The Future of the Biodefense Industry: Regulation, funding opportunities and company profiles

The Future of the Biodefense Industry: Regulation, funding opportunities and company profiles
Executive summary 12

Setting the scene 12

Regulatory environment 13

Major pathogens for BW 14

Biodefense funding 15

Company profiles 16

Future perspectives 17

Chapter 1 Setting the scene 20

Summary 20

Introduction 21

The use of biological agents in warfare and terrorism 22

Warfare 22

Offensive BW programs 23

Bioterrorism: Extent and nature of the threat 24

Creation 26

Bulk manufacture 27

Weaponization 27

Delivery 27

Future threats 29

Pre-2001 bioterrorism 29

2001 - September 11 and the anthrax attacks 30

Chapter 2 Regulatory environment 34

Summary 34

Introduction 35

US bodies 36

FDA 38

Center for Biologics Evaluation and Research (CBER) 38

Center for Drug Evaluation and Research (CDER) 40

Office of Counterterrorism and Emerging Threats 41

National Institutes of Health (NIH) 41

The National Institute of Allergy and Infectious Diseases (NIAID) 42

Biomedical Advanced Research and Development Authority (BARDA) 43

Public Health Emergency Medical Countermeasures Enterprise (PHEMCE) 44

Project Bioshield 45

Centers for Disease Control and Prevention (CDC) 48

US Army Medical Materiel Development Activity (USAMMDA) 49

European bodies 49

European Commission 50

European Council Health Security Committee 51

European Centre for Disease Prevention and Control (ECDC) 54

The European Medicines Agency (EMEA) 55

Economic Commission for Europe 57

Joint Commission/Pharmaceutical Industry Task Force 57

2009-2011 Indicative Program for the Instrument for Stability 58

International Agencies 58

World Health Organisation (WHO) 58

United Nations 59

Global Health Security Initiative (GHSI) 59

Chapter 3 Major pathogens for BW 62

Summary 62

Introduction 63

Anthrax 64

Developmental therapeutics and vaccines 65

Plague 68

Developmental therapeutics and vaccines 69

Tularemia 70

Developmental therapeutics and vaccines 70

Smallpox 71

Developmental therapeutics and vaccines 72

Viral Hemorrhagic Fevers - Ebola, Marburg, Crimean-Congo,

Hantavirus, Lassa 74

Developmental therapeutics and vaccines 75

Botulism 77

Developmental therapeutics and vaccines 77

Ricin 79

Developmental therapeutics and vaccines 80

Staphylococcal enterotoxins 81

Developmental therapeutics and vaccines 81

Brucella group 82

Developmental therapeutics and vaccines 82

Glanders 83

Developmental therapeutics and vaccines 83

Melioidosis (Whitmore’s disease) 84

Developmental therapeutics and vaccines 84

Q fever 85

Developmental therapeutics and vaccines 86

Conclusion 87

Chapter 4 Biodefense funding 90

Summary 90

US funding 91

Department of Health and Human Services (HHS) 93

Biomedical Research and Development Authority (BARDA) 94

Department of Homeland Security (DHS) 96

Other departments 97

Grants available for biodefense R&D in the US 97

NIH Challenge grants in health & science research 98

NIH Cooperative Research & Development Agreement (CRADA) 98

Small business awards 99

NIH small business biodefense program awards 100

Project BioShield awards 101

Regional Centers of Excellence for Biodefense and Emerging Infectious Diseases (RCE) 102

Defense Threat Reduction Agency (DTRA) 103

US Army Medical Research Institute of Infectious Diseases (USAMRIID) 103

Technology transfer 103

Defense Advanced Research Projects Agency (DARPA) 104

European funding 104

United Kingdom 105

France 106

Germany 106

EU biodefense programs 107

European Defense Agency (EDA) 107

EDA’s Joint R&D Investment Program 107

Bio-Edep 108

Bioterrorism Research Resilience Reaction (BIO3R) 108

Biodefense industry 108

New and Emerging Science and Technology (NEST) 109

Health Protection Agency (HPA) 110

Defense Science and Technology Laboratory (Dstl) 110

Japan 111

Chapter 5 Company profiles 114

Summary 114

Introduction 115

US 117

Achaogen 117

Contracts 118

Advanced Life Sciences 118

Contracts 119

Alnylam Pharmaceuticals 119

Contracts 119

Cleveland BioLabs 120

Contracts 120

Dynavax Technologies Inc. 121

Contracts 121

Dynport Vaccine Company Llc (DVC) 122

Contracts 122

Elusys Therapeutics 122

Contracts 123

Emergent BioSolutions 123

Contracts 125

Ichor Medical Systems 126

Contracts 126

Nanotherapeutics 127

Contracts 127

Peregrine Pharmaceuticals, Inc. 127

Contracts 128

PharmAthene 128

Contracts 129

SIGA Technologies 130

Contracts 131

VaxGen 132

Contracts 132

XOMA 132

Contracts 132

Canada 133

Affinium Pharmaceuticals 133

Contracts 133

Cangene 133

Contracts 135

ID Biomedical Corporation 135

Contracts 136

UK 136

Acambis 136

Contracts 137

Cambridge Biostability (CBL) 137

Contracts 138

MNLpharma 138

Contracts 138

Denmark 139

Bavarian Nordic 139

Contracts 140

Australia 141

Vaxine Pty 141

Contracts 141

India 141

Panacea Biotec Ltd. 142

Contracts 142

China 143

Sinovac Biotech Ltd. 144

Israel 144

Medison Pharma 145

Conclusion 145

Biodefense products: the risks 147

Chapter 6 Future perspectives 152

Summary 152

Introduction 153

Assessing future threats 153

GM technology 154

Synthetic biology 156

US funding – the reality 157

Has the US got value for its £50 billion-worth of funding since 2001? 159

Potential biodefense business 159

Getting a grant in the US 160

Project Bioshield 161

Is Europe doing enough? 162

Lack of coordination 164

US 164

EU 165

Biodefense – at the expense of other research? 166

Mismanagement of the stockpile 167

Case study: VaxGen 167

Problems of biosecurity 169

US 169

Russia and the FSU 171

Fulfilling unmet needs 172

viii

Conclusion 173

Chapter 7 Appendix 176

CDC characterization of biological threats 176

Glossary 177

Index 181

List of Figures

Figure 1.1: Bioterrorism incidents 1970-2008 26

Figure 2.1: US biodefense funding by department 2001-09 37

Figure 3.1: R&D funding pathway for biodefense medical countermeasures 43

Figure 4.1: Project BioShield procurement process 46

Figure 4.5: US biodefense funding ($m), 2001-12 93

Figure 4.6: BARDA funding forecasts ($m), 2009-14 96

Figure 5.7: Cleveland BioLabs research spending on Protectan CBL502 for biodefense applications ($m), 2008-09 121

Figure 5.8: Emergent BioSolutions operating results ($m), 2008-09 125

Figure 5.9: PharmAthene financial results ($m), 2008-09 130

Figure 5.10: Cangene financial results ($m), 2004-08 134

Figure 5.11: Bavarian Nordic financials (DKKm), 2004-08 140

Figure 6.12: US biodefense funding ($m), 2001-09 158

Figure 6.13: VaxGen financials ($m), 2005-08 168

Figure 6.14: NIH funded biocontainment laboratories 170

Figure 7.15: CDC characterization of biological threats 176

List of Tables

Table 2.1: US biodefense presidential acts 40

Table 2.2: US biodefense acts post-2001 48

Table 2.3: European Biodefense Legislation 54

Table 3.4: Developmental therapeutics and vaccines for anthrax 66

Table 3.5: Developmental therapeutics and vaccines for plague 69

Table 3.6: Developmental therapeutics and vaccines for tularemia 71

Table 3.7: Developmental therapeutics and vaccines for smallpox 73

Table 3.8: Developmental therapeutics and vaccines for VHF 76

Table 3.9: Developmental therapeutics and vaccines for botulism 78

Table 3.10: Developmental therapeutics and vaccines for ricin 80

Table 3.11: Developmental therapeutics and vaccines for staphylococcal enterotoxins 81

Table 3.12: Developmental therapeutics and vaccines for brucella 82

Table 3.13: Developmental therapeutics and vaccines for glanders 84

Table 3.14: Developmental therapeutics and vaccines for melioidosis 85

Table 3.15: Developmental therapeutics and vaccines for Q fever 86

Table 4.16: US biodefense funding by department ($m), 2001-10 92