Alpha-N-Acetylglucosaminidase (N-Acetyl-Alpha-Glucosaminidase or NAG or EC 3.2.1.50) - Pipeline Review, H1 2016

Published: Jun, 2016 | Pages: 54 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Alpha-N-Acetylglucosaminidase (N-Acetyl-Alpha-Glucosaminidase or NAG or EC 3.2.1.50) - Pipeline Review, H1 2016

Summary

Global Markets Direct's, 'Alpha-N-Acetylglucosaminidase (N-Acetyl-Alpha-Glucosaminidase or NAG or EC 3.2.1.50) - Pipeline Review, H1 2016', provides in depth analysis on Alpha-N-Acetylglucosaminidase (N-Acetyl-Alpha-Glucosaminidase or NAG or EC 3.2.1.50) targeted pipeline therapeutics. 

The report provides comprehensive information on the Alpha-N-Acetylglucosaminidase (N-Acetyl-Alpha-Glucosaminidase or NAG or EC 3.2.1.50), targeted therapeutics, complete with analysis by indications, stage of development, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in Alpha-N-Acetylglucosaminidase (N-Acetyl-Alpha-Glucosaminidase or NAG or EC 3.2.1.50) targeted therapeutics development and features dormant 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, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

The report 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.

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

Scope

- The report provides a snapshot of the global therapeutic landscape for Alpha-N-Acetylglucosaminidase (N-Acetyl-Alpha-Glucosaminidase or NAG or EC 3.2.1.50)
- The report reviews Alpha-N-Acetylglucosaminidase (N-Acetyl-Alpha-Glucosaminidase or NAG or EC 3.2.1.50) targeted therapeutics under development by companies and universities/research institutes based on information derived from company and industry-specific sources 
- The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages 
- The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities 
- The report reviews key players involved in Alpha-N-Acetylglucosaminidase (N-Acetyl-Alpha-Glucosaminidase or NAG or EC 3.2.1.50) targeted therapeutics and enlists all their major and minor projects 
- The report assesses Alpha-N-Acetylglucosaminidase (N-Acetyl-Alpha-Glucosaminidase or NAG or EC 3.2.1.50) targeted therapeutics based on mechanism of action (MoA), route of administration (RoA) and molecule type 
- The report summarizes all the dormant and discontinued pipeline projects 
- The report reviews latest news and deals related to Alpha-N-Acetylglucosaminidase (N-Acetyl-Alpha-Glucosaminidase or NAG or EC 3.2.1.50) targeted therapeutics

Reasons To Buy

- Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies
- Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage 
- Identify and understand the targeted therapy areas and indications for Alpha-N-Acetylglucosaminidase (N-Acetyl-Alpha-Glucosaminidase or NAG or EC 3.2.1.50)
- Identify the use of drugs for target identification and drug repurposing
- Identify potential new clients or partners in the target demographic
- Develop strategic initiatives by understanding the focus areas of leading companies 
- Plan mergers and acquisitions effectively by identifying key players and it's most promising pipeline therapeutics
- Devise corrective measures for pipeline projects by understanding Alpha-N-Acetylglucosaminidase (N-Acetyl-Alpha-Glucosaminidase or NAG or EC 3.2.1.50) development landscape 
- 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

 Table of Contents

Table of Contents 2
List of Tables 4
List of Figures 4
Introduction 5
Global Markets Direct Report Coverage 5
Alpha-N-Acetylglucosaminidase (N-Acetyl-Alpha-Glucosaminidase or NAG or EC 3.2.1.50) Overview 6
Therapeutics Development 7
Alpha-N-Acetylglucosaminidase (N-Acetyl-Alpha-Glucosaminidase or NAG or EC 3.2.1.50) - Products under Development by Stage of Development 7
Alpha-N-Acetylglucosaminidase (N-Acetyl-Alpha-Glucosaminidase or NAG or EC 3.2.1.50) - Products under Development by Therapy Area 8
Alpha-N-Acetylglucosaminidase (N-Acetyl-Alpha-Glucosaminidase or NAG or EC 3.2.1.50) - Products under Development by Indication 9
Alpha-N-Acetylglucosaminidase (N-Acetyl-Alpha-Glucosaminidase or NAG or EC 3.2.1.50) - Pipeline Products Glance 10
Late Stage Products 10
Early Stage Products 11
Alpha-N-Acetylglucosaminidase (N-Acetyl-Alpha-Glucosaminidase or NAG or EC 3.2.1.50) - Products under Development by Companies 12
Alpha-N-Acetylglucosaminidase (N-Acetyl-Alpha-Glucosaminidase or NAG or EC 3.2.1.50) - Therapeutics Assessment 14
Assessment by Monotherapy/Combination Products 14
Assessment by Mechanism of Action 15
Assessment by Route of Administration 17
Assessment by Molecule Type 19
Alpha-N-Acetylglucosaminidase (N-Acetyl-Alpha-Glucosaminidase or NAG or EC 3.2.1.50) - Companies Involved in Therapeutics Development 21
Abeona Therapeutics, Inc. 21
Alexion Pharmaceuticals, Inc. 22
BioMarin Pharmaceutical Inc. 23
Dorphan S.A. 24
Laboratorios Del Dr. Esteve S.A. 25
UniQure N.V. 26
Alpha-N-Acetylglucosaminidase (N-Acetyl-Alpha-Glucosaminidase or NAG or EC 3.2.1.50) - Drug Profiles 27
ABO-101 - Drug Profile 27
Product Description 27
Mechanism Of Action 27
R&D Progress 27
AMT-110 - Drug Profile 29
Product Description 29
Mechanism Of Action 29
R&D Progress 29
BMN-250 - Drug Profile 31
Product Description 31
Mechanism Of Action 31
R&D Progress 31
EGT-201 - Drug Profile 33
Product Description 33
Mechanism Of Action 33
R&D Progress 33
SBC-103 - Drug Profile 34
Product Description 34
Mechanism Of Action 34
R&D Progress 34
Small Molecule to Target Alpha-N-Acetylglucosaminidase for MPSIIIB - Drug Profile 37
Product Description 37
Mechanism Of Action 37
R&D Progress 37
Stem Cell Therapy to Activate NAG for Mucopolysaccharidosis III - Drug Profile 38
Product Description 38
Mechanism Of Action 38
R&D Progress 38
Alpha-N-Acetylglucosaminidase (N-Acetyl-Alpha-Glucosaminidase or NAG or EC 3.2.1.50) - Dormant Projects 39
Alpha-N-Acetylglucosaminidase (N-Acetyl-Alpha-Glucosaminidase or NAG or EC 3.2.1.50) - Featured News & Press Releases 40
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) 40
Apr 21, 2016: BioMarin Enrolls First Patient in Phase 1/2 Trial of NAGLU Fusion Protein BMN 250 for Treatment of MPS IIIB (Sanfilippo B Syndrome) 41
Mar 01, 2016: SBC-103 (rhNAGLU enzyme) Shows a 26.2 Percent Mean Reduction in Heparan Sulfate in Cerebrospinal Fluid at the Highest Dose Studied in Patients with Mucopolysaccharidosis IIIB in Phase 1/2 Study at Six Months 42
Feb 25, 2016: ESTEVE Provides Update On EGT-201 For Sanfilippo B Syndrome 43
Jan 11, 2016: Abeona Therapeutics Announces Initial European Regulatory Approval for Phase 1/2 Gene Therapy Clinical Study for Patients With Sanfilippo Syndrome 43
Sep 19, 2015: Positive Topline Results Announced From Phase I/II Trial in Sanfilippo B Syndrome Patients Using uniQure's Novel AAV5-Based Gene Therapy 44
Jun 04, 2015: Synageva BioPharma Completes Targeted Enrollment in Phase 1/2 Trial with SBC-103 for Mucopolysaccharidosis IIIB 45
May 20, 2015: PlasmaTech Biopharmaceuticals Announces Orphan Drug and Rare Pediatric Disease Designations For ABX-101 From FDA 46
Jan 26, 2015: Synageva BioPharma Announces Dosing Of Patients Commenced With SBC-103 In Phase 1/2 Study For Mucopolysaccharidosis IIIB And FDA Fast Track Designation Granted 46
Dec 15, 2014: Synageva BioPharma Announces Active Investigational New Drug Application For SBC-103 For The Treatment Of Mucopolysaccharidosis IIIB 47
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) 48
Feb 12, 2014: Synageva BioPharma Highlights SBC-103 Data at the Lysosomal Disease Network WORLD Symposium 48
Feb 11, 2014: BioMarin Announces Selection of NAGLU Fusion Protein Drug Development Candidate BMN 250 for the Treatment of Sanfilippo B 50
Jan 29, 2014: Synageva BioPharma Announces Data Presentations On SBC-103 at the 10th Annual Lysosomal Disease Network World Symposium 51
Jan 30, 2013: Synageva BioPharma To Present Scientific Data On SBC-103 At 9th Annual LDN WORLD Symposium 52
Appendix 53
Methodology 53
Coverage 53
Secondary Research 53
Primary Research 53
Expert Panel Validation 53
Contact Us 53
Disclaimer 54

List of Tables

Number of Products under Development for, H1 2016 7
Number of Products under Development by Therapy Area, H1 2016 8
Number of Products under Development by Indication, H1 2016 9
Comparative Analysis by Late Stage Development, H1 2016 10
Comparative Analysis by Early Stage Products, H1 2016 11
Number of Products under Development by Companies, H1 2016 12
Products under Development by Companies, H1 2016 13
Assessment by Monotherapy/Combination Products, H1 2016 14
Number of Products by Stage and Mechanism of Action, H1 2016 16
Number of Products by Stage and Route of Administration, H1 2016 18
Number of Products by Stage and Molecule Type, H1 2016 20
Pipeline by Abeona Therapeutics, Inc., H1 2016 21
Pipeline by Alexion Pharmaceuticals, Inc., H1 2016 22
Pipeline by BioMarin Pharmaceutical Inc., H1 2016 23
Pipeline by Dorphan S.A., H1 2016 24
Pipeline by Laboratorios Del Dr. Esteve S.A., H1 2016 25
Pipeline by UniQure N.V., H1 2016 26
Dormant Projects, H1 2016 39