GRNVAC DCVax-Brain AGS Glionix CYT004-MelQbG CDX-110 (PF ) GI TABLE 23 OCCURRENCE OF

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1 CHAPTER ONE: INTRODUCTION.1 STUDY GOALS AND OBJECTIVES... 1 REASONS FOR DOING THIS STUDY... 1 INTENDED AUDIENCE... 1 SCOPE OF REPORT... 2 METHODOLOGY... 2 INFORMATION SOURCES... 3 AUTHOR S CREDENTIALS... 3 RELATED BCC REPORTS... 3 BCC ONLINE SERVICES... 3 DISCLAIMER... 4 CHAPTER TWO: SUMMARY... 5 SUMMARY... 5 SUMMARY TABLE THE WORLDWIDE MARKET FOR THERAPEUTIC VACCINES BY INDICATION, THROUGH 2014 ($ MILLIONS)... 6 SUMMARY FIGURE THE WORLDWIDE MARKET FOR THERAPEUTIC VACCINES BY INDICATION, CHAPTER THREE: OVERVIEW... 7 DEFINITION OF A THERAPEUTIC VACCINE... 7 IMMUNE SYSTEM OVERVIEW: CAST OF CHARACTERS... 7 B-CELLS, T-CELLS AND ANTIGEN-PRESENTING CELLS... 7 B-Cells... 8 T-Cells... 8 Antigen-Presenting Cells... 8 CELLULAR VERSUS HUMORAL IMMUNITY... 8 INNOVATIVE APPROACHES TO VACCINE TECHNOLOGY... 9 DNA VACCINES... 9 CARBOHYDRATE VACCINES... 9 THE ROLE OF ADJUVANTS ATTRACTIVE FEATURES OF THERAPEUTIC VACCINES VERSUS OTHER CLASSES OF THERAPIES TABLE 1 FEATURES OF THERAPEUTIC VACCINES VERSUS OTHER DRUG CLASSES THERAPEUTIC VACCINES APPROVED FOR CLINICAL USE OR IN CLINICAL TRIALS TABLE 2 THERAPEUTIC VACCINES IN CLINICAL TRIALS, ORDERED BY PHASE TABLE 2 (CONTINUED) TABLE 2 (CONTINUED) TABLE 2 (CONTINUED) TABLE 2 (CONTINUED) TABLE 3 NUMBER OF THERAPEUTIC VACCINES IN CLINICAL TRIALS, BY PHASE... 15

2 FIGURE 1 VACCINES IN CLINICAL TRIALS BY INDICATION, 2010 (%) TABLE 4 VACCINES IN CLINICAL TRIALS BY TECHNOLOGY TABLE 5 COMPANIES WITH THERAPEUTIC VACCINES IN CLINICAL TRIALS CHAPTER FOUR: TECHNOLOGY OVERVIEW TYPES OF THERAPEUTIC VACCINES TABLE 6 COMPARISON OF AUTOLOGOUS VERSUS ALLOGENEIC VACCINES TABLE 7 NUMBER OF ALLOGENEIC AND AUTOLOGOUS THERAPEUTIC VACCINES IN DEVELOPMENT BY PHASE CHAPTER FIVE: PATENT EVALUATION PATENT LENGTHS AND MARKET EXCLUSIVITY TABLE 8 PATENT EXPIRATION DATES FOR THERAPEUTIC VACCINES IN LATE STAGES OF DEVELOPMENT CHAPTER SIX: PRODUCTS ADDICTION VACCINES TABLE 9 ADDICTION VACCINES IN DEVELOPMENT BY PHASE TABLE 10 THE WORLDWIDE MARKET FOR ADDICTION VACCINES, THROUGH 2014 ($ MILLIONS) COCAINE ADDICTION VACCINES The Market for Cocaine Addiction Vaccines TA-CD NICOTINE ADDICTION VACCINES The Market for Nicotine Addiction Vaccines NicVAX TABLE 11 NICOTINE ABSTINENCE RATE AT 12 MONTHS IN DIFFERENT STUDIES (%) NIC TA-NIC AUTOIMMUNE DISEASE VACCINES TABLE 12 AUTOIMMUNE DISEASE VACCINES IN DEVELOPMENT BY PHASE DIABETES (TYPE 1) Diamyd (rhgad65) Diamyd (rhgad65) (Continued) BHT Tovaxin TNFaKinoid CANCER VACCINES CANCER STATISTICS U.S. Cancer Statistics... 37

3 TABLE 13 INCIDENCE, MORTALITY, AND SURVIVAL RATES FOR COMMON CANCERS IN THE U.S., TABLE 14 CANCER PREVALENCE RATES IN THE U.S., 2006 (THOUSANDS) Current Treatment Methods Surgery Radiation Therapy Pharmaceutical Therapy CANCER VACCINES: A NEW MODALITY OF CARE The Primary Therapeutic Role for Cancer Vaccines The Immune System and Cancer Antigens: Their Role in Cancer Vaccines Antigens Used in Cancer Vaccine Development Carcinoembryonic Antigen Ganglioside Molecules Heat Shock Proteins MART Prostate-Specific Antigen Sialyl Tn Tyrosinase MAGE NY-ESO MUC Vaccine Development: Strategies Vaccine Development: (Continued) A Summary of Two Approaches: Low-technology vs. Hightechnology Vaccine Types Antigen/Adjuvant Vaccines Dendritic Cell Vaccines (DCs) DNA and Viral Vector Vaccines Idiotype Vaccines Oncolytic Virus Vaccines Whole Cell Tumor Vaccines TABLE 15 TYPES OF VACCINES IN DEVELOPMENT FOR CANCER TREATMENT The Long Road to Market The Market for Cancer Vaccines TABLE 16 ESTIMATED INITIAL U.S. MARKET OPPORTUNITY FOR CANCER VACCINES BY INDICATION TABLE 17 WORLDWIDE MARKET FOR HUMAN CANCER VACCINES BY TYPE, THROUGH 2014 ($ MILLIONS)... 53

4 TABLE 18 THE MARKET FOR HUMAN CANCER VACCINES, BY TECHNOLOGY (AUTOLOGOUS VERSUS ALLOGENEIC), WORLDWIDE, THROUGH 2014 ($ MILLIONS) FIGURE 2 NUMBER OF AUTOLOGOUS VERSUS ALLOGENEIC CANCER VACCINES IN DEVELOPMENT, 2010 (%) TABLE 19 THE WORLDWIDE MARKET FOR HUMAN CANCER VACCINES, BY INDICATION, THROUGH 2014 ($ MILLIONS) CANCER VACCINES IN DEVELOPMENT TABLE 20 CANCER VACCINES IN PHASE II OR LATER TRIALS BY PHASE TABLE 20 (CONTINUED) CANCER VACCINES APPROVED FOR USE ON THE WORLDWIDE MARKET Melacine Oncophage Oncophage (Continued) M-Vax M-Vax (Continued) CANCER VACCINES IN DEVELOPMENT Provenge (APC8015, Sipuleucel-T) TABLE 21 PROVENGE PHASE III CLINICAL TRIAL RESULTS Provenge (Continued) OncoVAX OncoVAX (Continued) MyVax BiovaxId BiovaxId (Continued) Multikine Multikine (Continued) GSK A TABLE 22 EXPRESSION OF MAGE-A3 ANTIGEN BY TUMOR TYPE (%) GSK A BLP25 Liposome Vaccine (Stimuvax) BLP25 Liposome (Continued) BLP25 Liposome (Continued) TroVax PR1 Peptide Vaccine Allovectin Allovectin-7 (Continued) Belagenpumatucel-L (Lucanix) GV CB MelCancerVac... 81

5 GRNVAC DCVax-Brain AGS Glionix CYT004-MelQbG CDX-110 (PF ) GI TABLE 23 OCCURRENCE OF RAS MUTATIONS IN VARIOUS SOLID TUMORS (%) HyperAcute-Pancreas HyperAcute Melanoma HyperAcute-Lung PEP IMA JX Onyvax-P (ONY-P1) Onyvax-P (ONY-P1) (Continued) TG4010 (MVA-MUC1-IL2) TG1042 (Ad-IFNγ) TG4001/R3484 (MVA-HPV-IL2) Abagovomab CV AE NeuVax (E75) ProstVac INFECTIOUS DISEASE VACCINES TABLE 24 INFECTIOUS DISEASE VACCINES IN DEVELOPMENT BY PHASE HIV/AIDS THE HIV INFECTION PROCESS HIV TRANSMISSION CONFUSION REGARDING THE PROPHYLACTIC VERSUS THERAPEUTIC ACTIVITY OF AIDS VACCINES BARRIERS TO DEVELOPING AND TESTING AIDS VACCINES Barriers to Developing (Continued) WHAT HAS BEEN LEARNED THE MARKET FOR AIDS VACCINES HIV THERAPEUTIC VACCINES IN DEVELOPMENT Results of Early Trials of HIV/AIDS Vaccines AIDSVAX, the Most Advanced HIV Vaccine Candidate but with Mixed Results The Failure of Merck s V520 Prophylactic and Therapeutic Vaccine

6 The Remune (AG1661, HIV-1 Immunogen) Saga The Remune (Continued) ALVAC-HIV (vcp1521), AIDSVAX Combination HIV Vaccine Prophylactic and/or Therapeutic HIV Vaccine Vacc-4x DermaVir (LC002) AGS OTHER INFECTIOUS DISEASE VACCINES (OUTSIDE HIV) HEPATITIS C GI TG4040 (MVA-HCV) IC NEUROLOGICAL DISEASE VACCINES TABLE 25 NEUROLOGICAL DISEASE VACCINES IN DEVELOPMENT THE MARKET FOR NEUROLOGICAL DISEASE VACCINES Alzheimer s Disease Vaccine (AN-1792) (Discontinued) ACC CAD PARKINSON S DISEASE VACCINE OTHER DISEASE VACCINES TABLE 26 OTHER DISEASE VACCINES IN DEVELOPMENT CYT006-AngQb Angiotensin Therapeutic Vaccine Angiotensin (Continued) CHAPTER SEVEN: MAJOR COMPANIES AND MARKET SHARE MARKET SHARE TABLE 27 FORECASTED SALES AND MARKET SHARE OF COMPANIES INVOLVED IN THERAPEUTIC VACCINES, 2014 ($ MILLIONS/%) COMPANY PROFILES AFFIRIS ANTIGENICS ARGOS THERAPEUTICS BAVARIAN NORDIC BIOVEST INTERNATIONAL CELLDEX THERAPEUTICS CEL-SCI CELTIC PHARMA CUREVAC CYTOS BIOTECHNOLOGY DENDREON DIAMYD MEDICAL ELAN

7 GENEXINE GERON GLAXOSMITHKLINE GLAXOSMITHKLINE U.S GLOBEIMMUNE KAEL MERCK KGAA (MERCK SERONO) NABI BIOPHARMACEUTICALS NEWLINK GENETICS NOVARTIS NOVARTIS INSTITUTES FOR BIOMEDICAL RESEARCH NOVARX ONCOTHYREON OXFORD BIOMEDICA PFIZER TRANSGENE VACCINOGEN VACCINOGEN U.S VICAL Vical (Continued) CHAPTER EIGHT: INTERNATIONAL ASPECTS AND REGULATORY ISSUES MARKET BY REGION TABLE 28 GLOBAL FORECAST FOR THERAPEUTIC VACCINES MARKET BY REGION, THROUGH 2014 ($ MILLIONS) MARKET BY REGION (CONTINUED) UNITED STATES The FDA Modernization Act of Fast-track Status Special Protocol Assessments New Surveillance and Safety Requirements Approvable Letters No More Medicare Other Healthcare Changes EUROPEAN UNION New Drug Approvals and Innovation New Drug (Continued) Drug Reimbursement and Reference Pricing Relocation of R&D Regulatory Climate JAPAN New Drug Approvals Regulatory Climate Relationship between Academia and Industry

8 CHAPTER NINE: FUTURE DIRECTIONS THERAPEUTIC VACCINES AS FIRST-LINE THERAPIES THE ROLE OF BIOMARKERS VACCINES AND CLINICAL TRIAL DESIGN COMBINATION THERAPIES