Clene (NasdaqCM:CLNN) Update / Briefing Transcript

Summary of Clene Nanomedicine's ALS Program Update Conference Call Company and Industry - Company: Clene Nanomedicine - Industry: Amyotrophic Lateral Sclerosis (ALS) treatment and research Key Points and Arguments Overview of CNM-Au8 and ALS Program - Clene has been developing CNM-Au8 for ALS treatment for 12 years, with significant data supporting its efficacy [3][4] - The company has conducted randomized double-blind clinical trials and expanded access protocols for ALS patients [3] FDA Interaction and Data Requirements - The FDA indicated that if Clene can substantiate the effects on neurofilament light (NFL), they would consider the 24-week data from the Healey trial for accelerated approval [4] - Clene is focusing on three areas: neurofilament change analysis, biomarker change analysis, and supportive ALS disease-specific biomarkers [6] Biomarkers and Clinical Relevance - Neurofilament light chain (NFL) and glial fibrillary acidic protein (GFAP) are key biomarkers for ALS, with NFL being a structural protein in neurons and GFAP indicating astrocytic activity [8][9] - Higher baseline NFL levels correlate with faster disease progression and higher mortality risk [10] Expanded Access Program Findings - The NIH Expanded Access Program provided a dataset of 291 participants, showing significant reductions in neurofilament levels across matched participants [12][14] - Bulbar onset patients showed a stronger reduction in neurofilament levels, indicating a clinically meaningful effect [16] Survival Analysis and Efficacy - CNM-Au8 demonstrated a 73% reduction in mortality risk at 12 months in the full analysis set and a 77% reduction in a comparable risk set [30] - Participants transitioning from placebo to CNM-Au8 showed a 51% reduction in mortality risk at 12 months [32] Future Steps and Regulatory Considerations - Clene plans to submit a new drug application to the FDA, with a focus on the totality of data supporting CNM-Au8's efficacy [36] - The upcoming phase 3 RESTORE-ALS trial will have survival as the primary endpoint, with ALSFRS as a secondary exploratory endpoint [66] Additional Insights - The data from the Expanded Access Program supports the hypothesis that CNM-Au8 can provide benefits even in more advanced ALS patients [60] - The relationship between neurofilament and GFAP changes and survival strengthens the case for CNM-Au8's clinical impact [27][33] Other Important Content - The call included discussions on the challenges of using matched controls in the Expanded Access Program and the importance of ensuring comparability in patient characteristics [55] - There was a focus on the potential implications of the findings for other neurodegenerative diseases, such as multiple sclerosis [75]