Oveporexton Boosts Wakefulness and Cuts Cataplexy in Narcolepsy Type 1, Phase 2 Trial Finds

The primary outcome was the change in mean sleep latency, measured by the Maintenance of Wakefulness Test (MWT), from baseline to week 8. This test assesses the ability to stay awake during the day, with higher scores indicating better wakefulness.

The key findings of the study were as follows:

• Patients receiving oveporexton showed significant improvements in sleep latency, with increases ranging from 12.5 to 25.4 minutes, while the placebo group saw a decrease of 1.2 minutes.
• All dosing groups of oveporexton demonstrated statistically significant benefits in sleep latency compared to placebo.
• The Epworth Sleepiness Scale scores improved by 8.9 to 13.8 points across oveporexton groups, whereas the placebo group showed a 2.5-point reduction.
• Participants on 2 mg twice daily or 2 mg escalating to 5 mg daily regimens reported significantly fewer cataplexy episodes than those on placebo.
• Oveporexton was generally well tolerated among participants.
• Common adverse effects included insomnia (48%), urinary urgency (33%), and urinary frequency (32%).
• Most insomnia cases were resolved within one week.
• No hepatotoxic effects were observed during the trial.

"These findings emphasize the potential of oveporexton as a targeted therapy for narcolepsy type 1 by directly addressing orexin deficiency. Over the eight-week trial period, the drug improved wakefulness, reduced sleepiness, and lowered the frequency of cataplexy attacks," the researchers concluded. "Further studies are expected to evaluate long-term efficacy and safety."

Reference:

DOI: 10.1056/NEJMoa2405847