Axoltis Pharma has reported topline findings from SEALS, its phase 2 study of NX210c in Amyotrophic Lateral Sclerosis. The company said the drug candidate, which aims to restore the blood brain barrier, provide neuroprotection and enhance neurotransmission, delivered encouraging signals across multiple clinical and biological measures.
The trial enrolled 82 patients across 16 French sites between October 2024 and October 2025. Participants were stratified by serum neurofilament light chain concentration and randomized to receive NX210c at 5mg/kg, 10mg/kg or placebo as 10 minute infusions three times weekly for four weeks.
NX210c did not meet its primary endpoint, but Axoltis highlighted consistent positive trends in secondary and exploratory parameters. In post-hoc analyses, the decline rate in ALSFRS-R score was substantially reduced versus placebo at week 6 and strengthened at week 10. The effect was maintained at month 4.
Motor function benefits were marked, with reductions of about 64% for 5mg/kg and 33% for 10mg/kg at weeks 6 and 10. Respiratory decline was also substantially attenuated at week 10 and month 4. A dose response trend in serum neurofilament light chain was observed, with more patients in active arms showing decreases of more than 10% at month 4.
Target engagement was confirmed through plasma claudin-5, which was significantly reduced at 10mg/kg at end of treatment and remained lower at month 4. Axoltis said this supports NX210c’s role in blood brain barrier restoration.
The company reported a good safety and tolerability profile, consistent with phase 1 observations.
Dr Annette Janus, neurologist and chief medical officer at Axoltis Pharma, said: “These results are promising and contribute to generating evidence that NX210c’s properties are beneficial in neurodegenerative diseases, especially ALS. These findings support advancing NX210c to the next stages of clinical development.”
Yann Godfrin, CEO at Axoltis, explained: “The observed effect of NX210c in reducing plasma claudin-5 opens new avenues for the use of NX210c to treat a broad range of indications where blood brain barrier dysfunction is observed, among them Alzheimer’s disease, Parkinson’s disease and multiple sclerosis.”
Axoltis is conducting further analyses, including a 10 month follow-up, and will present detailed results at Neuroscience 2026 in Washington DC in November.
Dr Gilles Avenard, chairman of Axoltis’ board, said: “These results represent an important milestone for Axoltis and provide a strong foundation for the next stages of clinical development.”
Dr Godfrin added: “The execution of the SEALS clinical study has been a success thanks to the FILSLAN and ACT4ALS/MND networks, the invaluable support of ARSLA, Les Invincibles, the Swiss ALS Foundation, the Auvergne-Rhône-Alpes Region and Bpifrance, as well as the backing of Axoltis’ investors.”
