Mucopolysaccharidosis III (MPS III) (Sanfilippo

Syndrome) - Pipeline Review, H2 2015 Is Released

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 1

Devise corrective measures for pipeline projects by understanding Mucopolysaccharidosis III (MPS III)
(Sanfilippo Syndrome) pipeline depth and focus of Indication therapeutics - Develop and design inlicensing 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.
with
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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
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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
4

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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