Decreased Mortality in Amyotrophic Lateral Sclerosis Seen in Open-Label Extension Study of Nanocrystalline Gold

Results from the long-term, open-label extension of the RESCUE-ALS trial (NCT04098406) showed significantly improved survival in participants with amyotrophic lateral sclerosis (ALS) treated with an oral suspension of nanocrystalline gold (CNM-Au8; Clene Nanomedicine, Salt Lake City, UT) vs placebo during the double-blind portion of this study. The unadjusted Kaplan-Meier survival analyses showed a significant survival benefit of 70% decreased risk of death (log-rank hazard ratio=0.301, 95% CI: 0.122 to 0.742, P=.0143). 

“We are very pleased to see these results and the apparent survival benefit that our investigational drug, CNM-Au8, appears to provide to people living with ALS,” said Rob Etherington, president and CEO, Clene. “At this point, we are awaiting top-line data from the HEALEY ALS Platform Trial, which focuses on endpoints measuring patient function, survival, and breathing over a 6-month period in a much larger cohort. Clene expects to announce these results this quarter. Based on the larger number of patients treated in the HEALEY trial and the higher dose of CNM-Au8 being tested, we are optimistic that we will be able to adequately characterize the effects of our drug on this devastating disease.”

Nanocrystalline gold was well-tolerated, and there were no significant safety findings reported during the long-term, open-label trial period. 

The trial enrolled 45 participants randomly assigned to receive 30 mg/day nanocrystalline gold (n=23) or placebo (n=22) for 36 weeks followed by treatment for all participants with 30 mg/day nanocrystalline gold in the open-label period for an additional 94 weeks. Survival was compared by treatment group from randomization through the latest vital status observation.