Clene announces long-term follow-up data of CNM-Au8 in HEALEY ALS Platform trial

Clene announced long-term follow-up data for patients treated with CNM-Au8 30mg for up to 133 weeks in the HEALEY ALS Platform Trial. These post hoc results show significantly improved survival with a 49% decreased risk of death for the covariate risk-adjusted analyses compared to the largest U.S. clinical database of previous amyotrophic lateral sclerosis trials. The HEALEY ALS Platform Trial is a perpetual multi-center, randomized, double-blind, placebo-controlled clinical trial program designed to evaluate the efficacy and safety of multiple investigational products in people living with ALS. Enrollment in the CNM-Au8 Regimen was initiated in the summer of 2020. Participants received CNM-Au8 in addition to ALS standard-of-care and were randomized to the drug or placebo during the 24-week double-blind period. CNM-Au8 was then offered to all participants who were eligible, and 92% elected to continue into the Open Label Extension. CNM-Au8 30mg was selected as the dosage going forward after the double-blind period. In ALS, clinical studies with shorter double-blind treatment duration such as 24 weeks have used historical placebo controls from prior trials to determine the relative survival benefit of investigational treatment over longer-term follow-up. The PRO-ACT dataset is derived from pooled ALS clinical trial data from 29 completed Phase 2 and Phase 3 ALS clinical trials. Millions of de-identified longitudinally collected records from more than 11,600 individuals with ALS were standardized across trials and merged to create the Pooled Resource Open-Access ALS Clinical Trials database. This database provides a useful and validated surrogate for survival status of past participants in ALS clinical trials with long-term follow-up. In this analysis, 59 participants who were originally randomized to CNM-Au8 30mg were compared to matched placebo participants derived from the PRO-ACT dataset: Originally randomized CNM-Au8 30mg treated participants demonstrated a statistically significant 49% decreased risk of death compared to PRO-ACT matched placebo patients through long-term follow-up. In a pooled analysis of the HEALEY ALS Platform Trial and the RESCUE-ALS Trial, participants originally randomized to CNM-Au8 30mg demonstrated a statistically significant 59% decreased risk of death compared to PRO-ACT matched placebo patients through long-term follow-up. More than 500 estimated years of collective exposure across ALS, multiple sclerosis, and Parkinson’s disease participants in CNM-Au8 clinical trials and Expanded Access Protocol programs without any observed safety signals. No serious adverse events have been assessed as related to CNM-Au8 treatment; adverse events observed with CNM-Au8 have been characterized as transient and predominantly mild-to-moderate in severity.