Interim Blinded Results Show Promise of Nanocrystalline Gold Treatment For Amyotrophic Lateral Sclerosis

Interim blinded data from an ongoing phase 2 clinical trial (NCT04098406) suggest treatment effect of catalytically active nanocrystalline gold (CNM-Au8; Clene Nanomedicine, Salt Lake City, UT) for amyotrophic lateral sclerosis (ALS).

In this placebo-controlled randomized trial, 45 participants were randomly assigned to receive the nanocrystalline gold solution or placebo in addition to their current standard of care over a 36-week treatment period. Blinded analyses show that the enrolled group had less decline, and some had improvement, on the MUNIX(4) sum, a validated EMG measure muscle function. 

In the overall study population (n=45; randomized 1:1 active therapy 30 mg daily to placebo), 34%, 26%, and 18% of patients who completed weeks 12, 24, and 36, respectively had MUNIX(4) sum improvement from baseline, in contrast to the typical continuous decline of -4.3% per month on MUNIX(4) for people with ALS. At 36 weeks, the mean decline for participants who completed the full 36 weeks (n=35) was -22% vs the expected -38.4% decline over this length of time.

Correlation analyses showed that subjects who demonstrated MUNIX(4)sum improvements also had less decline in ALS Functional Rating Scale-Revised (ALSFRS-R) and forced vital capacity (FVC) scores. The mean reduction in FVC for the overall study population was 10.5% (absolute change of the % predicted) at week 24 (n=42), an approximately 35% improvement from what is typically seen over the same time period.. 

“We are encouraged by this update from our ongoing phase 2 trial of CNM-Au8 for the treatment of ALS,” said Rob Etherington, president and chief executive officer, Clene Nanomedicine, “While blinded, we believe these data support CNM-Au8’s potential to become a breakthrough for this devastating disease. We look forward to delivering unblinded topline results by the end of this year. Concurrent with our advancement of the RESCUE-ALS study, we are also advancing CNM-Au8 through the Phase 3 HEALEY ALS Platform trial (NCT04297683). Through the parallel execution of these clinical programs, we aim to facilitate CNM-Au8’s clinical development and hopefully one day shift the paradigm in how we can treat and target the underlying bioenergetic deficits common to ALS patients.”

The treatment is a stable, aqueous suspension of catalytically active gold nanocrystals thought to act as a bioenergetic nanocatalyst to treat neurodegenerative disease by addressing bioenergetic failure, oxidative stress, and impaired proteostasis. 

These data were presented at the European Network to Cure ALS (ENCALS) 2021 Annual Meeting.