Harmony Biosciences has completed enrollment for its phase 3 registrational INTUNE study evaluating the safety and efficacy of pitolisant in adult patients with idiopathic hypersomnia (IH).
The completion comes nine months ahead of schedule. Topline results are now anticipated in the fourth quarter of 2023.
“Completing enrollment in the INTUNE study now puts us nine months ahead of plan and reflects the strong interest and enthusiasm from patients and healthcare professionals alike, the unmet medical need in the IH community, and the strong momentum of our broader pitolisant development program,” says Harmony’s chief medical officer, Kumar Budur MD, MS, in a release.
The INTUNE study is a double-blind, placebo-controlled, randomized withdrawal phase 3 registrational trial in approximately 200 adult patients with IH being conducted at 58 clinical trial sites across the US. The primary objective is to evaluate the safety and efficacy of pitolisant compared with placebo in treating excessive daytime sleepiness. Secondary objectives include assessing the impact of pitolisant on other important symptoms of IH, such as sleep inertia and cognitive impairment.
Other outcome measures include patient impression of overall change in symptoms, investigator assessment of overall disease severity, and functional status. Based on recent insurance claims data, the number of patients diagnosed with IH in the US ranges from 30,000 to 40,000, according to a release from Harmony Biosciences.
“At Harmony, we are harnessing a mechanism-based approach to the development of pitolisant, which is thought to target the histamine system to promote wakefulness in the brain,” says Budur in the release. “This approach is driven by our shared conviction that innovative science translates into therapeutic possibilities that have the potential to impact the lives of individuals with rare neurological diseases.”
Pitolisant is marketed as Wakix in the US for the treatment of excessive daytime sleepiness or cataplexy in adult patients with narcolepsy. Pitolisant is not approved for IH and is currently being evaluated as an investigational agent in adult patients with IH.