EXECUTIVE SUMMARY

• Scope of the analysis
• Datamonitor insight into the hematological malignancies market
• Key metrics
• Datamonitor pipeline assessment summary

PIPELINE OVERVIEW AND DYNAMICS

• Pipeline overview
- Products in late-phase development for hematological malignancies
- Products in Phase II development for hematological malignancies
- Products in Phase I development for hematological malignancies
• Pipeline by developmental phase and class
- There are 180 different products in the clinical developmental pipeline for hematological malignancies
• Pipeline by indication
- Over 50% of the pipeline products are being investigated in leukemia
- The 22 late-phase pipeline products target eight different hematological malignancies
• Pipeline by company
- Developmental pipeline dominated by small pharma/biotech players
- Only 17 (12%) companies/institutes have more than two candidates in the developmental pipeline for hematological malignancies

HEMATOLOGICAL MALIGNANCIES – MARKET POTENTIAL

• A diverse range of disease subtypes
• Genetic basis of cancer evolution
- Tumorigenesis is the result of co-operative accumulated mutations
• Existing pharmacotherapy approaches provide limited treatment benefit
- Cytotoxic drugs lack specificity
- Hormonal or endocrine therapy provides incremental benefit in selected tumors
- Optimizing current treatment strategies is paramount
• The emergence of targeted treatment heralds a revolution in cancer pharmacotherapy
• Dynamic cancer market offers significant commercial opportunity
- Ongoing sales growth drives the market
- Intensive R&D produces a rich developmental pipeline
• Epidemiology - Hematological Malignancies
- Cancer epidemiology - an expanding patient base
- Leukemia
- Lymphoma
- Multiple myeloma
- Myelodysplastic syndrome
- Disproportionate increase in prevalence results from improvements in diagnosis and treatment
• Significant areas of unmet need persist
- The need for more sophisticated pharmacotherapy
- Long-term control of advanced tumors is suboptimal
- Novel strategies required to reduce relapse rates in early-stage disease
- Toxicity of existing treatments jeopardizes quality of life and rates of treatment uptake
- Improvements in diagnostics and prognostic analysis will enhance cost-effectiveness of treatment
- Enhanced preventative strategies will ease the disease burden
• Clinical and strategic threats to the commercialization of cancer drugs
- Progressively rising R&D costs threaten industry productivity
- Pharmacoeconomic pressures drive payers to implement restrictive pricing and reimbursement policies
- Therapeutic and generic competition reduces periods of market exclusivity
- Segmentation of market will require changes in clinical trial methodology

R&D APPROACH

• Classification of pipeline products
- Molecular targeted therapies
- Cytotoxic Therapies
- Immunotherapeutic agents
• Evolution in oncology clinical trial design
- Patient selection is increasingly significant in the era of targeted treatment
- Clinical trials must have sufficient follow-up to establish true clinical benefit
- Diversity of targeted treatments will require an evolution in clinical trial design
- Most oncology clinical trials designate multiple endpoints
• Modification of accelerated approval process may impact significantly on approval times for hematologic oncology drugs

MOLECULAR TARGETED THERAPIES ANALYSIS AND FORECASTS

• Overview of molecular targeted therapies for hematological malignancies
- Pipeline summary
- Comparative forecasts
- Definition of current comparator therapy
• Tasigna (Nilotinib, AMN-107; Novartis)
- Drug overview
- Clinical trial data
- Datamonitor comments
- Forecasts to 2016
- Datamonitor drug assessment summary
• Ceflatonin (Myelostat; ChemGenex Pharmaceuticals)
- Drug overview
- Clinical trial data
- Datamonitor comments
- Forecasts to 2016
- Datamonitor drug assessment summary
• Sarasar (Lonafarnib; Schering-Plough)
- Drug overview
- Clinical trial data
- Datamonitor comments
- Forecasts to 2016
- Datamonitor drug assessment summary
• Torisel (Temsirolimus; Wyeth)
- Drug overview
- Clinical trial data
- Datamonitor comments
- Forecasts to 2016
- Datamonitor drug assessment summary
• Zarnestra (Tipifarnib; Janssen/Johnson & Johnson)
- Drug overview
- Clinical trial data
- Datamonitor comments
- Forecasts to 2016
- Datamonitor drug assessment summary
• Alvocidib (Flavopiridol; Sanofi-Aventis)
- Drug overview
- Clinical trial data
- Datamonitor comments
• Enzastaurin (LY317615; Eli Lilly)
- Drug overview
- Clinical trial data
- Datamonitor comments
- Forecasts to 2016
- Datamonitor drug assessment summary
• Lestaurtinib (CEP-701; Cephalon)
- Drug overview
- Clinical trial data
- Datamonitor comments
- Forecasts to 2016
- Datamonitor drug assessment summary
• Genasense (Oblimersen; Genta)
- Drug overview
- Clinical trial data
- Datamonitor comments
- Forecasts to 2016
- Datamonitor drug assessment summary

CYTOTOXIC THERAPIES ANALYSIS AND FORECASTS

• Overview of cytotoxic therapies for hematological malignancies
- Pipeline summary
- Comparative forecasts
• Clolar/Evoltra (Clofarabine; Genzyme/Bioenvision)
- Drug overview
- Clinical trial data
- Datamonitor comments
- Forecasts to 2016
- Datamonitor drug assessment summary
• Dacogen (decitabine; MGI Pharma)
- Drug overview
- Clinical trial data
- Datamonitor comments
- Forecasts to 2016
- Datamonitor drug assessment summary
• Cloretazine (VNP40101M; Vion Pharmaceuticals)
- Drug overview
- Clinical trial data
- Datamonitor comments
- Forecasts to 2016
- Datamonitor drug assessment summary
• Pixantrone (BBR-2778; Cell Therapeutics)
- Drug overview
- Clinical trial data
- Datamonitor comments
- Forecasts to 2016
- Datamonitor drug assessment summary
• Marqibo (Sphingosomal vincristine; Hana Biosciences)
- Drug overview
- Clinical trial data
- Datamonitor comments

IMMUNOTHERAPEUTIC AGENTS ANALYSIS AND FORECASTS

• Overview of immunotherapeutic agents for hematological malignancies
- Pipeline summary
- Comparative forecasts
- Definition of current comparator therapy
• Ceplene (Histamine dihydrochloride; Epicept)
- Drug overview
- Clinical trial data
- Datamonitor comments
- Forecasts to 2016
- Datamonitor drug assessment summary
• Galiximab (Anti-CD80 MAb; Biogen Idec)
- Drug overview
- Clinical trial data
- Datamonitor comments
- Forecasts to 2016
- Datamonitor drug assessment summary
• Lumiliximab (Anti-CD23 MAb; Biogen Idec)
- Drug overview
- Clinical trial data
- Datamonitor comments
- Forecasts to 2016
- Datamonitor drug assessment summary
• Ofatumumab (HuMax-CD20; Genmab/GlaxoSmithKline)
- Drug overview
- Clinical trial data
- Datamonitor comments
- Forecasts to 2016
- Datamonitor drug assessment summary
• Zanolimumab (HuMax-CD4; Merck Serono/Genmab)
- Drug overview
- Clinical trial data
- Datamonitor comments
- Forecasts to 2016
- Datamonitor drug assessment summary
• BIOVAXID (Accentia Biopharmaceuticals)
- Drug overview
- Clinical trial data
- Datamonitor comments
- Forecasts to 2016
- Datamonitor drug assessment summary
• FavId (Id-KLH; Favrille)
- Drug overview
- Clinical trial data
- Datamonitor comments
- Forecasts to 2016
- Datamonitor drug assessment summary
• MyVax (GTOP-99; Genitope)
- Drug overview
- Clinical trial data
- Datamonitor comments
- Forecasts to 2016
- Datamonitor drug assessment summary
• Comparison of anti-idiotype vaccines

APPENDIX

• List of tables
• List of figures
• Methodology
- Datamonitor forecast methodology
- Datamonitor drug assessment summary
• Abbreviations
• Contributing experts
• Key opinion leader interview transcripts
• Bibliography
• About Datamonitor
- About Datamonitor Healthcare
• Datamonitor Healthcare's therapy area capabilities
- About the Disease analysis team
- Disclaimer

LIST OF TABLES

Table 1: Late-phase pipeline products for hematological malignancies sales forecasts in the seven major markets ($m), 2007-2016
Table 2: Products in late-phase development for hematological malignancies, 2007
Table 3: Products in Phase II development for hematological malignancies, 2007
Table 4: Products in Phase I development for hematological malignancies, 2007
Table 5: Pipeline products in development for hematological malignancies by Phase and class, 2007
Table 6: Pipeline products by specific hematological malignancy, 2007
Table 7: Companies/Institutes with three or more products in the hematological malignancies pipeline, 2007
Table 8: Novartis' marketed oncology portfolio, 2007
Table 9: Novartis' hematological malignancies pipeline portfolio, 2007
Table 10: Biogen Idec's marketed oncology portfolio, 2007
Table 11: Biogen Idec's hematological malignancies pipeline portfolio, 2007
Table 12: Common mutations involved in tumor development
Table 13: Forecast incidence of hematological malignancies in the seven major pharmaceutical markets, 2002-2016
Table 14: Country-specific forecast incidence of the hematological malignancies, 2007
Table 15: Leukemia incidence in the seven major markets, 2002-2016

LIST OF FIGURES

Figure 1: Pipeline molecular targeted therapies sales forecasts ($m), 2007-2016
Figure 2: Pipeline cytotoxic therapies sales forecasts ($m), 2007-2016
Figure 3: Pipeline immunotherapeutic agents sales forecasts ($m), 2007-2016
Figure 4: Clinical and commercial attractiveness of pipeline molecular targeted therapies in development for hematological malignancies, 2007
Figure 5: Clinical and commercial attractiveness of pipeline cytotoxic therapies in development for hematological malignancies, 2007
Figure 6: Clinical and commercial attractiveness of pipeline immunotherapeutic agents in development for hematological malignancies, 2007
Figure 7: Pipeline products in development for hematological malignancies by Phase and class, 2007
Figure 8: Pipeline products in development for hematological malignancies by phase, 2007
Figure 9: Molecular targeted therapies pipeline candidates for hematological malignancies by developmental phase, 2007
Figure 10: Cytotoxic pipeline candidates for hematological malignancies by developmental phase, 2007
Figure 11: Immunotherapeutic pipeline candidates for hematological malignancies by developmental phase, 2007
Figure 12: Pipeline products in development for hematological malignancies by class, 2007
Figure 13: Pipeline products in development for hematological malignancies by subcategory within each class, 2007
Figure 14: Leukemia, lymphoma and myeloma pipeline products, 2007
Figure 15: Pipeline products by specific hematological malignancy, 2007
Figure 16: Late-phase pipeline products by specific hematological malignancy, 2007
Figure 17: Pipeline products in development for hematological malignancies by type of company, 2007
Figure 18: Global oncology sales ($m), 2002-2011
Figure 19: Oncology pipeline including supportive care, 2006
Figure 20: Forecast incidence of hematological malignancies in the seven major pharmaceutical markets, 2007 and 2016
Figure 21: Country specific forecast incidence of the hematological malignancies in 2007