USFDAAug 10, 2026, 08:14 AM
Clene Announces Biomarker Analyses for ALS NDA Accelerated Approval
AI Summary
Clene Inc. announced new biomarker analyses from its Phase 2 ALS trials (HEALEY and RESCUE-ALS) for CNM-Au8, which will be included in its planned New Drug Application (NDA) seeking accelerated approval. The analyses showed that CNM-Au8-treated patients whose neurofilament light chain (NfL) biomarker levels declined or stabilized lived significantly longer and had better functional outcomes than concurrently randomized controls. The company believes these findings address questions raised by the U.S. FDA and support the accelerated approval standard.
Key Highlights
- CNM-Au8-treated patients with NfL decline/stabilization showed significantly longer survival.
- CNM-Au8 30 mg reduced death risk by 74% vs. controls in HEALEY ALS trial (p=0.0124).
- NfL decline/stabilization linked to 38% lower death risk in HEALEY (HR 0.621, p=0.037).
- Average survival gain reached nearly six months at four years of follow-up (p=0.012).
- NfL responders scored better on Combined Assessment of Function and Survival (CAFS).
- Causal analysis showed 41% lower death risk in predicted NfL responders (HR 0.593, p=0.026).
- CNM-Au8 has over 1,100 participant-years in ALS with no serious adverse events.
- Planned Phase 3 trial (RESTORE-ALS) will prospectively evaluate NfL biomarker relationship.
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