Stakeholder Opinions: Therapeutic Vaccines - A far-off promise of greatness

Overview 1
Catalyst 1
Summary 1
About Datamonitor healthcare 2
About the healthcare research & analysis team 2
Executive Summary 3
Scope of the analysis 3
Datamonitor insight into the therapeutic vaccines market 3
Related reports 6
Upcoming reports 7
Therapeutic vaccination – an overview 16
Key findings 16
General considerations 16
Therapeutic vaccines induce either T-cell or B-cell mediated immune responses 17
Dendritic cells (DCs) play a key initial role in human immunity. 17
T-cell based immune responses lead to an eradication of pathogens or infected cells by cytotoxic T-cells 18
B-cell based immune responses lead to the production of antibodies 19
Monoclonal antibody therapy has laid a good foundation for therapeutic vaccine development in some disease areas 20
Potential advantages and challenges of therapeutic vaccination 21
Advantages 22
Therapeutic vaccines could offer targeted therapies with increased specificity 22
The longevity of an immune response would reduce therapy times 22
Therapeutic vaccination may be able to provide a cure for some diseases which currently require lifelong treatment 23
Therapeutic vaccines could significantly reduce treatment costs for some indications 24
Challenges 24
Lack of understanding of the disease mechanism handicaps therapeutic vaccine development in some indications 24
Lack of involvement of Big Pharma is threatening progress in the sector 25
Safety is a key concern for therapeutic vaccination approaches 26
Reaching efficacy remains the main challenge for therapeutic vaccines 29
Strategies used to increase the efficacy of therapeutic vaccines 30
Adjuvants can increase vaccine immunogenicity, but are often associated with safety concerns 30
Antigen design and delivery – advantages for organized antigen structures 32
Mucosal or dermal administration could offer benefits, but is perceived as less important by experts 33
Combination of therapeutic vaccines with standard therapy could increase efficacy 34
Regulatory situation 35
Overview of indications outside cancer targeted by therapeutic vaccines 37
Therapeutic vaccines in cardiovascular and metabolic diseases 42
Key findings 42
Overview 42
Hypertension vaccines 43
Rationale for development of vaccines against hypertension 43
Disease background: over 25% of adults are suffering from hypertension 43
Compliance is a key challenge for existing hypertension therapy 43
Pipeline overview 44
Profiles of key candidates 46
CYT-006-AngQb (Cytos Biotechnology) 46
Angiotensin Therapeutic Vaccine (BTG) 48
Outlook for hypertension vaccines 49
Atherosclerosis vaccines 50
Rationale for development of vaccines against atherosclerosis 50
Disease background: atherosclerosis is the main underlying condition for coronary heart disease or stroke 50
Current atherosclerosis treatment is based on statin therapy 51
Pipeline overview 52
Outlook for atherosclerosis vaccines 53
Diabetes vaccines 54
Rationale for development of vaccines against diabetes 54
Disease background: the prevalence of diabetes is increasing due to an aging population and sedentary lifestyle 54
Vaccination could add significant value to diabetes therapy, particularly in type 1 patients 55
Pipeline overview 57
Profiles of key candidates 57
Diamyd (Diamyd Medical AB) 57
Outlook for diabetes vaccines 60
Therapeutic vaccines in CNS diseases 61
Key findings 61
Overview 61
Substance dependence vaccines 62
Rationale for development of vaccines against substance dependence 62
Disease background: nicotine dependence is the most prevalent substance dependence 62
Current treatments are inadequate for preventing smoking relapse 63
Vaccination could provide a safe and more efficacious alternative to existing therapies 64
Pipeline overview 66
Profiles of key candidates 66
NicVAX (Nabi Biopharmaceuticals/GlaxoSmithKline) 66
NIC-002 (Novartis/Cytos Biotechnology) 69
TA-NIC (Celtic Pharma) 71
Niccine (Independent Pharmaceutica AB) 72
TA-CD (Celtic Pharma) 74
Outlook for substance dependence vaccines 75
Alzheimer’s disease vaccines 77
Rationale for development of vaccines against Alzheimer’s disease 77
Disease background: Abeta and tau as main targets for future drugs 77
The number of people suffering from Alzheimer’s disease is increasing across the seven major markets 77
Current treatments are symptomatic, with no disease-modifying drugs available 78
Therapeutic vaccination appears a promising concept for the therapy of Alzheimer’s disease 78
The termination of AN1792 has taught valuable lessons for vaccine design 80
Pipeline overview 81
Profiles of key candidates 82
ACC-001 (Johnson & Johnson/Pfizer) 82
CAD-106 (Novartis/Cytos Biotechnology) 85
Outlook for Alzheimer’s disease vaccines 86
Therapeutic vaccines in autoimmune disorders 89
Key findings 89
Overview 89
Rationale for development of vaccines against autoimmune diseases 90
Rheumatoid arthritis is a key target disease for vaccine makers 90
Disease background: rheumatoid arthritis is a chronic disease, mainly affecting the elderly 90
Antibody-based treatments have provided proof-of-concept for immunotherapy of rheumatoid arthritis 90
Vaccines could address several unmet needs in rheumatoid arthritis therapy 91
Multiple sclerosis is another key target for vaccine developers 92
Disease background: multiple sclerosis is a chronic disease causing severe damage to the central nervous system 92
Current treatment: interferons remain the backbone of multiple sclerosis therapy 93
Key unmet needs in multiple sclerosis therapy include improved efficacy and safety 93
Vaccination is a promising approach for the treatment of autoimmune diseases 94
Pipeline overview 95
Profiles of key candidates 96
Tovaxin (Opexa) 96
BHT-3009 (Bayhill Therapeutics) 99
Outlook for therapeutic vaccines against autoimmune diseases 100
Therapeutic vaccines in infectious diseases 104
Key findings 104
Overview 104
Hepatitis B therapeutic vaccines 105
Rationale for development of therapeutic vaccines against hepatitis B 105
Disease background: over 360 million people globally suffer from chronic hepatitis B 105
Current therapy is based on interferons and antivirals 106
Vaccination could improve efficacy, convenience and susceptibility to resistances 106
Pipeline overview 108
Profiles of key candidates 110
Hepatitis B virus vaccine (Emergent Biosolutions) 110
Outlook for therapeutic hepatitis B vaccines 112
Hepatitis C therapeutic vaccines 113
Rationale for development of therapeutic vaccines against hepatitis B 113
Disease background – hepatitis C is a key underlying condition for cirrhosis and liver cancer 113
Current treatment gold standard is combination therapy with peginterferon / ribavirin 114
Unmet needs include efficacy in genotype 1 patients, tolerability and shorter treatment duration 115
Hepatitis C is a challenging target for vaccine developers 116
Pipeline overview 118
Profiles of key candidates 119
GI-5005 / HepCVax (GlobeImmune) 119
IC41 (Intercell) 121
ChronVac-C (Tripep/Inovio) 123
Outlook for therapeutic hepatitis C vaccines 125
Human immunodeficiency virus (HIV) 126
Rationale for the development of therapeutic vaccines against HIV 126
Disease background – HIV prevalence is highest in the developing world 126
HAART has transformed HIV into a chronic disease, with many treatment options available 127
Viral eradication remains the most important unmet need in HIV therapy 128
Although promising in theory, vaccination against HIV faces significant challenges in practice 130
Pipeline overview 132
Clinical failure of Merck & Co’s prophylactic candidate has increased concerns regarding the feasibility of therapeutic HIV vaccination 134
Profiles of key candidates 135
Vacc-4x (Bionor Immuno) 135
AGS-004 (Argos Therapeutics/Kyowa Hakko Kirin) 136
Outlook for therapeutic HIV vaccines 137
Bibliography 140
Journals 140
Websites 148
Datamonitor reports 152
Appendix A 154
Data definitions, limitations and assumptions 154
Standard units 154
Country group definitions 154
Rest of European Union 154
Middle East and North Africa (MENA) 154
Rest of World 154
Appendix B 155
Contributing experts 155
Conferences attended 155
Report methodology 155
About Datamonitor 156
About Datamonitor Healthcare 156
About the Infectious Diseases analysis team 157
Key therapy team members 158
Holger Rovini, Head of Respiratory and Infectious Diseases 158
Hedwig Kresse, Senior Analyst, Infectious Diseases 158
Disclaimer 160