Clene goes for gold, and goes public

Company targeting neurodegenerative disease with gold nanocatalyst technology goes public; multiple human trials ongoing for MS, Parkinson’s and ALS.

Salt Lake City biopharma company Clene Nanomedicine is now a publicly traded company. Clene, which has developed a new class of neuro-therapeutics called “nanocatalysts”, already has several products in Phase 2 (and one in Phase 3) trials for diseases including multiple sclerosis, Parkinson’s and amyotrophic lateral sclerosis.

Longevity.Technology: With a unique technology addressing a class of neurodegenerative disease that the World Health Organization predicts will become the second most prevalent cause of death within the next 20 years, Clene is a company on a mission to change how we treat diseases of the brain. We caught up with its CEO Rob Etherington to find out more.

Having completed a $42 million Series D financing last summer, Clene was already contemplating an IPO in 2021, when the opportunity arose to move more quickly via a SPAC (special purpose acquisition company), which saw the company go public by merging with Tottenham Acquisition I Ltd last week. The transaction raised approximately $31.9 million, and the combined, publicly-traded company operates under the name Clene Inc., with its common stock trading on the NASDAQ Capital Market under the ticker symbol “CLNN.”

“With 2020 somewhat becoming the year of the SPAC included in biotechnology… we saw this as a slightly more expeditious, slightly less onerous, slightly less expensive path to a public offering,” says Etherington.

2020 was a good year for IPOs, but it was an even better year for Special Purpose Acquisition Companies (SPACs): 242 SPACs were introduced last year.

“This capital enables us to conclude all of our clinical programs and to effectively prove whether or not we have a bioenergetic catalyst that will do what we say it does – that is neurorepair and remyelination.”

Co-founded by physicist and materials scientist, Mark Mortenson, Clene’s technology is built around his considerable expertise and experience.

“Mark had the somewhat brilliant idea to trailblaze a brand new path for the treatment of disease by taking a completely novel approach,” says Etherington. “He and his team of engineers and scientists achieved that through a completely novel application of the principles of material science, physics, and hydroelectric crystal chemistry to grow clean-surfaced metallic nanocrystals in water and use these nanocrystals to treat human diseases.”

“We have built a drug discovery platform, which means if you can think of a so-called therapeutic elemental metal used in some compound, we think we can build an oral, water-based nanotherapeutic based on such metals that may have even greater disease treating benefits.”

The idea to use elements like gold to drive medicinal efficacy has been around for hundreds of years, but Clene’s approach to using clean-surfaced metallic nanocrystals to target these conditions is, according to Etherington, completely unique.

“Clene’s basic hypothesis was to see if we could make highly faceted, clean-surfaced gold nanocrystals and used them as metabolic catalysts in various pharmaceutical applications,” he says. “Traditional approaches to making metal nanocrystals had two problems which needed to be overcome by Mark and his team, namely the lack of any positive biological results and, more importantly, inherent toxicity issues associated with surface residues left by known synthetic chemistry preparation technique.”

“We decided to test this hypothesis to see if we could improve neuroprotection, enhance remyelination, and amplify the benefits of healthy intracellular bioenergetic reactions that younger people have and take for granted.”

“These bioenergetic reactions are necessary for cells and for people to thrive, and get compromised as we age.”

The company’s bioenergetic gold-based nanocatalyst drug candidate is designed to accelerate neurorepair for patients with neurodegenerative disease. Its lead candidate, CNM-Au8, uses gold nanocrystals to amplify bioenergetic reactions in patients that ultimately drive intracellular biological reactions.

Having been through years of preclinical development, Clene has concluded all of the safety and toxicity work required by regulatory agencies, and is now in a number of clinical studies.

“What we’re hoping to show in humans is exactly what we’ve already shown in animals, and that is an ability to promote neuronal repair, neural protection and remyelination,” says Etherington. “When the clinical studies wrap up, it is our hope to show that we can potentially reverse disease for patients with amyotrophic lateral sclerosis, multiple sclerosis and Parkinson’s disease.”

One of several Phase 2 studies underway for CNM-Au8, the Visionary MS study aims to evaluate the safety and efficacy as a remyelinating and neuro-reparative treatment in MS patients. Its primary endpoint is to assess improvement in chronic visual impairment typically seen in about half of MS patients. The company presented encouraging interim data from the study earlier this year, and expects to report on the trial’s conclusion by early 2022.

Clene is also engaged in Phase 2 trials for both MS and Parkinson’s designed to prove that CNM-Au8 is entering the body orally and then getting into the brains of patients with the diseases and driving biological energetic reactions. The company also announced positive interim results from these studies this year, with final results expected in summer next year.

“ … he goal is to build a multicenter, multi-regimen, placebo controlled Phase 3 study to evaluate the safety and efficacy of investigational products …”

For ALS, Clene has two Phase 2 trials ongoing, including one looking at preservation of motor neuron function in ALS patients. Excitingly, the company is also in a Phase 3 ALS trial called the Healey ALS Platform Trial, which is being led by Mass General Hospital. Clene is one of three companies whose products were selected from a pool of 30 to be part of the trial.

If any of the three products achieve the primary endpoint of ALS functional response score, then the companies can use this data to file for a new drug application.

“The goal is to build a multicenter, multi-regimen, placebo controlled Phase 3 study to evaluate the safety and efficacy of investigational products for the treatment of ALS,” says Etherington. “This is the first platform study ever done in the neurodegenerative disease space, and the idea here is to reduce trial time, to reduce costs, and to increase patient participation.”

Image courtesy of Clene Nanomedicine