RESCUE-ALS Phase 2 Trial Completes Enrollment Ahead of Schedule

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by Forest Ray PhD |

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Patient enrollment is now complete for the Phase 2 clinical trial of Clene Nanomedicine‘s CNM-Au8 as a potentially disease-modifying therapy for amyotrophic lateral sclerosis (ALS).

The trial, named RESCUE-ALS (NCT04098406) will test the safety, efficacy, pharmacokinetics, and pharmacodynamics — how the compound moves through the body and its effects, respectively — of CNM-Au8 in early symptomatic ALS patients.

The trial’s first participant was dosed in January and more than half the participants had been enrolled by late May.

“Completion of enrollment is ahead of schedule, clearly demonstrating the unmet medical need in this devastating disease,” said Robert Glanzman, MD, chief medical officer of Clene, in a press release.

The study’s primary goal is to show that CNM-Au8 improves muscle function, as compared to a placebo.

Improvements will be assessed using a technique called Motor Unit Number Index (MUNIX), which quantitatively measures the loss or preservation of motor neurons in ALS. This test will be performed at the beginning and end of the trial’s 36-week treatment period.

MUNIX is an electromyographic technique, meaning that it measures electrical activity in response to a nerve’s stimulation of a muscle and is an established way of measuring motor neuron loss in ALS.

“In multiple preclinical models of ALS, CNM-Au8 has been shown to promote motor neuron health, reduce neuron loss, and preserve motor neuron function. We believe the unique mechanisms of CNM-Au8 provide the potential to be an effective disease-modifying therapy for patients with ALS,” Glanzman said.

CNM-Au8 is a liquid suspension of gold nanocrystals that are thought to support biological reactions within cells, namely those that generate energy and those that remove toxic byproducts of cellular metabolism that lead to motor neuron death in ALS.

Phase 1 studies in healthy volunteers (NCT02755870) have shown the compound’s safety in humans.

RESCUE-ALS receives partial funding from FightMND, an Australian nonprofit patient advocacy group working to raise awareness of motor neuron disease in that country.

“Completing enrollment is a major milestone in our Phase 2 RESCUE-ALS trial. We are looking forward to unveiling the data expected in the summer of 2021. This key milestone could provide proof-of-concept for a novel nanotherapeutic agent for the treatment of ALS, and represents important advances for Clene,” said Rob Etherington, president and CEO of Clene.