Stakeholder Opinions: Hepatocellular cancer

Introduction
Despite the approval of Nexavar (sorafenib; Onyx pharmaceuticals/Bayer), patient outcomes remain poor. The large patient population and the lack of effective agents are driving R&D interest. The majority of pipeline drugs are molecular targeted therapies (MTTs) and their incorporation into treatment will improve both patient outcomes and commercial potential.

Scope of this research
Hepatocellular cancer overview, including disease definition, epidemiology, discussion of unmet needs, and market potential Discussion of current treatment options for hepatocellular cancer and their limitations Examination of the late-phase hepatocellular cancer pipeline, including drug profiles of late-stage agents Stakeholder opinions based on qualitative interviews with key opinion leaders from the US and Japan Research and analysis highlights
Hepatocellular cancer (HCC) is the fifth and seventh most common cancer in men and women respectively. The majority of patients with HCC are diagnosed with advanced-stage disease and have existing liver damage. The only approved treatment for late-stage disease is Nexavar which is not curative.

Chemotherapy is rarely used in hepatocellular cancer (HCC) as it is associated with low response rates. Regional therapies for early-stage patients have a high exclusion criteria and curative surgery is associated with relapse rates as high as 21.8%. Novel therapies are needed to improve survival rates in both early and late stage disease

There are currently 67 drugs in the pipeline, which is dominated by molecular targeted therapies (71%) and cytotoxic therapies (18%). Competition of late-phase pipeline drugs for first approval in HCC is fierce.

Key reasons to purchase this research
Estimate the number of treatable patients and identify unmet needs for future drug development opportunities Understand the current treatment of the disease, as well as opportunities and threats in the hepatocellular cancer market Analyze the current hepatocellular cancer pipeline and the potential of late-stage drugs
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TABLE OF CONTENTS
Overview 1
Catalyst 1
Summary 1
About Datamonitor healthcare 2
About the Oncology pharmaceutical analysis team 2
About the Epidemiology team 2
Executive Summary 3
Scope of the analysis 3
Datamonitor insight into the hepatocellular cancer market 3
Related reports 4
Upcoming reports 4
Patient Potential 12
Key findings 12
The healthy liver and its function 12
Liver anatomy 12
The function of the liver 13
Liver damage and cirrhosis 13
Hepatocellular cancer 14
Risk factors 15
Hepatitis infection 15
Hepatitis B 15
Hepatitis C 16
Alcohol 17
Aflatoxin exposure 18
Genetic predisposition 18
Diagnosis and screening 19
Complexities of early-stage diagnosis 19
Screening procedures 19
Current diagnosis 20
Staging 20
TNM classification 20
Child-Pugh 20
Cancer of the Liver Italian Program (CLIP) 21
The Barcelona Clinic Liver Classification (BCLC) 21
Treatment options for resectable HCC 22
Liver resection 22
Orthotopic liver transplantation (OLT) 23
Long waiting times due to organ shortage 23
Recurrence 24
Expense 24
Regional therapy; radiofrequency ablation (RFA) 25
Transcatheter arterial chemoembolization (TACE) 26
Percutaneous ethanol injection (PEI) 27
Treatment options for unresectable HCC 28
Chemotherapy: single agents 28
Doxorubicin 30
Cisplatin 31
Gemcitabine 31
Chemotherapy: combination regimens 31
Nexavar (sorafenib; Bayer/Onyx) 32
Nexavar as an adjuvant 36
Unmet need in hepatocellular cancer 37
Increased development of molecular targeted therapies (MTTs) 37
Greater focus on HCC prevention 38
Better designed clinical trials 38
Introduction and background 40
Key points 40
Disease definition and diagnosis criteria 41
Global variation and historical trends in incidence rates 41
Risk factors 46
Environmental risk factors 46
Host risk factors 48
Demographic risk factors 49
Epidemiologic forecasting of hepatocellular cancer 50
Cancer registries: age- and country-specific incidence rates 50
Population denominators 51
Hepatocellular cancer incidence statistical modeling 51
Incidence forecasting 52
Categorization and stratification 53
Age 53
Stage 54
Child-Pugh class 54
Epidemiologic forecast results 55
Estimated incident cases of hepatocellular cancer and future trends 55
Segmentation of incident cases: 2010 58
Segmentation by age group 58
Segmentation by stage 60
Segmentation by Child-Pugh class 61
Discussion 61
US 62
Japan 62
EU 63
Conclusions 63
Key findings 64
Current market overview 64
Target product profile versus current level of attainment 65
Opportunities and threats 66
Opportunities 66
High unmet need in the treatment of hepatocellular cancer 66
Increased interest in defining molecular targets for HCC 67
Growing patient population 67
Threats 67
Effective screening and increasing patient awareness could decrease patient population numbers 67
Pipeline Analysis 69
Key findings 69
Hepatocellular cancer pipeline overview 69
Afinitor (everolimus; Novartis) 75
Drug profile 75
Development overview 76
Product positioning 78
SWOT analysis 80
Tarceva (erlotinib; OSI Pharmaceuticals/Genentech/Roche) 81
Drug profile 81
Development overview 82
Product positioning 85
SWOT analysis 86
ThermoDox (heat activated doxorubicin; Celsion/Yakult Honsha) 87
Drug profile 87
Development overview 88
Product positioning 90
SWOT analysis 90
Brivanib (BMS-582664; Bristol-Myers Squibb) 91
Drug profile 91
Development overview 92
Significance of KRAS mutations for the effectiveness of brivanib 95
Product positioning 96
SWOT analysis 96
Linifanib (ABT-869; Abbott) 97
Drug profile 97
Development overview 98
Product positioning 100
SWOT analysis 101
Aptocine (talaporfin sodium; Light Science Oncology) 102
Drug profile 102
Development overview 103
Product positioning 104
SWOT analysis 105
Bibliography 107
Patient Potential 107
Journal papers 107
Websites 113
Datamonitor reports 115
Other 115
Epidemiology 115
Journal papers 115
Websites 119
Other 120
Datamonitor Reports 121
Market potential 121
Journal papers 121
Websites 121
Other 122
Pipeline analysis 122
Journal papers 122
Websites 124
Appendix 125
Contributing experts 125
Report methodology 125
About Datamonitor 126
About Datamonitor Healthcare 126
About the Oncology analysis team 127
Disclaimer 128


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