Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative motor neuron disease characterized by loss of upper and lower motor neurons, leading to worsening weakness, speech/swallowing impairment, and respiratory failure. It is also known as Lou Gehrig’s disease.
Actor Eric Dane died of ALS at age 53 in February 2026, less than a year after announcing his diagnosis. Former NFL star Chris Johnson also revealed his ALS diagnosis at age 39. The cases highlight the neurodegenerative disease, which affects about 33,000 people in the U.S. and has no cure.
NeuroSense Therapeutics reported that its ALS drug PrimeC achieved a statistically significant 65% reduction in the risk of death in a Phase 2b trial. Separately, the company was granted a South Korean patent covering PrimeC's composition, formulation, and use for ALS treatment through 2042.
Two experimental ALS therapies showed promise in recent clinical developments. Interim Phase 1/2 data suggest the antisense oligonucleotide QRL-201 restores a critical nerve protein and may slow disease decline, with a Phase 3 trial planned for 2027. Meanwhile, the oral therapy neflamapimod has been selected for testing in the UK's EXPERTS-ALS platform study, with first results expected this spring.
The FDA granted Fast Track designation to COYA 302 for ALS. Phase 1 data showed tolerability and biomarker effects, and the phase 2 ALSTARS trial is underway.
Amylyx is approaching key catalysts ahead of its May 7 earnings report, led by Phase 3 LUCIDITY data for avexitide in post-bariatric hypoglycemia due in the third quarter. The company is also expected to provide updates on AMX0035 in Wolfram syndrome and AMX0114 biomarker data in ALS.
CervoMed presents new MRI data showing neflamapimod may reduce basal forebrain atrophy in dementia with Lewy bodies and finalizes Phase 3 trial design. The drug has also been selected for a UK-funded ALS platform trial, with first dosing expected by end of 2026.
A phase 3 clinical trial has launched for pridopidine as a potential ALS treatment, while AI research identified 18 FDA-approved drugs that may extend survival. Separate studies found a promising three-drug combination for sporadic ALS using new cell models.
Prothena reported Q4 and full year 2025 financial results with $308.4 million in cash as partners Roche and Novo Nordisk initiated Phase 3 trials for prasinezumab and coramitug, both expected to complete in 2029.
PrimeC, an oral combination therapy, showed safety and signals of clinical benefit in a phase 2b trial, while PLL001, a gut-targeting therapy, demonstrated positive safety and tolerability in a phase I/II study for amyotrophic lateral sclerosis.
Northwestern Medicine scientists discovered that N-acetyl-L-leucine (NALL), an FDA-approved compound, promotes neuroprotective effects in experimental Parkinson's disease models by targeting multiple molecular pathways simultaneously.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07543367 |
INdependence Through Endovascular Neuroprosthetic Technology (INTENT): an Early Feasibility Study |
RECRUITING | NA |
| NCT07460037 |
Chinese-Specific Speech Imagery Coding Using High-Density ECoG |
RECRUITING | NA |
| NCT07446114 |
Functional Outcomes and Control Using Synchron BCI - Canada |
RECRUITING | NA |
| NCT07400393 |
Healey ALS MyMatch Common Screening Protocol |
NOT_YET_RECRUITING | |
| NCT07396818 |
Kamlanoflast In Amyotrophic Lateral Sclerosis |
NOT_YET_RECRUITING | PHASE1/PHASE2 |
| NCT07341334 |
Digital Speech Markers for Monitoring ALS in Spanish Speakers |
NOT_YET_RECRUITING | |
| NCT07193953 |
Intravenous Immunoglobulin (IVIG) and Blood-Brain Barrier Disruption in Amyotrophic Lateral Sclerosis (ALS) |
RECRUITING | PHASE1 |
| NCT07007390 |
Systems Biology of Amyotrophic Lateral Sclerosis (ALS) |
NOT_YET_RECRUITING | |
| NCT07006571 |
At-home Treatment With Cortico-spinal tDCS for Amyotrophic Lateral Sclerosis |
RECRUITING | NA |
| NCT06765564 |
Clinical Study of Induced Pluripotent Stem Cells Derived Motor Neuron Precursor Cell Therapy for Amyotrophic Lateral Sclerosis (ALS) |
RECRUITING | NA |
