Jazz Pharmaceuticals Announces Positive Top-line Results from Phase 3 Study of JZP-258 in Adult Narcolepsy Patients with Cataplexy and Excessive Daytime Sleepiness - Seite 2

"Narcolepsy is a chronic, debilitating disease and is associated with an increased risk of comorbidities," said Michael Thorpy, M.D., director, Sleep-Wake Disorders Center, Montefiore Health System in Bronx, New York. "An extensive body of evidence has established that excessive consumption of sodium is linked with an increased risk of stroke, cardiovascular disease and other adverse outcomes. Patients with narcolepsy may require lifelong medication, and there is a need for a new, low-sodium oxybate formulation."

About the Phase 3 Study
The Phase 3 study of JZP-258 was a global, double-blind, placebo-controlled, randomized-withdrawal, multicenter study evaluating the efficacy and safety of JZP-258 in the treatment of cataplexy in adult patients with narcolepsy. The primary endpoint was the change in the weekly number of cataplexy attacks and the key secondary endpoint was the change in the ESS score from JZP-258 and placebo over the randomized-withdrawal period. The study enrolled 201 patients and randomized 134 patients, comprising a heterogeneous patient population which included those previously treated with Xyrem, naïve to Xyrem, and with or without other anti-cataplectic treatments. A randomized-withdrawal study design aims to measure efficacy – specifically, maintenance of effect – for patients who remain on active treatment and worsening for patients who switch to placebo.

The study design included a titration period of up to 12 weeks, a JZP-258 stable-dose period of two weeks, followed by a 1:1 randomization to either JZP-258 or placebo for 2 weeks.  After the completion of the double-blind, placebo-controlled treatment period, patients had the opportunity to receive JZP-258 in an optional 24 week open-label safety extension period. More information about the study design is available at www.clinicaltrials.gov (identifier: NCT03030599).

About JZP-258
JZP-258 is an investigational product being evaluated in adult patients for the treatment of cataplexy and excessive daytime sleepiness in narcolepsy, as well as for the treatment of idiopathic hypersomnia. JZP-258 is a novel oxybate product candidate with a unique composition of cations resulting in 92% less sodium than Xyrem (sodium oxybate). The mechanism of action of JZP-258 is not fully understood, but it is hypothesized that the therapeutic effects of JZP-258 on sleep/wake symptoms are mediated through modulation of GABA_B during sleep.