ALS Agent CNM-Au8 Reduces NfL, Positive CLEAR-MIND Results, Decreased Learning Abilities in Narcolepsy

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Neurology News Network for the week ending January 6, 2023. [WATCH TIME: 3 minutes]

WATCH TIME: 3 minutes

Clene Nanomedicine recently announced positive data from the open-label extension phase of the HEALEY-ALS trial, with results showing that treatment with investigational CNM-Au8 resulted in reduced plasma neurofilament light levels and improved survival among patients with ALS. In the trial, 161 patients with ALS were randomly assigned to 30 mg CNM-Au8, 60 mg CNM-Au8, or placebo as adjunct to standard of care for a 24-week treatment period. Following the double-blind period, all patients were given the choice to enter the OLE. After 76 weeks of treatment, investigators observed a 16% reduction in NfL in those randomized to the 30 mg active group vs those initially randomized to placebo. For more context, over the original 24-week period, investigators observed a 10% relative reduction in this biomarker.

Longeveron recently announced positive topline data from its phase 2a CLEAR MIND study of Lomecel-B, with results showing that the agent met its primary end point of safety, with slowing of disease worsening in patients with mild Alzheimer disease. The company is planning to report additional exploratory end points of brain volumetry by MRI, biomarkers relevant to inflammation and endothelial/vascular system, and measures of cognitive function. At the conclusion of the 39-week treatment period, the agent met its primary end point of safety, with no new concerns. Investigators reported no incidences of hypersensitivity, no cases of amyloid-related imaging abnormalities (ARIA), no clinically asymptomatic microhemorrhages on MRI, and no notable changes in laboratory evaluations and electrocardiogram.

In a newly published study in Sleep Medicine, patients with narcolepsy type 1 (NT1) and other central disorders of hypersomnolence (CDH) showed a decrease in learning abilities when receiving negative feedback compared with positive feedback in a reinforcement learning task. These findings suggest observed alterations in decision-making abilities may be associated with vigilance disruptions and emphasize the importance of restoring adequate vigilance to potentially minimize cognitive impairments in patients with CDH. Authors noted that this pattern was not suggestive of dopamine deficiency, however, both participants with NT1 and other CDH had decreased learning abilities to avoid losses in the reinforcement learning task.

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