Amyotrophic lateral sclerosis drug pipeline

Upcoming readouts & recent results

• second half 2026AnticipatedUlefnersen (ION363)Topline dataNCT04768972

  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. ↗

• 2025-06-07ReportedUlefnersen (ION363)Full resultsmixed

  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. ↗

See the full catalyst calendar →

Compounds in development (1)

Trials recruiting now (58)

58 drug-interventional amyotrophic lateral sclerosis trials recruiting now, of 429 registered interventional trials. Browse all on ClinicalTrials.gov →

• Phase 4A Study to Learn More About the Long-Term Safety of Tofersen (Qalsody) in Chinese Participants With SOD-1 Amyotrophic Lateral Sclerosis (ALS)

  Tofersen

  Biogenn=12China · 1 siteStarted Dec 2025NCT07223723
• Phase 4Clinical Study to Evaluate the Efficacy and Safety of FB1006 in the Treatment of ALS Patients

  FB1006, Placebo

  Peking University Third Hospitaln=64China · 1 siteStarted Jan 2023NCT05923905
• Phase 3Pridopidine Phase 3 Study to Evaluate Efficacy and Safety in ALS

  Pridopidine, Placebo

  Prilenian=500Belgium, Canada +8 · 28 sitesStarted Feb 2026NCT07322003
• Phase 3Efficacy and Safety of Masitinib in Combination With SoC Versus Placebo in the Treatment of ALS Patients

  Masitinib 4.5 mg/kg/day, Placebo, Riluzole (100 mg)

  AB Sciencen=412Greece · 1 siteStarted Jan 2026NCT07174492
• Phase 3Efficacy and Safety of Masitinib Versus Placebo in the Treatment of ALS Patients

  Masitinib (6.0), Riluzole, Placebo, Masitinib (4.5)

  AB Sciencen=495Belgium, Denmark +15 · 56 sitesStarted Feb 2021NCT03127267
• Phase 2/3This Study Evaluates the Safety, Target Engagement, and Preliminary Efficacy of Galunisertib (TGF-βR1/ALK5 Inhibitor)Combined With Nerandomilast (PDE4 Inhibitor) in GREM2-positive ALS, a Biomarker-defined Subgroup Hypothesized to Reflect Heightened TGF-β/SMAD-driven Astrocytic and Fibrotic Signaling

  Galunisertib + Nerandomilast Combination

  Gipfel Life Sciences GmbHn=60Started Jun 2026NCT07321860
• Phase 2/3Efficacy and Safety of Tazbentetol in ALS Participants

  Tazbentetol, placebo

  Spinogenixn=430Started Mar 2026NCT07325591
• Phase 2/3HEALEY ALS Platform Trial - Regimen I NUZ-001

  NUZ-001, Matching placebo

  Merit E. Cudkowicz, MDn=160United States · 1 siteStarted Feb 2026NCT07410806
• Phase 2/3A Study to Evaluate the Efficacy and Safety of Different Doses of CB03-154 in Adult Patients With Amyotrophic Lateral Sclerosis (ALS)

  Test drug CB03-154 5mg group, Test drug CB03-154 10mg group, Test drug CB03-154 15mg group, Placebo Group

  Shanghai Zhimeng Biopharma, Inc.n=240Started Sep 2025NCT07082192
• Phase 2/3Trial on the Biological and Clinical Effects of Acetyl-L-carnitine in ALS

  Acetyl-l-carnitine, Placebo

  Mario Negri Institute for Pharmacological Researchn=246Australia, Italy · 19 sitesStarted Mar 2025NCT06126315
• Phase 2/3Nerve Growth Factor Encapsulated With 2-methacryloyloxyethyl Phosphorylcholine Nanocapsules in the Treatment of Amyotrophic Lateral Sclerosis

  Nerve Growth Factor

  Beijing Tiantan Hospitaln=60Started May 2024NCT06391645
• Phase 2/3HEALEY ALS Platform Trial - Master Protocol

  Zilucoplan, Verdiperstat, CNM-Au8, Pridopidine, SLS-005 Trehalose, ABBV-CLS-7262, DNL343, NUZ-001

  Merit E. Cudkowicz, MDn=1,500United States · 81 sitesStarted Jun 2020NCT04297683

Showing the 12 most advanced of 58 recruiting drug trials · see all on ClinicalTrials.gov →