Motor Neuron Disease Clinical Pipeline | 180+ Companies Advancing Next-Generation Therapies

The Motor Neuron Disease market is evolving rapidly, driven by cutting-edge research and innovative treatment approaches. Leading pharmaceutical companies like GeneCradle Therapeutics, Verge Genomics, QurAlis Corporation, and Zydus Lifesciences are at the forefront of revolutionizing MND care, introducing novel therapies that offer new hope to patients worldwide. As these breakthrough treatments progress through clinical pipelines, they hold the potential to reshape the future of MND management.

DelveInsight's 'Motor Neuron Disease Pipeline Insight 2025' report provides comprehensive global coverage of pipeline Motor Neuron Disease therapies in various stages of clinical development. Major pharmaceutical companies are working to advance the pipeline space and future growth potential of the Motor Neuron Disease pipeline domain.

For emerging Motor Neuron Disease drugs, the Motor Neuron Disease 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 Motor Neuron Disease Pipeline Report
• DelveInsight's Motor Neuron Disease Pipeline analysis depicts a robust space with 180+ active players working to develop 200+ pipeline drugs for Motor Neuron Disease treatment.
• The leading Motor Neuron Disease companies include Biohaven Pharmaceuticals, Inc., Prilenia Therapeutics, Helixmith Co., Ltd., Transposon Therapeutics, Inc., GeneCradle Therapeutics, Verge Genomics, QurAlis Corporation, Zydus Lifesciences Limited, Ra Pharmaceuticals, Guangzhou Magpie Pharmaceuticals Co., Ltd., Scholar Rock, Inc., Spinogenix, Seelos Therapeutics, Inc., Sanofi, Hoffmann-La Roche, Revalesio Corporation, Cytokinetics, Rapa Therapeutics LLC, Q Therapeutics, Inc., PTC Therapeutics, ProJenX, NeuroSense Therapeutics Ltd., Knopp Biosciences, MediciNova, Amylyx Pharmaceuticals Inc., Neuropore Therapies Inc, NMD Pharma A/, and others are evaluating their lead assets to improve the Motor Neuron Disease treatment landscape.
• Key Motor Neuron Disease pipeline therapies in various stages of development include Talditercept alfa, Pridopidine, GC 101, VRG 50635, QRL 201, Masitinib, Latozinemab (Al001/Gsk4527223), Nurown (Msc-Ntf Cells), Ulefnersen (Ion363), Ibudilast, Dnl343, Abbv-Cls-7262, Cnm-Au8, Sls-005 (Trehalose), Pridopidine, Rapa-501, Primec, Engensis (Vm202), Rns60, Anx005, Dazucorilant (Cort113176), 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 an 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 III clinical study and the plan for a future 505(b)(2) marketing application submission.
• In November 2024, Ractigen Therapeutics announced that the FDA granted Orphan Drug Designation (ODD) to its novel siRNA therapy, RAG-21, targeting the FUS gene for amyotrophic lateral sclerosis (ALS).

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Motor Neuron Disease Overview
Motor neuron diseases (MNDs) are a group of progressive neurological disorders that damage motor neurons, the nerve cells responsible for controlling voluntary muscle movements such as walking, speaking, swallowing, and breathing. This group includes amyotrophic lateral sclerosis (ALS), progressive bulbar palsy, primary lateral sclerosis, progressive muscular atrophy, spinal muscular atrophy (SMA), Kennedy's disease, and post-polio syndrome.

MND leads to progressive muscle weakness and loss of function, significantly impacting daily life. While there is no cure, treatments help manage symptoms and improve quality of life. The exact cause remains unclear, though genetic, environmental, and lifestyle factors are believed to contribute. About 10% of cases are inherited due to genetic mutations. Life expectancy varies depending on the type and progression of the disease.

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Motor Neuron Disease Treatment Analysis: Drug Profile
Talditercept Alfa: Biohaven Pharmaceuticals, Inc.
Taldefgrobep alfa (BMS-986089) is a modified adnectin specifically designed to target and bind to myostatin (GDF-8). Adnectins, derived from human fibronectin, possess key properties essential for a successful therapeutic agent, including high potency, specificity, stability, and a favorable half-life. Taldefgrobep alfa is currently in Phase III clinical development for the treatment of Spinal Muscular Atrophy (SMA).

Pridopidine: Prilenia Therapeutics
Pridopidine is an oral investigational drug administered as a small, easy-to-swallow capsule taken twice daily. Extensive clinical studies have provided insights into its safety, mechanism of action, and efficacy. Imaging studies confirm that pridopidine effectively penetrates the brain and spinal cord, where it activates the sigma-1 receptor (S1R). The drug is currently in the Phase II/III stage for the treatment of Amyotrophic Lateral Sclerosis (ALS).

VM202: Helixmith Co., Ltd.
Engensis (VM202) is an innovative gene therapy designed to promote nerve regeneration and induce microvascular blood vessel formation. As a non-viral plasmid DNA product, it expresses recombinant hepatocyte growth factor (HGF) in nerve and Schwann cells, facilitating nerve system repair. Clinical data indicate that Engensis is well tolerated and holds potential for durable symptomatic relief. The drug is currently in Phase II clinical development for Amyotrophic Lateral Sclerosis (ALS).

Key Motor Neuron Disease Therapies and Companies
• Talditercept alfa: Biohaven Pharmaceuticals, Inc.
• Pridopidine: Prilenia Therapeutics
• VM202: Helixmith Co., Ltd.
• TPN-101: Transposon Therapeutics, Inc.
• GC 101: GeneCradle Therapeutics
• VRG 50635: Verge Genomics
• QRL 201: QurAlis Corporation
• 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

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Motor Neuron Disease 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
• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical

By Molecule Type
• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy

Scope of the Motor Neuron Disease Pipeline Report
• Coverage: Global
• Key Motor Neuron Disease Companies: Biohaven Pharmaceuticals, Inc., Prilenia Therapeutics, Helixmith Co., Ltd., Transposon Therapeutics, Inc., GeneCradle Therapeutics, Verge Genomics, QurAlis Corporation, Zydus Lifesciences Limited, Ra Pharmaceuticals, Guangzhou Magpie Pharmaceuticals Co., Ltd., Scholar Rock, Inc., Spinogenix, Seelos Therapeutics, Inc., Sanofi, Hoffmann-La Roche, Revalesio Corporation, Cytokinetics, Rapa Therapeutics LLC, Q Therapeutics, Inc., PTC Therapeutics, ProJenX, NeuroSense Therapeutics Ltd., Knopp Biosciences, MediciNova, Amylyx Pharmaceuticals Inc., Neuropore Therapies Inc, NMD Pharma A/, and others.
• Key Motor Neuron Disease Pipeline Therapies: Talditercept alfa, Pridopidine, GC 101, VRG 50635, QRL 201, Masitinib, Latozinemab (Al001/Gsk4527223), Nurown (Msc-Ntf Cells), Ulefnersen (Ion363), Ibudilast, Dnl343, Abbv-Cls-7262, Cnm-Au8, Sls-005 (Trehalose), Pridopidine, Rapa-501, Primec, Engensis (Vm202), Rns60, Anx005, Dazucorilant (Cort113176), Ap-101, Sar443820/Dnl788, Ait-101 (Lam-002a), and others.

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Table of Contents
1. Introduction
2. Executive Summary
3. Motor Neuron Disease Pipeline: Overview
4. Analytical Perspective In-depth Commercial Assessment
5. Motor Neuron Disease Pipeline Therapeutics
6. Motor Neuron Disease Pipeline: Late-Stage Products (Phase III)
7. Motor Neuron Disease Pipeline: Late-Stage Products (Phase III)
8. Motor Neuron Disease Pipeline: Mid-Stage Products (Phase II)
9. Motor Neuron Disease 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

Contact Us:
Jatin Vimal
jvimal@delveinsight.com
+14699457679
Healthcare Consulting
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About DelveInsight
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This release was published on openPR.