Clene Inc. (NASDAQ: CLNN) is moving forward with a neurofilament biomarker analysis for its lead candidate CNM-Au8 in the treatment of amyotrophic lateral sclerosis (ALS), with the U.S. Food and Drug Administration (FDA) providing supportive feedback on the company's proposed statistical analysis plan. This development is significant as it could lead to an accelerated approval submission for CNM-Au8 later in 2025, offering a potential new treatment avenue for ALS patients who currently have limited options.
The analysis will focus on neurofilament light chain (NfL), a recognized biomarker for neurodegeneration, among nearly 200 ALS patients participating in the National Institutes of Health-sponsored Expanded Access Program (EAP) for CNM-Au8. The company aims to validate the neuroprotective effects observed in previous trials, comparing treated patients to matched controls at six and nine months. This research is crucial as it seeks to confirm the therapeutic potential of CNM-Au8 in slowing disease progression in ALS, a devastating neurodegenerative condition.
Benjamin Greenberg, MD, Head of Medical at Clene, highlighted the FDA's constructive feedback and the agency's collaborative approach. With additional FDA meetings scheduled to discuss long-term ALS survival results and the company's multiple sclerosis (MS) program, Clene is advancing its efforts to address unmet needs in neurodegenerative diseases. The potential for CNM-Au8 to receive accelerated approval underscores the importance of this research, not only for ALS patients but also for the broader field of neurodegenerative disease treatment.
Clene's commitment to improving mitochondrial health and protecting neuronal function represents a promising direction in the fight against ALS and MS. The upcoming biomarker analysis and FDA discussions could mark a significant step forward in the development of effective treatments for these challenging conditions, offering hope to patients and families affected by them.
