Global Huntington’s Disease Therapeutics Industry

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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 2009 through 2018. Also, a six-year historic analysis is provided for these markets .

The report profiles 35 companies including Alnylam Pharmaceuticals, Inc., AmpliPhi Biosciences Corp., Ceregene, Inc., Cortex Pharmaceuticals, Inc., Intellect Neurosciences Incorporation, Prana Biotechnology Limited, Raptor Pharmaceuticals Corp., Teva Pharmaceutical Industries Ltd., Trophos SA, Valeant Pharmaceuticals International, Inc., and Vertex Pharmaceuticals Incorporated. 

Market data and analytics are derived from primary and secondary research. Company profiles are primarily based on public domain information including company URLs.

Table of Contents

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
Prelude II-1
Huntington’s Disease – Few Quick Facts II-1
Market Opportunity II-2
Competition in the Global HD Therapeutics Market is Nonexistent II-3
Effectiveness of Current HD Management Strategies II-3
HD Treatment and Drugs II-4
Alternative Treatment Approaches for Huntington’s disease II-5
Alternate Approaches and Therapies II-5
Off-Label Prescriptions II-5
Select Off-Label Drugs Prescribed for Huntington’s Disease II-6

2. HD THERAPEUTICS MARKET TRENDS II-7
Companies Boost R&D Spending for HD Therapeutics II-7
Improved Diagnostics Fueling Interest of Companies in HD Research II-7
Support Organizations Play an Instrumental Role in HD Research
and Awareness II-7
Public-Private Partnerships: A Shot in the Arm II-8
HD Research Attracts ‘Big Pharma’ Investment II-8
Pre-Symptomatic Patients to Expand Market Base II-8
HDSA Pushes for Improvement in Medicare Funding for
Huntington’s Disease II-9
GINA for Reducing Discrimination Faced by People with HD II-9

3. HD RESEARCH AND DRUG PIPELINE II-11
A Quick Primer II-11
Drug Development Pipeline Reveals Promising Picture II-11
Select Drugs and Supplements in HD Research Pipeline (2011) II-11
Overview of Select Pipeline Drugs II-13
Huntexil™ (Pridopidine) II-13
RP103 (DR Cysteamine) II-13
PBT2 II-14
Other Novel Approaches for HD Treatment II-14
Cellular Delivery of Neurotropic Agents II-14
Cogane II-14
COPREXA II-15
Histone Deacetylases (HDAC) II-15
Melatonin, Coenzyme Q-Free Radical Scavengers II-15
RNAi – RNA Interference II-15
Small Molecule Inhibitor of Polyglutamine Interaction II-16
TELOMICS™ II-16

4. RESEARCH BREAKTHROUGHS IN HD ARENA 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-17
Latest TRACK-HD Findings Demonstrate Early Identification of
Huntington’s Disease Progression in Patients II-17
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-18
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-19
NUI Galway Announces Discovery of Experimental Drugs with
Potential for Slowing Progress of HD II-19
Lawrence Berkeley National Laboratory Designs Compound for
Suppressing HD Symptoms II-20
Development of HD Affected Human Brain Cells for Investigative
Purposes II-20
Device Implant in Brain Exhibits Therapeutic Potential for HD II-20
Sangamo Biosciences on Path to Discover a Therapy for HD II-21
Scientists at University of Rochester Medical Center
Demonstrate Use of Coenzyme Q10 for Reduction of Oxidative
Damage in HD Patients II-21
McMaster University Discovers New Drug Candidate for
Huntington’s Disease II-22
University of Central Florida Researchers Test Role of DRP1
Protein in HD II-22
CMMT and Brain Research Centre Decode Cellular Mechanism
Related to Alteration of Brain Cell Function in HD II-23

5. AN OVERVIEW OF HUNTINGTON’S DISEASE II-24
Introduction II-24
Historical Background II-24
Prognosis II-25
Stages of HD II-25
Symptoms and Signs II-26
Physical Symptoms II-26
Emotional Symptoms II-27
Mental Symptoms II-27
Genetics of Huntington’s Disease II-27
Inheritance II-28
Mechanism II-28
Functions of HTT II-29
Effects of mHTT on Cells II-29
Macroscopic Effects of mHTT II-29
Diagnosis of HD II-30
Clinical Diagnosis II-30
Embryonic Diagnosis II-30
Differential Diagnosis II-31
Diagnostic Tests for Huntington’s Disease II-31
Confirmatory Diagnostic Test II-31
Pre-symptomatic Diagnostic Test II-31
Prenatal Diagnostic Test II-31
Treatment Options II-31
Nutrition and Eating II-31
Social Activity II-32
Speech Therapy II-32
Physical Therapy II-32
Occupational Therapy II-33
Medication II-33

6. CLINICAL TRIALS II-34
Dimebon Fails Horizon Trials for Treatment of Cognitive
Dysfunction in HD Patients II-34
Huntexil’s Trials Prove only Borderline Benefits II-34
AFQ056 in Phase II Clinical Study for Treatment of Chorea II-34
Siena Biotech S.p.A. Undertakes Preparatory Steps for Phase II
study of SEN0014196 II-35
Elontril in the Set-up Stage for Phase II Study II-35
Alnylam Presents Positive Pre-clinical Data for its siRNA for
silencing the Mutant HTT II-35

7. RECENT INDUSTRY ACTIVITY II-36
Roche Enters into Alliance with Isis Pharmaceuticals II-36
Isis and CHDI Collaborate with KineMed II-36
Omeros Receives IND Approval for OMS824 for Huntington’s Disease II-37
Teva Pharmaceutical Industries Signs Asset Transfer Agreement
with NeuroSearch II-38
uniQure and Benitec Biopharma Ink Cross- Licensing Agreement II-38
Evotec and CHDI Foundation Extend Collaboration II-38
Raptor Acquires Exclusive Rights to Cysteamine and Associated
Compounds II-38
GNS Healthcare Enters into Collaboration with CHDI Foundation II-39
Lundbeck Collaborates with CHDI Foundation for HD Research II-39
Horizon Discovery Collaborates with Institute of Neurology,
London II-40
Thomson Reuters and CHDI Foundation Enter into MetaMiner
Partnership II-40
Targeted Genetics Changes Name to AmpliPhi Biosciences II-40
Intellect Neurosciences Commences Initial Development of
CONJUMAB-A Platform Technology II-41
CHDI Foundation and Galenea Collaborate II-41
CHDI Foundation and Isis Pharmaceuticals Renew Collaboration II-41
Sage Bionetworks and Takeda Pharmaceutical Enter into Research
Alliance II-42
Sage Bionetworks Extends Partnership with CHDI Foundation II-42
University of Dundee and GlaxoSmithKline to Collaborate over
HD Research II-43

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