CNM-Au8 is an experimental therapy by Clene Nanomedicine to treat patients with amyotrophic lateral sclerosis (ALS), Parkinson’s disease, and multiple sclerosis (MS).

How does CNM-Au8 work?

CNM-Au8 contains a suspension of nanocrystalline gold that acts to support biological reactions within cells that generate energy, as well as help remove the destructive byproducts of cellular metabolism.

Preclinical studies have demonstrated that CNM-Au8 is able to protect motor neurons from severe damage and death. Motor neurons are nerve cells that are responsible for the movement of muscles.

CNM-Au8 in clinical trials for ALS

A randomized, placebo-controlled, double-blind, and escalating dose Phase 1 clinical trial (NCT02755870) assessed the safety, tolerability, and pharmacokinetics (movement in the body) of CNM-Au8 in healthy volunteers. The results showed CNM-Au8 was safe and well-tolerated.

Ongoing clinical trials

Clene Nanomedicine selected CNM-Au8 for inclusion in the first platform trial (NCT04081714) in ALS. The treatment is among the candidates that researchers are testing in parallel under a similar protocol, the hallmark of a platform trial. Here, up to 20 ALS patients will receive oral CNM-Au8 daily for up to 24 weeks in the open-label study’s “first period.” Researchers will evaluate the treatment’s safety, pharmacokinetics, and pharmacodynamics (the effects on the body).

A Phase 2 clinical trial (NCT04098406), called RESCUE-ALS, aims to enroll 42 ALS patients at two locations in Sydney, Australia. Investigators will randomly assign patients to either CNM-Au8 or a matching oral placebo daily for 36 weeks. The study is assessing the safety, efficacy, pharmacokinetics, and pharmacodynamics of the treatment. Its primary goal is to observe changes from baseline to week 36 in MUNIX (motor unit number index). This is a quantitative neurophysiological method that reflects the loss of motor neurons in ALS.

Researchers presented interim blinded results of the trial at the 31st International Symposium on ALS/MND in December 2020. These showed that more than 40% of patients who had completed at least 12 weeks of treatment had an increase in MUNIX scores. There was an increase in the average MUNIX score for the whole population of participants. This included both patients receiving treatment or a placebo. The data is still blinded. However, it may indicate that CNM-Au8 has a restorative effect in ALS patients. Unblinded results of the trial are expected in the second half of 2021.

A second Phase 2 trial (NCT03843710) soon will be recruiting an estimated 24 ALS patients at UT Southwestern in Dallas, Texas, for an open-label pilot, sequential group study assessing the metabolic effects, safety, pharmacokinetics, and pharmacodynamics of CNM-Au8.

All patients will receive CNM-Au8, but both physicians and patients will be blinded as to the dose used. Investigators will divide patients into two groups. They will give a treatment dose to those in the second group based on the findings from the first group. Patients in the first group will receive one of four doses (7.5, 15, 30, or 60 mg in a liquid suspension) daily for 12 weeks. Those in the second group will receive the selected dose or doses daily for 12 weeks. 

The study’s primary goal (endpoint) is the ratio of two metabolic markers (NAD+ and NADH) using a technique called 31-phosphorous magnetic resonance spectroscopy (31P-MRS). These metabolic markers can indicate how much energy cells produce, and whether the energy balance has changed. Patients will go in for a 31P-MRS scan before the start of treatment (a four-week screening period), after 12 weeks of treatment, and four weeks after the end of treatment.

Other information

The U.S. Food and Drug Administration granted CNM-Au8 orphan drug status for the treatment of ALS in July 2019.


Last updated: Jan. 8, 2021


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