CAR-T Cell Therapy Market, 2018-2024 - Landmark Approvals of CAR-T Cell Therapies by the U.S. FDA and the EMA

DUBLIN, Sept. 7, 2018 /PRNewswire/ --

The "Global CAR-T Cell Therapy Market - Market Size, Forecasts, Trials & Trends" report has been added to ResearchAndMarkets.com's offering.

CAR-T cell therapy has swept the biotech industry by storm in recent years, creating hope that it could welcome in a new age of cancer treatment. However, the remarkable success stories have come from targeting CD19, which is now considered an antigen that holds the key to a limited range of blood cancers. Presently, this hematological arena is a highly competitive therapy space that is being shared between among leading CAR-T companies.

Scientists, investors and developers invariably agree that the key to longer-term success in this space depends on solving two major problems: identifying antigens other than CD19 that can be targeted with CAR-T therapy with strong efficacy and going beyond liquid cancers into solid tumor indications. CAR-T cell products to deal with solid tumors will undoubtedly offer a larger market potential.

However, it is not an easy task to identify the antigens found on the cells of solid tumors. There are reasons why CD19 is the most common target. It is seen solely on B cells, whose destruction via CAR-T therapy offers a straightforward route for treating B-cell leukemias and lymphomas. At the same time, loss of the body's B cells is not particularly problematical, because their antibody-producing function can be reinstated by injecting intravenous immunoglobulin (IVIG) to patients.

Currently, the only two non-CD19-directed CAR-T therapies are those that target CD22 in B-cell malignancies and B-cell maturation antigen (BCMA) in multiple myeloma. CD22 is structurally analogous to CD19, while BCMA is an antigen expressed on plasma cells, whose functional loss can also be replaced with IVIG.

The problem with solid tumors is that there is little evidence of CAR-T being able to overcome the numerous difficulties that exist for these to be targeted efficiently. However, both academic and commercial groups are racing against time to identify the antigens on solid tumor cells and develop suitable CAR-T cells, because it represents large market potential.

There are several reasons which make solid tumors difficult to treat using CAR-T cell therapies. Globally, solid tumors outnumber the hematological tumors by 10 to one. In 2015, Novartis and PENN reported that their CART-meso failed to show any effect in patients with mesothelioma, ovarian cancer and pancreatic cancer. Moreover, there was very poor persistence of CAR-T cells in the patients.

The difficulty with solid tumors is that they are usually surrounded by a hostile, immuno-suppressive microenvironment. This environment presents many inhibitory factors that prevent CAR-T cells from reaching them. A typical CAR-T approach will not have success under these conditions. For this reason, Juno and Novartis are engaging in constructing CAR-T cells with novel designs that incorporate additional elements to boost activity within this setting. Currently, these products are in preclinical development.

Driving Forces for the CAR-T Therapy Market

With growing demand for CAR-T therapies, CAR-T companies are proliferating. Growing numbers of these companies are supported by:

• Increasing investment flowing into CAR-T cell research
• Landmark approvals of CAR-T cell therapies by the U.S. Food & Drug Administration (FDA) and the European Medicines Agency (EMA)
• Major acquisitions within the CAR-T industry
• Large IPOs within the CAR-T industry

As mentioned, 2017 was the first year that the U.S. FDA approved a CAR-T cell therapy, approving Kymriah in August 2017 and Yescarta in October 2017. Novartis produced Kymriah, a CAR-T therapy used to treat acute lymphoblastic leukemia. Kite Pharma produced Yescarta, a CAR-T therapy to treat adult patients with certain types of large B-cell lymphoma who did not respond to or who relapsed after other kinds of treatment. In August 2018, both Kymriah and Yescarta secured approval in Europe from the EMA.

The approval of these first two CAR-T cell therapies has opened the gates for many other types of cell and gene therapies to claim respect, both from regulators, as well as from the scientific and medical community at large. These historic events demonstrate that CAR-T cell therapy is a market that has emerged, no longer one that is evolving in the future.

CAR-T funding is also on the rise. At first the trend was subtle, but the tide swelled as CAR-T therapies like Kymriah and Yescarta reached the marketplace and created a CAR-T funding craze. CAR-T start-ups have been richly funded by investors eager to get into this trending area of regenerative medicine. Following IPOs by CAR-T players Kite Pharma, Bellicum, Juno Therapeutis and Cellectis totaling over $750 million, CAR-T developer Autolus announced a $150 million IPO.

This has bought the total value of recent CAR-T initial public offerings (IPO's) to nearly $1 billion.

CAR-T Industry Deal-Making

The CAR-T industry has also witnessed aggressive deal-making in recent years. Celgene snagged Juno Therapeutics for a shocking $9 billion in January 2018 and Gilead acquired Kite Pharma for an astounding $11.9 billion in August 2017. After $20 billion of market capitalization from the CAR-T companies in 2018, the CAR-T market has continued to gain momentum.

There have also been more than a dozen CAR-T deals between pharmaceutical companies and academic institutions, with the best known being the partnership between Novartis and the University of Pennsylvania (UPenn). When Kymriah because the first CAR-T cell therapy to be approved in the U.S. in August 2017, it resulted from a 5-year collaboration between UPenn and Novartis.

CAR-T financing rounds have also proliferated. In one major example, Celularity raised $250 million in February 2018 to support development of placental-derived products, including T-cells that will be immune advantaged because of their derivation from the placenta. Celularity is looking to burst a major bottleneck in the industry by deriving T-cells from a single (allogeneic) cell line, potentially positioning itself to slash the price point for CAR-T treatments. In other significant example, London-based CAR-T start-up Allogene Therapeutics entered into an asset contribution deal with Pfizer in April 2018, as well as announced a $300 million Series A round.

Key Topics Covered:

1. Report Overview
1.1 Statement of the Report
1.2 Executive Summary
1.3 Introduction

2. History of CAR-T Cell Therapy
2.1 Timeline of CAR-T Cell Therapy Development

3. CAR-T Manufacturing Processes
3.1 Manufacturing Autologous CAR-T Cells
3.2 T-CellSource
3.3 T-Cell Activation
3.4 Genetic Modification of T-Cells
3.5 Expansion of CAR-T Cells
3.6 Clinical CAR-T Cell Manufacturing Quality Checkpoints
3.8 Allogeneic CAR-T Cells

4. Structure of a CAR-T Cell
4.1 First Generation CAR-T Cells
4.2 Second Generation CAR-T Cells
4.3 Third Generation CAR-T Cells
4.4 Fourth Generation CAR-T Cells
4.5 Mechanism of Action
4.6 New CAR Models and Concepts
4.7 Basic Components of CAR

5. Number of CAR-T Companies, their Recent Activities
5.1 Geographical Distribution of CAR-T Cell Therapeutic Companies
5.2 Immunocellular Therapy Companies by Cell Type
5.3 Market Leaders in CAR-T Cell Therapy and their Recent Activities

6. Tumor-Associated Target Antigens
6.1 Antigens on Solid Tumors
6.2 CAR-T Targets in Hematologic Malignancies
6.3 CAR-T Cell Trials Targeting CD19
6.4 CAR-T Cell Therapy for Solid Tumors
6.4.1 CAR-T Cell Targets in Solid Malignancies

7. Target Diseases for CAR-T Cell Therapy
7.1 Acute Lymphoblastic Leukemia (ALL)
7.2 Chronic lymphocytic leukemia (CLL)
7.3 Non-Hodgkin lymphoma
7.4 Acute myeloid leukemia (AML)
7.5 Neuroblastoma
7.6 Multiple myeloma (MM)

8. Pricing and Payment Models for CAR-T Therapies
8.1 Controversies over CAR-T Pricing
8.2 Hospital Mark-up Costs for Kymriah and Yescarta
8.3 Cost Effectiveness of Tisagenlecleucel and Axicabtagene
8.4 Value-Based Price Benchmarks
8.5 Unit Prices Needed to Reach Cost-Effectiveness Thresholds
8.6 Alternate Payment Strategies

9. Medical Facilities Offering CAR-T Therapies
9.1 CAR-T Recommended in Europe
9.2 CAR-T Cell Therapy in Chinese Hospital
9.3 Canada Joins the CAR-T Club

10. CAR-T Therapy Patent Landscape
10.1 Number of CAR-T Cell Patents, 2013-2018
10.2 CAR-T Patent Types
10.3 A Brief Snapshot of CAR-T Patent Landscape
10.4 Major CAR-T Patent Applicants

11. Deals, Fundings, Partnerships and Collaborations
11.1 Funding for CAR-T
11.2 CAR-T Deals
11.3 Initial Public Offering (IPO)
11.4 Key CAR-T Technology Deals
11.4.10 Trends in Oncology Licensing, Joint Venture and Research Deals

12. The Landscape of CAR-T Cell Therapy Clinical Trials
12.1 The Surge in Number of CAR-T Clinical Trials
12.2 Percentage (%) of Total CAR-T Clinical Trials by Target
12.3 Research Focus on CAR-T Trials by Indication
12.4 Clinical Trials using CAR-T Cells by Country as of 2018
12.5 CAR-T Clinical Trials to Watch
12.6 CAR-T Projects with Commercial Licensees
12.7 Clinical CAR-T Constructs with Sole Involvement from Academicia
12.8 Anti-CD19 CAR-T Studies
12.9 CAR-T Studies in Multiple Myeloma and Acute Myeloid Leukemia
11.10 CAR-T Cell Therapy for Solid Tumors
12.11 Studies of CAR-T Projects Transfected using mRNA Electroporation
12.12 CAR-T Projects Incorporating Suicide Genes
12.13 Early Stage CAR-T Assets
12.14 Anti-CD22 CAR-T Projects
12.15 Cytokine Release Syndrome (CRS) with CART19 Therapy
12.16 CAR-T Therapy Pipeline Distribution by Indication
12.17 CAR-T Therapy Pipeline Distribution by Target Antigen
12.18 Distribution of CAR-T Clinical Trials in China

13. CAR-T Cell Products in the Market
13.1 Tisagenlecleucel (Kymriah)
13.1.1 Evidences Supporting Effectiveness of Tisagenlecleucel (Kymriah)
13.1.2 Medical Necessities for Tisagenlecleucel (Kymriah)
13.1.3 Overall Remission Rates in Patients Treated with Kymriah
13.1.4 Overall Event-Free Survival Rate with Tisagenlecleucel (Kymriah)
13.1.5 Key Adverse Events in ELIANA Trial
13.1.6 Key Adverse Events in JULIET Trial
13.1.7 Budget Impact for Tisagenlecleucel
13.1.8 Overall Adverse Events with Tisagenlecleucel
13.1.9 Per-Patient Potential Budget Impact of Tisagenlecleucel
13.2 Axicabtagene Ciloleucel (Yescarta)
13.2.1 Evidences to Support the Effectiveness of Axicabtagene Ciloleucel
13.2.2 Medical Necessities for Axicabtagene Ciloleucel (Yescarta)
13.2.3 Clinical Benefits of Axicabtagene Ciloleucel (Yescarta)
13.2.4 Objective Response Rates (ORR) for Axicabtagene Ciloleucel
13.2.5 CRR for Axicabtagene Ciloleucel
13.2.6 Adverse Events in ZUMA-Trial
13.2.7 Base-Case Results
13.2.8 Value-Based Benchmark Prices
13.2.9 Per-Patient Budget Impact of Axicabtagene Ciloleucel
13.2.10 Unit Cost for Healthcare Utilization in CAR-T Therapy
13.2.10.1 Costs Associated with Adverse Events

14. Insurance Coverage for CAR-T Therapy
14.2 Coverage Policies for Tisagenlecleucel - B-ALL Patients
14.2 Coverage for Stem Cell Transplantation (SCT) - B-Cell ALL Patients
14.3 Coverage for Axicabtagene Ciloleucel - B-Cell NHL Patients
14.4 Coverage for Stem Cell Plantation (SCT) in B-Cell NHL Patients
14.5 Insurance Coverage by Medicare and Medicaid

15. Commercial Threats For CAR-T Industry
15.1 Competition in a Narrow Field
15.2 Competition from other Technologies
15.3 Threat from TCRs
15.4 Threat of Litigation

16. Challenges to Overcome
16.1 Lack of Persistence
16.2 Inadequate Activation
16.3 Transfection Method
16.4 Humanized Binding Domains
16.5 Antigen Escape
16.6 Lineage Switching
16.7 Lack of Safety
16.8 Benefits of CAR-T Cell Therapy
16.9 CAR-T Cell Therapy: Only the Beginning of the Story
16.10 A New Standard

17. Market Analysis
17.1 Global Market for CAR-T Therapy by Geography
17.2 Global Market for CAR-T Therapy by Country
17.3 Global Market for CAR-T Cell Therapy by Targeted Antigens
17.4 Competitive Landscape

18. Company Profiles

• AbbVie Inc.
• Adaptimmune Therapeutics PLC
• Amgen, Inc.
• Atara Biotherapeutics, Inc.
• Aurora Biopharma, Inc.
• Autolus Therapeutics PLC
• Bellicum Pharmaceuticals, Inc.
• BioAtla LLC
• bluebird bio
• Carina Biotech
• CARsgen Therapeutics, Ltd.
• CARTherics
• Cellectis
• Celularity
• Celyad SA
• Creative Biolabs
• DiaCarta, Inc.
• Endocyte, Inc.
• F1 Oncology, Inc.
• Fate Therapeutics Inc.
• Humanigen, Inc.
• Immune Therapeutics, Inc.
• Intrexon, Corp.
• Juno Therapeutics, Inc.
• Kite Pharma, Inc.
• Lion TCR Pte Ltd.
• MaxCyte, Inc.
• Mesoblast, Ltd.
• Minerva Biotechnologies Corp.
• Mustang Bio, Inc.
• Novartis AG
• Oxford BioMedica PLC.
• PeproMene Bio Inc.
• Pfizer, Inc.
• Posedia Therapeutics Inc.
• Precision Biosciences, Inc.
• ProMab Biotechnologies Inc.
• Servier Oncology
• Sorrento Therapeutics, Inc.
• TC Biopharm Ltd.
• Tessa Therapeutics Pte Ltd.
• TILT Biotherapeutics Ltd.
• Tmunity Therapeutics Inc.
• TrakCel Ltd.
• Xyphos
• ZIOPHARM Oncology, Inc.

For more information about this report visit https://www.researchandmarkets.com/research/s9r786/cart_cell?w=5

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