Recombinant Coagulation Factors 2015: Maturation of recombinant clotting factor pipeline and emergence of gene therapy and alternative procoagulants

Recombinant Coagulation Factors 2015:
Maturation of recombinant clotting factor pipeline and emergence of gene therapy and alternative procoagulants

A Pipeline Landscape Analysis and Comparative Assessment of Key Players

Table of Contents

1.         Executive Summary

2.         Recombinant Coagulation Factor Markets

2.1       Recombinant Factor VIII (rFVIII) Product Sales and Market Size
2.1.1    Advate/Recombinate Sales
2.1.2    Kogenate FS Sales
2.1.3    Helixate Sales
2.1.4    Xyntha / Refactor AF Sales
2.1.5    Eloctate Sales
2.1.6    2014 Performance of rFVIII Product Sales
2.1.7    rFVIII Market Size

2.2       Recombinant Factor IX (rFIX) Product Sales and Market Size
2.2.1    BeneFIX Sales
2.2.2    Alprolix Sales
2.2.3    2014 Performance of rFIX Product Sales
2.2.4    rFIX Market Size

2.3       Recombinant Factor VII (rFVII) Product Sales and Market Size
2.3.1    NovoSeven Sales
2.3.2    Coagil VII Sales
2.3.3    rFVII Market Size

2.4       Recombinant Thrombin Product Sales and Market Size

2.5       Total Recombinant Coagulation Factor Market Size

3.         Pipeline Update of Recombinant Coagulation Factors

3.1       Factor VIII Pipeline Changes and Drug Profile Updates
3.1.1    Wild-type, improved and biosimilar rFVIII
3.1.2    Long-acting rFVIII

3.2       Factor IX Pipeline Changes and Drug Profile Updates
3.2.1    Wild-type rFIX
3.2.2    Long-acting rFIX

3.3       Factor VII Pipeline Changes and Drug Profile Updates
3.3.1    Wild-type, improved and biosimilar rFVIIa
3.3.2    Long-acting rFVIIa

3.4       Pipeline Update for Other Recombinant Coagulation Factors

3.5       Pipeline Update for Alternative Procoagulants
3.5.1    Coagulation Factor Mimetics
3.5.2    Inhibitor Switch-off
3.5.3    Systemic Thrombin
3.5.4    Factor Xa Variants
3.5.5    Oral Procoagulants

3.6       Pipeline Update for Gene & Cell Therapy
3.6.1    Gene & Cell Therapy of Hemophilia B
3.6.2    Gene & Cell Therapy of Hemophilia A
3.6.3    Gene and Cell Therapy of Hemophilia A and B

3.7       Immune Tolerance Inducing (ITI) Agents

4.         Competitive Landscape Analysis of Recombinant Coagulation Factors

4.1       rFVIII Competitive Landscape Analysis
4.1.1    Wild-type, improved and biosimilar rFVIII Landscape
4.1.2    Long-acting rFVIII Landscape
4.2       rFIX Competitive Landscape Analysis
4.3       rFVII Competitive Landscape Analysis
4.4       Competitive Landscape of Other Recombinant Coagulation Factors
4.5       Competitive Landscape of Alternative Procoagulants for Systemic Use
4.6       Competitive Landscape of Gene & Cell Therapy for Hemophilia
4.7       Immune Tolerance Inducing (ITI) Agents

5.         Stakeholder Profiles

5.1       Coagulation Factor Portfolio Companies
5.1.1    Baxter
5.1.2    Novo Nordisk
5.1.3    CSL
5.1.4    Bayer HealthCare Pharmaceuticals
5.1.5    Pfizer
5.1.6    Octapharma
5.1.7    LFB
5.1.8    Biotest
5.1.9    The Medicines Company

5.2       Big Pharma & Biotech as New Entrants to Bleeding Disorder Field
5.2.1    Biogen Idec
5.2.2    Roche (Chugai)
5.2.3    AstraZeneca (MedImmune)

5.3       Specialty Pharma as New Entrants to Hemophilia Field
5.3.1    Shire
5.3.2    BioMarin Pharmaceutical
5.3.3    Emergent BioSolutions
5.3.4    OPKO Health

5.4       Biosimilar Companies
5.4.1    Generium Group
5.4.2    Aryogen

5.5       Protein Engineering Companies
5.5.1    Catalyst Biosciences
5.5.2    Pharming
5.5.3    The Chemo-Sero-Therapeutic Research Institute KAKETSUKEN

5.6       Gene & Cell Therapy Companies
5.6.1    uniQure
5.6.2    Spark Therapeutics
5.6.3    Dimension Therapeutics
5.6.4    Expression Therapeutics
5.6.5    Sangamo Biosciences
5.6.6    ReGenX
5.6.7    Sernova & Medicyte
5.6.8    Amarna Therapeutics

5.7       RNA Companies
5.7.1    Alnylam Pharmaceuticals
5.7.2    Arcturus Therapeutics

5.8       Immunology Companies
5.8.1    Apitope
5.8.2    Selecta Biosciences
5.8.3    EpiVax

6.         Strategic Analysis

6.1       Pipeline and Technology Overview and Trends
6.2       Short- and Mid-term Perspective of Recombinant Coagulation Factors
6.3       Key Product Profiles and Differentation Factors among Novel Products
6.4       Mid- and long-term perspective of new hemophilia treatments
6.5       Key Players in the Field

7          References

8          ADDENDUM: Development and Approval Timelines

Recombinant Coagulation Factors 2015:
Maturation of recombinant clotting factor pipeline and emergence of gene therapy and alternative procoagulants

A Pipeline Landscape Analysis and Comparative Assessment of Key Players

Tables

Table 1            Sales of Advate/Recombinate
Table 2            Sales of Kogenate (FS)
Table 3            Sales of Helixate (NexGen)
Table 4            Sales of ReFacto (AF)/Xyntha
Table 5            Growth of the Recombinant Factor VIII Market
Table 6            Market Shares of Recombinant Factor VIII Products
Table 7            Sales of BeneFIX / Growth of rFIX Market
Table 8            Sales of NovoSeven
Table 9            Sales of Coagil VII
Table 10          Growth of Recombinant Factor VIIa Market
Table 11          Sales of Recothrom
Table 12          Total Market Size of Recombinant Coagulation Factors in Hemophilia
Table 13          The US/EU Clinical Trial Program of GreenGene F
Table 14          The Clinical Trial Program of Nuwiq (simoctocog alfa)
Table 15          The LEOPOLD Clinical Trial Program of BAY 81-8973
Table 16          The AFFINITY Clinical Trial Program of CSL627
Table 17          The Clinical Trial Program of Eloctate
Table 18          The Clinical Trial Program of BAX 855
Table 19          The Clinical Trial Program of damoctocog alfa pegol
Table 20          The PATHFINDER Clinical Trial Program of turoctocog alfa pegol
Table 21          The Clinical Trial Program of nonacog gamma
Table 22          The Clinical Trial Program of trenonacog alfa
Table 23          The Clinical Trial Program of Alprolix
Table 24          The PROLONG-9FP Clinical Trial Program of CSL654
Table 25          The PARADIGM Clinical Trial Program of nonacog beta pegol
Table 26          The Clinical Trial Program of BAX 817
Table 27          The PERSEPT Clinical Trial Program of LR769
Table 28          The PROLONG-7FP Clinical Trial Program of CSL689
Table 29          R&D Stage and Timeline of wild-type, improved and biosimilar rFVIII
Table 30          Inhibitor Formation by wild-type and improved rFVIII
Table 31          Half-Life and Dosing Frequency of wild-type and improved rFVIII
Table 32          Efficacy of wild-type and improved rFVIII
Table 33          R&D Stage and Timeline of Long-Acting rFVIII
Table 34          Inhibitor Formation by Long-Acting rFVIII
Table 35          Half-Life and Dosing Frequency of Long-Acting rFVIII
Table 36          Efficacy of Long-Acting rFVIII
Table 37          R&D Stage and Timeline of wild-type, biosimilar and long-acting rFIX
Table 38          Inhibitor Formation by rFIX
Table 39          Half-Life and Dosing Frequency of rFIX
Table 40          Efficacy of rFIX
Table 41          R&D Stage and Timeline of wild-type, biosimlar and long-acting rFVII
Table 42          Inhibitor Formation by wild-type and long-acting rFVII
Table 43          Half-Life and Dosing Frequency of wild-type and long-acting rFVII
Table 44          Other Recombinant Coagulation Factors
Table 45          R&D Stage and Timelines of Alternative Procoagulants
Table 46          Product Profiles of Systemic Alternative Procoagulants
Table 47          R&D Stage and Timeline of Gene Therapy of Hemophilia B
Table 48          R&D Stage and Timeline of Gene & Cell Therapy of Hemophilia A
Table 49          R&D Stage and Timeline of Gene Therapy of Hemophilia A and B
Table 50          Immune Tolerance Inducing (ITI) Agents
Table 51          Baxter Hemophilia Recombinant Product Portfolio and R&D Pipeline
Table 52          Novo Nordisk Bleeding Disorder Product Portfolio and R&D Pipeline
Table 53          CSL Hemophilia Recombinant Product Portfolio and R&D Pipeline
Table 54          Bayer Hemophilia Product Portfolio and R&D Pipeline
Table 55          Pfizer Bleeding Disorder Recombinant Products and R&D Pipeline
Table 56          Octapharma Hemophilia Recombinant Product Portfolio
Table 57          LFB Hemophilia Recombinant R&D Pipeline
Table 58          The Medicines Company Bleeding Disorder Product and R&D Pipeline
Table 59          Biogen Idec Hemophilia Recombinant Products and R&D Pipeline
Table 60          AstraZeneca Hemophilia & Bleeding Disorder R&D Pipeline
Table 61          Shire and Sangamo Biosciences Bleeding Disorder R&D Pipeline
Table 62          BioMarin Pharmaceutical Hemophilia R&D Pipeline
Table 63          Emergent BioSolutions Hemophilia R&D Pipeline
Table 64          OPKO Health Hemophilia R&D Pipeline
Table 65          Generium Hemophilia Product Portfolio and R&D Pipeline
Table 66          Catalyst Biosciences Bleeding Disorder Recombinant R&D Pipeline
Table 67          Pharming Hemophilia & Bleeding Disorder R&D Pipeline
Table 68          uniQure Hemophilia R&D Pipeline
Table 69          Spark Therapeutics Hemophilia R&D Pipeline
Table 70          Dimension Therapeutics Hemophilia R&D Pipeline
Table 71          Expression Therapeutics Hemophilia R&D Pipeline
Table 72          ReGenX Biosciences Hemophilia R&D Pipeline
Table 73          Sernova & Medicyte Hemophilia R&D Pipeline
Table 74          Pipeline Overview and R&D Trends
Table 75          Target Launch Years for Novel rFVIII Molecules by Company Names
Table 76          Target Launch Years for Novel rFIX Molecules by Company Names
Table 77          Major Players and Hemophilia Portfolio Coverage

Recombinant Coagulation Factors 2015:
Maturation of recombinant clotting factor pipeline and emergence of gene therapy and alternative procoagulants

A Pipeline Landscape Analysis and Comparative Assessment of Key Players

Alnylam Pharmaceuticals
Alphamab
Alteogen
Amarna Therapeutics
Apitope
Arcturus Therapeutics
Aryogen
AstraZeneca
Baxter
Bayer HealthCare Pharmaceuticals
Biogen Idec
Biotest
Catalyst Biosciences
Chia Tai Tiangqing Pharma Group
Chiesi Farmaceutici
CSL
Dimension Therapeutics
Dong-A Pharmaceutical
Emergent BioSolutions
EpiVax
Expression Therapeutics
Generium Group
Green Cross
ISU Abxis
Nektar Therapeutics
Novo Nordisk
Octapharma
Opus Bio (Lentigen)
Pfizer
Pharming
Pharmstandard
REGENX Biosciences
Roche (Chugai)
Sangamo Biosciences
Sanofi (Genzyme)
Selecta Biosciences
Shanghai Institute for Pharmaceutical Industry (SIPI, a Sinopharma company)
Shire
Spark Therapeutics
Swedish Orphan Biovitrum (Sobi)
Teijin Pharma
The Chemo-Sero-Therapeutic Research Institute (KAKETSUKEN),
The Medicines Company
UCB
uniQure
Xenetic Biosciences