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1. RNAi for Therapeutic Applications:
Technology and Market 2014-2024
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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
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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
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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
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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
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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
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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
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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
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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 ($m, AGR %, CAGR %), 2013-2024: Optimistic Scenario
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10. Contents
Table 4.18 QPI-1002: Revenue Forecast ($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
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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
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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 ($m), 2013-2024:
Optimistic and Pessimistic Scenarios
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13. Contents
Figure 5.6 The BRIC RNAi Therapeutics Market: Revenue Forecast s($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
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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
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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)
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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
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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
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18. Contents
University of Massachusetts Medical School
VIRxSYS
World Health Organization (WHO)
Zeltia Group
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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
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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
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