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- Data Demonstrated Significant and Clinically Meaningful Improvements with the Potential to Redefine Narcolepsy Type 1 (NT1) Care Beyond Individual Symptoms
- Oveporexton was Generally Well-Tolerated with Safety Profile Consistent with Previous Clinical Studies
- Oveporexton is the First and Only Approved Orexin Agonist Designed to Treat the Underlying Cause of NT1 and is Now Approved in the U.S., China and Japan, with Additional Regulatory Submissions Underway
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OSAKA, Japan & CAMBRIDGE, Mass. — Takeda (TSE:4502/NYSE:TAK) announced that the New England Journal of Medicine published results from two Phase 3 studies evaluating oveporexton (ORZEYFUL), an oral orexin receptor 2 (OX2R) agonist, in people with narcolepsy type 1 (NT1).1 Oveporexton is the first and only medicine to treat the underlying cause of NT1.
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“People living with narcolepsy type 1 face persistent symptoms across the day and night, which can impact many aspects of daily life,” said Emmanuel Mignot, M.D., Ph.D., principal investigator for the FirstLight (TAK-861-3001) Phase 3 study. “The newly published Phase 3 data reinforce the potential of oveporexton to transform how we approach narcolepsy type 1 in clinical practice, shifting from managing individual symptoms to treating the disease itself.”
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NT1 is a chronic, rare neurological disease driven by orexin deficiency. As a result, people experience a range of daytime and nighttime symptoms including excessive daytime sleepiness, cataplexy (sudden loss of muscle tone), disrupted nighttime sleep, sleep paralysis, hallucinations and cognitive symptoms. The persistent, 24 hour-nature of the disease can severely impact many aspects of a person’s life, including work, education and social interactions.
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“Leveraging the extensive research we conducted on the impact of narcolepsy type 1, we designed our comprehensive Phase 3 program to reflect the complexity of the disease and the experiences of those living with it,” said Sarah Sheikh, M.Sc., B.M., B.Ch., MRCP, Head, Neuroscience Therapeutic Area Unit and Global Development at Takeda. “The magnitude of effect seen across the full range of symptoms evaluated reinforces the potential of oveporexton to redefine how people feel on treatment. We are grateful to the patients, caregivers and healthcare providers who have participated in our studies and are thrilled to bring the first orexin agonist to the community.”
Article contentFirstLight and Radiant Light Phase 3 Study DesignsArticle content
The FirstLight (TAK-861-3001) study enrolled 168 participants randomized to one of three dosing arms (twice-daily 2mg, twice-daily 1mg and placebo). The RadiantLight (TAK-861-3002) study enrolled 105 participants randomized to two dosing arms (twice-daily 2mg and placebo). The 14 primary and secondary endpoints from the studies assessed the effect of oveporexton on the broad disease impact compared to placebo over 12 weeks. More than 95 percent of the participants who completed the studies enrolled in the ongoing long-term extension (LTE) study.
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The data published in the New England Journal of Medicine included primary and key secondary efficacy results along with the safety and tolerability of oveporexton. Results from additional secondary and exploratory endpoints assessing quality of life measures and disease severity were also published.
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Article contentEfficacy ResultsArticle content
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- The two Phase 3 studies demonstrated consistent, clinically meaningful and statistically significant improvements compared to placebo in wakefulness, sleepiness, cataplexy, disease severity and quality of life measures (p-values of <0.001) across doses over 12 weeks. These improvements were observed at the earliest assessed timepoint for each measure and were sustained throughout the duration of the studies.
- The median percent reductions in weekly cataplexy rate ranged from 79.0% to 88.8% with oveporexton versus 27.7% to 39.1% with placebo at week 12.
- Oveporexton improved disease severity across all domains of the narcolepsy severity scale (NSS-CT) including EDS, cataplexy, hypnagogic hallucinations and sleep paralysis across both doses, and in the disrupted nighttime sleep domain with oveporexton 2mg. More than 70% of treated participants reported the lowest severity level on the NSS-CT (mild; score 0-14) across doses.
- Nearly all treated participants (97%) reported improvements in overall narcolepsy symptoms as assessed by the self-rated Patient Global Impression of Change (PGI-C) scale.
- All dose groups achieved normative thresholds for wakefulness on the Maintenance of Wakefulness Test (MWT) (≥20 minutes) and Epworth Sleepiness Scale (ESS) (score of ≤10). Most participants reached or exceeded normal thresholds for quality of life outcomes as measured by the 36-Item Short Form Survey (SF-36; secondary endpoint) and EuroQol-5 Dimensions 5-Levels (EQ-5D-5L; exploratory endpoint).2.3
Article contentSafety ResultsArticle content
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- Consistent across clinical studies to date, the most commonly reported treatment-emergent adverse events (TEAEs) across both studies were transient insomnia, urinary urgency, urinary frequency and excessive saliva.
- Most TEAEs were mild to moderate in intensity, started within two days of treatment and did not require medical intervention.
- Most insomnia events resolved within one week and did not impair or impact daytime functioning, unlike traditional insomnia symptoms. Approximately half of urinary events resolved by week 12 and unresolved events were all mild or moderate in severity.
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Oveporexton is the first and only orexin agonist approved in the United States, Japan and China to treat the disease holistically rather than individual symptoms. With additional regulatory submissions underway, Takeda continues to work with health authorities to bring oveporexton to more people living with NT1.