CNM-Au8 is an experimental therapy being developed 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. The compound is designed to support cellular reactions 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. In MS, there is a loss of the myelin sheath — the coating that protects nerve fibers from damage. CNM-Au8 may be able to protect the nerve cells from damaging cellular processes, as well as encouraging remyelination.

CNM-Au8 in clinical trials for MS

A randomized, placebo-controlled, double-blind, 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 that CNM-Au8 was safe and well-tolerated.

An open-label Phase 2 clinical trial (NCT03993171) called REPAIR-MS is currently recruiting an estimated 24 adult patients with relapsing-remitting MS (RRMS) in Texas to assess the safety, pharmacokinetics, and pharmacodynamics (effect on the body) of CNM-Au8. Patients will take one of four doses (7.5, 15, 30 or 60 mg) of CNM-Au8 in a liquid suspension every day for 12 weeks. The study’s primary 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 of the cell has changed. Patients will undergo 31P-MRS scans before the start of treatment, after 12 weeks of treatment, and four weeks after the end of treatment.

Another Phase 2 clinical trial (NCT03536559) called VISIONARY-MS is recruiting as many as 150 RRMS patients in Australia with lesions that affect their vision, to test the safety and efficacy of CNM-Au8 in remyelinating nerve cells related to vision. Patients will be randomly assigned to receive 15 mg or 30 mg of CNM-Au8 or placebo in a liquid suspension every day for six months. The primary endpoint of the study is the improvement in low-contrast vision as measured by low-contrast letter acuity (an eye exam using a chart with low contrast between the letters and the background of the chart) after 24 weeks of treatment. The secondary measure will be any improvements in remyelination as measured by visual evoked potentials (mfVEP, a vision test that measures the electrical potential across regions of the eye as the patient views an image) after 24 weeks of treatment.

Other information

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

 

Last updated: Feb. 14, 2020

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