Clene Announces Additional CNM-Au8 Biomarker Data Supporting Potential NDA Filing for Upcoming In-Person FDA Meeting

Core Viewpoint - Clene Inc. has received an in-person Type C Meeting from the FDA to discuss its biomarker and survival data related to its investigational treatment CNM-Au8 for neurodegenerative diseases, particularly ALS [1][8][13] Group 1: FDA Interaction and Meeting - The FDA has granted Clene an in-person Type C meeting scheduled for the first quarter of 2026 to review evidence supporting the use of neurofilament light chain (NfL) as a candidate biomarker for accelerated approval [1][8] - Clene has submitted a pre-meeting briefing package to the FDA, addressing previous requests regarding the clinical significance of NfL declines and their reproducibility [2][4] Group 2: Biomarker Evidence and Analysis - Clene's analyses indicate that longitudinal changes in NfL are associated with survival outcomes in ALS patients, independent of baseline disease severity [3][5] - Statistically significant reductions in NfL levels were observed in patients treated with CNM-Au8, demonstrating consistent pharmacodynamic effects across multiple studies [6][9] Group 3: Clinical Outcomes and Survival Benefits - Modest reductions in NfL (approximately 9-10%) were associated with an 8-13% lower risk of death in ALS patients, highlighting the potential clinical relevance of NfL as a surrogate endpoint [7][9] - In responders with IGFBP7 biomarker decline, CNM-Au8 treatment was associated with a 78% reduced mortality risk, indicating a strong link between biomarker changes and survival [8][10] Group 4: Mechanistic Insights and Future Directions - IGFBP7 has been identified as a key pharmacodynamic biomarker, with its decline correlating with improved survival and other disease-relevant biomarkers [11][12] - The findings suggest a mechanistic pathway linking CNM-Au8's action to neuroprotection, supported by genetic evidence related to IGFBP7 expression [12]