Motor Neuron Disease Pipeline Insight 2025: Gene-Targeted Therapies, Neuroprotective Agents, and Regenerative Approaches Aim to Change the Trajectory of ALS and Related Disorders | DelveInsight

DelveInsight's "Motor Neuron Disease - Pipeline Insight, 2025" highlights 200+ therapies in development for ALS and related motor neuron disorders. Despite supportive care being the current standard, limited disease-modifying options underline significant unmet needs.

The pipeline is expanding to include gene-targeted therapies for SOD1 and C9orf72 mutations, neuroprotective agents that address excitotoxicity and mitochondrial dysfunction, and cell-based interventions aimed at restoring motor function. Immunomodulatory and anti-inflammatory agents are also progressing, targeting the role of microglial and astrocytic activity in disease progression.

With several late-stage candidates, including antisense oligonucleotides and regenerative approaches, the treatment landscape is moving toward precision-driven, mechanism-based strategies that could alter disease trajectory and improve survival outcomes.

Interested in learning more about the current treatment landscape and the key drivers shaping the motor neuron disease pipeline? Click here: https://www.delveinsight.com/report-store/motor-neuron-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

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/S, Supernus Pharmaceuticals, Inc., Mitsubishi Tanabe Pharma America Inc., MaaT Pharma, 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, VM202, Censavudine, GC 101, VRG 50635, QRL 201, ZYIL1, Zilucoplan, Tetramethylpyrazine nitrone, SRK-015, SPG302, SLS-005, SAR443820, RO7204239, RNS60, Reldesemtiv, RAPA-501, Q-Cells, PTC857, Prosetin, PrimeC, Dexpramipexole, MN-166, AMX0035, NPT520-34, NMD670, MYOBLOC, MT-1186, MaaT033, and others.
• In March 2025, DiagnaMed Holdings Corp. announced a significant milestone in rare disease research, receiving Orphan Drug Designation (ODD) from the FDA for molecular hydrogen in the treatment of amyotrophic lateral sclerosis (ALS).
• In January 2025, Amylyx Pharmaceuticals (NASDAQ: AMLX) announced that the FDA has lifted the clinical hold on the Phase 1 trial of AMX0114, an antisense oligonucleotide targeting calpain-2 for ALS. With the hold lifted, Amylyx is now preparing to open U.S. sites for screening, enrollment, and dosing.
• 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 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.
• 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 Disease (MND) is a rare, progressive neurological disorder that affects the motor neurons-nerve cells responsible for controlling voluntary muscle activity such as walking, speaking, breathing, and swallowing. As these neurons degenerate and die, the muscles gradually weaken and waste away, leading to increasing disability. The most common form of MND is Amyotrophic Lateral Sclerosis (ALS). The exact cause is often unknown, though genetic and environmental factors may play a role. Symptoms typically begin with muscle weakness or stiffness and progress over time. While there is currently no cure, treatment focuses on managing symptoms, maintaining quality of life, and supporting breathing and mobility through a multidisciplinary approach.

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Motor Neuron Disease Treatment Analysis: Drug Profile
Talditercept Alfa: Biohaven Pharmaceuticals, Inc.
Taldefgrobep alfa (BMS-986089) is a modified adnectin designed to selectively target myostatin (GDF-8). Adnectins are a proprietary class of protein therapeutics based on human fibronectin, a naturally abundant extracellular protein. These molecules exhibit key drug-like properties, including high potency, specificity, stability, and an optimal 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 taken in a small, easy-to-swallow capsule twice daily. Extensive clinical studies have provided insights into its safety, mechanism of action, and potential efficacy. Imaging studies confirm that pridopidine penetrates the brain and spinal cord, where it activates the sigma-1 receptor (S1R), a protein linked to neuroprotection. It is currently in Phase II/III clinical development for the treatment of amyotrophic lateral sclerosis (ALS).

VM202 (Engensis): Helixmith Co., Ltd.
Engensis (VM202) is a gene therapy designed to promote tissue regeneration as a fundamental treatment approach. This non-viral plasmid DNA therapy expresses recombinant hepatocyte growth factor (HGF) protein in nerve and Schwann cells, supporting nerve regeneration and the formation of microvascular blood vessels. Clinical studies suggest Engensis is well tolerated and may provide long-term pain relief and symptomatic benefits across multiple conditions. It is currently in Phase II clinical development for the treatment of ALS.

Learn more about the novel and emerging Motor Neuron Disease pipeline therapies @ https://www.delveinsight.com/report-store/motor-neuron-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

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/S, Supernus Pharmaceuticals, Inc., Mitsubishi Tanabe Pharma America Inc., MaaT Pharma, and others.
• Key Motor Neuron Disease Pipeline Therapies: Talditercept alfa, Pridopidine, VM202, Censavudine, GC 101, VRG 50635, QRL 201, ZYIL1, Zilucoplan, Tetramethylpyrazine nitrone, SRK-015, SPG302, SLS-005, SAR443820, RO7204239, RNS60, Reldesemtiv, RAPA-501, Q-Cells, PTC857, Prosetin, PrimeC, Dexpramipexole, MN-166, AMX0035, NPT520-34, NMD670, MYOBLOC, MT-1186, MaaT033, and others.

Dive deep into rich insights for drugs used for Motor Neuron Disease treatment; visit @ https://www.delveinsight.com/report-store/motor-neuron-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

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
https://www.delveinsight.com/consulting-services

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This release was published on openPR.