Interim data from Zevra Therapeutics’ open-label dose titration phase of a phase 2 clinical trial evaluating KP1077 in patients with idiopathic hypersomnia (IH) suggests that KP1077 is well-tolerated across all doses and both dosing regimens studied. The data provides insights related to the effective dose range and schedule.
Zevra aims to provide topline data from the completed trial in the first half of 2024 after all patients have completed the double-blind withdrawal phase.
The combined open-label interim and upcoming topline data are expected to also provide information related to a number of secondary and exploratory endpoints, including excessive daytime sleepiness, sleep inertia, and brain fog.
“The open-label data yielded key insights for the design of the phase 3 trial and potential unique benefits of KP1077 as a treatment for IH, if approved,” says Rene Braeckman, PhD, senior vice president of clinical development at Zevra, in a release. “The interim data from the open-label portion of this trial demonstrate that KP1077 is well-tolerated at all dose levels and both dosing regimens with adverse events that are typical for stimulants and are mostly mild in severity.”
The results are consistent with data from Zevra’s previous phase 1 trial studying serdexmethylphenidate (SDX), a prodrug of d-methylphenidate and the sole active pharmaceutical ingredient in KP1077. The results indicate no greater cardiovascular safety risk despite higher overall exposure levels when compared to both immediate and long-acting methylphenidate products currently used off-label for the treatment of IH, according to Braeckman in the release.
“We believe KP1077, if approved, could provide a differentiated treatment option for patients underserved by currently available therapies,” she says in the release.
The interim data related to the secondary and exploratory endpoints showed marked improvements in patient-reported assessments of key IH symptoms from the open-label titration phase, including excessive daytime sleepiness, sleep inertia, and brain fog.
The improvements in symptoms were similar after both once-per-day dosing of KP1077 (in the evening, just before bedtime) and twice-per-day dosing (half the daily dose after awakening in the morning and half the daily dose just before bedtime).
The results from the completed phase 2 trial are expected to inform the final design of the anticipated phase 3 trial in IH, which is expected to be initiated by the end of 2024.