CNM-Au8 Displays Evidence of Survival Benefit in Amyotrophic Lateral Sclerosis


Results from phase 2 RESCUE-ALS trial show that treatment with CNM-Au8 in ALS demonstrated improvement in survival compared to the estimated median survival rate.

Robert Glanzman, MD, FAAN, chief medical officer, Clene Nanomedicine

Robert Glanzman, MD, FAAN

New data from the phase 2 RESCUE-ALS trial (NCT04098406) of CNM-Au8, an investigational treatment for amyotrophic lateral sclerosis (ALS) from Clene Nanomedicine, suggest that the therapy significantly improved survival among patients with ALS.1

Presented in a poster at the 2022 American Association of Neuromuscular & Electrodiagnostic Medicine Annual Meeting, held September 21-24, in Nashville, Tennessee, the findings showed that the CNM-Au8 treatment significantly improved survival benefit with, a hazard ratio of 0.31 (95%CI, 0.1328-0.7440; P = .011).

“These clinical and survival data from RESCUE-ALS contribute to the growing body of evidence supporting the potential for CNM-Au8 as a disease-modifying therapy for ALS,” Robert Glanzman, MD, FAAN, chief medical officer, Clene Nanomedicine, said in a statement.2

CNM-Au8 treated participants (n = 23) in phase 2 RESCUE-ALS trial completed the trial with 1 mortality event in the active-treatment group during the 36-week double-blind period.1 Among participants that were placebo treated (n = 22), 86% completed with 2 mortality events and 1 withdrew due to worsening of ALS disease, 14% death or withdrawal. The CNM-Au8 treatment was used in this study as it has been known to enhance neuronal metabolic energy, reduce oxidative stress, and improve protein homeostasis.1

The RESCUE-ALS trial was a phase 2 randomized, placebo-controlled, and double-blinded study of CNM-Au8 treatment in persons with early sporadic ALS. The trial had paired the main study with an ongoing process of an open-label extension in order to evaluate CNM-Au8 for its efficacy and long-term safety. Participants (n = 45) from RESCUE-ALS were randomly assigned using a ratio of 1:1 and then received 30 mg of CNM-Au8 or the placebo every day over the course of 36 weeks for the double-blind period of the trial. Following the double-blinded portion, an open-label extension was used with the CNM-Au8 treatment with the participants, 30 mg per day. The open-label extension participants were observed in a long-term survival analysis compared to the predicted median survival based acquired from the published ENCALS model.

Of the number of eligible participants during the main trial, 90% (n=36) entered the open-label extension. During the open-label extension, CNM-Au8 was well-tolerated, and no significant safety findings were reported. The data was censored either through February 1, 2022, or the participant’s last study contact (n = 1).

In a previous NeurologyLive® conversation with Matthew Kiernan, MBBS, PhD, DSc, FRACP, FAHMS, Bushell Chair of Neurology, and professor of medicine, Central Clinical School, about the RESCUE-ALS trial, he discussed positive interim data he presented earlier in 2022 at the American Academy of Neurology annual meeting.3

“Following the 36 weeks of placebo versus active compound, we put everyone on the active compound, and we've been following them up. We did the census of the data in March, and it really is quite dramatic, showing that the survival in patients is improved on nanocrystalline gold, CNM-Au8. And that was the case whether the patients were on the active part in the trial, or indeed on the placebo and then switched to the active part,” Kiernan told NeurologyLive® in April 2022. He added that the data showed not only improvement in quality of life but importantly, an improvement in survival. “There's less events for patients, less complications, more survival, and less deaths on the active compound, nanocrystalline gold. Really, this is quite a dramatic finding in ALS patients,” Kiernan said.

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1. Vucic S, Menon P, Huynh W, et al. Evidence for a Potential Survival Benefit in Amyotrophic Lateral Sclerosis with CNM-Au8 Treatment: Interim Results From the RESCUE-ALS Trial Long-term Open Label Extension. Presented at: AANEM 2022; September 21-24; Nashville, TN. Abstract 206.
2. Clene Nanomedicine Presents Updated Long-Term Survival Data from RESCUE-ALS Participants at 2022 AANEM Scientific Conference. News release. Clene Nanomedicine. September 21, 2022. Accessed September 22, 2022.
3. Updated Interim Data from Clene Nanomedicine’s RESCUE-ALS Open-Label Extension Study to be Presented in Emerging Science Program at 2022 AAN Annual Meeting. News release. Clene Nanomedicine. April 1, 2022. Accessed September 21, 2022.

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