New Class of Drugs Harnesses Gold Nanocrystals to Heal and Protect the Brain

Clene Chief Executive Officer Rob Etherington

Clene Chief Executive Officer Rob Etherington. Photo courtesy of Clene.

Clene Nanomedicine is trying to set a new “gold standard” in neurodegenerative diseases through the development of a new class of drugs called bioenergetic nanotherapeutics that harnesses the properties of gold nanocrystals.

The gold nanocrystals are used to amplify bioenergetic reactions in patients in order to drive intracellular biological reactions. Bioenergetic nanotherapeutics are a “clean break” from pharmaceutical drug development that uses classical synthetic chemistry, Clene Chief Executive Officer Rob Etherington told BioSpace in an interview. Clene’s lead asset is CNM-Au8, a bioenergetic nanocatalyst under development as an add-on treatment for neurodegenerative diseases like Parkinson’s disease, multiple sclerosis and Amyotrophic Lateral Sclerosis (ALS). CNM-Au8 is designed to catalyze bio cellular reactions, and so far the company has seen the asset live up to its promise in clinical studies. The company’s gold nanocrystals are grown in water and patients drink the asset down. Research has so far indicated that Clene and its golden asset could become a pioneer in therapeutic neurorepair and neuroprotection.

To date, CNM-Au8 has demonstrated safety in Phase I studies, remyelination and neuroprotective effects in preclinical models and is currently being assessed in a Phase II study for the treatment of chronic optic neuropathy in patients with multiple sclerosis. Additionally, CNM-Au8 is being studies in Phase II and Phase III studies for disease progression in patients with ALS. In September, Clene presented interim results from the REPAIR-MS and REPAIR-PD Phase II studies demonstrating the effects of its lead nanocatalytic therapeutic, CNM-Au8. The preliminary data demonstrate CNM-Au8-mediated modulation of key brain bioenergetic metabolites in relapsing multiple sclerosis (MS) and Parkinson's disease (PD) patients. Data from the studies indicate catalytic bioenergetic improvements across important CNS bioenergetic metabolites, including total nicotinamide adenine dinucleotide (NAD) levels, NAD+/NADH ratio, and adenosine triphosphate (ATP) levels, indicating a homeostatic effect of CNM-Au8 on brain bioenergetics, the company said.

Etherington said the data from the REPAIR-MS and REPAIR-PD studies indicate that CNM-Au8 is working mechanistically to address a foundational challenge common to many neurodegenerative diseases, which is that stressed or failing neurons need additional energy to survive and repair.

“We now have insights that CNM-Au8 is driving bioenergetics within the brain, which is a foundational insight for the further development of Clene's neurorepair clinical programs,” Etherington said. He added that should the data from the interim analysis pan out, it indicated that CNM-Au8 could effectively benefit millions of people across the globe suffering from multiple sclerosis and other neurodegenerative diseases.

There are multiple drugs already on the market for these neurodegenerative diseases. CNM-Au8 is not meant to replace those drugs, but to work alongside them. Etherington explained that CNM-Au8 is not designed to target a specific protein, nor it is designed to block or antagonize something, like most drugs. Rather, Clene’s compound is designed to enhance the intracellular biological actions necessary to repair and reverse neuronal damage, Etherington said.

“We are purposely seeking to reverse neurodegernation. We want to let the cell take care of its own housekeeping and enhance what’s naturally occurring in the central nervous system,” he said.

Etherington acknowledged the concept of drinking bits of gold nanocrystals may sound like something out of a Star Trek episode, but insisted the idea is sound. Gold-salt injections were historically used to treat rheumatoid arthritis decades ago, but were dropped due to health concerns. Clene had the idea to build a stable, oral nanotherapeutic, so they could see less toxicity and drive bioenergetics targets for a suite of neurodegenerative diseases, he said.

“It’s so out of the box that it can be a bit mind boggling. We’re breaking with the traditional path and shifting the paradigm to how we think neurodegenerative disease should be treated,” he said.

Not only is Clene moving forward in its clinical assessment of CNM-Au8, the company is planning to go public with a special purpose acquisition companies (SPAC) merger before the end of 2020. 2020 has been the busiest year for this kind of stock entry, with a 250% surge. As BioSpace recently reported, there have been nearly two dozen SPAC mergers in the biotech sector this year, targeting more than $3.5 billion in proceeds. When the company goes public, Etherington said Clene’s management team will remain the same and the funding raised from this reverse stock merger will provide the finances that can support the company’s ongoing research.

Featured Jobs on BioSpace

Back to news