For an Executive Summary of this report please contact ediz.ibrahim@visiongain.com (+44 (0)20 7549 9976) or refer to our website https://www.visiongain.com/Report/1284/Epigenetic-R-D-Therapies-and-Diagnostics-Industry-and-Market-Prospects-2014-2024

1. Epigenetic R&D, Therapies and
Diagnostics: Industry and Market
Prospects 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. www.visiongain.com
Contents
1. Executive Summary
1.1 Epigenetic Therapies, Technologies and Diagnostics: World Market Review
1.2 Global Epigenetics Market Segmentation
1.3 Why You Should Read This Report
1.4 How This Report Delivers
1.5 Main Questions Answered by This Analytical Report
1.6 Who is This Report For?
1.7 Methods of Research and Analysis
1.8 Frequently Asked Questions (FAQ)
1.9 Associated Reports
1.10 About Visiongain
2. Introduction to Generic Drugs
2.1 How is Epigenetics Defined?
2.1.1 Epigenetic Mechanisms
2.1.2 A Brief History of Epigenetic Research
2.2 The Links between Epigenetics and Disease
2.2.1 The Role of Lifestyle in Epigenetics
2.2.2 Epigenetics and Cancer
2.2.3 Epigenetics in Inherited Disorders
2.2.4 Epigenetics in Neurological Diseases
2.3 Epigenetics in Disease Diagnosis
2.3.1 Diagnosing for Breast Cancer
2.3.2 Colorectal Cancer: The Importance of Early Diagnosis
1. Report Overview
2. Introduction to Epigenetics and Uses in Pharma

3. www.visiongain.com
Contents
2.3.3 Diagnostics for Lung Cancer: Difficulty in Accessing Samples
2.3.4 Diagnostics for Prostate Cancer
2.4 The Use of Epigenetics in Treating Diseases
2.4.1 DNA Methlytransferase (DNMT) Inhibitors
2.4.1.1 Myelodysplastic Syndrome (MDS)
2.4.2 Histone Deacetylase (HDAC) Inhibitors
2.4.2.1 Cutaneous T Cell Lymphoma (CTCL)
2.4.2.2 Peripheral T Cell Lymphoma (PTCL)
2.5 Drug Discovery and Development Tools Using Epigenetics
2.5.1 Bisulphite Screening for DNA Methylation
2.5.2 Chromatin Immunoprecipitation (ChIP) for Histone Modification
3. Generic Drugs: World Market 2014-2024
3.1 The Global Epigenetic Therapies, Technologies and Diagnostics Market in 2013
3.1.1 Leading Segments of the Market in 2013
3.2 The Epigenetic Therapies, Technologies and Diagnostics Global Market 2014-2024
3.2.1 The Epigenetic Therapies, Technologies and Diagnostics Global Market: Revenue
Forecasts 2014-2024
3.2.2 What Will Expedite the Growth of Epigenetic Therapies, Technologies and Diagnostics
Market?
3.2.3 Increase in Epigenetic Diagnostics Market Share 2014-2024
3.3 Epigenetic Therapies Submarket 2014-2024
3.3.1 Epigenetic Therapies Submarket: Revenue Forecast 2014-2024
3. The Global Epigenetic Therapies, Technologies and Diagnostics
Market 2014-2024

4. www.visiongain.com
Contents
3.3.2 Rising Importance of Epigenetic Therapies Revenue 2014-2024
3.4 Epigenetic Technologies Submarket 2014-2024
3.4.1 Epigenetic Technologies Submarket: Revenue Forecast 2014-2024
3.4.2 Epigenetic Technologies Submarket: Drivers and Restraints 2014-2024
3.5 Epigenetic Diagnostics Submarket 2014-2024
3.5.1 Epigenetic Diagnostics Submarket: Revenue Forecast 2014-2024
3.5.2 Growth Driving Factor: New Product Launches 2014-2024
4. Leading Generic Drug Manufacturers
4.1 Leading National Markets for Epigenetic Therapies, Technologies and Diagnostics 2013
4.2 Leading National Markets: Revenue Forecasts 2014-2024
4.2.1 Global Market Will Be Dominated by the Western Countries 2014-2024
4.3 The Northern American Market 2014-2024
4.3.1 The US: The World’s Largest Market for the Epigenetic Therapies, Technologies and
Diagnostics 2014-2024
4.3.2 Canada: Revenue Forecast 2014-2024
4.4 European Union (EU) Epigenetics Market: Revenues 2013
4.4.1 Leading National Markets in EU: Revenue Forecasts 2014-2024
4.4.2 Germany: Revenue Forecast 2014-2024
4.4.3 The UK: A Leading Country for Epigenetic Research
4.5 Japanese Market: Increased Interest in Epigenetics
4.6 Epigenetics in Emerging National Markets
4. Leading National Epigenetic Therapies, Technologies and
Diagnostics Market 2014-2024

5. www.visiongain.com
Contents
4.6.1 BRIC Nations: Positive Growth Expected in Epigenetic Therapies, Technologies and
Diagnostics Markets 2014-2024
5. US Generics Market 2014-2024
5.1 The Future of the Epigenetic Therapies Market 2014-2024
5.2 Vidaza (Celgene Co. / Nippon Shinyaku): The First Therapy in the Epigenetic Market
5.2.1 Vidaza: Leading Epigenetic Therapy 2004-2012
5.2.2 How Was Vidaza Affected by Patent Expiry?
5.2.3 Will Oral Azacitidine and Combination Therapy Stimulate Vidaza Growth?
5.2.4 Vidaza: Revenue Forecast 2014-2024
5.3 Dacogen (Eisai/ Janssen-Cilag/ Otsuka Pharmaceutical Co.)
5.3.1 Dacogen: Historical Revenue Performance 2006-2012
5.3.2 EU Approval and Combination Therapy to Drive Sales Growth?
5.3.3 Will Oral Azacitidine and Combination Therapy Drive Vidaza Growth?
5.3.4 Dacogen: Revenue Forecast 2014-2024
5.4 Zolinza (Merck & Co./ Taiho Pharmaceutical)
5.4.1 Zolinza for Multiple Myeloma Treatment
5.4.2 Zolinza Revenue Forecast 2014-2024
5.5 Istodax: Second Epigenetic Drug by Celgene Co.
5.5.1 Istodax: Strong Revenue Growth 2010-2011
5.5.2 International Market Approvals to Drive Growth?
5.5.3 Istodax: Revenue Forecast 2014-2024
5.6 Epigenetic Therapy Market 2013: Rich Pipeline for HDAC Inhibitors
5.6.1 Beleodaq (TopoTarget/ Spectrum Pharmaceuticals): A Novel HDAC Inhibitor
5. Epigenetic Therapies Market 2014-2024

6. www.visiongain.com
Contents
5.6.2 Faridak (Novartis): Treatment for Velcade-Refractory Multiple Myeloma
5.6.3 Entinostat (Syndax Pharmaceuticals, Inc.): Breast and Lung Cancer
5.6.4 Resminostat (4SC/ Yakult Honsha Co.): Hepatocellular Carcinoma
5.6.5 Pracinostat (MEI Pharma): Treatment for MDS and AML
5.6.6 Mocetinostat: Initiatives of Mirati Therapeutics to Drive Development
5.6.7 Quisinostat (Janssen Pharmaceutica, NV/ NewVac LLC)
5.6.8 Rocilinostat (Acetylon Pharmaceuticals): One of Celgene’s Keen Interest
5.6.9 CHR-3396 (Chroma Therapeutics)
5.6.10 Givinostat (Italfarmaco): Chronic Myeloproliferative Neoplasms and Duchenne
Muscular Dystrophy
5.6.11 Shape (TetraLogic Pharmaceuticals):The First Topical HDAC inhibitor for CTCL
5.6.12 FRM-0334 (Forum Pharmaceuticals Inc): Neurodegenerative Diseases
5.6.13 4SC-202: Second Epigenetic Drug by 4SC Has Potential for Wider Indications
5.6.14 Tefinostat (Chroma Therapeutics): Haematological Cancer
5.6.15 CDX10 (Celleron Therapeutics): Part of Personalised Cancer Treatment Trial
5.6.16 RG2833 (The Scripps Research Institute): Friedreich’s Ataxia
5.6.17 Abexinostat (Pharmacyclics)
5.7 New Discoveries in the Field of HDAC 2013
5.7.1 Chemogenic Approaches to Spatiotemporal Regulation of HDAC Activity
5.7.2 Novel Lysine Pathways and Acetylation-Independent Mechanisms of HDACs
5.7.3 HDAC Inhibitor: Treatment of Pathological Muscle Remodelling
5.7.4 Design of Class I Isoform Selective Inhibitors for Use in Metabolic Indications
5.7.5 Zinc Binding Groups Inhibit Class IIa HDAC and Alters Immune Responses
5.8 DNMT Inhibitor R&D Pipeline 2013

7. www.visiongain.com
Contents
5.8.1 SGI-110 (Otsuka Pharmaceuticals): A Second Generation DNMT Inhibitor
5.8.2 FdCyd (National Cancer Institute): Solid Tumours
5.8.3 Hydralazine: Being Tested in 23 Clinical Trials
5.8.4 MG98 (Mirati Therapeutics)
5.8.5 CP-4200: A Second Generation Vidaza
5.8.6 RX3117 (Rexahn Pharmaceuticals)
5.9 Pipeline Treatments Targeting Other Epigenetic Enzymes 2013
5.9.1 EPZ-5676 (Epizyme Inc./ Celgene Co.): The First Clinical-Stage HMT Inhibitor
5.9.2 EPZ-5676 (Epizyme Inc./ Eisai): The Second Clinical-Stage HMT Inhibitor
5.9.3 ORY-1001(Roche): Orphan Drug Status in Early Stage AML
5.9.4 SP-2528 (Salarius Pharmaceuticals)
5.9.5 RVX-208 (Resverlogix): Advancing BET Protein Inhibitors
5.10 MiRNA Therapy Pipeline 2013
5.10.1 Miravirsen (Santaris Pharma A/S): The First miRNA Therapy to Enter Human Clinical
Trial
5.10.2 MRX34 (Mirna Therapeutics)
5.10.3 Collaborations of Regulus Therapeutics with Big Pharma Players
5.11 Leading Epigenetic Therapy Manufacturers and Developers
5.11.1 Celgene: Market Leader in 2013
5.11.2 Eisai
5.11.3 Janssen-Cilag
5.11.4 Otsuka Pharmaceuticals
5.11.5 Merck & Co.
5.11.6 TopoTarget to Merge with BioAlliance Pharma SA

8. www.visiongain.com
Contents
5.11.7 Syndax Pharmaceuticals
5.11.8 4SC
5.11.9 Curis: Focusing on Multi-Targeted Treatments
5.11.10 CellCentric: Collaborating with Big Pharma for Drug Development
5.11.11 Constellation Pharmaceuticals
5.11.12 EpiTherapeutics
5.11.13 Epizyme: Signed Multiple Collaboration Agreements with Big Pharma
5.11.14 IkerChem: HDAC and DNMT Inhibitors in Cancer and CNS Disorders
5.11.15 Karus Therapeutics: HDAC Inhibitors for Inflammatory Diseases and Cancer
6.
6.1 Epigenetic Technologies and Services Market 2014-2024
6.1.1 Should Epigenetic Discovery and Analysis Be Outsourced?
6.2 Next Generation Sequencing (NGS)
6.2.1 Higher Speed and Reduced Costs for Epigenetic Research
6.2.2 Are Current Bioinformatic Programmes a Setback?
6.2.3 Single Molecule Sequencing to Compete with NGS
6.3 The Future of Chromatin Immunoprecipitation
6.3.1 ChIP-Seq
6.4 Future Developments in Bisulphite Screening
6.4.1 Importance of Techniques to Distinguish 5hmC
6.5 High Throughput Screening
6.6 Leading Providers of Epigenetics Technologies 2013
6.6.1 Active Motif
6. Epigenetic Technologies: Markets 2014-2024

9. www.visiongain.com
Contents
6.6.2 Measurement Tools for Epigenetic Research by Agilent Technologies
6.6.3 Diagenode: Best –In-Class Methyl Binding Domain Based Kit
6.6.4 DiscoveRx
6.6.5 EMD Millipore
6.6.6 Epigenetek: Specialist in Epigenetic Technologies
6.6.7 Epiontis: Assessing Purity in Regenerative Medicine Products
6.6.8 Life Technologies: Reducing NGS Process Cost
6.6.9 Qiagen: Epigenetic Technologies and Diagnostics
6.6.10 Roche
7. Leading Asia-Pacific Generics Markets 2014-2024
7.1 Development of the Epigenetic Diagnostics Market 2014-2024
7.1.1 LDTs vs. IVDs: Two Diagnostics Development Strategies
7.2 Epigenetic Diagnostics Market 2013
7.2.1 Epi proColon 2.0 (Epigenomics)
7.2.2 ColoVantage (Quest Diagnostics)
7.2.3 Septin9 LDT (ARUP Laboratories)
7.2.4 ColoSure (LabCorp)
7.2.5 ConfirmMDx for Prostate Cancer
7.2.6 Gene Methylation in Prostate Cancer (LabCorp)
7.2.7 Epi proLung (Epigenomics)
7.2.8 MGMT Gene Methylation Assay (LabCorp)
7.2.9 PredictMDx (MDxHealth): Brain Cancer
7.2.10 Bladder Cancer Testing with CertNDx (Physician’s Choice Laboratory Services)
7. Epigenetic Diagnostics Market 2014-2024

10. www.visiongain.com
Contents
7.3 Epigenetic Diagnostics R&D Pipeline 2013
7.3.1 The Future of Colorectal Cancer Testing
7.3.1.1 Cologuard: Improved Sensitivity
7.3.1.2 Orion Colorectal Cancer Risk Test (Orion Genomics)
7.3.2 Pipeline of Prognostic Tools for Prostate Cancer
7.3.3 Lung Cancer
7.3.3.1 Lung Cancer Screening Test (Orion Genomics)
7.3.3.2 ConfirmMDx and InformMDx (MDxHealth)
7.3.4 The Future of Bladder Cancer Testing
7.3.4.1 Bladder Cancer Screening Test (Orion Genomics)
7.3.5 Other Cancer Tests Pipeline 2013
7.3.5.1 Pancreatic Cancer Prognostic Test (PrognosticDx Health)
7.4 Leading Epigenetic Diagnostic Developers 2013
7.4.1Epigenomics: Multiple Tests Marketed in the EU
7.4.1.1 Positive Revenue Growth 2012-2013
7.4.2 Exact Sciences: Colorectal Cancer Screening
7.4.3 MDxHealth: Targeting the US Diagnostics Markets
7.4.3.1 Strong Revenue Growth 2009-2013
7.4.4 Orion Genomics: More than 100 Markers Have Been Discovered
7.4.5 Physician’s Choice Laboratory Services (PCLS)
7.4.6 PrognosDx Health: Developing Epigenetic Prognostics
8. Research and Product Development Strategies in Epigenetics 2014

11. www.visiongain.com
Contents
8.1 Epigenetics Therapies, Technologies and Diagnostics Market by Disease 2014-2024
8.2 Present and Future Epigenetic Research in Cancer
8.2.1 Twin Studies Reveal Epigenetic Biomarkers in Breast Cancer
8.2.2 Colorectal Cancer: More Than Two Biomarkers Established
8.2.3 Lung Cancer: Prognostic Markers
8.2.4 Prostate Cancer: The Need for Progression in Epigenetic Therapies
8.3 The Role of DNA Hypomethylation in Autoimmune Diseases
8.3.1 Lupus
8.3.2 Rheumatoid Arthritis as a Target for Drugs and Diagnostics
8.3.2.1 Ignyta: Pioneer in Epigenetic Diagnostic for Rheumatoid Arthritis
8.3.3 Prevalence of Diabetes linked to Diet and Ageing
8.4 Neurodegenerative Diseases
8.4.1 Alzheimer’s Disease: Symptoms Improved With HDAC Inhibitors
8.4.2 Huntington’s Disease: Epigenetic Drugs in Clinical Development
8.5 Neurological Disorders
8.5.1 Drug Resistance in Schizophrenia
8.5.2 Hundreds of Genetic and Epigenetic Mutations linked to Autism
8.6 New Epigenetic Therapy Targets
8.6.1 BET Proteins Targeted by Multiple Therapeutic Areas
8.6.2 Importance of DNA Hydroxymethylation
8.6.3 Sirtuins: Undergoing Limited Research
8.6.4 Histone Methyltranferase
8.6.4.1 Lysine Methyltranferase
8.6.4.2 Domainex: Development of HMT Inhibitor

12. www.visiongain.com
Contents
8.6.4.3 Protein Arginine Methyltransferases: The Need for Improved Potency
8.6.4.4 Lysine Demethylases: Potential Cancer Treatment Target
9. Qualitative Analysis of the Generic Drugs Market 2014-2024
9.1 Epigenetic Therapies, Technologies and Diagnostics: Strengths and Weaknesses 2014-2024
9.1.1 Understanding Epigenetic Markers and Mechanisms
9.1.2 Specificity of Marketed Epigenetic Therapies
9.2 Epigenetics Therapies, Technologies and Diagnostics Market Opportunities and Threats
2014-2024
9.2.1 The Important Role of Translational Research
9.2.2 Development of Companion Diagnostics in Conjunction with Therapies
9.2.3 Rich Therapeutic and Diagnostic Pipelines
9.2.4 Combination Therapy with Epigenetic Drugs Show Promise
9.2.5 Epigenetic Drugs to Succeed Drug Resistance
9.2.6 Therapy Target Scope besides HDAC and DNMT Inhibition
9.3 Epigenetics Therapies, Technologies and Diagnostics: STEP Analysis 2014-2024
9.4 Social Factors Affecting Epigenetic Product Development
9.4.1 The Influence of Environment
9.4.2 The Role of Epigenetics in Ageing and Diseases
9.4.2.1 Increasing Prevalence of Global Disease to Drive Epigenetics Market Growth
9.4.3 Targeting Orphan Diseases and Patient Subpopulations
9.4.4 Use of Epigenetics in Personalised and Stratified Medicine
9.5 Advancing Epigenetic Technologies to Support Epigenetic Research
9. Epigenetic Therapies, Diagnostics and Related Technologies:
Industry and Market Trends 2014-2024

13. www.visiongain.com
Contents
9.5.1 NGS: Improving The Quality of Epigenetic Research
9.5.2 Epigenetics to Help Drive Stem Cell Research Development
9.5.3 Complementary and Competing Forces
9.5.3.1 Genomics
9.5.3.2 Proteomics
9.5.3.3 Metabolomics
9.6 Economic Factors
9.6.1 Recent Developments Helped Reduce Costs in Epigenetic Research
9.6.2 Importance of Big Pharma in Epigenetic Markets 2014-2024
9.6.2.1 Pfizer
9.6.2.2 Novartis
9.6.2.3 GSK: Part of Multiple Agreements in Epigenetics
9.6.3 Valuable Licensing Deals in Epigenetics
9.6.4 Epigenetic Business Models
9.7 Political Issues
9.7.1 Ethics in Epigenetic Research
9.7.2 Global Collaboration to Expedite Market Growth
9.7.2.1 Structural Genome Consortium
9.7.2.2 Roadmap Epigenomics Project (NIH)
9.7.2.3 International Human Epigenome Consortium
9.7.2.4 Blueprint Project
10.1 Interview with Dr Eric Marcotte, Strategic Lead, Canadian Epigenetics, Environment and
Health Research Consortium (CEEHRC)
10. Research Interview

14. www.visiongain.com
Contents
10.1.1 Canadian Institute of Health Research: Interest in Epigenetics
10.1.2 Canadian Epigenetic Therapy Market
10.1.3 Prominent Epigenetic Therapeutic Areas in the Future
10.1.4 Promising New Epigenetic Therapy and Diagnostics
10.1.5 Future of Canadian Epigenetics Market
10.1.6 CIHR and JST Partnership Outcome
11. Conclusions
11.1 Therapies Submarket Accounts for Most of the Epigenetic Market Revenues in 2013
11.2 Growth Expected in All Submarkets of the Epigenetic Market 2014-2024
11.2.1 Developed National Pharma Markets to Lead, 2014-2024
11.3 Many Opportunities Exist in the Epigenetics Industry and Market
11. Conclusions from the Research and Analysis

15. www.visiongain.com Page 83
Epigenetic R&D, Therapies and Diagnostics:
Industry and Market Prospects 2014-2024
of $143m by the middle of the forecast period (Table 4.5 and Figure 4.7). By 2024, the market will
be worth $257m, having grown with a CAGR of 11.3% since 2013.
Table 4.5 Epigenetic Therapies, Technologies and Diagnostics: Canadian Market Size
($m) Forecast, 2013-2024
2013 2014 2015 2016 2017 2018
Canadian Market Size ($m) 79 85 93 107 123 143
Annual Growth (%) 7 9 15 15 16
CAGR (%) 2013-2018 12.5
2019 2020 2021 2022 2023 2024
Canadian Market Size ($m) 163 184 210 231 245 257
Annual Growth (%) 14 13 14 10 6 5
CAGR (%) 2018-2024 10.2
CAGR (%) 2013-2024 11.3
Figure 4.7 Epigenetic Therapies, Technologies and Diagnostics: Canadian Market Size
($m) Forecast, 2013-2024
4.4 European Union (EU) Epigenetics Market: Revenues 2013
Europe - for the purposes of this reported considered to be the EU - was the second largest region
in the world epigenetic therapies and technologies market in 2013, behind North America. In that
0
50
100
150
200
250
300
2013 2014 2015 2016 2017 2018 2019 2020 2021 2022 2023 2024
MarketSize($m)
Year
Source: visiongain 2014
Source: visiongain 2014

16. www.visiongain.com Page 114
Epigenetic R&D, Therapies and Diagnostics:
Industry and Market Prospects 2014-2024
A MAA was submitted to the EMA in November 2007 for this indication. However, in October 2008,
the EMA’s Committee for Medicinal Products for Human Use (CHMP) adopted a negative opinion
on Zolinza, stating that data provided by Merck was not sufficient to approve the drug. In February
2009, Merck announced that it was withdrawing its application based on the opinion of the CHMP.
In July 2011, Zolinza was approved for use in CTCL in by the Ministry of Health, Labour and
Welfare (MHLW) in Japan, where it will be marketed by Taiho Pharmaceutical part of Otsuka
Holdings Co., Ltd. The drug is currently approved in the US, Canada, Japan and Australia for
similar indication. The patent expiry of Zolinza is scheduled for July 2015.
5.4.1 Zolinza for Multiple Myeloma Treatment
Revenue growth for Zolinza during the period 2014-2024 will be driven by approvals in new
regions, including the EU, and in new indications, such as multiple myeloma. In the US, 21,000
people were reported with multiple myeloma in 2013 (Table 5.10). Zolinza was granted orphan
drug status for that indication in the EU in April 2011. Orphan status has also been granted for
treating multiple myeloma in the US. Zolinza has already been shown to be effective in
combination with Velcade, with phase III results being released in December 2011. Patients in the
trial treated with a combination of Velcade and Zolinza were found to have a 23% lower risk of
disease progression than patients treated with Velcade on its own. Interim results from a phase II
trial had previously shown that Zolinza is effective in treating patients who are unresponsive to
treatment with Velcade. Currently, there are three phase III clinical trials investigating the efficacy
of vorinostat in treating multiple myeloma via combination therapy (Table 5.11).
The success in multiple myeloma contrasts with failures in other indications, visiongain notes. In
September 2011, Merck announced that a trial of the drug in mesothelioma had failed to meet its
primary endpoint of improving overall survival, for example. Beyond cancer, Zolinza has shown
efficacy in treating other diseases. Researchers from the University of North Carolina found that
the drug was effective in clearing latent HIV from patients receiving other treatments. The study
found that the HDAC inhibitor activates the disease within patients, which is then cleared by
regular HIV therapies. The researchers deduced that the vorinostat forces the virus to become
active, allowing the antiretroviral therapy the participants were already taking to do their job and
destroy the virus. They are continuing to investigate the study which is now in phase II and is
expected to finish in 2016. Merck & Co. and National Institute of Allergy and Infectious Disease
(NIAID) are collaborating with the researchers from the University of North Carolina in the study.

17. www.visiongain.com Page 126
Epigenetic R&D, Therapies and Diagnostics:
Industry and Market Prospects 2014-2024
Figure 5.16 HDAC Inhibitor Expected Approvals, 2015-2020
5.6.1 Beleodaq (TopoTarget/ Spectrum Pharmaceuticals): A Novel Pan-
HDAC Inhibitor
Beleodaq (belinostat, PXD-101) is a novel pan-HDAC inhibitor developed by TopoTarget. The drug
is primarily being developed for PTCL, although clinical trials are underway for many other cancer
indications. For a number of these TopoTarget is partnered with the US National Cancer Institute
(Table 5.17). In October 2012, Beleodaq was awarded orphan drug status in the EU for its primary
indication, guaranteeing it 10 years market exclusivity upon approval. Orphan drug designation has
also been granted in the US. The pivotal study of Beleodaq for the treatment of relapsed or
refractory (R/R) PTCL was closed for recruitment in September 2011 after the inclusion of 129
patients. Final data presented at the American Society for Clinical Oncology (ASCO) annual
meeting in 2013 showed an objective response rate (ORR) of 26% in all PTCL patients, 28% in
PTCL patients with platelet counts above 100,000/µL and 45.5% in patients with the PTCL subtype
angioimmunoblastic T-cell lymphoma (AITL). Safety data presented at the T-Cell Lymphoma
Forum in 2013 showed a favourable safety profile of Beleodaq when compared to the approved
treatments for patients with PTCL and it was emphasized that combining Beleodaq with cytotoxic
regimens is likely feasible. Beleodaq appears to have low myelosuppression and even patients
Source: visiongain 2014