The chief development officer at Clene Nanomedicine provided insight on the mechanistic function of CNM-Au8 and how it improves survival in patients with ALS. [WATCH TIME: 3 minutes]
WATCH TIME: 3 minutes
"CNM-Au8 is able to increase energetic support, acting as a catalyst or intracellular catalyst, supporting energy production in neurons. We’re seeing it across a variety of disease states and a variety of preclinical and clinical data sets that CNM-Au8 appears to help neuronal health and neuronal survival."
Treatment options remain among the top priority for patients and caregivers of amyotrophic lateral sclerosis (ALS), as only 2 drugs—edaravone (Radicava; Mitsubishi Tanabe Pharma) and riluzole (Rilutek; Sanofi)—have been approved to slow disease progression and improve survival. CNM-Au8 (Clene Nanomedicine), a suspension of clean-surfaced, catalytically active gold nanocrystals, presents as a new potential therapeutic, as it has shown to enhance neuronal metabolic energy, reduce oxidative stress, and improve protein homeostasis.
The ALS agent was most recently evaluated in a pivotal 45-patient, phase 2 study called RESCUE-ALS (NCT04098406). Here, patients were treated with either 30 mg of CNM-Au8 or placebo for 36 weeks, followed by an open-label extension, with change in the summated motor unit index scores as the primary end point. Although it did not reach statistical significance, a new analysis presented at the 2022 American Academy of Neuromuscular and Electrodiagnostic Medicine (AANEM) annual meeting, September 21-24, in Nashville, Tennessee, highlighted the significant impact the therapeutic has on survival.
To learn more about the results, NeurologyLive® sat down with Michael Hotchkin, chief development officer, Clene Nanomedicine. Hotchkin provided insight on the highlights the clinical community should be aware of, including the 70% decreased risk of mortality, as well as why the agent’s mechanism of action poses as a promising approach to treating ALS.