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

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

Companies mentioned

Acucela Inc.

Alkeus Pharmaceuticals, Inc.

Astellas Pharma Inc.

Copernicus Therapeutics, Inc.

Iris Pharma

Sanofi

Table of Contents

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

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

List of Figures

List of Figures

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

Comparative Analysis by Early Stage Products, H2 2016 13

Assessment by Monotherapy Products, H2 2016 22

Number of Products by Targets, H2 2016 23

Number of Products by Stage and Targets, H2 2016 23

Number of Products by Mechanism of Actions, H2 2016 25

Number of Products by Stage and Mechanism of Actions, H2 2016 25

Number of Products by Routes of Administration, H2 2016 27

Number of Products by Stage and Routes of Administration, H2 2016 27

Number of Products by Molecule Types, H2 2016 29

Number of Products by Stage and Molecule Types, H2 2016 29

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