Global Huntington's Disease Therapeutics Industry

LONDON, May 6, 2015 /PRNewswire/ -- This report analyzes the worldwide markets for Huntington's Disease Therapeutics in US$ Million. The report provides separate comprehensive analytics for the US, Europe, and Rest of World. Annual estimates and forecasts are provided for the period 2014 through 2020. Also, a five-year historic analysis is provided for these markets. Market data and analytics are derived from primary and secondary research. Company profiles are primarily based on public domain information including company URLs. The report profiles 25 companies including many key and niche players such as -

Alnylam Pharmaceuticals, Inc.
Auspex Pharmaceuticals, Inc.
Intellect Neurosciences Incorporation
Lundbeck A/S
Prana Biotechnology Limited



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I. INTRODUCTION, METHODOLOGY & PRODUCT DEFINITIONS

Study Reliability and Reporting Limitations I-1
Disclaimers I-2
Data Interpretation & Reporting Level I-2
Quantitative Techniques & Analytics I-3
Product Definitions and Scope of Study I-3


II. EXECUTIVE SUMMARY

1. INDUSTRY OVERVIEW II-1
A Prelude II-1
Current HD Treatment II-1
Huntington's Disease - Quick Facts II-2
Market Opportunity II-3
Huntington's Disease Prevalence in Major Region/Countries II-3
Competition in the Global HD Therapeutics Market is Non-existent II-3
Effectiveness of Current HD Management Strategies II-4
HD Treatment and Drugs: Brief Description of Disorder,
Medication and Side-Effects II-5
Alternative Treatment Approaches for Huntington's Disease II-5
Alternate Approaches and Therapies II-6
Off-Label Prescriptions II-6
Select Off-Label Drugs Prescribed for Huntington's Disease:
Drug Name & Manufacturer; Treatment Class; and Primary
Indication II-6
mHtt Aggregates in HD—Molecular Pathogenesis II-7

2. HD THERAPEUTICS MARKET TRENDS II-8
Therapeutics Focus on Reduction of mHtt Expression II-8
Companies Boost R&D Spending for HD Therapeutics II-8
Improved Diagnostics Fueling Interest of Companies in HD Research II-8
Support Organizations Play an Instrumental Role in HD Research
and Awareness II-9
Public-Private Partnerships: A Shot in the Arm II-9
HD Research Attracts 'Big Pharma' Investment II-9
Pre-Symptomatic Patients to Expand Market Base II-10
HDSA Pushes for Improvement in Medicare Funding for
Huntington's Disease II-10
GINA for Reducing Discrimination Faced by People with HD II-11

3. HUNTINGTON'S DISEASE PIPELINE ANALYSIS II-12
A Quick Primer II-12
Drug Development Pipeline Reveals a Promising Picture II-12
Select Drugs and Supplements in HD Research Pipeline (2014):
Drug Name, Manufacturer, Mode of Action and Current Status II-12
An Overview of Select Drugs in Late-Stage Clinical Trials II-13
SD-809 II-13
RP103 (Procysbi) II-13
PBT2 II-13
Huntexil™ (Pridopidine) II-14
Other Novel Approaches for HD Treatment II-15
Cellular Delivery of Neurotropic Agents II-15
Cogane II-15
COPREXA II-16
Histone Deacetylases (HDAC) II-16
Melatonin, Coenzyme Q-Free Radical Scavengers II-16
RNAi - RNA Interference II-16

4. RESEARCH BREAKTHROUGHS IN HD ARENA II-17
Triglyceride Oil 'Triheptanoin' Shows Promise for HD Therapeutics II-17
Reducing mTORC1 Activation through Drug/Diet May be Helpful II-17
Better Understanding of Early Neuronal Changes Lead to
Targeted Drug Development II-17
Lund University Researchers Successfully Map Functionality of
Hypothalamus in HD II-17
MIB, University of Lisbon and University of Leicester
Demonstrate Latest Findings on KMO Enzyme II-18
Latest TRACK-HD Findings Demonstrate Early Identification of
Huntington's Disease Progression in Patients II-18
Researchers at BUSM, MGH, Columbia University and McLean BTRC
Demonstrate Impact of Huntington's Disease on Human Brain II-18
Researchers Demonstrate Significance of TFEB and PGC-1alpha
Proteins in Prevention of HD II-19
Florey Neuroscience Institutes Demonstrates Importance of
Cognitive Stimulation in HD Patients II-19
University of Wisconsin-Madison Waisman Center Demonstrates
Stem Cell Therapy Potential for Treating HD II-20
NUI Galway Announces Discovery of Experimental Drugs with
Potential for Slowing Progress of HD II-20
Lawrence Berkeley National Laboratory Designs Compound for
Suppressing HD Symptoms II-20
Development of HD Affected Human Brain Cells for Investigative
Purposes II-21
Device Implant in Brain Exhibits Therapeutic Potential for HD II-21
Sangamo Biosciences on Path to Discover a Therapy for HD II-22
Scientists at University of Rochester Medical Center
Demonstrate Use of Coenzyme Q10 for Reduction of Oxidative
Damage in HD Patients II-22

5. AN OVERVIEW OF HUNTINGTON'S DISEASE II-23
Introduction II-23
Historical Background II-23
Prognosis II-24
Stages of HD II-24
Symptoms and Signs II-25
Physical Symptoms II-25
Emotional Symptoms II-26
Mental Symptoms II-26
Genetics of Huntington's Disease II-26
Inheritance II-27
Mechanism II-27
Functions of HTT II-28
Effects of mHTT on Cells II-28
Macroscopic Effects of mHTT II-28
Diagnosis of HD II-29
Clinical Diagnosis II-29
Embryonic Diagnosis II-30
Differential Diagnosis II-30
Diagnostic Tests for Huntington's Disease II-30
Confirmatory Diagnostic Test II-30
Pre-symptomatic Diagnostic Test II-30
Prenatal Diagnostic Test II-30
Treatment Options II-30
Nutrition and Eating II-31
Social Activity II-31
Speech Therapy II-31
Physical Therapy II-31
Occupational Therapy II-32
Medication II-32
Molecular Chaperones II-32

6. RECENT INDUSTRY ACTIVITY II-34
Auspex Reports Favorable Study Results of SD-809 II-34
Sangamo and Collaborators to Present ZFP Therapeutics® Data II-34
Sangamo Presents Positive Preclinical Data of ZFP Therapeutic® II-34
Sangamo BioSciences Acquires Ceregene II-34
Prana Completes Reach2HD Trial II-34
Teva Pharmaceuticals Forms Global R&D Collaboration for HD II-35
KineMed, Isis and CHDI Foundation Collaborate for HD Therapeutics II-35
CHDI Foundation Continues Collaboration with Medtronic II-35
Roche Enters into Alliance with Isis Pharmaceuticals II-35
Omeros Receives IND Approval for OMS824 for Huntington's Disease II-35
Teva Pharmaceutical Industries Signs Asset Transfer Agreement
with NeuroSearch II-35
uniQure and Benitec Biopharma Ink Cross-Licensing Agreement II-35
Evotec and CHDI Foundation Extend Collaboration II-36
Raptor Acquires Exclusive Rights to Cysteamine and Associated
Compounds II-36
GNS Healthcare Enters into Collaboration with CHDI Foundation II-36
Lundbeck Collaborates with CHDI Foundation for HD Research II-36
Horizon Discovery Collaborates with Institute of Neurology,
London II-36
Thomson Reuters and CHDI Foundation Enter into MetaMiner
Partnership II-36

7. FOCUS ON SELECT PLAYERS II-37
Alnylam Pharmaceuticals, Inc. (US) II-37
Auspex Pharmaceuticals, Inc. (US) II-37
Intellect Neurosciences Incorporation (US) II-37
Lundbeck A/S (Denmark) II-38
Prana Biotechnology Limited (Australia) II-38
Raptor Pharmaceuticals Corp. (US) II-38
Sangamo Biosciences, Inc. (US) II-39
Teva Pharmaceutical Industries Ltd (Israel) II-39
Trophos SA (France) II-39
Valeant Pharmaceuticals International, Inc. (Canada) II-40
Vertex Pharmaceuticals Incorporated (US) II-40

8. GLOBAL MARKET PERSPECTIVE II-41
Table 1: World Recent Past, Current & Future Analysis for
Huntington's Disease Therapeutics by Geographic Region - US,
Europe, and Rest of World Markets Independently Analyzed with
Annual Sales Figures in US$ Million for the Years 2014 through
2020 (includes corresponding Graph/Chart) II-41

Table 2: World Historic Review for Huntington's Disease
Therapeutics by Geographic Region - US, Europe, and Rest of
World Markets Independently Analyzed with Annual Sales Figures
in US$ Million for the Years 2009 through 2013 (includes
corresponding Graph/Chart) II-42

Table 3: World 12-Year Perspective for Huntington's Disease
Therapeutics by Geographic Region - Percentage Breakdown of
Dollar Sales for US, Europe, and Rest of World Markets for the
Years 2009, 2015 & 2020 (includes corresponding Graph/Chart) II-43


III. MARKET

1. THE UNITED STATES III-1
A.Market Analysis III-1
HDSA Brings about Improved Medicare Funding for Huntington's
Disease III-1
GINA for Reducing Discrimination Faced by People with HD III-1
Strategic Corporate Developments III-2
Select Players III-4
B.Market Analytics III-6
Table 4: The US Recent Past, Current & Future Analysis for
Huntington's Disease Therapeutics Analyzed with Annual Sales
Figures in US$ Million for the Years 2014 through 2020
(includes corresponding Graph/Chart) III-6

Table 5: The US Historic Review for Huntington's Disease
Therapeutics Analyzed with Annual Sales Figures in US$
Million for the Years 2009 through 2013 (includes
corresponding Graph/Chart) III-7

2. EUROPE III-8
Market Analysis III-8
Table 6: European Recent Past, Current & Future Analysis for
Huntington's Disease Therapeutics by Geographic Region -
France, Germany, UK, Spain and Rest of Europe Markets
Independently Analyzed with Annual Sales Figures in US$
Million for the Years 2014 through 2020 (includes
corresponding Graph/Chart) III-8

Table 7: European Historic Review for Huntington's Disease
Therapeutics by Geographic Region - France, Germany, UK,
Spain and Rest of Europe Markets Independently Analyzed with
Annual Sales Figures in US$ Million for the Years 2009
through 2013 (includes corresponding Graph/Chart) III-9

Table 8: European 12-Year Perspective for Huntington's
Disease Therapeutics by Geographic Region - Percentage
Breakdown of Dollar Revenues for France, Germany, UK, Spain
and Rest of Europe Markets for the Years 2009, 2015 & 2020
(includes corresponding Graph/Chart) III-10

2a. FRANCE III-11
A.Market Analysis III-11
Trophos Sa - A Key Player III-11
B.Market Analytics III-12
Table 9: French Recent Past, Current & Future Analysis for
Huntington's Disease Therapeutics Analyzed with Annual Sales
Figures in US$ Million for the Years 2014 through 2020
(includes corresponding Graph/Chart) III-12

Table 10: French Historic Review for Huntington's Disease
Therapeutics Analyzed with Annual Sales Figures in US$
Million for the Years 2009 through 2013 (includes
corresponding Graph/Chart) III-13

2b. GERMANY III-14
A.Market Analysis III-14
Strategic Corporate Development III-14
B.Market Analytics III-14
Table 11: German Recent Past, Current & Future Analysis for
Huntington's Disease Therapeutics Analyzed with Annual Sales
Figures in US$ Million for the Years 2014 through 2020
(includes corresponding Graph/Chart) III-14

Table 12: German Historic Review for Huntington's Disease
Therapeutics Analyzed with Annual Sales Figures in US$
Million for the Years 2009 through 2013 (includes
corresponding Graph/Chart) III-15

2c. THE UNITED KINGDOM III-16
A.Market Analysis III-16
Outlook III-16
Table 13: Juvenile HD Incidence Estimates in the
UK(includes corresponding Graph/Chart) III-16

Table 14: Juvenile HD Prevalence Estimates in the UK
(2006-2010) (includes corresponding Graph/Chart) III-16
Strategic Corporate Development III-17
B.Market Analytics III-17
Table 15: The UK Recent Past, Current & Future Analysis for
Huntington's Disease Therapeutics Analyzed with Annual Sales
Figures in US$ Million for the Years 2014 through 2020
(includes corresponding Graph/Chart) III-17

Table 16: The UK Historic Review for Huntington's Disease
Therapeutics Analyzed with Annual Sales Figures in US$
Million for the Years 2009 through 2013 (includes
corresponding Graph/Chart) III-18

2d. SPAIN III-19
A.Market Analysis III-19
KEY PLAYER - SOM BIOTECH III-19
B.Market Analytics III-20
Table 17: Spanish Recent Past, Current & Future Analysis for
Huntington's Disease Therapeutics Analyzed with Annual Sales
Figures in US$ Million for the Years 2014 through 2020
(includes corresponding Graph/Chart) III-20

Table 18: Spanish Historic Review for Huntington's Disease
Therapeutics Analyzed with Annual Sales Figures in US$
Million for the Years 2009 through 2013 (includes
corresponding Graph/Chart) III-21

2e. REST OF EUROPE III-22
A.Market Analysis III-22
Strategic Corporate Development III-22
B.Market Analytics III-23
Table 19: Rest of Europe Recent Past, Current & Future
Analysis for Huntington's Disease Therapeutics Analyzed with
Annual Sales Figures in US$ Million for the Years 2014
through 2020 (includes corresponding Graph/Chart) III-23

Table 20: Rest of Europe Historic Review for Huntington's
Disease Therapeutics Analyzed with Annual Sales Figures in
US$ Million for the Years 2009 through 2013 (includes
corresponding Graph/Chart) III-24

3. REST OF WORLD III-25
A.Market Analysis III-25
Strategic Corporate Developments III-25
Select Players III-25
B.Market Analytics III-27
Table 21: Rest of World Recent Past, Current & Future
Analysis for Huntington's Disease Therapeutics Analyzed with
Annual Sales Figures in US$ Million for the Years 2014
through 2020 (includes corresponding Graph/Chart) III-27

Table 22: Rest of World Historic Review for Huntington's
Disease Therapeutics Analyzed with Annual Sales Figures in
US$ Million for the Years 2009 through 2013 (includes
corresponding Graph/Chart) III-28


IV. COMPETITIVE LANDSCAPE

Total Companies Profiled: 25

The United States (13)
Canada (1)
Europe (8)
- France (1)
- Germany (1)
- The United Kingdom (2)
- Italy (1)
- Rest of Europe (3)
Asia-Pacific (Excluding Japan) (3)

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