WVE-N531's Impact on Exon Skipping, PROs Improved With Early DMT Initiation, Carbon Dioxide Improves Insomnia Sleep

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Welcome to this special edition of Neurology News Network. I’m Marco Meglio.

Wave Life Sciences has announced positive findings from its phase 1/2a proof-of-concept study, which showed that its investigational agent WVE-N531 led to significant concentrations of exon skipping in ambulatory boys with Duchenne muscular dystrophy (DMD) after just 3 weeks of biweekly multidosing at 10 mg/kg.WVE-N531, the first exon-skipping candidate from Wave to use its next-generation PN chemistry, resulted in a mean tissue concentration of 42 micrograms/gram, as well as resulted in mean exon skipping of 53% as measured by RT-PCR. The therapeutic was also safe and well-tolerated, as all adverse events, except for a COVID-19 infection of moderate intensity, were all mild. The analysis featured 3 ambulatory boys who were on escalating doses of 1,3,6, and 10 mg/kg in the multidose portion of the trial, followed by 3 doses of 10 mg/kg every other week. With the first patients dosed in October 2021, the trial is expected to include approximately 15 total individuals who have a documented mutation of the DMD gene that is amenable to exon 53 skipping intervention.

Findings from a nationwide observational cohort study of adults with relapsing multiple sclerosis (MS) who commenced disease-modifying treatment (DMT) within 4 years of disease onset showed that earlier commencement was associated with better patient-reported physical symptoms; however, general quality of life was unaffected. Investigators concluded that this indicates other factors may inform patients’ quality of life. From 2001 to 2016, investigators identified 2648 patients with relapsing MS who were categorized into early-treated (DMT initiation within 2 years of onset) and late-treated (DMT initiation 2-4 years after onset) groups. Outcomes were measured in the 4-to-10-year period from disease onset, and included the MSIS-29, a disease-specific questionnaire used to report physical and psychological symptoms of MS, and the EQ-5D-3L, a generic, disease-invariant measure of health-related quality of life consisting of 5 domains.

In a recent double-blind, randomized, sham-controlled cross-over study, results showed that adults with insomnia increased time spent asleep when exposed to a low concentration of carbon dioxide gas (CO2) with a sleep air device (Gosleep, NYX). This research report provides an opportunity for spreading more awareness on the importance of sleep health with respect to having more sleep-related products available. Adults who were exposed to the CO2 gas had spent a longer time in bed and longer total sleep time in comparison with the group exposed to room air. Additionally, both indices of total arousal and non-REM arousal were observably lower in the group exposed to CO2 gas in comparison with room air exposed group. Following the day after the sham treatment polysomnography, the average subjective sleep onset latency (SOL) observed was 42 minutes.

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