Axoltis misses the mark in phase 2 ALS study, but still strives to move towards the clinic

Biotech Axoltis misses the mark in phase 2 ALS study, but still strives to move towards the clinic By Marissa Russo Sep 15, 2026 3:00am amyotrophic lateral sclerosis failed trials peptide Clinical Data Axoltis Pharma announced today that its phase 2 trial for amyotrophic lateral sclerosis (ALS) has missed its primary endpoint, but the company said it observed consistent positive trends in secondary parameters. The French biotech will continue to develop its leading synthetic peptide, NX210c, for clinical development. The phase 2 study enrolled 82 patients with ALS in a double-blind, randomized, multicenter trial in France, to assess the efficacy, safety, tolerability and pharmacokinetics of multiple intravenous infusions of NX210c.

Patients received NX210c at either 5 mg/kg or 10 mg/kg or a placebo three times weekly for four weeks. At six weeks, patients did not show a significant change from baseline in blood neurofilament light chain (NfL), a test used to evaluate leakage of NfL protein into the bloodstream when nerve cells are injured. There was a trend of a decrease of NfL in the blood in 25.4% of patients in the NX210c arms of the study compared to 11.8% among the placebo-takers after four months, although these results were not significant.

Benefits in motor function scale, measured using the harmonized revised amyotrophic lateral sclerosis functional rating scale, were observed with 64% and 33% reductions in Patients treated with 5 mg/kg and 10 mg/kg, respectively. The reductions suggest improvement or slowing of decline in how well a person with ALS can control their physical and limb movements. There was also a significant decrease in blood claudin-5, a protein that is released into the bloodstream when the blood brain barrier (BBB) is disrupted, suggesting restoration of the BBB among the patients treated with 10 mg/kg of NX210c compared to those who received the placebo.

“The observed effect of NX210c in reducing plasma claudin-5 opens new avenues for the use of NX210c to treat a broad range of indications where blood brain barrier dysfunction is observed, among them Alzheimer’s disease, Parkinson’s disease and multiple sclerosis,” Yann Godfrin, Ph.D., CEO at Axoltis, said in the release. ALS, also known as Lou Gehrig’s disease, is a neurodegenerative disease that destroys the nerve cells in the brain and spinal cord. As these nerve cells die, they stop sending signals to your muscles leading to impaired walking, talking, chewing and breathing.

The blood brain barrier is the brain’s protective wall responsible for blocking toxins and other pathogens from entering the brain while allowing nutrients to pass through to the organ. The BBB is made of endothelial cells that are packed very tightly so that forbidden molecules cannot pass. If a molecule wants to enter the brain, it has to either be small enough to diffuse through the tightly packed cells or use a transporter, such as claudin-5, a tight junction protein that is one of the main gatekeepers of the brain.

Axoltis is attacking ALS with its synthetic peptide, NX210c, that has a three-pronged approach of targeting BBB restoration, protecting neurons and enhancing intercellular communication. It contains sequences from the subcommissural organ (SCO)-spondin, which is a glycoprotein essential for the process of neuron development that the SCO produces during embryonic development. ALS has been a tough market to crack recently.

In June 2025, the FDA missed Coya Therapeutics’ deadline for its IL-2 candidate called COYA 301, plus a CTLA4-Ig, for the potential treatment of ALS, dementia, Parkinson’s disease and Alzheimer’s disease, due to lack of resources and its current workload. In August 2025, Neurizon hit a similar roadblock on its way to repurposing a version of the veterinary drug monepantel as an ALS treatment. Meanwhile, California-based biotech Corcept Therapeutics is also in the race for ALS drugs and in June 2025 revealed promising data from its phase 2 study in ALS despite the initial failure to slow ALS progression.

Earlier that May, Eli Lilly penned a $415 million deal to license Alchemab’s early-stage ALS drug program. Axoltis plans to present the full data of its phase 2 trial during the society for neuroscience annual meeting in November 2026. “These results are promising and contribute to generating evidence that NX210c’s properties are beneficial in neurodegenerative diseases, especially ALS,” Annette Janus, M.D., chief medical officer at Axoltis, said in the release.

“These findings support advancing NX210c to the next stages of clinical development.” amyotrophic lateral sclerosis failed trials peptide Clinical Data Clinical Data Biotech