Axoltis Phase 2 ALS Trial Fails Primary Endpoint Though Shows Some Secondary Trends

Axoltis Pharma reported that its phase 2 study of the synthetic peptide NX210c in amyotrophic lateral sclerosis did not meet its primary goal. The double‑blind, randomized trial enrolled 82 French patients who received either 5 mg/kg, 10 mg/kg of the drug or placebo three times a week for four weeks.

The primary measure, change in blood neurofilament light chain (NfL) at six weeks, showed no statistically significant difference between treatment and placebo groups. A modest reduction in NfL was observed in about a quarter of treated patients versus roughly one‑tenth of those on placebo, but the finding did not reach significance.

Despite the primary miss, the company noted encouraging signals in secondary outcomes. Motor function scores improved in a larger proportion of patients receiving the lower dose compared with the higher dose, and the trend toward lower NfL levels was consistent across the treatment arms. Axoltis said it will continue to develop NX210c and pursue further clinical evaluation.