Amyotrophic Lateral Sclerosis Pipeline Insight 2025: 75+ Companies Accelerating Innovation Across Genetic and Neuroinflammatory Targets

Globe and Mail

2 days ago

The ALS pipeline is gaining momentum, with over 75 companies advancing R&D across key targets like SOD1 mutations, TDP-43, C9orf72 expansions, and neuroinflammation. Approaches include ASOs, gene therapies, mitochondrial modulators, neuroprotective agents, and immune biologics. A major milestone was the FDA's 2023 accelerated approval of Biogen and Ionis's Tofersen for SOD1-ALS.
DelveInsight's ' Amyotrophic Lateral Sclerosis (ALS) – Pipeline Insight, 2025 ' offers a detailed analysis of the evolving therapeutic landscape for ALS, a fatal neurodegenerative disease characterized by progressive loss of motor neurons, leading to muscle weakness, paralysis, and eventually respiratory failure. With most patients succumbing to the disease within 3–5 years of symptom onset, there remains a critical unmet need for effective disease-modifying treatments.
Several late-stage candidates are progressing through global Phase II/III studies, including Amylyx's AMX0035, which received full FDA approval in September 2022 as RELYVRIO, and is now being evaluated in confirmatory trials. Additionally, investigational programs from companies like Clene Nanomedicine, QurAlis, Wave Life Sciences, and BrainStorm Cell Therapeutics are advancing promising assets in both sporadic and familial ALS subpopulations, often with orphan drug or fast-track designations.
The regulatory landscape continues to support innovation in ALS with flexible pathways and growing biomarker guidance, including neurofilament light chain (NfL) levels, digital endpoints, and functional scores. Patient advocacy, real-world evidence generation, and adaptive trial designs are also helping de-risk development strategies and accelerate access.
As 2025 unfolds, the ALS pipeline reflects a shift from symptomatic care to precision medicine and disease modification, driven by robust collaboration among academia, biotech, regulatory bodies, and patient communities. With over 80 therapies in the pipeline, the future of ALS treatment is poised for transformative change.
Key Takeaways from the Amyotrophic Lateral Sclerosis Pipeline Report
• DelveInsight's amyotrophic lateral sclerosis pipeline analysis depicts a strong 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, 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 ION363, FB418, SRK-015, RNS60, QRL-201, SAR443820, MN-166, VM202, VRG-50635, Zilucoplan, and others.
• In July 2025, Neurizon Therapeutics submitted a formal response to the FDA addressing the clinical hold on its IND application for NUZ-001, its lead ALS therapy.
• In July 2025, Klotho Neurosciences received FDA Orphan Drug Designation for KLTO-202 (s-KL-AAV.myo), its novel gene therapy targeting ALS.
• In June 2025, Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) announced that the FDA granted Fast Track designation to AMX0114, an investigational antisense oligonucleotide targeting calpain-2 for treating ALS.
• In May 2025, BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI) announced FDA clearance to begin its Phase IIIb trial of NurOwn® for treating amyotrophic lateral sclerosis (ALS).
• In May 2025, Neuralink announced that it received the FDA's " breakthrough" designation for its device aimed at restoring communication for individuals with severe speech impairment. The device, designed to assist patients with conditions like ALS, stroke, spinal cord injury, cerebral palsy, and multiple sclerosis, represents a significant step forward in neurotechnology.
• 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 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 ALS.
Request a sample and discover the recent breakthroughs happening in the amyotrophic lateral sclerosis pipeline landscape.
Amyotrophic Lateral Sclerosis Overview
Amyotrophic Lateral Sclerosis (ALS) is a rare, progressive neurodegenerative disorder that affects nerve cells in the brain and spinal cord. It leads to the loss of muscle control, eventually impacting the ability to speak, move, eat, and breathe. ALS is often classified into sporadic (the most common form) and familial types, with the latter being linked to genetic mutations. Although the exact cause of ALS is unknown in most cases, environmental and genetic factors are believed to contribute to its development.
Currently, there is no cure for ALS, but several treatment options aim to slow disease progression and manage symptoms. FDA-approved drugs like riluzole and edaravone offer modest benefits in extending survival or preserving function. Ongoing research is focused on gene therapies, neuroprotective agents, and immune-targeting approaches to better address the underlying mechanisms of the disease and improve patient outcomes.
Find out more about amyotrophic lateral sclerosis medication at https://www.delveinsight.com/report-store/amyotrophic-lateral-sclerosis-als-pipeline-insight
Amyotrophic Lateral Sclerosis Treatment Analysis: Drug Profile
MN-166: MediciNova
MN-166 is an orally administered small molecule glial attenuator that reduces neuroinflammation by suppressing pro-inflammatory cytokines such as IL-1β, TNF-α, and IL-6, while potentially increasing the anti-inflammatory cytokine IL-10. It also functions as a toll-like receptor 4 (TLR4) antagonist, contributing further to its anti-inflammatory effects. MN-166 is currently in Phase II/III clinical trials for the treatment of ALS.
RNS60: Revalesio
RNS60 is designed to offer disease-modifying and potentially regenerative effects in neurological disorders. It works by activating signaling pathways that enhance mitochondrial function and reduce inflammation, offering neuroprotection and immune modulation. The FDA has granted RNS60 both Orphan Drug and Fast Track status for ALS. It is currently in Phase II clinical trials.
VM202: Helixmith
VM202 is a gene therapy aimed at tissue regeneration by promoting the production of hepatocyte growth factor (HGF) at the site of injection along peripheral nerves. HGF supports nerve protection, neuron growth, and muscle atrophy prevention. The FDA has granted VM202 Orphan Drug and Fast Track designations. It is being evaluated in Phase II trials for ALS treatment.
Learn more about the novel and emerging amyotrophic lateral sclerosis pipeline therapies.
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, and others.
• Key Amyotrophic Lateral Sclerosis Pipeline Therapies: ION363, FB418, SRK-015, RNS60, QRL-201, SAR443820, MN-166, VM202, VRG-50635, Zilucoplan, and others.
Explore detailed insights on drugs used in the treatment of amyotrophic lateral sclerosis here.
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: Mid-Stage Products (Phase II)
8. Amyotrophic Lateral Sclerosis Pipeline: Early Stage Products (Phase I)
9. Therapeutic Assessment
10. Inactive Products
11. Company-University Collaborations (Licensing/Partnering) Analysis
12. Key Companies
13. Key Products
14. Unmet Needs
15. Market Drivers and Barriers
16. Future Perspectives and Conclusion
17. Analyst Views
18. Appendix
About DelveInsight
DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform, PharmDelve.
Media Contact
Company Name: DelveInsight
Contact Person: Jatin Vimal
Email: Send Email
Phone: [phone]
Address: 304 S. Jones Blvd #2432
City: Las Vegas
State: Nevada
Country: United States
Website: https://www.delveinsight.com/