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Axoltis moves ALS drug forward after encouraging clinical findings

A new ALS candidate from French company Axoltis Pharma missed its primary endpoint in a Phase II study but showed several positive clinical effects.
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NX210c is a peptide‑based drug candidate being developed for neurodegenerative and trauma‑related disorders. It is designed to repair the blood–brain barrier, protect nerve cells, and improve signaling between neurons.

The study, which included 82 patients across 16 French clinical sites, did not meet its primary endpoint, which was the change in two biomarkers linked to the blood–brain barrier. However, post hoc analyses indicate that the treatment produced a clearly reduced decline in ALSFRS‑R, a common clinical scale used to measure functional loss in amyotrophic lateral sclerosis (ALS).

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