Neurofibromatoses Type I (Von Recklinghausens Disease)-Pipeline Review, H2 2016

Neurofibromatoses Type I (Von Recklinghausens Disease)-Pipeline Review, H2 2016


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Executive Summary

Neurofibromatoses Type I (Von Recklinghausen's Disease)-Pipeline Review, H2 2016

Summary

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

Neurofibromatosis type 1 (NF1), also called von Recklinghausen's disease, is a rare genetic disorder characterized by the development of multiple noncancerous (benign) tumors of nerves and skin (neurofibromas). This is transmitted on chromosome 17 and is caused by mutation of the NF1 gene. Symptoms include liver enlargement, glioma, Lisch nodules and pheochromocytoma. Treatment includes pain medications, surgery, chemotherapy and radiation therapy.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Neurofibromatoses Type I-Pipeline Review, H2 2016, provides comprehensive information on the therapeutics under development for Neurofibromatoses Type I (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 Neurofibromatoses Type I (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Neurofibromatoses Type I (Von Recklinghausen's Disease) and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase II, Preclinical and Discovery stages are 1, 2 and 1 respectively. Similarly, the Universities portfolio in Discovery stages comprises 1 molecules, respectively.

Neurofibromatoses Type I (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 Neurofibromatoses Type I (Genetic Disorders).

The pipeline guide reviews pipeline therapeutics for Neurofibromatoses Type I (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 Neurofibromatoses Type I (Genetic Disorders) therapeutics and enlists all their major and minor projects.

The pipeline guide evaluates Neurofibromatoses Type I (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 Neurofibromatoses Type I (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 Neurofibromatoses Type I (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 Neurofibromatoses Type I (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.



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Table of Contents

Table of Contents 2

List of Tables 3

List of Figures 4

Introduction 5

Global Markets Direct Report Coverage 5

Neurofibromatoses Type I (Von Recklinghausen's Disease) Overview 6

Therapeutics Development 7

Pipeline Products for Neurofibromatoses Type I (Von Recklinghausen's Disease)-Overview 7

Pipeline Products for Neurofibromatoses Type I (Von Recklinghausen's Disease)-Comparative Analysis 8

Neurofibromatoses Type I (Von Recklinghausen's Disease)-Therapeutics under Development by Companies 9

Neurofibromatoses Type I (Von Recklinghausen's Disease)-Therapeutics under Investigation by Universities/Institutes 10

Neurofibromatoses Type I (Von Recklinghausen's Disease)-Pipeline Products Glance 11

Clinical Stage Products 11

Early Stage Products 12

Neurofibromatoses Type I (Von Recklinghausen's Disease)-Products under Development by Companies 13

Neurofibromatoses Type I (Von Recklinghausen's Disease)-Products under Investigation by Universities/Institutes 14

Neurofibromatoses Type I (Von Recklinghausen's Disease)-Companies Involved in Therapeutics Development 15

Alexion Pharmaceuticals Inc 15

AstraZeneca Plc 16

Celldex Therapeutics Inc 17

Neurofibromatoses Type I (Von Recklinghausen's Disease)-Therapeutics Assessment 18

Assessment by Monotherapy Products 18

Assessment by Target 19

Assessment by Mechanism of Action 21

Assessment by Route of Administration 23

Assessment by Molecule Type 25

Drug Profiles 27

asfotase alfa-Drug Profile 27

Product Description 27

Mechanism Of Action 27

R&D Progress 27

CDX-0158-Drug Profile 31

Product Description 31

Mechanism Of Action 31

R&D Progress 31

selumetinib sulfate-Drug Profile 33

Product Description 33

Mechanism Of Action 33

R&D Progress 33

Small Molecules for Neurofibromatoses Type I-Drug Profile 38

Product Description 38

Mechanism Of Action 38

R&D Progress 38

VAL-0524-Drug Profile 39

Product Description 39

Mechanism Of Action 39

R&D Progress 39

Neurofibromatoses Type I (Von Recklinghausen's Disease)-Dormant Projects 40

Appendix 41

Methodology 41

Coverage 41

Secondary Research 41

Primary Research 41

Expert Panel Validation 41

Contact Us 41

Disclaimer 42

List of Figures

Number of Products under Development for Neurofibromatoses Type I (Von Recklinghausen's Disease), H2 2016 7

Number of Products under Development for Neurofibromatoses Type I (Von Recklinghausen's Disease)-Comparative Analysis, H2 2016 8

Number of Products under Development by Companies, H2 2016 9

Comparative Analysis by Early Stage Products, H2 2016 12

Assessment by Monotherapy Products, H2 2016 18

Number of Products by Targets, H2 2016 19

Number of Products by Stage and Targets, H2 2016 19

Number of Products by Mechanism of Actions, H2 2016 21

Number of Products by Stage and Mechanism of Actions, H2 2016 21

Number of Products by Routes of Administration, H2 2016 23

Number of Products by Stage and Routes of Administration, H2 2016 23

Number of Products by Molecule Types, H2 2016 25

Number of Products by Stage and Molecule Types, H2 2016 25

List of Tables

Number of Products under Development for Neurofibromatoses Type I (Von Recklinghausen's Disease), H2 2016 7

Number of Products under Development for Neurofibromatoses Type I (Von Recklinghausen's Disease)-Comparative Analysis, H2 2016 8

Number of Products under Development by Companies, H2 2016 9

Number of Products under Investigation by Universities/Institutes, H2 2016 10

Comparative Analysis by Clinical Stage Development, H2 2016 11

Comparative Analysis by Early Stage Development, H2 2016 12

Products under Development by Companies, H2 2016 13

Products under Investigation by Universities/Institutes, H2 2016 14

Neurofibromatoses Type I (Von Recklinghausen's Disease)-Pipeline by Alexion Pharmaceuticals Inc, H2 2016 15

Neurofibromatoses Type I (Von Recklinghausen's Disease)-Pipeline by AstraZeneca Plc, H2 2016 16

Neurofibromatoses Type I (Von Recklinghausen's Disease)-Pipeline by Celldex Therapeutics Inc, H2 2016 17

Assessment by Monotherapy Products, H2 2016 18

Number of Products by Stage and Target, H2 2016 20

Number of Products by Stage and Mechanism of Action, H2 2016 22

Number of Products by Stage and Route of Administration, H2 2016 24

Number of Products by Stage and Molecule Type, H2 2016 26

Neurofibromatoses Type I (Von Recklinghausen's Disease)-Dormant Projects, H2 2016 40

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Alexion Pharmaceuticals Inc, AstraZeneca Plc, Celldex Therapeutics Inc

Neurofibromatoses Type I (Von Recklinghausens Disease) Therapeutic Products under Development, Key Players in Neurofibromatoses Type I (Von Recklinghausens Disease) Therapeutics, Neurofibromatoses Type I (Von Recklinghausens Disease) Pipeline Overview, Neurofibromatoses Type I (Von Recklinghausens Disease) Pipeline, Neurofibromatoses Type I (Von Recklinghausens Disease) Pipeline Assessment

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