🧠The 5 ALS Clinical Trials Everyone’s Watching in 2025
Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig’s disease, continues to challenge the medical world—but 2025 might just be a turning point. With a surge of promising therapies in the pipeline, researchers, patients, and caregivers alike are keeping their eyes locked on a handful of clinical trials that could change everything.
Here are the five ALS clinical trials generating the most buzz this year—and why they matter.
1. Biogen’s Tofersen Expansion Trial
Tofersen, an antisense oligonucleotide therapy targeting the SOD1 gene mutation, has shown signs of slowing ALS progression in early studies. In 2025, Biogen is expanding this trial to test its efficacy in pre-symptomatic carriers. This could be monumental—it’s the first step toward preventative treatment for genetic ALS.
2. Amylyx’s AMX0035 Long-Term Efficacy Study
After receiving FDA approval in 2022, AMX0035 entered a long-term follow-up phase to assess how well the drug performs over time. 2025’s data will help determine if the benefits seen in initial trials truly hold up. For patients and physicians, this is a pivotal moment in deciding whether the drug should become a foundational therapy.
3. Clene Nanomedicine’s CNM-Au8 Regenerative Therapy Trial
This unique therapy uses gold nanocrystals to enhance brain energy metabolism. CNM-Au8 has already shown potential to improve neuron resilience and function. The 2025 trial is testing a higher dose over a longer duration. If it hits the mark, this could be the first regenerative-focused ALS therapy that works at the cellular level.
4. Calico & AbbVie’s Anti-Aging Angle
In a bold approach, Calico (Google’s longevity startup) and AbbVie are collaborating on a compound that targets aging pathways suspected to influence ALS progression. 2025 marks the first human trial phase. The industry is watching closely—if successful, this trial could open the door to a new class of ALS treatments that focus on slowing biological aging itself.
5. QurAlis’s Precision Neuro Therapy
This biotech firm is trialing a personalized neuroprotection therapy based on patient-specific biomarkers. QurAlis is betting big on tailoring treatment to the individual rather than a one-size-fits-all drug. With early preclinical success, its 2025 Phase 2 trial will test whether personalized ALS therapy could finally become reality.
