Axoltis Pharma is a pioneer in an innovative treatment for ALS and multiple neurodegenerative disorders.

Axoltis Pharma is developing NX210c, a first-in-class therapeutic candidate targeting amyotrophic lateral sclerosis (ALS) and a broad range of central nervous system (CNS) diseases. At the heart of our innovation is a unique peptide with extraordinary properties, notably the ability to repair the blood-brain barrier (BBB) . Disruption of this protective blood-brain barrier is now recognized as a key trigger and amplifier of several major neurodegenerative diseases, including Alzheimer’s, Parkinson’s, multiple sclerosis, and ALS. Given the strong scientific and medical rationale, and the fact that ALS is a significant unmet medical need affecting 500,000 patients worldwide without an effective treatment, it was selected as the first therapeutic indication for NX210c.
The disruption of the blood-brain barrier: a trigger and an accelerator of neurodegeneration
New evidence points to blood-brain barrier (BBB) dysfunction as a key factor in neurodegenerative diseases. The BBB acts as a crucial intermediary between the bloodstream and the brain, regulating the passage of nutrients and molecules. When it is compromised, harmful proteins infiltrate the brain, causing inflammation and neuronal damage. Diseases such as Alzheimer’s, Parkinson’s, multiple sclerosis, ALS, and many others share this underlying mechanism. Therefore, repairing the integrity of the BBB is a very promising avenue in the therapeutic arsenal for halting disease progression.
NX210c: a peptide with key properties
NX210c is a short synthetic peptide inspired by SCO-espondin, a multifunctional glycoprotein essential for neurogenesis, the process by which new neurons are formed in the brain . This peptide not only restores the blood-brain barrier (BBB) but also protects neurons and promotes intercellular communication. Its impact could be transformative, potentially slowing, halting, or even reversing neurological damage.
Focusing first on ALS: an urgent and unmet need
ALS is a rapidly progressing, fatal neuromuscular disease characterized by the death of motor neurons responsible for walking, speech, swallowing, and breathing. It affects more than 500,000 people worldwide, with an average survival of only 3 to 5 years after diagnosis. To date, there is no therapeutic option. By restoring the blood-brain barrier (BBB), NX210c offers a completely novel mechanism of action and new hope for patients.

A range of opportunities in the field of neurodegenerative diseases
The mechanism of action of NX210c opens up opportunities far beyond ALS. Its ability to restore the blood-brain barrier (BBB) could be relevant in a wide range of CNS conditions. The global market for neurodegenerative diseases is projected to nearly double over the next decade, growing from approximately $52 billion in 2024 to approximately $102.4 billion in 2034.

Promising preclinical and phase 1b results are driving rapid progress towards phase 2
Robust preclinical studies on blood-brain barrier restoration and in a murine model of ALS showed a delay in the decline of motor function and increased survival. In a 2023 phase 1b trial with healthy elderly volunteers, NX210c demonstrated an excellent safety and tolerability profile, with no serious adverse events. Preliminary pharmacodynamic signals confirmed its blood-brain barrier-restoring activity through biomarker analysis. (Click here to read the publication.) This paved the way for a phase 2 trial.
A top-level team ready to scale
Led by Dr. Yann Godfrin, CEO and serial biotech entrepreneur with 25 years of experience in corporate management, drug development, and innovation, Axoltis has assembled a multidisciplinary team of experts in neurology, clinical development, and regulatory affairs . The company is fully equipped to scale development quickly and efficiently.
A pioneering drug candidate with multiple patents
NX210c is an innovative disease-modifying drug, protected by seven patent families and granted orphan drug designation by the FDA and EMA. Orphan drugs benefit from market exclusivity once they receive marketing authorization for 10 years in the EU and 7 years in the US. Recently, in recognition of its innovative potential, Axoltis was nominated for the 2024 Prix Galien USA in the Best Startup category, considered the Nobel Prize of pharmaceutical innovation . Among its numerous and esteemed partners and sponsors, Axoltis is proud to have the valuable collaboration and support of ARSLA, the French Association for ALS Research.
Phase 2 in progress, first due diligence underway
A multicenter phase 2 trial in 80 ALS patients across France , coordinated by Dr. Bernard (HCL, Reference Center for ALS, Lyon, France), is currently recruiting participants. Preliminary results are expected in May 2026. Among several pharmaceutical companies interested in collaborating once the preliminary results are available, two have already initiated due diligence to explore licensing opportunities. This indicates strong market interest and an exceptional opportunity for investors, with a potential exit from X20 starting in 2026.

Axoltis’s mission
Axoltis is driven by an ambitious goal: to change the lives of millions of patients affected by neurodegenerative diseases, starting with ALS patients. With an innovative approach and a value creation strategy guided by a clear vision for drug development, we are at a pivotal turning point.
Join us now in the fight against ALS and help us bring to market a therapy with the potential to transform the future of neurological care.
