Spinal Muscular Atrophy (SMA) – Pipeline Review, H1 2018

Global Markets Direct’s, ‘Spinal Muscular Atrophy (SMA) – Pipeline Review, H1 2018’, provides an overview of the Spinal Muscular Atrophy (SMA) pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Spinal Muscular Atrophy (SMA), 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 Spinal Muscular Atrophy (SMA) 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 Spinal Muscular Atrophy (SMA)

– The report reviews pipeline therapeutics for Spinal Muscular Atrophy (SMA) 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 Spinal Muscular Atrophy (SMA) therapeutics and enlists all their major and minor projects

– The report assesses Spinal Muscular Atrophy (SMA) 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 Spinal Muscular Atrophy (SMA)

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 Spinal Muscular Atrophy (SMA)

- 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 Spinal Muscular Atrophy (SMA) 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

AveXis Inc

BioMarin Pharmaceutical Inc

Cytokinetics Inc

Exicure Inc

F. Hoffmann-La Roche Ltd

Genethon SA

Genzyme Corp

Kowa Co Ltd

Neurotune AG

Novartis AG

Paratek Pharmaceuticals Inc

Recursion Pharmaceuticals Inc

Sarepta Therapeutics Inc

Scholar Rock Inc

Spotlight Innovation Inc

Voyager Therapeutics Inc

Table of Contents

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

Spinal Muscular Atrophy (SMA) Overview

Spinal Muscular Atrophy (SMA) Therapeutics Development

Pipeline Overview

Pipeline by Companies

Pipeline by Universities/Institutes

Products under Development by Companies

Products under Development by Universities/Institutes

Spinal Muscular Atrophy (SMA) Therapeutics Assessment

Assessment by Target

Assessment by Mechanism of Action

Assessment by Route of Administration

Assessment by Molecule Type

Spinal Muscular Atrophy (SMA) Companies Involved in Therapeutics Development

AveXis Inc

BioMarin Pharmaceutical Inc

Cytokinetics Inc

Exicure Inc

F. Hoffmann-La Roche Ltd

Genethon SA

Genzyme Corp

Kowa Co Ltd

Neurotune AG

Novartis AG

Paratek Pharmaceuticals Inc

Recursion Pharmaceuticals Inc

Sarepta Therapeutics Inc

Scholar Rock Inc

Spotlight Innovation Inc

Voyager Therapeutics Inc

Spinal Muscular Atrophy (SMA) Drug Profiles

AAD-2004 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

ALB-111 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

amifampridine phosphate Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Antisense Oligonucleotide to Activate SMN2 for Spinal Muscular Atrophy Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Antisense Oligonucleotides for Spinal Muscular Atrophy Drug Profile

Product Description

Mechanism Of Action

R&D Progress

AVXS-101 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

branaplam Drug Profile

Product Description

Mechanism Of Action

R&D Progress

CK-2127107 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene Therapy to Activate SMN for Spinal Muscular Atrophy Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene Therapy to Activate SMN1 for Spinal Muscular Atrophy Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene Therapy to Activate SMN1 for Spinal Muscular Atrophy Drug Profile

Product Description

Mechanism Of Action

R&D Progress

K-828SP Drug Profile

Product Description

Mechanism Of Action

R&D Progress

NT-1654 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

olesoxime Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Oligonucleotides for Spinal Muscular Atrophy and Huntington Disease Drug Profile

Product Description

Mechanism Of Action

R&D Progress

PMO-25 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

PTKSMA-1 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

REC-0000716 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

REC-0001202 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

RG-7916 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Small Molecule to Inhibit GSK-3 for Spinal Muscular Atrophy Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Small Molecules for Central Nervous System Disorders Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Small Molecules for Spinal Muscular Atrophy Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Small Molecules for Spinal Muscular Atrophy Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Small Molecules for X-Linked Infantile Spinal Muscular Atrophy Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Small Molecules to Activate SMN2 for Spinal Muscular Atrophy Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Small Molecules to Activate SMN2 for Spinal Muscular Atrophy Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Small Molecules to Inhibit SRSF10 and BCLAF1 for HIV Infection, Spinal Muscular Atrophy and Colon Cancer Drug Profile

Product Description

Mechanism Of Action

R&D Progress

SRK-105 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

STL-182 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

VYSMN-101 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Xcel-hNu Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Xcel-hNuP Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Spinal Muscular Atrophy (SMA) Dormant Projects

Spinal Muscular Atrophy (SMA) Discontinued Products

Spinal Muscular Atrophy (SMA) Product Development Milestones

Featured News & Press Releases

Feb 02, 2018: Scholar Rock Announces Publication of Comprehensive Pharmacological Study of SRK-015, a Novel Inhibitor of Myostatin Activation, Supporting the Treatment of Muscle Atrophy Disorders

Jan 30, 2018: AveXis to Initiate Screening for Remaining Patients in Pivotal Trial of AVXS-101 for SMA Type 1 Following Review of Preliminary Data from First Three Patients

Jan 27, 2018: Preliminary Data from FIREFISH trial in Type 1 SMA Infants Presented at the International Scientific Congress on Spinal Muscular Atrophy

Jan 23, 2018: Data from RG7916 Programs in SMA to be Presented at the International Scientific Congress on Spinal Muscular Atrophy

Jan 23, 2018: Scholar Rock and Collaborators at Harvard Medical School and Northeastern University Elucidate Molecular Basis of Myostatin Activation a Key Physiological Process in Muscle Health

Jan 16, 2018: AveXis Announces Expanded Clinical Development Program for AVXS-101 in Spinal Muscular Atrophy

Jan 04, 2018: AveXis Announces Alignment with FDA on Next Steps Toward a BLA Submission for AVXS-101 in SMA Type 1

Jan 03, 2018: Cytokinetics Provides on CK-2127107

Dec 13, 2017: AveXis Announces Plan to Initiate Phase 1 Trial in SMA Type 2 Utilizing Intrathecal Delivery of AVXS-101

Dec 11, 2017: Cytokinetics Publishes Clinical Trial Data for CK-2127107

Nov 21, 2017: Catalyst Pharmaceuticals Announces Phase 2 Study of Firdapse in Ambulatory Patients with Spinal Muscular Atrophy (SMA)

Nov 01, 2017: AveXis Announces New England Journal of Medicine Publication of Phase 1 Data of AVXS-101 Gene Replacement Therapy in Spinal Muscular Atrophy Type 1

Oct 12, 2017: Spinal Muscular Atrophy Program Advances into Pivotal Study in SMA Patients

Oct 03, 2017: AveXis to Report Top-line Data from the Phase 1 Clinical Trial of AVXS-101 in SMA Type 1 at the International Annual Congress of the World Muscle Society

Oct 03, 2017: RG7916 Increased SMN Protein Production in SUNFISH Clinical Trial in Patients with Type 2/3 Spinal Muscular Atrophy

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

List of Tables

List of Tables

Number of Products under Development for Spinal Muscular Atrophy (SMA), H1 2018

Number of Products under Development by Companies, H1 2018

Number of Products under Development by Companies, H1 2018 (Contd..1), H1 2018

Number of Products under Development by Universities/Institutes, H1 2018

Products under Development by Companies, H1 2018

Products under Development by Companies, H1 2018 (Contd..1), H1 2018

Products under Development by Universities/Institutes, H1 2018

Number of Products by Stage and Target, H1 2018

Number of Products by Stage and Mechanism of Action, H1 2018

Number of Products by Stage and Route of Administration, H1 2018

Number of Products by Stage and Molecule Type, H1 2018

Spinal Muscular Atrophy (SMA) Pipeline by AveXis Inc, H1 2018

Spinal Muscular Atrophy (SMA) Pipeline by BioMarin Pharmaceutical Inc, H1 2018

Spinal Muscular Atrophy (SMA) Pipeline by Cytokinetics Inc, H1 2018

Spinal Muscular Atrophy (SMA) Pipeline by Exicure Inc, H1 2018

Spinal Muscular Atrophy (SMA) Pipeline by F. Hoffmann-La Roche Ltd, H1 2018

Spinal Muscular Atrophy (SMA) Pipeline by Genethon SA, H1 2018

Spinal Muscular Atrophy (SMA) Pipeline by Genzyme Corp, H1 2018

Spinal Muscular Atrophy (SMA) Pipeline by Kowa Co Ltd, H1 2018

Spinal Muscular Atrophy (SMA) Pipeline by Neurotune AG, H1 2018

Spinal Muscular Atrophy (SMA) Pipeline by Novartis AG, H1 2018

Spinal Muscular Atrophy (SMA) Pipeline by Paratek Pharmaceuticals Inc, H1 2018

Spinal Muscular Atrophy (SMA) Pipeline by Recursion Pharmaceuticals Inc, H1 2018

Spinal Muscular Atrophy (SMA) Pipeline by Sarepta Therapeutics Inc, H1 2018

Spinal Muscular Atrophy (SMA) Pipeline by Scholar Rock Inc, H1 2018

Spinal Muscular Atrophy (SMA) Pipeline by Spotlight Innovation Inc, H1 2018

Spinal Muscular Atrophy (SMA) Pipeline by Voyager Therapeutics Inc, H1 2018

Spinal Muscular Atrophy (SMA) Dormant Projects, H1 2018

Spinal Muscular Atrophy (SMA) Dormant Projects, H1 2018 (Contd..1), H1 2018

Spinal Muscular Atrophy (SMA) Discontinued Products, H1 2018

List of Figures

List of Figures

Number of Products under Development for Spinal Muscular Atrophy (SMA), H1 2018

Number of Products under Development by Companies, H1 2018

Number of Products under Development by Universities/Institutes, H1 2018

Number of Products by Top 10 Targets, H1 2018

Number of Products by Stage and Top 10 Targets, H1 2018

Number of Products by Top 10 Mechanism of Actions, H1 2018

Number of Products by Stage and Top 10 Mechanism of Actions, H1 2018

Number of Products by Routes of Administration, H1 2018

Number of Products by Stage and Routes of Administration, H1 2018

Number of Products by Molecule Types, H1 2018

Number of Products by Stage and Molecule Types, H1 2018

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