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Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline Review, H2 2015 Is Released

Press Release   •   Jan 30, 2016 12:00 IST

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline Review, H2 2015 Summary Global Markets Direct s, Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline Review, H2 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 Directs 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 Directs 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.

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Table of Contents

Introduction 7

Global Markets Direct Report Coverage 7

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Overview 8

Therapeutics Development 9

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

Pipeline Products for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Comparative Analysis 10

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Therapeutics under Development by Companies 11

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Therapeutics under Investigation by Universities/Institutes 12

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline Products Glance 13

Clinical Stage Products 13

Early Stage Products 14

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Products under Development by Companies 15

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Products under Investigation by Universities/Institutes 16

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Companies Involved in Therapeutics Development 17

Abeona Therapeutics, Inc. 17

Alexion Pharmaceuticals, Inc. 18

ArmaGen,Inc. 19

Axcentua Pharmaceuticals AB 20

BioMarin Pharmaceutical Inc. 21

Dorphan S.A. 22

Laboratorios Del Dr. Esteve S.A. 23

Lysogene 24

Phoenix Nest, Inc. 25

Shire Plc 26

Swedish Orphan Biovitrum AB 27

uniQure N.V. 28

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Therapeutics Assessment 29

Assessment by Monotherapy Products 29

Assessment by Target 30

Assessment by Mechanism of Action 32

Assessment by Route of Administration 34

Assessment by Molecule Type 36

Drug Profiles 38

ABO-101 - Drug Profile 38

Product Description 38

Mechanism of Action 38

R&D Progress 38

ABO-102 - Drug Profile 39

Product Description 39

Mechanism of Action 39

R&D Progress 39

AGT-184 - Drug Profile 40

Product Description 40

Mechanism of Action 40

R&D Progress 40

AMT-110 - Drug Profile 41

Product Description 41

Mechanism of Action 41

R&D Progress 41

AXP-10711 - Drug Profile 43

Product Description 43

Mechanism of Action 43

R&D Progress 43

BMN-250 - Drug Profile 45

Product Description 45

Mechanism of Action 45

R&D Progress 45

Gene Therapy to Activate Sulphamidase for Metabolic Disorders - Drug Profile 46

Product Description 46

Mechanism of Action 46

R&D Progress 46

LYS-SAF302 - Drug Profile 48

Product Description 48

Mechanism of Action 48

R&D Progress 48

Recombinant Enzyme to Replace N-Acetylgalactosamine-6-Sulfatase for Mucopolysaccharidosis Type IIID - Drug Profile 50

Product Description 50

Mechanism of Action 50

R&D Progress 50

SBC-103 - Drug Profile 51

Product Description 51

Mechanism of Action 51

R&D Progress 51

SHP-610 - Drug Profile 52

Product Description 52

Mechanism of Action 52

R&D Progress 52

Small Molecule for Sanfilippo Syndrome - Drug Profile 53

Product Description 53

Mechanism of Action 53

R&D Progress 53

Small Molecule for Sanfilippo Syndrome - Drug Profile 54

Product Description 54

Mechanism of Action 54

R&D Progress 54

Small Molecule to Target Alpha-N-Acetylglucosaminidase for MPSIIIB - Drug Profile 55

Product Description 55

Mechanism of Action 55

R&D Progress 55

SOBI-003 - Drug Profile 56

Product Description 56

Mechanism of Action 56

R&D Progress 56

Stem Cell Therapy for Sanfilippo Syndrome - Drug Profile 57

Product Description 57

Mechanism of Action 57

R&D Progress 57

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Recent Pipeline Updates 58

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Dormant Projects 63

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Product Development Milestones 64

Featured News & Press Releases 64

May 20, 2015: PlasmaTech Biopharmaceuticals Announces Orphan Drug and Rare Pediatric Disease Designations For ABX-101 From FDA 64

May 20, 2015: PlasmaTech Biopharmaceuticals Announces Orphan Drug and Rare Pediatric Disease Designations for ABX-102 from FDA 64

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) 64

Apr 02, 2014: Orphan designation granted for MPSIII 65

Feb 11, 2014: BioMarin Announces Selection of NAGLU Fusion Protein Drug Development Candidate BMN 250 for the Treatment of Sanfilippo B 65

Jul 25, 2013: New stem cell gene therapy gives hope to prevent inherited neurological disease 66

Apr 11, 2013: Axcentua granted patent on ten solid forms of genistein 67

Appendix 68

Methodology 68

Coverage 68

Secondary Research 68

Primary Research 68

Expert Panel Validation 68

Contact Us 68

Disclaimer 69

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