Other Metabolic Disorders – Pipeline Review, H2 2013

Summary

Global Markets Direct’s, ‘Other Metabolic Disorders – Pipeline Review, H2 2013’, provides an overview of the indication’s therapeutic pipeline. This report provides information on the therapeutic development for Other Metabolic Disorders, complete with latest updates, and special features on late-stage and discontinued projects. It also reviews key players involved in the therapeutic development for Other Metabolic Disorders. Other Metabolic Disorders – Pipeline Review, Half Year is built using data and information sourced from Global Markets Direct’s 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.

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

Scope

– A snapshot of the global therapeutic scenario for Other Metabolic Disorders.

– A review of the Other Metabolic Disorders products under development by companies and universities/research institutes based on information derived from company and industry-specific sources.

– Coverage of products based on various stages of development ranging from discovery till registration stages.

– A feature on pipeline projects on the basis of monotherapy and combined therapeutics.

– Coverage of the Other Metabolic Disorders pipeline on the basis of route of administration and molecule type.

– Key discontinued pipeline projects.

– Latest news and deals relating to the products.

Reasons to buy

– Identify and understand important and diverse types of therapeutics under development for Other Metabolic Disorders.

– Identify emerging players with potentially strong product portfolio and design effective counter-strategies to gain competitive advantage.

– Plan mergers and acquisitions effectively by identifying players of the most promising pipeline.

– Devise corrective measures for pipeline projects by understanding Other 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.

– Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from pipeline.

Table of Contents

Table of Contents 2

List of Tables 4

List of Figures 4

Introduction 5

Global Markets Direct Report Coverage 5

Other Metabolic Disorders Overview 6

Therapeutics Development 7

An Overview of Pipeline Products for Other Metabolic Disorders 7

Other Metabolic Disorders Therapeutics under Development by Companies 9

Mid Clinical Stage Products 10

Comparative Analysis 10

Early Clinical Stage Products 11

Comparative Analysis 11

Discovery and Pre-Clinical Stage Products 12

Comparative Analysis 12

Other Metabolic Disorders Therapeutics - Products under Development by Companies 13

Companies Involved in Other Metabolic Disorders Therapeutics Development 14

Obio Pharmaceutical Holdings Limited. 14

The Alize Pharma Group 15

Other Metabolic Disorders - Therapeutics Assessment 16

Assessment by Monotherapy Products 16

Assessment by Route of Administration 17

Assessment by Molecule Type 19

Drug Profiles 21

KP-624 - Drug Profile 21

Product Description 21

Mechanism of Action 21

R&D Progress 21

AZP-01 - Drug Profile 22

Product Description 22

Mechanism of Action 22

R&D Progress 22

UX-007 - Drug Profile 23

Product Description 23

Mechanism of Action 23

R&D Progress 23

XAF-5 - Drug Profile 24

Product Description 24

Mechanism of Action 24

R&D Progress 24

CAS-001 - Drug Profile 25

Product Description 25

Mechanism of Action 25

R&D Progress 25

CAS-003 - Drug Profile 26

Product Description 26

Mechanism of Action 26

R&D Progress 26

CAS-045 - Drug Profile 27

Product Description 27

Mechanism of Action 27

R&D Progress 27

Other Metabolic Disorders Therapeutics - Drug Profile Updates 28

Other Metabolic Disorders Therapeutics - Discontinued Products 30

Other Metabolic Disorders Therapeutics - Dormant Products 31

Other Metabolic Disorders - Product Development Milestones 32

Featured News & Press Releases 32

Aug 28, 2013: Ultragenyx Announces Presentation of two Abstract at the 12th International Congress of Inborn Errors of Metabolism (ICIEM 2013) 32

Aug 26, 2013: Ultragenyx Investigational New Drug Application for UX007 is Active 32

Aug 05, 2013: Ultragenyx Initiates New Development Program Studying Triheptanoin (UX007) for the Treatment of Glucose Transporter Type-1 Deficiency Syndrome (Glut1 DS) 33

Jan 11, 2012: Bridge BioResearch Announces Completion Of Preclinical Trials Of 2hydroxyoleic Acid 34

Nov 01, 2011: KYTHERA Announces Preliminary Two-Year Follow-Up Study Results And Poster Presentation Of Phase IIb Data From Fat Reduction Drug At ASDS 34

Apr 14, 2011: CHMP Announces Positive Opinion For Carbaglu In Hyperammonaemia 34

Feb 15, 2011: Raptor To Enroll Five Additional Patients In Phase III Clinical Trial Of DR Cysteamine For Treatment Of Nephropathic Cystinosis 35

Feb 04, 2011: Kythera Reports Positive Results From Phase IIB Study With ATX-101 36

Oct 06, 2010: Arena And Eisai Report Lorcaserin Presentations At Obesity 2010 36

Oct 04, 2010: Orexigen Announces Upcoming Data Presentations At 28th Annual Scientific Meeting Of Obesity Society 37

Appendix 38

Methodology 38

Coverage 38

Secondary Research 38

Primary Research 38

Expert Panel Validation 38

Contact Us 39

Disclaimer 39



List of Tables

Number of Products Under Development for Other Metabolic Disorders, H2 2013 7

Products under Development for Other Metabolic Disorders - Comparative Analysis, H2 2013 8

Number of Products under Development by Companies, H2 2013 9

Comparative Analysis by Mid Clinical Stage Development, H2 2013 10

Comparative Analysis by Early Clinical Stage Development, H2 2013 11

Comparative Analysis by Discovery and Pre-Clinical Stage Development, H2 2013 12

Products under Development by Companies, H2 2013 13

Obio Pharmaceutical Holdings Limited., H2 2013 14

The Alize Pharma Group, H2 2013 15

Assessment by Monotherapy Products, H2 2013 16

Assessment by Stage and Route of Administration, H2 2013 18

Assessment by Stage and Molecule Type, H2 2013 20

Other Metabolic Disorders Therapeutics - Drug Profile Updates 28

Other Metabolic Disorders Therapeutics - Discontinued Products 30

Other Metabolic Disorders Therapeutics - Dormant Products 31



List of Figures

Number of Products under Development for Other Metabolic Disorders, H2 2013 7

Products under Development for Other Metabolic Disorders - Comparative Analysis, H2 2013 8

Products under Development by Companies, H2 2013 9

Mid Clinical Stage Products, H2 2013 10

Early Clinical Stage Products, H2 2013 11

Discovery and Pre-Clinical Stage Products, H2 2013 12

Assessment by Monotherapy Products, H2 2013 16

Assessment by Route of Administration, H2 2013 17

Assessment by Stage and Route of Administration, H2 2013 18

Assessment by Molecule Type, H2 2013 19

Assessment by Stage and Molecule Type, H2 2013 20