The U.S. Food and Drug Administration (FDA) granted CNM-Au8 orphan drug status for the treatment of ALS.
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 to help remove the destructive by-products 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 that CNM-Au8 was safe and well-tolerated.
CNM-Au8 has been selected for inclusion in the first platform trial (NCT04081714) in ALS, which is being launched by the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital in Boston. CNM-Au8 is among the therapeutic candidates being tested in parallel under a similar protocol, the hallmark of a platform trial. Here, up to 20 ALS patients will be enrolled and given oral CNM-Au8 daily for up to 24 weeks in the open-label study’s “first period.” Safety, pharmacokinetics, and pharmacodynamics (the effects of treatment on the body) will be evaluated.
This platform study may be recruiting; information is available here.
A Phase 2 clinical trial (NCT04098406), called RESCUE-ALS, is currently enrolling 42 ALS patients at two locations in Sydney, Australia. Patients will be randomly assigned to either CNM-Au8 or a matching oral placebo daily for 36 weeks. The study will assess the safety, efficacy, pharmacokinetics, and pharmacodynamics of the treatment. Its primary goal will be changes from baseline to week 36 in MUNIX (motor unit number index), a quantitative neurophysiological method that reflects the loss of motor neurons in ALS.
A second Phase 2 trial (NCT03843710) is recruiting an estimated 24 ALS patients at UT Southwestern in Dallas, 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. Patients will be divided into two groups; and treatment dose given to those in the second group will be based on the findings from the first group. One of four doses (7.5, 15, 30 or 60 mg in a liquid suspension) will be given daily for 12 weeks in first group; then selected dose or doses (up to two may be chosen) daily for 12 weeks in the second group.
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.
Last updated: Jan. 28, 2020
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