The Future of Targeted Therapeutics: Key technologies, new therapy area applications and leading players

Table of Contents

Executive Summary

• Emergence of targeted therapeutics
• Analysis of stakeholder incentives
• Types of targeted therapeutics
• Role of targeted therapeutics in selected therapy areas

Chapter 1 Emergence of targeted therapeutics

• Summary
• Introduction
• Decline of the blockbuster model
• The "nichebuster" model
• The emergence of targeted therapeutics
• Convergence of biotechnology and pharmaceutical companies
  • Licensing incentives
  • Risk management
• Role of biomarkers and diagnostics
• Academic research and technology transfer

Chapter 2 Analysis of stakeholder incentives

• Summary
• Introduction
• Institutional payors
• Regulators
• Patients and patient advocacy groups
• Physicians
• Pharmaceutical companies

Chapter 3 Types of targeted therapeutics

• Summary
• Introduction
• Innovative small molecules
• Mechanism of action
• Examples
  • Gleevec
  • Lapatinib (Tykerd/Tyverb)
  • SNS-032
  • PLX4032
• Market synopsis
• Protein/Peptide drugs
• Mechanism of action
• Examples
  • AGRO100
  • PRX321
• Market synopsis
• Monoclonal antibodies and antibody-like structures
• Mechanism of action
• Examples
  • Rituximab (Rituxan)
  • Alemtuzumab (Campath)
  • Erbitux/cetuximab
  • Avastin/bevacizumab
  • Herceptin/trastuzumab
  • Ibritumomab tiuxetan (Zevalin)
  • Tositumomab (Bexxar)
• Market synopsis
• Nucleic acid therapies
• Mechanism of action
• Examples
  • Genasense (oblimersen sodium)
  • Nexagon
  • 4-Thio FAC
• Market synopsis
• RNA interference
• Mechanism of action
• Examples
  • CALAA-01
  • siRNA AV-34
  • Bioral SiRNA
• Market synopsis
• Stem cell therapies
• Market synopsis
• Future therapeutic applications
• Regulatory concerns
• Gene therapy
• Drug delivery and nanotechnology in targeted therapeutics
• Mechanism of action
• Examples
  • BioVant
  • ATI-1123 (docetaxel)
  • Rexin-G
  • Abraxane
  • 131I-TM-601
• Market synopsis
• Conclusion

Chapter 4 Role of targeted therapeutics in selected therapy areas

• Summary
• The cancer market
  • Targeting cancer cells for destruction
  • Blocking growth signalling pathways
• Epidemiology
• Current treatments
• Unmet needs and innovation
• Medical Needs
  • High toxicity and side effect profiles
  • Variable efficacy rates
  • The multifactorial nature of disease
  • Targeting brain tumors
• Economic Needs
  • Reimbursement policies
• Targeted therapeutics in cancer
• Antineoplastics
  • Innovative small molecule therapies
  • Monoclonal antibody therapies
  • Proteins and Peptides
  • Application of genetic technology
  • RNAi in cancer
  • Applications of nanotechnology
  • Novel drug delivery approaches
• The Cardiovascular Market
• Epidemiology
• Current market scenario
• Unmet needs and innovation
  • Pill burden and patient compliance
  • Inefficient mechanism of action
  • Drug delivery technology limitations
• Targeted therapeutics in cardiovascular diseases
  • Protein and nucleic acid therapies
  • Targeted small molecule drugs
  • Monoclonal antibody therapies
  • Stem cells
  • Nanotechnology
• The CNS Market
• Epidemiology
• Current treatments
• Unmet needs and innovation
• Crossing the blood-brain barrier
• Improving specificity and affinity
• Accuracy in determining drug bioavailability
• Improved side effect profiles
• Appropriate clinical trial design - defining target patient subsets
• Diagnosis-specific unmet needs
  • Depression
  • Schizophrenia
  • Sleep disorders
  • Alzheimer' s disease and other dementias
  • Epilepsy
  • Parkinson' s disease and movement disorders
  • Pain therapeutics
• Targeted therapeutics in CNS
• Innovative small molecules
• Nucleic acid therapies
• Monoclonal antibodies
• Stem cell and gene therapy
• Novel therapeutic mechanisms of action
  • Synthetic toxins
  • Targeted viral delivery systems
  • Innovative diagnostic devices
• The Infectious Diseases Market
• Epidemiology
• Viral Infections
  • Hepatitis
  • Influenza
  • Herpes simplex virus
  • Bacterial infections
  • Fungal infections
• Current market scenario
• Unmet Needs and Innovations
• Efficacy
• Emerging resistance to existing treatments and lack of innovation
• Immunoglobin (vaccination) drug delivery mechanisms
• Targeted therapeutics in infectious diseases
• Innovative small molecules
• Applications of genetic technology
  • Therapeutic proteins and peptides
  • RNAi in infectious diseases
  • Antibodies
  • Novel targeted drug delivery mechanisms

Chapter 5 Appendix

• Research methodology
• ATC Codes
• Cancer Therapies
• Antineoplastic and immunomodulating agents
  • L1: Cytostatics
  • L2: Cytostatic hormone therapy
  • L3: Immunostimulating agents
• Cardiovascular Therapies
  • B1: Anti-thrombotic agents
  • C1: Cardiac Therapy
  • C2 Antihypertensives
  • C3 Diuretics
  • C7 Beta Blockers
  • C8 Calcium antagonists
  • C9 Agents acting on the renin-angiotensin system
  • C10 Hypolipidaemics/Anti-Atheroma Preparations
• Index

List of Figures

• Figure 1.1: Forecast growth for the top 20 biopharmaceutical companies, 2006-12
• Figure 1.2: Evolution of the biopharmaceutical corporation
• Figure 1.3: Reliance on licensed products vs. forecast revenue growth, top 20 pharmaceutical firms, 2006-12
• Figure 1.4: FDA new drug approvals and Phase 3 failures, 2006-2007
• Figure 2.5: Typical health economic value proposition for innovative or personalized therapies
• Figure 3.6: Mechanism of action of targeted small-molecule inhibitors
• Figure 3.7: Schematic presentation of targeted delivery of therapeutic proteins and peptides to antigen-positive tumor cells or tumor vasculature
• Figure 3.8: Mechanism of action of monoclonal antibodies
• Figure 3.9: Mechanism of post-transcriptional gene silencing by siRNA
• Figure 4.10: Global cancer innovatives market, 2003-2006
• Figure 4.11: Targeted oncology pipeline by indication, 2006

List of Tables

• Table 1.1: Global revenues from blockbuster drugs, top 10 pharma companies, 2006 ($m)
• Table 1.2: Complementary strengths of pharma and biotech companies
• Table 4.3: Estimated annual incidence of cancer diagnoses (‘000s), global, 2002 census
• Table 4.4: Breakdown of the global antineoplastic market by drug class,
• Table 4.5: Leading therapies in the global antineoplastics market, 2005-07
• Table 4.6: Antineoplastic sales forecast, 2006-12
• Table 4.7: Chemotherapeutic siRNA targets in drug development pipelines
• Table 4.8: Key pipeline oncology drugs and technologies utilizing nanotechnology, 2008
• Table 4.9: Estimated prevalence of CVD across the seven major markets, 2006
• Table 4.10: Breakdown of the global CVD market by drug class, 2003-07
• Table 4.11: Estimated prevalence of CNS disorders across the seven major markets, 2006
• Table 4.12: Global sales of CNS products by disease indication, 2005-2007, $m
• Table 4.13: Top 10 CNS brands, sales revenues, global, 2007, $m
• Table 4.14: Medicinal chemical properties of a successful CNS drug
• Table 4.15: Estimated prevalence of infective diseases across seven major markets, 2005
• Table 4.16: The anti-infectives market, global sales revenues, 2003-2007, $m