Global Spinal Muscular Atrophy (SMA) Drug Pipeline Trends 2019: Discovery, Pre-clinical, Clinical, In Approval Therapeutics, Companies and Markets

August 2019 | 101 pages | ID: G122639A415EN
VPAResearch

Abstracts

High levels of pipeline activity are being observed in Spinal Muscular Atrophy (SMA) treatment during 2019. Clinical development activities are being undertaken by more than 25 companies including Armgo Pharma Inc, AurimMed Pharma Inc, AveXis Inc, Biogen Inc, BioMarin Pharmaceutical Inc and others.

A Significant contribution to the Spinal Muscular Atrophy (SMA) pipeline is being observed from emerging companies opting for new approaches to clinical development strategy. The early stage and late-stage Spinal Muscular Atrophy (SMA) pipeline included 26 compounds, driven by increasing number of targeted therapies. Progress is being made in accelerating compounds into advanced stages. However, fluctuating trends are being observed in average duration across three phases.

Increased understanding of underlying causes of Spinal Muscular Atrophy (SMA) condition and increased access to investments is encouraging growth of Spinal Muscular Atrophy (SMA) drug pipeline. However, despite high levels of pipeline activity, increasing clinical trial costs and decreasing drug approval rates is requiring companies to focus more on understanding clinical development pipeline compounds.

The research study is structured to provide clear and actionable insights into Spinal Muscular Atrophy (SMA) drug pipeline for companies to optimize spending levels by understanding competition, their pipeline products and prospect of further advances over the next few years.

The report addresses the discovery, research and pre-clinical stage active Spinal Muscular Atrophy (SMA) therapeutic candidates. Further, drugs in clinical stage including phase 1, phase 2, phase 3 and pre-registration phases are also analyzed.

The current report examines the current Spinal Muscular Atrophy (SMA) pipeline along with current development status, regulatory progress, mechanism of action, route of administration, collaborating companies, sponsors, molecule type, and research/discovery details.

In addition to drug overview, recent developments, the report also provides mechanism of action, Pre-clinical trial details and Clinical trial details for each of the drug candidates of Spinal Muscular Atrophy (SMA). Further, orphan drug status, fast track designation, grants awarded and other special status for Spinal Muscular Atrophy (SMA) pipeline compounds are also included.
This study was produced independently by VPAResearch from our proprietary databases, primarily to assist users to develop in-depth understanding of 2019 Spinal Muscular Atrophy (SMA) pipeline and formulate effective research and development strategies.

RESEARCH METHODOLOGY

Our integrated data reports are built through robust research methodology to ensure our users can build on the insights and data accuracy for their expansion plans. Both primary and secondary research methods are used with stringent quality checks at each stage to evaluate trends, analysis and forecasts.

Scope of the Research

Spinal Muscular Atrophy (SMA) Pipeline candidates-

• Pre-clinical Phase: Discovery, research, pre-clinical
• Early Phase: Phase 1 and Phase 2 Spinal Muscular Atrophy (SMA) drugs
• Late phase: Phase 3 and in-approval Spinal Muscular Atrophy (SMA) drugs

Companies involved in the Pipeline-

• Company overview
• Snapshot
• Spinal Muscular Atrophy (SMA) therapeutic treatment activities

Details for each Spinal Muscular Atrophy (SMA) drug candidate-

• Snapshot
• Drug Name
• Alternative Names
• Company
• Originator
• Phase
• Molecule Type
• Orphan Drug Status
• Drug Overview
• Mechanism of Action
• Current Status
• Trial Details

Other details

• Pre-clinical trials for each drug candidate
• Clinical trials for each drug candidate

Recent Spinal Muscular Atrophy (SMA) therapeutic candidates, clinical trials, investments, grants, partnerships, licenses, awards and other news

Contents

1 TABLE OF CONTENTS

1.1 List of Tables
1.2 List of Figures

2 EXECUTIVE SUMMARY

2.1 Spinal Muscular Atrophy (SMA)- Disease Overview
2.2 Spinal Muscular Atrophy (SMA)- Pipeline Snapshot
2.3 Spinal Muscular Atrophy (SMA)- Pipeline Drugs by Phase
2.4 Spinal Muscular Atrophy (SMA)- Pipeline Drugs by Company
2.5 Spinal Muscular Atrophy (SMA)- Pipeline Drugs by Mechanism of Action

3 COMPANY WISE PIPELINE ANALYSIS

3.1 Armgo Pharma Inc Spinal Muscular Atrophy (SMA) Drug Pipeline, H2- 2019
3.2 AurimMed Pharma Inc Spinal Muscular Atrophy (SMA) Drug Pipeline, H2- 2019
3.3 AveXis Inc Spinal Muscular Atrophy (SMA) Drug Pipeline, H2- 2019
3.4 Biogen Inc Spinal Muscular Atrophy (SMA) Drug Pipeline, H2- 2019
3.5 BioMarin Pharmaceutical Inc Spinal Muscular Atrophy (SMA) Drug Pipeline, H2- 2019
3.6 Biophytis SA Spinal Muscular Atrophy (SMA) Drug Pipeline, H2- 2019
3.7 Cytokinetics Inc Spinal Muscular Atrophy (SMA) Drug Pipeline, H2- 2019
3.8 Exicure Inc Spinal Muscular Atrophy (SMA) Drug Pipeline, H2- 2019
3.9 F. Hoffmann-La Roche Ltd Spinal Muscular Atrophy (SMA) Drug Pipeline, H2- 2019
3.10 Genea Biocells Spinal Muscular Atrophy (SMA) Drug Pipeline, H2- 2019
3.11 Genethon SA Spinal Muscular Atrophy (SMA) Drug Pipeline, H2- 2019
3.12 GNT Pharma Co Ltd Spinal Muscular Atrophy (SMA) Drug Pipeline, H2- 2019
3.13 Kowa Co Ltd Spinal Muscular Atrophy (SMA) Drug Pipeline, H2- 2019
3.14 Neurodyn Life Sciences Inc Spinal Muscular Atrophy (SMA) Drug Pipeline, H2- 2019
3.15 Neurotune AG Spinal Muscular Atrophy (SMA) Drug Pipeline, H2- 2019
3.16 Novartis AG Spinal Muscular Atrophy (SMA) Drug Pipeline, H2- 2019
3.17 Ono Pharmaceutical Co Ltd Spinal Muscular Atrophy (SMA) Drug Pipeline, H2- 2019
3.18 Ractigen Therapeutics Inc Spinal Muscular Atrophy (SMA) Drug Pipeline, H2- 2019
3.19 Reborna Biosciences Inc Spinal Muscular Atrophy (SMA) Drug Pipeline, H2- 2019
3.20 Recursion Pharmaceuticals Inc Spinal Muscular Atrophy (SMA) Drug Pipeline, H2- 2019
3.21 Sarepta Therapeutics Inc Spinal Muscular Atrophy (SMA) Drug Pipeline, H2- 2019
3.22 Scholar Rock Inc Spinal Muscular Atrophy (SMA) Drug Pipeline, H2- 2019
3.23 Shift Pharmaceuticals Spinal Muscular Atrophy (SMA) Drug Pipeline, H2- 2019
3.24 Spotlight Innovation Inc Spinal Muscular Atrophy (SMA) Drug Pipeline, H2- 2019
3.25 Vybion Inc Spinal Muscular Atrophy (SMA) Drug Pipeline, H2- 2019
3.26 Xcelthera Inc Spinal Muscular Atrophy (SMA) Drug Pipeline, H2- 2019

4 PHASE 1 PIPELINE DRUG DETAILS

4.1 Spinal Muscular Atrophy (SMA)- Phase 1 Drug Details
4.2 Spinal Muscular Atrophy (SMA)- Phase 1 Drug Overview
4.3 Spinal Muscular Atrophy (SMA)- Phase 1 Mechanism of Action
4.4 Current Status
4.5 Trial Details

5 PHASE 2 PIPELINE DRUG DETAILS

5.1 Spinal Muscular Atrophy (SMA)- Phase 2 Drug Details
5.2 Spinal Muscular Atrophy (SMA)- Phase 2 Drug Overview
5.3 Spinal Muscular Atrophy (SMA)- Phase 2 Mechanism of Action
5.4 Current Status
5.5 Trial Details

6 PHASE 3 PIPELINE DRUG DETAILS

6.1 Spinal Muscular Atrophy (SMA)- Phase 3 Drug Details
6.2 Spinal Muscular Atrophy (SMA)- Phase 3 Drug Overview
6.3 Spinal Muscular Atrophy (SMA)- Phase 3 Drug Mechanism of Action
6.4 Current Status
6.5 Trial Details

7 PRE-CLINICAL PHASE PIPELINE DRUG DETAILS

7.1 Spinal Muscular Atrophy (SMA)- Pre-clinical Phase Drug Details
7.2 Spinal Muscular Atrophy (SMA)- Pre-clinical Phase Drug Overview
7.3 Spinal Muscular Atrophy (SMA)- Pre-clinical Phase Drug Mechanism of Action
7.4 Current Status
7.5 Trial Details

8 RECENT NEWS AND DEVELOPMENTS

9 APPENDIX

9.1 About VPAResearch
9.2 Sources and Research Methodology