Clene Inc. (NASDAQ: CLNN) is preparing to conduct a neurofilament biomarker analysis for its lead candidate, CNM-Au8(R), in the treatment of amyotrophic lateral sclerosis (ALS) in early Q4 2025. This initiative follows a productive Type C meeting with the U.S. Food and Drug Administration (FDA), where the agency provided supportive feedback on Clene's proposed statistical analysis plan. The analysis could play a pivotal role in supporting an accelerated approval submission for CNM-Au8 later in the same year.
The neurofilament biomarker 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 study aims to validate the neuroprotective effects observed in previous results from the HEALEY Platform Trial, comparing treated patients to matched ALS controls at six and nine months of treatment.
Benjamin Greenberg, MD, Head of Medical at Clene, expressed optimism about the FDA's collaborative approach and the constructive feedback received. The company has already resubmitted its updated statistical analysis plan to the FDA, incorporating requested changes, with the expectation of finalizing the evaluation framework ahead of the biomarker review in Q4 2025.
Clene has scheduled two additional FDA meetings in Q3 2025 to discuss long-term ALS survival data and its multiple sclerosis (MS) program, including Phase 2 data from the VISIONARY-MS trial. These discussions are crucial for advancing Clene's ALS and MS programs, with the potential to bring innovative therapies to patients suffering from these neurodegenerative diseases.
The FDA's engagement underscores the potential of CNM-Au8, an oral suspension of gold nanocrystals designed to enhance cellular energy production, as a promising treatment for ALS and MS. With limited treatment options currently available for ALS, the successful outcome of the NfL assessment could significantly impact the lives of patients by paving the way for accelerated approval of CNM-Au8.
Clene's commitment to improving mitochondrial health and protecting neuronal function represents a significant step forward in the treatment of neurodegenerative diseases. The company's ongoing research and development efforts, supported by constructive FDA feedback, highlight the potential of CNM-Au8 to meet the urgent need for effective therapies in the ALS and MS communities.
