Spinal Muscular Atrophy (SMA) – Pipeline Review, H2 2018

Global Markets Direct’s, ‘Spinal Muscular Atrophy (SMA) – Pipeline Review, H2 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

AndroScience Corp

AveXis Inc

Biogen Inc

BioMarin Pharmaceutical Inc

Cytokinetics Inc

Exicure Inc

F. Hoffmann-La Roche Ltd

Genethon SA

Ionis Pharmaceuticals Inc

Kowa Co Ltd

Neurodyn Life Sciences Inc

Neurotune AG

Novartis AG

Ono Pharmaceutical Co Ltd

Paratek Pharmaceuticals Inc

Recursion Pharmaceuticals Inc

Sarepta Therapeutics Inc

Scholar Rock Inc

Spotlight Innovation Inc

Vybion 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

AndroScience Corp

AveXis Inc

Biogen Inc

BioMarin Pharmaceutical Inc

Cytokinetics Inc

Exicure Inc

F. Hoffmann-La Roche Ltd

Genethon SA

Ionis Pharmaceuticals Inc

Kowa Co Ltd

Neurodyn Life Sciences Inc

Neurotune AG

Novartis AG

Ono Pharmaceutical Co Ltd

Paratek Pharmaceuticals Inc

Recursion Pharmaceuticals Inc

Sarepta Therapeutics Inc

Scholar Rock Inc

Spotlight Innovation Inc

Vybion 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

ALG-802 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

Antisense Oligonucleotides to Activate SMN2 for Spinal Muscular Atrophy Drug Profile

Product Description

Mechanism Of Action

R&D Progress

ARM-210 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

ASC-J9/ASC-JM.X1 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

ASCJ-9 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

BIIB-110 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

branaplam 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

INT-41 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

LDN-5178 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

maresin-1 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

ND-602 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

NT-1654 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

onasemnogene abeparvovec 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

reldesemtiv Drug Profile

Product Description

Mechanism Of Action

R&D Progress

risdiplam Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Rycal 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 2 to Activate SMN2 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 Kennedy's Disease 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 Inhibit SRSF10 and BCLAF1 for HIV Infection, Spinal Muscular Atrophy and Colon Cancer Drug Profile

Product Description

Mechanism Of Action

R&D Progress

SMN-2 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

TEC-1 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

valproate sodium 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

Jul 24, 2018: Spotlight Innovation spinal muscular atrophy research collaborator professor Kevin Hodgetts receives $300,000 grant from Cure SMA

Jun 16, 2018: Cytokinetics Announces Data From Phase 2 Clinical Study of Reldesemtiv in Patients With Spinal Muscular Atrophy Presented at the 2018 Annual Cure SMA Conference

Jun 14, 2018: Exicure to Present Data at the Cure Spinal Muscular Atrophy Annual Conference in Dallas

Jun 08, 2018: Cytokinetics Announces Data From Phase 2 Clinical Study of Reldesemtiv in Patients With Spinal Muscular Atrophy to be Presented at the 2018 Annual Cure SMA Conference on June 16, 2018

May 31, 2018: Scholar Rock Announces Completion of Dosing of First Cohort in Phase 1 Clinical Trial of SRK-015

May 08, 2018: AveXis Issues Community Statement on the SPR1NT Trial

Apr 25, 2018: AveXis Announces First Patient Dosed in Phase 3 Trial of AVXS-101 in Pre-Symptomatic SMA Types 1, 2 and 3

Apr 24, 2018: AveXis Presents Initial Data from Pivotal U.S. Trial for SMA Type 1 and 24-Month Follow-Up Data from Phase 1 Trial of AVXS-101 in SMA Type 1 at the Annual Meeting of the American Academy of Neurology

Apr 24, 2018: Cytokinetics Announces Presentation of Data From Phase 2 Clinical Study of Reldesemtiv in Patients With Spinal Muscular Atrophy at the 2018 Annual Cure SMA Conference

Apr 19, 2018: AveXis to Present AVXS-101 Data at the Annual Meeting of the American Academy of Neurology

Mar 29, 2018: Scholar Rock Granted Orphan Drug Designation by the FDA for SRK-015 for the Treatment of Patients with Spinal Muscular Atrophy

Mar 27, 2018: AveXis Gene Therapy Awarded SAKIGAKE Designation for Spinal Muscular Atrophy Type 1

Mar 13, 2018: Newly Issued U.S. Patent Covers Spotlight Innovations Spinal Muscular Atrophy Drug Candidates

Feb 21, 2018: Phase 2 Clinical Trial Results for CK-2127107 Expected in Second Quarter of 2018

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

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), H2 2018

Number of Products under Development by Companies, H2 2018

Number of Products under Development by Companies, H2 2018 (Contd..1), H2 2018

Number of Products under Development by Universities/Institutes, H2 2018

Products under Development by Companies, H2 2018

Products under Development by Companies, H2 2018 (Contd..1), H2 2018

Products under Development by Companies, H2 2018 (Contd..2), H2 2018

Products under Development by Universities/Institutes, H2 2018

Number of Products by Stage and Target, H2 2018

Number of Products by Stage and Mechanism of Action, H2 2018

Number of Products by Stage and Route of Administration, H2 2018

Number of Products by Stage and Molecule Type, H2 2018

Spinal Muscular Atrophy (SMA) Pipeline by AndroScience Corp, H2 2018

Spinal Muscular Atrophy (SMA) Pipeline by AveXis Inc, H2 2018

Spinal Muscular Atrophy (SMA) Pipeline by Biogen Inc, H2 2018

Spinal Muscular Atrophy (SMA) Pipeline by BioMarin Pharmaceutical Inc, H2 2018

Spinal Muscular Atrophy (SMA) Pipeline by Cytokinetics Inc, H2 2018

Spinal Muscular Atrophy (SMA) Pipeline by Exicure Inc, H2 2018

Spinal Muscular Atrophy (SMA) Pipeline by F. Hoffmann-La Roche Ltd, H2 2018

Spinal Muscular Atrophy (SMA) Pipeline by Genethon SA, H2 2018

Spinal Muscular Atrophy (SMA) Pipeline by Ionis Pharmaceuticals Inc, H2 2018

Spinal Muscular Atrophy (SMA) Pipeline by Kowa Co Ltd, H2 2018

Spinal Muscular Atrophy (SMA) Pipeline by Neurodyn Life Sciences Inc, H2 2018

Spinal Muscular Atrophy (SMA) Pipeline by Neurotune AG, H2 2018

Spinal Muscular Atrophy (SMA) Pipeline by Novartis AG, H2 2018

Spinal Muscular Atrophy (SMA) Pipeline by Ono Pharmaceutical Co Ltd, H2 2018

Spinal Muscular Atrophy (SMA) Pipeline by Paratek Pharmaceuticals Inc, H2 2018

Spinal Muscular Atrophy (SMA) Pipeline by Recursion Pharmaceuticals Inc, H2 2018

Spinal Muscular Atrophy (SMA) Pipeline by Sarepta Therapeutics Inc, H2 2018

Spinal Muscular Atrophy (SMA) Pipeline by Scholar Rock Inc, H2 2018

Spinal Muscular Atrophy (SMA) Pipeline by Spotlight Innovation Inc, H2 2018

Spinal Muscular Atrophy (SMA) Pipeline by Vybion Inc, H2 2018

Spinal Muscular Atrophy (SMA) Dormant Projects, H2 2018

Spinal Muscular Atrophy (SMA) Dormant Projects, H2 2018 (Contd..1), H2 2018

Spinal Muscular Atrophy (SMA) Discontinued Products, H2 2018

List of Figures

List of Figures

Number of Products under Development for Spinal Muscular Atrophy (SMA), H2 2018

Number of Products under Development by Companies, H2 2018

Number of Products under Development by Universities/Institutes, H2 2018

Number of Products by Top 10 Targets, H2 2018

Number of Products by Stage and Top 10 Targets, H2 2018

Number of Products by Top 10 Mechanism of Actions, H2 2018

Number of Products by Stage and Top 10 Mechanism of Actions, H2 2018

Number of Products by Routes of Administration, H2 2018

Number of Products by Stage and Routes of Administration, H2 2018

Number of Products by Molecule Types, H2 2018

Number of Products by Stage and Molecule Types, H2 2018

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