Axoltis Pharma

Axoltis Pharma is developing NX210c, a groundbreaking treatment for Amyotrophic Lateral Sclerosis (ALS, also known as maladie de Charcot in French), the most frequent of the rare diseases and one of the most devastating. ALS progressively destroys motor neurons, leading to muscle weakness, paralysis, and ultimately death, with very limited treatment options today. NX210c is the first targeted - and most advanced - treatment addressing this high unmet medical need. This peptide restores the blood-brain barrier a key factor in ALS, Alzheimer’s, Parkinson’s, and other neurodegenerative diseases. With 7 patents and orphan drug designation in the USA and EU for ALS, NX210c has shown an excellent tolerability profile and is currently being evaluated in a Phase 2 clinical trial on 80 patients, with topline results expected in April 2026. Axoltis, nominated for the prestigious Prix Galien, is a pioneer in restoration and is accelerating the development of NX210c, bringing new hope to millions of patients facing neurodegenerative diseases.