Jazz Pharmaceuticals announced that its JZP-258 investigational medicine has met primary and key secondary endpoints in phase 3 narcolepsy trial.
The phase 3 global, double-blind, placebo-controlled, randomised-withdrawal, multicentre study has assessed the efficacy and safety of JZP-258 in the treatment of cataplexy and excessive daytime sleepiness (EDS) in adults with narcolepsy.
JZP-258, a novel oxybate product candidate with a unique composition of cations, is an investigational product being assessed in adults to treat cataplexy and excessive daytime sleepiness in narcolepsy. It is also being developed to treat idiopathic hypersomnia.
The change in the weekly number of cataplexy attacks is the primary endpoint, while the change in the Epworth Sleepiness Scale (ESS) score with JZP-258 compared to placebo is the major secondary endpoint of the study.
Jazz recruited 201 participants and randomised 134 participants, comprising a heterogeneous population, which included those previously treated with sodium oxybate and naïve to sodium oxybate, with or without other anticataplectic treatments.
The design of the trial included an optimisation and titration period of up to 12 weeks, a JZP-258 stable-dose period of two weeks, followed by 1:1 randomisation to either JZP-258 or placebo for two weeks.
The patients had the chance to secure JZP-258 in an optional 24-week open-label safety extension period, after the completion of the double-blind and placebo-controlled treatment period.
According to the company, the significant increase has been noticed in the median weekly number of cataplexy attacks in participants randomised to placebo compared with participants randomised to JZP-25 during the double-blind withdrawal period.
The significant increase has also been observed in median ESS scores in participants randomised to placebo compared with participants randomised to JZP- 258 at the end of the double-blind withdrawal period.
Jazz Pharmaceuticals sleep and neuroscience senior vice president Dr Jed Black said: “We are pleased with the positive results from the Phase 3 study of JZP-258, which demonstrate the efficacy of JZP-258 for the treatment of cataplexy and excessive daytime sleepiness in narcolepsy.
“These data support the efficacy and overall safety profile of a lower-sodium oxybate formulation for people living with narcolepsy, a chronic condition that may require lifelong therapy.”