Bushell Chair of Neurology; Professor of Medicine, Central Clinical School; Codirector, Discovery and Translation, Brain and Mind Centre; NHMRC Practitioner Fellow, University of Sydney; Neurologist, Institute of Clinical Neurosciences, Royal Prince Alfred Hospital

Matthew Kiernan, MBBS, PhD, DSc, FRACP, FAHMS

“It is a paradigm shift. A lot of this has come through understanding the disease itself. TDP43 and the connections to ALS was a relatively recent discovery in the last decade. Then, there’s been a lot of interest in ALS with the Ice Bucket Challenge and there’s been a lot of support from industry and philanthropy, and that’s driven the science through the roof. As a result, now we have a lot of compounds coming through.”

For neuromuscular disorder specialists such as Matthew Kiernan, MBBS, PhD, DSc, FRACP, FAHMS, there has been a paradigm shift in clinical intervention. One such example is Clene Nanomedicine’s investigational therapy for amyotrophic lateral sclerosis (ALS), CNM-Au8, which is an oral suspension of gold nanocrystals. In recently announced phase 2 data, the therapy showed promising results, demonstrating a 70% survival benefit among patients with ALS who entered the open-label extension of the trial (n = 36; 90% of eligible patients).

Data from that study, dubbed RESCUE-ALS (NCT04098406), were presented at the 

2022 American Academy of Neurology (AAN) Annual Meeting

, April 2-7, in Seattle, Washington, by Kiernan, who 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. The interim analysis of observed survival compared with estimated median survival derived from the validated European Network for the Cure of ALS prediction model, showed a significant signal for 

Kiernan, an investigator in the trial, sat down with 

 to discuss the newly announced interim results being presented at the AAN meeting, offering his thoughts on the clinical takeaways from RESCUE-ALS. Additionally, he spoke to the larger progress that therapies such as CNM-Au8 represent for the field of neuromuscular medicine, suggesting that a shift in thinking has occurred, noting that the insights gained in the last decade have vaulted the field into a new era of therapy.

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.
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.

Videos

The Bushell Chair of Neurology at the University of Sydney offered his perspective on phase 2 data of CNM-Au8 in amyotrophic lateral sclerosis and spoke to the advances that the field has made in recent years. [WATCH TIME: 3 minutes]

The Paradigm Shift in Therapeutic Intervention for ALS: Matthew Kiernan, MBBS, PhD, DSc, FRACP, FAHMS

“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.”

, 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).

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 

= .006, log-rank test) for those who entered the open-label extension. Kiernan, an investigator in the trial, sat down with 

® to discuss the newly announced interim results being presented at the AAN meeting.

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.

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.

The Bushell Chair of Neurology at the University of Sydney shared his insight into the latest data on CNM-Au8 as a potential treatment for amyotrophic lateral sclerosis and the frameshift in treatment that it represents. [WATCH TIME: 3 minutes]

Matthew Kiernan, MBBS, PhD, DSc, FRACP, FAHMS

Articles

The Paradigm Shift in Therapeutic Intervention for ALS: Matthew Kiernan, MBBS, PhD, DSc, FRACP, FAHMS

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

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