Alpha N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU or EC 3.2.1.50) – Pipeline Review, H2 2016

Global Markets Direct’s, ‘Alpha N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU or EC 3.2.1.50) – Pipeline Review, H2 2016’, provides in depth analysis on Alpha N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU or EC 3.2.1.50) targeted pipeline therapeutics.

The report provides comprehensive information on the Alpha N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU 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 NAGLU 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.

Updated report will be delivered in 48 hours of order confirmation.

Scope

The report provides a snapshot of the global therapeutic landscape for Alpha N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU or EC 3.2.1.50)

The report reviews Alpha N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU 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 NAGLU 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 NAGLU 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 NAGLU 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 NAGLU 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 NAGLU 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

Companies mentioned

Abeona Therapeutics Inc

Alexion Pharmaceuticals Inc

BioMarin Pharmaceutical Inc

Dorphan SA

Laboratorios Del Dr Esteve SA

UniQure NV

Table of Contents

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 NAGLU or EC

3.2.1.50) Overview 6

Therapeutics Development 7

Alpha N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU or EC

3.2.1.50) - Products under Development by Stage of Development 7

Alpha N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU or EC

3.2.1.50) - Products under Development by Therapy Area 8

Alpha N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU or EC

3.2.1.50) - Products under Development by Indication 9

Alpha N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU 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 NAGLU or EC

3.2.1.50) - Products under Development by Companies 12

Alpha N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU 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 NAGLU 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 SA 24

Laboratorios Del Dr Esteve SA 25

UniQure NV 26

Alpha N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU 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

lesinidase alfa - 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 NAGLU for Mucopolysaccharidosis IIIB - Drug Profile 38

Product Description 38

Mechanism Of Action 38

R&D Progress 38

Alpha N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU or EC

3.2.1.50) - Dormant Projects 39

Alpha N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU or EC

3.2.1.50) - Featured News & Press Releases 40

Jul 14, 2016: Alexion Presents New SBC-103 (rhNAGLU enzyme) Phase 1/2 Data on Brain MRI and Neurocognitive Assessments in Patients with Mucopolysaccharidosis IIIB (MPS IIIB) 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) 41

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) 42

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 43

Feb 25, 2016: ESTEVE Provides Update On EGT-201 For Sanfilippo B Syndrome 44

Jan 11, 2016: Abeona Therapeutics Announces Initial European Regulatory Approval for Phase 1/2 Gene Therapy Clinical Study for Patients With Sanfilippo Syndrome 44

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 45

Jun 04, 2015: Synageva BioPharma Completes Targeted Enrollment in Phase 1/2 Trial with SBC-103 for Mucopolysaccharidosis IIIB 46

May 20, 2015: PlasmaTech Biopharmaceuticals Announces Orphan Drug and Rare Pediatric Disease Designations For ABX-101 From FDA 47

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 47

Dec 15, 2014: Synageva BioPharma Announces Active Investigational New Drug Application For SBC-103 For The Treatment Of Mucopolysaccharidosis IIIB 48

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) 49

Feb 12, 2014: Synageva BioPharma Highlights SBC-103 Data at the Lysosomal Disease Network WORLD Symposium 49

Feb 11, 2014: BioMarin Announces Selection of NAGLU Fusion Protein Drug Development Candidate BMN 250 for the Treatment of Sanfilippo B 51

Jan 29, 2014: Synageva BioPharma Announces Data Presentations On SBC-103 at the 10th Annual Lysosomal Disease Network World Symposium 52

Appendix 54

Methodology 54

Coverage 54

Secondary Research 54

Primary Research 54

Expert Panel Validation 54

Contact Us 54

Disclaimer 55

List of Tables

List of Tables

Number of Products under Development for, H2 2016 7

Number of Products under Development by Therapy Area, H2 2016 8

Number of Products under Development by Indication, H2 2016 9

Comparative Analysis by Late Stage Development, H2 2016 10

Comparative Analysis by Early Stage Products, H2 2016 11

Number of Products under Development by Companies, H2 2016 12

Products under Development by Companies, H2 2016 13

Assessment by Monotherapy/Combination Products, H2 2016 14

Number of Products by Stage and Mechanism of Action, H2 2016 16

Number of Products by Stage and Route of Administration, H2 2016 18

Number of Products by Stage and Molecule Type, H2 2016 20

Pipeline by Abeona Therapeutics Inc, H2 2016 21

Pipeline by Alexion Pharmaceuticals Inc, H2 2016 22

Pipeline by BioMarin Pharmaceutical Inc, H2 2016 23

Pipeline by Dorphan SA, H2 2016 24

Pipeline by Laboratorios Del Dr Esteve SA, H2 2016 25

Pipeline by UniQure NV, H2 2016 26

Dormant Projects, H2 2016 39

List of Figures

List of Figures

Number of Products under Development for, H2 2016 7

Comparative Analysis by Early Stage Products, H2 2016 11

Assessment by Monotherapy/Combination Products, H2 2016 14

Number of Products by Mechanism of Actions, H2 2016 15

Number of Products by Stage and Mechanism of Actions, H2 2016 15

Number of Products by Routes of Administration, H2 2016 17

Number of Products by Stage and Routes of Administration, H2 2016 17

Number of Products by Molecule Types, H2 2016 19

Number of Products by Stage and Molecule Type, H2 2016 19

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