Drug candidate for ALS patients authorised for Phase II trial

Brain inflammation

Axoltis Pharma has received authorisation from ANSM, the French agency for the safety of health products, to launch the SEALS study for Amyotrophic Lateral Sclerosis (ALS).  

This Phase II clinical trial of drug candidate NX210c in patients with ALS is the first to target the integrity of the blood brain barrier (BBB). 

ALS is a fatal neurodegenerative disease affecting 50,000 individuals in Europe at any time, resulting in 10,000 deaths each year. It predominantly affects motor neurons in both the brain and spinal cord. This leads to muscular weakness and paralysis, with most patients succumbing to respiratory failure within, on average, two to five years. Currently, there is no cure for ALS and the only approved drug in the EU for the disease is Riluzole, prolonging survival for a median of just two months. Therefore, ALS remains a progressive and fatal neurologic disease of high unmet need. 

The integrity of the blood brain barrier

It has been shown that the BBB, which protects against undesirable blood components crossing into the brain, is impaired in several neurodegenerative conditions, such as ALS, and may even be disease-driving. Axoltis’ NX210c is a therapeutic agent with the ability to help the BBB recover its integrity, in addition to offering neuroprotection and promoting neurotransmission. NX210c is a cyclic peptide of 12 amino acids designed from the most conserved sequence of the type 1 thrombospondin repeats of the SCO-spondin. 

SEALS is a double-blind, randomised, placebo-controlled, multicentric Phase II study that will assess the efficacy, safety, tolerability and pharmacokinetics of NX210c treatment in ALS patients. Its primary objective is to assess the effect of NX210c via two markers: Neurofilament Light chain (NfL) concentration in the blood, as a diagnostic and prognostic of axonal damage relevant to ALS, as well as the ratio of albumin concentration between CerebroSpinal Fluid (CSF) and blood, which has long been a reliable biomarker of BBB integrity. The study will also evaluate the effect of NX210c on functional outcomes and select secondary biomarkers. The first results are expected by early 2026. 

Axoltis aims to enrol a total of 80 ALS patients in a trial planned in France with at least 15 investigating sites. Patients will receive a 10-minute IV infusion of the study drug at doses of 5 mg/kg or 10 mg/kg NX210c, or placebo, three times a week over four weeks. Patients will be able to continue the standard of care they have been receiving prior to receiving the study drug and will be followed-up for three months after the end of the treatment. Axoltis will receive operational support from the ACT4ALS/MND network.  

“Receiving the authorisation to start this Phase II clinical trial is a major achievement for the company, especially in ALS, where the unmet medical need is so important. In addition, there is a broad range of applications in CNS diseases where the BBB recovery could change their course, such as multiple sclerosis, Parkinson’s disease, Alzheimer’s disease, Huntington’s disease and more,” added Dr Yann Godfrin, Chief Executive Officer at Axoltis Pharma. 

The clinical trial will also incorporate statistical enrichment of the placebo group by adding virtual, in silico patients, based on computational methods of historical control data. This modelling, performed in collaboration with InSilicoTrials (Italy) will create predictions of disease progression in virtual subjects based on actual enrolled patients’ baseline characteristics. 

Part of the study is supported by the Region Auvergne-Rhône-Alpes and the French Government through i-Demo Régionalisé, a France 2030 grant. 

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