular cytotoxicity (ADCC) Technology Scorecard DONOR DERIVED C
Slide 17: AML: Pre-Clinical Data
AML: PRE-CLINICAL DATA Evidence of Significant Leukemia Killing CML, AML, MM IN VITRO KILLING IFN-G PRODUCTION PRIMARY AML KILLING CML AML MM CYNK-001 demonstrates robust killing (cytolytic) CYNK-001 exerted up to 60% specific lysis CYNK-001 activation releases high concentration against CML, AML, M
Slide 18: CYNK-001-AML-001 First-in-Human Study
CYNK-001-AML-001 FIRST-IN-HUMAN STUDY Phase I Study in Relapsed / Refractory Acute Myeloid Leukemia Showed Early Signs of Clinical Benefit PHASE I RESULTS PHASE I DESIGN § CYNK-001 well tolerated in a heavily pre-treated AML § Dose escalation study patient population § Conditioning with cyclophospha
Slide 19: CYNK-001-AML-001 First In-Human Study Translational Data
CYNK-001-AML-001 First In-Human Study Translational Data CYNK-001 demonstrated persistence up to 28 days (mean=11days) Persistent CYNK-001 cells matured and proliferated CYNK-001 demonstrated effector Absence of allo-HLA antibodies in No detectable exhaustion on CYNK-001 cells function post infusion
Slide 20: CYNK001-AML-002 Trial Design
CYNK001-AML-002 (RELAPSED/REFRACTORY AML) Phase 2 - Registrational SCREENING Lymphodepletion (Days -6, -5, -4, -3) NK Cell Induction (Up to 28 days) ENROLLMENT Cyclophosphamide IL-2 (6M IU) Relapsed/Refractory (Days 1,3, 5, 8, 10, 12 15) AML 900 mg/m2/day (IL-2 administered 1 - 3 24 Month Follow Up
Slide 21: CYNK001-AML-003 Trial Design
CYNK001-AML-003 (AML MINIMAL RESIDUAL DISEASE POSITIVE) Phase 2 - Registrational Lymphodepletion Placebo Induction (Days -6, -5, -4, -3) IL-2 (6M IU) Cyclophosphamide (Days 1,3, 5, 8, 10, 12 15) Allogeneic Stem Cell 12 Month Follow Up 900 mg/m2/day (IL-2 administered 1 - 3 Transplant RANDOMIZATION h
Slide 22: CYNK001-GBM-002 Glioblastoma Program
CYNK001-GBM-002 (GLIOBLASTOMA PROGRAM) Phase 1 Dose Escalation / Phase 2 Registrational Cohort 1 Screening Intravenous Intracavitary Up to 28 days Resection (2.4 x109 cells – D1,D8,D15) (100x106 cells – (3 out of 4 wks) recurrent resection eligible GBM Cohort 2 Intravenous Intracavitary Lymphodeplet
Slide 23: CYNK-101 HER2+ Advanced Esophageal / Gastric Adenocarcinoma
CYNK-101 HER2+ Advanced Esophageal / Gastric Adenocarcinoma
Slide 24: CYNK-101 in HER2+ Gastric Cancer Overview
CYNK-101 IN HER2+ GASTRIC CANCER Overview RATIONALE § Engineering CYNK cells with high affinity and cleavage resistant (CD16VP) expected to improve affinity for IgG1 therapeutic antibodies, resist activation induced cleavage and improve overall ADCC potential KEY HIGHLIGHTS § CYNK-101 adds “punching
Slide 25: CYNK-101 Demonstrates Effective Antitumor Activity
CYNK-101 DEMONSTRATES EFFECTIVE ANTITUMOR ACTIVITY Against Gastric Cancer Cell Lines in Conjunction with Anti-HER2 Monoclonal Antibody RESULTS Target cells alone Killing Index § Significant ADCC activity of CYNK-101 in combination with Herceptin against both gastric cancer cell lines was shown at E:
Slide 26: CYNK-101 Provides a Backbone for Combination Therapies
CYNK-101 PROVIDES A BACKBONE FOR COMBINATION THERAPIES Enhanced ADCC with Multiple Antibodies Forms the Basis of Combination Therapy ADCC WITH RITUXIMAB ADCC WITH ELOTUZUMAB ADCC WITH DARATUMUMAB % Specific Lysis % Specific Lysis % Specific Lysis E:T Ratio (Against Daudi) CYNK-101 + mAb CYNK + mAb C
Slide 27: CYNK101-HER2-001 Trial Design
CYNK101-HER2-001 (HER2+ GASTRIC/GEJ CANCER) Phase 1 - Dose Escalation / Phase 2a Expansion/Proof of Concept Induction NK Cell Re- Induction Maintenance (4 months) Lymphodepletion Pembrolizumab (Day 22 q 3 weeks) (Days -6, -5, -4, -3) 200 mg (Day 1) Pembrolizumab Pembrolizumab 200 mg q 3 weeks CR, PR
Slide 28: CyCART-19 B-Cell Malignancies
CyCART-19 B-Cell Malignancies
Slide 29: CyCART-19 Overview
CyCART-19 OVERVIEW Celularity Approach and Advantages RATIONALE CAR-T THERAPIES § Rationale for greater stemness, expandability, persistence Cell Therapy OTHER CELULARITY § Abundant renewable starting cell source for allogeneic therapies AUTOLOGOUS Technology Scorecard ALLOGENEIC CyCART-19 § Potenti
Slide 30: CyCART-19 Greater T Stem Cell Memory Characteristics
CELULARITY CyCART-19 Greater T Stem Cell Memory Characteristics Stem Cell Memory = Greatest Proliferative Potential, Persistence and Survival Benefit Established Robust Manufacturing Process Day 0 P-T cells Day 15 CD19 CAR+ T cells P -T c e ll D iffe r e n tia tio n S ta tu s C y C A R T -1 9 C A R
Slide 31: CyCART-19 Demonstrates Greater Anti-Lymphoma Activities & Survival
CyCART-19 DEMONSTRATES GREATER ANTI-LYMPHOMA ACTIVITIES & SURVIVAL Enhanced Efficacy & Persistence, Prolonged Immune Attack upon Tumor Recharging Survival CyCART: Durable Persistence 1 0 0 R e - c h a lle n g e 8 0 (d 1 2 2 ) S u r v iv a l 6 0 4 0 2 0 0 0 3 0 6 0 9 0 1 2 0 1 5 0 1 8 0 2 1 0 D a y s
Slide 32: CyCART-19 Cells Do Not Induce Xenogeneic GvHD In Vivo
CyCART-19 CELLS DO NOT INDUCE XENOGENEIC GvHD IN VIVO Efficient TCR Knockout as Risk Mitigation Endpoint PBMC (IV 30M) • Body Weight P-T cells (IV, 30M) • CD3+ T cells in blood P -C D 1 9 C A R T C R a /b + F re q u e n c y NCG mice Day 0 Day 45 1 0 0 8 0 (% ) C D 3+ 6 0 a /b + 100 W eek 2 W eek 4 P
Slide 33: CYCART19-BCM-001 Trial Design
CYCART19-BCM-001 (RELAPSED/REFRACTORY B-CELL MALIGNANCIES) Phase 1 Dose Escalation / Phase 2 Registrational Phase I Phase 2: Registrational Arms Safety and Dose Finding Stage 1 Stage 2 ARM 1: High Grade NHL CD-19 targeted therapy naïve (n= ~30) Dose Level 1: ARM 1: High Grade NHL • DLBCL 40 x106 cel
Slide 34: Degenerative Diseases
Degenerative Diseases
Slide 35: APPL-001 Overview
APPL-001 OVERVIEW Celularity Approach and Advantages RATIONALE § Immune-modulatory properties of mesenchymal-like adherent stromal cells (ASCs) have the potential to alleviate autoimmunity and possess anti-inflammatory activity § Off-the-shelf potential – ASCs are immune-privileged due to relatively
Slide 36: Allogeneic Placental Pluripotent Cells: Scalable & Off-the-Shelf
ALLOGENEIC PLACENTAL PLURIPOTENT CELLS: SCALABLE & OFF-THE-SHELF Clinical Stage Master Cell Stock Target Product Profile § Culture expanded, undifferentiated Mesenchymal-like Stromal Cells (MSCs) § Composition: CD34-, CD10+, CD105+, and CD200+ § MOA: Immune-modulatory, anti-inflammatory, Working Cel
Slide 37: Newly Developed APPL Program
NEWLY DEVELOPED APPL PROGRAM Leveraging Legacy Placental Mesenchymal-like Stromal Cell Studies to Expand to Degenerative Diseases IV Formulation IM Formulation 50+ patients dosed in multiple Crohn’s Disease studies 140+ patients dosed in Diabetic Foot Ulcer (DFU) and Diabetic Peripheral Neuropathy P
Slide 38: Newly Developed APPL Program: Genetically Modified APPL
NEWLY DEVELOPED APPL PROGRAM Leveraging PDA Cells and Develop New APPL Candidate Genetically Modified APPL with Greater Safety Profile § Tissue factor (TF) Knockout (KO) in APPL using CRISPR/Cas9 to reduce potential safety risk associated with TF § Identified two of four CRISPR guide RNAs showing >9
Slide 39: APPL in Crohn's Disease Summary
APPL IN CROHN’S DISEASE Summary KEY TAKEAWAYS CLINICAL PLAN § Culture-expanded, undifferentiated mesenchymal-like stromal cells § 1H22: IND Submission – Genetically modified with tissue factor (TF) knockout (KO) § 1H22: Phase I/IIa Study Start § Mechanism of Action: – Immune-modulatory, anti-inflamm
Slide 40: Corporate Summary
Corporate Summary
Slide 41: Experienced Management Team
EXPERIENCED MANAGEMENT TEAM With Deep Expertise in Cell Therapy Executive Leadership Team Robert J. Hariri, Andrew Pecora, John David Bradley Glover, Anne Jones, MD, PhD MD, FACP, CPE Haines Beers PhD PhD Founder & Senior Advisor Chief Operating Chief Financial Chief Technical Chief Business CEO Off
Slide 42: Near-Term Milestones
NEAR-TERM MILESTONES To Achieve the Next Advance in Placenta-based Cell Therapy Achievements to Date Key Near-Term Development Milestones April 2020: Received FDA Safe to proceed on IND CYNK-001 for CYNK-001 in COVID-19 § 2H21: Establish Phase 2 Dose (GBM) § 2H21: Dose Selection & Initiation of Expa
Slide 43: Appendix Clinical Programs
Appendix Clinical Programs Additional Detail
Slide 44: Broad IP Protection Across All Lead Programs
CELULARITY IMPACT™ PLATFORM Broad IP Protection Across All Lead Programs PNK Broad Background Patent Process Patents PLACENTAL-DERIVED CELLULAR PLATFORMS 15 PATENT PLACENTAL NK FAMILIES Genetically Modified PNK Patents Treatment of AML & MM Cryopreserved PNK (CYNK-001) Patents Treatment of GBM Proce
Slide 45: The Placenta as a Renewable Allogeneic Source
CELULARITY IMPACT™ PLATFORM The Placenta as a Renewable Allogeneic Source, with Purpose-Built Commercial Scale Manufacturing PROCESSES PRODUCTS INDICATION PLACENTAL-DERIVED CELLULAR PLATFORMS Acute Myeloid Leukemia Lymphocytic Progenitors CYNK-001 Glioblastoma Multiforme Placental NK CD34+ Cells Gas
Slide 46: Celularity Placental CAR-T: Solving the Downside of Autologous CAR-T
CELULARITY PLACENTAL CAR-T (CyCART) Solving the Downside of Autologous CAR-T Therapies AUTOLOGOUS CAR-T THERAPY ALLOGENEIC PLACENTAL CAR-T Status Quo Downside Celularity’s Scalable Solution Ø All CAR T-cell therapies on ü No apheresis capacity constraints § Complex, high COGS the market and most (~7
Slide 47: Celularity Placental CAR-T: Upside to Adult-donor Allogeneic CAR-T
CELULARITY PLACENTAL CAR-T (CyCART) Providing Upside to Adult-donor Allogeneic CAR-T Therapies ALLOGENEIC CAR-T THERAPY ALLOGENEIC PLACENTAL CAR-T Status Quo Downside Celularity’s Scalable Solution § Complex logistics, multistep Ø Requires selection, manufacturing process to ü No apheresis capacity
Slide 48: Celularity Placental NK Cells: Upside to Adult-donor NK Cells
CELULARITY PLACENTAL NK CELLS Providing Upside to both Adult-donor NK Cells ADULT DONOR NK CELL THERAPY ALLOGENEIC PLACENTAL NK Peripheral Blood NK iPSC NK Celularity’s Scalable Solution Ø Apheresis of peripheral blood § De-differentiated adult ü No apheresis capacity constraints from healthy donor
Slide 49: NK Cell Therapy for Cancer Immunotherapy
PIPELINE NK CELL THERAPY FOR CANCER IMMUNOTHERAPY Preclinical & Clinical Data Supporting Role of NK cells in the Treatment of Cancer NK CELLS ARE A MAJOR COMPONENT OF THE INNATE IMMUNE SYSTEM § Natural immune cells that eradicate both cancer and virus-infected cells – Directly via cytolytic granule
Slide 50: APPL Crohn's Disease Study Design
APPL CROHN’S DISEASE (CD) Study Design Study Design: Randomized, double-blind, Placebo-controlled study in adults with 5 doses of 1/4th unit APPL (~ 37 million cells) over 8 weeks vs. Humira treatment. Study Population: Moderate-to-Severe CD (CDAI score: 220-450) who are refractory to Corticosteroid
Slide 51: CONFIDENTIAL Page 51
CONFIDENTIAL Page 51