The Fastest Growing Biotechnology Companies: Growth strategies, comparative analyses and company profiles

Executive Summary 16
Summary of key findings 16
Comparitive analyses of growth strategies 17
Theravance, Inc - company profile and analysis 18
ISTA Pharmaceuticals - company profile and analysis 19
Palatin Technologies - company profile and analysis 20
Pharmion Corporation – company profile and analysis 21
Amylin Pharmaceuticals - company profile and analysis 22
Trimeris, Inc - company profile and analysis 23
ViroPharma Inc - company profile and analysis 24
NPS Pharmaceuticals - company profile and analysis 25
SIGA Technologies, Inc - company profile and analysis 26
Idenix Pharmaceuticals - company profile and analysis 27
Chapter 1 Key findings and industry
analysis 30
Summary 30
Overview of the biotechnology sector 31
Recent developments 32
Current applications 33
Structure of the biotechnology industry 35
The pharmaceutical-biopharmaceutical relationship 35
Emergence of the biopharmaceutical sector 36
Characteristics of the biopharmaceutical industry 40
Collaborative corporate environment 40
Highly technology and intellectual property 41
High venture capital investment 42
High risk and costs associated with drug development 42
Identifying fast growth companies- Methodology 44
Purpose and value of the analysis 45
Candidates identified as fast growth companies 46
Chapter 2 Comparitive analyses of growth
strategies 48
Summary 48
The Fastest Growing Biopharmaceutical Companies 49
The evolving nature of the biopharmaceutical industry 56
Comparative analysis of R&D strategies 57
Revenue versus R&D expenditure 57
Early Phase R&D Strategies 60
Late Stage Clinical Development 61
Sponsored Research Payment Strategies 62
Analysis by Therapeutic Area 64
Comparative analysis of commercialization strategies 67
Platform positioning 67
The modern extended biopharmaceutical enterprise 71
Comparative analysis of manufacturing strategies 74
Comparion of restructuring and cost containment strategies 75
Case Study: Downsizing R&D at Trimeris, Inc. 76
Chapter 3 Theravance, Inc. 80
Summary 80
Company address and contact details 81
Company overview and history 81
Business profile 82
Discussion and analysis of financial data 82
Source and details of income 82
Review of proprietary technologies 84
Patents and proprietary rights 84
Strategic approach and positioning 84
Strategic approach 84
SWOT Analysis 85
Strengths 85
Weaknesses 86
Opportunities 86
Threats 87
Joint ventures, collaborations and M&A activity 88
Theravance's collaboration with GlaxoSmithKline (GSK) 88
License, development and commercialization agreement with Astellas 89
License agreement with AstraZeneca AB 89
Product development programs 89
Bacterial infections 89
Telavancin 90
TD-1792 90
Respiratory 91
Horizon program (formerly Beyond Advair) 91
Inhaled bifunctional muscarinic anatgonist-beta2 agonist (MABA)
program 92
Inhaled long-acting muscarinic antagonist (LAMA) program 92
Gastrointestinal motility disorders 93
Research programs 93
Chapter 4 ISTA Pharmaceuticals, Inc. 96
Summary 96
Company Address and Contact Details 97
Company overview and history 97
Business profile 99
Discussion and analysis of financial data 99
Source and details of income 100
Review of proprietary technologies 100
Patents and proprietary rights 101
Strategic approach and positioning 104
Strategic approach 104
SWOT analysis 105
Strengths 105
Weaknesses 105
Opportunities 106
Threats 107
Joint ventures, collaborations and M&A activity 108
Xibrom, Istalol, Bepotastine, Ecabet Sodium, Latanoprost and Iganidipine
collaborations with Senju 108
Vitrase commercialization outside the US 108
Collaboration with Otsuka Pharmaceutical 109
Marketed products 109
Xibrom™ (bromfenac) 109
Istalol® 110
Vitrase® 111
Product development programs 111
T-Pred (tobramycin and prednisolone acetate combination product) 112
Bepotastine 112
Ecabet sodium 113
Strong steroid product 113
Chapter 5 Palatin Technologies Inc 118
Summary 118
Company address and contact details 119
Company overview and history 119
Business profile 120
Discussion and analysis of financial data 120
Source and details of income 121
Review of proprietary technologies 121
Patents and proprietary rights 122
Strategic approach and positioning 124
Strengths 124
Strength in MC expertise 124
Weaknesses 124
Termination of collaborative agreement with King Pharmaceuticals 124
Limited product pipeline portfolio 125
Opportunities 125
Threats 125
Joint ventures, collaborations and M&A activity 126
Strategic alliance with AstraZeneca AB 126
Collaborative development and marketing agreement with King
Pharmaceuticals 126
Strategic collaboration agreement with Mallinckrodt 127
Product development programs 128
Bremelanotide (formerly PT-141) 129
Male sexual dysfunction (MED) 129
Female sexual dysfunction (FSD) 129
Development of novel natriuretic receptor compounds 129
Chapter 6 Pharmion Corporation 134
Summary 134
Company address and contact details 135
Company overview and history 135
Business profile 138
Discussion and analysis of financial data 138
Source and details of income 138
Review of proprietary technologies 139
Patents and proprietary rights 140
Strategic approach and positioning 142
Strengths 142
Vidaza: potential future growth and revenue opportunities 142
Formation of strategic alliances 142
Weaknesses 143
Lack of manufacturing capabilities 143
Opportunities 144
Acquisition by Celgene 144
Inorganic growth 144
FDA approval of Vidaza NDA supplement for IV administration 144
Threats 145
Litigations 145
Intense competition 145
Cost containment pressures 146
Joint ventures, collaborations and M&A activity 147
M&A history 147
Acquisition of Cabrellis Pharmaceuticals 147
Joint ventures and collaborations 148
Marketed products 148
Vidaza (azacitidine for injection) 148
Thalidomide Pharmion 149
Innohep 149
Refludan 150
Product development programs 152
Amrubicin 152
Oral azacitidine 152
Satraplatin 152
MGCD0103 153
Chapter 7 Amylin Pharmaceuticals, Inc. 158
Summary 158
Company address and contact details 159
Company overview and history 159
Business profile 160
Discussion and analysis of financial data 160
Source and details of income 162
Review of proprietary technologies 162
Patents and proprietary rights 163
Strategic approach and positioning 164
Strengths 164
Strategic alliances 164
Byetta 164
New management team 165
Weaknesses 165
Reliance on third party manufacturers 165
Narrow customer concentration 166
Opportunities 167
Exenatide (long acting release) LAR 167
INTO obesity program 167
Threats 168
Competition 168
Joint ventures, collaborations and M&A activity 168
Joint ventures and collaborations 168
Marketed products 169
Byetta (exenatide) injection 169
Symlin 170
Product development programs 173
Diabetes product development 173
Exenatide LAR program 173
Obesity product development program 173
Pramlintide 173
Pramlintide and Leptin 174
Pramlintide PYY 3-36 174
Chapter 8 Trimeris, Inc. 178
Summary 178
Company address and contact details 179
Company overview and history 179
Business profile 181
Discussion, analysis of financial data 181
Details of income 181
Review of proprietary technologies 183
Patents and proprietary rights 183
Strategic approach and positioning 183
Strengths 184
Collaboration with Roche 184
Fuzeon 185
Weaknesses 185
T-1249 on hold 185
Opportunities 186
HIV drugs market 186
TRI-1144 186
Strategic shift in the company’s focus 187
Threats 188
Market acceptance of Fuzeon 188
Intense competition 188
Joint ventures, collaborations and M&A activity 189
Joint ventures and collaborations 189
Marketed products 190
Fuzeon 190
Product development programs 191
FUZEON 191
T-1249 191
Next generation fusion inhibitor peptide drug candidates 191
Chapter 9 ViroPharma Inc. 196
Summary 196
Company address and contact details 197
Company overview and history 197
Business profile 198
Discussion and analysis of financial data 198
Source and details of income 201
Review of proprietary technologies 201
Patents and proprietary rights 201
Strategic approach and positioning 202
Strengths 202
Strategic partnerships 202
Fast track status 203
Weaknesses 204
Lack of manufacturing capabilities 204
Customer concentration 204
Opportunities 205
Camvia™ (maribavir) 205
Threats 205
Dependence of continued sales of Vancocin 205
Joint ventures, collaborations and M&A activity 206
Joint ventures and collaborations 206
Cytomegalovirus and GlaxoSmithKline 206
Hepatitis C and Wyeth 207
Vancocin capsules and Eli Lilly 208
Picornaviruses and Schering-Plough Corporation 208
Marketed products 209
Vancocin 209
Product development programs 209
Camvia™ (maribavir) 209
HCV-796 210
NTCD (non-toxigenic C. difficile) 211
Antiviral discovery 211
Chapter 10 NPS Pharmaceuticals Inc. 214
Summary 214
Company Address and Contact Details 215
Company overview and history 215
Business profile 216
Discussion and analysis of financial data 216
Source and details of income 216
Review of proprietary technologies 217
Patents and proprietary rights 220
Strategic approach and positioning 220
Strategic approach 220
Outsource non-core competencies 220
Build a diversified pipeline of products addressing a variety of
medical conditions 221
Collaborate or out-license to reduce risk and accelerate the
commercialization of select product candidates 221
SWOT analysis 221
Strengths 221
Weaknesses 222
Opportunities 222
Threats 224
Joint ventures, collaborations and M&A activity 225
Joint ventures and collaborations 225
Amgen Inc 225
AstraZeneca 225
GlaxoSmithKline 226
Janssen 226
Kirin Brewery of Tokyo (pharmaceutical division) 226
Nycomed 227
Marketed products 228
Sensipar®/Mimpara® (Cinacalcet HCI) 228
Preotact™ (PREOS®) 228
Product development programs 230
Teduglutide (ALX-0600) 230
Calcilytics 230
Glycine reuptake inhibitors (GlyT-1) 230
Metabotropic glutamate receptors (mGluRs) 231
Chapter 11 SIGA Technologies, Inc. 234
Summary 234
Company address and contact details 235
Company overview and history 235
Business profile 237
Discussion and analysis of financial data 237
Source and details of income 240
Review of proprietary technologies 240
Patents and proprietary rights 241
Strategic approach and positioning 244
SWOT analysis 244
Strengths 244
Weaknesses 244
Opportunities 245
Threats 246
Joint ventures, collaborations and M&A activity 246
Collaborative research and licenses 246
Product development programs 248
Anti-infectives 248
Anti-smallpox drug 248
Other antivirals in development 248
Junín 249
Lassa fever 249
Ebola/Marburg 249
HTS discovery campaign 250
Dengue 250
Bunyavirus 250
Sortase 250
Chapter 12 Idenix Pharmaceuticals, Inc. 254
Summary 254
Company address and contact details 255
Company overview and history 255
Business profile 257
Discussion and analysis of financial data 257
Source and Details of Income 257
Review of proprietary technologies 260
Patents and proprietary rights 260
Strategic approach and positioning 261
Strategic approach 261
Focusing on HCV and HIV drug discovery and development
programs only 261
Discontinuation of the HBV franchise 261
Trimmed workforce 261
SWOT Analysis 262
Strengths 262
Weaknesses 263
Opportunities 263
Threats 264
Joint ventures, collaborations and M&A activity 264
Joint ventures and collaborations 264
Marketed products 266
Tyzeka®/Sebivo® (telbivudine) 266
Product development programs 267
IDX-899 267
IDX184 267
Chapter 13 Appendix 270
Methodology statement 270
Primary Data and Information Gathering 270
Secondary data and information gathering 270
Market share analysis and market forecast predictions 271
Definitions of Product-Life Cycle stages 272
Glossary of abbreviations and acronyms 275
Index 280
List of Figures
Figure 1.1: Evolution of the biopharmaceutical corporation 37
Figure 2.2: Analysis of R&D expenditure v revenues, 2006 59
Figure 2.3: Sponsored Research Payment Strategies 63
Figure 2.4: Sponsored research payment strategies 70
Figure 2.5: The principles of the extended enterprise 71
Figure 5.6: Disease area focus, Palatin Technologies 128
Figure 5.7: Annual product history, Palatin Technologies 130
Figure 7.8: Annual product history, Amylin Pharmaceuticals 171
Figure 7.9: Product pipeline, Amylin Pharmaceuticals 175
Figure 8.10: Annual product history, Trimeris 187
Figure 8.11: Product pipeline, Trimeris 192
Figure 9.12: Annual product history, ViroPharma 199
Figure 10.13: Annual product history, NPS Pharmaceuticals 218
Figure 10.14: Product pipeline, NPS 224
Figure 10.15: Disease area focus, NPS Pharmaceuticals 227
Figure 11.16: Annual product history, SIGA Technologies 238
Figure 11.17: Product pipeline, SIGA Technologies 247
Figure 12.18: Annual product history, Idenix Pharmaceuticals 258
Figure 12.19: Product pipeline, Idenix Pharmaceuticals 266
List of Tables
Table 1.1: Top 20 pharmaceutical companies ranked by revenues (2006) 39
Table 2.2: Summary analysis of the fastest growing biopharmaceutical companies, 2006 ($m) 51
Table 2.3: Fastest growing biopharmaceutical companies by net income/loss from operations,
2002-06 ($m) 52
Table 2.4: The top 20 biopharmaceutical companies by revenue in 2006 ($m) 53
Table 2.5: Summary analysis of fastest growing biopharmaceutical companies 2006 ($m) 54
Table 2.6: Fastest growing biopharmaceutical companies by revenues and % growth, 2002-06
($m) 54
Table 2.7: Fastest growing biopharmaceutical companies by R&D expenditure, 2002-06 ($m) 55
Table 2.8: Company Analysis: Proprietary Technology versus Therapeutic Area 66
Table 2.9: Fastest growing biopharmaceutical companies by revenues and % growth, 2002-06
($m) 69
Table 2.10: Company analysis by approved marketed product 69
Table 3.11: Company address and contact details 81
Table 3.12: Theravance Income Statement, 2002–2006 ($ millions) 83
Table 3.13: Theravance product development programs 94
Table 4.14: Company address and contact details 97
Table 4.15: ISTA Income Statement ($ millions 2002–2006) 102
Table 4.16: ISTA marketed product sales ($ millions 2005–2006) 103
Table 4.17: ISTA product development and pipeline 115
Table 5.18: Company address and contact details 119
Table 5.19: Palatin Income Statement ($ millions) year ended June 30, 2003–2007 123
Table 5.20: Palatin product development and pipeline 131
Table 6.21: Company address and contact details 135
Table 6.22: Pharmion Income Statement ($ millions 2002–2006) 141
Table 6.23: Pharmion marketed product sales ($ millions, 2002–2006) 151
Table 6.24: Pharmion product development and pipeline 155
Table 7.25: Company address and contact details 159
Table 7.26: Amylin Pharmaceuticals Income Statement, ($ millions, 2002–2006) 161
Table 7.27: Amylin Pharmaceuticals Income Statement ($ millions, 2002–2006) 172
Table 7.28: Amylin Pharmaceuticals product development and pipeline 176
Table 8.29: Company address and contact details 179
Table 8.30: Trimeris Income Statement ($ millions 2002–2006) 182
Table 8.31: Trimeris product development and pipeline 193
Table 9.32: Company address and contact details 197
Table 9.33: ViroPharma Income Statement ($ millions, 2002–2006) 200
Table 9.34: ViroPharma product development and pipeline 212
Table 10.35: Company address and contact details 215
Table 10.36: NPS Pharmaceuticals Income Statement ($ millions 2002–2006) 219
Table 10.37: Marketed products 220
Table 10.38: Marketed products 229
Table 10.39: NPS Pharmaceuticals product development and pipeline 232
Table 11.40: Company address and contact details 235
Table 11.41: SIGA Technologies Income Statement ($ millions, 2002–2006) 239
Table 11.42: SIGA Technologies- issued patents as of December 31, 2006 242
Table 11.43: SIGA Technologies provisional patents and patent applications as of Dec 31, 2006 243
Table 11.44: SIGA Technologies product development and pipeline 252
Table 12.45: Company address and contact details 255
Table 12.46: Idenix Pharmaceuticals Income Statement ($ millions, 2002–2006) 259
Table 12.47: Idenix Pharmaceuticals product development and pipeline 268