RNAi for Therapeutic Applications:
Technology and Market 2014-2024

©notice
This material is copyright by visiongain. It i...
Contents
1. Executive Summary
1.1 RNAi Therapeutics: World Market Review
1.2 Chapter Breakdown
1.3 Research and Analysis M...
Contents
3. Leading Therapeutic Applications for RNAi
3.1 Therapeutic Fields Stimulating the RNAi Therapeutics Market to 2...
Contents
4.8 SPC3649 (Miravirsen): Revenue Forecast, 2014-2024: Revolutionary Treatment Targeting All
Six HCV Subtypes?
4....
Contents
6. Leading Companies in the RNAi Therapeutics Market, 2014
6.1 Alnylam Pharmaceuticals
6.1.1 Profile
6.1.2 A Stro...
Contents
6.5.1 Profile
6.5.2 Locked Nucleic Acid (LNA) Platform: How Much Potential?
6.5.3 Sales and Recent Performance
6....
Contents
7.1.2 Lower Therapeutic Dosages Will Attract Patients
7.1.3 Shorter Lead Identification Times: Providing Advantag...
Contents
8.1.1 Sylentis
8.1.1.1 The Current Status of The RNAi Therapeutics Market
8.1.1.2 Challenges in RNAi Drug Develop...
Contents
List of Tables
Table 2.1 Advantages and Disadvantages of Using Chemically Modified Synthetic siRNAs
Table 2.2 Adv...
Contents
Table 4.18 QPI-1002: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024: Pessimistic
Scenario
Table 4.19 FANG: Reven...
Contents
Table 5.9 BRIC Nations’ RNAi Therapeutics Markets: Revenue Forecasts ($m, AGR %, CAGR %)
by Country, 2013-2024: O...
Contents
List of Figures
Figure 2.1 The Basic Process of RNAi
Figure 2.2 Model of RNA Interference: Cleavage Dependent Mec...
Contents
Figure 5.6 The BRIC RNAi Therapeutics Market: Revenue Forecast s($m) by Country, 2013-2024:
Optimistic Scenarios
...
Contents
Companies and Other Organisations Mentioned in This Report
Acuity Pharmaceuticals
Aegera Therapeutics
Agilent Tec...
Contents
Bristol-Myers Squibb
Calando Pharmaceuticals
Carnegie Institution for Science
Chinese University of Hong Kong
Cub...
Contents
Halo-Bio RNAi Therapeutics
Integrated DNA Technologies
Intradigm Corporation
Invitrogen
Isarna Therapeutics
ISIS ...
Contents
Quark Biotech
Quark Pharmaceuticals
Regado Biosciences
Regeneron Pharmaceuticals
Ribopharma AG
RiboTask
RXi Pharm...
Contents
University of Massachusetts Medical School
VIRxSYS
World Health Organization (WHO)
Zeltia Group

www.visiongain.c...
RNAi for Therapeutic Applications:
Technology and Market 2014-2024
(PKN3) - a protein which mediates malignant cell growth...
RNAi for Therapeutic Applications:
Technology and Market 2014-2024
other well-validated and known cancer drug targets. In ...
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RNAi for Therapeutic Applications: Technology and Market 2014-2024

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RNAi for Therapeutic Applications: Technology and Market 2014-2024

  1. 1. RNAi for Therapeutic Applications: Technology and Market 2014-2024 ©notice This material is copyright by visiongain. It is against the law to reproduce any of this material without the prior written agreement of visiongain. You cannot photocopy, fax, download to database or duplicate in any other way any of the material contained in this report. Each purchase and single copy is for personal use only.
  2. 2. Contents 1. Executive Summary 1.1 RNAi Therapeutics: World Market Review 1.2 Chapter Breakdown 1.3 Research and Analysis Methods 1.4 Scope of this Report 2. Introduction to RNA Interference (RNAi): Technologies and Uses 2.1 Ribonucleic Acid (RNA): From DNA to Protein 2.2 RNA Interference (RNAi): A Natural Cellular Gene Silencing Process 2.2.1 The Endogenous Process of RNAi: The RNAi Machinery 2.2.2 RNAi Inducers: The Role of siRNA and miRNA 2.2.2.1 Short or Small Interfering RNA (siRNA): Application in Genetic Diseases 2.2.2.2 microRNA (miRNA): Natural Gene Expression Regulators 2.3 Timeline of RNAi: From Discovery to Human Trials in Six Years 2.4 The Therapeutic Potential of RNAi 2.4.1 Development of RNAi-Based Therapeutic Agents 2.4.1.1 The Delivery Approach: Exogenous Introduction of siRNA 2.4.1.2 The Expressed/DNA-directed RNAi (ddRNAi) Approach: A Stable Expression of RNAi Inducers 2.4.1.2.1 Short Hairpin RNA (shRNA): Employing Viral Vectors 2.4.2 Ribopharma AG: The First Company Specialising in RNAi Therapeutics www.visiongain.com
  3. 3. Contents 3. Leading Therapeutic Applications for RNAi 3.1 Therapeutic Fields Stimulating the RNAi Therapeutics Market to 2024 3.2 Ocular Disease – Many Needs Remain to Benefit Patients 3.2.1 Age-related Macular Degeneration (AMD) 3.2.2 Glaucoma – an Eye Disorder Benefiting from Technological Advances 3.3 Lung Diseases and Respiratory Disorders – Life Improving Treatments 3.3.1 Respiratory Syncytial Virus (RSV) Infection 3.4 Infectious Diseases – Protecting and Saving Lives Through Medical Progress 3.4.1 Hepatitis B and C Virus (HBV and HCV) Infections 3.5 Kidney Disease – Investigations to Treat the Excretory System 3.5.1 Delayed Graft Function (DGF) 3.6 Cancer – Prominent Field of Medicine Holds Great Scope for New Therapies 4. The RNAi Therapeutics Market 2014-2024 4.1 Scope and Limitations 4.2 The RNAi Therapeutics Market Forecast, 2014-2024 4.2.1 The RNAi Therapeutics Market: Industry Trends, 2014-2024 4.3 PF-655: Revenue Forecast, 2014-2024: Novel Mode of Action 4.4 QPI-1007: Revenue Forecast, 2014-2024: Unique Structure Reduces Risk of Off-Target Effects 4.5 SYL040012: Revenue Forecast, 2014-2024: Improved Efficacy Over Market-Leading Drug 4.6 ALN-RSV01: Revenue Forecast, 2014-2024: First RNAi to Demonstrate Therapeutic Efficacy 4.7 ARC-520: Revenue Forecast, 2014-2024: Rapid Reduction in Viral Antigen an Industry First www.visiongain.com
  4. 4. Contents 4.8 SPC3649 (Miravirsen): Revenue Forecast, 2014-2024: Revolutionary Treatment Targeting All Six HCV Subtypes? 4.9 QPI-1002: Revenue Forecast, 2014-2024: Orphan Designation Offers Market Exclusivity 4.10 FANG: Revenue Forecast, 2014-2024: Personalised Therapy Targeting Multiple Cancer Subtypes 4.11 ALN-VSP02: Revenue Forecast, 2014-2024: Dual-Target Action Adds to Therapeutic Potential 4.12 Atu027: Revenue Forecast, 2014-2024: High Prospects for Combination Therapy 4.13 CALAA-01: Revenue Forecast, 2014-2024: Demonstrating a RNAi Mechanism 5. Leading National RNAi Therapeutics Markets 2014-2024 5.1 Scope and Limitations 5.2 Leading National Markets: Revenue Forecasts, 2014-2024 5.2.1 The US: The Leading Market for RNAi Therapeutics 5.2.2 The EU5 RNAi Therapeutics Market: Pivotal Region for Companies 5.2.2.1 RNAi Therapeutics in the EU5 Submarkets, 2014-2024 5.2.3 Japanese RNAi Therapeutics Market, 2014-2024: Aging Population to Raise Demand for New Therapies 5.2.4 The Potential for RNAi Therapeutics in Emerging National Markets, 2014-2024 5.2.4.1 China Steps Up Efforts in RNAi Drug Development 5.2.4.2 India: Company Collaborations to Stimulate Interest in RNAi 5.2.4.3 Brazil: Economic Growth Helps Drug Discovery Efforts 5.2.4.4 Russia: Strong Market Environment Encourages R&D www.visiongain.com
  5. 5. Contents 6. Leading Companies in the RNAi Therapeutics Market, 2014 6.1 Alnylam Pharmaceuticals 6.1.1 Profile 6.1.2 A Strong Portfolio in the RNAi Field 6.1.3 Sales and Recent Performance, 2012 6.1.4 Entering Into Strategic Alliances to Generate Revenues 6.1.5 Contract Manufacturing of siRNAs by Tekmira Pharmaceuticals 6.1.6 Future Growth Strategy – Commercial Outlook 6.2 Silence Therapeutics 6.2.1 Profile 6.2.2 Lead Candidate Atu027 Expected to Complete Phase II in 2014 6.2.3 Multiple Delivery Technologies Offers Promise 6.2.4 Sales and Recent Performance, 2012 6.2.5 Partnerships for Silence Therapeutics’ RNAi Platform 6.3 RXi Pharmaceuticals 6.3.1 Profile 6.3.2 RXI-109: Lead RNAi Candidate Preventing Scar Formation 6.3.3 Sales and Recent Performance, 2012 6.4 Quark Pharmaceuticals 6.4.1 Profile 6.4.2 A Broad siRNA Clinical Pipeline 6.4.3 Quark Partnerships to Develop and Promote RNAi Therapies 6.5 Santaris Pharma www.visiongain.com
  6. 6. Contents 6.5.1 Profile 6.5.2 Locked Nucleic Acid (LNA) Platform: How Much Potential? 6.5.3 Sales and Recent Performance 6.5.4 Targeting Rare Genetic Diseases Through Shire plc Alliance 6.6 Tekmira Pharmaceuticals Corporation 6.6.1 Profile 6.6.2 Therapeutic R&D Pipeline Based on Novel Drug Delivery System 6.6.3 Sales and Recent Performance, 2012 6.6.4 Out-Licensing Technology Adds Extra Source of Revenue 6.7 Arrowhead Research Corporation 6.7.1 Profile 6.7.2 Company Focus: A Nanoparticle-Based Drug Delivery Platform 6.7.3 Sales and Recent Performance, 2012 6.7.4 Roche Acquisition Strengthens Company’s Market Position 6.8 Sylentis 6.8.1 Profile 6.8.2 The First Company in Spain to Conduct Clinical Trial of an RNAi Medicine 6.9 Gradalis 6.9.1 Overview 6.9.2 Company Pipeline: Vaccine based on shRNA in Clinical Trials 7. RNAi Therapeutics: Industry Trends 2014-2024 7.1 RNAi Therapeutics: Strengths and Weaknesses, 2014-2024 7.1.1 Therapeutic Targeting: A Highly Selective Treatment www.visiongain.com
  7. 7. Contents 7.1.2 Lower Therapeutic Dosages Will Attract Patients 7.1.3 Shorter Lead Identification Times: Providing Advantage over Other Approaches 7.1.4 Manufacturing Synthetic RNAs: Cost Effective and Easier Than for Other Biologics 7.1.5 The Big Challenge of Drug Delivery 7.1.5.1 Poor Cellular Uptake and Low Stability of siRNAs in Blood Serum 7.1.5.2 Systemic and Targeted Delivery of RNAi-based Drug Candidates 7.1.5.3 Industry’s Approach to Overcome the Introduction Challenges 7.1.6 Off-target Binding, Immune Response and Oversaturation Elevates Risk of Side Effects 7.2 RNAi Therapeutics: Opportunities and Threats, 2014-2024 7.2.1 Treating Multifactorial Diseases: A Revolutionary Therapeutic? 7.2.2 Competing Technologies 7.2.2.1 Antisense Technology: First-to-Market Advantage 7.2.2.2 Aptamers: Chemical Antibodies with Little Effect on Immune Activation 7.2.3 Regulatory Threats: Novel Class of Treatment May Lead to Prolonged or Delayed Approval Processes 7.2.4 Large Scale Production of RNAs: A Potential Threat to Commercialisation? 7.3 RNAi Therapeutics: STEP Analysis, 2014-2024 7.3.1 Social Forces 7.3.2 Technological Developments 7.3.3 Economic Pressures 7.3.4 Political Issues 8. Research Interviews 8.1 Respondent From Sylentis www.visiongain.com
  8. 8. Contents 8.1.1 Sylentis 8.1.1.1 The Current Status of The RNAi Therapeutics Market 8.1.1.2 Challenges in RNAi Drug Development 8.1.1.3 Sylentis’ RNAi Candidates and Platform Technology 8.1.1.4 The Future Outlook for RNAi Therapeutics 8.2 Dr Geert Cauwenbergh, RXi Pharmaceuticals 8.2.1 RXi Pharmaceuticals 8.2.1.1 The Potential of RNAi Therapeutics 8.2.1.2 Company History and OPKO Health Partnership 8.2.1.3 RXi’s Self-Delivering RNAi Technology 8.2.1.4 RX-109: RNAi Candidate for Post-Surgical Scarring 8.2.1.5 Prospects for the RNAi Therapeutics Market 9. Conclusions from Our Study 9.1 The RNAi Therapeutics Market in 2013 9.2 Outlook for The RNAi Therapeutics Market, 2014-2024 9.2.1 Regional Outlook for the Market, 2014-2024 9.3 Future Opportunities in RNAi Therapeutics 9.4 Challenges and Potential for Success Exist Appendices About Visiongain’s Bespoke Research Service Appendix A: About Visiongain Appendix B: Visiongain Report Evaluation Form www.visiongain.com
  9. 9. Contents List of Tables Table 2.1 Advantages and Disadvantages of Using Chemically Modified Synthetic siRNAs Table 2.2 Advantages of ddRNAi over siRNA, 2014 Table 4.1 RNAi Therapeutics in Clinical Development, 2013 Table 4.2 World RNAi Therapeutics Market: Revenue Forecasts ($m, AGR %, CAGR %) for Leading Drugs, 2013-2024: Optimistic Scenario Table 4.3 World RNAi Therapeutics Market: Revenue Forecasts ($m, AGR %, CAGR %) for Leading Drugs, 2013-2024: Pessimistic Scenario Table 4.4 The RNAi Therapeutics Market: Drivers and Restraints, 2014-2024 Table 4.5 PF-655: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024: Optimistic Scenario Table 4.6 PF-655: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024: Pessimistic Scenario Table 4.7 QPI-1007: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024: Optimistic Scenario Table 4.8 QPI-1007: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024: Pessimistic Scenario Table 4.9 SYL040012: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024: Optimistic Scenario Table 4.10 SYL040012: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024: Pessimistic Scenario Table 4.11 ALN-RSV01: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024: Optimistic Scenario Table 4.12 ALN-RSV01: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024: Pessimistic Scenario Table 4.13 ARC-520: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024: Optimistic Scenario Table 4.14 ARC-520: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024: Pessimistic Scenario Table 4.15 SPC3649 (Miravirsen): Revenue Forecast ($m, AGR %, CAGR %), 2013-2024: Optimistic Scenario Table 4.16 SPC3649 (Miravirsen): Revenue Forecast ($m, AGR %, CAGR %), 2013-2024: Pessimistic Scenario Table 4.17 QPI-1002: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024: Optimistic Scenario www.visiongain.com
  10. 10. Contents Table 4.18 QPI-1002: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024: Pessimistic Scenario Table 4.19 FANG: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024: Optimistic Scenario Table 4.20 FANG: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024: Pessimistic Scenario Table 4.21 ALN-VSP02: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024: Optimistic Scenario Table 4.22 ALN-VSP02: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024: Pessimistic Scenario Table 4.23 Atu027: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024: Optimistic Scenario Table 4.24 Atu027: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024: Pessimistic Scenario Table 4.25 CALAA-01: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024: Optimistic Scenario Table 4.26 CALAA-01: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024: Pessimistic Scenario Table 5.1 The RNAi Therapeutics Market: Revenue Forecasts ($m, AGR %, CAGR %) by Region, 2013-2024: Optimistic Scenario Table 5.2 The RNAi Therapeutics Market: Revenue Forecasts ($m, AGR %, CAGR %) by Region, 2013-2024: Pessimistic Scenario Table 5.3 The US RNAi Therapeutics Market: Revenue Forecast ($m, AGR %, CAGR %), 20132024: Optimistic Scenario Table 5.4 The US RNAi Therapeutics Market: Revenue Forecast ($m, AGR %, CAGR %), 20132024: Pessimistic Scenario Table 5.5 The EU5 RNAi Therapeutics Market: Revenue Forecast ($m, AGR %, CAGR %) by Country, 2013-2024: Optimistic Scenario Table 5.6 The EU5 RNAi Therapeutics Market: Revenue Forecast ($m, AGR %, CAGR %) by Country, 2013-2024: Pessimistic Scenario Table 5.7 The Japanese RNAi Therapeutics Market: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024: Optimistic Scenario Table 5.8 The Japanese RNAi Therapeutics Market: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024: Pessimistic Scenario www.visiongain.com
  11. 11. Contents Table 5.9 BRIC Nations’ RNAi Therapeutics Markets: Revenue Forecasts ($m, AGR %, CAGR %) by Country, 2013-2024: Optimistic Scenario Table 5.10 BRIC Nations’ RNAi Therapeutics Markets: Revenue Forecasts ($m, AGR %, CAGR %) by Country, 2013-2024: Pessimistic Scenario Table 6.1 Alnylam Pharmaceuticals’ RNAi Drug Pipeline, 2013 Table 6.2: Alnylam Pharmaceuticals: Revenue From Collaborations ($,’000), 2011 and 2012 Table 6.3 Silence Therapeutics’ RNAi Drug Pipeline, 2013 Table 6.4 Silence Therapeutics: Revenues ($m) by Region, 2011 & 2012 Table 6.5 Quark Pharmaceuticals’ RNAi Drug Pipeline, 2013 Table 6.6 Santaris’ RNAi Drug Pipeline, 2013 Table 6.7 Santaris Pharma: Revenue ($m), 2010 and 2011 Table 6.8 Tekmira Pharmaceuticals’ RNAi Drug Pipeline, 2013 Table 6.9 Tekmira Pharmaceuticals: Revenue ($m), 2011 and 2012 Table 6.10 Arrowhead Research Corporations’ RNAi Drug Pipeline, 2013 Table 6.11 Arrowhead Research Corporation: Revenue ($m, CAGR %), 2009-2013 Table 6.12 Sylentis’ RNAi Drug Pipeline, 2013 Table 6.13 Gradalis’ RNAi Vaccine and Drug Pipeline, 2013 Table 7.1 The RNAi Therapeutics Market: Strengths and Weaknesses, 2014-2024 Table 7.2 Leading Contract Manufacturing Organisations Developing Oligonucleotide APIs, 2013 Table 7.3 Advantages of Local Delivery Over Systemic Delivery, 2014 Table 7.4 Leading Companies Developing RNAi Delivery Systems and Technologies, 2013 Table 7.5 The RNAi Therapeutics Market: Opportunities and Threats, 2014-2024 Table 7.6 Leading Companies Developing Antisense Drugs, 2013 Table 7.7 Leading Companies Developing Aptamer Drugs, 2013 Table 9.1 World RNAi Therapeutics Market: Overall Revenue Forecasts ($m, Three-Year Growth %, CAGR %) for Leading Drugs, 2012, 2015, 2018, 2021 and 2024 www.visiongain.com
  12. 12. Contents List of Figures Figure 2.1 The Basic Process of RNAi Figure 2.2 Model of RNA Interference: Cleavage Dependent Mechanism Figure 4.1 World RNAi Therapeutics Market: Revenue Forecasts ($m), 2013-2024: Optimistic and Pessimistic Scenario Figure 4.2 PF-655: Revenue Forecast ($m), 2013-2024: Optimistic and Pessimistic Scenarios Figure 4.3 QPI-1007: Revenue Forecast ($m), 2013-2024: Optimistic and Pessimistic Scenarios Figure 4.4 SYL040012: Revenue Forecast ($m), 2013-2024: Optimistic and Pessimistic Scenarios Figure 4.5 ALN-RSV01: Revenue Forecast ($m), 2013-2024: Optimistic and Pessimistic Scenarios Figure 4.6 ARC-520: Revenue Forecast ($m), 2013-2024: Optimistic and Pessimistic Scenarios Figure 4.7 SPC3649 (Miravirsen): Revenue Forecast ($m), 2013-2024: Optimistic and Pessimistic Scenarios Figure 4.8 QPI-1002: Revenue Forecast ($m), 2013-2024: Optimistic and Pessimistic Scenarios Figure 4.9 FANG: Revenue Forecast ($m), 2013-2024: Optimistic and Pessimistic Scenarios Figure 4.10 ALN-VSP02: Revenue Forecast ($m), 2013-2024: Optimistic and Pessimistic Scenarios Figure 4.11 Atu027: Revenue Forecast ($m), 2013-2024: Optimistic and Pessimistic Scenarios Figure 4.12 CALAA-01: Revenue Forecast ($m), 2013-2024: Optimistic and Pessimistic Scenarios Figure 5.1 The US RNAi Therapeutics Market: Revenue Forecast ($m), 2013-2024: Optimistic and Pessimistic Scenarios Figure 5.2 The EU5 RNAi Therapeutics Market: Revenue Forecast ($m), 2013-2024: Optimistic and Pessimistic Scenarios Figure 5.3 The EU5 RNAi Therapeutics Market: Revenue Forecasts ($m) by Country, 2013-2024: Optimistic Scenarios Figure 5.4 The EU5 RNAi Therapeutics Market: Revenue Forecasts ($m) by Country, 2013-2024: Pessimistic Scenarios Figure 5.5 The Japanese RNAi Therapeutics Market: Revenue Forecast ($m), 2013-2024: Optimistic and Pessimistic Scenarios www.visiongain.com
  13. 13. Contents Figure 5.6 The BRIC RNAi Therapeutics Market: Revenue Forecast s($m) by Country, 2013-2024: Optimistic Scenarios Figure 5.7 The BRIC RNAi Therapeutics Market: Revenue Forecast s($m) by Country, 2013-2024: Pessimistic Scenarios Figure 6.1 Alnylam Pharmaceuticals: Revenue Share (%) from Collaborations, 2012 Figure 6.2 Silence Therapeutics: Revenue Share (%) by Region, 2012 Figure 6.3 Santaris Pharma: Revenue ($m), 2010 and 2011 Figure 6.4 Arrowhead Research Corporation: Revenue ($m), 2009-2013 Figure 9.1 World RNAi Therapeutics Market: Overall Revenue Forecasts ($m), 2012, 2015, 2018, 2021 and 2024: Optimistic and Pessimistic Scenarios www.visiongain.com
  14. 14. Contents Companies and Other Organisations Mentioned in This Report Acuity Pharmaceuticals Aegera Therapeutics Agilent Technologies Alcon Allele Biotechnology Allergan Alnylam Pharmaceuticals Altogen Labs Antisense Therapeutics Antisoma Research Archemix Corporation ArmaGen Technologies Arrowhead Research Corporation Ascletis Pharmaceuticals AstraZeneca Atlantic Pharmaceuticals Atugen AG Avecia OligoMedicines AVI BioPharma Bayer Bio Synthesis Biocon Biogen Idec Biomatik Corporation Biomics Biotechnologies Bionexus BioSpring www.visiongain.com
  15. 15. Contents Bristol-Myers Squibb Calando Pharmaceuticals Carnegie Institution for Science Chinese University of Hong Kong Cubist Pharmaceuticals Dainippon Sumitomo Pharma Dharmacon RNAi Technologies Eleos Eli Lilly Enzo Therapeutics Enzon Pharmaceuticals Eurofins MWG-Biotech Eurogentec European Commission European Medicines Agency (EMA) European Union (EU) Expression Genetics Eyetech Pharmaceuticals EZBiolab Food and Drug Administration (US FDA) Gene Link Gene Signal International GeneCare Research Institute GeneDesign Genentech Genta Incorporated Genzyme Girindus America Gradalis GlaxoSmithKline (GSK) www.visiongain.com
  16. 16. Contents Halo-Bio RNAi Therapeutics Integrated DNA Technologies Intradigm Corporation Invitrogen Isarna Therapeutics ISIS Pharmaceuticals Kyowa Hakko Kirin Life Technologies Lorus Therapeutics Marina Biotech Mary Crowley Cancer Research Centre Max Planck Institute MedImmune Medtronic Merck & Co. Merz Pharmaceuticals Nitto Denko Corporation Novartis NovaRx Corporation Novosom Noxxon Pharma Ohio State University (OSU) Oligoengine OncoGenex Technologies Ophthotech Corporation OPKO Health OSI Pharmaceuticals Pfizer Protiva Biotherapeutics Qiagen www.visiongain.com
  17. 17. Contents Quark Biotech Quark Pharmaceuticals Regado Biosciences Regeneron Pharmaceuticals Ribopharma AG RiboTask RXi Pharmaceuticals Roche SABiosciences Samyang Corporation Santaris Pharma Sanofi Shire Sigma Aldrich Silence Therapeutics Sirnaomics Sirna Therapeutics Spanish Society for Glaucoma Solvay Group Suzhou Ribo Life Sciences Sylentis Talon Pharmaceuticals Takeda Targeted Genetics Corporation Tekmira Pharmaceuticals Teva Pharmaceuticals The Medicines Company Thermo Scientific Traversa Therapeutics University of Bayreuth www.visiongain.com
  18. 18. Contents University of Massachusetts Medical School VIRxSYS World Health Organization (WHO) Zeltia Group www.visiongain.com
  19. 19. RNAi for Therapeutic Applications: Technology and Market 2014-2024 (PKN3) - a protein which mediates malignant cell growth. Pre-clinical studies have demonstrated that Atu027 works by inhibiting blood supply to solid tumours. Combined with other currently marketed anti-cancer drugs, Atu027 has demonstrated additive effects. First-in-human studies involving Atu027 showed the treatment was well tolerated and no dose-dependent toxicities were observed. There was also evidence for anti-tumour efficacy of Atu-027, with the treatment leading to one patient with highly advanced neuroendocrine cancer reporting disease stabilisation for 9 months, and another patient with partial regression of pulmonary metastases. In June 2013, Atu027 became the first RNAi therapeutic to officially enter phase II clinical development for the treatment of pancreatic cancer, which assessed the safety and efficacy of Atu027 in combination with gemcitabine, a chemotherapy agent. The company plans to initiate further studies with Atu027 in two other separate cancer indications (head and neck cancer and breast cancer), later in 2014. Atu027 has a novel metastatic effect in preventing the spread of cancer cells into and out of blood vessels. It is this feature - visiongain believes - that makes Atu027 a promising clinical candidate as it can potentially be used - if approved - in a combination regimen with an antineoplastic agent such as gemcitabine (successful outcomes in any of the trials described above will strongly support this proposal). Ultimately, this will pave the way for a large scale maintenance therapy for cancer, thereby drawing the attention of big pharma companies such as Novartis and Pfizer (if they are looking to move into RNAi Therapeutics). This presents Silence Therapeutics with partnering opportunities in the development and commercialisation of Atu027. Also, by targeting pancreatic cancer, the company is investigating treatments for an indication for which there is a high unmet medical need. In these optimistic circumstances, visiongain predicts revenues for Atu027 will reach $614.9 in 2024 (Table 4.23). Table 4.23 Atu027: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024: Optimistic Scenario 2012 Revenue ($m) 2013 2014 2015 2016 2017 2018 0.0 0.0 0.0 0.0 0.0 92.1 134.9 Annual Growth (%) 46 2019 2020 2021 2022 2023 2024 Revenue ($m) 192.8 258.5 345.8 432.7 514.9 614.9 Annual Growth (%) 43 34 34 25 19 19 CAGR (%, 2017-2024) 31.2 Source: visiongain 2014 Conversely, Atu027 is considered a risk as little is known about the biology of the PKN3 target. This may turn out to be a disadvantage, especially when rival companies are probably investigating www.visiongain.com Page 66
  20. 20. RNAi for Therapeutic Applications: Technology and Market 2014-2024 other well-validated and known cancer drug targets. In this case, Atu027 may not transpire to be as successful for the company as expected, reaching revenues of only $284.8 in 2024 (Table 4.24). Table 4.24 Atu027: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024: Pessimistic Scenario 2012 Revenue ($m) 2013 2014 2015 2016 2017 2018 0.0 0.0 0.0 0.0 0.0 40.1 62.7 Annual Growth (%) 56 2019 2020 2021 2022 2023 2024 Revenue ($m) 89.4 124.4 164.4 200.9 244.9 284.8 Annual Growth (%) 43 39 32 22 22 CAGR (%, 2017-2024) 16 32.3 Source: visiongain 2014 Figure 4.11 illustrates both optimistic and pessimistic revenue growth forecasts for Atu027 from 2014-2024. Figure 4.11 Atu027: Revenue Forecast ($m), 2013-2024: Optimistic and Pessimistic Scenarios Optimistic Pessimistic 800.0 Revenue ($m) 600.0 400.0 200.0 0.0 Year Source: visiongain 2014 www.visiongain.com Page 67

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