Genetic Disorder Cluster Muscular Dystrophy Drug Development Pipeline Review, 2017

This report provides an overview of the genetic disorders pipeline landscape, specifically focusing on muscular dystrophies. The report provides comprehensive information on the therapeutics under development and key players involved in therapeutic development for Becker and Duchenne Muscular Dystrophy (BMD and DMD) and features dormant and discontinued projects. Both indications covered are inherited muscular dystrophies, a group of genetic, degenerative diseases primarily affecting voluntary muscles.

BMD, the first type of muscular dystrophy, leads to symptoms include pain and sensation, difficulty with running, hopping, and jumping, toe walking, breathing problems, cognitive problems and frequent falls. DMD is a condition which causes muscle weakness, and is an X-linked disorder. The gene for DMD is present on the X chromosome. It codes for a protein named dystrophin. Dystrophin is essential for providing structural support inside muscle cells. Symptoms usually appear before age 6 and may appear as early as infancy. They include fatigue, learning difficulties, intellectual disability, muscle weakness and progressive difficulty walking.

The size of these two pipelines ranges from just six in BMD to 108 in DMD. The majority of pipeline products for DMD target dystrophin which, as mentioned, is the causative gene for the disease. However, a number of products act on other molecular targets such as growth differentiation factor 8, nuclear factor kappa B and utrophin. The first two of these make up the pipeline for BMD, along with forkhead box protein 1B.

Scope

– Which companies are the most active within the pipeline for genetic disorder therapeutics?

– Which pharmaceutical approaches are the most prominent at each stage of the pipeline and within each indication?

– To what extent do universities and institutions play a role within this pipeline, compared to pharmaceutical companies?

– What are the most important R&D milestones and data publications to have happened in the field of genetic disorder therapeutics?

Reasons to buy

- Understand the overall pipeline, with an at-a-glance overview of all products in therapeutic development for each indication

- Assess the products in development in granular detail, with an up-to-date overview of each individual pipeline program in each indication, and a comprehensive picture of recent updates and milestones for each

- Analyze the companies, institutions and universities currently operating in the pipeline, and the products being fielded by each of these

- Understand the composition of the pipeline in terms of molecule type, molecular target, mechanism of action and route of administration

Table of Contents

Table of Contents

GBI Research Report Guidance 2

Executive Summary 3

Table of Contents 4

List of Tables 5

List of Figures 7

Introduction 8

Genetic Disorder Cluster Muscular Dystrophy Report Coverage 8

Becker Muscular Dystrophy – Overview 8

Duchenne Muscular Dystrophy – Overview 8

Therapeutics Development 9

Becker Muscular Dystrophy 9

Duchenne Muscular Dystrophy 12

Therapeutics Assessment 22

Becker Muscular Dystrophy 22

Duchenne Muscular Dystrophy 30

Companies Involved in Therapeutics Development 38

Becker Muscular Dystrophy 38

Duchenne Muscular Dystrophy 40

Dormant Projects 63

Becker Muscular Dystrophy 63

Duchenne Muscular Dystrophy 64

Discontinued Products 66

Duchenne Muscular Dystrophy 66

Product Development Milestones 67

Duchenne Muscular Dystrophy 67

Appendix 78

Methodology 78

Coverage 78

Secondary Research 78

Primary Research 78

Expert Panel Validation 78

Contact Us 79

Disclaimer 79

List of Tables

List of Tables

Number of Products under Development for Becker Muscular Dystrophy 9

Number of Products under Development by Companies, Becker Muscular Dystrophy 10

Products under Development by Companies, Becker Muscular Dystrophy 11

Number of Products under Development for Duchenne Muscular Dystrophy 12

Number of Products under Development by Companies, Duchenne Muscular Dystrophy 14

Number of Products under Development by Universities/Institutes, Duchenne Muscular Dystrophy 16

Products under Development by Companies, Duchenne Muscular Dystrophy 17

Products under Development by Universities/Institutes, Duchenne Muscular Dystrophy 21

Number of Products by Stage and Target, Becker Muscular Dystrophy 23

Number of Products by Stage and Mechanism of Action, Becker Muscular Dystrophy 25

Number of Products by Stage and Route of Administration, Becker Muscular Dystrophy 27

Number of Products by Stage and Molecule Type, Becker Muscular Dystrophy 29

Number of Products by Stage and Target, Duchenne Muscular Dystrophy 31

Number of Products by Stage and Mechanism of Action, Duchenne Muscular Dystrophy 33

Number of Products by Stage and Route of Administration, Duchenne Muscular Dystrophy 35

Number of Products by Stage and Molecule Type, Duchenne Muscular Dystrophy 37

Becker Muscular Dystrophy – Pipeline by Cardero Therapeutics Inc 38

Becker Muscular Dystrophy – Pipeline by Milo Biotechnology LLC 38

Becker Muscular Dystrophy – Pipeline by PTC Therapeutics Inc 39

Becker Muscular Dystrophy – Pipeline by ReveraGen BioPharma Inc 39

Becker Muscular Dystrophy – Pipeline by Sarepta Therapeutics Inc 40

Duchenne Muscular Dystrophy – Pipeline by Acceleron Pharma Inc 40

Duchenne Muscular Dystrophy – Pipeline by Akashi Therapeutics Inc 41

Duchenne Muscular Dystrophy – Pipeline by Asklepios BioPharmaceutical Inc 41

Duchenne Muscular Dystrophy – Pipeline by BioMarin Pharmaceutical Inc 41

Duchenne Muscular Dystrophy – Pipeline by Bristol-Myers Squibb Company 42

Duchenne Muscular Dystrophy – Pipeline by Capricor Therapeutics Inc 42

Duchenne Muscular Dystrophy – Pipeline by Cardero Therapeutics Inc 43

Duchenne Muscular Dystrophy – Pipeline by Catabasis Pharmaceuticals Inc 43

Duchenne Muscular Dystrophy – Pipeline by CRISPR Therapeutics 44

Duchenne Muscular Dystrophy – Pipeline by Daiichi Sankyo Company Ltd 44

Duchenne Muscular Dystrophy – Pipeline by Debiopharm International SA 45

Duchenne Muscular Dystrophy – Pipeline by Editas Medicine Inc 45

Duchenne Muscular Dystrophy – Pipeline by Eloxx Pharmaceuticals Ltd 46

Duchenne Muscular Dystrophy – Pipeline by FibroGen Inc 46

Duchenne Muscular Dystrophy – Pipeline by Galapagos NV 47

Duchenne Muscular Dystrophy – Pipeline by Genethon SA 47

Duchenne Muscular Dystrophy – Pipeline by GTx Inc 48

Duchenne Muscular Dystrophy – Pipeline by GW Pharmaceuticals Plc 48

Duchenne Muscular Dystrophy – Pipeline by Idera Pharmaceuticals Inc 49

Duchenne Muscular Dystrophy – Pipeline by Italfarmaco SpA 49

Duchenne Muscular Dystrophy – Pipeline by La Jolla Pharmaceutical Company 50

Duchenne Muscular Dystrophy – Pipeline by Lexicon Pharmaceuticals Inc 50

Duchenne Muscular Dystrophy – Pipeline by Marina Biotech Inc 51

Duchenne Muscular Dystrophy – Pipeline by Merck KGaA 51

Duchenne Muscular Dystrophy – Pipeline by Milo Biotechnology LLC 52

Duchenne Muscular Dystrophy – Pipeline by Mitobridge Inc 52

Duchenne Muscular Dystrophy – Pipeline by Mitochon Pharmaceuticals Inc 53

Duchenne Muscular Dystrophy – Pipeline by MyoTherix Inc 53

Duchenne Muscular Dystrophy – Pipeline by NicOx SA 54

Duchenne Muscular Dystrophy – Pipeline by Nippon Shinyaku Co Ltd 54

Duchenne Muscular Dystrophy – Pipeline by Nobelpharma Co Ltd 55

Duchenne Muscular Dystrophy – Pipeline by Novartis AG 55

Duchenne Muscular Dystrophy – Pipeline by Pfizer Inc 56

Duchenne Muscular Dystrophy – Pipeline by Pluristem Therapeutics Inc 56

Duchenne Muscular Dystrophy – Pipeline by Prothelia Inc 57

Duchenne Muscular Dystrophy – Pipeline by PTC Therapeutics Inc 57

Duchenne Muscular Dystrophy – Pipeline by RASRx LLC 58

Duchenne Muscular Dystrophy – Pipeline by ReveraGen BioPharma Inc 58

Duchenne Muscular Dystrophy – Pipeline by Santhera Pharmaceuticals Holding AG 59

Duchenne Muscular Dystrophy – Pipeline by Sarepta Therapeutics Inc 60

Duchenne Muscular Dystrophy – Pipeline by Strykagen Corp 61

Duchenne Muscular Dystrophy – Pipeline by Summit Therapeutics Plc 61

Duchenne Muscular Dystrophy – Pipeline by Taiho Pharmaceutical Co Ltd 62

Duchenne Muscular Dystrophy – Pipeline by WAVE Life Sciences Ltd 62

Becker Muscular Dystrophy – Dormant Projects 63

Duchenne Muscular Dystrophy – Dormant Projects 64

Duchenne Muscular Dystrophy – Discontinued Products 66

List of Figures

List of Figures

Number of Products under Development for Becker Muscular Dystrophy 9

Number of Products under Development by Companies, Becker Muscular Dystrophy 10

Number of Products under Development for Duchenne Muscular Dystrophy 12

Number of Products under Development by Companies, Duchenne Muscular Dystrophy 13

Number of Products under Development by Universities/Institutes, Duchenne Muscular Dystrophy 16

Number of Products by Targets, Becker Muscular Dystrophy 22

Number of Products by Stage and Targets, Becker Muscular Dystrophy 22

Number of Products by Mechanism of Actions, Becker Muscular Dystrophy 24

Number of Products by Stage and Mechanism of Actions, Becker Muscular Dystrophy 24

Number of Products by Routes of Administration, Becker Muscular Dystrophy 26

Number of Products by Stage and Routes of Administration, Becker Muscular Dystrophy 26

Number of Products by Molecule Types, Becker Muscular Dystrophy 28

Number of Products by Stage and Molecule Types, Becker Muscular Dystrophy 28

Number of Products by Top 10 Targets, Duchenne Muscular Dystrophy 30

Number of Products by Stage and Top 10 Targets, Duchenne Muscular Dystrophy 30

Number of Products by Top 10 Mechanism of Actions, Duchenne Muscular Dystrophy 32

Number of Products by Stage and Top 10 Mechanism of Actions, Duchenne Muscular Dystrophy 32

Number of Products by Routes of Administration, Duchenne Muscular Dystrophy 34

Number of Products by Stage and Routes of Administration, Duchenne Muscular Dystrophy 34

Number of Products by Molecule Types, Duchenne Muscular Dystrophy 36

Number of Products by Stage and Molecule Types, Duchenne Muscular Dystrophy 36

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