Unique Composition of CNM-Au8 to Treat MS and ALS: Michael Hotchkin

WATCH TIME: 2 minutes

"It is fascinating how many different neurodegenerative disorders share a commonality of energetic dysfunction. We do have preclinical models suggesting efficacy across a variety of different neurodegenerative diseases: MS, ALS, Alzheimer disease, and multiple other models."

CNM-Au8, an investigational agent consisting of a gold nanocrystal suspension developed by Clene Nanomedicine, poses as a unique candidate in the pipeline for neurological disorders. Currently, the therapeutic is being evaluated in patients with multiple sclerosis (MS) and those with amyotrophic lateral sclerosis (ALS), a neuromuscular disease for which there are only a handful of available treatments.

In August, the company announced positive topline results from its VISIONARY-MS study (NCT03536559), as the agent met both its primary and secondary end points without any significant safety signals found. All told, on Low Contrast Letter Acuity (LCLA) measures—the primary end point—the least squares mean difference was 3.13 (95% CI, –0.08 to 6.33; P = .056) compared with the placebo group in the clinically affected eye over 48 weeks of treatment.¹

In the company’s study in ALS, RESCUE-ALS (NCT04098406), despite not meeting its primary or secondary end points in change of Motor Unit Number Index, treatment with the agent resulted in a significant 70% decreased risk of death compared with those initial randomized to placebo.2 Those findings were presented at the 2022 American Association of Neuromuscular and Electrodiagnostic Medicine (AANEM) annual meeting, September 21-24, in Nashville, Tennessee.

To learn more about how this candidate successfully treats both neurologic conditions, NeurologyLive^® sat down with Michael Hotchkin, chief development officer, Clene Nanomedicine. Hotchkin provided insight on the crossover between these diseases, as well as whether there could be plans to expand CNM-Au8’s range into other disorders.

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REFERENCES
1. Clene Reports Positive Topline Results for CNM-Au8® in the Phase 2 VISIONARY-MS Trial in Multiple Sclerosis. News release. Clene Nanomedicine. August 15, 2022. Accessed September 23, 2022. https://www.globenewswire.com/news-release/2022/08/15/2498070/0/en/Clene-Reports-Positive-Topline-Results-for-CNM-Au8-in-the-Phase-2-VISIONARY-MS-Trial-in-Multiple-Sclerosis.html
2. 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.