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 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 predict. Our actual results, performance or achievements could differ materially from those expressed or implied by the forward-looking statements as a result of a number of factors, including, but not limited to, our reliance on, and our ability to grow, our point-of-care cell therapy platform, our ability to effectively use the net proceeds 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 obligation to revise or update any forward-looking statement for any reason. 2
Investment Highlights 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 Clean capital structure and solid balance sheet 3
Cell & Gene Therapy Market Early stage development candidates represent near-term opportunity in anticipated rapidly growing cell therapy market Global Cell & Gene Medicine Clinical Trials Global Cell & Gene Medicine Market (2017) (estimated) Gene Therapy Global market volume [USD bn ] 160 150 250 Global annual growth rate Gene-modified Cell Therapy 225 (CAGR, %) Cell Therapy 140 Tissue Engineering 200 120 170 105 +33% 144 100 150 75 113 80 106 +15% 56 90 100 60 44 36 40 30 26 23 38 21 50 30 20 11 9 5 5 0 0 2016 2017 2018 2019 2020 2021 2022 2023 2024 2025 Phase 1 Phase 2 Phase 3 ~$26 billion ~$150 billion +33% 946 53% Current value of Market volume Annual growth Clinical trials Clinical trials underway global Cell & Gene anticipated by 2025 expected between underway that utilize are in oncology medicine market 2020-2025 cell & gene technologies Source: Roland Berger FocusCell & Gene Medicine, September 2017 4
Built and Sold CDMO Subsidiary for $315 Million • Built and sold contract development manufacturing organization (CDMO) business for $315M, generating approximately $127 million net proceeds to Orgenesis • Sale reflects culmination of successful CDMO strategy initiated in 2015 • CDMO segment revenue increased from a run-rate of just $3 million in 2015 to a run-rate of approximately $30 million at the end of 2019 • Masthercell achieved a 59% 5- year CAGR of Masthercell under Orgenesis’ leadership • Orgenesis determined it was the right time to sell Masthercell to maximize value for shareholders and focus efforts around next generation POCare solutions • Net proceeds from this transaction intended to accelerate rollout of POCare platform 5
Cell & Gene Therapy - Unmet Need Costs associated with inefficiencies in workflow - preventing broader clinical accessibility Lack of global regulatory standardization Hospitals do not have the resources or business expertise to take innovative therapies from the research lab to the market and into patients at scale Iyer RK, Bowles PA, Kim H and Dulgar-Tulloch A (2018) Industrializing Autologous Adoptive Immunotherapies: Manufacturing Advances and Challenges. Front. Med. 5:150. doi: 10.3389/fmed.2018.00150 6
POCare Business Model Biotech company with unique development, processing, manufacturing and distribution model IN-LICENSES OUT-LICENSES • Orgenesis receives reduced fees + equity in therapeutics/technologies in exchange for: • Therapeutic product development assistance • POCare processing, manufacturing and production expertise • Therapeutic regulatory and clinical assistance • Therapeutic product commercialization and distribution • Initial out-licenses to regional partners providing development funding for therapies • Scalable business model design to maximize value for shareholders • Reduced risk and faster pathway to market versus traditional biotech drug development model 7
Advanced Therapy Medicinal Products (ATMPs) ATMPs Combined STMPs GTMPs TEPs ATMPs + medical device component Genetically Somatic Cell Combined Tissue Modified Therapy Advanced Engineered Therapy Medicinal Therapy Products Medicinal Products Medicinal Products Products Source: Roots Research Report: Cell Therapy Manufacturing Market, 2017-2027 European Medicines Agency: Advanced therapy medicinal products, 2 nd international awareness session 8
Licensed Therapeutics and Technology Milestones Validation of IP Strategy Feb 2012 May 2018 May 2018 Aug 2018 Nov 2018 Nov 2018 Jan 2019 Apr 2019 Nov 2019 Insulin Liver-Derived Cell-based Bioxomes for Hematological Metabolically Cell-based Cell-based Dependent Metabolic Tumor cell-based Cancer Intracellular Disorders optimized T Cancer Cancer Diabetes Disorders vaccine Immunother Delivery Human Postnatal cells Immunotherapy Liver-derived Immunotherapy DUVAC cancer vaccine Utilizing liver apy Bioxosome and Hemogenic CAR-T-CD19 - for Metabolically Autologous CAR-T-VAC69 - derived for Pancreatic, Hepatic NK-NHP - NK Redoxome for Liver Endothelial Cells optimized T cells B-lineage acute Insulin Ovarian cancer and mesenchymal- cells homing Disorders and Atopic (Hu-PHECs) for for Colorectal lymphoblastic Producing Cells and Multiple Cholangiocarcinoma like cells to treat protein Dermatitis Replacing Bone cancer and leukemia (B-ALL ) (AIPs) Myeloma and manage liver cancers targeting solid Marrow Transplants Melanoma diseases tumours Kecelletics Biotech.. Feb 2019 Feb 2020 Dec 2018 Sep 2019 Sep 2017 Nov 2018 June 2018 Next Generation Next generation Closed loop Automated Next Generation Next Generation Single use 3D bio-printing Scaffold/microca Systems for cell Scaffold/microca QC Scaffold/microcar Sensors for Generation of islets- processing rriers rriers Technology riers Bioreactors like spheroids Advanced gene Biodegradable and Vascularized Injectable Cell PGS delivery platforms Immune Tolerating using Baculovirus microcarriers Scaffold and vector Dissolvable Microcarriers 9
B Cell Acute Lymphoblastic Leukaemia Autologous CD-19.20 CAR-T • B-lineage acute lymphoblastic leukemia (B-ALL) is an aggressive neoplasm of B-lymphocyte precursors that express the pan B-cell marker CD19 in almost all cases • CD19.20 bi-specific CAR-T cells therapy for treatment B-ALL patients CD19 Antigen • The CAR construct targets unique sequence of CD-19 and CD-20 CD20 Antigen Anti- • The anti-CD20-CD19 bispecific CAR induced a full T cell CD19 response upon engagement of CD19 or CD20 on target cells in B-ALL CD CD patients 20 19 • The anti-CD20-CD19 bispecific CAR T cells may also Anti- CD20 reduce the risk of relapse through antigen-loss of leukemic cells in the long-term Modified from: Fry TJ et al. , Mol Ther Oncolytics, 2018 • A total of 200 patients were enrolled in clinical trials at Daopei Hospital 10
CAR-T Clinical Data • 200 cases of CD19 CAR-T treatment were conducted at Daopei Hospital • All cases were B-ALL patients with various complications • Most of the patients were under 20 years of age • Cell fusion: 3x10 5 cells/kg of patient weight: very low dosage • High efficacy: 97% completed remission achieved • Good safety profile: 92% patients had grade 2 or less Cytokine release syndromes (CRS) 11
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