What is the drug thought to do in the body?
CNM-Au8 is an oral liquid suspension of gold nanocrystals designed to improve nerve cells’ survival, function, and communication by supporting their energetic needs, while lowering oxidative stress. Laboratory studies, in models of MND, have shown that CNM-Au8 can improve nerve cell survival and motor neurone function.
Trial Outcome
The Phase 2 trial (RESCUE-ALS) investigated the safety and efficacy of CMN-Au8 in 45 people with MND for 36 weeks. The trial did not meet its primary endpoint, which looked at the change in a marker of motor neurone loss (Motor Unit Number Index - MUNIX). However, additional data from the trial, including the open-label extension, showed potential signs that people who received CMN-Au8 earlier and for longer had a survival benefit.
This trial was small and trends in each of the measures favouring CNM-Au8 suggest that a larger trial, better powered to see subtle, but potentially meaningful effects, will be valuable. This is one of the reasons CNM-Au8 was also trialed in a much larger clinical trial - the Healey Platform Trial. The results from this trial also highlighted a potential survival benefit, but this trial also did not meet its primary endpoint of changing the rate of disease progression.
Previous Clinical Trials
Phase 1
This Phase 1 trial was a randomised, placebo-controlled, double blind trial. It's primary outcome was determine the safety, tolerability and pharmacokinetics (movement in the body) in healthy volunteers. The therapy was found to be safe and tolerable. You can find out more about this trial here.
Latest News
2023
June 2023 - Additional results from the HEALEY Platform Trial were announced. A statistically significant reduction of a marker of nerve damage, called neurofilament light chain (NfL), was observed for those who received 24-weeks of treatment with CNM-Au8 compared to placebo. However, comparison to baseline levels was not reported. Further analysis in underway to understand more about what these results may mean. You can read more in a press release.
March 2023 - Additional results from the RESCUE-ALS trial were announced. Highlighting a potential benefit in disease progression for participants who received the treatment earlier and for longer. You can read more in a press release.
2022
Sept 2022 - Top-line results from the HEALEY platform trial regime of CNM-Au8 were announced. The trial did not meet its primary endpoint of slowing disease progression by ALSFRS-R but a potential impact on survival was observed. You can find out more here.
Sept 2022 - Additional survival data from the open label extension was reported. You can find out more here.
July 2022 - Clene announced a positive opinion from the EMA for their submission of Orphan Drug Status for CNM-Au8. You can find out more here.
July 2022 - Survival analysis from the open label extension was reported. You can find out more here.
March 2022 - Clene announced additional analysis from the trial. You can find out more here.
2021
Dec 2021 - Clene announced updated data from the trial.
Nov 2021 - Topline results of the trial were announced.
Resources
Blog Posts
The Scientific Advisory Council (SAC) Briefing Notes
The Scientific Advisory Council for the International Alliance of ALS/MND societies consists of 9 members from USA, UK (including MND Association's Head of Research - Dr Nick Cole), Sweden, Italy, South Africa, China, Australia and Canada.
They produce briefing notes as a trusted source of information about current experimental treatments in clinical development, such as CNM-Au8.
These notes are produced after many rounds of careful discussion between the SAC members.
You can find the briefing note for CNM-Au8 here.
Last updated: 21/08/2024