100+ Leading Amyotrophic Lateral Sclerosis Pipeline Companies are working to improve the Treatment Landscape

100+ Leading Amyotrophic Lateral Sclerosis Pipeline Companies are working to improve the Treatment Landscape

DelveInsight’s, “Amyotrophic Lateral Sclerosis Pipeline Insight, 2023,” report provides comprehensive insights about 100+ companies and 100+ pipeline drugs in the Amyotrophic Lateral Sclerosis pipeline landscape. It covers the Amyotrophic Lateral Sclerosis pipeline drug profiles, including Amyotrophic Lateral Sclerosis clinical trials 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.

 

In the Amyotrophic Lateral Sclerosis pipeline report, detailed description of the drug is given which includes mechanism of action of the drug, Amyotrophic Lateral Sclerosis clinical trials studies, Amyotrophic Lateral Sclerosis NDA approvals (if any), and product development activities comprising the technology, Amyotrophic Lateral Sclerosis (ALS) collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

 

Key takeaways from the Amyotrophic Lateral Sclerosis Pipeline Insight Report

 

  • DelveInsight’s Amyotrophic Lateral Sclerosis pipeline report depicts a robust space with 100+ active players working to develop 100+ pipeline therapies for Amyotrophic Lateral Sclerosis treatment.

 

  • The leading Amyotrophic Lateral Sclerosis Companies includes Biogen, Ionis Pharmaceuticals, Cytokinetics, AB Sciences, Alexion Pharmaceuticals, Orion Pharma, Orphazyme, Brainstorm-Cell Therapeutics, Amylyx Pharmaceuticals, Medicinova, Procypra Therapeutics, and others.

 

  • Promising Amyotrophic Lateral Sclerosis Pipeline Therapies includes BIIB067, ANX005, AMX0035, MN-166, BLZ945, AP-101, AS-202, Arimoclomol, ALZT-OP1a, AT1501, HK001, Q-Cells, and others.

 

  • The Amyotrophic Lateral Sclerosis companies and academics are working to assess challenges and seek opportunities that could influence Amyotrophic Lateral Sclerosis (ALS) R&D. The Amyotrophic Lateral Sclerosis pipeline therapies under development are focused on novel approaches to treat/improve Amyotrophic Lateral Sclerosis (ALS).

 

To explore more information on the latest breakthroughs in the Amyotrophic Lateral Sclerosis Pipeline treatment landscape of the report, click here @ Amyotrophic Lateral Sclerosis Pipeline Outlook

 

Amyotrophic Lateral Sclerosis Overview

Amyotrophic Lateral Sclerosis (ALS) (ALS) is a rare neurological disease that primarily affects the nerve cells (neurons) responsible for controlling voluntary muscle movement (those muscles we choose to move). 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. ALS belongs to a wider group of disorders known as motor neuron diseases, which are caused by gradual deterioration (degeneration) and death of motor neurons. Motor neurons are nerve cells that extend from the brain to the spinal cord and to muscles throughout the body.

 

Recent Developmental Activities in the Amyotrophic Lateral Sclerosis Treatment Landscape

  • In May 2021, Seelos Therapeutics announced it has received European Orphan Drug Designation for SLS-005 in amyotrophic lateral sclerosis (ALS) from the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP).
  • In May 2021, Aeterna Zentaris announced the commencement of the previously announced preclinical program to qualify macimorelin for clinical development as a potential treatment option for amyotrophic lateral sclerosis (ALS; Lou Gehrig’s disease).
  • In May 2021, Orphazyme announced that the ORARIALS-01 pivotal trial of arimoclomol in amyotrophic lateral sclerosis (ALS) did not meet its primary and secondary endpoints to show benefit in people living with ALS. No important safety signals were reported in the trial.
  • In March 2021, Retrotope announced that the first patient has been dosed in a multicenter Phase 2 clinical trial evaluating RT001, the company’s lead development candidate, in patients with amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease).

 

For further information, refer to the detailed Amyotrophic Lateral Sclerosis Unmet Needs, Amyotrophic Lateral Sclerosis Market Drivers, and Amyotrophic Lateral Sclerosis Market Barriers, click here for Amyotrophic Lateral Sclerosis Ongoing Clinical Trial Analysis

 

Amyotrophic Lateral Sclerosis Emerging Drugs Profile

 

AMX0035: Amylyx Pharmaceuticals

AMX0035 is an investigational product comprised of two complementary active agents, sodium phenylbutyrate (PB) and taurursodiol (TURSO), which were combined in a co-formulation to reduce neuronal death and dysfunction. AMX0035 was specifically co-formulated and manufactured by Amylyx to ensure proper absorption, exposure, and quality. AMX0035 targets endoplasmic reticulum and mitochondrial dependent neuronal degeneration pathways in ALS and other neurodegenerative diseases. The company has submitted a New Drug Submission (NDS) to Health Canada for AMX0035 (sodium phenylbutyrate (PB)-taurursodiol (TURSO)) for the treatment of ALS.

 

BIIB067: Biogen

BIIB067 (tofersen) is thought to reduce the production of SOD1 protein and is being evaluated in Phase III clinical trial to see if it can potentially slow the fatal progression of SOD1-ALS. Tofersen (BIIB067) previously called IONIS-SOD1Rx is an investigational therapy to slow the progression of familial Amyotrophic Lateral Sclerosis (ALS) (ALS). The therapy was developed in a collaboration between Ionis Pharmaceuticals and Biogen but is now being developed solely by Biogen.

 

Masitinib: AB Sciences

Masitinib is currently the only tyrosine kinase inhibitor in late-stage development for ALS. Masitinib distinguishes itself from other ALS developmental drugs by exerting neuroprotection in both central and peripheral nervous systems. Masitinib appears exceptional among other ALS-developmental drugs, exerting neuroprotection in both central nervous system and peripheral nervous system via selective kinase inhibition that modulates the functionality of different cells implicated in ALS pathogenesis. In recognition of the critical need for new treatments, masitinib received orphan drug designation for ALS from both the European Medicine Agency (EMA) and the U.S. Food and Drug Administration (FDA).

 

Ravulizumab: Alexion Pharmaceuticals

Ravulizumab is the first and only approved long-acting C5 complement inhibitor. It is administered intravenously every eight weeks or every four weeks for pediatric patients less than 20 kg, following a loading dose. ULTOMIRIS works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system. The terminal complement cascade, when activated in an uncontrolled manner, plays a role in severe ultra-rare disorders. ULTOMIRIS is approved in the U.S., Japan, and the EU as a treatment for adults with PNH and in the U.S. for aHUS to inhibit complement-mediated thrombotic microangiopathy (TMA) in adult and pediatric (one month of age and older) patients. The drug is being evaluated in Phase III clinical trial to treat patients with ALS.

 

Autologous MSC-NTF cells: BrainStorm Cell Therapeutics

BrainStorm has entered into agreements with Dana-Farber Cancer Institute (Dana-Farber) in Boston, Massachusetts and the City of Hope National Medical Center in Duarte, California to provide clean room facilities for production of autologous MSC-NTF cells. The company has completed a phase III clinical trial of autologous MSC-NTF cellular therapy in Amyotrophic Lateral Sclerosis.

 

Amyotrophic Lateral Sclerosis Pipeline Therapeutics Assessment

There are approx. 100+ key companies which are developing the therapies for Amyotrophic Lateral Sclerosis (ALS). The companies which have their Amyotrophic Lateral Sclerosis (ALS) drug candidates in the most advanced stage, i.e. Preregistration include, Amylyx Pharmaceuticals.

 

Request a sample and discover the recent advances in Amyotrophic Lateral Sclerosis Ongoing Clinical Trial Analysis and Medications, click here @ Amyotrophic Lateral Sclerosis Treatment Landscape

 

Scope of the Amyotrophic Lateral Sclerosis Pipeline Report

 

  • Coverage- Global

 

  • Amyotrophic Lateral Sclerosis Companies- Biogen, Ionis Pharmaceuticals, Cytokinetics, AB Sciences, Alexion Pharmaceuticals, Orion Pharma, Orphazyme, Brainstorm-Cell Therapeutics, Amylyx Pharmaceuticals, Medicinova, Procypra Therapeutics, and others.

 

  • Amyotrophic Lateral Sclerosis Pipeline Therapies- BIIB067, ANX005, AMX0035, MN-166, BLZ945, AP-101, AS-202, Arimoclomol, ALZT-OP1a, AT1501, HK001, Q-Cells, and others.

 

  • Amyotrophic Lateral Sclerosis Pipeline Segmentation: Product Type, Molecule Type, Mechanism of Action, Route of Administration

 

Dive deep into rich insights for drugs for Amyotrophic Lateral Sclerosis Market Drivers and Amyotrophic Lateral Sclerosis Market Barriers, click here @ Amyotrophic Lateral Sclerosis Unmet Needs and Analyst Views

 

Table of Content

  1. Introduction
  2. Executive Summary
  3. Amyotrophic Lateral Sclerosis (ALS): Overview
  4. Pipeline Therapeutics
  5. Therapeutic Assessment
  6. Late Stage Products (Preregistration)
  7. AMX0035: Amylyx Pharmaceuticals
  8. Drug profiles in the detailed report…..
  9. Mid Stage Products (Phase II)
  10. ANX005: Annexon
  11. Drug profiles in the detailed report…..
  12. Early stage products (Phase I/II)
  13. Q-cells: Q therapeutics
  14. Drug profiles in the detailed report…..
  15. Inactive Products
  16. Amyotrophic Lateral Sclerosis (ALS) Key Companies
  17. Amyotrophic Lateral Sclerosis (ALS) Key Products
  18. Amyotrophic Lateral Sclerosis (ALS)- Unmet Needs
  19. Amyotrophic Lateral Sclerosis (ALS)- Market Drivers and Barriers
  20. Amyotrophic Lateral Sclerosis (ALS)- Future Perspectives and Conclusion
  21. Amyotrophic Lateral Sclerosis (ALS) Analyst Views
  22. Amyotrophic Lateral Sclerosis (ALS) Key Companies
  23. Appendix

 

Got Queries? Find out the related information on Amyotrophic Lateral Sclerosis Mergers and acquisitions, Amyotrophic Lateral Sclerosis Licensing Activities @ Amyotrophic Lateral Sclerosis Emerging Drugs, and Recent Trends

 

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