Results of Clinical Trials for CNM-Au8, A Potential ALS Treatment: James Berry, MD, MPH

WATCH TIME: 5 minutes

"The main takeaway from that study was really that there was actually a difference in survival and those who started the drug early were randomized to the drug rather than placebo, although everybody went on to have drug during an open label phase of that trial."

CNM-Au8, an oral suspension of gold nanocrystals, was developed to restore neuronal health and function by increasing energy production and utilization. Although the treatment is shown in models to improve neuronal metabolic energy, it did not show benefit on primary or secondary endpoints in 2 trials, RESCUE-ALS and the HEALEY ALS Platform trial (NCT04297683; Regimen C). James Berry, MD, MPH, director of the Massachusetts General Hospital ALS Care Center, presented these findings of the survival analyses as an abstract at the 2023 American Academy of Neurology (AAN) Annual Meeting, April 22-27, in Boston, Massachusetts.

In RESCUE-ALS, analyses of all-cause mortality showed a significant survival benefit with CNM-Au8, resulting in more than a 70% decrease in risk of death (5 vs. 14 events, log-rank HR = 0.291; 95% CI, 0.118-0.718, P = .0115). In HEALEY-ALS, exploratory analysis demonstrated more than a 90% reduction in risk of death alone or death/permanently assisted ventilation (PAV) at week 24 for the 30mg dose, but not 60mg when adjusted for baseline imbalances (PAV and death, 4 vs. 1, P = .028, death only, 3 vs. 1, P = .057; unadjusted for multiple comparisons).¹

Berry sat down with NeurologyLive^® in an interview at AAN 2023 to provide an overview of the analyses and explain the significance of the findings for patients with ALS. He also talked about the differences in treatment responses observed in terms of the safety profile with the treatment. Additionally, he spoke about the next steps in research with CNM-Au8 including what should be studied further in future trials.

Click here for more coverage from AAN 2023.

REFERENCES
1. Berry J, Maragakis N, Paganoni S, et al. Evidence for Survival Benefit in ALS with CNM-Au8 Treatment Across Three Study Populations. Presented at: 2023 AAN Annual Meeting; April 22-27, Boston, Massachusetts.