Juvenile Macular Degeneration (Stargardt Disease) - Pipeline Review, H1 2016

Published: Mar, 2016 | Pages: 60 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Juvenile Macular Degeneration (Stargardt Disease) - Pipeline Review, H1 2016

Summary

Global Markets Direct's, 'Juvenile Macular Degeneration (Stargardt Disease) - Pipeline Review, H1 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 4
List of Figures 4
Introduction 5
Global Markets Direct Report Coverage 5
Juvenile Macular Degeneration (Stargardt Disease) Overview 6
Therapeutics Development 7
Pipeline Products for Juvenile Macular Degeneration (Stargardt Disease) - Overview 7
Juvenile Macular Degeneration (Stargardt Disease) - Therapeutics under Development by Companies 8
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline Products Glance 9
Clinical Stage Products 9
Early Stage Products 10
Juvenile Macular Degeneration (Stargardt Disease) - Products under Development by Companies 11
Juvenile Macular Degeneration (Stargardt Disease) - Companies Involved in Therapeutics Development 12
Acucela Inc. 12
Alkeus Pharmaceuticals, Inc. 13
Ocata Therapeutics, Inc. 14
Sanofi 15
Juvenile Macular Degeneration (Stargardt Disease) - Therapeutics Assessment 16
Assessment by Monotherapy Products 16
Assessment by Target 17
Assessment by Mechanism of Action 19
Assessment by Route of Administration 21
Assessment by Molecule Type 23
Drug Profiles 25
ALK-001 - Drug Profile 25
Product Description 25
Mechanism of Action 25
R&D Progress 25
echothiophate iodide - Drug Profile 27
Product Description 27
Mechanism of Action 27
R&D Progress 27
emixustat hydrochloride - Drug Profile 28
Product Description 28
Mechanism of Action 28
R&D Progress 28
MA09-hRPE - Drug Profile 30
Product Description 30
Mechanism of Action 30
R&D Progress 30
SAR-422459 - Drug Profile 32
Product Description 32
Mechanism of Action 32
R&D Progress 32
Small Molecule for Stargardt Disease - Drug Profile 34
Product Description 34
Mechanism of Action 34
R&D Progress 34
Stem Cell Therapy for Ophthalmic Disorders - Drug Profile 35
Product Description 35
Mechanism of Action 35
R&D Progress 35
VM-200 - Drug Profile 36
Product Description 36
Mechanism of Action 36
R&D Progress 36
VSM-20R - Drug Profile 37
Product Description 37
Mechanism of Action 37
R&D Progress 37
Juvenile Macular Degeneration (Stargardt Disease) - Recent Pipeline Updates 38
Juvenile Macular Degeneration (Stargardt Disease) - Product Development Milestones 52
Featured News & Press Releases 52
May 26, 2015: Ocata Therapeutics Receives Three New U.S. Patents for its RPE Therapy for Macular Degenerative Diseases 52
May 04, 2015: European Medicines Agency Grants Orphan Designation for Makindus' MI-100 for Stargardt's Disease 52
Mar 31, 2015: Ocata Therapeutics Successfully Completes Dosing in Phase 1/2 RPE Studies 53
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 53
Sep 25, 2014: Advanced Cell Technology Announces Final Patient Treated in Stargardt's Macular Degeneration Phase 1 Trial in the United Kingdom 54
Jul 17, 2013: ACT Secures Approval from Data Safety Monitoring Board to Complete Third Patient Cohort in All Three Clinical Trials 55
May 16, 2013: ACT Confirms Clinical Trial Participant Shows Improvement In Vision Following Treatment With Human Embryonic Stem Cells 56
Apr 23, 2013: ACT Initiates Higher-Dosage Patient Treatment In European Phase I Study For Macular Degeneration 56
Apr 15, 2013: ACT Treats First Patient With Better Vision In Clinical Trial For Stargardt's Macular Dystrophy 56
Apr 01, 2013: ACT Initiates Treatment Of Higher-dosage Cohort In Clinical Trials For Dry Age-related Macular Degeneration And Stargardt's Macular Dystrophy 57
Appendix 59
Methodology 59
Coverage 59
Secondary Research 59
Primary Research 59
Expert Panel Validation 59
Contact Us 59
Disclaimer 60

List of Tables

Number of Products under Development for Juvenile Macular Degeneration (Stargardt Disease), H1 2016 7
Number of Products under Development by Companies, H1 2016 8
Comparative Analysis by Clinical Stage Development, H1 2016 9
Comparative Analysis by Early Stage Development, H1 2016 10
Products under Development by Companies, H1 2016 11
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Acucela Inc., H1 2016 12
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Alkeus Pharmaceuticals, Inc., H1 2016 13
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Ocata Therapeutics, Inc., H1 2016 14
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Sanofi, H1 2016 15
Assessment by Monotherapy Products, H1 2016 16
Number of Products by Stage and Target, H1 2016 18
Number of Products by Stage and Mechanism of Action, H1 2016 20
Number of Products by Stage and Route of Administration, H1 2016 22
Number of Products by Stage and Molecule Type, H1 2016 24
Juvenile Macular Degeneration (Stargardt Disease) Therapeutics - Recent Pipeline Updates, H1 2016 38