FightMND Awards Clene More Than $900K to Advance Investigational ALS Therapy

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by Mary Chapman |

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Fight Motor Neuron Disease (FightMND) has awarded Clene Nanomedicine‘s Australian subsidiary $1.37 million AUD (approximately $924,000) to advance clinical studies of a new amyotrophic lateral sclerosis (ALS) candidate treatment.

These funds will support a Phase 2 clinical trial, named RESCUE-ALS, that will assess the efficacy and safety of the investigational CNM-Au8 in slowing the progression of ALS. The double-blind, placebo-controlled study will be led by ALS clinicians Steve Vucic and Matthew Kiernan at Westmead Hospital and the Sydney Brain and Mind Centre, both in Sydney, Australia.

CNM-Au8 is an orally administered gold nanocrystal suspension designed to overcome cells’ bioenergetic imbalance, allowing cells to have the energy needed to function efficiently.

The therapy is the first of a new class of neurotherapeutic medicines. It is designed to support bioenergetic cellular reactions and help remove the destructive byproducts of cellular metabolism that add to the breakdown of motor neurons in ALS.

“The nanocatalyst exerts unique effects on human motor neurons. We are excited by its potential therapeutic benefits in ALS,” Vucic said in a press release.

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“This is a really excellent opportunity to develop a new therapeutic approach to ALS,” Kiernan added.

Preclinical studies in multiple sclerosis models showed that CNM-Au8 can prevent the death of nerve cells that control movement through an innovative nanocatalytic mechanism. That mechanism enhances the cells’ ability to resist disease-associated stressors.

The compound was demonstrated to be safe in Phase 1 studies in healthy volunteers (NCT02755870).

“We are delighted to receive [FightMND’s] award, which will allow us to further develop our clean-surfaced, faceted gold nanocrystal therapeutic CNM-Au8 for the treatment of ALS,” said Rob Etherington, president and CEO of Clene. “With few truly disease-modifying options available for neurodegenerative diseases like ALS, Clene is striving to develop innovative new treatments, for we realize the urgency of patients who daily fight this devastating disease.”

CNM-Au8 also 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, Massachusetts. Five therapeutic candidates, including CNM-Au8, will be assessedduring the Healey ALS Platform Trial.

This new treatment candidate also has received a regulatory green light to advance to clinical research for Parkinson’s disease and multiple sclerosis, the company said.

“We would like to express our gratitude to FightMND and all of its contributors for helping to accelerate our efforts to make a safe and effective treatment available to ALS patients,” Etherington said.