Amyotrophic Lateral Sclerosis Als Pipeline Insight

DelveInsight’s, “Amyotrophic lateral sclerosis – Pipeline Insight, 2020,” report provides comprehensive insights about 250+ companies and 250+ pipeline drugs in Amyotrophic lateral sclerosis pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space. 

Geography Covered

  • Global coverage

Amyotrophic lateral sclerosis Understanding

Amyotrophic lateral sclerosis: Overview

Amyotrophic lateral sclerosis (ALS) is a group of rare neurological diseases that mainly involve the nerve cells (neurons) responsible for controlling voluntary muscle movement. Voluntary muscles produce movements like chewing, walking, and talking. The disease is progressive, meaning the symptoms get worse over time. Currently, there is no cure for ALS and no effective treatment to halt, or reverse, the progression of the disease.

Symptoms

The onset of ALS can be so subtle that the symptoms are overlooked but gradually these symptoms develop into more obvious weakness or atrophy that may cause a physician to suspect ALS. Some of the early symptoms include:

  • Fasciculations (muscle twitches) in the arm, leg, shoulder, or tongue
  • Muscle cramps
  • Tight and stiff muscles (spasticity)
  • Muscle weakness affecting an arm, a leg, neck or diaphragm.
  • Slurred and nasal speech
  • Difficulty chewing or swallowing.

Diagnosis

No one test can provide a definitive diagnosis of ALS. ALS is primarily diagnosed based on detailed history of the symptoms and signs observed by a physician during physical examination along with a series of tests to rule out other mimicking diseases. However, the presence of upper and lower motor neuron symptoms strongly suggests the presence of the disease. Physicians will review an individual’s full medical history and conduct a neurologic examination at regular

Treatment

No cure has yet been found for ALS. However, there are treatments available that can help control symptoms, prevent unnecessary complications, and make living with the disease easier.


The U.S. Food and Drug Administration (FDA) has approved the drugs riluzole (Rilutek) and edaravone (Radicava) to treat ALS. Riluzole is believed to reduce damage to motor neurons by decreasing levels of glutamate, which transports messages between nerve cells and motor neurons. Physicians can also prescribe medications to help manage symptoms of ALS, including muscle cramps, stiffness, excess saliva and phlegm, and the pseudobulbar affect (involuntary or uncontrollable episodes of crying and/or laughing, or other emotional displays).

Amyotrophic lateral sclerosis Emerging Drugs Chapters

This segment of the Amyotrophic lateral sclerosis report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Amyotrophic lateral sclerosis Emerging Drugs

  • Autologous MSC-NTF cells: BrainStorm Cell Therapeutics

Autologous MSC-NTF cells is being developed by BrainStorm Cell Therapeutics for the treatment of of ALS. BrainStorm is currently 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, supported by a grant from the California Institute for Regenerative Medicine.


  • Pegcetacoplan: Apellis Pharmaceuticals

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


  • BIIB078: Biogen

BIIB078 targets specific messenger RNA emerging from the C9orf72 gene and degrades it, removing the RNA and preventing production of the abnormal protein it encodes. Importantly, BIIB078 largely preserves the normal C9orf72 protein coming from the gene. Preclinical researchers have found this approach to be useful, decreasing ALS-related pathology in preclinical models and improving behavioral function. It is currently in phase I stage of development.


  • ALS-205: 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 shown similar effects on other cell types including lymphocytes, monocytes and monocyte-derived macrophages. It is also highly receptor selective, failing even at 100 μM concentrations to antagonize myeloperoxidase release from leukocytes stimulated with fMet-Leu-Phe, leukotriene B4, platelet-activating factor, C3a, or IL-8. ALS-205 is chemically stable to peptidase degradation in blood and gastric fluids and is both small enough and sufficiently lipophilic to be orally active at ≤1 mg/kg/day in vivo and readily accesses the brain. It is currently in preclinical stage of development.

Further product details are provided in the report……..

Amyotrophic lateral sclerosis: Therapeutic Assessment

This segment of the report provides insights about the different Amyotrophic lateral sclerosis drugs segregated based on following parameters that define the scope of the report, such as:


  • Major Players in Amyotrophic lateral sclerosis

There are approx. 250+ key companies which are developing the therapies for Amyotrophic lateral sclerosis. The companies which have their Amyotrophic lateral sclerosis drug candidates in the most advanced stage, i.e. phase III include BrainStorm Cell Therapeutics and others


  • Phases

DelveInsight’s report covers around 250+ products under different phases of clinical development like

  • Late-stage products (Phase III)
  • Mid-stage products (Phase II)
  • Early-stage products (Phase I) along with the details of
  • Pre-clinical and Discovery stage candidates
  • Discontinued & Inactive candidates
  • Route of Administration


Amyotrophic lateral sclerosis pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

  • Infusion
  • Intradermal
  • Intramuscular
  • Intranasal
  • Oral
  • Parenteral
  • Subcutaneous
  • Topical.
  • Molecule Type


Products have been categorized under various Molecule types such as

  • Gene therapies
  • Small molecule
  • Vaccines
  • Polymers
  • Peptides
  • Monoclonal antibodies
  • Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.


Amyotrophic lateral sclerosis: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase III, II, I, preclinical and discovery stage. It also analyses Amyotrophic lateral sclerosis therapeutic drugs key players involved in developing key drugs.


Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Amyotrophic lateral sclerosis drugs.

Report Highlights

  • The companies and academics are working to assess challenges and seek opportunities that could influence Amyotrophic lateral sclerosis R&D. The therapies under development are focused on novel approaches to treat/improve Amyotrophic lateral sclerosis.
  • ALS-205 has been granted orphan drug status by the US FDA for ALS
  • In October 2020, Catalent and BrainStorm Cell Therapeutics announced an agreement for the manufacture of NurOwn, BrainStorm’s autologous cellular therapy being investigated for the treatment of amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease or motor neuron disease.


Amyotrophic lateral sclerosis Report Insights

  • Amyotrophic lateral sclerosis Pipeline Analysis
  • Therapeutic Assessment
  • Unmet Needs
  • Impact of Drugs


Amyotrophic lateral sclerosis Report Assessment

  • Pipeline Product Profiles
  • Therapeutic Assessment
  • Pipeline Assessment
  • Inactive drugs assessment
  • Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

  • How many companies are developing Amyotrophic lateral sclerosis drugs?
  • How many Amyotrophic lateral sclerosis drugs are developed by each company?
  • How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Amyotrophic lateral sclerosis?
  • What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Amyotrophic lateral sclerosis therapeutics?
  • What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
  • What are the clinical studies going on for Amyotrophic lateral sclerosis and their status?
  • What are the key designations that have been granted to the emerging drugs?

Key Players

  • BrainStorm Cell Therapeutics
  • Apellis Pharmaceuticals
  • Biogen
  • Alsonex Pharmaceuticals
  • Astellas Pharma
  • Grifols Therapeutics
  • AL-S Pharma
  • Genuv
  • Annexon
  • Mitsubishi Tanabe Pharma Corporation

Key Products

  • ALS-205
  • BIIB078
  • Pegcetacoplan
  • Autologous MSC-NTF cells
  • Reldesemtiv
  • Albutein
  • BIIB067
  • AP-101
  • Trametinib
  • ANX005
  • MCI-186


Introduction

Executive Summary

Amyotrophic lateral sclerosis: Overview

• Causes

• Mechanism of Action

• Signs and Symptoms

• Diagnosis

• Disease Management

Pipeline Therapeutics

• Comparative Analysis

Therapeutic Assessment

• Assessment by Product Type

• Assessment by Stage and Product Type

• Assessment by Route of Administration

• Assessment by Stage and Route of Administration

• Assessment by Molecule Type

• Assessment by Stage and Molecule Type

Amyotrophic lateral sclerosis – DelveInsight’s Analytical Perspective

In-depth Commercial Assessment

• Amyotrophic lateral sclerosis companies’ collaborations, Licensing, Acquisition -Deal Value Trends

Amyotrophic lateral sclerosis Collaboration Deals

• Company-Company Collaborations (Licensing / Partnering) Analysis

• Company-University Collaborations (Licensing / Partnering) Analysis

Late Stage Products (Phase III)

• Comparative Analysis

Autologous MSC-NTF cells: BrainStorm Cell Therapeutics

• Product Description

• Research and Development

• Product Development Activities

Mid Stage Products (Phase II)

• Comparative Analysis

Pegcetacoplan: Apellis Pharmaceuticals

• Product Description

• Research and Development

• Product Development Activities

Drug profiles in the detailed report…..

Early Stage Products (Phase I)

• Comparative Analysis

BIIB078: Biogen

• Product Description

• Research and Development

• Product Development Activities

Drug profiles in the detailed report…..

Pre-clinical and Discovery Stage Products

• Comparative Analysis

ALS-205: Alsonex Pharmaceuticals

• Product Description

• Research and Development

• Product Development Activities

Inactive Products

• Comparative Analysis

Amyotrophic lateral sclerosis Key Companies

Amyotrophic lateral sclerosis Key Products

Amyotrophic lateral sclerosis- Unmet Needs

Amyotrophic lateral sclerosis- Market Drivers and Barriers

Amyotrophic lateral sclerosis- Future Perspectives and Conclusion

Amyotrophic lateral sclerosis Analyst Views

Amyotrophic lateral sclerosis Key Companies

Appendix

List of Table

 Table 1 Total Products for Amyotrophic lateral sclerosis

Table 2 Late Stage Products

Table 3 Mid Stage Products

Table 4 Early Stage Products

Table 5 Pre-clinical & Discovery Stage Products

Table 6 Assessment by Product Type

Table 7 Assessment by Stage and Product Type

Table 8 Assessment by Route of Administration

Table 9 Assessment by Stage and Route of Administration

Table 10 Assessment by Molecule Type

Table 11 Assessment by Stage and Molecule Type

Table 12 Inactive Products

List of Figures

 Figure 1 Total Products for Amyotrophic lateral sclerosis

Figure 2 Late Stage Products

Figure 3 Mid Stage Products

Figure 4 Early Stage Products

Figure 5 Preclinical and Discovery Stage Products

Figure 6 Assessment by Product Type

Figure 7 Assessment by Stage and Product Type

Figure 8 Assessment by Route of Administration

Figure 9 Assessment by Stage and Route of Administration

Figure 10 Assessment by Molecule Type

Figure 11 Assessment by Stage and Molecule Type

Figure 12 Inactive Products

• BrainStorm Cell Therapeutics

• Apellis Pharmaceuticals

• Biogen

• Alsonex Pharmaceuticals

• Astellas Pharma

• Grifols Therapeutics

• AL-S Pharma

• Genuv

• Annexon

• Mitsubishi Tanabe Pharma Corporation

  • Tags:
  • Amyotrophic lateral sclerosis (ALS)...
  • Amyotrophic lateral sclerosis (ALS)...
  • Amyotrophic lateral sclerosis (ALS)...
  • Amyotrophic lateral sclerosis (ALS)...
  • Amyotrophic lateral sclerosis (ALS)...
  • Amyotrophic lateral sclerosis (ALS)...
  • Amyotrophic lateral sclerosis (ALS)...
  • Amyotrophic lateral sclerosis (ALS)...

Forward to Friend

Need A Quote