Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Pipeline Review, H2 2016

Published: Nov, 2016 | Pages: 63 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Adrenoleukodystrophy - Pipeline Review, H2 2016, provides an overview of the Adrenoleukodystrophy (Genetic Disorders) pipeline landscape.

Adrenoleukodystrophy is a disorder that occurs most often in males. It mainly affects the nervous system and the adrenal glands, which are small glands located on top of each kidney. Abetalipoproteinemia is also a kind of Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) Bassen-Kornzweig syndrome is a rare disease passed down through families in which a person is unable to fully absorb dietary fats through the intestines. The disease is caused by a defect in gene that tells the body to create lipoproteins. It is sympotomized by curvature of spine, muscle weakness, decrease of vision over time. Treatment involves large doses of vitamin supplements containing fat-soluble vitamins.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Adrenoleukodystrophy - Pipeline Review, H2 2016, provides comprehensive information on the therapeutics under development for Adrenoleukodystrophy (Genetic Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

The Adrenoleukodystrophy (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase III, Phase I, IND/CTA Filed, Preclinical and Discovery stages are 2, 2, 1, 5 and 1 respectively. Similarly, the Universities portfolio in Phase II and Phase I stages comprises 1 and 1 molecules, respectively.Adrenoleukodystrophy.

Adrenoleukodystrophy (Genetic Disorders) pipeline guide 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. The guide 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. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Scope

- The pipeline guide provides a snapshot of the global therapeutic landscape of Adrenoleukodystrophy (Genetic Disorders).
- The pipeline guide reviews pipeline therapeutics for Adrenoleukodystrophy (Genetic Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.
- The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
- The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
- The pipeline guide reviews key companies involved in Adrenoleukodystrophy (Genetic Disorders) therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Adrenoleukodystrophy (Genetic Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.
- The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
- The pipeline guide reviews latest news related to pipeline therapeutics for Adrenoleukodystrophy (Genetic Disorders)

Reasons To Buy

- Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
- Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.
- Find and recognize significant and varied types of therapeutics under development for Adrenoleukodystrophy (Genetic Disorders).
- Classify potential new clients or partners in the target demographic.
- Develop tactical initiatives by understanding the focus areas of leading companies.
- Plan mergers and acquisitions meritoriously by identifying key players and it's most promising pipeline therapeutics.
- Formulate corrective measures for pipeline projects by understanding Adrenoleukodystrophy (Genetic Disorders) 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.
- Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what 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
Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) Overview 7
Therapeutics Development 8
Pipeline Products for Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Overview 8
Pipeline Products for Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Comparative Analysis 9
Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Therapeutics under Development by Companies 10
Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Therapeutics under Investigation by Universities/Institutes 11
Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Pipeline Products Glance 12
Late Stage Products 12
Clinical Stage Products 13
Early Stage Products 14
Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Products under Development by Companies 15
Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Products under Investigation by Universities/Institutes 16
Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Companies Involved in Therapeutics Development 17
Applied Genetic Technologies Corp 17
bluebird bio Inc 18
MedDay SA 19
Minoryx Therapeutics sl 20
Novartis AG 21
Pfizer Inc 22
ReceptoPharm Inc 23
Viking Therapeutics Inc 24
Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - 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
biotin - Drug Profile 34
Product Description 34
Mechanism Of Action 34
R&D Progress 34
DRX-065 - Drug Profile 38
Product Description 38
Mechanism Of Action 38
R&D Progress 38
DUOC-01 - Drug Profile 39
Product Description 39
Mechanism Of Action 39
R&D Progress 39
Gene Therapy to Activate ABCD1 for Adrenoleukodystrophy - Drug Profile 40
Product Description 40
Mechanism Of Action 40
R&D Progress 40
HSC-835 - Drug Profile 41
Product Description 41
Mechanism Of Action 41
R&D Progress 41
Lenti-D - Drug Profile 42
Product Description 42
Mechanism Of Action 42
R&D Progress 42
MIN-102 - Drug Profile 45
Product Description 45
Mechanism Of Action 45
R&D Progress 45
OP-101 - Drug Profile 46
Product Description 46
Mechanism Of Action 46
R&D Progress 46
pioglitazone hydrochloride - Drug Profile 47
Product Description 47
Mechanism Of Action 47
R&D Progress 47
RPI-78M - Drug Profile 48
Product Description 48
Mechanism Of Action 48
R&D Progress 48
Stem Cell Therapy to Activate Arylsulfatase A and ABCD1 for Metachromatic Leukodystrophy and Adrenoleukodystrophy - Drug Profile 50
Product Description 50
Mechanism Of Action 50
R&D Progress 50
temsirolimus - Drug Profile 51
Product Description 51
Mechanism Of Action 51
R&D Progress 51
VK-0214 - Drug Profile 54
Product Description 54
Mechanism Of Action 54
R&D Progress 54
Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Dormant Projects 56
Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Product Development Milestones 57
Featured News & Press Releases 57
Sep 26, 2016: Viking Therapeutics Presents Positive Proof-of-Concept Data for VK0214 in In Vivo Model of X-Linked Adrenoleukodystrophy at the 86th Annual Meeting of the American Thyroid Association 57
Sep 14, 2016: Positive Preclinical Data With MD1003 (High Dose Pharmaceutical Grade Biotin) in X-Linked Adrenoleukodystrophy to be Presented at European Committee for Treatment and Research in Multiple Sclerosis 2016 58
Jul 26, 2016: Viking Therapeutics Announces Positive Top-Line Results from Proof-of-Concept Study of VK0214 in In Vivo Model of X-Linked Adrenoleukodystrophy (X-ALD) 58
Jul 06, 2016: Minoryx Therapeutics Initiates Phase 1 clinical trial of MIN-102 59
Jun 14, 2016: Minoryx Therapeutics appoints Dr. Uwe Meya as Chief Medical Officer 59
Apr 03, 2014: Minoryx receives Orphan Drug Designation for MIN-101 60
Appendix 62
Methodology 62
Coverage 62
Secondary Research 62
Primary Research 62
Expert Panel Validation 62
Contact Us 62
Disclaimer 63

List of Tables

Number of Products under Development for Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex), H2 2016 8
Number of Products under Development for Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - 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
Products under Development by Companies, H2 2016 15
Products under Investigation by Universities/Institutes, H2 2016 16
Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Pipeline by Applied Genetic Technologies Corp, H2 2016 17
Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Pipeline by bluebird bio Inc, H2 2016 18
Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Pipeline by MedDay SA, H2 2016 19
Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Pipeline by Minoryx Therapeutics sl, H2 2016 20
Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Pipeline by Novartis AG, H2 2016 21
Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Pipeline by Pfizer Inc, H2 2016 22
Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Pipeline by ReceptoPharm Inc, H2 2016 23
Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Pipeline by Viking Therapeutics 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
Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Dormant Projects, H2 2016 56