Why ALS trial monitoring matters
Amyotrophic lateral sclerosis (ALS) affects approximately 30,000 Americans and 450,000 people worldwide, with a median survival of 2–5 years from diagnosis. Despite decades of research, only four FDA-approved treatments exist, riluzole, edaravone, sodium phenylbutyrate/taurursodiol, and tofersen. The race for more effective therapies is intensifying.
The ALS trial landscape is now stratified by genetic subtype, creating a more complex competitive matrix:
- SOD1 ALS: tofersen (Biogen) approved 2023; next-gen SOD1-targeting programs and combination strategies now entering trials; represents ~2% of ALS cases but validates the ASO approach
- C9orf72 ALS/FTD: the most common genetic form (~40% of familial ALS); multiple ASO and small molecule programs from Wave Life Sciences, AstraZeneca, and others
- TDP-43 programs: targeting TDP-43 aggregation, mislocalization, and UPS impairment across sporadic and genetic ALS; one of the most active areas in 2026
- FUS ALS: aggressive early-onset form; ION-363 (tofersen successor) in trials
- Sporadic ALS approaches: neuroinflammation, mitochondrial, and glial cell targets for the ~90% of cases without identified genetic cause
- Gene therapy: AAV delivery of protective genes, gene editing approaches from Precision Biosciences and others
Track every new ALS trial automatically
The ALS pipeline is increasingly complex. Get a clean daily digest of new trials and status changes relevant to your focus areas.
Get the Free DigestA primary completion date pushed out, enrollment cut, or a primary endpoint changed in the last 90 days. Active trials only.
| Trial | Warning signs | Status |
|---|---|---|
| A Multiple Dose Study to Investigate Safety, Tolerability and Pharmacokinetics… NCT04667013 | Primary completion moved 3 years later | Not Yet Recruiting |
21 more on the Trials at Risk page. See which trials →
Phase 2 and Phase 3 trials with a primary completion date in the next 180 days, as ClinicalTrials.gov estimates it today. Active trials only.
| Trial | Scheduled | Phase |
|---|---|---|
| Evaluation of the Combined Therapy of EH-301 and N-acetylcysteine Together With… NCT07414212 | Nov 25, 2026 · Estimated | Phase 1/2 |
17 more on the Completions page. See the calendar →
What we monitor for ALS
Our daily ClinicalTrials.gov connection can be configured for precise ALS monitoring:
- Condition keywords: "amyotrophic lateral sclerosis", "ALS", "motor neuron disease", "MND", "Lou Gehrig", "progressive muscular atrophy"
- Genetic subtype keywords: "SOD1", "C9orf72", "TDP-43", "TARDBP", "FUS", "UBQLN2", "OPTN", "TBK1", track any specific subtype
- Mechanism keywords: "antisense oligonucleotide ALS", "ASO motor neuron", "gene therapy ALS", "neuroinflammation ALS"
- Phase filter: Phase 1 (early competitive intelligence), Phase 2/3 (near-term competitive threats)
- Status changes: New trial registrations, phase progressions, enrollment completions, and study terminations
The antisense oligonucleotide wave in ALS
Tofersen's approval established ASOs as a validated approach in ALS, triggering a wave of follow-on programs. The ASO competitive landscape in ALS in 2026 includes:
- Ionis Pharmaceuticals and its partners with multiple CNS-delivered ASOs in ALS subtypes
- Wave Life Sciences with allele-selective C9orf72 programs
- Multiple academic and industry programs using intrathecal delivery to silence toxic gain-of-function mutations
- New generation of ASOs with improved CNS penetration and dosing intervals entering early trials
For BD teams evaluating platform technologies or pipeline acquisitions in neurodegeneration, tracking ALS ASO trial registrations on ClinicalTrials.gov is a real-time competitive intelligence source.
Who uses ALS trial monitoring
- Neurodegenerative disease BD teams: tracking competitor programs across ALS, FTD, and related motor neuron diseases
- ALS-focused biotech investors: monitoring pipeline milestones and competitive positioning for portfolio companies
- CROs and clinical trial sites: identifying enrollment competition for active ALS studies
- ALS patient advocacy organizations: keeping patients and families informed about available trial options
- Academic neurologists: monitoring the commercial pipeline for investigator-initiated study collaboration opportunities
The ALS pipeline won't wait
Set up your ALS digest in 2 minutes. Daily digest with new trials, status updates, and phase transitions.
Start Free: No Credit Card318 active trials, 189 recruiting.
Industry sponsors run 83 of these trials. The rest are run by universities, hospitals, government bodies and other non-commercial sponsors. The table below lists the 5 largest of 65.
| Top industry sponsor | Active trials |
|---|---|
| Neuralink Corp | 7 |
| Biogen | 4 |
| Eli Lilly and Company | 4 |
| Cognixion | 3 |
| AB Science | 2 |
| Trial | Status | Latest tracked change | Seen |
|---|---|---|---|
| A Study of LY3873862 in Participants With Sporadic or Familial Amyotrophic… NCT07883590 | Not Yet Recruiting | Trial sites expanded: 4 → 7 locations | 2026-11-14 |
| A Study to Learn About the Safety of BIIB147 and Its Effects in Adults With… NCT07855211 | Recruiting | Trial sites expanded: 0 → 3 locations | 2026-11-13 |
| Healey ALS MyMatch Common Screening Protocol NCT07400393 | Recruiting | Trial sites expanded: 0 → 4 locations | 2026-11-13 |
| Impact of Robotic Glove Use on Quality of Life, Grip Strength and Fine Motor… NCT07298486 | Enrolling by Invitation | Study completion pushed from Jul 30, 2026 to Jan 30, 2027 | 2026-11-11 |
| RE104 Safety and Efficacy Study in Adjustment Disorder in Cancer and Other… NCT07002034 | Recruiting | Study completion pushed from Dec 2026 to May 2027 | 2026-11-11 |
Each trial page shows every change DataLookout has recorded for that trial.
DataLookout checks ClinicalTrials.gov every day and records what changed on each trial: status, enrollment, phase, primary endpoint, completion dates, sites, sponsor, and the stated reason a trial stopped. Add a sponsor or a disease to your watchlist, or save any search, and the changes arrive in a daily or weekly email digest.
Free, $0 forever: 1 sponsor + 1 disease watchlist, 1 saved search in your email digest, track up to 5 individual trials, daily change detection, Trials at Risk (top 2 trials). No credit card.
Pro, $49/month or $490/year: 10 sponsor + 10 disease watchlists, 10 saved searches in your email digest, track up to 25 individual trials, full Trials at Risk list, Completions calendar, landscape reports, CSV export.