Clene Inc. said that analyses recommended by U.S. Food and Drug Administration (FDA) revealed statistically significant reductions in the biomarkers Neurofilament light (NfL) and Glial fibrillary acidic protein (GFAP) among people living with Amyotrophic lateral sclerosis (ALS) treated with its lead candidate CNM-Au8.
The company reported that in the NIH-sponsored Expanded Access Program (EAP), participants receiving CNM-Au8 experienced a significant Week-36 reduction in NfL compared to matched ALS controls (AUC geometric mean ratio 0.914; p = 0.0373). Concurrent declines in GFAP—an independent disease-relevant biomarker linked to astrocyte injury—were strongly correlated with NfL changes (Pearson r > 0.85; p < 0.0001).
In addition to biomarker improvements, updated survival analyses from the HEALEY ALS Platform Trial and its open-label extension showed that participants treated with CNM-Au8 30 mg had markedly reduced mortality risk at one-year follow-up. In the full analysis set, the hazard ratio was 0.27 (73% risk reduction; p = 0.0144), and in a risk-balanced subset it was 0.229 (77% reduction; p = 0.0151) compared with regimen controls.
Clene said these results strengthen the case for CNM-Au8 as a disease-modifying therapy and support its plan to request a Type C meeting with the FDA in the first quarter of 2026. The company intends to submit a New Drug Application under the accelerated approval pathway, with a confirmatory Phase 3 trial (RESTORE‑ALS) planned as a post-approval commitment.
Given the robust reductions in NfL and GFAP, their correlation with survival benefit, and the long-term safety data (over 1,000 patient-years), CNM-Au8 stands out as one of the most promising investigational therapies for ALS currently under regulatory review.
