Polycystic Kidney Disease – Pipeline Review, H2 2013

Summary

Global Markets Direct’s, ‘Polycystic Kidney Disease – Pipeline Review, H2 2013’, provides an overview of the indication’s therapeutic pipeline. This report provides information on the therapeutic development for Polycystic Kidney Disease, complete with latest updates, and special features on late-stage and discontinued projects. It also reviews key players involved in the therapeutic development for Polycystic Kidney Disease. Polycystic Kidney Disease – 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 Polycystic Kidney Disease.

– A review of the Polycystic Kidney Disease 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 Polycystic Kidney Disease 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 Polycystic Kidney Disease.

– 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 Polycystic Kidney Disease 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 5

List of Figures 6

Introduction 7

Global Markets Direct Report Coverage 7

Polycystic Kidney Disease Overview 8

Therapeutics Development 9

An Overview of Pipeline Products for Polycystic Kidney Disease 9

Polycystic Kidney Disease Therapeutics under Development by Companies 11

Polycystic Kidney Disease Therapeutics under Investigation by Universities/Institutes 13

Late Stage Products 14

Comparative Analysis 14

Mid Clinical Stage Products 15

Comparative Analysis 15

Early Clinical Stage Products 16

Comparative Analysis 16

Discovery and Pre-Clinical Stage Products 17

Comparative Analysis 17

Polycystic Kidney Disease Therapeutics - Products under Development by Companies 18

Polycystic Kidney Disease Therapeutics - Products under Investigation by Universities/Institutes 19

Companies Involved in Polycystic Kidney Disease Therapeutics Development 20

Emergent BioSolutions Inc. 20

Plexxikon Inc. 21

Pfizer Inc. 22

Synta Pharmaceuticals Corp. 23

Metabolic Solutions Development Co. 24

IC-MedTech, Inc. 25

Endocyte, Inc. 26

ManRos Therapeutics 27

Napo Pharmaceuticals, Inc. 28

DiscoveryBiomed, Inc. 29

Polycystic Kidney Disease - Therapeutics Assessment 30

Assessment by Monotherapy Products 30

Assessment by Combination Products 31

Assessment by Route of Administration 32

Assessment by Molecule Type 34

Drug Profiles 36

bosutinib - Drug Profile 36

Product Description 36

Mechanism of Action 36

R&D Progress 36

MSDC-0160 - Drug Profile 38

Product Description 38

Mechanism of Action 38

R&D Progress 38

tolvaptan - Drug Profile 40

Product Description 40

Mechanism of Action 40

R&D Progress 40

tolvaptan - Drug Profile 42

Product Description 42

Mechanism of Action 42

R&D Progress 42

tolvaptan - Drug Profile 44

Product Description 44

Mechanism of Action 44

R&D Progress 44

octreotide-LAR - Drug Profile 46

Product Description 46

Mechanism of Action 46

R&D Progress 46

TNFR x TWEAKR - Drug Profile 47

Product Description 47

Mechanism of Action 47

R&D Progress 47

lanreotide acetate - Drug Profile 48

Product Description 48

Mechanism of Action 48

R&D Progress 48

sirolimus - Drug Profile 49

Product Description 49

Mechanism of Action 49

R&D Progress 49

Polycystic Kidney Disease Program - Drug Profile 50

Product Description 50

Mechanism of Action 50

R&D Progress 50

Program For Polycystic Kidney Disease - Drug Profile 51

Product Description 51

Mechanism of Action 51

R&D Progress 51

Drug For Polycstic Kidney Disease - Drug Profile 52

Product Description 52

Mechanism of Action 52

R&D Progress 52

Drugs For Human Ciliopathies - Drug Profile 53

Product Description 53

Mechanism of Action 53

R&D Progress 53

GLG-302 - Drug Profile 54

Product Description 54

Mechanism of Action 54

R&D Progress 54

STA-2842 - Drug Profile 55

Product Description 55

Mechanism of Action 55

R&D Progress 55

Polycystic Kidney Disease Therapeutics - Drug Profile Updates 56

Polycystic Kidney Disease Therapeutics - Discontinued Products 64

Polycystic Kidney Disease Therapeutics - Dormant Products 65

Polycystic Kidney Disease - Product Development Milestones 66

Featured News & Press Releases 66

Aug 29, 2013: Otsuka Receives Complete Response Letter from U.S. Food and Drug Administration for Tolvaptan for Use in Patients with Autosomal Dominant Polycystic Kidney Disease 66

Aug 05, 2013: U.S. Food and Drug Administration's Cardiovascular and Renal Drugs Advisory Committee Recommends Not to Approve Otsuka Pharmaceutical's Tolvaptan for Use in Autosomal Dominant Polycystic Kidney Disease 66

Jun 03, 2013: Otsuka Pharma Submits New Drug Application In Japan For Tolvaptan For Treatment Of Autosomal Dominant Polycystic Kidney Disease 67

Apr 12, 2013: Otsuka Pharma Announces FDA Acceptance Of NDA For Tolvaptan 67

Mar 01, 2013: Otsuka Canada Pharma Updates Safety Information For Polycystic Kidney Disease Drug Samsca 67

Jan 25, 2013: Otsuka And FDA Notify Healthcare Professionals Of Significant Liver Injury Associated With Use Of Samsca 68

Nov 04, 2012: Otsuka's Investigational Compound For Autosomal Dominant Polycystic Kidney Disease, Tolvaptan, Meets Primary Endpoint In Phase III Clinical Trial 68

Oct 07, 2010: The Johns Hopkins Polycystic Kidney Disease Center includes DiscoveryBioMed, Inc. in its program. 70

Oct 26, 2009: Napo Acquires Rights To Additional CFTR Technology 70

May 05, 2009: Napo Pharmaceuticals Initiates Preclinical Development Of A Novel CFTR Inhibitor Using Preclinical Resources Offered By The National Institute Of Allergy And Infectious Diseases (NIAID) 71

Appendix 72

Methodology 72

Coverage 72

Secondary Research 72

Primary Research 72

Expert Panel Validation 72

Contact Us 73

Disclaimer 73



List of Tables

Number of Products Under Development for Polycystic Kidney Disease, H2 2013 9

Products under Development for Polycystic Kidney Disease - Comparative Analysis, H2 2013 10

Number of Products under Development by Companies, H2 2013 12

Number of Products under Investigation by Universities/Institutes, H2 2013 13

Comparative Analysis by Late Stage Development, H2 2013 14

Comparative Analysis by Mid Clinical Stage Development, H2 2013 15

Comparative Analysis by Early Clinical Stage Development, H2 2013 16

Comparative Analysis by Discovery and Pre-Clinical Stage Development, H2 2013 17

Products under Development by Companies, H2 2013 18

Products under Investigation by Universities/Institutes, H2 2013 19

Emergent BioSolutions Inc., H2 2013 20

Plexxikon Inc., H2 2013 21

Pfizer Inc., H2 2013 22

Synta Pharmaceuticals Corp., H2 2013 23

Metabolic Solutions Development Co., H2 2013 24

IC-MedTech, Inc., H2 2013 25

Endocyte, Inc., H2 2013 26

ManRos Therapeutics, H2 2013 27

Napo Pharmaceuticals, Inc., H2 2013 28

DiscoveryBiomed, Inc., H2 2013 29

Assessment by Monotherapy Products, H2 2013 30

Assessment by Combination Products, H2 2013 31

Assessment by Stage and Route of Administration, H2 2013 33

Assessment by Stage and Molecule Type, H2 2013 35

Polycystic Kidney Disease Therapeutics - Drug Profile Updates 56

Polycystic Kidney Disease Therapeutics - Discontinued Products 64

Polycystic Kidney Disease Therapeutics - Dormant Products 65



List of Figures

Number of Products under Development for Polycystic Kidney Disease, H2 2013 9

Products under Development for Polycystic Kidney Disease - Comparative Analysis, H2 2013 10

Products under Development by Companies, H2 2013 11

Products under Investigation by Universities/Institutes, H2 2013 13

Late Stage Products, H2 2013 14

Mid Clinical Stage Products, H2 2013 15

Early Clinical Stage Products, H2 2013 16

Discovery and Pre-Clinical Stage Products, H2 2013 17

Assessment by Monotherapy Products, H2 2013 30

Assessment by Combination Products, H2 2013 31

Assessment by Route of Administration, H2 2013 32

Assessment by Stage and Route of Administration, H2 2013 33

Assessment by Molecule Type, H2 2013 34

Assessment by Stage and Molecule Type, H2 2013 35