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Recent Analysis on Amyotrophic Lateral Sclerosis Clinical Trials

DelveInsight Business Research LLP
·8 min read

DelveInsight reports a complete review on Current and Emerging Therapies in the Amyotrophic Lateral Sclerosis pipeline enriched through the genetic breakthroughs along with recent discoveries in the field of stem-cell/gene therapy as well as molecular pathology.

Los Angeles, USA, Feb. 22, 2021 (GLOBE NEWSWIRE) -- Recent Analysis on Amyotrophic Lateral Sclerosis Clinical Trials

DelveInsight reports a complete review on Current and Emerging Therapies in the Amyotrophic Lateral Sclerosis pipeline enriched through the genetic breakthroughs along with recent discoveries in the field of stem-cell/gene therapy as well as molecular pathology.


DelveInsight’s “Amyotrophic Lateral Sclerosis (ALS) Pipeline Insight'' report provides comprehensive insights about 160+ companies and 160+ pipeline drugs in the Amyotrophic Lateral Sclerosis pipeline landscapes. It comprises ALS pipeline drug profiles, including clinical and non-clinical stage products. It also includes the Amyotrophic Lateral Sclerosis therapeutics assessment by product type, stage, route of administration, and molecule type and further highlights the inactive ALS pipeline products.


Some of the significant features of the Amyotrophic Lateral Sclerosis Pipeline Report

  • There is no cure for ALS. Nevertheless, treatments can help control symptoms, prohibit unnecessary complications, and make a more manageable living with the disease.

  • Key Players such as BrainStorm Cell Therapeutics, Apellis Pharmaceuticals, Biogen, Alsonex Pharmaceuticals, Astellas Pharma, Grifols Therapeutics, AL-S Pharma, Genuv, Annexon, Mitsubishi Tanabe Pharma Corporation, Q Therapeutics, Acurastem, Everfront Biotech Co. Ltd, MediciNova, Novartis, Amylyx Pharmaceuticals Inc., Orphazyme, and many others are developing therapies that are focused on novel approaches to treat Amyotrophic Lateral Sclerosis.

  • The U.S. Food and Drug Administration (FDA) has authorized the drugs riluzole (Rilutek) and edaravone (Radicava) for ALS treatment.

  • The US FDA has granted ALS-205 orphan drug status for Amyotrophic Lateral Sclerosis treatment.

  • In October 2020, Catalent and BrainStorm Cell Therapeutics announced a deal for the manufacture of NurOwn, BrainStorm’s autologous cellular therapy being investigated for the treatment of ALS.

  • BIIB067 is being developed to treat ALS that is also known as Tofersen is a second-generation antisense oligonucleotide (ASO) targeting the mRNA for superoxide dismutase. The therapy was developed in a partnership between Ionis Pharmaceuticals and Biogen but is currently being developed solely by Biogen. Biogen licensed tofersen from Ionis Pharmaceuticals, Inc. under a collaborative development and license agreement.

  • Recent developments in the comprehension of the underlying pathophysiologic processes contributing to Amyotrophic Lateral Sclerosis have led to several investigational therapies, with numerous in phase 3 trials.

Get an overview of pipeline landscape @ Amyotrophic Lateral Sclerosis Clinical Trial Analysis


Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig's disease, is a group of disorders known as motor neuron diseases. It is characterized by the progressive degeneration and death of nerve cells in the brain and spinal cord, eventually that aid in communication between the nervous system and voluntary muscles of the body. It affects both the upper and lower motor neurons that interrupt the transmission.


There is no cure for ALS. However, treatments available can help control symptoms, prevent unnecessary complications, and make living with the disease easier. Many pharmaceuticals companies are developing therapies that are focused on novel approaches to treat Amyotrophic Lateral Sclerosis.


ALS Emerging Drugs

  • Autologous MSC-NTF cells by BrainStorm Cell Therapeutics

Autologous MSC-NTF cells are being developed by BrainStorm Cell Therapeutics for ALS treatment. BrainStorm is recently enrolling a phase III pivotal trial using repeat-administration of autologous MSC-NTF cells in ALS at six leading clinical sites in the United States, backed by a grant from the California Institute for Regenerative Medicine.

  • Pegcetacoplan by Apellis Pharmaceuticals

Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive activation of the complement cascade. Apellis plans to commence a clinical trial to comprehend if targeting C3 with pegcetacoplan may have the potential to slow the ALS progression by reducing the inflammation that causes motor neuron death. It is currently in the phase II stage of development.

  • BIIB078 by Biogen

BIIB078 targets specific messenger RNA emerging from the C9orf72 gene and deteriorates it, eliminating the RNA and prohibiting the abnormal protein production it encodes. Importantly, BIIB078 largely preserves the normal C9orf72 protein emerging from the gene. It is currently in the phase I stage of development.

  • ALS-205 by Alsonex Pharmaceuticals

ALS-205 is a potent antagonist of the human C5a1 receptor with no activity at the C5a2 receptor. It binds to the human C5a1 receptor on white blood cells and is a functional insurmountable antagonist. ALS-205 has displayed similar effects on other cell types, comprising lymphocytes, monocytes, and monocyte-derived macrophages. It is currently in the preclinical stage of development.

For further product profiles, request @ ALS Pipeline Therapeutics


Scope of Amyotrophic Lateral Sclerosis Pipeline Drug Insight

  • Coverage: Global

  • Major Players: 160+ Key Players

  • Prominent Players: BrainStorm Cell Therapeutics, Apellis Pharmaceuticals, Biogen, Alsonex Pharmaceuticals, Astellas Pharma, Grifols Therapeutics, AL-S Pharma, Genuv, Annexon, Mitsubishi Tanabe Pharma Corporation, Q Therapeutics, Acurastem, Everfront Biotech Co. Ltd, MediciNova, Novartis, Amylyx Pharmaceuticals Inc. Orphazyme, and many others.

  • Key Drugs Profiles: 160+ Products

  • Phases:

  • Amyotrophic Lateral Sclerosis Therapies Late-stage (Phase III)

  • ALS Therapies (Phase II)

  • Amyotrophic Lateral Sclerosis Therapies (Phase I)

  • ALS Pre-clinical stage and Discovery candidates

  • Discontinued and Inactive candidates

  • Molecule Types:

  • Gene therapies

  • Small molecule

  • Vaccines

  • Polymers

  • Peptides

  • Monoclonal antibodies

  • Mechanism of Action:

  • Protease inhibitors

  • Immunomodulatory

  • Multiple kinase inhibitor

  • Route of Administration:

  • Infusion

  • Intradermal

  • Intramuscular

  • Intranasal

  • Oral

  • Parenteral

  • Subcutaneous

  • Topical

  • Product Types:

  • Monotherapy

  • Combination

Key Questions regarding Current Amyotrophic Lateral Sclerosis Treatment Landscape and Emerging Therapies Answered in the Pipeline Report

  • What are the current options for Amyotrophic Lateral Sclerosis treatment?

  • How many companies are developing therapies for the treatment of ALS?

  • How many are Amyotrophic Lateral Sclerosis emerging therapies in the early-stage, mid-stage, and late stages of development for the treatment of ALS?

  • What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, and significant licensing activities that will impact the Amyotrophic Lateral Sclerosis market?

  • Which are the dormant and discontinued products and the reasons for the same?

  • What is the unmet need for current therapies for the treatment of ALS?

  • What are the current novel therapies, targets, mechanisms of action, and technologies developed to overcome the limitation of existing Amyotrophic Lateral Sclerosis therapies?

  • What are the critical designations that have been granted for the emerging therapies for ALS?

  • How many patents are granted and pending for the emerging therapies to treat Amyotrophic Lateral Sclerosis?

Table of Contents

1

Amyotrophic Lateral Sclerosis Introduction

2

ALS Executive Summary

3

Amyotrophic Lateral Sclerosis Overview

4

ALS Analytical Perspective In-depth Commercial Assessment

5

Amyotrophic Lateral Sclerosis Pipeline Therapeutics

5.1

ALS Late Stage Products (Phase III)

5.1.1

BIIB067: Biogen

5.1.2

Arimoclomol: Orphazyme

5.2

Amyotrophic Lateral Sclerosis Mid Stage Products (Phase II/III)

5.2.1

MN-166: MediciNova

5.3

Amyotrophic Lateral Sclerosis Mid Stage Products (Phase II)

5.3.1

BLZ945: Novartis

5.4

ALS Early Stage Products (Phase I/II)

5.4.1

AP-101: AL-S Pharma

5.5

ALS Pre-clinical and Discovery Stage Products

5.5.1

AS-202: Acurastem

6

Amyotrophic Lateral Sclerosis Therapeutic Assessment

7

ALS Inactive Products

8

Company-University Collaborations (Licensing/Partnering) Amyotrophic Lateral Sclerosis Analysis

9

ALS Key Companies

10

Amyotrophic Lateral Sclerosis Key Products

11

ALS Unmet Needs

12

Amyotrophic Lateral Sclerosis Market Drivers and Barriers

13

ALS Future Perspectives and Conclusion

14

Amyotrophic Lateral Sclerosis Analyst Views

15

Appendix

16

About DelveInsight

Get customized pipeline report @ ALS Drugs Pipeline Report

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About DelveInsight
DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing end to end comprehensive solutions to improve their performance.

CONTACT: Contact Us: Shruti Thakur info@delveinsight.com +1(919)321-6187 www.delveinsight.com