Clene Submits FDA Application Seeking Accelerated Approval of CNM-Au8 as Treatment for Those with ALS
KEY TAKEAWAYS
- Clene submitted an NDA seeking accelerated approval of CNM-Au8 for ALS.
- The application includes biomarker and clinical evidence from previous studies, although randomized trials did not meet their primary efficacy endpoints.
- Clene is preparing the confirmatory phase 3 RESTORE-ALS trial.
Clene announced that it submitted a New Drug Application (NDA) to the FDA seeking accelerated approval of CNM-Au8, an investigational gold nanocrystal therapy for amyotrophic lateral sclerosis (ALS). The submission is supported by neurofilament light chain (NfL) biomarker analyses and clinical data from the RESCUE-ALS trial, HEALEY ALS Platform Trial, and an expanded access program.¹
As previously reported by Practical Neurology in March 2026, CNM-Au8 did not significantly slow ALS progression in the randomized HEALEY ALS Platform Trial.²˒³ Earlier RESCUE-ALS findings also failed to meet the primary neurophysiological endpoint, although exploratory analyses suggested potential survival benefits.⁴
In August 2026, Clene reported additional analyses from these studies associating NfL reductions or stabilization with improved survival and functional outcomes.⁵ These exploratory findings, rather than prospectively confirmed clinical benefits, form part of the evidence supporting the accelerated approval request.
CNM-Au8 is designed to improve neuronal energy metabolism and mitochondrial function while reducing oxidative stress. Clene is preparing the phase 3 RESTORE-ALS trial to further evaluate clinical benefit.¹˒⁷
The FDA has not approved CNM-Au8, and its efficacy remains unconfirmed.
References
- Clene Inc. Clene announces New Drug Application submission to FDA for CNM-Au8 for the treatment of ALS. Published October 19, 2026. Original announcement.
- Practical Neurology. CNM-Au8 did not slow ALS progression in HEALEY ALS Platform Trial. Published March 9, 2026. Read article.
- Writing Committee for the HEALEY ALS Platform Trial, HEALEY ALS Platform Trial Study Group. CNM-Au8 in amyotrophic lateral sclerosis: the HEALEY ALS Platform Trial. JAMA. 2025;333(13):1138-1149. doi:10.1001/jama.2024.27643.
- Practical Neurology. Results from RESCUE-ALS study demonstrate safety and efficacy of CNM-Au8 for treatment of ALS. Published September 20, 2023. Read article.
- Clene Inc. Clene announces biomarker analyses to be included in NDA submission supporting ALS accelerated approval. Published August 10, 2026. Original announcement.
- ALS Society of Canada. Clinical research & trials update, August 2026. Read article.
- Clene Inc. After successful FDA meeting, Clene filing accelerated approval NDA for ALS. Published May 4, 2026. Original announcement.