Clene Inc. (NASDAQ: CLNN) has taken a significant step forward in its regulatory strategy for CNM-Au8, a promising therapy for neurodegenerative diseases, following a productive Type C meeting with the FDA. The company has resubmitted a revised statistical analysis plan (SAP) for evaluating neurofilament light (NfL) biomarker data from its Expanded Access Protocol for amyotrophic lateral sclerosis (ALS), with FDA acceptance anticipated this summer. This development is crucial as it paves the way for NfL analyses in early Q4 2025, supporting a potential New Drug Application (NDA) submission under the accelerated approval pathway.
In addition to the progress in ALS treatment, Clene has confirmed two more FDA meetings in Q3 2025. One meeting will assess long-term ALS survival data for accelerated approval consideration, while another, an End-of-Phase 2 Type B meeting, will review Phase 2 multiple sclerosis (MS) trial results and discuss a Phase 3 study aimed at cognitive improvement. These meetings underscore the FDA's interest in CNM-Au8's potential to address critical unmet needs in neurodegenerative diseases.
The implications of these regulatory advancements are profound. For patients suffering from ALS and MS, CNM-Au8 represents a beacon of hope, offering the possibility of a therapy that not only slows disease progression but also improves quality of life. For the biopharmaceutical industry, Clene's progress highlights the importance of innovative approaches targeting mitochondrial health and neuronal function in treating neurodegenerative diseases. The potential accelerated approval of CNM-Au8 could set a precedent for future therapies in this challenging therapeutic area.
Clene's commitment to advancing CNM-Au8 through the regulatory process reflects the urgent need for effective treatments for ALS and MS. With millions of patients worldwide awaiting breakthroughs, the outcomes of the upcoming FDA meetings could have far-reaching consequences for the field of neurodegenerative disease research and therapy development.
