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

Global Markets Direct’s, ‘Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) – Pipeline Review, H2 2016’, provides an overview of the Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex), 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 Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) 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 Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex)

The report reviews pipeline therapeutics for Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) 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 Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) therapeutics and enlists all their major and minor projects

The report assesses Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) 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 Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex)

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 Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex)

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 Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) 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

Applied Genetic Technologies Corp

bluebird bio Inc

MedDay SA

Minoryx Therapeutics sl

Novartis AG

Pfizer Inc

ReceptoPharm Inc

Viking Therapeutics Inc

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

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

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

List of Figures

List of Figures

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 Clinical Stage Development, H2 2016 13

Comparative Analysis by Early Stage Products, H2 2016 14

Assessment by Monotherapy Products, H2 2016 25

Number of Products by Targets, H2 2016 26

Number of Products by Stage and Targets, H2 2016 26

Number of Products by Mechanism of Actions, H2 2016 28

Number of Products by Stage and Mechanism of Actions, H2 2016 28

Number of Products by Routes of Administration, H2 2016 30

Number of Products by Stage and Routes of Administration, H2 2016 30

Number of Products by Molecule Types, H2 2016 32

Number of Products by Stage and Molecule Types, H2 2016 32

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