CNM-Au8 is an experimental therapy that Clene Nanomedicine is developing 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. Researchers designed the compound 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 in motor neurons. The myelin sheath is the coating that protects nerve fibers from damage. CNM-Au8 may be able to protect the nerve cells from damaging cellular processes. It also may encourage remyelination (the formation of new myelin).
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.
Ongoing clinical trials
An open-label Phase 2 clinical trial (NCT03993171) called REPAIR-MS is currently recruiting an estimated 24 adults with relapsing-remitting MS (RRMS) in Texas. The aim is 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 goal is the ratio of two metabolic markers (NAD+ and NADH). This will be measured 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, after 12 weeks, and four weeks after the end of treatment.
Interim imaging results presented at the MSVirtual2020 conference showed significant engagement of CNM-Au8 with the central nervous system.
Another Phase 2 clinical trial (NCT03536559) called VISIONARY-MS is recruiting up to 150 RRMS patients in Australia who have lesions that affect their vision. The aim is to test the safety and effectiveness of CNM-Au8 in remyelinating nerve cells that play a role in vision.
Researchers will assign patients randomly to receive 15 mg or 30 mg of CNM-Au8 or a placebo in a liquid suspension every day for six months. The primary goal of the study is any improvements in low-contrast vision. Researchers will measure this using a low-contrast letter acuity (LCLA) test after 24 weeks of treatment. LCLA is an eye exam that uses a chart with low contrast between the letters and the background.
The secondary measure will be any improvements in remyelination. Researchers will measure this by visual evoked potentials (mfVEP) after 24 weeks of treatment. This is a vision test that measures the electrical potential across regions of the eye as the patient views an image.
Researchers presented interim results for the first 44 patients who received treatment for up to 36 weeks at MSVirtual2020. The results indicated a median improvement in LCLA, as well as three subscales of the multiple sclerosis functional composite (MSFC) test in patients receiving treatment. Safety data also indicated that the treatment was well-tolerated. Researchers expect to complete the trial in March 2021.
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: Oct. 23, 2020
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