Amyotrophic lateral sclerosis drug pipeline
- ICD-11 8B60.0
- MeSH D000690
3 compounds from 3 companies are in active clinical development for amyotrophic lateral sclerosis, including 2 at Phase 3 or beyond.
Mechanisms: Biologic (2) · Small molecule (1)
Pipeline data compiled September 2026 · every fact sourced.
Upcoming readouts & recent results
FUSION Phase 3 (ulefnersen vs placebo in FUS-ALS) — estimated primary completion in 2H 2026; would be the first controlled readout for a FUS-ALS therapy. ↗
ClinicalTrials.gov estimated primary completion of confirmatory Phase 3 AB19001 (NCT03127267): December 2027 (registry estimate, not company guidance). ↗
Lancet case series (n=12, investigator-initiated): jacifusen reduced CSF neurofilament light chain by up to 82.8% at 6 months; one participant showed objective functional recovery, another remained asymptomatic — but two deaths (deemed unrelated) and uncontrolled design temper interpretation. ↗
AB10015 Phase 2/3 published (Mora et al., ALS-FTD 2020, epub 2019-07-07): in the prespecified primary population (normal progressors, ALSFRS-R decline <1.1 pts/month), masitinib 4.5 mg/kg/day + riluzole slowed 48-week ALSFRS-R decline by 3.4 points vs placebo (95% CI 0.65-6.13, p=0.016; 27% slowing), with significant ALSAQ-40, FVC and time-to-event secondaries; no effect in the broader population or 3.0 mg/kg/day cohorts. ↗
Compounds in development (3)
| Compound | Company | Phase | Status | Mechanism |
|---|---|---|---|---|
| MasitinibEU Orphan Designation For ALS (Referenced In The EMA Alsitek Refusal Q&A); Swissmedic Orphan Drug Status For ALS (2022-12-27, Per AB Science) | AB Science | Phase 3 | Active | KIT Inhibitor |
| Ulefnersen (ION363) | Ionis Pharmaceuticals, Inc. | Phase 3 | Active | FUS Inhibitor |
| Latozinemab | Alector Inc. | Discontinued | Discontinued | Sortilin Antagonist |
Trials recruiting now (57)
57 drug-interventional amyotrophic lateral sclerosis trials recruiting now, of 429 registered interventional trials. Browse all on ClinicalTrials.gov →
Tofersen
- Phase 4Clinical Study to Evaluate the Efficacy and Safety of FB1006 in the Treatment of ALS Patients
FB1006, Placebo
Pridopidine, Placebo
Masitinib (6.0), Riluzole, Placebo, Masitinib (4.5)
Galunisertib + Nerandomilast Combination
Tazbentetol, placebo
NUZ-001, Matching placebo
Test drug CB03-154 5mg group, Test drug CB03-154 10mg group, Test drug CB03-154 15mg group, Placebo Group
Acetyl-l-carnitine, Placebo
Nerve Growth Factor
Zilucoplan, Verdiperstat, CNM-Au8, Pridopidine, SLS-005 Trehalose, ABBV-CLS-7262, DNL343, NUZ-001
Memantine Hydrochloride Oral Solution, Trazodone Hydrochloride oral solution, Placebo oral solution, Amantadine Hydrochloride Oral Solution, Tacrolimus 1Mg Cap, Placebo capsule
Showing the 12 most advanced of 57 recruiting drug trials · see all on ClinicalTrials.gov →
Frequently asked
- How many drugs are in development for amyotrophic lateral sclerosis?
- 3 compounds from 3 companies are in active clinical development for amyotrophic lateral sclerosis, including 2 at Phase 3 or beyond.
- Which amyotrophic lateral sclerosis drugs are in Phase 3?
- Phase 3 (or later) programs for amyotrophic lateral sclerosis include Masitinib and Ulefnersen (ION363).
- What are the next amyotrophic lateral sclerosis trial readouts?
- Upcoming catalysts include Ulefnersen (ION363) (second half 2026) and Masitinib (December 2027).
- How many clinical trials are recruiting for amyotrophic lateral sclerosis?
- 57 drug-interventional amyotrophic lateral sclerosis trials are currently recruiting participants, out of 429 registered interventional trials.