Frontier Pharma: Transplantation Therapeutics-A Highly Innovative Pipeline with a Range of Adaptive and Innate Immune-Targeting Programs Focusing on Graft-Versus-Host Disease and Kidney Transplantation

"The Report Frontier Pharma: Transplantation Therapeutics-A Highly Innovative Pipeline with a Range of Adaptive and Innate Immune-Targeting Programs Focusing on Graft-Versus-Host Disease and Kidney Transplantation provides information on pricing, market analysis, shares, forecast, and company profiles for key industry participants. - MarketResearchReports.biz"

In 2015 a total of 127,000 transplantations were performed worldwide, an increase of 5.8% from 2014, with 33,000 of these occurring in the EU and 32,000 in the US. Approximately 60% of these were kidney transplants, with liver, heart, lung, pancreas and small bowel transplantations accounting for the other most common procedures (Dominguez-Gil and Matesanz, 2017). When a graft is transplanted from a genetically non-identical individual, the recipients immune system recognizes the graft as foreign.

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This leads to an anti-graft immune response that involves T cells invading the new tissue, multiplying, and recruiting more immune cells to the transplant site in order to remove this foreign body. Depending on the nature of the incompatibility and the immune response, and acute or chronic rejection process can occur. Conversely, if the graft consists of hematopoietic stem cells or immune cells, there is a risk that they will mount an immune response against the host, known as graft versus host disease (GVHD). There are several safe pharmacological treatment options for acute rejections, but long-term treatment options remain unsatisfactory.

The risk of infection limits the effectiveness of these therapies, and improvements to their efficacy are also needed. Specific transplantation tolerance, in which alloreactive T cells are inactivated while the broader immune response is left intact, removing the need for broad immunosuppressant therapies, can be considered to be the ultimate goal for clinical transplantation. Compared with the overall immunology pipeline, in which there are 1,915 products, the transplantation pipeline is small, with only 244 products. However, of the 145 products in the transplantation pipeline with a disclosed molecular target, there are 65 first-in-class products, acting on a total of 63 distinct first-in-class targets, highlighting the fact that this pipeline displays strong levels of innovation.

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Scope

- There is a need for therapies that can achieve graft-specific immunosuppression, without having a general effect on the wider immune system. Which therapies and technologies currently in development are most likely to achieve this?
- There are 63 distinct first-in-class molecular targets in development for transplantation. Which of these hold the strongest potential in the clinic, and which are closest to reaching the market?
- How effective are current therapies for these indications, and how have they been able to improve the general prognosis in recent decades?
- Which molecule types and molecular targets are most prominent across transplantation therapy?
- Which specific types of transplantation are being most heavily studied across the pharmaceutical pipeline?

Reasons to buy

- Appreciate the current clinical and commercial landscapes by considering disease symptoms, pathogenesis, etiology, co-morbidities and complications, epidemiology, diagnosis, prognosis and treatment options for transplantation rejection.
- Identify leading products and key unmet needs within the market.
- Recognize trends in pipeline innovation by analyzing therapies by stage of development, molecule type and molecular target.
- Assess the therapeutic potential of first-in-class targets. Using a proprietary molecular target matrix assessment, first-in-class targets in the pipeline have been assessed and ranked according to clinical potential.
- Consider first-in-class pipeline products with no prior involvement in licensing and co-development deals, which may represent potential investment opportunities.

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Table of Contents
1 Table of Contents 2
1.1 List of Tables 3
1.2 List of Figures 3
2 Executive Summary 5
2.1 Strong Unmet need for Prevention of Chronic Rejection 5
2.2 Moderately-Sized by Highly Innovative Pipeline 5
2.3 Chemokines and Immune Surface Antigens Appear to Hold Promise as Novel Targets 5
3 The Case for Innovation 6
3.1 Growing Opportunities for Biologic Products 7
3.2 Diversification of Molecular Targets 7
3.3 Innovative First-in-Class Product Developments Remain Attractive 7
3.4 Regulatory and Reimbursement Policy Shifts Favor First-in-Class Product Innovation 8
3.5 Sustained Innovation 8
3.6 GBI Research Report Guidance 9
4 Clinical and Commercial Landscape 10
4.1 Disease Overview 10
4.2 Symptoms 11
4.2.1 Transplant Rejection 11
4.2.2 Graft-Versus-Host Disease 12
4.3 Diagnosis 12
4.4 Etiology and Pathophysiology 13
4.4.1 Major Histocompatibility Complex 13
4.4.2 Allogeneic Immune Response 13
4.4.3 Innate Immunity 15
4.4.4 The Role of Cytokines 15
4.4.5 Types of Rejection 15
4.5 Epidemiology and Prognosis 17
4.5.1 Transplant Rejection 17
4.5.2 Graft-Versus-Host Disease 18
4.6 Treatment Options 18
4.6.1 Antibody Therapies 19
4.6.2 Calcineurin Inhibitors 19
4.6.3 Anti-Proliferative Agents 19
4.6.4 mTOR Inhibitors 20
4.6.5 Corticosteroids 20
4.7 Overview of Marketed Products 21
4.8 Current Unmet Needs 23
5 Assessment of Pipeline Product Innovation 24
5.1 Pipeline by Stage of Development, Molecule Type and Molecular Target 24
5.2 First-in-Class Programs Targeting Novel Molecular Targets 28
6 Transplant Rejection Signaling Network, Disease Causation and Innovation Alignment 36
6.1 Complexity of Signaling Networks 36
6.2 Signaling Pathways and First-in-Class Molecular Target Integration 36
6.3 First-in-Class Matrix Assessment 36

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