Axoltis pharma

About Axoltis pharma

Axoltis Pharma is a clinical-stage biopharmaceutical company founded in 2003, developing treatments for neurodegenerative and neurotraumatic disorders. The company's primary focus is on its lead drug candidate, NX210c, a first-in-class peptide designed to address a range of central nervous system (CNS) diseases by repairing the blood-brain barrier (BBB). The breakdown of the BBB is a recognized trigger and amplifier in conditions like Amyotrophic Lateral Sclerosis (ALS), Alzheimer's, Parkinson's, and Multiple Sclerosis.

The company is headquartered in Clermont-Ferrand with offices in Lyon, France. Axoltis is led by CEO Dr. Yann Godfrin, a biotech entrepreneur with extensive experience in drug development and corporate management. The team includes experts in neurology, clinical development, and regulatory affairs. The company's business model is centered on developing its drug candidates through clinical trials to either partner with larger pharmaceutical companies for commercialization or seek acquisition. This strategy is supported by a portfolio of seven patent families protecting their lead candidate, NX210c.

NX210c has been granted Orphan Drug Designation by both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of ALS. Following promising preclinical studies and a Phase 1 trial that demonstrated a strong safety profile, Axoltis launched a Phase 2 clinical trial (the SEALS study) for NX210c in ALS patients in 2024, with top-line results anticipated in early 2026. The company is also exploring the potential of NX210c in other neurodegenerative diseases, including Parkinson's disease. Axoltis collaborates with academic and private laboratories and utilizes computational modeling to advance its research and development, aiming to address significant unmet medical needs in a neurodegenerative disease market projected to exceed $100 billion by 2034.