Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Pipeline Review, H2 2016

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

Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Pipeline Review, H2 2016

Summary

Global Markets Direct's, 'Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Pipeline Review, H2 2016', provides an overview of the Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) pipeline landscape. 

The report provides comprehensive information on the therapeutics under development for Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy), 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 Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 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 Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy)
- The report reviews pipeline therapeutics for Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 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 Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) therapeutics and enlists all their major and minor projects
- The report assesses Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 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 Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy)

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 Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy)
- 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 Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 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 5
Introduction 6
Global Markets Direct Report Coverage 6
Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) Overview 7
Therapeutics Development 8
Pipeline Products for Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Overview 8
Pipeline Products for Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Comparative Analysis 9
Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Therapeutics under Development by Companies 10
Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Therapeutics under Investigation by Universities/Institutes 11
Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Pipeline Products Glance 12
Late Stage Products 12
Clinical Stage Products 13
Early Stage Products 14
Unknown Stage Products 15
Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Products under Development by Companies 16
Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Products under Investigation by Universities/Institutes 17
Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Companies Involved in Therapeutics Development 18
Alkeus Pharmaceuticals, Inc. 18
Biovista Inc. 19
GenSight Biologics S.A. 20
Ixchel Pharma, LLC 21
Khondrion BV 22
Spark Therapeutics, Inc. 23
Stealth BioTherapeutics Inc. 24
Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Therapeutics Assessment 25
Assessment by Monotherapy Products 25
Assessment by Target 26
Assessment by Mechanism of Action 28
Assessment by Route of Administration 30
Assessment by Molecule Type 32
Drug Profiles 34
BVA-202 - Drug Profile 34
Product Description 34
Mechanism Of Action 34
R&D Progress 34
BVA-203 - Drug Profile 35
Product Description 35
Mechanism Of Action 35
R&D Progress 35
Drug for Lebers Hereditary Optic Neuropathy - Drug Profile 36
Product Description 36
Mechanism Of Action 36
R&D Progress 36
elamipretide - Drug Profile 37
Product Description 37
Mechanism Of Action 37
R&D Progress 37
Gene Therapy to Activate ND4 for Leber's Hereditary Optic Neuropathy - Drug Profile 43
Product Description 43
Mechanism Of Action 43
R&D Progress 43
GS-010 - Drug Profile 44
Product Description 44
Mechanism Of Action 44
R&D Progress 44
GS-011 - Drug Profile 46
Product Description 46
Mechanism Of Action 46
R&D Progress 46
IXC-201 - Drug Profile 47
Product Description 47
Mechanism Of Action 47
R&D Progress 47
IXC-203 - Drug Profile 48
Product Description 48
Mechanism Of Action 48
R&D Progress 48
KH-176 - Drug Profile 49
Product Description 49
Mechanism Of Action 49
R&D Progress 49
SPK-LHON - Drug Profile 51
Product Description 51
Mechanism Of Action 51
R&D Progress 51
Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Dormant Projects 52
Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Product Development Milestones 53
Featured News & Press Releases 53
Sep 08, 2016: GenSight Biologics Receives Approval to Include Teenage Patients in RESCUE and REVERSE Phase III Trials with GS010 in Leber's Hereditary Optic Neuropathy 53
Jun 08, 2016: GenSight Biologics Announces Additional Promising Results of Its Phase I/II Study with GS010 for the Treatment of Leber's Hereditary Optic Neuropathy (LHON) 54
Jun 01, 2016: Stealth BioTherapeutics Initiates Phase 2 Study of Elamipretide in Leber's Hereditary Optic Neuropathy 55
Feb 29, 2016: GenSight Biologics Enrolls First Patient in Pivotal Phase III Program of GS010 in Leber's Hereditary Optic Neuropathy 55
Sep 10, 2015: GenSight Biologics Receives IND Acceptance from FDA to Enter Phase III with GS010 57
Dec 15, 2014: New CMO joins Khondrion 57
Appendix 59
Methodology 59
Coverage 59
Secondary Research 59
Primary Research 59
Expert Panel Validation 59
Contact Us 59
Disclaimer 6

List of Tables

Number of Products under Development for Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy), H2 2016 8
Number of Products under Development for Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - 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 Late Stage Development, H2 2016 12
Comparative Analysis by Clinical Stage Development, H2 2016 13
Comparative Analysis by Early Stage Development, H2 2016 14
Comparative Analysis by Unknown Stage Development, H2 2016 15
Products under Development by Companies, H2 2016 16
Products under Investigation by Universities/Institutes, H2 2016 17
Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Pipeline by Alkeus Pharmaceuticals, Inc., H2 2016 18
Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Pipeline by Biovista Inc., H2 2016 19
Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Pipeline by GenSight Biologics S.A., H2 2016 20
Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Pipeline by Ixchel Pharma, LLC, H2 2016 21
Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Pipeline by Khondrion BV, H2 2016 22
Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Pipeline by Spark Therapeutics, Inc., H2 2016 23
Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Pipeline by Stealth BioTherapeutics Inc., H2 2016 24
Assessment by Monotherapy Products, H2 2016 25
Number of Products by Stage and Target, H2 2016 27
Number of Products by Stage and Mechanism of Action, H2 2016 29
Number of Products by Stage and Route of Administration, H2 2016 31
Number of Products by Stage and Molecule Type, H2 2016 33
Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Dormant Projects, H2 2016 52