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

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1 Published on Market Research Reports Inc. ( Home > Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Pipeline Review, H Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder- Addison Complex) - Pipeline Review, H Publication ID: GMD Publication November 30, 2016 Date: Pages: 63 Publisher: Global Markets Direct Countries: Global [1] $2, Publication License Type * SINGLE USER LICENSE (PDF), $2, SITE LICENSE (PDF), $4, GLOBAL LICENSE (PDF), $6, Please choose the suitable license type from above. More details are at given under tab "Report

2 License Types" below. Add to cart Diseases [2] Pharma & Healthcare [3] Description: Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Pipeline Review, H 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

3 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)

4 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 counterstrategies 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 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

5 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 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 Drug Profile 41

6 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 Drug Profile 45 Product Description 45 Mechanism Of Action 45 R&D Progress 45 OP 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 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

7 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 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 Jun 14, 2016: Minoryx Therapeutics appoints Dr. Uwe Meya as Chief Medical Officer 59 Apr 03, 2014: Minoryx receives Orphan Drug Designation for MIN 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), H Number of Products under Development for Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Comparative Analysis, H Number of Products under Development by Companies, H Number of Products under Investigation by Universities/Institutes, H Comparative Analysis by Late Stage Development, H Comparative Analysis by Clinical Stage Development, H Comparative Analysis by Early Stage Development, H Products under Development by Companies, H Products under Investigation by Universities/Institutes, H Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Pipeline by Applied Genetic Technologies Corp, H Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Pipeline by bluebird bio Inc, H Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Pipeline by MedDay SA, H Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Pipeline by Minoryx Therapeutics sl, H

8 Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Pipeline by Novartis AG, H Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Pipeline by Pfizer Inc, H Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Pipeline by ReceptoPharm Inc, H Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Pipeline by Viking Therapeutics Inc, H Assessment by Monotherapy Products, H Number of Products by Stage and Target, H Number of Products by Stage and Mechanism of Action, H Number of Products by Stage and Route of Administration, H Number of Products by Stage and Molecule Type, H Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Dormant Projects, H List of Figures Number of Products under Development for Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex), H Number of Products under Development for Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Comparative Analysis, H Number of Products under Development by Companies, H Number of Products under Investigation by Universities/Institutes, H Comparative Analysis by Clinical Stage Development, H Comparative Analysis by Early Stage Products, H Assessment by Monotherapy Products, H Number of Products by Targets, H Number of Products by Stage and Targets, H Number of Products by Mechanism of Actions, H Number of Products by Stage and Mechanism of Actions, H Number of Products by Routes of Administration, H Number of Products by Stage and Routes of Administration, H Number of Products by Molecule Types, H Number of Products by Stage and Molecule Types, H Companies Mentioned: Applied Genetic Technologies Corp bluebird bio Inc MedDay SA Minoryx Therapeutics sl

9 Novartis AG Pfizer Inc ReceptoPharm Inc Viking Therapeutics Inc License Types: Single User License (PDF) This license allows for use of a publication by one person. This person may print out a single copy of the publication. This person can include information given in the publication in presentations and internal reports by providing full copyright credit to the publisher. This person cannot share the publication (or any information contained therein) with any other person or persons. Unless a Enterprise License is purchased, a Single User License must be purchased for every person that wishes to use the publication within the same organization. Customers who infringe these license terms are liable for a Global license fee. Site License (PDF)* This license allows for use of a publication by all users within one corporate location, e.g. a regional office. These users may print out a single copy of the publication. These users can include information given in the publication in presentations and internal reports by providing full copyright credit to the publisher. These users cannot share the publication (or any information contained therein) with any other person or persons outside the corporate location for which the publication is purchased. Unless a Enterprise License is purchased, a Site User License must be purchased for every corporate location by an organization that wishes to use the publication within the same organization. Customers who infringe these license terms are liable for a Global license fee. Global License (PDF)* This license allows for use of a publication by unlimited users within the purchasing organization e.g. all employees of a single company. Each of these people may use the publication on any computer, and may print out the report, but may not share the publication (or any information contained therein) with any other person or

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