Juvenile Macular Degeneration (Stargardt Disease) - Pipeline Review, H2 2016

Published: Aug, 2016 | Pages: 68 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Juvenile Macular Degeneration (Stargardt Disease) - Pipeline Review, H2 2016

Summary

Global Markets Direct's, 'Juvenile Macular Degeneration (Stargardt Disease) - Pipeline Review, H2 2016', provides an overview of the Juvenile Macular Degeneration (Stargardt Disease) pipeline landscape. 

The report provides comprehensive information on the therapeutics under development for Juvenile Macular Degeneration (Stargardt Disease), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Juvenile Macular Degeneration (Stargardt Disease) and features dormant and discontinued projects. 

Global Markets Direct's report features investigational drugs from across globe covering over 20 therapy areas and nearly 3,000 indications. The report is built using data and information sourced from Global Markets Direct's proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

The report helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage.

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

Scope

- The report provides a snapshot of the global therapeutic landscape of Juvenile Macular Degeneration (Stargardt Disease)
- The report reviews pipeline therapeutics for Juvenile Macular Degeneration (Stargardt Disease) by companies and universities/research institutes based on information derived from company and industry-specific sources 
- The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages 
- The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities 
- The report reviews key players involved Juvenile Macular Degeneration (Stargardt Disease) therapeutics and enlists all their major and minor projects
- The report assesses Juvenile Macular Degeneration (Stargardt Disease) therapeutics based on drug target, mechanism of action (MoA), route of administration (RoA) and molecule type 
- The report summarizes all the dormant and discontinued pipeline projects 
- The report reviews latest news related to pipeline therapeutics for Juvenile Macular Degeneration (Stargardt Disease)

Reasons To Buy

- Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies
- Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage 
- Identify and understand important and diverse types of therapeutics under development for Juvenile Macular Degeneration (Stargardt Disease)
- Identify potential new clients or partners in the target demographic
- Develop strategic initiatives by understanding the focus areas of leading companies 
- Plan mergers and acquisitions effectively by identifying key players and it's most promising pipeline therapeutics
- Devise corrective measures for pipeline projects by understanding Juvenile Macular Degeneration (Stargardt Disease) 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 5
List of Figures 5
Introduction 6
Global Markets Direct Report Coverage 6
Juvenile Macular Degeneration (Stargardt Disease) Overview 7
Therapeutics Development 8
Pipeline Products for Juvenile Macular Degeneration (Stargardt Disease) - Overview 8
Pipeline Products for Juvenile Macular Degeneration (Stargardt Disease) - Comparative Analysis 9
Juvenile Macular Degeneration (Stargardt Disease) - Therapeutics under Development by Companies 10
Juvenile Macular Degeneration (Stargardt Disease) - Therapeutics under Investigation by Universities/Institutes 11
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline Products Glance 12
Clinical Stage Products 12
Early Stage Products 13
Juvenile Macular Degeneration (Stargardt Disease) - Products under Development by Companies 14
Juvenile Macular Degeneration (Stargardt Disease) - Products under Investigation by Universities/Institutes 15
Juvenile Macular Degeneration (Stargardt Disease) - Companies Involved in Therapeutics Development 16
Acucela Inc. 16
Alkeus Pharmaceuticals, Inc. 17
Astellas Pharma Inc. 18
Copernicus Therapeutics, Inc. 19
Iris Pharma 20
Sanofi 21
Juvenile Macular Degeneration (Stargardt Disease) - Therapeutics Assessment 22
Assessment by Monotherapy Products 22
Assessment by Target 23
Assessment by Mechanism of Action 25
Assessment by Route of Administration 27
Assessment by Molecule Type 29
Drug Profiles 31
ALK-001 - Drug Profile 31
Product Description 31
Mechanism Of Action 31
R&D Progress 31
echothiophate iodide - Drug Profile 32
Product Description 32
Mechanism Of Action 32
R&D Progress 32
emixustat hydrochloride - Drug Profile 33
Product Description 33
Mechanism Of Action 33
R&D Progress 33
Gene Therapy 1 to Activate ABCA4 for Stargardt Disease - Drug Profile 37
Product Description 37
Mechanism Of Action 37
R&D Progress 37
Gene Therapy to Activate ABCA4 for Stargardt Disease - Drug Profile 38
Product Description 38
Mechanism Of Action 38
R&D Progress 38
MA09-hRPE - Drug Profile 39
Product Description 39
Mechanism Of Action 39
R&D Progress 39
Oligonucleotides to Activate ABCA4 for Stargardt Disease - Drug Profile 49
Product Description 49
Mechanism Of Action 49
R&D Progress 49
ramiprilat - Drug Profile 50
Product Description 50
Mechanism Of Action 50
R&D Progress 50
SAR-422459 - Drug Profile 51
Product Description 51
Mechanism Of Action 51
R&D Progress 51
soraprazan - Drug Profile 53
Product Description 53
Mechanism Of Action 53
R&D Progress 53
Stem Cell Therapy for Ophthalmic Disorders - Drug Profile 54
Product Description 54
Mechanism Of Action 54
R&D Progress 54
VM-200 - Drug Profile 55
Product Description 55
Mechanism Of Action 55
R&D Progress 55
Juvenile Macular Degeneration (Stargardt Disease) - Dormant Projects 56
Juvenile Macular Degeneration (Stargardt Disease) - Product Development Milestones 57
Featured News & Press Releases 57
May 26, 2015: Ocata Therapeutics Receives Three New U.S. Patents for its RPE Therapy for Macular Degenerative Diseases 57
May 04, 2015: European Medicines Agency Grants Orphan Designation for Makindus' MI-100 for Stargardt's Disease 57
Mar 31, 2015: Ocata Therapeutics Successfully Completes Dosing in Phase 1/2 RPE Studies 58
Oct 15, 2014: ACT Announces Positive Results from Two Clinical Trials Published in The Lancet Using Differentiated Stem Cell-Derived Retinal Pigment Epithelium (RPE) Cells for the Treatment of Macular Degeneration 58
Sep 25, 2014: Advanced Cell Technology Announces Final Patient Treated in Stargardt's Macular Degeneration Phase 1 Trial in the United Kingdom 59
Jul 17, 2013: ACT Secures Approval from Data Safety Monitoring Board to Complete Third Patient Cohort in All Three Clinical Trials 60
May 16, 2013: ACT Confirms Clinical Trial Participant Shows Improvement In Vision Following Treatment With Human Embryonic Stem Cells 61
Apr 23, 2013: ACT Initiates Higher-Dosage Patient Treatment In European Phase I Study For Macular Degeneration 61
Apr 15, 2013: ACT Treats First Patient with Better Vision in Clinical Trial for Stargardt's Macular Dystrophy 61
Apr 01, 2013: ACT Initiates Treatment Of Higher-dosage Cohort In Clinical Trials For Dry Age-related Macular Degeneration And Stargardt's Macular Dystrophy 62
Mar 14, 2013: Advanced Cell Technology Receives DSMB Approval To Initiate Treatment Of Third Patient Cohort In All Three Clinical Trials Using hESC-derived RPE Cells 63
Jan 08, 2013: Advanced Cell Technology Achieves Clinical Milestone 64
Dec 17, 2012: ACT Completes Higher-Dosage Cohort In European Clinical Trial For Stargardt's Macular Dystrophy Using Retinal Pigment Epithelial Cells 64
Nov 28, 2012: ACT Completes Higher-Dosage Cohort In Both US Clinical Trials Using Retinal Pigment Epithelial Cells 65
Nov 08, 2012: Advanced Cell Technology Announces Interim Data From Three Ongoing Macular Degeneration Trials 65
Appendix 67
Methodology 67
Coverage 67
Secondary Research 67
Primary Research 67
Expert Panel Validation 67
Contact Us 67
Disclaimer 68

List of Tables

Number of Products under Development for Juvenile Macular Degeneration (Stargardt Disease), H2 2016 8
Number of Products under Development for Juvenile Macular Degeneration (Stargardt Disease) - Comparative Analysis, H2 2016 9
Number of Products under Development by Companies, H2 2016 10
Number of Products under Investigation by Universities/Institutes, H2 2016 11
Comparative Analysis by Clinical Stage Development, H2 2016 12
Comparative Analysis by Early Stage Development, H2 2016 13
Products under Development by Companies, H2 2016 14
Products under Investigation by Universities/Institutes, H2 2016 15
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Acucela Inc., H2 2016 16
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Alkeus Pharmaceuticals, Inc., H2 2016 17
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Astellas Pharma Inc., H2 2016 18
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Copernicus Therapeutics, Inc., H2 2016 19
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Iris Pharma, H2 2016 20
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Sanofi, H2 2016 21
Assessment by Monotherapy Products, H2 2016 22
Number of Products by Stage and Target, H2 2016 24
Number of Products by Stage and Mechanism of Action, H2 2016 26
Number of Products by Stage and Route of Administration, H2 2016 28
Number of Products by Stage and Molecule Type, H2 2016 30
Juvenile Macular Degeneration (Stargardt Disease) - Dormant Projects, H2 2016 56