Takeda has announced positive data from two phase 3 clinical trials of oveporexton in patients with narcolepsy type 1 (NT1).
NT1 is a rare, chronic neurological disorder characterized by excessive daytime sleepiness (EDS) and cataplexy. It is caused by reduced levels of orexin (aka hypocretin) in the brain.
Oveporexton (TAK-861) is an investigational first-in-class orexin receptor 2 (OX2R)-selective agonist that is designed to activate OX2R, restore signaling, and address orexin deficiency. FirstLight (NCT06470828) and RadiantLight (NCT06505031) are phase 3, global, multicenter, placebo-controlled studies evaluating the efficacy, safety and tolerability of oveporexton compared to placebo in patients with NT1 over 12 weeks.
The primary endpoint of both studies was improvement in EDS as measured by the Maintenance of Wakefulness Test. Key secondary endpoints included improvement in EDS measured by the Epworth Sleepiness Scale and in Weekly Cataplexy Rate. The FirstLight study enrolled 168 participants and the RadiantLight study enrolled 105 participants.
Statistically significant and clinically meaningful improvement compared to placebo was observed for all primary and secondary endpoints across all doses at week 12 in both studies. This included improvements in wakefulness, EDS, cataplexy, ability to maintain attention, overall quality of life, and daily life functions. Additionally, oveporexton was generally well-tolerated and no serious treatment-related adverse events were reported. The most common adverse events were insomnia and urinary urgency and frequency.
More than 95% of study participants have enrolled in the ongoing long-term extension study. Takeda has plans to present the results at upcoming medical congresses and is working to submit a New Drug Application to the U.S. Food and Drug Administration (FDA) and other global regulatory bodies in 2025.
To learn more about narcolepsy and other rare neurological conditions, visit https://checkrare.com/diseases/neurology-nervous-system-diseases/