What does the drug do?

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. Preclinical studies showed that CNM-Au8 improved nerve cell survival and motor neuron function in rodent models of MND and other neurodegenerative diseases, such as multiple sclerosis, and Parkinson’s disease.

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.

Phase 2 (RESCUE-ALS)

The Phase 2 trial is a randomised, double-blind, placebo-controlled study of the efficact, safety and pharmacokinetics and pharmacodynamics of CNM-Au8 in participants who became symptomatic in the last 24-months. The trial did not meet its primary or secondary endpoint of Motor Unit Number Index (MUNIX) biomarker or Forced Vital Capacity (FVC) at week 36. However, there were statistically significant reductions in other endpoints such as ALS disease progression and ALSFRS-R score and there was also evidence for potential long-term survival benefit. Additionally, it was found that CNM-Au8 demonstrated a stronger trend to efficacy in patients with both limb and blubar onset disease. These clinical benefits were described as extending the time to disease progression (defined as death, need for tracheostomy, non-invasive ventilation or gastrostomy tube). You can find out more about the trial here.

In March 2022 additional analysis from the trial was announced. This included evidence for significant survival benefit when compared to the validated ENCALS (European Network for the Cure of ALS) survival prediction model. This model predicts survival of patients with ALS based on baseline characteristics from over 11,000 patient records collected across Europe. Furthermore, the additional analysis looked into the MUNIX scores in more detail. They found that MUNIX scores changed very little for bulbar-onset participants. They hypothesised that this is due to the fact that the nerves used to calculate the MUNIX scores were controlled by the spinal cord and people living with bulbar-onset ALS often have less spinal cord damage. When the MUNIX scores were looked at for just people with limb-onset ALS they found that the score decreased by 40% in the placebo but little over 20% in the CNM-Au8 treatment. You can read more about these results here.

This trial does also have an optional open-label extension for up to 48 weeks.

This trial was recruiting in Australia only.

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 is also being trialed in a much larger clinical trial - the Healey Platform Trial.

Latest News

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 read more here

Sept 2022 - Additional survival data from the open label extension was reported. You can read more here

July 2022 - Clene announced a positive opinion from the EMA for their submission of Orphan Drug Status for CNM-Au8. You can read more here

July 2022 - Survival analysis from the open label extension was reported. You can read more about them here

March 2022 - Clene announced additional analysis from the trial. You can read more about them here.

Dec 2021 - Clene announced updated data from the trial. You can read more about them here.

Nov 2021 - Topline results of the trial were announced. You can read more about them here.


Want to find out more about CNM-Au8? Check out the resources below:


Update on clinical trials - part 1


Clene Inc. - Clene Inc. Presentation of RESCUE-ALS Topline Clinical Trial Results

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.

For further information and support contact our MND Connect helpline

mndconnect 0808 802 6262