Amyotrophic Lateral Sclerosis Pipeline: Advancing the Future with 50+ Innovators Leading Breakthrough Therapies

The amyotrophic lateral sclerosis market is rapidly advancing and is fueled by groundbreaking research and innovative therapies from companies such as Denali Therapeutics, AbbVie, Calico Life Sciences, and Clene Nanomedicine Biosciences. These industry pioneers are transforming treatment strategies and redefining the future of Amyotrophic Lateral Sclerosis care, bringing new hope to patients worldwide.

DelveInsight's "Amyotrophic Lateral Sclerosis Pipeline Insight, 2025" comprehensively analyzes the current clinical landscape and growth prospects in the Amyotrophic Lateral Sclerosis market. The report covers disease insights, treatment guidelines, and a detailed pipeline assessment from preclinical to marketed stages. It includes drug mechanisms, clinical studies, regulatory progress, and key developments such as collaborations, mergers, funding, and designations.

For emerging Amyotrophic Lateral Sclerosis drugs, the Amyotrophic Lateral Sclerosis pipeline analysis report provides a 360° view of the therapeutics landscape by development point, product type, route of administration, molecule type, and MOA. The pipeline research covers business opportunities, challenges, future partnerships, strong competitors, and growth strategies.

Key Takeaways from the Amyotrophic Lateral Sclerosis Pipeline Report
• DelveInsight's Amyotrophic Lateral Sclerosis Pipeline analysis depicts a robust space with 75+ active players working to develop 80+ pipeline drugs for Amyotrophic Lateral Sclerosis treatment.
• The leading Amyotrophic Lateral Sclerosis companies include Ionis Pharmaceuticals, 1st Biotherapeutics, Scholar Rock, Revalesio, QurAlis Corporation, Sanofi, MediciNova, Helixmith, Verge Genomics, UCB, GSK, Brainstorm Cell Therapeutics, Denali Therapeutics, AbbVie, Calico Life Sciences, Clene Nanomedicine Biosciences, Seelos Therapeutics, Prilenia Therapeutics, Rapa Therapeutics, Neurosense Therapeutics, Transposon Therapeutics, Annexon Biosciences, Corcept Therapeutics, Al-S Pharma, Orphai Therapeutics, and others are evaluating their lead assets to improve the Amyotrophic Lateral Sclerosis treatment landscape.
• Key Amyotrophic Lateral Sclerosis pipeline therapies in various stages of development include MN-166, RNS60, VM202, QRL-201, MASITINIB, LATOZINEMAB, NUROWN, ULEFNERSEN, IBUDILAST, DNL343, ABBV-CLS-7262, CNM-AU8, SLS-005, PRIDOPIDINE, RAPA-501, PRIMEC, ENGENSIS, TPN-101, RNS60, ANX005, DAZUCORILANT, AP-101, SAR443820/DNL788, AIT-101 (LAM-002A), and others.
• In March 2025, DiagnaMed Holdings Corp. announced a significant milestone in rare disease research, receiving Orphan Drug Designation (ODD) from the U.S. FDA for molecular hydrogen in the treatment of amyotrophic lateral sclerosis (ALS).
• In January 2025, the FDA granted approval for Zydus Lifesciences to proceed with a randomized Phase IIb trial of its oral NLRP3 inflammasome inhibitor, Usnoflast, for the treatment of amyotrophic lateral sclerosis (ALS).
• In December 2024, NeuroSense Therapeutics Ltd. announced the conclusion of a Type C meeting with the U.S. Food and Drug Administration (FDA) regarding PrimeC for the treatment of amyotrophic lateral sclerosis (ALS). The meeting focused on the design of a proposed Phase 3 clinical study and the plan for a future 505(b)(2) marketing application submission.

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Amyotrophic Lateral Sclerosis Overview
Amyotrophic Lateral Sclerosis (ALS), or "Lou Gehrig's disease," is a neurodegenerative disorder affecting motor neurons. Its exact cause remains unknown, with both genetic and sporadic factors contributing to its development. ALS impacts both upper and lower motor neurons, typically starting with lower motor neuron degeneration in the proximal limbs. As a progressive disease, it leads to paralysis and ultimately death.

The underlying mechanisms of sporadic ALS are still unclear, with theories including RNA processing defects, SOD1 mutations causing free radical toxicity, inflammatory responses, and glutamate excess. Familial ALS is often linked to C9ORF72 repeat expansions and SOD1 mutations, which cause protein misfolding and cell damage. Most genetic forms follow an autosomal dominant inheritance pattern, highlighting ALS as a complex disorder with multiple causes rather than a single origin.

Respiratory care is critical, as muscle weakness often leads to chronic respiratory failure. Noninvasive ventilation is advised for orthopnea, nocturnal hypoxia, or an FVC below 50%, while invasive ventilation may be required if noninvasive methods fail. Dysphagia is also common and should be managed with dietary adjustments, with enteral nutrition (G-tube) recommended for severe cases, as weight loss worsens prognosis.

ALS complications include respiratory decline, swallowing and speech difficulties, malnutrition, muscle spasms, spasticity, fatigue, and emotional disturbances. Some treatments can cause side effects, such as gastrointestinal issues and arrhythmias with mexiletine, liver enzyme elevations and weakness with Riluzole, and gait instability and headaches with edaravone.

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Amyotrophic Lateral Sclerosis Treatment Analysis: Drug Profile
MN-166: MediciNova
MN-166 is an orally bioavailable, first-in-class small molecule designed to reduce neuroinflammation by suppressing pro-inflammatory cytokines IL-1ß, TNF-a, and IL-6 while potentially increasing the anti-inflammatory cytokine IL-10. It also functions as a toll-like receptor 4 (TLR4) antagonist, further contributing to its neuroprotective effects. Currently, MN-166 is being evaluated in a Phase II/III clinical trial for ALS treatment.

RNS60: Revalesio
RNS60 is being developed as a disease-modifying and potentially restorative therapy for neurological disorders. It enhances mitochondrial function, reduces inflammation, and protects neurons and oligodendrocytes while modulating immune cell activity to restore balance. With Orphan Drug and Fast Track designations from the FDA, RNS60 is currently undergoing a Phase II clinical trial for ALS.

VM202: Helixmith
VM202 is an innovative gene therapy aimed at tissue regeneration. Injected along peripheral nerves, it promotes nerve protection and neuronal growth through the production of hepatocyte growth factor (HGF), potentially counteracting atrophic conditions. Recognized with Orphan Drug and Fast Track status by the FDA, VM202 is currently in a Phase II clinical trial for ALS treatment.

Key Amyotrophic Lateral Sclerosis Therapies and Companies
• MN-166: MediciNova
• RNS60: Revalesio
• VM202: Helixmith
• QRL-201: QurAlis Corporation
• Masitinib: AB Science
• Latozinemab (AL001/GSK4527223): Alector/Gsk
• Nurown (MSC-NTF CELLS): Brainstorm Cell Therapeutics
• Ulefnersen (ION363): Ionis Pharmaceuticals
• Ibudilast: Medicinova
• DNL343: Denali Therapeutics
• ABBV-CLS-7262: Abbvie/Calico Life Sciences
• CNM-AU8: Clene Nanomedicine Biosciences
• SLS-005 (Trehalose): Seelos Therapeutics
• Pridopidine: Prilenia Therapeutics
• RAPA-501: Rapa Therapeutics
• Primec: Neurosense Therapeutics
• EngensiS (VM202): Helixmith
• TPN-101: Transposon Therapeutics
• RNS60: Revalesio Corporation
• ANX005: Annexon Biosciences
• Dazucorilant (CORT113176): Corcept Therapeutics
• AP-101: AL-S PHARMA
• SAR443820/DNL788: Sanofi/Denali Therapeutics
• AIT-101 (LAM-002A): Orphai Therapeutics

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Amyotrophic Lateral Sclerosis Therapeutics Assessment

By Product Type
• Mono
• Combination
• Mono/Combination.

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

By Route of Administration
• Intravenous
• Subcutaneous
• Oral
• Intramuscular

By Molecule Type
• Monoclonal antibody
• Small molecule
• Peptide

Scope of the Amyotrophic Lateral Sclerosis Pipeline Report
• Coverage: Global
• Key Amyotrophic Lateral Sclerosis Companies: Ionis Pharmaceuticals, 1st Biotherapeutics, Scholar Rock, Revalesio, QurAlis Corporation, Sanofi, MediciNova, Helixmith, Verge Genomics, UCB, GSK, Brainstorm Cell Therapeutics, Denali Therapeutics, AbbVie, Calico Life Sciences, Clene Nanomedicine Biosciences, Seelos Therapeutics, Prilenia Therapeutics, Rapa Therapeutics, Neurosense Therapeutics, Transposon Therapeutics, Annexon Biosciences, Corcept Therapeutics, Al-S Pharma, Orphai Therapeutics, and others.
• Key Amyotrophic Lateral Sclerosis Pipeline Therapies: MN-166, RNS60, VM202, QRL-201, MASITINIB, LATOZINEMAB, NUROWN, ULEFNERSEN, IBUDILAST, DNL343, ABBV-CLS-7262, CNM-AU8, SLS-005, PRIDOPIDINE, RAPA-501, PRIMEC, ENGENSIS, TPN-101, RNS60, ANX005, DAZUCORILANT, AP-101, SAR443820/DNL788, AIT-101 (LAM-002A), and others.

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Table of Contents
1. Introduction
2. Executive Summary
3. Amyotrophic Lateral Sclerosis Pipeline: Overview
4. Analytical Perspective In-depth Commercial Assessment
5. Amyotrophic Lateral Sclerosis Pipeline Therapeutics
6. Amyotrophic Lateral Sclerosis Pipeline: Late-Stage Products (Phase III)
7. Amyotrophic Lateral Sclerosis Pipeline: Late-Stage Products (Phase III)
8. Amyotrophic Lateral Sclerosis Pipeline: Mid-Stage Products (Phase II)
9. Amyotrophic Lateral Sclerosis Pipeline: Early Stage Products (Phase I)
10. Therapeutic Assessment
11. Inactive Products
12. Company-University Collaborations (Licensing/Partnering) Analysis
13. Key Companies
14. Key Products
15. Unmet Needs
16. Market Drivers and Barriers
17. Future Perspectives and Conclusion
18. Analyst Views
19. Appendix

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This release was published on openPR.