Axoltis Pharma is pioneering a breakthrough 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 core of our innovation is a unique peptide with remarkable properties – most notably, the ability to repair the blood-brain barrier (BBB). The breakdown of this protective BBB 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 major unmet medical need affecting 500,000 patients worldwide with no effective treatment, it was selected as the first therapeutic indication for NX210c.
The blood-brain barrier breakdown: a trigger and an accelerator for neurodegeneration
Emerging evidence points to BBB dysfunction as a central driver in neurodegenerative pathologies. The BBB acts as a critical gatekeeper between the bloodstream and the brain, regulating the passage of nutrients and molecules. When compromised, harmful proteins infiltrate the brain, sparking inflammation and neuronal damage. Diseases such as Alzheimer, Parkinson’s, Multiple Sclerosis, ALS, and many others share this underlying mechanism. Repairing BBB integrity is therefore a very promising avenue in the therapeutic arsenal to stop disease progression.
NX210c: a peptide with key properties
NX210c is a short, synthetic peptide inspired by SCO-spondin, a multifunctional glycoprotein essential in neurogenesis, the process by which new neurons are formed in the brain. This peptide not only restores the BBB but also protects neurons and supports intercellular communication. Its impact could be transformative, potentially slowing, halting, or even reversing neurological damage.
Focusing first on ALS: an urgent and huge unmet need
ALS is a fatal, rapidly progressing neuromuscular disease marked by the death of motor neurons responsible for walking, speaking, swallowing, and breathing. The disease affects over 500,000 people globally, with a median survival of only 3–5 years post-diagnosis. There is no therapeutic option to date. By restoring the BBB, NX210c offers a completely novel mechanism of action and new hope for patients.
A pipeline of opportunities across neurodegenerative diseases
NX210c’s mechanism of action unlocks opportunities far beyond ALS. Its ability to restore the BBB could be relevant across a wide range of CNS conditions. The global market for neurodegenerative diseases is expected to nearly double over the next decade, rising from an estimated $52 billion in 2024 to around $102.4 billion by 2034.
Promising preclinical and Phase 1b results fuel rapid progress into Phase 2
Robust preclinical studies in blood brain barrier restoration and in an ALS mouse model showed delayed motor function impairment and increased survival. In a 2023 Phase 1b trial in elderly healthy volunteers, NX210c showed an excellent safety and tolerability profile with no serious adverse events. Preliminary pharmacodynamic signals confirmed its BBB-repair activity through biomarker analysis. (Click here to read the released publication). This paved the way for a phase 2 trial.
A top-tier team ready to scale
Led by CEO Dr. Yann Godfrin – a biotech serial entrepreneur with 25 years of experience in the corporate management, drug development and innovation – Axoltis has gathered a multidisciplinary team of experts spanning neurology, clinical development, and regulatory affairs. The company is fully equipped to scale development rapidly and efficiently.
A multi-patented pioneering drug candidate
NX210c is an innovative disease-modifying drug candidate, protected by 7 patent families and has been granted Orphan Drug Designation by FDA and EMA. Orphan medicines benefit from market exclusivity once they receive a marketing authorization from 10 years in EU and 7 years in USA. Recently, in recognition of its breakthrough potential, Axoltis has been nominated for Prix Galien USA 2024 – Best Start Up category, considered as the Nobel Prize of pharmaceutical innovation. Among its numerous esteemed partners and supporters, Axoltis is honored to benefit from the valuable collaboration and support of ARSLA, the French Association for Research on ALS.
Phase 2 underway, first due diligence in motion
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. Topline results are expected in May 2026. Among several Pharma companies interested in partnering once topline results are available, two have already initiated due diligence to explore licensing opportunities. This signals strong market interest and a rare opportunity for investors with a X20 potential exit as early as 2026.
Axoltis’s mission
Axoltis is driven by a bold ambition: 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 drug development vision, we are now at a decisive inflection point.
Join us now on in the fight against ALS – and help us bring to market a therapy with the potential to reshape the future of neurological care.
