Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline Review, H2 2016

 


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Abstract

 
Mucopolysaccharidosis type III (MPS III), also known as Sanfilippo syndrome, is an inherited metabolic disease caused by an absence or malfunctioning of certain enzymes needed to breakdown molecules called glycosaminoglycans (GAG). Symptoms include seizures, hyperactivity, liver and spleen enlargement, severe diarrhea or constipation and enlargement of tonsils and adenoids. Treatment includes enzyme replacement therapy (ERT). 

Description

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline Review, H2 2016, provides an overview of the Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) (Metabolic Disorders) pipeline landscape.

Mucopolysaccharidosis type III (MPS III), also known as Sanfilippo syndrome, is an inherited metabolic disease caused by an absence or malfunctioning of certain enzymes needed to breakdown molecules called glycosaminoglycans (GAG). Symptoms include seizures, hyperactivity, liver and spleen enlargement, severe diarrhea or constipation and enlargement of tonsils and adenoids. Treatment includes enzyme replacement therapy (ERT).
 

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Report Source Information

Supplied by: Research and Experts 
Date Published: 2016-12-21 

Report: Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline Review, H2 2016

 

Contents

Table of Contents
Table of Contents 2
List of Tables 5
List of Figures 6
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 SA 22
Laboratorios Del Dr Esteve SA 23
Lysogene SAS 24
Swedish Orphan Biovitrum AB 25
UniQure NV 26
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Therapeutics Assessment 27
Assessment by Monotherapy Products 27
Assessment by Target 28
Assessment by Mechanism of Action 30
Assessment by Route of Administration 32
Assessment by Molecule Type 34
Drug Profiles 36
ABO-101 - Drug Profile 36
Product Description 36
Mechanism Of Action 36
R&D Progress 36
ABO-102 - Drug Profile 38
Product Description 38
Mechanism Of Action 38
R&D Progress 38
AGT-184 - Drug Profile 41
Product Description 41
Mechanism Of Action 41
R&D Progress 41
AMT-110 - Drug Profile 42
Product Description 42
Mechanism Of Action 42
R&D Progress 42
AXP-10711 - Drug Profile 44
Product Description 44
Mechanism Of Action 44
R&D Progress 44
BMN-250 - Drug Profile 46
Product Description 46
Mechanism Of Action 46
R&D Progress 46
EGT-101 - Drug Profile 48
Product Description 48
Mechanism Of Action 48
R&D Progress 48
EGT-201 - Drug Profile 49
Product Description 49
Mechanism Of Action 49
R&D Progress 49
lesinidase alfa - Drug Profile 50
Product Description 50
Mechanism Of Action 50
R&D Progress 50
LYSSAF-301 - Drug Profile 53
Product Description 53
Mechanism Of Action 53
R&D Progress 53
LYSSAF-302 - Drug Profile 54
Product Description 54
Mechanism Of Action 54
R&D Progress 54
Proteins for Mucopolysaccharidosis Type IIIC - Drug Profile 55
Product Description 55
Mechanism Of Action 55
R&D Progress 55
Recombinant N-Acetylgalactosamine-6-Sulfatase Replacement for Mucopolysaccharidosis Type IIID - Drug Profile 56
Product Description 56
Mechanism Of Action 56
R&D Progress 56
Small Molecule for Sanfilippo Syndrome - Drug Profile 57
Product Description 57
Mechanism Of Action 57
R&D Progress 57
Small Molecule to Target Alpha-N-Acetylglucosaminidase for MPSIIIB - Drug Profile 58
Product Description 58
Mechanism Of Action 58
R&D Progress 58
SOBI-003 - Drug Profile 59
Product Description 59
Mechanism Of Action 59
R&D Progress 59
Stem Cell Therapy for Mucopolysaccharidosis Type IIIA - Drug Profile 60
Product Description 60
Mechanism Of Action 60
R&D Progress 60
Stem Cell Therapy for Sanfilippo Syndrome - Drug Profile 61
Product Description 61
Mechanism Of Action 61
R&D Progress 61
Stem Cell Therapy to Activate NAGLU for Mucopolysaccharidosis IIIB - Drug Profile 62
Product Description 62
Mechanism Of Action 62
R&D Progress 62
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Dormant Projects 63
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Discontinued Products 64
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Product Development Milestones 65
Featured News & Press Releases 65
Oct 19, 2016: European Commission Grants SOBI003 Orphan Designation for the Treatment of MPS IIIA 65
May 24, 2016: Abeona Therapeutics Announces FDA Allowance of Investigational New Drug for Phase 1/2 Clinical Study With ABO-101 Gene Therapy for Patients With Sanfilippo Syndrome Type B (MPS IIIB) 65
Feb 25, 2016: ESTEVE Provides Update On EGT-201 For Sanfilippo B Syndrome 66
Jan 11, 2016: Abeona Therapeutics Announces Initial European Regulatory Approval for Phase 1/2 Gene Therapy Clinical Study for Patients With Sanfilippo Syndrome 67
May 20, 2015: PlasmaTech Biopharmaceuticals Announces Orphan Drug and Rare Pediatric Disease Designations For ABX-101 From FDA 68
Apr 02, 2014: Orphan designation granted for MPSIII 68
Jul 25, 2013: New stem cell gene therapy gives hope to prevent inherited neurological disease 68
Apr 11, 2013: Axcentua granted patent on ten solid forms of genistein 69
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 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ), H2 2016 9
Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Comparative Analysis, H2 2016 10
Number of Products under Development by Companies, H2 2016 11
Number of Products under Investigation by Universities/Institutes, H2 2016 12
Comparative Analysis by Clinical Stage Development, H2 2016 13
Comparative Analysis by Early Stage Development, H2 2016 14
Products under Development by Companies, H2 2016 15
Products under Investigation by Universities/Institutes, H2 2016 16
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Abeona Therapeutics Inc, H2 2016 17
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Alexion Pharmaceuticals Inc, H2 2016 18
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by ArmaGen Inc, H2 2016 19
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Axcentua Pharmaceuticals AB, H2 2016 20
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by BioMarin Pharmaceutical Inc, H2 2016 21
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Dorphan SA, H2 2016 22
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Laboratorios Del Dr Esteve SA, H2 2016 23
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Lysogene SAS, H2 2016 24
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Swedish Orphan Biovitrum AB, H2 2016 25
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by UniQure NV, H2 2016 26
Assessment by Monotherapy Products, H2 2016 27
Number of Products by Stage and Target, H2 2016 29
Number of Products by Stage and Mechanism of Action, H2 2016 31
Number of Products by Stage and Route of Administration, H2 2016 33
Number of Products by Stage and Molecule Type, H2 2016 35
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Dormant Projects, H2 2016 63
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Discontinued Products, H2 2016 64
 

List of Figures

List of Figures
Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ), H2 2016 9
Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Comparative Analysis, H2 2016 10
Number of Products under Development by Companies, H2 2016 11
Comparative Analysis by Early Stage Products, H2 2016 14
Assessment by Monotherapy Products, H2 2016 27
Number of Products by Targets, H2 2016 28
Number of Products by Stage and Targets, H2 2016 28
Number of Products by Top 10 Mechanism of Actions, H2 2016 30
Number of Products by Stage and Top 10 Mechanism of Actions, H2 2016 30
Number of Products by Routes of Administration, H2 2016 32
Number of Products by Stage and Routes of Administration, H2 2016 32
Number of Products by Molecule Types, H2 2016 34
Number of Products by Stage and Molecule Types, H2 2016 34
 

Scope


- The pipeline guide provides a snapshot of the global therapeutic landscape of Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) (Metabolic Disorders).
- The pipeline guide reviews pipeline therapeutics for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) (Metabolic 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 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) (Metabolic Disorders) therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) (Metabolic 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 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) (Metabolic Disorders)

 

Highlights

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline Review, H2 2016, provides comprehensive information on the therapeutics under development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) (Metabolic 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 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) (Metabolic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase II, IND/CTA Filed, Preclinical and Discovery stages are 7, 1, 7 and 3 respectively. Similarly, the Universities portfolio in Preclinical stages comprises 1 molecules, respectively.

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) (Metabolic 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.

 

Classification

Language Code: EN 
Region: Global 
Report Type: Market Report 
Industry: Life Sciences 
Sector: Genetic Disorders 
Pages: 71 

Companies Featured

Abeona Therapeutics Inc Alexion Pharmaceuticals Inc ArmaGen Inc Axcentua Pharmaceuticals AB BioMarin Pharmaceutical Inc Dorphan SA Laboratorios Del Dr Esteve SA Lysogene SAS Swedish Orphan Biovitrum AB UniQure NV

 

Reasons to buy this report


- 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 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) (Metabolic 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 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) (Metabolic 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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Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline Review, H2 2016

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The global economy is the economy of all the world's countries. The total world population now stands at over 7 billion and the global unemployment rate is 9% with approximately half the world's population paid less than $2 a day. 

The global economy is picking up again after five years of uncertaintity and slow economic growth. The IMF is predicting global growth of 3.7% in 2014 rising to 3.9% in 2015. However, much uncertainity remains. Low inflation in developed economies means that these economies take longer to deal with debt burdens and are at increases risk of deflation. 

The second half of 2013 saw growth in the US and European economies (particularly UK, Germany and France).  

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Developing economies such as China and India will continue to have strong growth in 2014 and 2015. China has benefitted from increased investment with the Chinese govenment now focused on quality growth of 7.5% in 2014. India which is less reliant on exports expects growth of 5.4% in 2014 and 6.4% in 2015.

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Genetic Disorders

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The healthcare industry is changing rapidly due to changes in legislation (US Affordable Healthcare Act),  advances in IT and more pressures on governments and healthcare providers due to rising costs. 

According to a recent report by the OECD healthcare spending has fallen in one third of OECD member states due to the credit crunch and members are facing up to the challenge of providing greater care with fewer resources. Spending on healthcare has increased in Japan, Israel and Korea while it has dropped dramatically in Greece and Ireland.

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