T-Cell Immunotherapy Market,

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1 Product Sheet More information at T-Cell Immunotherapy Market, Published: 2015-OCT-13 Pages: 419 Format: PDF Price: 2199 USD (Single User License) Description WHO has stated that the number of new cancer cases is expected to rise by 70% in the coming two decades. In the US alone, over one million people are diagnosed with cancer each year. Although cancer therapeutics continues to be one of the most active areas in terms of drug development, there is still a significant unmet need. Specifically, immuno-oncology has been gradually nurtured by researchers over the last several years. Post an evident shift towards the development of therapeutics targeting cancer cells by harnessing immune system components, immunotherapeutics have gained a strong foothold in the pharmaceutical industry. After the early success of Immune Checkpoint Inhibitors, T-cell immunotherapy has emerged as another innovative and potent arm of this market. Characterised by key features such as target specificity, adaptability and memory, T-cells have been effectively used as tools to mediate an immune response. T-cell immunotherapy is structured into segments comprising of CAR-T, TCR and TIL therapies. Academicians across the globe have significantly contributed by convening the initial research of the candidates; this has served as the framework for several start-ups / established industry players to drive the research forward. The overall market is expected to make a huge leap over the coming decade. It is also important to highlight that various technology providers, aiming to develop T-cell immunotherapy products with improved efficacy and safety, have developed innovative platforms for engineering of the T-cells. Innovative research, lucrative rounds of VC funding, discovery of several novel targets, growing pipelines directed towards different therapeutic areas and encouraging clinical trial results have emerged as some of the other drivers of this market. <strong>synopsis</strong> The T-Cell Immunotherapy Market, report provides an extensive study of the field of T-cell immunotherapy. With no marketed products, this emerging field is still in its infancy; the report provides a comprehensive view of the market segmented into the following three sub-groups: CAR-T therapies, TCR therapies and TIL therapies. The market is characterised by a robust and opportunistic pipeline of products targeting both haematological cancers and solid tumours. As mentioned earlier, several research institutes have played a critical role in progression of the technology to its current stage. Post the initial research, industry players have collaborated with non-industry participants to fund the clinical and commercial development of these products. Some late stage products that have emerged out of such collaborations include CTL-019 (Novartis/University of Pennsylvania / The Children s Hospital of Philadelphia), JCAR017 (Juno Therapeutics / Seattle Children s Hospital), KTE-C19 and NY-ESO-1 TCR (Kite Pharma / National Cancer Institute), CD19-CAR (Takara Bio / Jichi Medical University) and LN-144 (Lion Biotechnologies / National Cancer Institute). Encouraging clinical results have accelerated the progress of these therapies as they have emerged as one of the most effective ways of leveraging the potential of immune system. The market is also seeing a growing interest from several technology providers, especially those with capabilities in genome editing and viral and non-viral gene transfer. In addition, several T-cell immunotherapy companies and technology providers are also developing innovative safety switches to manage known side effects such as cytokine release syndrome and B-cell aplasia. One of the key objectives of the study is to review and quantify the opportunities laid by the innovative programs of both small and big pharma firms. The unexploited and promising nature of this market supports the hopes pinned on multiple start-ups by several strategic investors and venture capital firms. Amongst other elements, the report elaborates on the

2 following key areas: The current state of the market with respect to key players, development stage of pipeline products (both clinical / preclinical) and targeted indications Partnerships which have taken place in the recent past covering research and development collaborations, manufacturing agreements, license agreements specific to technology platforms, product co-development and cocommercialisation Innovative technological platforms, such as safety switches, which have contributed significantly in overcoming the existing gaps in therapy regimen Various investments and grants received by the companies focused in this area Therapeutic areas forming the current focus of developers, the gradual drift and opportunity offered by nononcological therapeutic areas Competitive landscape, challenges and expected trend of market evolution within the three sub-segments Development and sales potential based on target consumer segments, likely adoption rate and expected pricing The research, analysis and insights presented in this report include potential sales of several CAR-T therapies, TCR therapies and TIL therapies; this analysis is backed by a deep understanding of key drivers behind the growth. With most products still in the early stage of development, we have provided three market forecast scenarios to add robustness to our model. The conservative, base and optimistic scenarios represent three different tracks of industry evolution. All actual figures have been sourced and analysed from publicly available information. The figures mentioned in this report are in USD, unless otherwise specified. <strong>example Highlights</strong> During the course of our research, we identified over 180 T-cell therapies across various phases of development; CAR-T therapies (56%) are the most common, followed by TCR (25%) and TIL (19%) therapies. Research institutions, through investment of time, expertise and effort, have played a critical role in the design and development of novel CAR-Ts, TCRs and TILs. In fact, non-industrial players are involved in the development of around 65% of all the molecules in the pipeline; of these, 23% molecules are being developed jointly with industry participants. Kite Pharma is the current flag-bearer; Novartis, Juno Therapeutics, CBMG, Celyad, Lion Biotechnologies, Immunocore and Adaptimmune are some other well-known players. In addition, several start-ups have also emerged; these include (in alphabetical order) Altor BioScience, Applied Immune Technologies, Autolus, Carsgen, Celdara Medical, Eureka Therapeutics, Formula Pharmaceuticals, Mustang Therapeutics, Theravectys, TILT Biotherapeutics and TNK Therapeutics. Engineered CAR-Ts with switch technologies are amongst the latest addition to the next-generation of T-cell immunotherapy pipeline. Players are developing CAR-Ts, TCRs and TILs which are more advanced and shall cater to a diverse array of patient needs. The coming few years are likely to witness a wave of innovative and successful T-cell therapies. Funding from VC firms and strategic investors has been a key enabler to the market s growth. Over the last six years, we identified investments close to USD 3 billion across several start-ups and small firms; this trend is unlikely to subside in the near future. Overall, we believe the T-cell therapy market will be worth USD 30 billion by 2030, expanding at an annualised growth rate of over 100%; as TCR and TIL therapies gain ground, CAR-T therapies are likely to garner the most attention in near future. <strong>research Methodology</strong> Most of the data presented in this report has been gathered via secondary and primary research. For all our projects, we conduct interviews with experts in the area (academia, industry, medical practice and other associations) to solicit their opinions on emerging trends in the market. This is primarily useful for us to draw out our own opinion on how the market will evolve across different regions and technology segments. Where possible, the available data has been checked for accuracy from multiple sources of information.

3 The secondary sources of information include Annual reports Investor presentations SEC filings Industry databases News releases from company websites Government policy documents Industry analysts views While the focus has been on forecasting the market over the coming ten years, the report also provides our independent view on various technological and non-commercial trends emerging in the industry. This opinion is solely based on our knowledge, research and understanding of the relevant market gathered from various secondary and primary sources of information. <strong>chapter Outlines</strong> Chapter 2 presents an executive summary of the report. It offers a high level view on where the T-cell immunotherapy market is headed in the mid-long term. Chapter 3 provides a general introduction to T-cell immunotherapy. In this section, we have briefly discussed the conventional therapies being used for oncology treatment, the advent of cancer immunotherapy and the reasons supporting the gradual emergence of T-cell therapies. Chapter 4 provides a comprehensive landscape of the T-cell immunotherapy market. It includes information on therapies which are currently in different stages of development (both clinical and preclinical/discovery). The detailed analysis of this pipeline includes information on most commonly targeted indications, current phases of development, the targets and the type of developers for each of the therapies. Chapter 5 focuses on CAR-T based therapies and highlights the prevailing trends pertaining to the on-going research in this field. It discusses the targets under investigation, current challenges, toxicity issues and several other relevant parameters. To credit the work of the eminent researchers across the globe, we have provided a mapping of prominent key opinion leaders in the CAR-T space. The chapter includes detailed profiles of the drugs under development. These profiles cover information such as history of development, clinical trial timeline, clinical trial results, manufacturing, estimated cost of treatment and treatment regimen. Chapter 6 elaborates on TCR based therapies and highlights the current scenario pertaining to the on-going research in this field. As with the CAR-T therapies, to credit the work of eminent researchers across the globe, we have provided a mapping of prominent key opinion leaders in the TCR space. The chapter also includes detailed drug profiles of several drugs under development. Chapter 7 elaborates on the TIL based therapies. As in the earlier two chapters, we have mapped the prominent key opinion leaders in the TIL space. The chapter also includes detailed profiles of the drugs under development. Chapter 8 elaborates on the monetary opportunity presented by these therapies. It provides a comprehensive market forecast analysis for molecules in advanced stages of development (phase I/II, phase II and phase II/III) taking into consideration the target patient population, competition, likely adoption rate and price points. Chapter 9 provides a competitive landscape of various T-cell therapies being developed for the treatment of commonly targeted indications such as haematological cancers and solid tumours. These indications have been the prime focus of companies developing T-cell immunotherapies. The chapter also highlights the epidemiological facts and currently available treatments for each indication.

4 Chapter 10 talks about emerging technological platforms which mediate/assist the growth of the T-cell market. It includes a detailed discussion on such novel and innovative technologies, along with brief information about the technology providers. Chapter 11 presents details on various investments and grants received by companies focused in the area of T-cell immunotherapy. The analysis highlights the growing interest from the VC community and other strategic investors. Chapter 12 provides detailed company profiles of the leading players in the market. Each company profile includes information such as financial performance, geographical presence, T-cell immunotherapy pipeline, recent collaborations and developments. Additionally, we have also provided details about funding instances relevant to each player. Chapter 13 is a collection of transcripts of interviews conducted during the course of this study. Chapter 14 summarises the overall report. In this chapter, we have provided a list of key takeaways and have expressed our independent opinion based on the research and analysis described in previous chapters. Chapters 15 is an appendix, which provides tabulated data and the list of companies covered in the report. Table of Contents 1 PREFACE 1.1. Scope of the Report 1.2. Research Methodology 1.3. Chapter Outlines 2 EXECUTIVE SUMMARY 3 INTRODUCTION 3.1. Chapter Overview 3.2. The Four Pillars of Cancer Therapy 3.3. Immunotherapy: Gaining a Strong Foothold 3.4. Immuno-Oncology (Cancer Immunotherapy) 3.5. Classification of Cancer Immunotherapies By Mechanism of Action Active Immunotherapy Passive Immunotherapy By Type of Target By Approach Activation and Suppression Immunotherapy By Product Class Monoclonal Antibodies Bispecific Antibodies Cytokines Cell Based Therapies 3.6. T-Cell Therapies: Historical Evolution 3.7. T-Cell Therapies: Key Considerations 3.8. Strategies for Redirection of T-Cells 3.9. Methodologies for Transduction / Transfection of T-Cells Retroviral Vectors Lentiviral Vectors Sleeping Beauty Transposon Curative Potential of T-Cell Therapies Roadblocks for T-Cell Therapies 4 MARKET LANDSCAPE 4.1. Introduction 4.2. Evolving T-Cell Therapy Space 4.3. T-Cell Therapy: A Promising and Robust Pipeline 4.4. CAR-T Cells Spearhead the T-Cell Market 4.5. Academia Research Leads On-going Activity in the T-Cell Space 4.6. T-Cell Market Poised to Expand Further: Several Molecules in Early Phases of Development 4.7. LimitedSuccess So Far: Restricted to Haematological Cancers/ Solid Tumours 4.8. Active Industry Players in the T-Cell Therapy Market 4.9. CD19 Continues to be the Most Popular Target for CAR-T Therapies Targets Well Distributed for TCR Therapies Other T-Cell Immunotherapies under Development

5 5 CHIMERIC ANTIGEN RECEPTOR-T CELL THERAPY 5.1. Introduction 5.2. History of Development 5.3. Current Research Landscape 5.4. Anatomical Layout of Chimeric Antigen Receptor 5.5. Generations of Chimeric Antigen Receptors 5.6. Development of CAR-T Cells 5.7. Lymphodepleting Therapy 5.8. Universal CAR-Ts 5.9. Route of Administration Toxicity Issues Cytokine Release Syndrome (CRS) On-Target Off-Tumour Toxicity Encephalopathy and B-Cell Aplasia Management of Toxicity Issues Target Selection Cell Persistence Receptor Expression CD19: An Attractive Target Other Targets Challenges Associated with CAR-T Therapy Competitive Risks Clinical Risks Regulatory Challenges Commercial Risks CTL019 (Novartis) Introduction History of Development Development Status Key Clinical Trial Results Dosage Regimen, Treatment Cost and Manufacturing JCAR (Juno Therapeutics) Introduction CAR-T Design Development Status Key Clinical Trial Results JCAR JCAR JCAR Dosage Regimen and Manufacturing KTE-C19 (Kite Pharma) Introduction Development Status Key Clinical Trial Results Dosage Regimen and Manufacturing Next Generation eact CAR Candidates CD19 CAR (Takara Bio) Introduction Development Status Key Clinical Trial Results Dosage Regimen and Manufacturing EFGRvIII CAR (Kite Pharma) Introduction Development Status Dosage Regimen and Manufacturing 6 T-CELL RECEPTOR (TCR) THERAPIES 6.1. Introduction 6.2. Structure of T-Cell Receptor 6.3. Difference between CAR and TCR 6.4. History of Development 6.5. Current Research Landscape 6.6. Mechanism of Action 6.7. Safety Issues Insertional Mutagenesis On-Target Off-Tumour Toxicity

6 Cross Reactivity Issues TCR Mismatch Pairing 6.8. Prerequisites of Antigen-Specific T-Cell Receptors 6.9. Identifying Strategies that Enhance the Anti-Tumour Efficacy Affinity Enhanced TCRs Soluble TCR Based Biologics NY-ESO-1 TCR (Kite Pharma) Introduction Development Status Dosage Regimen MAGE A3/A6 TCR (Kite Pharma) Introduction Development Status Dosage Regimen JTCR016 (Juno Therapeutics) Introduction Development Status Key Clinical Trial Results Dosage Regimen IMCgp100 (Immunocore) Introduction Development Status Key Clinical Trial Results Dosage Regimen 7. TUMOUR INFILTRATING LYMPHOCYTES (TIL) BASED THERAPIES 7.1. Introduction 7.2. Historical Background 7.3. Current Research Landscape 7.4. Strategies under Research to Enhance Efficacy 7.5. TIL: A Prognostic Tool for Various Indications 7.6. LN-144 (Formerly Contego) (Lion Biotechnologies) Introduction Development Status Key Clinical Trial Results Dosage Regimen and Manufacturing Patent Portfolio 8. MARKET OPPORTUNITY 8.1. Chapter Overview 8.2. Scope and Limitations 8.3. Forecast Methodology 8.4. Overall T-Cell Therapy Market, Overall Chimeric Antigen Receptor T-Cell (CAR-T) Market CTL019 (Novartis) Target Population Sales Forecast KTE-C19 (Kite Pharma) Target Population Sales Forecast CD19 CAR-T (Takara Bio) Target Population Sales Forecast EGFR CAR-T (Cellular Biomedicine Group) Target Population Sales Forecast CD30 CAR-T (Cellular Biomedicine Group) Target Population Sales Forecast Anti-CD19 CAR-T VectorTransducedT-Cells (Cellular Biomedicine Group)

7 Target Population Sales Forecast Anti-CD20 CAR T VectorTransduced T-Cells (Cellular Biomedicine Group) Target Population Sales Forecast CD19 EBV CAR-T (Autolus) Target Population Sales Forecast EGFRvIII (Kite Pharma) Target Population Sales Forecast JCAR017 (Juno Therapeutics) Target Population Sales Forecast JCAR0XX (Juno Therapeutics) Target Population Sales Forecast 8.6. Overall TCR Market IMCgp100 (Immunocore) Target Population Sales Forecast NY-ESO-1 TCR (Kite Pharma) Target Population Sales Forecast HPV-16 E6-TCR (Kite Pharma) Target Population Sales Forecast NY-ESO-1 TCR (Adaptimmune) Target Population Sales Forecast ALT-801 (Altor BioScience) Target Population Sales Forecast WT-1 TCR (Cell Therapy Catapult) Target Population Sales Forecast JTCR016 (Juno Therapeutics) Target Population Sales Forecast MAGE A3/A6 (Kite Pharma) Target Population Sales Forecast MAGE A3 (Kite Pharma) Target Population Sales Forecast 8.7. Overall TIL Therapy Market LN-144 (Lion Biotechnologies) Target Population Sales Forecast TIL Therapy (Nantes University) Target Population Sales Forecast TIL Therapy (Netherlands Cancer Institute) Target Population

8 Sales Forecast 9. KEY THERAPEUTIC AREAS FOR T-CELL THERAPIES 9.1. Chapter Overview 9.2. Introduction 9.3. Haematological Malignancies Leukaemia and Lymphoma Introduction and Epidemiology: Leukaemia Acute Myeloid Leukaemia (AML) Chronic Myeloid Leukaemia (CML) Acute Lymphocytic Leukaemia (ALL) Chronic Lymphocytic Leukaemia (CLL) Introduction and Epidemiology: Lymphoma Current Treatment Landscape Targeted Therapies T-Cell Immunotherapy and Research Landscape CAR-Ts and Leukaemia/Lymphoma TCRs and Leukaemia/Lymphoma Multiple Myeloma Introduction and Epidemiology Current Treatment Landscape T-Cell Immunotherapy and Research Landscape 9.4. Solid Tumours Metastatic Melanoma Introduction and Epidemiology Current Treatment Landscape T-Cell Immunotherapy and Research Landscape TILs and Metastatic Melanoma TIL Research at MD Cancer Research Centre, USA TIL Research at Sheba Medical Centre, Israel TCRs and Metastatic Melanoma CAR-Ts and Metastatic Melanoma Bladder Cancer Introduction and Epidemiology Current Treatment Landscape T-Cell Immunotherapy and Research Landscape Kidney Cancer Introduction and Epidemiology Current Treatment Landscape T-Cell Immunotherapy and Research Landscape Ovarian Cancer Introduction and Epidemiology Current Treatment Landscape T-Cell Immunotherapy and Research Landscape CAR-Ts and Ovarian Cancer TCRs and Ovarian Cancer Breast Cancer Introduction and Epidemiology Current Treatment Landscape T-Cell Immunotherapy and Research Landscape CAR-Ts and Breast Cancer TCRs and Breast Cancer TILs and Breast Cancer Glioblastoma Introduction and Epidemiology Current Treatment Landscape T-Cell Immunotherapy and Research Landscape 10 EMERGING TECHNOLOGIES Chapter Overview Introduction Genome Editing Driving the T-Cell Therapies Applications of Genome Editing

9 Genome Editing Technologies CRISPR/Cas9 System Structural Features Modularity of the System Mechanism of Action Targeting Efficiency and Challenges Next-GEN CRISPR Technology Technology Providers Editas Medicine Intellia Therapeutics CRISPR Therapeutics TALENs Structural Features Mechanism of Action Advantages and Challenges Technology Providers Cellectis Editas Medicine Endonuclease and megatal Technology Providers bluebird bio Zinc Finger Nuclease Benefits Technology Providers Sangamo Biosciences Designing T-Cell Therapies with ImprovedCharacteristics Technology for Targeting Multiple Cancers Antibody Coupled T-Cell Receptor, UnumTherapeutics NK Cell Receptor, Celyad Long Term Tumour free Survival Durable Anti-Tumour Immunity Technology for Improved Safety Armoured CAR, Juno Therapeutics RheoSwitch Therapeutic System, Intrexon Inducible Caspase9 Safety Switch, BellicumPharmaceuticals CaspaCIDe Safety Switch Technology CIDeCAR Switch Technology GoCAR-T Switch Technology On-Off Switch, Multiple Companies Inhibitory CAR (icar), Juno Therapeutics On-Off Switch, Theravectys Allogeneic Technology CIK CAR-T Cells, Formula Pharmaceuticals Allogeneic Platform, Celyad Allogeneic Platform, Cellectis 11 VENTURE CAPITAL SUPPORT Chapter Overview Investors and Innovative Therapies: Trending Together Instances of Investments / Grants in T-Cell Therapies Growing Venture Capitalist Interest Funding Instances: Distribution by Type offunding Funding Amount: Distribution by Type of Funding 12 COMPANY PROFILES Chapter Overview Adaptimmune Company Overview Financial Information Product Portfolio Manufacturing Capabilities Collaborations Progenitor Cell Therapy Thermofisher / Life Technologies GlaxoSmithKline

10 Future Outlook Altor Bioscience Corporation Company Overview Financial Information Product Portfolio Patents Portfolio Collaborations Massachusetts General Hospital and Howard Hughes Medical Institute Future Outlook bluebird bio Company Overview Financial Information Product Portfolio Collaborations Celgene Corporation Pregenen Five Prime Therapeutics Kite Pharma Future Outlook Cellectis Company Overview Financial Information Product Portfolio Collaborations CELLforCURE OncoDesign Biotechnology Ohio University Weill Cornell Medical College Ordering Order Online - Order by Fax - using the form below Order by Post - print the order form below and send to: BioMarket Group Björnnäsvägen STOCKHOLM SWEDEN

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