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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

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

Summary


in 2015, more than of 125,000 transplantations were performed worldwide, an increase of over 5.5% from 2014, with around 33,000 of these occurring in the EU and a further 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.

Generally, there are a modest number of 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 needed regardless. 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 as an end-goal for clinical transplantation.

Compared with the overall immunology pipeline, in which there are over 1,900 products, the transplantation pipeline is small, with only about 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 first-in-class targets. First-in-class products account for 44% of transplantation pipeline products with a disclosed molecular target.

Of the 65 first-in-class pipeline products in development for transplantation and GVHD, those which emerged as being the most promising based on underlying scientific data included cell surface antigens such as CD40 and 47, and chemokines such as CXCR1 and 2. Many of these have yielded promising Preclinical data, although the availability of published clinical data is limited. Nonetheless, many drugs have reached a late stage of development, such as Dompe Farmaceutica’s reparixin, and appear poised to be valuable additions to the relatively stagnant range of treatments for the prevention of transplant rejection, and if successful will be able to drive higher long-term post-transplant survival rates.

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 helps to identify innovative pipeline programs across a therapy area, with a focus on indications with high R&D activity and strong innovation, as well as a competitive commercial landscape and high levels of deal activity. Monitoring innovative new product developments is becoming an increasingly vital part of competitive intelligence for all market participants, and increasingly important for companies seeking strategic partnerships or looking to acquire technologies, products or other companies.

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.


  • Strong Unmet need for Prevention of Chronic Rejection
    • Moderately-Sized by Highly Innovative Pipeline
    • Chemokines and Immune Surface Antigens Appear to Hold Promise as Novel Targets
  • The Case for Innovation
    • Table Figure 1: Transplantation Therapeutics, US, Innovation Trends in Product Approvals, 1987–2014
    • Growing Opportunities for Biologic Products
    • Diversification of Molecular Targets
    • Innovative First-in-Class Product Developments Remain Attractive
      • Table Figure 2: Transplantation Therapeutics, US, Sales Performance of First-in-Class and Non-First-in-Class Products Post Marketing Approval, 2006–2013
    • Regulatory and Reimbursement Policy Shifts Favor First-in-Class Product Innovation
    • Sustained Innovation
    • GBI Research Report Guidance
  • Clinical and Commercial Landscape
    • Disease Overview
    • Symptoms
      • Transplant Rejection
        • Table Transplantation Therapeutics, Global, Organ-Specific Symptoms of Transplant Rejection, 2017
      • Graft-Versus-Host Disease
        • Table Transplantation Therapeutics, Global, Organ-Specific Symptoms of Acute GVHD, 2017
        • Table Transplantation Therapeutics, Global, Organ-Specific Symptoms of Chronic GVHD, 2017
    • Diagnosis
    • Etiology and Pathophysiology
      • Major Histocompatibility Complex
      • Allogeneic Immune Response
      • Innate Immunity
      • The Role of Cytokines
      • Types of Rejection
    • Epidemiology and Prognosis
      • Transplant Rejection
      • Graft-Versus-Host Disease
    • Treatment Options
      • Antibody Therapies
      • Calcineurin Inhibitors
      • Anti-Proliferative Agents
      • mTOR Inhibitors
      • Corticosteroids
    • Overview of Marketed Products
      • Table Figure 3: Transplantation Therapeutics, Global, Overview of Marketed Products, 2017
    • Current Unmet Needs
  • Assessment of Pipeline Product Innovation
    • Pipeline by Stage of Development, Molecule Type and Molecular Target
      • Table Figure 4: Transplantation Therapeutics, Global, Overall Pharmaceutical Industry Pipeline by Therapy Area, 2017
      • Table Figure 5: Transplantation Therapeutics, Global, Pipeline by Stage of Development and Molecule Type, 2017
      • Table Figure 6: Transplantation Therapeutics, Global, Key Indications by Stage of Development and Molecule Type, 2017
      • Table Figure 7: Transplantation Therapeutics, Global, Pipeline by Molecular Target, 2017
      • Table Figure 8: Transplantation Therapeutics, Global, Key Indications by Molecular Target, 2017
    • First-in-Class Programs Targeting Novel Molecular Targets
      • Table Figure 9: Transplantation Therapeutics, Global, Distribution of Pipeline First-in-Class and Non-First-in-Class Products by Stage of Development and Molecular Target, 2017
      • Table Figure 10: Transplantation Therapeutics, Global, Percentage Distribution of First-in-Class and Non-First-in-Class Pipeline Products with a Disclosed Target by Stage of Development (%), 2017
      • Table Figure 11: Transplantation Therapeutics, Global, Percentage Distribution of First-in-Class and Non-First-in-Class Pipeline Products by Molecular Target (%), 2017
      • Table Figure 12: Transplantation Therapeutics, Global, Ratio of First-in- Class Products to First-in-Class Targets by Stage of Development, 2017
      • Table Figure 13: Transplantation Therapeutics, Global, Ratio of First-in- Class Products to First-in-Class Targets by Molecular Target, 2017
      • Table Figure 14: Transplantation Therapeutics, Global, Pipeline Products, 2017
  • Transplant Rejection Signaling Network, Disease Causation and Innovation Alignment
    • Complexity of Signaling Networks
    • Signaling Pathways and First-in-Class Molecular Target Integration
    • First-in-Class Matrix Assessment
      • Table Figure 19: Transplantation Therapeutics, Global, First-in-Class Matrix Assessment, 2017
  • First-in-Class Target and Pipeline Program Evaluation
    • Pipeline Programs Targeting C-X-C Chemokine Receptor Type 1 and C-X-C Chemokine Receptor Type 2
      • Table Transplantation Therapeutics, Global, Key Features of CX- C Chemokine Receptor Type 1, 2017
      • Table Transplantation Therapeutics, Global, Key Features of C-XC Chemokine Receptor Type 2, 2017
      • Table Transplantation Therapeutics, Global, Pipeline Programs Targeting C-X-C Chemokine Receptor Type 1 and C-X-C Chemokine Receptor Type 2, 2017
    • Pipeline Programs Targeting Tumor Necrosis Factor Receptor Superfamily Member 5
      • Table Transplantation Therapeutics, Global, Key Features of Tumor Necrosis Factor Receptor Superfamily Member 5, 2017
      • Table Transplantation Therapeutics, Global, Pipeline Programs Targeting Tumor Necrosis Factor Receptor Superfamily Member 5, 2017
    • Pipeline Programs Targeting Stimulator of Interferon Genes Protein
      • Table Transplantation Therapeutics, Global, Key Features of Stimulator of Interferon Genes Protein, 2017
      • Table Transplantation Therapeutics, Global, Pipeline Programs Targeting Stimulator of Interferon Genes Protein, 2017
    • Pipeline Programs Targeting Leukocyte Surface Antigen CD47
      • Table Transplantation Therapeutics Global, Key Features of Leukocyte Surface Antigen CD47, 2017
      • Table Transplantation Therapeutics, Global, Pipeline Programs Targeting Leukocyte Surface Antigen CD47, 2017
    • Pipeline Programs Targeting Tyrosine Protein Kinase SYK
      • Table Transplantation Therapeutics, Global, Key Features of Tyrosine Protein Kinase SYK, 2017
      • Table Transplantation Therapeutics, Global, Pipeline Programs Targeting Tyrosine Protein Kinase SYK, 2017
    • Pipeline Programs Targeting Potassium Voltage Gated Channel Subfamily A Member 3
      • Table Transplantation Therapeutics, Global, Key Features of Potassium Voltage Gated Channel Subfamily A Member 3, 2017
      • Table Transplantation Therapeutics, Global, Pipeline Programs Targeting Potassium Voltage Gated Channel Subfamily A Member 3, 2017
  • Strategic Consolidations
    • Industry-Wide First-in-Class Deals
      • Table Figure 20: Pharmaceutical Industry, Global, Licensing Deals by Stage of Development, 2006–2015
      • Table Figure 21: Pharmaceutical Industry, Global, Industry-Wide Licensing Deals by Deal Value, Upfront Payment Value, Stage of Development and First-in-Class Status ($m), 2006–2015
    • Licensing Deals
      • Deals by Region, Year and Value
        • Table Figure 22: Transplantation Therapeutics, Global, Licensing Deals by Region, Value and Year, 2006–2017
      • Deals by Stage of Development and Value
        • Table Figure 23: Transplantation Therapeutics, Global, Licensing Deals by Stage of Development, Deal Value and Upfront Payment Value, 2006–2017
      • Deals by Molecule Type, Molecular Target and Value
        • Table Figure 24: Transplantation Therapeutics, Global, Number and Aggregate Deal Value of Licensing Deals by Molecule Type and Molecular Target, 2006-2017
      • List of Deals with Disclosed Deal Values
        • Table Figure 25: Transplantation Therapeutics, Global, Licensing Deals with Disclosed Deal Values, 2006–2017
    • Co-development Deals
      • Deals by Region, Year and Value
        • Table Figure 26: Transplantation Therapeutics, Global, Co-development Deals by Region, Value and Year, 2006–2017
      • Deals by Stage of Development and Value
        • Table Figure 27: Transplantation Therapeutics, Global, Co-development Deals by Stage of Development, Deal Value and Upfront Payment Value, 2006–2017
      • Deals by Molecule Type, Molecular Target and Value
        • Table Figure 28: Transplantation Therapeutics, Global, Number and Aggregate Deal Value of Co-development Deals by Molecule Type and Molecular Target, 2006–2017
      • List of Deals with Disclosed Deal Values
        • Table Figure 29: Transplantation Therapeutics, Global, Co-development Deals with Disclosed Deal Values, 2006–2017
    • List of First-in-Class Pipeline Products with and without Prior Deal Involvement
      • Table Figure 30: Transplantation Therapeutics, Global, First-in-class Programs in Active Development Without Recorded Prior Deal Involvement, 2017
  • Appendix
    • Abbreviations
    • Disease List
    • References
    • Research Methodology
      • Data integrity
      • Innovative and meaningful analytical techniques and frameworks
      • Evidence based analysis and insight
    • Secondary Research
      • Market Analysis
      • Pipeline Analysis
      • Licensing and Co-development Deals
    • Contact Us
    • Disclaimer

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