Jazz hits a high note with positive narcolepsy results
- In a potential boost for patients with narcolepsy, Jazz Pharmaceuticals has announced that its selective dopamine and norepinephrine reuptake inhibitor (DNRI), JZP-110, met the co-primary endpoints in the TONES 2 study in patients with narcolepsy at the two highest doses. The open-label long-term maintenance efficacy portion of the study is ongoing, and Jazz plans to submit a New Drug Application in late 2017.
- Patients treated with 300mg or 150mg JZP-110 were significantly more likely to stay awake (maintenance of wakefulness test) and report lower levels of sleepiness (Epworth sleepiness scale) than placebo, throughout the 12-week study. The lower 75mg dose statistically improved wakefulness but not sleepiness.
- There were two serious adverse events in the study but these were not deemed to be drug related. The most common side effects were headache, nausea, decreased appetite, nasopharyngitis, dry mouth and anxiety.
Things are waking up for Jazz Pharmaceuticals' sleep disorder franchise, with the second piece of JZP-110 news this month. Earlier in March the company reported data from its TONES 3 and TONES 4 Phase 3 studies in excessive sleepiness associated with obstructive sleep apnea (OSA). These showed that the drug significantly improved both primary endpoints, the ability to stay awake and the levels of sleepiness, and this could be maintained for 12 weeks, at doses of 75mg and above.
An NDA in both excessive sleepiness in obstructive sleep apnea and narcolepsy is planned for late-2017 for JZP-110. Jazz is also conducting a Phase 2 study for the drug in excessive sleepiness in Parkinson's disease, which will begin treating patients this quarter, and markets Xyrem (sodium oxybate) for narcolepsy.
"I believe this is an important growth driver for Jazz and fits in nicely with the Xyrem franchise. In fact, JZP-110 may be more important than [the company's] acute myeloid leukemia program, Vyxeos," said Umer Raffat, analyst at Evercore ISI, in a note to clients. Raffat predicts peak sales of around $500 million for narcolepsy and obstructive sleep apnea.
Narcolepsy affects the brain's ability to regulate its sleep-wake cycle, and occurs in around one in 2,000 people in the U.S. About half of the people with narcolepsy are undiagnosed, and diagnosis can take as long as ten years. People with narcolepsy can lapse into drowsiness or sleep during the day, and may also experience cataplexy, affecting education, work and leisure, including the ability to drive.
"Excessive sleepiness occurs in all patients with narcolepsy and when combined with frequent irresistible sleep attacks, can lead to a considerable burden of illness and adverse effect on patients' health," said Michael Thorpy, Director of the Sleep-Wake Disorders Center at the Montefiore Medical Center, Bronx, New York.
- Jazz Pharmaceuticals Statement
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