Neurodegenerative Diseases – Pipeline Review, H2 2013

Summary

Global Markets Direct’s, ‘Neurodegenerative Diseases – Pipeline Review, H2 2013’, provides an overview of the indication’s therapeutic pipeline. This report provides information on the therapeutic development for Neurodegenerative Diseases, complete with latest updates, and special features on late-stage and discontinued projects. It also reviews key players involved in the therapeutic development for Neurodegenerative Diseases. Neurodegenerative Diseases – Pipeline Review, Half Year is built using data and information sourced from Global Markets Direct’s proprietary databases, Company/University websites, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources, put together by Global Markets Direct’s team.

Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data for the indicated disease.

Scope

– A snapshot of the global therapeutic scenario for Neurodegenerative Diseases.

– A review of the Neurodegenerative Diseases products under development by companies and universities/research institutes based on information derived from company and industry-specific sources.

– Coverage of products based on various stages of development ranging from discovery till registration stages.

– A feature on pipeline projects on the basis of monotherapy and combined therapeutics.

– Coverage of the Neurodegenerative Diseases pipeline on the basis of route of administration and molecule type.

– Key discontinued pipeline projects.

– Latest news and deals relating to the products.

Reasons to buy

– Identify and understand important and diverse types of therapeutics under development for Neurodegenerative Diseases.

– Identify emerging players with potentially strong product portfolio and design effective counter-strategies to gain competitive advantage.

– Plan mergers and acquisitions effectively by identifying players of the most promising pipeline.

– Devise corrective measures for pipeline projects by understanding Neurodegenerative Diseases pipeline depth and focus of Indication therapeutics.

– Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope.

– Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from pipeline.

Table of Contents

Table of Contents 2

List of Tables 8

List of Figures 10

Introduction 11

Global Markets Direct Report Coverage 11

Neurodegenerative Diseases Overview 12

Therapeutics Development 13

An Overview of Pipeline Products for Neurodegenerative Diseases 13

Neurodegenerative Diseases Therapeutics under Development by Companies 15

Neurodegenerative Diseases Therapeutics under Investigation by Universities/Institutes 21

Mid Clinical Stage Products 24

Comparative Analysis 24

Early Clinical Stage Products 25

Comparative Analysis 25

Discovery and Pre-Clinical Stage Products 26

Comparative Analysis 26

Neurodegenerative Diseases Therapeutics - Products under Development by Companies 27

Neurodegenerative Diseases Therapeutics - Products under Investigation by Universities/Institutes 34

Companies Involved in Neurodegenerative Diseases Therapeutics Development 36

Bristol-Myers Squibb Company 36

F. Hoffmann-La Roche Ltd. 37

Biogen Idec Inc. 38

Apotex, Inc. 39

ExonHit Therapeutics SA 40

Isotechnika Pharma Inc. 41

EnVivo Pharmaceuticals 42

Les Laboratoires Servier 43

Pfizer Inc. 44

Merck KGaA 45

Addex Pharmaceuticals 46

Neuralstem, Inc. 47

Genfit 48

Nymox Pharmaceutical Corporation 49

ACADIA Pharmaceuticals Inc. 50

Yungjin Pharm Ind. Co., Ltd. 51

WhanIn Pharmaceutical Co., Ltd. 52

Suven Life Sciences Ltd. 53

MediPost Co., Ltd. 54

Newron Pharmaceuticals S.p.A. 55

Intas Pharmaceuticals Ltd. 56

Oryzon 57

Angelini Group 58

SCYNEXIS, Inc. 59

Chipscreen Biosciences Ltd 60

Quantum Pharmaceuticals 61

Oncodesign SA 62

Aegera Therapeutics Inc. 63

Medeia Therapeutics Ltd. 64

AngioChem Inc. 65

Phoenix Biotechnology, Inc. 66

Antyra, Inc. 67

TetraLogic Pharmaceuticals 68

NEURONAX 69

PsychoGenics, Inc. 70

GenKyoTex S.A. 71

Affichem 72

NeuroNascent, Inc. 73

Siena Biotech S.p.A. 74

NovaLead Pharma Pvt. Ltd. 75

N-Gene Research Laboratories, Inc. 76

BT PHARMA 77

Biovista Inc. 78

Nexgenix Pharmaceuticals, LLC 79

Proteostasis Therapeutics, Inc. 80

M's Science Corporation 81

Encore Therapeutics Inc. 82

Georgia Tech Research Corporation 83

MAKScientific, LLC 84

GENEXTRA S.p.A. 85

Ausio Pharmaceuticals, LLC 86

Histocell S.L. 87

Synthon BV 88

Varinel, Inc. 89

Sigmoid Pharma. 90

Neurodegenerative Diseases - Therapeutics Assessment 91

Assessment by Monotherapy Products 91

Assessment by Route of Administration 92

Assessment by Molecule Type 94

Drug Profiles 96

EVP-0334 - Drug Profile 96

G-15750 - Drug Profile 98

TGFTX-1 Program - Drug Profile 99

TGFTX-3 Program - Drug Profile 100

sarizotan hydrochloride - Drug Profile 101

CSO-240-0600 - Drug Profile 103

NeuroTrans Program - Drug Profile 104

NXD-5150 - Drug Profile 105

NNI-370 - Drug Profile 106

AF-243 - Drug Profile 107

Necrostatin Program - Drug Profile 108

AUS-131 - Drug Profile 109

EHT/AGN-0002 - Drug Profile 111

VAR-10300 - Drug Profile 112

EPO Mimetics - Drug Profile 114

MC-116 - Drug Profile 115

AMT-090 - Drug Profile 116

NXD-30001 - Drug Profile 117

VAR-10200 - Drug Profile 118

ponezumab - Drug Profile 119

ANG-3101 - Drug Profile 121

ANG-3201 - Drug Profile 122

GSK-3 Beta Inhibitor - Drug Profile 123

EHT/AGN-0003 - Drug Profile 124

Immunoglobulin - Drug Profile 125

NSI-189 - Drug Profile 126

NLP-61 - Drug Profile 128

YJP-60107 - Drug Profile 129

NST-0021 - Drug Profile 130

NST-0005 - Drug Profile 131

NST-0037 - Drug Profile 132

NG-094 - Drug Profile 133

FRP-XX - Drug Profile 134

AF-250s - Drug Profile 135

Alpha Synuclein Program - Drug Profile 136

TDP-43 Program - Drug Profile 137

NOX 2/1 Inhibitors - Drug Profile 138

diazoxide - Drug Profile 139

P2X7 Compounds - Drug Profile 141

TRKB PAM - Drug Profile 142

RPh-201 - Drug Profile 143

SUVN-501 - Drug Profile 145

SUVN-507 - Drug Profile 146

SUVN-901 - Drug Profile 147

SUVN-976 - Drug Profile 148

Drug Targeting Nicotinic Acetylcholine Receptor Alpha-4 Beta-2 For Neuro Degenerative Diseases - Drug Profile 149

ER-Beta Program - Drug Profile 150

PT-00114 - Drug Profile 151

PT-00311 - Drug Profile 152

Glutaminyl Cyclase Inhibitors - Drug Profile 153

TDI-154 - Drug Profile 154

NST-0060 - Drug Profile 155

NPS-0155 - Drug Profile 156

FRP-924 - Drug Profile 157

NPT-002 - Drug Profile 158

PBI-04711 - Drug Profile 159

NT-KO-009 - Drug Profile 160

NT-KO-012 - Drug Profile 161

NT-CC-017 - Drug Profile 162

F-637 - Drug Profile 163

WIN-1001X - Drug Profile 164

RDC-5 - Drug Profile 165

Program-3 - Drug Profile 166

JRP-655 - Drug Profile 167

JRP-900 - Drug Profile 168

MAC-i Class I - Drug Profile 169

MAC-i Class II - Drug Profile 170

EAB-318 - Drug Profile 171

Drug Targeting HSP90 - Drug Profile 172

S-Allymercapto-N-Acetylcysteine - Drug Profile 173

Nonimmunosuppressive Cyclosporin Analog Molecule Program - Drug Profile 174

CS-410 - Drug Profile 176

meldonium - Drug Profile 177

caffeic acid phenethyl ester - Drug Profile 178

TDI-185 - Drug Profile 179

Adenosine Antagonists - Drug Profile 180

Sphingosine Kinase-1 Inhibitor - Drug Profile 181

Sphingosine Kinase-2 Inhibitor - Drug Profile 182

miR-29b - Drug Profile 183

Estrogen Receptor Beta Ligand - Drug Profile 184

enreptin - Drug Profile 185

RE-024 - Drug Profile 186

Gamma-Secretase Modulators - Drug Profile 187

Retinal Neuroprotective Compounds - Drug Profile 188

Drug For Neuroprotection And Neurite-Promoting Activity - Drug Profile 189

mPTP Inhibitors - Drug Profile 190

BVA-202 - Drug Profile 191

BVA-203 - Drug Profile 192

UBP-608 - Drug Profile 193

VCE-006 - Drug Profile 194

CDNF Protein Therapy For Other Neurodegenerative Disease - Drug Profile 195

mGluR3 PAMs - Drug Profile 196

mGluR4 PAMs Backup Series - Drug Profile 197

mGluR4 PAMs Lead Series - Drug Profile 198

Drug For Tau-Related Diseases - Drug Profile 199

HAT Inhibitor Program - Drug Profile 200

Drugs For Neurodegenerative Diseases - Drug Profile 201

AM-5206 - Drug Profile 202

Alpha Synuclein - Drug Profile 204

MDA-107 - Drug Profile 205

MDA-107 Back Up - Drug Profile 207

Cyclohetero Cycloalkylaminoaryl Derivatives - Drug Profile 209

Drug For Neurodegenerative Disorders - Drug Profile 210

Drug 2 For Neurodegenerative Diseases - Drug Profile 211

Drug 1 For Neurodegenerative Diseases - Drug Profile 212

Glycolysis Inhibitors - Drug Profile 213

NS-640 - Drug Profile 214

ORY-2001 - Drug Profile 215

Biosimilar-2 - Drug Profile 216

Drug For Cancer And Neurodegenerative Disease - Drug Profile 217

LMW Immunomodulator - Drug Profile 218

Pluripotent Stem Cells From Embryonic Tissues - Drug Profile 219

HR-010 - Drug Profile 220

P7C3 - Drug Profile 221

Insulin-Regulated Amino Peptidase Inhibitor - Drug Profile 222

Secretory IgA - Drug Profile 223

Glutathione Peroxidase Mimetic Compounds - Drug Profile 224

Lipophilic Phenolic Antioxidants - Drug Profile 225

Tetraamine derivatives - Drug Profile 226

Aza-Peptide Epoxides - Drug Profile 227

PC-41 - Drug Profile 228

NST-0076 - Drug Profile 229

NST-0078 - Drug Profile 230

Salt-Inducible Kinase 2 Inhibitor - Drug Profile 231

Cell-Division Cycle Protein-Like Kinase Family Inhibitor - Drug Profile 232

GDNFOS Peptides - Drug Profile 233

Antibody Against GDNFOS3 - Drug Profile 234

Morphinans - Drug Profile 235

Opioid Peptides - Drug Profile 236

Tripeptide GGF - Drug Profile 237

Neurodegenerative Disease Program - Drug Profile 238

Compound-12 - Drug Profile 239

Compound-69 - Drug Profile 240

Compound-149 - Drug Profile 241

Drugs Targeting Leukocyte Immunoglobulin Like Receptor - Drug Profile 242

Compound-1 - Drug Profile 243

Compound-2 - Drug Profile 244

Neurodegenerative Diseases Therapeutics - Drug Profile Updates 245

Neurodegenerative Diseases Therapeutics - Discontinued Products 256

Neurodegenerative Diseases Therapeutics - Dormant Products 257

Neurodegenerative Diseases - Product Development Milestones 259

Featured News & Press Releases 259

Appendix 265

Methodology 265

Coverage 265

Secondary Research 265

Primary Research 265

Expert Panel Validation 265

Contact Us 266

Disclaimer 266



List of Tables

Number of Products Under Development for Neurodegenerative Diseases, H2 2013 22

Products under Development for Neurodegenerative Diseases - Comparative Analysis, H2 2013 23

Number of Products under Development by Companies, H2 2013 25

Number of Products under Development by Companies, H2 2013 (Contd..1) 26

Number of Products under Development by Companies, H2 2013 (Contd..2) 27

Number of Products under Development by Companies, H2 2013 (Contd..3) 28

Number of Products under Development by Companies, H2 2013 (Contd..4) 29

Number of Products under Investigation by Universities/Institutes, H2 2013 31

Number of Products under Investigation by Universities/Institutes, H2 2013 (Contd..1) 32

Comparative Analysis by Mid Clinical Stage Development, H2 2013 33

Comparative Analysis by Early Clinical Stage Development, H2 2013 34

Comparative Analysis by Discovery and Pre-Clinical Stage Development, H2 2013 35

Products under Development by Companies, H2 2013 36

Products under Development by Companies, H2 2013 (Contd..1) 37

Products under Development by Companies, H2 2013 (Contd..2) 38

Products under Development by Companies, H2 2013 (Contd..3) 39

Products under Development by Companies, H2 2013 (Contd..4) 40

Products under Development by Companies, H2 2013 (Contd..5) 41

Products under Development by Companies, H2 2013 (Contd..6) 42

Products under Investigation by Universities/Institutes, H2 2013 43

Products under Investigation by Universities/Institutes, H2 2013 (Contd..1) 44

Bristol-Myers Squibb Company, H2 2013 45

F. Hoffmann-La Roche Ltd., H2 2013 46

Biogen Idec Inc., H2 2013 47

Apotex, Inc., H2 2013 48

ExonHit Therapeutics SA, H2 2013 49

Isotechnika Pharma Inc., H2 2013 50

EnVivo Pharmaceuticals, H2 2013 51

Les Laboratoires Servier, H2 2013 52

Pfizer Inc., H2 2013 53

Merck KGaA, H2 2013 54

Addex Pharmaceuticals, H2 2013 55

Neuralstem, Inc., H2 2013 56

Genfit, H2 2013 57

Nymox Pharmaceutical Corporation, H2 2013 58

ACADIA Pharmaceuticals Inc., H2 2013 59

Yungjin Pharm Ind. Co., Ltd., H2 2013 60

WhanIn Pharmaceutical Co., Ltd., H2 2013 61

Suven Life Sciences Ltd., H2 2013 62

MediPost Co., Ltd., H2 2013 63

Newron Pharmaceuticals S.p.A., H2 2013 64

Intas Pharmaceuticals Ltd., H2 2013 65

Oryzon, H2 2013 66

Angelini Group, H2 2013 67

SCYNEXIS, Inc., H2 2013 68

Chipscreen Biosciences Ltd, H2 2013 69

Quantum Pharmaceuticals, H2 2013 70

Oncodesign SA, H2 2013 71

Aegera Therapeutics Inc., H2 2013 72

Medeia Therapeutics Ltd., H2 2013 73

AngioChem Inc., H2 2013 74

Phoenix Biotechnology, Inc., H2 2013 75

Antyra, Inc., H2 2013 76

TetraLogic Pharmaceuticals, H2 2013 77

NEURONAX, H2 2013 78

PsychoGenics, Inc., H2 2013 79

GenKyoTex S.A., H2 2013 80

Affichem, H2 2013 81

NeuroNascent, Inc., H2 2013 82

Siena Biotech S.p.A., H2 2013 83

NovaLead Pharma Pvt. Ltd., H2 2013 84

N-Gene Research Laboratories, Inc., H2 2013 85

BT PHARMA, H2 2013 86

Biovista Inc., H2 2013 87

Nexgenix Pharmaceuticals, LLC, H2 2013 88

Proteostasis Therapeutics, Inc., H2 2013 89

M's Science Corporation, H2 2013 90

Encore Therapeutics Inc., H2 2013 91

Georgia Tech Research Corporation, H2 2013 92

MAKScientific, LLC, H2 2013 93

GENEXTRA S.p.A., H2 2013 94

Ausio Pharmaceuticals, LLC, H2 2013 95

Histocell S.L., H2 2013 96

Synthon BV, H2 2013 97

Varinel, Inc., H2 2013 98

Sigmoid Pharma., H2 2013 99

Assessment by Monotherapy Products, H2 2013 100

Assessment by Stage and Route of Administration, H2 2013 102

Assessment by Stage and Molecule Type, H2 2013 104

Neurodegenerative Diseases Therapeutics - Drug Profile Updates 254

Neurodegenerative Diseases Therapeutics - Discontinued Products 265

Neurodegenerative Diseases Therapeutics - Dormant Products 266

Neurodegenerative Diseases Therapeutics - Dormant Products (Contd..2) 267



List of Figures

Number of Products under Development for Neurodegenerative Diseases, H2 2013 22

Products under Development for Neurodegenerative Diseases - Comparative Analysis, H2 2013 23

Products under Development by Companies, H2 2013 24

Products under Investigation by Universities/Institutes, H2 2013 30

Mid Clinical Stage Products, H2 2013 33

Early Clinical Stage Products, H2 2013 34

Discovery and Pre-Clinical Stage Products, H2 2013 35

Assessment by Monotherapy Products, H2 2013 100

Assessment by Route of Administration, H2 2013 101

Assessment by Stage and Route of Administration, H2 2013 102

Assessment by Molecule Type, H2 2013 103

Assessment by Stage and Molecule Type, H2 2013 104