Urea Cycle Disorders – Pipeline Review, H2 2012

07:48 EDT 22 Oct 2012 | BioPortfolio Reports

Recently added to the BioPortfolio report store, Urea Cycle Disorders – Pipeline Review, H2 2012 is a new report from Global Markets Direct published on 2012-09-24. This 43-page report is available in PDF from $2000.

Urea Cycle Disorders – Pipeline Review, H2 2012


Global Markets Direct’s, 'Urea Cycle Disorders - Pipeline Review, H2 2012', provides an overview of the indication’s therapeutic pipeline. This report provides information on the therapeutic development for Urea Cycle 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 Urea Cycle Disorders. Urea Cycle 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.


- A snapshot of the global therapeutic scenario for Urea Cycle Disorders.
- A review of the Urea Cycle 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 Urea Cycle 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 Urea Cycle 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 Urea Cycle 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
Urea Cycle Disorders Overview 6
Therapeutics Development 7
An Overview of Pipeline Products for Urea Cycle Disorders 7
Urea Cycle Disorders Therapeutics under Development by Companies 9
Urea Cycle Disorders Therapeutics under Investigation by Universities/Institutes 10
Late Stage Products 11
Comparative Analysis 11
Mid Clinical Stage Products 12
Comparative Analysis 12
Early Clinical Stage Products 13
Comparative Analysis 13
Urea Cycle Disorders Therapeutics – Products under Development by Companies 14
Urea Cycle Disorders Therapeutics – Products under Investigation by Universities/Institutes 15
Companies Involved in Urea Cycle Disorders Therapeutics Development 16
Aspen Pharmacare Holdings Ltd 16
Ocera Therapeutics, Inc. 17
Hyperion Therapeutic, Inc. 18
Promethera Biosciences 19
Urea Cycle Disorders – Therapeutics Assessment 20
Assessment by Monotherapy Products 20
Assessment by Route of Administration 21
Assessment by Molecule Type 23
Drug Profiles 25
glycerol phenylbutyrate - Drug Profile 25
Product Description 25
Mechanism of Action 25
R&D Progress 25
ornithine phenylacetate - Drug Profile 27
Product Description 27
Mechanism of Action 27
R&D Progress 27
carglumic acid - Drug Profile 29
Product Description 29
Mechanism of Action 29
R&D Progress 29
Human Heterologous Liver Cells - Drug Profile 31
Product Description 31
Mechanism of Action 31
R&D Progress 31
carglumic acid - Drug Profile 32
Product Description 32
Mechanism of Action 32
R&D Progress 32
Promethera HepaStem - Drug Profile 33
Product Description 33
Mechanism of Action 33
R&D Progress 33
Urea Cycle Disorders Therapeutics – Drug Profile Updates 34
Urea Cycle Disorders – Product Development Milestones 37
Featured News & Press Releases 37
May 14, 2012: Promethera Biosciences Treats First Patients With Promethera HepaStem 37
Oct 19, 2011: Cytonet's Liver Cell Therapy Trial For Children With UCD Expands To Canada 37
May 17, 2011: Cytonet's Liver Cell Therapy Receives Orphan Drug Designation From FDA 38
Apr 13, 2011: Cytonet Launches Phase II Trial For Hereditary Urea Cycle Disorders (UCD) In Children 38
Nov 02, 2010: Hyperion' Glycerol Phenylbutyrate Meets Primary Endpoint In Phase III Pivotal Study In Urea Cycle Disorders 39
Oct 18, 2010: Hyperion Receives FDA Fast Track Designation For Glycerol Phenylbutyrate For Treatment Of Urea Cycle Disorders 40
Nov 02, 2009: UCL (University College London) Liver Failure Group Present Two Studies On A Novel Treatment Option (OCR-002) For Complications Of Hyperammonemia In Liver Failure At The 60th Annual AASLD Meeting 40
Feb 18, 2005: Medicis Announces FDA Approval of AMMONUL for Urea Cycle Disorder 41
Appendix 42
Methodology 42
Coverage 42
Secondary Research 42
Primary Research 42 For more information open Urea Cycle Disorders – Pipeline Review, H2 2012.


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