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Coagulation Factors 2009: Target Pipeline and Corporate Benchmark Analysis

Published by: La Merie Publishing

Published: Aug. 1, 2009 - 386 Pages


Table of Contents


1 Executive Summary

2 Overview: What you will find in the report and where

3 Introduction


4 Target Pipeline Analysis


5 Corporate Benchmark Analysis

5.1.1 Baxter

5.2 The Diversifying Companies
5.2.1 CSL
5.3 The Emerging Companies
5.3.1 Zymogenetics


5.4 The Facilitator Companies
6 References

7 Tables in the Text

Table 1 Prevalence of rare coagulation factor disorders

Table 2 2008 Sales of Recombinant Factor VIII Products

Table 3 Growth of the recombinant Factor VIII market (US$ mln)

Table 4 BeneFix Sales (1999 - 2008)

Table 5 NovoSeven Sales (1999 - 2008)

Table 6 Total Sales of recombinant coagulation factors in 2008

Table 7 2008 Sales of selected pd coag. factors for systemic use

Table 8 2008 Sales of selected pd coagulation factors for topical use

Table 9 2008 Sales of coagulation factor product lines per company

Table 10 Changes of manufacturing/formulation in rec. coag. factors

Table 11 Approval / launches of rec coag. factors in the US and EU

Table 12 The TOP coag. factor companies in terms of 2008 sales

Table 13 Product characteristics of improved rhu F. VIII products

Table 14 Overview of R&D pipeline of rec factor VIII products

Table 15 Target profiles of rec wild-type F. VIII products in R&D

Table 16 Target profiles of rec long-acting F. VIII products in R&D

Table 17 Product characteristics of marketed wt rhu F. IX

Table 18 Overview of R&D pipeline of rec factor IX products

Table 19 Target profiles of rec wild-type F. IX products in R&D

Table 20 Target profiles of rec long-acting F. IX products in R&D

Table 21 Product characteristics of marketed wt rhu F. VII

Table 22 Overview of R&D pipeline of rec factor VIIa products

Table 23 Target profile of rec wild-type F. VIIa product in R&D

Table 24 Target profiles of rec long-acting F. VIIa products in R&D

Table 25 Overview on Baxter’s R&D Pipeline in rec coag. factors

Table 26 SWOT analysis of Baxter

Table 27 Overview on Novo Nordisk’s R&D pipeline in coag. factors

Table 28 SWOT analysis of Novo Nordisk

Table 29 Overview on Bayer’s R&D pipeline in rec coag. factors

Table 30 SWOT analysis of Bayer Schering Pharma

Table 31 Overview on Wyeth’s R&D pipeline in rec coag. factors

Table 32 SWOT analysis of Wyeth

Table 33 Overview on TOP League’s R&D pipeline in r coag. factors

Table 34 The largest plasma fractionation companies by plasma vol.

Table 35 The largest plasma fractionation companies by sales

Table 36 SWOT analysis of CSL

Table 37 SWOT analysis of Octapharma

Table 38 SWOT analysis of LFB

Table 39 SWOT analysis of Zymogenetics

Table 40 SWOT analysis of Biogen Idec

Table 41 SWOT analysis of Biovitrum

Table 42 SWOT analysis of Inspiration Biopharmaceuticals

Table 43 SWOT analysis of Ipsen

Table 44 SWOT analysis of Green Cross

Table 45 Protein engineering companies (ref. rec coagulation factors)

Table 46 Drug delivery companies (ref. rec coagulation factors)

Table 47 Product portfolio of traditional plasma fract. companies

Table 48 R&D portfolio of traditional plasma fractionation companies

ADDENDUM

A) Executive Drug Profiles

AV513

BAY 79-4980

BAY 86-6150

CSL689

FIXFc

GreenGene

IB1001

NN1731

NN1841

NN7008

NN7128

OBI-1

Octagenate

rVWF

B) Company Profiles

Amsterdam Molecular Therapeutics

Apitope

Asklepios Biopharmaceutical

Baxter

Bayer Schering Pharma

Coagulation Factors 2009

August 2009 Page: 6 / 386

Bio Products Laboratory (BPL)

Biogen Idec

Biotest

Biovitrum

Catalyst Biosciences

Celestial Biologicals

China Biologic Products

Crucell

CSL

Ethicon (J&J)

Green Cross

Grifols

GTC Biotherapeutics

Haemacure

Inspiration Biopharmaceuticals

Intas Biopharmaceuticals

Ipsen

Jerini (Shire)

Kaketsuken

Kamada

Kedrion

King Pharmaceuticals

LFB

Lipoxen

Maxygen

MDRNA

MediVas

Mochida Pharmaceutical

National Bioproducts Institute (NBI)

Nautilus Biotech (HanAll Pharmaceutical)

Coagulation Factors 2009

August 2009 Page: 7 / 386

Nektar Therapeutics

Neose Technologies

Novo Nordisk

Nycomed

Octapharma

Oxford Biomedica

Pharming

ProFibrix

ProGenetics

PROLOR Biotech

Recoly

Sanquin

Shanghai RAAS

SK Chemicals

Talecris Biotherapeutics

Thrombotargets

VIRxSYS

Wyeth

Zymogenetics

C) Tabulated Overviews on Profiles of Plasma Derived Coagulation Factors

Tab I: Profile of Human Plasma-Derived Coagulation Factor VIII Concentrates

Tab II: Profile of Highly Purified Human Plasma-Derived Coagulation Factor IX Concentrates

Tab III: Profile of Purified Human Plasma-Derived Prothrombin Complex Concentrates

Tab IV: Profile of Other Human Plasma-Derived Coagulation Factor Concentrates

Tab V: Profile of Topical Coagulation Factor Products & Projects

Coagulation Factors 2009

August 2009 Page: 8 / 386

D) Tabulated Target Pipelines of Coagulation Factors

Tab VI: Human Plasma-Derived Coagulation Factor VIII Concentrates

Tab VII: Recombinant Coagulation Factor VIII Products

Tab VIII: Human Plasma-Derived Coagulation Factor IX Products

Tab IX: Recombinant Coagulation Factor IX Products

Tab X: Human Plasma-Derived Factor VII

Tab XI: Recombinant Coagulation Factor VII Products

Tab XII: Human Plasma-Derived Coagulation Factor Concentrates for vWD

Tab XIII: Recombinant Human Von Willebrand Factor Products

Tab XIV: Human Plasma-Derived Fibrinogen (Factor I)

Tab XV: Recombinant Human Fibrinogen Products

Tab XVI: Recombinant Human Factor V

Tab XVII Human Plasma-Derived Factor XI

Tab XVIII: Human Plasma-Derived Factor X

Tab XIX: Recombinant Human Factor X

Tab XX: Human Plasma-Derived Factor XIII

Tab XXI: Recombinant Human Factor XIII

Tab XXII: Other Recombinant Factors

Tab XXIII: Other Experimental Hemophilia Therapies

Tab XXIV: Gene Therapy

Tab XXV: Topical Human Plasma-Derived Fibrinogen and Thrombin Products

Tab XXVI: Topical Plasma-Derived Thrombin Products

Tab XXVII: Human Plasma-Derived Prothrombin Complex Concentrates

E) Tabulated Corporate Product Portfolios and R&D Pipelines

Abraxis Biosciences

Amsterdam Molecular Therapeutics

Angiotech

Apitope

Asklepios Biopharmaceutical

Coagulation Factors 2009

August 2009 Page: 9 / 386

Baxter

Bayer Schering Pharma

Bio Products Laboratory (BPL)

Biogen Idec

Biotest

Biovitrum

Catalyst Biosciences

China Biologic Products

Crucell

CSL

Ethicon (J&J)

Genzyme

Green Cross

Grifols

GTC Biotherapeutics

Haemacure

Inspiration Biopharmaceuticals

Intas Biopharmaceuticals

Ipsen

Jerini (Shire)

Kaketsuken

Kamada

Kedrion

King Pharmaceuticals

LFB

Lipoxen

Maxygen

MDRNA

MediVas

Mochida Pharmaceutical

Coagulation Factors 2009

August 2009 Page: 10 / 386

National Bioproducts Institute (NBI)

Nautilus Biotech (HanAll Pharmaceutical)

Nektar Therapeutics

Neose Technologies

Novo Nordisk

Nycomed

Octapharma

Oxford Biomedica

Pharming

ProFibrix

ProGenetics

PROLOR Biotech

Recoly

Roche

Sanquin

Shanghai RAAS

Sinobiomed

SK Chemicals

Talecris Biotherapeutics

Thrombotargets

VIRxSYS

Wyeth

Zymogenetics

Abstract

The “Coagulation Factors 2009: Target Pipeline and Corporate Benchmark Analysis” report is the most complete and up-to-date evaluation and assessment of the recombinant and plasma-derived coagulation factor pipelines. The authors analyze and assess the target pipeline for each of the coagulation factors used for systemic and topical administration. Companies active in the therapeutic coagulation business are evaluated and the strengths, weaknesses, opportunities and threats (SWOT) in their R&D pipeline benchmarked in the respective peer group. Technologies used for creation of next generation coagulation factors are discussed and assessed. The report identifies strategies to overcome weaknesses in the portfolio and suggests development strategies

Background

The market size of recombinant and plasma-derived coagulation factor products exceeded US$ 7.5 bln in 2008, the majority (US$ 5.5 bln) generated by recombinant proteins. Replacement of plasma-derived coagulation factors by marketed recombinant molecules is ongoing and will further increase by new recombinant coagulation factors in development. In light of ongoing expiration of substance patents, about 40 new recombinant coagulation factor projects are in the pipeline indicating a changing marketplace over the next decade. New players are competing and will challenge established manufacturers and marketers of coagulation factor products.

Scope of the report

R&D pipeline overview; In depth profiles of most promising coagulation factors in the pipeline pipeline analysis for each coagulation factor ; evaluation and assessment of protein engineering and drug delivery technologies overview and analysis of marketed coagulation factors; profiles of more than 50 companies active in this business; corporate product portfolios and R&D pipelines benchmark and SWOT analysis of corporate competitors within peer group;

Highlights

Ongoing patent expirations and failed life cycle management efforts leave the established companies vulnerable regarding the future growth perspective of their recombinant coagulation factor business, but at the same time opens the door to potential new market entrants.

The recombinant factor VIII pipeline consists of more than ten novel molecules, four of them already in clinical stages, some are wild-type constructs, but many are longer acting variants of factor VIII. The recombinant factor IX pipeline even counts 15 developments with three in clinical phases. In the recombinant factor VIIa pipeline, all projects except one are modified versions, either short-acting or longer acting with four projects in the clinic and two more shortly to follow.

All of the next generation molecules need systemic intravenous administration, attempts to allow subcutaneous or even non-injectable administration regimes so far have not succeeded and leave space for future optimization.

SWOT analysis revealed Novo Nordisk as well positioned in contrast to market leader Baxter.

Benefits from the report Understand your position in a highly competitive environment Avoid costly mistakes Learn from your competitors Challenge your strategy with a view from outside the organization Revise weaknesses of your molecule and development plan Identify threats to your business plan Realize new opportunities suggested by the report Reassure and build upon your strengths Find applications for your technology

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