Clene Inc. submitted a New Drug Application to the U.S. Food and Drug Administration in October 2026 seeking accelerated approval for CNM-Au8, an investigational therapy for amyotrophic lateral sclerosis. The Salt Lake City-based biotechnology company, which trades on Nasdaq under the ticker CLNN, stated that the submission relies on data from a neurofilament light chain biomarker and clinical outcomes.
Rob Etherington, President and Chief Executive Officer of Clene, described the filing as an important achievement for the company and for individuals living with ALS. He noted that the company values the FDA's constructive engagement throughout the process and anticipates working closely with the agency during its review. Etherington attributed the milestone to years of dedicated work by Clene's team, clinical investigators, patients, families, and partners.
Clene continues preparing for a confirmatory Phase 3 study known as RESTORE-ALS while advancing CNM-Au8 through the regulatory process. The company believes the therapy has the potential to restore and protect neuronal health and function, which could improve survival for patients with ALS. ALS is a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord.
CNM-Au8 is a first-in-class therapy designed to improve the survival and function of central nervous system cells. It operates by targeting mitochondrial function and the NAD pathway while reducing oxidative stress. Clene, along with its subsidiary Clene Nanomedicine Inc., focuses on improving mitochondrial health to treat neurodegenerative diseases, including Parkinson's disease and multiple sclerosis. The company maintains research and development and manufacturing operations in Maryland.
The press release includes forward-looking statements regarding the timing of the RESTORE-ALS study and the potential benefits of CNM-Au8. Clene cautions that these statements involve risks and uncertainties, including whether clinical trials will demonstrate efficacy and safety to regulatory satisfaction, actions by regulatory agencies, and the company's ability to obtain additional funding. Actual results may differ from those expressed or implied in these statements.