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Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)-Pipeline Review, H1 2015

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)-Pipeline Review, H1 2015


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Executive Summary

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)-Pipeline Review, H1 2015

Summary

Global Markets Direct's, 'Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)-Pipeline Review, H1 2015', provides an overview of the Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)'s therapeutic pipeline.

This report provides comprehensive information on the therapeutic development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome), complete with comparative analysis at various stages, therapeutics assessment by drug target, mechanism of action (MoA), route of administration (RoA) and molecule type, along with latest updates, and featured news and press releases. It also reviews key players involved in the therapeutic development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) and special features on late-stage 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, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources, put together by Global Markets Direct's team. Drug profiles/records featured in the report undergoes periodic updation following a stringent set of processes that ensures that all the profiles are updated with the latest set of information. Additionally, processes including live news & deals tracking, browser based alert-box and clinical trials registries tracking ensure that the most recent developments are captured on a real time basis.

The report enhances decision making capabilities and help to create effective counter strategies to gain competitive advantage. It strengthens R&D pipelines by identifying new targets and MOAs to produce first-in-class and best-in-class products.

Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data for the indicated disease.

Scope

The report provides a snapshot of the global therapeutic landscape of Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)

The report reviews key pipeline products under drug profile section which includes, product description, MoA and R&D brief, licensing and collaboration details & other developmental activities

The report reviews key players involved in the therapeutics development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) and enlists all their major and minor projects

The report summarizes all the dormant and discontinued pipeline projects

A review of the Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) products under development by companies and universities/research institutes based on information derived from company and industry-specific sources

Pipeline products coverage based on various stages of development ranging from pre-registration till discovery and undisclosed stages

A detailed assessment of monotherapy and combination therapy pipeline projects

Coverage of the Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) pipeline on the basis of target, MoA, route of administration and molecule type

Latest news and deals relating related to pipeline products

Reasons To Buy

Provides strategically significant competitor information, analysis, and insights to formulate effective R&D development strategies

Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage

Develop strategic initiatives by understanding the focus areas of leading companies

Identify and understand important and diverse types of therapeutics under development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)

Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline

Devise corrective measures for pipeline projects by understanding Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 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

Table of Contents

Table of Contents 2

List of Tables 5

List of Figures 5

Introduction 6

Global Markets Direct Report Coverage 6

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) Overview 7

Therapeutics Development 8

Pipeline Products for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)-Overview 8

Pipeline Products for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)-Comparative Analysis 9

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)-Therapeutics under Development by Companies 10

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)-Therapeutics under Investigation by Universities/Institutes 11

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)-Pipeline Products Glance 12

Clinical Stage Products 12

Early Stage Products 13

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)-Products under Development by Companies 14

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)-Products under Investigation by Universities/Institutes 15

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)-Companies Involved in Therapeutics Development 16

ArmaGen Technologies, Inc. 16

Axcentua Pharmaceuticals AB 17

BioMarin Pharmaceutical Inc. 18

Dorphan S.A. 19

Laboratorios Del Dr. Esteve S.A. 20

Shire Plc 21

Synageva BioPharma Corp. 22

uniQure N.V. 23

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)-Therapeutics Assessment 24

Assessment by Monotherapy Products 24

Assessment by Target 25

Assessment by Mechanism of Action 27

Assessment by Route of Administration 29

Assessment by Molecule Type 31

Drug Profiles 33

ABX-101-Drug Profile 33

Product Description 33

Mechanism of Action 33

R&D Progress 33

ABX-102-Drug Profile 34

Product Description 34

Mechanism of Action 34

R&D Progress 34

AGT-184-Drug Profile 35

Product Description 35

Mechanism of Action 35

R&D Progress 35

AMT-110-Drug Profile 36

Product Description 36

Mechanism of Action 36

R&D Progress 36

AXP-10711-Drug Profile 37

Product Description 37

Mechanism of Action 37

R&D Progress 37

BMN-250-Drug Profile 39

Product Description 39

Mechanism of Action 39

R&D Progress 39

Gene Therapy for Sanfilippo A Syndrome-Drug Profile 40

Product Description 40

Mechanism of Action 40

R&D Progress 40

HGT-3010-Drug Profile 41

Product Description 41

Mechanism of Action 41

R&D Progress 41

SAF-301-Drug Profile 42

Product Description 42

Mechanism of Action 42

R&D Progress 42

SBC-103-Drug Profile 43

Product Description 43

Mechanism of Action 43

R&D Progress 43

SHP-610-Drug Profile 44

Product Description 44

Mechanism of Action 44

R&D Progress 44

Small Molecule for Sanfilippo Syndrome-Drug Profile 45

Product Description 45

Mechanism of Action 45

R&D Progress 45

Small Molecule to Target Alpha-N-Acetylglucosaminidase for MPSIIIB-Drug Profile 46

Product Description 46

Mechanism of Action 46

R&D Progress 46

Stem Cell Therapy for Sanfilippo Syndrome-Drug Profile 47

Product Description 47

Mechanism of Action 47

R&D Progress 47

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)-Recent Pipeline Updates 48

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)-Dormant Projects 52

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)-Product Development Milestones 53

Featured News & Press Releases 53

Dec 10, 2014: FDA Grants BioMarin Orphan Drug Designation for NAGLU Fusion Protein, BMN 250, for the Treatment of MPS IIIB (Sanfilippo Syndrome Type B) 53

Apr 02, 2014: Orphan designation granted for MPSIII 53

Feb 11, 2014: BioMarin Announces Selection of NAGLU Fusion Protein Drug Development Candidate BMN 250 for the Treatment of Sanfilippo B 54

Jul 25, 2013: New stem cell gene therapy gives hope to prevent inherited neurological disease 55

Apr 11, 2013: Axcentua granted patent on ten solid forms of genistein 56

Appendix 57

Methodology 57

Coverage 57

Secondary Research 57

Primary Research 57

Expert Panel Validation 57

Contact Us 57

Disclaimer 58

List of Tables

Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ), H1 2015 8

Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)-Comparative Analysis, H1 2015 9

Number of Products under Development by Companies, H1 2015 10

Number of Products under Investigation by Universities/Institutes, H1 2015 11

Comparative Analysis by Clinical Stage Development, H1 2015 12

Comparative Analysis by Early Stage Development, H1 2015 13

Products under Development by Companies, H1 2015 14

Products under Investigation by Universities/Institutes, H1 2015 15

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)-Pipeline by ArmaGen Technologies, Inc., H1 2015 16

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)-Pipeline by Axcentua Pharmaceuticals AB, H1 2015 17

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)-Pipeline by BioMarin Pharmaceutical Inc., H1 2015 18

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)-Pipeline by Dorphan S.A., H1 2015 19

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)-Pipeline by Laboratorios Del Dr. Esteve S.A., H1 2015 20

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)-Pipeline by Shire Plc, H1 2015 21

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)-Pipeline by Synageva BioPharma Corp., H1 2015 22

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)-Pipeline by uniQure N.V., H1 2015 23

Assessment by Monotherapy Products, H1 2015 24

Number of Products by Stage and Target, H1 2015 26

Number of Products by Stage and Mechanism of Action, H1 2015 28

Number of Products by Stage and Route of Administration, H1 2015 30

Number of Products by Stage and Molecule Type, H1 2015 32

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) Therapeutics-Recent Pipeline Updates, H1 2015 48

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)-Dormant Projects, H1 2015 52

List of Figures

Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ), H1 2015 8

Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)-Comparative Analysis, H1 2015 9

Number of Products under Development by Companies, H1 2015 10

Comparative Analysis by Clinical Stage Development, H1 2015 12

Comparative Analysis by Early Stage Products, H1 2015 13

Assessment by Monotherapy Products, H1 2015 24

Number of Products by Top 10 Targets, H1 2015 25

Number of Products by Stage and Top 10 Targets, H1 2015 26

Number of Products by Top 10 Mechanism of Actions, H1 2015 27

Number of Products by Stage and Top 10 Mechanism of Actions, H1 2015 28

Number of Products by Top 10 Routes of Administration, H1 2015 29

Number of Products by Stage and Top 10 Routes of Administration, H1 2015 30

Number of Products by Top 10 Molecule Types, H1 2015 31

Number of Products by Stage and Top 10 Molecule Types, H1 2015 32

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ArmaGen Technologies, Inc.

Axcentua Pharmaceuticals AB

BioMarin Pharmaceutical Inc.

Dorphan S.A.

Laboratorios Del Dr. Esteve S.A.

Shire Plc

Synageva BioPharma Corp.

uniQure N.V.

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) Therapeutic Products under Development, Key Players in Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) Therapeutics, Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) Pipeline Overview, Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) Pipeline, Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) Pipeline Assessment


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