Investment Landscape: Amyotrophic Lateral Sclerosis covers the current R&D investment,
clinical trial pipeline, and funding trends for Amyotrophic Lateral Sclerosis research.
Last updated: 2026-04-01 09:38 PT
Total Clinical Trials: 1569
Active Trials (Recruiting/Active): 434
| Phase | Count |
|---|---|
| PHASE1 | 138 |
| PHASE1, PHASE2 | 77 |
| PHASE2 | 216 |
| PHASE2, PHASE3 | 49 |
| PHASE3 | 71 |
| PHASE4 | 20 |
| NA | 527 |
| Not Applicable | 440 |
| Status | Count |
|---|---|
| RECRUITING | 273 |
| ACTIVE_NOT_RECRUITING | 78 |
| NOT_YET_RECRUITING | 83 |
| COMPLETED | 747 |
| TERMINATED | 103 |
| WITHDRAWN | 39 |
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.
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.
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.
Based on trial count analysis, the following mechanism categories represent either well-invested areas or underserved opportunities:
Continued Phase 2/3 readouts expected for leading mechanisms. Focus on biomarker-positive trials for enrichment. Regulatory pathways becoming clearer for disease-modifying therapies.
Gene therapies and RNA-targeting modalities expected to expand. Combination therapy trials likely to increase. Patient stratification through genetic and biomarker testing becoming standard.
Prevention trials in pre-symptomatic populations. Personalized medicine approaches based on genetic profiles. Disease-modifying therapies potentially becoming standard of care.
Last updated: 2026-04-01