Clene Inc. and its wholly owned subsidiary, Clene Nanomedicine Inc., a clinical-stage biopharmaceutical company focused on revolutionizing the treatment of neurodegenerative diseases, reported new data demonstrating a statistically significant reduction of plasma neurofilament light chain (NfL) levels for CNM-Au8® treated participants compared to placebo after 24 weeks of treatment in the double-blind, placebo-controlled period of the HEALEY ALS Platform Trial.
NfL is a key biomarker of neurodegeneration. NfL is released from neurons following axonal injury, especially in people living with ALS, where higher levels of NfL have been found to predict more rapid decline in clinical function and increased mortality risk. Surrogate biomarkers such as NfL have recently been used to support an FDA approval for the treatment of ALS. The results are based on an analysis of the plasma NfL biomarker across all Regimen C participants (CNM-Au8 or placebo, n=161), representing a broad ALS population, as the least-square mean (LS mean) change of the natural logarithm (Ln) of the plasma NfL values with the standard error (SE): CNM-Au8 treatment reduced plasma NfL levels compared to placebo; LS Means on a Ln Scale for the 24-week difference of plasma NfL: CNM-Au8 = -0.024 (SE: 0.024); placebo = +0.076 (SE: 0.042); CNM-Au8 vs. placebo difference of LS Means on a Ln Scale = -0.100 (SE: 0.048), p=0.040.
In addition to the full analysis across all Regimen C participants, sensitivity analyses showed consistent significant reduction in plasma NfL levels versus placebo observed in specific populations generally considered at greater risk of ALS disease progression, including: Faster progressors (baseline pre-treatment ALSFRS-R slope >0.45 points/month (post hoc, n=107); Difference of LS Means on a Ln Scale (SE) = -0.144 (0.058); p=0.014. Definite or probable ALS diagnosis per El Escorial criteria (post hoc, n=125); Difference of LS Means on a Ln Scale (SE) = -0.124 (0.054); p=0.023. Higher mortality risk (baseline plasma NfL > median, post hoc, n=79); Difference of LS Means on a Ln Scale (SE) = -0.150 (0.068); p=0.031. Analyses of NfL from serum samples specified as the primary blood matrix for analysis are underway. Additional biomarker and long-term survival data from the HEALEY ALS Platform Trial double-blind and open-label extension periods have been collected and are undergoing testing preparatory for analysis to be reported later this year.