Hypertrophic Cardiomyopathy – Pipeline Review, H2 2016

Global Markets Direct’s, ‘Hypertrophic Cardiomyopathy – Pipeline Review, H2 2016’, provides an overview of the Hypertrophic Cardiomyopathy pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Hypertrophic Cardiomyopathy, complete with analysis by stage of development, drug target, 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 therapeutic development for Hypertrophic Cardiomyopathy 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 of Hypertrophic Cardiomyopathy

The report reviews pipeline therapeutics for Hypertrophic Cardiomyopathy 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 Hypertrophic Cardiomyopathy therapeutics and enlists all their major and minor projects

The report assesses Hypertrophic Cardiomyopathy therapeutics based on drug target, 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 related to pipeline therapeutics for Hypertrophic Cardiomyopathy

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 important and diverse types of therapeutics under development for Hypertrophic Cardiomyopathy

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 Hypertrophic Cardiomyopathy 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

Companies mentioned

Gilead Sciences Inc

Heart Metabolics Ltd

Lead Discovery Center GmbH

miRagen Therapeutics Inc

MyoKardia Inc

Table of Contents

Table of Contents

Table of Contents 2

List of Tables 4

List of Figures 5

Introduction 6

Global Markets Direct Report Coverage 6

Hypertrophic Cardiomyopathy Overview 7

Therapeutics Development 8

Pipeline Products for Hypertrophic Cardiomyopathy - Overview 8

Pipeline Products for Hypertrophic Cardiomyopathy - Comparative Analysis 9

Hypertrophic Cardiomyopathy - Therapeutics under Development by Companies 10

Hypertrophic Cardiomyopathy - Therapeutics under Investigation by Universities/Institutes 11

Hypertrophic Cardiomyopathy - Pipeline Products Glance 12

Late Stage Products 12

Clinical Stage Products 13

Early Stage Products 14

Hypertrophic Cardiomyopathy - Products under Development by Companies 15

Hypertrophic Cardiomyopathy - Products under Investigation by Universities/Institutes 16

Hypertrophic Cardiomyopathy - Companies Involved in Therapeutics Development 17

Gilead Sciences Inc 17

Heart Metabolics Ltd 18

Lead Discovery Center GmbH 19

miRagen Therapeutics Inc 20

MyoKardia Inc 21

Hypertrophic Cardiomyopathy - Therapeutics Assessment 22

Assessment by Monotherapy Products 22

Assessment by Target 23

Assessment by Mechanism of Action 25

Assessment by Route of Administration 27

Assessment by Molecule Type 28

Drug Profiles 30

eleclazine - Drug Profile 30

Product Description 30

Mechanism Of Action 30

R&D Progress 30

HCM-2 - Drug Profile 32

Product Description 32

Mechanism Of Action 32

R&D Progress 32

LUS-1 - Drug Profile 33

Product Description 33

Mechanism Of Action 33

R&D Progress 33

MGN-9103 - Drug Profile 34

Product Description 34

Mechanism Of Action 34

R&D Progress 34

MYK-461 - Drug Profile 36

Product Description 36

Mechanism Of Action 36

R&D Progress 36

perhexiline maleate - Drug Profile 39

Product Description 39

Mechanism Of Action 39

R&D Progress 39

ranolazine ER - Drug Profile 41

Product Description 41

Mechanism Of Action 41

R&D Progress 41

Small Molecule for Cardiac Hypertrophy - Drug Profile 44

Product Description 44

Mechanism Of Action 44

R&D Progress 44

Small Molecules to Inhibit RhoGEF12 for Cardiovascular Diseases - Drug Profile 45

Product Description 45

Mechanism Of Action 45

R&D Progress 45

Hypertrophic Cardiomyopathy - Dormant Projects 46

Hypertrophic Cardiomyopathy - Discontinued Products 47

Hypertrophic Cardiomyopathy - Product Development Milestones 48

Featured News & Press Releases 48

Nov 11, 2016: MyoKardia Announces Exploratory Digital Health Substudy as Part of PIONEER-HCM Trial of MYK-461 in Symptomatic, Obstructive Hypertrophic Cardiomyopathy Patients 48

Nov 02, 2016: MyoKardia Announces Dosing of First Patient in Phase 2 PIONEER-HCM Study of MYK-461 in Symptomatic, Obstructive Hypertrophic Cardiomyopathy 48

Sep 21, 2016: MyoKardia Outlines Path to Registration for MYK-461 in Symptomatic, Obstructive Hypertrophic Cardiomyopathy Patients and Provides Pipeline and Research Updates at Inaugural R&D Day 49

Aug 31, 2016: Heart Metabolics Announces Initiation of Phase 2b Study of Perhexiline for Hypertrophic Cardiomyopathy 51

Jul 14, 2016: SHaRe Registry Data Published in PNAS Demonstrate the Power of Combining Clinical, Genetic and Structural Data to Gain Insight into Hypertrophic Cardiomyopathy 52

Jul 11, 2016: MyoKardia Announces Topline Clinical Data Supporting Advancement of MYK-461 to Phase 2 PIONEER-HCM Study in Symptomatic, Obstructive Hypertrophic Cardiomyopathy Patients 53

May 02, 2016: MyoKardia Receives Orphan Drug Designation for MYK-461 for Treatment of Symptomatic Obstructive Hypertrophic Cardiomyopathy 54

Mar 15, 2016: Heart Metabolics Announces Multiple Senior Appointments to Management Team 55

Feb 04, 2016: MyoKardia Publishes Article in Science Demonstrating That MYK-461 Prevents and Reverses Disease in Genetic Mouse Models of Hypertrophic Cardiomyopathy 55

Oct 06, 2015: MyoKardia Provides Update on Two Phase 1 Trials of MYK-461 for the Treatment of Hypertrophic Cardiomyopathy 56

Apr 15, 2015: Heart Metabolics and FDA Reach Agreement on Special Protocol Assessment for Single Phase 3 Trial of Perhexiline in Hypertrophic Cardiomyopathy 57

Mar 03, 2015: MyoKardia Announces Initiation of Phase 1 Clinical Study of MYK-461 for Hypertrophic Cardiomyopathy 58

Appendix 59

Methodology 59

Coverage 59

Secondary Research 59

Primary Research 59

Expert Panel Validation 59

Contact Us 59

Disclaimer 60

List of Tables

List of Tables

Number of Products under Development for Hypertrophic Cardiomyopathy, H2 2016 8

Number of Products under Development for Hypertrophic Cardiomyopathy – Comparative Analysis, H2 2016 9

Number of Products under Development by Companies, H2 2016 10

Number of Products under Investigation by Universities/Institutes, H2 2016 11

Comparative Analysis by Late Stage Development, H2 2016 12

Comparative Analysis by Clinical Stage Development, H2 2016 13

Comparative Analysis by Early Stage Development, H2 2016 14

Products under Development by Companies, H2 2016 15

Products under Investigation by Universities/Institutes, H2 2016 16

Hypertrophic Cardiomyopathy – Pipeline by Gilead Sciences Inc, H2 2016 17

Hypertrophic Cardiomyopathy – Pipeline by Heart Metabolics Ltd, H2 2016 18

Hypertrophic Cardiomyopathy – Pipeline by Lead Discovery Center GmbH, H2 2016 19

Hypertrophic Cardiomyopathy – Pipeline by miRagen Therapeutics Inc, H2 2016 20

Hypertrophic Cardiomyopathy – Pipeline by MyoKardia Inc, H2 2016 21

Assessment by Monotherapy Products, H2 2016 22

Number of Products by Stage and Target, H2 2016 24

Number of Products by Stage and Mechanism of Action, H2 2016 26

Number of Products by Stage and Route of Administration, H2 2016 27

Number of Products by Stage and Molecule Type, H2 2016 29

Hypertrophic Cardiomyopathy – Dormant Projects, H2 2016 46

Hypertrophic Cardiomyopathy – Discontinued Products, H2 2016 47

List of Figures

List of Figures

Number of Products under Development for Hypertrophic Cardiomyopathy, H2 2016 8

Number of Products under Development for Hypertrophic Cardiomyopathy – Comparative Analysis, H2 2016 9

Number of Products under Development by Companies, H2 2016 10

Comparative Analysis by Early Stage Products, H2 2016 14

Assessment by Monotherapy Products, H2 2016 22

Number of Products by Targets, H2 2016 23

Number of Products by Stage and Targets, H2 2016 23

Number of Products by Mechanism of Actions, H2 2016 25

Number of Products by Stage and Mechanism of Actions, H2 2016 25

Number of Products by Stage and Routes of Administration, H2 2016 27

Number of Products by Molecule Types, H2 2016 28

Number of Products by Stage and Molecule Types, H2 2016 28

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