Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) – Pipeline Review, H2 2017

Global Markets Direct’s, ‘Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) – Pipeline Review, H2 2017’, 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

Pfizer Inc

ReceptoPharm Inc

SOM Biotech SL

Viking Therapeutics Inc

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

Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Overview 7

Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Therapeutics Development 8

Pipeline Overview 8

Pipeline by Companies 9

Pipeline by Universities/Institutes 11

Products under Development by Companies 12

Products under Development by Universities/Institutes 13

Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Therapeutics Assessment 14

Assessment by Target 14

Assessment by Mechanism of Action 16

Assessment by Route of Administration 18

Assessment by Molecule Type 20

Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Companies Involved in Therapeutics Development 22

Applied Genetic Technologies Corp 22

bluebird bio Inc 22

MedDay SA 23

Minoryx Therapeutics sl 23

Pfizer Inc 24

ReceptoPharm Inc 24

SOM Biotech SL 25

Viking Therapeutics Inc 25

Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Drug Profiles 26

biotin - Drug Profile 26

Product Description 26

Mechanism Of Action 26

R&D Progress 26

DRX-065 - Drug Profile 30

Product Description 30

Mechanism Of Action 30

R&D Progress 30

DUOC-01 - Drug Profile 31

Product Description 31

Mechanism Of Action 31

R&D Progress 31

Gene Therapy to Activate ABCD1 for Adrenoleukodystrophy - Drug Profile 32

Product Description 32

Mechanism Of Action 32

R&D Progress 32

Lenti-D - Drug Profile 33

Product Description 33

Mechanism Of Action 33

R&D Progress 33

MIN-102 - Drug Profile 37

Product Description 37

Mechanism Of Action 37

R&D Progress 37

OP-101 - Drug Profile 39

Product Description 39

Mechanism Of Action 39

R&D Progress 39

pioglitazone hydrochloride - Drug Profile 40

Product Description 40

Mechanism Of Action 40

R&D Progress 40

RPI-78M - Drug Profile 41

Product Description 41

Mechanism Of Action 41

R&D Progress 41

sobetirome - Drug Profile 43

Product Description 43

Mechanism Of Action 43

R&D Progress 43

SOM-1201 - Drug Profile 45

Product Description 45

Mechanism Of Action 45

R&D Progress 45

Stem Cell Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy - Drug Profile 46

Product Description 46

Mechanism Of Action 46

R&D Progress 46

temsirolimus - Drug Profile 47

Product Description 47

Mechanism Of Action 47

R&D Progress 47

VK-0214 - Drug Profile 50

Product Description 50

Mechanism Of Action 50

R&D Progress 50

VK-2809 - Drug Profile 53

Product Description 53

Mechanism Of Action 53

R&D Progress 53

Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Dormant Projects 58

Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Product Development Milestones 59

Featured News & Press Releases 59

Oct 23, 2017: Viking Therapeutics Presents Results from Proof-of-Concept Study of VK0214 in In Vivo Model of X-Linked Adrenoleukodystrophy at the 87th Annual Meeting of the American Thyroid Association 59

Oct 04, 2017: Viking Therapeutics Announces Top-Line Results from Proof-of-Concept Study of VK0214 in In Vivo Model of X-Linked Adrenoleukodystrophy (X-ALD) 60

Sep 28, 2017: NeuroVia Appoints Daniel Bradbury and Edward Kaye to its Board of Directors 60

Jul 21, 2017: SOM Biotech Opens New Office in US for Development of Adrenoleukodystrophy Drug, SOM1201 62

Mar 21, 2017: Minoryx Therapeutics successfully completes phase 1 clinical trial for lead candidate MIN-102 62

Feb 22, 2017: Minoryx Therapeutics receives Orphan Drug Designation from the US FDA for its lead candidate MIN-102 63

Jan 18, 2017: Minoryx Therapeutics further strengthens its Scientific Advisory Board (SAB) 63

Dec 14, 2016: Minoryx Therapeutics receives European Orphan Drug Designation for its lead candidate MIN-102 65

Dec 06, 2016: Viking Therapeutics Receives Orphan Drug Designation for VK0214 from the U.S. Food and Drug Administration for the Treatment of X-Linked Adrenoleukodystrophy 65

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 66

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) 66

Jul 06, 2016: Minoryx Therapeutics Initiates Phase 1 clinical trial of MIN-102 67

Jun 14, 2016: Minoryx Therapeutics appoints Dr. Uwe Meya as Chief Medical Officer 67

Apr 03, 2014: Minoryx receives Orphan Drug Designation for MIN-101 68

Appendix 70

Methodology 70

Coverage 70

Secondary Research 70

Primary Research 70

Expert Panel Validation 70

Contact Us 70

Disclaimer 71

List of Tables

List of Tables

Number of Products under Development for Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex), H2 2017 8

Number of Products under Development by Companies, H2 2017 10

Number of Products under Development by Universities/Institutes, H2 2017 11

Products under Development by Companies, H2 2017 12

Products under Development by Universities/Institutes, H2 2017 13

Number of Products by Stage and Target, H2 2017 15

Number of Products by Stage and Mechanism of Action, H2 2017 17

Number of Products by Stage and Route of Administration, H2 2017 19

Number of Products by Stage and Molecule Type, H2 2017 21

Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) – Pipeline by Applied Genetic Technologies Corp, H2 2017 22

Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) – Pipeline by bluebird bio Inc, H2 2017 23

Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) – Pipeline by MedDay SA, H2 2017 23

Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) – Pipeline by Minoryx Therapeutics sl, H2 2017 23

Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) – Pipeline by Pfizer Inc, H2 2017 24

Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) – Pipeline by ReceptoPharm Inc, H2 2017 24

Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) – Pipeline by SOM Biotech SL, H2 2017 25

Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) – Pipeline by Viking Therapeutics Inc, H2 2017 25

Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) – Dormant Projects, H2 2017 58

List of Figures

List of Figures

Number of Products under Development for Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex), H2 2017 8

Number of Products under Development by Companies, H2 2017 9

Number of Products under Development by Universities/Institutes, H2 2017 11

Number of Products by Targets, H2 2017 14

Number of Products by Stage and Targets, H2 2017 14

Number of Products by Mechanism of Actions, H2 2017 16

Number of Products by Stage and Mechanism of Actions, H2 2017 16

Number of Products by Routes of Administration, H2 2017 18

Number of Products by Stage and Routes of Administration, H2 2017 18

Number of Products by Molecule Types, H2 2017 20

Number of Products by Stage and Molecule Types, H2 2017 20

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