Investment Landscape: ALS

Investment Landscape: Amyotrophic Lateral Sclerosis

Overview

Investment Landscape: Amyotrophic Lateral Sclerosis covers the current R&D investment, [@clinicaltrialsgov]
clinical trial pipeline, and funding trends for Amyotrophic Lateral Sclerosis research.

Last updated: 2026-03-17 19:20 PT

Clinical Trial Pipeline

Total Clinical Trials: 1569 Active Trials (Recruiting/Active): 434

Trial Phases

| Phase | Count |
|-------|-------|
| PHASE1 | 138 |
| PHASE1, PHASE2 | 77 |
| PHASE2 | 216 |
| PHASE2, PHASE3 | 49 |
| PHASE3 | 71 |
| PHASE4 | 20 |
| NA | 527 |
| Not Applicable | 440 |

Trial Status

| Status | Count |
|--------|-------|
| RECRUITING | 273 |
| ACTIVE_NOT_RECRUITING | 78 |
| NOT_YET_RECRUITING | 83 |
| COMPLETED | 747 |
| TERMINATED | 103 |
| WITHDRAWN | 39 |

Top Therapeutic Approaches

• mitochondrial: 696 trials
• genetic: 117 trials
• amyloid: 74 trials
• [tau](/proteins/tau): 37 trials
• tauro: 37 trials
• neuroinflammation: 14 trials
• metal: 14 trials
• metabolic: 12 trials

Investment Context

Amyotrophic lateral sclerosis has 1,569 total clinical trials, representing a significantly smaller research investment compared to AD and PD. The limited Phase 3 count (71) reflects the challenge of conducting large-scale trials for this rapidly progressive disease. The high termination rate (103 trials) highlights the need for better therapeutic targets.

Key Investment Themes

...

Investment Landscape: Amyotrophic Lateral Sclerosis

Overview

Investment Landscape: Amyotrophic Lateral Sclerosis covers the current R&D investment, [@clinicaltrialsgov]
clinical trial pipeline, and funding trends for Amyotrophic Lateral Sclerosis research.

Last updated: 2026-03-17 19:20 PT

Clinical Trial Pipeline

Total Clinical Trials: 1569 Active Trials (Recruiting/Active): 434

Trial Phases

| Phase | Count |
|-------|-------|
| PHASE1 | 138 |
| PHASE1, PHASE2 | 77 |
| PHASE2 | 216 |
| PHASE2, PHASE3 | 49 |
| PHASE3 | 71 |
| PHASE4 | 20 |
| NA | 527 |
| Not Applicable | 440 |

Trial Status

| Status | Count |
|--------|-------|
| RECRUITING | 273 |
| ACTIVE_NOT_RECRUITING | 78 |
| NOT_YET_RECRUITING | 83 |
| COMPLETED | 747 |
| TERMINATED | 103 |
| WITHDRAWN | 39 |

Top Therapeutic Approaches

• mitochondrial: 696 trials
• genetic: 117 trials
• amyloid: 74 trials
• [tau](/proteins/tau): 37 trials
• tauro: 37 trials
• neuroinflammation: 14 trials
• metal: 14 trials
• metabolic: 12 trials

Investment Context

Amyotrophic lateral sclerosis has 1,569 total clinical trials, representing a significantly smaller research investment compared to AD and PD. The limited Phase 3 count (71) reflects the challenge of conducting large-scale trials for this rapidly progressive disease. The high termination rate (103 trials) highlights the need for better therapeutic targets.

Key Investment Themes

• Genetic Subtypes: Growing stratification based on SOD1, C9orf72, and other genetic markers
• Neuroprotection: Emphasis on preserving motor neuron function
• Repositioning: Drug repurposing from other neurological conditions
• Biomarkers: Critical need for trial enrichment biomarkers

Emerging Investment Areas

With 103 terminated trials, ALS remains one of the most challenging neurodegenerative indications. RNAi-based therapies targeting SOD1 and C9orf72 have shown promise in early trials. Astrocyte modulation represents a novel approach to restore neuronal support. Metabolic interventions targeting energy dysfunction are gaining traction as a complementary strategy.

Priority Research Gaps

Late-Stage Development Bottleneck

Only 4.5% of trials are in Phase 3, indicating a significant gap between early discovery and late-stage clinical development. Investment in clinical trial infrastructure and regulatory engagement could accelerate late-stage programs.

Recommended Priorities

Phase 2→3 Translation: Enhance predictive biomarkers and clinical endpoints

Trial Design Innovation: Adaptive trials and platform protocols

Patient Recruitment: Investment in trial-ready cohorts and registry infrastructure

Therapeutic Target Priorities

Based on trial count analysis, the following mechanism categories represent either well-invested areas or underserved opportunities:

• mitochondrial: 696 trials - Well-established
• genetic: 117 trials - Well-established
• amyloid: 74 trials - Well-established
• tau: 37 trials - Growing area
• tauro: 37 trials - Growing area

Recent Investment Developments (2024-2025)

Approved Therapies

ALS has seen meaningful therapeutic advances:

• Tofersen (Qalsody, Biogen): First FDA-approved ASO for SOD1-ALS (2023), validated genetic targeting
• Riluzole: Continued use; AMX0035 (Cyren) showed survival benefit but FDA decision pending
• Edaravone (Radicava): Approved 2017, remains standard of care

Key Pipeline Updates (2025)

• Rozanolixizumab (UCB): Anti-glycan antibody in Phase 3 for C9orf72-ALS
• Ibudilast ( MediciNova): Neuroinflammatory modulator in Phase 2/3
• CNM-Au8 (Clene): Catalytic gold nanoparticles in Phase 3 for ALS/PD
• TPM-0032 (Transposon): Gene therapy for C9orf72 advancing to Phase 2
• Masitinib (AB Science): Tyrosine kinase inhibitor with Phase 3 data expected 2025

Investment Themes

• Genetic stratification: SOD1 and C9orf72 targeting drives investment
• RNA therapeutics: Multiple ASOs and RNAi programs in development
• Neuroprotection: Broad focus on preserving motor neuron function
• Astrocyte modulation: Novel approach to restore neuronal support

Recent Deals

• Biogen/Longitude: Acquisition of ALS pipeline including tofersen follow-ons
• Lily/Prevail: Strategic partnership for gene therapy in genetic forms of ALS

Investment Outlook

Near-Term Opportunities (1-3 Years)

Continued Phase 2/3 readouts expected for leading mechanisms. Focus on biomarker-positive trials for enrichment. Regulatory pathways becoming clearer for disease-modifying therapies.

Medium-Term Opportunities (3-5 Years)

Gene therapies and RNA-targeting modalities expected to expand. Combination therapy trials likely to increase. Patient stratification through genetic and biomarker testing becoming standard.

Long-Term Vision (5-10 Years)

Prevention trials in pre-symptomatic populations. Personalized medicine approaches based on genetic profiles. Disease-modifying therapies potentially becoming standard of care.

Related Pages

• [Amyotrophic Lateral Sclerosis](/diseases/amyotrophic-lateral-sclerosis)
• [TDP-43 Proteinopathy](/mechanisms/tdp-43-proteinopathy)
• [C9orf72](/entities/c9orf72)
• [Clinical Trials: ALS](/clinical-trials)

External Links

• [ALS Association](https://www.als.org/) - Non-profit fighting ALS
• [ClinicalTrials.gov ALS Studies](https://clinicaltrials.gov/search?cond=Amyotrophic+Lateral+Sclerosis) - Current ALS clinical trials
• [NEALS Consortium](https://www.nealsconsortium.org/) - Clinical research for ALS and related disorders

See Also

• [Neurodegenerative Disease Research](/diseases/neurodegeneration)
• [Therapeutic Development](/therapeutics/therapeutic-development)
• [Clinical Trials](/clinical-trials/clinical-trials-index)
• [Biomarkers](/mechanisms/neurodegenerative-biomarkers)
• [Drug Discovery](/mechanisms/drug-discovery)
• [Investment Analysis](/investments/investment-analysis)

References

Unknown, ClinicalTrials.gov (n.d.)