Celularity Inc. · NASDAQ:CELU
Celularity — Investor Presentation (October 2021)
44 slides from Celularity's investor presentation, October 2021
Slide 1: The Next Evolution in Cellular Medicine
THE NEXT EVOLUTION IN CELLULAR MEDICINE October 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: Celularity's Vision
CELULARITY’S VISION To deliver innovative off-the-shelf allogeneic cellular medicines for patients with high unmet need at unparalleled scale, quality and cost. Lead the evolution in placental-derived therapeutics: advance the discovery of the placenta as a limitless, renewable source of neonatal ce
Slide 4: Celularity: Company History
CELULARITY: COMPANY HISTORY Celgene Spin-out (2017) Leveraging 20+ Years of Cellular Therapeutics Innovation Celularity and GX Acquisition FIH Genetically Corp. Announce cryopreserved Allogeneic -modified Merger Celularity allogeneic Placental NK cell Agreement to Enters into formed from placental-
Slide 5: Celularity Placental-Derived Product Platform
CELULARITY PLACENTAL-DERIVED PRODUCT PLATFORM Capitalizing on the Benefits of Placental-Derived Cells to Target Multiple Diseases One Placenta → Many Patients Cryopreserved - On Demand T Cell Isolation + Genetic Modification • Universal donor material & Off-The-Shelf • No requirement for matching •
Slide 6: Single-Source, Placenta-Based Platform Driving Broad Pipeline
SINGLE-SOURCE, PLACENTA-BASED PLATFORM DRIVING BROAD PIPELINE 4 Key Cell Types Driving 6 Clinical Indications and Potential for Broad Expansion FUTURE OPPORTUNITIES AND INDICATIONS SINGLE SOURCE 4 ALLOGENEIC 6 INITIAL 4 KEY PROGRAMS MATERIAL CELL TYPES INDICATIONS Potential Future CAR Constructs for
Slide 7: 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 8: Our Experienced Leadership Team
OUR EXPERIENCED LEADERSHIP TEAM With Deep Expertise in Cell Therapy Executive Leadership Team Robert J. Hariri, Andrew Pecora, John David Bradley Glover, Anne Jones, Beloo Mirakhur, Dr. Stephen Keary MD, PhD MD, FACP, CPE Haines Beers PhD PhD MD, PhD Brigido Dunn, Esq. Founder, Chairman President Ch
Slide 9: Pipeline
PIPELINE Overview CELL TYPE PROGRAM INDICATION 2021 2022 Program Milestones CYNK-001 Acute Myeloid Phase I Phase II (cryopreserved) Leukemia (AML) CYNK-001 Unmodified ▪ 2H21: Dose Selection & Initiation Natural of Expansion Cohorts (AML) Killer Cell CYNK-001 Glioblastoma ▪ 2H21: Establish Phase II D
Slide 10: 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, Placenta-derived pluripotent stem cells placental-derived pluripo
Slide 11: 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 12: Enhanced Efficacy & Persistence, Prolonged Immune Attack upon Tumor Rechallenge
Enhanced Efficacy & Persistence, Prolonged Immune Attack upon Tumor Rechallenge 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 s
Slide 13: Persistence, Maturation and Proliferation with Absence of Allo-HLA Antibodies
Persistence, Maturation and Proliferation with Absence of Allo-HLA Antibodies PNK-007 demonstrated persistence up to 28 days (mean=11days) Persistent PNK-007 cells matured and proliferated PNK-007 demonstrated effector function Absence of allo-HLA antibodies in No detectable exhaustion on PNK-007 ce
Slide 14: CYNK-001 AML & GBM
CYNK-001 AML & GBM
Slide 15: CYNK-001 (unmodified NK cellular therapy) Overview
CYNK-001(unmodified NK cellular therapy) Overview RATIONALE ▪ NK cells are natural immune cells that eradicate both cancer and virus- NK CELL THERAPIES infected cells ADULT CELULARITY Cell Therapy DONOR CYNK-001 & – Key mediators of antibody-dependent cellular cytotoxicity (ADCC) Technology Scorecar
Slide 16: PNK-007-AML-001 First-in-Human Study
PNK-007-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 ▪ PNK-007 well tolerated in a heavily pre-treated AML patient population ▪ Dose escalation study – 11 r/r AML patients enrolled, 10
Slide 17: Phase 1 Study Schema – CYNK001-AML-001
CYNK001-AML-001 (MRD+/RELAPSED REFRACTORY AML) Phase 1 Study Schema – In Progress LD Regimen #1 In Progress Lymphodepletion for Cohorts 1 to 3 Cohort 4a: Cohort 5a: Cohort 6a: (Days -5, -4, -3) Completed AML MRD+ AML MRD+ AML MRD+ EXPANSION COHORT Cyclophosphamide AML MRD+ (n=7) 1.8B Cells of 1.8B C
Slide 18: CYNK001-GBM-002 (Glioblastoma Program)
CYNK001-GBM-002 (GLIOBLASTOMA PROGRAM) Planned Phase 1 Dose Escalation / Phase 2 Proof of Concept Cohort 1 Screening Intravenous Resection Intracavitary Up to 28 days (2.4 x109 cells – D1,D8,D15) (100x106 cells – (3 out of 4 wks) recurrent resection eligible GBM Cohort 2 Intravenous Resection Intrac
Slide 19: CYNK-101 HER2+ Advanced Esophageal / Gastric Adenocarcinoma
CYNK-101 HER2+ Advanced Esophageal / Gastric Adenocarcinoma
Slide 20: CYNK-101 in HER2+ Gastric Cancer
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 21: 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 Trastuzumab against both gastric cancer cell lines was shown at
Slide 22: 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 23: CYNK101-HER2-001 (HER2+ Gastric/GEJ Cancer)
CYNK101-HER2-001 (HER2+ GASTRIC/GEJ CANCER) Planned Phase 1 - Dose Escalation / Phase 2a Expansion/Proof of Concept Induction NK Cell Re- Induction Maintenance (5 cycles every Lymphodepletion (every 21 days) 21 days) Pembrolizumab (3 days) 200 mg (Day 1 of cycle) Pembrolizumab Pembrolizumab CR, PR,
Slide 24: CyCART-19 B-Cell Malignancies
CyCART-19 B-Cell Malignancies
Slide 25: 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 26: CYCART19-BCM-001 (Relapsed/Refractory B-Cell Malignancies)
CYCART19-BCM-001 (RELAPSED/REFRACTORY B-CELL MALIGNANCIES) Planned Phase 1 Dose Escalation / Phase 2 Registrational Phase 1 Phase 2: Safety and Dose Finding Potential Registrational Cohorts Cohort A: High Grade NHL CD-19 targeted therapy naïve (n= ~66) • DLBCL Dose Level 1: • High Grade B-Cell lymph
Slide 27: Imugene Collaboration
IMUGENE COLLABORATION Exclusive Strategic Partnership Combining CD19 Oncolytic Virus + CyCART-19 in Solid Tumors • Based on Dr. Yuman Fong’s oncolytic virus technology from City of Hope • OnCARlytics infects tumor cells → replicates and produces CF33-CD19 on the tumor cell surface OnCARlytics – Enab
Slide 28: Degenerative and Autoimmune Diseases
Degenerative and Autoimmune Diseases
Slide 29: 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 30: 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 31: 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 APPL-TFKO Late passage ▪ Identified two of four CRISP
Slide 32: Next Steps: Clinical Program Milestones
Next Steps: • Clinical Program Milestones
Slide 33: 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 34: Appendix
Appendix Additional Detail
Slide 35: Celularity IMPACT Platform: Broad IP Protection
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 36: Celularity IMPACT Platform: Placenta as 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 37: Celularity Placental CAR-T: Solving 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 ➢ All CAR T-cell therapies on ✓ No apheresis capacity constraints ▪ Complex, high COGS the market and most (~7
Slide 38: 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 ➢ Requires selection, manufacturing process to ✓ No apheresis capacity
Slide 39: 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 + F re q u e n c y NCG mice 1 0 0 Day 0 Day 45 8 0 (% ) C D 3+ 6 0 + 100 W eek 2 W eek 4 P
Slide 40: 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 ➢ Apheresis of peripheral blood ▪ De-differentiated adult ✓ No apheresis capacity constraints from healthy donor
Slide 41: APPL Crohn's Disease (CD) 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 42: Manufacturing Process
MANUFACTURING PROCESS Celularity Purpose-built Commercial Scale150,000 sq. ft. Manufacturing Facility Network of Longstanding Cell Isolation and Partnerships Selection Product Cryopreservation • Birthing Centers • Proprietary perfusion • In-process cooling/cryopreservation of drug • Obstetricians me
Slide 43: 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 44: 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 PNK-007 demonstrates robust killing (cytolytic) PNK-007 exerted up to 60% specific lysis against PNK-007 activation releases high concentration against CML, A