InSilicoTrials partners with Axoltis Pharma to leverage advanced modeling technology, generating predictive analyses of disease progression in virtual subjects based on the baseline characteristics of actual enrolled patients. This collaboration will enhance the development of Axoltis’s NX210c by accelerating the treatment process, improving safety, and reducing development costs.
Clermont-Ferrand and Lyon, France, April 10, 2024 – Axoltis Pharma, a French biopharmaceutical company dedicated to developing therapeutic solutions for neurodegenerative diseases, today announces the authorization from ANSM, the French agency for the safety of health products, to launch the SEALS study. This phase II clinical trial of drug candidate NX210c in patients with Amyotrophic Lateral Sclerosis (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.
Today, 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 therapeutic approach of recovering BBB integrity in ALS patients is very
promising; here at Axoltis we are proud to have a marked head start in the clinic with NX210c,” said Dr. Annette Janus, neurologist and chief medical officer at Axoltis. “The use of advanced analytical methods will offer a fresh approach to tackling ALS and contribute to the understanding of neurodegenerative diseases overall.”
SEALS is a double-blind, randomized, 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.
In another pioneering step, 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.
Read the full press release here: https://www.axoltis.com/wp-content/uploads/2024/04/240410-Axoltis-phase-II-trial-ALS-EN.pdf