Zevra Therapeutics Announces Top-Line Data from the Phase 2 Clinical Trial of KP1077 for Idiopathic Hypersomnia - Seite 2

The positive top-line results of the Phase 2 trial support the safety and tolerability of KP1077 as measured by the primary endpoint of the study. The study also successfully fulfilled the objectives of providing key information for the design of a potentially pivotal efficacy trial, and the results of the secondary efficacy endpoints were supportive of initiating a Phase 3 trial of KP1077.

KP1077 was shown to be well-tolerated at all dose levels evaluated in the trial, including the highest dose of 320 mg daily, regardless of dosing regimen (once or twice daily). The most common adverse events were insomnia, headache, anxiety, nausea, and decreased appetite. Due to its unique pharmacokinetic profile, adverse events were mostly mild in severity despite higher overall exposure levels when compared to both immediate and long-acting methylphenidate products currently used off-label for the treatment of IH.

Topline results of the Phase 2 study include:

• KP1077 produced clinically meaningful improvement in excessive daytime sleepiness (EDS), as assessed by change from baseline in the Epworth Sleepiness Scale (ESS), that was maintained during both the five-week open-label titration period and throughout the 2-week double-blind withdrawal period for both dosing regimens.
  
• Patients administered KP1077 showed benefits in change from baseline for the IH Severity Scale (IHSS), Sleep Inertia Visual Analog Scale (SIVAS) and Brain Fog severity Scale (BFS) at the end of the open-label dose titration, and at the end of the double-blind withdrawal period.
  
• The results from the completed Phase 2 trial provide key information for the design of a potentially pivotal Phase 3 trial of KP1077 in patients with IH.
  

The Company plans to request an end-of-Phase 2 (EOP2) meeting with the U.S. Food and Drug Administration to seek guidance on the Phase 3 clinical trial design.

About the KP1077 Phase 2 Trial

The Phase 2 clinical trial was a double-blind, placebo-controlled, randomized-withdrawal, dose-optimizing, multi-center study that evaluated the safety and efficacy of KP1077 for the treatment of IH. Part 1 of the trial consisted of a five-week open-label dose titration phase during which patients were optimized to one of four doses of KP1077 (80, 160, 240, or 320 mg/day). Part 2 of the trial entailed a two-week randomized, double-blind, withdrawal phase, during which two-thirds of the trial participants continued to receive their optimized dose while the remaining one-third received placebo. Participants were assigned into two evenly divided cohorts. The first cohort received a single daily dose just before bedtime, and the second cohort received half the daily dose shortly after awakening and the second half prior to bedtime. Zevra enrolled 66 adult patients with IH in 24 centers in the U.S into the open-label titration phase of the study and 50 of those patients continued into the double-blind phase.