Clene and its subsidiary Clene Nanomedicine have reported data from RESCUE-ALS long-term, open-label extension clinical trial, where CNM-Au8 substantially boosted survival in patients with amyotrophic lateral sclerosis (ALS).
An oral suspension of gold nanocrystals, CNM-Au8 was developed for restoring neuronal health and function by boosting energy production and usage.
The trial enrolled 45 participants who were randomised into a 1:1 ratio to receive 30mg CNM-Au8 or placebo daily for 36 weeks during the trial’s double-blind portion.
Subsequently, in the open-label portion, the subjects received treatment for up to 130 weeks from randomisation.
As of the data cutoff on 5 July this year, five deaths were reported in the group which was randomised to receive CNM-Au8 compared to 14 in the group initially randomised to receive placebo.
Due to inadequate mortality events, median survival from randomisation in the CNM-Au8 group was undefined versus 23.1 months in the placebo arm.
According to the unadjusted Kaplan-Meier survival assessments, a substantial survival benefit was demonstrated in subjects initially randomised to the CNM-Au8 treatment group compared to those who received a placebo initially, indicating a 70% decline in mortality risk.
Furthermore, CNM-Au8 was found to be well-tolerated without any significant safety findings observed during the long-term, open-label study period.
Clene president and CEO Rob Etherington said: “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.
“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 six-month period in a much larger cohort.”
