Amyotrophic lateral sclerosis (ALS) has been a challenging disease to tackle in terms of drug development, with several setbacks in recent years. Despite this, the ALS community remains hopeful as researchers continue to explore new treatment options and potential breakthroughs in the field.
One such hopeful candidate is Clene Nanomedicine’s CNM-Au8, which is currently seeking accelerated approval for its ALS treatment by the end of this year. CNM-Au8 is an oral suspension of gold nanocrystals designed to enhance nerve cell health and increase energy production to improve survival rates. Clene has been working closely with the FDA on a pathway towards accelerated approval, with plans to leverage biomarker data to support their case.
Another promising candidate is NX210c from Axoltis Pharma, which targets the integrity of the blood-brain barrier and neural circuits in ALS patients. Their phase 2 trial is currently underway, with phase 1b results showing positive effects on neuroprotection and neurotransmission. The biotech is expected to release phase 2 results in the second quarter of 2026.
MediciNova is also making strides with their ALS candidate, MN-166, a PDE4 inhibitor targeting inflammation in ALS patients. The company is currently enrolling participants in a phase 2/3 trial and has seen positive correlations in their interim study results, indicating potential benefits for ALS patients.
Coya Therapeutics is taking a unique approach with their ALS drug candidate, COYA-302, which targets the overactive immune system in ALS patients. By enhancing Treg cells, a subset of T cells, Coya aims to reduce neuroinflammation and slow disease progression. Top-line results from their phase 2 trial are expected in the second half of 2026, with additional data on biomarkers and oxidative stress markers to be published in 2025.
Rapa Therapeutics is also targeting T cells with their ex vivo cell therapy, RAPA-501, which modifies T cells to produce an anti-inflammatory response. The study is currently in phase 2/3 testing and is expected to reach its primary completion in July 2026.
Finally, Amylyx Pharmaceuticals, despite previous setbacks with Relyvrio, is forging ahead with their investigational antisense oligonucleotide, AMX0114, which targets the calcium-activated protease calpain-2. The company recently received fast track designation from the FDA and is currently in phase 1 testing, with early cohort data expected later this year.
Overall, the ALS landscape is evolving rapidly, with promising candidates on the horizon and ongoing research efforts to uncover new treatment options for this devastating disease. The ALS community remains hopeful for breakthroughs that could change the course of ALS treatment in the near future.
