CNM-Au8 Data From the RESCUE-ALS Open-Label Extension: Matthew Kiernan, MBBS, PhD, DSc, FRACP, FAHMS

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“Importantly, though, linked to improved survival [was that] patients reported improved quality of life, and that was quite dramatic. There are less events per patient, less complications, more survival, and less deaths on the active compound, nanocrystalline gold. Really, this is quite a dramatic finding in ALS patients.”

At the 2022 American Academy of Neurology (AAN) Annual Meeting, April 2-7, in Seattle, Washington, data from the open-label extension of the phase 2 RESCUE-ALS trial (NCT04098406) of Clene Nanomedicine’s investigational agent, CNM-Au8, are being presented by Matthew Kiernan, MBBS, PhD, DSc, FRACP, FAHMS. Announced on April 1, the data demonstrate a 70% survival benefit among patients with amyotrophic lateral sclerosis (ALS) who entered the open-label extension (n = 36; 90% of eligible patients).¹

This updated interim analysis of observed survival compared to estimated median survival derived from the validated European Network for the Cure of ALS, or ENCALS, prediction model significantly favored CNM-Au8 treatment with an HR of 0.3 (P = .006, log-rank test) for those who entered the open-label extension. Kiernan, an investigator in the trial, sat down with NeurologyLive® to discuss the newly announced interim results being presented at the AAN meeting.^1,2

Kiernan is the Bushell Chair of Neurology and professor of medicine at the Central Clinical School, as well as the codirector of Discovery and Translation at the Brain and Mind Centre of the University of Sydney and a neurologist in the Institute of Clinical Neurosciences at Royal Prince Alfred Hospital. He shared his perspective on the data and offered up some of his experience with CNM-Au8 in its development for ALS, noting that these results potentially mark the opening of a new era of medicine in ALS.

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REFERENCES
1. 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 April 1, 2022. https://www.globenewswire.com/news-release/2022/04/01/2414686/0/en/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.html
2. Kiernan MC. Long-Term Survival of Randomized Participants Enrolled in the Phase 2 RESCUE-ALS Trial of CNM-Au8, A Cellular Energetic Catalyst. Presented at: AAN Annual Meeting; April 2-7, 2022; Abstract 007.