CNM-Au8 May Be Source of Motor Neuron Gains Being Seen in Phase 2 Trial

CNM-Au8 May Be Source of Motor Neuron Gains Being Seen in Phase 2 Trial
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Nearly half of all patients in a Phase 2 trial of CNM-Au8, Clene Nanomedicine’s lead candidate for the treatment of amyotrophic lateral sclerosis (ALS), showed improvements in the health and functioning of their motor neurons, according to the interim trial data.

These data are still blinded, meaning it’s unknown at this point if these benefits are in patients using the investigative oral therapy or those on a placebo in the RESCUE-ALS (NCT04098406) clinical trial.

But if CNM-Au8 is the source, these findings suggest the therapy is helping to maintain motor neurons — the nerve cells responsible for controlling movement that are gradually lost over the course of ALS.

Blinded interim study data were presented by Clene in the e-poster “RESCUE-ALS Trial, A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study of CNM-Au8 to Slow Disease Progression in Amyotrophic Lateral Sclerosis Patients: Design and Interim Blinded Results” (on page 23), at the Virtual 31st International Symposium on ALS/MND, held Dec. 9–11, 2020.

Clene expects to announce full study data later this year.

“This blinded interim analysis suggests that CNM-Au8 is working mechanistically to address a foundational challenge common to many neurodegenerative diseases, namely that stressed or failing neurons need additional energy for their survival, repair, and improved function,” Rob Etherington, president and CEO of Clene, said in a press release.

“We eagerly anticipate final results and are encouraged that these blinded interim results may provide hope for ALS patients and their families as they search for new therapies to treat this devastating disease,” Etherington added.

CNM-Au8 is an oral liquid suspension of gold nanocrystals designed to improve nerve cells’ survival, function, and communication by supporting their metabolic needs, while lowering oxidative stress — cellular damage resulting from the buildup of harmful oxidant molecules.

In preclinical studies, the therapy was found to benefit nerve cell survival and response to stress, and to improve motor neuron function in rodent models of ALS, multiple sclerosis, and Parkinson’s disease. An earlier Phase 1 trial (NCT02755870) involving 86 healthy volunteers also showed that CNM-Au8 was safe.

RESCUE-ALS is assessing the safety, efficacy, and pharmacological properties of CNM-Au8 in 44 people with newly symptomatic ALS, meaning those whose symptoms started within two years of study screening, or within a year following diagnosis.

Participants have been randomly assigned to either a liquid suspension containing 30 mg of CNM-Au8, or a placebo, being given each morning for 36 consecutive weeks (about eight months), in addition to standard of care therapies.

The study’s main goal is the effects of treatment on the number, function, and health of motor neurons, which will be evaluated based on changes in the Motor Unit Number Index (MUNIX) score from the trial’s beginning until the end of the 36-week treatment. MUNIX allows for a measure of motor neuron loss and overall health.

Changes in the scores of the ALS Functional Rating Scale revised (ALSFRS-R; a measure of disability), forced vital capacity (FVC; assess lung health), and other measures of quality of life will also be assessed.

These blinded interim findings covered trial data gathered up until Oct. 27. They showed that 40% of enrolled patients who had completed the first 12 weeks of their assigned treatment saw their average MUNIX scores increase since the study’s start.

“Emerging MUNIX data potentially indicate preservation of motor units, which is promising,” Etherington said.

Importantly, this increase surpassed the expectations set by statistical models, which predicted MUNIX scores would gradually drop from those recorded at the study’s start.

Despite being blinded, these data suggest that CNM-Au8 may have promising neuro-reparative properties that can help preserve motor neuron function in ALS patients.

“Although blinded to treatment assignment, these data are encouraging. We believe Clene’s breakthrough approach … may hold the potential to revolutionize the treatment of neurodegenerative diseases such as ALS and other motor neuron diseases,” said Robert Glanzman, MD, chief medical officer of Clene.

Joana holds a BSc in Biology, a MSc in Evolutionary and Developmental Biology and a PhD in Biomedical Sciences from Universidade de Lisboa, Portugal. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells — cells that made up the lining of blood vessels — found in the umbilical cord of newborns.
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Inês holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she specialized in blood vessel biology, blood stem cells, and cancer. Before that, she studied Cell and Molecular Biology at Universidade Nova de Lisboa and worked as a research fellow at Faculdade de Ciências e Tecnologias and Instituto Gulbenkian de Ciência. Inês currently works as a Managing Science Editor, striving to deliver the latest scientific advances to patient communities in a clear and accurate manner.
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Joana holds a BSc in Biology, a MSc in Evolutionary and Developmental Biology and a PhD in Biomedical Sciences from Universidade de Lisboa, Portugal. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells — cells that made up the lining of blood vessels — found in the umbilical cord of newborns.
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