Sections
| Title | Starting Page | Number of Pages |
|---|
| Table of Contents | 2 | 6 |
| List of Tables | 6 | 1 |
| List of Figures | 7 | 1 |
| Introduction | 8 | 1 |
| Global Markets Direct Report Coverage | 8 | 1 |
| Fragile X Syndrome Overview | 9 | 1 |
| Therapeutics Development | 10 | 2 |
| Pipeline Products for Fragile X Syndrome Overview | 10 | 1 |
| Pipeline Products for Fragile X Syndrome Comparative Analysis | 11 | 1 |
| Fragile X Syndrome Therapeutics under Development by Companies | 12 | 2 |
| Fragile X Syndrome Therapeutics under Investigation by Universities/Institutes | 14 | 1 |
| Fragile X Syndrome Pipeline Products Glance | 15 | 3 |
| Late Stage Products | 15 | 1 |
| Clinical Stage Products | 16 | 1 |
| Early Stage Products | 17 | 1 |
| Fragile X Syndrome Products under Development by Companies | 18 | 2 |
| Fragile X Syndrome Products under Investigation by Universities/Institutes | 20 | 1 |
| Fragile X Syndrome Companies Involved in Therapeutics Development | 21 | 16 |
| Aelis Farma SAS | 21 | 1 |
| Alcobra Ltd | 22 | 1 |
| AMO Pharma Limited | 23 | 1 |
| Anavex Life Sciences Corp. | 24 | 1 |
| Confluence Pharmaceuticals LLC | 25 | 1 |
| DRI Biosciences Corporation | 26 | 1 |
| Eli Lilly and Company | 27 | 1 |
| GlaxoSmithKline Plc | 28 | 1 |
| GW Pharmaceuticals Plc | 29 | 1 |
| Kareus Therapeutics, SA | 30 | 1 |
| Marinus Pharmaceuticals, Inc. | 31 | 1 |
| Neuren Pharmaceuticals Limited | 32 | 1 |
| Neuron Biopharma SA | 33 | 1 |
| Ovid Therapeutics Inc. | 34 | 1 |
| Sage Therapeutics, Inc. | 35 | 1 |
| Zynerba Pharmaceuticals, Inc. | 36 | 1 |
| Fragile X Syndrome Therapeutics Assessment | 37 | 9 |
| Assessment by Monotherapy Products | 37 | 1 |
| Assessment by Target | 38 | 2 |
| Assessment by Mechanism of Action | 40 | 2 |
| Assessment by Route of Administration | 42 | 2 |
| Assessment by Molecule Type | 44 | 2 |
| Drug Profiles | 46 | 55 |
| acamprosate calcium Drug Profile | 46 | 1 |
| ACT-01 Drug Profile | 47 | 1 |
| AMO-01 Drug Profile | 48 | 1 |
| ANAVEX-273 Drug Profile | 49 | 8 |
| Bryostatin-1 Drug Profile | 57 | 4 |
| cercosporamide Drug Profile | 61 | 1 |
| Drug to Antagonize mGluR5 for Fragile X Syndrome and 16p11.2 Microdeletion Syndrome Drug Profile | 62 | 1 |
| flindokalner Drug Profile | 63 | 1 |
| gaboxadol Drug Profile | 64 | 2 |
| ganaxolone Drug Profile | 66 | 5 |
| JRP-655 Drug Profile | 71 | 1 |
| KU-046 Drug Profile | 72 | 1 |
| metadoxine ER Drug Profile | 73 | 6 |
| NNZ-2591 Drug Profile | 79 | 2 |
| NST-0076 Drug Profile | 81 | 1 |
| SGE-872 Drug Profile | 82 | 1 |
| Small Molecule for Fragile X-Associated Tremor Ataxia Syndrome Drug Profile | 83 | 1 |
| Small Molecule to Activate FMRP for Fragile X Syndrome Drug Profile | 84 | 1 |
| Small Molecule to Antagonize CB1R for Fragile X Syndrome and Down Syndrome Drug Profile | 85 | 1 |
| Small Molecules to Activate Slack Channels for Fragile X Syndrome Drug Profile | 86 | 1 |
| Small Molecules to Agonize Cannabinoid Receptor 2 for Fragile X Syndrome, Pervasive Developmental Disorder and Rett Syndrome Drug Profile | 87 | 1 |
| Small Molecules to Block Kv3.1 for Fragile X Syndrome Drug Profile | 88 | 1 |
| Small Molecules to Inhibit PI3K for Autism and Fragile X Syndrome Drug Profile | 89 | 1 |
| SRT-278 Drug Profile | 90 | 1 |
| TC-2153 Drug Profile | 91 | 1 |
| trofinetide Drug Profile | 92 | 6 |
| ZYN-002 Drug Profile | 98 | 3 |
| Fragile X Syndrome Dormant Projects | 101 | 2 |
| Fragile X Syndrome Discontinued Products | 103 | 1 |
| Fragile X Syndrome Product Development Milestones | 104 | 12 |
| Featured News &Press Releases | 104 | 1 |
| Jul 19, 2016: Alcobra Granted European Orphan Drug Designation for Metadoxine in Fragile X Syndrome | 104 | 1 |
| Jun 28, 2016: Results from Phase 2 Exploratory Clinical Study Support Continued Development of Ganaxolone in Fragile X Syndrome | 104 | 1 |
| Jun 27, 2016: Marinus Pharmaceuticals to Host Conference Call on Tuesday, June 28 | 105 | 1 |
| Jun 27, 2016: Zynerba Pharmaceuticals Announces Positive Top Line Results from ZYN002 CBD Gel Phase 1 Multiple Rising Dose Trial and Initiation of Phase 2 Study in Adult Epilepsy Patients with Refractory Focal Seizures | 106 | 1 |
| Jun 22, 2016: Neuren receives grant of new Australian patent for trofinetide to treat Autism Spectrum Disorders | 107 | 1 |
| Jun 21, 2016: Bryostatin, a PKC epsilon Activator, Generates New Synapses Through Accumulation of the Synaptic Anchoring Protein PSD-95 at Neuronal Membranes | 107 | 1 |
| Jun 13, 2016: Neurotrope's Chief Scientific Officer Dr. Daniel Alkon Presents at the Gordon Conference: Drugs That Target Common Mechanisms for Synaptic Restoration in Mental Retardation and Alzheimer's Disease | 108 | 1 |
| Jun 07, 2016: AMO Pharma Presents Pre-clinical Data Showing AMO-01 Successfully Inhibits Activated Pathway Associated with Fragile X Syndrome | 108 | 1 |
| Jun 06, 2016: Anavex Announces Positive Preclinical Results with ANAVEX 2-73 in Fragile X Autism-Related Disorders | 109 | 1 |
| Apr 12, 2016: Cannabis and Cannabinoid Research Publishes Data Demonstrating the Degradation of Cannabidiol to Psychoactive Cannabinoids when Exposed to Simulated Gastric Fluid | 110 | 1 |
| Mar 08, 2016: Neurotrope Announces Publication Demonstrating Synaptic Regeneration with the PKC epsilon Activator Bryostatin, in a Fragile X Disease Late Brain Development Model | 111 | 1 |
| Feb 25, 2016: Zynerba Pharmaceuticals Announces ZYN002 Granted Orphan Drug Designation for Fragile X Syndrome | 111 | 1 |
| Jan 06, 2016: Zynerba Pharmaceuticals Announces Positive Initial Results From ZYN002 CBD Gel Phase 1 Single Rising Dose Trial and Initiation of Phase 1 Multiple Rising Dose Trial | 112 | 1 |
| Dec 07, 2015: Neuren s trofinetide successful in proof of concept Phase 2 clinical trial in Fragile X syndrome | 113 | 2 |
| Nov 30, 2015: Neuren receives grant of composition patent in Europe for NNZ-2591 | 115 | 1 |
| Appendix | 116 | 2 |
| Methodology | 116 | 1 |
| Coverage | 116 | 1 |
| Secondary Research | 116 | 1 |
| Primary Research | 116 | 1 |
| Expert Panel Validation | 116 | 1 |
| Contact Us | 116 | 1 |
| Disclaimer | 117 | 1 |
