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Amyotrophic Lateral Sclerosis Pipeline Booms as 100+ Therapies Likely to Enter in the Treatment Domain | DelveInsight

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DelveInsight Business Research, LLP

21 Feb, 2023, 15:30 GMT

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The prevalence of amyotrophic lateral sclerosis has been rising over the past few years, which prompts the growing demand for treatment options. The increasing prevalence of amyotrophic lateral sclerosis and the growing research and development activities to develop novel therapies to treat amyotrophic lateral sclerosis to drive the market. The companies developing the potential therapies in the last stage of development include Biogen, AB Sciences, Helixmith, and several others.

LAS VEGAS, Feb. 21, 2023 /PRNewswire/ -- DelveInsight's 'Amyotrophic Lateral Sclerosis Pipeline Insight – 2023' report provides comprehensive global coverage of available, marketed, and pipeline amyotrophic lateral sclerosis therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the amyotrophic lateral sclerosis pipeline domain. 

Key Takeaways from the Amyotrophic Lateral Sclerosis Pipeline 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. 
  • Key amyotrophic lateral sclerosis companies such as Biogen, Ionis Pharmaceuticals, Ferrer Internacional S.A., AbbVie, Calico Life Sciences LLC, Genuv Inc., Kadimastem, Corcept Therapeutics, AL-S Pharma, Rapa Therapeutics LLC, Cytokinetics, MediciNova, Retrotope, Inc. Woolsey Pharmaceuticals, Sanofi, PTC Therapeutics, Helixmith Co., Ltd., Annexon, Inc., Denali Therapeutics Inc., Revalesio Corporation, Clene Nanomedicine, Ashvattha Therapeutics, Inc., Apellis Pharmaceuticals, Inc., Procypra Therapeutics, Knopp Biosciences, InFlectis BioScience, AI Therapeutics, Inc., Cellenkos, ZZ Biotech, LLC, QurAlis Corporation, Alector Inc., NeuroSense Therapeutics Ltd., Novartis Pharmaceuticals, Eledon Pharmaceuticals,  and others are evaluating new amyotrophic lateral sclerosis drugs to improve the treatment landscape.
  • Promising amyotrophic lateral sclerosis pipeline therapies in various stages of development include BIIB067, FAB122, ABBV-CLS-7262, Trametinib, BIIB105, AstroRx, Dazucorilant, AP-101,  RAPA-501, ION-363, Reldesemtiv, MN-166, RT001, Fasudil,  SAR443820, PTC857, ANX005, DNL343, RNS60, BIIB100, CNMAu8, 18F-OP-801, Pegcetacoplan (APL-2), Cu(II)ATSM, KNS-760704, IFB-088, LAM-002A,  CK 0803, 3K3A-APC,  QRL-201, Dexpramipexole, AL001, PrimeC, BLZ945, AT-1501,  and others.
  • In February 2023, NeuroSense Therapeutics Ltd. and QuantalX Neuroscience Ltd; the developer of Delphi-MD, a clinically objective neurodiagnostic medical device, announced a collaboration to improve early diagnosis and treatment of neurodegenerative diseases.In addition, the companies agreed that QuantalX's Delphi-MD would be used for early diagnosis and ongoing monitoring of trial participants in NeuroSense's planned future pivotal Phase III efficacy trial of PrimeC in people living with amyotrophic lateral sclerosis (ALS), pending the successful conclusion of its ALS Phase IIb trial.
  • In February 2023, Amylyx Pharmaceuticals, Inc. announced the completion of enrollment in PHOENIX, a global, 48-week, randomized, placebo-controlled Phase III clinical trial of AMX0035 (sodium phenylbutyrate and taurursodiol [also known as ursodoxicoltaurine]) in people living with amyotrophic lateral sclerosis (ALS). Amylyx anticipates topline results in 2024. The study enrolled 664 participants living with ALS.
  • In February 2023, UMass Chan Medical School signed a three-year sponsored research agreement with NeuShen Therapeutics Inc. to investigate a gene therapy treatment for amyotrophic lateral sclerosis, a rare neurological disease. Based in Shanghai and Boston, NeuShen is focused on developing innovative treatments for central nervous system disorders using adeno-associated virus-based gene therapy and small molecule delivery.
  • In January 2023, Inflamed Pharma, part of the incubator and accelerator company Xlife Sciences,  partnered with researchers at the University Medical Center Gottingen in Germany to develop its investigational therapy ProcCluster for the treatment of amyotrophic lateral sclerosis (ALS).The collaboration, led by Jan C. Koch, MD, a principal investigator at the university's Department of Neurology, will be focused particularly on  investigating ProcCluster in cellular models of ALS.
  • In January 2023, Amylyx Pharmaceuticals and Neopharm signed a license and distribution agreement to commercialize AMX0035 (sodium phenylbutyrate and ursodoxicoltaurine) to treat amyotrophic lateral sclerosis (ALS).Neopharm will commercialize the oral, fixed-dose medication in Israel, West Bank, Gaza, and the Palestinian Authority, subject to regulatory review and clearance.According to the agreement, Amylyx will grant exclusive rights to Neopharm for commercializing the therapy in the covered territory.
  • In January 2023, uniQure N.V. and Apic Bio announced that they have entered into a global licensing agreement for APB-102 to treat superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS), a rare, genetic form of ALS. Under the agreement, uniQure acquires global rights for the development and commercialization of APB-102, adding to its pipeline of gene therapies to treat neurological disorders.

Request a sample and discover the recent advances in amyotrophic lateral sclerosis drug treatment @ Amyotrophic Lateral Sclerosis Pipeline Report

The amyotrophic lateral sclerosis pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage amyotrophic lateral sclerosis drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the amyotrophic lateral sclerosis clinical trial landscape.

Amyotrophic Lateral Sclerosis Overview

Amyotrophic Lateral Sclerosis (ALS) is a rare neurological disease that affects the nerve cells (neurons) that control voluntary muscle movement (those muscles we choose to move). Voluntary muscles are responsible for movements such as chewing, walking, and talking. The disease is progressive, which means that the symptoms worsen over time. There is currently no cure for ALS and no effective treatment to slow or reverse the disease's progression.

Muscle weakness or stiffness are common early ALS symptoms. Individuals gradually lose strength and the ability to speak, eat, move, and even breathe as all voluntary muscles are affected. Most people with ALS die from respiratory failure, usually within 3 to 5 years of the onset of symptoms. However, approximately 10% of people with ALS live for ten years or more.

There is no single test that can definitively diagnose ALS. A detailed history of the symptoms observed by a physician during physical examination, as well as a review of the individual's full medical history and a series of tests to rule out other diseases, are used to make the ALS diagnosis.

Find out more about drugs for amyotrophic lateral sclerosis @ New Amyotrophic Lateral Sclerosis Drugs 

A snapshot of the Amyotrophic Lateral Sclerosis Pipeline Drugs mentioned in the report:

Drugs

Company

Phase 

MoA

RoA

BIIB067

Biogen

Preregistration

RNA interference; Superoxide dismutase 1 expression inhibitors

Intrathecal

Masitinib

AB Sciences

Preregistration

Angiogenesis inhibitors; Colony stimulating factor inhibitors; Coronavirus-3C-like-proteinase inhibitors; Endopeptidase Clp inhibitors; Focal adhesion protein-tyrosine kinase inhibitors; Lyn protein-tyrosine kinase inhibitors; Macrophage colony-stimulating factor receptor modulators; Mast cell inhibitors; Platelet-derived growth factor receptor antagonists; Protein tyrosine kinase inhibitors; Proto oncogene protein c-kit inhibitors; Proto-oncogene protein c-fyn modulators; Type 3 fibroblast growth factor receptor antagonists

Oral

Engensis

Helixmith

Phase II

Angiogenesis inducing agents; Gene transference; Hepatocyte growth factor expression stimulants

Intramuscular

RNS60

Revalesio Corporation

Phase II

G protein-coupled receptor modulators; HSP90 heat-shock protein modulators; Ion channel modulators

Inhalation

EPI-589 

PTC Therapeutics

Phase II

Antioxidants; NAD(P)H dehydrogenase (quinone) modulators

Oral

AT-1501 

Eledon Pharmaceuticals

Phase II

CD40 ligand inhibitors

Intravenous

ANX005 

Annexon

Phase II

Complement C1 inhibitors

Intravenous

Pegcetacoplan 

Apellis Pharmaceuticals

Phase II

Complement C3 inhibitors

Subcutaneous

PrimeC 

NeuroSense Therapeutics

Phase II

Cyclo-oxygenase 2 inhibitors; DNA gyrase inhibitors

NA

Learn more about the emerging amyotrophic lateral sclerosis pipeline therapies @ Amyotrophic Lateral Sclerosis Clinical Trials

Amyotrophic Lateral Sclerosis Therapeutics Assessment

The amyotrophic lateral sclerosis pipeline report proffers an integral view of amyotrophic lateral sclerosis emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration.

Scope of the Amyotrophic Lateral Sclerosis Pipeline Report 

  • Coverage: Global 
  • Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination
  • Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III
  • Therapeutics Assessment By Route of Administration: Oral, Parenteral, Intravenous, Subcutaneous, Topical
  • Therapeutics Assessment By Molecule Type: Monoclonal Antibody, Peptides, Polymer, Small molecule, Gene therapy
  • Therapeutics Assessment By Mechanism of Action: RNA interference, Superoxide dismutase 1 expression inhibitors, Angiogenesis inhibitors, Colony stimulating factor inhibitors, Coronavirus-3C-like-proteinase inhibitors, Endopeptidase Clp inhibitors, Focal adhesion protein-tyrosine kinase inhibitors, Lyn protein-tyrosine kinase inhibitors, Macrophage colony-stimulating factor receptor modulators, Mast cell inhibitors, Platelet-derived growth factor receptor antagonists, Protein tyrosine kinase inhibitors, Proto oncogene protein c-kit inhibitors, Proto-oncogene protein c-fyn modulators, Type 3 fibroblast growth factor receptor antagonists, Angiogenesis inducing agents, Gene transference, Hepatocyte growth factor expression stimulants, G protein-coupled receptor modulators, HSP90 heat-shock protein modulators, Ion channel modulators, Antioxidants, NAD(P)H dehydrogenase (quinone) modulators, CD40 ligand inhibitors, Complement C1 inhibitors, Cyclo-oxygenase 2 inhibitors, DNA gyrase inhibitors
  • Key Amyotrophic Lateral Sclerosis Companies: Biogen, Ionis Pharmaceuticals, Ferrer Internacional S.A., AbbVie, Calico Life Sciences LLC, Genuv Inc., Kadimastem, Corcept Therapeutics, AL-S Pharma, Rapa Therapeutics LLC, Cytokinetics, MediciNova, Retrotope, Inc. Woolsey Pharmaceuticals, Sanofi, PTC Therapeutics, Helixmith Co., Ltd., Annexon, Inc., Denali Therapeutics Inc., Revalesio Corporation, Clene Nanomedicine, Ashvattha Therapeutics, Inc., Apellis Pharmaceuticals, Inc., Procypra Therapeutics, Knopp Biosciences, InFlectis BioScience, AI Therapeutics, Inc., Cellenkos, ZZ Biotech, LLC, QurAlis Corporation, Alector Inc., NeuroSense Therapeutics Ltd., Novartis Pharmaceuticals, Eledon Pharmaceuticals, and others.
  • Key Amyotrophic Lateral Sclerosis Pipeline Therapies: BIIB067, FAB122, ABBV-CLS-7262, Trametinib, BIIB105, AstroRx, Dazucorilant, AP-101,  RAPA-501, ION-363, Reldesemtiv, MN-166, RT001, Fasudil,  SAR443820, PTC857, ANX005, DNL343, RNS60, BIIB100, CNMAu8, 18F-OP-801, Pegcetacoplan (APL-2), Cu(II)ATSM, KNS-760704, IFB-088, LAM-002A, CK 0803, 3K3A-APC,  QRL-201, Dexpramipexole, AL001, PrimeC, BLZ945, AT-1501, and others.

Dive deep into rich insights for new drugs for amyotrophic lateral sclerosis treatment; visit @ Amyotrophic Lateral Sclerosis Medications

Table of Contents

1.

Amyotrophic Lateral Sclerosis Pipeline Report Introduction

2.

Amyotrophic Lateral Sclerosis Pipeline Report Executive Summary

3.

Amyotrophic Lateral Sclerosis Pipeline: Overview

4.

Analytical Perspective In-depth Commercial Assessment

5.

Amyotrophic Lateral Sclerosis Clinical Trial Therapeutics

6.

Amyotrophic Lateral Sclerosis Pipeline: Late Stage Products (Pre-registration)

6.1.

BIIB067: Biogen

7.

Amyotrophic Lateral Sclerosis Pipeline: Late Stage Products (Phase III)

7.1.

ION363: Ionis Pharmaceuticals, Inc.

8.

Amyotrophic Lateral Sclerosis Pipeline: Mid Stage Products (Phase II)

8.1.

RNS60: Revalesio Corporation

9.

Amyotrophic Lateral Sclerosis Pipeline: Early Stage Products (Phase I)

10.

Amyotrophic Lateral Sclerosis Pipeline Therapeutics Assessment

11.

Inactive Products in the Amyotrophic Lateral Sclerosis Pipeline

12.

Company-University Collaborations (Licensing/Partnering) Analysis

13.

Key Companies

14.

Key Products in the Amyotrophic Lateral Sclerosis Pipeline

15.

Unmet Needs

16.

Market Drivers and Barriers

17.

Future Perspectives and Conclusion

18.

Analyst Views

19.

Appendix

For further information on the amyotrophic lateral sclerosis pipeline therapeutics, reach out @ Amyotrophic Lateral Sclerosis Drug Treatment

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