KemPharm initiates Phase 2 trial evaluating KP1077

KemPharm announced the initiation of a Phase 2 clinical trial evaluating KP1077 as a treatment for idiopathic hypersomnia, IH. KP1077 is comprised solely of serdexmethylphenidate, SDX, KemPharm’s proprietary prodrug of d-methylphenidate. 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. 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 primary endpoint is the safety and tolerability of SDX. The major secondary efficacy endpoint is the change in Epworth Sleepiness Scale total score. Additional exploratory endpoints include the Patient Global Impression of Severity, the Clinical Global Impression of Severity, change in total score on the Idiopathic Hypersomnia Severity Scale, and a new scale to assess the symptoms and severity of "Brain Fog". "Initiation of the Phase 2 clinical trial investigating KP1077 for the treatment of IH is a significant milestone for KemPharm and a key inflection point as we continue to pursue our strategic transformation to develop or acquire novel therapeutics designed to address a range of underserved rare diseases," said Travis C. Mickle, Ph.D., President and CEO of KemPharm. "We believe the design of the Phase 2 study will allow us to evaluate KP1077’s effect on several symptoms associated with IH, including excessive daytime sleepiness, extreme difficulty waking, and severe brain fog. Additionally, this study provides an opportunity to explore both the optimal SDX dose range and dosing regimen(s) – nighttime dosing or twice daily – which will inform the design of future Phase 3 studies for the IH and narcolepsy indications. We expect to report interim data from the trial as early as Q3 2023."