Clene announces new data from HEALEY ALS Platform Trial

Clene and its wholly owned subsidiary Clene Nanomedicine reported new data from the 12-month long-term open label extension of the CNM-Au8 treatment arm in the HEALEY ALS Platform Trial. Long-Term Plasma NfL Biomarker Findings from Regimen C in the HEALEY ALS Platform Trial Open Label Extension: Plasma neurofilament light chain, a blood-based biomarker associated with neurodegeneration, declined by 16% from baseline to 76 weeks of treatment in the HEALEY ALS Platform Trial Open Label Extension in participants randomized to CNM-Au8 30 mg relative to participants initially randomized to placebo. CNM-Au8 30mg treatment reduced plasma NfL levels compared to baseline: Mixed Model with Repeat Measures, Least Squared Means on a Natural Log Scale for the 76-week change from baseline of plasma NfL: CNM-Au8 = -0.075; placebo = +0.098; CNM-Au8 30mg vs. original placebo difference of LS Means on a Ln Scale = -0.173. Long-Term Survival Improvement from Regimen C in the HEALEY ALS Platform Trial Open Label Extension: Long-term survival analyses included the prespecified rank-preserving structural failure time model to account for the effects of CNM-Au8 in participants randomized to placebo who crossed-over to treatment with CNM-Au8. Under an assumption of a constant common treatment effect from CNM-Au8, treatment with CNM-Au8 demonstrated a 60% decreased risk of long-term all-cause mortality in participants originally randomized to treatment with CNM-Au8 compared to those originally randomized to placebo, after adjusting for the estimated benefit received after switching to CNM-Au8. Post-hoc Analysis Validates Association of NfL levels with Clinical Morbidity Outcomes and the Effects of CNM-Au8 in High Risk ALS Patients. To investigate the role of NfL in the incidence of ALS clinical worsening events, the pooled population of the HEALEY ALS Platform and the RESCUE-ALS trial were stratified by baseline plasma NfL levels by quartile. The average number of ALS clinical worsening events including death, tracheostomy, feeding tube placement, and initiation of assisted ventilation were calculated for each treatment group during the double-blind periods. Results of these analyses suggested a beneficial effects of CNM-Au8 in delaying occurrence of clinical worsening events in the highest risk NfL quartiles.