Investor Presentation
March 2020
Transforming development & commercialization
- f cell and gene therapies (CGTs) through
point-of-care (POCare) solutions.
Investor Presentation March 2020 Forward Looking Statements This - - PowerPoint PPT Presentation
Transforming development & commercialization of cell and gene therapies (CGTs) through point-of-care (POCare) solutions. Investor Presentation March 2020 Forward Looking Statements This Presentation contains forward-looking statements
Transforming development & commercialization
point-of-care (POCare) solutions.
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This Presentation contains forward-looking statements which are made pursuant to the safe harbor provisions of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities and Exchange Act of 1934, as amended. These forward-looking statements involve substantial uncertainties and risks and are based upon our current expectations, estimates and projections and reflect our beliefs and assumptions based upon information available to us at the date of this release. We caution readers that forward-looking statements are predictions based on our current expectations about future events. These forward-looking statements are not guarantees of future performance and are subject to risks, uncertainties and assumptions that are difficult to
by the forward-looking statements as a result of a number of factors, including, but not limited to, our reliance
from the sale of Masthercell, our ability to achieve and maintain overall profitability, the sufficiency of working capital to realize our business plans, the development of our POCare strategy, our trans-differentiation technology as therapeutic treatment for diabetes which could, if successful, be a cure for Type 1 Diabetes, the technology behind our in-licensed ATMPs not functioning as expected, our ability to retain key employees, our competitors developing better or cheaper alternatives to our products and the risks and uncertainties discussed under the heading "RISK FACTORS" in Item 1A of our Annual Report on Form 10-K for the fiscal year ended November 30, 2018, and in our other filings with the Securities and Exchange Commission. We undertake no
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Cell & gene therapy industry estimated to grow from $26.7B in 2018 to $150B in 2025 Orgenesis rapidly built and sold its contract development manufacturing organization (CDMO) business for $315M in February 2020—generating approximately $127 million net proceeds and significant value for shareholders Therapeutic pipeline built around academic and hospital collaborations Rolling out next generation point-of-care (POCare) platform
Cell processing and treatment within the patient care setting Highly scalable & cost-effective model Growing global network of leading healthcare facilities
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Source: Roland Berger FocusCell & Gene Medicine, September 2017
Early stage development candidates represent near-term opportunity in anticipated rapidly growing cell therapy market
21 23 26 30 36 44 56 75 105 150 20 40 60 80 100 120 140 160
2016 2017 2018 2019 2020 2021 2022 2023 2024 2025
Global Cell & Gene Medicine Market
Global market volume [USD bn] Global annual growth rate (CAGR, %)
~$150 billion
Market volume anticipated by 2025
+15% +33%
+33%
Annual growth expected between 2020-2025
~$26 billion
Current value of global Cell & Gene medicine market
113 170 30 106 144 9 90 225 38 5 11 5 50 100 150 200 250 Phase 1 Phase 2 Phase 3
Gene Therapy Gene-modified Cell Therapy Cell Therapy Tissue Engineering
Global Cell & Gene Medicine Clinical Trials
946
Clinical trials underway that utilize cell & gene technologies
53%
Clinical trials underway are in oncology
(estimated) (2017)
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$315M, generating approximately $127 million net proceeds to Orgenesis
approximately $30 million at the end of 2019
shareholders and focus efforts around next generation POCare solutions
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Iyer RK, Bowles PA, Kim H and Dulgar-Tulloch A (2018) Industrializing Autologous Adoptive Immunotherapies: Manufacturing Advances and Challenges.
doi: 10.3389/fmed.2018.00150
the research lab to the market and into patients at scale
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IN-LICENSES OUT-LICENSES
model
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Source: Roots Research Report: Cell Therapy Manufacturing Market, 2017-2027 European Medicines Agency: Advanced therapy medicinal products, 2nd international awareness session
Somatic Cell Therapy Medicinal Products Genetically Modified Therapy Medicinal Products Tissue Engineered Products Combined Advanced Therapy Medicinal Products ATMPs TEPs STMPs GTMPs Combined ATMPs
+ medical device component
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Sep 2017 Next Generation Scaffold/microca rriers
Biodegradable and Injectable Cell PGS microcarriers
June 2018 Single use Sensors for Bioreactors Nov 2018 Next Generation Scaffold/microcar riers
Vascularized Immune Tolerating Scaffold and Dissolvable Microcarriers
Next generation 3D bio-printing
Generation of islets- like spheroids
Dec 2018
Next Generation Scaffold/microca rriers
Advanced gene delivery platforms using Baculovirus vector
Feb 2019 Aug 2018 Feb 2012 Insulin Dependent Diabetes
Liver-derived Autologous Insulin Producing Cells (AIPs)
May 2018 Liver-Derived Metabolic Disorders
Utilizing liver derived mesenchymal- like cells to treat and manage liver diseases
May 2018 Cell-based Cancer Immunother apy
NK-NHP - NK cells homing protein targeting solid tumours
Bioxomes for Intracellular Delivery
Bioxosome and Redoxome for Liver Disorders and Atopic Dermatitis
Nov 2018 Hematological Disorders
Human Postnatal Hemogenic Endothelial Cells (Hu-PHECs) for Replacing Bone Marrow Transplants
Tumor cell-based vaccine
DUVAC cancer vaccine for Pancreatic, Hepatic and Cholangiocarcinoma cancers
Jan 2019 Apr 2019 Cell-based Cancer Immunotherapy
CAR-T-VAC69 - Ovarian cancer and Multiple Myeloma
Metabolically
cells
Metabolically
for Colorectal cancer and Melanoma
Nov 2018
Kecelletics Biotech..
Cell-based Cancer Immunotherapy
CAR-T-CD19 - for B-lineage acute lymphoblastic leukemia (B-ALL)
Nov 2019 Sep 2019 Automated QC Technology Closed loop Systems for cell processing Feb 2020
Autologous CD-19.20 CAR-T
CD 19 CD 20
CD19 Antigen CD20 Antigen Anti- CD20 Anti- CD19 Modified from: Fry TJ et al. , Mol Ther Oncolytics, 2018
neoplasm of B-lymphocyte precursors that express the pan B-cell marker CD19 in almost all cases
response upon engagement of CD19 or CD20 on target cells in B-ALL patients
reduce the risk of relapse through antigen-loss of leukemic cells in the long-term
at Daopei Hospital
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Daopei Hospital
dosage
Cytokine release syndromes (CRS)
system and direct it against cancer cells.
macrophages pulsed with relevant modified cancer cells antigens.
source of antigens. Targeting the entire repertoire of tumor cell antigens leaves little or no possibility for cancer cells to evade the immune system.
antigens for stimulating a robust and long-lasting humoral B-cell and cellular T-cell immune response in vivo.
clinical grade manufacturing, reducing cost of goods while ensuring high quality and availability for a variety of tumor types.
Universidad in Buenos Aires Argentina demonstrated significant improvement in quality and duration of life in lung cancer and ovarian cancer patients post vaccination. .
Figdor CG et al, Nature Materials, 2018
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Semi-Personalized Flexibility: effectiveness could bedemonstrated in prophylactic as well as therapeutic regimens Capability to produce a procedure-based vaccine as a product Synergy between developmental components Integrating basic components:simple,fast, affordable Potential on-top-of- therapy approach
multiple tumor Ag; based off of tumor cell lines which are readily available.
most comprehensive source of antigens.
antigens leaves little or no possibility for cancer cells to evade the immune system.
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recognize tumor antigens.
production and cytolytic activity, leading to the failure of cancer elimination.
faster, safer and metabolically optimal culturing and expansion process.
MOTC.
Colorectal Cancer and Metastatic Melanoma.
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Current T cell biomanufacturing process presents several weaknesses:
poor product quality.
integrity and reproducibility of T cells for potential clinical use. Orgenesis Solution:
closed system to produce high-quality and large-scale human T cells.
bioproduction, and outstanding scalability due to the precise process control of pH, temperature, dissolved
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Description
Liver tissue
is being increased
are ready for bio-banking Mesenchymal liver-cells-like
infecting the cells with gene/protein PDX1 & NEUROD1 Progenitor beta cells-like
the protein MAFA is added Pre-mature beta cells-like
within the transdifferentiation process 1 2 3 4
Endocrine Differentiation
MAFA
Processing Amplification (P4) Mincing Overnight incubation in collagenase
1-2 billion of liver-derived Insulin producing cells
Expansion (3-4 weeks) Hepatic De-differentiation
PDX1 & NEUROD1
1 2 3 4
Mesenchymal liver-cells- like Progenitor beta cells-like Pre-mature beta cells-like Liver tissue Cryopreservation 40M cells minimum
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Biodegradable and Injectable cell PGS scaffold Secant Group B Single use sensors for Bioreactors MIRCOD C Bioxome and redoxome Excellbio Ltd
D
Automated point of care immune cells processing system A
manufacturing cGMP system for CAR T’s, TLR’s, TIL’s, DC and NK cells
process
use outside of Grade B/C hospital clean rooms
cell culturing of engineered poly glycerol sebacate (PGS)
higher viability and efficacy
the high surface to volume ratio and reduce the downstream steps resulting in reduced cost of goods, improved quality and safety for the Insulin Producing Cells and other adherent cells such as MSC’s
enter the cell through fusion and endocytosis with the cell membrane, delivering designed target cargo
platforms such as cell transfection, in-vitro toxicity assays, animal modeling, gene therapy
development and manufacturing projects
securely for further evaluation
monitors ensures that there is no contamination because it maintains a closed environment and no loss of power to sensors
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Biodegradable and Injectable cell PGS scaffold Secant Group Single use sensors for Bioreactors MIRCOD Automated point of care immune cells processing system
system for CAR T’s, TLR’s, TIL’s, DC and NK cells
Grade B/C hospital clean rooms
for cell culturing of engineered poly glycerol sebacate (PGS)
in higher viability and efficacy
the high surface to volume ratio and reduce the downstream steps resulting in reduced cost of goods, improved quality and safety for the Insulin Producing Cells and other adherent cells such as MSC’s
manufacturing projects
further evaluation
ensures that there is no contamination because it maintains a closed environment and no loss of power to sensors
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Manufacturing technology lacking behind A No industrial and distribution infrastructure C Disconnect between hospitals and industry D Challenges Orgenesis’ solution No defined commercial pathway B
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Bioreactor - Immune Cells (Suspension) Bioreactor - Adherent cells - AIP, MSCs, BM alternatives Viral Vector Production system development (Lenti, AAV, AV) Exosomes / Secretome Extraction NK-NHP
Solid tumor
CAR-T/NK
Ovarian cancer and Melanoma
CAR-T CD-19
B-ALL
MOTC
Melanoma, Sarcoma, Colorectal cancer
AIP
Diabetes Type 1
DUVAC
Pancreatic, Hepatic and Cholangio carcinoma cancers
Cord tissue/ liver cells (HU- PHECS)
Alternative To Bone Marrow Transplants (Leukemia)
HepaCell
Liver metabolic diseases
Bioxomes
Liver damage
Exosomes, MSCs secretomes
Atopic Dermatitis
Small scale viral manufacturing for personalizes TCR therapy
Enabling production in Open Class C facilities = Reducing capex and operating costs
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can occur. Time saving and maximum efficiency.
by incorporated PLC & SCADA system.
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Fully Automated
Cell Phenotyping and Enumeration
Single Use Microfluidic Cartridge
Unitized and Dried Reagents
Portable and easy to use
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How Setting up a network of leading healthcare facilities with interest in developing our autologous therapy products. Leverage IP, technical and processing expertise to allow a closed system processing approach for our development stage products.
Goal
Bring autologous therapies in a cost effective, high quality and scalable manner to patients.
Why
Hybrid therapies fall between a traditional drug and a procedure such as bone marrow transplant.
Why Orgenesis?
5 years and $40M + invested to date in industrial capability, the best people, network, services, know-how & IP. Focused on gaining and sharing expertise in an emerging scientific field.
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North America Europe Asia Middle-East
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Unique Know-How
processes
system
Innovation Technology
& AI based systems for biological production
processing cells
technologies
system providers
Systems Therapies Portfolio
immuno-oncology related technologies
therapies
therapies
Cell & Gene Products Infrastructure / Distribution Channels
systems in major hospitals
Europe, North America, Asia, South America, etc.
system network serve as distribution channels
Distribution 1 2 3 4 Grow internal asset pipeline Leverage unique know-how and expertise Provide modular cell production cGMP systems Enable commercialization and distribution
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Rationale
production solutions and automation, the estimated cost of production of a CAR T therapy could potentially be 84% lower than conventional manufacturing
significantly lowered with Orgenesis’ highly efficient processes and technology for POCare cell production: 1.Labor costs 2.Logistics 3.Quality control 4.Disposables 5.Cleaning costs
Costs of conventional manufacturing vs. automated POCare production
Automation Level
2018 2019 2024 Year Production costs for CAR T batch Current market prices Potential costs with Orgenesis technology & processes
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David Sidransky – Director
named and profiled by TIME magazine in 2001 as one of the top physicians and scientists in America, recognized for his work with early detection of cancer. Since 1994, Dr. Sidransky has been the Director of the Head and Neck Cancer Research Division at Johns Hopkins University School of Medicine's Department of Otolaryngology and Professor of Oncology, Cellular & Molecular Medicine, Urology, Genetics, and Pathology at the John Hopkins University School of Medicine. Dr. Sidransky is one of the most highly cited researchers in clinical and medical journals in the world in the field of oncology during the past decade, with over 460 peer reviewed publications. Dr. Sidransky is a founder of a number of biotechnology companies and holds numerous biotechnology
ImClone Systems, Inc., a global biopharmaceutical company committed to advancing oncology care. He is serving, or has served
company), among others and is currently on the board of Galmed and Rosetta Genomics Ltd. and chairs the board of Advaxis and Champions Oncology, Inc. Dr. Sidransky served as Director from 2005 until 2008 of the American Association for Cancer Research (AACR). He was the chairperson of AACR International Conferences during the years 2006 and 2007 on Molecular Diagnostics in Cancer Therapeutic Development: Maximizing Opportunities for Personalized Treatment. Dr. Sidransky is the recipient of a number
Ochsner Award Relating Smoking and Health by the American College of Chest Physicians, and the 2004 Richard and Hinda Rosenthal Award from the American Association of Cancer Research. We believe Mr. Sidransky is qualified to serve on our Board
the public markets. Mario Philips - Director
France that has developed a diagnostic platform technology for neurodegenerative diseases in combination with a therapy to cure neurodegenerative diseases such as ALS and Parkinson’s. Mr. Philips also acts as strategic partner for the private equity fund, Archimed, and is Chairman of the Board for its portfolio company, Clean Biologics. Prior to that, in 2017 Mr. Philips acted as Vice President and General Manager for Danaher Pall’s (“Pall”) biotech business, with full P&L responsibility for a $1.3 billion business
Disposable Lab (France) and Artelis (Belgium). Mr. Philips earned an Engineering degree in Biochemistry at CTL, Gent, and has a postgraduate degree in Marketing from Groep T Leuven.
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Ashish Nanda - Director Ashish Nanda was appointed a director on February 22, 2017. Since 1990, Mr. Nanda has been the Managing Director of Innovations Group, one of the largest outsourcing companies in the financial sector that employs close to 14,000 people working across various financial sectors. Prior to that, from 1991 to 1994, Mr. Nanda held the position of Asst. Manager Corporate Banking at Emirates Banking Group where he was involved in establishing relationship with business houses owned by UAE nationals and expatriates in order to set up banking limits and also where he managed portfolios of USD $26 billion. Mr. Nanda holds a Chartered Accountancy from the Institute of Chartered Accountants from India. We believe that Mr. Nanda is qualified to serve on our Board because of his business experience and strategic understanding of advancing the valuation
Yaron Adler - Director Yaron Adler was appointed as our director on April 17, 2012. In 1999 Mr. Adler co-founded IncrediMail Ltd. and served as its Chief Executive Officer until 2008 and President until 2009. In 1999, prior to founding IncrediMail,
served as a Product Manager from 1997 to 1999, and as a software engineer from 1994 to 1997, at Tecnomatix Technologies Ltd., a software company that develops and markets production engineering solutions to complex automated manufacturing lines that fill the gap between product design and production, and which was acquired by UGS Corp. in April 2005. In 1993, Mr. Adler held a software engineer position at Intel Israel Ltd. He has a B.A. in computer sciences and economics from TelAviv University. We believe Mr. Adler is qualified to serve on our Board of Directors because of his education, success with early-stage enterprises and his business acumen in the public markets.
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Guy Yachin - Director Guy Yachin has served as a director since April 2, 2012. Mr. Yachin has served as CEO of NasVax Ltd., a company focused on the development of improved immunotherapeutics and vaccines and as CEO MultiGene Vascular Systems Ltd., a cell therapy company focused on blood vessels disorders, leading the company through clinical studies in the U.S. and Israel, financial rounds, and a keystone strategic agreement with Teva Pharmaceuticals Industries Ltd. He was CEO and founder of Chiasma Inc., a biotechnology company focused on the oral delivery of macromolecule drugs, where he built the company's presence in Israel and the U.S., concluded numerous financial rounds, and guided the company's strategy and operation for over six years. Earlier he was CEO of Naiot Technological Center Ltd., and provided seed funding and guidance to more than a dozen biomedical startups such as Remon Medical Technologies Ltd., Enzymotec Ltd. and NanoPass Technologies Ltd. He holds a BSc. in Industrial Engineering and Management and an MBA from the Technion - Israel Institute of Technology. We believe Mr. Yachin is qualified to serve on our Board of Directors because of his education, experience within the life science industry and his business acumen in the public markets. Vered Caplan - President, Chief Executive Officer and Director Vered Caplan has been the CEO since August 14, 2014, prior to which she was Interim President and CEO since December 23,
dermatology purposes. From 2004 to 2007, Ms. Caplan was Chief Executive Officer of GammaCan International Inc., a company focused on the use of immunoglobulins for treatment of cancer. During the previous five years, Ms. Caplan has been a director of the following companies: Opticul Ltd., a company involved with optic based bacteria classification; Inmotion Ltd., a company involved with self-propelled disposable colonoscopies; Nehora Photonics Ltd., a company involved with noninvasive blood monitoring; Ocure Ltd., a company involved with wound management; Eve Medical Ltd., a company involved with hormone therapy for Menopause and PMS; and Biotech Investment Corp., a company involved with prostate cancer diagnostics. Ms. Caplan has a M.Sc. in biomedical engineering from TelAviv University specializing in signal processing; management for engineers from TelAviv University specializing in business development; and a B.Sc. in mechanical engineering from the Technion-Israel Institute of Technology specialized in software and cad systems.
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IN-LICENSES OUT-LICENSES
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*Includes approximately $127 million net proceeds from the sale of the CDMO business
Contact Us Vered Caplan, CEO E-mail: vered.c@orgenesis.com Investor Relations: David Waldman President & CEO Crescendo Communications Office: +1 212 671 1020 Mobile: +1 917 355 2239 Dwaldman@crescendo-ir.com www.crescendo-ir.com