Press release
KemPharm Announces Initiation of Phase 2 Clinical Trial Investigating KP1077 for the Treatment of Idiopathic Hypersomnia (IH)
Trial designed as a multi-center, dose-optimizing, double-blind, placebo-controlled, randomized-withdrawal study to evaluate safety and efficacy of KP1077, as
About this update from Zevra Therapeutics, Inc.
[{"type":"text","content":"Trial designed as a multi-center, dose-optimizing, double-blind, placebo-controlled, randomized-withdrawal study to evaluate safety and efficacy of KP1077, as well as to assess the symptoms and severity of “brain fog” Interim efficacy and safety data expected as early as Q3 2023 CELEBRATION, Fla., Dec. 21, 2022 (GLOBE NEWSWIRE) -- KemPharm, Inc. (NasdaqGS: KMPH) (KemPharm, or the Company), a rare disease therapeutics company focused on the development of treatments for rare CNS, neurodegenerative diseases, lysosomal storage disorders and related treatment areas, today announced the initiation of a Phase 2 clinical trial evaluating KP1077 as a treatment for idiopathic hypersomnia (IH), a rare neurological sleep disorder. KP1077 is comprised solely of serdexmethylphenidate (SDX), KemPharm’s proprietary prodrug of d-methylphenidate. SDX was recently granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for the treatment of IH. The Phase 2 clinical trial is a double-blind, placebo-controlled, randomized-withdrawal, dose-optimizing, multi-center study evaluating the efficacy and safety of KP1077 for the treatment of IH. KemPharm expects to enroll approximately 48 adult patients with IH in more than 30 centers in the United States. Part 1 of the trial will consist of a five-week open-label titration phase during which patients will be optimized to one of four doses of SDX (80, 160, 240, or 320 mg/day). Part 2 of the trial will entail a two-week randomized, double-blind, withdrawal phase, during which two-thirds of the trial participants will continue to receive their optimized dose while the remaining one-third will receive placebo. Participants will be further assigned into two evenly divided cohorts. The first cohort will receive a single daily dose just before bedtime, and the second cohort will receive half the daily dose shortly after awakening and half the daily dose prior to bedtime. The primary endpoint is the safety and tolerability of SDX. The major secondary efficacy endpoint is the change in Epworth Sleepiness Scale (ESS) total score. Additional exploratory endpoints include the Patient Global Impression of Severity (PGI-S), the Clinical Global Impression of Severity (CGI-S), change in total score on the Idiopathic Hypersomnia Severity Scale (IHSS), and a new scale to assess the symptoms and severi...